uniQure rockets on map to market for Huntington's therapy

uniQure says it has reached an agreement with the FDA on a pathway to accelerated approval for its Huntington's disease gene therapy based on a phase 1/2 trial.

The announcement led to a doubling in uniQure's share price as investors considered the prospect of an earlier-than-expected marketing application based on an ongoing study of the AMT-130 gene therapy in the US and Europe.

In principle, the trial – which is comparing AMT-130 to a natural history external control – could be the main element in a biologics license application (BLA) and would not need to be backed up by an additional study.

Crucially for the Netherlands-based biopharma company, the FDA has said that the composite Unified Huntington's Disease Rating Scale (cUHDRS) can serve as an intermediate clinical endpoint for its review, along with the use of neurofilament light chain (NfL) levels in cerebrospinal fluid (CSF) as a biomarker for efficacy.

Last year, Biogen secured FDA approval for its Qalsody (tofersen) therapy for a form of amyotrophic lateral sclerosis (ALS), another neurodegenerative disorder, using NfL as an endpoint.

uniQure has said it plans to meet with the FDA in the first half of next year to discuss statistical analysis plans and technical manufacturing requirements for AMT-130 in preparation for its BLA.

Interim results from the phase 1/2 trial reported in the summer showed that AMT-130 was able to slow down disease progression in patients with early-stage Huntington's over two years compared to an untreated control group, and also reduce levels of NfL in the CSF.

Additional data from the study is expected in mid-2025, including three-year follow-up data for some patients.

"This is an important milestone for the Huntington's disease community, as it puts us on the most rapid and efficient pathway to deliver a potentially life-changing therapy to people living with this devastating neurodegenerative disorder," commented uniQure's chief medical officer, Walid Abi-Saab.

uniQure's Nasdaq-listed shares rose 110% to $15.30 after news of the agreement with the FDA was announced, giving the company a value of almost $750 million.

Last month, it started a cost-stripping programme to save $75 million a year, shedding two-thirds of its staff and shelving early-stage R&D programmes, in order to devote its attention to AMT-130.

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