Acer Therapeutics Announces Topline Results from its Bioequivalence Trial of ACER-001 Compared to BUPHENYL Under Fed Conditions

Targeting a pre-NDA meeting with FDA in Q2 2021

NEWTON, Mass., Feb. 11, 2021 (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 topline results from its bioequivalence trial in which ACER-001 showed similar relative bioavailability compared to BUPHENYL (sodium phenylbutyrate) under fed conditions. ACER-001 powder is a proprietary, taste-masked, immediate release formulation of sodium phenylbutyrate (NaPB) in development for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD).

The single-center, single-blind, randomized, single-dose crossover trial evaluated bioequivalence (BE) of ACER-001 compared to BUPHENYL when administered under fed conditions in 36 healthy adults. The topline data from this trial showed ACER-001 to have similar pharmacokinetic (PK) profiles for both phenylbutyrate (PBA) and phenylacetate (PAA) compared to BUPHENYL under fed conditions. Acer is initially developing ACER-001 for the treatment of patients with UCDs under Section 505(b)(2) of the Federal Food, Drug and Cosmetic Act, which provides a potentially streamlined path for sponsors that have developed drug products that rely upon data from drug products previously approved by the FDA.

“With topline data now in hand, we are moving forward with our plans to conduct a pre-NDA meeting with the FDA in the second quarter of 2021, assuming successful and timely completion of the ongoing development activities, including evaluation of long-term product stability data,” said Chris Schelling, CEO and Founder of Acer. “Assuming no additional data is requested by the Agency during our pre-NDA meeting, we will plan to submit an NDA for ACER-001 for the treatment of UCDs in mid-2021. If ACER-001 is approved by the FDA, we believe its unique formulation will provide clinicians with an alternative to existing sodium phenylbutyrate-based treatments.”

ACER-001 is an investigational product being studied for the treatment of patients with UCDs and MSUD and has not been approved by FDA for any indication. There can be no assurance that if submitted, a New Drug Application will be accepted by the FDA for filing and review or, if filed, that it will be approved.

About UCDs
UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy, somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches, confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric symptoms.^1,2