Caspian Therapeutics Unveils KO-7246 as Diabetes Treatment Candidate

Caspian Therapeutics, Inc., a company pioneering menin-directed therapies in diabetes and other cardiometabolic diseases, today announced comprehensive preclinical data supporting KO-7246 as a potential best-in-class menin inhibitor for diabetes. The data, generated by Kura Oncology prior to Caspian’s launch in September 2026, were presented by Francis Burrows, Ph.D., Chief Scientific Officer of Kura Oncology, at the 62nd European Association for the Study of Diabetes Annual Meeting (EASD) in Milan, Italy.

KO-7246 has demonstrated the ability to regenerate functional β-cell mass and produce durable glycemic control in both Type 1 and Type 2 diabetes models. The findings highlight a potentially disease-modifying approach designed to restore the body’s own endogenous insulin-producing capacity. Caspian Therapeutics is building a leadership position in menin-directed therapies for diabetes and other cardiometabolic diseases.

The preclinical data indicate that KO-7246 can normalize fasting blood glucose in a majority of animals, increase stimulated C-peptide levels, and regenerate pancreatic islets to 40-90% of levels seen in healthy controls by Day 56. Importantly, the normalized glucose levels and increased C-peptide were maintained for at least one month after treatment was stopped, while residual cell proliferation was negligible. These findings suggest that KO-7246 can rebuild functional β-cell capacity and that the benefits persist after dosing ends.

In a mouse model of Type 2 diabetes, KO-7246 significantly reduced fasting blood glucose, increased insulin and C-peptide levels, and produced a 3.4-fold increase in β-cell mass. The compound also demonstrated enhanced activity in combination with semaglutide, highlighting its potential to complement existing GLP-1 therapies. The increase in insulin was not associated with hypoglycemia, suggesting that KO-7246 could provide a differentiated therapeutic benefit as either a monotherapy or a complement to widely used glucose-lowering treatments.

In human pancreatic islet models, KO-7246 increased β-cell proliferation and the proportion of β-cells without stimulating growth of other islet cells, and improved insulin release in response to glucose. These findings provide additional evidence that the regenerative effects observed in animal models extend to human β-cell systems. The selective effect on β-cells further differentiates KO-7246 from broader proliferative approaches that could stimulate non-β-cell growth.

The preclinical findings also underscore KO-7246’s differentiated pharmacology compared to other targeted therapies in clinical development for diabetes. KO-7246 demonstrated potent menin inhibition in biochemical and cellular assays, while BMF-219 (icovamenib) showed no discernable activity against menin in the biochemical assay but instead inhibited several kinases, including CDK9. In vivo evidence of bona fide menin pharmacology was demonstrated with KO-7246 in a menin-dependent leukemia model, while BMF-219 showed no efficacy even at an 8-fold higher daily dose. In the Type 1 diabetes model, where KO-7246 restored glycemic control and increased β-cell mass, BMF-219 did not produce either of these effects. Taken together, these comparative results support KO-7246’s profile as a bona fide and highly selective menin inhibitor with potential as a differentiated therapy for diabetes.

Caspian Therapeutics will host a webcast and conference call on October 13, 2026, at 4:30 p.m. ET / 1:30 p.m. PT, to discuss the findings and provide an update on the development of KO-7246.

Caspian Therapeutics is a company pioneering menin-directed therapies in diabetes and other cardiometabolic diseases. Built on more than a decade of menin-inhibitor research at Kura Oncology, Caspian combines deep expertise in menin biology and medicinal chemistry with a development strategy focused on the requirements of diabetes and other cardiometabolic diseases. Caspian’s lead compound, KO-7246, is a next-generation, highly selective and orally bioavailable menin inhibitor intended for metabolic applications and is currently in IND-enabling development. Caspian plans to evaluate KO-7246 in a Phase 1 program designed to assess its potential to restore functional β-cell capacity and endogenous insulin production in patients with diabetes. Caspian also plans to advance a second development candidate for additional cardiometabolic indications.

The comprehensive preclinical data supporting KO-7246 as a potential best-in-class menin inhibitor for diabetes mark a significant milestone in Caspian’s development strategy. These findings provide a strong foundation for KO-7246 to become a best-in-class menin inhibitor and for Caspian to lead the development of menin-directed therapies for diabetes and other cardiometabolic diseases.