Kyverna Therapeutics, under new CEO Warner Biddle, has refined its strategic focus for its CAR-T cell therapy program, KYV-101, prioritizing stiff person syndrome (SPS) as its initial target for approval. Here’s a summary:

  • New Strategic Focus: Kyverna aims to be the first company to achieve approval for a CAR-T therapy in an autoimmune disease.
  • Lead Program (KYV-101):
    • Primary Indication: Stiff Person Syndrome (SPS): This rare neurological disorder, recently brought into public awareness by Céline Dion’s diagnosis, is now Kyverna’s top priority. Biddle believes targeting this unmet need will allow Kyverna to establish itself as a leader in autoimmune CAR-T. A Phase 2 trial (KYSA-8) is underway with 40% enrollment complete and full enrollment expected mid-2025. Top-line data is expected in H1 2026, with a potential application in 2026.
    • Secondary Indication: Myasthenia Gravis: This condition is now the second priority. Kyverna plans to confirm a regulatory path in H1 2025 and report interim Phase 2 (KYSA-6 trial) data in H2 2025.
    • Tertiary Indication: Lupus: While lupus was the initial focus and drove significant investment, it has been moved to third priority due to increased competition. Kyverna is completing enrollment in the Phase 1 lupus nephritis trial and aims to report data from nine patients (including six at the target dose) in H2 2025 to inform the design of a pivotal trial. They are focusing on lupus nephritis specifically due to the high risk of progression to late-stage renal disease in this patient population.
  • Rationale for Prioritization: Biddle cited the significant unmet need in SPS and the potential for faster clinical trial execution as reasons for prioritizing this indication. The smaller patient population in SPS also offers a more manageable initial target.
  • Past Performance and Market Context: Kyverna experienced a significant stock price drop after its IPO despite initial enthusiasm for CAR-T in lupus following promising academic results. KYV-101 is one of the first CAR-T therapies to report clinical data in lupus, showing promise for patients with lupus nephritis. A single infusion has demonstrated deep B-cell depletion, halted disease activity, and restored kidney function, eliminating the need for immunosuppressants and steroids. However, the field is becoming increasingly competitive with both large pharmaceutical companies and other biotechs entering the space. A patient relapse in an earlier KYV-101 lupus trial highlights the challenges of this therapeutic approach.
  • Other Programs: KYV-101 is also being tested in multiple sclerosis and systemic sclerosis. Initial Phase 2 data in multiple sclerosis was inconclusive. Kyverna’s second candidate, KYV-102 (designed to eliminate the apheresis process in CAR-T manufacturing), aims for an IND filing in H2 2025.
  • Financial Position: Kyverna has sufficient cash to operate into 2027 and is open to partnerships that contribute to future growth.

 

In summary, Kyverna is strategically pivoting to prioritize SPS for its lead CAR-T therapy, aiming for a faster path to market and establishing a strong foothold in the autoimmune CAR-T field. While lupus remains a key target, it has been deprioritized due to competitive pressures.

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