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 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 18 FDA-approved drugs for IBD in 2018, only 9 distinct molecules were represented, with others being different formulations of the same molecule or biosimilars.
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 2016 was estimated at $10.5 billion and is expected to grow to $14.8 billion by 2024. 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 2018, there were 15 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 $16.1 billion in 2016, and is expected to be a $27.4 billion market by 2025. 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.
The American Cancer Society predicts 1.7 million people will be diagnosed with cancer in 2018, with over 600,000 dying from the disease. Patients have historically been treated with radiation and chemotherapy, resulting in prolongation of life, but rarely complete remission. Recently, a new class of oncology agents referred to as checkpoint inhibitors have been approved by the FDA and have resulted in complete remission in a small percentage of patients with certain cancers, notably melanoma and non-small cell lung cancer, among others. The market size for checkpoint inhibitors was over $10 billion in 2017 and is expected to grow to over $20 billion by 2020.
Some patients, however, are unable to tolerate treatment with checkpoint inhibitors and are forced to terminate treatment due to colitis or other gastrointestinal toxicities. An agent that would prevent gastrointestinal toxicities would enable additional patients to benefit from checkpoint inhibition and would ease the course for patients with low grade toxicities. Furthermore, recent work emerging from microbiome research is intriguingly pointing to potential avenues for enhancement of checkpoint inhibitor efficacy. We are advancing Reglemer programs that have the potential to decrease gastrointestinal toxicities and enhance the efficacy of checkpoint inhibitors.