The U.S. Food and Drug Administration communicated on January 30, 2026 that uniQure’s Phase I/II data for AMT‑130 were insufficient to support a marketing application, and the company announced the decision publicly on March 2, 2026. The FDA recommended a new randomized, double‑blind, sham‑surgery controlled Phase III trial to establish efficacy and safety.
The decision follows a 2024 agreement that the Phase I/II protocol would use an external control derived from the Enroll‑HD natural history dataset. UniQure had believed the data would support a marketing application, but the FDA found the comparison to the external control inadequate.
UniQure’s Q4 2025 earnings showed an EPS of $‑0.56, beating the consensus estimate of $‑0.93 by $0.37, a 39.9% beat. The company’s revenue of $5.57 million fell short of the $10.35 million estimate, a miss of $4.78 million or 46.2%. Revenue declined from $16.1 million in 2025 to $27.1 million in 2024, reflecting a slowdown in collaboration and contract manufacturing income.
Management emphasized continued dialogue with the FDA. Matt Kapusta said, “While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting.” He added, “We remain committed to engaging with the FDA to determine a clear, scientifically grounded, and efficient path forward for AMT‑130.” He also noted, “We are deeply grateful for the resilience and support of the Huntington’s disease community and remain committed to standing with patients and their families as we advance this potentially transformative therapy for a community in need.”
The new trial requirement is expected to add several years to the approval timeline and increase development costs, potentially eroding the projected $40 billion lifetime revenue for AMT‑130. UniQure reported $622.5 million in cash, cash equivalents, and investments at the end of 2025, which the company says supports operations into the second half of 2029. Guidance for the next quarter projects an EPS of $‑0.90 and a full‑year EPS of $‑3.32, reflecting the company’s cautious outlook amid the regulatory setback.
Investors reacted negatively to the announcement, citing the FDA’s rejection of the Phase I/II data and the requirement for a new Phase III trial as the primary driver of the market’s response.
The content on EveryTicker is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.