FDA requires Phase III trials uniQure(QURE.US) Huntington's disease gene therapy launch faces obstacles, stock plunges approximately 33%

LootboxPhobia · 2026-03-03T05:08:42+00:00

Gene therapy company uniQure's Huntington's disease candidate drug AMT-130 did not receive FDA accelerated approval and requires Phase III clinical trials, leading to a roughly 33% drop in stock price. Although Phase I data showed significant drug efficacy, the FDA believes the current data is insufficient to support a marketing application. uniQure plans to discuss subsequent plans with the FDA.

LootboxPhobia

2026-03-03 05:08:42

Abstract generation in progress

Proactive Financial APP learned that gene therapy company uniQure (QURE.US) originally hoped that its Huntington’s disease candidate drug AMT-130 would receive accelerated approval from the U.S. FDA, but this expectation was recently dashed. The FDA explicitly informed the biopharmaceutical company that it needs to conduct a randomized, double-blind, sham-controlled Phase III clinical trial.

Following this news, the company’s stock price closed down about 33% on Monday.

According to a press release from the company, uniQure had previously planned to apply for approval based on data from a Phase I/II study comparing it to external controls. However, the FDA believes that the existing data are not sufficient as “the primary evidence of effectiveness needed to support the BLA submission for AMT-130.”

uniQure stated that it will request a Type B meeting with the FDA in the second quarter to discuss the next steps.

In a subsequent conference call, CEO Matthew Kapusta revealed that Phase I/II data showed the drug could slow disease progression by 75% and reduce the decline in overall functional capacity by 60%. However, he also admitted that during a pre-BLA meeting held last October, “the FDA indicated that the data submitted at that time… were unlikely to serve as the main basis for a BLA submission.”

View Original

This page may contain third-party content, which is provided for information purposes only (not representations/warranties) and should not be considered as an endorsement of its views by Gate, nor as financial or professional advice. See Disclaimer for details.