UniQure's AMT-130 Achieves Landmark Success in Huntington's Disease Trial, QURE Stock Surges 194%

UniQure's AMT-130 has achieved a significant milestone in the treatment of Huntington's disease, demonstrating a 75% reduction in disease progression over three years in its pivotal Phase I/II trial. This breakthrough positions AMT-130 as a potential first-in-class gene therapy for this devastating neurodegenerative disorder.

Following the announcement, UniQure’s stock (NASDAQ: QURE) surged 194%, reflecting strong investor confidence in AMT-130’s potential. As of the latest trading session, QURE is priced at $39.56, with a day's range between $14.07 and $42.63, and a trading volume exceeding 30 million shares. Analysts have reiterated a "Strong Buy" rating, with a 12-month price target of $39.91.

Clinical Trial Overview

The Phase I/II study involved 29 patients, with 17 receiving a high-dose and 12 a low-dose of AMT-130. The primary endpoint was met with a statistically significant 75% slowing of disease progression, as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS), in the high-dose group compared to a propensity score-matched external control. Additionally, a key secondary endpoint, Total Functional Capacity (TFC), showed a 60% reduction in disease progression in the high-dose group.

Other secondary endpoints, including the Symbol Digit Modalities Test (SDMT) and Stroop Word Reading Test (SWRT), also indicated favorable trends, with the high-dose group showing an 88% and 113% slowing of disease progression, respectively. Furthermore, cerebrospinal fluid neurofilament light chain (NfL) levels remained below baseline at 36 months, suggesting a reduction in neuronal damage.

Safety and Tolerability

AMT-130 was generally well-tolerated, with a manageable safety profile. No new drug-related serious adverse events were observed since December 2022, and most common adverse events were procedure-related and resolved without complications.

Regulatory Outlook

UniQure plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first quarter of 2026, with the goal of making AMT-130 available to patients later that year. The therapy has already received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, which could expedite the approval process.

Conclusion

The positive results from this pivotal trial offer new hope to individuals and families affected by Huntington's disease. AMT-130's potential to meaningfully slow disease progression represents a significant advancement in the field and underscores the importance of continued investment in gene therapy research.

For more information, please refer to the official press release.