Roche and Isis Pharmaceuticals have formed an alliance to develop treatments for Huntington's disease.
Initially, research will focus on Isis' lead drug candidate that blocks production of all forms of the huntingtin (HTT) protein, which is responsible for the inherited genetic brain disorder. The US firm is also conducting research into treatments that specifically block production of the disease-causing forms of the HTT protein which has the potential to treat subsets of HD patients.
Cashwise, Roche will pay an upfront fee of $30 million and up to $362 million in milestone payments, plus tiered royalties. The Swiss major has the option to license the drugs from Isis through the completion of Phase I.
Commenting on the deal, Luca Santarelli, head of neuroscience and small molecules research at Roche, noted that HD is a severely debilitating neurodegenerative disease and represents a large unmet medical need. He added that the Isis approach in combination with Roche's 'brain shuttle' technology, which aims to improve penetration of antisense drugs, "represent one of the most advanced programmes targeting the cause of HD with the aim of slowing down or halting the progression of this disease."
Shafique Virani, head of neuroscience, cardiovascular and metabolism at Roche Partnering, added that this "dual track development programme ensures whichever candidate compound proves to be most promising - Isis' lead target or Roche's brain shuttle version - can be taken forward to pivotal clinical trials".