# EDGAR Filing Document

**Accession Number:** 0001590560
**File Stem:** 0001104659-23-008609
**Filing Date:** 2023-1
**Character Count:** 14345
**Document Hash:** d6f2b8355d516beaa831579e4a96eac6
**Contains OCR:** False
**Source Format:** 

## Filing Content

## Filing Summary
**0001104659-23-008609.hdr.sgml**: 20230131

**ACCESSION NUMBER**: 0001104659-23-008609

**CONFORMED SUBMISSION TYPE**: 8-K

**PUBLIC DOCUMENT COUNT**: 14

**CONFORMED PERIOD OF REPORT**: 20230131

**ITEM INFORMATION**: Other Events

**ITEM INFORMATION**: Financial Statements and Exhibits

**FILED AS OF DATE**: 20230131

**DATE AS OF CHANGE**: 20230131

**FILER**: 

**COMPANY DATA:**
- **COMPANY CONFORMED NAME:** uniQure N.V.
- **CENTRAL INDEX KEY:** 0001590560
- **STANDARD INDUSTRIAL CLASSIFICATION:** PHARMACEUTICAL PREPARATIONS [2834]
- **IRS NUMBER:** 000000000
- **FISCAL YEAR END:** 1231

**FILING VALUES:**
- **FORM TYPE:** 8-K
- **SEC ACT:** 1934 Act
- **SEC FILE NUMBER:** 001-36294
- **FILM NUMBER:** 23572739

**BUSINESS ADDRESS:**
- **STREET 1:** PAASHEUVELWEG 25A
- **CITY:** AMSTERDAM
- **STATE:** P7
- **ZIP:** 1105 BP
- **BUSINESS PHONE:** 1-339-970-7000

**MAIL ADDRESS:**
- **STREET 1:** PAASHEUVELWEG 25A
- **CITY:** AMSTERDAM
- **STATE:** P7
- **ZIP:** 1105 BP

**FORMER COMPANY:**
- **FORMER CONFORMED NAME:** uniQure B.V.
- **DATE OF NAME CHANGE:** 20131030

?xml version="1.0" encoding="utf-8"?

**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): **January 31, 2023**

**uniQure N.V.**

(Exact Name of Registrant as Specified in Charter)

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| | | |
|:---|:---|:---|
| **The Netherlands** | **001-36294** | **N/A** |
| (State or Other<br> Jurisdiction of Incorporation) | (Commission<br> File Number) | (IRS Employer<br> Identification No.) |

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| | |
|:---|:---|
| **Paasheuvelweg 25a,**<br> **1105 BP Amsterdam, The Netherlands** | &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;**N/A** |
| (Address of Principal Executive Offices) | &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;(Zip Code) |

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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):

◻&nbsp;&nbsp;&nbsp;&nbsp; Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

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

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

◻&nbsp;&nbsp;&nbsp;&nbsp; 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:

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| | | |
|:---|:---|:---|
| **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<br> The Nasdaq Global Select Market |

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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. ◻

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| | |
|:---|:---|
| **Item 8.01** | **Other Events.** |

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On January 31, 2023, uniQure N.V. issued a press release announcing its execution of a global licensing agreement with Apic Bio. A copy of the press release is attached to this Current Report on Form 8-K as Exhibit 99.1 and incorporated herein by reference.

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| | |
|:---|:---|
| **Item 9.01** | **Financial Statements and Exhibits.** |

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(d)&nbsp;&nbsp;&nbsp;&nbsp; Exhibit

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| | |
|:---|:---|
| Exhibit No. | Description |
| [99.1](tm234964d1_ex99-1.htm) | [Press Release of uniQure N.V. dated January 31, 2023 announcing its execution of a global licensing agreement with Apic Bio.](tm234964d1_ex99-1.htm) |
| 104 | Cover Page Interactive Data File (embedded with the Inline XBRL document). |

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**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.

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| | | |
|:---|:---|:---|
|  | **UNIQURE N.V.** | **UNIQURE N.V.** |
| Date: January 31, 2023 | By: | /S/ DAVID J. CERVENY |
|  |  | David J. Cerveny |
|  |  | Chief Legal Officer |

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## Exhibit 99.1

**Exhibit 99.1**

**uniQure and Apic Bio enter into global licensing agreement for APB-102,** 

**a clinical stage gene therapy for patients with ALS caused by mutations in SOD1**

~ *License of APB-102 further strengthens uniQure's pipeline of innovative gene therapies to treat neurological disorders and miRNA-based gene silencing programs ~*

 

*~APB-102 and uniQure's c9orf72-ALS program have the potential to* 

*address most inherited forms of ALS ~*

 

*~ uniQure plans to initiate a Phase I/II trial of APB-102 in the second half of 2023 ~*

**Lexington, MA, Amsterdam, the Netherlands, and Cambridge, MA,** January 31, 2023 — <u>uniQure N.V</u>. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, and Apic Bio, an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, today announced that they have entered into a global licensing agreement for APB-102 to treat superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. Under the agreement, uniQure acquires global rights for the development and commercialization of APB-102, adding to its pipeline of gene therapies to treat neurological disorders. The U.S. Food and Drug Administration has cleared the investigational new drug (IND) application for APB-102 and has granted Orphan Drug and Fast Track designations.

APB-102 is designed to be a novel, one-time, intrathecally administered gene therapy for ALS caused by mutations in SOD1, a rapidly progressing, rare motor neuron disease that leads to loss of everyday functions and is uniformly fatal. Mutations in the SOD1 gene of ALS account for approximately one-fifth of all inherited forms of this fatal disease<sup>1</sup>. APB-102 is comprised of a recombinant AAVrh10 vector that expresses a micro ribonucleic acid (miRNA) designed to knock down the expression of SOD1 with the goal of slowing down or potentially reversing the progression of ALS in patients with SOD1 mutations.

"The licensing of APB-102 provides uniQure with another clinical stage program that is strategically aligned with our current pipeline and highly complementary with our AMT-161 program for the treatment of ALS caused by mutations in the c9orf72 gene," stated Ricardo Dolmetsch, Ph.D. president of research and development at uniQure. "Together, these ALS gene therapy candidates have the potential to address most familial forms of ALS and transform the lives of thousands of patients around the world suffering from this devasting disease. We look forward to initiating a Phase I/II clinical study of APB-102 in the second half of 2023."

The clinical development of APB-102 is based on nearly 30 years of research demonstrating the link between the SOD1 gene mutation and ALS. Preclinical studies in a SOD1-ALS mouse model demonstrated that APB-102 greatly enhanced survival in affected mice. Relevant SOD1 reduction in spinal cord motor neurons also was demonstrated in rodents, as well as in non-human primates at proposed clinical doses.

"I am very proud of the contributions Apic Bio has made to bring APB-102 to the cusp of clinical development," stated John Reilly, co-founder and chief executive officer of Apic Bio. "uniQure is at the forefront of the field of miRNA gene therapies for neurological disorders and is the ideal partner to achieve the goal of rapidly advancing the clinical development of APB-102 for the potential benefit of SOD1-ALS patients."

Under the terms of the agreement, uniQure will make an initial cash payment of $10 million. In addition, UniQure will pay Apic Bio up to $45 million in milestones upon achievement of regulatory approvals in the U.S. and Europe and pre-specified annual net sales, and a tiered royalty on net sales ranging from the mid-single digits to low double digits.

SVB Securities acted as exclusive financial advisor to uniQure in the transaction.

Torreya Partners acted as exclusive financial advisor to Apic Bio in the transaction.

**About SOD1-ALS**

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by loss of motor neurons, leading to muscle weakness and eventual paralysis. Most patients face mortality within five years of disease onset due to respiratory failure<sup>2</sup>. ALS can be caused by multiple genetic mutations and can be sporadic (spontaneous mutations) or familial (inherited mutations). Familial mutations account for approximately ten percent of ALS cases, and of these, approximately twenty percent are linked to a mutation in the SOD1 gene that codes for the enzyme superoxide dismutase 1<sup>1</sup>. SOD1-linked ALS is most likely caused by toxic mutant forms of the superoxide dismutase 1 (SOD1) protein (a gain-of-function mutation)<sup>1</sup>. Current approved ALS treatments only delay disease progression without addressing the underlying genetic causes of the disease.

**About uniQure** 

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The recent approval of our gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represents 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 platform to rapidly advance a <u>pipeline</u> of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. <u>www.uniQure.com</u>

**About Apic Bio**

Apic Bio is an innovative gene therapy company focused on developing first-in-class treatment options for rare, undertreated neurological and liver diseases. The Company's lead program to date has been an adeno-associated (AAV)-based gene therapy for the treatment of SOD1 ALS. Preclinical studies of additional genetic forms of ALS (C9orf72) and Alpha-1 Antitrypsin Deficiency (Alpha-1) are ongoing. The Company is also advancing discovery programs for two undisclosed CNS indications that leverage its proprietary silence and replace THRIVE™ platform. The Company is backed by leading and disease-centric investors, including Morningside Venture Investments, ALS Investment Fund, and The Alpha-1 Project (TAP). For more information, please visit www.apic-bio.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. These forward-looking statements include, but are not limited to, statements about whether uniQure's ALS gene therapy candidates have the potential to address most familial forms of ALS or transform the lives of thousands of patients around the world, whether uniQure will initiate a Phase I/II clinical study of APB-102 in the second half of 2023, and whether uniQure will be able to rapidly advance the clinical development of APB-102 for the potential benefit of SOD1-ALS patients. The Company's actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the regulatory approval and commercial launch of HEMGENIX<sup>®</sup>, our clinical trial for Huntington's disease, the impact of financial and geopolitical events on our Company and the wider economy and health care system, our Commercialization and License Agreement with CSL Behring, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in the Company's periodic securities filings, including its Annual Report on Form 10-K filed February 25, 2022. 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.*

**References:**

1. Brown CA, Lally C, Kupelian V, Flanders WD. Estimated Prevalence and Incidence of Amyotrophic Lateral Sclerosis and SOD1 and C9orf72 Genetic Variants. Neuroepidemiology. 2021

&nbsp;&nbsp;&nbsp;&nbsp;2. Brown RH, Al-Chalabi A. Amyotrophic Lateral Sclerosis. N Engl J Med. 2017 Jul 13

**uniQure Contacts:**

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| | | |
|:---|:---|:---|
| **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 |
| <u>m.cantor@uniQure.com</u> | <u>c.russo@uniQure.com</u> | <u>t.malone@uniQure.com</u> |

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**Apic Bio Contacts:**

**FOR INVESTORS/MEDIA:**

David Rosen

<u>david.rosen@argotpartners.com</u>

646.461.6387