Age-related macular degeneration (AMD) affects nearly 10 million people in the U.S (Research to Prevent Blindness Foundation). Recently approved anti-VEGF therapy is the only cure for wet AMD as of today. Recent studies have demonstrated the role of the alternative pathway (AP) in AMD. Gradual loss of vision eventually resulting in blindness is the key symptom of AMD in the adult population. Using the mouse AMD model, Bora et al demonstrated that complement factor B of the alternative pathway plays an important role in the disease. With the use of siRNA techniques, Dr. Bora demonstrated that silencing of the factor B gene prevents the induction and persistence of AMD. Encouraged by these results, we decided to evaluate the effect of our blocking anti-factor B monoclonal antibody (Bikaciomab) in inhibiting the development and progression of AMD. Bikaciomab binds factor B protein in human blood and blocks its function in AP mediated complement activation. Bikaciomab prevents alternative pathway mediated formation of C3a, C5a, and C5b-9, the key anaphylatoxins responsible for the inflammatory responses. We will test the effect of this blocking anti-factor B antibody in prophylaxis and established models of the disease to provide direct clinical relevance of the drug under testing.

Public Health Relevance

Age-related macular degeneration (AMD) affects nearly 10 million people in the U.S (Research to Prevent Blindness Foundation). Recently approved anti-VEGF therapy is the only cure for wet AMD as of today. There is an unmet medical need in the area of macular degeneration. NovelMed's current approach focuses on neutralizing factor B function and providing disease benefit. Using the previously developed animal models of AMD, NovelMed intends to test its lead complement inhibitor for the treatment of AMD. Potent activity of Bikaciomab in vitro, ex vivo and in vivo makes the drug as a potential therapeutic for not only AMD but also for several diseases where alternative pathway plays an important role in disease pathology.

Agency
National Institute of Health (NIH)
Institute
National Eye Institute (NEI)
Type
Small Business Innovation Research Grants (SBIR) - Phase I (R43)
Project #
1R43EY019191-01A1
Application #
7612275
Study Section
Special Emphasis Panel (ZRG1-ETTN-E (12))
Program Officer
Wujek, Jerome R
Project Start
2009-02-15
Project End
2011-02-14
Budget Start
2009-02-15
Budget End
2011-02-14
Support Year
1
Fiscal Year
2009
Total Cost
$212,118
Indirect Cost
Name
Novelmed Therapeutics, Inc.
Department
Type
DUNS #
190155171
City
Cleveland
State
OH
Country
United States
Zip Code
44106