Roche’s tominersen could help Huntington’s disease market reach over $1 billion in sales by 2030, says GlobalData

Ionis Pharmaceuticals’ partner Roche recently announced that it will initiate a new Phase II trial to reassess its pipeline antisense therapy, tominersen, in the treatment of Huntington’s disease (HD). Tominersen offers Roche the opportunity to bring one of the first disease-modifying treatments to this market, in a move that could help nudge the HD market from its current forecast of $917.7 million to over $1 billion in sales by 2030*, says GlobalData, a leading data and analytics company.

Sarah Elsayed, Neurology Analyst at GlobalData, comments: “Roche’s decision to bring back tominersen in a newly designed trial marks a surprising twist in the drug’s story, as its development was halted in March 2021** due to failure to show evidence of clinical benefit.  However, Roche’s bid to revive tominersen is justifiable, given that the choice of approved treatments is extremely limited across the US, the UK, and Germany. There are only two symptomatic treatments—tetrabenazine and deutetrabenazine—approved for the treatment of HD-associated chorea.”

GlobalData’s report, ‘Huntington’s Disease: Opportunity Assessment and Forecast to 2030’, notes that, the greatest clinical unmet need in the HD market is the development of a disease-modifying drug. Thus, tominersen still has the potential to fulfil this need even though Roche’s new tominersen trial will focus on a smaller subset of HD patients than were studied previously.

Roche recently presented an update on new tominersen data, as part of the Huntington’s Disease Society of America Research Webinar Series, noting that the new Phase II trial will evaluate the safety and efficacy of different doses of tominersen in younger adult patients with low disease burden. This will be assessed using the CAG-repeat Age Product (CAP) score, with specific details on the trial design and start date to be shared at a later date.

During the webinar, Roche presented new clinical endpoints from the hypothesis-driven, post-hoc exploratory analyses of GENERATION HD1 that support the company’s decision to continue the development of tominersen. The data showed that treatment with tominersen every 16 weeks in the low age, low CAP subgroup resulted in better overall outcomes compared to placebo, as measured by the cUHDRS, total functional capacity (TFC), and total motor score (TMS), as well as comparable overall adverse events.

Elsayed continues: “Based on positive findings from an exploratory post-hoc analyses of the GENERATION HD1 trial, Roche’s new trial will assess the efficacy of tominersen in a younger group of adult early manifest HD patients with lower disease burden. If tominersen can score an approval in this patient group, it is highly likely to see strong uptake as no disease-modifying drug has yet been approved for HD patients.”

* In the US, UK, and Germany
** In the Phase III GENERATION HD1 (NCT03761849) study

More Media