BioPharma, Pharma

Kura Oncology Gets $330M to Kick Off Global Leukemia Drug Pact With Kyowa Kirin

Kura Oncology already had a broad development plan for ziftomenib in leukemias, but the agreement with Kyowa Kirin provides the cash to execute it and a partner to commercialize the drug globally. Ziftomenib is in the same class of medicines as Syndax Pharmaceuticals’ Revuforj, which won FDA approval last week.

By Frank Vinluan on November 21, 2024

The FDA recently approved the first drug in a new class of leukemia medicines. Kura Oncology’s endeavor to follow that newly blazed regulatory trail is now infused with $330 million from a collaboration agreement with Kyowa Kirin.

The Kura drug, ziftomenib, has completed a Phase 2 test designed to support a new drug application that the companies expect will be submitted to the FDA in 2025 seeking approval as a treatment for advanced cases of acute myeloid leukemia (AML). The two companies also plan to develop the drug broadly across leukemias as an earlier line of therapy, in combination with other drugs, and as a maintenance treatment following the stem cell transplant procedure that is currently one leukemia treatment option.

The $330 million sum is an upfront payment. According to deal terms announced after Wednesday’s market close, Kura and Kyowa Kirin will jointly develop and commercialize ziftomenib; San Diego-based Kura will lead those efforts in the U.S. and the two companies will share equally in the profits. Tokyo-based Kyowa Kirin has exclusive commercialization rights in the rest of the world and will pay Kura royalties from product sales. Milestone payments to Kura could reach up to $1.2 billion.

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Ziftomenib is a potential treatment for patients whose leukemia is driven by mutations to the KMT2A gene. Genetic rearrangement of this gene can lead to aggressive leukemias that are prone to relapse and drug resistance. In patients whose cancer is characterized by this genetic signature, the interaction of KMT2A fusion proteins with a protein called menin drives disease progression. Kura’s drug is a small molecule designed to block menin.

Last week’s FDA approval of Revuforj made the Syndax Pharmaceuticals drug the first menin inhibitor to pass the agency’s regulatory bar. The approval covers the treatment of advanced cases of acute leukemia. Syndax has also tested the drug in AML driven by mutations to the NPM1 gene. The company is planning a first half 2025 regulatory submission seeking to expand the drug’s approval to AML driven by that mutation.

Kura had a broad plan for the development of ziftomenib prior to striking the Kyowa Kirin alliance. The company has been evaluating the drug as an earlier line of leukemia treatment and for leukemia driven by NPM1 mutations. A Phase 1 test is evaluating ziftomenib in combination with standard AML treatments. Kura plans to present updated data next month during the annual meeting of the American Society of Hematology, the company said in an investor presentation.

Under the terms of its deal with Kyowa Kirin, Kura is eligible to earn $420 million in near-term milestone payments tied to the launch of ziftomenib in advanced AML. The agreement gives Kyowa Kirin opt-in rights to develop and commercialize ziftomenib in gastrointestinal stromal tumors and other solid tumors. Exercising that right could trigger up to $228 million in additional milestone payments. Kura also revealed plans for two placebo-controlled Phase 3 studies evaluating the drug as a first-line treatment of AML driven by either the KMT2A or NPM1 mutations. In the investor presentation, Kura said this fully funded study is expected to start in mid-2025.

Leerink Partners analyst Jonathan Chang said in a Thursday research note that Kura’s deal with Kyowa Kirin is positive, as it provides the biotech with the cash and capabilities to aggressively pursue broad development and commercialization opportunities for ziftomenib.

“Overall, we continue to view ziftomenib as a highly promising agent in the emerging class of menin-MLL inhibitors and view KURA as well-positioned long term to execute on their development strategy for ziftomenib and the farnesyl transferase inhibitor programs,” Chang said in the note.

The farnesyl transferase inhibitor is tipifarnib, a drug that Kura is developing for certain head and neck squamous cell carcinomas. Kura is conducting a Phase 1/2 study evaluating tipifarnib in combination with Novartis’s Piqray, a drug that blocks the PI3K pathway that, when mutated, drives cancer growth. Piqray is approved as a treatment for breast cancer driven by PI3K mutations. Kura said in its third quarter 2024 financial report that it expects data from its study will be presented at a medical meeting in the first half of next year.

Public domain image of acute myelocytic leukemia from the National Cancer Institute