NEWTON, MA – March 31, 2022 – 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 received notice from The Nasdaq Stock Market (Nasdaq) on March 30, 2022 informing Acer that it has regained compliance with the minimum market value of listed securities standard under Nasdaq Listing Rule 5550(b)(2) for continued listing on the Nasdaq Capital Market, and the matter is now closed.
On December 29, 2021, Nasdaq notified Acer that it had failed to maintain a minimum market value of listed securities of $35 million over the previous 30 consecutive business days as required by the Nasdaq Capital Market set forth in Listing Rule 5550(b)(2). Following its notification in December 2021, Nasdaq determined that Acer regained compliance with the Rule after the Company’s market value of listed securities was $35 million or greater for 10 consecutive business days from March 16 through March 29, 2022.
About Acer Therapeutics Inc. 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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.www.acertx.com.
Investor Contact: Hans Vitzthum LifeSci Advisors Ph: 617-430-7578 [email protected]
Jim DeNike Acer Therapeutics Inc. Ph: 844-902-6100 [email protected]
NEWTON, MA – March 30, 2022 – 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 the enrollment of the first patient in its Phase 2a randomized, double-blind, placebo-controlled, dose-ranging trial evaluating the efficacy, safety, and pharmacokinetics (PK) of ACER-801 (osanetant) at different doses, compared to placebo, for the treatment of moderate to severe Vasomotor Symptoms (VMS) associated with menopause.
The trial is intended to enroll approximately 56 post-menopausal women aged 40-65, who experience moderate to severe hot flashes. Subjects are to receive twice daily doses of either 50 mg, 100 mg, or 200 mg of ACER-801 or placebo (14 subjects per treatment arm) over a 14-day treatment period, followed by a 14-day safety follow-up assessment. Additional trial details will be available at ClinicalTrials.gov. Results from this trial could provide proof of concept data in post-menopausal women and could inform ACER-801 dosing and a development path forward in patients with induced Vasomotor Symptoms (iVMS).
“Vasomotor symptoms, either induced or as part of peri- and post-menopause, are a significant and debilitating burden on patients. We are excited to start evaluating the safety and efficacy of ACER-801 in this dose-ranging study in post-menopausal women with moderate to severe VMS. We believe this study marks an important first step in finding a meaningful treatment for patients suffering from iVMS,” said Adrian Quartel, MD, CMO of Acer. “We are looking forward to the results of this trial to inform us on the potential next steps in our development program of ACER-801.”
About iVMS and ACER-801 VMS are generally defined as hot flashes, flushing and night sweats that most often occur in women entering or in menopause. VMS can also be induced (iVMS) by anti-androgen and anti-estrogen cancer therapies and surgical procedures that can lead to treatment non-compliance.1,2 VMS are caused by low estrogen levels leading to increased stimulatory signaling of neurokinin B (NKB) on the KNDy neuron in the hypothalamus. A non-hormonal treatment to manage iVMS is needed as estrogen is contraindicated for the management of VMS in patients with hormone-positive tumors, including breast and prostate tumors.
ACER-801 (osanetant) is a novel, non-hormonal, neurokinin 3 receptor (NK3R) antagonist that could offer a potential treatment option with meaningful improvement of VMS for patients with iVMS by blocking the stimulatory signaling of NKB on the KNDy neurons. Direct human safety evidence is available from 23 completed Phase 1 and 2 studies in which approximately 400 healthy subjects and 820 patients were treated with osanetant for schizophrenia, depression and other indications. Data from these studies indicated no major safety concerns after single-dose and repeat-dose administration.3 ACER-801 is orally bioavailable4 and readily crosses the blood-brain barrier.5 Acer believes that several disorders involving the hypothalamus-pituitary-gonadal axis could be investigated for potential benefit from treatment with an NK3R antagonist.
ACER-801 is an investigational product candidate which has not been approved by FDA or any other regulatory authority. There is no guarantee that this product candidate will receive regulatory authority approval in any territory or become commercially available for any indications.
About Acer Therapeutics Inc. 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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.www.acertx.com.
References
Kotsopoulos J, Huzarski T, Gronwald J, Moller P, Lynch HT, Neuhausen SL, et al. Hormone replacement therapy after menopause and risk of breast cancer in BRCA1 mutation carriers: a case-control study. Breast Cancer Research and Treatment 2016;155(2):365–73.
Guidozzi F. Hormone therapy after prophylactic risk-reducing bilateral salpingo-oophorectomy in women who have BRCA gene mutation. Climacteric 2016;19(5): 419–22.
Meltzer H, et al. Placebo-controlled evaluation of four novel compounds for the treatment of schizophrenia and schizoaffective disorder. June 2004; 161(6):975-84.
Single and Repeated Ascending Oral Dose Tolerability Study of SR142801 in Healthy Male Subjects. Sanofi Clinical Study Report February 2001.
Gueudet C, et al. Blockade of neurokinin3 receptors antagonizes drug-induced population response and depolarization block of midbrain dopamine neurons in guinea pigs. Synapse. 1999 Jul;33(1):71-9.
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 regarding strategy, future operations, timelines for clinical study enrollment or regulatory actions, or otherwise, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions for ACER-001, ACER-801, EDSIVO™ or our other products; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. 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 our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. 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. You may access these documents for no charge at http://www.sec.gov.
NEWTON, MA – March 7, 2022 – 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 convertible note and loan financing facilities for up to $48.5 million with affiliates of Marathon Asset Management L.P. (Marathon) and SWK Holdings Corporation (Nasdaq: SWKH), subject to certain conditions. Proceeds from these financings would be used to advance Acer’s pipeline.
Summaries of the financing facilities are as follows:
Marathon $6.0 Million Secured Convertible Note Under the terms of a secured convertible note agreement, and subject to certain customary closing conditions, Marathon is expected to fund $6.0 million at a closing no later than March 14, 2022. The secured convertible note issued to Marathon would have a 3-year term, bear interest at 6.5% per annum, be secured by a lien on all of Acer’s assets, be convertible at Marathon’s option into shares of Acer common stock at a conversion price of $2.50 per share, and be redeemable at Marathon’s option during the 30-day periods beginning 12 months, 18 months and 24 months after issuance.
SWK $6.5 Million Secured Loan Under the terms of a senior secured term loan agreement, and subject to certain customary closing conditions as well as the funding of the Marathon secured convertible note referenced above, SWK is expected to fund $6.5 million at a closing no later than March 14, 2022. If the Company’s drug candidate ACER-001 (sodium phenylbutyrate) receives FDA approval for marketing (referred to as ACER-001 Marketing Approval) on or before September 30, 2022, then this loan would be payable within 12 business days of such approval; otherwise, this loan would have a 2-year term. As background, FDA has assigned a PDUFA target action date of June 5, 2022, to Acer’s pending New Drug Application for ACER-001 for the treatment of patients with urea cycle disorders. This loan would bear interest per annum at the sum of 3-month LIBOR (with a floor of 1%) plus 11% and would be secured by a senior lien on all of Acer’s assets. If the loan is paid off or ACER-001 Marketing Approval occurs prior to September 30, 2022, SWK would receive an exit fee from Acer yielding a return (inclusive of principal, interest and origination and other fees) of 1.3x the outstanding principal; otherwise, SWK would receive an exit fee from Acer yielding a return of 1.5x the outstanding principal. SWK would also receive a warrant with a 7-year term to acquire 150,000 shares of Acer common stock at an exercise price of $2.46 per share.
Proceeds from the foregoing two facilities would currently be expected to finance into mid-2022 Acer’s planned additional investments in ACER-001 pre-commercial activities as well as activities relating to a planned ACER-801 (osanetant) Phase 2a proof of concept trial in postmenopausal women and a planned EDSIVO™ (celiprolol) pivotal Phase 3 trial in COL3A1 positive vascular Ehlers-Danlos syndrome (vEDS) patients. A portion of the proceeds from these financings would also be required to pay the cost of obtaining such financing, including origination fees, transaction costs and financial advisor fees, as well as a commitment fee and certain other costs associated with the contingent Marathon lending arrangement referenced below. Note that additional capital will be required to conduct beyond mid-2022 and complete the planned pivotal Phase 3 trial of EDSIVO™.
Marathon $42.5 Million Secured Loan Acer has also entered into a senior secured loan agreement with Marathon for an additional $42.5 million which, in addition to customary conditions, is contingent upon ACER-001 Marketing Approval occurring not later than December 31, 2022. This loan would have a 6-year term, bear interest at 13.5% per annum (with Acer having the option to capitalize up to 4% for the first 3 years), be secured by a senior lien on all of Acer’s assets, have the outstanding principal amortize at a monthly rate of 2.78% commencing on the third anniversary, and be subject to prepayment fees ranging from 5% if repaid prior to the third anniversary to 1% if repaid after five years. Acer and Marathon have also entered into a synthetic royalty agreement whereby, if this loan is funded, Acer would pay Marathon, on a quarterly basis, 2% of certain aggregate revenue from ACER-001 during that quarter (i.e., 2% of the net sales and of the amount of certain other payments), subject to a cap on the aggregate amount of such payments of $15 million. The funds from this facility would be used to pay off the SWK loan referenced above (i.e., $6.5 million in principal, plus an exit fee as noted above, leaving loans of $48.5 million in principal between the two Marathon facilities) as well as provide capital for an ACER-001 commercial product launch, if ACER-001 is approved, and advancement of other pipeline programs and ongoing operations into the second half of 2023 based upon current expectations. A portion of the proceeds from this financing would also be used to pay certain costs of obtaining such financing, including a commitment fee, transaction costs and financial advisor fees. If this loan is funded, then Acer may also request an increase in the loan amount by $50 million, although any such increase is subject to Marathon’s sole discretion.
“Accessing these loan facilities is a significant achievement for Acer as they provide capital to fund advancement of our pipeline programs at what we believe will be a lower cost of capital than equity financing,” said Chris Schelling, CEO and Founder of Acer. “Our financing agreements with Marathon and SWK significantly extend our cash runway and we appreciate their commitment to our mission of delivering much-needed therapies to patients in need.”
“This transaction represents the beginning of Marathon’s partnership with Acer,” said Dr. Evan Bedil, Head of Healthcare at Marathon. “Acer is led by an experienced and highly talented management team with a suite of innovative medications to advance the health and wellbeing of those affected by rare life-threatening metabolic disease and to improve the quality of life of post-menopausal women suffering from vasomotor symptoms. Our financing commitments provide Acer with growth capital to commercialize their products and advance the Company’s pipeline.”
“The proceeds from our loan, together with Marathon’s convertible note, will provide Acer the necessary funding to potentially achieve near-term value inflection points,” said Winston Black, CEO of SWK. “We look forward to Acer’s continued progress as it prepares for ACER-001 PDUFA in June and initiates important clinical trials.”
Each of these loan transactions includes additional terms which are customary in transactions of this type. Further information with respect to the debt financing agreements with Marathon and SWK is contained in a Current Report on Form 8-K filed by Acer with the Securities and Exchange Commission. ACER-001, ACER-801 and EDSIVO™ are investigational product candidates that have not been approved by FDA. There is no guarantee that any of these product candidates will receive regulatory authority approval in any territory or become commercially available for the indications under investigation.
Reedland Capital Partners, acting through Weild & Co., member FINRA|SIPC, served as financial advisor to Acer in connection with these financing transactions. For more information, please visit www.reedland.com.
About ACER-001 ACER-001 (sodium phenylbutyrate) is being developed for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD). ACER-001 is a nitrogen-binding agent in development for use as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). ACER-001’s multi-particulate dosage formulation for oral administration is designed to minimize the aversive taste and odor1 of sodium phenylbutyrate while quickly dissolving in the stomach. The ACER-001 NDA for UCDs is currently under FDA review with a PDUFA target action date of June 5, 2022. ACER-001 is also being developed for MSUD and has been granted orphan drug designation by the FDA for this indication. ACER-001 is an investigational product candidate which has not been approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority.
About Acer Therapeutics Inc. 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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including UCDs and MSUD; ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics Holding SA for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.
About Marathon Asset Management LP Marathon Asset Management LP, with $24 billion of assets under management, was formed in 1998 by Bruce Richards (Chairman & Chief Executive Officer) and Louis Hanover (Chief Investment Officer). The firm seeks attractive absolute returns through investments in the global capital markets and the private credit markets whereby it is known for its ability to provide capital solutions to companies across industries. Marathon’s healthcare team, led by Dr. Evan Bedil, is known for buying pharmaceutical royalties in addition to providing capital solutions across the capital structure to emerging biopharmaceutical companies. Marathon has significant experience investing in companies through multiple cycles and possesses a broad-based skill set and proprietary platform to research, analyze and act upon complex capital structures and situations. For additional information, please visit www.marathonfund.com.
About SWK Holdings Corporation SWK Holdings Corporation (SWKH.OB) is a specialized finance company with a focus on the global healthcare sector. SWK partners with ethical product marketers and royalty holders to provide flexible financing solutions at an attractive cost of capital to create long-term value for both SWK’s business partners and its investors. SWK believes its financing structures achieve an optimal partnership for companies, institutions and inventors seeking capital for expansion or capital and estate planning by allowing its partners to monetize future cash flow with minimal dilution to their equity stakes. Additional information is available on the company’s website at www.swkhold.com.
References
Peña-Quintana L, et al. Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Prefer Adherence. 2017 Sep 6;11:1489-1496.
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 regarding regulatory submissions, actions or approvals, financings, cash position, liquidity, strategy, future operations, timelines, future financial position, future revenues, projected expenses, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; our ability to close upon and obtain the proceeds of any proposed financing as well as to satisfy the ongoing conditions and requirements for maintaining related financing facilities and avoiding default or an accelerated repayment requirement; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability, in addition to currently proposed financings, to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. 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 our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. 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 Quarterly Reports on Form 10-Q and 10-Q/A, and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.
Investor Contact: Hans Vitzthum LifeSci Advisors Ph: 617-430-7578 [email protected]
Jim DeNike Acer Therapeutics Inc. Ph: 844-902-6100 [email protected]
NEWTON, MA –March 2, 2022 – 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 fourth quarter and full year ended December 31, 2021 and provided an update on the Company’s recent corporate developments.
“We made significant progress this year in advancing our pipeline programs, including filing of our New Drug Application and key patent issuances for ACER-001, and receipt of two Investigational New Drug application clearances for our ACER-801 proof-of-concept clinical trial and our EDSIVO pivotal clinical trial,” said Chris Schelling, CEO and Founder of Acer. “We also recently welcomed Dr. Adrian Quartel as Chief Medical Officer who brings extensive experience and a proven track record in the clinical development, approval, and launch of treatments for rare disease or rare genetic disorders, including KUVAN®, VIMIZIM® and Brineura®.”
Q4 2021 and Recent Highlights
ACER-001 (sodium phenylbutyrate)
Announced in October 2021 that FDA accepted for filing the New Drug Application (NDA) for ACER-001 for the treatment of patients with urea cycle disorders (UCDs) and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 5, 2022
Announced in October 2021 that the U.S. Patent and Trademark Office (USPTO) issued a new U.S. patent to Acer for certain claims related to ACER-001. Patent 11,154,521 covers pharmaceutical composition claims related to ACER-001’s taste-masked, multi-particulate dosage formulation for oral administration and has an expiration date in 2036
In December 2021, the USPTO also issued a new U.S. patent 11,202,767 to Acer that covers certain methods of use claims related to ACER-001 and has an expiration date in 2036
Announced in January 2022 the acceptance of four ACER-001 abstracts for poster presentations at the upcoming Society for Inherited Metabolic Disorders (SIMD) Annual Meeting on April 10-13, 2022, and the Genetic Metabolic Dieticians International (GMDI) Conference on May 4-7, 2022. Acer will also be exhibiting at the American College of Medical and Genomics (ACMG) Annual Clinical Genetics Meeting March 23-25, 2022
ACER-801 (osanetant)
Announced in December 2021 FDA clearance of Acer’s Investigational New Drug (IND) application for its selective non-peptide neurokinin 3 receptor (NK3R) antagonist, ACER-801, for the potential treatment of induced Vasomotor Symptoms (iVMS)
EDSIVO™ (celiprolol)
In January 2022, FDA cleared the EDSIVOTM IND for the treatment of patients with COL3A1+ vascular Ehlers-Danlos Syndrome (vEDS). Acer is in discussions with FDA, through a special protocol assessment (SPA), to seek agreement on its planned DiSCOVER (Decentralized Study of Celiprolol on vEDS-related Event Reduction) trial
In February 2022, Acer submitted to FDA its request for EDSIVO™ Breakthrough Therapy Designation
Corporate
Announced in February 2022 the appointment of Adrian Quartel, M.D., FFPM, as Chief Medical Officer. Dr. Quartel is an industry veteran with over 20 years of drug development experience tasked with overseeing Acer’s clinical development, medical affairs, regulatory affairs and other scientific and medical functions. Dr. Quartel joins Acer from Adamas Pharmaceuticals where he served as Chief Medical Officer overseeing research and development, as well as medical affairs and regulatory functions. Prior to Adamas, Dr. Quartel held senior medical leadership positions at BioMarin Pharmaceuticals Inc., Astellas, Chiltern, and ICON Clinical Research
Ended Q4 2021 with $12.7 million in cash and cash equivalents. Acer believes its cash and cash equivalents available as of December 31, 2021 and the $5.0 million received in January 2022 under Acer’s Collaboration and License Agreement with Relief Therapeutics Holding AG (Relief Collaboration) will be sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for the planned ACER-001 (MSUD), ACER-801 and EDSIVO™ clinical trials
Anticipated Milestones
ACER-001 (sodium phenylbutyrate)
June 5, 2022: FDA has assigned a PDUFA target action date of June 5, 2022, following its acceptance for filing of the NDA for ACER-001 (sodium phenylbutyrate) for the treatment of patients with UCDs
H2 2022: Acer plans to initiate in H2 2022 a clinical study evaluating ACER-001 in MSUD, subject to additional capital
ACER-801 (osanetant)
Q1 2022: Acer plans to initiate in Q1 2022 a Phase 2a clinical trial designed to evaluate the effect of ACER-801 at different doses, compared to placebo, on the frequency and severity of vasomotor symptoms associated with menopause, subject to additional capital
H2 2022: Assuming such initiation, the Phase 2a clinical trial results would be anticipated in H2 2022, which, if positive, could provide proof of concept data informing ACER-801 dosing and a development path forward in patients with iVMS
Q2 2022: If SPA agreement is reached with FDA, in Q2 2022 Acer intends to initiate the pivotal Phase 3, randomized, double-blind, placebo-controlled, decentralized clinical trial for EDSIVO™ for patients with COL3A1+ vEDS, subject to additional capital. The DiSCOVER trial is estimated to take approximately 3.5 years to complete, once fully enrolled
Q2 2022: A response to Acer’s request for EDSIVO™ Breakthrough Therapy Designation from FDA is anticipated in Q2 2022
ACER-2820 (emetine)
Ongoing: Further advancement of the emetine program for treatment of certain viruses, including cytomegalovirus, zika, dengue, ebola, and COVID-19, is dependent on Acer’s ability to raise non-dilutive capital for this program
Q4 and Full Year 2021 Financial Results
Cash position. Cash and cash equivalents were $12.7 million as of December 31, 2021, compared to $5.8 million as of December 31, 2020. Acer believes its cash and cash equivalents available as of December 31, 2021, plus the $5.0 million second tranche of the Second Development Payment received on January 14, 2022 per the Relief Collaboration, will be sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for the planned ACER-001 (MSUD), ACER-801 and EDSIVO™ clinical trials.
Research and Development Expenses. Research and development expenses were $1.7 million, net of collaboration funding of $1.3 million, for the three months ended December 31, 2021, compared to $3.5 million for the three months ended December 31, 2020. Research and development expenses for the three months ended December 31, 2021 were comprised of $1.1 million related to ACER-001, offset by $1.3 million of collaboration funding; $0.7 million related to ACER-801; $0.7 million related to EDSIVO™; $0.1 million related to ACER-2820; and $0.4 million related to other development activities. Research and development expenses were $6.5 million, net of collaboration funding of $6.1 million, for the year ended December 31, 2021, as compared to $11.8 million for the year ended December 31, 2020. This decrease of $5.3 million was primarily due to the recognition of $6.1 million of the collaboration funding from the Relief Collaboration, as well as a decrease in contract research expenses, partially offset by increases in employee-related expenses, regulatory and filing fees, and expenses for consulting and professional services.
General and Administrative Expenses. General and administrative expenses were $3.1 million, net of collaboration funding of $1.6 million for the three months ended December 31, 2021, compared to $2.7 million for the three months ended December 31, 2020. General and administrative expenses were $10.7 million, net of collaboration funding of $3.2 million, for the year ended December 31, 2021, as compared to $11.0 million for the year ended December 31, 2020. This decrease of $0.3 million was primarily due to the recognition of $3.2 million of the collaboration funding from the Relief Collaboration as well as a decrease in legal and consulting expenses, partially offset by increases in employee-related expenses and in precommercial activities.
Net Loss. Net loss for the three months ended December 31, 2021 was $4.4 million, or $0.31 net loss per share (basic and diluted), compared to a net loss of $6.2 million, or $0.50 net loss per share (basic and diluted), for the three months ended December 31, 2020. Net loss for the year ended December 31, 2021 was $15.4 million, or $1.08 loss per share (basic and diluted), compared to a net loss of $22.9 million, or $2.06 loss per share (basic and diluted), for the year ended December 31, 2020.
For additional information, please see Acer’s Annual Report on Form 10-K filed today with the SEC.
About Acer Therapeutics Inc.
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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics Holding AG for development and commercialization of ACER-001. 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 regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. 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 our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. 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 Quarterly Reports on Form 10-Q and 10-Q/A, and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.
NEWTON, MA – Feb. 22, 2022 – 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 the appointment of Adrian Quartel, M.D., FFPM, as Chief Medical Officer. Dr. Quartel will oversee Acer’s clinical development, medical affairs, regulatory and other scientific and medical functions.
“We are pleased to welcome Dr. Quartel to our experienced leadership team,” said Chris Schelling, CEO and Founder of Acer. “Dr. Quartel’s proven track record of guiding the clinical development, approval and launch of six rare disease products or treatments in the U.S. and Europe, and his experience as a board-certified physician in pharmaceutical medicine, will be a welcome addition to our team and provide vital strategic oversight as our product pipeline continues to advance and grow.”
Adrian Quartel, M.D., FFPM, added, “The chance to join Acer at such a pivotal time in the company’s history is compelling. Acer’s innovative business model and its unwavering devotion to patients offers the opportunity to make an immediate and positive difference in people’s lives. I look forward to leading Acer’s medical efforts in support of its mission to bring much-needed treatments to patients.”
Dr. Quartel joins Acer Therapeutics from Adamas Pharmaceuticals, a company focused on drug discovery, development, and delivery of treatments for neurological diseases, where he served as Chief Medical Officer since 2020. In his role at Adamas, Dr. Quartel oversaw research and development, as well as medical affairs and regulatory functions. Prior to Adamas, he served as the Group Vice President, Global Medical Affairs, at BioMarin Pharmaceuticals Inc. where he led the launch of six treatments for rare disease or rare genetic disorders, including KUVAN®, VIMIZIM® and Brineura®. Prior to BioMarin, Dr. Quartel held senior medical leadership roles at Astellas, Chiltern, and ICON Clinical Research where he was responsible for clinical development, pharmacovigilance, and medical affairs. Earlier in his career, Dr. Quartel worked as a clinical research fellow at UCLA Cedar Sinai and as a resident in cardio-thoracic surgery at Erasmus University Medical Center. Dr. Quartel received an M.D. from Erasmus University Medical School, Rotterdam, and a post graduate specialization in pharmaceutical medicine from the Faculty of Pharmaceutical Medicine in London. He is board certified by the General Medical Council (GMC) in pharmaceutical medicine in the United Kingdom.
Inducement Grant Under Nasdaq Marketplace Rule 5635(c)(4)
In connection with his appointment as Acer’s Chief Medical Officer, Dr. Quartel was issued a grant of a non-qualified option to purchase 200,000 shares of Acer’s common stock. The award was approved by the independent members of Acer’s Board of Directors and granted under the inducement award provision of Acer’s 2018 Stock Incentive Plan, with a grant date of February 21, 2022, as an inducement material to Dr. Quartel in entering into employment with Acer, in accordance with Nasdaq Marketplace Rule 5635(c)(4).
The per share exercise price of the option is equal to the closing price of Acer’s common stock on the trading day before the grant date. The option vests over a four-year period commencing on the date of grant, with 25% vesting on the one-year anniversary of the grant date and the remaining 75% vesting quarterly over the remaining three years (assuming continued service in all instances), with a standard post-service exercise period of 90 days. In addition, the option will accelerate and become fully vested immediately prior to a change in control but only to the extent that the optionee remains in the service of the Company immediately prior to such change in control. The option has a ten-year term.
Acer is providing this information in compliance with Nasdaq Marketplace Rule 5635(c)(4).
About Acer Therapeutics Inc.
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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of ACER-001. 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 regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. 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 our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. 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 Quarterly Reports on Form 10-Q and 10-Q/A, and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.
Investor Contact: Hans Vitzthum LifeSci Advisors Ph: 617-430-7578 [email protected]
Jim DeNike Acer Therapeutics Inc. Ph: 844-902-6100 [email protected]
NEWTON, MA –Feb. 1, 2022 – 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 Acer’s management team will virtually participate in the BIO CEO & Investor Conference on February 14-17, 2022.
Format: On-demand recorded corporate presentation and one-on-one virtual meetings Dates: February 14-17, 2022 Presentation: https://www.acertx.com/investor-relations/events-presentations/
About Acer Therapeutics Inc.
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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.
NEWTON, MA and GENEVA, SWITZERLAND – Jan. 27, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (“Acer”) and its collaboration partner, Relief Therapeutics Holding SA (SIX: RLF, OTCQB: RLFTF) (“Relief”), today announced the acceptance of four ACER-001 abstracts for poster presentations at the upcoming Society for Inherited Metabolic Disorders (SIMD) Annual Meeting on April 10-13, 2022 in Orlando, Florida, and the Genetic Metabolic Dieticians International (GMDI) Conference on May 4-7, 2022, in Las Vegas, Nevada. The ACER-001 New Drug Application (NDA) for urea cycle disorders (UCDs) is currently under U.S. Food and Drug Administration (FDA) review with a Prescription Drug User Fee Act (PDUFA) target action date of June 5, 2022.
“Many UCDs patients currently taking nitrogen scavengers may struggle with elevated ammonia levels potentially associated with consequences of treatment non-compliance,” said Chris Schelling, Chief Executive Officer and Founder of Acer. “The data from our bioequivalence and taste assessment studies accepted for presentations at the SIMD and GMDI conferences further support our belief that, if approved, ACER-001 could offer an alternative to current therapies that may lead to meaningful clinical outcomes in UCDs patients.”
Raghuram (Ram) Selvaraju, Chairman of Relief, added, “The acceptance of ACER-001 data at these prestigious industry conferences is another important milestone for this potential treatment for patients with UCDs. We are highly encouraged by the continued progress of the ACER-001 program and look forward to the FDA’s decision on the PDUFA target action date in June.”
Details of the presentations are as follows:
2022 SIMD 43rd Annual Meeting
Title: The Pharmacokinetics of Taste-Masked Sodium Phenylbutyrate (Acer-001) for the Treatment of Urea Cycle Disorders Under Fasting and Fed Conditions in Healthy Volunteers Presenter: Dr. Robert Steiner Poster #: 84 Date/Time: April 11, 2022; 7:00-8:00 pm ET
Title: Taste-Masked Coating of Sodium Phenylbutyrate (Acer-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders Presenter: Dr. Stephen Cederbaum Poster #: 18 Date: April 11, 2022; 7:00-8:00 pm ET
Additionally, Acer is sponsoring a lunchtime symposium at SIMD on April 12th at 12:45 pm ET. The symposium, entitled: “Addressing Unmet Needs in Screening, Diagnosis, Therapeutics and Advocacy for Urea Cycle Disorders (UCDs),” will be hosted by Robert D Steiner, MD, FAAP, FACMG; Professor (Clinical) University of Wisconsin; Editor in Chief of Genetics in Medicine; Wisconsin Newborn Screening Program Medical Consultant; and Chief Medical Officer of PreventionGenetics.
2022 GMDI Conference
Title: ACER-001: a Potential Alternative to Sodium and Glycerol Phenylbutyrate for Treatment of Urea Cycle Disorders Presenter: Dr. Robert Steiner Poster #: 26 Date: May 5, 2022; 5:30-7:00 pm PT
Title: Taste-masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders Presenter: Dr. Stephen Cederbaum Poster #: 10 Date/Time: May 5, 2022; 5:30-7:00 pm PT
Additionally, Acer is sponsoring a breakfast symposium at GMDI on May 5th at 7:00 am PT. The symposium, entitled: “Energy and Protein Needs in UCD Patients: What We Know and What We Need to Know to Move Towards Personalized Care,” will be hosted by Deborah Geary Hook, MPH, RDN, FAND, PhDc. PhD Candidate, University of California, Davis.
Parties interested in the ACER-001 program for UCDs may sign up for updates at:
UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.1,2 The current treatment of patients with UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.
Current medical treatments for patients with UCDs include nitrogen scavengers, RAVICTI® and BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate and sodium phenylbutyrate, respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports3, while nitrogen scavenging medications have been shown to be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include aversive taste and odor4, frequency with which medication must be taken, required number of pills, and the high cost of the medication.
About ACER-001
ACER-001 (sodium phenylbutyrate) is being developed for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 is a nitrogen-binding agent in development for use as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). ACER-001’s multi-particulate dosage formulation for oral administration is designed to minimize the aversive taste and odor4 of sodium phenylbutyrate while quickly dissolving in the stomach. The ACER-001 NDA for UCDs is currently under FDA review with a PDUFA target action date of June 5, 2022. ACER-001 is also being developed for MSUD and has been granted orphan drug designation by the FDA for this indication. ACER-001 is an investigational product candidate which has not been approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority.
About Acer Therapeutics Inc.
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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.
About RELIEF THERAPEUTICS Holding SA
Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. Relief’s lead drug candidate, RLF-100™ (aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the U.S. for the treatment of respiratory deficiency due to COVID-19 through Relief’s collaboration partner in the U.S., NeuroRx, Inc. As part of its pipeline diversification strategy, in March 2021, Relief entered into a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001. ACER-001 is a taste-masked and immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment of Urea Cycle Disorders and Maple Syrup Urine Disease. Finally, Relief’s recently completed acquisitions of APR Applied Pharma Research SA and AdVita Lifescience GmbH, bring to Relief a diverse pipeline of marketed and development-stage programs.
RELIEF THERAPEUTICS Holding SA is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbols RLFTF and RLFTY. For more information, visit www.relieftherapeutics.com.
References
Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.
Peña-Quintana L, et al. Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Prefer Adherence. 2017 Sep 6;11:1489-1496.
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 regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. 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 our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. 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 Quarterly Reports on Form 10-Q and 10-Q/A, and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.
Relief Forward-Looking Statements
This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding SA and its businesses. Such statements involve certain known and unknown risks, uncertainties and other factors, including (i) whether the FDA will approve Acer’s NDA for ACER-001, (ii) whether RELIEF THERAPEUTICS Holding SA will be able to submit an application for approval of ACER-001 in Europe in Q2/Q3 2022 (or at all), (iii) whether any such application submitted to European authorities seeking marketing authorization for ACER-001 for the treatment of patient in Europe with UCDs will be approved, (iv) whether RLF-100 (aviptadil) will receive emergency use authorization in the United States, (v) whether RLF-100 (aviptadil) will ever be submitted for authorization in Europe, (vi) whether RELIEF THERAPEUTICS Holding SA’s ongoing disputes with its U.S. collaboration partner for RLF-100 (aviptadil) can be resolved amicably, and (vii) those other risks, uncertainties and factors described in RELIEF THERAPEUTICS Holding SA’s press releases, periodic filings with the SIX Stock Exchange, all of which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding SA to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding SA is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise.
CORPORATE CONTACTS
Acer Therapeutics: Jim DeNike Acer Therapeutics Inc. [email protected] +1 844-902-6100
RELIEF Therapeutics Holding SA: Jack Weinstein Chief Financial Officer and Treasurer [email protected]
INVESTOR RELATIONS CONTACTS
Acer Therapeutics: Hans Vitzthum LifeSci Advisors [email protected] +1 617-430-7578
Relief Therapeutics Holding SA:
Michael Miller Rx Communications Group [email protected] +1-917-633-6086
NEWTON, MA –Dec. 22, 2021 – 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 Acer’s management team will participate in the upcoming 11th Annual LifeSci Partners Corporate Access virtual event and the H.C. Wainwright BioConnect Virtual Conference.
About Acer Therapeutics Inc. 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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.
Investor Contact: Hans Vitzthum LifeSci Advisors Ph: 617-430-7578 [email protected]
Jim DeNike Acer Therapeutics Inc. Ph: 844-902-6100 [email protected]
NEWTON, MA – Dec. 9, 2021 – 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 U.S. Food and Drug Administration (FDA) clearance of its Investigational New Drug (IND) application for its selective non-peptide neurokinin 3 receptor (NK3R) antagonist, ACER-801 (osanetant), for the potential treatment of induced Vasomotor Symptoms (iVMS).
The initial randomized, double-blind, placebo-controlled, dose-ranging Phase 2a trial is expected to begin in Q1 2022, subject to additional capital required for ACER-801 clinical development. The trial is designed to evaluate the effect of ACER-801 at different doses, compared to placebo, on the frequency and severity of vasomotor symptoms associated with menopause. The trial will also evaluate the safety and pharmacokinetics profile of ACER-801 at different doses in postmenopausal women.
“Submission and clearance of our ACER-801 IND application is an important step toward the clinical development of our novel NK3R antagonist for treatment of iVMS,” said Chris Schelling, Chief Executive Officer and Founder of Acer. “This drug has been extensively studied, with over 1,200 individuals having received it in clinical trials for other non-iVMS indications. We’re excited to start this Phase 2a trial which, if positive, will provide proof of concept data and help define ACER-801’s optimal dose and development path forward in patients with iVMS.”
ACER-801 is an investigational product candidate which has not been approved by FDA or any other regulatory authority. There is no guarantee that this product candidate will receive regulatory authority approval in any territory or become commercially available for any indications.
About iVMS and ACER-801
VMS are generally defined as hot flashes, flushing and night sweats that most often occur in women entering or in menopause. VMS can also be induced (iVMS) by anti-androgen and anti-estrogen cancer therapies and surgical procedures that can lead to treatment non-compliance. VMS are caused by low estrogen levels leading to increased stimulatory signaling of neurokinin B (NKB) on the KNDy neuron in the hypothalamus. A non-hormonal treatment to manage iVMS is needed as estrogen is contraindicated for the management of VMS in patients with hormone-positive tumors, including breast and prostate tumors.
ACER-801 (osanetant) is a novel, non-hormonal, NK3R antagonist that could offer a potential treatment option with meaningful improvement of VMS for patients with iVMS by blocking the stimulatory signaling of NKB on the KNDy neuron. Direct human safety evidence is available from 23 completed Phase 1 and 2 studies of whom approximately 400 healthy subjects and 820 patients were treated with osanetant for schizophrenia, depression, and other indications. Data from these studies indicated no major safety concerns after single-dose and repeat-dose administration.
About Acer Therapeutics Inc.
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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of ACER-001. 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 regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. 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 our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. 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 Quarterly Reports on Form 10-Q Q and 10-Q/A, and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.
NEWTON, MA –Nov. 19, 2021 – 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 third quarter ended September 30, 2021 and provided an update on Acer’s recent corporate developments.
“This quarter was all about preparing regulatory filings to advance our pipeline products,” said Chris Schelling, CEO and Founder of Acer. “For ACER-001, we successfully submitted a 505(b)(2) NDA in August and received FDA’s acceptance for filing and substantive review of our NDA in October, setting up a PDUFA target action date in June 2022. For ACER-801 and EDSIVO™, our team has been working diligently to prepare INDs, with the goal to submit both to the FDA in Q4. Additionally, we further enhanced the value in the company through the issuance of a key formulation patent by the USPTO related to ACER-001 with an expiration date of 2036, and expansion of our leadership team with the recent addition of Alka Chawla, M.D., Vice President, Medical Director.”
Q3 and Recent Highlights
ACER-001 (sodium phenylbutyrate)
Announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for ACER-001 (sodium phenylbutyrate) for the treatment of patients with Urea Cycle Disorders (UCDs), and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 5, 2022
Announced that the U.S. Patent and Trademark Office (USPTO) has issued a key new ACER-001 (sodium phenylbutyrate) formulation composition of matter U.S. patent (No. 11,154,521), strengthening Acer’s proprietary position in the U.S. until 2036
ACER-801 (osanetant)
Submitted an Investigational New Drug (IND) application to FDA for ACER-801, a selective non-peptide neurokinin 3 receptor (NK3R) antagonist for the treatment of induced Vasomotor Symptoms (iVMS)
EDSIVO™ (celiprolol)
Continued preparation for an IND submission and Special Protocol Assessment (SPA) request for a Phase 3, randomized, double-blind, placebo-controlled, decentralized clinical trial (DiSCOVER) for EDSIVO™ for patients with COL3A1+ vascular Ehlers-Danlos Syndrome (vEDS)
Hired Alka Chawla, M.D., Vice President, Medical Director, in September 2021. Dr. Chawla brings to Acer over 20 years of biopharmaceutical industry experience, including work in the development and optimization of drug-binding and cell-based assays and the purification of proteins, as well as expertise in the therapeutic areas of hematology, metabolic disorders, and oncology. Dr. Chawla has served as a key team member for an FDA Advisory Committee Meeting, led clinical development programs, and has been responsible for monitoring and evaluating the clinical safety of numerous product candidates
Ended Q3 2021 with $14.2 million in cash and cash equivalents. Acer believes its cash and cash equivalents available as of September 30, 2021, plus the $10.0 million Second Development Payment in two tranches following FDA acceptance for filing and review of an NDA for ACER-001 in a UCD per the Collaboration Agreement with Relief Therapeutics Holding AG (Relief), will be sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for the planned ACER-801 and EDSIVO™ clinical trials
Anticipated Milestones
ACER-001 (sodium phenylbutyrate)
June 5, 2022: FDA has assigned a PDUFA target action date of June 5, 2022, following its acceptance for filing of the NDA for ACER-001 (sodium phenylbutyrate) for the treatment of patients with UCDs
ACER-801 (osanetant)
Q1 2022: Phase 2a clinical trial initiation is expected in Q1 2022 designed to evaluate ACER-801’s pharmacokinetics, efficacy and safety in postmenopausal women and to identify the optimal dosing strategy for subsequent efficacy trials in certain cancer patients with iVMS, dependent upon IND clearance and subject to additional capital
Q4 2021: Submission of the proposed EDSIVO™ pivotal trial in an IND and SPA request, and request for breakthrough therapy designation are anticipated in Q4 2021
Q1 2022: Initiation of the EDSIVO™ DiSCOVER pivotal clinical trial is expected in Q1 2022 subject to successful IND submission and clearance and SPA agreement, and obtaining additional capital required to conduct and complete the trial
ACER-2820 (emetine)
Ongoing: Further advancement of the emetine program for treatment of certain infectious diseases, including COVID-19, is dependent on Acer’s ability to raise non-dilutive capital
ACER-001, ACER-801 and EDSIVO™ (celiprolol) are investigational product candidates that have not been approved by FDA. There is no guarantee that these product candidates will receive regulatory authority approval in any territory or become commercially available for the indications under investigation.
Q3 2021 Financial Results
Cash position. Cash and cash equivalents were $14.2 million as of September 30, 2021, compared to $5.8 million as of December 31, 2020. Acer believes its cash and cash equivalents available as of September 30, 2021, plus the $10.0 million Second Development Payment in two tranches following FDA acceptance for filing and review of an NDA for ACER-001 in a UCD per the Collaboration Agreement with Relief, will be sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for planned ACER-801 and EDSIVO™ clinical trials.
Research and Development Expenses. Research and development expenses were $1.4 million, net of collaboration funding of $3.5 million for the three months ended September 30, 2021, as compared to $3.2 million for the three months ended September 30, 2020. This decrease of $1.8 million was primarily due to the recognition of the $3.5 million of collaboration funding from the Collaboration Agreement with Relief, partially offset by increases in contract manufacturing expenses and employee-related expenses. Research and development expenses for the three months ended September 30, 2021 were comprised of $3.6 million related to ACER-001, offset by $3.5 million of collaboration funding; $0.9 million related to ACER-801; $0.3 million related to EDSIVOTM; and $0.1 million related to other development activities.
General and Administrative Expenses. General and administrative expenses were $1.8 million, net of collaboration funding of $1.0 million for the three months ended September 30, 2021, as compared to $2.7 million for the three months ended September 30, 2020. This decrease of $0.9 million was primarily due to the recognition of the $1.0 million of collaboration funding from the Collaboration Agreement with Relief, as well as a decrease in legal expenses, partially offset by increases in precommercial expenses and employee-related expenses.
Net Loss. Net loss for the three months ended September 30, 2021 was $3.3 million, or $0.23 net loss per share (basic and diluted), compared to a net loss of $5.9 million, or $0.51 net loss per share (basic and diluted), for the three months ended September 30, 2020.
For additional information, please see Acer’s Quarterly Report on Form 10-Q filed today with the SEC.
Completion of Financial Restatement
Acer has completed the restatement of its previously issued financial statements for the fiscal quarters ended March 31, 2021 and June 30, 2021 (Restated Periods), and has filed amended Quarterly Reports on Form 10-Q/A for the Restated Periods with the SEC. As previously disclosed in its Current Report on Form 8-K filed November 16, 2021, a non-cash error was made in the application of certain complex accounting guidance for collaboration agreements, and the revenue recognition associated with the previously disclosed collaboration and license agreement between Acer and Relief, which was entered into on March 19, 2021. The correction of this non-cash error resulted in a restatement of Acer’s unaudited condensed interim financial statements and financial data for the Restated Periods.The restatement had no impact on Acer’s cash position or operating expenses or its ongoing operations or future plans.
About Acer Therapeutics Inc.
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. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of ACER-001. 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 regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. 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 our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. 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 Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.
