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news.The US Food and Drug Administration (FDA) has granted orphan drug designation for H3 Biomedicine's H3B-8800, its lead clinical compound for the treatment of patients with Acute Myelogenous Leukemia (AML) and Chronic Myelomonocytic Leukemia (CMML).
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H3B-8800, which is a potent, selective and orally bioavailable small molecule modulator of wild-type and mutant SF3b complexes, is currently in Phase 1 clinical trials.
“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.
“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.”
The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S. The orphan drug designation provides H3 Biomedicine with certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
About Acute Myelogenous Leukemia (AML)
According to the American Cancer Society, acute myeloid leukemia (AML) is also called acute myelocytic leukemia, acute myelogenous leukemia, acute granulocytic leukemia, acute non-lymphocytic leukemia, or sometimes just AML. It is most common in older people. For more information, please visit www.cancer.org/cancer/acute-myeloid-leukemia.html.
About Chronic Myelomonocytic Leukemia (CMML)
According to the American Cancer Society, Chronic myelomonocytic leukemia (CMML) is a type of cancer that starts in blood-forming cells of the bone marrow and invades the blood. It affects mainly older adults. For more information, please visit www.cancer.org/cancer/chronic-myelomonocytic-leukemia.html.
About H3B-8800
H3B-8800 is an oral, potent and selective small molecule modulator of splicing factor 3b subunit 1 (SF3B1) that is being developed by H3 Biomedicine as an anticancer therapeutic agent. In pre-clinical studies, H3B-8800 showed dose dependent modulation of canonical and aberrant splicing when dosed orally at tolerated doses.
Oral administration of H3B-8800 demonstrated preferential antitumor activity in several pre-clinical xenograft models carrying spliceosome mutations. H3 Biomedicine’s lead research and discovery programs in splicing are designed to develop drugs that target the vulnerabilities related to deregulated RNA homeostasis in cancer.
