uniQure (QURE) Guggenheim Securities Inaugural Healthcare Innovation Conference summary

Regulatory engagement and development plans

• Scheduled a multidisciplinary Type B meeting with the FDA for late November to align on development and communication plans, focusing on clinical, CMC, and safety aspects.

• Aims to gather FDA feedback to structure future interactions, with follow-up meetings expected in the first half of next year.

• Will disclose progress only after alignment with the FDA, likely providing updates around JPMorgan, but not through interim press releases.

• Next major data update planned for mid-2025, aligning with the availability of natural history data and regulatory milestones.

Clinical data and expert feedback

• Key opinion leaders express excitement over data from 29 patients, especially regarding functional composite endpoints and neurofilament light (NfL) reductions.

• NfL reduction of 10% is considered meaningful in Huntington’s, given the natural history shows a 25% increase over two years.

• Placebo-controlled studies are seen as impractical due to disease rarity and slow progression; external controls and functional endpoints are favored.

Study design and patient population

• Early intervention is preferred, with a focus on treating patients as early as possible, including pre-symptomatic individuals.

• Considering relaxing inclusion criteria such as minimal striatal volume for future studies.

• Exploring procedural improvements to make treatment more efficient, though not required for registration.