Roche and Isis set to silence Huntington’s in $392 million deal

isis roche aso antisense brain shuttle (Dr Steven Finkbeiner)

Roche and Isis Pharmaceuticals have announced that they have formed an alliance to develop treatments for Huntington's disease. The alliance looks to develop Isis' antisense oligonucleotide (ASO) technology, and the companies will also collaborate on the development of a drug that utilises Roche's "brain shuttle" program to improve brain penetrance.

Initial research will focus on developing Isis' lead drug candidate that blocks production of the huntingtin (HTT) protein, some forms of which are responsible for the disorder. The research will also lead on to developing treatments that will specifically block production of the disease-causing forms of the HTT protein.

Roche will pay an upfront fee of $30 million, and up to $362 million depending on whether various milestones are met. Roche has the option to license the drugs from Isis through the completion of the first Phase 1 trial.

“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,” said Luca Santarelli, Head of Neuroscience and Small Molecules Research at Roche, in a press release.

“We are excited to be working with Roche,” said Frank Bennett, Senior Vice President of Research at Isis. “We believe our mature antisense drug discovery platform is a perfect fit for Roche’s neuroscience franchise, and we anticipate a fruitful collaboration to advance our pre-clinical compounds.”

If you're interested in exploratory development and early-phase clinical trials, you might be interested in attending the Exploratory Clinical Development World Europe, 4-6 June 2013, London. Click here to download the brochure >

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