Pilavapadin

– No objections raised by FDA to advancement of pilavapadin into Phase 3 development utilizing two-arm registrational studies of 10 mg daily dose compared to placebo – Jan. 21, 2026 -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced the successful completion of the End-of-Phase 2 (EOP2) Meeting with the U.S. Food and Drug Administration (FDA) for pilavapadin in diabetic peripheral neuropathic pain (DPNP). During the meeting, the FDA raised no objections to the advancement of pilavapadin into Phase 3 development, which would include two placebo-controlled, 12-week, two arm registrational studies comparing the 10 mg daily dose to placebo. The primary endpoint of the Phase 3 studies will be the change in average daily pain score (ADPS) from baseline to Week 12. “Our End-of-Phase 2 meeting with FDA was productive and provided Lexicon with the insights needed to design a robust Phase 3 program, with the goal of making the first non-opioid DPNP medicine available to patients in over two decades,” said Mike Exton, Ph.D., chief executive officer and director of Lexicon. “Furthermore, the FDA did not require any unanticipated preclinical or clinical studies that would be expected to complicate or delay the advancement of this program into Phase 3 development and a potential regulatory submission.” Discovered using Lexicon’s unique approach to gene science, pilavapadin is a potent, once-daily, orally delivered, selective, investigational small molecule inhibitor of AAK1, a novel target for neuropathic pain which inhibits reuptake and recycling of neurotransmitters involved in pain signaling without affecting opiate pathways. Lexicon identified AAK1 in its target discovery efforts as a promising approach for the treatment of neuropathic pain. Preclinical studies of pilavapadin demonstrated central nervous system penetration and reduction in pain behavior in models of neuropathic pain without affecting opiate pathways. Lexicon is a biopharmaceutical company with a mission of pioneering medicines that transform patients’ lives. Through the Genome5000™ program, Lexicon’s unique genomics target discovery platform, Lexicon scientists studied the role and function of nearly 5,000 genes and identified more than 100 protein targets with significant therapeutic potential in a range of diseases. Through the precise targeting of these proteins, Lexicon is pioneering the discovery and development of innovative medicines to treat disease safely and effectively. Lexicon has a pipeline of promising drug candidates in discovery and clinical and preclinical development in neuropathic pain, hypertrophic cardiomyopathy (HCM), obesity, metabolism and other indications. The content above comes from the network. if any infringement, please contact us to modify.

iStock, Yutthana Gaetgeaw The company also stands to gain from recent regulatory FDA guidance aimed at streamlining the development of non-opioid painkillers, Jefferies analysts suggested. Following a successful end-of-Phase II meeting with the FDA, Lexicon Pharmaceuticals can now move forward with the late-stage development of its non-opioid analgesic pilavapadin, taking one step closer to what Jefferies estimates is a market opportunity that exceeds $1 billion. Lexicon closed Wednesday’s trading session at $1.56, a 19% jump from its closing price of $1.31 on Tuesday. “Pilavapadin’s development could be helped by regulatory tailwinds, supporting the use of non-opioid alternatives in chronic pain indications,” the Jefferies analysts wrote in a Wednesday note to investors, pointing specifically to a recently published draft guidance to help drugmakers develop non-opioid options for chronic pain. Lexicon stands to gain from such regulatory flexibility, Jefferies said Wednesday. “The regulatory environment in chronic pain is improving, as the FDA is collaborating with sponsors on non-addictive alternatives,” they added. Lexicon is advancing pilavapadin for diabetic peripheral neuropathic pain (DPNP)—a market opportunity that could exceed $1 billion, according to Jefferies. During the regulatory meeting, the FDA raised no issues regarding advancing pilavapadin into Phase III, Lexicon said in its news release. The company was also able to align with the agency on the pivotal design of the trial: two placebo-controlled studies that would run for 12 weeks, using a 10-mg daily oral dose. Change in average daily pain score was set as the program’s primary outcome. At the meeting, the FDA also “did not require any unanticipated preclinical or clinical studies that would be expected to complicate or delay” the development of pilavapadin, Lexicon CEO Mike Exton said in a statement. If approved, pilavapadin would become the first non-opioid therapy for DPNP “in over two decades,” he added. In March last year, Lexicon announced that the drug was unable to significantly ease pain in a Phase IIb study of DPNP as compared with placebo—results that at the time sank the biotech’s shares by 50%. Still, Lexicon decided to push pilavapadin into late-stage development, pointing to its “ enormous potential ” in chronic pain. This decision was vindicated months later, in September, when a post-hoc data analysis found “ nominally significant separation from placebo ” in terms of pain improvement. Jefferies also noted that beginning Phase III would be “contingent upon securing a BD partnership,” which the firm said could be helped by this positive FDA outcome.