uniQure reaches FDA alignment on AMT-130 BLA submission; securities class action filed

Filed July 29, 2026 · Period ending June 30, 2026 · Compared to 10-Q Jul 29, 2025 · ~2 min read

Key Changes

• high

  FDA confirmed in July 2026 that a BLA submission under accelerated approval for AMT-130 is reasonable based on existing clinical data, but now requires alignment on confirmatory study design before submission and expects the confirmatory trial to be well underway or fully enrolled at approval time. Company expects Q3 2026 BLA submission.

  Risk Factors: AMT-130 regulatory pathway verify on EDGAR →
• high

  Securities class action filed February 2026 alleging false/misleading statements about AMT-130 study data and BLA filing timing during September-October 2025. Amended complaint filed July 2026; company has not accrued any liability, stating loss is not probable or reasonably estimable. Motion to dismiss due September 2026.

  Legal Proceedings verify on EDGAR →
• high

  June 2026 equity raise added $242.7M net proceeds at $45.50/share (168% higher price than January 2025 raise), extending cash runway from second half of 2027 to into 2030. Total cash and investments increased 114% to $810.3M.

  MD&A: Liquidity verify on EDGAR →