uniQure signs EU commercialization agreement with Chiesi Farmaceutici for first approved gene therapy treatment, and announces EUR 45 million (USD 58 million) in equity and collaboration financing

uniQure signs EU commercialization agreement with Chiesi Farmaceutici for first approved gene therapy treatment, and announces EUR 45 million (USD 58 million) in equity and collaboration financing

Amsterdam, the Netherlands, July 9, 2013 — uniQure B.V., a leader in human gene therapy, today announced it has signed collaboration agreements with Chiesi Famaceutici SpA for the commercialization of Glybera, the first gene therapy to receive regulatory approval in Europe, as well as the co-development of a gene therapy for hemophilia B. In connection with this transaction, uniQure has received EUR 17 million (USD 21.8 million) in collaboration financing and EUR 14 million (USD 18 million) in equity financing from Chiesi, and has converted into equity the previously announced EUR 14.1 million (USD 18.1 million) investment led by Coller Capital (London, UK) with participation by existing investors.

uniQure's agreement with Chiesi, an international company headquartered in Parma, Italy, gives Chiesi exclusive rights to commercialize Glybera, the first gene therapy product approved in the European Union for the treatment of the orphan disease lipoprotein lipase deficiency (LPLD) for which there is currently no treatment, as well as for uniQure's pipeline product for hemophilia B, in Europe and selected other countries (Brazil, Mexico, Pakistan, Turkey, Russia, and the CIS countries, plus China for Glybera only). Commercial rights for the US, Japan, and parts of Latin America and Asia, and Australasia remain with uniQure. In exchange, uniQure stands to receive net royalties that range from 20 to 30 percent over time on sales of both products. Furthermore, Chiesi will fund half of the remaining development costs for uniQure's hemophilia B program, as well as take an equity stake in uniQure.

The investment by Chiesi will also result in the conversion into new uniQure shares of the EUR 14.1 million (USD 18.1 million) in convertible debt the Company recently raised from Coller Capital, a leading global private equity investor, and existing investors Forbion Capital Partners, Gilde Healthcare Partners, Lupus Alpha, Grupo Netco and others.

"The agreement with Chiesi is a key component of our strategy to rapidly develop and commercialize multiple gene therapy based treatments as well as being a validation of our technology platform," said Jörn Aldag, CEO of uniQure. "With its focus on innovative therapeutics, Chiesi is a strong partner for the commercialization of Glybera in Europe. The investment from Coller Capital, supplemented by our existing investors, allows us to continue apace with the development of what we believe is the richest and most advanced gene therapy pipeline in the industry. In the next 12-18 months, we anticipate clarifying the path toward an FDA filing for Glybera in the US, reporting results from a Phase I/II study in acute intermittent porphyria, and starting at least two Phase I/II studies for additional pipeline programs."

About uniQure
uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. Our approach is validated by multiple partnerships and the regulatory approval of our lead product Glybera. www.uniqure.com.

About Chiesi Farmaceutici
Founded in 1935 in Parma, Italy, Chiesi Farmaceutici currently has 25 affiliates worldwide and markets its therapeutics in over 60 countries. Chiesi's manufacturing plants in Parma, Blois (France) and Santana de Parnaiba (Brazil), and R&D centers in Parma, Paris, Rockville (USA) and Chippenham (UK) integrate their efforts to advance the Group's pre-clinical, clinical and registration programs. At the end of 2012, the Chiesi Group's total staff stood at over 3,800 people, more than 350 of whom are dedicated to R&D. The main areas of activity are in respiratory therapeutics and specialist medicine areas.

This press release contains forward-looking statements based on uniQure's current expectations. These forward-looking statements include statements regarding the commercialization of Glybera, regulatory approval matters and the development of additional gene therapies. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties regarding further regulatory requirements, the success of further clinical trials, and competitive pressures. uniQure assumes no responsibility to update such forward-looking statements.

Jörn Aldag
CEO uniQure
T: +31 20 566 8014
[email protected]

Massimo Zaninelli
Communication Advisor Chiesi Farmaceutici Spa
Tel: +39 335 7893658
[email protected]

Media inquiries:
MacDougall Biomedical Communications
Doug MacDougall (US)
+1 781 235_3060
[email protected]

Gretchen Schweitzer (Europe)
+49 172 861 8540
[email protected]