Full Press Release Details
Tonix Pharmaceuticals Holding Corp. 8-K
Pharmaceuticals Announces FDA Orphan-Drug Designation for TNX-2900 for the Treatment of Prader-Willi Syndrome
N.J., March 3, 2022 (GLOBE NEWSWIRE) - Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage
biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan-Drug Designation for TNX-2900*
(intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome.
Designation by the FDA is an important milestone and further validates our efforts to investigate the utility of TNX-2900 for Prader-Willi
syndrome," said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. "It underscores the urgent, unmet
medical need for patients diagnosed with this disease, and will benefit us as we continue to advance our program."
recently announced, Tonix entered into a sponsored research agreement with Inserm Transfert, the private subsidiary of Inserm, on behalf
of Inserm (the French National Institute of Health and Medical Research) and Aix-Marseille Universit to study oxytocin in the
genetically engineered mouse model of Prader-Willi syndrome, a rare genetic disorder that causes distinct, but related pathological eating
disorders in adults and newborns. In adults, Prader-Willi causes hyperphagia, or pathological over-eating, which leads to obesity and
other complications associated with significant mortality. In newborns, Prader-Willi causes a deficiency in suckling, which has been
shown to be normalized by oxytocin treatment.
FDA's Office of Orphan Drug Products grants orphan status to the active moiety of drugs and biologics that demonstrate promise
for the treatment of diseases or conditions affecting fewer than 200,000 people in the United States. Orphan drug designation provides
Tonix Pharmaceuticals with certain development incentives, including tax credits for qualified clinical testing, exemptions from certain
FDA application fees, and potential market exclusivity for seven years, if approved.
is an investigational new drug and has not been approved for any indication.
Prader-Willi Syndrome
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.
for Prader-Willi Research (fpwr.org).
TNX-2900 and Tonix's Potentiated Oxytocin Platform
is based on Tonix's patented intranasal potentiated oxytocin formulation intended for use by adults and adolescents. 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 oxytocin. 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 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 discontinued, and the New Drug Application was withdrawn.
is a trademark of Par Pharmaceutical, Inc.
is a trademark of BGP Products Operations GmbH
Tonix Pharmaceuticals Holding Corp.
is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics
to treat and prevent human disease and alleviate suffering. Tonix's portfolio is composed of immunology, infectious disease, and
central nervous system (CNS) product candidates. Tonix's immunology portfolio includes biologics to address organ transplant rejection,
autoimmunity and cancer, including TNX-15001 which is a humanized monoclonal antibody targeting CD40 ligand being developed
for the prevention of allograft rejection treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in
the second half of 2022. Tonix's infectious disease pipeline includes next-generation vaccines to prevent COVID-19, an antiviral
to treat COVID-19, and a potential treatment for Long COVID. The pipeline also includes a vaccine in development to prevent smallpox.
Tonix's lead vaccine candidate for COVID-19, TNX-18002, is a live virus vaccine based on Tonix's recombinant pox
vaccine (RPV) platform. TNX-35003 (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute
COVID-19 and is in the pre-IND stage of development. TNX-102 SL4 , (cyclobenzaprine HCl sublingual tablets), is a small
molecule drug being developed to treat Long COVID, a chronic post-COVID condition. Tonix expects to initiate a Phase 2 study in Long
COVID in the first half of 2022. The Company'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, is in mid-Phase 3 development for the management
of fibromyalgia with a new Phase 3 study expected to start in the first half of 2022. Finally, TNX-13005 is a biologic designed
to treat cocaine intoxication that is expected to start a Phase 2 trial in the first quarter of 2022.
is an investigational new biologic and has not been approved for any indication.
is an investigational new biologic and has not been approved for any indication. TNX-1800 is based on TNX-801, live horsepox
virus vaccine for percutaneous administration, which is in development to protect against smallpox and monkeypox. TNX-801 is an investigational
new biologic and has not been approved for any indication.
is an investigational new drug at the pre-IND stage of development and has not been approved for any indication.
SL is an investigational new drug and has not been approved for any indication.
is an investigational new biologic and has not been approved for any indication.
press release and further information about Tonix can be found at www.tonixpharma.com.
statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These
statements may be identified by the use of forward-looking words such as "anticipate," "believe," "forecast,"
"estimate," "expect," and "intend," among others. These forward-looking statements are based on Tonix's
current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ
materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the
development of TNX-2900, the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties
caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our
need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement;
limited research and development efforts and dependence upon third parties; and substantial
competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization
of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the
risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2020, as filed with the Securities and Exchange
Commission (the "SEC") on March 15, 2021, and periodic reports filed with the SEC on or after the date thereof. All of Tonix's
forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth
herein speaks only as of the date thereof.