Roche has announced a licensing agreement with PTC Therapeutics to develop treatments for a genetic muscle-weakening disease in a deal that could be worth $490 million.
The Swiss major has signed up to get access to PTC's spinal muscular atrophy (SMA) programme. SMA is a genetic neuromuscular disorder that causes muscle weakness and one in every 10,000 children born is affected by it.
There is currently no effective treatment for SMA which is caused by a missing or defective SMN1 gene, which results in reduced levels of the survival motor neuron protein. Roche noted that given the rarity of SMA, "it could be eligible for orphan status by regulatory authorities, thereby potentially reducing the time needed for a drug to reach patients".
Under the terms of the agreement, Roche gains an exclusive worldwide licence to PTC’s SMA programme, which includes three compounds currently in preclinical development, as well as potential back-up compounds. PTC will pocket a $30 million upfront fee and up to $460 million upon successful completion of certain development and commercialisation milestones, plus double-digit royalties.__
PTC's programme has been developed in partnership with the SMA Foundation and, interestingly, development will be overseen by a joint steering committee comprised of members from the latter group, as well as Roche and PTC. Dinakar Singh, chairman and founder of the SMA Foundation, which has given out over $100 million for research, said "Roche can help us realise what we have been working so hard to achieve".__
Luca Santarelli, head of neuroscience at the Basel-headquartered giant, said "we found the science behind this programme very compelling, with the potential to help treat a currently incurable condition". He went on to claim that "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 and Roche are old friends and signed an agreement in September 2009 to develop orally bioavailable small molecules using PTC’s GEMS technology, focusing initially on four CNS disease targets. Cashwise, Roche made an upfront payment of $12 million and the potential of that deal is in the region of $1 billion.