Roche, PTC strike potential $490M deal for genetic disease program

Swiss drug giant Roche has bought exclusive rights to PTC Therapeutics' program for spinal muscular atrophy (SMA) for $30 million upfront, providing PTC with another major pharma collaboration to fuel development of its pipeline. The deal, which offers up to $460 million in future payments to PTC, gives the South Plainfield, NJ-based developer a boost after suffering the setback of Genzyme walking away from PTC's troubled rare disease program in September.

Roche, which previously partnered with PTC in 2009, now has exclusive global rights to three preclinical compounds in development and backups against SMA. The companies say there are no good drugs for the disease, which is caused by a missing or mutated SMN1 gene and leads to lower levels of the survival motor neuron protein. It weakens muscles, interferes with key survival functions, and often claims the lives of its victims during childhood. PTC initially developed the program with the SMA Foundation, which will stay on as part of the collaboration with Roche.

"We found the science behind this program very compelling, with the potential to help treat a currently incurable condition," Luca Santarelli, global head of Roche Neuroscience, said in a statement. "This is the essence of Roche's entire strategy, focused on solid science and high unmet clinical need, and these compounds bolster our rich pipeline in central nervous system diseases."

PTC has been able to find larger drugmakers such as Roche and AstraZeneca ($AZN) to help take its programs forward, a sound strategy as funding remains tight for private biotech outfits. The new Roche deal, of course, is heavily back-ended, and PTC will have to achieve development and commercial goals to collect the bulk of the money. If the program succeeds, PTC is also in line for double-digit royalties on product sales.

- here's the release
- read this Bloomberg article