Breakthrough in Huntington's Disease Treatment: Gene Therapy Shows Promise Amid Ongoing Research
In a groundbreaking development for Huntington's disease (HD) treatment, uniQure's gene therapy has demonstrated significant success in slowing disease progression. This milestone has reinvigorated the field, with several other promising therapies now in advanced stages of development. As the pharmaceutical industry races to bring the first disease-modifying treatment to market, we examine the latest advancements and their potential impact on patients.
UniQure's AMT-130: A Potential Game-Changer
UniQure's gene therapy, AMT-130, has reported "historic positive results" in a pivotal trial, showing a 75% reduction in disease progression over three years in 29 patients. This breakthrough has been hailed as the first successful treatment of Huntington's disease, marking a significant turning point not only for HD but for adult-onset neurodegenerative diseases as a whole.
Despite the excitement, experts urge caution, citing the small patient sample and potential risks associated with the intracranial surgery required for treatment. Nevertheless, uniQure is moving forward with plans to submit a biologics license application to the FDA in Q1 2026, aiming for a potential launch later that year.
Competing Therapies in Late-Stage Development
Wave Life Sciences' WVE-003
Wave Life Sciences is advancing WVE-003, an antisense oligonucleotide (ASO) that selectively targets mutant huntingtin (mHTT) mRNA. Recent data from the Phase Ib/IIa SELECT-HD trial showed a 46% reduction in mHTT levels in patients' cerebrospinal fluid after 24 weeks of treatment. The company has reached an agreement with the FDA on a path to accelerated approval, using slowing of caudate atrophy as a clinical surrogate endpoint.
Prilenia Therapeutics' Pridopidine
Despite setbacks, including a rejection by the European Medicines Agency, Prilenia Therapeutics continues to develop pridopidine, a small molecule agonist of the sigma-1 receptor. New analyses of two-year data from the PROOF-HD study have shown significant slowing of disease progression in patients not taking antidopaminergic medicines. A global confirmatory trial is planned for early 2026.
PTC Therapeutics and Novartis' PTC518
PTC518, a small molecule that reduces mHTT production, has shown promise in the Phase II PIVOT-HD trial. The lowest dose achieved a 23% reduction in blood HTT protein levels in both early- and late-stage patients. While questions remain about the correlation between HTT reduction and clinical benefit, analysts believe the data may be sufficient for discussions with the FDA regarding a registrational path for earlier-stage patients.
As these therapies progress through clinical trials and regulatory processes, the Huntington's disease community remains cautiously optimistic about the potential for effective treatments in the near future. The success of uniQure's gene therapy has set a new benchmark, while the diverse approaches of other candidates offer hope for a multi-faceted treatment landscape for this devastating disease.
References
- 4 Huntington’s Therapies Chasing UniQure
Last month, “historic positive results” from uniQure’s gene therapy snapped the Huntington’s community out of years of failure. As the biotech prepares to submit for FDA approval, BioSpace looks at four more candidates on the near horizon.
Explore Further
What are the long-term safety concerns associated with uniQure's AMT-130 gene therapy, particularly given the intracranial surgery requirement?
How does Wave Life Sciences' WVE-003 compare to uniQure's AMT-130 in terms of clinical efficacy and potential market competitiveness?
What are the specific challenges that Prilenia Therapeutics faced during the European Medicines Agency's review of pridopidine, and how do they plan to address these in the future trial?
What is the significance of PTC Therapeutics and Novartis' PTC518 achieving a reduction in HTT protein levels, and how might this correlate to patient outcomes?
What are the potential impacts of FDA accelerated approvals on the competitive landscape of Huntington's disease treatments, particularly for gene therapies and next-generation drugs?