Drug Development

FDA forces Concert Pharma to hit the brakes on a mid-stage study so it can review tox studies

Concert Pharmaceuticals has been slammed with a clinical hold on a Phase IIa study of its drug CTP-543 for alopecia areata.

The biotech made it sound innocent enough. The FDA, it says, needs to review “certain recently completed non-clinical toxicology studies.” The agency did not cite a safety concern, Concert added in a brief statement, and regulators wanted to review the data to support its plans for a one-year dosing schedule of their new version of Jakafi.

Investors registered their disapproval of the news, but didn’t panic. Concert’s stock $CNCE dropped 10% in pre-market trading.

Concert keyed their program for this drug — their only clinical effort not partnered — on academic efforts that showed Jakafi has potential to treat an autoimmune disease that can cause partial or complete loss of hair. The biotech has been pioneering the use of deuterium to stabilize a drug, making it safer and potentially more effective over a longer period of time with fewer doses.

That profile recently attracted Vertex to the table, taking control of CTP-656 with an upfront payment of $160 million plus $90 million in milestones. The drug is a longer acting formulation of Kalydeco for cystic fibrosis, which has gone a long way to funding Vertex’s transition to a CF company.


Get Endpoints News in your inbox

News reports for those who discover, develop, and market drugs. Join 16,000+ biopharma pros who read Endpoints News articles by email every day. Free subscription.

Quick Subscribe

You're subscribing to Endpoints News

John Carroll, Editor and Co-Founder

We produce two daily newsletters designed to give you a complete picture of what's important in biopharma.

Early Edition is a skimmable digest of original sources you need to see by ~7:15a ET, and our Main Edition is the daily chronicle of biotech, with every story inside the email ~11:55a ET.
2x/weekdays. Privacy policy




EvaluatePharma World Preview 2017