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, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Study Supports Use of TNX102-SL in Long COVID Symptoms. Tonix presented a large, retrospective study to determine the incidence of symptoms in patients with Long COVID (PASC, or Post-Acute Sequelae of COVID-19). The findings showed a significant overlap between symptoms of Long COVID and fibromyalgia, including multi-site pain, fatigue, and insomnia. It also found significantly higher opioid use in patients that have multi-site pain. Since TNX-102 SL has been shown to relieve symptoms of fibromyalgia, we believe the study supports the Phase 2 PREVAIL trial of TXN-102 SL in Long COVID.
Retrospective Analysis of Patient Database Identifies Long COVID Symptoms. Tonix analyzed a large patient database using published criteria to identify Long COVID patients in a network of health care providers. In the sample of 260,082 COVID patients, 20.1% (n=52,322) had Long COVID symptoms. Of these 41.5% (n=21,694) had multi-site pain.
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., Sept. 22, 2022 (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 Company will be participating in-person in the Ladenburg Thalmann 2022 Healthcare Conference, which is taking place at the Sofitel Hotel in New York, NY.
Steven Fruchtman, M.D., President & CEO of Onconova, will present a corporate overview to include an update on the company’s portfolio, followed by a 10-minute analyst-led fireside chat, on September 29th, 2022, at 12:00 p.m. ET. The presentation can be viewed here or on the “Corporate Events and Presentations” section of the Onconova website and will be archived for 90 days. Dr. Fruchtman and other members of the Onconova management team will also be available for in-person one-on-one meetings during the conference on September 29th, 2022. Those interested in requesting a meeting should contact their Ladenburg Thalmann representative.
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.
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.
Approximately 40% of Long COVID Patients Experience Multi-Site Pain Similar to Overlapping Chronic Pain Syndromes Fibromyalgia and ME/CFS
Approximately 50% of Long COVID Patients with Multi-Site Pain and Sleep Disturbance Use Opiates
CHATHAM, N.J., Sept. 22, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced data from a retrospective observational database study in patients diagnosed with Long COVID at the International Association for the Study of Pain (IASP) 2022 World Congress on Pain, being held September 20-23, 2022, in Toronto, Canada. The study was motivated to identify the frequency of symptoms of multi-site pain, fatigue and insomnia in Long COVID patients because these are the hallmarks of Overlapping Chronic Pain Syndromes like fibromyalgia and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). A copy of the poster is available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com.
The poster presentation titled, “Retrospective Observational Database Study of Patients with Long COVID with Multi-site Pain, Fatigue, and Insomnia: A Real-World Analysis of Symptomatology and Opioid Use,” include data from the study showing that:
Approximately 40% of patients with symptoms of Long COVID had fibromyalgia-like multi-site pain.
The rate of opioid use in Long COVID patients with multi-site pain was 34%, compared to 19% of Long COVID patients without multi-site pain.
In patients with multi-site pain, opioid use increased to approximately 50% of patients when sleep disturbance was also present.
“The recently released U.S. HHS National Research Action Plan on Long COVID1 repeatedly addresses the overlap of Long COVID with ME/CFS, which, like fibromyalgia is one of the overlapping chronic pain syndromes with central sensitization,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Previously, central sensitization had been observed in approximately two-thirds of Long COVID patients2. The data from the new retrospective study revealed that U.S. Long COVID patients are turning to opioids for symptomatic relief, revealing the urgency to provide effective non-opioid, non-addictive analgesics that address multi-site pain. Currently, there is no therapy approved by the U.S. Food and Drug Administration for the treatment of multi-site pain associated with Long COVID.”
In August 2022, the Company announced that the first participant was enrolled in the Phase 2 PREVAIL study of TNX-102 SL as a potential treatment for patients with Long COVID syndrome (Long COVID) whose symptoms overlap with fibromyalgia. Long COVID is known officially as Post-Acute Sequelae of COVID-19 (PASC).
Dr. Lederman added, “TNX-102 SL is a centrally-acting non-opioid analgesic with no recognized abuse potential that has shown to decrease daily pain in a Phase 3 study of fibromyalgia. The finding that approximately 40% of Long COVID patients have fibromyalgia-like multi-site pain symptoms in the retrospective observational database study suggests that we should be able to recruit a robust cohort of participants to test the effects of TNX-102 SL in treating multi-site pain in Long COVID in our ongoing Phase 2 study.”
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 a new Phase 3 study launched in the second quarter of 2022 and 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. Tonix initiated a Phase 2 study in Long COVID in the third quarter of 2022 and expects interim data in the first half 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 first quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is expected to enter the clinic with a Phase 2 study in the fourth quarter of 2022. 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 first half of 2023. Tonix’s infectious disease pipeline consists of a vaccine in development to prevent smallpox and monkeypox, next-generation vaccines to prevent COVID-19, and a platform to make fully human monoclonal antibodies to treat COVID-19. TNX-801, Tonix’s vaccine in development to prevent smallpox and monkeypox, also serves as the live virus vaccine platform or recombinant pox vaccine (RPV) platform for other infectious diseases. A Phase 1 study of TNX-801 is expected to be initiated in Kenya in the first half of 2023. Tonix’s lead vaccine candidate for COVID-19 is TNX-1850, a live virus vaccines based on Tonix’s recombinant pox live virus vector vaccine platform. A Phase 1 study of the COVID-19 vaccine is expected to be initiated in the second half of 2023.
*All ofTonix’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, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, 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.
TNX-1500 Treatment Prevents Organ Rejection and Preserves Function for Both Allograft and Xenograft Transplants in Animal Studies
CHATHAM, N.J., Sept. 15, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced data from three oral presentations by faculty at the Center for Transplantation Sciences, Massachusetts General Hospital Center at the 29th International Congress of The Transplantation Society (TTS 2022) held September 10-14, 2022 in Buenos Aires, Argentina. The data involve studies of Tonix’s TNX-1500 (Fc modified anti-CD40L monoclonal antibody) 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. Copies of the presentations are available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com.
The presentations titled, “Long-term rejection-free renal allograft survival with Fc-modified anti-CD154 antibody monotherapy in nonhuman primates,” and “Monotherapy with TNX-1500, a Fc-modified anti-CD154mAb, prolongs cardiac allograft survival in cynomolgus monkeys,” include data demonstrating that TNX-1500 treatment 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 cardiac and kidney allograft models without clinical thrombosis.
The presentation titled, “Long-term (>1 year) rejection-free survival of kidney xenografts with triple xenoantigen knockout and multiple human transgenes in nonhuman primates,” includes data demonstrating that TNX-1500 treatment showed activity in preventing xenograft kidney rejection and was well tolerated in non-human primates. Xenografts are transplanted organs from donors of a different species from the recipient, and in this study, the xenografts originated from genetically engineered pigs. Blockade of CD40L with TNX-1500 monotherapy consistently and safely prevented pathologic xenoimmunity in non-human primate kidney xenograft models 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. We believe TNX-1500 has the potential for treating and preventing organ transplant rejection in both allograft and xenograft transplants. Tonix expects to initiate a Phase 1 trial of TNX-1500 in the first half of 2023. Preventing transplant rejection is the first indication we are pursuing, but based on results of anti-CD40L in numerous animal models, we believe that TNX-1500 has the potential for treating a number of autoimmune conditions.”
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 a new Phase 3 study launched in the second quarter of 2022 and 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. Tonix initiated a Phase 2 study in Long COVID in the third quarter of 2022 and expects interim data in the first half 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 first quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is expected to enter the clinic with a Phase 2 study in the fourth quarter of 2022. 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 infectious disease pipeline consists of a vaccine in development to prevent smallpox and monkeypox, next-generation vaccines to prevent COVID-19, and a platform to make fully human monoclonal antibodies to treat COVID-19. TNX-801, Tonix’s vaccine in development to prevent smallpox and monkeypox, also serves as the live virus vaccine platform or recombinant pox vaccine (RPV) platform for other infectious diseases. A Phase 1 study of TNX-801 is expected to be initiated in Kenya in the first half of 2023. Tonix’s lead vaccine candidate for COVID-19 is TNX-1850, a live virus vaccines based on Tonix’s recombinant pox live virus vector vaccine platform. A Phase 1 study of the COVID-19 vaccine is expected to be initiated in the second half of 2023.
*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, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, 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, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Data Shows Efficacy After Treatment Failures. Onconova presented positive interim data from its Phase 1/2a trial testing rigosertib at the European Society for Medical Oncology (ESMO). The Investigator-sponsored trial tested the combination of rigosertib with nivolumab, an anti-PD1 checkpoint inhibitor, in non-small cell lung cancer patients that had progressive non-small cell lung cancer (NSCLC) following treatment with checkpoint inhibitor therapies.
Patients Showed Both Complete and Partial Responses. The interim data reported was for 14 out of 19 patients enrolled in the study. Four of the 14 (29%) showed disease control responses, including 1 complete response, 2 partial responses, and 1 stable disease. Mean survival for these responders was 6.75 months, compared with 1-2 months for the non-responders. Each responding patient had a different mutation in the KRAS gene, showing activity against multiple variants.
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.
Data show an early signal of efficacy in an extensively pre-treated population with 1 complete response and 2 partial responses achieved in 14 evaluable patients
Responses achieved in patients with 3 distinct and different KRAS mutations, confirming the MOA of rigosertib being KRAS+ agnostic
4 of 14 (29%) evaluable patients demonstrated disease control
The combination of rigosertib and nivolumab has been well tolerated with no synergistic toxicities observed to-date
NEWTOWN, Pa., Sept. 12, 2022 (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 updated data from an investigator-sponsored Phase 1/2a trial of oral rigosertib plus the anti-PD-1 immune checkpoint inhibitor (ICI) nivolumab in advanced KRAS-mutated (KRAS+) non-small cell lung cancer (NSCLC). The data, which are featured in a poster at the European Society for Medical Oncology (ESMO) Congress 2022, show an early and encouraging signal of efficacy in the trial’s extensively pre-treated population. The studied doublet has been well tolerated to-date.
“The emerging data being presented at ESMO are encouraging, as treatment with rigosertib plus nivolumab led to both complete and partial responses in patients with KRAS-mutated lung cancers who failed prior ICI therapy,” said Dr Rajwanth Veluswamy, the principal investigator of the study. “Objective responses showcased rigosertib’s KRAS mutation-agnostic mechanism of action, as each responding patient had a tumor with a different underlying variant. This differentiates rigosertib from agents targeting a single KRAS mutation variant, and positions it to potentially address the unmet needs of a much broader patient population. In addition, the ESMO data demonstrated activity in multiple patients with both low PD-L1 expression at diagnosis and STK11/LKB1 co-mutations, both poor predictive features for current lung cancer treatments.”
Key data from the presentation include:
Demographics:
All enrolled patients failed at least one line of prior therapy with a PD-1 checkpoint inhibitor (includes evaluable and non-evaluable patients)
80% of enrolled patients failed at least two lines of prior therapy
Response results (as of August 15th, 2022-data cutoff date):
3 of 14 evaluable patients achieved an objective response
1 patient achieved a complete response (CR) as per RECIST Criteria, with complete resolution of the primary lung tumor as well as sites of metastatic disease.
2 patients achieved a partial response (PR)
Responses were achieved in patients with 3 distinct KRAS mutations (CR: KRAS G12V; PRs: KRAS G12C/STK11 and Q61H/STK11)
The mean duration of response is 6.75 months
4 of 14 evaluable patients achieved disease control (CR, PR, or stable disease)
Safety results:
The studied doublet has been generally well tolerated. Treatment-related adverse events (TRAE) have been mostly mild and manageable.
One dose limiting toxicity of grade 3 hyponatremia has been observed (previously documented with rigosertib)
Urinary toxicities well documented with rigosertib are the most common TRAE
No unexpected safety events or synergistic toxicities have been observed
Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova, commented, “The evidence of efficacy observed in the trial’s highly challenging population suggests rigosertib may synergize with ICI and potentially provide clinical benefit to patients with limited therapeutic options. This hypothesis is supported both by these latest clinical data and the results of preclinical studies in multiple indications. Looking forward, we expect the maturation of the trial’s current results, as well as the new data we expect to collect by enrolling additional patients, to provide key insights that will inform the next steps for rigosertib’s current investigator-sponsored study program.”
The ESMO poster (#1018P) is titled “Phase 1/2 Trial of Rigosertib and Nivolumab for KRAS Mutated Non-Small Cell Lung Cancer (NSCLC) Patients.” It is currently available for viewing on the congress’s virtual platform and is being presented by the trial’s principal investigator, Rajwanth Veluswamy, M.D., Assistant Professor, Medicine, Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, today during Poster Session 14. The poster is available on the “Scientific Presentations” section of the Onconova website.
About the Investigator-sponsored Phase 1/2a Trial
This Phase 1/2a trial is designed to evaluate the combination of rigosertib and nivolumab in advanced KRAS+ metastatic NSCLC patients who have progressed on standard-of-care with anti-PD-1 monotherapy or anti-PD-1 in combination with chemotherapy. It includes a dose-escalating Phase 1 portion followed by a Phase 2a dose-expansion portion. Patients in the trial receive oral rigosertib twice daily on days 1-21, and intravenous nivolumab on days 1 and 15 of 28-day cycles. The primary endpoints of the trial are safety assessments to determine maximum tolerated dose, and overall response rate. Secondary endpoints include progression-free survival and overall survival. For more information on the trial, see ClinicalTrials.gov Identifier: NCT04263090.
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.
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.
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 the timing of Onconova’s and investigator-initiated clinical development and trial data, and the mechanisms, therapeutic effects, and indications for Onconova’s product candidates. 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., Sept. 06, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced three oral presentations by academic collaborators at the 29th International Congress of The Transplantation Society (TTS 2022) being held September 10-14, 2022 in Buenos Aires, Argentina, and virtually. Copies of the presentations will be available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com following the conference.
Oral Presentation Details
Title:
Long-term rejection free renal allograft survival with Fc-modified anti-CD154 antibody monotherapy in nonhuman primates
Date:
Monday, September 12, 2022
Time:
4:35 p.m. EDT (17:35 ART)
Session:
Campfire Session: Models, mechanisms & therapies
Presenter
Grace Lassiter, M.D., Research Fellow of the Kawai Lab
Title:
Monotherapy with TNX-1500, a Fc-modified anti-CD154mAb, prolongs cardiac allograft survival in cynomolgus monkeys
Date:
Tuesday, September 13, 2022
Time:
3:25 p.m. EDT (16:25 ART)
Session:
Mini-Oral Abstracts Session: Snap-shots of thoracic transplantation
Presenter
Kohei Kinoshita, M.D., Research Fellow of the Pierson Lab
Title:
Long-term (>1 year) rejection/TMA free survival of kidney xenografts with triple xenoantigen knockout and multiple human transgenes in nonhuman primates
Date:
Wednesday, September 14, 2022
Time:
10:00 a.m EDT (11:00 ART)
Session:
Mini-Oral Abstracts Session: Xenotransplantation
Presenter
Grace Lassiter, M.D., Research Fellow of the Kawai Lab
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 a new Phase 3 study launched in the second quarter of 2022 and 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. Tonix initiated a Phase 2 study in Long COVID in the third quarter of 2022 and expects interim data in the first half 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 first quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is expected to enter the clinic with a Phase 2 study in the fourth quarter of 2022. 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 first half of 2023. Tonix’s infectious disease pipeline consists of a vaccine in development to prevent smallpox and monkeypox, next-generation vaccines to prevent COVID-19, and a platform to make fully human monoclonal antibodies to treat COVID-19. TNX-801, Tonix’s vaccine in development to prevent smallpox and monkeypox, also serves as the live virus vaccine platform or recombinant pox vaccine (RPV) platform for other infectious diseases. A Phase 1 study of TNX-801 is expected to be initiated in Kenya in the first half of 2023. Tonix’s lead vaccine candidate for COVID-19 is TNX-1850, a live virus vaccines based on Tonix’s recombinant pox live virus vector vaccine platform. A Phase 1 study of the COVID-19 vaccine is expected to be initiated in the second half of 2023.
*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, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, 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.
The Differences Investors Should Know Between Biotech Companies and Pharmaceutical Manufacturers
In mid-June of this year, the stock market seemed to have changed. The oil sector that had been up for most of the year began trending down, and most other industries that had been hard hit began moving up in a mid-year “V” shape. The new leader replacing oil was biotech, which had been one of the hardest hit at the beginning of the year. It makes sense that biotech trades with its own cycles – it’s a very different sector. There is reason to believe that fundamentals favor the biotech sector now, below we discuss the nuances of these very interesting companies, and explain how they are different than other drug companies..
With biotech stocks, regulators, financiers, the highly educated, geniuses, technology, and investors all come together to help people in the most important way – life and health. For investors, it provides its own set of challenges, and at least two possible rewards. If an investor uncovers the stock that has the right mix of science, money and regulatory approval, they may not only cash in like few other sectors, but also be part of improving the health of generations to come.
Above, I did use the word “possible”. This is true of all stocks in all sectors, but the volatility in the biotech sector is a demonstration of the adage that for bigger potential rewards, one would have to increase their risk. Companies in this industry, often startups with great patents, burn through millions of dollars, often with zero earnings, and with any potential for earnings years off and contingent on being right about their science and the products it can provide. This means stock selection in biotech requires a deeper understanding of the company and potential for its work than in other sectors.
What’s included in the Biotechnology Sector?
Biotechnology focuses on novel drug development and clinical research aimed at treating diseases and medical conditions.
They are almost always unprofitable, and many have no revenue at all. In fact, some stock analysts would suggest that the main distinction between a biotech and a pharmaceutical company lies in profitability. Biotechnology is often characterized as being more research and development than manufacturing. They serve as incubators for the ideas that lead to the next “miracle” cures. But, drug development has a long development timeline. A successful candidate can take a decade or more to go from idea to available medicine. That’s if it is among the roughly 10% of prospective candidates that reaches approval for use.
Why would anyone invest in a company that is developing a pipeline of potential products that each have about a 10% chance of approval? Because breakthrough drugs, and the potential for breakthroughs, each step forward on the path toward approval often provides a huge upward move in the stock price. Even if not eventually approved. Conversely failure to move forward often comes with a rapid decline from disappointment.
Differences Between Biotech and Pharmaceuticals
There is a lot of overlap and a gray area between what is biotech and what is pharmaceutical. Here are some guidelines of where the differences often lay.
Biotechnology ventures tend to be more entrepreneurial pursuits with a lot on the line for the success of an idea, while pharmaceutical companies have an ongoing business and can better gauge and manage business risk. One reason is pharmaceutical companies often have a large diversified portfolio of approved drugs they market. They may also have drugs in various stages of research and development, but these costs are usually investments in future products using current revenue.
A biotechnology firm is often based on “new” science and driven to develop a useful and profitable application for the science. An example of a growing segment of biotech is biopharma which develops drugs originating living organisms.
Large pharmaceutical companies often pay stable dividends to shareholders, this is rare in companies that fall under the biotech umbrella, if there are earnings it may be reinvested in moving forward the study of drugs in development. Many biotechs’ strength is to explore and develop, not to market the successful result. Large pharmaceutical companies, like Johnson and Johnson (JNJ)or Eli Lilly (LLY) are marketing machines, they look to smaller biotech to be an incubator to keep their portfolio of products fresh.
The two industries also stand apart when it comes to valuation and business evaluation. Models and valuation derived from cash flow are quite relevant in assessing pharmaceutical stocks. While many analysts gamely attempt to construct discounted cash flow models for early-stage biotechs, the reality is that success is often thumbs-up or thumbs-down (“drug works” or “drug doesn’t work”).
The FDA Has Final Say
As the regulatory body that approves new drugs for the U.S. market, as well as issues permits for human clinical trials, the Food and Drug Administration (FDA) is the gatekeeper for every biotech firm. Investors should have some understanding of the FDA process and requirements or follow an analyst covering the sector that does. In order to get FDA approval, biotechs must establish a sufficient body of information that the drug is safe and effective. This is generally done through a series of at least three clinical trials.
If the trials meet the goals for safety and efficacy, the company files a request for approval called a New Drug Application (NDA). If the NDA is approved, the FDA sets the date a decision will be made by on the application.
Product Pipeline
A biotech’s pipeline is the future of the company. Investors focus a great deal on the number of entrants in the pipeline, the stages of study they are at, and the overall science, and competing products (if any). It’s critical to establish valuation forecasts.
Different biotech companies tend to operate in different stages of a product’s development. The stages are, most commonly, preclinical testing, enabling studies, clinical trials, and biologics licensing. A company that is focused on the early stage of research and development will conduct preliminary research to determine whether an idea may successfully be used against a disease or ailment. It attempts to answer, “can this technology become a product?”. This step requires translating the technology into a product. Preclinical testing is the phase of product development when a company must show that its proposed product is safe. They must also determine what the pharmacokinetics (PK) pharmacodynamics (PD) are. PK shows what happens to a drug once it enters the body, ie: how quickly its absorbed, distributed, metabolized, and excreted. Studies done for the express purpose of convincing the FDA to approve the start of clinical trials in humans are called Investigational New Drug (IND)-enabling.
Once a startup has received approval from the FDA to begin clinical trials for their product, they progress through a series of phases depending on how their product fares. These phases are:
Phase 1 This first trial’s main purpose is to determine that the product is safe for use in humans. The number of participants is kept as small as possible, and may be limited to patients who are farther along in a disease state or who have exhausted other options for treatment.
Phase 2 The second phase continues to confirm that the product is safe, but increases the number of participants and tries to determine the optimum dose and regimen.
Phase 3 Typically the last clinical trial phase and is designed to determine whether the treatment is truly effective, and is expanded to include enough people so the potential side effects are more likely to be captured in the data.
The biologics license application (BLA) is the process by which a biologic that has successfully completed clinical trials applies to the FDA to be sold to the public. A new drug application (NDA) is the same process, but for pharmaceuticals.
Take Away
Biotech investing can be very rewarding. Investors need to understand the company and the risks, and also the stage in the pipeline and the success of recent trials. Failed attempts by companies in this sector at devising the next wonder drug outnumber successes. One success may easily make up for some failures for investors. With research into the company and even reliance on trusted analysts that specialize in biotechs, an investor can increase their chances of uncovering oversized profits. We encourage you, if you are not signed up for Channelchek and the research by Noble Capital Markets equity analysts delivered to your inbox each morning, to take the time and register with us now.
