Roche drops Huntington's disease trial with once-promising drug tominersen

ZURICH: Roche is calling it quits on a late-stage trial of its Huntington's disease hopeful tominersen, the Swiss drugmaker said on Monday, a blow for sufferers of the rare, debilitating disease with no treatments to stop or reverse it.

The drug, an antisense oligonucleotide originally developed by Ionis and licensed for $45 million in 2017, had raised hopes in an earlier trial when it showed dose-dependent reductions of the mutant protein that drives this complex disease that impairs a person's ability to think, feel and move.

But the Basel-based company is now calling the study off and pausing a separate open-label extension study after an unblinded Independent Data Monitoring Committee made the recommendation to go no further, based on tominersen's potential benefit/risk profile.

"This is very unfortunate news to deliver on the tominersen Phase III study and we know it will be especially difficult for people with Huntington's disease to hear," said Levi Garraway, Roche's chief medical officer.

Roche's drugs division head, Bill Anderson, in February predicted no "game changing data" for tominersen until 2022, so even though the company on Monday didn't provide specifics on why it ditched the trial, getting bad news so quickly clearly came as a surprise.

Roche is still pursuing Huntington's disease, via its Spark gene therapy unit that it bought in late 2019. Its cross-town rival Novartis is also studying a drug, called branaplam, that it has repurposed from spinal muscular atrophy to Huntington's disease.