Acer Reaches Agreement With FDA; Phase 3 Trial of Edsivo to Begin Soon
Acer Therapeutics expects to launch its pivotal Phase 3 DiSCOVER clinical trial — which will test Edsivo (celiprolol) in patients with COL3A1-positive vascular Ehlers-Danlos syndrome (vEDS) — by the end of June.
The announcement follows an agreement between Acer and the U.S. Food and Drug Administration (FDA) on a special protocol assessment (SPA) regarding the overall protocol design of the study. More information on the DISCOVER trial is available here.
As part of the SPA process, developers may ask to meet with the FDA to discuss specific details about the design of planned clinical trials or other studies.
“Receiving SPA agreement from FDA is a significant milestone in our ongoing development of EDSIVO for patients with COL3A1-positive vEDS, who urgently need an approved treatment for this devastating disease,” Adrian Quartel, MD, Acer’s chief medical officer, said in a press release.
“The SPA underscores our alignment with FDA on important regulatory, clinical and scientific requirements for our planned Phase 3 trial and reflects our ongoing commitment to the vEDS patient community. We are planning for trial initiation by the end of [quarter two] 2022,” Quartel said.
The Decentralized Study of Celiprolol on vEDS-related Event Reduction (DiSCOVER) will be conducted in the U.S. and intends to enroll about 150 patients with a COL3A1 gene mutation, the chief cause of vEDS. Participants will be randomly assigned to Edsivo or a placebo in a 2:1 ratio, meaning that twice the number of patients will receive Edsivo.
The main goal is to see if Edsivo reduces the occurrence of vEDS-related clinical events that require medical intervention — namely heart and arterial events, uterine and intestinal ruptures, as well as unexplained sudden death — compared to placebo.
The trial is expected to run for nearly 3.5 years and an interim analysis is planned at about two years after full enrollment. The trial’s progress beyond June will require additional capital, according to the press release.
vEDS is characterized by deficient production of collagen, a structural protein, which leads to abnormally weak blood vessels and internal organs. Originally developed as a treatment for high blood pressure, Edsivo may raise collagen production in blood vessels.
In April, the FDA granted Edsivo breakthrough therapy designation for COL3A1-positive vEDS. This is meant to quicken the development and review of medicines that seek to treat a serious condition.
Data from a Phase 4 trial called BBEST (NCT00190411) showed Edsivo could reduce the risk of artery, intestinal, and uterine ruptures in vEDS patients.
Acer, supported by these findings, requested FDA approval of Edsivo in 2018. However, the agency rejected the application and asked for an additional trial to further assess its effectiveness. A formal appeal of the rejection was denied.