Kura Oncology Gains on $30M Milestone for Leukemia Drug Trial
Payment from partner Kyowa Kirin follows the start of a pivotal Phase 3 study for ziftomenib, with a key FDA decision for the drug expected next month.
Kura Oncology Inc. (NASDAQ: KURA) shares edged higher Friday after the company announced it received a $30 million milestone payment from its partner, Kyowa Kirin, bolstering its finances as it advances its lead leukemia drug candidate, ziftomenib, into a crucial late-stage trial.
The payment was triggered by the dosing of the first patient in a Phase 3 clinical trial, known as KOMET-017, evaluating ziftomenib in patients with newly diagnosed acute myeloid leukemia (AML) who have specific genetic mutations. Shares of the San Diego-based biopharmaceutical company climbed approximately 2.9% to $9.36 in morning trading on the news, reflecting investor confidence in the drug's expanding clinical program.
For a clinical-stage company with a market capitalization of roughly $790 million, the infusion of non-dilutive capital is significant. It strengthens Kura’s balance sheet and extends its operational runway as it navigates the costly late stages of drug development.
The new Phase 3 study is designed to test ziftomenib in combination with standard chemotherapy regimens for frontline treatment of AML patients with NPM1 mutations or KMT2A rearrangements. The company confirmed the milestone in a press release Friday morning, marking a critical step in potentially expanding the drug's application to a larger patient population.
This latest development adds to a period of significant clinical momentum for Kura. The advancement into a frontline AML trial comes as the company awaits a major regulatory decision from the U.S. Food and Drug Administration (FDA). Kura previously submitted a New Drug Application (NDA) for ziftomenib as a monotherapy for patients with relapsed or refractory NPM1-mutant AML, based on positive results from its pivotal Phase 2 KOMET-001 trial.
The FDA has granted that application Breakthrough Therapy Designation and set a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025. A potential approval next month would validate ziftomenib's novel mechanism as a menin inhibitor and provide Kura with its first commercial product.
Ziftomenib works by blocking the interaction of two proteins, menin and KMT2A, which is a key driver for certain forms of AML. The positive pivotal data from the KOMET-001 study, which was recently published in the Journal of Clinical Oncology, showed the drug could induce durable remissions in a heavily pre-treated patient population that has limited therapeutic options.
Wall Street remains broadly optimistic about Kura's prospects. The consensus analyst price target for the stock sits at $27.00, suggesting significant potential upside from its current trading level. Of the 15 analysts covering the company, 13 currently rate the stock as a "Buy" or "Strong Buy," with only two maintaining a "Hold" rating.
The partnership with Kyowa Kirin, a global specialty pharmaceutical company based in Japan, is central to Kura's strategy. The collaboration is focused on the development and commercialization of ziftomenib in Japan and other Asian markets, giving Kura access to a major pharmaceutical market without bearing the full cost of expansion.
Kura is a leader in a new class of targeted cancer drugs called menin inhibitors, but it faces competition. Syndax Pharmaceuticals is developing a rival menin inhibitor, revumenib, creating a competitive race to market for treating these genetically defined leukemias. However, analysts have noted that ziftomenib's safety profile, particularly its low incidence of a side effect known as differentiation syndrome, could be a key advantage.
As Kura pushes ziftomenib forward on two fronts—a potential approval for relapsed patients and a new Phase 3 trial for frontline therapy—investors are closely watching the upcoming PDUFA date as the next major inflection point for the company and its novel approach to treating acute myeloid leukemia.