Roche Embraces Three-Way Rare-Disease Play With PTC and Patient Group

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2009 on a separate technology-related alliance. In June, at a meeting focused on SMA, Roche and PTC began serious discussions about a formal partnership, Hirawat recalls.

Under the deal, PTC will receive $30 million up front, and Roche will get an exclusive worldwide license to three compounds in early development, plus back-up drugs. PTC will be eligible for $460 million in milestone payments, plus double-digit royalties on any drug that makes it to market. Hirawat says PTC and Roche have not yet ironed out the timeline for getting a drug into human clinical trials, but she is confident SMA will be granted orphan-disease status, which could shorten the FDA-approval process.

Hirawat says that one of the main reasons PTC picked Roche was that the drug giant was willing to include the SMA Foundation in the development process. “Roche has an innate appreciation of the role of the foundation,” Hirawat says. The foundation will participate in a joint steering committee with scientists from both companies, she says. That committee will oversee all the decisions about which compounds to take forward into clinical trials. The foundation, Hirawat says, “gets an actual vote.”

PTC was founded in 1998 with a focus on developing therapies for neuromuscular disorders. In addition to working with Roche, PTC has formed alliances with a number of other Big Pharma companies, including Pfizer, Merck, AstraZeneca, and Celgene. And it has won grants from such organizations as the Muscular Dystrophy Association and the Cystic Fibrosis Foundation. SMA Foundation has awarded PTC a total of $13 million in grant funding.

Hirawat says she’s unaware of any other three-way collaboration between two pharma companies and a foundation. But she’s hopeful such alliances will catch on in drug development. “If there’s one key word that needs to be embraced in rare disorders it is ‘collaboration,'” Hirawat says. “We all have limited tools, limited information. It’s critical that everyone pulls together.”

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