- Acer Therapeutics Inc. had a welcoming first week on the Nasdaq stock exchange, seeing shares sruge more 10% Monday after verifying positive clinical results published almost seven years ago from a study of celiprolol, the active ingredient in its lead drug candidate.
- The study tested celiprolol against placebo in patients with vascular Ehlers-Danlos Syndrome (vEDS), a rare, life-threatening disorder that causes arteries and organs to easily rupture. After receiving treatment for about four years on average, 20% of the participants in the investigational arm experienced arterial events versus 50% of those in the control arm.
- While those pivotal results are nothing new, Acer contends its "retrospective source verified analysis" allows the company to use the data in an approval filing for Edsivo, its most advanced drug. Acer plans to submit a New Drug Application (NDA) for Edsivo in the first half of 2018.
There are 13 types of EDS, though healthcare professionals largely consider vascular to be the most severe. Estimates on exactly how prevalent EDS have changed over time, with more recent research suggesting the disease affects between one in every 10,000 to 25,000 people.
Yet even with such a small patient population, there's plenty of market opportunity. The Food and Drug Administration hasn't approved any treatments for the disease — meaning, should Edsivo ultimately get a thumbs up from regulators, it could sit in blissful isolation from competitors. It also doesn't appear as though there are many other potential therapies coming down the pike.
"We have studied celiprolol for nearly two decades in vEDS patients and this is the only drug to ever demonstrate a clinical benefit in this difficult to treat patient population in a randomized, controlled clinical study," said Pierre Boutouyrie, co-director of clinical pharmacology service at the Georges-Pompidou European Hospital in Paris and principal investigator for the celiprolol study, which was published in The Lancet in 2010.
"Having established celiprolol as the standard of care in France for vEDS patients, we are excited to collaborate with Acer to help bring celiprolol to U.S. patients who are suffering from this devastating, life-threatening disease," he added.
Acer's investors shared that excitement, trading the company's stock up nearly 11% to $11 per share at Monday's close.
The Cambridge, Massachusetts-based drugmaker started trading on the Nasdaq on Sept. 21 — the result of a reverse merger with Opexa Therapeutics Inc.. Following the deal, Acer shareholders owned close to 90% of the Opexa's common stock on a pro forma basis while the target's shareholders held on to roughly 10%. The combined company's key focus was to advance Edsivo toward an NDA filing.
Also helping that cause was $15.7 million in financing from an investor syndicate led by TVM Capital Life Sciences. In July, Acer CEO Chris Schelling said the proceeds from the concurrent financing should be enough to get an Edsivo submission in front of U.S. regulators by June 2018 at the latest.
Schelling elaborated in a Sept. 25 statement that his company has been working on "modernizing manufacturing and assembling other components of the regulatory package" for Edsivo, as well as establishing a Centers of Excellence and a vEDS registry for patient care and support.