Full Press Release Details
Tonix Pharmaceuticals Holding Corp. 8-K
Tonix Pharmaceuticals Completes Clinical
Stage of Phase 1 Trial for TNX-1500
(Fc-modified humanized anti-CD40L mAb) in Healthy Volunteers
results expected in the third quarter of 2024; planning a Phase 2 Trial for prevention of kidney transplant rejection
has multiple possible indications in addition to solid organ and bone marrow transplantation including autoimmune diseases: potential
pipeline in a product
recently published results on their Fc-modified humanized anti-CD40L mAb, frexalimab, for multiple sclerosis in the New England Journal
CHATHAM, N.J., February 28, 2024 (GLOBE
NEWSWIRE) - Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a
biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the completion of the
clinical stage of its Phase 1, single ascending dose escalation trial of TNX-1500 (Fc-modified humanized anti-CD40L monoclonal antibody,
or mAb)* in healthy volunteers. TNX-1500 is in development for the prevention of rejection in solid organ and bone marrow transplant and
for the treatment of autoimmune disorders.
"Despite advancements in the field
of solid organ transplantation, there remains a significant need for new treatments with improved activity and tolerability," said
Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. "Anti-CD40L modulates T cell function and has the potential
to promote tolerance of transplanted organs. We are excited to have completed the clinical stage of this Phase 1 trial of TNX-1500, a
third-generation Fc-modified anti-CD40L mAb that has been designed by protein engineering to eliminate the risk of thrombosis associated
with first-generation anti-CD40L mAbs. Preclinical studies in non-human primates did not result in any thrombotic complications, suggesting
that the protein engineering of TNX-1500's Fc region has achieved its design goals."
Dr. Lederman continued, "Recently,
positive clinical data with other CD40L blockers have been reported by Sanofi, with its Fc-modified humanized anti-CD40L mAb frexalimab
in treating relapsing multiple sclerosis.2 Eledon Pharmaceuticals is developing tegoprubart, a non-covalent dimer antibody
with no heavy-light or heavy-heavy interchain disulfide bridges for the prevention of rejection of kidney transplants3."
Dr. Lederman concluded, "We believe
TNX-1500 has the potential to prevent organ transplant rejection and improve graft survival with reduced long-term toxicity burden of
current immunosuppressive regimens. In addition, TNX-1500 has the potential to address multiple other indications, including several autoimmune
diseases. We look forward to the results of this Phase 1 trial, which are expected in the third quarter of this year, and to continuing
the development of TNX-1500 as a promising candidate in an important therapeutic space."
TNX-1500 (Fc-modified humanized anti-CD40L
mAb) is a humanized monoclonal antibody that interacts with the CD40-ligand (CD40L), also known as CD154. TNX-1500 is being developed
for the prevention of allograft and xenograft rejection, for the prevention of graft-versus-host disease (GvHD) after hematopoietic stem
cell transplantation (HCT) and for the treatment of autoimmune diseases. The first-in-human Phase 1 trial of TNX-1500 was initiated in
the third quarter of 2023. The primary objective of the Phase 1 trial is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics
of intravenous (i.v.) TNX-1500. Eligible participants enrolled in the Phase 1 trial were distributed across three dosing cohorts
(3 mg/kg, 10 mg/kg, and 30 mg/kg, respectively) and evaluated regularly over a 120-day period after dosing. This first-in-human trial
is intended to support dosing in a planned Phase 2 trial in kidney transplant recipients. Two published articles in the American Journal
of Transplantation demonstrate TNX-1500 prevents rejection, prolongs survival and preserves graft function as a single agent or in
combination with other drugs in non-human primate renal and heart allografts.4,5
About anti-CD40L Therapeutics in Development
No anti-CD40L mAb has been approved in any
jurisdiction. In addition to TNX-1500, frexalimab and tegoprubart, tn03 fusion protein dazodalibep is being developed by Amgen (formerly
Horizon Therapeutics Public Limited Company) for the treatment of Sj gren's Syndrome .6,7 Dapirolizumab pegol, an anti-CD40L
pegylated Fab, is being developed by UCB for the treatment of systemic lupus erythematosus.8
*TNX-1500 is an investigational new biologic and
is not approved for any indication
Tonix Pharmaceuticals Holding Corp.*
Tonix is a biopharmaceutical company focused on developing,
licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix's development portfolio
is focused on central nervous system (CNS) disorders. Tonix's priority is to submit a New Drug Application (NDA) to the FDA in the
second half of 2024 for Tonmya1, a product candidate for which two positive Phase 3 studies have been completed for the management
of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction as well as fibromyalgia-type Long COVID. Tonix's
CNS portfolio includes TNX-1300 (cocaine esterase) a biologic designed to treat cocaine intoxication with Breakthrough Therapy designation.
Tonix's immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including
TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft
rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease
and infectious disease. Tonix Medicines, our commercial subsidiary, markets Zembrace SymTouch (sumatriptan injection) 3 mg and
Tosymra (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
*Tonix's product development candidates are
investigational new drugs or biologics and have not been approved for any indication.
1Tonmya is conditionally accepted
by the U.S. Food and Drug Administration (FDA) as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been
approved for any indication.
Zembrace SymTouch and Tosymra are registered trademarks
of Tonix Medicines. All other marks are property of their respective owners.
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 failure to obtain FDA
clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products;
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, 2022, as filed with the Securities and Exchange Commission (the "SEC") on March 13, 2023, 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.