Acer Therapeutics Announces ACER-001 for Urea Cycle Disorders Trial Shows Bioequivalence to BUPHENYL

Anticipate submitting New Drug Application in early 2021 assuming successful completion of additional non clinical work and 12-month long-term stability data

NEWTON, Mass., Feb. 24, 2020 (GLOBE NEWSWIRE) -- 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.