Kura Oncology Inc. and Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor, ziftomenib, met the primary endpoints in the phase II registrational Komet-001 trial in patients with relapsed/refractory NPM1-mutant acute myeloid leukemia, and Kura expects to submit its NDA to the U.S. FDA in the second quarter of 2025. Although details were scant, the Komet-001 trial achieved its primary endpoint of complete remission (CR) plus CR with partial hematologic recovery, and the primary endpoint was statistically significant, Kura said, with safety and tolerability consistent with previous reports.

Accelerated approval, mRNA therapies in recent UK drug regulations

The U.K. is continuing to shape up regulation, adding reform of its accelerated drug approval process and its draft guidance on personalized mRNA cancer vaccines to new clinical trial regulations that will come into force early in 2026. The Innovative Licensing and Access Pathway has been relaunched following a review of the industry’s experience of the scheme since its introduction in January 2021, and it will be open for applications from next month. The MHRA also this week launched an eight-week consultation on how it intends to regulate personalized mRNA cancer therapies.

Low sales, safety concerns doom biopharma’s ROI for mega-M&As

BioWorld’s three-part analysis of M&As sought to discover successful transactions and to understand the trend of multibillion-dollar deals that have become commonplace in the last decade. Instead, more than 80% of the acquisitions explored simply indicate that buyers are paying too much, suggesting that transactions meant to restore pipelines and revenues with innovative and marketed products are sometimes akin to high-stakes gambling. In part three, the final part of this series, we examine four more disappointments in which the return on investment (ROI) remains well behind the price paid in acquiring the company.

Henlius lands European approval of PD-1 lung cancer drug

The European Commission (EC) on Feb. 5 cleared Shanghai Henlius Biotech Inc.’s serplulimab (HLX-02) under the brand name of Hetronifly as a first-line combination therapy with carboplatin and etoposide to treat extensive-stage small-cell lung cancer (ES-SCLC). The EC’s go-ahead, issued to Henlius Europe GmbH, makes Hetronifly the first and only anti-PD-1 monoclonal antibody approved for use in all 27 EU member states and those in the European Economic Area, including Norway, Iceland and Liechtenstein, to treat patients with ES-SCLC.

PI3Kα target ever more alluring post-Lilly’s Scorpion takeover

Eli Lilly and Co.’s potential $2.5 billion deal signed recently to take over Scorpion Therapeutics Inc. and gain rights to phase I/II-stage STX-478 for breast cancer and other solid tumors piqued an already lively interest in the PI3Kα space. Other developers at work on the target include Roche AG, plus smaller entities such as Celcuity Inc., Menarini Group, Onkure Therapeutics Inc., Relay Therapeutics Inc. and Totus Medicines Inc.

Also in the news

60 Degrees, Actinogen, Adicet, Algernon, Amgen, Aqilion, Arbor, Astellas, Azitra, Cantargia, Creative Medical Technology, Cytoimmune, Ensysce, Eyepoint, Formycon, GH Research, Gradalis, Greenstone, Henlius, Iveena, Lotus, Nuvectis, Pasithea, Qlaris, Roche, Serac, Tubulis, Ultragenyx, Valneva