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Investigational Therapy TNX-102 SL Receives FDA Fast Track for Agitation in Alzheimer Disease

TNX-102 SL’s impact on sleep disturbance highlights a mechanism of action that could result in positive effects on agitation in AD.

Seth Lederman, MD

Tonix Pharmaceuticals announced today that the FDA has granted a Fast Track designation to TNX-102 SL, an investigational new therapy intended to treat agitation in Alzheimer disease (AD).

Currently, the therapy is being assessed in a phase II trial in patients with AD agitation after 8 weeks of bedtime treatment, in addition to genomic DNA analyzation to attempt to identify biomarkers associated with treatment response.

"Fast Track designation reflects the recognition by the FDA that TNX-102 SL has the potential to address a large unmet medical need for a serious condition," said Seth Lederman, MD, the chief executive officer of Tonix, in a statement.1 "Currently, there are no FDA-approved treatments for agitation in Alzheimer's disease, despite a high disease burden and a need for an effective therapy. We are eager to work closely with the FDA to advance [the] development of TNX-102 SL for the treatment of agitation in Alzheimer's disease."

In May, the FDA granted clearance of Tonix’s Investigational New Drug Application (IND), which supported the initiation of the phase II, pivotal efficacy study of the therapy in a 5.6-mg dose. At the time, Lederman said that the new indication would potentially expand Tonix’s pipeline, and would leverage the company’s understanding of the TNX-102 SL mechanism of action in AD.2

“The investigation of TNX-102 SL for agitation in Alzheimer’s disease may bring a new treatment targeting sleep disturbances in patients experiencing this condition,” Lederman said in May. “Currently there are no FDA-approved treatments for agitation in Alzheimer’s disease, despite a high disease burden and a need for an effective therapy. A Fast Track designation request will be filed next month to potentially accelerate the development and review of this unmet medical need.”

Lederman noted after a pre-IND submission meeting with the FDA, in December 2017, that the therapy’s potential to treat agitation in Alzheimer stems from its mechanism of action that positively impacts sleep quality for patients.3 Agitation in dementia, and moderate cognitive impairment, has been associated with sleep disruption, making the low-dosage strength of the therapy and its late-night dosing regimen an “ideal candidate” for this indication, according to Tonix’s chief medical officer, Gregory Sullivan, MD.

In a phase II trial of the therapy in 231 patients with posttraumatic stress disorder (PTSD), led by Sullivan, that compared the therapy in 2 doses—2.8 mg and 5.6 mg—to placebo, the therapy’s effect on sleep disturbance was assessed by a comparison of the mean change from baseline.3 That trial found that the Least Squares (LS) mean change for Clinician-Administered PTSD Scale for DSM-5 (CAPS-5) item E6, measuring sleep disturbance, was reduced significantly at weeks 2, 4, 8, and 12 for the 5.6-mg dose in comparison to placebo (P <.05 for all).

Previously, the therapy, while marketed as Tonmya and seeking an indication for the treatment of PTSD, was granted a Breakthrough Therapy designation. It is currently being tested for that indication in a phase III trial for military-related PTSD. That analysis is expected in Q3 of 2018.


1. Tonix Pharmaceuticals Receives Fast Track Designation from the U.S. FDA for TNX-102 SL for Treatment of Agitation in Alzheimer’s Disease [press release]. New York, NY: Tonix Pharmaceuticals; July 16, 2018. tonixpharma.com/news-events/press-releases/detail/1109/tonix-pharmaceuticals-receives-fast-track-designation-from. Accessed July 16, 2018.

2. Tonix Pharmaceuticals Receives IND Clearance by U.S. FDA for TNX-102 SL in Agitation in Alzheimer’s Disease [press release]. New York, NY: Tonix Pharmaceuticals; May 1, 2018. tonixpharma.com/press-releases/detail/1102/tonix-pharmaceuticals-receives-ind-clearance-by-u-s-fda. Accessed July 16, 2018.

3. Sullivan G, Gendreau J, Gendreau RM, et al. The AtEase Study: A Phase 2 Multicenter Randomized Clinical Trial of the Safety and Efficacy of TNX-102 SL in the Treatment of Military-Related PTSD. Poster presented


57th American Society of Clinical Psychopharmacology Annual Meeting; June 1, 2016; Scottsdale, AZ. content.equisolve.net/tonixpharma/media/14e76561cc098ad018f50d7f37832e87.pdf. Accessed July 16, 2018.