uniQure prospectus: $281M net proceeds, AMT-130 positive 36-month topline data
uniQure N.V. (QURE) is conducting a registered offering of 5,789,473 ordinary shares and, in lieu of shares to certain investors, 526,316 pre-funded warrants exercisable at $0.0001 per share. The prospectus supplement cites a Nasdaq last sale price of $52.65 on September 25, 2025. Estimated net proceeds are approximately $281.45 million (or $323.75 million if the underwriters' option is exercised) to fund commercialization readiness, a potential AMT-130 launch, development of other clinical candidates and general corporate purposes. Recent clinical updates include positive 36-month topline Phase I/II results for AMT-130: a statistically significant 75% slowing on cUHDRS (p=0.003) and 60% slowing on TFC (p=0.033) for the high-dose group, supportive biomarker change (CSF NfL -8.2%), and a planned pre-BLA meeting with the FDA ahead of an intended BLA submission in Q1 2026. Other programs: AMT-260 (GenTLE) reported a 92% seizure reduction in an early participant; AMT-191 showed sustained GLA activity in four patients; AMT-162 (SOD1-ALS) enrollment is voluntarily paused after a dose-limiting toxicity in one patient. Separately, Dutch tax authorities informed the company that a 2023 EUR 342 million royalty upfront should be treated as 2023 income for Dutch tax purposes; uniQure estimates a tax liability in the mid single- to low double-digit millions of dollars and will record a current tax liability in the quarter ended September 30, 2025.
Positive
- AMT-130 36-month topline efficacy: 75% slowing on cUHDRS (p=0.003) and 60% slowing on TFC (p=0.033) in high-dose group
- Supportive biomarker change: CSF NfL declined by 8.2% in treated patients, a supportive neurodegeneration marker
- Planned regulatory path: Pre-BLA meeting with FDA and intent to submit a BLA in Q1 2026 for AMT-130
- Additional positive clinical signals: AMT-260 reported a 92% seizure reduction in an early GenTLE participant; AMT-191 showed sustained GLA activity in four patients
- Capital raise to fund commercialization: Estimated net proceeds of ~$281.45M (or ~$323.75M with option) to support launch readiness and pipeline development
Negative
- Program safety pause: Voluntary pause in AMT-162 enrollment after a dose-limiting toxicity in one patient
- Dutch tax outcome: Tax authorities view the EUR 342M 2023 royalty upfront as taxable income in 2023, creating a current tax liability estimated in the mid single- to low double-digit millions of dollars
- Immediate dilution: New investors face immediate dilution of $44.74 per ordinary share at the stated public offering price
- Pre-funded warrant limitations: No public market for pre-funded warrants, limited liquidity, ownership-based exercise caps, and exercise will not materially fund the company
Insights
TL;DR: Offering funds commercialization and development; AMT-130 pivotal topline supports regulatory path but issuance causes meaningful near-term dilution.
The offering is sized to provide roughly $281M of net proceeds to support commercialization readiness and pipeline development, which materially extends cash resources. The reported AMT-130 36-month topline—75% slowing on cUHDRS (p=0.003) and 60% on TFC (p=0.033)—is a material clinical outcome that management intends to use in a BLA discussion and potential Q1 2026 submission. Key risks to valuation include immediate dilution to new investors (stated as $44.74 per share) and a Dutch tax position related to the 2023 royalty financing that produces a mid single- to low double-digit million dollar liability. Overall, clinical data improve the company’s regulatory and commercial outlook while the financing and tax items affect near-term capital structure.
TL;DR: AMT-130 36-month functional and biomarker signals are clinically meaningful; safety appears manageable but other programs show mixed operational status.
The AMT-130 high-dose group demonstrates statistically significant slowing on primary and key secondary functional endpoints at 36 months and supportive biomarker change (CSF NfL -8.2%), consistent with a dose-dependent effect. These results justify planned regulatory interactions. Early AMT-260 and AMT-191 signals are promising (92% seizure reduction in a single GenTLE participant; sustained GLA increases in four AMT-191 patients) but are limited by small sample sizes and early follow-up. The voluntary pause in AMT-162 after a dose-limiting toxicity introduces program-specific safety uncertainty and will require further data review. Overall, clinical efficacy signals are strong for AMT-130; other programs remain early-stage.
(To prospectus dated January 7, 2025)
Pre-Funded Warrants to Purchase 526,316 Ordinary Shares
| | | |
Per ordinary
share |
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Per pre-funded
warrant |
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Total
|
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Public offering price
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| | | $ | 47.50 | | | | | $ | 47.4999 | | | | | $ | 299,999,925 | | |
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Underwriting discounts and commissions(1)
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| | | $ | 2.85 | | | | | $ | 2.8500 | | | | | $ | 17,999,999 | | |
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Proceeds to uniQure N.V. (before expenses)
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| | | $ | 44.65 | | | | | $ | 44.6499 | | | | | $ | 281,999,926 | | |
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Leerink Partners
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| | Stifel | | |
Guggenheim Securities
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Van Lanschot Kempen
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Page
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ABOUT THIS PROSPECTUS SUPPLEMENT
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SPECIAL CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
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PROSPECTUS SUPPLEMENT SUMMARY
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| | | | S-1 | | |
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THE OFFERING
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| | | | S-5 | | |
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RISK FACTORS
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| | | | S-7 | | |
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USE OF PROCEEDS
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| | | | S-11 | | |
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DIVIDEND POLICY
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| | | | S-11 | | |
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DILUTION
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| | | | S-12 | | |
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DESCRIPTION OF PRE-FUNDED WARRANTS
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| | | | S-14 | | |
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MATERIAL DUTCH AND U.S. FEDERAL INCOME TAX CONSIDERATIONS
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| | | | S-16 | | |
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UNDERWRITING
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| | | | S-27 | | |
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LEGAL MATTERS
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| | | | S-32 | | |
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EXPERTS
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| | | | S-33 | | |
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WHERE YOU CAN FIND MORE INFORMATION
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| | | | S-34 | | |
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INCORPORATION OF CERTAIN INFORMATION BY REFERENCE
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| | | | S-35 | | |
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Page
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ABOUT THIS PROSPECTUS
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| | | | 1 | | |
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THE COMPANY
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RISK FACTORS
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SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
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| | | | 4 | | |
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USE OF PROCEEDS
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| | | | 6 | | |
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DESCRIPTION OF ORDINARY SHARES AND ARTICLES OF ASSOCIATION
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| | | | 7 | | |
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DESCRIPTION OF DEBT SECURITIES
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| | | | 13 | | |
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DESCRIPTION OF WARRANTS
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| | | | 19 | | |
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DESCRIPTION OF RIGHTS
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DESCRIPTION OF UNITS
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CONVERTIBLE OR EXCHANGEABLE SECURITIES
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| | | | 22 | | |
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FORM, EXCHANGE AND TRANSFER
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BOOK-ENTRY PROCEDURES AND SETTLEMENT
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PLAN OF DISTRIBUTION
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| | | | 26 | | |
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LEGAL MATTERS
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| EXPERTS | | | | | 30 | | |
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WHERE YOU CAN FIND MORE INFORMATION
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INCORPORATION OF CERTAIN INFORMATION BY REFERENCE
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shares
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Public offering price per ordinary share
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| | | | | | | | | $ | 47.50 | | |
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Net tangible book value per ordinary share as of June 30, 2025
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| | | $ | (2.08) | | | | | | | | |
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Increase per ordinary share attributable to new investors
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| | | $ | 4.84 | | | | | | | | |
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As adjusted net tangible book value per ordinary share as of June 30, 2025 after giving effect to this offering
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| | | | | | | | | $ | 2.76 | | |
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Dilution per ordinary share to new investors purchasing ordinary shares in this offering
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| | | | | | | | | $ | 44.74 | | |
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Underwriter
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Number of
Ordinary Shares |
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Number of
Pre-Funded Warrants |
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|
Leerink Partners LLC
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| | | | 2,431,579 | | | | | | 221,053 | | |
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Stifel, Nicolaus & Company, Incorporated
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| | | | 1,389,474 | | | | | | 126,316 | | |
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Guggenheim Securities, LLC
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| | | | 1,215,789 | | | | | | 110,526 | | |
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Van Lanschot Kempen (USA) Inc
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| | | | 578,947 | | | | | | 52,632 | | |
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H.C. Wainwright & Co., LLC
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| | | | 173,684 | | | | | | 15,789 | | |
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Total
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| | | | 5,789,473 | | | | | | 526,316 | | |
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Total
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Per Share
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Per
Pre-Funded Warrant |
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Without
Option |
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With
Option |
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Initial public offering price
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| | | $ | 47.50 | | | | | $ | 47.4999 | | | | | $ | 299,999,925 | | | | | $ | 344,999,905 | | |
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Underwriting discounts and commissions
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| | | $ | 2.85 | | | | | $ | 2.8500 | | | | | $ | 17,999,999 | | | | | $ | 20,699,998 | | |
|
Proceeds, before expenses, to us
|
| | | $ | 44.65 | | | | | $ | 44.6499 | | | | | $ | 281,999,926 | | | | | $ | 324,299,907 | | |
1 Hartwell Place
Lexington, MA 02421
Attn: Investor Relations
+1 339 970 7000
Warrants
Rights
Debt Securities
Units
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Page
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ABOUT THIS PROSPECTUS
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| | | | 1 | | |
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THE COMPANY
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| | | | 2 | | |
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RISK FACTORS
|
| | | | 3 | | |
|
SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS
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| | | | 4 | | |
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USE OF PROCEEDS
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| | | | 6 | | |
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DESCRIPTION OF ORDINARY SHARES AND ARTICLES OF ASSOCIATION
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| | | | 7 | | |
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DESCRIPTION OF DEBT SECURITIES
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| | | | 13 | | |
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DESCRIPTION OF WARRANTS
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| | | | 19 | | |
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DESCRIPTION OF RIGHTS
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| | | | 20 | | |
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DESCRIPTION OF UNITS
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| | | | 21 | | |
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CONVERTIBLE OR EXCHANGEABLE SECURITIES
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| | | | 22 | | |
|
FORM, EXCHANGE AND TRANSFER
|
| | | | 23 | | |
|
BOOK-ENTRY PROCEDURES AND SETTLEMENT
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| | | | 24 | | |
|
PLAN OF DISTRIBUTION
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| | | | 26 | | |
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LEGAL MATTERS
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| | | | 29 | | |
| EXPERTS | | | | | 30 | | |
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WHERE YOU CAN FIND MORE INFORMATION
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| | | | 31 | | |
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INCORPORATION OF CERTAIN INFORMATION BY REFERENCE
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| | | | 32 | | |
1 Hartwell Place
Lexington, MA 02421
Attn: Investor Relations
+1 339 970 7000
Pre-Funded Warrants to Purchase 526,316 Ordinary Shares
| |
Leerink Partners
|
| |
Stifel
|
| |
Guggenheim Securities
|
| |
Van Lanschot Kempen
|
|