The clinical-stage pharma company Acer Therapeutics Inc. (Nasdaq:ACER) has begun screening patients for its Phase 3 DiSCOVER (Decentralized Study of Celiprolol on vEDS-related Event Reduction) clinical study of EDSIVO (celiprolol) to treat COL3A1-positive vascular Ehlers-Danlos syndrome (vEDS).
There are presently no FDA-approved drugs available for vEDS, which is associated with a risk of internal bleeding, internal injuries and aneurysms.
The study will determine whether the Beta 1 adrenergic receptor antagonist celiprolol reduces the likelihood of vEDS-related clinical events needing medical attention. Such events, including cardiac or arterial events, uterine rupture or intestinal rupture, can sometimes be fatal.
Vascular Ehlers-Danos syndrome is relatively rare, affecting between 6,000 and 8,000 people in the U.S., according to the VEDS Movement.
To optimize recruiting, Acer Therapeutics will use a decentralized study design. The company aims to recruit roughly 150 participants randomized 2:1 to receive celiprolol or a placebo.
The company expects the trial duration to be 3.5 years, with an interim analysis scheduled for 24 months after enrollment is completed.
The prospective DiSCOVER study will be randomized, double-blind and placebo-controlled, with an independent adjudication committee.
Eligible participants will have genetically confirmed COL3A1-positive vEDS.
The COL3A1 gene guides the production of instructions for making type III collagen.
In 2015, celiprolol received orphan drug designation for vEDS from FDA in 2015.
Acer filed a new drug application for the indication in 2018, but in 2019, Acer received an FDA Complete Response Letter (CRL).
In April 2022, however, FDA granted celiprolol Breakthrough Therapy designation for COL3A1-positive vEDS.
ACER shares were up 18.84% to $1.45 in mid-day trading.