uniQure NV Q1 FY2025 earnings call

• Pipeline Progress: Advancing AMT-130 towards a planned BLA submission, with recent interactions with the FDA on preparations for the BLA. Expanded clinical pipeline with initiation of studies in refractory temporal lobe epilepsy, Fabry disease, and SOD1-ALS.
• Clinical Updates: Completed enrollment of Cohort 3 in the Phase 1/2 trial of AMT-130, with positive safety data; ongoing enrollment in Fabry and SOD1-ALS studies; screening for TLE clinical study and expected preliminary results from the first patient dosed in the TLE study later in the month.
• Financials: Strong financial position with over $400 million in cash on hand as of March 31, 2025, resulting from divestiture of a manufacturing facility and a follow-on offering, providing flexibility to advance the pipeline and support clinical programs.

In the first quarter of 2025, uniQure's revenue was $1.6 million, a decrease of $6.9 million from the same period in 2024. This decline was mainly due to a $3.3 million drop in collaboration revenue and a $4 million decrease in contract manufacturing revenue. Research and development expenses were $36.1 million in the quarter, down from $40.7 million in 2024, primarily due to reductions in employee-related and facility expenses. Selling, general and administrative expenses were $10.9 million, down from $13.9 million in 2024, also due to reduced employee-related expenses. Cash, cash equivalents, and investment securities totaled $409 million as of March 31, 2025, up from $367.5 million at the end of 2024, primarily from the net proceeds of an $80.5 million follow-on offering in the first quarter.

• 2025 is anticipated to be a transformative year with multiple value-driving milestones, including presenting data from all clinical programs in the next 12 months and providing updates on AMT-130's regulatory interactions, including the planned timing of a BLA submission later in the quarter.
• Cash, cash equivalents, and investment securities are expected to be sufficient to fund operations into the second half of 2027.

• Uncertainty in regulatory outcomes, as actual results could differ materially from forward-looking statements due to factors in uniQure's most recent SEC filings.
• Risks associated with clinical trial outcomes, including potential challenges in enrolling patients, achieving study endpoints, or encountering safety issues with clinical programs.

Q: What is your view on the new CBER head Dr. Prasad and its impact on uniQure's interactions with the FDA regarding AMT-130?

A: Matt Kapusta stated he looks forward to working with Dr. Prasad, believes in the data supporting AMT-130, and notes that the approach for AMT-130 is based on clinical outcomes and a clinical measure as a primary endpoint, differentiating it from reliance on surrogate biomarkers.

Q: How confident are you on the three-year follow-up data on the cUHDRS for AMT-130?

A: Walid Abi-Saab stated they have no indication of losing efficacy over time, believe the dose-dependent reduction in cUHDRS observed at two years will be maintained at three years, and feel confident in the results going forward.

Q: Will there be additional regulatory interactions ahead of the BLA filing for AMT-130?

A: Matt Kapusta mentioned they've had constructive interactions with the FDA and will provide a regulatory update later in the quarter, but emphasized it's good housekeeping to wait for formal meeting minutes to fully understand and clarify interactions.

Q: Can you elaborate on the immunosuppression-related SAEs in Cohort 3 of AMT-130?

A: Walid Abi-Saab explained there were three serious adverse events related to immunosuppression (mania, MRSA infection, fever), all of which resolved with standard of care, and they believe a short two-week course of steroids is an appropriate immunosuppression strategy going forward.

Q: What is the current thinking on commercial plans and rollout for AMT-130?

A: Matt Kapusta stated they are excited about the potential in Huntington's disease, focused on regulatory interactions in the U.S. first, and will engage with European regulators later, with commercialization preparations underway and strategic interest from large biotechs and pharma companies.