Pharma

PTC Therapeutics licensing agreement with Roche valued up to $490 million

PTC Therapeutics has agreed to a licensing deal with Swiss pharmaceutical firm Roche for its […]

PTC Therapeutics has agreed to a licensing deal with Swiss pharmaceutical firm Roche for its spinal muscular atrophy program that could be worth as much as $490 million.

Under the terms of the agreement, Roche gains an exclusive worldwide license to PTC’s SMA program, which includes three compounds currently in preclinical development, as well as potential backup compounds. PTC will receive $30 million up front and up to $460 million if it meets development and commercialization milestones. PTC will also receive royalties on commercial sales.

Development of the treatment program will be overseen by a joint steering committee with representatives from Roche, PTC and the SMA Foundation. The SMA Foundation was started in 2003 by hedge fund manager Dinakar Singh of TPG-Axon Capital Management to help find a cure for his daughter, Arya, and other patients with the disease.

Spinal muscular atrophy, or SMA, is a genetic muscle-weakening disease and affects 18,000 children per year, according to the SMA Foundation. Because the disease affects less than 200,000 people, the treatment is considered an orphan drug and is eligible for expedited approval from the U.S. Food and Drug Administration. There is currently no cure for the disease.

Roche’s Luca Santarelli, who serves as the global head of Roche Neuroscience, said: “We found the science behind this program very compelling, with the potential to help treat a currently incurable condition.”

Claudia Hirawat, senior vice president for corporate development at PTC, told Bloomberg BusinessWeek that the three-way collaboration of Roche, PTC and the SMA Foundation is breaking new ground. “I am not aware of any collaboration that follows exactly this structure.”

 

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