Kyverna secures RMAT status for stiff-person syndrome cell therapy

Six months after receiving fast track status for treating multiple sclerosis, Kyverna Therapeutics’ KYV-101 has been granted another significant designation by the US Food and Drug Administration (FDA). This time, the treatment has earned a regenerative medicine advanced therapy (RMAT) status for its potential in addressing stiff-person syndrome.

The RMAT designation provides numerous benefits, such as priority review and accelerated approval, similar to the fast track and breakthrough therapy pathways. Additionally, it allows the use of surrogate endpoints in clinical trials, which can expedite the approval process. Following this announcement, shares in Kyverna Therapeutics rose by 9.5% from their pre-announcement levels on 15 July, reflecting investor optimism. The company currently has a market capitalization of $427 million.

KYV-101 is a CD19 chimeric antigen receptor (CAR)-T cell therapy developed for B cell-driven autoimmune disorders. Its current evaluation is in a Phase II trial for patients suffering from treatment-refractory stiff-person syndrome. The trial, designated KYSA-8, was recently approved by the FDA. According to a press release dated 16 July, the RMAT application for KYV-101 was bolstered by positive clinical outcomes observed in patients treated in Germany under a named patient treatment option.

CEO Peter Maag expressed the company's eagerness to start gathering data from the US trial, although further details about the study have not been disclosed. Stiff-person syndrome is a chronic, progressive autoimmune disorder that results in severe muscle stiffness and spasms, primarily affecting the torso and limbs. This condition can lead to significant complications, as patients are prone to breaking bones due to a lack of muscle reflexes necessary to prevent falls. The exact cause of stiff-person syndrome remains unknown.

Kyverna is venturing beyond traditional blood cancer treatments with its cell therapy KYV-101. The therapy is currently being tested in various sponsored Phase I/II and Phase II trials across the US and Germany for conditions such as refractory lupus nephritis, multiple sclerosis, and myasthenia gravis. Earlier this year, Kyverna announced positive Phase I results for KYV-101 in multiple sclerosis patients, an indication for which it already has fast track status from the FDA. To date, approximately 50 patients with cancer or autoimmune disorders have been treated with KYV-101.

In addition to its clinical progress, Kyverna Therapeutics went public earlier this year, successfully raising $319 million through its initial public offering (IPO). The IPO saw the company sell 14.5 million shares at $22 each, surpassing initial expectations.

Celine Dion has also brought attention to stiff-person syndrome, a rare autoimmune disorder, further highlighting the importance of developing effective treatments for this debilitating condition. The recent advancements by Kyverna Therapeutics signify a promising step forward in the fight against various autoimmune diseases.