VERTANICAL Announces Positive Results From the VER-01 Phase 3 Program for the Treatment of Chronic Low Back Pain (CLBP)

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• VER-01 significantly reduced pain intensity in chronic low back pain compared with placebo, with effects sustained during 12 months of treatment. 
• VER-01 was generally well tolerated, with no signs of dependence, or withdrawal.
• In a second Phase 3 head-to-head study comparing VER-01 with opioids, VER-01 demonstrated superior pain reduction and gastrointestinal tolerability.
• VERTANICAL submitted for marketing authorization in several European countries for the treatment of chronic low back pain.
• A Phase 3 study in the U.S. is planned to start in early 2026 to support a submission for FDA approval. 

MUNICH, Oct. 1, 2025 /PRNewswire/ -- Vertanical today announced publication of results from two Phase 3 clinical studies investigating the safety and efficacy of VER-01, a first-in-class standardized full-spectrum extract from Cannabis sativa DKJ127 L, for the treatment of chronic low back pain (CLBP). The Phase 3 findings point to a potential therapeutic breakthrough for the treatment of CLBP.

Published in Nature Medicine and Pain & Therapy, the results demonstrate that VER-01 provides superior pain relief and gastrointestinal tolerability compared to opioids, without evidence of dependence or abuse¹. VER-01 also improved key symptoms of chronic pain – including sleep disturbances and impaired physical function – resulting in a significant improvement in patients' quality of life ².

"These findings provide powerful evidence that VER-01 could transform how we care for patients with chronic lower back pain, if approved," says Prof. Charles E. Argoff, Professor of Neurology at Albany Medical College and Past President of the American Academy of Pain Medicine. "The results of the Phase 3 studies bring hope to millions living with chronic pain that, if approved, VER-01 could deliver effective pain relief while addressing key safety challenges of current therapies."

CLBP affects more than half a billion people worldwide and is the leading cause of disability and reduced quality of life^3,4. Yet current pharmacological treatment options remain largely limited to nonsteroidal anti-inflammatory drugs and opioids, both of which have been associated with significant safety concerns and potentially serious side effects, particularly with long-term use ^5,6,7.

VER-01 shows superiority over placebo in pivotal Phase 3 study

In a placebo-controlled double-blind Phase 3 study enrolling 820 patients with CLBP published in Nature Medicine, VER-01 demonstrated significant pain reduction compared with placebo, meeting the study's primary endpoint. After 12 weeks of treatment, patients receiving VER-01 achieved a pain reduction of -1.9 points on the 0- to 10-point pain numerical rating scale (NRS). In an open-label extension phase, pain intensity further decreased to -2.9 points after six months and was sustained over 12 months continued treatment. Effects of VER-01 were particularly pronounced in participants with neuropathic pain and those with severe pain. Study participants receiving VER-01 also reported significantly greater improvements in sleep quality and physical function compared with placebo.

VER-01 was generally well tolerated with no evidence of dependence or withdrawal. While mild to moderate adverse events, generally related to dizziness, headache, fatigue, nausea, dry mouth, and somnolence, were more frequent with VER-01 than placebo, most side effects occurred only during the initial 3-week titration phase of the study and were generally transient. The incidence of serious adverse events was comparable to placebo showing no safety signals.

VER-01 shows superiority over opioids in ELEVATE head-to-head comparator study

A second phase 3 study ("ELEVATE") directly compared VER-01 with opioids in patients with chronic low back pain (CLBP). Published in Pain & Therapy, the study demonstrated superiority of VER-01 to opioids in both efficacy and gastrointestinal tolerability. The Phase 3 open-label study enrolling 384 patients met its primary endpoint, demonstrating a significantly higher incidence of constipation in the opioid group compared to the VER-01 group. Participants receiving VER-01 were four times less likely to develop constipation than those receiving opioids and three times less likely to use laxatives. VER-01 was more effective in alleviating pain than opioids with patients also experiencing improved sleep throughout the 6 months of treatment. Pain reduction was particularly pronounced in participants with neuropathic pain and those with severe pain.

"The two Phase 3 studies show that VER-01 could be a transformative therapy," says Prof. Roger Knaggs, Professor of Pain Management and President of the British Pain Society. "Patients and physicians have long sought novel treatments for chronic pain. If approved, VER-01 could represent the first new class of chronic pain medicine for a very long time."

About VER-01

VER-01 is an investigational standardized full-spectrum extract from the Cannabis sativa strain DKJ127 L. Its unique phytochemical composition contains a distinct and complex mixture of cannabinoids, terpenes and other bioactive compounds, such as Cannabigerol, β-caryophyllene and α-bisabolol, that act synergistically and contribute to its overall pharmacological effects ("entourage effect"). VER-01 is manufactured using a proprietary extraction process, preserving the plant's full spectrum of bioactive compounds.

Based on the Phase 3 data, VERTANICAL submitted VER-01 for marketing authorization in multiple European countries for the treatment of chronic low back pain. An additional pivotal Phase 3 study in the U.S. is planned to start in early 2026 to support the marketing application for VER-01 in the U.S.

About VERTANICAL

VERTANICAL is a leading biopharmaceutical company dedicated to developing and commercializing innovative therapies for chronic pain. Guided by the vision of a world free of chronic pain, the company is committed to advancing clinical research and development in chronic pain diseases. With over 20 completed preclinical and clinical studies and a comprehensive development program across multiple chronic pain indications, VERTANICAL is recognized as one of the global innovation leaders in the field of chronic pain.

VERTANICAL was founded in 2007 and has its global headquarters in Munich, Germany. The company operates one of the world's most advanced GMP facilities for the development and manufacturing of biopharmaceuticals. It maintains an international network of several hundred research and clinical trial sites across the U.S. and multiple European countries.

¹ Meissner W, Argoff C, Sator S, Schoder V, Karst M. VER-01 shows enhanced gastrointestinal tolerability, superior pain relief, and improved sleep quality compared to opioids in treating chronic low back pain: a randomized phase 3 clinical trial. Pain Ther (2025), published online on September 30, 2025, doi:10.1007/s40122-025-00773-z
² Karst M, Meissner W, Sator S, Keßler J, Schoder V, Häuser W. Full-spectrum extract from Cannabis sativa DKJ127 for chronic back pain: a randomized, placebo-controlled phase 3 study. Nat Med (2025), published online on September 29, 2025, doi: 10.1038/s41591-025-03977-0
³ Wu A, March L, Zheng X, et al. Global low back pain prevalence and years lived with disability from 1990 to 2017: estimates from the Global Burden of Disease Study 2017. Ann Transl Med. 2020;8(6):299. doi:10.21037/atm.2020.02.175
⁴ GBD 2021 Low Back Pain Collaborators. Global, regional, and national burden of low back pain, 1990-2020, its attributable risk factors, and projections to 2050: a systematic analysis of the Global Burden of Disease Study 2021. Lancet Rheumatol. 2023 May 22;5(6):e316-e329. doi: 10.1016/S2665-9913(23)00098-X. PMID: 37273833; PMCID: PMC10234592.
⁵ Salvo F, Antoniazzi S, Duong M, et al. Cardiovascular events associated with the long-term use of NSAIDs: a review of randomized controlled trials and observational studies. Expert Opin Drug Saf. 2014;13(5):573-585. doi:10.1517/14740338.2014.907792
⁶ Wirth T, Lafforgue P, Pham T. NSAID: Current limits to prescription. Joint Bone Spine. 2024 Jul;91(4):105685. doi: 10.1016/j.jbspin.2023.105685. Epub 2023 Dec 29. PMID: 38159794.
⁷ Vowles KE, McEntee ML, Julnes PS, Frohe T, Ney JP, van der Goes DN. Rates of opioid misuse, abuse, and addiction in chronic pain: a systematic review and data synthesis. Pain. 2015;156(4):569-576. doi:10.1097/01.j.pain.0000460357.01998.f1

SOURCE Vertanical