Acer Therapeutics Inc (NASDAQ: ACER) has announced that its celiprolol therapy has received Breakthrough Therapy Designation from the FDA. The company has developed Celiprolol to treat vascular Ehlers Danlos Syndrome.
Ehlers Danlos syndrome has a high unmet need
The FDA gives Breakthrough therapy designation to drugs that have the potential to treat severe diseases. The designation is to accelerate the review and development of drugs. For the drug to receive this designation, the developer must demonstrate in a preliminary study that it can improve particular disease endpoints and is more effective than the therapies available.
According to Acer’s CMO, Adrian Quartel, the company is glad to receive the designation as theirs is no medication for Ehlers Danlos syndrome. For this reason, Acer is working to provide medication for an unmet need.
Quartel adds that the company is happy to move forward with the SPA procedure and continue the discussion with the FDA. These discussions ensure that the company can successfully initiate the Pivotal Phase III DISCOVER study. Acer hopes to start the clinical trial before Q2 2022 ends.
The company is trying to agree on a SPA
Celiprolol, also known as EDSIVO, received an IND from the FDA in January. The company and the regulator are now trying to agree on a SPA. AN SPA is a process where sponsors could have discussions with the FDA to discuss the size and design of animal or clinical trials.
Acer is hoping to recruit 150 patients from the country for its study. Once this is done, the company could conduct the study for about three and a half years. If the FDA and the trial’s sponsors conclude, the company could soon start the trial as long as they have enough capital to continue it past 2022.
Acer hopes to receive an NDA from the FDA. However, this is not a surety and will depend solely on the data it gets from the study. Furthermore, the SPA could enable the company to successfully market the drug if it shows success, as it shows Acer’s willingness to comply with the regulator’s guidelines.
The disease is a genetic disorder that presents on a spectrum. One of its most common presentations is the vascular form which makes blood vessels unusually fragile.
