QURE: The Wind At The FDA Has Shifted And We Would Be Buyers Into Strength

Research evidence excerpt

QURE: The Wind At The FDA Has Shifted And We Would Be Buyers Into Strength

uniQure N.V.

Target/Upside/Downside Scenarios Investment summary

We rate QURE Outperform, Speculative Risk. We continue touniQure N.V.

like the set-up despite the recent run now that QURE aligned

80 125 Weeks 25JAN24 - 17JUN26 with the FDA on a potential path to accelerated approval for

70 Huntington's disease post the Prasad and Makary departures

TARGETTARGET 65.0065.00

60 from the Agency. Huntington’s is high risk/reward, but we

50 CURRENTCURRENT 48.1648.16 like clarity on genetic root cause and QURE’s approach as: 1)

30 potentially one-and-done; 2) MoA targeting disease causing

20 exon 1; and 3) intrastriatal is invasive but ensures delivery to

10 the primary site of the pathology. We also like that this is a

0 company with a proven history of getting GTx over the finish

150m line (HEMGENIX first GTx approved for Hem-B and partnered

100m

50m with CSL in 2020 for $450 million upfront), and potential for

2024 2025 2026 J F M A M J J A S O N D J F M A M J J A S O N D J F M A M J material upside as the rest of the pipeline makes progress

QURE US Rel. S&P 500 COMPOSITE MA 40 weeks (MTLE enrollment progressing and promising early data for

Source: Bloomberg and RBC Capital Markets estimates for Target Fabry).

Valuation

Our $65 price target is based on a DCF analysis that assumes a FDA Alignment Leaves The Door Open On HD With Potential

10% WACC (same for all stocks in our coverage), 3% terminal Pathway To Accelerated Approval. QURE recently announced

growth rate (with a 0–3% range applied to our coverage that following their Type B meeting with the FDA, the Agency

depending on the relative maturity of the platforms), 100% has decided that the 3-year data from the Phase I/II study