Lantern Pharma (LTRN) announced preclinical data for LP-184 in atypical teratoid rhabdoid tumors, a rare and aggressive pediatric brain cancer. The results were presented by Eric Raabe of Johns Hopkins University School of Medicine at the Society for Neuro-Oncology’s 8th Biennial Pediatric Neuro-Oncology Conference held May 15-17 in San Diego, California. The data further validates the research that supported Lantern Pharma’s Rare Pediatric Disease Designation from the FDA for LP-184 in ATRT and strengthens the scientific foundation for the company’s planned pediatric clinical trial expected to begin in late 2025 or early 2026. The data demonstrated that LP-184, a next-generation acylfulvene clinical-stage drug candidate, significantly extended survival in mouse models of ATRT. In the CHLA06 model, median survival increased from 20 days in the control group to 89 days in the LP-184 treatment group, representing a 345% improvement. In the BT37 model, median survival increased from 68 days to 98 days. Key highlights from the presentation include: LP-184 demonstrated potent anti-tumor activity across multiple ATRT cell lines representing different molecular subtypes, with IC50 values ranging from 17.5 nM to 161 nM; treatment with LP-184 significantly decreased cancer cell proliferation and increased apoptosis in ATRT cells; LP-184 showed strong blood-brain barrier penetrance, with reported Cmax of 730 nM in brain tissue; no apparent toxicity was observed in the mouse models, with stable weight maintained throughout the treatment period; treatment with LP-184 resulted in statistically significant survival benefits in two different orthotopic xenograft ATRT models. ATRT is characterized by the deletion or inactivation of the SMARCB1 gene, an epigenetic regulator. LP-184’s mechanism of action may be particularly effective against tumors with epigenetic dysregulation, potentially explaining the strong preclinical anti-tumor activity observed in this tumor type. The company highlighted that the pediatric Phase I trial for LP-184 in brain tumors is targeted to open in winter 2025 or early 2026, following completion of the ongoing Phase I trial in adult solid tumors and obtaining future funding and approvals from the pediatric consortium.
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