Lantern Pharma announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to LP-284 for the treatment of soft tissue sarcomas.

This marks the third orphan designation for LP-284, following previous orphan designations in Mantle Cell Lymphoma (MCL) in January 2023 and High-Grade B-Cell Lymphoma (HGBL) with MYC and BCL2 rearrangements in November 2023. It is the sixth overall orphan designation granted to Lantern Pharma's clinical programmes.

"Receiving orphan drug designation for LP-284 in soft tissue sarcomas expands this molecule’s potential beyond haematologic malignancies into solid tumours. Adult soft tissue sarcomas are a compelling opportunity for LP-284. Unlike paediatric 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 per cent 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," said Panna Sharma, CEO, Lantern Pharma.

Soft tissue sarcomas are a diverse group of rare cancers that develop in the tissues that connect, support and surround other body structures, including muscle, fat, blood vessels, nerves, tendons and the lining of joints. According to the National Cancer Institute's SEER Program, approximately 13,520 new cases of soft tissue sarcoma are expected to be diagnosed in the United States in 2025. Over 79 per cent of soft tissue sarcoma cases occur in patients 40 years of age or older.

Globally, soft tissue sarcoma represents a significant health burden with approximately 96,200 new cases diagnosed worldwide in 2021, according to the Global Burden of Disease Study. The seven major pharmaceutical markets (United States, France, Germany, Italy, Spain, United Kingdom and Japan) for soft tissue sarcoma therapeutics reached a value of approximately USd 2.4 billion in 2025 and are projected to reach approximately USD 4.7 billion by 2035.

Adult soft tissue sarcomas exhibit distinct molecular characteristics compared to paediatric sarcomas. While paediatric sarcomas are largely driven by specific gene fusions, adult sarcomas more commonly harbor complex genomic alterations, chromosomal instability and DNA repair deficiencies that may be susceptible to agents that target DNA repair pathways.

According to the Sarcoma Foundation of America’s 2025 Sarcoma Statistics, treatment options for advanced or metastatic soft tissue sarcomas remain limited, with five-year survival rates for distant disease at approximately 16-17 per cent, representing a significant unmet medical need in adult oncology.

LP-284 is a novel small molecule with a synthetic lethal mechanism targeting DNA repair deficiencies via Transcription-Coupled Nucleotide Excision Repair (TC-NER). A member of the acylfulvene class, LP-284 has demonstrated promising activity across multiple cancer types in pre-clinical studies and early clinical development, with observed activity regardless of TP53 mutation status or surface antigen expression.

LP-284 is currently being evaluated in a phase I clinical trial (NCT06132503) for B-cell non-Hodgkin lymphomas, including MCL and HGBL. With three orphan designations spanning both haematologic malignancies and solid tumours, LP-284 represents a versatile therapeutic development candidate with potential applications across diverse cancer types characterised by DNA repair vulnerabilities.

The FDA's Orphan Drug Designation programme provides incentives for the development of drugs intended to treat rare diseases affecting fewer than 200,000 people in the United States. Benefits of orphan drug designation include seven years of market exclusivity upon regulatory approval, tax credits for qualified clinical trials, exemption from FDA user fees and FDA assistance in clinical trial design.