Vidasym, Inc — Department of Health and Human Services SBIR Phase II: NIAMS

Description

DESCRIPTION provided by applicant Osteoporosis is a major public health threat The American Association of Orthopaedic Surgeons estimates that the cost of treating patients with a low energy fracture in was US$ billion and projected costs for care of osteoporosis and low energy fractures over the next two decades are $ billion Current treatments for osteoporosis include several anti resorptive or anti catabolic and some anabolic agents VDRMs such as calcitriol are currently used to treat osteoporosis in several ex US countries VDRMs influence bone metabolism through regulation of intestinal calcium absorption parathyroid hormone receptor activator of nuclear factor kB ligand and also via direct effects on osteoblasts and osteoclasts Despite encouraging data on VDRMandapos s benefits for treating osteoporosis VDRMs are not used in osteoporosis patients in the US likely due to the fact that those currently on the market have substantial hypercalcemic toxicity that interferes with calcium homeostasis Consequently VDRM therapy requires frequent serum calcium monitoring and dose titration An ideal VDRM should be with little or no hypercalcemic toxicity in the efficacious dose range Since VDRMs are well known to have anabolic effects on the bone a VDRM without hypercalcemic toxicity for the treatment of osteoporosis will provide significant benefits to patients receiving the standard of care Vidasym has taken a unique approach to discover and develop novel VDRMs such as VS Data from extensive animal studies show that VS has an exceptionally wide therapeutic index TI at andgt fold vs TI of calcitriol at fold Results from SBIR Phase I confirm that VS exhibits potent anabolic effects on the bone in a dose range that does not affect serum calcium The mechanism of action studies demonstrate that while VS is potent in stimulating osteoblast activities osteoclast activitie are reduced leading to an increase in bone growth bone mineral density and bone volume These positive data strongly support the advancement of VS into Phase II SBIR The specific aims of this Phase II study are To complete synthesis and formulation of VS under Good Manufacturing Practice GMP in preparation for a Phase I clinical study To file an IND with the FDA for the indication of osteoporosis in preparation for a Phase I clinical study with VS Vidasym plans to develop VS into a reimbursable prescription drug to treat osteoporosis in patients receiving the standard of care According to a report from GBI Research the osteoporosis therapeutics market in was at $ billion and is forecasted to grow at a CAGR of to $ billion in An independent report by EvaluatePharma stated that osteoporosis medications achieved US$ billion in annual worldwide sales in and this number is likely to increase based on the projections of increased patient numbers Assuming Vidasymandapos s VS has a modest penetration into the osteoporosis market when launched in the estimated annual sales will be US$ billion PUBLIC HEALTH RELEVANCE Data from SBIR Phase I confirm that VS exhibits potent anabolic effects on the bone in a dose range that does not have side effects These positive data strongly support the advancement of VS into the next development phase which consists of manufacturing GMP compliant VS and filing an IND with the FDA in preparation for a Phase I clinical study The cost of treating patients with a low energy fracture in was US$ billion and projected costs for care of osteoporosis over the next two decades are $ billion Current treatments for osteoporosis have serious side effects and low compliance issues A new treatment approach such as VS that is efficacious with minimal toxicity offers a significant opportunity for improved outcomes with substantial societal benefit