Our novel all antibody condition regimen seeks to address the limitations of current stem cell transplantation, or SCT, conditioning regimens, which utilize chemotherapy and/or radiation to kill HSCs and make space for transplanted cells. These regimens are highly toxic, induce immune suppression and may require hospitalization. Numerous comorbidities are often observed, including severe infections, impaired brain development, infertility, endocrine dysfunction, secondary malignancies and organ damage. As a result, many patients are ineligible for, or choose not to undergo, SCT.
In the preclinical data presented at ASH,FSI-174 demonstrated binding affinity to both human and NHP cKIT receptors, and induced phagocytosis as well as antibody-dependent cell-mediated cytotoxicity in a dose-dependent manner. Additionally,FSI-174 was well-tolerated when administered to NHPs as a single agent, with no dose-limiting toxicities. Theno-observed-adverse-effect level was established at 50 mg/kg, the highest dose tested.
When administered together,FSI-174 and magrolimab demonstrated a synergistic benefit, promoting the highest level of phagocytosis of cKIT expressing target cells and inducing significant HSC depletion nine days after infusion compared to placebo. As expected, there were no changes in blood cell counts over the course of the study, with no cytopenias observed with either monotherapy or combination treatment. These pharmacodynamic and NHP data demonstrate the specificity and safety ofFSI-174 and magrolimab, and reveal a window for SCT where the cKIT antibody is washed out, but the depletion of HSCs is sustained long enough to enable the transplantation of donor HSCs.
We believe that a safe, well-tolerated conditioning regimen may substantially broaden the population eligible for SCT, potentially expanding its use for a wide range of genetic blood disorders, as well as for autoimmune diseases, leukemias and lymphomas.
We expect to complete investigational new drug (IND)-enabling studies by the end of 2019, and subject to approval of the IND by the FDA, we plan to initiate a Phase 1 clinical trial evaluatingFSI-174 in healthy volunteers in the first quarter of 2020.
Corporate Information
We were incorporated in Delaware in 2014 as CD47 Sciences, Inc. Our principal executive offices are located at 1490 O’Brien Drive, Suite A, Menlo Park, California 94025, and our telephone number is (650)352-4150.
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