The therapy is already being evaluated in patients with lupus nephritis and diffuse cutaneous systemic sclerosis.
The FDA has granted investigational new drug (IND) clearance for an open-label, phase 2 trial of Kyverna Therapeutics’ KYV-101 chimeric antigen receptor (CAR) T-cell therapy in 10 patients with myasthenia gravis.1
"We have seen firsthand the transformative effects of KYV-101 in MG patients treated with the investigational therapy in our clinic," said Prof. Dr. med. Aiden Haghikia, Director, Department of Neurology, Medical Faculty, Otto-von-Guericke University, Magdeburg, Germany, said in a statement.1 "I welcome the FDA's decision and look forward to more clinical data to further our knowledge about CAR T-cell therapy in patients with severe neurological autoimmune diseases."
KYV-101 will be evaluated for safety and tolerability in the new indication in the KYSA-6 trial. KYV-101 is also being evaluated in patients with lupus nephritis in the phase 1 KYSA-1 trial (NCT05938725) in the United States (US) and the phase 1/2 KYSA-3 clinical trial in Germany. For patients with diffuse cutaneous systemic sclerosis (scleroderma), KYV-101 is being assessed in the phase 1/2 KYSA-5 trial in the US.
"We are grateful that the FDA's decision to clear the IND for our Phase 2 KYSA-6 trial will allow Kyverna to offer this potentially paradigm-shifting investigational treatment to patients that may benefit from a deep B cell depletion and possibly durable reset of their immune system," Peter Maag, PhD, chief executive officer, Kyverna, added to the statement.1
KYV-101 is an autologous, fully human CD19 CAR T-cell designed to treat B-cell driven autoimmune diseases which was in part designed by the National Institutes of Health for oncological indications. Kyverna plans to explore the use of KYV-101 in other autoimmune diseases. The company stated that it had also obtained clearance of 2 additional INDs for investigator-initiated trials of KYV-101 in the US.
The IND clearance for Kyverna’s scleroderma program was granted in October 2023.2 Earlier, in September, the company announced that the first patient with lupus nephritis treated in the US and the therapy had been well-tolerated with 28 days of follow-up.3 The patient did not experience any immune effector cell-associated neurotoxicity syndrome (ICANS). The US trial is seeking to recruit approximately 9 to 12 patients with lupus nephritis in total across several clinical sites
"We welcome Kyverna's enthusiasm and interest to push forward with their CAR-T approach in scleroderma patients with the KYSA-5 trial," Luke Evnin, PhD, the chairman of the Scleroderma Research Foundation, added to the statement.2 "We await the results with optimism that CAR-T driven B-cell ablation may ultimately deliver efficacy with adequate safety for a broader range of our patients than immune-ablative chemotherapy alone."