Therapeutic applications

Our lead product is an orally-active Reglemer™ that brings a novel mechanism of action to the treatment of immune-mediated indications in which regulatory T cell induction is known to have therapeutically beneficial effects. The first two indications that we are targeting are:

Inflammatory Bowel Disease (IBD)

Inflammatory bowel disease is a chronic and progressive gastrointestinal disease characterized by uncontrolled activation of the intestinal immune system resulting in severe gastrointestinal inflammation. The two broad subsets of IBD are Crohn’s disease (CD) and ulcerative colitis (UC), together affecting over 1.5 million patients in the United States alone, making IBD a serious medical, social and economic problem. Of the 13 FDA-approved drugs for IBD in 2014, only 9 distinct molecules were represented, with others being different formulations of the same molecule.

Existing therapies are associated with significant adverse events related to immune suppression, and several of the therapies coming down the pipeline carry substantial immunologic long-term impact for the recipient. The market size for IBD drugs in 2013 was estimated at $7.8 billion and is expected to grow to $8.3 billion by 2021 – just in the seven major markets. A safe and efficacious oral immunomodulator that does not result in an overwhelming immune suppression is needed, and our lead program is expected to fill that role in IBD therapy.

Multiple Sclerosis (MS)

Multiple sclerosis is a chronic demyelinating inflammatory disease of the human CNS, and the most common neurological disease of young adults, affecting over 450,000 patients in the US and over 3.5 million patients worldwide. Several subtypes of MS have been described, which include relapsing remitting multiple sclerosis (RRMS), secondary progressive multiple sclerosis (SPMS) and primary progressive multiple sclerosis (PPMS). The largest subtype of MS patients is RRMS, and all currently approved MS therapeutics have been approved only for this indication.

As of 2014, there were 12 FDA-approved drugs for MS, with 5 of the drugs being interferon-betas. Currently approved and pipeline disease-modifying drugs in MS have been associated with significant adverse events related to immune suppression, and the FDA has required a black box warning for some of the more recently approved therapies. Several of the therapies coming down the pipeline and before the FDA for registration carry substantial immunologic long-term impact for the recipient. The market for MS therapeutics was estimated at $14 billion in 2013, and is expected to be a $17 billion market by 2021 – just in the US, EU and Japan. A safe and efficacious oral immunomodulator that does not result in an overwhelming immune suppression is needed, and our lead program is expected to fill that role in MS therapy.