3 Reasons Why Uniqure Could Overtake Hemophilia B Virus Gene Therapy

Uniqure NV QURE 12.49%, 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.

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