FDA Grants Orphan Drug Designation to Investigational Drug for Mantle Cell Lymphoma


LP-284, which now has orphan drug designation from the FDA for mantle cell lymphoma, yielded positive preclinical data at the 2022 American Society of Hematology Annual Meeting and Exposition.

The FDA has granted orphan drug designation to the investigational small molecule agent LP-284 for the management of mantle cell lymphoma (MCL), according to a press release from Lantern Pharma.1

LP-284 received the designation following reports of positive preclinical data shared at the 2022 American Society of Hematology (ASH) Annual Meeting, which indicated that the agent produced anti-tumor activity in MCL. In particular, LP-284 shrunk and delayed progression in MCL xenograft tumors tumors that had developed resistance following pretreatment with ibrutinib (Imbruvica) and bortezomib (Velcade).2

Data indicated that LP-284 yielded near complete tumor growth inhibition in pretreated tumors in xenograft animal models, and that no tumor regrowth or animal deaths were observed 15 days following the last dose of LP-284. At 17 days following treatment, LP-284 yielded a tumor growth inhibition rate of 113% and 63% at a dosing level of 4 mg/kg and 2 mg/kg, respectively; ibrutinib and bortezomib produced respective tumor growth inhibition rates of 8% and 22%.

“Receiving orphan drug designation is an important milestone for our latest drug candidate, LP-284, and further validates our data-driven approach to oncology drug discovery and development,” Panna Sharma, president and chief executive officer of Lantern Pharma, said in the press release. He also noted that the findings presented at ASH were “critically pertinent due to the high relapse rate and poor prognosis of the majority of patients [with MCL].”

LP-284 is a novel agent that preferentially damages DNA expressed in cancer cells with mutations in DNA damage repair pathways. The agent was designed to manage several types of B-cell non-Hodgkin lymphoma, including MCL and double hit lymphoma.

In the preclinical investigation of LP-284, investigators assessed the survival benefit and efficacy of the agent in xenograft mice with MCL compared with ibrutinib and bortezomib. Investigators administered 2 mg/kg or 4 mg/kg per dose of LP-284 intravenously vs 50 mg/kg per dose of oral ibrutinib or 1 mg/kg of bortezomib as intraperitoneal therapy.

Other primary objectives from the study included exploring the agent’s mechanism of action in inducing DNA damage and blocking transcription as well as evaluating its toxicity profile and pharmacokinetics in dogs.

LP-284’s safety profile was assessed in dogs and was conducted via a non-GLP repeat dose toxicity study. Reported adverse effects (AEs) pertained to body weight, clinical pathology, and pathology, and were observed in doses of 0.6 mg/kg or greater. Additionally, hematologic changes related to LP-284 were observed in doses of 0.3 mg/kg or greater. Investigators identified a No Observed AE Level of 0.3 mg/kg per dose.

The manufacturers of LP-284 will file an investigational new drug application with the FDA and organize a first-in-human phase 1 trial for the agent in the management of B-cell non-Hodgkin lymphomas including MCL by mid 2023.

In addition to the designation for LP-284, the FDA had previously granted orphan drug designations to LP-184 for treating patients with malignant gliomas, atypical teratoid rhabdoid tumors, and pancreatic cancer. The FDA also gave the agent a rare pediatric disease designation for atypical teratoid rhabdoid tumors.


  1. FDA grants Lantern Pharma orphan drug designation for drug candidate LP-284 in mantle cell lymphoma. News release. Lantern Pharma. January 5, 2023. Accessed January 16, 2023. yhoo.it/3H82EiR
  2. Zhou J, Kulkarni A, Love C, et al. Development of a potent DNA damaging agent LP-284 for treatment of mantle cell lymphoma. Presented at: 64th American Society of Hematology Annual Meeting and Exposition; December 10-13, 2022; New Orleans, Louisiana. Abstract 2156.
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