Central Nervous System

Central Nervous System

Tonix is committed to developing therapeutics that improve the lives of patients suffering from chronic, often overlooked conditions, with a focus on Central Nervous System (CNS) disorders. Relying on in-house discovery as well as external collaborations, Tonix has transformed into a company that now includes biologic product candidates, in addition to small molecule drug candidates, targeting an array of CNS disorders.


Fibromyalgia (FM) is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. FM afflicts an estimated 6-12 million adults in the U.S.1, approximately 90% of whom are women. Symptoms include chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled.

1 American Chronic Pain Association (www.theacpa.org, 2019)
Vincent A, Lahr BD, Wolfe F, Clauw DJ, Whipple MO, Oh TH, Barton DL, St Sauver J. Prevalence of fibromyalgia: a population-based study in Olmsted County, Minnesota, utilizing the Rochester Epidemiology Project. Arthritis Care Res (Hoboken). 2013;65(5):786-92.
Robinson RL. Kroenke K, Mease P, Williams DA, Chen, Y, D’Souza D, Wohlreich, M, McCarberg, B. Burden of illness and treatment patters for patients with fibromyalgia. Pain Medicne, 2012; 13:1366-1376.

Posttraumatic Stress Disorder

Posttraumatic Stress Disorder (PTSD) is a serious chronic psychiatric illness defined as a maladaptive prolonged stress response which occurs after experiencing severely injurious traumatic event(s). It is a chronic and severely debilitating condition in which patients re-experience the horrific traumas that resulted in the condition in the forms of intrusive memories, flashbacks, and nightmares. Based on U.S. 12-month prevalence rates1,2, it is estimated that PTSD affects approximately 12 million Americans adults. PTSD typically is characterized by disrupted sleep, anxiety, agitation, avoidance, emotional numbness and estrangement from family and friends, guilt, or negative beliefs about self, and sometimes is associated with clinical depression and suicidal thinking. Individuals who suffer from PTSD usually have significant impairment in social functioning, occupational disability, and an overall poor quality of life. PTSD is sometimes associated with substance abuse and unpredictable violent or suicidal behaviors.

Goldstein RB, Smith SM, Chou SP, Saha TD, Jung J, Zhang H, Pickering RP, Ruan WJ, Huang B, Grant BF. The epidemiology of DSM-5 posttraumatic stress disorder in the United States: results from the National Epidemiologic Survey on Alcohol and Related Conditions-III. Soc Psychiatry Psychiatr Epidemiol. 2016;51(8):1137-1148.
Pietrzak RH, Goldstein RB, Southwick SM, Grant BF. Prevalence and Axis I comorbidity of full and partial posttraumatic stress disorder in the United States: results from Wave 2 of the National Epidemiologic Survey on Alcohol and Related Conditions. J Anxiety Disord. 2011;25(3):456-465.
IMS. Market Sizing and Treatment Dynamics: PTSD Patients. On file 2016.


Long COVID or Post-Acute Sequelae of COVID-19 (PASC), is a condition that survivors of COVID-19 infection experience at varying degrees. It is a chronic disabling condition that is expected to result in a significant global health and economic burden.1 The symptoms include intense fatigue, fevers, sleep problems, pain, and cognitive issues (“brain fog”). Post infection, many patients experience one or many of the symptoms of Long COVID: some patients have initial symptoms that become prolonged; others manifest entirely new syndromes that impact more than one system or organ. According to a recent publication in the Journal of American Medical Association (JAMA), over 1 in 10 healthcare workers who had recovered from COVID-19 were still coping with at least one moderate to severe symptom eight months later.2 Several cohort studies have reported that persistence of symptoms following SARS-CoV-2 infection occurs in more than 30% of patients.3 There is currently no approved drug for the treatment of Long COVID.

1 Briggs, Andrew, and Anna Vassall. “Count the cost of disability caused by COVID-19.” (2021): 502-505.
Symptoms and functional impairment assessed 8 Months after mild COVID-19 among health care workers. Havervall S, Rosell A, Phillipson M, Mangsbo SM, Nilsson P, Hober S, Thålin C. JAMA. 2021 Apr 7.
3 Nalbandian, Ani, et al. “Post-acute COVID-19 syndrome.” Nature Medicine (2021): 1-15.

Cocaine Intoxication

Cocaine is an illegal recreational drug which is taken for its pleasurable effects and associated euphoria. Pharmacologically, cocaine blocks the reuptake of the neurotransmitter dopamine from central nervous system synapses, resulting in the accumulation of dopamine within the synapse and an amplification of dopamine signaling and its role in creating positive feeling. With the continued use of cocaine, however, intense cocaine cravings occur resulting in a high potential for abuse and addiction (dependence), as well as the risk of cocaine intoxication. Cocaine intoxication refers to the deleterious effects on other parts of the body, especially those involving the cardiovascular system. Common symptoms of cocaine intoxication include tachyarrhythmias and elevated blood pressure, either of which can be life-threatening. As a result, individuals with known or suspected cocaine intoxication are sent immediately to the emergency department, preferably by ambulance in case cardiac arrest occurs during transit. There are approximately 505,000 emergency room visits for cocaine abuse each year in the U.S., of which 61,000 require detoxification services.

According to the National Institute on Drug Abuse, about 16,000 individuals died of cocaine overdose in 2019.According to a recent report by the U.S. Centers for Disease Control and Prevention2, and covered by news reports3,4, among all 2017 U.S. drug overdose deaths, approximately 20% involved cocaine. Overdose deaths involving cocaine increased 34 percent from 2016 to 2017.

1 Overdose Death Rates — National Institute on Drug Abuse — https://www.drugabuse.gov/related-topics/trends-statistics/overdose-death-rates
2 Kariisa, M, Scholl, L, Wilson, N, Seth, P, Hoots, B. Drug Overdose Deaths Involving Cocaine and Psychostimulants with Abuse Potential — United States, 2003–2017. MMWR Weekly / May 3, 2019 / 68(17);388–395 — https://www.cdc.gov/mmwr/volumes/68/wr/mm6817a3.htm?s_cid=mm6817a3_w; accessed May 11, 2019
3 Fottrell, Q. MarketWatch, Fatal drug overdoses involving cocaine and other stimulants have surged by over 52%8, May 3, 2019 — https://www.marketwatch.com/story/fatal-drug-overdoses-involving-cocaine-and-other-stimulants-have-surged-by-over-52-2019-05-03; accessed May 11, 2019
4 Cocaine deaths up in U.S. and opioids are a big part of it. Associated Press. https://www.msn.com/en-us/news/us/cocaine-deaths-up-in-us-and-opioids-are-a-big-part-of-it/ar-AAAOxs8?ocid=se; accessed May 11, 2019


Migraine is a neurological condition that manifests in throbbing headache, often on one side of the head, that lasts at least four hours. It can also be accompanied by nausea, vomiting, visual disturbances, and sensitivity to bright light, strong smells, and loud noises1. Epidemiological studies indicate that globally, approximately 1.2 billion individuals suffer from migraines annually.2 In the U.S., approximately 39 million Americans suffer from migraines and among these individuals, approximately four million experience chronic migraines (15 or more headache days per month).2

Depression/Major Depressive Disorder

Major Depressive Disorder (MDD) is a commonly occurring serious mood disorder that affects one’s thoughts, causing severe sadness that can hinder functioning of daily life. It is a condition characterized by symptoms such as a depressed mood or loss of interest or pleasure in daily activities most of the time for two weeks or more, accompanied by appetite changes, sleep disturbances, motor restlessness or retardation, loss of energy, feelings of worthlessness or excessive guilt, poor concentration, and suicidal thoughts and behaviors. These symptoms cause clinically significant distress or impairment in social, occupational, or other important areas of functioning. According to the National Institute of Mental Health, depression affects approximately 17 million adults in the U.S.1 have had at least one major depressive episode, with approximately 2.5 million adults treated with adjunctive therapy.2,3 The majority of people who suffer from depression do not respond adequately to initial antidepressant therapy.4

National Institute of Mental Health. (2017). Major Depression. Retrieved from http://www.nimh.nih.gov/health/statistics/major-depression.shtml
IMS NSP, NPA, NDTI MAT-24-month data through Aug 2017.
PLOS One, Characterization of Treatment Resistant Depression Episodes in a Cohort of Patients from a US Commercial Claims Database, Oct 2013, Vol 8, Issue 10.
Rush AJ, et al. (2007) Am J. Psychiatry 163:11, pp. 1905-1917 (STAR*D Study).

Agitation in Alzheimer’s Disease

Agitation in Alzheimer’s disease (AAD) is a common behavioral symptom experienced by a majority of patients with Alzheimer’s Disease. AAD, which includes emotional lability, restlessness, irritability, and agitation,1 is one of the most distressing and debilitating of the behavioral complications of Alzheimer’s disease. Both sleep disturbances and agitation are common and co-morbid features of Alzheimer’s disease.1,2,3 It is associated with significant negative consequences for both patients as well as their caregivers. Development of agitation, or its worsening, is one of the most common reasons for patients having to transition to nursing homes and other long-term care settings.

To date, there are no FDA approved treatment for behavioral symptoms such as agitation and aggression which affects the quality of life of both the patients and caregivers1.

Agitation is likely to affect more than half of the 5.3 million Americans who currently suffer from Alzheimer’s disease, and this number is expected to nearly triple by 2050.4 The presence of agitation nearly doubles the cost of caring for patients with Alzheimer’s disease, and agitation is estimated to account for more than 12 percent of the $256 billion in healthcare and societal cost of associated with Alzheimer’s disease for the year 2017 in the United States. 4

AAD is recognized by the FDA as a serious condition with an unmet medical need.

1 Canevelli M, Valletta M, Trebbastoni A, Sarli G, D’Antonio F, Tariciotti L, de Lena C, Bruno G. Sundowning in Dementia: Clinical Relevance, Pathophysiological Determinants, and Therapeutic Approaches. Front Med (Lausanne). 2016;3:73.
2 Rose K, Specht J, Forch W. Correlates among nocturnal agitation, sleep, and urinary incontinence in dementia. Am J Alzheimers Dis Other Demen. 2015;30(1):78-84.
3 Shih YH, Pai MC, Huang YC, Wang JJ. Sundown Syndrome, Sleep Quality, and Walking Among Community-Dwelling People With Alzheimer Disease. J Am Med Dir Assoc. 2017;18(5):396-401.
4 The Alzheimer’s Association. Alzheimer’s Disease Facts and Figures. https://www.alz.org/alzheimers-dementia/facts-figures.

Alcohol Use Disorder

Alcohol use disorder (AUD) is a medical condition characterized by an impaired ability to stop or control alcohol use despite adverse social, occupational, or health consequences. It encompasses the conditions that some people refer to as alcohol abuse, alcohol dependence, alcohol addiction, and the colloquial term, alcoholism. Considered a brain disorder, AUD can be mild, moderate, or severe. Lasting changes in the brain caused by alcohol misuse perpetuate AUD and make individuals vulnerable to relapse. According to a national survey, 14.1 million U.S. adults ages 18 and older1(5.6 percent of this age group2) had AUD in 2019. Among youth, an estimated 414,000 adolescents ages 12–171 (1.7 percent of this age group2) had AUD during this timeframe.

1 Substance Abuse and Mental Health Services Administration (SAMHSA), Center for Behavioral Health Statistics and Quality. 2019 National Survey on Drug Use and Health. Table 5.4A—Alcohol Use Disorder in Past Year Among Persons Aged 12 or Older, by Age Group and Demographic Characteristics: Numbers in Thousands, 2018 and 2019. https://www.samhsa.gov/data/sites/default/files/reports/rpt29394/NSDUHDetailedTabs2019/NSDUHDetTabsSect5pe2019.htm?s=5.4&#tab5-4a. Accessed November 6, 2020
2 SAMHSA, Center for Behavioral Health Statistics and Quality. 2019 National Survey on Drug Use and Health. Table 5.4B—Alcohol Use Disorder in Past Year Among Persons Aged 12 or Older, by Age Group and Demographic Characteristics: Percentages, 2018 and 2019. https://www.samhsa.gov/data/sites/default/files/reports/rpt29394/NSDUHDetailedTabs2019/NSDUHDetTabsSect5pe2019.htm?s=5.4&#tab5-4b