CASI Pharmaceuticals presented Phase 1 open‑label data for its anti‑CD38 monoclonal antibody CID‑103 in adult patients with immune thrombocytopenia (ITP) at the 67th American Society of Hematology (ASH) Annual Meeting in Orlando, Florida, on December 7, 2025.
The study enrolled 11 patients across five dose‑escalation cohorts (30 mg, 150 mg, 300 mg, 600 mg, and 900 mg) with a priming dose of either 30 mg or 150 mg. Eight of the 11 patients (73%) met the primary efficacy endpoint, and six of those eight responders (75%) achieved a complete response. Platelet improvement was observed as early as one week after dosing, and the safety profile was manageable, with two Grade 3 treatment‑related events and no dose‑limiting toxicities; infusion‑related reactions were low‑grade and associated with the priming dose.
In its most recent quarterly report, CASI reported a net loss of $10.9 million for the third quarter of 2025, up from $8.4 million in the same period last year. Revenue for the quarter fell to $3.1 million, a 60% decline from $7.8 million in Q3 2024, largely driven by goods returns for its EVOMELA product. The company’s cash and cash equivalents have also decreased, underscoring the financial headwinds it faces as it advances CID‑103.
Global Chief Medical Officer Dr. Alex Zukiwski said the safety and tolerability of CID‑103 were encouraging and that the 73% primary endpoint rate was a positive signal for the drug’s potential in ITP. CEO David Cory reiterated the company’s focus on advancing CID‑103 for organ transplant rejection and autoimmune diseases, noting that the Phase 1 data represent the first clinical evidence of the antibody outside its multiple‑myeloma program.
The results provide a promising first step toward a new therapeutic option for ITP, but CASI’s financial trajectory remains a concern. The company’s sole remaining asset is CID‑103, and its continued development will be critical to reversing the current revenue decline and restoring investor confidence. The Phase 1 data, while encouraging, must be followed by larger, controlled studies to confirm efficacy and safety before the drug can reach the market.
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