uniQure Faces Critical Moment Ahead of Huntington's Gene Therapy Data

uniQure, a gene therapy company, is awaiting critical data for its Huntington's disease treatment. Despite a 35% share price increase since February, investors are getting nervous about the upcoming results. The company's stock has risen significantly due to its innovative treatments and strong financial performance.

Amsterdam, Netherlands — uniQure (NASDAQ: QURE), a gene therapy company specializing in rare diseases, is gearing up for a critical data readout for its Huntington’s disease treatment, AMT-130. Despite a significant 35% increase in share price since February, investor sentiment has been volatile, with nerves rising ahead of the upcoming results. The company's stock has seen substantial gains due to its innovative treatments and strong financial performance.

uniQure, based in Amsterdam, is valued at a market cap of $1.2 billion, with shares trading at approximately $18 per share. The company has secured approvals for Glybera and Hemgenix, but its present focus is on AMT-130, a gene therapy candidate for Huntington’s disease. AMT-130 has been granted Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations by the FDA, and the company expects to present topline three-year data in September 2025.

The recent Q2 earnings report revealed a cash position of $377 million, expected to last until the second half of 2027. Net loss for the quarter was $37.7 million, or $0.69 per share. Management expects to present AMT-130 data in September 2025, which could be a significant catalyst for the company. CEO Matthew Kapusta stated that the primary efficacy analysis will compare the 3-year change in cUHDRS in high-dose patients to a propensity score matched external control arm using data from the ENROLL-HD natural history study.

The FDA has aligned with uniQure on the AMT-130 statistical analysis plan and chemistry, manufacturing, and control (CMC) requirements to support a planned BLA submission in the first quarter of 2026. The company's Chief Medical Officer, Walid Abi-Saab, noted that the agency endorsed their CMC strategy, suggesting that prior knowledge from Hemgenix manufacturing may be sufficient to support process validation for the BLA submission.

Investors are cautiously optimistic about the upcoming data, but there are concerns regarding the use of natural history data and the switch from propensity score reading to propensity matching. The company has also announced a fourth cohort in the Phase I/II trial of AMT-130, which is expected to initiate in the third quarter. This open-label single-arm U.S. study will evaluate the safety of the high dose of AMT-130 in at least six patients with lower striatal volumes.

The upward trajectory of the share price across the past few weeks can tentatively be interpreted as a positive, with the CEO expressing confidence in the company's progress. However, investors remain nervous about the upcoming data and the potential impact on the company's future prospects.

References:
[1] https://seekingalpha.com/article/4820086-uniqure-getting-nervous-ahead-of-critical-huntingtons-gene-therapy-data