Tonix Pharmaceuticals is currently recruiting participants in a Phase 3 trial for military-related posttraumatic stress disorder.  For more information, please visit www.thehonorstudy.com or www.clinicaltrials.gov (NCT03062540).

Posttraumatic Stress Disorder

Therapeutic Areas: Posttraumatic Stress Disorder

Posttraumatic Stress Disorder Overview

Posttraumatic stress disorder (PTSD) is a serious psychiatric illness that is associated with devastating symptoms, be chronic in nature, and result in significant disability and disruption of life. PTSD can result from experiencing a terrifying, traumatic event which may be re-experienced in the form of flashbacks and nightmares. Sufferers may experience impaired social functioning, occupational disability, intense anxiety and avoidance, emotional numbness, intense guilty or worry, agitation and an overall poor quality of life. Symptoms of PTSD may begin to appear months to years after the traumatic event which may last for years or even decades. PTSD is also often associated with substance abuse and unpredictable or violent behaviors, making it an important public health concern.

In December 2016, Tonix was granted Breakthrough Therapy designation by the FDA for TNX-102 SL, as a treatment for PTSD.

Who is Affected

It is estimated that 3.5% of American adults, or approximately 8.5 million individuals, suffer from PTSD each year, and about half of these patients are receiving some form of treatment. PTSD is often associated with veterans returning from an armed conflict, but anyone who experience a serious accidents, natural or human-caused disasters, exposure to terrorist attacks, violent personal assaults or sexual abuse, or even sudden and major emotional losses are at risk for developing PTSD.

Current Landscape

Various forms of psychotherapy, including cognitive behavior therapy (CBT), eye movement desensitization and reprocessing (EMDR), exposure therapy or virtual reality exposure (VRE) are used to treat PTSD; yet there is a lack of appropriately trained mental health professionals to administer these therapies. Furthermore, although particular trauma-focused therapies, such as CBT and prolonged exposure (PE), have been shown to be efficacious in PTSD, the gains are modest for most patients, particularly for combat veterans.Attribution

The SSRIs (selective serotonin reuptake inhibitors) sertraline and paroxetine are the only drugs approved for the treatment of PTSD. While these drugs may be effective at reducing PTSD symptoms in some patients, their use can be limited by side effects including worsening sleep disturbance. Off-label use of anti-anxiety and sleep medicines are sometimes helpful but have not been adequately studied. Furthermore, prescription sleep medications are not ideal due to their short-term efficacy and safety concerns, as well as potential for abuse.

Many patients rely on both psychotherapy and pharmacotherapy for treatment but it has been more than 15 years since a new drug for PTSD has been approved by the FDA.

Breakthrough Therapy Designation

The FDA's Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned for use to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The benefits of Breakthrough Therapy designation include eligibility for priority review of the NDA in 6 months instead of 10 months under standard review and rolling submission of portions of the application ahead of completion of the full NDA dossier, and an organizational commitment involving FDA's senior managers with more intensive guidance from the FDA.  In some cases, the development program for the Breakthrough Therapy could be shorter than for other drugs intended to treat the disease being studied. However, FDA notes that a compressed drug development program still must generate adequate data to demonstrate that the drug is safe, effective and meets the statutory standard for approval. Breakthrough Therapy designation does not change the standards for approval.  If a clinical development program granted Breakthrough Therapy designation does not continue to meet the criteria, FDA may rescind the designation.

According to an FDA presentation on December 14, 2016, there have been a total of 404 Breakthrough Therapy designation requests since inception in July 9, 2012 through November 30, 2016. Of those, psychiatry projects constitute approximately 5% of the requests.  Out of all 404 Breakthrough Therapy designation requests, 141 (35%) have been granted so far. Of those granted, nine (6%) were for psychiatric products.

The commitment of the FDA Psychiatry Division to expediting approval of medicines with Breakthrough Therapy designation for serious psychiatric condition is exemplified in the first approval of a Breakthrough Therapy psychiatry product, NUPLAZID® (pimavanserin) for hallucinations associated with Parkinson's disease.  NUPLAZID was designated as a Breakthrough Therapy in September 2014, the NDA was submitted in September 2015 and NDA approval was received in April 2016.