Biosplice Therapeutics Announces First Patient Dosed In NCI-Sponsored Clinical Trial Of Cirtuvivint In Acute Myeloid Leukemia And Myelodysplastic Syndromes

(MENAFN- GlobeNewsWire - Nasdaq) 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.

In addition to the NCI-sponsored AML/MDS trial, cirtuvivint is being evaluated in several other clinical programs:

• An ongoing Phase 2 trial in advanced soft tissue sarcomas (NCT05633307)
• An upcoming combination trial with olaparib in BRCA-mutated or homologous recombination-deficient platinum-resistant ovarian cancer (NCT06856499)
• An upcoming combination trial with irinotecan in relapsed small-cell lung cancer (NCT07155200)
  

About Cirtuvivint
Cirtuvivint is a small molecule inhibitor of CLK and DYRK kinases, which are increasingly recognized as key drivers of various cancers. By targeting these kinases, cirtuvivint modulates alternative pre-mRNA splicing, which reduces the expression of genes vital for tumor growth, survival, and drug resistance. The compound has shown broad anti-tumor activity across a range of solid and liquid tumors in extensive preclinical studies.

Cirtuvivint has also been investigated for the treatment of advanced solid tumors in two clinical trials. The first, SM08502-ONC-01 (NCT03355066), was a first-in-human dose escalation study designed to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with advanced solid tumors. The second, SM08502-ONC-03 (NCT05084859), was a Phase 1b trial assessing the efficacy of cirtuvivint in combination with standard treatments for patients with advanced castration-resistant prostate cancer, advanced non-small cell lung cancer, and advanced colorectal cancer.

About Biosplice
Biosplice stands at the forefront of research concentrating on the study and regulation of Cdc2-like kinases (CLKs) and dual-specificity tyrosine-regulated kinases (DYRKs). These kinases play pivotal roles in cell cycle regulation, splicing, and neurodevelopment, marking them as critical targets for therapeutic intervention in a range of diseases, including osteoarthritis, cancer, neurological disorders, and diabetes. With a robust chemical platform for kinase inhibition and a deep understanding of kinase signaling pathways, Biosplice leverages cutting-edge technologies to discover and develop highly selective kinase inhibitors. Biosplice's drugs in clinical development include lorecivivint for knee osteoarthritis (completed Phase 3) and cirtuvivint for numerous cancers, with a broad preclinical pipeline that encompasses Alzheimer's disease, diabetes, and other degenerative conditions.

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Corporate Contact:

Phil Wilson

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