Pharma, R&D

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.

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Associate Director CMC
Elektroki Boston, MA
Director Process Development
Elektroki Boston, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

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