Roche is to partner with Isis Pharmaceuticals to research medicines for the Huntington’s disease – a rare, genetic brain disorder for which there is currently no effective treatment.
Antisense specialists Isis could make up to $392m plus royalties depending on the success of the programme, which initially will focus on research into Isis’ lead candidate that blocks the production of all forms of the huntingtin protein, responsible for the disorder.
In addition, Isis is also conducting research into other treatments that block production of specific forms of the huntingtin protein, noting their potential for use in subsets of patients with Huntington’s disease.
To complement this research, Roche will combine the antisense oligonucleotide (ASO) technology behind these compounds with its ‘brain shuttle’ programme that aims to help drugs overcome the blood-brain barrier and improve penetration of antisense medicines for neurodegenerative conditions.
“Treatments are urgently needed, said Luca Santarelli, head of neuroscience and small molecules research at Roche, “and we believe that the Isis approach in combination with Roche’s brain shuttle represent one of the most advanced programmes targeting the cause of Huntington’s disease with the aim of slowing down or halting the progression of this disease.”
The unmet medical need for Huntingdon’s disease is compounded by its severity, with patients enduring progressive loss of both mental faculties and physical control.
Symptoms usually appear between the ages of 30 to 50, and worsen over a 10 to 25 year period, while death usually occurs when a patient succumbs to a complication, such as pneumonia or heart failure.
Its rareness and difficulty to treat may have contributed to lack of previous research into potential treatments, and Isis’ work was supported by funding from the CHDI Foundation – a non-profit foundation exclusively dedicated to the development of therapies that slow the progression of Huntington’s disease.
The CHDI Foundation will receive $1.5m as part of the agreement with Roche and will be further reimbursed by Isis from milestone payment.
Roche’s payment to Isis breaks down as a $30m upfront fee and licence fee and pre- and post-licensing milestone payments of up to $362m, including up to $80m in commercial milestone payments.
