Caspian Therapeutics Unveils KO-7246 at EASD as a Potential Best-In-Class Menin Inhibitor for Diabetes

The findings come from animal and human-islet models; Caspian still plans clinical evaluation of KO-7246.

Rhea-AI Summary

Kura Oncology (KURA) and Caspian Therapeutics presented preclinical KO-7246 diabetes findings at the EASD meeting in Milan.

In a Type 1 diabetes rat model, KO-7246 normalized fasting glucose in a majority of animals. Among responders, pancreatic islets regenerated to 40-90% of healthy-control levels by Day 56. Normal glucose levels and increased C-peptide persisted for at least one month after treatment stopped. In a Type 2 diabetes mouse model, β-cell mass rose 3.4-fold, and combining KO-7246 with semaglutide enhanced activity. Human-islet experiments showed increased β-cell proliferation and improved insulin release in response to glucose. KO-7246 is in IND-enabling development; Caspian plans a Phase 1 evaluation.

Positive

• Minor pointType 1 diabetes rats reached normal fasting glucose in a majority of treated animals.
• Minor pointPancreatic islets regenerated to 40-90% of healthy-control levels among responders by Day 56.
• Minor pointGlucose control and increased C-peptide persisted for at least one month after treatment stopped.
• Minor pointType 2 diabetes mice showed a 3.4-fold increase in β-cell mass.
• Minor pointSemaglutide combination enhanced KO-7246 activity in the Type 2 diabetes mouse model.
• Minor pointHuman islets showed increased β-cell proliferation and improved glucose-responsive insulin release.
• Minor point. Forward-looking: it has not happened yet and may not happen.Phase 1 evaluation is planned by Caspian for KO-7246.

Negative

• Minor point. Forward-looking: it has not happened yet and may not happen.Phase 1 evaluation remains planned; KO-7246 is currently in IND-enabling development.

Market Context

The $50 million financing announced on Sep 09 was expected to support KO-7246 through IND-enabling d...

Analysis

The $50 million financing announced on Sep 09 was expected to support KO-7246 through IND-enabling development and initial clinical proof of concept, linking earlier program funding to the preclinical findings reported here.

Key Figures

Pancreatic islet regeneration: 40–90% of healthy-control levels Post-treatment durability: At least one month β-cell mass increase: 3.4-fold +1 more

Pancreatic islet regeneration

40–90% of healthy-control levels

Among responder animals in a rat Type 1 diabetes model by Day 56

Post-treatment durability

At least one month

Normalized glucose levels and increased C-peptide persisted after treatment stopped

β-cell mass increase

3.4-fold

Mouse model of Type 2 diabetes

Comparative daily dose

8-fold higher

BMF-219 showed no efficacy at this higher daily dose in a menin-dependent leukemia model

Historical Context

1 past event · Latest: Sep 09

1 event

1. Sep 09

   Program financing

   24h Move

   -3.4%

   Caspian's financing was expected to support KO-7246 through IND-enabling work and initial clinical proof of concept.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

c-peptide, hypoglycemia, glp-1, ind-enabling

4 terms

c-peptide medical

"increased stimulated C-peptide, a marker of endogenous insulin production."

C‑peptide is a short protein fragment released at the same time the pancreas produces insulin; because it lingers in the blood longer than insulin itself, clinicians measure C‑peptide levels as a clear sign of how much natural insulin a person still makes. For investors, C‑peptide matters because it’s used as a measurable outcome in diabetes drug and device trials, in diagnostic tests, and by regulators to judge treatment benefit — results that can affect clinical success, approvals, and market value.

hypoglycemia medical

"The increase in insulin was not associated with hypoglycemia."

Hypoglycemia is a condition where blood sugar falls too low to fuel the body and brain, causing symptoms like shakiness, confusion, sweating or fainting. Think of it as a car running low on gas: systems slow or fail until fuel is restored. Investors care because treatments, devices or drugs that cause or prevent hypoglycemia affect patient safety, regulatory approval, product labeling and market acceptance, which influence a company’s commercial prospects.

glp-1 medical

"highlighting its potential to complement existing GLP-1 therapies."

GLP-1 (glucagon-like peptide-1) is a natural hormone in the body that helps regulate blood sugar levels and appetite. Its significance to investors lies in its role as the basis for a class of medications that address conditions like type 2 diabetes and obesity, which are large and growing markets. Advances or investments in GLP-1-based treatments can signal opportunities in healthcare innovation and potentially impact pharmaceutical companies’ growth.

ind-enabling regulatory

"currently in IND-enabling development."

Ind-enabling describes the preclinical tests and safety work a drug candidate must pass before a company can ask regulators for permission to start human trials (an Investigational New Drug or IND filing). Think of it as the mechanical inspection and crash-testing a prototype car needs before it can legally be driven on public roads; for investors, successful ind-enabling work reduces technical and regulatory risk and makes clinical progress and potential value creation more likely.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Add on Google Not now

– KO-7246 regenerated functional β-cell mass and produced durable glycemic control in Type 1 and Type 2 diabetes models –

– KO-7246 has potential to complement existing diabetes therapies with a novel mechanism of action, evidenced by post-treatment durability and combination benefit with semaglutide –

– Findings highlight a potentially disease-modifying approach designed to restore the body’s own endogenous insulin-producing capacity –

– Caspian is building a leadership position in menin-directed therapies for diabetes and other cardiometabolic diseases –

– Management to host webcast and conference call on October 13, 2026, at 4:30 p.m. ET / 1:30 p.m. PT –

SAN DIEGO, Sept. 29, 2026 (GLOBE NEWSWIRE) -- Caspian Therapeutics, Inc. (“Caspian”) and Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) today announced comprehensive preclinical data supporting KO-7246 as a potential best-in-class menin inhibitor for diabetes. KO-7246 regenerated functional β-cell mass, demonstrated durable glycemic control, and increased endogenous insulin production in preclinical models of Type 1 and Type 2 diabetes. In human islets, KO-7246 selectively increased β-cell proliferation and improved glucose-responsive insulin secretion. Together, the findings establish a differentiated preclinical profile including validated activity as a menin inhibitor, durable disease-relevant activity, and confirmed translation to a human-islet setting.

The data were generated by Kura Oncology prior to the launch of Caspian in September 2026. Francis Burrows, Ph.D., Chief Scientific Officer of Kura Oncology, presented the findings today at the 62^nd European Association for the Study of Diabetes Annual Meeting (EASD) in Milan, Italy.

Menin acts as a molecular brake on pancreatic β-cell proliferation. With its menin inhibitor platform, Caspian is pursuing a potentially disease-modifying approach designed to increase functional β-cell mass and restore the body’s insulin-producing capacity.

“Menin is a compelling target in diabetes because multiple lines of evidence, from human physiology and genetics to pharmacologic studies, point to its role in regulating β-cell mass,” said Dr. Burrows. “KO-7246 gives us a potent, selective way to test whether menin inhibition can harness this biology to restore endogenous insulin-producing capacity in both Type 1 and Type 2 diabetes. Its potency and selectivity as a menin inhibitor, as well as its activity in a range of diabetes preclinical models, distinguish KO-7246 from earlier approaches and support its potential to become a best-in-class menin inhibitor for diabetes.”

In a rat model of Type 1 diabetes, KO-7246 normalized fasting blood glucose in a majority of animals and increased stimulated C-peptide, a marker of endogenous insulin production. Among responder animals, pancreatic islets regenerated to 40-90% of levels seen in healthy controls by Day 56. Importantly, normalized glucose levels and increased C-peptide were maintained for at least one month after treatment was stopped, while residual cell proliferation was negligible. Together, these findings suggest that KO-7246 rebuilt functional β-cell capacity and that the benefit persisted after dosing ended. The dependence of response on residual β-cell capacity at the baseline was also consistent with the proposed regenerative mechanism.

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. KO-7246 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. These findings suggest 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 presentation included data highlighting 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.

“We believe KO-7246 has one of the most comprehensive preclinical profiles reported for a menin inhibitor being developed for diabetes. It combines bona fide menin pharmacology with durable β-cell regeneration, activity in both Type 1 and Type 2 diabetes models, and potent and selective effects on β cells in human islets,” said Robert Spencer, Ph.D., President and Chief Operating Officer of Caspian Therapeutics. “Caspian was formed to translate this biology into potentially disease-modifying medicines. We believe these data 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. We are advancing KO-7246 through IND-enabling development toward initial clinical evaluation.”

Virtual Investor Event

Caspian and Kura management will host a webcast and conference call on October 13, 2026 at 4:30 p.m. ET / 1:30 p.m. ET. The live webcast and replay will be available on www.kuraoncology.com under the Investors tab in the Events and Presentations section.

About Caspian Therapeutics

Caspian Therapeutics is 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. To learn more, visit www.caspiantherapeutics.com.

Kura Oncology Forward-Looking Statements

This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the potential for KO-7246 to be a best-in-class menin inhibitor for diabetes, represent a disease-modifying approach to the disease, and restore the body’s endogenous insulin-producing capacity; the strength of the preclinical findings related to KO-7246; the therapeutic potential of, opportunity for and differentiated approach for KO-7246; the potential for KO-7246 to provide a differentiated therapeutic benefit as either a monotherapy or a complement to widely used glucose-lowering treatments such as GLP-1 therapies; the potential for the regenerative effects of KO-7246 observed in animal models to extend to human β-cell systems; the potential for Caspian to lead the development of menin-directed therapies for diabetes and other cardiometabolic diseases; and Caspian’s 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 and to advance a second development candidate for additional cardiometabolic indications. Factors that may cause actual results to differ materially include risks associated with the conduct of preclinical studies and clinical trials; the risk of the FDA not permitting Kura’s or Caspian’s planned trials to proceed; the risk that Kura’s or Caspian’s product candidates may not receive regulatory approval; the potential for Kura’s or Caspian’s product candidates to have unexpected adverse side effects; the risk that Kura or Caspian may not be able to obtain additional financing; the risks associated with reliance on outside financing to meet capital requirements; the risk that compounds that appeared promising in early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials; risks associated with reliance on third parties to successfully conduct clinical trials; and other risks associated with the process of discovering, developing and commercializing drugs. You are urged to consider statements that include the words “may,” “will,” “would,” “could,” “should,” “believes,” “estimates,” “projects,” “potential,” “expects,” “plans,” “anticipates,” “intends,” “continues,” “designed,” “goal,” or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties Kura faces, please refer to Kura’s periodic and other filings with the Securities and Exchange Commission, which are available at www.sec.gov. Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts
Caspian Therapeutics
Robert Spencer, Ph.D.
rob@caspiantx.com

Kura Oncology
Greg Mann
858-987-4046
gmann@kuraoncology.com

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did KO-7246 show in Kura Oncology’s Type 1 diabetes model?

KO-7246 normalized fasting blood glucose in a majority of rats in the Type 1 diabetes model. Among responders, pancreatic islets regenerated to 40-90% of healthy-control levels by Day 56; normal glucose levels and increased C-peptide persisted for at least one month after treatment stopped.

What affected response to KO-7246 in the Type 1 diabetes rat model?

Response depended on how much β-cell capacity remained before treatment. The findings do not establish how patients with different levels of remaining β-cell capacity would respond.