SWITZERLAND – Roche has outlined a new clinical path forward for tominersen, a closely watched Huntington’s disease drug, ten months after disappointing Phase 3 study results appeared to spell the end for the experimental medicine.

According to a statement from development partner Ionis Pharmaceuticals, Roche determined the drug may help younger adult patients with a lower disease burden after reviewing study data.

The company is planning a Phase 2 trial to put the theory to the test. It didn’t go into specifics.

Roche halted the phase 3 clinical trial in March 2021 in response to an interim review of the data in patients with Huntington’s disease, a rare genetic, progressive condition.

The Swiss Big Pharma used a Huntington’s event the following month to share a look at the results that prompted the decision.

Participants who received tominersen every 16 weeks performed similarly to their placebo counterparts. Those who received tominersen every eight weeks performed worse than the placebo group.

Tominersen is an experimental antisense drug that is designed to reduce the production of all forms of the huntingtin protein.

Following the discontinuation of tominersen dosing in the phase 3 GENERATION HD1 trial, exploratory post-hoc analyses revealed a possible benefit of tominersen in younger adult patients with lower disease burden; however, a randomized, placebo-controlled study is required to confirm these findings.

The fact that patients performed worse the more tominersen they received painted a bleak picture of the drug’s chances.

Nonetheless, after sifting through the results, Roche has discovered indications that the drug may benefit younger adult patients with lower disease burden.

According to Ionis, the Swiss drugmaker is in the “early stages” of designing the new trial, which will compare different doses of tominersen in younger patients with less severe disease.

Roche will share the trial’s design with Huntington’s disease specialists at medical meetings later this year.

Roche’s Huntington’s collaboration with Ionis began in 2013, when the big drugmaker paid US$30 million for initial licensing rights and promised up to US$362 million in future payments.

Following encouraging results in early testing, Roche paid US$45 million to license tominersen and cover clinical development and commercial costs.

Huntington’s disease appears to be a good target for a medicine that can block the mutation, which results in a faulty version of a protein called huntingtin.

Tominersen is an experimental antisense drug that is designed to reduce the production of all forms of the huntingtin protein.

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