QURE

Etranacogene dezaparvovec (AMT-061)

Hemophilia B

Stage (next event)

Expected Date

Pre-BLA meeting with the FDA

Q1 2021

Catalyst Info & Data Links

TITLE: Etranacogene dezaparvovec (AMT-061) for Hemophilia B - BLA Submission

  • ClinicalTrials.gov (NCT03569891): HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients


WHAT IS THE CATALYST EVENT?


WHEN WILL THE EVENT (OR DID THE EVENT) OCCUR?

  • 1. Q1 2021

  • 2. 2021


PRIOR DATA/EVENTS

PRESS RELEASE

Mechanism of Action

MECHANISM OF ACTION

  • Etranacogene dezaparvovec, also known as AMT-061, consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). AAV5-based gene therapies have been demonstrated to be safe and well tolerated in many clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with the Company’s AAV5-based gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates. Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.

MARKET

  • Disease prevalence: ~6,000 patients in the United States  and ~14,000 patients in Europe

Updated by HC

Prior Data (click to view full image)

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Trial Design / Revenue (click to view full image)

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