Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) has unveiled significant progress in its suzetrigine pain program, potentially introducing the first new class of medication for acute and neuropathic pain in over twenty years. Suzetrigine, an oral selective NaV1.8 pain signal inhibitor (previously VX-548), has garnered attention for its promising developments.
Building on the positive Phase 3 outcomes in acute pain disclosed in January 2024, the Food and Drug Administration (FDA) has initiated a rolling New Drug Application (NDA) submission for suzetrigine in moderate-to-severe acute pain. Vertex has already commenced this submission process and is set to conclude it by the second quarter of 2024. Suzetrigine had previously been granted FDA Fast Track and Breakthrough Therapy designations for moderate-to-severe acute pain.
In the realm of neuropathic pain, Vertex has shared encouraging results from its Phase 2 study in December 2023 and recently concluded a fruitful end-of-phase 2 meeting with the FDA. The company is now gearing up to launch a Phase 3 pivotal program for suzetrigine in patients with DPN (Diabetic Peripheral Neuropathy) in the latter half of 2024. Moreover, suzetrigine has received Breakthrough Therapy designation from the FDA for treating pain associated with DPN.
The Phase 3 initiative will entail two identical 12-week randomized, double-blind, placebo-controlled studies, evaluating the efficacy and safety of suzetrigine (70 mg once daily) in DPN patients. The primary endpoint for both studies will be the alteration from baseline in the weekly average of daily pain intensity, measured on the numeric pain rating scale (NPRS) at Week 12 compared to placebo. Furthermore, both studies will feature a significant secondary endpoint, examining the change from baseline in the weekly average of daily pain intensity on the NPRS at Week 12 compared to pregabalin. Each Phase 3 study anticipates enrolling approximately 1,100 patients. Post-completion of the randomized controlled studies, patients may transition to an open-label study to assess the long-term safety and efficacy of suzetrigine in DPN.
Additionally, Vertex is actively enrolling participants for its Phase 2 study of suzetrigine targeting patients with lumbosacral radiculopathy (LSR), a condition characterized by pain stemming from nerve root impairment or injury in the lumbar spine region. The company aims to wrap up enrollment for the Phase 2 LSR study by year-end.
Dr. Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, remarked, “Today marks a significant milestone on our journey to redefine the treatment of pain. Given the favorable benefit/risk profile demonstrated by suzetrigine across the entire clinical program and the positive interactions with regulators, we are excited by the opportunity to rapidly advance suzetrigine, a new non-opioid potential treatment, for the millions of patients suffering from acute and peripheral neuropathic pain.”
Looking ahead, Vertex is committed to advancing preclinical and clinical development of additional NaV1.8 and NaV1.7 pain signal inhibitors, either alone or in combination, for acute and neuropathic pain. The company plans to progress its next-generation NaV1.8 pain signal inhibitor VX-993 oral formulation into Phase 2 acute pain and peripheral neuropathic pain studies later this year. Moreover, Vertex anticipates commencing a Phase 1 study of an intravenous formulation of VX-993 within the same timeframe.
Acute pain, defined as pain persisting for less than 3 months, is a debilitating condition affecting millions of individuals annually in the U.S. The limited treatment options underscore an unmet need in acute pain management, highlighting the urgency for enhancing patient experiences and alleviating the economic and societal burdens associated with the condition.
