On Monday it was announced that Roche had exercised its option on an orphan drug for the treatment of Huntington’s disease (HD), a genetic, progressive, neurodegenerative disease. There are currently approximately 30,000 people with symptomatic HD in the United States and there is no approved effective disease modifying treatment for the condition.
Roche initially announced a collaboration with Isis Pharmaceuticals (since wisely renamed as Ionis Pharmaceuticals) in 2013. The aim of the collaboration was to develop therapies for HD, with the focus to be on developing Ionis’ lead drug candidate. The drug works by blocking production of huntingtin protein, which is the protein responsible for HD.
Under the terms of the agreement, Ionis has been responsible for discovery and development of the drug through completion of the first Phase 1/2 trial. Ionis recently completed a 46-patient Phase 1/2a randomized, placebo-controlled, dose escalation study. The drug demonstrated a suitable safety and tolerability profile and there were dose-dependent reductions of huntingtin protein observed in participants. Roche has subsequently exercised its option to license the drug and will now be responsible for all global development, regulatory and commercialization activities.
Roche paid Ionis a US$30 million upfront on the establishment of the companies’ collaboration in 2013 and has since paid another US$25 million in milestones. A further US$45 million will now be paid as license fee. Total potential payments under the deal agreed to in 2013 were US$362 million with Ionis also entitled to receive tiered royalties on sales if the drug is approved.
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