PTC Therapeutics has leveraged its know-how on small molecule drug development into two new discovery pacts. The developer raked in $12 million upfront to help Roche find four new drugs for CNS diseases with its Gene Expression Modulation by Small-molecules (GEMS) technology platform. The deal includes an option on four more programs and up to $239 million for each in milestone payments, representing a potential haul of just under $2 billion. And PTC announced a separate deal with Celgene--which exercised an option to partner on a cancer drug--but kept the "substantial" numbers under wraps.
GEMS has generated some big deals for PTC, which has its own experimental therapies in the pipeline. BioWorld notes three: A $1.2 billion deal with Pfizer, a $345 million pact with CV Therapeutics and a $212 million partnership with Schering-Plough.
"PTC's expertise in small molecules addressing RNA biology and the demonstrated productivity of their GEMS technology platform make this an attractive collaboration," said Dr. Christer Nordstedt, head of CNS Discovery of Roche. "We believe that this novel and highly innovative technology will enable Roche to address important disease mechanisms that were intractable with conventional approaches. This may have profound impact on the treatment and management of several CNS disorders and therefore lead to great benefits for the patient."