Category: Uncategorized

NEWTON, MA – August 13, 2020 – 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, 2020, and provided an update on the Company’s recent corporate developments.

“The second quarter of 2020 was marked by significant progress in two of our lead programs – emetine and ACER-001,” said Chris Schelling, CEO and Founder of Acer. “The need for a therapeutic to treat confirmed COVID-19 patients before they progress and become hospitalized remains a global priority. The team has been working diligently for the past several months to advance emetine from a concept to a pre-IND opportunity and we look forward to the clinical evaluation of emetine, subject to the availability of the required capital. Regarding ACER-001, we reported encouraging new data showing that when ACER-001 is administered in a fasted state it may provide better disease management over currently approved therapies for UCDs that require administration with food. To that end, we submitted a Type C briefing package to the FDA to discuss these results and are expecting feedback later in the third quarter. We continue to target an ACER-001 NDA filing in the first half of 2021, subject to additional capital.”

Second Quarter 2020 and Recent Highlights

• Emetine
  • Announced a research collaboration agreement with the National Center for Advancing Translational Sciences (NCATS), one of the National Institutes of Health (NIH), to develop emetine hydrochloride as a potential treatment for COVID-19, the disease caused by infection with the SARS-CoV-2 coronavirus
  • Working with federal agencies and private research organizations in an effort to secure non-dilutive funding in support of emetine development
  • Obtained multiple rounds of pre-Investigational New Drug (IND) feedback from the Division of Antivirals (DAV) at the U.S. Food and Drug Administration (FDA)
  • Secured active pharmaceutical ingredient (API) from extract for initiation of the planned Phase 2/3 trial evaluating emetine in high-risk COVID-19 outpatients, assuming the availability of capital. In parallel, Acer successfully established a fully synthetic development pathway for API exclusively in the U.S.
• ACER-001
  • Announced results from a food effect study in healthy volunteers showing that administration of ACER-001 in fasted state increased systemic exposure of phenylbutyrate (PBA) levels compared to fed state, which according to in silico modeling may provide improved disease management in patients with urea cycle disorders (UCDs) when compared to currently approved treatments requiring administration with food
  • Submitted a Type C briefing package to the FDA
• EDSIVO™
  • Continued to assess possible paths forward that could provide the confirmatory evidence needed to demonstrate the substantial evidence of effectiveness needed to support a potential resubmission of the EDSIVO™ NDA
• Osanetant
  • Advanced API manufacturing and other steps to support IND submission
• Corporate
  • Hired Stacey Bain, Ph.D. as Vice President, Clinical Operations, who brings 22 years of international clinical operational and drug development experience in pharmaceutical, biotechnology, and clinical research organization settings
  • Ended the second quarter with $5.9 million in cash and cash equivalents. Acer believes its cash position at the end of the second quarter, combined with an additional $4.7 million of net proceeds subsequently received from sales of common stock from the ATM facility and the insider private placement of common stock, will be sufficient to fund its current operations into the first quarter of 2021, excluding support for the planned emetine Phase 2/3 clinical trial

Upcoming Milestones

• Emetine
  • Seek to obtain potential non-dilutive funding
  • Targeting in the first half of 2021 an IND submission and clearance, followed by potential initiation of the planned Phase 2/3 trial evaluating emetine in high-risk COVID-19 outpatients, subject to ongoing discussions with the FDA and additional capital
• ACER-001
  • Anticipate submitting NDA for urea cycle disorders (UCDs) in the first half of 2021, subject to additional capital, and assuming successful completion of nonclinical work and evaluation of long-term product stability data
• EDSIVO™
  • Expect to request a meeting with FDA by the end of fourth quarter of 2020 to discuss Acer’s proposed plan to provide sufficient confirmatory evidence and, if successful, potentially satisfy the substantial evidence of effectiveness needed to support a possible resubmission of the EDSIVO™ NDA. Neither EDSIVO™ NDA resubmission nor approval is assured
• Osanetant
  • Anticipate submitting osanetant IND in the first quarter of 2021
  • Targeting initiation of Phase 1/2 pharmacokinetic/pharmocodynamic and safety trial in the first half of 2021, subject to additional capital, that will evaluate osanetant in patients with medically and/or surgically induced vasomotor symptoms (iVMS)

Financial Results for the Second Quarter 2020

Cash position. Cash and cash equivalents were $5.9 million as of June 30, 2020, compared to $12.1 million as of December 31, 2019. Acer believes its cash position at the end of the second quarter, combined with an additional $4.7 million of net proceeds subsequently received from sales of common stock under the ATM facility and the insider private placement of common stock, will be sufficient to fund its current operations into the first quarter of 2021, excluding support for the planned emetine Phase 2/3 clinical trial.

Research and Development Expenses. Research and development expenses were $2.8 million for the three months ended June 30, 2020, compared to $4.2 million for the three months ended June 30, 2019. This decrease of $1.4 million was primarily due to decreases in employee-related expenses and clinical and regulatory consulting as a direct result of the Complete Response Letter for EDSIVO™ received from the FDA in June 2019, partially offset by increases in contract research and contract manufacturing expenses. Research and development expenses for the three months ended June 30, 2020 were primarily comprised of $1.0 million related to emetine, $0.9 million related to ACER-001, and $0.8 million related to osanetant.

General and Administrative Expenses. General and administrative expenses were $3.0 million for the three months ended June 30, 2020, compared to $6.9 million for the three months ended June 30, 2019. This decrease of $3.9 million was primarily due to decreases in precommercial and employee-related expenses as a direct result of the Complete Response Letter for EDSIVO™ received from the FDA in June 2019, partially offset by costs related to implementing Acer’s equity line purchase agreement with Lincoln Park Capital Fund, LLC.

Net Loss. Net loss for the three months ended June 30, 2020 was $5.8 million, or $0.56 net loss per share (basic and diluted), compared to a net loss of $11.0 million, or $1.09 net loss per share (basic and diluted), for the three months ended June 30, 2019.

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 clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; ACER-001 (a taste-masked, immediate release formulation of 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; and osanetant for the treatment of induced Vasomotor Symptoms (iVMS). Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.www.acertx.com.

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 the emetine program; the adequacy of our capital to support our future operations and our ability to successfully 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 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 – July 8, 2020 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced data from a food effect study in healthy volunteers showing that administration of ACER-001 in a fasted state increased systemic exposure of phenylbutyrate (PBA), phenylacetate (PAA) and phenylacetylglutamine (PAGN) levels compared to fed state, and therefore based on modeling data may improve disease management in patients with urea cycle disorders (UCDs) when compared to currently approved treatments requiring administration with food.

Results from Part B of the ACER-001 bioequivalence (BE) trial in healthy volunteers (n=36) announced in February 2020 showed that ACER-001 was bioequivalent to BUPHENYL® (sodium phenylbutyrate) and were within the parameters recommended by the FDA’s Guidance for Industry, “Statistical Approaches to Establishing Bioequivalence.” The BE trial included a food effect study, which evaluated the pharmacokinetics (PK) of sodium phenylbutyrate (NaPBA) showing that administration of ACER-001 in a fasted state achieved more than two times the maximum concentration (C_max) of PBA compared to administration of the same dose of ACER-001 in a fed state. These results are consistent with previously published data by Nakano, et al¹ that evaluated PK of NaPBA in patients with progressive familial intrahepatic cholestasis, also demonstrating that administration of NaPBA in a fasted state significantly increased PBA peak plasma concentration compared to administration of NaPBA in a fed state.

Currently approved therapies for UCDs, including BUPHENYL®² and RAVICTI®³ (glycerol phenylbutyrate), are required to be administered with food. BUPHENYL® is required to be administered in a fed state due to its aversive odor and taste, with side effects including nausea, vomiting and headaches, which often lead to discontinuation of treatment.⁴ Additionally, prescribing information states that BUPHENYL® food effect is unknown. RAVICTI® PK and pharmacodynamic (PD) properties were determined to be indistinguishable in fed or fasted states.⁵ ACER-001 is uniquely formulated with its multi-particulate, taste-masked coating to allow for administration in a fasted state, while still allowing for rapid systemic release.

Based on the results from the food effect study within the ACER-001 BE trial, Acer commissioned Rosa & Co. LLC to create a PhysioPD® PK model to evaluate the potential food effect on exposure, tolerability and efficacy of ACER-001 in UCDs patients. Results from this in silico model suggest that administration of ACER-001 in a fasted state required approximately 30% less PBA to achieve comparable therapeutic benefit in a fed state. In addition, the model predicted that administration of ACER-001 in a fasted state compared to administration of BUPHENYL® or RAVICTI® (same amounts of PBA) in their required fed states is expected to result in higher peak blood PBA, PAA and PAGN concentrations, predicting a 43% increase in urinary PAGN levels (a negative correlation between blood ammonia area under the curve and 24-hour urinary PAGN amount has been demonstrated⁶).

“For nearly a quarter century, phenylbutyrate has been prescribed to UCD patients with food while its effect on phenylbutyrate absorption was never determined. The results of the ACER-001 food effect study, published literature and in silico modeling suggest that ACER-001 administered in a fasted state, and likely just 10 minutes prior to meals, could offer UCD patients a safe and better disease management option compared to currently approved products that are required to be taken with food,” said Chris Schelling, CEO and Founder of Acer. “We formulated ACER-001 to specifically improve palatability and tolerability, and we expect that this formulation should allow ACER-001 to be successfully administered without food. We look forward to discussing these findings with the FDA later in the third quarter.” Schelling continued “Interestingly, the increased exposure seen under fasted conditions may have benefit in other patient populations we intend to study, such as Maple Syrup Urine Disease (MSUD), where the C_max of phenylbutyrate is the active moiety.”

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.^7,8

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 (GPBA) and sodium phenylbutyrate (NaPBA), respectively. According to a 2016 study by Shchelochkov et al., published in Molecular Genetics and Metabolism Reports, while nitrogen scavenging medications can be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons given for non-compliance include the unpleasant taste associated with available medications, the frequency with which medication must be taken, the number of pills, and the high cost of the medication.⁹

About ACER-001

ACER-001 is a taste-masked, immediate-release proprietary formulation of sodium phenylbutyrate developed by Acer using a microencapsulation process. ACER-001 is being developed for the treatment of various inborn errors of metabolism, including UCDs and Maple Syrup Urine Disease (MSUD). ACER-001 microparticles consist of a core center, a layer of active drug, and a taste-masking coating that quickly dissolves in the stomach, allowing taste to be neutralized while still allowing for rapid systemic release. This taste-masked formulation may result in better patient tolerability allowing for administration in a fasted state, and likely prior to a meal. 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 will receive FDA approval or become commercially available for the uses being investigated.

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 clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.www.acertx.com.

References

1. Nakano S, et al. Effect of food on the pharmacokinetics and therapeutic efficacy of 4-phenylbutyrate in progressive familial intrahepatic cholestasis. Sci Rep 9, 17075 (2019).
2. https://www.hzndocs.com/BUPHENYL-Prescribing-Information.pdf
3. https://www.hzndocs.com/RAVICTI-Prescribing-Information.PDF
4. Pena-Qintana L, et al. Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives. Patient Preference and Adherence Volume 11:1489-1496, September 2017.
5. United States Patent number US8642012B2.
6. Lee et al. Phase 2 Comparison of A Novel Ammonia Scavenging Agent With Sodium Phenylbutyrate In Patients With Urea Cycle Disorders: Safety, Pharmacokinetics And Ammonia Control. Mol Genet Metab. 2010 July; 100(3): 221–228.
7. Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University of Washington, Seattle; 1993.
8. Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
9. 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.

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 the ACER-001 program; the adequacy of our capital to support our future operations and our ability to successfully 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 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 – May 14, 2020 – 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, 2020 and provided an update on the Company’s recent corporate developments.

“Over the last several months, we made significant progress in the expansion and further diversification of our product pipeline with the recent addition of emetine, a broad-acting and potent antiviral for the treatment of COVID-19 patients,” said Chris Schelling, CEO and Founder of Acer. “We are very pleased to be collaborating with NCATS on the development of emetine and look forward to advancing this important program. We continued to advance our ACER-001 program for UCDs following the successful completion of our pivotal bioequivalence trial and are targeting NDA submission in early 2021, subject to additional capital. In addition, we are preparing to submit an IND for osanetant by the end of the year, and we intend to initiate a Phase 1/2 trial in patients with induced vasomotor symptoms in the first quarter of next year, subject to additional capital. To that end, we entered into a stock purchase agreement with Lincoln Park Capital.”

First Quarter 2020 and Recent Highlights

• Emetine
  • Announced a research collaboration agreement with the National Center for Advancing Translational Sciences (NCATS), one of the National Institutes of Health (NIH), to develop emetine hydrochloride as a potential treatment for COVID-19, the disease caused by infection with the SARS-CoV-2 coronavirus
  • In discussion with the Division of Antivirals (DAV) at FDA after receiving its written responses to the Company’s pre-Investigational New Drug (pre-IND) package
  • Presented at the Biomedical Advanced Research and Development Authority (BARDA) CoronaWatch meeting on May 7, 2020. BARDA CoronaWatch is a funding program providing government support for selected coronavirus projects
• ACER-001
  • Initiated nonclinical work and evaluation of long-term product stability following the successful completion of the pivotal trial showing that ACER-001 is bioequivalent to BUPHENYL® (sodium phenylbutyrate)
• EDSIVO™
  • Received appeal response from the FDA’s Office of New Drugs (OND) stating it denied Acer’s appeal of the Complete Response Letter (CRL) in relation to the New Drug Application (NDA) for EDSIVO™. Acer is assessing possible paths forward, as described in the OND’s response, that could provide the substantial evidence of effectiveness needed to support a potential resubmission of the EDSIVO™ NDA
• Ended the first quarter with $7.0 million in cash and cash equivalents. Acer believes its cash position will be sufficient to fund its current operations into the fourth quarter of 2020, excluding support for a planned emetine clinical trial and for EDSIVO™ development and precommercial activities
• Announced a common stock purchase agreement to potentially sell up to $15 million worth of shares to Lincoln Park Capital Fund, LLC over a 36-month period, following the filing and effectiveness of a registration statement and subject to various limitations including those under the Nasdaq listing rules

Upcoming Milestones

• Emetine
  • Pursuing several potential financing options, including federally-funded research and grants, to support emetine development
  • Working toward an IND submission in mid-2020
  • Targeting initiation in the third quarter of 2020, subject to additional capital, an adaptive design Phase 2/3 randomized, blinded, placebo-controlled multi-center trial in high-risk, symptomatic adult COVID-19 patients not requiring hospitalization
• ACER-001
  • Anticipate submitting NDA for urea cycle disorders (UCDs) in the first quarter of 2021, subject to additional capital, and assuming successful completion of nonclinical work and evaluation of long-term product stability data
• Osanetant
  • Anticipate submitting osanetant IND in the fourth quarter of 2020
  • Targeting initiation of Phase 1/2 pharmacokinetic/pharmocodynamic and safety trial in the first quarter of 2021, subject to additional capital, evaluating osanetant in patients with medically and/or surgically induced vasomotor symptoms (iVMS) in which Hormone Replacement Therapy (HRT) is contraindicated
• EDSIVO™
  • Continuing to evaluate possible next steps with the goal of resubmission of the EDSIVO™ NDA. Neither resubmission nor EDSIVO™ approval is assured

Financial Results for the First Quarter 2020

Cash position. Cash and cash equivalents were $7.0 million as of March 31, 2020, compared to $12.1 million as of December 31, 2019. Acer believes its cash position will be sufficient to fund its current operations into the fourth quarter of 2020, excluding support for a planned emetine clinical trial and for EDSIVO™ development and precommercial activities.

Research and Development Expenses. Research and development expenses were $2.3 million for the three months ended March 31, 2020, compared to $3.9 million for the three months ended March 31, 2019. This decrease of approximately $1.6 million was primarily due to decreases in clinical and regulatory consulting, contract manufacturing, and employee-related expenses as a direct result of the Complete Response Letter for EDSIVO™ received from the FDA in June 2019, partially offset by an increase in contract research expenses. Research and development expenses for the three months ended March 31, 2020 were primarily comprised of approximately $0.5 million related to EDSIVO™ and approximately $1.4 million related to ACER-001.

General and Administrative Expenses. General and administrative expenses were $2.6 million for the three months ended March 31, 2020, compared to $4.2 million for the three months ended March 31, 2019. This decrease of $1.6 million was primarily due to decreases in precommercial and employee-related expenses as a direct result of the Complete Response Letter for EDSIVO™ received from the FDA in June 2019.

Net Loss. Net loss for the three months ended March 31, 2020 was $4.9 million, or $0.49 net loss per share (basic and diluted), compared to a net loss of $8.0 million, or $0.79 net loss per share (basic and diluted), for the three months ended March 31, 2019.

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 clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.www.acertx.com.

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 the emetine program; the adequacy of our capital to support our future operations and our ability to successfully 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 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 – May 11, 2020 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced it has entered into a research collaboration agreement with the National Center for Advancing Translational Sciences (NCATS), one of the National Institutes of Health (NIH), to develop emetine hydrochloride as a potential treatment for patients with COVID-19, the disease caused by infection with the SARS-CoV-2 virus. Under the terms of the agreement, Acer and NCATS will collaborate to accelerate the clinical development of emetine, a broad-acting and potent antiviral according to various preclinical and clinical studies.

Acer is in ongoing discussion with the Division of Antivirals (DAV) at the FDA after receiving its initial written responses to the Company’s pre-Investigational New Drug (pre-IND) package. Acer is working toward an IND submission in mid-2020 and targeting clinical trial initiation in the third quarter of 2020, subject to additional capital. The Company has proposed an adaptive design Phase 2/3 randomized, blinded, placebo-controlled multi-center trial to evaluate the safety and antiviral activity of emetine in high-risk, symptomatic adult patients with confirmed COVID-19 infection not requiring hospitalization. The trial objectives as planned are to determine the safety and efficacy of emetine via clinical status at a specific timepoint in addition to disease resolution.

Acer is concurrently pursuing several financing options, including federally-funded research and grants, to support emetine development. For example, the Biomedical Advanced Research and Development Authority (BARDA) invited the Company to present the emetine development program at the BARDA CoronaWatch meeting on May 7, 2020. BARDA CoronaWatch is a funding program providing government support for selected coronavirus projects. While Acer plans to advance emetine through IND submission, initiation of the clinical trial of emetine is contingent on the timely availability of additional capital to fund this program.

Emetine will be delivered as a sterile subcutaneous injection. Acer will oversee the contract synthesis and manufacturing of emetine for clinical development and potential commercialization.

“We are very pleased to be selected by NCATS and look forward to collaborating on the development of emetine, a broad-acting and potent antiviral identified by NCATS as their best preclinical opportunity for further clinical development for the treatment of COVID-19,” said Chris Schelling, CEO and Founder of Acer. “With a collaboration agreement in place, ongoing discussions with the FDA toward IND submission, and several potentially non-dilutive funding sources being pursued, we believe we are well-positioned to advance the clinical development of emetine.”

Conference Call and Webcast Details

Interested parties can access the live call and webcast on Monday, May 11, 2020, at 5:30 pm Eastern Time (2:30 pm Pacific Time) from the Investors section of Acer’s website or directly at

http://public.viavid.com/index.php?id=139741. Participants can also access the call by dialing 800-458-4148 (US Toll Free) or 1-720-543-0206 (International Toll Number) and providing the Conference ID 8855853. A replay of the call will also be available under the Investors section of Acer’s website.

About Emetine Hydrochloride

Acer and NCATS are working together to develop emetine for the treatment of patients with COVID-19, the disease  caused by the SARS-CoV-2 virus. Emetine is an active pharmaceutical ingredient of syrup of ipecac, given orally to induce emesis, and has also been formulated as an injectable to treat thousands of individuals with amebiasis. Several independent in vitro studies have demonstrated nanomolar potency against both DNA and RNA-replicating viruses, including Zika virus, Ebola virus¹, Rabies Lyssavirus, human cytomegalovirus, human immunodeficiency virus 1, influenza A virus, Rift Valley fever virus, echovirus 1, human metapneumovirus, and herpes simplex virus type 2². Clinically, emetine has been used to treat approximately 700 patients (including pediatrics) with viral hepatitis ³ and varicella-zoster virus⁴. Additionally, emetine is a potent inhibitor of multiple genetically-distinct coronaviruses and demonstrated in vitro the strongest anti-coronavirus activity in one study that screened and identified approved compounds with broad-spectrum efficacy against the replication of four coronaviruses⁵ and specifically against SARS-CoV-2.⁶

Acer intends to initially seek FDA approval to market emetine in the U.S. using a regulatory pathway established under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act that allows applicants to rely at least in part on third party data for approval. The Company intends to rely in part on the existing preclinical and clinical safety data for emetine, while supplementing with the COVID-19 safety and efficacy data to be generated in the Phase 2/3 trial as well as chemistry, manufacturing and controls information. If the Phase 2/3 trial is completed successfully, following IND submission and clearance, Acer anticipates submitting to the FDA the 505(b)(2) NDA for emetine for the treatment of COVID-19. The potential initiation of the Phase 2/3 trial, its conduct and completion and NDA submission are subject to the Company’s ability to generate sufficient capital resources to fund this program. Emetine is an investigational drug for COVID-19 and is not currently FDA approved for any indication.

About Acer Therapeutics Inc.

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes four clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; EDSIVO™ (celiprolol), for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate), for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant, for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.www.acertx.com.

References

1. Yang S, et al. Emetine inhibits Zika and Ebola virus infections through two molecular mechanisms: inhibiting viral replication and decreasing viral entry. Cell Discov (2018) 4:31. doi:10.1038/s41421-018-0034-1
2. Andersen, P.I., et al. Novel Antiviral Activities of Obatoclax, Emetine, Niclosamide, Brequinar, and Homoharringtonine. Viruses 2019, 11, 964
3. Del Puerto et al. Pren. méd. argent., 55: 818, 1968
4. Annamalai et al. Emetine Hydrochloride in the Treatment of Herpes Zoster. 1968
5. Shen L, et al. High-Throughput Screening and Identification of Potent Broad-Spectrum Inhibitors of Coronaviruses. J Virol. 2019 May 29;93(12)
6. Choy et al. Remdesivir, lopinavir, emetine, and homoharringtonine inhibit SARS-CoV-2 replication in vitro. Antiviral Res. 2020 Jun; 178: 104786

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 the emetine program; the adequacy of our capital to support our future operations and our ability to successfully 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 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 – April 30, 2020 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced it has entered into a common stock purchase agreement for up to $15 million with Lincoln Park Capital Fund, LLC (LPC), a Chicago-based institutional investor.

Under the terms of the purchase agreement and following the filing and effectiveness of a registration statement, Acer will have the right at its sole discretion, but not the obligation, to sell to LPC up to $15 million worth of shares over the 36-month term of the agreement, subject to various limitations including those under the Nasdaq listing rules. There are no upper limits to the price per share LPC may pay to purchase the shares, and the purchase price of the shares will be based on the then prevailing market prices at the time of each sale to LPC. Acer controls the timing and amount of any future sales of its stock to LPC. There are no warrants, derivatives, financial or business covenants associated with the agreements, and LPC has agreed not to cause or engage in any direct or indirect short selling or hedging of Acer’s common stock. Acer may terminate the purchase agreement at any time, at its discretion, without any cost or penalty. 

Acer intends to use any net proceeds from the sale of its common stock to LPC for working capital and general corporate purposes. In consideration for LPC entering into the purchase agreement, Acer issued shares of its common stock to LPC as a commitment fee.

Additional information regarding the purchase agreement with LPC is available in the Current Report on Form 8-K that Acer filed today with the Securities and Exchange Commission.

The offer and sale of the securities in the above transaction have not been registered under the Securities Act of 1933, as amended (the “Securities Act”), or any state securities laws and may not be offered or sold in the United States absent registration under the Securities Act and any applicable state securities laws or an applicable exemption from such registration.

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

About Lincoln Park Capital Fund, LLC

LPC is a long-only institutional investor headquartered in Chicago, Illinois. LPC’s experienced professionals manage a portfolio of investments in public and private entities. These investments are in a wide range of companies and industries emphasizing life sciences and technology. LPC’s investments range from multi-year financial commitments to fund growth to special situation financings to long-term strategic capital offering companies’ flexibility and consistency. For more information, please visit www.lpcfunds.com.

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 three clinical-stage candidates: EDSIVO™ (celiprolol), for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate), for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant, for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.www.acertx.com.

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 possible sales of common stock pursuant to the purchase agreement with LPC and our  financing needs are forward-looking statements. 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, including, without limitation, uncertainties related to our working capital, our ability to carry on our existing operations and to obtain needed financing. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors. 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 Current Reports on Form 8-K, 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 18, 2020 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today reported financial results for the fourth quarter and full year ended December 31, 2019 and provided an update on the Company’s recent corporate developments.

“Over the last 12 months, we have made considerable progress in advancing our pipeline of clinical-stage product candidates,” said Chris Schelling, CEO and Founder of Acer. “While the Office of New Drugs recently denied our appeal of the EDSIVO™ Complete Response Letter, we are encouraged by its description of possible paths forward. While neither resubmission nor the prospect of approval is assured, we are evaluating our possible next steps with the goal of resubmission of the EDSIVO™ NDA. Concurrently, we continue to advance our ACER-001 program for Urea Cycle Disorders following the successful completion of our pivotal bioequivalence trial and are working toward a New Drug Application submission in early 2021. In addition, we are preparing to initiate an osanetant Phase 1/2 trial in patients with induced vasomotor symptoms by the end of the year, subject to additional capital.”

Fourth Quarter 2019 and Recent Highlights

• EDSIVO™
  • Today announced the Office of New Drugs (OND) of the U.S. Food and Drug Administration (FDA) denied Acer’s appeal of the Complete Response Letter (CRL) in relation to the New Drug Application (NDA) for EDSIVO™. In its Appeal Denied letter, the OND describes possible paths forward for Acer to explore that could provide the substantial evidence of effectiveness needed to support a potential resubmission of the EDSIVO™ NDA for the treatment of patients with vascular Ehlers-Danlos syndrome (vEDS) with a confirmed COL3A1 mutation

• ACER-001
  • Announced successful completion and final data from Part B of the pivotal trial showing that ACER-001 was bioequivalent to BUPHENYL® (sodium phenylbutyrate)
  • Initiated remaining nonclinical work and evaluation of long-term product stability

• Ended the fourth quarter with $12.1 million in cash and cash equivalents. Acer believes its cash position will be sufficient to fund its current operations through the end of 2020, excluding support for EDSIVO™ development and precommercial activities and the planned osanetant clinical trial

Upcoming Milestones

• EDSIVO™
  • While neither resubmission nor the prospect of approval is assured, evaluate possible next steps with the goal of resubmission of the EDSIVO™ NDA

• ACER-001
  • Submit NDA for UCDs in early 2021, subject to additional capital, and assuming successful completion of nonclinical work and evaluation of long-term product stability data

• Osanetant
  • Submit osanetant IND in 2H 2020
  • Target initiation of Phase 1/2 pharmacokinetic/pharmocodynamic and safety trial by end of 2020, subject to additional capital, evaluating osanetant in patients with medically and/or surgically induced vasomotor symptoms (iVMS) in which Hormone Replacement Therapy (HRT) is contraindicated

Financial Results for the Fourth Quarter and Full Year 2019

Cash position. Cash and cash equivalents were $12.1 million as of December 31, 2019, compared to $41.7 million as of December 31, 2018. Acer believes its cash position will be sufficient to fund its current operations through the end of 2020, excluding support for EDSIVO™ development and precommercial activities and the planned osanetant clinical trial.

Research and Development Expenses. Research and development expenses were $2.8 million for the three months ended December 31, 2019, compared to $5.3 million for the three months ended December 31, 2018. Research and development expenses for the three months ended December 31, 2019 were primarily comprised of approximately $0.3 million related to EDSIVO™ and approximately $2.4 million related to ACER-001. Research and development expenses were $13.9 million for the year ended December 31, 2019, compared to $12.5 million for the year ended December 31, 2018. This increase of approximately $1.4 million was primarily due to increases in spending related to contract manufacturing services, regulatory consulting, and medical affairs services during the first half of 2019 in preparation for the potential launch of EDSIVO^TM, as well as to an increase in employee-related expenses. The increase in employee-related expenses was driven by increased headcount during the first half of 2019, as well as $0.5 million restructuring expense and increased stock-based compensation expense. These increases were partially offset by a decrease in spending related to license fees.

General and Administrative Expenses. General and administrative expenses were $2.4 million for the three months ended December 31, 2019, compared to $3.4 million for the three months ended December 31, 2018. General and administrative expenses were $16.0 million for the year ended December 31, 2019, compared to $9.3 million for the year ended December 31, 2018. This increase of $6.7 million was primarily due to a $4.6 million increase in employee-related expenses, which included $1.0 million restructuring expense, increased headcount and travel during the first half of 2019, and increased stock-based compensation expense. The remaining increase in general and administrative expenses was primarily due to an increase in expenses related to precommercial activities.

Net Loss. Net loss for the three months ended December 31, 2019 was $5.2 million, or $0.51 net loss per share (basic and diluted), compared to a net loss of $8.5 million, or $0.85 net loss per share (basic and diluted), for the three months ended December 31, 2018. Net loss for the year ended December 31, 2019 was $29.4 million, or $2.91 loss per share (basic and diluted), compared to a net loss of $21.3 million, or $2.49 loss per share (basic and diluted), for the year ended December 31, 2018.

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

About Acer Therapeutics Inc.

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes three clinical-stage candidates: EDSIVO™ (celiprolol), for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate), for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant, for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.www.acertx.com.

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; the adequacy of our capital to support our future operations and our ability to successfully 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 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 18, 2020 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced that the Office of New Drugs (OND) of the U.S. Food and Drug Administration (FDA) has denied Acer’s appeal of the Complete Response Letter (CRL) in relation to the New Drug Application (NDA) for EDSIVO™. In its Appeal Denied letter, the OND describes possible paths forward for Acer to explore that could provide the substantial evidence of effectiveness needed to support a potential resubmission of the EDSIVO™ NDA for the treatment of patients with vascular Ehlers-Danlos syndrome (vEDS) with a confirmed COL3A1 mutation.

“We appreciate the OND’s time and attention in thoughtfully considering this FDRR,” said Chris Schelling, CEO and Founder of Acer. “While neither resubmission nor the prospect of approval of the EDSIVO™ NDA is assured, we are evaluating our possible next steps with the goal of resubmission of the EDSIVO™ NDA.”

Acer believes its cash position will be sufficient to fund its current operations through the end of 2020, excluding support for EDSIVO™ development and precommercial activities and the planned osanetant clinical trial.

About vEDS and EDSIVO™ (celiprolol)

Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. vEDS is the most severe subtype where patients suffer from life threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. There is currently no approved treatment option for vEDS. The median age of death is 51 years.¹ An Acer-commissioned patient-finder study phenotypically identified 4,169 vEDS patients in the U.S. from an analysis of a commercially available patient claims database with data of approximately 190 million unique patient lives. Based on that information, Acer estimates the prevalence of phenotypically-defined vEDS in the U.S. could be greater than 1 in 45,000. Currently, there are no FDA-approved therapies for vEDS. Acer is advancing EDSIVO™, a new chemical entity (NCE), for the treatment of vEDS based on published results from a randomized controlled clinical trial of celiprolol.² The FDA granted a priority review of the EDSIVO™ NDA and subsequently issued a CRL in June 2019. The OND denied an Acer appeal of the CRL in March 2020, but made reference to the FDA Guidance document issued in December 2019, where substantial evidence of effectiveness can be provided by two or more adequate and well-controlled studies demonstrating efficacy, or a single positive adequate and well-controlled study plus confirmatory evidence³. EDSIVO™ received FDA Orphan Drug Designation for the treatment of vEDS in 2015.

About Acer Therapeutics Inc.

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes three clinical-stage candidates: EDSIVO™ (celiprolol), for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate), for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant, for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. Each of Acer’s product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA. For more information, visit www.www.acertx.com.

References

1. Pepin M, et al. Clinical and genetic features of Ehlers-Danlos syndrome type IV, the vascular type. N Engl J Med. 2000; 342:673-80
2. Ong KT, et al. Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. Lancet. 2010;376(9751):1476-1484
3. FDA guidance “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products”, December 2019

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; the adequacy of our capital to support our future operations and our ability to successfully 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 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 – Feb. 24, 2020 – 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 successful completion and final data from Part B of its pivotal trial evaluating the bioavailability and bioequivalence of ACER-001 to BUPHENYL® (sodium phenylbutyrate). ACER-001 is a proprietary, taste-masked formulation of sodium phenylbutyrate, in development for the treatment of Urea Cycle Disorders (UCDs).

Part B of the trial evaluated 36 healthy adults in a single-center, single-blind, randomized, single-dose crossover study designed to evaluate bioequivalence of ACER-001 compared to BUPHENYL®. Consistent with observations from Part A of the trial, data from Part B showed ACER-001 to be bioequivalent to BUPHENYL® and were within the parameters recommended by the FDA’s Guidance for Industry, “Statistical Approaches to Establishing Bioequivalence.” Acer is developing ACER-001 under Section 505(b)(2) of the Federal Food, Drug and Cosmetic Act, which provides a potentially streamlined path for sponsors that have developed improvements to drug products previously approved by the FDA.

“We are pleased to see data from the trial showing ACER-001 bioequivalence to BUPHENYL®, marking the successful completion of an important step in the advancement of ACER-001,” said William Andrews, M.D., FACP, Chief Medical Officer of Acer. “These data support our belief that if approved, ACER-001 could represent a cost-effective, taste-masked, immediate-release alternative for patients suffering from UCDs. We plan to submit a New Drug Application in early 2021, subject to additional capital, and assuming successful completion of remaining nonclinical work and 12-month long-term stability data.”

About Section 505(b)(2)

The ACER-001 pivotal trial is designed to make use of the 505(b)(2) alternative pathway to approval established in the Hatch-Waxman Amendments to the Federal Food, Drug, and Cosmetic Act (FFDCA).

Section 505(b)(2) provides an alternative pathway for submission of an NDA, referred to as a 505(b)(2) application, when some or all of the safety and efficacy investigations relied on for approval were not conducted by or for the applicant and for which the applicant has not obtained a right of reference but for which the relevant information is publicly available. The Hatch-Waxman Amendments also provide pharmaceutical products approved under Section 505(b)(2) with potential market exclusivity for three years from FDA approval.

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 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 can be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons given for non-compliance include the unpleasant taste associated with available medications, the frequency with which medication must be taken, the number of pills, and the high cost of the medication³.

About ACER-001

ACER-001 is a taste-masked, immediate-release proprietary formulation of sodium phenylbutyrate developed by Acer using a microencapsulation process. ACER-001 is being developed for the treatment of various inborn errors of metabolism, including UCDs and Maple Syrup Urine Disease (MSUD). ACER-001 microparticles consist of a core center, a layer of active drug, and a taste-masking coating which dissolves in the stomach, allowing taste to be neutralized while still allowing for rapid systemic release. 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 will receive FDA approval or become commercially available for the uses being investigated.

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 three clinical-stage candidates: EDSIVO™ (celiprolol), for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate), for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant, for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. 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.

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.

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 ACER-001 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; the adequacy of our capital to support our future operations and our ability to successfully 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 meet our business objectives and operational requirements, including our ability to raise additional capital, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results or actual outcomes, 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 – Dec. 16, 2019 – 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 full enrollment of Part B  of its pivotal trial evaluating bioavailability and bioequivalence of ACER-001 (sodium phenylbutyrate) for the treatment of Urea Cycle Disorders (UCDs). Part B was initiated following successful completion of Part A and identification of an optimal ACER-001 formulation. Acer is developing ACER-001 under Section 505(b)(2) of the Federal Food, Drug and Cosmetic Act.

Results from Part A of the trial evaluated bioavailability of three different oral suspension formulations of ACER-001 compared to BUPHENYL® (sodium phenylbutyrate) in 20 healthy adult subjects. These data, along with results from a concurrent taste assessment trial evaluating palatability of the three formulations of ACER-001 compared to BUPHENYL®, informed Acer’s selection of the single, optimal formulation of ACER-001 for ongoing evaluation in Part B that will be taken through to potential commericalization. Part B of this trial is a single-center, single-blind, randomized, single-dose crossover study designed to demonstrate bioequivalence of ACER-001 compared to BUPHENYL® in 36 healthy adult subjects.

“Successful completion of Part A and full enrollment of Part B mark important progress in the ongoing development of ACER-001,” said William Andrews, M.D., FACP, Chief Medical Officer of Acer. “Over the next year, we aim to complete Part B of the bioequivalence trial in the first quarter of 2020, enroll and complete Part B of the Taste Assessment study, and per FDA feedback, conduct some additional nonclinical work while monitoring the long-term stability data of the product. Assuming successful outcomes with these activities, we should be on track to proceed with submission of a New Drug Application (NDA) in early 2021.”

About Section 505(b)(2)

The ACER-001 pivotal trial is designed to make use of the 505(b)(2) alternative pathway to approval established in the Hatch-Waxman Amendments to the Federal Food, Drug, and Cosmetic Act (FFDCA).

The Hatch-Waxman Amendments established Section 505(b)(2) of the FFDCA that provides an alternative pathway for submission of an NDA, referred to as a 505(b)(2) application, when some or all of the safety and efficacy investigations relied on for approval were not conducted by or for the applicant and for which the applicant has not obtained a right of reference. The Hatch-Waxman Amendments also established market exclusivity to provide pharmaceutical products approved under Section 505(b)(2) with potential market exclusivity for three years from FDA approval.

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 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 can be effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with treatment is common. Reasons given for non-compliance include the unpleasant taste associated with available medications, the frequency with which medication must be taken, the number of pills, and the high cost of the medication³.

About ACER-001

ACER-001 is a fully taste-masked, immediate-release proprietary formulation of sodium phenylbutyrate developed by Acer using a microencapsulation process and being developed for the treatment of various inborn errors of metabolism, including UCDs and Maple Syrup Urine Disease (MSUD). ACER-001 microparticles consist of a core center, a layer of active drug, and a taste-masking coating which dissolves in the stomach, allowing taste to be neutralized while still allowing for rapid systemic release. Acer is initially developing ACER-001 as a taste-masked, cost-effective alternative treatment for patients with UCDs. If the pivotal bioavailability and bioequivalence trial is successful, Acer plans to submit an NDA under Section 505(b)(2) for ACER-001 in UCDs in the first quarter of 2021. Acer also intends to develop ACER-001 for patients with Maple Syrup Urine Disease (MSUD) and has been granted orphan drug designation by the FDA in this indication.

About Acer Therapeutics Inc.

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes three clinical-stage candidates: EDSIVO™ (celiprolol), for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate), for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant, for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. 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.

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.

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 ACER-001 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; the adequacy of our capital to support our future operations and our ability to successfully 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 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 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 – Nov. 13, 2019 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development, and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today reported financial results for the third quarter ended September 30, 2019 and provided an update on the Company’s recent corporate developments.

“We have been very active as we continue to progress the development of our pipeline products, including continued work in support of potential EDSIVO™ approval, and the ongoing development of ACER-001,” said Chris Schelling, CEO and Founder of Acer. “We have completed our EDSIVO™ Type A meeting following receipt of the Complete Response Letter (CRL) and are evaluating plans to appeal the decision. At the same time, we are well underway with our ACER-001 pivotal bioequivalence trial for urea cycle disorders (UCDs), and we are advancing osanetant toward clinical development for induced Vasomotor Symptoms (iVMS), both as previously announced.”

Third Quarter 2019 and Recent Events

• EDSIVO™
  • Conducted a Type A meeting in October 2019 with the U.S. Food and Drug Administration (FDA) regarding the EDSIVO™CRL and engaged with industry experts to determine an optimal path forward
  • Presented data at the Academy of Managed Care Pharmacy (AMCP) Nexus 2019 conference from a pilot study designed to estimate the healthcare costs associated with clinical events in patients with vEDS in the United States. The poster can be found here: https://www.acertx.com/edsivo-publications-and-presentations/

• ACER-001
  • Fully enrolled Part A of a two-part pivotal bioavailability and bioequivalence (BE) trial to bridge ACER-001 to BUPHENYL® for treatment of UCDs.  Part A is a single-center, single-blind, randomized, single-dose crossover trial designed to evaluate the relative bioavailability of three different oral suspension formulations of ACER-001 compared to BUPHENYL® in 20 healthy adult subjects
  • Initiated a taste assessment trial of three different formulations of ACER-001 (multi-particulate powder) assessed relative to BUPHENYL^® (powder) using certified taste-testers
  • Received Type C written comments from the FDA in October 2019:
    • Proceeding with clinical trials as planned
    • The FDA stated that 12 months of long-term and 6 months of accelerated stability data would be required for submission of a 505(b)(2) New Drug Application (NDA)
• Ended the third quarter with $16.1 million in cash and cash equivalents, which the Company believes will be sufficient to fund its current operating and capital requirements through the end of 2020

Upcoming Milestones

• EDSIVO™
  • Evaluating an appeal via Formal Dispute Resolution Request (FDRR) to the Office of New Drugs (OND) with potential submission by the end of 2019

• ACER-001
  • Two-part pivotal BE trial:
    • Results expected for Part A in Q4 2019
    • Anticipate enrolling the first subjects in Part B in Q4 2019.  Part B is a single-center, open-label, randomized, single-dose crossover trial to demonstrate bioequivalence of the optimal formulation of ACER-001 (chosen from Part A) compared to BUPHENYL® in 36 healthy adult subjects.  Trial completion expected in Q1 2020
  •  Taste assessment trials:
    • Results expected in Q4 2019 for taste assessment trial evaluating three different formulations of ACER-001
    • Anticipate enrolling the first subjects in 1H 2020 for the taste study comparing the optimal ACER-001 formulation with BUPHENYL®. The design and size of the trial will be determined following further discussions with the FDA
  • Submit NDA for UCDs in Q1 2021, subject to additional capital, assuming successful outcome in BE trial and 12-month long-term stability data

• Osanetant
  • Submit osanetant Investigational New Drug Application (IND) in Q2 2020
  • Aim to initiate Phase 1/2 trial in 2H 2020, subject to additional capital, evaluating osanetant in patients with medically and/or surgically induced VMS in which Hormone Replacement Therapy (HRT) is contraindicated

Financial Results for the Third Quarter 2019

Cash position. Cash and cash equivalents were $16.1 million as of September 30, 2019, compared to $41.7 million as of December 31, 2018. The Company believes its cash position will be sufficient to fund its current operating and capital requirements through the end of 2020.

Research and Development Expenses. Research and development expenses were $2.8 million during the three months ended September 30, 2019, compared to $2.4 million during the three months ended September 30, 2018. This increase of $0.4 million was primarily due to increases in expenses related to manufacturing services, partially offset by decreases in employee-related expenses and in expenses related to research services and consulting services. Research and development expenses for the three months ended September 30, 2019 were primarily comprised of approximately $0.8 million related to EDSIVO™ and approximately $1.6 million related to ACER-001.

General and Administrative Expenses. General and administrative expenses were $2.5 million for the three months ended September 30, 2019 compared to $1.7 million for the three months ended September 30, 2018. The increase of $0.8 million was primarily due to increases in employee-related expenses.

Net Loss. Net loss for the three months ended September 30, 2019 was $5.3 million, or $0.52 net loss per share (basic and diluted), compared to a net loss of $4.0 million, or $0.43 net loss per share (basic and diluted), for the three months ended September 30, 2018. The increase in loss per share (basic and diluted) was driven largely by increases in expenses related to manufacturing services, partially offset by an increase in the number of shares outstanding at September 30, 2019 as compared to September 30, 2018.

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 three clinical-stage pharmaceutical product candidates: EDSIVO™ (celiprolol), for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; ACER-001 (a fully taste-masked, immediate-release formulation of sodium phenylbutyrate), for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); and osanetant, for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is likely contraindicated. 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.

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 expectations regarding our capital resources; the potential for EDSIVO™ (celiprolol), ACER-001 and osanetant 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; the adequacy of our capital to support our future operations and our ability to successfully initiate and complete clinical trials and regulatory submissions; our progress toward possible approval for EDSIVO™ in light of the Complete Response Letter we received earlier this year; 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, our ability to reduce our operating expenses and conserve cash on a net basis as a result of our prior or any future corporate restructuring initiative, the availability of sufficient resources 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 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]