uniQure Announces Multiple Presentations and Investor Webcast on Hemophilia B Gene Therapy Program at the 62nd American Society of Hematology (ASH) Annual Meeting

Lexington, Massachusetts, UNITED STATES

~ Strong Presence at ASH Featuring Five Presentations, Including Late-Breaking
Oral Presentation on HOPE-B Pivotal Trial ~

~ uniQure to Host Investor Webcast Tuesday, December 8, 2020 at 5:00 p.m. ET ~

LEXINGTON, Mass. and AMSTERDAM, Nov. 30, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan, will present clinical data from the HOPE-B pivotal trial of etranacogene dezaparvovec in hemophilia B at the virtual 62nd American Society of Hematology (ASH) Annual Meeting.

  • Dr. Pipe, principal investigator of the HOPE-B pivotal trial, will participate in the ASH press briefing scheduled for Monday, December 7 from 10:30 a.m. – 11 a.m. E.T.
  • Dr. Pipe will present a late-breaking oral presentation on the HOPE-B data on Tuesday, December 8 at 11:45 a.m. E.T.

uniQure management along with Dr. Pipe will host an investor webcast on Tuesday, December 8, 2020, at 5:00 p.m. ET. To access the live webcast with presentation slides, please visit the Investor Relations section of uniQure’s website at www.uniQure.com. The webcast will be archived for 90 days. The event also may be accessed by dialing (877) 870 – 9135 for domestic callers and +44 020 719 283 38 for international callers. The passcode is 3164585. Please specify to the operator that you would like to join the “uniQure Conference Call.”

Late-Breaking Oral Presentation
Title:First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies
Presenter:Steven Pipe, M.D.
Session Name:Late-Breaking Abstracts, LBA-6
Date:Tuesday, December 8, 2020
Presentation Time:11:45 a.m. ET (8:45 a.m. PT)
Oral Presentation 
Title:Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Two Year Data from a Phase 2b Trial
Presenter:Annette von Drygalski, M.D., PharmD
Session Name:801. Gene Editing, Therapy and Transfer I
Date:Monday, December 7, 2020
Presentation Time:2:45 p.m. ET (11:45 a.m. PT)
Poster Presentations 
Title:AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 5 Years
Session Name:801. Gene Editing, Therapy and Transfer: Poster III
Date:Monday, December 7, 2020
Presentation Time:10:00 a.m. - 6:30 p.m. ET (7:00 a.m. - 3:30 p.m. PT)
Title:A Single Administration of AAV5-hFIX in Newborn, Juvenile and Adult Mice Leads to Stable hFIX Expression up to 18 Months after Dosing 
Session Name:801. Gene Editing, Therapy and Transfer: Poster II
Date:Sunday, December 6, 2020
Presentation Time:10:00 a.m. - 6:30 p.m. ET (7:00 a.m. - 3:30 p.m. PT)
Title:Examining the Hemophilia Disability Paradox
Session Name:904. Outcomes Research—Non-Malignant Conditions: Poster II
Date:Sunday, December 6, 2020
Presentation Time:10:00 a.m. - 6:30 p.m. ET (7:00 a.m. - 3:30 p.m. PT)

The conference abstracts are available and can be accessed through this link: ASH abstracts.

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 gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.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," "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, whether we will present long-term follow-up data from our hemophilia B gene therapy studies, including two years of follow-up on the Phase IIb clinical trial of etranacogene dezaparvovec (AMT-061) and up to five years of follow-up from the Phase I/II clinical trial of AMT-060, and whether we will announce top-line data from the pivotal HOPE-B study of etranacogene dezaparvovec before the end of this year. 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, clinical results, 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 October 27, 2020. 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:

Maria E. CantorChiara RussoTom Malone
Direct: 339-970-7536Direct: 617-306-9137Direct: 339-970-7558
Mobile: 617-680-9452Mobile: 617-306-9137Mobile:339-223-8541