Tonix Pharmaceuticals Holding Corp. · NASDAQ:TNXP
Tonix Pharmaceuticals — Technical Presentation (September 2025)
20 slides from Tonix Pharmaceuticals's technical presentation, Sep 29, 2025
Slide 1: TNX-2900 Prader-Willi Syndrome
© 2025 Tonix Pharmaceuticals Holding Corp. Version P0545 September 29, 2025 TNX - 2900 Prader - Willi Syndrome NASDAQ: TNXPJH1
Slide 2: Cautionary Note on Forward-Looking Statements
2 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Cautionary Note on Forward - Looking Statements Certain statements in this presentation regarding strategic plans, expectations and objectives for future operations or results are “forward - looking statements” as defined by the Pri
Slide 3: TNX-2900: Prader-Willi Syndrome (PWS) Development Program Profile
3 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Patents Issued PROFILE DEVELOPMENT PROGRAM TNX - 2900*: Prader - Willi Syndrome (PWS) Intranasal Potentiated Oxytocin (OT) with Magnesium Prader - Willi Syndrome is the most common genetic cause of life - threatening childhood obesi
Slide 4: Prader-Willi Syndrome
4 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Prader - Willi Syndrome Cause Prevalence Symptoms a 65% of cases are due to a new deletion on paternal chromosome 15; first genetic imprinting disorder recognized in humans Diagnosis 1 in 10,000 to 1 in 30,000 1,2 ; most common synd
Slide 5: Progression of Prader-Willi Syndrome
5 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Progression of Prader - Willi Syndrome 1 0 yrs 0 to 9 months 9 to 25 months 2 to 4.5 years 4.5 to 8 years 8 years to adulthood Adulthood Average age at death: 22.1 years 2 Phase 0 Low birth weight Phase 1a Failure to thrive, feeding
Slide 6: Dangers of PWS Hyperphagia
6 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Dangers of PWS Hyperphagia Caretaker Burden 1 - 4 : • 24/7 supervision • Restricted food intake • Low - calorie diet • Locking cabinets and refrigerators Behaviors around food 1 - 4 : • Foraging or hoarding • Temper tantrums and mel
Slide 7: Abnormalities of the Oxytocin System in Patients with PWS
7 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Abnormalities of the Oxytocin System in Patients with PWS 1. Correa - da - Silva F, et al. J Neuroendocrinol . 2021;33(7):e12994. 2. Jurek B, et al. Physiol Rev . 2018;98(3):1805 - 1908. PWS patients have PVN=paraventricular nucleus
Slide 8: History of Oxytocin Use
8 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO History of Oxytocin Use 1. den Hertog CE, et al. Eur J Obstet Gynecol Reprod Biol . 2001;94(1):8 - 12. 2. Bakermans - Kranenburg MJ, et al. Transl Psychiatry . 2013;3(5):e258. 3. Oxytocin. Package insert. Hikma Pharmaceuticals USA I
Slide 9: Functions of Natural and Therapeutic Oxytocin
9 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Functions of Natural and Therapeutic Oxytocin 1. McCormack SE , et al. Endocr Rev. 2020;41(2):121 - 145. 2. Kuwabara Y, et al. Arch Gynecol Obstet . 1987;241(1):13 - 23. 3. Boie S, et al. Cochrane Database Syst Rev . 2018;8(8):CD012
Slide 10: Oxytocin Plays Major Role in Satiety
10 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Oxytocin Plays Major Role in Satiety 1 - 3 1. Correa - da - Silva F, et al. J Neuroendocrinol . 2021;33(7):e12994. 2. McCormack SE , et al. Endocr Rev. 2020;41(2):121 - 145. 3. Kerem L, et al. Int J Mol Sci. 2021;22(14):7737 . Oxyt
Slide 11: Intranasal Use of Oxytocin
11 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Intranasal Use of Oxytocin • Intranasal oxytocin was introduced as a lactation aid in the early 1960s 1 • Numerous studies have investigated chronic and acute intranasal oxytocin for the treatment of neuropsychiatric disorders and
Slide 12: Intranasal (IN) Oxytocin As PWS Treatment
12 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Intranasal (IN) Oxytocin As PWS Treatment 1. McCormack SE , et al. Endocr Rev. 2020;41(2):121 - 145. 2. Einfeld SL, et al. Am J Med Genet A. 2014;164A(9):2232 - 2239. 3. Kuppens RJ, et al. Clin Endocrinol ( Oxf ). 2016;85(6):979 -
Slide 13: Challenges in Intranasal Oxytocin Studies in PWS
13 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Challenges in Intranasal Oxytocin Studies in PWS 1. Quintana DS, et al. Mol Psychiatry. 2021;26(1):80 - 91. 2. Bharadwaj VN, et al. Pharmaceutics. 2022;14(5):1105. 3. Meyerowitz JG, et al. Nat Struct Mol Biol . 2022;29(3):274 - 281
Slide 14: Oxytocin Receptor (OXTR)
14 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Oxytocin Receptor (OXTR) 1 Jurek B, et al. Physiol Rev . 2018;98(3):1805 - 1908. 2 Meyerowitz JG, et al. Nat Struct Mol Biol. 2022;29(3):274 - 281. OXTR Signaling Cascade OXTR G q /11 Phospho - lipase C Ca 2+ Protein synthesis Unkn
Slide 15: Oxytocin+Mg2+ Activates OXTR Secondary Messengers
15 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Oxytocin+Mg 2+ Activates OXTR Secondary Messengers Magnesium is needed not only for oxytocin binding to OXTR but also for OXTR activation BRET=bioluminescence resonance energy transfer; EDTA= etheylenediaminetetraacetic acid; HEK=h
Slide 16: Oxytocin Requires Magnesium for Receptor Binding
16 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Oxytocin Requires Magnesium for Receptor Binding • OXTR exists in 2 conformational states 1 : • Low affinity • High affinity • Magnesium ions are necessary for the high - affinity state 1,2 • Without magnesium ions present, oxytoci
Slide 17: Phase 2 Study to Investigate TNX-2900 for Hyperphagia in PWS
17 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Phase 2 study to investigate TNX - 2900 for the treatment of hyperphagia in PWS • FDA has cleared the IND for a Phase 2 randomized, double - blind, placebo - controlled, parallel design study to evaluate the safety, tolerability, a
Slide 18: Addition of Mg2+ Potentially Expands Useful Dose Range of Intranasal Oxytocin
18 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Addition of Mg 2+ Potentially Expands the in vivo Useful Dose Range of Intranasal Oxytocin in Animals Bharadwaj VN, et al. Pharmaceutics . 2022;14(5):1105. • A nonlinear dose response has been demonstrated in the use of intranasal
Slide 19: Highlights
19 © 2025 Tonix Pharmaceuticals Holding Corp. RARE DISEASE PORTFOLIO Highlights • Hyperphagia in Prader - Willi syndrome (PWS) is severe and life - threatening. • There is a high unmet need for safe and effective treatments for hyperphagia in PWS. • Oxytocin is one of the hormones responsible for si
Slide 20: Thank You
© 2025 Tonix Pharmaceuticals Holding Corp. THANK YOU
Tonix Pharmaceuticals Holding — Technical Presentation (September 2025) on 6ix