Full Press Release Details
Tonix Pharmaceuticals Holding
Tonix Pharmaceuticals Receives Federal Grant from
the National Institute on Drug Abuse (NIDA) to Advance Development of TNX-1300 as a Treatment for Cocaine Intoxication
There is No FDA-Approved Product for Cocaine Intoxication
TNX-1300 Has Been Granted Breakthrough Therapy
Designation by the FDA
Phase 2 Single-Blind, Placebo-Controlled,
Potential Pivotal Study Expected to Start in Fourth Quarter 2022, Pending FDA Agreement
CHATHAM, N.J., August 2, 2022 (GLOBE NEWSWIRE)
- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today
announced that it has received a Cooperative Agreement grant from the National Institute on Drug Abuse (NIDA), part of the National Institutes
of Health (NIH), to support development of TNX-1300 (T172R/G173Q double-mutant cocaine esterase 200 mg, i.v. solution) for the
treatment of cocaine intoxication. TNX-1300 is a recombinant enzyme that efficiently degrades and metabolizes cocaine. Cocaine intoxication
refers to a state in which cocaine has deleterious effects on several body systems, especially the cardiovascular system. TNX-1300 demonstrated
activity on reversing the physiological effects of i.v. cocaine challenge in people who use cocaine in a prior Phase 2a randomized,
double-blind, placebo-controlled clinical study.1
The grant is intended to support continued development of TNX-1300 as
a treatment for life threatening cocaine intoxication. In 2021, more than 24,900 individuals in the U.S. died from drug overdose deaths
"This grant award underscores the unmet
need for safe and effective treatments for cocaine intoxication and validates the progress we have achieved to date with TNX-1300,"
said Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals. "Cocaine intoxication remains a serious
issue in the U.S. where there is currently no specific pharmacotherapy indicated treatments. By targeting the cause rather than the symptoms
of cocaine intoxication, we believe TNX-1300 may offer significant advantages to the current standard of care for cocaine intoxication."
Tonix recently announced the design of a new
single-blind, open-label, placebo-controlled, randomized Phase 2 clinical trial of TNX-1300 for the treatment of cocaine intoxication.
The Phase 2 study, which has the potential to serve as a pivotal trial, is anticipated to start in the fourth quarter of 2022, pending
U.S. Food and Drug Administration (FDA) agreement. TNX-1300 has been granted Breakthrough Therapy designation by the FDA. As a biologic
and new molecular entity, TNX-1300 is eligible for 12 years of U.S. market exclusivity upon approval by the FDA, in addition to expected
patent protection through 2029.
"The research Tonix is pursuing is a
bright light in our shared goal of reducing overdose deaths and harm as we continue to battle the crisis in substance use disorders in
New Jersey and across the country, which has only been compounded by the pandemic. This targeted treatment could bring down healthcare
costs and, most importantly, loss of life due to cocaine overdose," said Representative Mikie Sherrill (NJ-11). "With this
federal grant, Tonix will be able to move one step closer to FDA authorization and getting this potentially life-saving treatment into
the hands of emergency room doctors and nurses, as well as EMS and other first responders. I am proud to have Tonix's headquarters
based here in NJ-11."
"Tonix has been an incredible partner and job creator in Maryland,
and I commend their efforts in fighting against the substance use disorder crisis that our nation continues to face. With over 100,000
Americans killed by drug overdoses just last year, we need to work together to curb the loss and set our sights on prevention, harm reduction,
treatment and recovery. That starts with medical innovation in our own communities and reliable investment in our country's brightest
leaders. This funding from the National Institute on Drug Abuse will do just that," said Representative David Trone (MD-06).
Research reported in this press release was
supported by the National Institute on Drug Abuse of the National Institutes of Health under award number U01DA056245. The content is
solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
TNX-1300 (T172R/G173Q double-mutant cocaine
esterase 200 mg, i.v. solution) is being developed under an Investigational New Drug (IND) application for the treatment of cocaine
intoxication. TNX-1300 is a recombinant protein enzyme produced through rDNA technology in a non-disease-producing strain of E.
coli bacteria. Cocaine esterase (CocE) was identified in bacteria (Rhodococcus) that uses cocaine as its sole source
of carbon and nitrogen and that grows in soil surrounding coca plants.3 The gene encoding CocE was identified and the
protein was extensively characterized.3-6 CocE catalyzes the breakdown of cocaine into metabolite ecgonine methyl ester
and benzoic acid. Wild-type CocE is unstable at body temperature, so targeted mutations were introduced in the CocE gene and resulted
in the T172R/G173Q double-mutant CocE, which is active for approximately 6 hours at body temperature.6 In a Phase 2
study, TNX-1300, at 100 mg or 200 mg i.v. doses, was well tolerated and rapidly reduced cocaine effects after a cocaine 50 mg
About Cocaine Intoxication
Cocaine is an illegal recreational drug which
is taken for its pleasurable effects and associated euphoria, as well as mental alertness, in some cases. 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 which reinforces the drug taking. With the continued use of cocaine, however,
intense cocaine cravings can occur, resulting in a high potential for continued use and addiction, as well as the risk of cocaine intoxication.
Cocaine intoxication refers to the deleterious effects of cocaine on several body systems, 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. According to the U.S. Centers for Disease Control and Prevention (CDC), in 2021
the number of overdose death involving cocaine reached 24,900 individuals.2 Also according to a recent report by the CDC,
among all 2020 U.S. drug overdose deaths, approximately nearly 1 in 5 involved cocaine.7 In 2020, Black Americans experienced
the highest death rate for overdoses involving cocaine, at 14 per 100,000.7
C. A randomized, double-blind, placebo-controlled trial of RBP-8000 in cocaine abusers:
pharmacokinetic profile of rbp-8000 and cocaine and effects of RBP-8000 on cocaine-induced physiological effects. J
Addict Dis. 2014;33(4):289-302.
2 Centers for Disease Control
and Prevention (CDC) - https://www.cdc.gov/nchs/nvss/vsrr/drug-overdose-data.htm
NC. Gene cloning and nucleotide sequencing and properties of a cocaine esterase from
Rhodococcus sp. strain MB1. Appl
Environ Microbiol. 2000. 66(3):904-8.
IA. Crystal structure of a bacterial cocaine esterase. Nat
Struct Biol. 2002. 9(1):17-21.
RA. Biochemical characterization and structural analysis of a highly proficient cocaine
esterase. Biochemistry.
2002. 41(41):12297-307.
RK, Zhan CG. Thermostable
variants of cocaine esterase for long-time protection against cocaine toxicity. Mol
Pharmacol. 2009. 75(2):318-23.
7 National Institute on
About Tonix Pharmaceuticals Holding
Tonix is a clinical-stage
biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease
and alleviate suffering. Tonix's portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious
disease product candidates. Tonix's CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric
and addiction conditions. Tonix's lead CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development
for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022 and interim data expected in the first
quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate
a Phase 2 study in Long COVID in the third quarter of 2022. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication
that is mid-Phase 2 with a new potentially pivotal Phase 2 study expected to be initiated in the fourth quarter of 2022. TNX-1300 has
been granted Breakthrough Therapy Designation by the FDA. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development
for chronic migraine, is expected to enter the clinic with a Phase 2 study in the second half of 2022. Tonix's rare disease portfolio
includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug
Designation by the FDA. Tonix's immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and
cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft
and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the