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UNIQURE N.V.[QURE]

Date Filed : Jun 23, 2021

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EX-99.12tm2120374d1_ex99-1.htmEXHIBIT 99.1

Exhibit 99.1

 

 

uniQure to Acquire Corlieve Therapeuticsand Advance its
Gene Therapy Program to Treat Temporal Lobe Epilepsy (TLE)

 

~ Expands uniQure’s Pipelineof Innovative Gene Therapies to Treat Neurological Disorders ~

 

~ Strengthens uniQure’sGlobal Leadership in the Development of Gene Therapies that Employ miRNA 

Silencing Technology ~

 

~ Large Opportunity Targeting an Estimated1.3 million TLE Patients in the U.S. and Europe with
Approximately 800,000 Drug-Resistant Patients ~

 

~ Preclinical Proof-of-Concept Datafrom Disease Model and from Resected Hippocampi from Refractory TLE
Patients Demonstrate Clear Suppression of Chronic Spontaneous EpilepticSeizures ~

 

Lexington, MA; Amsterdam, the Netherlands;and Paris, France, June 22, 2021 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformativetherapies for patients with severe medical needs, today announced it has entered into a definitive agreement to acquire Corlieve Therapeuticsand its lead program, which will be known as AMT-260, to treat temporal lobe epilepsy, the most common form of focal epilepsy.

 

Corlieve’s lead gene therapy program employsmiRNA silencing technology to target suppression of aberrantly expressed kainate receptors in the hippocampus of patients with temporallobe epilepsy (TLE). TLE affects approximately 1.3 million people in the U.S. and Europe alone, of which approximately 800,000 patientsare unable to adequately control acute seizures with currently approved anti-epileptic therapies. Patients with refractory TLE experienceincreased morbidity, excess mortality, and poor quality of life.

 

AMT-260 was originally developed by Corlieve incollaboration with Christophe Mulle, Ph.D., CNRS Research Director, at the Interdisciplinary Institute of Neurosciences, CNRS, Universityof Bordeaux, Valerie Crepel, Ph.D., Inserm Research Director at the Institut de Neurobiologie de la Méditerranée, INSERM,Aix-Marseille University, and REGENXBIO Inc. Drs. Mulle and Crepel will continue their collaborations with uniQure.

 

“The acquisition of Corlieve provides anextraordinary opportunity to transform the lives of hundreds of thousands of patients around the world suffering from epilepsy and alignswith our vision of pursuing unmet medical needs for disorders that impact large populations and can be addressed with gene therapies directedto the CNS and liver,” stated Matt Kapusta, Chief Executive Officer of uniQure. “The groundbreaking work of the Corlieve team,in collaboration with Drs. Mulle and Crepel and others, has led to compelling preclinical results in temporal lobe epilepsy thatwe believe can strategically leverage uniQure’s leading position in developing and delivering gene therapies that employ miRNA silencingtechnology. We look forward to welcoming the Corlieve team into the uniQure family as we join forces to advance this important and potentiallytransformative therapy into clinical studies.”

 

“I am very proud of what Corlieve hasachieved in such a short amount of time. With the dedication and focus of our team and our collaboration partners, we have taken apromising therapeutic approach discovered by our scientific founders and created a potential transformative therapeutic opportunityfor patients with refractory TLE,” said Richard Porter, Ph.D., founderand Chief Executive Officer of Corlieve. “As leaders in the field for miRNA gene therapy for neurological conditions, uniQure isthe ideal long-term partner for us, and we look forward to working together to advance our program rapidly to the clinic for the benefitof the patients we serve.”

 

 

 

 

Upon the closing of the transaction, Dr. Porterwill assume the role at uniQure of General Manager of the Corlieve subsidiary.

 

“As a founding investor, we are delightedto see that the translational work conducted by Corlieve is recognized by uniQure, further reinforcing our commitment to company creationbased on partnering with the industry,” said Vanessa Malier, Managing Partner at Kurma Partners and Chairman of Corlieve.

 

Transaction Details

 

Under the terms of the agreement, uniQure willpay €46.3 million in an upfront payment of cash to acquire Corlieve.

 

Corlieve shareholders are eligible to receivethe following additional payments of which up to 25% will be payable in uniQure ordinary shares at uniQure’s election: up to €43.7million in development milestones through Phase I/II and €160 million in milestones associated with Phase III development and theapprovals of AMT-260 in the U.S and European Union.

 

Corlieve has an established license and collaborationagreement with REGENXBIO that includes an exclusive license to AAV9 for the specific genetic target of AMT-260. Under the license andcollaboration agreement, REGENXBIO received equity in Corlieve and is eligible to receive milestone payments and royalties on net salesof AMT-260.

 

The transaction has been approved by theBoards of both companies and does not require uniQure shareholder approval. The transaction is subject to customary closingconditions as well as review by the French Ministry of Economy, Finance and Recovery (Ministère de l’Economie, desFinances et de la Relance) pursuant to articles L.151-3 and R.151-1 and seq. of the French Code Monétaire et Financier.Currently, the transaction is anticipated to be completed early in the third quarter of 2021.

 

SVB Leerink LLC is acting as sole financial advisorand Morgan Lewis is acting as legal advisor to uniQure. McDermott Will & Emery is acting as legal advisor to Corlieve.

 

About Corlieve Therapeutics 

 

Corlieve Therapeutics is a biotechnology company focused on bringingnovel therapeutic options to patients with severe neurological disorders. The lead project is targeting aberrantly expressed kainatereceptors in the hippocampus of patients with refractory TLE using a gene therapy approach. Corlieve was founded by Kurma Partners 4Q2019on the basis of a partnership with REGENXBIO Inc., SATT Aquitaine Science Transfert, and Inserm Transfert. Corlieve is supported by itsinvestors and partners Kurma Partners, Eurazeo, Pureos Bioventures, SATT Aquitaine Science Transfert, Inserm Transfert, InsermCNRS, and REGENXBIO. For more information, please visit www.corlieve.com.

 

About uniQure 

 

uniQure is delivering on the promise of gene therapy – singletreatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipelineof proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type3 and other diseases. www.uniQure.com

 

 

 

 

uniQure Forward-Looking Statements

 

This press release contains forward-looking statements. Allstatements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements arebased 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, whether uniQure or Corlieve will advance the AMT-260 program tothe clinic rapidly or at all, and whether the transaction is completed early in the third quarter of 2021 or ever. uniQure’sactual results could differ materially from those anticipated in these forward-looking statements for many reasons, including,without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy andhealth care system, our risks associated with the integration of Corlieve and the execution of the AMT-260 development efforts,regulatory review of the transaction, our clinical development activities, clinical results, collaboration arrangements, regulatoryoversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factorsdescribed under the heading "Risk Factors" in uniQure’s Quarterly Report on Form 10-Q filed on May 10,2021. Given these risks, uncertainties, and other factors, you should not place undue reliance on these forward-looking statements,and we assume no obligation to update these forward-looking statements, even if new information becomes available in thefuture.

 

Corlieve Contacts:

 

Amy Conrad 

Juniper Point

858-366-3243

amy@juniper-point.com 

Or

contact@corlieve.com

 

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

 

 

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