Ono Pharma plans further ONO-1110 studies in three pain indications after Phase 2 trials missed primary endpoints despite exploratory efficacy signals.
Written By: Chavi Kalal, PharmD
Reviewed By: Pharmacally Editorial Team
Ono Pharmaceutical announced on October 29, 2026, results from five Phase 2 clinical studies of ONO-1110, an orally available investigational endocannabinoid modulator. The company reported exploratory pain-score efficacy signals in postherpetic neuralgia (PHN), fibromyalgia (FM) and Hunner-type interstitial cystitis (HIC). However, all three studies failed to meet their primary endpoints. Two additional studies in major depressive disorder (MDD) and social anxiety disorder (SAD) showed no efficacy signals.
Based on the findings, Ono plans further clinical studies in PHN, FM and HIC but does not plan additional studies in MDD or SAD. The results provide a basis for continued investigation in selected pain indications, although the available data do not establish clinical efficacy.
ONO-1110 and Its Clinical Rationale
ONO-1110 is an orally available endocannabinoid modulator developed by Ono. Endocannabinoids are naturally produced substances involved in regulating physiological functions, including pain processing and aspects of the central nervous system.
According to the company, ONO-1110 is intended to modulate endocannabinoids produced in response to neuronal activity, including pain transmission. The approach is being investigated as a potential way to address chronic pain through an endogenous physiological mechanism.
The announcement does not specify the drug’s molecular target or provide detailed pharmacological data. ONO-1110 remains investigational across all five indications evaluated.
Three Pain Studies Miss Primary Endpoints
The three pain studies were multicenter, randomized, placebo-controlled Phase 2 trials conducted in Japan. They evaluated the efficacy, safety, tolerability and pharmacokinetics of oral ONO-1110 compared with placebo.
- Postherpetic neuralgia (ONO-1110-04): The study had a target enrollment of 140 participants, with treatment administered once daily for eight weeks.
- Fibromyalgia (ONO-1110-05): The study had a target enrollment of 160 participants and used once-daily treatment for eight weeks.
- Hunner-type interstitial cystitis (ONO-1110-06): The study had a target enrollment of 50 participants, with once-daily treatment for twelve weeks.
All three studies failed to meet their primary endpoints. Ono nevertheless reported signals suggesting improvement in Numerical Rating Scale (NRS) pain scores in prespecified subgroup analyses and exploratory post hoc analyses.
The announcement does not disclose the specific subgroups, treatment-effect estimates, p-values or adjustments for multiple comparisons. It also does not report actual enrollment or the number of participants included in the analyses. Consequently, the magnitude and robustness of the reported signals cannot be independently assessed from the available information.
No Efficacy Signals in Depression or Social Anxiety
The two remaining Phase 2 studies evaluated ONO-1110 in MDD and SAD. Ono reported no efficacy signals in either indication and does not plan further clinical studies in these populations.
The announcement does not provide detailed study designs, participant numbers, treatment durations or numerical efficacy results for these two studies. The reasons for the absence of further development beyond the reported lack of efficacy signals were not elaborated.
Safety Findings Remain Preliminary
Ono characterized ONO-1110’s safety profile as favorable across all five studies. However, the announcement provides no numerical adverse-event rates, serious adverse-event data, discontinuation rates or detailed information on central nervous system effects.
Without these data, the overall safety characterization cannot be assessed in detail or compared quantitatively with placebo. The reported findings should therefore not be interpreted as establishing the drug’s safety profile.
Clinical Interpretation and Next Steps
The results represent a mixed Phase 2 readout. Although subgroup and post hoc analyses identified potential pain efficacy signals in PHN, FM and HIC, the failure of all three studies to meet their primary endpoints remains the principal efficacy finding.
Prespecified subgroup analyses can help identify populations that may warrant further investigation, while exploratory post hoc analyses can generate hypotheses for subsequent testing. However, findings from these analyses require cautious interpretation, particularly when the primary endpoint has not been met. The announcement does not provide enough information to determine the statistical robustness of the reported signals or the extent to which multiple comparisons were addressed.
The studies’ eight- to twelve-week treatment periods also leave longer-term efficacy and safety uncertain. The target enrollment of 50 participants in the HIC study indicates a smaller planned study scale than in PHN and FM, although actual enrollment figures are unavailable.
Ono’s decision to continue development in the three pain indications suggests that the reported findings warrant further investigation, rather than demonstrating an established treatment benefit. The company has not disclosed the designs, timing, endpoints or size of the planned studies.
Further trials with clearly defined primary endpoints, appropriately specified target populations and complete efficacy and safety reporting will be needed to determine whether ONO-1110 provides a clinically meaningful benefit over placebo.
For now, ONO-1110 remains an investigational agent, and its reported pain signals should be viewed as hypothesis-generating rather than confirmatory evidence of efficacy.
Reference
Ono Pharma Announces Results from Five Phase 2 Clinical Studies of ONO-1110, an Endocannabinoid Modulator, Ono Pharmaceuticals, October 29, 2026.
About the Writer
Charvi Kalal (LinkedIn) is a Pharm.D intern and aspiring medical writer with hands-on experience in medical writing, clinical research, pharmacovigilance, and clinical pharmacy. She has experience in medical content writing through CliMed, along with exposure to ADR monitoring, prescription analysis, patient counselling, literature review, and research documentation. Her research focuses on ADR monitoring and reporting in breast cancer patients receiving targeted therapy. With certifications in GCP, biomedical research, scientific writing, and clinical research, she is passionate about transforming scientific evidence into accurate, engaging, and accessible healthcare content.