Lexicon Pharmaceuticals (NASDAQ:LXRX) successfully completed an End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration for its lead pain candidate, pilavapadin.
The FDA raised no objections to the company’s proposal to advance the 10 mg daily dose of pilavapadin into a pivotal Phase 3 program, targeting diabetic peripheral neuropathic pain (DPNP)—a market that hasn't seen a novel, non-opioid oral therapy approved in over 20 years.
The planned Phase 3 program will consist of two identical 12-week, placebo-controlled registrational studies.
The primary endpoint will measure the change from baseline in the average daily pain score (ADPS) at Week 12.
Crucially for investors, Lexicon confirmed that the FDA did not mandate any additional preclinical or unanticipated clinical studies, removing a significant potential hurdle for the therapy's regulatory timeline.
Pilavapadin, a first-in-class AAK1 inhibitor, represents a structural shift away from traditional pain management.
Unlike opioids, the small molecule works by inhibiting the reuptake and recycling of neurotransmitters involved in pain signaling without engaging opiate pathways.
Recent Phase 2b data showed that the 10 mg dose achieved a clinically meaningful two-point reduction in pain scores while maintaining a safety profile characterized by mild-to-moderate side effects, primarily nausea and dizziness.
With roughly 9 million Americans suffering from DPNP, Lexicon is positioning pilavapadin as a potential blockbuster alternative in an era of heightened opioid scrutiny.