Dive Brief:

As 2021 began, Huntington's drug research appeared to be on the verge of turning a corner.

Both Roche and Wave Life Sciences were advancing drugs that were similarly designed to block production of a protein implicated in disease progression. For Roche, the stakes were particularly high: A successful result could've led to an approval application for the first drug that might slow the march of the deadly, inherited disease.

By the end of the first quarter, however, both companies reported negative data, raising questions about their drugs as well as researchers' understanding of Huntington's. It also dialed up pressure on companies with earlier stage projects, such as UniQure and Passage Bio.

But it turns out Roche isn't done with tominersen after all, a decision that could have implications for others. While the new clinical trial will be a small, Phase 2 test that will require follow-up studies, tominersen remains one of the most advanced disease-modifying drug in clinical development for Huntington's.

The Swiss drugmaker is in "the early stages" of designing the new trial, Ionis said, which will evaluate different doses of tominersen in younger patients with less severe disease. Roche will share the design of the trial with Huntington's disease specialists in medical meetings later this year, according to Ionis.

Ahead of the trial design presentations, Roche later this week will begin a series of webinars to discuss with Huntington's specialists the after-the-fact analysis of GENERATION-HD1 that hinted at a benefit for the younger, less severe patients.

Roche's Huntington's collaboration with Ionis dates back to 2013, when the big drugmaker acquired initial licensing rights for $30 million and promised up to $362 million in future payouts. Following positive signs in early testing, Roche paid $45 million to license tominersen and cover clinical development as well as commercial costs.

As a disease caused by an identifiable genetic defect, Huntington's seems to be a good target for a medicine that can block the mutation, which results in a flawed version of a protein called huntingtin. Tominersen is a type of medicine known as antisense oligonucleotide and works by going after the RNA that encodes for the protein.

Gene therapies are also aimed at the disease's genetic cause, but work by replacing the defective gene with what could in theory be a one-time treatment. Tominersen, by comparison, was given to patients once every eight or 16 weeks in GENERATION-HD1.

An estimated 41,000 people in the U.S. have Huntington's disease, although many are undiagnosed.

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Roche revives a closely watched Huntington's disease drug - BioPharma Dive