Acute Myelogenous Leukemia Treatment Gets Orphan Drug Designation

Mathew Shanley

H3 Biomedicine’s lead clinical compound, H3B-8800, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) earlier this morning for the treatment of patients with acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML).
H3B-8800 is an orally-administered, small molecule modulator of wild-type and mutant SF3b complexes. It is currently in Phase 1 clinical trials.
AML is a rapidly-progressing cancer of the blood and bone marrow that is characterized by the speedy expansion of white blood cells. These cells, when grown, interfere with the production of normal blood cells. In CMML, there are an increased amount of large white blood cells, or monocytes, and immature blood cells, or precursor cells, in the bordering blood and bone marrow.
Per the American Cancer Society, both AML and CMML are most common in older adults.
“Receiving the orphan drug designation for H3B-8800 is a critical milestone for H3’s ongoing cancer genomics driven drug discovery program,” said Markus Warmuth, M.D., President and CEO of H3 Biomedicine in a press release. “We are pleased with the progress our scientific and clinical teams are making, and look forward to continue investigating H3B-8800 as a potential treatment option for patients with these diseases.”
Preclinical data indicates that H3B-8800 modulates ribonucleic acid (RNA) splicing, and displays dose-dependent modulation of common and uncommon splicing. H3B-8800 exhibits preferential antitumor activity in several pre-clinical xenograft models carrying spliceosome mutations.
Initial clinical development is ongoing in patients with hematological malignancies like AML and CMML that may carry mutations in the core spliceosome genes. These clinical trials will assess the safety and preliminary efficacy of H3B-8800.
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