Experimental cell therapy KYV-101 wins RMAT designation for MG
Status intended to speed treatment's development, review
Kyverna Therapeutics’ experimental cell therapy, KYV-101, has been granted regenerative medicine advanced therapy (RMAT) status by the U.S. Food and Drug Administration (FDA) for treating progressive myasthenia gravis (MG).
RMAT designation is intended to expedite the development and FDA review of experimental regenerative treatments that treat, modify, reverse, or cure a serious or life-threatening condition and have preliminary clinical evidence that shows they can address an unmet need with that disease.
“The RMAT designation underscores the attention and interest by the FDA in the development of potentially transforming therapies targeting a severe autoimmune disease such as myasthenia gravis,” Srikanth Muppidi, MD, a neuromuscular disorder specialist at Stanford Medicine, said in Kyverna’s press release.
In most cases, MG is caused by self-reactive antibodies that target and attack important proteins required for nerve-muscle communication, leading to progressive muscle weakness and other symptoms. KYV-101 targets and eliminates B-cells, the immune cells that produce disease-causing antibodies.
Testing KYV-101 in MG, other diseases
A type of treatment known as CAR T-cell therapy wherein a patient’s own T-cells, another type of immune cell, are collected and modified to contain a man-made receptor called a chimeric antigen receptor (CAR). This CAR directs T-cells to attack a target.
With KYV-101, the CAR’s target is CD19, a protein on the surface of B-cells. When the engineered T-cells are infused back into the patient, they should target and eliminate CD19-expressing B-cells, reducing disease-causing antibodies.
So far, 50 patients with autoimmune conditions or cancer have been treated with the CAR in KYV-101 in more than 15 locations in Europe and the U.S., according to the company. In one case outside of any clinical trial, KYV-101 safely and effectively improved muscle strength and reduced fatigue in a woman with severe, treatment-resistant generalized myasthenia gravis (gMG).
Kyverna is also sponsoring several ongoing clinical trials testing KYV-101 for other autoimmune and/or neurological conditions.
The company announced KYSA-6, an open-label, multicenter Phase 2 clinical trial (NCT06193889) to test KYV-101 in people with MG in November 2023. KYSA-6 will enroll 20 patients, all with refractory, or treatment-resistant, MG who test positive for self-reactive antibodies. The study primarily aims to assess the therapy’s safety and tolerability, along with its efficacy at reducing disease severity.
“We are witnessing an era of profound changes in the approach to autoimmune conditions and ultimately, we hope this leads to a symptom-free state for patients,” said Muppidi, who’s also a principal investigator in the KYSA-6 trial.
RMAT status is similar in many ways to the FDA’s fast track designation, which the drug received in December 2023. Both incentivize early and intensive interactions between drug developers and the agency to expedite drug development and FDA review.
“We are very happy with the constructive scientific rapport established between Kyverna and the FDA,” said Peter Maag, PhD, CEO at Kyverna. “We believe the RMAT designation may ultimately add to our rigorous approach to KYV-101 development in the hope of benefiting the most deserving patients.”
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