10-K: Kura Oncology Announces Global Collaboration with Kyowa Kirin; Reports Positive Phase 2 Results for Ziftomenib

Kura Oncology partners with Kyowa Kirin in a global collaboration for ziftomenib, while also reporting positive topline results from its Phase 2 KOMET-001 trial.

Summary

• Kura Oncology has entered into a global strategic collaboration with Kyowa Kirin to develop and commercialize ziftomenib.
• The collaboration includes an upfront payment of $330 million to Kura and potential milestone payments totaling up to $1.161 billion.
• Kura will lead development, regulatory, and commercial strategy in the U.S., with a 50/50 profit share.
• Kyowa Kirin will lead development, regulatory, and commercial strategy outside the U.S., and Kura will receive tiered double-digit royalties on net product sales.
• Kura also announced positive topline results from the Phase 2 registration-directed portion of the KOMET-001 trial for ziftomenib in relapsed or refractory NPM1-mutant AML.
• The KOMET-001 trial achieved its primary endpoint of CR plus CR with partial hematological recovery, or CRh.
• Topline data from the Phase 2 portion of KOMET-001 is expected to be presented at an international medical meeting in the second quarter of 2025.
• Based on KOMET-001 trial results and pre-NDA meeting with the FDA, Kura expects to submit an NDA to the FDA for ziftomenib for the treatment of relapsed or refractory NPM1-mutant AML in the second quarter of 2025.
• The FDA is anticipated to assign a PDUFA date in the second half of 2025.
• Kura and Kyowa Kirin also announced plans for KOMET-017, a global protocol evaluating ziftomenib in combination with standards of care for adults with newly diagnosed NPM1-mutant or KMT2A-rearranged AML.
• The KOMET-017 trial will be comprised of two independent, global, randomized, double-blind, placebo-controlled Phase 3 trials to evaluate ziftomenib in combination with both intensive and non-intensive regimens in patients with newly diagnosed NPM1-mutant or KMT2A-rearranged AML.
• The End-of-Phase 1 meetings were intended to serve as the key regulatory interaction ahead of initiating the KOMET-017 protocol.
• The registrational KOMET-017-IC (Intensive Chemotherapy) trial will evaluate the combination of ziftomenib with induction chemotherapy (7+3) in patients with newly diagnosed NPM1-mutant or KMT2A-rearranged AML.
• The KOMET-017-IC trial will assess MRD-negative CR and event-free survival, or EFS, as dual-primary endpoints to support potential U.S. accelerated approval and full approval, respectively.
• We expect to initiate the KOMET-017-IC trial in the second half of 2025.
• Based on our current assumptions, we believe we may have topline results from the MRD-negative CR accelerated endpoint in the intensive chemotherapy setting in 2028.
• The registrational KOMET-017-NIC (Non-Intensive Chemotherapy) trial will evaluate the combination of ziftomenib with venetoclax plus azacitidine in patients with newly diagnosed NPM1-mutant AML who are unfit to receive intensive chemotherapy.
• The KOMET-017-NIC trial will assess CR and OS as dual-primary endpoints to support potential U.S. accelerated approval and full approval, respectively.
• We expect to initiate the KOMET-017-NIC trial in the second half of 2025.

Sentiment

Score: 8

Explanation: The document presents a positive outlook due to the strategic collaboration, significant upfront payment, and promising Phase 2 results. The company is well-positioned to advance its ziftomenib program.

Positives

• The collaboration with Kyowa Kirin provides significant financial resources and expertise to support the development and commercialization of ziftomenib.
• Positive Phase 2 results for ziftomenib in relapsed or refractory NPM1-mutant AML support its potential as a treatment option for this patient population.
• Planned Phase 3 trials in frontline AML could expand the market opportunity for ziftomenib.
• Breakthrough Therapy Designation from the FDA for ziftomenib may expedite its development and review.

Negatives

• The collaboration agreement gives Kyowa Kirin significant control over the development and commercialization of ziftomenib outside the U.S.
• The success of ziftomenib is not guaranteed, and it may not receive regulatory approval or achieve commercial success.
• The company is still in the clinical stage and has no approved products or historical product revenue.

Risks

• Clinical trials may not be successful, and regulatory approvals may not be obtained.
• The company may need to raise additional capital in the future, which could dilute existing stockholders.
• The company faces competition from other companies developing treatments for AML.
• The company is dependent on third parties for manufacturing and clinical trial services.
• The company's stock price may be volatile.

Future Outlook

Kura Oncology anticipates that the collaboration funding, along with its current cash balance, will support the ziftomenib AML program through commercialization in the frontline combination setting. The company plans to launch multiple Phase 2 and Phase 3 studies of ziftomenib in AML and other hematologic malignancies over the next several years.

Management Comments

• Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology, stated that the collaboration is an important step toward fulfilling Kura's commitment to precision medicines and advances its goal of building a sustainable, fully integrated biopharmaceutical company.
• Yasuo Fujii, MBA, Chief Strategy Officer, Managing Executive Officer of Kyowa Kirin, stated that ziftomenib is a very promising investigational treatment for genetically defined AML patients and will complement Kyowa Kirin's existing hemato-oncology portfolio.

Industry Context

This announcement reflects the ongoing trend in the pharmaceutical industry towards strategic collaborations to share the costs and risks of drug development and commercialization. The focus on precision medicines and targeted therapies is also a key trend in oncology.

Comparison to Industry Standards

• Syndax Pharmaceuticals recently received FDA approval for revumenib (Revuforj) in relapsed or refractory KMT2A-rearranged acute leukemia, indicating a competitive landscape in menin inhibitor development.
• AbbVie/Genentech's venetoclax (VENCLEXTA) and Astellas' gilteritinib (XOSPATA) are approved therapies for AML, setting a high bar for efficacy and safety that ziftomenib will need to meet.
• The collaboration structure, with Kura leading U.S. activities and Kyowa Kirin leading ex-U.S. activities, is a common model in the pharmaceutical industry for global drug development and commercialization.

Stakeholder Impact

• Shareholders: Potential for increased stock value due to the collaboration and positive clinical trial results.
• Patients: Potential for new treatment options for AML.
• Employees: Increased job security and opportunities for growth within the company.

Next Steps

• Present topline data from the Phase 2 portion of KOMET-001 at an international medical meeting in the second quarter of 2025.
• Submit an NDA to the FDA for ziftomenib for the treatment of relapsed or refractory NPM1-mutant AML in the second quarter of 2025.
• Initiate the KOMET-017-IC and KOMET-017-NIC trials in the second half of 2025.

Key Dates