DALLAS — Lantern Pharma Inc., a clinical-stage biopharmaceutical company using artificial intelligence to advance oncology drug discovery and development, said the U.S. Food and Drug Administration has granted Orphan Drug Designation to its investigational therapy LP-284 for the treatment of soft tissue sarcomas.
The designation marks the third FDA orphan designation for LP-284, following prior designations in mantle cell lymphoma in January 2023 and high-grade B-cell lymphoma with MYC and BCL2 rearrangements in November 2023. It is the sixth orphan drug designation overall awarded to Lantern Pharma’s A.I.-driven precision oncology pipeline.
“Receiving orphan drug designation for LP-284 in soft tissue sarcomas expands this molecule’s potential beyond hematologic malignancies into solid tumors,” said Panna Sharma, CEO of Lantern Pharma. “Adult soft tissue sarcomas are a compelling opportunity for LP-284. Unlike pediatric sarcomas driven by specific gene fusions, adult sarcomas commonly exhibit complex genomic alterations, chromosomal instability, and DNA damage response deficiencies – including BRCA-ness and homologous recombination repair defects – that align with LP-284’s synthetic lethal mechanism. With over 79% of cases occurring in adults, this designation addresses a distinct patient population with significant unmet need. Our RADR® AI platform identified these DNA repair vulnerabilities, demonstrating its ability to uncover biomarker-driven precision oncology opportunities in rare cancers with limited treatment options.”
Soft tissue sarcomas are a rare and diverse group of cancers that arise in connective and supportive tissues such as muscle, fat, blood vessels, nerves, tendons, and joint linings. In the United States, more than 13,500 new cases are expected to be diagnosed in 2025, with the majority occurring in adults aged 40 and older. Globally, soft tissue sarcomas account for an estimated 96,000 new cases annually, representing a significant unmet medical need.
Lantern said adult soft tissue sarcomas differ biologically from pediatric forms of the disease. While pediatric sarcomas are often driven by specific gene fusions, adult sarcomas more frequently show complex genomic alterations and deficiencies in DNA repair pathways, making them potential targets for therapies that exploit synthetic lethal mechanisms. Treatment options for advanced or metastatic disease remain limited, with five-year survival rates for distant disease reported at approximately 16 to 17 percent.
LP-284 is a novel small-molecule therapy designed to target DNA repair deficiencies through transcription-coupled nucleotide excision repair. In preclinical studies and early clinical development, the compound has shown activity across multiple cancer types, independent of TP53 mutation status or surface antigen expression.
In July 2025, Lantern reported that LP-284 achieved a complete metabolic response in a heavily pretreated patient with aggressive diffuse large B-cell lymphoma enrolled in an ongoing Phase 1 clinical trial. The patient had previously failed multiple advanced therapies, including chemo-immunotherapy, CAR-T cell therapy, and bispecific antibody treatment. Lantern said the response supports the drug’s synthetic lethal mechanism and highlights its potential to address treatment-resistant cancers.
LP-284 is currently being evaluated in a Phase 1 clinical trial for B-cell non-Hodgkin lymphomas, including mantle cell lymphoma and high-grade B-cell lymphoma. With orphan designations spanning both hematologic malignancies and solid tumors, the company said LP-284 represents a versatile development candidate for cancers characterized by DNA repair vulnerabilities.
The FDA’s Orphan Drug Designation program is intended to encourage the development of treatments for rare diseases affecting fewer than 200,000 people in the United States and provides benefits including market exclusivity following approval, tax credits for clinical trials, and regulatory support during development.
