uniQure's AMT-130 at Four Years: 44% Slowing of Huntington's Progression — but the Key Score Loses Statistical Significance, and the Stock Craters
September 29–30, 2026. uniQure disclosed four-year data from the pivotal Phase 1/2 trial of AMT-130, its one-time gene therapy for Huntington's disease — and investors reacted as if the durability story had cracked. Per BioPharma Dive: "UniQure lost about a billion dollars in market value Tuesday" as the stock fell 40% to just below $24 at the open.
The numbers: In the 12 high-dose participants followed to four years, disease progression on the cUHDRS scale (cognition, motor skills, daily living) was slowed 44% versus an updated external-control patient database — but "that difference wasn't enough to reach statistical significance." On the TFC scale (daily-function management), progression slowed 61%, which uniQure called a "favorable treatment difference." The company said the collective results show AMT-130 "continues to have a 'meaningful' effect on disease," and argued the four-year effect was "likely understated" because 53% of the 48-month reference data in the updated natural-history database were missing; a company after-the-fact analysis put the slowing at 54% (cUHDRS) and 68% (TFC).
The durability optics: alongside the four-year data, uniQure provided three-year data from 15 high-dose patients that looked stronger — 80% and 67% slowing on the two scales. Leerink analysts flagged that the external-control database gets updated over time and loses data as patients progress, but still wrote that slowing of any kind, "especially on TFC which has remained stable ... is absolutely unheard of," adding "AMT-130 is clearly doing something." Stifel's Paul Matteis called the four-year results "still impressive generally" despite "some regression," while the fresh three-year data "look robust" and are "arguably most important, since this data cut is the crux" of uniQure's alignment with FDA — and "the big picture here continues to support" accelerated approval.
Regulatory stakes: uniQure submitted its BLA late this summer after a "lengthy and highly unusual back and forth" with FDA, requesting a rapid review that could produce a verdict in about eight months. Leerink previously estimated a $1.7M U.S. price and $2.5B worldwide sales by 2030 if approved. The FDA's decision on the BLA is tracked as a standing watch on this topic.