Category: Uncategorized

NEWTON, MA and GENEVA, SWITZERLAND – October 26, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER) (“Acer”) and its collaboration partner, Relief Therapeutics Holding SA (SIX: RLF, OTCQB: RLFTF) (“Relief”), today announced that the U.S. Patent and Trademark Office (USPTO) has issued a new U.S. patent to Acer for certain claims related to ACER-001 (sodium phenylbutyrate). Patent 11,154,521 covers pharmaceutical composition claims related to ACER-001’s taste-masked, multi-particulate dosage formulation for oral administration. The newly issued patent has an expiration date in 2036.

“We are extremely pleased that our ACER-001 formulation patent has been issued, adding key protection to our growing intellectual property portfolio for ACER-001 as we continue to advance its development to potentially treat patients with Urea Cycle Disorders (UCDs), Maple Syrup Urine Disease (MSUD) and other possible indications,” said Jeff Davis, Chief Business Officer at Acer. “This patent issuance is an important step in our pursuit of possible ACER-001 commercialization, and we intend to submit it for listing by the U.S. Food and Drug Administration (FDA) in its Approved Drug Products with Therapeutic Equivalence Evaluations, or Orange Book, should ACER-001 receive marketing approval.”

Jack Weinstein, Chief Financial Officer and Treasurer of Relief, added, “In parallel to the patent application efforts in the U.S., Acer and Relief are pursuing similar claims in the European Patent Office to cover ACER-001 as we continue to execute on our plan to submit a Marketing Authorization Application for ACER-001 for the treatment of patients with UCDs in Europe in Q2/Q3 2022.”

Parties interested in the ACER-001 program for UCDs may sign up for updates at:

https://www.acertx.com/rare-disease-research/acer-001-for-urea-cycle-disorders-ucds/

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 this product candidate will receive regulatory authority approval for marketing in any territory or become commercially available for the indications under investigation.

About UCDs

UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.^1,2   The current treatment of patients with UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.

Current medical treatments for patients with UCDs include nitrogen scavengers, RAVICTI® and BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate and sodium phenylbutyrate, respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications have been shown to be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, frequency with which medication must be taken, required number of pills, and the high cost of the medication.³

About ACER-001

ACER-001 (sodium phenylbutyrate) is being developed for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 is a nitrogen-binding agent in development for use as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). The formulation is a multi-particulate dosage formulation for oral administration consisting of a core center, a layer of active drug, and a taste-masked coating designed to avoid the bitter taste of sodium phenylbutyrate in the mouth while quickly dissolving in the low pH of the stomach. The ACER-001 NDA for UCDs is currently under FDA review with a PDUFA target action date of June 5, 2022. ACER-001 is also being developed for MSUD and has been granted orphan drug designation by the FDA for this indication. ACER-001 is an investigational product candidate which has not been approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority.

About Acer Therapeutics Inc.

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

About RELIEF THERAPEUTICS Holding SA

Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. Relief’s lead drug candidate RLF-100™ (aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the U.S. for the treatment of respiratory deficiency due to COVID-19. As part of its pipeline diversification strategy, in March 2021, Relief entered into a Collaboration and License Agreement with Acer Therapeutics for development and commercialization of ACER-001. ACER-001 is a taste-masked and immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment of Urea Cycle Disorders and Maple Syrup Urine Disease. In addition, Relief’s recently completed acquisitions of APR Applied Pharma Research SA and AdVita Lifescience GmbH, bring to Relief a diverse pipeline of marketed and development-stage programs.

RELIEF THERAPEUTICS Holding SA is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbol RLFTF. For more information, visit

www.relieftherapeutics.com. Follow Relief on LinkedIn.

References

1. Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
2. Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
3. Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.

Acer Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

Relief Forward-Looking Statements

This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding SA and its businesses.  Such statements involve certain known and unknown risks, uncertainties and other factors, including (i) whether the FDA will approve Acer’s NDA for ACER-001, (ii) whether RELIEF THERAPEUTICS Holding SA will be able to submit an application for approval of ACER-001 in Europe in Q2/Q3 2022 (or at all), (iii) whether any such application submitted to European authorities seeking marketing authorization for ACER-001 for the treatment of patient in Europe with UCDs will be approved, and (iv) those other risks, uncertainties and factors described in RELIEF THERAPEUTICS Holding SA’s annual and periodic filings with the SIX Stock Exchange, all of which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding SA to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding SA is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise.

CORPORATE CONTACTS

Acer Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
[email protected]
+1 844-902-6100

RELIEF THERAPEUTICS Holding SA:
Jack Weinstein
Chief Financial Officer and Treasurer
[email protected]

INVESTOR RELATIONS CONTACTS

Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
[email protected]
+1 617-430-7578

RELIEF THERAPEUTICS Holding SA:
Michael Miller
Rx Communications Group
[email protected]
+1-917-633-6086

NEWTON, MA and GENEVA, SWITZERLAND – October 7, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER) (“Acer”) and its collaboration partner, Relief Therapeutics Holding SA (SIX: RLF, OTCQB: RLFTF) (“Relief”), today announced that the U.S. Patent and Trademark Office (USPTO) has issued a Notice of Allowance for Acer’s patent application No. 17/196,416 for certain claims related to ACER-001 (sodium phenylbutyrate). The allowed patent claims in the application titled, “Palatable Compositions Including Sodium Phenylbutyrate and Uses Thereof,” include pharmaceutical composition claims covering ACER-001’s taste-masked, multi-particulate dosage formulation for oral administration.

The USPTO issues a patent Notice of Allowance after it determines a patent should be granted upon completion of any outstanding administrative requirements. Acer’s patent is expected to be issued in the fourth quarter of 2021 and expire in 2036. If it receives marketing approval for ACER-001, Acer intends to submit the patent for listing by the U.S. Food and Drug Administration (FDA) in its Approved Drug Products with Therapeutic Equivalence Evaluations, or Orange Book.

“We are extremely pleased to have received this Notice of Allowance from the USPTO for our proprietary formulation of ACER-001 as we continue to advance its development to potentially treat patients with Urea Cycle Disorders (UCDs), Maple Syrup Urine Disease (MSUD) and other possible indications,” said Jeff Davis, Chief Business Officer at Acer. “This Notice marks another important milestone in our pursuit of possible ACER-001 commercialization while we prepare to bring this treatment to UCDs patients, subject to FDA’s approval of our ACER-001 New Drug Application.”

Jack Weinstein, Chief Financial Officer and Treasurer of Relief, added, “In parallel to the patent application efforts in the U.S., Acer and Relief are pursuing similar claims in the European Patent Office to cover ACER-001 as we continue to execute on our plan to submit a Marketing Authorization Application for ACER-001 for the treatment of patients with UCDs in Europe in Q2/Q3 2022.”

Parties interested in the ACER-001 program for UCDs may sign up for updates at:

https://www.acertx.com/rare-disease-research/acer-001-for-urea-cycle-disorders-ucds/

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 this product candidate will receive regulatory authority approval for marketing in any territory or become commercially available for the indications under investigation.

About UCDs

UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.^1,2   The current treatment of patients with UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.

Current medical treatments for patients with UCDs include nitrogen scavengers, RAVICTI® and BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate and sodium phenylbutyrate, respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications have been shown to be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, frequency with which medication must be taken, required number of pills, and the high cost of the medication.³

About ACER-001

ACER-001 (sodium phenylbutyrate) is being developed for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 is a nitrogen-binding agent in development for use as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). The formulation is a multi-particulate dosage formulation for oral administration consisting of a core center, a layer of active drug, and a taste-masked coating designed to avoid the bitter taste of sodium phenylbutyrate in the mouth while quickly dissolving in the low pH of the stomach. The ACER-001 NDA for UCDs is currently under FDA review with a PDUFA target action date of June 5, 2022. ACER-001 is also being developed for MSUD and has been granted orphan drug designation by the FDA for this indication. ACER-001 is an investigational product candidate which has not been approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority.

About Acer Therapeutics Inc.

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

About RELIEF THERAPEUTICS Holding SA

Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. Relief’s lead drug candidate RLF-100™ (aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the U.S. for the treatment of respiratory deficiency due to COVID-19. As part of its pipeline diversification strategy, in March 2021, Relief entered into a Collaboration and License Agreement with Acer Therapeutics for development and commercialization of ACER-001. ACER-001 is a taste-masked and immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment of Urea Cycle Disorders and Maple Syrup Urine Disease. In addition, Relief’s recently completed acquisitions of APR Applied Pharma Research SA and AdVita Lifescience GmbH, bring to Relief a diverse pipeline of marketed and development-stage programs, a commercial infrastructure in selected European countries and an internal R&D capability, which Relief hopes to leverage for both internal pipeline products as well as for third party product development on a fee for service basis.

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 symbol RLFTF. For more information, visit www.relieftherapeutics.com. Follow Relief on LinkedIn.

References

1. Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
2. Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
3. Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.

Acer Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

Relief Forward-Looking Statements

This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding SA and its businesses.  Such statements involve certain known and unknown risks, uncertainties and other factors, including (i) whether the FDA will approve Acer’s NDA for ACER-001, (ii) whether RELIEF THERAPEUTICS Holding SA will be able to submit an application for approval of ACER-001 in Europe in Q2/Q3 2022 (or at all), (iii) whether any such application submitted to European authorities seeking marketing authorization for ACER-001 for the treatment of patient in Europe with UCDs will be approved, (iv) whether RLF-100 (aviptadil) will receive emergency use authorization in the United States, (v) whether RLF-100 (aviptadil) will ever be submitted for authorization in Europe, (vi) whether RELIEF THERAPEUTICS Holding SA’s ongoing disputes with its U.S. collaboration partner for RLF-100 (aviptadil) can be resolved amicably, and (vii) those other risks, uncertainties and factors described in RELIEF THERAPEUTICS Holding SA’s annual and periodic filings with the SIX Stock Exchange, all of which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding SA to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding SA is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise.

CORPORATE CONTACTS

Acer Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
[email protected]
+1 844-902-6100

RELIEF THERAPEUTICS Holding SA:
Jack Weinstein
Chief Financial Officer and Treasurer
[email protected]

INVESTOR RELATIONS CONTACTS

Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
[email protected]
+1 617-430-7578

RELIEF THERAPEUTICS Holding SA:
Michael Miller
Rx Communications Group
[email protected]
+1-917-633-6086

#  #  #

NEWTON, MA and GENEVA, SWITZERLAND – October 6, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER) (“Acer”) and its collaboration partner, Relief Therapeutics Holding SA (SIX: RLF, OTCQB: RLFTF) (“Relief”), today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for ACER-001 (sodium phenylbutyrate) for the treatment of patients with Urea Cycle Disorders (UCDs). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 5, 2022.

Acer’s 505(b)(2) NDA is supported by results from two previously announced bioequivalence (BE) trials in which ACER-001 showed similar relative bioavailability for both phenylbutyrate (PBA) and phenylacetate (PAA), the active metabolite of sodium phenylbutyrate, compared to the reference listed drug, BUPHENYL® (sodium phenylbutyrate).

“With FDA commencing a substantive review of our NDA, ACER-001 is one step closer to potentially providing an alternative treatment option for patients with UCDs,” said Chris Schelling, Chief Executive Officer and Founder of Acer. “We look forward to working with the FDA during their review of our application. In addition, we continue to collaborate with our partners to ensure we are well positioned to support a successful commercial launch of ACER-001, subject to FDA approval.”

Jack Weinstein, Chief Financial Officer and Treasurer of Relief, added, “Our collaboration with Acer is thriving and we are pleased with the progress they have made in advancing ACER-001. In parallel with Acer’s activities, we continue to execute on our global commercial strategy for ACER-001 which includes our intended submission of a Marketing Authorization Application (MAA) for the treatment of patients with UCDs in Europe in Q2/Q3 2022.”

Parties interested in the ACER-001 program for UCDs may sign up for updates at:

https://www.acertx.com/rare-disease-research/acer-001-for-urea-cycle-disorders-ucds/

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 is no guarantee that this product candidate will receive regulatory authority approval in any territory or become commercially available for the indications under investigation.

About UCDs

UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.^1,2   The current treatment of patients with UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.

Current medical treatments for patients with UCDs include nitrogen scavengers, RAVICTI® and BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate and sodium phenylbutyrate, respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications have been shown to be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, frequency with which medication must be taken, required number of pills, and the high cost of the medication.³

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). The formulation is a multi-particulate dosage formulation for oral administration consisting of a core center, a layer of active drug, and a taste-masked coating designed to avoid the bitter taste of sodium phenylbutyrate in the mouth while quickly dissolving in the low pH of the stomach. Acer is also being developed for MSUD and has been granted orphan drug designation by the FDA for this indication. ACER-001 is an investigational product candidate which has not been approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority.

About Acer Therapeutics Inc.

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

About RELIEF THERAPEUTICS Holding SA

Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. Relief’s lead drug candidate RLF-100™ (aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the U.S. for the treatment of respiratory deficiency due to COVID-19. As part of its pipeline diversification strategy, in March 2021, Relief entered into a Collaboration and License Agreement with Acer Therapeutics for development and commercialization of ACER-001. ACER-001 is a taste-masked and immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment of Urea Cycle Disorders and Maple Syrup Urine Disease. In addition, Relief’s recently completed acquisitions of APR Applied Pharma Research SA and AdVita Lifescience GmbH, bring to Relief a diverse pipeline of marketed and development-stage programs, a commercial infrastructure in selected European countries and an internal, R&D capability, which Relief hopes to leverage for both internal pipeline products as well as for third party product development on a fee for service basis.

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 symbol RLFTF. For more information, visit www.relieftherapeutics.com. Follow Relief on LinkedIn.

References

1. Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
2. Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
3. Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.

Acer Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

Relief Forward-Looking Statements

This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding SA and its businesses.  Such statements involve certain known and unknown risks, uncertainties and other factors, including (i) whether the FDA will approve Acer’s NDA for ACER-001, (ii) whether Relief will be able to submit an application for approval of ACER-001 in Europe in Q2/Q3 2022 (or at all), (iii) whether any such application submitted to European authorities seeking marketing authorization for ACER-001 for the treatment of patient in Europe with UCDs will be approved, (iv) whether RLF-100 (aviptadil) will receive emergency use authorization in the United States, (v) whether RLF-100 (aviptadil) will ever be submitted for authorization in Europe, (vi) whether Relief’s ongoing disputes with its U.S. collaboration partner for RLF-100 (aviptadil) can be resolved amicably, and (vii) those other risks, uncertainties and factors described in Relief’s annual and periodic filings with the SIX Stock Exchange, all of which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding SA to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding SA is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise.

CORPORATE CONTACTS
Acer Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
[email protected]
+1 844-902-6100

RELIEF Therapeutics Holding SA:
Jack Weinstein
Chief Financial Officer and Treasurer
[email protected]

INVESTOR RELATIONS CONTACTS
Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
[email protected]
+1 617-430-7578

Relief Therapeutics Holding SA:
Michael Miller
Rx Communications Group
[email protected]
+1-917-633-6086

#  #  #

NEWTON, MA –August 30, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced that Acer’s management team will present and host one-on-one investor meetings at the upcoming H.C. Wainwright Global Investment Conference September 13-15, 2021.

Conference: H.C. Wainwright 23rd Annual Global Investment Conference
Format: Virtual on demand presentation and one-on-one meetings
Presentation: Available on demand beginning September 13, 2021 at 7 AM ET                
Webcast: https://www.acertx.com/investor-relations/events-presentations/

About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced vasomotor symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]

Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]

#  #  #

NEWTON, MA –August 10, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today reported financial results for the second quarter ended June 30, 2021 and provided an update on the Company’s recent corporate developments.

“This quarter marks significant, meaningful progress across our entire pipeline, including the very recent submission of our NDA for ACER-001 for the treatment of UCDs,” said Chris Schelling, CEO and Founder of Acer. “As we anticipate working closely with the FDA on the review process for ACER-001, we continue to make significant progress across the rest of our pipeline, including preparation of two IND submissions targeted for Q4 of this year. These INDs are expected to support initiating a dose-ranging Phase 2a study for ACER-801 (osanetant) for the treatment of vasomotor symptoms, and a pivotal Phase 3 study for EDSIVO™ for the treatment of COL3A1+ vEDS. On the corporate front, we have enhanced our management team with four important senior-level hires across their respective disciplines, including program and alliance management, marketing, and clinical development. I warmly welcome our new team members, who bring deep industry knowledge and tremendous experience, and look forward to their contributions as we position ourselves for growth.”

Q2 2021 and Recent Highlights

• ACER-001 (sodium phenylbutyrate)
  • Following completion of a Q2 2021 pre-NDA meeting with the U.S. Food and Drug Administration (FDA), submitted a New Drug Application (NDA) in August 2021 for ACER-001 for the treatment of Urea Cycle Disorders (UCDs)
  • Reached agreement with FDA on the Company’s initial Pediatric Study Plan (iPSP) which outlines an approach that addresses the needs of pediatric patients with UCDs
• ACER-801 (osanetant)
  • Continued to advance toward an Investigational New Drug Application (IND) submission for the treatment of vasomotor symptoms (VMS)
• EDSIVO™ (celiprolol)
  • Following completion of a Type B meeting with FDA, in June 2021 Acer announced plans to conduct a pivotal Phase 3, randomized, double-blind, placebo-controlled, decentralized clinical trial for EDSIVO™ for patients with COL3A1+ vascular Ehlers-Danlos Syndrome (vEDS), subject to available capital
    • The DiSCOVER (Decentralized Study of Celiprolol on vEDS-related Event Reduction) trial is estimated to take approximately 3.5 years to complete, once fully enrolled
    • FDA indicated agreement with Acer’s plans to submit a proposed protocol under the IND Special Protocol Assessment (SPA) process with possible breakthrough designation
  • Launched www.discoverceliprolol.com as an outreach tool for interested parties
• Corporate
  • Hired Tanya Hayden, Vice President of Program and Strategic Alliance Management, in June 2021. During her nearly 20-year tenure at Lonza (formerly Bend Research/Capsugel), Ms. Hayden was responsible for oral drug delivery technologies, clinical and commercial contract manufacturing, operational excellence and client-focused project management
  • Hired John Hilton, Vice President of Marketing and Strategic Insights, in July 2021. Mr. Hilton brings over 20 years of biopharmaceutical industry experience including marketing, sales, KOL development, pipeline marketing, and access strategy functions, as well as oversight of launch planning for ten new products in the last ten years
  • Hired Terrie Kellmeyer, Ph.D., Vice President of Clinical Development, in August 2021. Dr. Kellmeyer brings to Acer over 23 years of experience in the pharmaceutical industry spanning the areas of Clinical Development, Regulatory Affairs, Medical Writing and Medical Affairs
  • Hired Jeffrey Edwards, Ph.D., Vice President of Clinical Sciences, in August 2021. Dr. Edwards brings to Acer over 16 years of pharmaceutical experience in clinical and preclinical studies and support for regulatory submissions
  • Ended Q2 2021 with $22.1 million in cash and cash equivalents. Acer believes its cash and cash equivalents available as of June 30, 2021, plus a $10.0 million Second Development Payment conditioned upon FDA accepting an NDA for ACER-001 in a UCD for filing and review per the Collaboration Agreement with Relief Therapeutics, will be sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for the planned ACER-801 and EDSIVO™ clinical trials

Anticipated Milestones

• ACER-001 (sodium phenylbutyrate)
  • Q4 2021: Based on standard FDA review timelines, Acer expects to receive notification from FDA on the potential acceptance of the NDA for filing within 60 days of submission and subsequent substantive review
  • ACER-001 is an investigational product candidate which has not been approved by FDA. There is no guarantee that this product candidate will be accepted for substantive review, or if accepted, receive regulatory authority approval in any territory, or become commercially available for the indications under investigation
• ACER-801 (osanetant)
  • Q4 2021: IND submission for the treatment of VMS is anticipated in Q4 2021
  • Q1 2022: Phase 2a clinical trial initiation is expected in Q1 2022 designed to evaluate ACER-801’s PK, efficacy and safety in postmenopausal women and to identify the optimal dosing strategy for subsequent efficacy studies in certain cancer patients with induced vasomotor symptoms (iVMS), dependent upon successful IND submission and clearance, and subject to additional capital
• EDSIVO™ (celiprolol)
  • Q4 2021: Submission of the proposed EDSIVO™ pivotal trial protocol and IND, request for breakthrough therapy designation, coordination with FDA on SPA agreement, and beginning to identify COL3A1+ vEDS patients for potential trial participation are anticipated in Q4 2021
  • Q1 2022: Initiation of the EDSIVO™ DiSCOVER pivotal clinical trial is expected in Q1 2022 subject to successful IND submission and clearance, FDA SPA agreement, and obtaining additional capital required to conduct and complete the trial
• ACER-2820 (emetine)
  • Ongoing: With most of the emetine IND-enabling work complete, further advancement of the emetine program for treatment of certain infectious diseases, including COVID-19, is dependent on Acer’s ability to raise non-dilutive capital

Q2 2021 Financial Results

Cash position. Cash and cash equivalents were $22.1 million as of June 30, 2021, compared to $5.8 million as of December 31, 2020. Acer believes its cash and cash equivalents available as of June 30, 2021, plus a $10.0 million Second Development Payment conditioned upon FDA accepting an NDA for ACER-001 in a UCD for filing and review per the Collaboration Agreement with Relief Therapeutics, will be sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for planned ACER-801 and EDSIVO™ clinical trials.

Research and Development Expenses. Research and development expenses were $1.5 million, net of collaboration funding of $0.9 million for the three months ended June 30, 2021, as compared to $2.8 million for the three months ended June 30, 2020. This decrease of $1.3 million was primarily due to the recognition of the $0.9 million of collaboration funding from the Collaboration Agreement with Relief, as well as a decrease in expenses for contract research and contract manufacturing, partially offset by an increase in consulting and professional fees. Research and development expenses for the three months ended June 30, 2021 were comprised of $1.1 million related to ACER-001, offset by $0.9 million of collaboration funding; $0.5 million related to emetine; $0.3 million related to ACER-801; $0.3 million related to EDSIVO^TM; and $0.2 million related to other development activities.

General and Administrative Expenses. General and administrative expenses were $2.3 million, net of collaboration funding of $0.5 million for the three months ended June 30, 2021, as compared to $3.0 million for the three months ended June 30, 2020. This decrease of $0.7 million was primarily due to the recognition of the $0.5 million of collaboration funding from the Collaboration Agreement with Relief, as well as a reduction in one-time costs related to the Lincoln Park agreement recognized in 2020, partially offset by an increase in employee-related expenses.

Net Loss. Net loss for the three months ended June 30, 2021 was $3.2 million, or $0.23 net loss per share (basic and diluted), compared to a net loss of $5.8 million, or $0.56 net loss per share (basic and diluted), for the three months ended June 30, 2020.

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

About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced vasomotor symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]

Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]

NEWTON, MA and GENEVA, SWITZERLAND – August 9, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER) (“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, and Relief Therapeutics Holding AG (SIX: RLF, OTCQB: RLFTF) (“Relief”), a biopharmaceutical company seeking to provide patients therapeutic relief from serious diseases with high unmet need, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ACER-001 (sodium phenylbutyrate) for the treatment of Urea Cycle Disorders (UCDs).  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).

Based on standard FDA review timelines, Acer expects to receive notification from FDA on the potential acceptance of the NDA for filing within 60 days of submission and subsequent substantive review.

The 505(b)(2) NDA submission is supported by results from two previously announced bioequivalence (BE) trials in which ACER-001 showed similar relative bioavailability for both phenylbutyrate (PBA) and phenylacetate (PAA), the active metabolite of sodium phenylbutyrate, compared to BUPHENYL® (sodium phenylbutyrate). Acer has also received an Agreement Letter from FDA in response to the Company’s initial Pediatric Study Plan (iPSP) which outlines an agreed upon approach that addresses the needs of pediatric patients with UCDs.

“The submission of our NDA for ACER-001 marks an important step toward bringing this potential alternative treatment option to patients with UCDs,” said Chris Schelling, Chief Executive Officer and Founder of Acer. “We look forward to working with the FDA through the review process and will continue advancing our preparations for a potential launch of ACER-001, while also assisting Relief toward regulatory submissions in Europe.”

Jack Weinstein, Chief Financial Officer and Treasurer of Relief Therapeutics, added, “We are excited about the progress made to date in support of a potential regulatory approval of ACER-001 for UCDs in the U.S. With the NDA now submitted, we will continue our efforts to back ACER-001’s development in Europe by targeting submission of a Marketing Authorization Application (MAA) for the treatment of UCDs in Europe by the end of 2021.”

ACER-001 is an investigational product candidate which has not been approved by FDA or the European Medicines Agency (EMA). There is no guarantee that this product candidate will be accepted for substantive review, or if accepted, receive regulatory authority approval in any territory, or become commercially available for the indications under investigation.

About UCDs
UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.^1,2   The current treatment of UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.

Current medical treatments for UCDs include nitrogen scavengers, RAVICTI® and BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate (GPB) and sodium phenylbutyrate, respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications have been shown to be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, frequency with which medication must be taken, required number of pills, and the high cost of the medication.³

About ACER-001
ACER-001 (sodium phenylbutyrate) is being developed for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 is a nitrogen-binding agent in development for use as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). The formulation is a multi-particulate dosage formulation for oral administration consisting of a core center, a layer of active drug, and a taste-masked coating designed to avoid the bitter taste in the mouth while quickly dissolving in the low pH of the stomach. ACER-001’s taste-masked formulation is aimed to improve the palatability of sodium phenylbutyrate. Acer is also being developed for Maple Syrup Urine Disease (MSUD) and has been granted orphan drug designation by the FDA for the MSUD indication. ACER-001 is an investigational product candidate which has not been approved by FDA or the European Medicines Agency (EMA).

About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

About RELIEF THERAPEUTICS Holding AG
Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. Relief’s lead drug candidate RLF-100™ (aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the U.S. for the treatment of respiratory deficiency due to COVID-19. As part of its pipeline diversification strategy, in March 2021, Relief entered into a Collaboration and License Agreement with Acer Therapeutics for development and commercialization of ACER-001. ACER-001 is a taste-masked and immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment of Urea Cycle Disorders and Maple Syrup Urine Disease. In addition, Relief’s recently completed acquisition of APR Applied Pharma Research SA brings a diverse pipeline of marketed and development-stage programs.

RELIEF THERAPEUTICS Holding AG is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbol RLFTF. For more information, visit www.relieftherapeutics.com. Follow Relief on LinkedIn.

References

1. Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
2. Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
3. Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.

Acer Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

Relief Forward-Looking Statements
This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding AG and its businesses. The results reported herein may or may not be indicative of the results of future and larger clinical trials for ACER-001 for the treatment of UCDs and MSUD, nor whether the ongoing clinical trials of Relief’s lead compound, RLF-100™ (aviptadil) in advanced clinical development to treat respiratory deficiency due to COVID-19, will be successful. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding AG to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding AG 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.
+1 844-902-6100
[email protected]

RELIEF THERAPEUTICS Holding AG:
Jack Weinstein
Chief Financial Officer and Treasurer
[email protected]

INVESTOR RELATIONS CONTACTS

Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
+1 617-430-7578
[email protected]

Relief Therapeutics:
Michael Miller
Rx Communications Group
+1-917-633-6086
[email protected]

#  #  #

NEWTON, MA –June 10, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced that following a recent Type B meeting with the U.S. Food and Drug Administration (FDA) regarding Acer’s proposed EDSIVO™ (celiprolol) development plan, the company is now planning a pivotal clinical trial in patients with COL3A1+ vascular Ehlers-Danlos Syndrome (vEDS).

In the Type B meeting, Acer discussed conducting a U.S.-based prospective, randomized, double-blind, placebo-controlled, decentralized clinical trial in patients with COL3A1+ vEDS, and sought the Agency’s opinion on various proposed design features of the study. The official meeting minutes Acer recently received from the FDA confirm details of that discussion, including: the acceptability of a decentralized (virtual) clinical trial design and use of an independent centralized adjudication committee; acceptability of a primary endpoint based on clinical events associated with disease outcome; agreement with modest safety data collection (based on the known safety profile of the drug^1,2,3,4) and no pharmacokinetic (PK) data needing to be collected; and a statistical plan that considers the rare disease classification of vEDS.

In the second half of 2021, Acer intends to submit a protocol for the prospective study, along with an investigational new drug application (IND), request breakthrough therapy designation, and coordinate with FDA to reach agreement on a Special Protocol Assessment (SPA). Based on initial power calculations, Acer anticipates that the trial should enroll approximately 200 COL3A1+ vEDS patients in the U.S., and the duration of the clinical trial is estimated to be approximately 3.5 years to complete once fully enrolled.

“We appreciate the guidance and flexibility provided by FDA on the design of this proposed clinical trial,” said Chris Schelling, CEO and Founder of Acer. “This trial, along with the results from the prior BBEST trial¹, should answer whether EDSIVO™ demonstrates substantial evidence of effectiveness by reducing vEDS-related events. Over the next six months, we intend to work closely with FDA to finalize the protocol for the pivotal trial, collaborate with vEDS advocacy groups to identify COL3A1+ patients who may be interested in participating in the trial in order to expedite enrollment, and achieve first patient dosed as quickly as possible. Our progress to date on the EDSIVO™ program is the direct result of our unwavering and ongoing commitment to patients with rare diseases. We look forward to continuing its development for treatment of vEDS patients who desperately need a therapy.”

For more information on the trial as it becomes available, please visit: www.discoverceliprolol.com.

The conduct and completion of the EDSIVO™ pivotal clinical trial is subject to Acer’s ability to obtain up to approximately $10 million of funding. Such funding may come from one or more of a number of current and future sources, including but not limited to current cash, a strategic partnership, other non-dilutive sources and potential ACER-001 revenue. Acer believes its cash and cash equivalents available as of March 31, 2021, plus up to $20.0 million of Development Payments per the ACER-001 Collaboration Agreement with Relief Therapeutics, are sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for planned EDSIVO™ and osanetant clinical trials.

EDSIVO™ is an investigational drug and is not currently FDA approved for any indication. There can be no assurance that the funding to conduct and complete the clinical trial will be obtained, that the clinical trial will be successful, or that the resubmission of the original New Drug Application will be approved.

About Breakthrough Therapy Designation

The FDA’s breakthrough therapy designation is intended to expedite the development and review of new treatments for serious or life-threatening conditions. The designation entitles the company developing a therapy to more intensive FDA guidance on an efficient and accelerated development program, as well as eligibility for other actions to expedite FDA review, such as rolling submission and priority review. To earn breakthrough designation, a treatment must show encouraging early clinical results demonstrating substantial improvement over available therapies with regard to a clinically significant endpoint, or it must fulfill an unmet need.

About vEDS

Ehlers-Danlos syndrome (EDS) is an inherited disorder caused by mutations in the genes responsible for the structure, production, or processing of collagen, an important component of the connective tissues in the human body, or proteins that interact with collagen. EDS is a spectrum disorder where patients present with various forms, the most serious of which is vEDS, also known as EDS type IV, which is generally caused by a mutation in the COL3A1 gene. vEDS causes abnormal fragility in blood vessels, which can give rise to aneurysms, abnormal connections between blood vessels known as arteriovenous fistulas, arterial dissections, and spontaneous vascular ruptures, all of which can be potentially life-threatening. Gastrointestinal and uterine fragility or rupture also commonly occur in vEDS patients. Spontaneous arterial rupture has a peak incidence in the third or fourth decade of life in vEDS patients but may occur earlier and is the most common cause of sudden death in vEDS patients. Arterial rupture or dissection events occur in about 25% of patients before the age of 20 but increase to roughly 90% of patients by the age of 40. The median survival age of vEDS patients in the U.S. is 51 years, with arterial rupture being the most common cause of sudden death.⁵

About EDSIVO™ (celiprolol)

Acer is developing EDSIVO™, a new chemical entity (NCE), for the treatment of COL3A1+ vEDS patients. Acer’s EDSIVO™ NDA was originally submitted based on data obtained from the BBEST trial¹ and accepted for filing in October 2018 with priority review. Following FDA review, Acer received a Complete Response Letter (CRL) stating that it will be necessary to conduct an adequate and well-controlled trial to determine whether celiprolol reduces the risk of clinical events in patients with vEDS. Acer subsequently appealed the FDA decision. While FDA denied the appeal, it described possible paths forward for Acer to explore. Following its Type B meeting with FDA in the second quarter of 2021, Acer has decided to conduct a clinical trial for EDSIVO™. There can be no assurance that the resulting data from the trial would be adequate to support approval of an NDA, although having a SPA agreement in place would indicate concurrence by FDA with the adequacy and acceptability of specific critical elements of overall protocol design (e.g., conduct, entry criteria, dose selection, endpoints, and planned analyses) for a study intended to support a future marketing application.

About Acer Therapeutics Inc.

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

References

1. 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.
2. Nawarskas J, et al. Cardiology in Review 2017;25: 247–253.
3. Frank M, et al. Vascular Ehlers-Danlos Syndrome: Long-Term Observational Study. J Am Coll Cardiol. 2019 Apr, 73 (15) 1948–1957.
4. Baderkhan, H, et al. Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome. European Journal of Vascular and Endovascular Surgery. November 20, 2020.
5. 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.

Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines for clinical study enrollment or regulatory actions, or otherwise, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions for EDSIVO™ or our other products; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, the substantial costs and diversion of management’s attention and resources which could result from pending securities litigation, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov .

Investor Contacts:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]

Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100                                                                                                                                          
[email protected]

#  #  #

NEWTON, MA and GENEVA, SWITZERLAND –May 25, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER)(“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, and Relief Therapeutics Holding AG (SIX: RLF, OTCQB: RLFTF)(“Relief”), a biopharmaceutical company with its lead compound RLF-100^TM (aviptadil) in advanced clinical development to treat severe COVID-19 patients, today announced the outcome of Acer’s pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) for ACER-001 for the treatment of Urea Cycle Disorders (UCDs). ACER-001 is a proprietary immediate release multi-particulate powder formulation of sodium phenylbutyrate (NaPB) with a taste-masked coating. ACER-001 is being developed in collaboration with Relief.

The purpose of the pre-NDA meeting was to discuss the content of Acer’s planned NDA submission. Based on FDA feedback, the Companies believe the proposed data package will be sufficient to support an NDA submission under the Section 505(b)(2) regulatory pathway of ACER-001 for the treatment of patients with UCDs. Following NDA submission and FDA determination of acceptance for filing, the FDA will conduct a substantive review before deciding upon the action on the application.

“We are pleased with the outcome of our recent pre-NDA meeting with FDA, supporting our belief that the studies and data we intend to include in our planned NDA for ACER-001 should be sufficient for NDA submission,” said Chris Schelling, CEO and Founder of Acer. “We remain on track to complete the NDA submission in Q3 2021, provided that we obtain agreement with the FDA on our initial pediatric study plan (iPSP).”

Jack Weinstein, CFO and Treasurer of Relief Therapeutics, added: “The outcome of the pre-NDA meeting with the FDA is highly encouraging and marks an important milestone in support of the development and potential commercialization of ACER-001 worldwide. We are pleased with the progress of the ACER-001 program and look forward to working with the Acer team to potentially bring to market an alternative to existing NaPB-based treatments.”

ACER-001 is an investigational product candidate which has not been approved by FDA. There can be no assurance that if submitted, an NDA will be accepted for filing and substantive review or, if filed, that an NDA would be approved.

About UCDs
UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.^1,2 The current treatment of UCDs consists of dietary management to limit ammonia production in conjunction with medications that provide alternative pathways for the removal of ammonia from the bloodstream. Some patients may also require individual branched-chain amino acid supplementation.

Current medical treatments for UCDs include nitrogen scavengers, RAVICTI® and BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate (GPB) and sodium phenylbutyrate (NaPB), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications have been shown to be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons referenced for non-compliance associated with some available medications include unpleasant taste, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.³

About ACER-001
ACER-001 is being developed for the treatment of various inborn errors of metabolism, including UCDs and MSUD. ACER-001 is a proprietary immediate release powder formulation of sodium phenylbutyrate (NaPB). The formulation is a multi-particulate dosage formulation for oral administration consisting of a core center, a layer of active drug, and a taste-masked coating designed to avoid the bitter taste in the mouth while quickly dissolving in the low pH of the stomach. ACER-001’s taste-masked formulation is aimed to improve the palatability of NaPB. Acer has been granted orphan drug designation by the FDA for the MSUD indication. ACER-001 is under clinical investigation and its safety and efficacy have not been established. There is no guarantee that this product candidate will receive U.S. FDA approval or become commercially available for the indications under investigation.

About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for the worldwide development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

About RELIEF THERAPEUTICS Holding AG
Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing and use in human patients or a strong scientific rationale. Relief’s lead drug candidate RLF-100™ (aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the U.S. for the treatment of respiratory deficiency due to COVID-19. As part of its pipeline diversification strategy, in March 2021, Relief entered into a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001. ACER-001 is a taste-masked and immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment of Urea Cycle Disorders and Maple Syrup Urine Disease.

RELIEF THERAPEUTICS Holding AG is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbol RLFTF. For more information, visit

www.relieftherapeutics.com. Follow Relief on LinkedIn.

References

1. Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
2. Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
3. Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.

Acer Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, the substantial costs and diversion of management’s attention and resources which could result from pending securities litigation, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

Relief Forward-Looking Statements
This communication expressly or implicitly contains certain forward-looking statements concerning RELIEF THERAPEUTICS Holding AG and its businesses. The results reported herein may or may not be indicative of the results of future and larger clinical trials for ACER-001 for the treatment of UCDs and MSUD, nor whether the ongoing clinical trials of Relief’s lead compound, RLF-100™ (aviptadil) in advanced clinical development to treat respiratory deficiency due to COVID-19, will be successful. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of RELIEF THERAPEUTICS Holding AG to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. RELIEF THERAPEUTICS Holding AG 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.
+1 844-902-6100
[email protected]

RELIEF THERAPEUTICS Holding AG:
Jack Weinstein
Chief Financial Officer and Treasurer
[email protected]

MEDIA CONTACTS

Relief (Europe):
Anne Hennecke
MC Services AG
[email protected]
+49 (0) 211-529-252-22

INVESTOR RELATIONS CONTACTS

Acer Therapeutics:
Hans Vitzthum
LifeSci Advisors
+1 617-430-7578
[email protected]

Relief (Europe):
Anne Hennecke
MC Services AG
[email protected]
+49 (0) 211-529-252-22

#  #  #

NEWTON, MA –May 17, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today reported financial results for the first quarter ended March 31, 2021 and provided an update on the Company’s recent corporate developments.

“We made significant progress in advancing our pipeline programs and strengthening our balance sheet in Q1 2021,” said Chris Schelling, CEO and Founder of Acer. “Regarding ACER-001, we executed the Collaboration and License Agreement with Relief Therapeutics; we conducted a pre-NDA meeting with the FDA and are awaiting FDA meeting minutes; and we remain on track to submit an NDA in mid-2021 for ACER-001 as a treatment for UCDs, provided that no additional data are requested by the FDA and we reach agreement on the Pediatric Study Plan (PSP). In addition, we conducted a Type B meeting with the FDA to discuss our proposed path forward for EDSIVO™, and our dialogue is ongoing. We continue to prepare and target an IND submission for ACER-801 (osanetant) in late Q3 2021. Finally, we continue to seek non-dilutive capital to advance development of ACER-2820 (emetine).”

Q1 2021 and Recent Highlights

• ACER-001
  • Announced in February 2021 topline results from Acer’s bioequivalence trial in which ACER-001 showed similar relative bioavailability compared to BUPHENYL® (sodium phenylbutyrate) under fed conditions
  • Announced in March 2021 that Acer and Relief Therapeutics Holding AG (Relief) entered into a Collaboration and License Agreement (Collaboration Agreement) for worldwide development and commercialization of ACER-001. Under the terms of the agreement, Relief has paid Acer $20 million, and will pay Acer up to an additional $21 million in development expense reimbursement and milestones plus royalties in exchange for net profit share and territory rights
  • Conducted a Type B pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) – awaiting FDA meeting minutes
• EDSIVO™ (celiprolol)
  • Conducted a Type B meeting with the FDA to discuss Acer’s proposed plan to collect additional data with the goal of providing further evidence in support of celiprolol’s potential benefit in treating COL3A1-positive vEDS patients – awaiting FDA meeting minutes
• ACER-801 (osanetant)
  • Continued to advance development activities in support of an Investigational New Drug Application (IND) submission
• Corporate
  • Hired Bill DeVincenzi in March 2021 as Vice President, Quality. Mr. DeVincenzi brings to Acer 29 years of experience in quality assurance, supporting the development and commercialization of several small-molecule pharmaceuticals across a wide spectrum of dosage forms
  • Ended Q1 2021 with $15.9 million in cash and cash equivalents. Acer believes its cash and cash equivalents available as of March 31, 2021, plus up to $20.0 million of Development Payments per the Collaboration Agreement with Relief, are sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for a planned osanetant clinical trial

Anticipated Milestones

• ACER-001
  • Q2 2021: Awaiting minutes from the Type B pre-NDA meeting with the FDA
  • Mid-2021: ACER-001 NDA submission for treatment of patients with Urea Cycle Disorders (UCDs) is anticipated in mid-2021, provided that no additional data are requested by the FDA and ongoing development activities are successfully completed (including evaluation of product stability data and reaching agreement on the Pediatric Study Plan (PSP))
• EDSIVO™
  • Q2 2021: Awaiting minutes from the Type B meeting with the FDA
• ACER-801 (osanetant)
  • Q3 2021: IND submission for osanetant is anticipated in late Q3 2021
  • Q4 2021: Clinical trial initiation is expected in late Q4 2021 designed to evaluate osanetant’s PK/PD and safety and to identify the optimal dosing strategy for future efficacy studies in patients with induced Vasomotor Symptoms (iVMS), dependent upon successful IND submission and clearance, and subject to additional capital
• ACER-2820 (emetine)
  • Ongoing: The Company believes that most of the emetine IND-enabling work is complete. Further advancement of the emetine program for treatment of certain infectious diseases, including COVID-19, is dependent on Acer’s ability to raise non-dilutive capital

Q1 2021 Financial Results

Cash position. Cash and cash equivalents were $15.9 million as of March 31, 2021, compared to $5.8 million as of December 31, 2020. Acer believes its cash and cash equivalents available as of March 31, 2021, plus up to $20.0 million of Development Payments per the Collaboration Agreement with Relief, are sufficient to fund its currently anticipated operating and capital requirements into mid-2022, excluding support for a planned osanetant clinical trial.

Revenue. Acer recognized revenue of $4.0 million during the three months ended March 31, 2021, related to the license of intellectual property as part of the Collaboration Agreement signed during the quarter.

Research and Development Expenses. Research and development expenses were $2.0 million for the three months ended March 31, 2021, as compared to $2.3 million for the three months ended March 31, 2020. An increase in employee-related expenses, which was driven by bonuses expensed during the quarter, was offset by a decrease in expenses for contract research and contract manufacturing, as well as an offset of $0.3 million associated with the recognition of collaboration funding from the Collaboration Agreement with Relief. Research and development expenses for the three months ended March 31, 2021 were comprised of $1.2 million related to ACER-001 offset by $0.3 million of collaboration funding, $0.5 million related to osanetant, $0.4 million related to emetine, and $0.2 million related to other development activities.

General and Administrative Expenses. General and administrative expenses were $3.5 million for the three months ended March 31, 2021, as compared to $2.6 million for the three months ended March 31, 2020. This increase of $0.9 million was primarily due to an increase in employee-related expenses, driven by bonuses expensed during the quarter, partially offset by a decrease in expenses related to precommercial activities.

Net Loss. Net loss for the three months ended March 31, 2021 was $1.5 million, or $0.11 net loss per share (basic and diluted), compared to a net loss of $4.9 million, or $0.49 net loss per share (basic and diluted), for the three months ended March 31, 2020.

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

About Acer Therapeutics Inc.

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for worldwide development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, the substantial costs and diversion of management’s attention and resources which could result from pending securities litigation, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You may access these documents for no charge at http://www.sec.gov.

Investor Contact:

Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]

Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]

NEWTON, MA –March 29, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced that Acer’s management team will present and host one-on-one investor meetings at the upcoming 20^th Annual Needham Virtual Healthcare Conference April 12-15, 2021.

Conference: 20^th Annual Needham Virtual Healthcare Conference
Format: Virtual presentation and one-on-one meetings
Presentation:  April 15, 2021             
Time: 11 a.m. Eastern Time
Webcast: https://www.acertx.com/investor-relations/events-presentations/

About Acer Therapeutics Inc.
Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four programs: ACER-001 (sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the U.S. FDA. On March 19, 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for worldwide development and commercialization of ACER-001. For more information, visit www.www.acertx.com.

Investor Contact:
Hans Vitzthum
LifeSci Advisors
Ph: 617-430-7578
[email protected]

Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100
[email protected]