BASEL, Switzerland--Roche has announced the formation of a new partnershipthis week, establishing a licensing agreement with PTC Therapeutics,Inc. and the Spinal Muscular Atrophy (SMA) Foundation for PTC's SMAprogram.
Per the terms of the agreement, Roche will gain an exclusive worldwidelicense to PTC's SMA program, which consists of three compounds that are currently in preclinical development, in addition to potential back-upcompounds. Roche will pay PTC $30 million in an upfront payment, withpotential additional payments of up to $460 million based on theachievement of certain development and commercialization milestones. PTC will also receive up to double-digit royalties on commercial sales. The development of the SMA compounds will be overseen by a joint steeringcommittee made up of members from all three organizations.
"Having been a partner with Roche for several years, we have every confidencethat the combination of our own expertise and Roche's considerablecapabilities in clinical development, biomarkers and diagnostics willhelp us maximize the potential for this program," Stuart W. Peltz, Ph.D., President and CEO of PTC, said in a press release. "We aredelighted that the SMA Foundation continues to be an active participantin the collaboration as we share a strong commitment to advancing thisinnovative potential treatment as rapidly as possible."
PTC's SMA program was developed in partnership with the SMA Foundation, andthat partnership will remain active within the new collaboration.
"This collaboration brings us one step closer to developing a treatment for a condition that has a profound effect on the lives of many thousands ofchildren and their parents worldwide," Dinakar Singh, Chairman of the SMA Foundation, said in a press release. "We are veryoptimistic that, by building on the pioneering efforts of PTCTherapeutics, Roche can help us realize what we have been working sohard to achieve."
SMA, a genetic neuromuscular disorder that leads to muscle weakness, iscaused by an absent or defective SMN1 gene, which in turn results inreduced levels of the survival motor neuron (SMN) protein. PTC'scompounds work by treating the underlying cause of SMA, and havedemonstrated increases in SMN levels in nervous system, muscles andother tissues in SMA models. SMA is a rare disorder, with one in every10,000 children born affected with the disorder. No effective treatments currently exist for SMA, and it could be eligible for orphan status,which could help reduce the time needed for a related drug to reachpatients.
"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 press release. "This isthe essence of Roche's entire strategy, focused on solid science andhigh unmet clinical need, and these compounds bolster our rich pipelinein 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 tomake a significant impact in the treatment of SMA."
The first collaboration between Roche and PTC, for the development oforally bioavailable small molecules, was announced in September 2009.The collaboration was centered on the utilization of PTC's technology,Gene Expression Modulation by Small-molecules (GEMS). PTC has developedits SMA program through a different scientific approach known asalternative splicing.
SOURCE: PTC Therapeutics press release