UniQure Reports Growth With Advancing Clinical Stage Hemophilia (B) HOPE-2 Trial

 UniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. UniQure is leveraging AAV based gene delivery platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with severe genetic diseases. The AAV5 variant or serotype is utilized to deliver product candidates. 

Stock Symbol: QURE

Platform: AAV5 based vector to deliver target genes constructs that encode for specific protein or miRNA.  

Q3 2020 Company Highlight
  • Enrolled First Four Patients in Phase I/II Clinical Trial of AMT-130 in Huntington’s Disease
  • Top-Line Data from HOPE-B Pivotal Trial in Hemophilia B Expected Before Year-End
  • Initiated IND-enabling Studies for AMT-150 in Spinocerebellar Ataxia Type 3 (SCA3)
  • Cash equivalent: 
  • Net Loss: $53.8 million, or $1.21 loss per share
Therapeutic Area 
Hemophilia B
Fabry disease
Huntington's disease
SCA Type 3 

Analyst Review and Growth
Successful Hemophilia B Clinical Program is considered as Inflection Point for the company

Analyst Recommendations- Strong Buy (wsj.com)

AMT-061 for the treatment of Hemophilia B (Clinical Program Update)
  • Advancing late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B
  • Patient dosing was completed in the HOPE-B pivotal trial of etranacogene dezaparvovec in March, 2020, and a 26-week follow-up on all patients has now been achieved. The Company is looking forward to reporting 26-week Factor IX data for all 54 patients enrolled in the global clinical trial before the end of 2020.
  • In June 2020, the Company and CSL Behring entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene dezaparvovec. Under the terms of the agreement, which is subject to regulatory review in the United States, Australia, and the United Kingdom, the Company will receive a $450 million upfront cash payment and be eligible to receive up to $1.6 billion in payments based on regulatory and commercial milestones. The Company will also be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percentage of net product sales arising from the collaboration. Under the terms of the licensing agreement, CSL Behring is responsible for the submission of a Biologics License Application, which is expected in the second half of 2021.
AMT-130  for the treatment of Huntington’s disease (Clinical Program Update)
  • In September 2020, the independent Data Safety Monitoring Board (DSMB), which is responsible for evaluating patient safety in the Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease, reviewed the 90-day follow-up data on the first two patients and observed no significant safety concerns to prevent further dosing in the study.
  • In early October 2020, the next two patient procedures were completed in the clinical trial. A total of four patients have been enrolled to date in this randomized, double-blinded study - two patients receiving AMT-130 and two patients who have received imitation (sham) surgery.
  • In accordance with study protocol, patient enrollment is expected to continue after a DSMB meeting to review the 90-day follow-up data on the two patients enrolled in October 2020 and the 6-month data on the two patients enrolled in June 2020. The Company expects this DSMB review will take place early next year and that the remaining six patients in the 10-patient first dose cohort will be enrolled by mid-2021.
Research pipeline of gene therapy candidates toward the clinic

AMT-150- expected submission of an IND application in 2021 
AMT is a novel gene therapy for the treatment of Spinocerebellar Ataxia Type 3 (SCA3) . 

AMT-190 - Advancing into IND enabling studies

Third Quater 2020 Financial Update 
  • Cash Position: As of September 30, 2020, the Company held cash and cash equivalents of $279.5 million, compared to $314.3 million as of June 30, 2020.
  • Revenues: Revenue for the three months ended September 30, 2020 was $1.8 million, compared to $1.0 million during the same period in 2019.
  • R&D Expenses: Research and development expenses were $36.3 million for the three months ended September 30, 2020, compared to $23.6 million during the same period in 2019. The change was primarily related to increased activities associated with our ongoing clinical studies of etranacogene dezaparvovec and AMT-130, increased share-based compensation, increased license expenses and the recruitment of personnel to support the development of our product candidates.
  • SG&A Expenses: Selling, general and administrative expenses were $10.8 million for the three months ended September 30, 2020, compared to $8.9 million during the same period in 2019. The change was primarily related to increases in personnel and consulting expenses and share-based compensation expenses.
  • Other non-operating items, net: Other expense was $9.3 million for the three months ended September 30, 2020, compared to other income of $7.7 million during the same period in 2019 primarily related to foreign exchange results related to changes in the exchange rates between the Euro and the U.S. Dollar.
  • Net Loss: The net loss for the three months ended September 30, 2020 was $53.8 million, or $1.21 loss per share, compared to $23.6 million, or $0.58 loss per share during the same period in 2019
Visit UniQure www.uniqure.com for more details


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