Roche, Ionis herald a big win in early-stage Huntington’s disease trial, demonstrate disease modifying potential

Ionis $IONS and its partners at Roche have unveiled the biomarker data from a Phase I/II study that recently drove the pharma giant to follow through with a $45 million payment to option rights to IONIS-HTT Rx (RG6042).

Researchers said Thursday evening they tracked a mean reduction of 40% in mutant huntingtin protein at the two highest doses of the therapy. Some patients saw up to 60% reductions. And that, they add, is a first for any Huntington’s drug. It’s that toxic protein that lays waste to neurons in the brain, causing a severe neurodegenerative disease marked by declining muscle coordination and cognitive abilities, which typically slowly kills patients over a course of 20 years.

If they’re right, the developers are on track to the first potential drug that could change the course of the disease, though some steep hurdles on safety and efficacy remain to be cleared.

The research team add the dose dependent response demonstrated that mutant protein levels were still on the decline after the last readout, raising expectations that they can do even better. An open-label extension of the study is underway. Roche has now taken control of the drug and is responsible for mounting a pivotal trial.

Sarah Tabrizi

Roche first signed on to work with Ionis on this drug back in 2013.

Ionis has been on an upswing recently, buoyed by royalties from Spinraza. The biotech also has two drugs under review, including inotersen, which is widely expected to get trounced on the market by Alnylam’s rival patisiran. Still, no one was holding back on the celebration today.

“For nearly twenty years, I have seen many families devastated from losses to this progressive neurodegenerative disease.  With IONIS-HTTRx (RG6042), the HD community has new hope for a therapy that can reduce the cause of HD, and therefore, may slow the progression and potentially prevent the disease in future generations, which is truly groundbreaking,” said Dr Sarah Tabrizi, professor of clinical neurology, director of the University College London’s Huntington’s Disease Centre and the global lead investigator on the study. “I look forward to a longer-term, larger study that can establish the benefit of reducing the toxic mutant huntingtin protein in people with HD.”

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Recursion Pharmaceuticals Salt Lake City, UT
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