[424B5] uniQure N.V. Prospectus Supplement (Debt Securities)
uniQure N.V. is offering up to $200,000,000 of ordinary shares and pre-funded warrants, with ordinary shares listed on Nasdaq under the ticker QURE and a reported last sale price of $47.50 on September 24, 2025. The supplement highlights positive pivotal Phase I/II topline 36-month data for AMT-130 in Huntington's disease: a 75% slowing in disease progression by cUHDRS (p=0.003) and a 60% slowing by TFC (p=0.033) versus a propensity score‑matched external control, and the company plans a pre‑BLA meeting with the FDA and intends to submit a BLA in Q1 2026. Other programs: AMT-260 (MTLE) shows early seizure reduction signal; AMT-191 (Fabry) reports sustained GLA activity in cohort 1; AMT-162 (SOD1-ALS) enrollment is voluntarily paused after a dose‑limiting toxicity in cohort 2 while data are evaluated. The company disclosed a Dutch tax assessment related to a 2023 royalty financing upfront payment and expects a current tax liability in the mid single- to low double-digit millions. Financials show a net tangible book value deficit of $(114) million as of June 30, 2025; proceeds are intended for commercialization readiness, potential AMT-130 launch, development, and general corporate purposes.
- AMT-130 high-dose met primary endpoint: 75% slowing on cUHDRS (p=0.003) at 36 months versus external control
- Key secondary met: 60% slowing on TFC (p=0.033) supporting functional benefit
- Regulatory path engaged: Company plans pre-BLA meeting with FDA and intends BLA submission in Q1 2026
- Pipeline breadth: Positive early data for AMT-191 (Fabry) with sustained GLA activity and AMT-260 (MTLE) with early seizure reduction signal
- Capital raise: Up to $200,000,000 offering intended to fund commercialization readiness and development
- Enrollment pause in AMT-162 (SOD1-ALS) after a dose-limiting toxicity in cohort 2, creating program uncertainty
- Dutch tax assessment for the 2023 royalty financing upfront payment; estimated current tax liability in the mid single- to low double-digit millions
- Net tangible book deficit of $(114) million as of June 30, 2025, indicating dilution risk for new investors
- No public market for the pre-funded warrants and the company will not list them, limiting liquidity for warrant holders
Insights
TL;DR: AMT-130 36-month topline results demonstrate statistically significant functional benefit at high dose and support planned regulatory engagement.
The reported 36-month primary and key secondary outcome improvements versus a propensity score-matched external control are notable for a one-time gene therapy in Huntington's disease. A 75% slowing on cUHDRS (p=0.003) and 60% on TFC (p=0.033) at high dose meet prespecified analyses and, combined with supportive biomarker trends (CSF NfL reduction), provide a plausible basis for discussion with the FDA at a pre-BLA meeting. Safety appears manageable with no new drug-related SAEs since December 2022. Pooling U.S. and EU cohorts and use of Enroll-HD as an external control were pre-agreed with FDA, which reduces but does not eliminate regulatory risk.
TL;DR: Offering proceeds bolster runway, but material near-term risks include tax assessment, balance sheet deficit, dilution and a paused ALS trial.
The $200M shelf offering aims to fund commercialization and development but follows a net tangible book deficit of $(114)M as of June 30, 2025, implying dilution for new investors. The company disclosed a Dutch tax determination on the 2023 royalty financing upfront payment with an estimated liability in the mid single- to low double-digit millions, recorded in the quarter ended September 30, 2025. The voluntary pause in AMT-162 after a DLT introduces program-specific safety and timeline risk. These factors, combined with standard biotech execution and regulatory risks, merit careful investor consideration.
(To prospectus dated January 7, 2025)
Pre-Funded Warrants to Purchase Ordinary Shares
| | | |
Per ordinary
share |
| |
Per pre-funded
warrant |
| |
Total
|
| |||||||||
|
Public offering price
|
| | | $ | | | | | $ | | | | | $ | | | |||
|
Underwriting discounts and commissions(1)
|
| | | $ | | | | | $ | | | | | $ | | | |||
|
Proceeds to uniQure N.V. (before expenses)
|
| | | $ | | | | | $ | | | | | $ | | | | ||
| |
Leerink Partners
|
| | Stifel | | |
Guggenheim Securities
|
| |
Van Lanschot Kempen
|
|
| | | |
Page
|
| |||
|
ABOUT THIS PROSPECTUS SUPPLEMENT
|
| | | | S-ii | | |
|
SPECIAL CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
|
| | | | S-iv | | |
|
PROSPECTUS SUPPLEMENT SUMMARY
|
| | | | S-1 | | |
|
THE OFFERING
|
| | | | S-5 | | |
|
RISK FACTORS
|
| | | | S-7 | | |
|
USE OF PROCEEDS
|
| | | | S-11 | | |
|
DIVIDEND POLICY
|
| | | | S-11 | | |
|
DILUTION
|
| | | | S-12 | | |
|
DESCRIPTION OF PRE-FUNDED WARRANTS
|
| | | | S-14 | | |
|
MATERIAL DUTCH AND U.S. FEDERAL INCOME TAX CONSIDERATIONS
|
| | | | S-16 | | |
|
UNDERWRITING
|
| | | | S-27 | | |
|
LEGAL MATTERS
|
| | | | S-32 | | |
|
EXPERTS
|
| | | | S-33 | | |
|
WHERE YOU CAN FIND MORE INFORMATION
|
| | | | S-34 | | |
|
INCORPORATION OF CERTAIN INFORMATION BY REFERENCE
|
| | | | S-35 | | |
| | | |
Page
|
| |||
|
ABOUT THIS PROSPECTUS
|
| | | | 1 | | |
|
THE COMPANY
|
| | | | 2 | | |
|
RISK FACTORS
|
| | | | 3 | | |
|
SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
|
| | | | 4 | | |
|
USE OF PROCEEDS
|
| | | | 6 | | |
|
DESCRIPTION OF ORDINARY SHARES AND ARTICLES OF ASSOCIATION
|
| | | | 7 | | |
|
DESCRIPTION OF DEBT SECURITIES
|
| | | | 13 | | |
|
DESCRIPTION OF WARRANTS
|
| | | | 19 | | |
|
DESCRIPTION OF RIGHTS
|
| | | | 20 | | |
|
DESCRIPTION OF UNITS
|
| | | | 21 | | |
|
CONVERTIBLE OR EXCHANGEABLE SECURITIES
|
| | | | 22 | | |
|
FORM, EXCHANGE AND TRANSFER
|
| | | | 23 | | |
|
BOOK-ENTRY PROCEDURES AND SETTLEMENT
|
| | | | 24 | | |
|
PLAN OF DISTRIBUTION
|
| | | | 26 | | |
|
LEGAL MATTERS
|
| | | | 29 | | |
| EXPERTS | | | | | 30 | | |
|
WHERE YOU CAN FIND MORE INFORMATION
|
| | | | 31 | | |
|
INCORPORATION OF CERTAIN INFORMATION BY REFERENCE
|
| | | | 32 | | |
shares
| |
Public offering price per ordinary share
|
| | | | | | | | | $ | | | |
| |
Net tangible book value per ordinary share as of June 30, 2025
|
| | | $ | (2.08) | | | | | | | | |
| |
Increase per ordinary share attributable to new investors
|
| | | $ | | | | | | | | | |
| |
As adjusted net tangible book value per ordinary share as of June 30, 2025 after giving effect to this offering
|
| | | | | | | | | $ | | | |
| |
Dilution per ordinary share to new investors purchasing ordinary shares in this offering
|
| | | | | | | | | $ | | | |
|
Underwriter
|
| |
Number of
Ordinary Shares |
| |
Number of
Pre-Funded Warrants |
|
|
Leerink Partners LLC
|
| | | | | | |
|
Stifel, Nicolaus & Company, Incorporated
|
| | | | | | |
|
Guggenheim Securities, LLC
|
| | | | | | |
|
Van Lanschot Kempen (USA) Inc
|
| | | | | | |
|
Total
|
| | | | | | |
| | | | | | | | | | | | | | | |
Total
|
| |||||||||
| | | |
Per Share
|
| |
Per
Pre-Funded Warrant |
| |
Without
Option |
| |
With
Option |
| ||||||||||||
|
Initial public offering price
|
| | | $ | | | | | $ | | | | | $ | | | | | $ | | | ||||
|
Underwriting discounts and commissions
|
| | | $ | | | | | $ | | | | | $ | | | | | $ | | | ||||
|
Proceeds, before expenses, to us
|
| | | $ | | | | | $ | | | | | $ | | | | | $ | | | | |||
1 Hartwell Place
Lexington, MA 02421
Attn: Investor Relations
+1 339 970 7000
Warrants
Rights
Debt Securities
Units
| | | |
Page
|
| |||
|
ABOUT THIS PROSPECTUS
|
| | | | 1 | | |
|
THE COMPANY
|
| | | | 2 | | |
|
RISK FACTORS
|
| | | | 3 | | |
|
SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
|
| | | | 4 | | |
|
USE OF PROCEEDS
|
| | | | 6 | | |
|
DESCRIPTION OF ORDINARY SHARES AND ARTICLES OF ASSOCIATION
|
| | | | 7 | | |
|
DESCRIPTION OF DEBT SECURITIES
|
| | | | 13 | | |
|
DESCRIPTION OF WARRANTS
|
| | | | 19 | | |
|
DESCRIPTION OF RIGHTS
|
| | | | 20 | | |
|
DESCRIPTION OF UNITS
|
| | | | 21 | | |
|
CONVERTIBLE OR EXCHANGEABLE SECURITIES
|
| | | | 22 | | |
|
FORM, EXCHANGE AND TRANSFER
|
| | | | 23 | | |
|
BOOK-ENTRY PROCEDURES AND SETTLEMENT
|
| | | | 24 | | |
|
PLAN OF DISTRIBUTION
|
| | | | 26 | | |
|
LEGAL MATTERS
|
| | | | 29 | | |
| EXPERTS | | | | | 30 | | |
|
WHERE YOU CAN FIND MORE INFORMATION
|
| | | | 31 | | |
|
INCORPORATION OF CERTAIN INFORMATION BY REFERENCE
|
| | | | 32 | | |
1 Hartwell Place
Lexington, MA 02421
Attn: Investor Relations
+1 339 970 7000
Pre-Funded Warrants to Purchase Ordinary Shares
| |
Leerink Partners
|
| |
Stifel
|
| |
Guggenheim Securities
|
| |
Van Lanschot Kempen
|
|