QURE · uniQure N.V.

This disclosure represents a HIGH material regulatory milestone for uniQure’s lead pipeline asset AMT-130 in Huntington’s disease. The successful UK MHRA pre-submission meeting confirms alignment on the data package and manufacturing requirements needed to support a Marketing Authorization Application, which the company now expects to submit in Q3 2026. Critically, the filing explicitly anchors the MAA submission to the three-year analysis from the ongoing U.S. and European Phase I/II trials — not new data — and cites a statistically significant 75% slowing of disease progression at the high dose (p=.003) using the composite Unified Huntington’s Disease Rating Scale versus a propensity score-matched external control. While this topline efficacy signal is encouraging, the press release cautions that it is based on limited, non-randomized, externally controlled data, and the forward-looking statements section underscores substantial risks — including uncertainty whether regulators will accept the external control methodology or deem the endpoint robust enough for approval. The concurrent FDA Type B meeting scheduled for Q2 2026 to discuss Phase III design and the four-year analysis plan further highlights the pivotal nature of upcoming data and regulatory decisions. Importantly, no financial results, capital events, executive changes, or agreements are disclosed; the entire materiality derives from the timing, scope, and evidentiary basis of the impending MAA — a key inflection point for valuation and development risk assessment. Investors should monitor the Q3 2026 MAA submission and subsequent MHRA feedback, as well as any updates on the FDA meeting outcomes and four-year data timing.