Executive Summary

We are focused on developing and marketing first-in-class regenerative therapies for osteoarthritis. Our proprietary platform technology harnesses the healing potential of the purinergic system which plays a critical role in maintaining cartilage homeostasis. Our lead product is a proprietary formulation for regenerating cartilage in joints with established osteoarthritis, targeting a rapidly growing joint preservation market. Our goal is to offer patients a disease modifying therapy with uncompromising efficacy relative to existing technologies. By developing this disease modifying therapy, we will significantly improve clinical outcomes and patient quality of life, and reduce the total health care delivery costs associated with regenerative medicine.

Technical Validation

The purinergic system plays a critical role in maintaining cartilage homeostasis. We found that lack of adenosine A2A receptor can cause spontaneous osteoarthritis (OA) in mice, and that chondrocytes in OA patients generate much less adenosine than normal chondrocytes. Adenosine has an extremely short half-life in biological fluids (1-2 seconds). A key aspect of the intellectual property lies in the formulation of the two assets in our development pipeline which prolong the activity of adenosine. These two novel formulations were tested for efficacy with intra-articular injections in two models simulating the most common causes of OA: a rat model with established OA following ACL rupture, and a model in which mice develop OA due to weight gain. In both models, we demonstrated that restoring adenosine levels in affected joints prevented the progression of OA, reversed cartilage degradation and restored cartilage thickness in the treated joints. The exogenous adenosine induces cartilage regeneration by activating the TGF-beta signaling associated with chondrocyte proliferation & maturation, and by downregulation of factors which lead to cell death.