ACT Brief: Smarter Study Planning, New AML Approval, and Biomarker Gaps in Cancer Care

This is the Applied Clinical Trials Brief—your fast track to the latest insights in clinical research operations. In under three minutes, we’ll recap top stories, highlight expert perspectives, and keep you current on what’s moving the industry.

• A new feature article examines why enrollment failures continue to drive demand for clinical trial rescue services—and how proactive planning may help avoid them. Sponsors and CROs are increasingly leveraging real-world data and AI tools to better assess patient availability, evaluate site feasibility, and simulate enrollment before a study begins. The goal: build more realistic protocols upfront and reduce the delays, dropouts, and costly course-corrections that too often derail modern trials.
• In regulatory news, the FDA has approved Komzifti, the first once-daily oral menin inhibitor for adults with relapsed or refractory NPM1-mutated AML. Backed by Phase I/II KOMET-001 results, the therapy offers a new option for a difficult-to-treat population with limited alternatives. The approval reflects both meaningful remission activity and a manageable safety profile, and is expected to support broader development of the drug across additional AML settings.
• And in a new Pharm Exec podcast, Jaspal Singh, MD, discusses persistent barriers preventing many cancer patients from receiving timely biomarker testing. He highlights gaps in specimen management, education, and clinical workflows, noting that delayed or incomplete testing can limit access to precision therapies. Proposed solutions include reflex testing and stronger clinician-patient education to ensure molecular insights are available early enough to guide treatment decisions.

That’s all for today’s ACT Brief. Join us tomorrow for more updates shaping clinical operations and drug development. Thanks for listening.