Our focus is to develop innovative therapies to treat autoimmune diseases where the body’s natural immune system mistakenly attacks normal healthy cells. Autoimmune diseases affect a wide range of organs that can have a profound effect on human health. There are over 100 known autoimmune diseases where, in many cases, women are disproportionally affected.
Many demyelinating diseases are inflammatory autoimmune diseases where the body’s own immune system attacks and destroys the myelin sheaths that envelop axons in the central nervous system.
Multiple Sclerosis (MS) is an autoimmune disease where myelin in the CNS is damaged due to inflammation.
Optic Neuritis (ON) is an autoimmune disease where inflammation leads to myelin damage in the optic nerve.
Patients experience diminished acuity and blurry vision most often in one eye. Although symptoms typically resolve within a few months, ON can signal the onset of MS in about 20-30% of MS patients. This condition is known as Multiple Sclerosis associated ON (MS-ON). Ultimately, up to 50% of MS patients experience ON during the course of their disease.
There are other forms of ON that are distinct from MS-ON, such as Neuromyelitis Optica (NMO) and Myelin Oligodendrocyte Glycoprotein Antibody Disease (MOGAD). These are rare chronic diseases that lead to progressive worsening of vision until blindness.
Safe and effective therapies that promote repair of myelin are urgently needed.
We are developing remyelinating agents to repair and rebuild damaged myelin
We are developing novel remyelinating agents that target a transmembrane receptor complex located on oligodendrocytes which regulates myelin formation in the CNS. When the receptor complex is activated, it blocks remyelination by oligodendrocytes. Our remyelinating agents are designed to disrupt the receptor complex and remove the barriers to remyelination.
Our lead molecule (FTX-101) is a peptide that targets the transmembrane region of this receptor to promote remyelination. FTX-101 is currently in IND-enabling studies and is expected to enter clinical studies in 2023.
Diseases of the GI tract
We have identified certain GPCRs that are involved in autoimmune diseases of the GI tract with a significant unmet need.
GPCRs are the largest and most diverse superfamily of membrane receptors that mediate most of our physiological responses to hormones, neurotransmitters, and environmental stimulants. They are characterized by seven transmembrane domains and activate associated G proteins responsible for intracellular signaling. It has been estimated that 30% of all FDA-approved drugs act on GPCRs.
We are actively pursuing several discovery programs to find peptides and small molecules directed against these GPCRs to develop as potential therapies.