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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): May 7, 2024

uniQure N.V.

(Exact Name of Registrant as Specified in Charter)

The Netherlands

  

001-36294

  

N/A

(State or Other
Jurisdiction of Incorporation)

(Commission
File Number)

(IRS Employer
Identification No.)

Paasheuvelweg 25a,
1105 BP Amsterdam, The Netherlands

  

N/A

(Address of Principal Executive Offices)

(Zip Code)

Registrant’s telephone number, including area code: +31-20-566-7394

(Former Name or Former Address, if Changed Since Last Report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class:

  

Trading Symbol(s)

  

Name of each exchange on which registered:

Ordinary Shares, par value €0.05 per share

QURE

The Nasdaq Stock Market LLC
The Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.

Item 2.02

Results of Operations and Financial Condition.

On May 7, 2024, uniQure N.V. (the “Company”) issued a press release announcing its financial results for the quarter ended March 31, 2024, and highlighting Company progress. A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.

The information furnished pursuant to this Item 2.02, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities under that section, and shall not be deemed to be incorporated by reference into any filing of the Company under the Securities Act of 1933, as amended, or the Exchange Act, except as shall be expressly set forth by specific reference in such filing.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits.

Exhibit No.

    

Description

99.1

Press Release of uniQure N.V. dated May 7, 2024

104

Cover Page Interactive Data File (embedded with the Inline XBRL document).

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.

 

UNIQURE N.V.

 

 

 

 

 

Date: May 7, 2024

By:

/s/ JEANNETTE POTTS

 

 

Jeannette Potts

 

 

Chief Legal and Compliance Officer

Graphic

Exhibit 99.1

uniQure Announces First Quarter 2024 Financial Results

and Highlights Recent Company Progress

~ On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024 ~

~ Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 ~

~ Comprehensive review of operations and options to reduce expenses underway and expected to be completed in mid-2024 ~

Lexington, MA and Amsterdam, the Netherlands, May 7, 2024 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the first quarter of 2024 and highlighted recent progress across its business.

“In the first quarter, we made solid progress across three key business priorities – clarifying the road ahead for AMT-130 in Huntington’s disease, initiating three new Phase I/II clinical trials, and prudently conserving our capital,” stated Matt Kapusta, chief executive officer of uniQure.  “We are on track to initiate FDA interactions on AMT-130 this quarter and look forward to presenting longer-term follow-up data from our Phase I/II trials mid-year, which will include up to three years of patient follow-up on 29 treated patients.  We have also made significant progress towards the initiation of new Phase I/II studies. We have obtained IRB approvals for initial sites, which are poised for imminent activation, and have also identified prospective patients. Enrollment for the Fabry disease study is on track to begin in the second quarter of 2024, with the SOD1-ALS and MTLE studies expected to begin in third quarter.”  

“In addition, we are actively engaged in a comprehensive review of uniQure’s operations and options to reduce expenses and increase shareholder value,” he added. “We believe deeply in the significant value of our assets, as well as the need to achieve a focused and sustainable level of investment in order to maximize this potential. We expect to complete this evaluation mid-2024.”

Recent Updates

Advancing AMT-130 for the treatment of Huntington’s disease

The Company is on track to initiate interactions with the U.S. Food and Drug Administration in the second quarter of 2024 with the goal of defining the future clinical and regulatory pathway for AMT-130. These interactions, which are expected to continue throughout the year, are expected to  include discussions of the interim data from the ongoing Phase I/II clinical trials and the potential to leverage natural history comparators alongside our long-term Phase I/II clinical data. The Company expects to provide an update on its regulatory plans as part of the next clinical update on AMT-130 mid-year and have greater clarity regarding a potential approval pathway for AMT-130 before the end of the year.

In mid-2024, the Company expects to provide an update from its ongoing Phase I/II clinical trials of AMT-130. The data will include up to three years of follow-up data on 29 treated patients in both

the low- and higher-dose cohorts, of which 21 patients will have a minimum of two years of follow-up.

Patient dosing is ongoing in a third cohort of up to 12 patients to further evaluate both doses of AMT-130 in combination with perioperative immunosuppression, with a focus on evaluating near-term safety and tolerability. Enrollment in this third cohort is expected to be completed in the second half of 2024.  

Initiating new Phase I/II clinical studies

AMT-191 for the treatment of Fabry disease - Patient enrollment in a Phase I/IIa clinical trial is expected to begin in the second quarter of 2024. The Phase I/IIa clinical trial will be a U.S.-based, multi-center, open-label trial consisting of two cohorts enrolling up to six adult male patients each. The study is designed to evaluate safety, tolerability, and early signs of efficacy.

AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – Patient enrollment in a Phase I/II clinical trial is expected to begin in the third quarter of 2024. The Phase I/II clinical trial will be a U.S.-based, multi-center, open-label trial consisting of three cohorts with up to four patients each receiving a one-time intrathecal infusion with immunosuppression. The study is designed to evaluate safety, tolerability, and early signs of efficacy.

AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE) – Patient enrollment in a Phase I/IIa clinical trial is expected to begin in third quarter of 2024. The Phase I/II will be a U.S.-based consisting of two parts. The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs for efficacy of AMT-260 in patients with refractory MTLE.  The second part is expected to be a randomized, controlled trial to generate proof of concept (POC) data.  

Upcoming Investor Events

RBC Capital Markets 2024 Global Healthcare Conference, May 15th – New York, NY

Financial Highlights

Cash position: As of March 31, 2024, the Company held cash and cash equivalents and investment securities of $555.7 million, compared to $617.9 million as of December 31, 2023. The Company expects cash, cash equivalents and investment securities will fund operations into the second quarter of 2027.  

Revenues: Revenue for the three months ended March 31, 2024 was $8.5 million, compared to $5.3 million in the same period in 2023. The net increase of $3.2 million in revenue resulted from an increase of $2.9 million from collaboration revenue and an increase of $1.2 million in license revenue partially offset by a decrease of $0.9 million from contract manufacturing of HEMGENIX®  for CSL.

Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were $9.1 million for the three months ended March 31, 2024, compared to $2.4 million for the same period in 2023. The increase primarily relates to expensing costs previously capitalized as inventory.  

R&D expenses: Research and development expenses were $40.7 million for the three months ended March 31, 2024, compared to $60.8 million during the same period in 2023. The $20.1 million decrease was primarily related to an $8.1 million decrease in external spend for the ALS SOD1 program, as prior year costs included a $10.0 million one-time cost for the acquisition of the program from Apic Bio. Additionally, there was a $7.0


million decrease in preclinical and other program spend, a $2.0 million decrease in costs incurred related to preclinical supplies and a $2.7 million decrease in employee related expenses.

SG&A expenses: Selling, general and administrative expenses were $13.9 million for the three months ended March 31, 2024, compared to $17.8 million during the same period in 2023. The $3.9 million decrease was primarily related to a $1.7 million decrease in professional and intellectual property fees, as well as a reduction of expenses for information technology.

Other non-operating items, net:

Other non-operating items, net was an expense of $10.7 million for the three months ended March 31, 2024, compared to $4.3 million for the same period in 2023. The $6.5 million increase in other non-operating items, net was primarily related to an increase in non-cash interest expense of $12.5 million related to the royalty agreement that the Company entered into in May 2023, which partially was offset by an increase of $4.8 million in interest income earned on investment securities and cash on hand.

Net loss:

The net loss for the three months ended March 31, 2024, was $65.6 million, or $1.36 basic and diluted loss per ordinary share, compared to $77.2 million net loss for the same period in 2023, or $1.63 basic and diluted loss per ordinary share.

About uniQure

uniQure’s mission is to reimagine the future of medicine by delivering innovative cures that transform lives.  The recent approvals of our gene therapy for hemophilia B – a historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. We are now leveraging our modular and validated technology and manufacturing platform to advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis (ALS), Fabry disease, and other severe diseases. www.uniQure.com

uniQure Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," “establish,” "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," “seek,” "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning the Company’s cash runway and its ability to fund its operations into the second quarter of 2027; the Company’s ongoing review of its operations and options to reduce expenses, the Company’s expectations that such review will lead to a strategy that, if effectively executed, will reduce operating expenses and increase shareholder value, and the expected timing of the completion of such review; the Company’s plans to announce additional follow-up data from its ongoing U.S. and European Phase I/II clinical studies of AMT-130; the Company’s plans to initiate interactions with the FDA regarding the further development of AMT-130, the timing of such interactions and expectations regarding regulatory clarity from such interactions; the Company’s plans regarding the third cohort in its AMT-130 clinical trial and the timing of enrollment for such cohort; and the Company’s plans to initiate patient enrollment for AMT-191 in the second quarter of 2024, and to initiate patient enrollment for AMT-260 and AMT-162 in the third quarter of 2024. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing


of the Company’s programs; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to approval; the Company’s ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; the Company’s ability to fund its operations and to raise additional capital as needed; and the impact of global economic uncertainty, rising inflation, rising interest rates or market disruptions on its business. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including its Annual Report on Form 10-K filed February 28, 2024 and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.

uniQure Contacts:

FOR INVESTORS:

FOR MEDIA:

Maria E. Cantor

Chiara Russo

Tom Malone

Direct: 339-970-7536

Direct: 617-306-9137

Direct: 339-970-7558

Mobile: 617-680-9452

Mobile: 617-306-9137

Mobile:339-223-8541

m.cantor@uniQure.com

c.russo@uniQure.com

t.malone@uniQure.com


uniQure N.V.

 

UNAUDITED CONSOLIDATED BALANCE SHEETS

March 31, 

December 31, 

2024

2023

(in thousands, except share and per share amounts)

Current assets

 

 

Cash and cash equivalents

$

243,062

$

241,360

Current investment securities

312,621

376,532

Accounts receivable and contract asset

10,717

4,193

Inventories

7,672

12,024

Prepaid expenses

18,839

15,089

Other current assets and receivables

3,092

2,655

Total current assets

596,003

651,853

Non-current assets

 

 

Property, plant and equipment, net

44,554

46,548

Operating lease right-of-use assets

27,695

28,789

Intangible assets, net

59,111

60,481

Goodwill

25,795

26,379

Deferred tax assets, net

11,594

12,276

Other non-current assets

5,298

5,363

Total non-current assets

174,047

179,836

Total assets

$

770,050

$

831,689

Current liabilities

Accounts payable

$

5,231

$

6,586

Accrued expenses and other current liabilities

22,658

30,534

Current portion of contingent consideration

27,587

28,211

Current portion of operating lease liabilities

7,997

8,344

Total current liabilities

63,473

73,675

Non-current liabilities

Long-term debt

102,120

101,749

Liability from royalty financing agreement

405,398

394,241

Operating lease liabilities, net of current portion

26,983

28,316

Contingent consideration, net of current portion

14,625

14,795

Deferred tax liability, net

7,376

7,543

Other non-current liabilities

3,321

3,700

Total non-current liabilities

559,823

550,344

Total liabilities

623,296

624,019

Shareholders' equity

 

 

Total shareholders' equity

146,754

207,670

Total liabilities and shareholders' equity

$

770,050

$

831,689


uniQure N.V.

 

UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS

Three months ended March 31,

2024

2023

(in thousands, except share and per share amounts)

Total revenues

$

8,485

$

5,325

Operating expenses:

Cost of license revenues

(150)

Cost of contract manufacturing revenues

(9,076)

(2,435)

Research and development expenses

(40,692)

(60,809)

Selling, general and administrative expenses

(13,937)

(17,848)

Total operating expenses

(63,855)

(81,092)

Other income

1,376

1,811

Other expense

(234)

(216)

Loss from operations

(54,228)

(74,172)

Non-operating items, net

(10,734)

(4,262)

Loss before income tax (expense) / benefit

$

(64,962)

$

(78,434)

Income tax (expense) / benefit

(656)

1,207

Net loss

$

(65,618)

$

(77,227)

 

 

 

Basic and diluted net loss per ordinary share

$

(1.36)

$

(1.63)

Weighted average shares used in computing basic and diluted net loss per ordinary share

48,384,510

47,436,335


v3.24.1.u1
Document and Entity Information
May 07, 2024
Document and Entity Information [Abstract]  
Document Type 8-K
Document Period End Date May 07, 2024
Entity File Number 001-36294
Entity Registrant Name uniQure N.V.
Entity Incorporation, State or Country Code P7
Entity Tax Identification Number 00-0000000
Entity Address, Address Line One Paasheuvelweg 25a
Entity Address, City or Town Amsterdam
Entity Address, Postal Zip Code 1105 BP
City Area Code +31
Local Phone Number 20-566-7394
Title of 12(b) Security Ordinary Shares, par value €0.05 per share
Trading Symbol QURE
Security Exchange Name NASDAQ
Entity Emerging Growth Company false
Entity Central Index Key 0001590560
Amendment Flag false
Entity Address, Country NL
Written Communications false
Soliciting Material false
Pre-commencement Tender Offer false
Pre-commencement Issuer Tender Offer false

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