uniQure surges on new Huntington's gene therapy news

Shares of uniQure saw a remarkable surge of up to 60% on Tuesday following the release of promising interim data from its ongoing Phase I/II trials for AMT-130, a gene therapy for Huntington's disease. The data indicated a notable deceleration in disease progression, marking a significant milestone for the treatment. The findings are based on up to two years of data from both U.S. and European trials of AMT-130. In total, 29 patients received one of two doses of the therapy, while 10 control patients underwent sham surgeries. By the cut-off date of March 31, 21 patients had completed 24 months of follow-up.

The company highlighted that patients administered with the higher dose of AMT-130 exhibited a mean change of -0.2 on the composite Unified Huntington's Disease Rating Scale (cUHDRS), in contrast to a -1.0 change in the propensity score-weighted external control group. This translates to an 80% reduction in the rate of disease progression. Patients on the lower dose showed an average change of -0.7 compared to controls, indicating a 30% slowing in disease progression.

However, the journey for AMT-130 has had its hurdles. In 2022, uniQure temporarily suspended the high-dose regimen due to severe adverse reactions in three patients. Treatment resumed later with enhanced risk mitigation strategies. Additionally, uniQure shares dropped dramatically last year after a mixed update on AMT-130's progress. In October, the company announced a restructuring plan, including a 20% workforce reduction and the discontinuation of over half its research projects, to concentrate resources on key programs like AMT-130.

The latest update builds upon positive momentum from December, when uniQure reported that AMT-130-treated patients continued to maintain neurological function. Despite this, shares dipped at that time due to the absence of a mutant huntingtin protein (mHTT) signal in the cerebrospinal fluid (CSF).

Tuesday's announcement also emphasized a statistically significant reduction in neurofilament light chain (NfL), a biomarker related to neurodegeneration. Patients treated with AMT-130 experienced an average 11% reduction in CSF NfL levels compared to baseline at the 24-month mark. "We believe this is the first clinical trial of any investigational medicine for Huntington's disease to show evidence of potential long-term clinical benefit and reduction of a key marker of neurodegeneration," stated Walid Abi-Saab, the chief medical officer at uniQure.

The company also noted that AMT-130 continues to demonstrate a manageable safety profile, with no new therapy-related serious adverse events reported in this latest analysis. Looking forward, uniQure plans to engage with the FDA in the second half of the year to discuss possible expedited clinical development pathways and accelerated approval. Additionally, the therapy has received the first-ever Regenerative Medicine Advanced Therapy (RMAT) designation for a Huntington's disease treatment.