Kyverna Therapeutics Conference Call Summary Company Overview - **Company**: Kyverna Therapeutics (NasdaqGS:KYTX) - **Focus**: Development of CAR-T therapies for autoimmune diseases, specifically targeting stiff person syndrome (SPS) with their lead product MIVCEL (mevalcaptagene autolysel) Key Industry Insights - **Industry**: Autoimmune therapies, particularly CAR-T cell therapies - **Market Opportunity**: Significant unmet medical need in SPS with no FDA-approved therapies currently available Core Points and Arguments 1. **Top-Line Data from KYZA-8 Trial**: - The trial met primary and all secondary endpoints with high statistical significance, demonstrating MIVCEL's effectiveness in reversing disability in SPS patients after a single dose [4][12][25] - MIVCEL showed a 74% improvement in the Timed 25-Foot Walk test, with patients' times improving from an average of 17.3 seconds to 4.5 seconds [8][18] 2. **Regulatory Pathway**: - BLA submission to the FDA is on track for the first half of 2026, supported by the trial's results and designations such as RMAT and orphan drug status [4][13][25] 3. **Patient Population**: - The trial included 26 patients with a median age of 56, all of whom had previously inadequate responses to off-label immunotherapies [15][16] - 88% of patients were GAD65 positive, the most common autoantibody associated with SPS [16] 4. **Efficacy and Safety**: - MIVCEL demonstrated deep and broad B-cell depletion, with durable efficacy beyond 24 months in some patients [10][11][31] - The safety profile was well-tolerated, with no high-grade CRS or ICANS observed [21][22] 5. **Market Dynamics**: - Approximately 6,000 diagnosed patients in the U.S. with SPS, with 2,000 to 2,500 patients identified as having the highest unmet need [26][27] - The company aims to establish MIVCEL as the first FDA-approved therapy for SPS, targeting patients with inadequate responses to current treatments [27][29] 6. **Commercial Strategy**: - Focused commercialization strategy leveraging existing CAR-T ecosystem and targeting academic centers where patients are concentrated [29][30] - Plans to expand into broader indications, including myasthenia gravis, following the potential approval of MIVCEL [5][26] Additional Important Insights - **Patient Impact**: The psychological and physical burden of SPS is significant, with many patients experiencing severe mobility issues and requiring walking aids [9][10] - **Transformative Potential**: MIVCEL's ability to eliminate the need for chronic immunotherapies and improve quality of life is emphasized as a game changer for SPS patients [19][24] - **Future Directions**: Kyverna is also exploring other autoimmune diseases, with promising early data in multiple sclerosis and rheumatoid arthritis [31][54] Conclusion - Kyverna Therapeutics is positioned to potentially lead the CAR-T therapy market for autoimmune diseases, with MIVCEL showing promising results in treating stiff person syndrome and a clear path towards regulatory approval and commercialization.

Cabaletta Bio (NasdaqGS:CABA) FY Conference December 02, 2025 08:20 AM ET Company ParticipantsSteven Nichtberger - CEOSteve Gavel - Chief Commercial OfficerDavid Chang - Chief Medical OfficerModeratorAll right, we're on. Thanks for tuning in, everyone. Next up, we have the full team from Cabaletta. We have CEO Steven Nichtberger, David Chang, CMO, and Steve Gavel, Chief Commercial Officer. Thanks for joining us, guys. Steven, I'm going to turn it over to you just for an overview of the company, where things ...