Roche inks $490 million deal with PTC Therapeutics
pharmafile | November 30, 2011 | News story | Research and Development, Sales and Marketing | PTC Therapeutics, Roche
Roche has signed a $490 million deal with specialist biotech firm PTC Therapeutics.
Roche is paying PTC $30 million upfront for rights to its spinal muscular atrophy (SMA) programme, and could add $460 million in future milestone payments.
Roche previously partnered with PTC in 2009 and has now extended this agreement to include exclusive global rights to three pre-clinical compounds in development against SMA.
Luca Santarelli, global head of Roche neuroscience, said: “We found the science behind this programme very compelling, with the potential to help treat a currently incurable condition.
“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.
“As an established partner of Roche, we already have experience with PTC’s scientific approach. Together with the involvement of the SMA Foundation, we now have the opportunity to make a significant impact in the treatment of SMA.”
The disease 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 is the leading genetic cause of death in infants and toddlers, according to PTC.
The New Jersey, US-based firm initially developed the program with the SMA Foundation, which said it would stay on as part of the collaboration with Roche.
PTC is focused on the discovery and development of oral, small-molecule drugs that target post-transcriptional control processes. These regulate the rate and timing of protein production and are of central importance to proper cellular function.
PTC’s pipeline of drugs includes treatments for rare genetic disorders, oncology and infectious diseases, but also it licenses out its proprietary GEMS technology which identifies small molecules that modulate post-transcriptional control mechanisms.
The firm has secured several deals with major pharma firms based on this pipeline – including AstraZeneca and Pfizer – but suffered a setback in September when Genzyme dropped out of a deal with its rare disease programme.
Ben Adams
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