Topline Results from Four CNS Clinical Trials Expected in the Third and Fourth Quarters of 2023
Fibromyalgia Topline Results Expected in the Fourth Quarter of 2023 for Phase 3 Potentially NDA-Enabling Study of the Centrally-Acting Non-Opioid Analgesic TNX-102 SL
Completed Strategic Acquisition of Two Marketed Acute Migraine Products: Zembrace® SymTouch® (sumatriptan injection) and Tosymra® (sumatriptan nasal spray)
“We have recently completed enrollment in four clinical trials for central nervous system (CNS) indications, representing our continuing progress towards bringing forth new treatments for serious CNS conditions that affect large patient populations,” said
On
“We plan to submit an IND to study TNX-2900 (intranasal potentiated oxytocin) in Prader-Willi syndrome, for which Orphan Drug Designation has already been granted by the FDA,” said
Recent Highlights—Marketed Products
On
Recent Highlights—Key Product Candidates*
Upcoming Data Readouts in Central Nervous System (CNS) Pipeline During 2023
Fibromyalgia (4Q): Phase 3 Potentially NDA-Enabling Study of TNX-102 SL
Fibromyalgia-Type Long COVID (3Q): Phase 2 Proof-of-Concept Study of TNX-102 SL
Chronic Migraine (4Q): Phase 2 Proof-of-Concept Study of TNX-1900
Depression (4Q): Phase 2 Proof-of-Concept Study of TNX-601 ER
Central Nervous System (CNS) Pipeline
TNX-102 SL (cyclobenzaprine HCl sublingual tablets): once-daily at bedtime small molecule for the management of fibromyalgia (FM) – centrally-acting, non-opioid analgesic.
- In
August 2023 , the Company announced that it completed enrollment of its potentially confirmatory Phase 3 RESILIENT trial of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) 5.6 mg in fibromyalgia. A total of 457 participants were randomized in the trial, which, if successful, may serve as the final, well-controlled efficacy trial required for submission of an NDA for approval by the FDA. RESILIENT is a registration-quality, double-blind, placebo-controlled study. - Topline results from the RESILIENT trial are expected in the fourth quarter of 2023
TNX-102 SL for the treatment of Fibromyalgia-Type Long COVID, also known as Post-Acute Sequelae of COVID-19 (PASC)
- In
August 2023 , the Company announced it completed the clinical phase of the PREVAIL study, a Phase 2 proof-of-concept study of TNX-102 SL for fibromyalgia-type Long COVID, as the last patient completed their final study visit. PREVAIL is a registration-quality, double-blind, placebo-controlled study. - Topline results from the PREVAIL Phase 2 trial are expected in the third quarter of 2023.
TNX-1900 (intranasal potentiated oxytocin): small peptide for migraine, craniofacial pain, social anxiety disorder, insulin resistance and related disorders, and adolescent obesity and binge eating disorder
- Tonix has developed a formulation of intranasal oxytocin that contains magnesium (Mg2+) which is believed to potentiate the effects of oxytocin and may address the “inverted U” dose response of oxytocin. The company believes that if Mg2+ reduces the high dose inhibition of oxytocin alone, then the new formulation may improve consistency in clinical trials.
- Enrollment has been completed in the proof-of-concept Phase 2 PREVENTION study of TNX-1900 for the prevention of migraine headache in chronic migraineurs with a total of 88 patients enrolled. PREVENTION is a registration-quality, double-blind, placebo-controlled study.
- Topline results from the PREVENTION Phase 3 trial are expected in the fourth quarter of 2023.
- In
July 2023 , Tonix announced that the first participant was enrolled in the investigator-initiated Phase 2 STROBE Study of TNX-1900 for the treatment of binge-eating disorder at theMassachusetts General Hospital (MGH) under the direction of Principal InvestigatorElizabeth Lawson . Tonix is supporting the STROBE study through a clinical trial agreement with MGH. - In
July 2023 , Tonix announced that the first participant was enrolled in a Phase 2 investigator-initiated, proof-of-concept study of TNX-1900 for enhancing social safety learning in social anxiety disorder (SAD) under the direction of Principal InvestigatorAngela Fang . Tonix entered into an agreement with theUniversity of Washington to examine the potential role of TNX-1900 in enhancing vicarious extinction learning in SAD, compared to healthy controls. - In
July 2023 , Tonix announced that the first participant was enrolled in the Phase 2 POWER study of TNX-1900 for the treatment of pediatric obesity with MGH under the direction of Principal InvestigatorElizabeth Lawson . MGH is the sponsor of theNational Institutes of Health-funded trial, being conducted under an investigator-initiated IND. - In
May 2023 , Tonix entered into a research collaboration agreement with the principal investigator of the study, Dr.Antoinette Maassen van den Brink , Professor of Neurovascular Pharmacology,Erasmus University Medical Center , to evaluate the effect of TNX-1900 on capsaicin- or electrical stimulation-induced forehead dermal blood flow in health female volunteers.
TNX-601 ER (tianeptine hemioxalate extended-release tablets): a once-daily orally administered small molecule for the treatment of major depressive disorder (MDD), posttraumatic stress disorder (PTSD), neurocognitive dysfunction associated with corticosteroid use, and potentially Alzheimer’s disease
- Enrollment has been completed in the proof-of-concept Phase 2 UPLIFT study for the treatment of MDD, with 116 patients enrolled. UPLIFT is a registration-quality, double-blind, placebo-controlled study.
- Topline results for the UPLIFT Phase 2 trial are expected in the fourth quarter of 2023.
- Scientists at Tonix’s
Research and Development Center (RDC) inFrederick, Md. established the molecular mechanism of action of tianeptine, the active ingredient of TNX-601 ER. The research supports a direct role for restoring neuroplasticity and neurogenesis, and upsets previously held beliefs about the significance of neurotransmitters in treating depression. It also provides clarity on why tianeptine does not cause sexual dysfunction, weight gain or several other treatment-limiting toxicities associated with traditional antidepressants.
TNX-4300 (estianeptine): small molecule oral therapeutic for MDD, bipolar disorder, Alzheimer’s disease and Parkinson’s disease
- In
July 2023 , the Company announced that it would focus resources on the development of single isomer TNX-4300 as a first-in-class oral therapy for mood disorders, Alzheimer’s disease and other psychiatric and neurodegenerative conditions with memory deficits. The decision follows the Company’s announcement inMay 2023 of the isolation and characterization of the (S)-isomer of tianeptine, which activates PPAR-β/δ, restores neuroplasticity in neuronal tissue culture and is free of µ-opioid receptor activity.
TNX-1300 (recombinant double mutant cocaine esterase): biologic for life-threatening cocaine intoxication
- Tonix expects to initiate a potentially pivotal Phase 2 clinical study of TNX-1300 for the treatment of cocaine intoxication in the third quarter of 2023. In 2022, Tonix was awarded a Cooperative Agreement grant from the
National Institute on Drug Abuse (NIDA), part of theNational Institutes of Health (NIH), to support development of TNX-1300. - TNX-1300 has been granted Breakthrough Therapy designation by the FDA.
Rare Disease Pipeline
TNX-2900 (intranasal potentiated oxytocin): small peptide for the treatment of Prader-Willi syndrome (PWS)
- TNX-2900 has been granted Orphan Drug designation from the FDA for the treatment of PWS and the preparation of the IND is in process.
- TNX-2900 is in development to treat PWS and the planned clinical study will study its effects on hyperphagia, or pathological over-eating, in children and young adult patients with PWS.
Immunology Pipeline
TNX-1500 (anti-CD40L monoclonal antibody): third generation anti-CD40L monoclonal antibody for prophylaxis of organ transplant rejection and treatment of autoimmune disorders.
- In
May 2023 , the IND for prevention of organ rejection in patients receiving a kidney transplant was cleared by FDA. A first-in-human Phase 1 study is expected to start in the third quarter of 2023. The first indication for TNX-1500 will be prophylaxis of organ rejection in adult patients receiving a kidney transplant, but multiple additional indications are possible, including autoimmune diseases. Two peer reviewed publications described the work at theMassachusetts General Hospital on allogeneic transplants in animals.1,2
*All of Tonix’s product candidates are investigational new drugs or biologics and none have been approved for any indication.
1Lassiter, G., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate renal allograft survival.
2Miura, S., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate cardiac allograft survival.
Recent Highlights—Financial
As of
The acquisition cost of Zembrace SymTouch and Tosymra was
On
On
Second Quarter 2023 Financial Results
R&D expenses for the second quarter 2023 were approximately
G&A expenses for the second quarter 2023 were
Net loss available to common stockholders was
Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia, having completed enrollment in a potentially registration-enabling study, and with topline data expected in the fourth quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 study has been completed, and topline results are expected in the third quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), is a once-daily formulation being developed as a treatment for major depressive disorder (MDD). Enrollment is now complete in the UPLIFT trial of TNX-601 ER in MDD and topline results are expected in the fourth quarter of 2023. TNX-4300 (estianeptine) is a small molecule oral therapeutic in preclinical development to treat MDD, Alzheimer’s disease and Parkinson’s disease. TNX-1900 (intranasal potentiated oxytocin), is in development for chronic migraine, and the PREVENTION study has completed enrollment with topline data expected in the fourth quarter of 2023. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the third quarter of 2023. Tonix’s rare disease development portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology development portfolio includes 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. A Phase 1 study of TNX-1500 is expected to be initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, classes of broad-spectrum small molecule antivirals.
*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.
Tonix Medicines has contracted to acquire the Zembrace SymTouch and Tosymra registered trademarks. Intravail is a registered trademark of
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
TONIX PHARMACEUTICALS HOLDING CORP. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In Thousands, Except Share and Per Share Amounts) (unaudited) | ||||||||||||||||
Three Months Ended | Six Months Ended | |||||||||||||||
2023 | 2022 | 2023 | 2022 | |||||||||||||
COSTS AND EXPENSES: | ||||||||||||||||
Research and development | $ | 21,976 | $ | 16,579 | $ | 48,487 | $ | 35,001 | ||||||||
General and administrative | 7,026 | 6,757 | 14,417 | 14,771 | ||||||||||||
29,002 | 23,336 | 62,904 | 49,772 | |||||||||||||
Operating loss | (29,002 | ) | (23,336 | ) | (62,904 | ) | (49,772 | ) | ||||||||
Interest and other income, net | 646 | 196 | 1,543 | 215 | ||||||||||||
Net loss | (28,356 | ) | (23,140 | ) | (61,361 | ) | (49,557 | ) | ||||||||
Preferred stock deemed dividend | — | 4,255 | — | 4,255 | ||||||||||||
Net loss available to common stockholders | $ | (28,356 | ) | $ | (27,395 | ) | $ | (61,361 | ) | $ | (53,812 | ) | ||||
Net loss per common share, basic and diluted | $ | (2.68 | ) | $ | (7.64 | ) | $ | (5.88 | ) | $ | (17.28 | ) | ||||
Weighted average common shares outstanding, basic and diluted | 10,587,096 | 3,584,699 | 10,428,678 | 3,113,965 | ||||||||||||
See the accompanying notes to the condensed consolidated financial statements |
CONDENSED CONSOLIDATED BALANCE SHEETS (In Thousands) (Unaudited) | |||||
Assets | |||||
Cash and cash equivalents | $ | 25,617 | $ | 120,229 | |
Inventory | 13,700 | - | |||
Prepaid expenses and other | 12,012 | 10,548 | |||
Total current assets | 51,329 | 130,777 | |||
Other non-current assets | 108,407 | 94,913 | |||
Total assets | $ | 159,736 | $ | 225,690 | |
Liabilities and stockholders' equity | |||||
Total liabilities | $ | 19,273 | $ | 18,508 | |
Stockholders' equity | 140,463 | 207,182 | |||
Total liabilities and stockholders' equity | $ | 159,736 | $ | 225,690 |
1The condensed consolidated balance sheet for the year ended
Investor Contact
investor.relations@tonixpharma.com
(862) 904-8182
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505
Media Contact
ICR Westwicke
ben.shannon@westwicke.com
(919) 360-3039
Zembrace® SymTouch® (sumatriptan Injection): IMPORTANT SAFETY INFORMATION
Zembrace SymTouch (Zembrace) can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop use and get emergency help if you have any signs of a heart attack:
- discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
- severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
- pain or discomfort in your arms, back, neck, jaw or stomach
- shortness of breath with or without chest discomfort
- breaking out in a cold sweat
- nausea or vomiting
- feeling lightheaded
Zembrace is not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam shows no problem.
Do not use Zembrace if you have:
- history of heart problems
- narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
- uncontrolled high blood pressure
- hemiplegic or basilar migraines. If you are not sure if you have these, ask your provider.
- had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
- severe liver problems
- taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, dihydroergotamine.
- are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
- an allergy to sumatriptan or any of the components of Zembrace
Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.
Zembrace can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.
Zembrace may cause serious side effects including:
- changes in color or sensation in your fingers and toes
- sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
- cramping and pain in your legs or hips; feeling of heaviness or tightness in your leg muscles; burning or aching pain in your feet or toes while resting; numbness, tingling, or weakness in your legs; cold feeling or color changes in one or both legs or feet
- increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
- medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
- serotonin syndrome, a rare but serious problem that can happen in people using Zembrace, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
- hives (itchy bumps); swelling of your tongue, mouth, or throat
- seizures even in people who have never had seizures before
The most common side effects of Zembrace include: pain and redness at injection site; tingling or numbness in your fingers or toes; dizziness; warm, hot, burning feeling to your face (flushing); discomfort or stiffness in your neck; feeling weak, drowsy, or tired.
Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Zembrace. For more information, ask your provider.
This is the most important information to know about Zembrace but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit www.upsher-smith.com or call 1-888-650-3789.
You are encouraged to report adverse effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
INDICATION AND USAGE
Zembrace is a prescription medicine used to treat acute migraine headaches with or without aura in adults who have been diagnosed with migraine.
Zembrace is not used to prevent migraines. It is not known if it is safe and effective in children under 18 years of age.
Tosymra® (sumatriptan nasal spray): IMPORTANT SAFETY INFORMATION
Tosymra can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop Tosymra and get emergency medical help if you have any signs of heart attack:
- discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
- severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
- pain or discomfort in your arms, back, neck, jaw, or stomach
- shortness of breath with or without chest discomfort
- breaking out in a cold sweat
- nausea or vomiting
- feeling lightheaded
Tosymra is not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam is done and shows no problem.
Do not use Tosymra if you have:
- history of heart problems
- narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
- uncontrolled high blood pressure
- severe liver problems
- hemiplegic or basilar migraines. If you are not sure if you have these, ask your healthcare provider.
- had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
- taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider if you are not sure if your medicine is listed above.
- are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
- an allergy to sumatriptan or any ingredient in Tosymra
Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.
Tosymra can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.
Tosymra may cause serious side effects including:
- changes in color or sensation in your fingers and toes
- sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
- cramping and pain in your legs or hips, feeling of heaviness or tightness in your leg muscles, burning or aching pain in your feet or toes while resting, numbness, tingling, or weakness in your legs, cold feeling or color changes in one or both legs or feet
- increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
- medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
- serotonin syndrome, a rare but serious problem that can happen in people using Tosymra, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
- hives (itchy bumps); swelling of your tongue, mouth, or throat
- seizures even in people who have never had seizures before
The most common side effects of Tosymra include: tingling, dizziness, feeling warm or hot, burning feeling, feeling of heaviness, feeling of pressure, flushing, feeling of tightness, numbness, application site (nasal) reactions, abnormal taste, and throat irritation.
Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Tosymra. For more information, ask your provider.
This is the most important information to know about Tosymra but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit www.upsher-smith.com or call 1-888-650-3789.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
INDICATION AND USAGE
Tosymra is a prescription medicine used to treat acute migraine headaches with or without aura in adults.
Tosymra is not used to treat other types of headaches such as hemiplegic or basilar migraines or cluster headaches.
Tosymra is not used to prevent migraines. It is not known if Tosymra is safe and effective in children under 18 years of age.
Source:
2023 GlobeNewswire, Inc., source