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Acer Therapeutics Announces ACER-801 IND Clearance for the Treatment of Induced Vasomotor Symptoms

Press Releases

Phase 2a trial initiation expected in Q1 2022

Trial designed to generate proof of concept data for ACER-801 and identify optimal dose for subsequent efficacy trials in patients with iVMS

NEWTON, MA – Dec. 9, 2021 – Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced U.S. Food and Drug Administration (FDA) clearance of its Investigational New Drug (IND) application for its selective non-peptide neurokinin 3 receptor (NK3R) antagonist, ACER-801 (osanetant), for the potential treatment of induced Vasomotor Symptoms (iVMS).

The initial randomized, double-blind, placebo-controlled, dose-ranging Phase 2a trial is expected to begin in Q1 2022, subject to additional capital required for ACER-801 clinical development.  The trial is designed to evaluate the effect of ACER-801 at different doses, compared to placebo, on the frequency and severity of vasomotor symptoms associated with menopause. The trial will also evaluate the safety and pharmacokinetics profile of ACER-801 at different doses in postmenopausal women.

“Submission and clearance of our ACER-801 IND application is an important step toward the clinical development of our novel NK3R antagonist for treatment of iVMS,” said Chris Schelling, Chief Executive Officer and Founder of Acer. “This drug has been extensively studied, with over 1,200 individuals having received it in clinical trials for other non-iVMS indications. We’re excited to start this Phase 2a trial which, if positive, will provide proof of concept data and help define ACER-801’s optimal dose and development path forward in patients with iVMS.”

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

About iVMS and ACER-801

VMS are generally defined as hot flashes, flushing and night sweats that most often occur in women entering or in menopause. VMS can also be induced (iVMS) by anti-androgen and anti-estrogen cancer therapies and surgical procedures that can lead to treatment non-compliance. VMS are caused by low estrogen levels leading to increased stimulatory signaling of neurokinin B (NKB) on the KNDy neuron in the hypothalamus. A non-hormonal treatment to manage iVMS is needed as estrogen is contraindicated for the management of VMS in patients with hormone-positive tumors, including breast and prostate tumors.

ACER-801 (osanetant) is a novel, non-hormonal, NK3R antagonist that could offer a potential treatment option with meaningful improvement of VMS for patients with iVMS by blocking the stimulatory signaling of NKB on the KNDy neuron. Direct human safety evidence is available from 23 completed Phase 1 and 2 studies of whom approximately 400 healthy subjects and 820 patients were treated with osanetant for schizophrenia, depression, and other indications. Data from these studies indicated no major safety concerns after single-dose and repeat-dose administration.

About Acer Therapeutics Inc.

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

Acer Forward-Looking Statements

This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, timelines, future financial position, future revenues, projected expenses, regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to the potential for our product candidates to safely and effectively treat diseases and to be approved for marketing; the commercial or market opportunity of any of our product candidates in any target indication and any territory; our ability to secure the additional capital necessary to fund our various product candidate development programs; the adequacy of our capital to support our future operations and our ability to successfully fund, initiate and complete clinical trials and regulatory submissions; the ability to protect our intellectual property rights; our strategy and business focus; and the development, expected timeline and commercial potential of any of our product candidates. We may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient resources to fund our various product candidate development programs and to meet our business objectives and operational requirements, the fact that the results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by our intellectual property, risks related to the drug development and the regulatory approval process, including the timing and requirements of regulatory actions, and the impact of competitive products and technological changes. We disclaim any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. You should review additional disclosures we make in our filings with the Securities and Exchange Commission, including our Quarterly Reports on Form 10-Q Q and 10-Q/A, 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

Jim DeNike
Acer Therapeutics Inc.
Ph: 844-902-6100

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