Kuro Oncology and partner Kyowa Kirin are on track for an NDA submission for ziftomenib in the second quarter of this year.
Kura Oncology and development partner Kyowa Kirin on Wednesday reported “positive” topline findings for the registrational KOMET-001 study of their oral menin blocker ziftomenib for acute myeloid leukemia.
The companies did not provide specific data, revealing only that ziftomenib hit its primary endpoint of complete response (CR) plus CR with partial hematological recovery (CRh) in relapsed or refractory acute myeloid leukemia (AML) patients harboring a certain mutation in the NPM1 gene.
The ziftomenib benefit-risk profile in KOMET-001 was “highly encouraging,” according to Kuro and Kyowa, with safety and tolerability findings being consistent with what had been previously reported.
Full data from and analyses of KOMET-001 will be presented at a medical congress scheduled for the second quarter of the year, according to the partners. The companies also announced on Wednesday that they had wrapped up “successful” talks with the FDA, and that a New Drug Application (NDA) for ziftomenib is on-track for submission in the second quarter.
Investors appear underwhelmed by the news, however, sending Kura’s stocks dipping around 6% to $8.50 in after-hours trading, from $9.05 at market close.
In a note on Wednesday evening, analysts at Mizuho Securities called the selloff an “over-reaction,” noting that while there were no data revealed, the partners’ readout “sounds fine to us.” The companies also explained to Mizuho that the decision to withhold specific data in their press announcement was “largely due to conference rules.”
On ziftomenib’s prospects, the Mizuho note pointed out that KOMET-001 “was powered to detect a CR/CRh rate of 20-30%, so we believe it’s in that range,” and noted that the filing of the NDA further boosts the firm’s confidence that “the CR/CRh was in the 20-30% range.”
Wednesday’s readout comes after Kuro and Kyowa inked their AML partnership in November 2024. For $330 million upfront and up to $420 million in near-term milestones—as well as a maximum of $741 million in developmental, regulatory and commercial milestones—Kuro gave the Japanese pharma developmental and joint commercial rights over ziftomenib outside the U.S.
Kuro will remain responsible for the asset in the U.S., taking charge of its R&D, regulatory and commercialization activities.
The partners, however, are playing catch-up to Syndax, which in November 2024 won the FDA’s first approval for a menin inhibitor with its Revuforj, indicated for relapsed or refractory acute leukemia with KMT2A mutations. Pivotal data for Revuforj show a CR/CRh rate of 21%.
