Trial of Intranasal Oxytocin for Chronic Migraine Cleared by FDA


Approval permits the initiation of a phase 2 study to ascertain the efficacy of intranasal potentiated oxytocin in patients with chronic migraine.

Seth Lederman, MD, president and CEO, Tonix

Seth Lederman, MD

The FDA has cleared an investigational new drug application supporting initiation of a phase 2 study of TNX-1900 (intranasal potentiated oxytocin) in patients with chronic migraine, Tonix Pharmaceuticals announced recently. The treatment is also anticipated to be eligible for the 505(b)(2) pathway for FDA approval—an option for new formulations of approved drugs. 

TNX-1900 is a proprietary formulation of oxytocin also being developed for treatment of craniofacial pain, insulin resistance, and other conditions, in addition to prophylaxis of chronic migraine. Based on an intranasal actuator device, the combination product delivers oxytocin into the nose, concentrating in the trigeminal system where it binds to receptors on neurons, preventing the release of CGRP and transmission of pain signals. 

“We are excited to have received the FDA’s IND clearance to begin clinical trials for TNX-1900 in prevention of migraine headaches in chronic migraineurs,” Seth Lederman, MD, president and CEO, Tonix, said in a statement. “An estimated 4 million individuals in the United States suffer from chronic migraine. We believe that by engaging and stimulating oxytocin receptors in the trigeminal ganglia, TNX-1900 has the potential to help chronic migraine sufferers. TNX-1900 contains magnesium, which potentiates the action of oxytocin at oxytocin receptors in animal models.”

The phase 2 study is anticipated to begin enrolling patients in the second half of 2022, Lederman said. 

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TNX-1900 isn’t the only intranasal migraine treatment being evaluated. Data presented earlier this year at the 2021 Virtual American Headache Society (AHS) 63rd Annual Scientific Meeting, June 3-6, suggest that treatment with Impel Neuropharma’s intranasal formulation of dihydroergotamine (DHE), INP104, had an acceptable safety profile for treatment of migraine. Data was from the long-term, phase 3 STOP 301 trial (NCT03557333), in which investigators noted that safety should be monitored with intermittent use of the medication. 

“Patients underwent endoscopy, so looking at it anatomically with an upper nasal endoscopy, and we did not find any issues that were related to INP104,” Sheena Aurora, MD, vice president, Medical Affairs – Migraine, Impel Neuropharma, and adjunct clinical associate professor of anesthesiology, perioperative, and pain medicine, Stanford Health Care, told NeurologyLive® at the time. “What is good for clinicians and patients is that adverse event reporting was just as sensitive as the findings. We didn’t find any permanent changes. The most common adverse event was nasal congestion.”

Investigators analyzed data from the full safety set of 354 patients from the STOP study, of which, 36.7% (n = 130) reported at least 1 treatment-related, treatment-emergent adverse event (TEAE) during the 24-week treatment period. Over 52 weeks and 6332 doses of INP104, 30 patients reported 39 nausea TEAEs (0.6% of doses), and although mild nasal congestion was not uncommon (n = 59; 16.7%), no concerning nasal or cardiovascular TEAEs were reported.2

The treatment, marketed as Trudhesa, was approved by the FDA in September 2021 for the acute treatment of migraine headaches with or without aura in adults. The approval made the treatment the first and only approved therapy that utilized Precision Olfactory Delivery technology, a novel delivery system that specifically targets the upper nasal space.3

1. Tonix Pharmaceuticals announces FDA clearance of the IND for potentiated intranasal oxytocin (TNX-1900) for the prevention of migraine headache in chronic migraineurs. News release. Tonix Pharmaceuticals. November 23, 2021. Accessed December 3, 2021.
2. Auruora SK. DHE Pharmacology Revisited: Does a Broad Receptor Profile Molecule Treat the Whole Migraine? Presented at AHS 2021 Scientific Annual Meeting; June 3-6.
3. Impel NeuroPharma announces US FDA approval of Trudhesa (Dihydroergotamine mesylate) nasal spray for the acute treatment of migraine. News release. Impel NeuroPharma. September 3, 2021. Accessed December 3, 2021.
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