On May 12, 2026 BlossomHill Therapeutics, Inc., a privately-held, clinical-stage biopharmaceutical company applying an intentional, chemistry-based approach to develop innovative small molecule medicines for the treatment of cancer, reported that initial dose escalation data from its Phase 1/1b trial of BH-30236, a macrocyclic CDC-link kinase (CLK) inhibitor, in relapsed or refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndrome (HR-MDS) will be presented during a poster session on June 12, 2026, at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress in Stockholm, Sweden. Additionally, BH-30236 has been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of AML.
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"We are highly encouraged by the initial safety data and early signs of anti-leukemic activity observed with BH-30236, both as a monotherapy and in combination with venetoclax," said Jean Cui, Ph.D., Founder and Chief Executive Officer of BlossomHill Therapeutics. "By modulating aberrant alternative mRNA splicing, our potential first-in-class CLK inhibitor, BH-30236, represents a novel approach for patients with relapsed or refractory AML and higher-risk MDS, who face a significant unmet medical need. We look forward to presenting additional data from more patients with longer follow-up at the meeting and to advancing BH-30236 in both monotherapy and combination settings."
BH-30236 was designed to target the CLK kinase family, leading to the modulation of aberrant alternative mRNA splicing, a defining feature implicated in cancer progression and therapeutic resistance across both hematologic malignancies and solid tumors. Our Phase 1/1b trial evaluating BH-30236 was initiated following encouraging preclinical data, including evidence of synergistic activity between BH-30236 and venetoclax, a BCL-2 inhibitor and an established standard of care therapy for patients with AML. The trial is currently enrolling patients in dose escalation, evaluating BH-30236 as both a monotherapy and in combination with venetoclax.
As of the January 23, 2026 cutoff date for the EHA (Free EHA Whitepaper) abstract submission, 28 patients received BH-30236 monotherapy at 5-120 mg on a continuous daily administration schedule (QD) and 11 patients received BH-30236 at 20-60mg QD in combination with venetoclax. Initial findings demonstrated:
BH-30236 was generally well tolerated as both a monotherapy and in combination with venetoclax, with most treatment-related adverse events being low-grade and manageable, as well as one grade 3 DLT (diarrhea)
Dose escalation showed predictable pharmacokinetics without drug accumulation or reduction, and no significant drug-drug interactions were observed with venetoclax
Early signs of clinical activity were observed:
In the monotherapy cohort, 29% (n=5) of evaluable patients achieved at least a 50% reduction in bone marrow blast counts, including one HR-MDS patient treated at 60 mg with ongoing blast count reduction with duration of treatment 7.6 months.
In the combination cohort, 55% (n=5) of evaluable patients experienced at least a 50% blast reduction, including one patient refractory to all prior therapy including venetoclax, who achieved a minimal residual disease (MRD)-negative complete remission.
"In March of this year, the U.S. Food and Drug Administration granted Orphan Drug Designation to BH-30236 for the treatment of acute myeloid leukemia, underscoring the need for new therapies in this rare malignancy," said Dr. Geoff Oxnard, Chief Medical Officer of BlossomHill Therapeutics. "This designation is an important milestone that reflects the potential of BH-30236 to address a significant unmet need and provides benefits that may support our ongoing clinical development, as well as potential market exclusivity upon approval."
Poster Session Details
Title: A First-in-Human Study of the Oral CLK Inhibitor BH-30236 in Adults with Relapsed/Refractory Acute Myeloid Leukemia or Higer-Rish Myelodysplastic Syndrome: Monotherapy and Venetoclax Combination
Presenting Author: Eytan M. Stein, MD, Chief of the Leukemia Service, Associate Attending Physician, Clinical Investigator and Director of the Program for Drug Development in Leukemia on the Leukemia Service at Memorial Sloan Kettering Cancer Center
Date & Time: Friday, June 12 from 12:45 to 1:45 ET / 18:45 – 19:45 CEST
Abstract Code: PF494
About BH-30236
BH-30236 is an investigational orally bioavailable, macrocyclic inhibitor of the CDC-like kinase (CLK) family. BH-30236 is designed to modulate aberrant alternative splicing in cancerous tissue, targeting the same aberrant splicing machinery that drives acute myeloid leukemia (AML) and higher-risk myelodysplastic syndrome (HR-MDS) disease biology and that cancer cells exploit to develop resistance to venetoclax, FLT3 inhibitors and cytarabine. BH-30236 is currently in clinical development for the treatment of relapsed or refractory AML (R/R AML) and HR-MDS.
BH-30236 is being evaluated in a Phase 1/1b multicenter, open-label, first-in-human dose escalation and expansion trial in adults with R/R AML and HR-MDS. The U.S. Food and Drug Administration has granted orphan drug designation to BH-30236 for the treatment of AML.
(Press release, BlossomHill Therapeutics, MAY 12, 2026, View Source [SID1234665580])