H3 Biomedicine Inc., a clinical stage biopharmaceutical company specializing in the discovery and development of next-generation cancer medicines using its data science and precision chemistry product engine, today announced novel findings from a comprehensive genomic analysis of 6,235 patients across 15 hematologic malignancies. The results include the first-ever observance of recurrent RNA splicing factor mutations in non-Hodgkin’s lymphoma (NHL) and multiple myeloma (MM). While splicing factor mutations have been observed in other hematologic malignancies, including myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML) and chronic lymphocytic leukemia (CLL), the presence of these mutations in NHL and MM has not been reported previously.
Presented today at the 2018 American Association for Cancer Research (AACR) Annual Meeting in Chicago, these new findings demonstrate the continued emergence of splicing factor mutations as a hallmark of dozens of hematologic and solid tumor cancers, their potential role in tumor formation and growth, and, thus, the opportunity to advance a new class of therapies.
At the AACR meeting, Dominic Reynolds, Ph.D., Vice President of Chemistry at H3 Biomedicine, also gave an oral presentation discussing the discovery of H3B-8800, the Company’s first-in-class potent, selective and orally bioavailable small molecule modulator of the SF3b complex currently in Phase I clinical trials in patients with AML, CMML and MDS with splicing factor mutations.
"We continue to uncover new insights into the prevalence of splicing factor mutations across a broad spectrum of hematologic and solid tumor cancers and are leveraging this knowledge for our existing development programs and to inform the discovery of new targets and drugs," said Peter Smith, Ph.D., chief scientific officer at H3 Biomedicine. "For example, our work presented at AACR describes mutations that are addressed by our lead splicing modulator, H3B-8800, which we’re already evaluating in AML, MDS and CMML patients in an ongoing Phase 1 clinical study. These new findings of mutations in non-Hodgkin’s lymphoma and multiple myeloma could expand the addressable patient population for H3B-8800."
Dr. Smith continued, "Beyond our own development efforts, we hope the novel insights from this research will help advance the oncology community’s understanding of the pathogenesis of multiple myeloma and non-Hodgkin’s lymphoma and stimulate new drug discovery programs to help patients whose cancer cannot be effectively treated or cured with existing therapies."
The findings presented today were the result of an ongoing collaboration between H3 Biomedicine and Foundation Medicine Inc. (NASDAQ:FMI) to help advance the discovery and development of precision medicines in oncology. H3 Biomedicine scientists and scientists from Foundation Medicine jointly uncovered the mutations through computational biology based on the genomics data from FoundationOne®Heme, Foundation Medicine’s comprehensive genomic profiling (CGP) assay for hematologic malignancies and sarcomas. H3 Biomedicine is now performing additional translational research to validate the findings.
"Comprehensive genomic profiling (CGP) is a critical tool to drive the discovery and development of precision medicines in both hematologic and solid tumor cancers," said Vincent Miller, M.D., chief medical officer at Foundation Medicine. "The inherent complexity of all cancers mandates the use of an unbiased comprehensive approach in genomic profiling to speed target identification and therapeutic options. These results obtained using FoundationOneHeme and our FoundationCore™ database further underscore that premise. We look forward to understanding how these findings may translate to potential new therapeutic strategies for patients."
About the Findings
H3 Biomedicine and Foundation Medicine scientists surveyed somatic mutations of several splicing factors (SF3B1, SRSF2, U2AF1, ZRSR2, DDX3X, ZMYM3, PCBP1 and U2AF2) in 6,235 patients across 15 hematological malignancies. While these mutations have been observed in MDS, AML, CMML and CLL, the frequency of these ...
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