Lantern Pharma Receives FDA Guidance for Pediatric CNS Cancer Trial

Lantern Pharma has successfully completed a Type C meeting with the U.S. Food and Drug Administration, receiving crucial guidance on the regulatory pathway and design for its planned pediatric trial focused on central nervous system cancers. The FDA supported the company's proposal for a parallel cohort targeting Atypical Teratoid Rhabdoid Tumor and the potential inclusion of spironolactone as a combination agent with LP-184/STAR-001, Lantern's lead investigational therapy. This regulatory milestone enables the company to proceed with an Investigational New Drug amendment reflecting the FDA's feedback, with trial initiation targeted for the first quarter of 2026.

The significance of this development lies in addressing the critical unmet medical needs in pediatric oncology, particularly for rare and aggressive CNS cancers like ATRT that have limited treatment options. The program holds both Rare Pediatric Disease and Orphan Drug designations, highlighting the FDA's recognition of the importance of developing therapies for these underserved patient populations. The planned multi-site study will focus on clinically meaningful endpoints including progression-free survival, overall response rate, and quality-of-life outcomes, providing comprehensive data on the therapy's potential benefits.

Through its subsidiary Starlight Therapeutics, Lantern Pharma leverages its proprietary AI and machine learning platform, RADR®, which utilizes over 200 billion oncology-focused data points to accelerate drug development. This approach represents a transformative shift in oncology research, potentially reducing the cost and timeline of bringing new therapies to market. The company's growing pipeline of therapies spans multiple cancer indications, including both solid tumors and blood cancers, with an estimated combined annual market potential exceeding $15 billion.

The FDA's guidance and support for this pediatric trial design underscore the regulatory agency's commitment to facilitating the development of innovative treatments for rare pediatric diseases. The inclusion of quality-of-life metrics alongside traditional efficacy endpoints demonstrates an evolving approach to cancer drug development that considers the holistic impact on young patients. This trial represents an important step forward in applying advanced AI-driven drug discovery approaches to some of the most challenging pediatric cancers, potentially offering new hope for patients and families affected by these devastating diseases.