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Wave Chasing Huntington’s Accelerated Approval Based On Caudate Atrophy Surrogate

 
• By Alaric DeArment

The biotech plans a pivotal trial that would support accelerated approval of the Takeda-partnered antisense oligonucleotide, WVE-003, and will discuss it with regulators before the end of the year.

Wave believes a registrational trial with about 150 patients could last 12 to 18 months. • Source: Shutterstock

Wave Life Sciences Ltd. expects to pitch using caudate atrophy as a surrogate endpoint to support accelerated approval of its WVE-003 in Huntington's disease to regulators by the end of the year.

Key Takeaways

  • Wave announced positive efficacy data for WVE-003 in Huntington’s disease, with statistically significant reductions in mutant huntingtin protein and promising imaging biomarker data.

  • The...

The Cambridge, MA-based biotech's case may be helped by positive Phase Ib/IIa efficacy data, although potential safety concerns about neurofilament light (NfL) protein levels could complicate the decision.

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