3 Reasons Why Uniqure Could Overtake Hemophilia B Virus Gene Therapy

Uniqure NV QURE, a gene therapy company that focuses on severe unmet medical needs, could "dominate" the space with its AMT-061 therapy, according to B. Riley FBR.

The Analyst

B. Riley FBR's Madhu Kumar initiated coverage of Uniqure with a Buy rating and $50 price target.

The Thesis

Uniqure's AMT-061 can potentially disrupt and transform hemophilia B treatment on three fronts, Kumar said in the initiation note:

• The company's recently issued patent should eliminate any concerns tied to the patent estate, which makes the company "the dominant firm" in hemophilia B AAV GT from an intellectual property point of view, the analyst said. 
• Preclinical and clinical data suggests an AAV5-based GT has a substantially modest neutralizing antibody rate, and the company's Phase 3 study of its AMT-061 should show the potential to be used in almost all hemophilia B patients, Kumar said. 
• The regulatory environment at the FDA suggests that hemophilia factor production could serve as a surrogate endpoint for approval, with downstream functional measures included bleed rates as a post-approval metric, the analyst said. This bodes well for AMT-061's rapid approval from the regulatory bodies, he said. 

From an intellectual property, scientific and regulatory point of view, Uniqure boasts many advantages in hemophilia B, according to B. Riley FBR. 

Price Action

Uniqure shares were rallying more than 13 percent to $38.39 at the time of publication Thursday afternoon. 

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