First bispecific CAR-T therapy for MS receives green light from FDA to start phase 1

The California-based company announced this week that the FDA had cleared an investigational new drug application (IND) for its drug candidate IMPT-514, a CD19/CD20 bispecific CAR-T cell therapy in development for patients with multiple sclerosis (MS).

Almost three million people worldwide have been diagnosed with MS. The condition chronically affects the central nervous system and is thought to be an autoimmune disorder in which the body’s own immune cells attack the myelin protein surrounding the nerve fibers. With the destruction of myelin, the nerves cannot properly conduct electrical impulses to and from the brain, impacting the body’s muscle functions. While some people only have mild symptoms, others can lose their ability to see, talk, or move. 

ImmPACT Bio’s IMPT-514 is a bispecific CAR-T therapy that targets CD19 and CD20 membrane proteins found on the surface of most B cells, a type of immune cell. CAR-T cell therapies consist of patient-sourced T cells engineered to carry a fusion protein – known as a chimeric antigen receptor (CAR) – that allows the T cells to specifically bind to targets such as CD19 and CD20.

While CAR-T therapies have been used as a promising immunotherapy for blood cancers​, IMPT-514 is the first and only known CAR-T cell therapy for multiple sclerosis patients. In the case of IMPT-514, the therapy was manufactured using T cells from heavily immunosuppressed autoimmune disease patients with proven B cell elimination.

“The dual-targeting nature of IMPT-514 offers a unique opportunity to potentially ablate autoreactive B cells and the likely pathogenic CD20-expressing T cells,” explained Jonathan Benjamin, chief medical officer of ImmPACT Bio in a press statement.

“Moreover, the ability of CAR-T cells to cross the blood-brain barrier could potentially overcome the limitations of current disease-modifying therapies such as anti-CD20 antibodies that are not effective at crossing the blood-brain barrier, where potentially pathogenic cells reside.”

Now that ImmPACT Bio has received FDA clearance to start a Phase 1 dose exploration trial, the company expects to dose the first patient in the first half of 2025. The trial will aim to slow or even stop the progression of disability in MS patients whose condition remains suboptimal despite receiving disease-modifying therapies.

“IND clearance for our bispecific CAR T-cell therapy in MS marks an exciting achievement that further expands clinical development of our autoimmune program," said Sumant Ramachandra, CEO of ImmPACT Bio. "As an intended one-time treatment, IMPT-514 has the potential to reset the immune system by depleting a broad range of autoreactive immune cells implicated in the pathogenesis of MS in patients.”