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

Xconomy New York — 

Yesterday, European drug giant Roche and South Plainfield, NJ-based PTC Therapeutics announced they had entered into a drug-development pact that could be worth as much as $490 million to PTC. Such alliances are nothing new, especially these days, when Big Pharma companies are increasingly looking towards their smaller rivals for innovations. But this deal is unusual in that it includes a third partner—a not-for-profit organization that is so dedicated to finding cures for a rare but devastating disease that it “earned a seat at the table” for the development process, says Claudia Hirawat, senior vice president for corporate development at PTC.

The disease is called spinal muscular atrophy (SMA), and the organization is the SMA Foundation, based in New York. SMA is a genetic disease that causes muscles to weaken and atrophy over time. About 9,000 people in the United States have the disease, and it is the number-one cause of gene-related deaths in infants and toddlers, according to the SMA Foundation. The organization—founded in 2003 by Loren Eng and Dinakar Singh, who have a child with SMA—has so far devoted $100 million to SMA research efforts.

PTC started collaborating with the SMA Foundation in 2006, Hirawat says, and generated several compounds designed to target the root of the disease. SMA is caused by a defect in a gene called SMN1, which makes a protein that’s critical for maintaining healthy motor neurons—the nerve cells in the spinal cord that are responsible for muscle contraction. Using a process called “alternative splicing,” PTC generated drugs that prompt a backup gene to produce more of the vital protein.

Last year, PTC started to generate promising data in animal trials, Hirawat says. So the company began talking to potential development partners, including Roche, which had been working with PTC since2009 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.”