Human trial to start this year for next-generation CAR T-cell therapy for MG
Treatment acts like a wanted poster in autoimmune diseases, targeting proteins
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- A human trial for TPST-4003, a next-generation CAR T-cell therapy for autoimmune diseases, is slated to start later this year.
- Testing will first focus on the treatment's safety and effectiveness in myasthenia gravis and multiple sclerosis.
- TPST-4003 is an in vivo therapy, meaning it works inside the body rather than being modified in a lab.
TPST-4003, Tempest Therapeutics’ next-generation CAR T-cell therapy for autoimmune diseases such as myasthenia gravis (MG), will soon be tested in people for the first time, the developer announced.
The upcoming investigator-initiated clinical trial, to be conducted in China by Senlang Biotechnology, will assess the effects of this new infusion therapy in 10 people with autoimmune diseases that affect the nervous system. Clinical testing will initially focus on MG and multiple sclerosis, according to a company press release providing updates on Tempest’s lead treatment candidate.
The trial is expected to start recruitment and dosing between September and December, and initial data are expected in the first half of 2027. Under a collaboration agreement between Tempest and Senlang, the latter may choose to negotiate and enter into a definitive license agreement for TPST-4003 in China.
“We look forward to advancing TPST-4003 into clinical development later this year,” said Matt Angel, PhD, Tempest’s president and CEO.
MG is an autoimmune disease driven by self-reactive antibodies that interfere with communication between nerve and muscle cells, leading to symptoms such as muscle weakness and fatigue. Antibodies are immune proteins produced by B-cells, a type of immune cell.
First clinical trial testing TPST-4003 to focus on MG, MS
TPST-4003 belongs to a relatively new class of treatments called CAR T-cell therapies. These take advantage of the body’s immune T-cells, which can kill other cells, to mount immune responses against specific targets.
The cells are modified to carry a human-made protein called a chimeric antigen receptor, or CAR, which acts sort of like a wanted poster, directing the T-cells to attack a specific target protein.
In TPST-4003, the CAR specifically targets two proteins that are produced by B-cells: CD19 and BCMA. By targeting both of these proteins at the same time, the experimental therapy is designed to trigger T-cells to eliminate a wide swath of B-cells, including those that make MG-driving antibodies.
According to Angel, this next-generation therapy “is being designed to enable broad B-cell lineage depletion and reset with its dual CD19/BCMA targeting architecture, with the goal of potentially delivering improved efficacy and durability compared with single-target CAR approaches.”
Traditionally, CAR T-cell therapies involve the collection of a patient’s T-cells, their modification in the lab to carry the CAR, and their infusion back into the patient. This process is laborious, time-consuming, and expensive.
TPST-4003 is an in vivo CAR T-cell therapy, meaning it’s designed to modify the T-cells within the patient’s body. It uses tiny fatty vesicles to deliver genetic instructions to produce the CAR specifically to T-cells by binding to CD7, a protein found at the cells’ surface.
This approach is expected to “achieve meaningful biological activity at lower doses, supporting improved manufacturing efficiency, reduced cost of goods, and the potential for scalable, repeatable treatment,” the release stated.
[This next-generation CAR T-cell therapy] is being designed … with the goal of potentially delivering improved efficacy and durability compared with single-target CAR approaches.
Angel added: “We believe our differentiated in vivo CAR-T platform may enable broader addressable T-cell coverage and support scalable repeat dosing once developed and commercialized, supporting future commercial attractiveness of in vivo CAR-T therapy.”
The main goal of the upcoming trial will be to track the treatment’s safety, cellular changes, and biological activity. Researchers will also explore TPST-4003’s effects on standard disease-specific clinical measures, the company stated.
Initial safety and biological activity data are anticipated in the first half of next year, while preliminary efficacy results are expected in the second half.
“We are excited to announce the strategic prioritization of our next-generation in vivo CAR-T pipeline and our plan to advance TPST-4003 toward an anticipated first patient dosing later this year,” Angel said.
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