~ Pivotal Trial in Hemophilia B on Track to Begin in Q3 2018, with Initial AMT-061 Clinical Data by Year End ~
~ AMT-130 Expected to be First AAV-Based Gene Therapy for Huntington’s Disease to Enter Clinic ~
~ Strong Cash Position Sufficient to Fund Company into 2020 ~
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Jan. 08, 2018 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced its near-term objectives following a transformational year of achievement and corporate execution. Key business accomplishments during 2017 included uniQure’s alignment with regulatory authorities to advance AMT-061 into late-stage clinical development in hemophilia B, progress with its research pipeline including AMT-130 in Huntington’s disease, further enhancement of the Company’s manufacturing capabilities, and the strengthening of its financial position.
“We ended 2017 with significant momentum across all of our programs and the cash to fund operations into 2020,” stated Matthew Kapusta, chief executive officer of uniQure. “Now that we have established robust, commercial-scale manufacturing in our Lexington facility, we are highly focused on the near-term initiation of our pivotal study of AMT-061 in hemophilia B and Phase I/II trial of AMT-130 in Huntington’s disease.”
“We also are intensifying efforts to further expand our gene therapy product pipeline. In addition to AMT-126, our BMS-partnered gene therapy candidate for congestive heart failure, we have a number of promising early-stage projects focused on liver-directed and CNS disorders. We expect multiple new INDs to be filed over the next two years, further leveraging our industry-leading manufacturing and technology platform.”
uniQure management is meeting with investors and analysts this week in conjunction with the 36th Annual J.P. Morgan Healthcare Conference taking place in San Francisco. An updated corporate presentation has been posted to the investor page of the Company’s website, www.uniqure.com.
Key Near-Term Objectives
Financial Guidance
As of December 31, 2017, the Company had approximately $159 million in cash and cash equivalents, and expects cash on hand will be sufficient to fund operations into 2020.
Major Accomplishments Throughout 2017
Hemophilia B – AMT-061
Huntington’s Disease – AMT-130
Congestive Heart Failure – AMT-126 (partnered with Bristol-Myers Squibb)
Manufacturing Capabilities
Research on AAV5
Corporate Developments
About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and cardiovascular diseases.
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," "estimate," "expect," "goal," "intend," "look forward to," "may," "plan," "potential," "predict," "project," "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, the development of our gene therapy product candidates, the transition to our AMT-061 product candidate, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates, and the scope of protection provided by our patent portfolio. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators' clinical development activities, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's Quarterly Report on Form 10-Q filed on November 1, 2017. 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 the future.
uniQure Contacts
For Investors:
Maria E. Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor@uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com
For Media:
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone@uniQure.com