Acer therapeutics announces full enrollment of part a in acer-001 pivotal bioavailability and bioequivalence trial for urea cycle disorders

Acer therapeutics inc. 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. 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.
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