Prior data provide clinical proof-of-concept for narazaciclib’s mechanism of action in endometrial cancer
Preliminary data from trial’s Phase 1 portion expected in 4Q 2023
NEWTOWN, Pa., May 11, 2023 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that the first patient has been dosed in the Company’s Phase 1/2a trial evaluating narazaciclib combined with letrozole in recurrent metastatic low-grade endometrioid endometrial cancer (LGEEC). Narazaciclib is a multi-kinase inhibitor targeting CDK 4, CDK 6, and other kinases important for cell proliferation and motility. Preliminary data from the trial’s Phase 1 portion are expected in 4Q 2023.
Endometrial cancer arises in the uterine lining and is the most common cancer of the female reproductive organs. Endometrioid endometrial cancer is the most common subtype of endometrial cancer, accounting for approximately 75% of cases. Data from prior randomized and single-arm trials have demonstrated the anti-cancer activity of letrozole combined with CDK 4/6 inhibition in recurrent endometrial cancer1-3. Currently, there is no health authority-approved CDK 4/6 inhibitor for the treatment of endometrial cancer.
“Improved treatment options for recurrent LGEEC are urgently needed, as the CDK 4/6 inhibitors currently used off-label for this indication are marked by limitations related to safety, tolerability, and treatment resistance,” said Bhavana Pothuri, M.D., Professor, Department of Obstetrics and Gynecology at NYU Grossman School of Medicine and Director, Gynecologic Oncology Research; Perlmutter Cancer Center and Principal Investigator of the trial. “Narazaciclib’s kinase inhibitory profile suggests it can overcome each of these limitations thanks to reduced activity against kinases whose inhibition is associated with bone marrow toxicity and diarrhea, and increased activity against those implicated in pro-tumor immune suppression and cancer cell survival. This hypothesis is supported by data from in vitro and murine cancer models, and I look forward to its continued evaluation in the ongoing Phase 1/2a trial.”
Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova, commented, “Our recurrent LGEEC program provides an opportunity to establish narazaciclib as a best-in-class therapy in an indication where clinical proof-of-concept for its mechanism of action has been demonstrated. We, therefore, view the program as a key avenue for value creation and look forward to our Phase 1/2a trial’s preliminary data readout expected later this year.”
About the Phase 1/2a Trial
The Phase 1/2a trial is an open-label, multicenter study evaluating narazaciclib in combination with letrozole as a second or third-line treatment for patients with recurrent metastatic LGEEC. Both narazaciclib and letrozole are administered orally with a continuous daily dosing schedule. The trial begins with a Phase 1 dose escalation phase before moving to a Phase 2 expansion cohort designed to enroll approximately 30 patients. The primary objective of the Phase 1 portion of the trial is to evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics in order to determine a recommended Phase 2 dose (RP2D) of the combination. The primary objective of the Phase 2 portion will be to evaluate the efficacy of the combination at the RP2D, as measured by progression-free survival at 24 weeks. The estrogen/progesterone receptor status of participants will be recorded as part of an exploratory objective. The trial will be conducted at sites including NYU Langone Health, the site of the Principal Investigator of the study, sites affiliated with MD Anderson Cancer Center, and U.S. Oncology Research sites.
References
1. Mirza MR. ESMO Virtual Congress 2020. Abstr. LBA28.
2. Konstantinopoulos PA, et al.; 2022 SGO Annual Meeting on Women’s Cancer; March 18-21, 2022. Phoenix, AZ
Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.
Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in a combination trial with estrogen blockade in advanced endometrial cancer. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also evaluating opportunities for combination studies with narazaciclib in additional indications.
Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies, including a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, and a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa.
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding its clinical development and trials, its product candidates, its business and financial position. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
CHATHAM, N.J., May 09, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that it will effect a 1-for-6.25 reverse stock split of its outstanding common stock. This will be effective for trading purposes as of the commencement of trading on May 10, 2023.
The reverse stock split was previously approved by the Board of Directors of Tonix in accordance with Nevada law, under which no stockholder approval is required, and is intended to increase the per share trading price of Tonix’s common stock to satisfy the $1.00 minimum bid price requirement for continued listing on The NASDAQ Capital Market (Rule 5550(a)(1)). Tonix’s common stock will continue to trade on the NASDAQ Capital Market under the symbol “TNXP” and under a new CUSIP number, 890260854. As a result of the reverse stock split, every six and one-quarter pre-split shares of common stock outstanding will become one share of common stock. The reverse stock split will also proportionately reduce the number of shares of authorized common stock from 1 billion to 160 million shares. The reverse split will also apply to common stock issuable upon the exercise of Tonix’s outstanding warrants and stock options.
Tonix’s transfer agent, VStock Transfer LLC, which is also acting as the exchange agent for the reverse split, will provide instructions to shareholders regarding the process for exchanging share certificates. Any fractional shares of common stock resulting from the reverse stock split will be rounded up to the nearest whole post-split share and no shareholders will receive cash in lieu of fractional shares.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS 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 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-1900 (intranasal potentiated oxytocin), in development for chronic migraine, is currently enrolling with topline data expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), a once-daily formulation being developed as a treatment for major depressive disorder (MDD), is also currently enrolling with interim 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 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 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, for which a Phase 1 study is expected to be initiated in the second half of 2023. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease portfolio also includes TNX-3900 and TNX-4000, classes of broad-spectrum small molecule oral antivirals.
*All of Tonix’s product candidates are investigational new drugs or biologics and none has been approved for any indication.
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; delays and uncertainties caused by the global COVID-19 pandemic; 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.
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-15001 which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-29002 for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-8013, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-18504, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-35005 (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL6, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022. Finally, TNX-13007 is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
1Q23 Reported Within Expectations. Tonix reported a 1Q23 loss of $33.0 million or $(0.52) per share, consistent with our expectations of a loss of $34.8 million or $(0.57) per share. During the quarter, the company reported a shift in pipeline priorities toward its CNS programs and discontinuation of some early-stage programs. There are several clinical data presentations expected during 2H23 that we see as potential drivers of the stock in 2H23. The company ended the quarter with $72.0 million in cash.
Looking Forward To Phase 3 Results In Fibromyalgia. Data was presented for the completed RELIEF Phase 3, the first of the two studies needed for an NDA. The interim analysis for the Phase 3 RESILIANT trial has been cancelled, avoiding the “statistical penalty” for the early analysis as well as saving time and money. The RESILIANT data announcement is expected in 4Q23.
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This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).
*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
NEWTOWN, Pa., May 08, 2023 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova” or “the Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced an upcoming presentation at the International Society of Investigative Dermatology (ISID) International Epidermolysis Bullosa Symposium, which is being held in Osaka, Japan through May 9, 2023.
The presentation will take place on May 9, 2023, at 1:00 p.m. Japan Standard Time. During the presentation, Onconova and the principal investigators will provide an overview of its investigator-sponsored clinical program evaluating rigosertib monotherapy in squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC). The Company previously announced that both of the program’s evaluable participants achieved a complete clinical response of all cancerous skin lesions. Onconova plans to review these findings with regulators to determine the most expeditious path toward approval for rigosertib in RDEB-associated SCC.
Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova, commented, “RDEB-associated SCC is an ultra-rare disease with a tragically poor prognosis. The most common cause of death for patients with RDEB is the development of metastatic SCC, driven by the overexpression of PLK-1. Our investigator-sponsored clinical program has produced very promising results based on the complete resolution of the SCCs in patients with RDEB treated to date, suggesting rigosertib can address a pressing unmet need in this indication and may have therapeutic potential in other more common SCCs driven by PLK-1. We look forward to discussing these results with the clinical and scientific community at the upcoming ISID symposium.”
A copy of the slides from the oral presentation will be available on the “Scientific Presentations” section of the Onconova website following the conclusion of the symposium.
About RDEB-associated SCC
RDEB is caused by insufficient expression of type VII collagen protein, which is responsible for anchoring the skin’s inner layer to its outer layer. This leads to extreme skin fragility as well as chronic blistering and wound formation with recurrent infections in RDEB patients, many of whom go on to develop metastatic squamous cell carcinoma driven by overexpression of polo like kinase 1 (PLK-1). RDEB-associated SCC tumors show a highly aggressive and early metastasizing course that makes them the primary cause of death for these patients, with a cumulative risk of death of 70% and 78.7% by ages 45 and 55, respectively1,2. RDEB-associated SCC can appear in pediatric patients or in young adults. Currently available treatments such as targeted therapies and conventional chemo- and/or radiotherapy have demonstrated limited response rates and poor durability in RDEB-associated SCC1,3.
About Onconova Therapeutics, Inc.
Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.
Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose escalation and expansion studies. These trials are currently underway in the United States and China. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also planning a combination trial of narazaciclib with estrogen blockade in advanced endometrial cancer, as well as evaluating opportunities for potential clinical studies in additional indications.
Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies, including a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, and a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC).
Fine et al. J Am Acad Dermatol. 2009 Feb; 60(2):203-11. doi: 10.1016/j.jaad.2008.09.035.
Stratigos et al. Eur J Cancer. 2020 Mar;128:83-102. doi: 10.1016/j.ejca.2020.01.008.
Forward Looking Statements
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding its clinical development and trials, its product candidates, its business and financial position. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
Prioritizing Late-Stage Clinical CNS Programs in Fibromyalgia, Depression, Migraine, and Cocaine Intoxication
Topline Results Expected in Fourth Quarter 2023 for Potentially Confirmatory Phase 3 Trial of TNX-102 SL for Fibromyalgia
Potentially Pivotal Phase 2 Trial of TNX-601 ER for Major Depressive Disorder (MDD) Enrolling; TNX-601 ER Represents an Innovative Approach for MDD through Restoration of Neuroplasticity and Neurogenesis
Cash and Cash Equivalents of Approximately $72.0 Million at March 31, 2023
CHATHAM, N.J., May 08, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced financial results for the first quarter ended March 31, 2023, and provided an overview of recent operational highlights.
“With much achieved already, we expect 2023 will continue to serve as an important milestone year for Tonix,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “The prioritization of key programs in our pipeline highlights our commitment to helping bring relief and value to patients suffering from diseases with limited or insufficient therapeutic options. We are pleased with the enrollment in our current RESILIENT Phase 3 study for TNX-102 SL (cyclobenzaprine HCl sublingual tablets) in fibromyalgia. We are looking forward to topline results from the trial in the fourth quarter of this year. If successful, we believe it will be the second and final adequate and well-controlled efficacy trial required for filing a New Drug Application (NDA) for approval by the U.S. Food and Drug Administration (FDA). Moreover, we believe we have satisfied all the other clinical and non-clinical requirements for an NDA submission. In addition, we are excited to have initiated enrollment in the potentially pivotal UPLIFT Phase 2 study of TNX-601 ER (tianeptine hemioxalate extended release tablets) for major depressive disorder (MDD). TNX-601 ER represents a novel approach to treating depression in the U.S., since the active ingredient tianeptine restores neuroplasticity and neurogenesis rather than modulating neurotransmitter levels and activity.”
Recent Highlights—Key Product Candidates*
Central Nervous System (CNS) Pipeline
TNX-102 SL: small molecule for the management of fibromyalgia (FM)
In March 2023 at the 5th International Congress on Controversies in Fibromyalgia, the Company presented positive efficacy and safety data from Phase 3 RELIEF Study of TNX-102 SL for the management of fibromyalgia. Titled “Efficacy and Safety of TNX-102 SL (Sublingual Cyclobenzaprine) for the Treatment of Fibromyalgia: Results from the Randomized, Placebo Controlled RELIEF Trial”, the presentation displayed the data that TNX-102 SL met its pre-specified primary endpoint in the Phase 3 RELIEF trial, significantly reducing daily pain compared to placebo (p=0.01) in participants with fibromyalgia. In addition, TNX-102 SL was well tolerated with the most common adverse event associated with active treatment being oral numbness or hypoaesthesia, an administration site reaction that is typically transient, was never rated as severe, and led to only one discontinuation.
The Company recently announced new plans to eliminate the interim analysis to streamline the ongoing Phase 3 RESILIENT trial and to provide topline data in the fourth quarter of 2023. Target enrollment for the TNX-102 SL fibromyalgia study remains approximately 470 participants.
TNX-102 SL for the treatment of Fibromyalgia-Type Long COVID, also known as Post-Acute Sequelae of COVID-19 (PASC)
In April 2023, enrollment of 63 participants was completed in the PREVAIL study, a Phase 2 study of TNX-102 SL for fibromyalgia-type Long COVID.
Topline results from the PREVAIL Phase 2 trial are expected in the third quarter of 2023.
In February 2023 at a virtual event co-hosted by BIO and Solve M.E. titled, “Long COVID: What Will it Take to Accelerate Therapeutic Progress?”, the Company presented its analysis that the majority of Long COVID patients present with a constellation of symptoms including multisite pain, fatigue, sleep disorders and cognitive dysfunction or brain fog. These symptoms overlap with fibromyalgia and chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME). Fibromyalgia-type Long COVID, like fibromyalgia and CFS/ME, appears to be one of several chronic overlapping pain conditions that have in common the neurological process called central sensitization.
TNX-601 ER: a once-daily orally-administered small molecule for the treatment of MDD, Posttraumatic Stress Disorder (PTSD), neurocognitive dysfunction associated with corticosteroid use and potentially Alzheimer’s disease
In March 2023, enrollment was initiated in the potentially pivotal Phase 2 ‘UPLIFT’ Study for the treatment of MDD. Results from a planned interim analysis are expected to be released in the fourth quarter of 2023.
TNX-601 ER represents a novel approach to treating depression in the U.S., since the active ingredient tianeptine induces a neuroprotective and resilient phenotype in both neurons and microglia under conditions of stress. The Phase 2 UPLIFT study is a double-blind, randomized, multicenter, placebo-controlled study to evaluate the efficacy and safety of TNX-601 ER taken orally once-daily for 6 weeks to treat MDD. It is a parallel design study with two arms, a TNX-601 ER 39.4 mg arm and a placebo arm. A total of 300 participants will be randomized in a 1:1 ratio into the two arms across approximately 30 U.S. sites, enrolling adult patients 18-65 years old. The primary efficacy endpoint is mean change from baseline in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score at Week 6.
TNX-1900 (intranasal potentiated oxytocin): small peptide for migraine, craniofacial pain, insulin resistance and related disorders, and obesity-associated binge eating disorder
In February 2023, enrollment began in the Phase 2 PREVENTION study of TNX-1900 for the prevention of migraine headache in chronic migraineurs. The double-blind, placebo-controlled study has a target enrollment of 150 participants at approximately 25 sites across the U.S, with topline results expected in the fourth quarter of 2023.
In January 2023, data from clinical and nonclinical studies were presented at the 16th Annual Headache Cooperative of the Pacific (HCOP) Winter Conference by collaborator Professor David Yeomans. The oral presentation titled, “Primary vs Secondary Sex Hormones and Migraine,” includes research sponsored and licensed by Tonix. Preliminary results from a positron emission tomography study in humans showed that intranasal delivery of a radioisotope of magnesium-potentiated oxytocin is delivered to the trigeminal ganglia, which have known roles in migraine headaches. In addition, preliminary results of data collected from isolated human trigeminal ganglia neurons in vitro show co-expression of oxytocin receptors and calcitonin gene-related peptide, which are believed to represent the first observation of oxytocin receptors in human trigeminal ganglia. Furthermore, the presentation highlights data which suggest a sex difference in oxytocin potency.
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.
As previously disclosed, in 2022, Tonix received a Cooperative Agreement grant from the National Institute on Drug Abuse (NIDA), part of the National 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.
In March 2023, Tonix delivered a presentation titled, “TNX-2900 (Intranasal Oxytocin + Magnesium) in Development for the Treatment of Hyperphagia in Adolescents and Young Adults with Prader-Willi Syndrome” at the Rare Disease Innovation and Partnership Summit. The presentation displayed data showing the enhancing effects of magnesium (Mg2+) on the activation of oxytocin receptors. The Mg2+ enhanced formulation of intranasal oxytocin is the basis for TNX-2900, in development to treat 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 initiate 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.
Tonix announced a research agreement with Boston Children’s Hospital to study TNX-1500 for the prevention of graft-versus-host diseases (GvHD) after hematopoietic stem cell transplantation (HCT) in animals. HCT from unrelated donors is a component of the treatment protocol for several hematologic malignancies, but GvHD complicates treatment and limits the success of engraftment after HCT. In April 2023, the Company announced the online publication of two papers in the American Journal of Transplantation by faculty at the Center for Transplantation Sciences, Massachusetts General Hospital (MGH) in collaboration with Tonix Pharmaceuticals1,2. The publications include data demonstrating that TNX-1500 showed activity in preventing organ rejection and was well tolerated in non-human primates. To date, there has not been a humanized anti-CD40L antibody that can effectively prevent transplant rejections with an acceptable level of safety.
Infectious Disease Pipeline
TNX-801 (live horsepox virus vaccine for percutaneous administration): vaccine to protect against smallpox and monkeypox (mpox) designed as a single-administration vaccine to elicit T cell immunity.
As previously announced, a Phase 1 study is expected to start in the second half of 2023.
In January 2023, the Company appointed Zeil Rosenberg, M.D., M.P.H., as Executive Vice President, Medical for Infectious Disease programs. Dr. Rosenberg is responsible for leading the Company’s clinical development efforts for vaccines, including TNX-801.
A publication describing the activity of TNX-801 to protect non-human primates against a lethal challenge with intra-tracheal monkeypox was published in the peer-reviewed journal, Viruses3.
*All of Tonix’s product candidates are investigational new drugs or biologics and none has 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. American Journal of Transplantation. https://doi.org/10.1016/j.ajt.2023.03.022
2Miura, S., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate cardiac allograft survival. American Journal of Transplantation. https://doi.org/10.1016/j.ajt.2023.03.025
3Noyce RS, et al. (2023). Single Dose of Recombinant Chimeric Horsepox Virus (TNX-801) Vaccination Protects Macaques from Lethal Monkeypox Challenge. Viruses. 15(2):356. doi: 10.3390/v15020356
Recent Highlights—Corporate and Other
In April 2023, Tonix announced it is reallocating resources and cash to streamline its pipeline and focus on its mid- and late-stage clinical programs within its core CNS portfolio. The pipeline realignment prioritizes key near-term value drivers, reduces investment in several longer-term programs, particularly COVID-19-related studies, and delays the start of a PTSD study in Kenya.
Recent Highlights—Financial
As of March 31, 2023, Tonix had $72.0 million of cash and cash equivalents, compared to $120.2 million as of December 31, 2022. Net cash used by financing activities was approximately $11.5 million for first quarter 2023, compared to net cash provided by financing activities of $13.1 million for the same period in 2022.
On April 8, 2020, the Company entered into a sales agreement (the “Sales Agreement”) with AGP of up to $320.0 million in at-the-market offerings (“ATM”) sales. During the quarter ended March 31, 2023, the Company sold approximately 3.2 million shares of common stock under the Sales Agreement, for net proceeds of approximately $2.0 million. Subsequent to March 31, 2023, the Company has sold 0.9 million shares of common stock under the Sales Agreement, for net proceeds of approximately $0.5 million.
In January 1, 2023, the Company repurchased 16,700,269 shares of common stock under its share repurchase programs at an average price per share of $0.82 for a gross aggregate cost of approximately $13.6 million.
Cash used in operations was approximately $32.9 million for the first quarter ended March 31, 2023, compared to $31.0 million for the first quarter ended March 31, 2022.
Cash used by investing activities for the first quarter ended March 31, 2023, and 2022 was approximately $3.8 million and $20.2 million, respectively, related to the purchase of property and equipment.
First Quarter 2023 Financial Results
R&D expenses for the first quarter 2023 were $26.5 million, compared to $18.4 million for the same period in 2022. As planned, R&D expenses will be increasing during 2023 as we move our clinical development programs forward and invest in our development pipeline.
G&A expenses for the first quarter 2023 were $7.4 million, compared to $8.0 million for the same period in 2022. The decrease is primarily due to decreased employee-related and financial reporting expenses.
Net loss was $33.0 million, or $0.52 per share, basic and diluted, for the first quarter 2023, compared to net loss of $26.4 million, or $1.61 per share, basic and diluted, for the same period in 2022. The basic and diluted weighted average common shares outstanding for the first quarter 2023 was 63,352,898 compared to 16,445,010 shares for the same period in 2022.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS 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 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-1900 (intranasal potentiated oxytocin), in development for chronic migraine, is currently enrolling with topline data expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), a once-daily formulation being developed as a treatment for major depressive disorder (MDD), is also currently enrolling with interim 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 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 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, for which a Phase 1 study is expected to be initiated in the second half of 2023. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease portfolio also includes TNX-3900 and TNX-4000, classes of broad-spectrum small molecule oral antivirals.
*All of Tonix’s product candidates are investigational new drugs or biologics and none has been approved for any indication.
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; delays and uncertainties caused by the global COVID-19 pandemic; 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.
TONIX PHARMACEUTICALS HOLDING CORP. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In Thousands, Except Share and Per Share Amounts) (unaudited)
Three Months Ended March 31,
2023
2022
COSTS AND EXPENSES:
Research and development
$
26,511
$
18,422
General and administrative
7,391
8,014
33,902
26,436
Operating loss
(33,902
)
(26,436
)
Interest income
897
19
Net loss
$
(33,005
)
$
(26,417
)
Net loss per common share, basic and diluted
$
(0.52
)
$
(1.61
)
Weighted average common shares outstanding, basic and diluted
63,352,898
16,445,010
See the accompanying notes to the condensed consolidated financial statements
1The condensed consolidated balance sheet for the year ended December 31, 2022 has been derived from the audited financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements.
Company to host conference call and webcast at 4:30 p.m. ET on Monday, May 15, 2023
NEWTOWN, Pa., May 04, 2023 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova” or “the Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that the Company intends to release its first quarter 2023 financial results on Monday, May 15, 2023. Management plans to host a conference call and live webcast at 4:30 p.m. ET on the same day to discuss these results and provide an update on its pipeline programs.
Conference Call and Webcast Information
Interested parties who wish to participate in the conference call may do so by dialing (800) 715-9871 for domestic and (646) 307-1963 for international callers and using conference ID 9090989.
Those interested in listening to the conference call via the internet may do so by visiting the investors and media page on the Company’s website at www.onconova.com and clicking on the webcast link. In addition to the live webcast, a replay will be available on the Onconova website for 90 days following the call.
About Onconova Therapeutics, Inc.
Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.
Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose escalation and expansion studies. These trials are currently underway in the United States and China. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also planning a combination trial of narazaciclib with estrogen blockade in advanced endometrial cancer, as well as its clinical study in additional indications.
Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies, including a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, and a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC).
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding its clinical development and trials, its product candidates, its business and financial position. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
Multiple Additional Indications Possible, Including Autoimmune Diseases: Pipeline within a Product
Published Non-Human Primate Studies Show TNX-1500 Prolongs Renal and Heart Allograft Survival
Phase 1 Clinical Trial of TNX-1500 Expected to Start Third Quarter 2023
CHATHAM, N.J., May 04, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, today announced the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application to support a Phase 1 clinical trial with TNX-1500 (anti-CD40L monoclonal antibody [mAb]). The first indication Tonix is seeking for TNX-1500 is the prevention of organ rejection in patients receiving a kidney transplant. The Company expects to initiate enrollment in the Phase 1 study in the third quarter of 2023.
The IND application for TNX-1500 was supported by preclinical allotransplantation studies conducted at the Massachusetts General Hospital (MGH), led by principal investigators Tatsuo Kawai, MD, PhD, A. Benedict Cosimi Chair in Transplant Surgery, MGH and Professor of Surgery, Harvard Medical School (HMS), and Richard N. Pierson III, M.D., scientific director of the Center for Transplantation Sciences in the Department of Surgery at MGH and Professor of Surgery at HMS.
“This is an important milestone as we advance TNX-1500 into clinical development,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Despite advancements in the field of solid organ transplantation, there remains a significant need for new treatments with improved activity and tolerability to prevent organ transplant rejection. Our primary focus of early development will be allotransplantation in which the donor organ comes from another human. However, in the longer term we hope to develop TNX-1500 for xenograft transplantation in which the donor organ comes from a genetically engineered pig.”
Dr. Lederman continued, “We view TNX-1500 as a pipeline within a product, because of its potential to treat a number of autoimmune diseases. Anti-CD40L mAbs have demonstrated activity and tolerability in autoimmune diseases like systemic lupus erythematosus and Sjögren’s Syndrome. An anti-CD40L mAb is also in development for multiple sclerosis. CD40L is a member of the TNFα super gene family. Other TNFα super gene members have been the targets of successful mAb therapeutics: TNFα and RANKL for autoimmune diseases and osteoporosis, respectively. Still other TNFα super gene family members are targeted by mAbs in development including TNF-like ligand 1A (TL1A) and CD30L for ulcerative colitis and Ox40L for atopic dermatitis.”
TNX-1500 is a third generation anti-CD40L mAb that has been designed by protein engineering to decrease FcγRIIA binding and to therefore reduce the potential for thrombosis. Preclinical studies in non-human primates demonstrated that TNX-1500 showed activity in preventing allograft organ rejection and was well tolerated1,2.
About TNX-1500
TNX-1500 (Fc-modified anti-CD40L mAb) is a humanized monoclonal antibody that interacts with the CD40-ligand (CD40L), which is also known as CD154. TNX-1500 is being developed for the prevention of allograft and xenograft rejection, for the treatment of autoimmune diseases and for the prevention of graft-versus-host disease (GvHD) after hematopoietic stem cell transplantation (HCT). A Phase 1 study of TNX-1500 is expected to be initiated in the third quarter of 2023. Two articles have recently published in the American Journal of Transplantation that demonstrate TNX-1500 prolongs non-human primate renal and heart allograft survival1,2.
1Lassiter, G., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate renal allograft survival. American Journal of Transplantation. April 3, 2023. https://doi.org/10.1016/j.ajt.2023.03.022
2Miura, S., et al. (2023) TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate cardiac allograft survival. American Journal of Transplantation. April 6, 2023. https://doi.org/10.1016/j.ajt.2023.03.025
Tonix Pharmaceuticals Holding Corp.*
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS 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 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-1900 (intranasal potentiated oxytocin), in development for chronic migraine, is currently enrolling with topline data expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), a once-daily formulation being developed as a treatment for major depressive disorder (MDD), is also currently enrolling with interim 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 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 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 and xenograft 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, for which a Phase 1 study is expected to be initiated in the second half of 2023. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease portfolio also includes TNX-3900 and TNX-4000, classes of broad-spectrum small molecule oral antivirals.
*All of Tonix’s product candidates are investigational new drugs (IND) or biologics and have not been approved for any indication. TNX-801, TNX-2900, TNX-3900 and TNX-4000 are in pre-IND stage of development and have not been approved for any indication.
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; delays and uncertainties caused by the global COVID-19 pandemic; 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.
Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation. Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two Phase 1 dose-escalation and expansion studies. These trials are currently underway in the United States and China. Onconova’s product candidate rigosertib is being studied in an investigator-sponsored study program, including in a dose-escalation and expansion Phase 1/2a investigator-sponsored study with oral rigosertib in combination with nivolumab for patients with KRAS+ non-small cell lung cancer.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Data Presented At AACR Annual Meeting. Onconova presented two posters at the American Association of Cancer Research annual meeting, held April 14 to April 19. These posters from preclinical studies gave additional details on the activity, targets, and mechanism of action for narazaciclib, its CDK4/6 inhibitor, and comparisons with other approved CDK4/6 inhibitors.
Study On Naraciclib Targets. The first poster showed that narazaciclib inhibits CDK4/6 as well as additional kinases in cancer progression and metastasis. Data included the BUB1 kinase that is overexpressed in breast and uterine cancers, showing correlations between BUB1 expression and poor survival. BUB1 was inhibited by narazaciclib, but not palbociclib (Ibrance, from Pfizer) or abemaciclib (Verzenio, from Lilly). The poster also presented data showing that narazaciclib may lead to the expression of immune modulators in tumor cells that lead to anti-tumor immunity, an important mechanism for preventing recurrence.
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This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).
*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
Research Directed by Faculty of the Center for Transplantation Sciences, Massachusetts General Hospital
CHATHAM, N.J., April 17, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, today announced the on-line publication of two papers1,2 in the American Journal of Transplantation by faculty at the Center for Transplantation Sciences, Massachusetts General Hospital (MGH) in collaboration with Tonix Pharmaceuticals. The data involve studies of Tonix’s TNX-1500 (Fc-modified anti-CD40L humanized monoclonal antibody [mAb]) product candidate in development for the prevention of organ transplant rejection. The molecular target of TNX-1500 is CD40-ligand (CD40L), which is also known as CD154, T-BAM or 5c8 antigen. The publications include data demonstrating that TNX-1500 showed activity in preventing organ rejection and was well tolerated in non-human primates. Blockade of CD40L with TNX-1500 monotherapy consistently and safely prevented pathologic alloimmunity in non-human primate models of cardiac and kidney allograft model without clinical thrombosis.
“There remains a significant need for new treatments with improved activity and tolerability to prevent organ transplant rejection,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “To date, there has not been a humanized anti-CD40L antibody that can effectively prevent transplant rejections with an acceptable level of tolerability. TNX-1500 is a third generation anti-CD40L mAb that has been designed by protein engineering to decrease FcγRII binding and to reduce the potential for thrombosis. The animal studies found that TNX-1500 retains activity to prevent rejection and preserve graft function. Tonix expects to start a first-in-human Phase 1 study in the second quarter of 2023 of TNX-1500 for prophylaxis of organ rejection in adult patients receiving a kidney transplant.”
Tatsuo Kawai, M.D., Ph.D., A. Benedict Cosimi Chair in Transplant Surgery, MGH and Professor of Surgery, Harvard Medical School (HMS) and senior author of the kidney transplant publication, said, “The blockade of the CD40L-CD40 pathway with anti-CD40L mAbs has been the most promising immunomodulatory approach to prevent allograft rejection. However, long-term graft and patient survival following transplantation of kidneys and other solid organs are constrained by side effects of the existing medications. Our data demonstrate a favorable safety profile associated with TNX-1500, since neither non-human primate nor human platelet activation were observed in-vitro when exposed to TNX-1500-sCD40L immune complexes. The therapeutic effects of TNX-1500 to consistently inhibit rejection of mismatched kidney allografts were not associated with infectious or thromboembolic complications, suggesting that clinical studies are warranted to evaluate TNX-1500 for transplant indications.”
Richard N. Pierson III, M.D., scientific director of the Center for Transplantation Sciences in the Department of Surgery at MGH and Professor of Surgery at HMS and senior author of the heart transplant paper said, “Anti-CD40L therapy has a unique activity in controlling the immune response to organ transplants. There remains a significant need for new treatments with improved activity and tolerability to prevent or treat organ transplant rejection. Anti-CD40L has shown great promise to facilitate transplant tolerance in multiple preclinical transplant models. A safe, effective anti-CD40L also has potential to enable use of genetically modified or humanized pig organs to treat humans with advanced organ failure or diabetes, an emerging field known as xenotransplantation.”
Tonix Pharmaceuticals Holding Corp.*
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS 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 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-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is currently enrolling with topline data expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), a once-daily formulation of tianeptine being developed as a treatment for major depressive disorder (MDD), is also currently enrolling with interim 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 second quarter of 2023. Tonix’s rare disease 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 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 and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox, for which a Phase 1 study is expected to be initiated in the second half of 2023. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease portfolio also includes TNX-3900, a class of broad-spectrum small molecule oral antivirals.
*All of Tonix’s product candidates are investigational new drugs or biologics and none has been approved for any indication.
1Lassiter, G., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs nonhuman primate renal allograft survival. American Journal of Transplantation. April 3, 2023. https://doi.org/10.1016/j.ajt.2023.03.022
2Miura, S., et al. (2023) TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs nonhuman primate cardiac allograft survival. American Journal of Transplantation. April 6, 2023. https://doi.org/10.1016/j.ajt.2023.03.025
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; delays and uncertainties caused by the global COVID-19 pandemic; 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.
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-15001 which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-29002 for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-8013, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-18504, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-35005 (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL6, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022. Finally, TNX-13007 is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Interim Analysis Cancelled For Two Trials. Tonix announced that it has eliminated the interim analyses for the Phase 3 RESILIENT trial testing TNX-102 SL in fibromyalgia and the Phase 2 PREVENTION trial testing TNX-601 in chronic migraine. Both trials were due to report interim analyses in 2Q23, and both trials will continue treatment with top-line results expected in 4Q23 as planned. These interim results were not important drivers for the stock, and we expect little to no impact from their elimination.
Elimination Of The Interim Analysis Eliminates The Statistical Penalty. Clinical trials that include interim analysis have a “statistical penalty” for analyzing data before the conclusion of the trial. This often requires the final analysis to reach higher levels of statistical significance or show benefits of the drug in larger patient populations. By eliminating the interim analysis in its trials, Tonix can reduce the target enrollment for the PREVENTION trial from 300 to about 150 patients. This reduces the cost and time for the trial.
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This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).
*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
Streamlining Phase 3 Fibromyalgia and Phase 2 Chronic Migraine Trials by Eliminating Interim Analyses
Topline Results Expected for Both Programs in Fourth Quarter 2023
CHATHAM, N.J., April 13, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, today announced that it is eliminating the interim analyses in its registration-enabling, confirmatory Phase 3 RESILIENT study of TNX-102 SL for fibromyalgia and its Phase 2 PREVENTION study of TNX-1900 for chronic migraine. The modifications to the RESILIENT and PREVENTION studies are designed to streamline the trials and to provide topline data for both programs in 2023. Target enrollment for the core TNX-102 SL fibromyalgia study remains approximately 470 participants while target enrollment for the TNX-1900 chronic migraine study will be reduced from approximately 300 participants to approximately 150 participants, to accommodate the new topline timing.
“In an effort to expedite and deliver on clinical timelines, we are modifying the designs of our confirmatory, registration-enabling Phase 3 trial in fibromyalgia and our Phase 2 trial in chronic migraine,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “By eliminating the interim analyses, we remove the statistical penalties associated with this type of analysis, conserve resources, and can plan on topline results for each of these studies in the fourth quarter of 2023. Fibromyalgia and migraine each affect millions of people, and we remain committed to aligning our operational and scientific efforts on these core CNS programs. We are excited to progress these programs closer to FDA approval, upon achieving positive topline data.”
Key Anticipated 2023 Milestones
Updated Guidance
Eliminating interim analysis of Phase 3 RESILIENT study of TNX-102 SL (sublingual cyclobenzaprine tablets) for fibromyalgia.
Eliminating interim analysis of Phase 2 PREVENTION study of TNX-1900 (intranasal potentiated oxytocin) for chronic migraine; topline results now expected in the fourth quarter of 2023.
Unchanged Guidance
Topline results of Phase 3 RESILIENT study of TNX-102 SL (sublingual cyclobenzaprine tablets) for fibromyalgia in the fourth quarter of 2023.
Topline results of Phase 2 PREVAIL study of TNX-102 SL for fibromyalgia-type Long COVID in the third quarter of 2023.
Interim analysis results of Phase 2 UPLIFT study of TNX-601 ER (tianeptine hemioxalate extended-release tablets) for major depressive disorder in the fourth quarter of 2023.
Initiate enrollment in a potentially pivotal Phase 2 study of TNX-1300 (recombinant double-mutant cocaine esterase for injection) for the treatment of cocaine intoxication in the second quarter of 2023.
Initiate enrollment in a Phase 1 study of TNX-1500 (anti-CD40L monoclonal antibody) for the prophylaxis of rejection in kidney transplantation in the second quarter of 2023.
Initiate enrollment in a Phase 1 study of TNX-801 (live virus vaccine for percutaneous administration), a potential vaccine to protect against smallpox and mpox (formerly known as monkeypox), in the second half of 2023.
Continue development of TNX-2900 (intranasal potentiated oxytocin), a small peptide for the treatment of hyperphagia in Prader-Willi syndrome (PWS), for which the FDA has granted Orphan Drug designation.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS 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 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-1900 (intranasal potentiated oxytocin), in development for chronic migraine, is currently enrolling with topline data expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), a once-daily formulation being developed as a treatment for major depressive disorder (MDD), is also currently enrolling with interim 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 second quarter of 2023. Tonix’s rare disease 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 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 and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox, for which a Phase 1 study is expected to be initiated in the second half of 2023. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease portfolio also includes TNX-3900 and TNX-4000, classes of broad-spectrum small molecule oral antivirals.
*All of Tonix’s product candidates are investigational new drugs (IND) or biologics and have not been approved for any indication. TNX-801, TNX-1500, TNX-2900, TNX-3900 and TNX-4000 are in pre-IND stage of development and have not been approved for any indication.
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; delays and uncertainties caused by the global COVID-19 pandemic; 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.
NEWTOWN, Pa., April 06, 2023 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova” or “the Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that its Chief Medical Officer (CMO) Mark Gelder, M.D., recently passed away unexpectedly. Michael Saunders, M.D., has been appointed the interim Chief Medical Officer of the Company.
“The entire Onconova family is deeply saddened by the loss of our beloved friend and colleague,” said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova. “Dr. Gelder dedicated his career to caring for and improving the treatment options available to patients with cancer and his thought leadership and scientific passion will be sorely missed by the oncology community. Mark trained and worked as a gyn oncologist. Because of that experience, he was dedicated to advancing the care of women with reproductive tract cancers. Our goal is to complete the mission Mark started by advancing our trial in endometrial cancer and other cancers as well. We are immensely grateful for his many contributions to Onconova and express our deepest condolences to his family, friends, and colleagues during this difficult time. May he rest in peace.”
Dr. Gelder served as Onconova’s CMO since June 2021. He was an accomplished industry leader with more than 35 years of experience in clinical development, medical affairs, and medical marketing. His previously held the role of CMO at Elevar Therapeutics, Pierian Biosciences (formerly DiaTech Oncology), Accelovance, Inc., and Heron Therapeutics, Inc. Dr. Gelder also had extensive experience at large pharmaceutical companies including Pfizer, Wyeth, and Bayer. He earned his M.D. from the University of Virginia School of Medicine, completed a fellowship in gynecologic oncology, and was a Fellow of the American College of Physicians and the American College of Obstetrics and Gynecology.
Onconova’s Board of Directors will convene at an appropriate time to appoint a new CMO. A search is currently underway by the Company. Onconova does not expect any material impact to the stated timelines associated with its ongoing or planned clinical trials due to this sad news. The Company notes that Dr. Michael Saunders, a noted drug developer and colleague, has been working with Onconova as a consultant and is thus well acquainted with the Company’s compounds in development.
About Onconova Therapeutics, Inc.
Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.
Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose escalation and expansion studies. These trials are currently underway in the United States and China. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also planning a combination trial of narazaciclib with estrogen blockade in advanced endometrial cancer, as well as its clinical study in additional indications.
Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies, including a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, and a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC).
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding its clinical development and trials, its product candidates, its business and financial position. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
Data from Animal Studies on TNX-1700 (recombinant TFF2 – albumin fusion peptide) in Syngeneic Models of Colorectal and Gastric Cancer Will be Presented
CHATHAM, N.J., April 05, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, today announced that two posters with research results on TNX-1700 (recombinant TFF2 – albumin fusion peptide) will be presented as posters at the American Association for Cancer Research (AACR) Annual Meeting being held April 14-19, 2023, in Orlando, Fla. These data demonstrate that targeting myeloid-derived suppressor cells (MDSCs) using mTNX-1700, a murine TFF2 – murine serum albumin fusion peptide (mTFF2-MSA) provides additive benefits to PD-1 blockade therapy in advanced and metastatic syngeneic mouse models of colorectal and gastric cancer.
Copies of the Company’s posters will be available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com following the conference. Additional meeting information can be found on the AACR website.
Bruce L. Daugherty1, Rebecca J. Boohaker2, Rebecca Johnstone2, Karr Stinson2, Jin Qian3, Timothy C. Wang3, Seth Lederman1
1. Tonix Pharmaceuticals, Inc., 26 Main Street, Suite 101, Chatham, NJ 07928 2. Southern Research, 2000 9th Ave S, Birmingham, AL 35205 3. Division of Digestive and Liver Diseases, Irving Cancer Research Center, Columbia University Medical Center, New York, NY 10032, USA
Topic:
Oncolytic Viruses, Anticancer Vaccines, and Other Immunomodulatory Therapies
Jin Qian1, Sandra Ryeom1, Bruce Daugherty2, Seth Lederman2, Timothy C. Wang12.
1. Division of Digestive and Liver Diseases, Irving Cancer Research Center, Columbia University Medical Center, New York, NY 10032, USA
2. Tonix Pharmaceuticals, Inc., 26 Main Street, Suite 101, Chatham, NJ 07928
Title:
Combination Immunotherapies 1
Location:
Orange County Convention Center, Orlando, Fla.
Section:
21, #5088
Date:
Tuesday, April 18, 2023
Time:
1:30 p.m. – 5:00 p.m. ET
Tonix Pharmaceuticals Holding Corp.*
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS 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 with interim data expected in the second 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-1900 (intranasal potentiated oxytocin), in development for chronic migraine, is currently enrolling with interim data expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), a once-daily formulation being developed as a treatment for major depressive disorder (MDD), is also currently enrolling with interim 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 second quarter of 2023. Tonix’s rare disease 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 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 and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox, for which a Phase 1 study is expected to be initiated in the second half of 2023. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease portfolio also includes TNX-3900 and TNX-4000, classes of broad-spectrum small molecule oral antivirals.
*All of Tonix’s product candidates are investigational new drugs or biologics and have not been approved for any indication.
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; delays and uncertainties caused by the global COVID-19 pandemic; 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.
