Leading Independent Proxy Advisory Firms ISS and Glass Lewis Recommend Acer
Therapeutics Shareholders Vote “FOR” the Proposed Merger and Related Proposals

Acer Shareholders – Please Vote Today!

NEWTON, MA – November 2, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical
company focused on the acquisition, development and commercialization of therapies for
serious, rare and life-threatening diseases with significant unmet medical needs, today
announced that Institutional Shareholder Services Inc. (ISS) and Glass, Lewis & Co. LLC (Glass
Lewis) recommended that Acer shareholders vote “FOR” the proposed merger with Zevra
Therapeutics, Inc. and the related proposals in the Company’s proxy statement and prospectus
for the special meeting of its shareholders to be held on November 8, 2023 at 11:00 a.m.
Eastern Time.
ISS and Glass Lewis are widely recognized as the leading independent voting and corporate
governance advisory firms. Their analysis and recommendations are relied on by many major
institutional investment firms, mutual funds and fiduciaries throughout North America.
In its report, ISS stated, among other things, that “The transaction warrants support in light of
the reasonably thorough review of alternatives, the positive market reaction, the upside
potential provided by the stock and CVR forms of consideration, and the downside risk of non-
approval.”
Glass Lewis concluded that the transaction would allow Acer shareholders to participate in a
larger and better capitalized pharmaceutical company, while also retaining significant upside
potential through the CVR consideration, at a time when Acer appears to have few, if any,
viable alternatives. Glass Lewis also noted that the total implied value of the proposed
consideration represents a substantial premium to the unaffected trading price of Acer shares
and the merger consideration compares favorably with the expected outcome in a liquidation
scenario, in which Acer shareholders were not expected to receive any proceeds.
Commenting on the proxy advisors’ reports, Chris Schelling, CEO and Founder of Acer, said:
“The ISS and Glass Lewis recommendations are consistent with our view that the merger with
Zevra is in the best interest of Acer shareholders.”
The merger and related agreements have been unanimously approved by the boards of
directors of both companies. The merger and related proposals have been unanimously
approved by Acer’s board of directors.
Failure to vote or an abstention from voting will have the same effect as a vote “AGAINST” the
merger proposal. All shareholders are asked to vote “FOR” all proposals as soon as possible.

THE MERGER WILL NOT GO FORWARD UNLESS THE MERGER AND RELATED PROPOSALS ARE

APPROVED.

ACER SHAREHOLDERS – PLEASE VOTE TODAY!

If the merger is not approved on November 8, ACER will begin trading on OTC Pink Market
starting on November 9 because of the failure by the Company to regain compliance, during the
previously granted 180 calendar day grace period, with Nasdaq’s requirement of having at least
$35 million in market value of listed securities, resulting in the trading suspension of ACER on
Nasdaq.
If the merger is not subsequently consummated, Acer will not be able to fund its business
operations and will likely be forced to terminate operations, liquidate or file for bankruptcy.
If you are an Acer shareholder and you have questions or require assistance in submitting your
proxy or voting your shares, please contact Acer’s proxy solicitor:
ADVANTAGE PROXY, INC.
Toll Free: 1-877-870-8565
Collect: 1-206-870-8565
Email: ksmith@advantageproxy.com
Additional Information about the Proposed Merger between Acer and Zevra, the Special
Meeting and Where to Find It
In connection with the proposed merger, Zevra has filed a registration statement on Form S-4
with the Securities and Exchange Commission (the “SEC”), including a proxy statement /
prospectus. The registration statement was declared effective on October 10, 2023.
Additionally, Acer’s proxy statement was filed on October 10, 2023. Acer shareholders are
urged to read these materials because they contain important information about Acer, Zevra
and the proposed merger. The proxy statement / prospectus and other relevant materials, and
any other documents filed by Zevra and Acer with the SEC, may be obtained free of charge at
the SEC website at www.sec.gov. In addition, Acer shareholders will be able to attend the Acer
special meeting via the Internet at https://www.cstproxy.com/acertx/sm2023 and view the
Acer 2023 Special Meeting Proxy Statement and the Zevra Therapeutics, Inc. Forms 10-K, 10-Qs
and 8-Ks. Acer shareholders are urged to read the proxy statement / prospectus and the other
relevant materials before making any voting or investment decision with respect to the
proposed merger.
No Offer or Solicitation
This communication is for informational purposes only and not intended to and does not
constitute an offer to subscribe for, buy or sell, the solicitation of an offer to subscribe for, buy
or sell or an invitation to subscribe for, buy or sell any securities or the solicitation of any vote
or approval in any jurisdiction pursuant to or in connection with the proposed transaction or
otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in

contravention of applicable law. No offer of securities shall be made except by means of a
prospectus meeting the requirements of Section 10 of the Securities Act of 1933, as amended
(the “Securities Act”), and otherwise in accordance with applicable law.
Participants in the Solicitation
Acer, Zevra and their respective directors and executive officers may be considered participants
in the solicitation of proxies in connection with the proposed transaction. Information about
the directors and executive officers of Acer is set forth in its Annual Report on Form 10-K for the
year ended December 31, 2022, which was filed with the SEC on March 27, 2023, and its proxy
statement for its 2023 annual meeting of shareholders, which was filed with the SEC on April
14, 2023. Information about the directors and executive officers of Zevra is set forth in its
Annual Report on Form 10-K for the year ended December 31, 2022, which was filed with the
SEC on March 7, 2023, and its proxy statement for its 2023 annual meeting of stockholders,
which was filed with the SEC on March 15, 2023, the definitive proxy statement filed by Daniel
J. Mangless, together with the other participants named therein, which was filed with the SEC
on March 17, 2023, and Zevra’s Current Reports on Form 8-K, filed with the SEC on March 30,
2023, May 8, 2023, May 15, 2023, and August 7, 2023. Other information regarding the
participants in the proxy solicitations and a description of their direct and indirect interests, by
security holdings or otherwise, is set forth in the proxy statement/prospectus and other
relevant materials filed with the SEC and may be obtained free of charge from the sources
indicated above.
About Acer Therapeutics
Acer is a pharmaceutical company focused on the acquisition, development and
commercialization of therapies for serious rare and life-threatening diseases with significant
unmet medical needs. In the U.S., OLPRUVA® (sodium phenylbutyrate) is approved for the
treatment of UCDs involving deficiencies of CPS, OTC, or AS. Acer is also advancing a pipeline of
investigational product candidates for rare and life-threatening diseases, including: OLPRUVA®
(sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine
Disease (MSUD); and EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome
(vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. For more information,
visit www.www.acertx.com.
Forward-Looking Statements
DISCLOSURE NOTICE: This press release contains “forward-looking statements” within the
meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act of
1934, as amended, related to Acer, Zevra and the proposed acquisition of Acer by Zevra. All
statements other than statements of historical fact are forward-looking statements for
purposes of federal and state securities laws. These forward-looking statements involve
uncertainties that could significantly affect the financial or operating results of Acer, Zevra or
the combined company. These forward-looking statements may be identified by terms such as
anticipate, believe, foresee, expect, intend, plan, may, will, could, should and would and the
negative of these terms or other similar expressions. Forward-looking statements in this
document include, among other things, statements about the potential benefits of the

proposed acquisition; statements about contingent cash consideration and related milestones
as contemplated by the CVR Agreement; the anticipated timing of closing of the acquisition; the
delisting of Acer’s stock from Nasdaq and resulting move to OTC Pink Market; and that, if the
merger is not subsequently consummated, Acer will not be able to fund its business operations
and will likely be forced to terminate operations, liquidate or file for bankruptcy. These
forward-looking statements involve substantial risks and uncertainties that could cause actual
results to differ materially from those expressed or implied by such statements. Risks and
uncertainties include, among other things, risks related to the satisfaction of the conditions to
closing the acquisition (including the failure to obtain necessary stockholder approval) in the
anticipated timeframe or at all; risks related to the ability to realize the anticipated benefits of
the acquisition, including the possibility that the expected benefits from the proposed
acquisition will not be realized or will not be realized within the expected time period; risks
related to the contingent cash consideration and related milestones as contemplated by the
CVR Agreement, including that such milestone may not be achieved and thus the related cash
consideration would not become payable; the risk that the businesses will not be integrated
successfully; disruption from the transaction making it more difficult to maintain business,
contractual and operational relationships; the unfavorable outcome of the legal proceedings
that have been or may be instituted against Acer, Zevra or the combined company; the ability
to retain key personnel; negative effects of this announcement or the consummation of the
proposed acquisition on the market price of the capital stock of Acer and Zevra and on Acer’s
and Zevra’s operating results; risks relating to the value of Zevra’s shares to be issued in the
transaction; significant transaction costs, fees, expenses and charges; unknown liabilities; the
risk of litigation and/or regulatory actions related to the proposed acquisition; the financing of
the transaction and Acer’s interim operations; the occurrence of any event, change or other
circumstances that could give rise to the termination of the merger agreement; other business
effects, including the effects of industry, market, economic, political or regulatory conditions;
future exchange and interest rates; changes in tax and other laws, regulations, rates and
policies; future business combinations or disposals; and competitive developments.
A further description of risks and uncertainties relating to Acer and Zevra can be found in their
respective most recent Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q and
Current Reports on Form 8-K, all of which are filed with the SEC and available at www.sec.gov.
Neither Acer nor Zevra intends to update the forward-looking statements contained in this
document as the result of new information or future events or developments, except as
required by law.
Corporate Contact
Harry Palmin
Chief Financial Officer
Acer Therapeutics Inc.
investors@www.acertx.com
+1-844-902-6100

Zevra Therapeutics to Acquire Acer Therapeutics, Expanding its Rare Disease Portfolio and Adding Commercial Product

Celebration, FL and Newton, MA – August 31, 2023 – Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (“Zevra”), a rare disease company melding science, data and patient need to create transformational therapies for diseases with limited or no treatment options, and Acer Therapeutics Inc. (Nasdaq: ACER) (“Acer”), a pharmaceutical company focused on development and commercialization of therapies for rare and life-threatening diseases, today announced the companies have entered into a definitive agreement pursuant to which Zevra would acquire Acer in a merger transaction having a total potential value for Acer stockholders of up to $91 million, consisting of (i) approximately 2.96 million shares of Zevra common stock valued at $15 million, or 0.121 shares of Zevra’s common stock per share of Acer common stock based on the volume weighted average trading price (VWAP) of shares of Zevra’s common stock during the 20 consecutive trading days ending on the trading date prior to today, and (ii) up to an additional $76 million in a series of potential cash payments pursuant to non-transferable Contingent Value Rights (CVRs) upon achievement of certain commercial and regulatory milestones for Acer’s OLPRUVA (sodium phenylbutyrate) and Acer’s EDSIVO (celiprol) within specified time periods. Certain additional cash payments are also possible pursuant to the CVRs with respect to milestones involving Acer’s early-stage program ACER-2820 (emetine), as described further below. Zevra has also purchased Acer’s secured debt at a discount from Nantahala Capital (Nantahala) through a series of transactions in capital efficient structure. In addition, Zevra has agreed to provide Acer with a bridge loan facility for up to $16.5 million, subject to certain terms and conditions. Both companies are deeply committed to developing and commercializing treatments for rare diseases with a strong focus on patients and remain dedicated to supporting communities with little or no existing therapeutic options. The merger is expected to expand Zevra’s rare disease portfolio, as well as increase and diversify its revenues with the addition of a U.S. commercial asset, OLPRUVA, indicated for the treatment of UCDs. The transaction is subject to certain customary closing conditions, including, but not limited to, approval by Acer’s stockholders.

“We believe that Acer’s portfolio of rare disease programs, including the recent U.S. commercial approval of OLPRUVA for UCDs, is a perfect strategic fit for Zevra and creates significant opportunity for us to positively impact patient lives while creating shareholder value,” said Joshua Schafer, Chief Commercialization Officer and Executive Vice President of Business Development of Zevra Therapeutics. “We are excited about Acer’s clinical programs and are confident in the potential of OLPRUVA to bring UCD patients a more convenient and cost-effective treatment option over current therapies. Acer would bring unique rare disease operations and capabilities that would serve as a foundation to support the commercialization of Zevra’s pipeline as it advances.” 

Chris Schelling, Acer Therapeutics’ Chief Executive Officer and Founder, added, “Following years of product development and commitment to rare disease communities, culminating in the FDA approval of OLPRUVA, we are pleased to see our assets, pipeline and team positioned to unite under the Zevra umbrella. We look forward to working with the Zevra team to ensure a smooth transition as we work together on behalf of patients.”

“This merger would support Zevra’s vision of becoming a leading rare disease company bringing life-changing therapies to patients with a significant unmet need,” said Christal Mickle, Zevra’s interim Chief Executive Officer and Chief Development Officer. “The commercial launch of OLPRUVA in the U.S. requires a small, highly-focused commercial team, which is complementary to what we intend to build for arimoclomol, our product candidate for the treatment of Niemann-Pick disease Type C (NPC). We believe there is potential to realize significant synergies across our commercial organizations as both UCDs and NPC are metabolic related conditions and there is overlap among those physicians that treat both disorders.”
Financial Details and Terms of the Transactions:
The transactions, which have been approved by the Boards of Directors of both companies, are currently anticipated to close in the fourth quarter of 2023, subject to Acer stockholder approval, as well as other customary closing conditions. The merger is expected to accelerate Zevra’s pathway to becoming a commercial-stage company by adding OLPRUVA, an FDA-approved asset, which is expected to add to Zevra’s revenue. There are potential synergies to be realized by combining Acer’s operations with Zevra’s capabilities in preparation for the potential launch of arimoclomol. In addition, Zevra expects to acquire significant net operating loss (NOL) tax assets as part of this merger, providing potential tax savings against future earnings.
Under the terms of the definitive agreement, at closing, Zevra would issue 0.121 of a share of Zevra’s common stock in respect of each share of Acer’s common stock. In addition, Acer stockholders of record as of immediately prior to the effective time of the merger would receive non-transferable CVRs entitling the holders to receive up to $34 million in cash upon the achievement of certain commercial milestones for OLPRUVA, and up to an additional $42 million in cash upon the achievement of certain regulatory milestones for OLPRUVA and EDSIVO.

Approximately 2.96 million shares of Zevra common stock to be issued in the merger is calculated by dividing $15.0 million by the VWAP of Zevra’s shares of common stock during the 20 consecutive trading days through yesterday, which was $5.0667. The 20-day trailing VWAP value represents a discount of approximately 2% to yesterday’s closing share price for Acer.

The non-transferable CVRs will entitle the Acer stockholders of record to receive up to $34 million in cash upon the achievement of certain commercial milestones for OLPRUVA, and an additional $42 million in cash upon the achievement of certain regulatory milestones for other development programs. The proposed transactions also include non-transferable CVRs for ACER-2820, Acer’s early phase emetine program.

Based on the number of Zevra shares issued and outstanding as of June 30, 2023, together with the equity issued to Nantahala as part of the debt acquisition as described below, the aggregate number of shares issuable to Acer stockholders in the merger is expected to represent approximately 7.6% of the issued and outstanding common stock of Zevra following the merger.

To provide for a smooth transition and uninterrupted operations, and subject to certain conditions, Zevra has extended a bridge loan facility to Acer of up to $16.5 million to provide additional working capital to, among other things, support the commercial launch of OLPRUVA until the expected closing of the merger transaction, and to provide the $10 million payment to Acer’s termination of the 2021 collaboration and license agreement by and between Acer and Relief Therapeutics, and Acer’s related entry into an exclusive license agreement with Relief for the development and commercialization rights for OLPRUVA in geographical Europe.
Additionally, Zevra has purchased Acer’s secured debt from Nantahala representing an aggregate of principal, accrued interest other fees and premiums of approximately $35.3 million, for a total of $28.5 million to be paid using a combination of $12 million in cash financed from Zevra’s existing margin line of credit facility, $5 million in a new promissory note held by Nantahala, with a three-year maturity and bearing interest initially at 9% per annum (increasing to 12% per annum if the note remains outstanding six months after issuance), and $11.5 million in Zevra’s common stock based on the 20-day trailing VWAP calculation described above, or approximately 2.27 million shares, or approximately 5.8% of the issued and outstanding common stock of Zevra following the merger.
Bryan Cave Leighton Paisner LLP served as legal advisor to Zevra and Canaccord Genuity LLC served as exclusive financial advisor to Zevra for the transactions. Pillsbury Winthrop Shaw Pittman LLP served as legal advisor to Acer, and William Blair & Company, LLC served as exclusive financial advisor to Acer.
Conference Call Information:
Zevra will host a conference call and live audio webcast with a slide presentation today, August 31, 2023, at 8:30 a.m. ET, to discuss details of the acquisition agreement with Acer.
 
The audio webcast with a slide presentation will be accessible via the Investor Relations section of the Company’s website, http://investors.zevra.com/. An archive of the webcast and presentation will be available for 90 days beginning at approximately 9:30 a.m. ET, on August 31, 2023. 
 
Additionally, interested participants and investors may access the conference call by dialing either:  
(800) 245-3047 (U.S.)
+1 (203) 518-9765 (International)
Conference ID: ZevraUpdate
About Urea Cycle Disorders:
UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments, if ammonia levels are not controlled. Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels.

About OLPRUVA:
ACER-001 (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and has recently been marketed under the brand name, OLPRUVA. OLPRUVA (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Please see Important Safety Information and full Prescribing Information, including Patient Information.

Important Safety Information:
Certain medicines may increase the level of ammonia in your blood or cause serious side effects when taken during treatment with OLPRUVA. Tell your doctor about all the medicines you or your child takes especially if you or your child takes corticosteroids, valproic acid, haloperidol, and/or probenecid.

OLPRUVA can cause serious side effects, including: 1) nervous system problems (neurotoxicity). Symptoms include sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, confusion, 2) low potassium levels in your blood (hypokalemia) and 3) conditions related to swelling (edema). OLPRUVA contains salt (sodium), which can cause swelling from salt and water retention. Tell your doctor right away if you or your child get any of these symptoms. Your doctor may do certain blood tests to check for side effects during treatment with OLPRUVA. If you have certain medical conditions such as heart, liver or kidney problems, are pregnant/planning to get pregnant or breast-feeding, your doctor will decide if OLPRUVA is right for you.

The most common side effects of OLPRUVA include absent or irregular menstrual periods, decreased appetite, body odor, bad taste or avoiding foods you ate prior to getting sick (taste aversion). These are not all of the possible side effects of OLPRUVA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

For additional information, please see Important Safety Information and full Prescribing Information, including Patient Information and discuss with your doctor.

About Niemann-Pick disease type C (NPC):
Niemann-Pick disease type C (NPC) is an ultra-rare and progressive, neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various tissue areas, including brain tissue. The disease is caused by mutations in the NPC1 or NPC2 genes which are responsible for making lysosomal proteins and is an autosomal recessive trait. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease progression is irreversible and can be fatal within months or take years to be diagnosed and advance in severity.

About Arimoclomol:
Arimoclomol, Zevra’s orally-delivered, first-in-class investigational product candidate for the treatment of NPC, has been granted orphan drug designation, Fast Track designation, Breakthrough Therapy designation and rare pediatric disease designation for NPC by the FDA, and orphan medicinal product designation for the treatment of NPC by the European Medicines Agency (EMA). The arimoclomol NDA is currently being prepared for resubmission to the FDA.

About Zevra Therapeutics:
Zevra Therapeutics is a rare disease company melding science, data, and patient need to create transformational therapies for diseases with limited or no treatment options. With unique, data-driven clinical, regulatory, and commercialization strategies, the Company is overcoming complex drug development challenges to bring much-needed therapies to patients. With both regulatory and clinical stage product candidates, the Company is building its commercial capability to make new therapies available to the rare disease community.

Early access programs are made available by Zevra Therapeutics and its affiliates and are subject to the Company’s Early Access Program (EAP) policy as published on its website at zevra.com. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician’s discretion.

About Acer Therapeutics:
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); and EDSIVO (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. For more information, visit www.www.acertx.com.

Cautionary Note Concerning Forward Looking Statements:
This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, and which can be identified by the use of words such as “may,” “will,” “expect,” “project,” “estimate,” “anticipate,” “plan,” “believe,” “potential,” “should,” “continue,” “could,” “intend,” “target,” “predict,” or the negative versions of those words or other comparable words or expressions, although not all forward-looking statements contain these identifying words or expressions. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements include without limitation statements regarding the proposed merger transaction, its timing and its consummation, the anticipated financial performance of Zevra and Acer related thereto, including the anticipated closing of, benefits of, and synergies related to the proposed merger transaction, potential strategic implications as a result of the proposed merger transaction, Zevra’s intention to provide bridge financing to Acer and the availability of such financing to Acer, Zevra’s path to profitability, Zevra’s strategic and product development objectives, including with respect to becoming a leading, commercially-focused rare disease company, Zevra’s plans to build out commercial teams for products or product candidates, Zevra’s commercial infrastructure investments and the impact of the proposed transactions on them, Zevra’s industry, plans, goals and expectations concerning market position, future operations and other financial and operating information, and the potential for achievement of the milestones that would trigger cash payments pursuant to the CVRs that would be issued to the Acer stockholders in the merger. Forward-looking statements are based on information currently available to Zevra and Acer and their respective current plans or expectations, and are subject to several known and unknown uncertainties, risks, and other important factors that may cause actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements, including, but not limited to, uncertainties involving the following: the potential timing of the consummation of the proposed merger transaction and the ability of the parties to consummate the proposed merger; the satisfaction of the conditions precedent to consummation of the proposed merger, including the approval of Acer’s stockholders; the ability to obtain required regulatory approvals at all or in a timely manner; any litigation related to the proposed transactions; disruption of Acer’s or Zevra’s current plans and operations as a result of the proposed transactions; the ability of Acer or Zevra to retain and hire key personnel; competitive responses to the proposed transactions; unexpected costs, charges or expenses resulting from the proposed transactions; the ability of Zevra to successfully integrate Acer’s operations, products, product candidates and technology; the ability of Zevra to implement its plans, forecasts and other expectations with respect to Acer’s business after the completion of the proposed transactions and realize additional opportunities for growth and innovation; the ability of Zevra to realize the anticipated synergies and related benefits from the proposed transactions in the anticipated amounts or within the anticipated timeframes or at all; and the ability to maintain relationships with Zevra’s and Acer’s respective employees, customers, other business partners and governmental authorities. These and other important factors are described in detail in the “Risk Factors” section of Zevra’s and Acer’s Annual Reports on Form 10-K for the year ended December 31, 2022, as updated in Zevra’s and Acer’s Quarterly Reports on Form 10-Q for the quarter ended June 30, 2023, and Zevra’s and Acer’s other filings with the Securities and Exchange Commission. While either Zevra or Acer may elect to update such forward-looking statements at some point in the future, each party disclaims any obligation to do so, except as required by law, even if subsequent events cause their respective views to change. Although each party believes the expectations reflected in such forward-looking statements are reasonable, neither party can assure that such expectations will prove correct.  These forward-looking statements should not be relied upon as representing the views of either Acer as of any date after the date of this press release. 

Important Additional Information Regarding the Transactions Will Be Filed With the SEC
In connection with the proposed transactions, Zevra and Acer will file relevant materials with the SEC, including a Zevra registration statement on Form S-4 that will include a proxy statement of Acer and will also constitute a prospectus of Zevra, and a definitive proxy statement will be mailed to stockholders of Acer. INVESTORS AND SECURITY HOLDERS OF ZEVRA AND ACER ARE URGED TO READ THE PROXY STATEMENT/PROSPECTUS THAT WILL BE INCLUDED IN THE REGISTRATION STATEMENT ON FORM S-4, AND OTHER RELEVANT DOCUMENTS FILED OR TO BE FILED WITH THE SEC IN CONNECTION WITH THE PROPOSED TRANSACTIONS OR INCORPORATED BY REFERENCE IN THE PROXY STATEMENT/PROSPECTUS (IF ANY) CAREFULLY AND IN THEIR ENTIRETY WHEN THEY BECOME AVAILABLE BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTIONS, THE PARTIES TO THE PROPOSED TRANSACTIONS AND THE RISKS ASSOCIATED WITH THE PROPOSED TRANSACTIONS. Investors and security holders will be able to obtain, without charge, a copy of the registration statement, the proxy statement/prospectus and other relevant documents filed with the SEC (when available) from the SEC’s website at www.sec.gov. Copies of the documents filed with the SEC by Zevra will be available free of charge on Zevra’s investor relations website at investors.zevra.com under the tab “SEC Filings.” Copies of the documents filed with the SEC by Acer will be available free of charge on Acer’s investor relations website at www.www.acertx.com/investor-relations under the tab “SEC Filings.”

Participants in the Solicitation
Zevra, Acer and certain of their directors, executive officers and other members of management may be deemed to be participants in the solicitation of proxies with respect to the proposed transactions. Information regarding the persons who may, under the rules of the SEC, be deemed participants in the solicitation of the stockholders of Acer in connection with the proposed transactions, including a description of their direct or indirect interests, by security holdings or otherwise, will be set forth in the proxy statement/prospectus when it is filed with the SEC. Information regarding Zevra’s directors and executive officers is contained in Zevra’s definitive proxy statement, which was filed with the SEC on March 15, 2023, the definitive proxy statement filed by Daniel J. Mangless, together with the other participants named therein, which was filed with the SEC on March 17, 2023, and Zevra’s Current Reports on Form 8-K, filed with the SEC on March 30, 2023, May 8, 2023, May 15, 2023, and August 7, 2023. Information regarding Acer’s directors and executive officers is contained in Acer’s definitive proxy statement, which was filed with the SEC on April 14, 2023. Security holders and investors may obtain additional information regarding the interests of such persons, which may be different than those of Zevra’s security holders generally, by reading the proxy statement/prospectus and other relevant documents regarding the transactions, which will be filed with the SEC. You may obtain these documents (when they become available) free of charge through the website maintained by the SEC at www.sec.gov and Zevra’s or Acer’s investor relations websites as described above.

No Offer or Solicitation
This communication is not intended to and does not constitute an offer to sell or the solicitation of an offer to subscribe for or buy or an invitation to purchase or subscribe for any securities or the solicitation of any vote or approval in any jurisdiction pursuant to the proposed transactions or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. This communication does not constitute a prospectus or prospectus equivalent document. No offering of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act. In connection with the proposed transactions, Zevra will file a registration statement on Form S-4 that will include a proxy statement of Acer and will also constitute a prospectus of Zevra. INVESTORS AND SECURITY HOLDERS OF ZEVRA AND ACER ARE URGED TO READ THE PROXY STATEMENT/PROSPECTUS AND OTHER DOCUMENTS THAT WILL BE FILED WITH THE SEC CAREFULLY AND IN THEIR ENTIRETY WHEN THEY BECOME AVAILABLE BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION.

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Zevra Contacts: 
 
Nichol Ochsner 
+1 (732) 754-2545 
nochsner@zevra.com  
 
Janine Bogris
+1 (201) 245-6838
Janine.bogris@canalecomm.com

Acer Contacts:

Harry Palmin
+1 (844) 902-6100
investors@www.acertx.com

Nick Colangelo
+1 (332) 895-3226
nick@gilmartinIR.com

Acer Therapeutics Reacquires Worldwide Development, Commercialization and Economic Rights to OLPRUVA™ from Relief Therapeutics, Excluding the Geographical Europe

NEWTON, MA – August 30, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today announced it has entered into definitive agreements to reacquire worldwide development, commercialization and economic rights to OLPRUVA™ (sodium phenylbutyrate) from Relief Therapeutics, excluding the European Union, Liechtenstein, San Marino, Vatican City, Norway, Iceland, Principality of Monaco, Andorra, Gibraltar, Switzerland, United Kingdom, Albania, Bosnia, Kosovo, Montenegro, Serbia and North Macedonia (Geographical Europe). OLPRUVA™ is approved in the U.S. for the treatment of certain urea cycle disorders (UCDs).

Under the prior Collaboration and License Agreement (CLA) between the parties, Acer would have paid Relief 60% of Acer’s OLPRUVA™ net profits in the U.S., Canada, Brazil, Turkey and Japan (the Acer territories), while Acer would have received a 15% royalty on Relief’s net sales in the rest of the world (the Relief territories). Acer and Relief have now agreed to terminate the CLA, where Acer is no longer required to pay Relief 60% of OLPRUVA™ net profits in the Acer territories and have regained all development and commercialization rights in rest of the world, excluding the Geographical Europe. In return, Relief will receive an upfront payment by Acer of $10 million, to be made within 5 business days of the signature date, with an additional payment of $1.5 million due on the first-year anniversary of the $10 million payment. Acer has also agreed to pay a 10% royalty on net sales in the Acer territories, and 20% of any value received by Acer from certain third parties relating to OLPRUVA™ licensing or divestment rights, all of the foregoing which are capped at $45 million, for total payments to Relief of up to $56.5 million. Additionally, Acer and Relief have entered into a new Exclusive License Agreement (ELA) in which Relief will retain development and commercialization rights for OLPRUVA™ in the Geographical Europe, where Acer will have the right to receive a royalty of up to 10% of the net sales of OLPRUVA™.

“Regaining exclusive worldwide rights to OLPRUVA™ without profit sharing, subject only to Relief’s rights in the Geographical Europe, unlocks value for Acer as we continue the U.S. launch of OLPRUVA™ and begin to provide treatments to UCD patients in need,” said Chris Schelling, CEO and Founder of Acer. “Reacquiring greater economic rights to OLPRUVA™ allows Acer to reinvest more capital into potential lifecycle expansion opportunities for OLPRUVA™, in additional inborn errors of metabolism, such as Maple Syrup Urine Disease and other potential indications.”

“We are very happy to have restructured our collaboration with Acer Therapeutics which we believe will benefit those suffering from UCDs and other potential rare metabolic conditions,” said Jack Weinstein, CEO of Relief Therapeutics. “Our collective goal with Acer is to maximize the global commercialization of OLPRUVA™ to ensure as many patients as possible will access this much needed, differentiated and convenient alternative.”

Additional information regarding the termination of the March 2021 CLA and execution of the new ELA for European rights is available on form 8-K at https://www.acertx.com/investor-relations/sec-filings-list/.

OLPRUVA™: Now Available by Prescription in U.S.
ACER-001 (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and is marketed under the brand name, OLPRUVA™. OLPRUVA™ (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a BSA of 1.2 m2 or greater, with UCDs, involving deficiencies of CPS, OTC, or AS.1 Please see full Prescribing Information, including Patient Information.

Important Safety Information
Certain medicines may increase the level of ammonia in the blood or cause serious side effects when taken during treatment with OLPRUVA™. Tell your doctor about all the medicines you or your child takes, especially if you or your child takes corticosteroids, valproic acid, haloperidol, and/or probenecid.

OLPRUVA™ can cause serious side effects, including: 1) nervous system problems (neurotoxicity) such as, sleepiness, tiredness, lightheadedness, vomiting, nausea, headache, and/or confusion, 2) low potassium levels in your blood (hypokalemia) and 3) conditions related to swelling (edema). OLPRUVA™ contains salt (sodium), which can cause swelling from salt and water retention. Tell your doctor right away if you or your child get any of these symptoms. Your doctor may do certain blood tests to check for side effects during treatment with OLPRUVA. If you have certain medical conditions such as heart, liver or kidney problems, are pregnant/planning to get pregnant or breast-feeding, your doctor will decide if OLPRUVA™ is right for you.

The most common side effects of OLPRUVA™ include absent or irregular menstrual periods, decreased appetite, body odor, bad taste or avoiding foods you ate prior to getting sick (taste aversion). These are not all of the possible side effects of OLPRUVA™. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

For additional Important Safety Information, see full Prescribing Information, Patient Package Insert and discuss with your doctor.

About Acer Therapeutics
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of UCDs involving deficiencies of CPS, OTC, or AS. Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); and EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. For more information, visit www.www.acertx.com.

References
OLPRUVA™ (sodium phenylbutyrate) for oral suspension. Prescribing information. Newton, MA: Acer Therapeutics Inc.

Acer Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, expected payments between Acer and Relief in connection with the ELA and terminated CLA, the impact of the revised business arrangement between Acer and Relief on the Company and the Company’s investment plans related thereto, plans with respect to the U.S. launch of OLPRUVA™, and the possible side effects of OLPRUVA™. Additionally, the Company’s effort to advance a pipeline of investigational product candidates and related plans and expectations is an example of such forward-looking statements. Our efforts to commercialize OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS are at an early stage, we currently do not have fully developed marketing and sales capabilities, and there is no guarantee that we will be successful in our commercialization efforts. Our pipeline products (including OLPRUVA™ for indications other than UCDs) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the availability of financing to fund our commercialization efforts, our pipeline product development programs and general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Corporate and IR Contacts
Harry Palmin
Chief Financial Officer
Acer Therapeutics Inc.
investors@www.acertx.com
+1-844-902-6100

Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

Acer Therapeutics Reports Second Quarter 2023 Financial Results and Provides Corporate Update

NEWTON, MA – August 14, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today reported financial results for the second quarter ended June 30, 2023 and provided a corporate update.

“The second quarter of 2023 marked continued progress for Acer as we execute on our OLPRUVA™ launch strategy,” said Chris Schelling, CEO and Founder of Acer. “We are pleased to offer OLPRUVA™ in the U.S. to certain UCD patients as a novel alternative option to current treatments that was specifically designed for palatability and convenience.1,2 With OLPRUVA™ kits now available in all dosage strengths, we are committed to supporting patients from initial prescription to support throughout the treatment process and look forward to making OLPRUVA™ available to patients in need.”

Program and Corporate Highlights

  • OLPRUVA™ (sodium phenylbutyrate) for oral suspension
    • OLPRUVA™ kits are now available in all dosage strengths and ready to ship to patients
    • A dedicated OLPRUVA™ Navigator Team at CVS Specialty is now available to provide prescription fulfillment support throughout the treatment process; healthcare provider and patient resources are also now available at www.OLPRUVAHCP.com and www.OLPRUVA.com
    • Acer Therapeutics is in discussions with both commercial and government payers and is actively engaged with the major pharmacy benefits managers (PBM) and group purchasing organizations (GPO) representing a substantial majority of covered lives
  • Corporate
    • Ended Q2 2023 with $1.6 million in cash and cash equivalents. Acer believes its cash and cash equivalents available as of June 30, 2023 will be sufficient to fund its anticipated operating and capital requirements through mid-Q3 2023

Anticipated Milestones (Subject to Available Capital)

  • Q3 2023: Acer expects to begin attaining OLPRUVA™ commercial insurance coverage in Q3 2023
  • Q3 2023: Acer expects to begin attaining insurance coverage for OLPRUVA™ Medicaid patients starting in Q3 2023
  • H1 2024: Acer anticipates enrollment completion in H1 2024 of its pivotal Phase 3 DiSCOVER trial of EDSIVO™ in patients with COL3A1-positive vEDS
  • Acer also intends to pursue additional opportunities for potential OLPRUVA™ label expansion, including the potential for additional dosage strengths to address patients with lower weights/body surface areas, and potential administration using a gastrostomy tube (G-tube)

Q2 2023 Financial Results

Cash Position. Cash and cash equivalents were $1.6 million as of June 30, 2023, compared to $2.3 million as of December 31, 2022. Acer believes its cash and cash equivalents at June 30, 2023 will be sufficient to fund its anticipated operating and capital requirements through the middle of the third quarter of 2023.

Research and Development Expenses. Research and development expenses were $1.4 million, net of collaboration funding of $0.6 million, for the three months ended June 30, 2023, as compared to $3.4 million, net of collaboration funding of $1.6 million, for the three months ended June 30, 2022. This decrease of $2.0 million was primarily due to decreases in expenses for clinical studies, employee-related expenses, expenses for consulting and professional services, and contract manufacturing expenses. Research and development expenses related to ACER-001 decreased in the three months ended June 30, 2023, resulting in a decrease in the recognition of the collaboration funding from the Collaboration Agreement with Relief. Research and development expenses for the three months ended June 30, 2023 were comprised of $1.0 million related to EDSIVOTM; $0.6 million related to ACER-001, offset by $0.6 million of collaboration funding; $0.2 million related to ACER-801; and $0.2 million related to other development activities.

General and Administrative Expenses. General and administrative expenses were $2.9 million, net of collaboration funding of $1.4 million, for the three months ended June 30, 2023, as compared to $3.6 million, net of collaboration funding of $3.3 million, for the three months ended June 30, 2022. This decrease of $0.7 million was primarily due to decreases in marketing expenses, expenses for consulting and professional services, and employee-related expenses. General and administrative expenses related to ACER-001 decreased in the three months ended June 30, 2023, resulting in a decrease in the recognition of the collaboration funding from the Collaboration Agreement with Relief.

Net Loss. Net loss for the three months ended June 30, 2023 was $8.1 million, or $0.33 net loss per share (basic and diluted), compared to a net loss of $2.7 million, or $0.17 net loss per share (basic and diluted), for the three months ended June 30, 2022.

For additional information, please see Acer’s Quarterly Report on Form 10-Q filed today with the Securities and Exchange Commission (SEC).

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of UCDs involving deficiencies of CPS, OTC, or AS. Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); and EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. For more information, visit www.www.acertx.com.

References

  1. OLPRUVA™ (sodium phenylbutyrate) for oral suspension. Prescribing information. Newton, MA: Acer Therapeutics Inc.
  2. Appel LE, Shockey JR, Schelling DC, inventors; Acer Therapeutics Inc, assignee. Palatable compositions including sodium phenylbutyrate and uses thereof. US patent 11,154,521 (B2). October 26, 2021.

Acer Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements about plans and strategy for the commercialization of OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with certain UCDs, including progress with respect to discussions with commercial and government insurance providers, physicians outreach and awareness, and patient support and fulfillment, statements with respect to our EDSIVO clinical trial for patients with vEDS, including enrollment and timing milestones related thereto, statements about our anticipated 2023 milestones, statements about our investment of OLPRUVA revenue, and statements about our capital requirements and sufficiency and duration of our current cash and cash equivalents. Our efforts to commercialize OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS are at an early stage, we currently do not have fully developed marketing, sales or distribution capabilities, and there is no guarantee that we will be successful in our commercialization efforts.  Our pipeline products (including OLPRUVA™ for indications other than UCDs as well as EDSIVO™ and ACER-801) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the availability of financing to fund our commercialization efforts, our pipeline product development programs and our general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Corporate and IR Contact

Jim DeNike
Acer Therapeutics Inc.
jdenike@www.acertx.com
+1-844-902-6100

Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

Acer Therapeutics Receives $1 Million Capital Infusion from Chief Executive Officer and Founder

NEWTON, MA – June 26, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today announced it has received $1,000,000 in funding in exchange for the issuance of an unsecured, subordinated promissory note for that principal amount to Christopher Schelling, the Company’s Chief Executive Officer and Founder, a member of the Company’s Board of Directors, and the beneficial owner of more than 10% of the Company’s outstanding common stock. Acer expects receipt of this funding to extend its cash runway into early Q3 2023.

“This capital infusion will provide additional runway and allow us to continue to execute on our OLPRUVA™ launch and provide additional working capital for the ongoing clinical development of EDSIVO™ and other ongoing initiatives,” said Mr. Schelling. “We expect OLPRUVA™ will be available by prescription beginning July 5 in the U.S. for the treatment of urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).”

The principal amount of the promissory note will accrue interest at a rate of 6% per annum, and all principal and accrued interest will be due and payable on the maturity date on August 21, 2023.

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); and EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. For more information, visit www.www.acertx.com.

Acer Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements about the sufficiency and duration of the Company’s cash and cash equivalents and statements about OLPRUVA™ launch activities, including timing thereof. Our efforts to commercialize OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS are at an early stage, we currently do not have fully developed marketing and sales capabilities, and there is no guarantee that we will be successful in our commercialization efforts.  Our pipeline products (including OLPRUVA™ for indications other than UCDs) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund the Company’s various product candidate development programs, to fund the Company’s commercialization efforts for OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS, and to meet the Company’s business objectives and operational requirements, and risks and uncertainties associated with drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Corporate and IR Contacts

Jim DeNike
Acer Therapeutics Inc.
jdenike@www.acertx.com
+1-844-902-6100

Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

Acer Therapeutics Reports Q1 2023 Financial Results and Provides Corporate Update

NEWTON, MA – May 15, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today reported financial results for the first quarter ended March 31, 2023 and provided a corporate update.

“The first quarter of 2023 was marked by considerable progress and a number of significant milestones in support of our commercial launch of OLPRUVA™, an innovative treatment option for patients with certain Urea Cycle Disorders (UCDs),” said Chris Schelling, CEO and Founder of Acer. “As a result, we are now ahead of our anticipated launch schedule with drug-in-channel expected in mid-June 2023. We have also made significant progress in the other key areas of our OLPRUVA™ commercialization strategy, including ongoing discussions with commercial and government insurance providers, physician outreach and awareness, and patient support and fulfillment. We look forward to continued progress in all of these areas and to delivering OLPRUVA™ to patients in mid-June 2023.”

Mr. Schelling added, “We have also started a broad outreach program to physicians who treat vascular Ehlers-Danlos Syndrome (vEDS) patients and have received overwhelming support for our ongoing Decentralized Study of Celiprolol on vEDS-related Event Reduction (DiSCOVER) Phase 3 EDSIVO™ (celiprolol) clinical trial. As a result, we anticipate enrollment in this trial to be completed by the end of this year. All of these activities are subject to additional capital.”

Q1 2023 and Recent Highlights

  • OLPRUVA™ (sodium phenylbutyrate) for oral suspension
    • In March 2023, Acer announced the presentation of data at the Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD) from a survey of UCD healthcare providers sponsored by Acer that indicated taste and odor were the most important attributes when considering treatment options and treatment adherence
    • Also at the SIMD Annual Meeting, Acer representatives met with 33 metabolic treatment providers — including nurse practitioners, registered dieticians, and physicians – from 24 metabolic treatment centers throughout the U.S. Of those metabolic treatment providers surveyed by Acer, 70% expressed a high interest in treating at least one of their patients with OLPRUVA™ in 2023
    • In preparation for OLPRUVA™’s planned drug-in-channel in mid-June 2023, the Company is in discussions with the major pharmacy benefits managers (PBM) and group purchasing organizations (GPO) representing a substantial majority of covered lives
    • Acer has also established a responsible and competitive pricing strategy designed to offer UCD patients a new treatment option at a significant discount to RAVICTI® while delivering predictable pricing that will not increase beyond the rate of inflation. Acer also plans to invest a portion of OLPRUVA™ revenue back into additional solutions aimed at improving outcomes for UCD patients
    • Finally, Acer has established and staffed its patient support program, Navigator by Acer Therapeutics, that includes a suite of services designed to provide streamlined and efficient prescription management — including benefits verification, education, and home delivery — and personalized support for OLPRUVA™ patients
  • EDSIVO™ (celiprolol)
    • Acer continues to enroll patients in its ongoing, pivotal Phase 3 Decentralized Study of Celiprolol on vEDS-related Event Reduction (DiSCOVER) clinical trial of EDSIVO™ (celiprolol) in patients with COL3A1-positive vEDS. The double-blind portion of the DiSCOVER trial is designed to include an interim analysis conducted after 28 vEDS related events, which could occur as early as approximately 18 months after completion of full enrollment; and end if statistical significance is reached after 46 vEDS-related events, estimated to occur as early as approximately 40 months after completion of full enrollment
  • ACER-801
    • In March 2023, Acer announced topline results from its Phase 2a trial evaluating ACER-801 (osanetant) for the treatment of moderate to severe Vasomotor Symptoms (VMS) associated with menopause. The trial showed that ACER-801 was generally safe and well-tolerated but did not achieve statistically significant decrease in frequency or severity of hot flashes in postmenopausal women. As a result, Acer has paused the ACER-801 program until it has completed a thorough review of the full data set from the Phase 2a trial
  • Corporate
    • Ended Q1 2023 with $6.4 million in cash and cash equivalents. Acer believes its cash and cash equivalents available at March 31, 2023, together with $0.4 million from Acer’s ATM facility subsequent to March 31, 2023, will be sufficient to fund its anticipated operating and capital requirements into late in the second quarter of 2023

Anticipated Milestones (Subject to Available Capital)

  • Mid-to-Late May 2023: Acer expects to publish OLPRUVA™’s list price, or wholesale acquisition cost (WAC), in mid-to-late May 2023
  • June 2023: Acer anticipates OLPRUVA™ drug availability in mid-June 2023
  • 2H 2023: Acer expects to begin attaining OLPRUVA™ commercial insurance coverage in 2H 2023
  • Q3 2023: Acer expects to begin attaining insurance coverage for OLPRUVA™ Medicaid patients starting in Q3 2023
  • Year End 2023: Acer anticipates enrollment completion by end of 2023 in its ongoing, pivotal Phase 3 DiSCOVER trial of EDSIVO™ in patients with COL3A1-positive vEDS
  • Acer also intends to pursue additional opportunities for potential OLPRUVA™ label expansion, including the potential for additional dosage strengths to address patients with lower weights/body surface areas, and potential administration using a gastrostomy tube (G-tube)

Q1 2023 Financial Results

Cash Position. Cash and cash equivalents were $6.4 million as of March 31, 2023, compared to $2.3 million as of December 31, 2022. Acer believes its cash and cash equivalents available at March 31, 2023, together with $0.4 million from Acer’s ATM facility subsequent to March 31, 2023, will be sufficient to fund its anticipated operating and capital requirements into late in the second quarter of 2023.

Research and Development Expenses. Research and development expenses were $2.4 million, net of collaboration funding of $0.7 million, for the three months ended March 31, 2023, as compared to $3.2 million, net of collaboration funding of $3.0 million, for the three months ended March 31, 2022. This decrease of $0.8 million was primarily due to decreases in expenses for contract manufacturing and contract research, employee-related expenses, and clinical studies related to ACER-801 and EDSIVOTM. Research and development expenses related to ACER-001 decreased in the three months ended March 31, 2023, resulting in a decrease in the recognition of the collaboration funding from the Collaboration Agreement with Relief. Research and development expenses for the three months ended March 31, 2023 were comprised of $1.0 million related to EDSIVOTM; $0.8 million related to ACER-001, offset by $0.7 million of collaboration funding; $0.8 million related to ACER-801; and $0.5 million related to other development activities.

General and Administrative Expenses. General and administrative expenses were $2.6 million, net of collaboration funding of $1.1 million, for the three months ended March 31, 2023, as compared to $3.9 million, net of collaboration funding of $2.4 million, for the three months ended March 31, 2022. This decrease of $1.3 million was primarily due to decreases in employee-related expenses, precommercial expenses, and consulting fees. General and administrative expenses related to ACER-001 decreased in the three months ended March 31, 2023, resulting in a decrease in the recognition of the collaboration funding from the Collaboration Agreement with Relief.

Net Loss. Net loss for the three months ended March 31, 2023 was $16.3 million, or $0.77 net loss per share (basic and diluted), compared to a net loss of $9.2 million, or $0.64 net loss per share (basic and diluted), for the three months ended March 31, 2022.

For additional information, please see Acer’s Quarterly Report on Form 10-Q filed today with the Securities and Exchange Commission (SEC).

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders; EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-801 (osanetant) for treatment of Vasomotor Symptoms (VMS), post-traumatic stress disorder (PTSD) and prostate cancer. For more information, visit www.www.acertx.com. 

Acer Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements about plans and strategy for the commercialization of OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with certain UCDs, including launch schedule and timing of drug in channel, progress with respect to discussions with commercial and government insurance providers, physicians outreach and awareness, and patient support and fulfillment, statements with respect to our EDSIVO clinical trial for patients with vEDS, including enrollment and timing milestones related thereto, statements about our anticipated 2023 milestones, statements about our investment of OLPRUVA revenue, and statements about our capital requirements and sufficiency and duration of our current cash and cash equivalents. Our efforts to commercialize OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS are at an early stage, we currently do not have fully developed marketing, sales or distribution capabilities, and there is no guarantee that we will be successful in our commercialization efforts.  Our pipeline products (including OLPRUVA™ for indications other than UCDs as well as EDSIVO™ and ACER-801) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the availability of financing to fund our commercialization efforts, our pipeline product development programs and our general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Corporate and IR Contact

Jim DeNike
Acer Therapeutics Inc.
jdenike@www.acertx.com
+1-844-902-6100

Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

Acer Therapeutics Announces OLPRUVA™ Commercial Launch Progressing Ahead of Schedule

NEWTON, MA – May 1, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today announced an update on progress in support of its commercial launch of OLPRUVA™ (sodium phenylbutyrate) for oral suspension, including drug availability now expected earlier than anticipated in mid-June 2023, subject to additional capital.

“Since the U.S. FDA approval of OLPRUVA™ at the end of 2022, we and our partners have been working diligently to bring this innovative treatment option to UCD patients in need as soon as possible,” said Chris Schelling, CEO and Founder of Acer. “As a result of these efforts, I am pleased to report we are ahead of our anticipated launch schedule and now expect drug availability beginning in mid-June 2023. We have also made significant progress in our ongoing discussions with payers regarding reimbursement, increased physician awareness and interest, and built out our patient support and fulfillment program. We look forward to continued progress across these and other launch initiatives and to delivering OLPRUVA™ to patients starting in mid-June 2023, subject to additional capital.”

Availability
As a result of the ongoing launch readiness efforts by Acer representatives and its manufacturing partners, Acer now expects select OLPRUVA™ dose levels to be available to patients beginning in mid-June 2023, at which time representatives from Acer’s patient support service will be available to begin accepting prescriptions. Acer expects to publish OLPRUVA™’s list price, or wholesale acquisition cost (WAC), in mid-to-late May.

Patient and Physician Awareness
Acer has also made significant progress in support of its objective to raise awareness for OLPRUVA™ as a new, alternative treatment option for certain UCD patients. Most recently, the Company attended and exhibited at the 44th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), in March 2023.

During the annual meeting, the Company met with 33 metabolic treatment providers — including nurse practitioners, registered dieticians, and physicians – from 24 metabolic treatment centers throughout the U.S. Of those metabolic treatment providers surveyed by Acer, 70% expressed a high interest in treating at least one of their patients with OLPRUVA™ in 2023. Providers surveyed also stated they viewed OLPRUVA™ as an attractive alternative therapy for UCD patients citing that despite available nitrogen scavengers in the market today, there are still unmet needs for UCD patients that may likely be addressed by prescribing OLPRUVA™.

Reimbursement
Acer Therapeutics has been engaged with both commercial and government payers as the Company anticipates approximately 50% of OLPRUVA™ prescriptions to be reimbursed through Medicaid, 45% through commercial payers, and 5% Medicare Part D. The Company is in discussions with the major pharmacy benefits managers (PBM) and group purchasing organizations (GPO) representing a substantial majority of covered lives, and Acer believes it will begin attaining OLPRUVA™ commercial insurance coverage in 2H 2023. The Company is also in negotiations with Medicaid payers in key priority states with the goal of attaining reimbursement for OLPRUVA™ Medicaid patients starting in Q3 2023.

Patient Support
Acer has also established and staffed its patient support program, Navigator by Acer Therapeutics, that includes a suite of services designed to provide streamlined and efficient prescription management — including benefits verification, education, and home delivery — and personalized support for OLPRUVA™ patients.

New FDA-Approved UCD Treatment Option: OLPRUVA
ACER-001 (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and is marketed under the brand name, OLPRUVA™. OLPRUVA™ (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).1 Please see Important Safety Information and full Prescribing Information, including Patient Information.

About Acer Therapeutics
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-801 (osanetant) for treatment of Vasomotor Symptoms (VMS), post-traumatic stress disorder (PTSD) and prostate cancer. For more information, visit www.www.acertx.com.

References

  1. OLPRUVA™ (sodium phenylbutyrate) for oral suspension. Prescribing information. Newton, MA: Acer Therapeutics Inc.

Acer Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements about survey data to be presented, including the timing, detail and content thereof, and OLPRUVA™ launch activities, including timing thereof, the the addition of resources, the launch of patient support services, discussions with payers and organizations, and the Company’s pricing strategy and related plans and expectations. Our efforts to commercialize OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS are at an early stage, we currently do not have fully developed marketing and sales capabilities, and there is no guarantee that we will be successful in our commercialization efforts.  Our pipeline products (including OLPRUVA™ for indications other than UCDs) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the availability of financing to fund our commercialization efforts, our pipeline product development programs and general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Corporate and IR Contacts
Jim DeNike
Acer Therapeutics Inc.
jdenike@www.acertx.com
+1-844-902-6100

Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

Acer Therapeutics Reports Q4 and Full Year 2022 Financial Results and Provides Corporate Update

NEWTON, MA – March 27, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today reported financial results for Q4 and full year ended December 31, 2022, and provided an update on the Company’s recent corporate developments.

“2022 marked Acer’s first U.S. Food and Drug Administration (FDA) product approval, for OLPRUVA™ (sodium phenylbutyrate), an innovative and responsibly priced treatment option for certain people with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS),” commented Chris Schelling, CEO and Founder of Acer. “We remain committed to our mission to provide transformative treatments to underserved and overlooked patients with rare and life-threatening diseases and will continue to focus our resources on launch preparation for OLPRUVA™ as well as continued pivotal Phase 3 clinical trial evaluation of EDSIVO™ for vEDS, both of which are subject to additional capital.”

Q4 2022 and Recent Highlights

  • OLPRUVA™ (sodium phenylbutyrate) for oral suspension
    • In October 2022, Acer was issued a Notice of Allowance from the US Patent and Trademark Office (USPTO) for US patent application No. 16/624,834 (now issued as US Patent No. 11,517,547) for claims related to a kit comprising a combination therapeutic product composed of sodium phenylbutyrate or glycerol phenylbutyrate and sodium benzoate
    • In December 2022, Acer announced FDA approval of OLPRUVA™ (sodium phenylbutyrate) for oral suspension, a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of CPS, OTC or AS. OLPRUVA™ is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. More information is available at www.OLPRUVA.com
    • In March 2023, Acer announced an update on its OLPRUVA™ U.S. commercial launch activities, including the ongoing addition of commercial and medical affairs resources, the introduction of its OLPRUVA™ Navigator by Acer Therapeutics patient support service, a predictable and responsible price commitment, and anticipated drug availability by early July 2023 (subject to additional capital)
    • In March 2023, Acer announced results from a survey designed to quantify preferences of healthcare providers for Urea Cycle Disorders (UCDs) presented at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting. The authors concluded that optimizing nitrogen-binding medications for UCD treatment to facilitate and encourage increased patient adherence through masking taste/odor and/or enhancing other aspects of the patient experience may support improved outcomes in UCDs
  • EDSIVO™ (celiprolol)
    • In October 2022, Acer announced that the USPTO issued a Notice of Allowance for Acer’s patent application No. 16/930,208 and subsequently issued on December 13, 2022, as US Patent #11,523,997, for claims related to certain methods of treating vascular Ehlers-Danlos syndrome (vEDS) with celiprolol
  • ACER-801 (osanetant)
    • In March 2023, Acer announced that topline results from Acer’s Phase 2a proof of concept clinical trial to evaluate ACER-801 (osanetant) as a potential treatment for moderate to severe Vasomotor Symptoms (VMS) associated with menopause showed that ACER-801 was safe and well-tolerated but did not achieve statistical significance when evaluating ACER-801’s ability, when compared to placebo, to decrease the frequency or severity of hot flashes in postmenopausal women. As a result, Acer is pausing the ACER-801 program until Acer has conducted a thorough review of the full data set
  • Corporate
    • Ended Q4 2022 with $2.3 million in cash and cash equivalents. Acer believes its cash and cash equivalents available at December 31, 2022, together with the gross proceeds of $7.0 million from a second term loan with SWK Funding LLC which closed on January 31, 2023, $4.1 million from  Acer’s ATM facility subsequent to December 31, 2022, and $2.7 million from a sale of securities (including pursuant to a registered direct offering of shares of common stock and prefunded warrants) which closed on March 24, 2023, will be sufficient to fund Acer’s anticipated operating and capital requirements into the middle of Q2 2023

Anticipated Milestones (Subject to Available Capital)

  • Q2 2023: Acer intends to continue to add resources to establish its commercial and medical affairs presence in the U.S. in support of its OLPRUVA™ launch expected in Q2 2023 
  • Q2 2023: Negotiations with the major commercial payers and state Medicaid organizations regarding access for OLPRUVA™ are ongoing, and representatives from Acer’s OLPRUVA™ Navigator by Acer Therapeutics patient support service are expected to begin accepting prescriptions in late Q2 2023
  • Q2 2023: Acer expects OLPRUVA™ wholesale acquisition cost (WAC) pricing to be listed publicly in Q2 2023
  • Early July 2023: Acer anticipates OLPRUVA™ drug availability in early July 2023
  • Q4 2023: Acer anticipates completing enrollment in its ongoing, pivotal Phase 3 DiSCOVER trial of EDSIVO™ (celiprolol) in patients with COL3A1-positive vEDS. The double-blind portion of the DiSCOVER trial is designed to end if statistical significance is reached at an interim analysis which occurs at accrual of 28 vEDS-related events, estimated to occur as early as approximately 18 months after completion of full enrollment, or after accrual of 46 vEDS-related clinical events
  • Acer intends to explore additional lifecycle opportunities for OLPRUVA™ (sodium phenylbutyrate) in various disorders where proof of concept data exists, including in Maple Syrup Urine Disease (MSUD), Pyruvate Dehydrogenase Complex Deficiency (PCDC), rare pediatric epilepsies and various liver disorders

Q4 and Full Year 2022 Financial Results

Cash Position. Cash and cash equivalents were $2.3 million as of December 31, 2022, compared to $12.7 million as of December 31, 2021. Acer believes its cash and cash equivalents as of December 31, 2022, together with the gross proceeds of $7.0 million from a second term loan with SWK Funding LLC which closed on January 31, 2023, $4.1 million from Acer’s ATM facility subsequent to December 31, 2022, and $2.7 million from a sale of securities (including pursuant to a registered direct offering of shares of common stock and prefunded warrants) which closed on March 24, 2023, will be sufficient to fund its currently anticipated operating and capital requirements into the middle of Q2 2023.

Research and Development Expenses. Research and development expenses were $2.6 million, net of collaboration funding of $1.0 million, for the three months ended December 31, 2022, as compared to $1.7 million, net of collaboration funding of $1.3 million, for the three months ended December 31, 2021. Research and development expenses for the three months ended December 31, 2022 were comprised of $1.1 million related to ACER-001, offset by $1.0 million of collaboration funding; $1.2 million related to ACER-801; $1.0 million related to EDSIVO™; and $0.3 million related to other development activities. Research and development expenses were $11.9 million, net of collaboration funding of $7.8 million, for the year ended December 31, 2022, as compared to $6.5 million, net of collaboration funding of $6.1 million, for the year ended December 31, 2021. This increase of $5.4 million was primarily due to increases in employee-related expenses and expenses related to clinical studies, partially offset by the increase in recognition of contra-expense from the collaboration funding from Acer’s Collaboration and License Agreement with Relief Therapeutics Holding AG (Relief) for the development and commercialization of OLPRUVA™ (Collaboration Agreement).

General and Administrative Expenses. General and administrative expenses were $2.6 million, net of collaboration funding of $1.2 million, for the three months ended December 31, 2022, as compared to $3.1 million, net of collaboration funding of $1.6 million, for the three months ended December 31, 2021. General and administrative expenses were $12.7 million, net of collaboration funding of $8.2 million, for the year ended December 31, 2022, as compared to $10.7 million, net of collaboration funding of $3.2 million, for the year ended December 31, 2021. This increase of $2.0 million was primarily due to increases in precommercial activities, employee-related expenses, professional services, as well as information technology, partially offset by the increase in the recognition of contra-expense from the collaboration funding from the Collaboration Agreement with Relief.

Net Loss. Net loss for the three months ended December 31, 2022 was $9.4 million, or $0.54 net loss per share (basic and diluted), compared to a net loss of $4.4 million, or $0.31 net loss per share (basic and diluted), for the three months ended December 31, 2021. Net loss for the year ended December 31, 2022 was $26.2 million, or $1.66 net loss per share (basic and diluted), compared to a net loss of $15.4 million, or $1.08 net loss per share (basic and diluted), for the year ended December 31, 2021.

For additional information, please see Acer’s Annual Report on Form 10-K filed today with the SEC.

About Acer Therapeutics
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-801 (osanetant) for treatment of Vasomotor Symptoms (VMS), post-traumatic stress disorder (PTSD) and prostate cancer. For more information, visit www.www.acertx.com.  

Acer Forward-Looking Statements This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements about plans for the commercialization of OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS, including negotiations with commercial payers and Medicaid organizations regarding access as well as the timing of drug availability and the expected commercial launch, statements about plans and potential milestones for the continued clinical development of OLPRUVA™ in other indications, statements about plans and potential milestones for the continued clinical development of EDSIVO™ for treatment of vEDS in patients with a confirmed type III collagen (COL3A1) mutation, statements about plans for the development of ACER-801, statements about our anticipated 2023 milestones, and statements about our capital requirements and sufficiency and duration of our current cash and cash equivalents. Our efforts to commercialize OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS are at an early stage, we currently do not have fully developed marketing, sales or distribution capabilities, and there is no guarantee that we will be successful in our commercialization efforts.  Our pipeline products (including OLPRUVA™ for indications other than UCDs as well as EDSIVO™ and ACER-801) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the availability of financing to fund our commercialization efforts, our pipeline product development programs and our general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Corporate and IR Contact

Jim DeNike
Acer Therapeutics Inc.
jdenike@www.acertx.com
+1-844-902-6100

Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

Acer Therapeutics Announces Data Presented at Society for Inherited Metabolic Disorders Annual Meeting Identifying Preferred Urea Cycle Disorder Treatment Attributes

NEWTON, MA – March 22, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today announced data was presented from a survey designed to quantify preferences of healthcare providers for Urea Cycle Disorders (UCDs) at the 44th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), March 18th-21st in Salt Lake City, Utah. Results from the discrete choice experiment presented showed that taste and odor were the most important attributes, as identified by UCD healthcare providers, that influence overall prescription of, and patient adherence to, UCD treatments when evaluating nitrogen-binding medications (such as sodium phenylbutyrate and glycerol phenylbutyrate).

“Nitrogen-binding medications, such as sodium phenylbutyrate or glycerol phenylbutyrate, can be efficacious in the treatment of UCDs if patients are adherent to their prescribed treatment1,2,” stated Robert Steiner, M.D., Professor at the University of Wisconsin School of Medicine and Public Health. “However, 25% of life-threatening hyperammonemic crises in patients with UCDs may be precipitated by a lack of adherence to medications and/or diet and certain attributes of existing nitrogen-binding medications may negatively impact adherence3. Given these results, alternative treatment options are urgently needed.”

Healthcare Provider UCD Treatment Preference Data Presented at SIMD 2023
A copy of the poster presentation from the 2023 SIMD Meeting is available on Acer’s website at: https://www.acertx.com/wp-content/uploads/2023/03/2023-SIMD-DCE-Poster.pdf

Poster #97: Quantifying Preferences for Urea Cycle Disorder Treatments Using a Discrete-Choice Experiment4

This poster presented at SIMD summarizes results from a web-based, quantitative survey study conducted on Acer’s behalf, using a discrete choice experiment (DCE) methodology.  This study was designed to quantify the most-desired product attributes that may influence overall prescription of, and patient adherence to, nitrogen-binding medications (such as sodium phenylbutyrate and glycerol phenylbutyrate) for the treatment of UCDs as identified by the survey participants.

Of the 51 healthcare providers that completed the survey, most HCPs reported dissatisfaction with current treatment options [mean rating (SD)=5.4 (1.7); Likert scale with 1 = not at all satisfied through 9 = extremely satisfied]. The results of the survey showed that taste and odor were the most important attributes for both prescribing and for patient adherence and compliance . The authors concluded that optimizing nitrogen-binding medications for UCD treatment to facilitate and encourage increased patient adherence through masking taste and odor, and/or enhancing other aspects of the patient experience, may support improved outcomes in UCDs.

“OLPRUVA™5 leverages the well-established efficacy of sodium phenylbutyrate in an innovative dual-coating formulation designed for palatability,” said Adrian Quartel, MD, FFPM, CMO of Acer. “We are pleased to offer an innovative, responsibly priced treatment option to patients with UCDs and remain committed to working closely with the UCD community to ensure that those affected by this terrible disease live their lives with as few challenges and interruptions as possible.”

ACER-001 (sodium phenylbutyrate) Palatability Data Presented at SIMD and GMDI 2022
Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders6,7

Results from two Phase 1, open-label, repeated measures, taste assessment studies of ACER-001 (sodium phenylbutyrate) suspension and sodium phenylbutyrate (BUPHENYL®) powder were presented at the SIMD Annual Meeting and Genetic Metabolic Dieticians International (GMDI) Conference in April 2022 and May 2022, respectively. ACER-001 for the treatment of certain UCDs is now marketed in the U.S. as OLPRUVA™ (sodium phenylbutyrate) following FDA approval in December 2022. Results from both studies concluded that ACER-001 (sodium phenylbutyrate) suspension was shown to have overall lower flavor intensity scores than sodium phenylbutyrate (BUPHENYL®) powder when administered within five minutes of preparation.

New FDA-Approved UCD Treatment Option: OLPRUVA
ACER-001 (sodium phenylbutyrate) was approved for the treatment of certain UCDs in December 2022 and is marketed under the brand name, OLPRUVA™. OLPRUVA™ (sodium phenylbutyrate) for oral suspension is a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).4 Please see Important Safety Information and full Prescribing Information, including Patient Information.

About Acer Therapeutics
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-801 (osanetant) for treatment of Vasomotor Symptoms (VMS), post-traumatic stress disorder (PTSD) and prostate cancer. For more information, visit www.www.acertx.com.

References

  1. Batshaw ML, Tuchman M, Summar M, Seminara J, Members of the Urea Cycle Disorders C. A longitudinal study of urea cycle disorders. Mol Genet Metab. 2014;113(1-2):127-130.
  2. Pena-Quintana L, Llarena M, Reyes-Suarez D, Aldamiz-Echevarria L. Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Prefer Adherence. 2017;11:1489-1496.
  3. Pena-Quintana L, Llarena M, Reyes-Suarez D, Aldamiz-Echevarria L. Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Prefer Adherence. 2017;11:1489-1496.
  4. Edelblut J, et al. Quantifying Preferences for Urea Cycle Disorder Treatments Using a Discrete-Choice Experiment. SIMD March 2023
  5. OLPRUVATM (sodium phenylbutyrate) for oral suspension. Prescribing information. Newton, MA: Acer Therapeutics Inc.
  6. Steiner R, et al. The Pharmacokinetics of Taste-Masked Sodium Phenylbutyrate (ACER-001) for the Treatment of Urea Cycle Disorders Under Fasting and Fed Conditions in Healthy Volunteers. SIMD April 2022
  7. Cederbaum S, et al. Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders. GMDI May 2022

Acer Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements about survey data to be presented, including the timing, detail and content thereof, and OLPRUVA™ launch activities, including the addition of resources, the launch of patient support services, discussions with payers and organizations, and the Company’s pricing strategy and related plans and expectations. Our efforts to commercialize OLPRUVA™ for oral suspension in the U.S. for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC, or AS are at an early stage, we currently do not have fully developed marketing, sales or distribution capabilities, and there is no guarantee that we will be successful in our commercialization efforts.  Our pipeline products (including OLPRUVA™ for indications other than UCDs) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the availability of financing to fund our commercialization efforts, our pipeline product development programs and general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Corporate and IR Contacts
Jim DeNike
Acer Therapeutics Inc.
jdenike@medinfo+1-844-902-6100

Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

Acer Therapeutics Announces $2.675 Million Registered Direct Offering Priced At-the-Market Under Nasdaq Rules

NEWTON, MA – March 22, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today announced that it has entered into a definitive agreement for the purchase and sale of 2,920,306 shares of the Company’s common stock (or common stock equivalents) at a purchase price of $0.916 per share of common stock (or common stock equivalent) in a registered direct offering priced at-the-market under Nasdaq rules. In addition, in a concurrent private placement, the Company will issue warrants to purchase up to 2,920,306 shares of common stock. The warrants will have an exercise price of $0.791 per share, will be exercisable immediately following issuance and have a term of five and one-half years from the issuance date.

H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The closing of the offering is expected to occur on or about March 24, 2023, subject to the satisfaction of customary closing conditions. The gross proceeds to the Company from the offering are expected to be approximately $2.675 million, before deducting the placement agent’s fees and other offering expenses payable by the Company. The Company intends to use the net proceeds from this offering for general corporate purposes and working capital.

The shares of common stock described above (but not the warrants issued in the concurrent private placement or the shares of common stock underlying such warrants) are being offered by the Company pursuant to a “shelf” registration statement on Form S-3 (File No. 333-261342) initially filed with the Securities and Exchange Commission (the “SEC”) on November 24, 2021 and declared effective by the SEC on December 7, 2021. The offering of the shares of common stock is made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the registered direct offering will be filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained, when available, on the SEC’s website at http://www.sec.gov or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (212) 865-5711 or e-mail at placements@hcwco.com.

The warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”), and, along with the shares of common stock underlying the warrants, have not been registered under the Securities Act, or applicable state securities laws. Accordingly, the warrants and underlying shares of common stock may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About Acer Therapeutics
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-801 (osanetant) for treatment of Vasomotor Symptoms (VMS), post-traumatic stress disorder (PTSD) and prostate cancer. For more information, visit www.www.acertx.com.

Acer Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements regarding the consummation of the offering, the satisfaction of closing conditions and the use of proceeds from the offering, as well as statements about the continued development of ACER-801 for treatment of VMS or other indications, the continued development of EDSIVO™, and launch preparations for OLPRUVA™. Our pipeline products (including ACER-801) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, market and other conditions, the availability of financing to fund our pipeline product development programs, commercialization efforts and general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Corporate and IR Contacts
Jim DeNike
Acer Therapeutics Inc.
jdenike@medinfo+1-844-902-6100

Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-332-895-3226

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