Huntington’s disease

Sage Therapeutics discontinued development of its lead candidate dalzanemdor after a third clinical failure, leading analysts to question the biotech’s future profitability.

After previously failing studies in Parkinson’s and Alzheimer’s, dalzanemdor’s latest stumble in Huntington’s disease has pushed Sage Therapeutics to pull the plug on the NMDA receptor modulator.

The past four years have brought disappointment for the Huntington’s community, but optimism is growing as companies including Prilenia and Wave Life Sciences eye paths to approval of therapies that could address the underlying cause of the disease.

A fatal, highly hereditary illness with no disease-modifying treatments, Huntington’s is long overdue for a therapeutic win. Here, BioSpace looks at five candidates that could change the trajectory for patients.

Wave Life Sciences in a Tuesday filing with the SEC said Takeda has elected to terminate its option to continue work on Wave’s WVE-003 clinical-stage Huntington’s disease program—a potential $5 billion commercial opportunity, according to the biotech.

Following a disappointing readout last year, uniQure on Tuesday posted promising Phase I/II data for its investigational gene therapy AMT-130 and nabbed the first-ever Regenerative Medicine Advanced Therapy designation from the FDA in Huntington’s disease.

After back-to-back failures in 2021, Wave Life Sciences has finally aced a Phase Ib/IIa Huntington’s disease trial and is looking to a potential accelerated approval for its investigational antisense oligonucleotide.

PTC Therapeutics said Thursday the FDA has lifted a partial clinical hold on its Huntington’s disease candidate PTC518 after displaying favorable clinical trends in a mid-stage study.

While Sage Therapeutics’ drug candidate showed a slight difference compared to placebo in a mid-stage Huntington’s disease trial, William Blair analysts in a Tuesday note to investors said they “remain cautious” on dalzanemdor and “do not view the small numerical changes as definitive.”

Following a series of clinical failures, optimism builds for the first disease-modifying treatment.