Roche cuts late-stage study of Huntington's med, Ionis import with prior safety flags

 Hoping to tackle the absolutely brutal Huntington’s disease, Roche inked a licensing pact with Ionis back in 2017 for a novel antisense therapy targeting a protein implicated in the disease. Now, after riding out earlier safety red flags, Roche is calling a late-stage study quits on a data committee’s orders.

Roche has halted dosing for its Phase III study of tominersen, an antisense drug targeting the huntingtin protein and a mutant variant, after an independent data committee “made its recommendation based on the investigational therapy’s potential benefit/risk profile for study participants,” the Swiss drug giant said.

Roche in-licensed tominersen, formerly known as IONIS-HTTRx, back in 2017 and had enrolled 791 patients in 18 countries so far in the Phase III GENERATION HD1 study prior to stopping dosing. The double-blind, placebo-controlled study tested tominersen in patients with manifest Huntington’s over 25 months, randomized to receive either a 120-milligram dose of the drug every two months, a 120-milligram dose every four months or placebo.

Roche also stopped dosing in an open-label extension study dubbed GEN-EXTEND dosing patients on the same high- and low-dose protocol. The company said it would provide further updates on its plan for the drug after data are further evaluated and planned to keep monitoring patients’ safety signals through the study’s trial window.

“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,” CMO Levi Garraway said in a statement.

Roche pointed out that new safety risks weren’t at play in the committee’s decision. That would put to rest some worries that safety signals that derailed another Phase I study had carried over into the late-stage test.

Last March, Roche suspended enrollment of that study, dubbed GEN-PEAK, after investigators noted two cases of intrathecal catheter-related infections that weren’t tied to the drug. That study was later continued, and Roche said it has no plans to discontinue now.

Ionis bagged a $45 million licensing fee from Roche when they followed through on the pact in late 2017. Roche believes it is on to a new and better way to shut down production of huntingtin (HTT) protein, linked to the development of the lethal disease.

https://endpts.com/roche-cuts-bait-on-late-stage-study-of-huntingtons-med-tominersen-an-ionis-import-with-prior-safety-flags/