Single Therapy for Wet AMD &Geographic Atrophy

Period of Performance: 09/01/2015 - 08/31/2016


Phase 2 SBIR

Recipient Firm

Novelmed Therapeutics, Inc.
Cleveland, OH 44106
Principal Investigator


DESCRIPTION (provided by applicant): Approximately 85% of the total age-related macular degeneration (AMD) patients have the Dry form. Geographic atrophy (GA) is the advanced form of Dry AMD and is caused by the degeneration of the retinal epithelial cells. While GA is less common than Wet AMD, it is responsible for 10-20% of legal blindness in the US. There is currently no FDA approved treatment for GA. However, the latest results from a trial evaluating the drug Lampalizumab, an anti-Factor D therapeutic antibody targeted to the alternative complement pathway, provide convincing evidence that the alternative complement pathway plays a key role in the development of vision loss in patients with GA. Complement-mediated destruction of RPE, photoreceptor cells, and Bruch's membrane are the hallmarks of GA. Lampalizumab demonstrated nearly 44% benefit for patients with Dry AMD. NovelMed's lead drug candidate, NM5072, is a superior AP inhibitor and is expected to be more effective and longer lasting, at lower dosages, when compared to Lapalizumab. We expect NM5072 to inhibit both the CNV and the GA before it can be known which of the two pathologies a patient might develop later on (if not treated). NovelMed's lead drug candidate is a selective inhibitor of the alternative complement pathway. The molecule; a) binds the target with high affinity and selectivity, b) prevents formation of new blood vessels at potent dose levels, c) prevents inflammation, and d) has longer pharmacokinetics for improved dosing schedule, Given the role of the alternative complement pathway in GA, we expect our drug to prevent the onset and progression of CNV and GA without affecting tissue repair. There is no therapy available today that could control both the Wet and the Dry form of AMD that leads to vision loss. The drug has been manufactured to FDA specifications and ready for GLP toxicity studies and for human clinical trial. We intend to develop NM5072 as a single therapy for both Wet and Dry AMD. We expect NM5072 to inhibit both CNV and GA at an early stage of AMD, before it can be known which of the two pathologies a patient might develop later on (if left untreated). By treating AMD prior to onset of CNV and GA, AMD driven vision loss could be prevented entirely.