Roche gets some regulatory love for its breakthrough quest on a PhIII Huntington’s drug

Roche got a boost today in its quest to steer a potentially landmark Huntington’s drug through pivotal development. The EMA handed Roche its PRIME designation for RG6042 — once called IONIS-HTTRx when Ionis was doing the work. 

Similar to the FDA’s breakthrough therapy designation, European regulators will give Roche an open door at the drug agency as the pharma giant launches a Phase III study of the drug, which has joined the lineup of Roche’s late-stage showcase efforts.

Just a few months ago Roche investigators outlined a mean reduction of 40% in mutant huntingtin protein they saw at the two highest doses of the therapy tested in a Phase I/II trial. 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. 

Now Roche — which picked up the option on the drug late last year — has to prove that hitting that biomarker in patients will deliver an important effect on the course of the disease. That won’t be easy, but the potential rewards for success would be big.

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

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