uniQure: BLA submission in 3Q a major positive, upgrade to OW

Research evidence excerpt

uniQure: BLA submission in 3Q a major positive, upgrade to OW

ely the

FDA will reverse stance on communications to sponsors at this point. We upgrade the stock to

Price Performance Exchange-NasdaqOverweight and raise our price target to $65 from $25.

52 Week range USD 71.50-8.73

Specifically, yesterday QURE provided a regulatory update (see our note) indicating that at a

Type B meeting, the FDA communicated a path to accelerated approval (after alignment on

confirmatory study design). QURE plans to file BLA in 3Q for accelerated approval in

Huntington's; we note that with priority review this could put a US launch as early as

1H27. Notably, for the confirmatory study, it seems a sham control is off the table, which we also

view as a major positive.

Source: IDC

For QURE shares, we think the opportunity in Huntington's is significant (~$3-5B+ market). We Link to Barclays Live for interactive charting

think if there is a pathway to accelerated approval, we see upside into launch; we note QURE

shares at the ~$60/sh level in fall '25 when this was previously seen as viable. From the 3-year data, U.S. Small & Mid Cap Biotechnology

we think AMT-130 works, and see a major unmet need in this progressive, fatal disease. We note Eliana Merle, CFA

that while per QURE the FDA communicated a path to accelerated approval at the recent Type B +1 212 526 9445

meeting, we lack visibility into future regulatory decisions (confirmatory study alignment, eliana.merle@barclays.com

acceptance/approval of BLA) & some of the prior issues raised by FDA could become review issues. BCI, US

However, we note that prior to this update when there was greater uncertainty around the Jasmine Fels, PhD

regulatory pathway in Huntington's, shares were at the ~$27/sh level (close on 6/17). +1 212 526 0865