Workflow
Kezar Life Sciences(KZR) - 2024 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The Portola trial enrolled 24 patients, with 21 in a prespecified subgroup on steroid therapy. 36% of patients receiving zetomipsemib achieved complete biochemical remission by week 24 compared to none in the placebo arm [4][16] - The safety profile of zetomipsemib was favorable, with most treatment-emergent adverse events being grade one and two, primarily related to injection site reactions [5][19] Business Line Data and Key Metrics Changes - The trial demonstrated that responses to zetomipsemib were durable, with no flares reported for patients achieving complete remission [4][16] - 20 patients completed the double-blind treatment period, with 16 eligible for the open-label extension, indicating a strong continuation of treatment [11] Market Data and Key Metrics Changes - Autoimmune hepatitis affects nearly 100,000 patients in the United States, with a significant unmet need for effective treatments [5][21] - The trial results suggest that zetomipsemib could transform the treatment landscape for autoimmune hepatitis, which currently lacks additional therapeutic options [7][21] Company Strategy and Development Direction - The company aims to respond to the FDA regarding the partial clinical hold on the Portola study and plans to align with regulatory bodies for a potential registrational study of zetomipsemib in autoimmune hepatitis [51] - Future studies may mandate steroid tapering as a key endpoint, reflecting a commitment to improving treatment protocols [61] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the data from the Portola study, highlighting the potential for zetomipsemib to address the needs of patients with autoimmune hepatitis [22][51] - The management acknowledged the challenges in designing a phase three study due to the lack of regulatory precedent but emphasized the importance of biochemical remission and histologic improvement as endpoints [80] Other Important Information - The trial included an open-label extension for some patients, with ongoing data collection to assess long-term outcomes [11][51] - The company highlighted the need for new agents in the treatment of autoimmune hepatitis, as current therapies have significant side effects and limited efficacy [21][47] Q&A Session Summary Question: Concerns with zetomipsemib and lupus nephritis - Craig Lammert noted that injection site reactions were minor and manageable, with no significant safety concerns observed in the Portola study [56][57] - Christopher Kirk added that systemic injection reactions were consistent with previous studies, indicating a tolerable safety profile [59] Question: Biochemical remission without steroid taper - Christopher Kirk explained that the protocol suggested but did not mandate steroid tapering, with physicians making decisions based on individual patient responses [61] - Craig Lammert emphasized the real-world challenges in managing autoimmune hepatitis and the need for a more rigorous approach in future studies [63][64] Question: Infection rates in the trial - Divya Rao inquired about the higher infection rates in the placebo arm, to which Christopher Kirk responded that overall infection rates were consistent with previous trials [67] - Gideon Hirschfield noted that corticosteroids are a significant risk factor for infections, highlighting the unmet need for better therapies [70] Question: Clinical meaningfulness of the data - Craig Lammert discussed the importance of reducing liver test abnormalities and corticosteroid dependency, emphasizing the potential impact on patient quality of life [76] - Gideon Hirschfield agreed, stating that the trial population reflected patients struggling to control their disease, and the results indicated a meaningful biological effect [78] Question: Designing a phase three study - Christopher Kirk acknowledged the lack of regulatory guidance but emphasized the need for alignment with the FDA on trial design and endpoints [80]