UniQure Stock (QURE) Rallying – Analysts Upgrade on Huntington’s Gene Therapy Data

UniQure N.V. (QURE) witnessed a remarkable surge in its stock price following the release of positive topline data from its Phase I/II study of AMT-130, a gene therapy targeting Huntington's disease. The announcement spurred significant market enthusiasm, reflecting confidence in the potential of AMT-130 to address this challenging neurodegenerative condition.

The markets reacted swiftly to the news, with uniQure's stock experiencing a dramatic climb. Shares skyrocketed nearly 250%, reaching $45.80. This surge represents a substantial gain and positions the stock at levels unseen in recent times.

The positive data stemmed from the pivotal Phase I/II study, where high-dose AMT-130 demonstrated a statistically significant 75% slowing of disease progression, as measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 36 months, compared to a propensity score-matched external control.

Furthermore, the study met a key secondary endpoint, achieving a statistically significant 60% slowing of disease progression as measured by Total Functional Capacity (TFC) at 36 months compared to the control group. Favorable trends were also observed in secondary endpoint measures of motor and cognitive function, reinforcing the potential benefits of the therapy. Notably, a mean reduction from baseline in cerebrospinal neurofilament light protein of -8.2% suggests a potential reduction in neurodegeneration.

The FDA granted Breakthrough Therapy designation to AMT-130 earlier in the year, further bolstering the prospects of the gene therapy. This designation is designed to expedite the development and review of drugs demonstrating substantial improvement over existing therapies. It reflects the FDA's recognition of the potential of AMT-130 to address the unmet needs in Huntington's disease treatment.

Financial analysts have responded positively to the data, with several firms raising their price targets for uniQure. H.C. Wainwright reiterated a Buy rating with a $70 price target, highlighting the robust, statistically significant, and dose-dependent slowing of disease progression observed in the trials.

The firm believes the data establish AMT-130 as a leading candidate in Huntington's and support a biologics license application (BLA) filing in Q1 2026, potentially leading to accelerated approval. Stifel raised its price target to $65 from $30, maintaining a Buy rating, noting the three-year AMT-130 data suggest a larger market opportunity and a higher probability of approval.

Leerink increased its price target to $68 from $48, keeping an Outperform rating, citing the topline results as a “game-changer” and a “definitive win” for uniQure, making a clear difference on progression across multiple measures of function and cognition.

uniQure's strong cash position, reported at approximately $409 million as of March 31st, is expected to fund operations into the second half of 2027. The company plans to submit a Biologics License Application (BLA) for AMT-130 in the first quarter of 2026, with an anticipated U.S. launch later that year, pending approval. This timeline provides a clear roadmap for the potential commercialization of AMT-130 and its impact on the Huntington's disease treatment landscape.

The significant stock price appreciation reflects the markets' optimism regarding AMT-130's potential and uniQure's progress in developing a meaningful treatment for Huntington's disease. The positive clinical data, regulatory support, and solid financial foundation have collectively contributed to the surge in market confidence.

The coming months will be crucial as uniQure prepares for the BLA submission and continues to advance its gene therapy program. The market's response underscores the potential for future growth and value creation.