Roche, Isis sign $392m Huntington’s disease alliance

Hannah Blake

pharmaphorum

Roche has formed an alliance with Isis Pharmaceuticals to develop treatments for the genetic brain disorder, Huntington’s disease. The alliance will be based on Isis' antisense oligonucleotide (ASO) technology and therefore Roche will pay Isis US$30 million upfront and up to $362 million in licensing and milestone payments.

"We are pleased to be working with Roche, a global leader in drug development with significant experience in developing and commercializing drugs to treat neurological diseases. We believe that Roche's expertise in developing CNS drugs, along with their clinical development experience, will greatly enhance our development efforts for this program and allow us to move forward more rapidly.”

B. Lynne Parshall, Chief Operating Officer of Isis.

Initially, research will focus on Isis' lead drug candidate that blocks production of all forms of the huntingtin (HTT) protein, the protein responsible for HD and thus has the potential to treat all HD patients. In addition, Isis and Roche will also be collaborating to combine Isis' ASOs and Roche's proprietary "brain shuttle" program with the objective of increasing the brain penetration of ASOs with systemic administration.

"Huntington's is a severely debilitating neurodegenerative disease and a large unmet medical need. Patients experience gradually worsening motor function and psychological disturbances, with a significant shortening of life expectancy after the disease is diagnosed. Treatments are urgently needed, and we believe that the Isis approach in combination with Roche's brain shuttle represent one of the most advanced programs targeting the cause of HD with the aim of slowing down or halting the progression of this disease."

Luca Santarelli, Head of Neuroscience and Small Molecules Research at Roche.

Approximately five to 10 out of every 100,000 people in Western Europe suffer from Huntington’s disease and there’s an estimated 30,000 people affected in the US.