Have you ever heard of Orphan Drug designation? This designation, granted by the U.S. Food and Drug Administration (FDA), is given to drugs or biologics that are designed to treat, prevent, or diagnose rare conditions. A rare condition is one affecting fewer than 200,000 people nationwide. Orphan Drug designation comes with benefits such as fee waivers, tax credits, and up to seven years of market exclusivity if/when the drug is approved. Overall, this designation was developed to incentivize drug development within the rare disease sphere. According to reporting from OncLive, the FDA granted Orphan Drug designation to LP-284 for the treatment of high-grade B-cell lymphomas with MYC or BCL2 rearrangements. This is the second Orphan Drug designation granted to LP-284; it previously received this status for the treatment of mantle cell lymphoma (MCL).
What is High-Grade B-Cell Lymphoma?
The Lymphoma Research Foundation describes high-grade B-cell lymphoma as:
an aggressive type of B-cell non-Hodgkin lymphoma (NHL) characterized by rearrangements (parts of genes switch places within chromosomes) in two particular genes…the MYC gene, and… the BCL2 gene.
Symptoms may include fever and night sweats; losing weight without trying; fatigue; appetite loss; shortness of breath; pain; and swollen lymph nodes in the groin, neck, or underarms.
LP-284 is a small-molecule acylfulvene therapy that causes cancer cells to die. In murine (mice) models of B-cell NHL with MYC/BCL2 rearrangements, LP-284 was shown to significantly prevent tumor growth by 57%. When combined with rituximab, the percentage rose to 93%.
Moving forward, researchers plan to evaluate LP-284 for solid tumors and relapsed/refractory lymphoma in a Phase 1 clinical study. Participants must be adults whose condition is relapsed or refractory following 2+ prior therapies. During the first portion of the trial, researchers will perform dose escalation to identify the ideal dose for future studies. In the second portion of the study, researchers will focus on dose expansion, as well as how safe and well-tolerated the treatment is.
