Kura Oncology and Kyowa Kirin Report Positive Results in AML Trial, Prepare for FDA Filing

Kura Oncology and its development partner Kyowa Kirin have announced positive topline results from their registrational KOMET-001 study, evaluating the oral menin blocker ziftomenib in acute myeloid leukemia (AML) patients. The companies are now gearing up for a New Drug Application (NDA) submission to the FDA, expected in the second quarter of this year.

Pivotal Trial Success

The KOMET-001 study met its primary endpoint, demonstrating efficacy in relapsed or refractory AML patients with NPM1 gene mutations. While specific data were not disclosed, the companies reported that ziftomenib achieved the desired complete response (CR) plus CR with partial hematological recovery (CRh) in the target patient population.

The benefit-risk profile of ziftomenib was described as "highly encouraging," with safety and tolerability findings consistent with previous reports. Full data and analyses from the KOMET-001 trial are slated for presentation at a medical conference in the second quarter.

Regulatory Progress and Market Reaction

Following successful discussions with the FDA, Kura Oncology and Kyowa Kirin have confirmed their plans to submit an NDA for ziftomenib in the second quarter of 2025. This regulatory milestone comes on the heels of their partnership agreement, inked in November 2024, which saw Kyowa Kirin secure development and joint commercial rights for ziftomenib outside the United States.

Despite the positive announcement, investor reaction has been lukewarm. Kura's stock experienced a 6% dip in after-hours trading, closing at $8.50 from $9.05 at market close. Analysts at Mizuho Securities, however, view this selloff as an "over-reaction," noting that the lack of specific data in the press release was likely due to conference presentation rules rather than any concerns about the results.

Competitive Landscape

As Kura Oncology and Kyowa Kirin prepare for their regulatory submission, they face competition in the menin inhibitor space. Syndax Pharmaceuticals gained a head start in November 2024 with the FDA's first approval of a menin inhibitor, Revuforj, for relapsed or refractory acute leukemia with KMT2A mutations. Pivotal data for Revuforj showed a CR/CRh rate of 21%, setting a benchmark for the field.

While specific efficacy data for ziftomenib remain undisclosed, industry watchers speculate that the CR/CRh rate likely falls within the 20-30% range, based on the study's design and the companies' confidence in moving forward with the NDA submission.