CASI Pharmaceuticals Announces Preliminary Results of CID-103 Clinical Trial

CASI Pharmaceuticals announced data presented from its Phase 1 open-label study of CID-103 in adult patients with immune thrombocytopenia at the 67th American Society of Hematology Annual Meeting and Exposition on December 7, 2025, in Orlando, Florida. This open-label Phase 1 dose escalation study assigned patients to sequential dose-escalating cohorts of intravenous infusions of CID-103 at 30 mg, 150 mg, 300 mg, 600 mg, and 900 mg, with a priming dose of CID-103 of either 30 mg or 150 mg administered prior to the cohort dose. Primary objective is to evaluate safety and tolerability of multiple escalating doses of CID-103. Primary efficacy endpoint is the proportion of patients achieving a platelet response, defined as a platelet count greater than or equal to 50 x 109/L and greater than or equal to 20 x 109/L above baseline on at least 2 consecutive measurements at least 7 days apart within the first twelve weeks of treatment. Other endpoints were evaluated including pharmacokinetic and pharmacodynamic markers. As of cut-off date of November 28, eleven patients have been enrolled and dosed. Interim Results: CID-103 demonstrated a manageable safety profile with only two Grade 3 treatment-related events and no dose limiting toxicities observed; All infusion-related reactions occurred with priming dose and are due to low grade AEs; Primary Efficacy Endpoint achieved in 8 of 11 patients; 6 of 8 patients achieved Complete Response with platelet improvement observed as early as one week post dose; Reduction of PD markers is consistent with the presumed CID-103 MOA resulting in the observed platelet response. "We are pleased with the safety and tolerability of CID-103 and encouraged by the 73% of patients achieving the primary efficacy endpoint," said Alex Zukiwski, CMO. "Importantly, this study provides important clinical proof of concept supporting further development of CID-103 in autoimmune disorders, solid organ transplant rejection, and other CD38 mediated diseases with large unmet medical need."