Iaso Bio’s experimental CAR T-cell therapy cleared for testing in MS
Eque-cel also being tested for other autoimmune conditions
Iaso Biotherapeutics has received the green light from the U.S. Food and Drug Administration (FDA) to start clinical trials of its investigational CAR T-cell therapy equecabtagene autoleucel (eque-cel) in people with multiple sclerosis (MS).
The FDA’s clearance of the company’s investigational new drug (IND) application makes MS the second autoimmune condition this year for which eque-cel will be assessed, following difficult-to-treat generalized myasthenia gravis (gMG).
“The IND approval of Eque-cel in the treatment of MS from the FDA is another strong evidence of IASO Bio’s ongoing dedication and technological advancements in the treatment of autoimmune diseases,” Yongke Zhang, MD, PhD, Iaso’s chief scientific officer, said in a company press release.
MS is marked by damage to the brain and spinal cord due to a misguided immune response against the myelin sheath, a fatty substance that surrounds nerve fibers. This inflammatory attack is driven by several immune cell types, including B-cells, which are the cells that make antibodies.
Eque-cel designed to reduce inflammatory attack in autoimmune conditions
Eque-cel, also known as CT103A, is designed to reduce the inflammatory attack that drives MS and other autoimmune conditions by targeting a protein at the surface of B-cells, called B-cell maturation antigen, or BCMA.
The treatment belongs to the class of CAR T-cell therapies, where a patient’s own T-cells are collected and genetically engineered in the lab to produce a man-made receptor, called a chimeric antigen receptor (CAR), that recognizes a specific target.
By targeting BCMA, eque-cel is designed to cause B-cell death and reduce the levels of self-reactive antibodies, which is expected to reduce MS relapses and lesions and slow the progression of disability.
Eque-cel’s safety and efficacy in people with autoimmune conditions is being tested in an investigator-initiated Phase 1 clinical trial called CARTinNS trial (NCT04561557). The trial is being conducted at the Tongji Hospital of the Huazhong University of Science and Technology, in China.
A total of 36 patients with autoimmune conditions are expected to enroll, including those with MS, MG, and neuromyelitis optica spectrum disease (NMOSD), a related autoimmune condition similarly marked by damage to myelin for which eque-cel is also being explored. MS patients with either relapsing or progressive forms of the disease are eligible to participate.
In the trial, patients will undergo a round of chemotherapy before receiving the CAR T-cell therapy to make room for the new cells. Then, they will receive a single into-the-bloodstream infusion of eque-cel at one of three doses: 0.25 million, 0.5 million, or 1 million CAR T-cells.
Treated NMOSD patients experienced improvements in disability, life quality
Interim data from the first 12 adults with NMOSD, who had failed to respond adequately to available treatments, showed all but one remained free from relapses over 5.5 months of follow-up, and all had improvements in disability and quality of life.
The treatment appeared to have a manageable safety profile, although all patients experienced severe or life-threatening conditions, most commonly low levels of immune cells and other blood cells.
All patients developed cytokine release syndrome, a potentially life-threatening side effect marked by an excessive immune response, and more than half had infections after the treatment.
While no results from MS patients have been published to date, “eque-cel has shown promising efficacy in 6 autoimmune diseases” in that trial, Zhang said.
“Through close collaboration and in-depth exchanges with international clinical research institutions, we aim to accelerate the development and commercialization of more innovative drugs, bringing greater benefits to patients worldwide,” he added.
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