SAN DIEGO, Nov. 03, 2025 (GLOBE NEWSWIRE) -- BlossomHill Therapeutics, Inc., a privately-held, clinical-stage biopharmaceutical company focused on the design and development of next-generation medicines for cancer, today announced an upcoming poster presentation at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, being held December 6-9, 2025 in Orlando, FL. The trial-in-progress poster will highlight BlossomHill’s ongoing, first-in-human, Phase 1/1b dose escalation study of BH-30236 in relapsed or refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndrome (HR-MDS). BH-30236 is a novel, orally available, macrocyclic inhibitor of CDC-like kinase (CLK) designed to address dysregulated RNA splicing events that drive tumor growth and disease progression.
“Targeting the spliceosome offers a new opportunity to expand the targetable proteome, potentially addressing treatment resistance in a new way. We are excited to present design details for our first-in-human trial for our CLK inhibitor, BH-30236, now that we have initiated our first therapeutic combination to test this thesis,” said Geoff Oxnard, M.D., Chief Medical Officer of BlossomHill Therapeutics.
“Aberrant alternative splicing is an important driver of disease progression and therapeutic resistance in AML and MDS,” said Eytan Stein, M.D., Chief, Leukemia Service, Memorial Sloan Kettering Cancer Center. “By targeting CLK as a master regulator of RNA splicing machinery, BH-30236 represents an innovative therapeutic approach for patients with limited treatment options.”
The multi-center, open-label, Phase 1/1b dose escalation study is designed to evaluate the safety, tolerability, pharmacokinetics and preliminary anti-leukemic activity of BH-30236 as a monotherapy or in combination with venetoclax in adult participants with R/R AML or HR-MDS. For additional information, including a list of study sites and how to enroll, please visit at clinicaltrials.gov (NCT06501196).
Details of the poster presentation are as follows:
Title: A phase 1/1b open-label, dose escalation, first-in-human study to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-leukemic activity of the orally available clk inhibitor, BH-30236, in adults with R/R AML or HR-MDS
Publication Number: 1655
Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster I
Session Date and Time: Saturday, December 6, 2025, 5:30 - 7:30 p.m. ET
Location: Orange County Convention Center - West Halls B3-B4
About BH-30236
BH-30236 is a novel, oral, macrocyclic, ATP-competitive, highly potent and selective inhibitor of CDC-like kinase (CLK). By modulating RNA splicing through selective inhibition of CLK enzymes, BH-30236 aims to address aberrant splicing events that drive tumor growth and disease progression in certain hematological malignancies such as acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). In preclinical studies, BH-30236 demonstrated in vitro efficacy in patient-derived AML cells, including cells insensitive to venetoclax. BH-30236 also modulated DNA repair pathway and apoptosis families, leading to enhanced cancer cell apoptosis via splicing regulation. Further, BH 30236 demonstrated in vitro and in vivo synergy with venetoclax and the combination of BH 30236 with venetoclax demonstrated substantially better efficacy than standard of care venetoclax + azacytidine in venetoclax resistant CDX AML models.
BH-30236 is currently being evaluated as a monotherapy or in combination with venetoclax in a first-in-human, Phase 1/1b dose escalation study in adult participants with relapsed or refractory acute myeloid leukemia (R/R AML) or higher-risk myelodysplastic syndrome (HR-MDS).
About BlossomHill Therapeutics
BlossomHill Therapeutics, Inc. is a privately held, clinical-stage biopharmaceutical company focused on designing and developing next-generation targeted therapies for cancer. Founded and led by industry veterans with a proven track record in oncology drug discovery and development – including multiple FDA-approved therapies – BlossomHill applies cutting-edge science to address key oncogenic drivers and improve patient outcomes in difficult-to-treat cancers. The company’s lead clinical programs include BH-30643, a first-in-class, macrocyclic, non-covalent, mutant selective OMNI-EGFRTM inhibitor for the treatment of EGFR- or HER2-mutated non-small cell lung cancer (NSCLC), and BH-30236, a macrocyclic CLK inhibitor for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML) or higher-risk myelodysplastic syndrome (HR-MDS), representing a first-in-class opportunity. BlossomHill Therapeutics is headquartered in San Diego, California and is supported by leading life sciences investors, including Cormorant Asset Management, OrbiMed, Vivo Capital and Colt Ventures. For more information, visit bhtherapeutics.com and follow us on LinkedIn and X.
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1AB
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