uniQure Announces Presentations at Upcoming International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress

Rhea-AI Summary

uniQure N.V. (NASDAQ: QURE) announced it will present eight studies at the ISTH Virtual Congress from July 17-21, 2021, focusing on gene therapy for hemophilia B. Highlights include five-year data on FIX expression stability and outcomes from the Phase 3 HOPE-B trial. Notable presentations include the clinical outcomes of patients with pre-existing AAV5 antibodies and a case report on liver safety. The company continues to advance gene therapies for severe conditions, emphasizing its commitment to innovative treatment solutions.

Positive

• Eight presentations at ISTH, showcasing ongoing research and commitment to gene therapy.
• Five-year data confirms stable FIX expression and reduced bleeding post-AMT-060 therapy.
• Focus on significant clinical outcomes in Phase 3 HOPE-B trial could enhance market confidence.

Negative

• None.

LEXINGTON, Mass. and AMSTERDAM, The Netherlands, July 02, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that eight data presentations, of which three are oral presentations, will be delivered at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress being held July 17-21, 2021.

Specific details on uniQure’s presentations at ISTH include:

• Title: Five Year Data Confirms Stable FIX Expression and Sustained Reductions in Bleeding and Factor IX Use Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B (Abstract OC 26.3)
  Oral Session Title: Hemostatic Agents and Hemostatic Efficacy
  Date and Time: Monday, July 19, 11:00 – 12:00 p.m. EDT

• Title: Clinical Outcomes in Adults with Hemophilia B With and Without Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data from the Phase 3 Etranacogene Dezaparvovec HOPE-B Gene Therapy Trial (Abstract OC 67.3)
  Oral Session Title: Safety and Efficacy in Gene Therapy for Hemophilia
  Date and Time: Wednesday, July 21, 10:00 – 11:00 a.m. EDT

• Title: Liver Safety Case Report from the Phase 3 HOPE-B Gene Therapy Trial in Adults with Hemophilia B (Abstract OC 67.4)
  Oral Session Title: Safety and Efficacy in Gene Therapy for Hemophilia
  Date and Time: Wednesday, July 21, 10:00 – 11:00 a.m. EDT

• Title: Etranacogene dezaparvovec (AAV5-Padua hFIX variant, AMT-061), an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: 2.5 Year data from a Phase 2b Trial (Live Poster, Abstract LPB0020)
  Poster Session Title: Oligonucleotide Therapeutics
  Live ePoster Date and Time: Sunday, July 18, 3:00 – 4:00 p.m. EDT

• Title: 52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults with Severe or Moderate-Severe Hemophilia B: Data from the Phase 3 HOPE-B Gene Therapy Trial (Abstract PB0653)
  Date and Time: All ePosters will be made available on Saturday, July 17
  
  
• Title: Management of Infusion Reactions: Lessons from the Phase 3 HOPE-B Gene Therapy Trial of Etranacogene Dezaparvovec in Adults with Hemophilia B (Abstract PB0659)
  Date and Time: All ePosters will be made available on Saturday, July 17

• Title: Real-world Outcomes in People with Severe Hemophilia B Receiving FIX Prophylaxis across Europe: A CHESS II Analysis (Abstract PB0463)
  Date and Time: All ePosters will be made available on Saturday, July 17
  

• Title: Preferences of People with Hemophilia A and B for Treatments Including Gene Therapies in the US: A Discrete Choice Experiment (Abstract PB0658)
  Date and Time: All ePosters will be made available on Saturday, July 17

uniQure Vice President of Clinical Development David Cooper, M.D., also will give an invited presentation to the ISTH SSC (Scientific and Standardization Committee) subcommittee entitled “Factor VIII, Factor IX and Rare Coagulation Disorders.” His pre-recorded presentation, Rationale for Adeno-associated Virus (AAV) Mediated Gene Therapy in Patients with Pre-existing anti-AAV5 antibodies, will take place on Saturday, July 17 from 9:24 to 9:30 a.m.

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 severe genetic diseases of the central nervous system (CNS) and liver, including clinical programs in hemophilia B and Huntington's disease and preclinical candidates in Fabry disease, spinocerebellar ataxia Type 3, temporal lobe epilepsy, Alzheimer’s, Parkinson’s, and ALS. www.uniQure.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

FAQ

What will uniQure present at the ISTH Congress 2021?

uniQure will present eight studies focusing on gene therapy for hemophilia B at the ISTH Congress from July 17-21, 2021.

What is the significance of the five-year data presented by uniQure?

The five-year data highlights stable FIX expression and sustained reductions in bleeding and factor IX use in patients treated with AMT-060 gene therapy.

When is uniQure's oral presentation scheduled at ISTH?

uniQure's oral presentations are scheduled for July 19 and July 21, 2021.

What are the key topics of uniQure's presentations at the ISTH Congress?

Key topics include the clinical outcomes in hemophilia B patients, liver safety reports, and the efficacy of Etranacogene Dezaparvovec.

How does uniQure's gene therapy research impact investors?

The positive data and ongoing research efforts may enhance investor confidence in uniQure's potential for innovative treatments and market growth.