Tonix Pharmaceuticals Licenses Technology for Treating Prader-Willi Syndrome, a Rare Genetic Eating Disorder, from the French National Institute of Health and Medical Research (Inserm) Expands Proprietary Uses of Tonix's

Tonix Pharmaceuticals Holding Corp. 8-K

Tonix Pharmaceuticals Licenses Technology

for Treating Prader-Willi Syndrome, a Rare Genetic Eating Disorder, from the French National Institute of Health and Medical Research

Expands Proprietary Uses of Tonix's

Potentiated Oxytocin for Intranasal Administration

Disorder Stunts Growth of Newborns and,

Paradoxically, Can Cause Excessive Hunger During Childhood and Beyond

CHATHAM, N.J., February 11, 2021 (GLOBE NEWSWIRE)

-- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today

announced an agreement whereby Tonix has licensed technology using oxytocin-based therapeutics for the treatment of Prader-Willi

syndrome and non-organic failure to thrive disease from Inserm. The licensing agreement has been negotiated and signed by Inserm

Transfert, the private subsidiary of Inserm, on behalf of Inserm (the French National Institute

of Health and Medical Research), Aix-Marseille Universit and Centre Hospitalier Universitaire of Toulouse.

The co-exclusive license allows Tonix to expand

its intranasal potentiated oxytocin development program to a new indication. The new program at Tonix has the designation TNX-2900

(intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. The patents covering the technology are expected

to provide market exclusivity for the co-licensees in the U.S. and Europe through 2031, which exclusivity could be extended after

marketing authorization by a Supplemental Protection Certificate in Europe or a Patent Term Extension in the U.S., independent

of other Tonix-held patents covering the formulation and oxytocin potentiation technologies for intranasal administration.

"Prader-Willi syndrome is a rare genetic

disorder of failure to thrive in infancy and uncontrolled appetite and obesity in childhood and adulthood with no approved treatments

available," said Tonix's President and Chief Executive Officer, Seth Lederman, M.D. "With the license from Inserm

Transfert , we have the opportunity to expand our ongoing efforts with intranasal potentiated oxytocin to this new indication.

Since Prader-Willi syndrome is an orphan disease that occurs in approximately one in 15,000 births, we plan at the appropriate

time to submit an application to the U.S. Food and Drug Administration for Orphan Drug and Fast Track designations for TNX-2900."

Prader-Willi syndrome results in physical,

mental and behavioral problems. A key feature of Prader-Willi syndrome in infants is a lack of suckling and poor muscle strength

which leads to malnutrition and failure to thrive. However, paradoxically in children and adults, the key feature of Prader-Willi

syndrome is a constant sense of hunger (hyperphagia), which leads to severe obesity. Intranasal oxytocin improves suckling in newborn

animals but also suppresses feeding behaviors in adult animal models. Tonix's patented potentiated oxytocin formulation is

believed to increase specificity for oxytocin receptors relative to vasopressin receptors as well as to enhance the potency of

About Prader-Willi Syndrome

Prader-Willi syndrome is recognized as the

most common genetic cause of life-threatening childhood obesity1 and affects males and females with equal frequency

and all races and ethnicities. The hallmarks of Prader-Willi syndrome are lack of suckling in infants and, in children and adults,

severe hyperphagia, an overriding physiological drive to eat, leading to severe obesity and other complications associated with

significant mortality. There is currently no approved treatment for either the suckling deficit in babies or the obesity and hyperphagia

in older children associated with Prader-Willi syndrome.

1Foundation for Prader-Willi Research

About TNX-2900* and Tonix's Potentiated Oxytocin

TNX-2900 is based on Tonix's patented

intranasal potentiated oxytocin formulation. Tonix is also developing a different intranasal formulation and device, designated

TNX-1900, for prophylaxis of chronic migraine and for the treatment of insulin resistance and related conditions. Oxytocin is a

naturally occurring human hormone that acts as a neurotransmitter in the brain. It was originally approved by the U.S. Food and

Drug Administration (FDA) as Pitocin **, an intravenous infusion or intramuscular injection drug, for use in pregnant women

to induce labor. An intranasal form of oxytocin was marketed in the U.S. by Novartis to assist in the production of breast milk

as Syntocinon *** (oxytocin nasal 40 units/ml), but the product was withdrawn, and the New Drug Application (NDA) has been

discontinued. TNX-2900 and TNX-1900 are in the pre-Investigational New Drug (IND) stage and have not been approved for any indication.

*TNX-2900 is an investigational new drug and has not been approved

**Pitocin is a trademark of Par Pharmaceutical, Inc.

***Syntocinon is a trademark of BGP Products Operations

About TNX-1900* (Intranasal Potentiated

TNX-1900 is based on a proprietary potentiated

formulation of oxytocin and is currently being developed as a candidate for prophylaxis of chronic migraine and for the treatment

of insulin resistance and related conditions.

TNX-1900 for Migraine: In clinical

and preliminary research, it has been observed that low oxytocin levels in the body can lead to increase in headache frequency,

and that increased oxytocin levels can relieve headaches. Oxytocin, when delivered via the nasal route, results in enhanced binding

of oxytocin to receptors on neurons in the trigeminal system, inhibiting transmission of pain signals. Intranasal oxytocin has

been well tolerated in several clinical trials in adults and children. Intranasal oxytocin has been shown to block calcitonin gene-related

peptide (CGRP) release in animals, a pathway known to be critical to the pathogenesis of migraine attacks. TNX-1900 is believed

to interrupt pain signals at the trigeminal ganglia by suppressing electrical impulses, a potentially different activity than drugs

that just block CGRP. Migraine attacks are caused, in part, by the release of CGRP from pain-sensing nerve cells that are part

of the trigeminal system. Targeted delivery results in low systemic exposure and lower risk of non-nervous system, off-target effects

which could potentially occur with systemic CGRP antagonists. For example, CGRP has roles in dilating blood vessels in response

to ischemia, including in the heart. Tonix believes targeted delivery of oxytocin could translate into selective blockade of CGRP

release in the trigeminal ganglion and not throughout the body, which could be a potential safety advantage over systemic CGRP

inhibition. TNX-1900 is also believed to provide augmented analgesia in the treatment of pain, relative to oxytocin.

TNX-1900 for Insulin Resistance:

Tonix recently acquired the exclusive license to develop TNX-1900 for the treatment of insulin resistance and related conditions

from the University of Geneva. The license allows Tonix to expand its intranasal potentiated

oxytocin development program into cardiometabolic syndromes, which include insulin resistance, impaired glucose tolerance, obesity,

diabetes management and related metabolic complications. The patents covering the

technology are expected to provide Tonix market exclusivity in the U.S. and Europe through 2031 which exclusivity could be extended

after marketing authorization by a Supplemental Protection Certificate in Europe or a Patent Term Extension in the U.S., independently

of other Tonix-held patents covering the formulation and potentiation technologies related to TNX-1900. The University of Geneva

technology is based on the discovery that oxytocin administration in an animal model of obesity improved lipid metabolism by increasing

lipolysis and fatty acid- -oxidation in adipose tissue accompanied by improvements in glucose intolerance and insulin resistance,

independent of food intake1. A number of studies have shown that intranasal oxytocin has effects on insulin resistance

and weight2-4,". Intranasal oxytocin has been reported to improve glucose

homeostasis, improve pancreatic -cell responsivity, decrease energy-induced and reward-induced eating, and support cognitive

control of food choices.2-9

*TNX-1900 is an investigational new drug and has not been approved

1Deblon N, et al. (2011) PLoS

ONE 6(9): e25565. doi:10.1371/journal.pone.0025565

Nat Rev Endocrinol. 13(12):700-709. doi: 10.1038/nrendo.2017.115. PMID: 28960210

et al. (2017) Curr Opin Endocrinol Diabetes Obes. 24(5):320-325. doi: 10.1097/MED.0000000000000351. PMID: 28590323.

(2019) Obes Rev. 2019 Jan;20(1):22-40. doi: 10.1111/obr.12757. PMID: 30253045.

5Lawson EA, et al. (2015) Obesity. 23:950-956.

DOI: 10.1002/oby.21069 PMID: 25865294

6Klement, J et al. (2017) Diabetes 66(2) 264-271; DOI: 10.2337/db16-0569

7Ott V, et al. (2013) Diabetes. 62:3418-3425.

DOI: 10.2337/db13-0663 PMID: 23835346

8Thienel M, et al. (2016) Int J Obes. 40(11):1707-1714.

DOI: 10.1038/ijo.2016.149 PMID:

9Striepens N, et al. (2016) Human Brain Mapp.

37(12):4276-4285. DOI: 10.1002/hbm.23308 PMID: 27381253

About Inserm Transfert

Inserm Transfert, the private subsidiary of the French National

Institute of the Health and Medical Research (Inserm), is responsible for value creation of Inserm innovations in human health