Biosplice Therapeutics Announces First Patient Dosed in NCI-Sponsored Clinical Trial of Cirtuvivint in Acute Myeloid Leukemia and Myelodysplastic Syndromes

Excerpt from the Press Release:

SAN DIEGO, Sept. 09, 2025 (GLOBE NEWSWIRE) — Biosplice Therapeutics, Inc. (“Biosplice”), a clinical-stage biotechnology company pioneering advancements in small molecule inhibition of CDC-like kinases (CLK) and Dual-specificity tyrosine phosphorylation-regulated (DYRK) kinases, today announced that the first patient has been dosed in a Phase 1 clinical trial of cirtuvivint in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The study is sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health (NIH), and conducted by the NCI funded Experimental Therapeutics Clinical Trials Network, under a Cooperative Research and Development Agreement (CRADA) with Biosplice. This milestone highlights the ongoing collaboration between Biosplice and the NCI’s Cancer Therapy Evaluation Program (CTEP). The trial (NCT06484062) is evaluating the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of cirtuvivint, both as a monotherapy and in combination with ASTX727 (INQOVI, oral decitabine/cedazuridine), a hypomethylating agent developed by Taiho Oncology, Inc. and FDA-approved for MDS. The study is enrolling patients with relapsed or refractory AML or MDS, and is led by Principal Investigator Dr. Evan Chen at Dana-Farber Cancer Institute.

“We are pleased to see continued momentum in cirtuvivint’s development with this important milestone in our collaboration with the NCI,” said Yusuf Yazici, M.D., Chief Medical Officer at Biosplice. “AML and MDS remain areas of significant unmet need, and we are hopeful that cirtuvivint’s unique mechanism, modulating RNA splicing to disrupt oncogenic signaling, will demonstrate benefit for patients with these challenging malignancies. The combination with a well-established agent such as ASTX727 offers a compelling approach to enhancing therapeutic response.”

Cirtuvivint is a selective pan-CLK, pan-DYRK inhibitor that modulates alternative RNA splicing, including transcripts involved in oncogenic pathways. Preclinical studies have shown that CLK/DYRK inhibition alters splice patterns in malignant cells, suppresses oncogenic variants, and enhances apoptosis. The combination with ASTX727 aims to leverage two complementary mechanisms, hypomethylation (ASTX727) and splicing modulation (cirtuvivint), to target leukemic cells more effectively.

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