Sept. 27 Quick Takes: Roche and Ionis taking another approach to Huntington disease

Roche (SIX:ROG; OTCQX:RHHBY) and Ionis Pharmaceuticals Inc. (NASDAQ:IONS) are taking another crack at Huntington disease, and adding Alzheimer’s to their partnership, through a new deal that grants Ionis $60 million up front for two preclinical antisense therapies. The partners’ tominersen, an antisense therapy that targets both wild type and mutant HTT, performed worse than placebo in the Phase III GENERATION HD1 trial to treat Huntington. But post hoc analyses suggested the less frequent of two dosing schedules may have had some benefit in a subset of patients with less advanced disease, prompting Roche to run a new Phase II trial in the subpopulation. One possible explanation for the apparent harmful effect of tominersen may be that it results in loss of wild type HTT function.

The pair is now developing a preclinical mutant HTT-selective therapy to get around that problem. The company did not respond in time for publication to an inquiry about whether the mutant-selective therapy is the subject of the new deal, or whether the deal entails a third candidate. The target of the preclinical Alzheimer’s program is undisclosed. In addition to the upfront fee, the deal includes milestones and tiered royalties...