Acer Therapeutics Announces Publication of Vascular Ehlers-Danlos Syndrome (vEDS) Patient Registry Data from Sweden - Seite 2

An infographic accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/f7935f3b-34a5-4373 ...

About EDSIVO
Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. vEDS is the most severe subtype where patients suffer from life threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. The median age of death in the U.S. is 51 years.³ An Acer-commissioned patient-finder study phenotypically identified 4,169 vEDS patients in the U.S. from an analysis of a commercially available patient claims database with data of approximately 190 million unique patient lives. Based on that information, Acer estimates the prevalence of phenotypically-defined vEDS in the U.S. could be greater than 1 in 45,000. Currently, there are no FDA-approved therapies for vEDS.

Acer is advancing EDSIVO, a new chemical entity (NCE), for the treatment of COL3A1-positive vEDS based on published results from a randomized controlled clinical trial of celiprolol (BBEST).² In addition, long-term data has now been published from separate French⁴ and Swedish¹ vEDS patient registries.

The FDA granted a priority review of the EDSIVO NDA and subsequently issued a Complete Response Letter (CRL) in June 2019. The CRL stated that it will be necessary to conduct an adequate and well-controlled trial to determine whether celiprolol reduces the risk of clinical events in patients with vEDS. In March 2020, Acer received a response to its Formal Dispute Resolution Request (FDRR) from the OND of the FDA stating that it had denied its appeal of the CRL in relation to the NDA for EDSIVO. In its Appeal Denied letter, the OND described possible paths forward for Acer to explore that could provide the substantial evidence of effectiveness needed to support a potential resubmission of the EDSIVO NDA for the treatment of patients with vEDS with a confirmed COL3A1 mutation. The OND referred to the FDA Guidance document issued in December 2019⁵, where substantial evidence of effectiveness can be provided by two or more adequate and well-controlled studies demonstrating efficacy, or a single positive adequate and well-controlled study plus confirmatory evidence. Neither resubmission nor the prospect of approval of the EDSIVO NDA is assured. EDSIVO received FDA Orphan Drug Designation for the treatment of vEDS in 2015. EDSIVO is an investigational drug candidate that has not been approved by the FDA for any indication.