Lantern Pharma Announces FDA Orphan Drug Designation for LP-284 and USPTO Patent Allowance for LP-184
Lantern Pharma's LP-284 received FDA Orphan Drug Designation for soft tissue sarcomas, while the USPTO allowed a patent for biomarker-guided use of LP-184 in multiple cancers. LP-284 showed a complete metabolic response in a Phase 1 DLBCL patient, and LP-184 demonstrated a 45% disease control rate in advanced solid tumors.
Lantern Pharma announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to LP-284 for soft tissue sarcomas, and the U.S. Patent and Trademark Office has issued a Notice of Allowance for a patent covering biomarker-guided use of its investigational therapy LP-184 (zirdafulven) in ovarian, primary liver, kidney, and thyroid cancers.
The FDA designation for LP-284 marks the third orphan indication for the drug, following previous designations in mantle cell lymphoma (January 2023) and high-grade B-cell lymphoma with MYC and BCL2 rearrangements (November 2023), and is the sixth overall orphan designation for Lantern Pharma's clinical programs. Adult soft tissue sarcomas, which represent over 79% of the approximately 13,520 expected U.S. cases in 2025, are characterized by complex genomic alterations, chromosomal instability, and DNA repair deficiencies that align with LP-284's synthetic lethal mechanism. The CEO of Lantern Pharma noted that the RADR® AI platform identified these DNA repair vulnerabilities, demonstrating its ability to uncover biomarker-driven precision oncology opportunities in rare cancers.
In July 2025, Lantern reported that LP-284 achieved a complete metabolic response in a heavily pretreated patient with diffuse large B-cell lymphoma in its ongoing Phase 1 trial. The 41-year-old patient, who had failed chemo-immunotherapy, CAR-T cell therapy, and bispecific antibody therapy, enrolled in April 2025 and showed non-avid lesions after two 28-day cycles of LP-284. The drug is a novel small molecule targeting DNA repair deficiencies via transcription-coupled nucleotide excision repair (TC-NER).
Soft tissue sarcoma represents a significant unmet need, with approximately 96,200 new cases globally in 2021, according to the Global Burden of Disease Study. The seven major pharmaceutical markets for soft tissue sarcoma therapeutics reached a value of approximately $2.4 billion in 2025 and are projected to reach $4.7 billion by 2035. Five-year survival rates for distant disease remain approximately 16-17%.
The USPTO allowance for LP-184 covers methods of selecting patients based on elevated expression of PTGR1, PTPN14, and ASPH. LP-184 is a next-generation acylfulvene prodrug activated inside tumor cells by PTGR1, leading to DNA cross-links and double-strand breaks that exploit synthetic lethality in cells with deficient DNA damage repair, such as BRCA1/2-mutant tumors. A completed 63-patient Phase Ia trial in recurrent or refractory advanced solid tumors showed a 45% disease control rate among patients treated at or above the effective therapeutic dose. Lantern plans to advance LP-184 into multiple biomarker-guided Phase Ib/II trials during 2026, including the indications named in the newly allowed patent. LP-184 has also received FDA Fast Track designation for triple-negative breast cancer and glioblastoma, as well as Orphan Drug designations for malignant gliomas, pancreatic cancer, and atypical teratoid rhabdoid tumor.