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Demyelinating diseases

Science

Repairing myelin in the CNS

Our scientific approach focuses on enabling remyelination by targeting mechanisms that regulate myelin formation and repair in the central nervous system.

Promising research

Demyelinating diseases

Many demyelinating diseases are inflammatory autoimmune diseases where the body’s own immune system attacks and destroys the myelin sheaths that envelop and support axons in the central nervous system.

Chronic Optic Neuropathy is the result of chronic, progressive myelin damage of the optic nerve and visual tracts in the brain.

In central nervous system inflammatory diseases, such as Multiple Sclerosis (MS), autoimmune reactions cause inflammation leading to destruction of myelin and myelinated axons in the visual pathway, resulting in chronic visual impairment and loss of quality of life. Two forms of visual impairment associated with MS are Acute Optic Neuritis (AON) and Chronic Optic Neuropathy (CON).

Many people with MS, will experience attacks of AON, characterized by subacute onset of pain and diminished vision, usually in one eye. Acute optic neuritis is often treated with corticosteroids and most people recover from individual attacks within a few months. CON on the other hand, is a more debilitating condition characterized by serious visual impairment, with irreversible loss of vision. CON does not respond to corticosteroid or disease modifying immunomodulatory MS treatment. There is a major unmet medical need to find safe and effective treatments that can repair damaged myelin in the optic nerve and visual tracts of the brain to preserve axonal function and restore vision in CON.

Up to 50% of people with MS will experience an episode of optic neuritis during the course of their disease. In the United States, there are an estimated prevalence of 90,000 people who suffer from Chronic Optic Neuropathy.

There are other forms of Optic Neuropathy that are distinct from CON and AON. These include Neuromyelitis Optica (NMO) and Myelin Oligodendrocyte Glycoprotein Antibody Disease (MOGAD) that also involve loss of myelin in the optic nerve due to specific inappropriate auto-immune responses leading to destruction of central nervous system myelin. These are rare chronic diseases that can lead to worsening of vision until blindness. NMO and MOGAD each have a prevalence of approximately 10,000 people in the United States.

Safe and effective therapies that promote repair of myelin are urgently needed.

Although numerous immunomodulatory drugs modulating the immune and inflammatory components of MS have been approved over the past 20 years, there is a major unmet need for drugs that can repair myelin in people with CON and other demyelinating diseases such as MS.

We are developing remyelinating agents to repair and rebuild 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 migration to areas of damage and 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 has completed IND-enabling studies and is expected to enter Phase 1 clinical studies in Q4 2024.