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.
“Orphan-Drug 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.”
As 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
Université 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.
The 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.
*TNX-2900 is an investigational new drug and has
not been approved for any indication.
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
(fpwr.org).
About TNX-2900 and Tonix’s Potentiated
Oxytocin Platform
TNX-2900 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.
*Pitocin® is a trademark of Par Pharmaceutical,
Inc.
**Syntocinon® is a trademark of BGP Products
Operations GmbH.
About Tonix Pharmaceuticals Holding
Corp.
Tonix 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.
1TNX-1500 is an investigational new biologic and
has not been approved for any indication.
2TNX-1800 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.
3TNX-3500 is an investigational new drug at the
pre-IND stage of development and has not been approved for any
indication.
4TNX-102 SL is an investigational new drug and
has not been approved for any indication.
5TNX-1300 is an investigational new biologic and
has not been approved for any indication.
This press release and further information about
Tonix can be found at www.tonixpharma.com.
Forward-Looking Statements
Certain 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.
Contacts
Jessica Morris (corporate)Tonix
Pharmaceuticalsinvestor.relations@tonixpharma.com(212) 688-9421
Olipriya Das, Ph.D. (media)Russo
Partnersolipriya.das@russopartnersllc.com (646) 942-5588
Peter Vozzo (investors)ICR
Westwickepeter.vozzo@westwicke.com (443) 213-0505
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