FDA Rejects uniQure's AMT-130 Data as Insufficient for Approval, Demands Sham-Controlled Trial
The FDA informed uniQure that Phase 1/2 study data for AMT-130 in Huntington's disease is insufficient to support a biologics license application, recommending a prospective, randomized, double-blind, sham surgery-controlled study instead.
The Food and Drug Administration informed uniQure BV that data from Phase 1/2 studies of AMT-130 compared to an external control are insufficient to provide the primary evidence of effectiveness required to support a biologics license application filing. The agency's position was conveyed in final meeting minutes following a Type A meeting held on January 30, with feedback received by the company on February 27.
The FDA strongly recommends that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study. The company expects a Type B meeting with the FDA to be held in the second quarter to discuss study designs.
The stock currently trades at $10.50, down 56% over the past week and 56% year-to-date from its 52-week high of $71.50. Wells Fargo downgraded uniQure to Equal Weight from Overweight and lowered its price target to $15 from $60. The firm said it updated its model assumptions on the Huntington's disease program probability of success and launch timeline.
Stifel maintained a Buy rating and $40.00 price target on uniQure following the regulatory feedback. The firm noted the development is disappointing but not surprising following the FDA Commissioner's CNBC interview last week. Stifel said a sham-controlled study introduces meaningful risk.
Mizuho downgraded uniQure to Neutral from Outperform, citing lower confidence in regulatory flexibility for its AMT-130 treatment following recent meeting minutes. RBC Capital downgraded the stock to Sector Perform from Outperform, pointing to the FDA's recommendation for a more rigorous study for AMT-130. Cantor Fitzgerald also downgraded uniQure to Neutral from Overweight due to regulatory feedback that delays the gene therapy program. Leerink Partners reduced its price target to $35 from $60 while maintaining an Outperform rating, noting delays in the U.S. launch.
The company reported a net loss of $199 million, or $3.46 per share, for fiscal year 2025, while ending the year with $622.5 million in cash and equivalents, which is expected to sustain operations into the second half of 2029.
A securities class action lawsuit is pending in the United States District Court for the Southern District of New York against uniQure and certain of its executives. The lawsuit alleges that the Company failed to disclose material information between September 24, 2025 and October 31, 2025, violating federal securities laws. During that period, the Company represented to investors that there was a high likelihood that AMT-130 would receive accelerated approval from the FDA after the Company's planned biologics license application submission in the first quarter of 2026. On November 3, 2025, the Company disclosed that "the FDA currently no longer agrees that the data from the Phase I/II studies of AMT-130 in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission" and as a result, "the timing of the BLA submission for AMT-130 is now unclear." The price of uniQure's shares plummeted $33.40 per share, or more than 49%, from a close of $67.69 per share on October 31, 2025, to close at $34.29 per share on November 3, 2025. Investors have until April 13, 2026 to file lead plaintiff applications.