Tonix Pharmaceuticals

Tonix Pharmaceuticals (Tonix Pharmaceuticals Holding Corp.) is a pharmaceutical company based in New York City that focuses on repurposed drugs for central nervous system conditions and as of 2020 was also pursuing a vaccine for COVID-19 and a biodefense project.[1]

Tonix Pharmaceuticals

Type	Public
Traded as	NASDAQ: TNXP
Industry	Biotechnology Healthcare
Headquarters	New York, New York , United States
Key people	Seth Lederman, MD – CEO
Website	www.tonixpharma.com

The company's predecessors were Tamandare Explorations Inc. which had been formed in 2007 as a mining wildcat vehicle focused on land in Nevada and the shares of which were traded over the counter, and L & L Technologies, LLC, which had been formed in 1996 by Seth Lederman and Donald Landry to repurpose drugs for CNS development. L&L had formed Janus Pharmaceuticals, Inc., which later became Vela Pharmaceuticals, Inc. to develop some of its inventions, and Vela returned those assets to L&L in 2006. L&L placed them in a subsidiary called Krele Pharmaceuticals, Inc., and that subsidiary and Tamandare Explorations Inc. performed a reverse merger in October 2011; the new entity was renamed Tonix Pharmaceuticals Holding Corp. and Lederman was named CEO.[2] It was listed on the NASDAQ exchange in 2013 under the symbol TNXP.[2]

The current lead candidates in the pipeline are the Phase 3 study of TNX-102 SL for the bedtime treatment of fibromyalgia, the Phase 2 study of TNX-1300 for cocaine intoxication and overdose and the preclinical study of TNX-1800, a potential coronavirus vaccine.[3]

In February 2020 Tonix began development of a potential vaccine to protect against COVID-19 with collaboration with Southern Research based on its live horsepox vaccine platform.[4] In May 2020 it announced an additional collaboration with the University of Alberta to develop more horsepox-based vaccines, TNX-1810, TNX-1820 and TNX-1830, partnering with FUJIFILM Diosynth Biotechnologies in College Station, TX, to manufacture the TNX-1800 vaccine candidate.[5]

In November 2019 Tonix announced the initiation of the Phase 3 study of TNX-102 SL for the management of fibromyalgia with interim analysis expected in September 2020 and topline data in the first quarter of 2021.[6] In January 2020, Tonix reported it has finished the essential fed-fasting and dose proportionality tests in support of a NDA.

Tonix continued work on TNX-102 SL in PTSD; the IND for that use had been accepted in 2014[7] and in December 2016 after the Phase IIa trial was done, TNX-102 was granted Breakthrough Therapy designation by the FDA in December 2017[8] which was rescinded in January 2019, but then restored in March 2019.[9] A Phase 3 trial of military-related PTSD began in February 2017, but was stopped in July 2018 after an interim analysis of 50% of target participants were evaluated, for "inadequate separation on primary efficacy endpoint".[10] In March 2020, a later Phase 3 trial of TNX-102 SL for PTSD in from mostly civilian traumas also showed futility at an interim analysis of 50% of its target participants patients were evaluated and enrollment was discontinued. FDA Breakthrough Therapy designation was rescinded again in May 2020.[11][12]

In July 2017 the news division of the journal, Science, reported that Tonix had sponsored research and collaborating with scientists at the University of Alberta, David Evans and Ryan Noyce, and that the work had led to the generation of an extinct horsepox virus using synthetic biology — the lab had bought pieces of DNA from a reagent company and had built the horsepox genome with them. This invention was licensed to Tonix and Tonix announced that it intended to further develop it into a smallpox vaccine, TNX-801, which is a biodefense business model.[13][14][15] The research was published in 2018.[16]

In May 2019, Tonix reported in-licensing the Phase 2 asset, TNX-1300 (T172R/G173Q double-mutant cocaine esterase 200 mg, i.v. solution), for the treatment of cocaine intoxication.[17][18] TNX-1300 (formerly known as RBP-8000) is being developed under an Investigational New Drug application (IND) for the treatment of cocaine intoxication.[19] 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 use cocaine as its sole source of carbon and nitrogen and that grow in soil surrounding coca plants.[20] The gene encoding CocE was identified and the protein was extensively characterized.[21][22][23] 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.[23] In a Phase 2 study, TNX-1300 at 100 mg or 200 mg i.v. doses was well tolerated and interrupted cocaine effects after cocaine 50 mg i.v. challenge.[19] TNX-1300 is designated as a breakthrough therapy by the U.S. Food and Drug Administration (FDA).

In September, 2019, Tonix licensed immuno-oncology treatment technology based on Trefoil Family Factor 2 (TFF2) from Columbia University.[24] A poster from that work was presented at the American Association of Cancer Research (AACR) meeting in June 2020.[25]

In December 2019, the Phase 1 trial for TNX-601 CR (tianeptine oxalate), an indirect glutamatergic pathway modulator, was completed and results were reported at the American Society of Clinical Psychopharmacology (ASCP) meeting in May 2020.[26]

In June 2020, Tonix acquired the migraine and pain treatment technologies of Trigemina for the TNX-1900 program based on a proprietary formulation of oxytocin.