Novelmed Therapeutics Inc — Department of Health and Human Services SBIR Phase II: W

Description

DESCRIPTION provided by applicant Approximately 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 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 Bruchandapos s membrane are the hallmarks of GA Lampalizumab demonstrated nearly benefit for patients with Dry AMD NovelMedandapos s lead drug candidate NM is a superior AP inhibitor and is expected to be more effective and longer lasting at lower dosages when compared to Lapalizumab We expect NM 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 NovelMedandapos 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 NM as a single therapy for both Wet and Dry AMD We expect NM 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 PUBLIC HEALTH RELEVANCE Geographic atrophy GA is the advanced atrophic form of age related macular degeneration AMD No treatment is currently available to prevent the either the onset and or progression of GA The latest results from anti Factor D an alternative pathway inhibitor show the involvement of the complement system Geographic atrophy is the advanced form of Dry AMD and is caused by the degeneration of the retinal epithelial cells These cells protect the rods and cones that are responsible for central vision Although GA is less common than Wet AMD it is responsible for of cases of legal blindness in the US The global prevalence of GA is in all ages occurring in of people between the ages of years old in of people between the ages of and in of people over years old The prevalence of GA increases to after years of age