Six months after winning fast track status for multiple sclerosis, Kyverna Therapeutics’ KYV-101 has secured another US Food and Drug Administration (FDA) designation; this time, a regenerative medicine advanced therapy (RMAT) tag for the treatment of stiff-person syndrome.
An RMAT designation includes those benefits included in the fast track and breakthrough therapy pathways such as a priority review, along with early and enhanced FDA interactions. The RMAT tag also permits the use of surrogate endpoints in clinical trials studying the therapy, which can lead to accelerated approval.
Shares in the US biotech rose 9.5% at market open compared to a pre-announcement 15 July market close. Kyverna has a market cap of $427m.
Kyverna’s KYV-101 is a CD19 chimeric antigen receptor (CAR)-T cell therapy designed for B cell-driven autoimmune disorders. It is currently being evaluated in a Phase II trial in patients with treatment-refractory stiff-person syndrome. The study, named KYSA-8, received the FDA greenlight last month.
The RMAT application’s success was based on the positive clinical outcomes of KYV-101 in patients treated in Germany under a named patient treatment option, according to a 16 July press release.
Kyverna has not revealed further information about the US trial, with the biotech’s CEO Peter Maag saying “we are eager to begin generating data”.
Stiff-person syndrome is a progressive autoimmune disorder that causes muscle stiffness and spasms in the torso and limbs. Many patients can break bones from falling as they lack the necessary muscle reflexes for correction. It is not known what causes the disease.
Kyverna is considering cell therapy treatments beyond blood cancers, the space in which many CAR-T treatments are currently approved to treat. KVY-101 is being evaluated in sponsored Phase I/II and Phase II trials in the US and Germany in indications such as refractory lupus nephritis (NCT05938725), multiple sclerosis, and myasthenia gravis. The biotech reported positive Phase I results for the cell therapy in multiple sclerosis patients in April this year, the indication for which the drug has fast track status from the FDA.
In total, Kyverna says that 50 patients with cancer or autoimmune conditions have been treated with KYV-101 so far.
Kyverna went public earlier this year, raising $319m in the process. The initial public offering (IPO), which was larger than expected, saw the company sell 14.5 million shares for $22 each.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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