FBR Still Positive on Abeona Therapeutics (ABEO) Following DSMB Review of ABO-102

October 5, 2016 10:43 AM EDT
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FBR & Co. affirmed Abeona Therapeutics Inc. (Nasdaq: ABEO) with an Outperform rating and $15 price target after the company announced that the Data Safety Monitoring Board (DSMB) has reviewed the initial safety data from the low dose cohort (n=3) in the Phase 1/2 clinical trial of ABO-102 (AAV-SGSH) enrolling at Nationwide Children's Hospital in Columbus, Ohio.

Analyst Vernon T. Bernardino sees the DSMB ruling as a positive. The analyst commented, The Data Safety Monitoring Board (DSMB) for ABEO’s Phase I/II trial for ABO-102, ABEO's investigational gene therapy for patients with the metabolic disorder mucopolysaccharidosis Type IIIA (MPS IIIA or Sanfilippo A), has reviewed the initial safety data from the low-dose cohort (n=3) and, as a result, has authorized ABEO’s collaborators at Nationwide Children’s Hospital (NCH) to proceed with dose escalation in a second cohort of MPS IIIA patients. As a reminder, MPS IIIA is a deadly lysosomal storage disease caused by a single gene defect that occurs in 1 in every 70,000 births and has no treatments available. While other gene therapy strategies have been employed to treat MPS IIIA, we believe ABO-102 has significant advantages over previous attempts, as it delivers a normal copy of the gene for heparin N-sulfatase via the intravenous route as a single treatment. We also view continued supervision of the study by NCH as positive, as NCH is one of the top 10 National Institutes of Health–funded pediatric research centers in the U.S. and has a strong background in research on rare genetic diseases.

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