Acer Therapeutics announced that although it is going through a restructuring process, it stays committed to advancing the development of Edsivo (celiprolol), a potential treatment for vascular Ehlers-Danlos syndrome (vEDS).
The FDA said that before clearing the therapy, it wanted additional data from “an adequate and well-controlled trial” to demonstrate that Edsivo can reduce “the risk of clinical events in patients with vEDS.”
“While we are disappointed by the CRL, we intend to continue our dialogue with the FDA to fully understand its response, and work toward our goal of approval of Edsivo for confirmed COL3A1+ vEDS patients, who currently have no approved treatment options,” Chris Schelling, CEO and founder of Acer, said in a press release.
“Nevertheless, in light of the CRL, it was necessary to reduce our expenses, extend our cash runway, and focus our resources on a potential path forward for Edsivo, as well as continued development of our other pipeline opportunities,” Schelling said.
Edsivo was originally developed to treat high blood pressure, but it started being investigated for vEDS after researchers noted it could promote the production of normal collagen in blood vessels.
It also lowers blood pressure, which might help reduce vEDS patients’ risk of a blood vessel rupture by reducing the mechanical stress on affected vessels.
vEDS, also called EDS type IV, is an uncommon and most severe form of EDS. It is marked by impaired production of the tissue’s structural protein collagen, most of the time due to mutations in the COL3A1 gene. No cure is available; treatment and management are focused on preventing serious complications and relieving signs and symptoms of the disease.
Acer submitted a new drug application with the FDA for Edsivo as treatment for vEDS at the end of 2018. It was largely based on results of a Phase 4 trial (NCT00190411). In the study, fewer patients on oral Edsivo experienced a ruptured artery compared to untreated patients (20% vs. 50%). Also, patients taking Edsivo were less likely to have intestinal or uterine ruptures (24% vs. 61%).
Acer says its restructuring is expected to provide the resources needed for the company to conduct its planned business operations through 2020.