Author: AcerUser

NEWTON, MA – October 28, 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 A in a pivotal trial evaluating bioavailability and bioequivalence of ACER-001 for the treatment of Urea Cycle Disorders (UCDs). 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 ACER-001 trial design consists of two parts. 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. The goal of Part A of this study is to provide data to help identify the optimal formulation of ACER-001 for further evaluation in Part B.  Part B will be a single-center, open-label, randomized, single-dose crossover trial to demonstrate bioequivalence of the optimal formulation of ACER-001 compared to BUPHENYL® in 36 healthy adult subjects.

“The initiation of this trial represents a significant milestone as we continue to advance the development of our pipeline product candidates,” said William Andrews, M.D., FACP, Chief Medical Officer of Acer. “We believe ACER-001 could represent a taste-masked, cost-effective alternative treatment for patients with UCDs.  We look forward to completion of the two-part bioequivalence trial in the first quarter of 2020, and to progressing our work on ACER-001 for this serious, rare disease.”

About Section 505(b)(2)

The Hatch-Waxman Amendments established Section 505(b)(2) of the FFDCA that provides an alternative pathway for submission of a New Drug Application (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. ACER-001 is being developed under Section 505(b)(2).

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 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, 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. The most common side effects of BUPHENYL® include absent or irregular menstrual periods, decreased appetite, body odor and bad taste.

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 – August 27, 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 that Acer’s management team will provide a corporate overview at the 21^st Annual H.C. Wainwright Global Investment Conference being held at the Lotte New York Palace Hotel in New York City on Tuesday, September 10, 2019.

21^st Annual H.C. Wainwright Global Investment Conference Details

Date:               Tuesday, September 10, 2019                       

Time:               2:35 pm ET

Location:         The Lotte Hotel, New York

Webcast:         http://wsw.com/webcast/hcw5/acer/

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes 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. Acer’s product candidates are 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, future financial position, future revenues, projected expenses, regulatory 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 anticipated future reduction in operating and cash conservation benefits associated with our corporate restructuring initiative; 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; 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™; 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 securities class action litiation, 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 Contacts:

Hans Vitzthum

LifeSci Advisors

Ph: 617-535-7743

[email protected]

Jim DeNike

Acer Therapeutics Inc.

Ph: 844-902-6100

[email protected]

NEWTON, MA – August 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 second quarter ended June 30, 2019 and provided an update on the Company’s recent corporate developments.

“Following receipt of the previously announced EDSIVO™ Complete Response Letter, we plan to meet with the FDA to better understand its views. We are continuing to evaluate paths forward for EDSIVO™ on behalf of the COL3A1+ vascular Ehlers-Danlos syndrome patients, who currently have no approved treatment options,” said Chris Schelling, CEO and Founder of Acer. “As we continue to proactively manage our cash, we have taken prudent steps, including a significant restructuring of the Company, to allow for the continued advancement of our pipeline products through the end of 2020. We look forward to reporting progress on a number of important milestones throughout the second half of 2019 and into 2020.”

Second Quarter 2019 and Recent Events

• ACER-001 Investigational New Drug (IND) application cleared for initiation of pivotal bioequivalence bridging and taste assessment trials of ACER-001 compared to sodium phenylbutyrate
• Disclosed osanetant clinical development plan for patients with induced Vasomotor Symptoms (iVMS), which may include patients receving tamoxifen treatment for breast cancer and/or leuprolide treatment for prostate cancer and/or women who elect to have prophylactic bilateral oophorectomy (surgical removal of ovaries) where Hormone Replacement Therapy (HRT) is likely contraindicated
• Ended the second quarter with $23.5 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™
  • Conduct Type A meeting with FDA in Q3 2019 to discuss EDSIVO™ Complete Response Letter
• ACER-001:
  • Conduct Type C meeting with FDA in 2H 2019 to discuss ACER-001 development plans
  • Initiate ACER-001 pivotal bioequivalence bridging and taste assessment trials in Q4 2019
  • Submit ACER-001 New Drug Application (NDA) for UCD patients in mid-2020, assuming successful outcomes in bridging trials and sufficient drug product stability data to enable NDA filing
• Osanetant:
  • Submit osanetant IND in Q4 2019
  • Initiate Phase 1/2 trial in mid-2020 evaluating osanetant in patients with iVMS, which may include patients receving tamoxifen treatment for breast cancer and/or leuprolide treatment for prostate cancer and/or women who elect to have prophylactic bilateral oophorectomy (surgical removal of ovaries) where Hormone Replacement Therapy (HRT) is likely contraindicated

Financial Results for the Second Quarter 2019

In order to reduce operating expenses and conserve cash resources, the Company implemented a corporate restructuring initiative on June 28, 2019 which included a reduction of approximately 60% of its full-time workforce of 48 employees and a halt of pre-commercial activities for EDSIVO™. The Company implemented the restructuring in light of the Complete Response Letter received from the FDA after market close on June 24, 2019 regarding its NDA for EDSIVO™ for the treatment of vascular Ehlers-Danlos syndrome (vEDS). The Company recorded a one-time severance-related charge of approximately $1.5 million associated with the workforce reduction in the second quarter of 2019. 

Cash position. Cash and cash equivalents were $23.5 million as of June 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 $4.2 million during the three months ended June 30, 2019, compared to $2.7 million during the three months ended June 30, 2018. This increase of $1.5 million was principally due to a $1.1 million increase in employee-related expenses, which included expense of $0.5 million related to the restructuring. The remaining increase in research and development expenses was primarily due to increases in expenses related to manufacturing services, partially offset by decreases in spending for research services. Research and development expenses for the three months ended June 30, 2019 were primarily comprised of approximately $3.0 million related to EDSIVO™ and approximately $1.0 million related to ACER-001.

General and Administrative Expenses. General and administrative expenses were $6.9 million for the three months ended June 30, 2019 compared to $2.2 million for the three months ended June 30, 2018. The increase of $4.7 million was primarily due to a $2.7 million increase in employee-related expenses, which included expense of $1.0 million related to the restructuring. The remaining increase in general and administrative expenses was primarily due to increases in expenses related to pre-commercial activities and professional services.

Net Loss. Net loss for the three months ended June 30, 2019 was $11.0 million, or $1.09 loss per share (basic and diluted), compared to a net loss of $4.8 million, or $0.64 loss per share (basic and diluted), for the three months ended June 30, 2018. The increase in loss per share (basic and diluted) was driven largely by increases in employee-related expenses, which included expenses related to the restructuring, as well as increases in expenses related to pre-commercial activities, expenses related professional services, and expenses related to manufacturing services, partially offset by a decrease in spending for contract research and by an increase in the number of shares outstanding at June 30, 2019 as compared to June 30, 2018.

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

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes 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. Acer’s product candidates are 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, future financial position, future revenues, projected expenses, regulatory 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 anticipated future reduction in operating and cash conservation benefits associated with our corporate restructuring initiative; 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; 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™; 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 securities class action 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: 617-535-7743

[email protected]

Jim DeNike

Acer Therapeutics Inc.

Ph: 844-902-6100

[email protected]

NEWTON, MA – August 1, 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 that Acer’s management team will provide a corporate overview at the 2019 Wedbush PacGrow Healthcare Conference being held at the Parker New York Hotel in New York City on Wednesday, August 14, 2019. 2019 Wedbush PacGrow Healthcare Conference Details

2019 Wedbush PacGrow Healthcare Conference Details:

• Date:               Wednesday, August 14, 2019            
• Time:              8:35 am ET
• Location:       The Parker New York Hotel, New York
• Webcast:        https://www.acertx.com/investor-relations/events-presentations/

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes three clinical-stage candidates: 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); osanetant for the treatment of induced Vasomotor Symptoms (iVMS) where Hormone Replacement Therapy (HRT) is contraindicated; and EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. Acer’s product candidates are 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, future financial position, future revenues, projected expenses, regulatory 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 anticipated future reduction in operating and cash conservation benefits associated with our corporate restructuring initiative; 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; 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™; 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 securities class action litiation, 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: 617-535-7743

[email protected]

Jim DeNike

Acer Therapeutics Inc.

Ph: 844-902-6100

[email protected]

NEWTON, MA – July 25, 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 it will host a conference call and webcast on Wednesday, July 31, 2019 at 11:00 am Eastern Time to discuss a detailed update on each of its pipeline programs, including ACER-001 for Urea Cycle Disorders (UCDs) and Maple Syrup Urine Disease (MSUD), osanetant for neuroendocrine disorders, and EDSIVO™ for vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation.

Conference Call and Webcast Details

Interested parties can access the live call and webcast on Wednesday, July 31, 2019 at 11:00 am Eastern Time (8:00 am Pacific Time) from the Investors section of Acer’s website or directly at http://public.viavid.com/index.php?id=135556. Participants can also access the call by dialing 1-877-407-0789 (US Toll Free) or 1-201-689-8562 (International Toll Number) and providing the Conference ID 13692996. A replay of the call will also be available under the Investors section of Acer’s website.

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. Acer’s pipeline includes three clinical-stage candidates: 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); osanetant for the treatment of various neuroendocrine disorders; and EDSIVO™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. Acer’s product candidates are 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, future financial position, future revenues, projected expenses, regulatory 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; 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™; 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, 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: 617-535-7743

[email protected]

Jim DeNike

Acer Therapeutics Inc.

Ph: 844-902-6100

[email protected]

NEWTON, MA – July 5, 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 a corporate restructuring and update on its pipeline programs. Acer’s headcount has been reduced from 48 to 19 employees and pre-commercial activities of EDSIVO™ (celiprolol) have been halted. The restructuring is expected to provide the resources needed for Acer to conduct its planned business operations through 2020. Acer intends to pursue discussions with the U.S. Food and Drug Administration (FDA) regarding its previously announced Complete Response Letter (CRL) for Acer’s New Drug Application (NDA) for EDSIVO™ for the treatment of vascular Ehlers-Danlos syndrome (vEDS), and to continue the development of Acer’s additional pipeline programs, including ACER-001 and osanetant. 

“While we are disappointed by the CRL, we intend to continue our dialogue with the FDA to fully understand its response and work toward our goal of approval of EDSIVO™ for confirmed COL3A1+ vEDS patients, who currently have no approved treatment options,” said Chris Schelling, CEO and Founder of Acer. “Nevertheless, in light of the CRL it was necessary to reduce our expenses, extend our cash runway, and focus our resources on a potential path forward for EDSIVO™ as well as continued development of our other pipeline opportunities.” 

ACER-001 for Urea Cycle Disorders (UCDs)

Acer expects to initiate a pivotal bridging study in Q4 2019 of ACER-001, a fully taste-masked, immediate release formulation of sodium phenylbutyrate, in patients with a Urea Cycle Disorder (UCD) under an active Investigational New Drug (IND) application. Sodium phenylbutyrate has been found to help control blood ammonia levels in conjunction with a restricted diet for people with UCD.^1,2 However, non-compliance with treatment is common due to the unpleasant taste and the high cost associated with the medications currently approved by the FDA. Acer believes the taste-masked properties and competitive pricing of ACER-001 could make it a compelling alternative to existing treatments.

The purpose of the bridging study is to establish bioequivalence to sodium phenylbutyrate. The trial is expected to enroll approximately 35-70 healthy subjects. Acer will also conduct an independent taste assessment comparing the two formulations. If the studies are successful, and pending feedback from a Type C meeting with the FDA, Acer plans to file an NDA for ACER-001 in UCD patients in the first half of 2020. The Company 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. 

Osanetant for Neuroendocrine Disorders

Acer is also developing osanetant, a clinical-stage, selective, non-peptide tachykinin NK3 receptor antagonist. The Company acquired worldwide rights to osanetant from Sanofi in December 2018. Osanetant was originally developed by Sanofi for the treatment of symptoms associated with schizophrenia and has been studied in more than 1,500 healthy subjects and schizophrenic patients with clinical and laboratory safety data from 21 completed Phase 1 and Phase 2 trials. Clinical proof of concept studies with other NK3R antagonists have demonstrated rapid and clinically-meaningful improvement in vasomotor symptoms and polycystic ovarian syndrome.

Acer expects to disclose specific indications for the development of osanetant in late July 2019 and to initiate a clinical study by mid-2020 to evaluate pharmacokinetics, pharmacodynamics and safety of osanetant in patients with various rare and life-threatening neuroendocrine disorders.

About Acer Therapeutics

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. 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 various neuroendocrine disorders. Acer’s product candidates are 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. Sirrs SM, et al. Barriers to Transplantation in Adults with Inborn Errors of Metabolism. JIMD Rep. 2013;8:139-144.
2. Camp KM, et al.  Phenylketonuria Scientific Review Conference: State of the science and future research needs. Mol Genet Metab. 2014;112(2):87-122. 

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, future financial position, future revenues, projected expenses, regulatory 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; 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™; 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, 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: 617-535-7743

[email protected]

Jim DeNike

Acer Therapeutics Inc.

Ph: 844-902-6100

[email protected]