Acer Therapeutics announces corporate update, anticipated milestones

Acer Therapeutics announced a corporate update and provided anticipated key development milestones for 2023. "The U.S. approval of OLPRUVA for oral suspension by the U.S. Food and Drug Administration is a culmination of Acer‘s collective efforts and ongoing dedication to develop and provide new treatments to patients suffering from rare diseases. Moreover, OLPRUVA’s approval marks a significant milestone for patients in need, offering a new, responsibly priced sodium phenylbutyrate treatment option, that will be supported by Navigator by Acer Therapeutics, our patient services program designed to support patients and caregivers," stated Chris Schelling, CEO and Founder of Acer. "Our commitment to patients is the cornerstone of our mission, and we are proud to have secured our first FDA approval. We look forward to providing further updates in due course as the commercial launch of OLPRUVA(TM) progresses." Expected 2023 Development Milestones: Q1 2023: Acer expects to announce topline results from its ongoing Phase 2a randomized, double-blind, placebo-controlled, dose-ranging trial of ACER-801 for the treatment of moderate to severe VMS in post-menopausal women; Q2 2023: Acer expects initiation of the UNC investigator-sponsored trial to evaluate the potential for ACER-801 to reduce the frequency and severity of PTSD and other trauma related disorders; Q4 2023: Acer anticipates completing enrollment in its ongoing, pivotal Phase 3 DiSCOVER trial of EDSIVO in patients with COL3A1-positive vEDS. The double-blind portion of DiSCOVER trial is intended to end if statistical significance is reached at an interim analysis which occurs at accrual of 28 vEDS-related events, estimated to occur as early as approximately 18 months after completion of full enrollment, or after accrual of 46 vEDS-related clinical events; Acer intends to explore additional lifecycle opportunities for OLPRUVA in various disorders where proof of concept data exists, including in Maple Syrup Urine Disease, Pyruvate Dehydrogenase Complex Deficiency, rare pediatric epilepsies and various liver disorders