FDA Clears IND Application for CID-103 in Immune Thrombocytopenia

The US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for CID-103 for the treatment of adults with chronic immune thrombocytopenia (ITP).¹

Announced by CASI Pharmaceuticals on May 15, 2024, the IND application was initially submitted to the FDA in April to support a phase ½ study of CID-103 in adults with ITP. On May 13, the company received a letter from the FDA stating the study may move forward.

“ITP is a serious autoimmune blood disorder characterized by autoantibody-mediated platelet destruction and impaired platelet production, leading to thrombocytopenia and an increased risk of life-threatening bleeding episodes,” Wei-Wu He, PhD, chief executive officer of CASI Pharmaceuticals, said in a statement. “We are excited to advance this program into clinical development as CID-103 has the potential to represent a new therapeutic option to help alleviate the disease burden in this patient community."

The therapy, CID-103, is a fully human IgG1 monoclonal antibody (Mab) directed against the cell glycoprotein ADP-ribosyl cyclase 1 (CD83), with potential antineoplastic activity. At administration, CID-103 specifically targets and binds to CD38 expressions. CD83 is a type II glycoprotein present on various immune cells and hematologic malignancies.

Early preclinical data of CID-103 have suggested enhanced activity against malignancies and a better preclinical safety profile, compared with other anti-CD38 monoclonal antibodies, according to CASI Pharmaceuticals. The company is also evaluating CID-103 in a phase 1 dose escalation and expansion trial for relapsed or refractory multiple myeloma.

“CID-103 has previously shown encouraging preclinical efficacy, a favorable safety profile and greater antibody-dependence cellular cytotoxicity activity over other anti-CD38 mAbs, and we are hopeful this will translate into patient benefit,” He previously stated. "This Phase 1 trial will generate valuable information and has the potential to provide early evidence of clinical activity in the treatment of multiple myeloma.”

References

1. _{Pharmaceuticals C. Casi Pharmaceuticals receives FDA clearance on the Investigational New Drug (IND) application for CID-103 in immune thrombocytopenia (ITP). PR Newswire: press release distribution, targeting, monitoring and marketing. May 15, 2024. Accessed May 15, 2024. https://www.prnewswire.com/news-releases/casi-pharmaceuticals-receives-fda-clearance-on-the-investigational-new-drug-ind-application-for-cid-103-in-immune-thrombocytopenia-itp-302145848.html.}
2. _{CASI Pharmaceuticals Announces Dosing Of First Patient Of CID-103 In Phase 1 Clinical Trial For Relapsed Or Refractory Multiple Myeloma. June 2021. Accessed May 15, 2024. https://www.casipharmaceuticals.com/mediapress/19.153.html.}