Acer Therapeutics NasdaqCM:ACER Stock Report

Acer Therapeutics and Relief Therapeutics Announce FDA Acceptance for Review of NDA Resubmission for ACER-001 for Treatment of UCDs

Acer Therapeutics Inc. Resubmittes the ACER-001 New Drug Application (NDA) to the FDA for the Treatment of Urea Cycle Disorders

RELIEF THERAPEUTICS Holding SA announced that its collaboration partner, ACER Therapeutics Inc. for ACER-001 (sodium phenylbutyrate) for oral suspension for the treatment of patients with urea cycle disorders (UCDs), has resubmitted its New Drug Application (NDA) for ACER-001 to the U.S. Food and Drug Administration (FDA). Relief has been advised by Acer that it believes that the resubmission addresses, in full, the items raised by the FDA in the Complete Response Letter (CRL). In June 2022, as previously announced, the FDA issued Acer a CRL stating that satisfactory inspection of its third-party contract packaging manufacturer is required before the ACER-001 NDA may be approved. Relief is advised that Acer notified the FDA in the resubmission that the third-party contract packaging manufacturer is ready for inspection. FDA did not cite any other approvability issues in the CRL pertaining to the NDA, nor request any additional clinical or pharmacokinetic studies be conducted prior to FDA action. According to Acer, additional existing nonclinical information as requested by the FDA in the CRL but identified as “not an approvability issue,” as well as labeling and other routine updates to the original NDA, were provided in the resubmission of the NDA.

Acer Therapeutics Announces Initiation of its Pivotal Phase 3 DiSCOVER Trial of EDSIVO™ (celiprolol) for the Treatment of Vascular Ehlers-Danlos Syndrome

Acer Therapeutics Inc. announced the initiation of patient screening in its Phase 3 DiSCOVER (Decentralized Study of Celiprolol on vEDS-related Event Reduction) clinical trial of EDSIVO™ (celiprolol) for the treatment of patients with COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS).The DiSCOVER trial is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy trial designed to evaluate EDSIVO™ (celiprolol) in patients with genetically confirmed COL3A1-positive vEDS using a decentralized clinical trial design and an independent adjudication committee. The primary objective of the trial is to determine whether EDSIVO™ (celiprolol) reduces the occurrence of vEDS-related clinical events requiring medical attention, including fatal and non-fatal cardiac or arterial events, uterine rupture, intestinal rupture, and/or unexplained sudden death, relative to placebo as measured by time to event. Acer plans to enroll approximately 150 COL3A1-positive vEDS patients, all in the U.S., randomized 2:1 to receive either EDSIVO™ (celiprolol) or placebo, respectively. Once the trial is fully enrolled, the duration of the DiSCOVER trial is currently estimated to be up to approximately 3.5 years to completion (based on statistical power calculations and number of primary events), which will require additional capital beyond Third Quarter 2022. One interim analysis (based on number of primary events) is also planned at approximately 24 months after full enrollment. EDSIVO™ (celiprolol) is an investigational product candidate which has not been approved by the U.S. Food and Drug Administration (FDA). There is no guarantee that this product candidate will receive regulatory authority approval or become commercially available for any indications in the U.S.