Acer Therapeutics Highlights Key 2022 Achievements and Pipeline Advancements, and Provides Anticipated 2023 Milestones

NEWTON, MA January 09, 2023 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today announced a corporate update and provided anticipated key development milestones for 2023.

“The U.S. approval of OLPRUVA™ (sodium phenylbutyrate) for oral suspension by the U.S. Food and Drug Administration (FDA) is a culmination of Acer’s collective efforts and ongoing dedication to develop and provide new treatments to patients suffering from rare diseases. Moreover, OLPRUVA™’s approval marks a significant milestone for patients in need, offering a new, responsibly priced sodium phenylbutyrate treatment option, that will be supported by Navigator by Acer Therapeutics, our patient services program designed to support patients and caregivers,” stated Chris Schelling, CEO and Founder of Acer. “Our commitment to patients is the cornerstone of our mission, and we are proud to have secured our first FDA approval. We look forward to providing further updates in due course as the commercial launch of OLPRUVA™ progresses.”

Mr. Schelling continued, “In addition to the approval of OLPRUVA™, our clinical team has been diligently executing on the clinical trials for ACER-801, being investigated in Vasomotor Symptoms (VMS), Post-traumatic Stress Disorder (PTSD) and now prostate cancer. We are eager to report the topline results from our ongoing Phase 2a trial of ACER-801 in moderate to severe VMS in post-menopausal women in Q1 of 2023. These trial results will provide important insight into ACER-801’s therapeutic potential in induced VMS (iVMS). Additionally, our pivotal Phase 3 trial of EDSIVO™ (celiprolol) in COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS) patients remains ongoing as planned, and we look forward to providing an update on full enrollment later this year.”

Review of Acer’s Pipeline

  • OLPRUVA™ (sodium phenylbutyrate) for oral suspension
    • On December 27, 2022, Acer announced that the U.S. Food and Drug Administration (FDA) approved OLPRUVA™ (sodium phenylbutyrate) for the treatment of certain patients with UCDs involving deficiencies of CPS, OTC or AS
    • OLPRUVA™ is a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with UCDs, involving deficiencies of CPS, OTC or AS. OLPRUVA™ is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment. For additional Important Safety Information, see full Prescribing Information, Patient Information and discuss with your doctor
    • Formation of Navigator by Acer Therapeutics: Navigator is Acer’s patient access program, a suite of integrated patient support services designed to help provide more convenient access to OLPRUVA™. The Navigator by Acer Therapeutics support services are intended to facilitate support, education, and treatment adherence to the UCD patient community
  • ACER-801 (osanetant)
    • Acer’s ongoing Phase 2a randomized, double-blind, placebo-controlled, dose-ranging trial of ACER-801 (osanetant) for the treatment of moderate to severe VMS in post-menopausal women is expected to readout in Q1 2023. The data from the Phase 2a trial will inform Acer’s development path for ACER-801 (osanetant) in iVMS
    • In October 2022, Acer announced the expansion of its ACER-801 (osanetant) program into PTSD, a disorder that affects over 12 million adults in the U.S. each year.1 Related to this program expansion, Acer licensed the worldwide exclusive rights and corresponding intellectual property portfolio from Emory University based on preclinical data that showed osanetant’s ability to block fear memory consolidation in mice
    • Concurrent with this expansion, the US Department of Defense (DoD) awarded a $3 million grant to the University of North Carolina Institute for Trauma Recovery to support a proposed investigator-sponsored Phase 2 trial evaluating osanetant in 180 trauma patients. The randomized, placebo-controlled Phase 2 trial is currently expected to begin in Q2 2023
    • In January 2023, Acer announced the initiation of two Phase 2, single-arm investigator-sponsored trials evaluating ACER-801 (osanetant) in men with adenocarcinoma of the prostate. The POSH-MAP (Pilot of Osanetant for Severity of Hot Flashes in Men with Adenocarcinoma of the Prostate) and PORT-MAP (Pilot of Osanetant to Reduce Testosterone in Men with Adenocarcinoma of the Prostate) trials are being sponsored and conducted by The University of Kansas Cancer Center in partnership with Acer
  • EDSIVO™ (celiprolol)
    • Acer’s ongoing Phase 3 DiSCOVER trial is a randomized, double-blind, placebo-controlled efficacy trial designed to evaluate EDSIVO™ (celiprolol) in patients with genetically confirmed COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS)

Expected 2023 Development Milestones (Subject to Available Capital)

  • Q1 2023: Acer expects to announce topline results from its ongoing Phase 2a randomized, double-blind, placebo-controlled, dose-ranging trial of ACER-801 (osanetant) for the treatment of moderate to severe VMS in post-menopausal women
  • Q2 2023: Acer expects initiation of the UNC investigator-sponsored trial to evaluate the potential for ACER-801 (osanetant) to reduce the frequency and severity of PTSD and other trauma related disorders
  • Q4 2023: Acer anticipates completing enrollment in its ongoing, pivotal Phase 3 DiSCOVER trial of EDSIVO™ (celiprolol) in patients with COL3A1-positive vEDS. The double-blind portion of DiSCOVER trial is intended to end if statistical significance is reached at an interim analysis which occurs at accrual of 28 vEDS-related events, estimated to occur as early as approximately 18 months after completion of full enrollment, or after accrual of 46 vEDS-related clinical events
  • Acer intends to explore additional lifecycle opportunities for OLPRUVA™ (sodium phenylbutyrate) in various disorders where proof of concept data exists, including in Maple Syrup Urine Disease (MSUD), Pyruvate Dehydrogenase Complex Deficiency (PCDC), rare pediatric epilepsies and various liver disorders

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS), Post-traumatic Stress Disorder (PTSD) and prostate cancer; 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. National Center for PTSD.  How Common is PTSD in Adults?

Acer Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release are forward-looking statements. Examples of such statements include, but are not limited to, statements about the role we believe ACER-801 could play in reducing the frequency and severity of PTSD, the planned clinical evaluation of ACER-801 for such indication, the continued development of ACER-801 for treatment of iVMS and our expected 2023 milestones. Our pipeline products (including ACER-801) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the availability of financing to fund our pipeline product development programs and general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Corporate and IR Contact

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

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

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Acer Therapeutics Announces Initiation of Two Investigator-Sponsored Trials of ACER-801 (Osanetant) in Men with Adenocarcinoma of the Prostate

NEWTON, MA – Jan. 5, 2023 –Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced the initiation of two Phase 2, single-arm investigator-sponsored trials evaluating ACER-801 (osanetant) in men with adenocarcinoma of the prostate. The POSH-MAP (Pilot of Osanetant for Severity of Hot Flashes in Men with Adenocarcinoma of the Prostate) and PORT-MAP (Pilot of Osanetant to Reduce Testosterone in Men with Adenocarcinoma of the Prostate) trials are being sponsored and conducted by The University of Kansas Cancer Center in partnership with Acer.

POSH-MAP Trial
The POSH-MAP trial will evaluate the ability of ACER-801 to reduce hot flash frequency and severity and improve quality of life measures in men with prostate cancer following 28 days of therapy. Approximately 10 participants will receive 200mg of osanetant twice daily. Following the completion of treatment on day 28 participants will re-test hormone levels and report final patient outcome measures. More information on this trial can be found here.  

“Prostate cancer’s responsiveness to hormone-based treatments that decrease testosterone levels such as leuprolide or other forms of androgen deprivation therapy (ADT) have established them as the standard of care for treatment of prostate cancer,” said Elizabeth Wulff-Burchfield, MD, Principal Investigator of the POSH-MAP trial, Divisions of Medical Oncology and Palliative Medicine, Department of Medicine, The University of Kansas Health System. “However, for men on ADT, this causes a secondary decline in estrogen that results in dysfunctional thermoregulation and development of vasomotor symptoms (VMS) which can affect quality of life and lead to treatment non-adherence.1 With clinical data showing the potential of neurokinin 3 receptor (NK3R) antagonism to reduce menopausal related VMS in women,2 we look forward to evaluating the ability of ACER-801 to help mediate ADT-related VMS in men with prostate cancer.”

PORT-MAP Trial
The second trial, PORT-MAP, will evaluate the ability of ACER-801 to suppress testosterone production in men with prostate cancer within 28 days prior to a planned prostatectomy. Approximately 10 participants will receive 200mg of osanetant twice daily for 28 days, followed by a one week wash out period. Following the one week wash out period, patients will undergo a prostatectomy between days 35-39. The overall effect of osanetant on testosterone levels and the proportion of men achieving castrate levels of testosterone (<50ng/ml) will be assessed, with hormone level assessment occurring on days 2, 3, 14, 28 and day 77. More information on this trial can be found here.

“Early studies in healthy male volunteers treated with various NK3R antagonists have shown an inhibitory effect on the levels of testosterone,” said William Parker, MD, Principal Investigator, Division of Urologic Oncology, Department of Urology, The University of Kansas Health System. “However, the ability of NK3R antagonists to reduce testosterone to castrate levels in prostate cancer patients has not been evaluated to date. Based on these data, we look forward to evaluating the potential of ACER-801 and its ability to reduce testosterone in men with prostate cancer.”

“We are pleased to partner with The University of Kansas Cancer Center to evaluate ACER-801 in men with prostate cancer currently receiving ADT treatment in these investigator-sponsored trials,” said Adrian Quartel, MD, FFPM, Chief Medical Officer of Acer. “With the recent expansion of our ACER-801 (osanetant) program into stress-related trauma disorders, including PTSD, and now men with prostate cancer, we look forward to the ongoing evaluation of ACER-801 in multiple indications and reporting topline results from our ongoing Phase 2a trial for the treatment of moderate to severe VMS in post-menopausal women in Q1 2023.”

Rationale for ACER-801 (osanetant) NK3R Antagonist Evaluation in Prostate Cancer 
Prostate cancer is a hormonally driven cancer, and the management of this disease for many men is through suppression of testosterone production – called androgen deprivation therapy (ADT). Currently, most men on ADT are treated with medications that suppress hormone production which can cause dysfunctional thermoregulation and development of vasomotor symptoms (VMS), also known as hot flashes. Up to 75% of men on ADT experience VMS, resulting in high rates of distress and ADT treatment noncompliance, with approximately 20% of men with high-risk prostate cancer prematurely discontinuing ADT.3 Early pharmacokinetic studies in men and women with various NK3R antagonists have shown an inhibitory effect on the levels of luteinizing hormone and testosterone. However, the degree of effect relative to a therapeutic goal of castrate levels of testosterone (≤ 50ng/mL) remains unexplored.1,2 A non-hormonal treatment to lower testosterone levels and manage induced VMS is needed as estrogen is contraindicated for the management of VMS in patients with hormone-positive tumors, including breast and prostate tumors.

ACER-801 is an investigational product candidate which has not been approved by FDA or any other regulatory authority. There is no guarantee that this product candidate will receive regulatory authority approval in any territory or become commercially available for any indications.

About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS), Post-traumatic Stress Disorder (PTSD) and prostate cancer; 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. Challapalli, Amarnath, et al. “Evaluating the Prevalence and Predictive Factors of Vasomotor and Psychological Symptoms in Prostate Cancer Patients Receiving Hormonal Therapy: Results from a Single Institution Experience.” Clinical and Translational Radiation Oncology, Elsevier, 21 Mar. 2018
  2. Prague J. et al. Neurokinin 3 receptor antagonism rapidly improves vasomotor symptoms with sustained duration of action. Menopause. 2018 Aug; 25(8): 862–869.
  3. Trinity Partners 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 are forward-looking statements. Examples of such statements include, but are not limited to, statements about the role we believe ACER-801 could play in mediating ADT-related VMS in men with prostate cancer and reducing testosterone levels in men with prostate cancer, the planned clinical evaluation of ACER-801 for such indications, plans with respect to the POSH-MAP trial and PORT-MAP trial, including enrollment, timing, outcome and participants, the continued development of ACER-801 for multiple indications, and our plans, including timing, to report topline results from our ongoing Phase 2a trial for the treatment of moderate to severe VMS in post-menopausal women. Our pipeline products (including ACER-801) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the availability of financing to fund our pipeline product development programs and general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Acer Contacts

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

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

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Acer Therapeutics Compliant with All Nasdaq Listing Criteria

NEWTON, MA – Dec. 29, 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 The Nasdaq Stock Market LLC has formally notified Acer that the company has regained compliance with the $35 million market value of listed securities requirement, and otherwise satisfies all other criteria necessary, for continued listing on The Nasdaq Capital Market. Accordingly, the listing matter is now closed and the previously-scheduled hearing before the Nasdaq Hearings Panel has been cancelled.

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various other inborn errors of metabolism, including Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS) and Post-traumatic Stress Disorder (PTSD); 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 Contacts

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

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

# # #

Acer Therapeutics and Relief Therapeutics Announce U.S. FDA Approval of OLPRUVA™for Patients with Urea Cycle Disorders

NEWTON, MASS. and GENEVA – Dec. 27, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (“Acer”) and its collaboration partner, RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (“Relief”), today announced that the U.S. Food and Drug Administration (FDA) has approved OLPRUVA™ (sodium phenylbutyrate) for oral suspension in the U.S. for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

“The FDA’s approval of OLPRUVA™, an innovative formulation of sodium phenylbutyrate packaged for the first time in single-dose envelopes, marks the culmination of our ongoing dedication to develop new and differentiated treatment options for those affected by rare diseases,” said Chris Schelling, chief executive officer and founder of Acer. “Patients who are living with UCDs now have an alternative treatment option with OLPRUVA™, to address some of the challenges they may have with existing therapy. We are pleased to be able to provide a new, approved treatment choice for those living with this challenging disease.”

Mr. Schelling continued, “This approval represents the first FDA-approved product for Acer, validating our ability to identify and develop treatments where science can be applied in novel ways and make them available to patients as quickly and efficiently as possible. In addition, this approval unlocks our Marathon debt funding option and provides us with resources to advance our pipeline of investigational product candidates.”

“The daily challenges of living with a UCD can be overwhelming and emotionally draining for patients and their families,” said Tresa Warner, president of the National Urea Cycle Disorders Foundation. “We welcome new treatment options that can help patients, caregivers and their healthcare teams manage UCDs.”

OLPRUVA’s™ approval triggers the availability of a $42.5 million term loan to Acer under the previously announced March 2022 loan agreement the Company entered into with affiliates of Marathon Asset Management L.P. If Acer requests and receives the loan proceeds, management believes it will have sufficient resources to fund current operations into H2 2023.

OLPRUVA™ has been approved as an oral suspension by the FDA for the treatment of patients with UCDs. UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, potentially resulting in brain damage and neurocognitive impairments, if ammonia levels are not controlled.1 Any increase in ammonia over time is serious. Therefore, it is important to adhere to any dietary protein restrictions and have alternative medication options to help control ammonia levels.

“This FDA approval is a significant milestone for patients with UCDs in the U.S., offering an additional choice to manage their condition,” added Jack Weinstein, chief executive officer of Relief. “We look forward to building on OLPRUVA™’s approval in the U.S. and expanding its availability into other territories outside of the U.S.”

OLPRUVA™ received FDA approval under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act (FDCA), a regulatory pathway that allows applicants to rely, at least in part, on third party data for approval. In its New Drug Application (NDA), Acer cited preclinical and clinical safety and efficacy data from the reference listed drug (RLD), BUPHENYL® powder, which is approved as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of CPS, OTC or AS. In its NDA, Acer also provided additional data including studies that evaluated the bioavailability and bioequivalence of OLPRUVA™ compared to BUPHENYL® powder. The data from these studies, presented at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting in April 2022 and the Genetic Metabolic Dieticians International (GMDI) Conference in May 2022, showed that OLPRUVA™ was bioequivalent to BUPHENYL® powder.

Commitment to Patient Access

Acer intends to offer Navigator by Acer Therapeutics, a suite of integrated patient support services designed to facilitate access to therapy. Navigator by Acer Therapeutics is designed to assist UCD patients with support, access, education, and adherence.

Financial Outlook

Acer is not currently providing specific revenue or operating expense guidance for OLPRUVA™. Based on current forecasted operating expenses and revenue, and assuming receipt of the $42.5 million term loan funds from its March 2022 term loan arrangement with Marathon (less the amount to repay the bridge loan and fees), and Acer’s existing cash and equivalents, Acer believes its cash resources will be sufficient to fund its operations into H2 2023. Further information with respect to Acer’s March 2022 term loan arrangement, as well as a bridge loan facility (as amended in August 2022) and a convertible note financing which also funded in March 2022 can be found in the SEC Filings section of Acer’s website.

About OLPRUVA

OLPRUVA™ is a prescription medicine used along with certain therapy, including changes in diet, for the long-term management of adults and children weighing 44 pounds (20 kg) or greater and with a body surface area (BSA) of 1.2 m2 or greater, with urea cycle disorders (UCDs), involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinic acid synthetase (AS). OLPRUVA™ is not used to treat rapid increase of ammonia in the blood (acute hyperammonemia), which can be life-threatening and requires emergency medical treatment.

Important Safety Information

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

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

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

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

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. In the U.S., OLPRUVA™ (sodium phenylbutyrate) is approved for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: OLPRUVA™ (sodium phenylbutyrate) for treatment of various other inborn errors of metabolism, including Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS) and Post-traumatic Stress Disorder (PTSD); 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. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of OLPRUVA™ in which Acer retains development and commercialization rights in the U.S., Canada, Brazil, Turkey, and Japan. For more information, visit www.acertx.com.

About RELIEF THERAPEUTICS Holding SA

RELIEF THERAPEUTICS Holding SA is a Swiss, commercial-stage, biopharmaceutical company focused on identification, development and commercialization of novel, patent protected products intended for the treatment of rare and ultra-rare diseases including metabolic disorders, pulmonary diseases and connective tissue disorders. Relief’s diversified pipeline consists of assets with the potential to effectively address significant unmet medical needs, including PKU GOLIKE®, engineered with the proprietary Physiomimic technology, which is the first prolonged-release amino acid product commercialized for the dietary management of phenylketonuria (PKU). 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 urea cycle disorders (UCDs) and maple syrup urine disease (MSUD). Relief also continues to develop aviptadil for several rare pulmonary indications. Further, Relief is in clinical development for APR-TD011, a differentiated acid oxidizing solution of hypochlorous acid intended for the treatment of epidermolysis bullosa (EB), a group of rare, genetic, life-threatening connective tissue disorders; APR-TD011 has been granted orphan drug designation by the U.S. FDA. Finally, Relief is commercializing several legacy products via licensing and distribution partners. 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, please visit www.relieftherapeutics.com. You may also follow Relief Therapeutics on LinkedIn.

References

  1. 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.
  2. 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.
  3. Gerstein MT, Markus AR, Gianattasio KZ, et al. Choosing between medical management and liver transplant in urea cycle disorders: a conceptual framework for parental treatment decision-making in rare disease. J Inherit Metab Dis. 2020;43(3):438-458.
  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 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 and potential of OLPRUVA™ for the treatment of patients with UCDs, including the opportunity for approval in territories outside of the United States; 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 OLPRUVATM in MSUD, ACER-801, EDSIVO™ or our other investigational 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 product candidates are under investigation, 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 ability to launch successfully and sustain commercial viability of OLPRUVA™ for the treatment of patients with UCDs in the United States, 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 RELIEF THERAPEUTICS Holding SA will submit an application for approval of ACER-001 in Europe and the timing of filing such application, (ii) 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 (iii) 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.

TM and ® denote trademarks and registered trademarks, respectively, of their respective owners.

CORPORATE CONTACTS

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

RELIEF Therapeutics Holding SA:
Catherine Day
Vice President, IR & Communications
contact@relieftherapeutics.com

INVESTOR RELATIONS CONTACTS

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

RELIEF Therapeutics Holding SA:
Irina Koffler
LifeSci Advisors
ikoffler@lifesciadvisors.com
+1-917-734-7387

#  #  #

Acer Therapeutics Announces $1.5M Private Placement

NEWTON, MA November 30, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious, rare and life-threatening diseases with significant unmet medical needs, today announced that it has entered into definitive agreements with its CEO as well as its Chairman for a private placement of its securities for gross proceeds of $1.5 million.

On November 29, 2022, Acer entered into a securities purchase agreement for the sale and issuance of an aggregate of 1,229,508 shares of the Company’s common stock, for an aggregate purchase price of $1,499,999.76, in a private placement transaction at a price per share of $1.22, which represented a 5.2% premium to the $1.16 closing price of the common stock on that day. The investors are Chris Schelling, CEO and Founder of Acer, and Steve Aselage, Chairman of Acer’s Board of Directors. The private placement is scheduled to close on or about December 2, 2022, subject to the satisfaction or waiver of customary closing conditions.

The proceeds from the private placement will be used by Acer for working capital and general corporate purposes and, together with Acer’s existing cash and cash equivalents, are expected to be sufficient to fund the Company’s anticipated operating and capital requirements through the fourth quarter of 2022.

About Acer Therapeutics
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. 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) and post-traumatic stress disorder (PTSD); 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 are forward-looking statements. Examples of such statements include, but are not limited to, statements regarding the sufficiency and duration of our cash and cash equivalents as well as the consummation of the proposed private placement transaction (which is subject to customary closing conditions). 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, the Company’s actual operating and capital requirements and whether the closing conditions to the proposed private placement transaction are satisfied. 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.

Acer Contacts

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

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

# # #

Acer Therapeutics Reports Q3 2022 Financial Results and Provides Corporate Update

NEWTON, MA November 14, 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 third quarter ended September 30, 2022 and provided an update on Acer’s recent corporate developments.

“I am pleased with our team’s progress in the third quarter, highlighted by the timely resubmission of our New Drug Application (NDA) following the Complete Response Letter (CRL) issued by the US Food and Drug Administration (FDA) in response to ACER-001’s NDA submission,” stated Chris Schelling, CEO and Founder of Acer. “As the FDA continues its review of our resubmitted NDA for UCDs, we remain focused on advancing ACER-001 for the treatment of Maple Syrup Urine Disease (MSUD), having submitted our (Investigational New Drug) IND application to the FDA for a Phase 2a trial and receiving Orphan Drug Designation (ODD) from the European Commission.”

“In addition to the advancement of ACER-001, we made significant progress with the continued development of our other pipeline programs, including the initiation of our pivotal, Phase 3 DiSCOVER trial of EDSIVO™ (celiprolol) for the treatment of COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS) patients — the only ongoing clinical trial in this patient population, to our knowledge — and expansion of our ACER-801 program into treatment and prevention of Post-traumatic Stress Disorder (PTSD) through an investigator-sponsored trial to be conducted by the University of North Carolina (UNC),” Schelling continued. “And early next year, we look forward to reporting topline results from our ongoing Phase 2a clinical trial of ACER-801 for the treatment of moderate to severe Vasomotor Symptoms (VMS) in post-menopausal women.”

Q3 and Recent Highlights

  • ACER-001 (sodium phenylbutyrate)
    • In July 2022, Acer resubmitted its NDA for ACER-001 to the FDA following receipt of a CRL in June 2022, in which the FDA stated that satisfactory inspection of Acer’s third-party contract packaging manufacturer is required before the ACER-001 NDA may be approved
    • In July 2022, the FDA accepted Acer’s NDA resubmission (Class 2) and assigned a Prescription Drug User Fee Act (PDUFA) target action date of January 15, 2023
    • In July 2022, the China National Intellectual Property Administration (CNIPA) issued a utility model patent for ACER-001 covering certain dosage form claims related to ACER-001’s polymer-coated formulation, with an expiration date of August 24, 2031
    • In July 2022, Acer submitted its IND application to the FDA, which is now in effect, for the investigation of ACER-001 in a Phase 2a, open-label dose-ranging efficacy clinical trial that would evaluate the treatment effect of different doses of ACER-001 on blood leucine and other branched-chain amino acid (BCAA) levels in MSUD patients (initiation of this trial is subject to additional capital)
    • In August 2022, the European Commission granted orphan medicinal product designation in the EU to ACER-001 for the potential treatment of MSUD
    • In October 2022, Acer received a Notice of Allowance from the US Patent and Trademark Office (USPTO) for US patent application No. 16/624,834 for claims related to a kit comprising sodium phenylbutyrate or glycerol phenylbutyrate and sodium benzoate 
  • ACER-801 (osanetant)
    • In October 2022, Acer announced expansion of its ACER-801 (osanetant) program into PTSD, an indication that affects over 12 million adults in the US alone. The worldwide exclusive rights, related patents and patent applications, were licensed from Emory University based on preclinical data in which osanetant was able to block fear memory consolidation in mice
    • In October 2022, the Department of Defense (DoD) awarded a $3 million grant to the UNC Institute for Trauma Recovery to support a proposed 180-patient, randomized, placebo-controlled trial evaluating osanetant in trauma patients
  • EDSIVO™ (celiprolol)
    • In October 2022, Acer announced the USPTO issued a Notice of Allowance for Acer’s patent application No. 16/930,208 for claims related to certain methods of treating vEDS with celiprolol
  • Corporate
    • Ended Q3 2022 with $6.4 million in cash and cash equivalents, which Acer believes will be sufficient to fund its currently anticipated operating and capital requirements into, but not through, the fourth quarter of 2022

Anticipated Milestones (Subject to Available Capital)

  • ACER-001 (sodium phenylbutyrate)
    • January 15, 2023: The FDA has assigned a new PDUFA target action date of January 15, 2023, on the pending NDA, following the FDA’s acceptance for review of Acer’s resubmitted NDA for ACER-001 for the treatment of patients with UCDs
    • 2023: Acer plans to initiate in 2023 its Phase 2a trial to evaluate the efficacy and safety of ACER-001 for the potential treatment of patients with MSUD
  • ACER-801 (osanetant)
    • Q1 2023: Acer expects to announce topline results in Q1 2023 from its ongoing Phase 2a randomized, double-blind, placebo-controlled, dose-ranging trial of ACER-801 for the treatment of moderate to severe VMS in post-menopausal women
    • H1 2023: UNC expects to initiate in H1 2023 the proposed investigator-sponsored trial (OASIS) to evaluate the potential for ACER-801 to reduce the frequency and severity of PTSD
  • EDSIVO™ (celiprolol)
    • Q4 2023: Acer anticipates the DiSCOVER trial for celiprolol in patients with COL3A1-positive vEDS will be fully enrolled in Q4 2023. Once 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). One interim analysis (based on the number of primary events occurring) is also planned at approximately 24 months after full enrollment

Q3 2022 Financial Results

Cash Position. Cash and cash equivalents were $6.4 million as of September 30, 2022, compared to $12.7 million as of December 31, 2021. Acer believes its cash and cash equivalents available as of September 30, 2022 will be sufficient to fund its currently anticipated operating and capital requirements into, but not through, the fourth quarter of 2022.

Research and Development Expenses. Research and development expenses were $2.7 million, net of collaboration funding of $2.2 million, for the three months ended September 30, 2022, as compared to $1.4 million, net of collaboration funding of $3.5 million, for the three months ended September 30, 2021. This increase of $1.3 million was primarily due to increases in expenses for clinical studies related to ACER-801 and EDSIVO™, contract research, and employee-related expenses, partially offset by decreases in expenses related to contract manufacturing. Research and development expenses related to ACER-001 decreased in the three months ended September 30, 2022, resulting in a decrease in the recognition of the collaboration funding from the Collaboration Agreement with Relief. Research and development expenses for the three months ended September 30, 2022 were comprised of $2.3 million related to ACER-001, offset by $2.2 million of collaboration funding; $1.2 million related to ACER-801; $1.0 million related to EDSIVO; and $0.4 million related to other development activities.

General and Administrative Expenses. General and administrative expenses were $2.6 million, net of collaboration funding of $1.4 million, for the three months ended September 30, 2022, as compared to $1.8 million, net of collaboration funding of $1.0 million, for the three months ended September 30, 2021. This increase of $0.8 million was primarily due to increases in employee-related expenses, precommercial expenses, and audit and consulting fees, partially offset by an increase in the recognition of the collaboration funding from the Collaboration Agreement with Relief.

Net Loss. Net loss for the three months ended September 30, 2022 was $5.0 million, or $0.31 net loss per share (basic and diluted), compared to a net loss of $3.3 million, or $0.23 net loss per share (basic and diluted), for the three months ended September 30, 2021.

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

Completion of Financial Restatement

Acer has completed the restatement of its previously issued financial statements for the fiscal quarter ended June 30, 2022 (Restated Period), and has filed an amended Quarterly Report on Form 10-Q/A for the Restated Period with the SEC. A non-cash error was made in applying certain interpretive accounting guidance related to ASC 260, Earnings Per Share, which affected the Company’s presentation of its diluted earnings per share calculation. The correction of this non-cash error resulted in a restatement of Acer’s unaudited condensed interim financial statements and financial data for the Restated Period. The restatement had no impact on Acer’s cash position or operating expenses or its ongoing operations or future plans.

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. 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) and post-traumatic stress disorder (PTSD); 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 are forward-looking statements. Examples of such statements include, but are not limited to, statements regarding the sufficiency and duration of our cash and cash equivalents, our anticipated milestones, and our plans with respect to future clinical trials, results and timing thereof for ACER 001, ACER 801 and EDSIVO™. Our pipeline products (including ACER-801) are under investigation and their safety and efficacy have not been established and there is no guarantee that any of our investigational products in development will receive health authority approval or become commercially available for the uses being investigated. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the availability of financing to fund our pipeline product development programs and general corporate operations as well as risks related to drug development and the regulatory approval process, including the timing and requirements of regulatory actions. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. You may access these documents for no charge at http://www.sec.gov.

Acer Contacts

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

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

Acer Therapeutics Receives Notice of Allowance of Key US Patent Application Covering EDSIVO™ (celiprolol)

NEWTON, MA – Oct. 26, 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 that the US Patent and Trademark Office (USPTO) has issued a Notice of Allowance for Acer’s patent application No. 16/930,208, exclusively licensed from Assistance Publique—Hôpitaux de Paris, for claims related to certain methods of treating vascular Ehlers-Danlos syndrome (vEDS) with celiprolol. The allowed patent claims in the application titled, “Method of Providing Celiprolol Therapy to a Patient,” include the dosing regimen in Acer’s ongoing 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 vEDS.

“We are extremely pleased to receive this Notice of Allowance from the USPTO for our method of treatment of vEDS with celiprolol, as we continue to advance its development for the potential treatment of COL3A1-positive vEDS patients and other possible indications,” said Jeff Davis, Chief Business Officer at Acer. “These allowed method of use claims are based on the higher survival rate observed in vEDS patients when using a dose titration regimen to reach what we believe could be the preferred celiprolol dose as previously described in the ‘Long-Term Observational Study’ published in the Journal of American College of Cardiology (JACC)1. When issued, this patent will build on the potential regulatory exclusivities from celiprolol’s Orphan Drug Designation and New Chemical Entity (NCE) status. We look forward to the continued advancement of this program.”

Acer’s patent is expected to be issued in Q4 2022 and expire in 2038. If it receives marketing approval for EDSIVO™ after completion of the DiSCOVER trial and NDA resubmission, Acer intends to submit the patent for listing by the FDA in its Approved Drug Products with Therapeutic Equivalence Evaluations, or Orange Book.

About the 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 an 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, 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™ or placebo, respectively. Individuals seeking more information on the EDSIVO™ 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 in Q4 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 FDA. There is no guarantee that this product candidate will receive regulatory authority approval or become commercially available for any indications in the US.

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.2 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.3 In addition, data from long-term observational studies of patients treated with celiprolol in France and Sweden were published in JACC in April 20191 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, the FDA granted celiprolol Breakthrough Therapy designation in the US for the treatment of patients with COL3A1-positive vEDS. In May 2022, Acer confirmed agreement with the FDA under an SPA for its Pivotal Phase 3 clinical trial of EDSIVO™ 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) and post-traumatic stress disorder (PTSD); 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. Frank M, et al. Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study. J Am Coll Cardiol. 2019 Apr, 73 (15) 1948–1957
  2. 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.
  3. 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
  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 Company’s plans with respect to the continued advancement of the method of treatment of vEDS with celiprolol, the expected patent issuance date and duration, that the Company intends to submit the patent for listing by the FDA in its Approved Drug Products with Therapeutic Equivalence Evaluations program, 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 in Q4 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.

Acer Contacts

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

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

# # #

Acer Therapeutics Announces University of North Carolina Receives Department of Defense Grant to Support the Proposed Investigator Sponsored OASIS Trial of ACER-801 (Osanetant) to Reduce the Frequency and Severity of Post-Traumatic Stress Disorder

NEWTON, MA — October 6, 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 that the University of North Carolina (UNC) Institute for Trauma Recovery has been awarded a $3 million grant from the Department of Defense (DoD) to investigate the potential of ACER-801 (osanetant) to reduce the frequency and severity of acute stress disorder and post-traumatic stress disorder (PTSD). Acute stress disorder refers to the body’s immediate response to trauma, whereas PTSD is the long-term effects of trauma.

“Historically, we have been able to provide emergency care to address immediate and long-term problems after visible wounds using tools such as sutures and antibiotics. However, we still have nothing to offer trauma survivors, whether in the emergency department or on the battlefield immediately after trauma, to prevent the development of ‘invisible wounds’,” said Samuel McLean, MD, Professor at UNC School of Medicine, and lead principal investigator of the proposed study. Dr. McLean continued, “We need to investigate potential treatments like ACER-801 in an effort to better address these challenges.”

The proposed Osanetant After Stress to Increase recovery Success (OASIS) trial will examine the safety and efficacy of ACER-801 to reduce acute stress response symptoms, post-traumatic stress disorder symptoms and behavioral changes among patients presenting to the emergency department after a motor vehicle collision. It is intended to enroll a total of 180 subjects who will be randomized in the emergency department, to receive a low or high dose of ACER-801 or placebo in the emergency department and be discharged with a two-week supply of study drug. Participating sites would include Washington University in St. Louis, University of Massachusetts Chan Medical School, Rhode Island Hospital, University of Florida College of Medicine – Jacksonville, and Indiana University School of Medicine.

Initiation of patient enrollment in the proposed investigator-sponsored OASIS trial is anticipated in the first half of 2023, subject to Investigational New Drug (IND) application filing and US Food and Drug Administration (FDA) clearance.

The OASIS trial will build upon a foundation of knowledge and infrastructure developed through the UNC-led, $40 million AURORA initiative. The AURORA study is a major national research initiative to improve the understanding, prevention, and recovery of individuals who have experienced a traumatic event. AURORA is supported by funding from NIH, One Mind, private foundations, and partnerships with leading tech companies such as Mindstrong Health and Verily Life Sciences, the healthcare arm of Google’s parent company Alphabet.

“We are proud to be partnering with a leading academic institution in the field of trauma recovery as we seek to explore ACER-801 as a treatment option to reduce the frequency and severity of PTSD. Leveraging the support from the AURORA initiative allows Acer and UNC to streamline trial efficiency, reduce costs and increase trial power through enriching the target patient population, while utilizing the Department of Defense’s non-dilutive capital to primarily fund OASIS,” said Adrian Quartel, MD, FFPM, Chief Medical Officer of Acer. “The data from thousands of motor vehicle collisions collected through the AURORA initiative should allow us to better predict the correlation of the emergence of acute stress disorder or PTSD symptoms following a motor vehicle collision. We look forward to the planned OASIS trial following IND filing and FDA clearance.”

Added Brandon Staglin, President of One Mind “We are thrilled to see how our funding to the AURORA initiative over the last five years is accelerating further advancements such as the OASIS Trial. The targeted outcomes of the OASIS Trial are the types of results that One Mind supports and of incredible value to anyone who experiences trauma and traumatic stress.”

Acute and chronic stress disorders can affect both civilian and military populations. According to the National Center for PTSD, in the US about 60% of men and 50% of women experience at least one trauma in their lives.2 In the US alone, one-third of emergency department visits (40-50 million patients per year) are for evaluation after trauma exposures, and in a 2014 study involving 3,157 US veterans, 87% reported exposure to at least one potentially traumatic event during their service.3 Moreover, as many as 500,000 US troops who served in wars between 2001 and 2015 were diagnosed with PTSD.4

Rationale for ACER-801 (osanetant) Evaluation in Post-Traumatic Stress Disorder
The Tacr3 gene encodes tachykinin receptor 3 (NK3R), which belongs to the tachykinin receptor family. This family of proteins includes typical G protein-coupled receptors and belongs to the rhodopsin subfamily. NK3R functions by binding to its high-affinity ligand, Neurokinin B (NKB), which is encoded by the Tac3 (human) gene. The role of NKB-NK3R in growth and reproduction has been extensively studied, but NKB-NK3R is also widely expressed in the nervous system from the spinal cord to the brain and is involved in both physiological and pathological processes in the nervous system.5  In animal models, Tac2 (mice) mRNA levels are rapidly up-regulated during fear consolidation 30 minutes after fear conditioning, and subsequent NKB-NK3R activation can lead to over stress sensitization and the consolidation of fear,6 and treatment with osanetant has been shown to block a critical fear/stress sensitization step in the brain.7,8,9 An effective therapeutic to reduce acute and persistent/long-term psychological and somatic symptoms would fulfill a large unmet need.

About Acer Therapeutics
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. 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) and post-traumatic stress disorder (PTSD); 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. Sidran Institute.  Traumatic Stress Education & Advocacy Fact Sheet.
  2. National Center for PTSD.  How Common is PTSD in Adults?
  3. Wisco et al. 2014 J Clin Psychiatry.  Posttraumatic stress disorder in the US veteran population: results from the National Health and Resilience in Veterans Study. J Clin Psychiatry . 2014 Dec;75(12):1338-46
  4. Thompson 2015. Unlocking the secrets of PTSD. Time 2015;185:40–43
  5. Zhang et al. ACS Chemical Neuroscience 2020 11 (19), 2935-2943
  6. Al Abed et. Al, Biological Psychiatry 2021
  7. Andero R, Dias BG, Ressler KJ. A role for Tac2, NkB, and Nk3 receptor in normal and dysregulated fear memory consolidation. Neuron. 2014;83(2):444-454
  8. Andero R, Daniel S, Guo JD, et al. Amygdala-Dependent Molecular Mechanisms of the Tac2 Pathway in Fear Learning. Neuropsychopharmacology. 2016;41(11):2714-2722
  9. Zelikowsky M, Ding K, Anderson DJ. Neuropeptidergic Control of an Internal Brain State Produced by Prolonged Social Isolation Stress. Cold Spring Harb Symp Quant Biol. 2018;83:97-103

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

Acer Contacts

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

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

# # #

Acer Therapeutics Announces Expansion of ACER-801 (osanetant) Development Indications to Include Post-Traumatic Stress Disorder

NEWTON, MA October 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 the expansion of ACER-801 (osanetant) into a new indication, for the reduction of the frequency and severity of acute stress disorder and post-traumatic stress disorder (PTSD). Acute stress disorder refers to the body’s immediate response to trauma, whereas PTSD is defined as the long-term effects of trauma.

Studies conducted at Emory University screened thousands of genes that were activated in the brains of mice following fear conditioning events. The top gene identified was Tac2, which is responsible for the production of the peptide, Neurokinin B (NKB), in mice. The researchers showed that the Tac2 gene, expressed by neurons specifically within the amygdala, is required for modulating fear memories, and that NKB, and its specific receptor, NK3R, are also involved in the consolidation of fear memories. By administering the potent and specific NK3R antagonist, osanetant, they were able to block fear memory consolidation shortly after exposure to a trauma, potentially providing a novel therapeutic approach for disorders with altered fear learning such as PTSD.1

“Immediately – hours to several days – after trauma exposure, memory remains in a labile state, called the memory consolidation period, and blocking fear memory consolidation after trauma exposure could lower the frequency and severity of PTSD in trauma patients,” said Kerry Ressler, MD, PhD, Chief Scientific Officer and James and Patricia Poitras Chair in Psychiatry at McLean Hospital.

“Activation of the NK3 receptor pathway in the central amygdala is necessary and sufficient for the modulation of fear memories, and we have learned that by blocking this pathway with osanetant, we can block the consolidation of fear memories in animal models,”1 added Dr. Ressler. “Osanetant is a promising agent that could reduce the frequency and or severity of PTSD for millions of people who experience a traumatic event.” 

Acer previously entered into an agreement with Emory for an exclusive worldwide license to US Patent No. 10,314,835, US Application 15/320,952, and European Patent No. EP3160469 covering certain methods of treating or preventing PTSD with osanetant. 

“We are pleased to further expand our ACER-801 development program into PTSD, an increasingly prevalent psychiatric disorder that affects millions every year. Today’s announcement further validates Acer’s strategy of identifying and developing treatments based on promising technology that can be applied in new ways for use in diseases with high unmet need,” commented Chris Schelling, CEO and Founder of Acer Therapeutics. “While the role of the NK3R pathway in the hypothalamus to manage thermoregulation is well-established in clinical trials, this opportunity explores an entirely different mechanism of action for the drug. We look forward to presenting our clinical development plan for ACER-801 for the reduction of frequency and severity of PTSD in the near future.”

According to the National Center for PTSD, in the US about 6 of every 10 men (or 60%) and 5 of every 10 women (or 50%) experience at least one trauma in their lives leading to about 12 million adults in the U.S. have PTSD during a given year.3  In the US alone, one-third of emergency department visits are for evaluation after trauma exposures and up to 20% of people who have experienced a traumatic event will develop PTSD.4

Rationale for ACER-801 (osanetant) Evaluation in Post-Traumatic Stress Disorder
The Tacr3 gene encodes tachykinin receptor 3 (NK3R), which belongs to the tachykinin receptor family. This family of proteins includes typical G protein-coupled receptors and belongs to the rhodopsin subfamily. NK3R functions by binding to its high-affinity ligand, Neurokinin B (NKB), which is encoded by the Tac3 (human) gene. The role of NKB-NK3R in growth and reproduction has been extensively studied, but NKB-NK3R is also widely expressed in the nervous system from the spinal cord to the brain and is involved in both physiological and pathological processes in the nervous system.5  In animal models, Tac2 (mice) mRNA levels are rapidly up-regulated during fear consolidation 30 minutes after fear conditioning, and subsequent NKB-NK3R activation can lead to over stress sensitization and the consolidation of fear,6 and treatment with osanetant has been shown to block a critical fear/stress sensitization step in the brain.1,7,8  An effective therapeutic to reduce acute and persistent/long-term psychological and somatic symptoms would fulfill a large unmet need.

About Acer Therapeutics
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. 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) and post-traumatic stress disorder (PTSD); 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. Andero R, Dias BG, Ressler KJ. A role for Tac2, NkB, and Nk3 receptor in normal and dysregulated fear memory consolidation. Neuron. 2014;83(2):444-454
  2. Sidran Institute.  Traumatic Stress Education & Advocacy Fact Sheet.
  3. National Center for PTSD.  How Common is PTSD in Adults?
  4. Sidran Institute.  Traumatic Stress Education & Advocacy Fact Sheet.
  5. Zhang et al. Tacr3/NK3R: Beyond Their Roles in Reproduction. ACS Chemical Neuroscience 2020 11 (19), 2935-2943
  6. Al Abed et. Al, Biological Psychiatry 2021
  7. Andero R, Daniel S, Guo JD, et al. Amygdala-Dependent Molecular Mechanisms of the Tac2 Pathway in Fear Learning. Neuropsychopharmacology. 2016;41(11):2714-2722
  8. Zelikowsky M, Ding K, Anderson DJ. Neuropeptidergic Control of an Internal Brain State Produced by Prolonged Social Isolation Stress. Cold Spring Harb Symp Quant Biol. 2018;83:97-103

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

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

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

# # #

Acer Therapeutics and Relief Therapeutics Announce Receipt of Notice of Allowance of US Patent Application Covering a Kit Comprising Phenylbutyrate and Sodium Benzoate

NEWTON, MA and GENEVA, SWITZERLAND – October 3, 2022 – Acer Therapeutics Inc. (Nasdaq: ACER) (Acer) and RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF, RLFTY) (Relief) today announced that the US Patent and Trademark Office (USPTO) has issued a Notice of Allowance to Acer for US patent application No. 16/624,834 for claims related to a kit comprising a combination therapeutic product composed of sodium phenylbutyrate or glycerol phenylbutyrate and sodium benzoate. The patent application is exclusively licensed to Acer from Baylor College of Medicine.

“This Notice of Allowance expands ACER-001’s patent protection and adds an additional component to our product expansion strategy as we evaluate how to maximize its potential,” said Jeff Davis, Chief Business Officer at Acer. “The combination of phenylbutyrate and sodium benzoate was synergistic at removing ammonia in healthy subjects based on data from a study published in Genetics in Medicine in 2018.1 As a result, this combination offers the potential to use lower drug doses of each agent while maintaining equivalent ammonia removal in urea cycle disorder patients and becomes part of our lifecycle planning for ACER-001, subject to FDA approval in this indication.”

About Phenylbutyrate and Sodium Benzoate
Phenylbutyrate and sodium benzoate are nitrogen-binding agents that are used in the prevention and treatment of hyperammonemia in patients with UCDs.  Sodium benzoate for oral administration is available from compounding pharmacies and is widely used as a food preservative but has not been approved as a single agent by the U.S. Food and Drug Administration (FDA) or any regulatory authority for the treatment of UCDs. Sodium benzoate in combination with sodium phenylacetate is approved in the US and marketed as AMMONUL® (sodium phenylacetate and sodium benzoate) Injection.2 

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-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 is a Swiss, commercial-stage, biopharmaceutical company focused on identification development and commercialization of novel, patent protected products intended for the treatment of metabolic, dermatological and pulmonary rare diseases with a portfolio of clinical and marketed assets that serve unmet patient needs.  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). The FDA has accepted for review Acer’s New Drug Application (“NDA”) resubmission under the 505(b)(2) pathway for ACER-001, for oral suspension, for the treatment of patients with UCDs. The FDA designated the NDA as a Class 2 resubmission and set a PDUFA target action date of January 15, 2023. Relief also continues to develop aviptadil for several rare pulmonary indications; 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.

Reference

  1. Nagamani et al. A randomized trial to study the comparative efficacy of phenylbutyrate and benzoate on nitrogen excretion and ureagenesis in healthy volunteers. Genet Med. 2018 Jul; 20(7): 708–716.
  2. https://www.accessdata.fda.gov/drugsatfda_docs/label/2005/020645lbl.pdf

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 expected patent issuance date and duration, our strategy, our positioning, our products and regulatory actions 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.

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) the intellectual property protection provided by the patent discussed above, (ii) whether the FDA will approve Acer’s NDA for ACER-001 for the treatment of UCDs, (iii) whether RELIEF THERAPEUTICS Holding SA will submit an application for approval of ACER-001 in Europe for the treatment of UCDs and the timing of filing such application, (iv) whether any application submitted to European authorities seeking marketing authorization for ACER-001 for the treatment of patients in Europe with UCDs will be approved, (v) whether the FDA will approve Acer’s IND to evaluate ACER-001 for the treatment of MSUDs, (vi) the timing of Acer’s Phase 2b trial evaluating ACER-001 for the treatment of MSUDs, (vii) whether ACER-001’s currently proposed trial and any future required trials of ACER-001 for MSUDs will be undertaken and successful, (viii) whether ACER-001 will ever be approved for the treatment of MSUDs in the United States, (ix) whether Relief will ever file the necessary applications in Europe to seek the right to commercialize ACER-001 in Europe for the treatment of MSUDs and whether any such applications filed will be granted, and (x) 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:
Irina Koffler
LifeSci Advisors
ikoffler@lifesciadvisors.com
+1-917-734-7387

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