On October 11, 2016 Cellerant Therapeutics, Inc., a clinical-stage company developing innovative cell- and antibody-based immunotherapies for hematologic malignancies (blood cancers) and other blood-related disorders, reported preclinical data showing potent killing of leukemic blast and stem cells by CSC030-ADC, the Company’s antibody drug-conjugate (ADC) product candidate being developed as a treatment for acute myeloid leukemia (AML) (Press release, Cellerant Therapeutics, OCT 11, 2016, View Source [SID:SID1234515756]). CSC030-ADC targets CSC030, or the C-type-like lectin 1 (CLL-1), a cell surface antigen widely expressed in nearly all AML cell subtypes, including leukemic stem cells. However, CSC030 is not expressed on normal hematopoietic stem and progenitor cells, making it a highly attractive target for an AML therapeutic. The data was presented today by Jagath Reddy Junutula, Ph.D., Vice President, Antibody Discovery & Development of Cellerant Therapeutics, at the 7th Annual World ADC meeting being held in San Diego, October 10-13, 2016.

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"We have shown that CSC030-ADC can specifically target and kill leukemic blast and stem cells," said Dr. Junutula. "Leukemic stem cells are believed to be responsible for tumor relapse, so an ADC that can target the leukemic stem cells precisely while minimally affecting normal stem cells would potentially have an important safety advantage compared to other targeted therapies for AML."

CSC030-ADC is a humanized monoclonal ADC that utilizes a potent DNA-damaging payload to target CSC030, an antigen expressed specifically on AML blasts and leukemic stem cells. The Company and others have shown that CSC030 is expressed in approximately 90% of all AML patient types, including all French American British (FAB) classifications, all cytogenetic risk categories, and in patients independent of FLT-3 status. When combined with a cytotoxic payload, the CSC030-ADC displayed potent target-dependent activity on several AML cell lines and robust tumor colony inhibition. CSC030-ADC also demonstrated a broad therapeutic index, e.g., greater than 10-fold therapeutic efficacy to toxicity, in AML tumor models. In addition, CSC030-ADC showed killing of both proliferating and quiescent CSC030-expressing cells. Importantly, CSC030 is not expressed on normal hematopoietic stem or progenitor cells and thus should have minimal effect on the formation of normal blood cell types. In contrast, the CD33 antigen is expressed on both normal stem and progenitor cells.

"We are excited with the preclinical results of CSC030-ADC and we look forward to advancing our product candidate to the clinic," said Ram Mandalam, Ph.D., President and Chief Executive Officer of Cellerant Therapeutics. "CSC030 is the first of multiple candidates identified through our stem cell target discovery efforts. In combination with Cellerant’s engineered antibodies and payload technology, we are well positioned to develop next generation of ADCs for the treatment of cancer."