MedCity News December 2, 2024
Frank Vinluan

Novartis is securing global rights to a PTC Therapeutics Huntington’s disease drug candidate that has encouraging Phase 2 data that will be discussed with the FDA to plan a pivotal study. Last year, Novartis discontinued its Huntington’s program after safety issues emerged in Phase 2 testing.

Nearly six months ago, PTC Therapeutics reported positive interim data for a drug in mid-stage development for Huntington’s disease, an inherited neurological disorder with limited treatment options and a history of stymied drug research efforts. The PTC drug’s data readout prompted another company to make an unsolicited bid for the program.

Rather than jump at the offer, PTC decided to see if others might also be interested in the asset. Novartis has won this...

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