uniQure N.V. (QURE) Earnings

Management highlights

### Regulatory Progress for Lead Program AMT-130 (Huntington's Disease) - Reached alignment with the FDA at a June 2026 Type B meeting that 3-year Phase 1-2 data is an acceptable primary basis for a Biologics License Application (BLA) under the accelerated approval pathway, with alignment confirmed in final meeting minutes. - FDA alignment confirms that a sham-controlled confirmatory trial is no longer required; the agency instead recommends a randomized standard-of-care controlled trial with Total Functional Capacity (TFC) at 36 months as the primary endpoint. - BLA submission to the FDA and regulatory submission to the UK MHRA are both on track for Q3 2026; broader engagement with the EMA is planned for 2027. - 4-year AMT-130 Phase 1-2 data analysis is on track for presentation in September 2026, using the updated 26,000-participant EnrollHD natural history control database. ### Clinical Pipeline Progress - **AMT-260 (refractory mesial temporal lobe epilepsy):** Initial low-dose cohort Phase 1-2 data readout presented in Q2 2026: 3 of 6 patients achieved 79% to 100% reductions in disabling seizures; no treatment-related serious adverse events, and no immunosuppression required. Enrollment in the higher-dose cohort is expected to complete imminently, with updated results expected in H1 2027. - **AMT-191 (Fabry disease):** All 11 patients across 3 dose levels experienced dose-dependent increases in alpha-gal-A activity, with stable plasma lyso-GB3 levels and all patients remaining withdrawn from enzyme replacement therapy (ERT). Two Grade 3 liver enzyme elevations in the mid-dose cohort have resolved after immunosuppression; additional dosing in mid/high-dose cohorts remains paused pending FDA agreement on a new monitoring plan. Further updates are expected in H1 2027. ### Commercial Preparation - Core priorities for the potential AMT-130 launch include: (1) ongoing deep engagement with Huntington's Disease Centers of Excellence in the U.S. and UK to prepare for procedures; (2) patient and provider community education and care pathway development; (3) payer engagement supported by a robust health economics and outcomes research program. - Pre-approval early access programs are planned for additional geographies (Middle East, Latin America, Central and Eastern Europe) if approval is secured in the U.S. or UK. ### Financial Position - As of June 30, 2026, cash, cash equivalents, and investment securities totaled $810.3 million, up from $622.5 million at December 31, 2025, following a successful follow-on offering that strengthened the balance sheet.

Guidance

- Management expects current cash reserves will be sufficient to fund all planned operations, including the confirmatory trial for AMT-130, potential commercial launch preparation, ongoing pipeline clinical trials, and advancement of other pipeline candidates into late-stage development, into 2030. - BLA and UK MHRA submissions for AMT-130 are confirmed on track for Q3 2026, with 4-year data readout expected in September 2026. - Updated clinical data for AMT-260 and AMT-191 is expected to be released in the first half of 2027. - Full engagement with the EMA for AMT-130 is planned to begin in 2027. - Management expects a reasonable chance of FDA approval of priority review for AMT-130, given the program holds Breakthrough Therapy, RMAT, and Fast Track designations, with a final decision from the FDA pending BLA submission.

Segment performance

Unicure is a clinical-stage biotechnology company focused on gene therapy development, with no commercial product segments as of Q2 2026. All revenue is derived from licensing activities: Q2 2026 total revenue was $5.8 million, a $0.5 million increase compared to the prior year period. Total operating expenses for Q2 2026 were $51.4 million, consisting of: 1) Research and Development (R&D) expenses: $34 million (66.1% of total operating expenses), a $1.4 million decrease from Q2 2025. The decrease stemmed from lower facility, labor, and contingent consideration fair value costs, partially offset by higher direct spending on pipeline programs AMT-260, AMT-162, and AMT-191, and lower spending on lead program AMT-130. 2) Selling, General and Administrative (SG&A) expenses: $17.4 million (33.9% of total operating expenses), a $3.9 million increase from Q2 2025. The increase was driven by higher labor costs for new commercial team hires to support the potential AMT-130 launch, plus higher intellectual property and IT costs, partially offset by lower professional fees.

Risks & headwinds

- Final confirmatory trial design is still being finalized with the FDA, and failure to reach alignment on study design could delay BLA submission or approval. - Retention and recruitment in the global confirmatory trial for AMT-130 carries inherent risk, including potential dropout rates and slower-than-expected enrollment in geographies where the product is not yet commercially available. - If the FDA determines the confirmatory trial is not feasible or not sufficiently advanced at the time of BLA review, it may withhold accelerated approval. - The FDA may still request an Advisory Committee (adcom) meeting for the AMT-130 BLA, where external advisors could raise concerns about efficacy or safety that impact the approval decision. - AMT-191 clinical development is currently paused for mid and high-dose cohorts pending FDA agreement on a revised safety monitoring plan, which could delay overall program progress. - Commercial launch of AMT-130 faces operational risks related to variable institutional workflows across treatment centers, which could impact initial patient throughput and uptake.

Analyst Q&A

• Q: What role will four-year AMT-130 data play in the BLA review, and how quickly can the confirmatory trial be operationalized?

  A: The BLA submission is self-contained based on the agreed three-year data, so the four-year data will only be included if the FDA specifically requests it for the review or an adcom. Preparatory work for the global confirmatory trial is already complete: the team will prioritize recruitment in countries where AMT-130 is not yet commercially available to hit enrollment timelines, and management is confident the trial can be completed on schedule. Final sample size is still pending FDA alignment and has not been disclosed.

• Q: What operational bottlenecks for AMT-130 delivery have you identified at treatment centers, and how are you addressing them for launch?

  A: The team has learned that institutional workflows vary widely across centers, so they are individually mapping processes across all involved specialties (neurology, neurosurgery, etc.) at each planned center ahead of potential approval. Management does not see these workflow differences as an unresolvable bottleneck, and is working to ensure centers are ready to treat patients as quickly as possible after approval. Initial center capacity per site is still being finalized, and more details will be shared in the coming months.

• Q: Is accelerated approval for AMT-130 dependent on completing confirmatory trial enrollment before approval? What does the 2030 cash runway include?

  A: Accelerated approval does not require completed enrollment; the FDA only requires that the trial can be completed in a timely manner post-approval, and management is confident it can meet this requirement. The 2030 cash runway includes funding for the confirmatory trial, commercial launch preparations, all ongoing pipeline clinical trials, and potential investment to advance other pipeline candidates into late-stage development.

• Q: Given recent skeptical FDA briefing documents for other gene therapy adcoms, do you have concerns about FDA pushback on AMT-130?

  A: Every program is evaluated on its own individual merits, data, and patient population, so it is not appropriate to draw comparisons to other programs. Management notes that interactions with the FDA have been consistently constructive, and the data for AMT-130 speaks for itself. If an adcom is requested, Unicure welcomes the opportunity to present and discuss the data.

• Q: What are key unique characteristics of the AMT-130 launch to consider, and are there relevant launch analogs?

  A: Key priorities are having the right number of prepared treatment centers, securing payer understanding of unmet need and treatment value, and fully mapping patient referral and care pathways. Zolgensma (a one-time gene therapy delivered via hospital procedure) and Elivie are reasonable general capacity analogs, though no analog is perfect, as every disease and therapy has unique market dynamics.