Author: AcerUser

NEWTON, MA – June 27, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER), a clinical stage 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 initiation of patient screening in its Phase 3 DiSCOVER (Decentralized Study of Celiprolol on vEDS-related Event Reduction) clinical trial of EDSIVO™ (celiprolol) for the treatment of patients with COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS).

“With no approved treatments available for vEDS, ongoing clinical evaluation of EDSIVO™ (celiprolol) is an important step that we hope could lead to the first available treatment option for this fatal disorder,” said Adrian Quartel, MD, FFPM, Chief Medical Officer of Acer. “The innovative, decentralized study design will allow for greater access and ease of administration for vEDS patients, mobile nurses, remote clinical research coordinators and investigators. We’d like to thank all the patients, their family members, the advocacy groups and clinicians who have expressed interest in learning more about participating in the study, and look forward to rapidly enrolling the trial.”

The DiSCOVER trial is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy trial designed to evaluate EDSIVO™ (celiprolol) in patients with genetically confirmed COL3A1-positive vEDS using a decentralized clinical trial design and an independent adjudication committee. The primary objective of the trial is to determine whether EDSIVO™ (celiprolol) reduces the occurrence of vEDS-related clinical events requiring medical attention, including fatal and non-fatal cardiac or arterial events, uterine rupture, intestinal rupture, and/or unexplained sudden death, relative to placebo as measured by time to event. Acer plans to enroll approximately 150 COL3A1-positive vEDS patients, all in the U.S., randomized 2:1 to receive either EDSIVO™ (celiprolol) or placebo, respectively. Individuals seeking more information on the EDSIVO™ (celiprolol) pivotal clinical trial are invited to visit www.discoverceliprolol.com.

Once the trial is fully enrolled, the duration of the DiSCOVER trial is currently estimated to be up to approximately 3.5 years to completion (based on statistical power calculations and number of primary events), which will require additional capital beyond Q3 2022. One interim analysis (based on number of primary events) is also planned at approximately 24 months after full enrollment.

EDSIVO™ (celiprolol) is an investigational product candidate which has not been approved by the U.S. Food and Drug Administration (FDA). There is no guarantee that this product candidate will receive regulatory authority approval or become commercially available for any indications in the U.S.

About vEDS

Ehlers-Danlos syndrome (EDS) is an autosomal inherited disorder caused by mutations in the genes responsible for the structure, production, or processing of collagen, an important component of the connective tissues in the human body, or proteins that interact with collagen. EDS is a spectrum disorder where patients present with various forms, the most serious of which is vascular Ehlers-Danlos syndrome (vEDS), also known as vEDS type IV, which is generally caused by a mutation in the COL3A1 gene resulting in reduced collagen levels. vEDS causes abnormal fragility in blood vessels, which can give rise to aneurysms, abnormal connections between blood vessels known as arteriovenous fistulas, arterial dissections, and spontaneous vascular ruptures, all of which can be potentially life-threatening. Gastrointestinal and uterine fragility or rupture also commonly occur in vEDS patients. Spontaneous arterial rupture has a peak incidence in the third or fourth decade of life in vEDS patients but may occur earlier and is the most common cause of sudden death in vEDS patients. Arterial rupture or dissection events occur in about 25% of patients before the age of 20 but increase to roughly 90% of patients by age 40. The median survival age of vEDS patients in the U.S. is 51 years, with arterial rupture being the most common cause of sudden death.¹ Based on an analysis of diagnosed vEDS patients from the Truven MarketScan® database and U.S. population data, Acer projects the total COL3A1-positive vEDS patient prevalence in the U.S. could be as high as 7,000 patients. Currently, there are no approved pharmacologic therapies anywhere in the world for vEDS.

About EDSIVO™ (celiprolol)

EDSIVO™ (celiprolol) is a new chemical entity (NCE) currently in Phase 3 development for the treatment of COL3A1-positive vEDS patients to potentially reduce the risk of arterial and other hollow organ clinical events. In October 2010, data was published in the Lancet from the BBEST trial designed to assess the preventative effect of celiprolol for major cardiovascular events in patients with vEDS via a multicenter, prospective, randomized, open trial with blinded evaluation of clinical events.² In addition, data from long-term observational studies of patients treated with celiprolol in France and Sweden were published in the Journal of the American College of Cardiology (JACC) in April 2019³ and in the European Journal of Vascular and Endovascular Surgery (EJVES) in November 2020⁴, respectively. Data from these and other publications can be found at www.acertx.com. Acer’s original NDA was submitted based on data obtained from the BBEST trial and accepted for filing in October 2018 with priority review. Following FDA review, Acer received a Complete Response Letter (CRL) in June 2019 stating that it will be necessary to conduct an adequate and well-controlled trial to determine whether EDSIVO™ reduces the risk of clinical events in patients with vEDS. In April 2022, FDA granted celiprolol Breakthrough Therapy designation in the U.S. for the treatment of patients with COL3A1-positive vEDS. In May 2022, Acer reached agreement with FDA under an SPA for its Pivotal Phase 3 clinical trial of EDSIVO™ (celiprolol) for the treatment of patients with COL3A1-positive vEDS, and patient screening was initiated in June 2022. Celiprolol received FDA Orphan Drug Designation for the treatment of vEDS in 2015.

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 investigational 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 viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more information, visit www.acertx.com.

References

1. Pepin, et al. Survival is affected by mutation type and molecular mechanism in vascular Ehlers–Danlos syndrome (EDS type IV). Genet Med. 2014 Dec;16(12):881-8.
2. Ong KT, et al. Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. Lancet. 2010;376(9751):1476-1484
3. Frank M, et al. Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study. J Am Coll Cardiol. 2019 Apr, 73 (15) 1948–1957
4. Björck M, et al. Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome. European Journal of Vascular and Endovascular Surgery. November 20, 2020.

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, including statements regarding the objectives, potential results, and duration of the DiSCOVER trial, including with respect to the Company’s plans for patient enrollment, the interim analysis and timing thereof, and the need for additional capital beyond Q3 2022, as well as statements regarding any future regulatory approval or commercial availability of our product candidate EDSIVO™ (celiprolol), are forward-looking statements. Our pipeline products 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 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 and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

CORPORATE CONTACT

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

INVESTOR RELATIONS CONTACT

Acer Therapeutics:
Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-339-225-1047

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NEWTON, MA – June 21, 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 promotion of Tanya Hayden to Chief Operating Officer (COO). Harry S. Palmin, who had been serving as the Company’s Chief Operating Officer as well as its Chief Financial Officer, will continue to serve as the Company’s Chief Financial Officer.

In her new role as COO, Ms. Hayden will have primary responsibility for supporting operational and commercial growth and effectiveness, as well as establishing, improving, and scaling the company’s executional operations. Ms. Hayden joined Acer in June 2021 as Vice President of Program and Strategic Alliance Management and has played a critical role in the advancement of Acer’s investigational programs, including the Company’s ongoing preparations for the potential commercial launch of ACER-001 (sodium phenylbutyrate) for treatment of urea cycle disorders (UCDs), and management of its strategic collaborations. Ms. Hayden will continue to oversee program and strategic alliance management responsibilities in her new role as COO.

“Tanya’s leadership and strategic oversight of Acer’s commercial and operational readiness in preparation for our potential commercial approval and launch of ACER-001 in UCDs, along with her role in the clinical advancement of our product candidates, has been instrumental in shaping our growth in the last year,” stated Chris Schelling, CEO and Founder of Acer. “Tanya’s deep expertise in, and responsibility for, drug development and delivery, clinical and commercial contract manufacturing, and alliance management, will be instrumental in our future growth as our ACER-001, ACER-801 and EDSIVO™ (celiprolol) programs continue to advance toward key near-term milestones throughout the rest of 2022.”

Prior to joining Acer, Ms. Hayden spent 20 years at Lonza (formerly Bend Research/Capsugel) and was responsible for business unit planning, operational excellence, clinical and commercial contract manufacturing, and program management. Throughout her career, Ms. Hayden has had the opportunity to lead internal cross-functional teams to introduce new capabilities and improve performance, as well as collaborate with external partners to advance new products to the market. Ms. Hayden received a B.S. degree in Chemistry from Gonzaga University.

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 investigational 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 viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more information, visit 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 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 investigational 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 financing arrangements as well as to satisfy the ongoing conditions and requirements for maintaining the financing facilities and avoiding default or an accelerated payment requirement; 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 product candidates; 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. Our pipeline products 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 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 and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

CORPORATE CONTACTS

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

INVESTOR RELATIONS CONTACTS

Acer Therapeutics:
Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-339-225-1047

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NEWTON, MA and GENEVA, SWITZERLAND – June 21, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (Acer) and its collaboration partner, RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief), announced today that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for ACER-001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with urea cycle disorders (UCDs).

The CRL indicates that the FDA cannot approve the NDA in its current form. The CRL states: “[The FDA’s] field investigator could not complete inspection of [Acer’s third-party contract packaging manufacturer], because the facility was not ready for inspection. Satisfactory inspection is required before [the NDA] may be approved. Please notify us in writing when this facility is ready for inspection.”

The FDA did not cite any other approvability issues in the CRL pertaining to the NDA, nor request any additional clinical or pharmacokinetic studies be conducted prior to FDA approval. The FDA did provide one comment in the CRL (identified as “not an approvability issue”) requesting additional existing nonclinical information to be provided in the resubmission of the NDA.

Acer is actively collaborating with its third-party contract packaging manufacturer and cooperating with the FDA to address the FDA’s comments as soon as reasonably possible and currently intends to resubmit the updated NDA for ACER-001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with UCDs in early-to-mid Q3 2022.

“While the outcome of the NDA review was not what we had hoped for, multiple rounds of labeling negotiations have already been conducted to date and we believe the recommendations raised by FDA can be appropriately addressed. We should be able to resubmit the NDA relatively quickly,” said Chris Schelling, CEO and Founder of Acer. “We remain committed to bringing a new treatment option to patients in the U.S. with UCDs.”

About ACER-001

ACER-001 (sodium phenylbutyrate) is being developed for the treatment of various inborn errors of metabolism, including 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 is a polymer coated formulation that, when taken within 5 minutes, helps prevent the coating from dissolving. ACER-001 has been granted orphan drug designation by the FDA for MSUD. ACER-001 is an investigational product candidate which has not been approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority. There can be no assurance that Acer will be able to meet its intended resubmission timeline for the NDA, that FDA inspection of the third-party contract packaging manufacturer facility will be satisfactory, that such inspection is the only impediment to FDA approval of a resubmitted NDA, that a resubmitted NDA will otherwise be approved by the FDA, or that ACER-001 will be approved for any indication.

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 investigational 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 viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more information, visit 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 has a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including UCDs and Maple Syrup Urine Disease (MSUD). Relief also continues to study aviptadil for several possible lung related conditions. Finally, Relief’s 2021 acquisitions of APR Applied Pharma Research SA and AdVita Lifescience GmbH brought 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 Follow Relief on LinkedIn.

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 investigational product candidates to safely and effectively treat diseases and to be approved for marketing; our ability to submit regulatory filings (including for ACER-001 for oral suspension for the treatment of patients with UCDs) within intended timeframes, to address satisfactorily the requirements for regulatory approval (including through FDA inspection of our third-party contract packaging manufacturer for ACER-001 for oral suspension for the treatment of patients with UCDs), or to otherwise address satisfactorily the requirements for and to obtain regulatory approval (including approval by the FDA of a resubmitted NDA for ACER-001 for oral suspension for the treatment of patients with UCDs); our ability to close upon and obtain the proceeds of any financing arrangements as well as to satisfy the ongoing conditions and requirements for maintaining the financing facilities and avoiding default or an accelerated payment requirement; 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 product candidates; 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. Our pipeline products 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 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 and Quarterly Reports on Form 10-Q. 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 submit an application for approval of ACER-001 in Europe and the timing of filing such application, (iii) whether any such application submitted to European authorities seeking marketing authorization for ACER-001 for the treatment of patients in Europe with UCDs will be approved, and (iv) those other risks, uncertainties and factors described in RELIEF THERAPEUTICS Holding SA’s press releases and filings with the SIX Swiss Exchange and the U.S. Securities and Exchange Commission, 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.
jdenike@acertx.com
+1-844-902-6100

RELIEF Therapeutics Holding SA:
Jack Weinstein
Chief Financial Officer and Treasurer
contact@relieftherapeutics.com

INVESTOR RELATIONS CONTACTS

Acer Therapeutics:
Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-339-225-1047

Relief Therapeutics Holding SA:
Michael Miller
Rx Communications Group
mmiller@rxir.com
+1-917-633-6086

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NEWTON, MA – June 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 not yet received a decision from the U.S. Food and Drug Administration (FDA) on its 505(b)(2) New Drug Application (NDA) for ACER-001 (sodium phenylbutyrate) for the treatment of urea cycle disorders (UCDs). The NDA for ACER-001 for UCDs was accepted for review by FDA on October 5, 2021 at which time FDA assigned a PDUFA target action date of Sunday, June 5, 2022. FDA has informed Acer that review for ACER-001 is ongoing and the agency currently does not have a set target date. Under the Prescription Drug User Fee Act (PDUFA), FDA’s review performance goal is to review and act on 90 percent of NDA submissions by the target action date.

Acer will issue a press release once FDA has provided its written decision to the Company.

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 investigational 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 viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more information, visit 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 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 investigational 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 identified financing arrangements as well as to satisfy the ongoing conditions and requirements for maintaining the financing facilities and avoiding default or an accelerated payment requirement; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability, in addition to the currently identified 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 for ACER-001, ACER-801, EDSIVO™ or our other product candidates; 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. Our pipeline products 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 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 and Quarterly Report on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

CORPORATE CONTACTS

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

INVESTOR RELATIONS CONTACTS

Acer Therapeutics:
Nick Colangelo
Gilmartin Group
nick@gilmartinIR.com
+1-339-225-1047

#  #  #

NEWTON, MA –May 16, 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 first quarter ended March 31, 2022 and provided an update on the Company’s recent corporate developments.

“Progress in Q1 2022 was marked by a transformative debt financing in March, with continued advancement toward potential commercial launch of ACER-001 for UCDs and further development of our other pipeline programs,” said Chris Schelling, CEO and Founder of Acer. “Our advances to date have positioned Acer for potential achievement of a number of important planned milestones through the rest of 2022, including an FDA decision on our ACER-001 New Drug Application in June, the planned initiation of our EDSIVO™ pivotal Phase 3 trial in vascular Ehlers-Danlos Syndrome under our SPA agreement with FDA by end of Q2 2022, and ACER-801 Phase 2a trial results in Vasomotor Symptoms in H2 2022.”

Q1 2022 and Recent Highlights

• ACER-001 (sodium phenylbutyrate)
  • Announced in April 2022 the presentation of data evaluating the bioavailability, bioequivalence and taste attributes of ACER-001 (sodium phenylbutyrate) compared to sodium phenylbutyrate (BUPHENYL®) powder during poster sessions at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting
  • Announced in May 2022 the presentation of data evaluating the bioavailability, bioequivalence and taste attributes of ACER-001 (sodium phenylbutyrate) compared to sodium phenylbutyrate (BUPHENYL®) powder during poster sessions at the Genetic Metabolic Dieticians International (GMDI) Conference. Publications from SIMD and GMDI are available at: https://www.acertx.com/publications-and-presentations/
  • Launched SeeUCDifferently, a national U.S. disease awareness campaign intended to provide education and information about urea cycle disorders (UCDs), including www.SeeUCDifferently.com, a new online resource that provides general UCDs diagnosis and disease education information
• ACER-801 (osanetant)
  • Announced in March 2022 enrollment of the first patient in a Phase 2a (NCT05325775) randomized, double-blind, placebo-controlled, dose-ranging trial evaluating the pharmacokinetics (PK), safety, and efficacy of ACER-801 at different doses, compared to placebo, for the treatment of moderate to severe Vasomotor Symptoms (VMS) in post-menopausal women. 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)
• EDSIVO™ (celiprolol)
  • In January 2022, the U.S. Food and Drug Administration (FDA) cleared the celiprolol IND for the treatment of patients with COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS)
  • In April 2022, announced FDA granted celiprolol Breakthrough Therapy designation (BTD) in the U.S. for the treatment of patients with COL3A1-positive vEDS
  • In May 2022, announced agreement with FDA under a Special Protocol Assessment (SPA) for pivotal Phase 3 clinical trial of celiprolol for the treatment of patients with COL3A1-positive vEDS. More information on the DiSCOVER (Decentralized Study of Celiprolol on vEDS-related Event Reduction) trial is available at https://discoverceliprolol.com/
• Corporate
  • In February 2022, announced 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 Pharmaceutical Inc., Astellas, Chiltern, and ICON Clinical Research

• In March 2022, announced 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, subject to certain conditions.  Proceeds from these financings will be used to advance pipeline and for general corporate purposes. Further information with respect to the debt financing agreements with Marathon and SWK is contained in a Current Report on Form 8-K filed with the Securities and Exchange Commission on March 7, 2022
• Ended Q1 2022 with $20.8 million in cash and cash equivalents, which Acer believes will be sufficient to fund its currently anticipated operating and capital requirements into Q3 2022

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 for the treatment of patients with UCDs
  • End of Q2 2022:  Acer plans to submit an Investigational New Drug (IND) application by the end of Q2 2022 for a clinical study evaluating ACER-001 in Maple Syrup Urine Disease (MSUD)
• ACER-801 (osanetant)
  • H2 2022: Results from the ongoing ACER-801 Phase 2a clinical trial in women with moderate to severe VMS are anticipated in H2 2022
• EDSIVO™ (celiprolol)
  • End of Q2 2022: Acer intends to initiate by end of Q2 2022 the pivotal Phase 3, randomized, double-blind, placebo-controlled, decentralized clinical trial for celiprolol for patients with COL3A1-positive vEDS under its SPA agreement with FDA. The duration of the DiSCOVER clinical trial is currently estimated to be approximately 3.5 years to completion, once fully enrolled, and will require additional capital beyond Q3 2022

Q1 2022 Financial Results

Cash position. Cash and cash equivalents were $20.8 million as of March 31, 2022, compared to $12.7 million as of December 31, 2021. Acer believes its cash and cash equivalents available as of March 31, 2022 will be sufficient to fund its currently anticipated operating and capital requirements into Q3 2022.

Research and Development Expenses. Research and development expenses were $3.2 million, net of collaboration funding of $3.0 million, for the three months ended March 31, 2022, compared to $2.0 million, net of collaboration funding of $0.3 million, for the three months ended March 31, 2021. This increase of $1.2 million was primarily due to increases in contract manufacturing expenses, employee-related expenses including a one-time bonus accrual, and clinical and medical affairs expenses, partially offset by the recognition of $3.0 million of the collaboration funding from the Collaboration Agreement with Relief. Research and development expenses for the three months ended March 31, 2022 were comprised of $3.1 million related to ACER-001, offset by $3.0 million of collaboration funding; $1.3 million related to ACER-801; $1.2 million related to EDSIVO™; $0.5 million related to other development activities.

General and Administrative Expenses. General and administrative expenses were $3.9 million, net of collaboration funding of $2.4 million for the three months ended March 31, 2022, compared to $3.5 million for the three months ended March 31, 2021. This increase of $0.4 million was primarily due to increases in precommercial expenses, audit and consulting fees, and employee-related expenses including a one-time bonus accrual, partially offset by the recognition of $2.4 million of the collaboration funding from the Collaboration Agreement with Relief.

Net Loss. Net loss for the three months ended March 31, 2022 was $9.2 million, or $0.64 net loss per share (basic and diluted), compared to a net loss of $4.6 million, or $0.33 net loss per share (basic and diluted), for the three months ended March 31, 2021.

For additional information, please see Acer’s Quarterly Report on Form 10-Q 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 investigational 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 viruses, including cytomegalovirus, zika, dengue, ebola and COVID-19. For more information, visit 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 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 investigational 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 identified financing arrangements as well as to satisfy the ongoing conditions and requirements for maintaining the financing facilities and avoiding default or an accelerated payment requirement; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability, in addition to the currently identified 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 for ACER-001, ACER-801, EDSIVO™ or our other product candidates; 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. Our pipeline products 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 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 and Quarterly Report on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

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

Investor Relations Contact:
Nick Colangelo
Gilmartin Group
+1-339-225-1047
nick@gilmartinIR.com

NEWTON, MA – May 09, 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 it has reached agreement with the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for its pivotal Phase 3 DiSCOVER (Decentralized Study of Celiprolol on vEDS-related Event Reduction) clinical trial of EDSIVO™ (celiprolol) for the treatment of patients with COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS). In April 2022, FDA granted celiprolol Breakthrough Therapy designation in the U.S.

The SPA agreement is a process through which sponsors may seek agreement with FDA on the design and size of certain clinical trials, clinical studies, or animal studies. Agreement on Acer’s Phase 3 trial design indicates concurrence by FDA with the adequacy and acceptability of specific critical elements of the overall protocol design that could support a future regulatory submission and marketing application if the trial results meet the agreed-upon criteria. For more information on Special Protocol Assessments, please visit: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/special-protocol-assessment-guidance-industry.

“Receiving SPA agreement from FDA is a significant milestone in our ongoing development of EDSIVO™ for patients with COL3A1-positive vEDS, who urgently need an approved treatment for this devastating disease,” said Adrian Quartel, MD, CMO of Acer. “The SPA underscores our alignment with FDA on important regulatory, clinical and scientific requirements for our planned Phase 3 trial and reflects our ongoing commitment to the vEDS patient community. We are planning for trial initiation by the end of Q2 2022.”

About the EDSIVO™ DiSCOVER Trial
The DiSCOVER trial is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy trial designed to evaluate EDSIVO™ in patients with genetically confirmed COL3A1-positive vEDS using a decentralized clinical trial design and independent adjudication committee. The primary objective of the trial is to determine whether EDSIVO™ reduces the occurrence of vEDS-related clinical events requiring medical attention (fatal and non-fatal cardiac or arterial events, uterine rupture, intestinal rupture, and/or unexplained sudden death) relative to placebo as measured by time to event. One interim analysis (based on number of primary events) is planned at approximately 24 months after full enrollment. Acer plans to enroll approximately 150 COL3A1-positive vEDS patients, all in the U.S., randomized 2:1 to receive either EDSIVO™ or placebo, respectively.

The duration of the DiSCOVER trial is currently estimated to be approximately 3.5 years to completion (based on statistical power calculations and number of primary events), once fully enrolled, which will require additional capital beyond mid-2022. In April 2022, FDA granted celiprolol Breakthrough Therapy designation in the U.S. for the treatment of patients with COL3A1-positive vEDS. EDSIVO™ (celiprolol) is an investigational product candidate which has not been approved by FDA. There is no guarantee that this product candidate will receive regulatory authority approval or become commercially available for any indications in the U.S.

More information on the planned EDSIVO™ pivotal clinical trial can be found at www.discoverceliprolol.com.

About vEDS
Ehlers-Danlos syndrome (EDS) is an inherited disorder caused by mutations in the genes responsible for the structure, production, or processing of collagen, an important component of the connective tissues in the human body, or proteins that interact with collagen. EDS is a spectrum disorder where patients present with various forms, the most serious of which is vascular Ehlers-Danlos syndrome (vEDS), also known as vEDS type IV, which is generally caused by a mutation in the COL3A1 gene resulting in reduced collagen levels. vEDS causes abnormal fragility in blood vessels, which can give rise to aneurysms, abnormal connections between blood vessels known as arteriovenous fistulas, arterial dissections, and spontaneous vascular ruptures, all of which can be potentially life-threatening. Gastrointestinal and uterine fragility or rupture also commonly occur in vEDS patients. Spontaneous arterial rupture has a peak incidence in the third or fourth decade of life in vEDS patients but may occur earlier and is the most common cause of sudden death in vEDS patients. Arterial rupture or dissection events occur in about 25% of patients before the age of 20 but increase to roughly 90% of patients by the age of 40. The median survival age of vEDS patients in the U.S. is 51 years, with arterial rupture being the most common cause of sudden death.¹

About EDSIVO™ (celiprolol)
Acer is developing EDSIVO™ (celiprolol), a new chemical entity (NCE), for the treatment of COL3A1-positive vEDS patients. Celiprolol received FDA Orphan Drug Designation for the treatment of vEDS in 2015. The EDSIVO™ NDA was originally submitted based on data obtained from the BBEST trial² and accepted for filing in October 2018 with priority review. Following FDA review, Acer received a Complete Response Letter (CRL) in June 2019 stating that it will be necessary to conduct an adequate and well-controlled trial to determine whether EDSIVO™ reduces the risk of clinical events in patients with vEDS. In April 2022, FDA granted celiprolol Breakthrough Therapy designation in the U.S. for the treatment of patients with COL3A1-positive vEDS. In May 2022, Acer reached agreement with FDA under a SPA for its pivotal Phase 3 clinical trial of EDSIVO™ (celiprolol) for the treatment of patients with COL3A1-positive vEDS.

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 viruses, including cytomegalovirus, zika, dengue, ebola and 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.acertx.com.

References

1. Pepin, et al. Survival is affected by mutation type and molecular mechanism in vascular Ehlers–Danlos syndrome (EDS type IV). Genet Med. 2014 Dec;16(12):881-8.
2. Ong KT, et al. Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. Lancet. 2010;376(9751):1476-1484

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 product candidates; 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.

Corporate and IR Contact:
Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
jdenike@acertx.com

#  #  #

NEWTON, MA and GENEVA, SWITZERLAND – May 6, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (“Acer”) and its collaboration partner, Relief Therapeutics Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (“Relief”), today announced the presentation of data evaluating the bioavailability, bioequivalence and taste attributes of ACER-001 (sodium phenylbutyrate) compared to sodium phenylbutyrate (BUPHENYL®) powder during poster sessions at the recent Genetic Metabolic Dieticians International (GMDI) Conference on May 5, 2022, in Las Vegas, Nevada.

“We are pleased to present results from these two studies at another renowned genetic metabolic conference,” said Adrian Quartel, MD, CMO of Acer. “These data further support our belief that, if approved, ACER-001 could offer an alternative to current therapies that may lead to meaningful clinical outcomes in patients with Urea Cycle Disorders (UCDs).”

“Despite available treatments, there remains an unmet medical need for additional management options for the treatment of UCDs,” added Raghuram (Ram) Selvaraju, Chairman of Relief. “We look forward to the Prescription Drug User Fee Act (PDUFA) target action date on June 5, 2022 and potential approval by the U.S. Food and Drug Administration (FDA) of Acer’s new drug application for ACER-001 for the treatment of UCDs. Assuming a favorable decision from the FDA, we plan to submit a Marketing Authorization Application in the EU for the product during 2022.”

ACER-001 Data Presented at GMDI
A copy of each poster presentation from the 2022 GMDI Conference is available on Acer’s website at: https://www.acertx.com/publications-and-presentations/.  

ACER-001: A Potential Alternative to Sodium and Glycerol Phenylbutyrate for Treatment of Urea Cycle Disorders¹
This poster summarizes results from two Phase 1 bridging studies that evaluated the bioavailability and bioequivalence of ACER-001 (sodium phenylbutyrate) compared to sodium phenylbutyrate (BUPHENYL®) powder. The objectives of the two studies were to determine the bioequivalence of ACER-001 administered as a suspension relative to sodium phenylbutyrate (BUPHENYL®) powder administered as a solution in healthy adult volunteers after a single dose under fasting and fed conditions, and to assess the effect of a high-fat meal on the pharmacokinetics (PK) of ACER-001.

The data presented concluded that ACER-001 was bioequivalent to sodium phenylbutyrate (BUPHENYL®) powder under both fed and fasting conditions. Higher levels of phenylbutyrate (PBA) and phenylacetate (PAA), a conjugate base of phenylacetic acid, were observed when ACER-001 was administered under fasting versus fed conditions. A similar reduction in the PK of sodium phenylbutyrate (BUPHENYL®) powder under fed conditions was observed between the fasted and fed studies. Adverse events in these studies showed no major safety signals and were similar to sodium phenylbutyrate (BUPHENYL®). These studies suggest investigating administration of nitrogen scavengers under fasting conditions, which may ultimately provide lower dose options and increase dosing flexibility.

Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders²
The second poster presented at GMDI details results from two Phase 1, open-label, repeated measures, taste assessment studies of polymer coated sodium phenylbutyrate (ACER-001) and sodium phenylbutyrate (BUPHENYL®) powder. The studies included healthy panelists who were required to complete a training program for a minimum of six months that educated panelists on the identification, description, and quantification of sensory attributes of products.

The objective of the two taste assessment studies was to identify and quantify the intensity of perceived flavor attributes of ACER-001 relative to sodium phenylbutyrate (BUPHENYL®) powder. Results from both studies concluded that ACER-001 was shown to have overall lower flavor intensity scores than sodium phenylbutyrate (BUPHENYL®) powder when administered within five minutes of preparation.

About UCDs
The urea cycle is a series of biochemical reactions that occur primarily in the liver, which converts toxic ammonia produced by the breakdown of protein and other nitrogen-containing molecules in the human body into urea for excretion³. UCDs are a group of disorders caused by genetic mutations that result in a deficiency in any one of the six enzymes or two of the amino acid transporters, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia.⁴ 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.

Medications for UCDs are primarily comprised of nitrogen scavenger drugs, which are substances that provide alternative excretion pathways for nitrogen by bypassing the urea cycle.⁵ The use of these alternative pathways for nitrogen removal is important for the management of acute episodes of hyperammonemia and are also included as part of a long-term treatment regimen for UCDs patients. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports⁶, while nitrogen scavenging medications are effective in helping to manage UCDs, non-compliance with treatment is common. Reasons given for non-compliance include the unpleasant taste associated with some available medications, the frequency with which medication must be taken 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 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 polymer coated formulation for oral administration is designed to minimize the aversive taste 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 viruses, including cytomegalovirus, zika, dengue, ebola and 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.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 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. Relief also has a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including Urea Cycle Disorders and Maple Syrup Urine Disease. Acer’s new drug application for ACER-001 for use as a treatment of urea cycle disorders was recently accepted by the FDA for filing with a PDUFA decision date of June 5, 2022. Finally, Relief’s acquisitions last summer of APR Applied Pharma Research SA and AdVita Lifescience GmbH brought 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. Follow Relief on LinkedIn.

References

1. Steiner R, et al. ACER-001: A Potential Alternative to Sodium and Glycerol Phenylbutyrate for Treatment of Urea Cycle Disorders. GMDI May 2022.
2. 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.
3. Weiner ID, Mitch WE, Sands JM. Urea and ammonia metabolism and the control of renal nitrogen excretion. Clin J Am Soc Nephrol. 2015;10(8):1444-1458
4. Ah Mew N, et al. Urea cycle disorders overview [updated June 22, 2017]. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews® [Internet]. University of Washington; 1993-2022. Accessed March 20, 2022.
5. Häberle J, Boddaert N, Burlina A, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet J Rare Dis. 2012;7:32.
6. Shchelochkov et al. Molecular Genetics & Metabolism Reports 8 (2016) 43-47.

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 product candidates; 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.

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 submit an application for approval of ACER-001 in Europe and the timing of filing such application, (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, and (iv) those other risks, uncertainties and factors described in RELIEF THERAPEUTICS Holding SA’s press releases and filings with the SIX Swiss Exchange and the U.S. Securities and Exchange Commission, 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.
jdenike@acertx.com
+1 844-902-6100

RELIEF Therapeutics Holding SA:
Jack Weinstein
Chief Financial Officer and Treasurer
contact@relieftherapeutics.com

INVESTOR RELATIONS CONTACTS
Acer Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
jdenike@acertx.com
+1 844-902-6100

Relief Therapeutics Holding SA:
Michael Miller
Rx Communications Group
mmiller@rxir.com
+1-917-633-6086

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NEWTON, MA –May 5, 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 present at, and participate in, the H.C. Wainwright Global Investment Conference May 24-26, 2022.

Format: On-demand recorded virtual presentation and one-on-one virtual meetings
Date: On-demand presentation available beginning at 7:00 am ET, May 24, 2022           
Webcast: https://journey.ct.events/view/2b639762-a11f-4570-8694-012c299884ce

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 viruses, including cytomegalovirus, zika, dengue, ebola and 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.acertx.com.

Corporate and IR Contact:
Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
jdenike@acertx.com

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NEWTON, MA and GENEVA, SWITZERLAND – April 12, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (“Acer”) and its collaboration partner, Relief Therapeutics Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (“Relief”), today announced the presentation of data evaluating the bioavailability, bioequivalence and taste attributes of taste-masked sodium phenylbutyrate (ACER-001) compared to sodium phenylbutyrate (BUPHENYL®) powder during poster sessions at the recent Society for Inherited Metabolic Disorders (SIMD) Annual Meeting on April 10-13, 2022 in Orlando, Florida.

“These data 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,” said Adrian Quartel, MD, CMO of Acer. “We are pleased with the outcomes of these studies and look forward to presenting them at future conferences.”

“We are highly encouraged by the results of these bioavailability, bioequivalence and taste attribute studies of ACER-001,” added Raghuram (Ram) Selvaraju, Chairman of Relief. “We look forward to the U.S. Food and Drug Administration’s decision on the Prescription Drug User Fee Act (PDUFA) target action date on June 5, 2022.”

ACER-001 Data Presented at SIMD
A copy of each poster presentation from the SIMD Meeting is available on Acer’s website at: https://www.acertx.com/publications-and-presentations/.  

The Pharmacokinetics of Taste-Masked Sodium Phenylbutyrate (ACER-001) for the Treatment of Urea Cycle Disorders Under Fasting and Fed Conditions in Healthy Volunteers¹

This poster summarizes results from two Phase 1 bridging studies that evaluated the bioavailability and bioequivalence of taste-masked sodium phenylbutyrate (ACER-001) compared to sodium phenylbutyrate (BUPHENYL®) powder. The objectives of the two studies were to determine the bioequivalence of taste-masked sodium phenylbutyrate (ACER-001) administered as a suspension relative to sodium phenylbutyrate (BUPHENYL®) powder administered as a solution in healthy adult volunteers after a single dose under fasting and fed conditions, and to assess the effect of a high-fat meal on the pharmacokinetics (PK) of taste-masked sodium phenylbutyrate (ACER-001).

The data presented concluded that taste-masked sodium phenylbutyrate (ACER-001) was bioequivalent to sodium phenylbutyrate (BUPHENYL®) powder under both fed and fasting conditions. Higher levels of phenylbutyrate (PBA) and phenylacetate (PAA), a conjugate base of phenylacetic acid, were observed when taste-masked sodium phenylbutyrate (ACER-001) was administered under fasting versus fed conditions. A similar reduction in the PK of sodium phenylbutyrate (BUPHENYL®) powder under fed conditions was observed between the fasted and fed studies. Adverse events in these studies showed no major safety signals and were similar to sodium phenylbutyrate (BUPHENYL®). These studies suggest investigating administration of nitrogen scavengers under fasting conditions, which may ultimately provide lower dose options and increase dosing flexibility.

Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders²

The second poster presented at SIMD details results from two Phase 1, open-label, repeated measures, taste assessment studies of taste-masked sodium phenylbutyrate (ACER-001) and sodium phenylbutyrate (BUPHENYL®) powder. The studies included healthy panelists who were required to complete a training program for a minimum of 6 months that educated panelists on the identification, description, and quantification of sensory attributes of products.

The objective of the two taste assessment studies was to identify and quantify the intensity of perceived flavor attributes of taste-masked sodium phenylbutyrate (ACER-001) relative to sodium phenylbutyrate (BUPHENYL®) powder. Results from both studies concluded that taste-masked sodium phenylbutyrate (ACER-001) was shown to have overall lower flavor intensity scores than sodium phenylbutyrate (BUPHENYL®) powder when administered within five minutes of preparation.

About UCDs
The urea cycle is a series of biochemical reactions that occur primarily in the liver, which converts toxic ammonia produced by the breakdown of protein and other nitrogen-containing molecules in the human body into urea for excretion. UCDs are a group of disorders caused by genetic mutations that result in a deficiency in any one of the six enzymes or two of the amino acid transporters, 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.

Medications for UCDs are primarily comprised of nitrogen scavenger drugs, which are substances that provide alternative excretion pathways for nitrogen by bypassing the urea cycle. The use of these alternative pathways for nitrogen removal is important for the management of acute episodes of hyperammonemia and are also included as part of a long-term treatment regimen for UCDs patients. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports³, while nitrogen scavenging medications are effective in helping to manage UCDs, non-compliance with treatment is common. Reasons given for non-compliance include the unpleasant taste associated with some available medications, the frequency with which medication must be taken 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.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 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. Relief also has a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including UCDs and MSUD. Acer’s new drug application for ACER-001 for use as a treatment of urea cycle disorders was recently accepted by the FDA for filing with a PDUFA decision date of June 5, 2022. Finally, Relief’s acquisitions last summer of APR Applied Pharma Research SA and AdVita Lifescience GmbH brought 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. Follow Relief on LinkedIn.

References

1. 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.
2. Cedarbaum S, et al. Taste-Masked Coating of Sodium Phenylbutyrate (ACER-001) Improves the Palatability of Sodium Phenylbutyrate for Treatment of Urea Cycle Disorders. SIMD April 2022.
3. Shchelochkov et al. Molecular Genetics & Metabolism Reports 8 (2016) 43-47.
4. 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 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.

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, and (iv) those other risks, uncertainties and factors described in RELIEF THERAPEUTICS Holding SA’s press releases and filings with the SIX Swiss Exchange and the U.S. Securities and Exchange Commission, 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.
jdenike@acertx.com
+1 844-902-6100

RELIEF Therapeutics Holding SA:
Jack Weinstein
Chief Financial Officer and Treasurer
contact@relieftherapeutics.com

INVESTOR RELATIONS CONTACTS
Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
hans@lifesciadvisors.com
+1 617-430-7578

Relief Therapeutics Holding SA:
Michael Miller
Rx Communications Group
mmiller@rxir.com
+1-917-633-6086

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NEWTON, MA – April 4, 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 U.S. Food and Drug Administration (FDA) has granted celiprolol Breakthrough Therapy designation (BTD) in the U.S. for the treatment of patients with COL3A1-positive vascular Ehlers-Danlos syndrome (vEDS).

BTD is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on at least one clinically significant endpoint(s) over available therapy.¹

“With no currently approved treatments for vEDS anywhere in the world, this designation by FDA marks an important step forward in support of our goal to provide treatment options like EDSIVO™ to rare disease patients, who are often overlooked or underserved,” said Adrian Quartel, MD, Chief Medical Officer of Acer.  “We look forward to continuing our discussions with FDA, through the SPA process, to seek agreement on the protocol design of the proposed pivotal Phase 3 DiSCOVER trial that we plan to initiate by the end of Q2 2022 once agreement is reached.”

EDSIVO™ (celiprolol) Regulatory Path Forward
In December 2021, Acer submitted to FDA a protocol for its proposed prospective pivotal trial, along with an IND which received FDA clearance in January 2022. Acer is working with FDA to reach agreement on a special protocol assessment (SPA), a process in which sponsors may ask to meet with FDA to reach agreement on the design and size of certain clinical trials, clinical studies, or animal studies.² Based on initial statistical power calculations, the Company anticipates that the trial would plan to enroll approximately 150 COL3A1-positive vEDS patients, all in the U.S. The duration of the clinical trial is currently estimated to be approximately 3.5 years to completion, once fully enrolled.

If agreement on the design of the protocol is reached with FDA through the SPA, initiation of the EDSIVO™ Decentralized Study of Celiprolol on vEDS-related Event Reduction (DiSCOVER) pivotal clinical trial is expected by the end of Q2 2022, subject to additional capital required to conduct and complete the trial beyond mid-2022. There can be no assurance that the resulting data from the trial would be adequate to support approval of an NDA, or that the NDA will be approved, although having a SPA agreement in place would indicate concurrence by FDA with the adequacy and acceptability of specific critical elements of overall protocol design (e.g., study conduct, entry criteria, dose selection, endpoints, and planned statistical and other analyses) for a study intended to support a future marketing application. EDSIVO™ is an investigational drug and is not currently FDA approved for any indication. More information is available at www.discoverceliprolol.com.

About vEDS
Ehlers-Danlos syndrome (EDS) is an inherited disorder caused by mutations in the genes responsible for the structure, production, or processing of collagen, an important component of the connective tissues in the human body, or proteins that interact with collagen. EDS is a spectrum disorder where patients present with various forms, the most serious of which is vascular Ehlers-Danlos syndrome (vEDS), also known as vEDS type IV, which is generally caused by a mutation in the COL3A1 gene resulting in reduced collagen levels. vEDS causes abnormal fragility in blood vessels, which can give rise to aneurysms, abnormal connections between blood vessels known as arteriovenous fistulas, arterial dissections, and spontaneous vascular ruptures, all of which can be potentially life-threatening. Gastrointestinal and uterine fragility or rupture also commonly occur in vEDS patients. Spontaneous arterial rupture has a peak incidence in the third or fourth decade of life in vEDS patients but may occur earlier and is the most common cause of sudden death in vEDS patients. Arterial rupture or dissection events occur in about 25% of patients before the age of 20 but increase to roughly 90% of patients by the age of 40. The median survival age of vEDS patients in the U.S. is 51 years, with arterial rupture being the most common cause of sudden death.³

About EDSIVO™ (celiprolol)
Acer is developing EDSIVO™, a new chemical entity (NCE), for the treatment of COL3A1-positive vEDS patients. Celiprolol received FDA Orphan Drug Designation for the treatment of vEDS in 2015. The EDSIVO™ NDA was originally submitted based on data obtained from the BBEST trial⁴ and accepted for filing in October 2018 with priority review. Following FDA review, Acer received a Complete Response Letter (CRL) in June 2019 stating that it will be necessary to conduct an adequate and well-controlled trial to determine whether EDSIVO™ reduces the risk of clinical events in patients with vEDS. Acer subsequently appealed the FDA decision and while FDA denied the appeal, it described possible paths forward for Acer to explore. In a Type B meeting in May 2021, the Company discussed with FDA the conduct of an U.S.-based prospective, randomized, double-blind, placebo-controlled, decentralized clinical trial in patients with COL3A1-positive vEDS, and sought FDA’s opinion on various proposed design features of the study. The official Type B meeting minutes outlined: the acceptability of a decentralized (virtual) clinical trial design and use of an independent centralized adjudication committee; acceptability of a primary endpoint based on clinical events associated with disease outcome; agreement with modest safety data collection (based on the known safety profile of the drug^4,5,6,7); and a statistical plan that considers the rare disease classification of vEDS.

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.acertx.com.

References

1. U.S. Food and Drug Administration (FDA). Breakthrough Therapy. https://www.fda.gov/forpatients/approvals/fast/ucm405397.htm Updated January 4, 2018. Accessed February 10, 2022
2. U.S. Food and Drug Administration (FDA). Special Protocol Assessment. https://www.fda.gov/media/97618/download Updated April 2018. Accessed April 3, 2022
3. Pepin, et al. Survival is affected by mutation type and molecular mechanism in vascular Ehlers–Danlos syndrome (EDS type IV). Genet Med. 2014 Dec;16(12):881-8.
4. Ong KT, et al. Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. Lancet. 2010;376(9751):1476-1484
5. Frank M, et al. Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study. J Am Coll Cardiol. 2019 Apr, 73 (15) 1948–1957
6. Nawarskas J, et al. Cardiology in Review 2017;25: 247–253.
7. Björck M, et al. Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome. European Journal of Vascular and Endovascular Surgery. November 20, 2020.

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.

Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
hans@lifesciadvisors.com

Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
jdenike@acertx.com

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