Company to host conference call and webcast at 4:30 p.m. Eastern Time on Monday, November 14, 2022
NEWTOWN, Pa., Nov. 07, 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 intends to release its third quarter 2022 financial results on Monday, November 14, 2022. 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 6078502.
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.
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.
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.
3Q22 Reported With Clinical Progress. Tonix reported a net loss for 3Q22 of $29.0 million or $(0.69) per share. Cash balance at the end of the quarter was $140.0 million, excluding a private placement in October that raised gross proceeds of $15 million. The company made progress in several programs during the quarter, reiterating milestones for current trials and plans for new trials in 2023.
Current Trials Have Data Milestones In 2023. The Phase 3 RESILIENT study testing TNX102 SL in fibromyalgia continues to enroll patients. A planned interim analysis is expected to be announced in 2Q23. The Phase 2 PREVAIL study in Long COVID began enrolling patients in August, with an interim analysis planned for 2Q23.
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.
PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of molecularly targeted cancer and infectious disease immunotherapies based on the Company’s proprietary Versamune® and Infectimune™ T-cell activating technology platforms. Our Versamune®-based products have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. The Company’s pipeline products address various cancers including HPV16-associated cancers (anal, cervical, head and neck, penile, vaginal, vulvar) and breast, colon, lung, prostate and ovarian cancers.
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 To Be Presented At SITC. PDS Biotechnology released two PDS0101 abstracts to be presented at the SITC (Society for Immunotherapy of Cancer) meeting later this week. Data will be from the Phase 2 IMMUNOCERV trial in cervical cancer in combination with chemotherapy and radiation and the trial known as the Triple Combination Study or NCI Study, testing PDS0101 in combination with two immunomodulating agents. We believe both studies show positive results that are consistent with our expectations.
IMMUNOCERV Trial Highlights. The Phase 2 IMMUNOCERV trial is testing PDS0101 in combination with standard-of-care chemotherapy in advanced cervical cancer. Out of 17 patients enrolled in the study, 8 had been reached the 170-day status evaluation for by PET CT. Complete responses (CR) were seen in 7 out of 8 (87.5%) of the patients. One year survival was all 8 out of 8 (100%), with 7 out of 8 disease free (87.5%). One patient who only received 3 out of 5 doses showed signs of residual disease. The study is still ongoing.
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.
Patients with high-risk, locally advanced cervical cancer on IMMUNOCERV demonstrated increased tumor-infiltrating polyfunctional CD8+ (killer) T cells, and 1-year overall survival of 100%
Data from study across several checkpoint inhibitor refractory HPV-positive cancers demonstrate an increase in HPV-specific T cells following treatment with PDS0101-based triple combination
FLORHAM PARK, N.J., Nov. 07, 2022 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, today announced upcoming poster presentations of clinical data from two Phase 2 clinical trials of PDS0101 at the 37th Annual Meeting for the Society for Immunotherapy of Cancer (SITC 2022) being held November 8-12, 2022 in Boston. PDS0101 is PDS Biotech’s lead candidate being developed as a potential treatment for HPV-positive cancers.
The first abstract accepted for presentation, titled, “IMMUNOCERV, an ongoing Phase II trial combining PDS0101, an HPV-specific T cell immunotherapy, with chemotherapy and radiation for treatment of locally advanced cervical cancers,” highlights data from The University of Texas MD Anderson Cancer Center-led IMMUNOCERV Phase 2 clinical trial (NCT04580771). The study is investigating PDS0101 in combination with standard-of-care chemoradiotherapy (CRT) for the potential treatment of cervical cancer in patients with large tumors over 5cm in size and/or cancer that has spread to the lymph nodes (lymph node metastasis). Highlights from the study being presented at SITC 2022 include:
17 patients have been enrolled in the trial.
8 of the 17 patients had completed a Day 170 post-treatment Positron Emission Tomography, Computed Tomography (PET CT) scan to assess the status of the cancer.
87.5% (7/8) of patients treated with the combination of PDS0101 and CRT demonstrated a complete response (CR) on Day 170 by PET CT. One patient who received 3 of the 5 scheduled doses of PDS0101 showed signs of residual disease.
In comparison, 74.1% (40/54) of locally advanced patients who received CRT alone and were monitored at The University of Texas MD Anderson Cancer Center on a prospective protocol independent of IMMUNOCERV had a CR on PET CT at Day 170.
The 1-year overall survival is 100% (8/8) in patients treated with the combination of PDS0101 and CRT.
The observed 1-year disease-free survival rate for IMMUNOCERV patients is 87.5% (7/8).
Patients treated with the combination of PDS0101 and CRT had a 71% increase in multi-cytokine-inducing (polyfunctional) killer (CD8+) T cells within the tumors from baseline to end of treatment (38% to 65%). This increase in activated T cells was not seen in patients receiving standard-of-care CRT.
Toxicity of PDS0101 was limited to low-grade local injection site reactions.
The second abstract, titled “Immune Correlates Associated with Clinical Benefit in Patients with Checkpoint Refractory HPV-Associated Malignancies Treated with Triple Combination Immunotherapy,” reports data from the Phase 2 triple combination trial (NCT04287868), which is being led by the Center for Cancer Research at the National Cancer Institute (NCI), part of the National Institutes of Health. The study is investigating PDS0101 in combination with two investigational immune-modulating agents: M9241, a tumor-targeting IL-12 (immunocytokine), and bintrafusp alfa, a bifunctional checkpoint inhibitor (PD-L1/ TGF-β). The triple combination is being studied in checkpoint inhibitor (CPI)-naïve and -refractory patients with advanced HPV-positive anal, cervical, head and neck, vaginal, and vulvar cancers who have failed prior therapy. For most patients who are CPI refractory, there is no effective therapy. The immune correlates before and after treatment in the CPI refractory patient population were studied. Highlights from the study being presented at SITC 2022 include:
A more than two-fold increase in HPV16-specific T cells in the blood of 79% (11/14 tested) of the evaluated patients.
Immune responses were associated with increases in natural killer cells, soluble granzyme B (associated with active killer T cells), IFN-γ, TNF-α, etc., two weeks after the first treatment cycle thus signaling a pro-inflammatory response.
These immunogenicity findings highlight the potential role of the combination in altering immune suppressive forces, and support previously announced results documenting promising clinical outcomes in the CPI-refractory population receiving the triple combination.
“We are very pleased that research describing PDS0101’s therapeutic potential will be highlighted in two poster presentations at SITC 2022, including encouraging preliminary efficacy results from the ongoing IMMUNOCERV Phase 2 clinical trial,” said Dr. Frank Bedu-Addo, CEO of PDS Biotech. “Taken together, the data being presented at SITC 2022 demonstrate the potential ability of PDS0101 to elicit in patients the right type and quality of therapeutic immune response. This seems to allow PDS0101 to work in combination with a variety of therapeutic agents to generate clinical responses that appear to exceed current standards of care and allow for improved outcomes in patients with HPV-positive cancers. We look forward to continued progression of our Phase 2 clinical trials evaluating the efficacy, safety and tolerability of PDS0101 in combination with other therapies.”
Details of the posters being presented at SITC 2022 are as follows:
Abstract Number: 674 Abstract Title: IMMUNOCERV, an ongoing Phase II trial combining PDS0101, an HPV-specific T cell immunotherapy, with chemotherapy and radiation for treatment of locally advanced cervical cancers Presenting Author: Dr. Ann Klopp, The University of Texas MD Anderson Cancer Center Session Date: Friday, Nov. 11
Abstract Number: 695 Abstract Title: Immune correlates associated with clinical benefit in patients with checkpoint refractory HPV-associated malignancies treated with triple combination immunotherapy Presenting Author: Dr. Meg Goswami, National Cancer Institute Session Date: Thursday, Nov. 10
About PDS Biotechnology PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune® and Infectimune™ T cell-activating technology platforms. We believe our targeted Versamune® based candidates have the potential to overcome the limitations of current immunotherapy by inducing large quantities of high-quality, potent polyfunctional tumor specific CD4+ helper and CD8+ killer T cells. To date, our lead Versamune® clinical candidate, PDS0101, has demonstrated the potential to reduce tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV-expressing cancers in multiple Phase 2 clinical trials. Our Infectimune™ based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.
About PDS0101
PDS Biotech’s lead candidate, PDS0101, combines the utility of the Versamune® platform with targeted antigens in HPV-expressing cancers. In partnership with Merck & Co., PDS Biotech is evaluating a combination of PDS0101 and KEYTRUDA® in a Phase 2 study in first-line treatment of recurrent or metastatic head and neck cancer, and also in second line treatment of recurrent or metastatic head and neck cancer in patients who have failed prior checkpoint inhibitor therapy. A National Cancer Institute-supported Phase 2 clinical study of PDS0101 in a triple combination therapy is also being conducted in checkpoint inhibitor refractory patients with multiple advanced HPV-associated cancers. A third Phase 2 clinical trial in first line treatment of locally advanced cervical cancer is being led by The University of Texas MD Anderson Cancer Center. A final Phase 2 clinical trial of PDS0101 monotherapy in first line treatment of newly diagnosed patients HPV16+ head and neck cancer patients is being conducted at the Mayo Clinic.
KEYTRUDA® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.
Forward Looking Statements This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune® and Infectimune™ based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune® and Infectimune™ based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
Versamune® is a registered trademark and Infectimune™ is a trademark of PDS Biotechnology.
Five Potentially Pivotal Phase 2 or 3 Studies for CNS Programs Expected to be in the Clinic by First Quarter 2023
Data from Planned Interim Analyses of TNX-102 SL in Phase 3 Fibromyalgia Study and Phase 2 Long COVID Study Expected Second Quarter 2023
Advanced Development Center in Dartmouth, Mass. and Infectious Disease Research and Development Facility in Frederick, Md. Operational
Cash and Cash Equivalents Totaled Approximately $140 Million at September 30, 2022
CHATHAM, N.J., Nov. 07, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced financial results for the third quarter ended September 30, 2022, and provided an overview of recent operational highlights.
“Tonix continues to make meaningful progress in the development of multiple programs within its robust pipeline, having already commenced a confirmatory Phase 3 study for fibromyalgia in the second quarter of this year and a potentially pivotal Phase 2 study for Long COVID in the third quarter of this year,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “We look forward to the interim data from both of these TNX-102 SL studies in the second quarter of 2023. Additionally, we look forward to advancing our other central nervous system or CNS product candidates including TNX-102 SL for PTSD, TNX-1900 for chronic migraine, TNX-1300 for cocaine intoxication, and TNX-601 ER for depression, all of which we expect to be in the clinic by first quarter 2023. Finally, we continue to make strides in immunology with TNX-1500 for preventing organ transplant rejection expected to enter into a Phase 1 study in the first half of 2023, as well as in infectious diseases with TNX-801, a vaccine to prevent smallpox and monkeypox, expected to enter into a Phase 1 study also in the first half of next year.”
Recent Highlights—Key Product Candidates*
Central Nervous System (CNS) Pipeline
TNX-102 SL (cyclobenzaprine HCl sublingual tablet): small molecule for the management of fibromyalgia (FM)
Enrollment continues in the RESILIENT study, a double-blind, randomized, placebo-controlled, potentially pivotal confirmatory Phase 3 study of TNX-102 SL for the management of fibromyalgia. Results from a planned interim analysis are expected in the second quarter of 2023.
TNX-102 SL for the treatment of Long COVID, also known as Post-Acute Sequelae of COVID-19 (PASC)
Enrollment continues in the PREVAIL study, a potentially pivotal Phase 2 study of TNX-102 SL for Long COVID. Results from a planned interim analysis are currently anticipated in the second quarter of 2023.
Tonix presented data from a previously announced retrospective observational database study in patients with Long COVID at the International Association for the Study of Pain (IASP) 2022 World Congress on Pain. 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,” included data showing that approximately 40% of patients had fibromyalgia-like multi-site pain, the rate of opioid use in Long COVID patients with multi-site pain was 34%, which increased to approximately 50% when sleep disturbance was also present. These findings support the feasibility of the currently enrolling Phase 2 study for patients with Long COVID whose symptoms overlap with fibromyalgia.
TNX-102 SL for the treatment of Posttraumatic Stress Disorder (PTSD)
Tonix expects to begin enrolling a Phase 2 study of TNX-102 SL in police in Kenya in the fourth quarter of 2022.
Tonix expects to initiate a new, potentially pivotal, Phase 2 clinical study of TNX-1300 for the treatment of cocaine intoxication in the first quarter of 2023, pending agreement with the U.S. Food and Drug Administration (FDA).
In August 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.
TNX-1900 (intranasal potentiated oxytocin): small peptide for migraine, craniofacial pain, insulin resistance and related disorders, and obesity associated binge eating disorder
The Company expects to begin enrollment in a Phase 2 study of TNX-1900 for the prevention of migraine headache in chronic migraineurs in the fourth quarter of 2022.
Tonix announced that U.S. Patent 11,389,473 issued in July 2022. The patent, entitled “Magnesium-Containing Oxytocin Formulations and Methods of Use” claims methods and compositions for treating pain, including migraine headaches, using intranasal magnesium-containing oxytocin formulations. This patent, excluding possible patent term extensions, is expected to provide Tonix with U.S. market exclusivity until January 2036.
TNX-601 ER (tianeptine hemioxalate extended-release tablets): a once-daily small molecule for the treatment of major depressive disorder (MDD), PTSD, and neurocognitive dysfunction associated with corticosteroid use.
In October 2022, Tonix announced that the FDA has cleared the Investigational New Drug (IND) application to support a Phase 2 study of TNX-601 ER for the treatment of MDD, which the Company expects to initiate in the first quarter of 2023.
TNX-601 ER is being developed as a monotherapy and first-line treatment for MDD. No tianeptine-containing product has been approved by the FDA.
Rare Disease Pipeline
TNX-2900 (intranasal potentiated oxytocin): small peptide for the treatment of Prader-Willi syndrome (PWS)
In July 2022, 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 World Orphan Drug Congress USA.
TNX-2900 has been granted Orphan Drug designation from the FDA for the treatment of PWS.
Immunology Pipeline
TNX-1500 (anti-CD40L monoclonal antibody): third generation monoclonal antibody for prophylaxis of organ transplant rejection and treatment of autoimmune disorders.
Tonix announced data from three oral presentations at the 29th International Congress of The Transplantation Society (TTS 2022) by faculty at the Center for Transplantation Sciences, Massachusetts General Hospital for TNX-1500 targeting CD40-ligand (CD40L), which is also known as CD154.
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. These presentations suggested that 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. These presentations suggested that blockade of CD40L with TNX-1500 monotherapy consistently and safely prevented pathologic xenoimmunity in non-human primate kidney xenograft models without clinical thrombosis.
A Phase 1 study of TNX-1500 is expected to start in the first half of 2023.
Infectious Disease Pipeline
TNX-801 (live horsepox virus vaccine for percutaneous administration): vaccine against smallpox and monkeypox designed as a single-administration vaccine to elicit T cell immunity
As previously mentioned, Tonix announced a collaboration with the Kenya Medical Research Institute (KEMRI) to plan, seek regulatory approval for and conduct a Phase 1 clinical study in Kenya to develop TNX-801 as a vaccine to protect against monkeypox and smallpox. The study is expected to start in the first half of 2023.
Tonix presented data from a research collaboration with The University of Alberta in a poster presentation at the 4th Symposium of the Canadian Society for Virology on June 5, 2022. The poster titled, “Synthetic Chimeric Horsepox Virus (scHPXV) Vaccination Protects Macaques from Monkeypox,” describes data from animals vaccinated with TNX-801 to protect against monkeypox. The poster presentation reports that all animals (n=8) vaccinated with TNX-801 were fully protected with sterilizing immunity from a challenge with intra-tracheal monkeypox. The vaccinations with TNX-801 were well tolerated. Synthetic horsepox virus is the basis for the Company’s TNX-801 vaccine in development to protect against monkeypox and smallpox and for the Company’s Recombinant Pox Virus (RPV) platform to protect against other pathogens, including SARS-CoV-2.
*All of Tonix’s product candidates are investigational new drugs or biologics and have not been approved for any indication.
Recent Highlights–Financial
As of September 30, 2022, Tonix had $140.0 million of cash and cash equivalents, compared to $178.7 million as of December 31, 2021.
In October 2022, Tonix issued 1,400,000 shares of Series A convertible redeemable preferred stock and 100,000 shares of Series B convertible redeemable preferred stock to certain institutional investors in a private placement for gross proceeds of $15.0 million. The Company expects to use the proceeds to redeem the preferred stock.
Cash used in operations was approximately $23.5 million for the three months ended September 30, 2022, compared to $12.9 million for the same period in 2021. The increase in cash outlays was primarily due to an increase in research and development activities. Capital expenditures were approximately $8.8 million for the three months ending September 30, 2022 compared to $7.8 million for the same period in 2021. The increase was primarily due to the continued buildout of the ADC in North Dartmouth, Mass.
Third Quarter 2022 Financial Results
Research and development (R&D) expenses for the three months ended September 30, 2022 were $22.2 million, compared to $13.1 million for the same period in 2021. The increase is predominately due to increased clinical, manufacturing, non-clinical, employee-related and laboratory expenses. The Company continues to expect R&D expenses to increase throughout the remainder of 2022 as it moves its clinical development programs forward and invests in its development pipeline.
General and administrative (G&A) expenses for the three months ended September 30, 2022 were $7.4 million, compared to $5.5 million for the same period in 2021. The increase is primarily due to increased employee-related and financial reporting expenses.
Net loss available to common stockholders was $29.0 million, or $0.69 per share, basic and diluted, for the three months ended September 30, 2022, compared to net loss of $18.5 million, or $1.60 per share, basic and diluted, for the same period in 2021. The basic and diluted weighted average common shares outstanding for the three months ended September 30, 2022 was 41,944,289 compared to 11,581,367 shares for the same period in 2021.
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 second 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 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. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily formulation of tianeptine being developed as a potential treatment for major depressive disorder (MDD) with a Phase 2 study expected to be initiated in the first 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 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.
*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.
TONIX PHARMACEUTICALS HOLDING CORP. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (In Thousands, Except Share and Per Share Amounts) (unaudited)
Three Months Ended September 30,
Nine Months Ended September 30,
2022
2021
2022
2021
COSTS AND EXPENSES:
Research and development
$
22,201
$
13,082
$
57,202
$
46,542
General and administrative
7,390
5,453
22,161
16,291
29,591
18,535
79,363
62,833
Operating loss
(29,591
)
(18,535
)
(79,363
)
(62,833
)
Interest income, net
610
7
825
99
Net loss
(28,981
)
(18,528
)
(78,538
)
(62,734
)
Preferred stock deemed dividend
—
—
4,255
—
Net loss available to common stockholders
$
(28,981
)
$
(18,528
)
$
(82,793
)
$
(62,734
)
Net loss per common share, basic and diluted
$
(0.69
)
$
(1.60
)
$
(3.06
)
$
(6.02
)
Weighted average common shares outstanding, basic and diluted
1The condensed consolidated balance sheet for the year ended December 31, 2021 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.
What is Inflammation? Two Immunologists Explain How the Body Responds to Everything from Stings to Vaccination and Why it Sometimes Goes Wrong
When your body fights off an infection, you develop a fever. If you have arthritis, your joints will hurt. If a bee stings your hand, your hand will swell up and become stiff. These are all manifestations of inflammation occurring in the body.
We are two immunologists who study how the immune system reacts during infections, vaccination and autoimmune diseases where the body starts attacking itself.
This article was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts. It represents the research-based findings and thoughts of Prakash Nagarkatti, Professor of Pathology, Microbiology and Immunology, University of South Carolina and Mitzi Nagarkatti Professor of Pathology, Microbiology and Immunology, University of South Carolina
While inflammation is commonly associated with the pain of an injury or the many diseases it can cause, it is an important part of the normal immune response. The problems arise when this normally helpful function overreacts or overstays its welcome.
What is Inflammation?
Generally speaking, the term inflammation refers to all activities of the immune system that occur where the body is trying to fight off potential or real infections, clear toxic molecules or recover from physical injury. There are five classic physical signs of acute inflammation: heat, pain, redness, swelling and loss of function. Low-grade inflammation might not even produce noticeable symptoms, but the underlying cellular process is the same.
Take a bee sting, for example. The immune system is like a military unit with a wide range of tools in its arsenal. After sensing the toxins, bacteria and physical damage from the sting, the immune system deploys various types of immune cells to the site of the sting. These include T cells, B cells, macrophages and neutrophils, among other cells.
The B cells produce antibodies. Those antibodies can kill any bacteria in the wound and neutralize toxins from the sting. Macrophages and neutrophils engulf bacteria and destroy them. T cells don’t produce antibodies, but kill any virus-infected cell to prevent viral spread.
Additionally, these immune cells produce hundreds of types of molecules called cytokines – otherwise known as mediators – that help fight threats and repair harm to the body. But just like in a military attack, inflammation comes with collateral damage.
The mediators that help kill bacteria also kill some healthy cells. Other similar mediating molecules cause blood vessels to leak, leading to accumulation of fluid and influx of more immune cells.
This collateral damage is the reason you develop swelling, redness and pain around a bee sting or after getting a flu shot. Once the immune system clears an infection or foreign invader – whether the toxin in a bee sting or a chemical from the environment – different parts of the inflammatory response take over and help repair the damaged tissue.
After a few days, your body will neutralize the poison from the sting, eliminate any bacteria that got inside and heal any tissue that was harmed.
Asthma is caused by inflammation that leads to swelling and a narrowing of airways in the lungs, as seen in the right cutaway in this image. BruceBlaus/Wikimedia Commons, CC BY-SA
Inflammation as a Cause of Disease
Inflammation is a double-edged sword. It is critical for fighting infections and repairing damaged tissue, but when inflammation occurs for the wrong reasons or becomes chronic, the damage it causes can be harmful.
Allergies, for example, develop when the immune system mistakenly recognizes innocuous substances – like peanuts or pollen – as dangerous. The harm can be minor, like itchy skin, or dangerous if someone’s throat closes up.
Chronic inflammation damages tissues over time and can lead to many noninfectious clinical disorders, including cardiovascular diseases, neurodegenerative disorders, obesity, diabetes and some types of cancers.
The immune system can sometimes mistake one’s own organs and tissues for invaders, leading to inflammation throughout the body or in specific areas. This self-targeted inflammation is what causes the symptoms of autoimmune diseases such as lupus and arthritis.
Another cause of chronic inflammation that researchers like us are currently studying is defects in the mechanisms that curtail inflammation after the body clears an infection.
While inflammation mostly plays out at a cellular level in the body, it is far from a simple mechanism that happens in isolation. Stress, diet and nutrition, as well as genetic and environmental factors, have all been shown to regulate inflammation in some way.
There is still a lot to be learned about what leads to harmful forms of inflammation, but a healthy diet and avoiding stress can go a long way toward helping maintain the delicate balance between a strong immune response and harmful chronic inflammation.
CARLSBAD, Calif.–(BUSINESS WIRE)–Nov. 3, 2022– Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today announced that it will report its third quarter 2022 financial and operating results on Thursday, November 10, 2022, following the close of the U.S. financial markets. Lineage management will also host a conference call and webcast on Thursday, November 10, 2022, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its third quarter 2022 financial and operating results and to provide a business update.
Interested parties may access the conference call by dialing (800) 715-9871 from the U.S. and Canada and should request the “Lineage Cell Therapeutics Call” or provide conference ID number5262180. A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through November 17, 2022, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 5262180.
About Lineage Cell Therapeutics, Inc.
Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical and preclinical programs are in markets with billion dollar opportunities and include five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelial cell therapy in development for the treatment of geographic atrophy secondary to age-related macular degeneration, is being developed under a worldwide collaboration with Roche and Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer; (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy; and (v) PNC1, a photoreceptor neural cell therapy for the potential treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit www.lineagecell.com or follow the company on Twitter @LineageCell.
BOTHELL, Wash., Nov. 02, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) announces that management will participate in two upcoming investor conferences as follows:
2022 BioFlorida Annual Conference: James Martin, CFO and interim co-CEO, will participate in a panel titled “BioTrends: Learning from the Pandemic: The Fight Against Infectious Diseases” on Thursday, November 3 at 2:05 p.m. Eastern time. The conference is being held at Hyatt Regency Miami. More information about the conference is available here.
Q4 Investor Summit: Mr. Martin will present a company overview on Monday, November 14 at 11:30 a.m. Eastern time. The conference is sponsored by the Investor Summit Group and is being held at the Sheraton New York Time Square Hotel. More information about the conference is available here.
A webcast of the Q4 Investor Summit presentation will be posted to the IR Calendar page of the company website.
About Cocrystal Pharma, Inc. Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), hepatitis C viruses and noroviruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.
Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product, Covaxin, is a killed-virus vaccine for COVID-19 in-licensed from Bharat Biotech (India). The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.
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.
Gene Therapy In The Spotlight. Ocugen held an R&D Day to highlight its programs in gene therapy and COVID-19. The presentations discussed the novel mechanisms behind its gene therapy products and the nature of the ophthalmic disease indications in development. While the company has been best known for COVAXIN and COVID-19, we have always seen the gene therapy indications as highly promising and underappreciated.
Modifier Gene Therapy Platform. The first presentations discussed the company’s novel approach to gene therapy. In contrast to other approaches that seek to transfer a functional copy of a gene to replace a gene that has mutated or become dysfunctional, Ocugen’s Modifier Gene Therapy targets a “master control gene” that controls the expression of multiple gene pathways, allowing products to be developed for diseases caused by downstream gene pathways and combinations of genetic mutations.
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.
Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using compositions of endogenous metabolic modulators (EMMs). The company’s product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to restore cellular homeostasis in multiple key biological pathways and improve cellular energetic efficiency. Axcella’s pipeline includes lead therapeutic candidates in Phase 2 development for the treatment of Long COVID and non-alcoholic steatohepatitis (NASH), and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. The company’s unique model allows for the evaluation of its EMM compositions through non-IND clinical studies or IND clinical trials. For more information, please visit www.axcellatx.com.
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.
Financial Results. Axcella reported a 3Q22 loss of $17.8 million or $(0.34) per share, compared with our estimate of a loss of $21.7 million or $(0.43) per share. The company sold convertible notes raising $6.0 million to end the quarter with $25.4 million in cash and equivalents. After the quarter ended, it added $28.2 million in cash through a direct offering of common stock. Based on our 4Q22 estimate of a loss of $18.1 million, we project the cash balance at YE22 to be around $35.5 million.
Long COVID Study Results Were Encouraging. Results from a Phase 2a study testing AXA1125 in Long COVID were announced in early August, as discussed in our note from August 3. The data showed statistically significant improvements in both physical and mental fatigue symptoms. We believe this is clinically relevant for an estimated 20% to 30% of the people who have been infected with COVID-19. Axcella is in currently in discussions with regulatory authorities to design the next study, planned to begin in 2023.
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.
BOTHELL, Wash., Nov. 01, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) announces it has been granted European patent EP3866778 titled “Combinations Of Inhibitors of Influenza Virus Replication,” which covers the Company’s broad-spectrum oral PB2 inhibitor CC-42344 in combination with approved influenza antiviral drugs including Tamiflu®, Xofluza® or Favipiravir. Cocrystal recently announced the completion of enrollment in a Phase 1 study with CC-42344 for the treatment of pandemic and seasonal influenza A, with topline study results expected later this year.
“This new patent provides meaningful protection for our PB2 clinical program in the European Union and is the first to be granted as we pursue a similar patent strategy in other jurisdictions,” said James Martin, Cocrystal’s CFO and interim Co-CEO. “The granting of this patent is an important step in our efforts to expand protection of our novel, best-in-class antiviral candidates both in the U.S. and internationally.”
About CC-42344 and Influenza CC-42344 is an oral PB2 inhibitor discovered using Cocrystal’s proprietary structure-based drug discovery platform technology. It is specifically designed to be effective against all significant pandemic and seasonal influenza A strains and to have a high barrier to resistance due to the way the virus’ replication machinery is targeted. CC-42344 targets the influenza polymerase, an essential replication enzyme with several highly essential regions common to multiple influenza strains. In vitro testing showed CC-42344’s excellent antiviral activity against influenza A strains, including pandemic and seasonal strains, as well as against strains resistant to Tamiflu® and Xofluza®, while also demonstrating favorable PK and safety profiles.
According to a June 2022 report by Precision Reports, the global influenza therapeutics market is projected to reach $9.5 billion by 2027, up from $6.6 billion in 2020 and growing at a 4.8% CAGR between 2021 and 2027.
About Cocrystal Pharma, Inc. Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), hepatitis C viruses and noroviruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.
Cocrystal Pharma Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements the potential design and efficacy of CC-42344, and the demand and potential market size for products designed to treat influenza. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, the possibility that assumptions underlying market projections prove to be incorrect, the availability of federal government funding and budgetary issues that may arise, the risks and uncertainties arising from any future impact of the Russian invasion of Ukraine, and/or inflation and interest rate increases on the global economy, the U.K. and on our Company and collaboration partners, including supply chain disruptions and our continued ability to proceed with our programs such as obtaining the requisite regulatory approvals including from the United Kingdom Medicines and Healthcare Products Regulatory Agency, the ability of the CRO to recruit patients into clinical trials, and the results of the studies for CC-42344. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2021. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.
Promising Results from Phase 2a Placebo Controlled Clinical Trial for Long COVID
Positive Interim Data from Phase 2b EMMPACT Study of AXA1125 in Nonalcoholic Steatohepatitis (NASH)
$34.2 Million Registered Direct Offering of Common Stock Priced At The Market
Appointment of Two New Board Members
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Nov. 1, 2022– Axcella Therapeutics (Nasdaq: AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, today announced financial results for the third quarter ended September 30, 2022 and provided a business update.
“This has been an extremely exciting period for Axcella,” said Bill Hinshaw President and Chief Executive Officer of Axcella. “Through the third quarter, we made important progress in our clinical development of AXA1125 and strengthened our balance sheet with a new financing. Specifically, we reported positive data from both our Phase 2 study of Long COVID and our Phase 2b EMMPACT study in non-alcoholic steatohepatitis (NASH). In addition, we completed a financing worth $34.2 million that will allow us to advance the Long COVID program and our EMMPACT Phase 2b clinical trial in NASH.”
Mr. Hinshaw continued, “As the company advances toward late-stage clinical trials, we are pleased to have the additions of Mr. Rosiello and Mr. Straight Nissen to our Board, whose extensive leadership experience will help guide Axcella as we focus on taking steps toward delivering these treatments to patients.”
Clinical Results in the Quarter:
The quarter saw progress in the clinical development of AXA1125, an orally delivered potent and safe compound addressing two very large markets: Long-COVID and NASH. This included the release of top-line results in Long COVID and an interim analysis of the Phase 2b EMMPACT trial in NASH.
Long COVID is a persistent and growing challenge of the pandemic, affecting an estimated one hundred million patients worldwide, with fatigue as the most common symptom. In a release of top-line results, subjects who received AXA1125 had improvements in measures of mental and physical fatigue that were both highly statistically significant and clinically relevant compared to those who received placebo. Mean changes in total, physical and mental scores in the CFQ-11 versus placebo were -4.30 (p=0.0039), -2.94 (p=0.0097) and -1.32 (p=0.0097), respectively.
NASH affects an estimated 40 million people in the U.S., including approximately 10% of U.S. children. The complex pathogenesis of NASH includes dysregulation of metabolism, inflammation and fibrosis. In this interim analysis, at 24-weeks there were statistically significant improvements in the liver stiffness measurement (LSM) using vibration-controlled transient elastography compared to placebo in the high dose arm for all subjects. Absolute changes in LSM were 0.13, -2.01, and -4.07 kilopascals (kPa) in the placebo, low dose and high dose arms, respectively (p= 0.0992 and 0.0096 for the low and high dose, respectively, compared to placebo). These results were supported by statistically significant improvements in other non-invasive measures of liver fibrosis: ELF and FIB-4. Statistically significant improvements in alanine aminotransferase (ALT) were seen at both weeks 12 and 24 in all subjects (placebo-adjusted difference of -28.61% (p=0.0183) and -36.3% (p=0.0017) for the low and high doses, respectively). All subjects experienced significantly greater changes from baseline in MRI-PDFF at 12-weeks compared to the change from baseline in the placebo group (placebo adjusted difference of -18.98% (p=0.0082) and -21.24% (p=0.0014) for the low and high doses, respectively).
Financing and Board:
In September 2022, the Company received $6.0 million from the issuance of convertible notes to funds associated with Flagship Pioneering. In October 2022, the Company secured an additional $28.2 million in gross proceeds through a registered direct offering of common stock, which also resulted in the conversion of the convertible notes into common stock. In total, the Company sold 20,847,888 shares of common stock at a purchase price of $1.64 per share. Since this offering was made without an underwriter or a placement agent, Axcella did not pay any underwriting or placement agent commissions. Simultaneous with the financing, the company appointed Robert Rosiello and Torben Straight Nissen to its Board of Directors, and Mr. Rosiello also became Chairman of the Board. After five years of service, David Epstein stepped off the Board, and will continue to support the Company as a consultant.
Financial Results:
Cash Position: As of September 30, 2022, cash, cash equivalents, and marketable securities totaled $25.4 million, compared to $55.0 million as of December 31, 2021. In September 2022, the Company received $6.0 million from the issuance of convertible notes to funds associated with Flagship Pioneering and, in October 2022, the Company secured an additional $28.2 million in gross proceeds through a registered direct offering of common stock. As part of the registered direct offering, the $6.0 million convertible notes automatically converted into the Company’s common stock. Axcella expects that its current cash balance will be sufficient to meet its operating needs into the second quarter of 2023, provided that, if the Company is unable to satisfy the cash covenants contained in its loan and security agreement with SLR Investment Corp., and SLR Investment Corp. seeks immediate repayment of the loan in full, the Company believes that its cash and cash equivalents will be sufficient to fund its operations into the late first quarter of 2023.
R&D Expenses: Research and development expenses for the quarter and nine months ended September 30, 2022 were $13.3 million and $43.7 million, respectively. Research and development expenses for the same periods ended September 30, 2021 were $10.1 million and $30.7 million, respectively. These increases are the result of the Company’s EMMPACT and Long COVID Phase 2 clinical trials, as well as closure costs for its EMMPOWER Phase 2 clinical trial.
G&A Expenses: General and administrative expenses for the quarter and nine months ended September 30, 2022 were $3.8 million and $12.3 million, respectively. General and administrative expenses for the same periods ended September 30, 2021 were $4.8 million and $14.0 million. These decreases are primarily the result of lower non-cash stock-based compensation expenses.
Net Loss: Net loss for the quarter and nine months ended September 30, 2022 was $17.8 million, or $0.34 per basic and diluted share, and $58.2 million, or $1.19 per basic and diluted share, respectively. This compares with a net loss of $15.6 million, or $0.41 per basic and diluted share, and $46.7 million, or $1.23 per basic and diluted share, for the quarter and nine months ended September 30, 2021.
Internet Posting of Information
Axcella uses the “Investors and News” section of its website, www.axcellatx.com, as a means of disclosing material nonpublic information, to communicate with investors and the public, and for complying with its disclosure obligations under Regulation FD. Such disclosures include, but may not be limited to, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, and public conference calls and webcasts. The information that we post on our website could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
About Axcella Therapeutics (Nasdaq: AXLA)
Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using compositions of endogenous metabolic modulators (EMMs). The company’s product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to restore cellular homeostasis in multiple biological pathways, improve cellular energetics and restore homeostasis. Axcella’s pipeline includes lead therapeutic candidates in Phase 2 development for the treatment of Long COVID, and NASH. The company’s unique model allows for the evaluation of its EMM compositions through non-IND clinical studies or IND clinical trials. For more information, please visit www.axcellatx.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the timing of the company’s clinical trial data readouts, its expected cash runway and the expected benefits of Mr. Rosiello’s and Mr. Straight Nissen’s service on the Board of Directors of Axcella. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company’s ability to conduct and complete its ongoing or planned clinical studies and clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its clinical trials of AXA1125, other potential impacts of COVID-19 on the company’s business and financial results, including with respect to its ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company’s clinical trial plans and timing, clinical trial design and target indications for AXA1125, the clinical development and safety profile of AXA1125 and its therapeutic potential, whether and when, if at all, the company’s product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company’s target indications, and other risks identified in the company’s SEC filings, including Axcella’s Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.
DENVER, Nov. 1, 2022 /CNW/ – Medicine Man Technologies operating as Schwazze, (OTCQX: SHWZ) (NEO: SHWZ) (“Schwazze” or the “Company”), is pleased to announce that Justin Dye, Chairman & CEO; Nirup Krishnamurthy, President; and Nancy Huber, CFO will host the Schwazze Third Quarter conference call on November 9, 2022, at 5:00 pm ET.
Investors and stakeholders may participate in the conference call by dialing 416-764-8650 or by dialing North American toll free 1-888-664-6383 or listen to the webcast from the Company’s website at https://ir.schwazze.com The webcast will be available on the Company’s website and on replay until November 16, 2022, and may be accessed by dialing 1-888-390-0541 / 997573 #.
Following their prepared remarks, Chief Executive Officer, Justin Dye; President, Nirup Krishnamurthy; and Chief Financial Officer, Nancy Huber will answer investor questions. Investors may submit questions in advance or during the conference call itself through the weblink: https://app.webinar.net/x0q6rpnP84n This weblink has been posted to the Company’s website and will be archived on the website. All Company SEC filings can also be accessed on the Company website at https://ir.schwazze.com/sec-filings
Upcoming Events: MJ BizCon – Las Vegas, November 16-18, 2022 Justin Dye, Chairman & CEO and Nancy Huber, CFO will be attending MJ BizCon Las Vegas November 16-18 at the Las Vegas Convention Centre. Management will also be participating in one-on-one investor meetings at the event (Booth #3007). For more information or to schedule a meeting please contact Joanne Jobin, IRO at [email protected].
Benchmark 11th Annual Discovery Conference – New York City – December 1, 2022 Justin Dye, Chairman & CEO and Nancy Huber, CFO will participate in the afternoon session of the Benchmark 11th Annual Discovery Conference at the New York Athletic Club, 180 Central Park South in Manhattan. Management will be participating in one-on-one investor meetings throughout the conference. For more information, please contact your Benchmark representative.
About Schwazze Schwazze (OTCQX:SHWZ, NEO:SHWZ) is building a premier vertically integrated regional cannabis company with assets in Colorado and New Mexico and will continue to take its operating system to other states where it can develop a differentiated regional leadership position. Schwazze is the parent company of a portfolio of leading cannabis businesses and brands spanning seed to sale. The Company is committed to unlocking the full potential of the cannabis plant to improve the human condition. Schwazze is anchored by a high-performance culture that combines customer-centric thinking and data science to test, measure, and drive decisions and outcomes. The Company’s leadership team has deep expertise in retailing, wholesaling, and building consumer brands at Fortune 500 companies as well as in the cannabis sector. Schwazze is passionate about making a difference in our communities, promoting diversity and inclusion, and doing our part to incorporate climate-conscious best practices. Medicine Man Technologies, Inc. was Schwazze’s former operating trade name. The corporate entity continues to be named Medicine Man Technologies, Inc.
Schwazze derives its name from the pruning technique of a cannabis plant to enhance plant structure and promote healthy growth.
Forward-Looking Statements This press release contains “forward-looking statements.” Such statements may be preceded by the words “plan,” “will,” “may,”, “predicts,” or similar words. Forward-looking statements are not guarantees of future events or performance, are based on certain assumptions, and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control and cannot be predicted or quantified. Consequently, actual events and results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) our inability to manufacture our products and product candidates on a commercial scale on our own or in collaboration with third parties; (ii) difficulties in obtaining financing on commercially reasonable terms; (iii) changes in the size and nature of our competition; (iv) loss of one or more key executives or scientists; (v) difficulties in securing regulatory approval to market our products and product candidates; (vi) our ability to successfully execute our growth strategy in Colorado and outside the state, (vii) our ability to consummate the acquisition described in this press release or to identify and consummate future acquisitions that meet our criteria, (viii) our ability to successfully integrate acquired businesses and realize synergies therefrom, (ix) the ongoing COVID-19 pandemic, * the timing and extent of governmental stimulus programs, (xi) the uncertainty in the application of federal, state and local laws to our business, and any changes in such laws, and * out ability to satisfy the closing conditions for the private finding described in this press release. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company’s filings with the Securities and Exchange Commission (SEC), including the Company’s Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC’s website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise except as required by law.
