PTC could earn $460 million in milestones plus double-digit royalties.

Roche is paying PTC Therapeutics $30 million up front for an exclusive, worldwide license to the latter’s spinal muscular atrophy (SMA) program. It includes three preclinical-stage candidates and potential back-up programs. PTC has been developing the program in partnership wtih the SMA Foundation, which will retain an active role in the collaboration. The deal with Roche could see PTC receive up to $460 million in development and commercialization milestones plus potentially double-digit royalties on commercial sales.

Roche and PTC have been collaborating since 2009 on the development of orally available small molecules based on the latter’s GEMS™ (gene expression modulation by small molecules) platform. The SMA program developed by PTC is based on alternative splicing. “We found the science behind this program very compelling,” notes Luca Santarelli, global head of Roche Neuroscience.

PTC is focused on the discovery and development of oral small molecule drugs that target post-transcriptional control processes. The firm’s internal pipeline includes candidates against diseases in multiple therapeutic areas including rare genetic disorders, oncology, and infectious diseases.

The firm’s lead therapeutic protein candidate, ataluren, is currently undergoing pivotal trials as a treatment for nonsense mutation (nm) Duchenne/Becker muscular dystrophy (DBMD) and nm cystic fibrosis. It also is in Phase II development for nm hemophilia Aand B and nm methylmalonic academia.

In September the firm restructured its deal with Genzyme for development of the drug. Under the original deal PTC held commercial rights to ataluren in the U.S. and Canada, and Genzyme held commercial rights in all other countries. Under the restructured agreement PTC regains worldwide rights to ataluren, and Genzyme retains an option to commercialize ataluren in indications other than nmDBMD outside the U.S. and Canada. PTC will continue to conduct worldwide development in all indications with a near-term focus on late-stage programs in nmDBMD and nonsense mutation (nm) cystic fibrosis.

PTC’s clinical pipeline also includes the anticancer candidate PTC299, which is in Phase II development for the treatment of type 2 neurofibromatosis, Kaposi sarcoma, metastastic breast cancer, and solid tumors. PTC299 is an designed to inhibit the production of VEGF by targeting the post-transcriptional processes that regulate VEGF synthesis.

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