Wow! QURE to File a BLA After All

Research evidence excerpt

Wow! QURE to File a BLA After All

Biotechnology

uniQure N.V. (QURE)

EQUITY RESEARCH Company Update

June 17, 2026

Wow! QURE to File a BLA After All Price: $26.99

Price Target: $61.00 (From $18.00) What Happened?

Rating: Overweight (From Neutral)

●Today (6/17), uniQure announced plans to submit a BLA for Key Statistics: accelerated approval for AMT-130 (AAV-gene therapy) in Huntington's Symbol NSQ: QURE

52-Week Range 8.73 - 71.50 disease (HD) in 3Q26.

Market Cap 1,702.2 ●This is based on the previously disclosed three-year analysis data from ADV (3 mo) 1,760,257

Shares Out (M) 63.1 the Phase 1/2 study.

●This comes after the recent Type B meeting conducted with the FDA,

Research Analysts: where the company expects to receive final minutes within 30 days.

Kristen Kluska

212-915-1927 ●The key remaining step is to align on the confirmatory study design

Kristen.Kluska@cantor.com prior to submitting, where the company is guiding to consideration of

Ross Fladeland a concurrent control on SoC therapy vs. a sham procedure. 212-915-1803

ross.fladeland@cantor.com ●Let us know if you want our raw notes from our discussion with

Ayan Hussein, PhD management. 212-915-1122

Ayan.Hussein@cantor.com ●We are upgrading to OW from N and increasing our 12-mo PT to $61

from $18; more details on this below. REV ($M)

FYE Dec 2025A 2026E 2027E

1Q $1.6 $3.6A $2.5 So, What Changed? What Did We Learn From Our Discussion with

2Q $5.3 $2.3E $2.5 Management?

3Q $3.7 $2.4E $6.9

Prev - - $2.5 ●The team doesn't know exactly what happened inside the FDA in

4Q $5.6 $4.6E $20.2 terms of actual discussions, but they suspect there were really serious Prev - - $2.5

Year $16.1 $12.9E $32.1 discussions amongst senior leadership and the review team around

Prev - - $10.0 their situation.