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Other RARγ Agonists

Our work in FOP, MO, and ocular disorders, as well as in non-clinical studies designed to elucidate RARγ agonist biology in a variety of cell systems has provided us with unique insights into the biological effects of systemically administered RARγ agonists and their potential therapeutic applications. We believe that RARγ selective agonists have substantial untapped therapeutic potential because of their potential antifibrotic, tissue regeneration and repair activities, as well as their predicted safety profile.

We undertook a systematic search for second generation RARγ agonists which we could deploy in different indications, which led us to our license agreement with Galderma for several novel RARγ agonists in their portfolio. These agonists, derived from four structural families, possess numerous advantageous properties including high selectivity to the RARγ receptor.

An initial focus for the development of novel RARγ agonists will be for therapeutic use in diseases, like FOP or MO, which involve pathological bone formation. There are several other potential indications for the prevention of HO, such as ankylosing spondylitis, a type of arthritis associated with excess BMP signaling. Other indications, such as those characterized by excessive fibrosis or scarring, are also potential target indications for RARγ agonists. On the basis of our scientific know-how and other clinical and commercial insights, a number of indications have been prioritized for animal model proof-of-concept studies in 2017 and 2018. Should these studies be successful, we plan to initiate the pre-IND activities necessary to initiate clinical trials in these new indications.

We believe that RARγ agonists represent a new class of compounds with broad therapeutic potential comparable to other hormones such as corticosteroids. Our plans are to fully exploit this potential internally and in partnership with others.