Tonix Prescription drugs Receives Uncommon Pediatric Illness Designation from the FDA for TNX-2900 for the Therapy of Prader-Willi Syndrome

CHATHAM, N.J., March 25, 2024 (GLOBE NEWSWIRE) — Tonix Prescription drugs Holding Corp. (Nasdaq: TNXP) (Tonix or the Firm), a biopharmaceutical firm with marketed merchandise and a pipeline of growth candidates, as we speak introduced the U.S. Meals and Drug Administration (FDA) has granted Uncommon Pediatric Illness Designation to TNX-2900* (intranasal potentiated oxytocin), a proprietary magnesium (Mg2⁺)-potentiated formulation of intranasal oxytocin, to deal with Prader-Willi syndrome (PWS) in kids and adolescents. TNX-2900 was beforehand granted Orphan Drug designation by the FDA in 2022 for the therapy of PWS and the investigational new drug (IND) utility was cleared by the FDA in 2023. The Firm could also be eligible to obtain a transferable Precedence Assessment Voucher if TNX-2900 for PWS is authorized for advertising. Not too long ago, vouchers have offered for roughly $100 million.

“The Uncommon Pediatric Illness Designation is a vital regulatory milestone within the growth of TNX-2900. With PWS being the commonest genetic reason behind life-threatening childhood weight problems, we’re excited that the FDA has acknowledged this vital unmet want in kids and adolescents, notably for PWS hyperphagia, which at the moment has no authorized therapies¹^,²,” mentioned Seth Lederman, M.D., Chief Govt Officer of Tonix Prescription drugs. “As PWS is a genetic dysfunction related to abnormalities of the oxytocin system, Tonix believes TNX-2900’s distinctive formulation has the potential to enhance intranasal oxytocin’s therapeutic motion by addressing limitations in efficacy noticed at high-dose intranasal oxytocin that’s not Mg2⁺-potentiated^3,4.”

The FDA defines a uncommon pediatric illness as a severe or life-threating illness that primarily impacts people aged from delivery to 18 years and impacts below 200,000 folks in the USA.

About FDA’s Uncommon Pediatric Illness Precedence Voucher Program

The FDA’s Uncommon Pediatric Illness Precedence Voucher Program is meant to encourage the event of latest medication to deal with sure uncommon pediatric ailments. Below the FDA’s Uncommon Pediatric Illness Designation and Voucher Program, if TNX-2900 is authorized for advertising, Tonix could qualify for a precedence evaluation voucher that may be redeemed to obtain precedence evaluation of a subsequent advertising utility for a unique product. Precedence evaluation vouchers may additionally be offered or transferred to a different sponsor. The brand new sponsor can redeem the voucher to obtain precedence evaluation for a unique product, which reduces the evaluation time of NDAs from 10 months to 6 months. There isn’t any restrict on the variety of instances a precedence evaluation voucher will be transferred. A 2020 U.S. Authorities Accounting Workplace evaluation⁵ of the voucher program discovered that within the ten years since launch of this system in 2009, the value of shopping for precedence evaluation vouchers ranged from $67 million to $350 million. Extra just lately, precedence evaluation vouchers had been acquired by Novo Nordisk for $110 million in June of 2022, and by Novartis for $100 million from Marinus in July of 2022.⁶ Bluebird Bio offered vouchers for $102 million, $95 million and $103 million in November 2022, January 2023, and October 2023, respectively.^7-9 In June of 2023, Novartis purchased a precedence evaluation voucher from Pharming for $21 million, a worth that had been negotiated as a part of a purchase order settlement when Pharming acquired the asset from Novartis.⁵

About Prader-Willi Syndrome (PWS)

PWS is acknowledged as the commonest genetic reason behind life-threatening childhood weight problems and impacts men and women with equal frequency and all races and ethnicities. PWS outcomes from the absence of expression of a gaggle of genes on the paternally acquired chromosome 15. The hallmarks of PWS are lack of suckling in newborns and, in kids and adolescents, extreme hyperphagia – an overriding physiological drive to eat, resulting in extreme weight problems and different problems related to vital mortality. A scientific evaluation of the morbidity and mortality as a consequence of hyperphagia in PWS discovered that the common age of dying in PWS was 22.1 years.¹⁰ There isn’t any authorized remedy to deal with poor feeding in newborns or hyperphagia in kids and adolescents with PWS. Given the intense or life-threatening manifestations of those situations, there’s a vital want for efficient therapies to lower morbidity and mortality, enhance high quality of life, and enhance life expectancy in folks with PWS. Oxytocin has potent results in correcting behavioral traits of the Magel2 knock-out mouse mannequin for PWS and autism.¹¹^-13 Six medical trials have investigated intranasal oxytocin as a therapy in pediatric sufferers with PWS. 4 research confirmed proof for enchancment in PWS-related behaviors/signs¹⁴^–¹⁷; three medical research reported proof for enchancment in hyperphagia¹⁴^,15,17; and one medical examine confirmed an enchancment in sucking in infants¹⁶.

About TNX-2900 and Tonix’s Potentiated Oxytocin Platform

TNX-2900 relies on Tonix’s patented intranasal Mg²⁺-potentiated oxytocin formulation supposed to be used by kids and adolescents. This formulation is believed to boost the efficiency of oxytocin in addition to enhance specificity for oxytocin receptors relative to vasopressin receptors, probably lowering undesirable unwanted effects from activating vasopressin receptors. Tonix can be growing a unique intranasal formulation, designated TNX-1900 for adolescent weight problems, binge consuming dysfunction, bone well being in autism, and social anxiousness dysfunction. Oxytocin is a naturally occurring human hormone that acts as a neurotransmitter within the mind. Oxytocin is believed to be greater than 600 million years outdated and is current in vertebrates together with mammals, birds, reptiles, amphibians, and fish.¹⁸ It was initially authorized by the U.S. Meals and Drug Administration as Pitocin®**, an intravenous infusion or intramuscular injection drug, to be used in pregnant girls to induce labor and management postpartum bleeding or hemorrhage. An intranasal formulation of oxytocin is marketed in some European international locations to help in breast milk manufacturing as Syntocinon®*** (oxytocin nasal 40 items/ml).

Tonix Prescription drugs Holding Corp.^*

Tonix is a biopharmaceutical firm centered on growing, licensing and commercializing therapeutics to deal with and forestall human illness and alleviate struggling. Tonix’s growth portfolio is targeted on central nervous system (CNS) issues. Tonix’s precedence is to submit a New Drug Software (NDA) to the FDA within the second half of 2024 for Tonmya, a product candidate for which two optimistic Section 3 research have been accomplished for the administration of fibromyalgia. TNX-102 SL can be being developed to deal with acute stress response in addition to fibromyalgia-type Lengthy COVID. Tonix’s CNS portfolio contains TNX-1300 (cocaine esterase) a biologic designed to deal with cocaine intoxication with Breakthrough Remedy designation. Tonix’s immunology growth portfolio consists of biologics to deal with organ transplant rejection, autoimmunity and most cancers, together with TNX-1500, which is a humanized monoclonal antibody focusing on CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the therapy of autoimmune ailments. Tonix additionally has product candidates in growth within the areas of uncommon illness and infectious illness. Tonix Medicines, our business subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the therapy of acute migraine with or with out aura in adults.

*Tonix’s product growth candidates are investigational new medication or biologics and haven’t been authorized for any indication. Tonmya™ is conditionally accepted by the U.S. Meals and Drug Administration because the tradename for TNX-102 SL for the administration of fibromyalgia.

**Pitocin® is a trademark of Par Pharmaceutical, Inc.

***Syntocinon® is a trademark of BGP Merchandise Operations GmbH

Citations

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U.S. Authorities Accounting Workplace, Jan 31, 2020. “Drug Growth: FDA’s Precedence Assessment Voucher Packages”. GAO-20-251. https://www.gao.gov/merchandise/gao-20-251.

Waldron, J. Fierce Biotech. June 1, 2023. “Novartis buys precedence evaluation voucher from Pharming for {discount} worth of $21 M.” www.fiercebiotech.com/biotech/novartis-buys-priority-review-voucher-pharming-discount-price-21m

BlueBird bio Press Launch. Nov. 30, 2022 “bluebird bio Sells Precedence Assessment Voucher for $102 Million”. https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-sells-priority-review-voucher-102-million

Kansteiner, F. Fierce Pharma. Jan 6, 2023 “Bluebird scores $95M nest egg after promoting second FDA precedence evaluation voucher to BMS”. http://www.fiercepharma.com/pharma/bluebird-scores-another-nest-egg-after-selling-second-fda-review-voucher-bms-95m

Enterprise Wire. October 30, 2023. “bluebird bio Enters into Advance Settlement to Promote Precedence Assessment Voucher, if Granted, for $103 Million.” http://finance.yahoo.com/information/bluebird-bio-enters-advance-agreement-120000768.html

Bellis SA, et al. Eur J Med Genet. 2022. 65(1):104379.

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Meziane H, et al. Biol Psychiatry. 2015. 78: 85-94.

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Gruber CW. Exp Physiol. 2014. 99(1):55-61. doi: 10.1113/expphysiol.2013.072561.

Zembrace SymTouch and Tosymra are registered logos of Tonix Medicines. All different marks are property of their respective house owners.

This press launch and additional details about Tonix will be discovered at www.tonixpharma.com.

Ahead Wanting Statements

Sure statements on this press launch are forward-looking inside the which means of the Personal Securities Litigation Reform Act of 1995. These statements could also be recognized by way of forward-looking phrases resembling “anticipate,” “imagine,” “forecast,” “estimate,” “count on,” and “intend,” amongst others. These forward-looking statements are primarily based on Tonix’s present expectations and precise outcomes might differ materially. There are a selection of things that would trigger precise occasions to vary materially from these indicated by such forward-looking statements. These components embrace, however should not restricted to, dangers associated to the failure to acquire FDA clearances or approvals and noncompliance with FDA rules; dangers associated to the failure to efficiently market any of our merchandise; dangers associated to the timing and progress of medical growth of our product candidates; our want for added financing; uncertainties of patent safety and litigation; uncertainties of presidency or third celebration payor reimbursement; restricted analysis and growth efforts and dependence upon third events; and substantial competitors. As with all pharmaceutical below growth, there are vital dangers within the growth, regulatory approval and commercialization of latest merchandise. Tonix doesn’t undertake an obligation to replace or revise any forward-looking assertion. Traders ought to learn the danger components set forth within the Annual Report on Type 10-Okay for the yr ended December 31, 2022, as filed with the Securities and Change Fee (the “SEC”) on March 13, 2023, and periodic stories filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly certified by all such danger components and different cautionary statements. The knowledge set forth herein speaks solely as of the date thereof.

Supply: Tonix Prescription drugs Holding Corp.

Posted: March 2024