QURE: Highlights from the RBC Healthcare Conference

研报英文原文证据摘录

QURE: Highlights from the RBC Healthcare Conference

EQUITY RESEARCH QUICK TAKE

RBC Capital Markets, LLC

Luca Issi, Ph.D. (Analyst)

Lisa Walter, Ph.D. (Analyst)

Shelby Hill, Ph.D. (Senior Associate)

Cassie Yuan, Ph.D. (Senior Associate)

May 19, 2026

uniQure N.V.

Highlights from the RBC Healthcare Conference

NASDAQ: QURE | USD 24.93 | Outperform | Speculative Risk | Price Target USD 35.00

Sentiment: Neutral

We hosted QURE’s Matt Kapusta (CEO) for a fireside chat. Our top takeaways include: 1) QURE remains deeply convicted in the data

and reiterates Type A meeting feedback was centered on the lack of pre-specification; (2) the upcoming Type B meeting will cover

the SAP and control protocol with the 4-year data analysis; (3) QURE emphasizes that the ex-US opportunity is more meaningful

than appreciated, with a successful pre-submission meeting with the UK MHRA and plans to submit an MAA based on 3-year data

in 3Q26. We reiterate our Outperform, Speculative Risk rating.

Type B Meeting For HD Upcoming - Recall, FDA feedback from Type A meeting suggested that the Agency was not willing to approve

AMT-130 based on comparison to natural history and is recommending a prospective, randomized, double-blind, sham-controlled

study. QURE has since been granted a Type B meeting in 2Q26 to discuss a new clinical trial design and the SAP with the 4-year data

expected in 3Q26. Mgmt noted that the FDA was not dismissive of the clinical data itself given the progressive improvement in

cUHDRS at 1, 2, and 3 years, but that the analysis was not prespecified. QURE notes that on one hand, such requirement would not

have been achievable given trial initiated back in 2018 when natural history data pool was largely immature; on the other hand,