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UniQure Announces Pricing of Initial Public Offering

By Press Release, UniQure
Press Release.


Amsterdam, the Netherlands, February 5, 2014 – uniQure B.V. (“uniQure”), a leader in human gene therapy, today announced the pricing of its initial public offering of 5,400,000 ordinary shares at an initial public offering price of $17.00 per ordinary share. In addition, uniQure has granted the underwriters a 30-day option to purchase up to an additional 810,000 ordinary shares from uniQure at the public offering price, less underwriting discounts. After deducting the underwriting discounts and other estimated offering expenses payable by uniQure, the net proceeds of the initial public offering are expected to be approximately $81.9 million. The offering is expected to close on or about February 10, 2014, subject to customary closing conditions. uniQure’s ordinary shares have been approved for listing on the NASDAQ Global Select Market and are expected to begin trading under the symbol “QURE” on February 5, 2014.

Jefferies LLC and Leerink Partners LLC are acting as joint book-running managers for the offering. Piper Jaffray & Co. is acting as lead manager for the offering.

A registration statement relating to these securities was declared effective by the Securities and Exchange Commission on February 4, 2014. The offering will be made only by means of a prospectus, copies of which may be obtained from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 12th Floor, New York, NY 10022, or by email at, or by phone at 877-547-6340; or from Leerink Partners LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, or by email at, or by phone at 800-808-7525.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About uniQure
uniQure is delivering on the promise of gene therapy through single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease-modifying therapies to patients with severe disorders. We are engaged in multiple partnerships and have obtained regulatory approval of our lead product, Glybera, in the European Union for a subset of patients with LPLD.

Jörn Aldag
CEO uniQure
T: +31 20 566 8014

Media inquiries:
Gretchen Schweitzer
MacDougall Biomedical Communications
T: +49 172 861 8540

uniQure signs EU commercialization agreement with Chiesi Farmaceutici

By Press Release, UniQure
Press Release.


uniQure signs EU commercialization agreement with Chiesi Farmaceutici for first approved gene therapy treatment, and raises EUR 45 million (USD 59 million)

  • Partnership with Chiesi Farmaceutici for commercialization of Glybera and co-development of hemophilia B gene therapies in  Europe and certain other territories
  • Investment by Coller Capital and other investors

Amsterdam, the Netherlands, July XX, 2013 — uniQure B.V., the leader in human gene therapy, today announced it has successfully raised a total of EUR 45 million, with EUR 31 million from Chiesi Farmaceutici SpA (Parma, Italy), and EUR 14.1 million in an investment led by Coller Capital (London, UK) with participation by existing investors.

uniQure’s agreement with Chiesi Farmaceutici (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. Commercial rights for the US, Japan, and parts of Latin America and Asia, and Australasia remain with uniQure. In exchange, uniQure stands to receive high double-digit royalties 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.

Separately, uniQure recently announced a raise of EUR 13.5 million in convertible debt led by Coller Capital, a leading global private equity investor; existing investors Forbion Capital Partners, Gilde Healthcare Partners, Lupus Alpha and Grupo Netco also participated. An additional EUR 0.6 million in equity funding was contributed by staff and other private investors.

“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 tremendous validation of our technology platform,”

said Jörn Aldag, CEO of uniQure.

“With its focus on innovative therapeutics, Chiesi is the ideal partner for the commercialization of Glybera in Europe. The additional investment from Coller Capital, supplemented by our existing investors, allows us to continue apace with the development of 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 potential 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

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.

Jörn Aldag                           Hans Herklots
CEO                                     Managing Director
uniQure                               Capricorn One
Tel : +31 20 566 8014        Tel: +41 79 598 7149

First gene therapy in Western world receives positive opinion in Europe from CHMP

By Press Release, UniQure
Press Release.


uniQure’s gene therapy Glybera® recommended for approval

• First gene therapy in the Western world to reach important regulatory approval milestone, culminating 40 years of research

• First therapy for LPL deficient patients, a severe disease with no alternative treatment

• Validates uniQure’s unique AAV-based gene therapy platform, consisting of a modular, plug-and-play vector system and unrivaled GMP manufacturing capabilities on a commercial scale

• Heralds new phase in uniQure’s development, including potential revenues from salesand partnerships

• Technology platform can now be leveraged to find solutions for many more severe genetic and other disorders

Amsterdam, The Netherlands – July 20, 2012 – uniQure announced today that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera® (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances. LPLD is a very rare, inherited disease. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain and pancreatitis.

The European Commission (EC) generally follows the recommendations of the CHMP.

“We expect final approval from the EC within 3 months after the CHMP decision,”

says Jörn Aldag, CEO of uniQure.

“After today’s positive recommendation, Glybera is poised to become the first in a class of gene therapy products approved in Europe to treat orphan diseases, rare conditions with a very high unmet medical need.”

Marketing authorization covers all 27 European Union member states.

Mr. Aldag continued:

“Patients with LPLD are afraid of eating a normal meal because it can lead to acute and extremely painful inflammation of the pancreas, often resulting in a visit to intensive care. Now, for the first time, a treatment exists for these patients that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection. The positive recommendation from the CHMP for Glybera therefore represents a major breakthrough for both LPLD patients and for medicine as a whole. Restoring the body’s natural ability to break down fat particles in the blood in order to prevent pancreatitis and excruciating abdominal pain suffered by patients, is what gene therapy is all about: curing disease at the genetic level.”

“At uniQure we are developing treatments for a number of other rare diseases as well, such as acute intermittent porphyria and Sanfilippo B. But the potential of gene therapy stretches far beyond rare diseases. As shown recently in a publication in the New England Journal of Medicine (N Engl J Med 2011; 365:2357-2365, December 22, 2011), hemophilia patients treated with our proprietary gene are showing a sustained clinical effect over several years, which has allowed prophylaxis treatment to be stopped. In addition, we are advancing programs in degenerative diseases such as Parkinson’s. We believe that just like antibodies, gene therapy will one day be a mainstay in clinical practice,”

Mr Aldag added.

As part of the approval, treatment with Glybera will be offered through dedicated centers of excellence with expertise in treating LPLD and by specially trained doctors to ensure ongoing safety of this novel treatment paradigm. uniQure has also committed to building a patient registry for continued understanding of this devastating, under-researched disease. The Company is now preparing to apply for regulatory approval in the US, Canada, and other markets.

Glybera has been tested in three interventional clinical studies conducted in the Netherlands and in Canada, in which a total of 27 LPLD patients participated. In all three clinical trials, Glybera was well tolerated, with no relevant safety issues observed. Data from these clinical trials indicate that a single dose administration of Glybera resulted in a long-term biological activity of the LPL protein.

About Glybera®
uniQure has developed Glybera as a therapy for patients with the genetic disorder lipoprotein lipase deficiency.

LPLD is an orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. Glybera has orphan drug designation in the EU and US. LPL Deficiency affects 1-2 persons per million. For further information on LPLD visit

About uniQure
uniQure is a world leader in the development of human gene based therapies. uniQure has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson’s disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world’s first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows uniQure to pursue its strategy of focusing on this sector of the industry. uniQure’s largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at