Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-15001 which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-29002 for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-8013, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-18504, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-35005 (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL6, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022. Finally, TNX-13007 is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
RESILIENT Study Shows Strong Results In Fibromyalgia. Tonix announced that the Phase 3 RESILIENT study testing TNX-102 SL in fibromyalgia met its primary endpoint of pain reduction and its six secondary endpoints. The fibromyalgia population is estimated at 6 to 12 million patients, making this a significant market for Tonix. The company plans to submit an application for FDA approval in 2H2024, consistent with our projected product launch in 2025.
Tonix Raised Funds With Stock and Warrants. The company also announced a $144 million Registered Direct offering that will raise $30 million upon closing and a potential $114 million through warrant exercise. We believe the offering offset the positive news from the RESILIENT clinical trial, causing the stock to decline despite good clinical news.
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*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.
Phase 3 RESILIENT study of TNX-102 SL successfully demonstrated daily pain reduction over placebo (primary endpoint, p = 0.00005)
All six key secondary endpoints, including patient global impression, fibromyalgia-specific symptoms and dysfunction, fatigue and sleep measures were significantly improved (all p ≤ 0.001)
Positive results support planned New Drug Application (NDA) submission to the FDA in the second half of 2024 An estimated 6 million to 12 million adults in the U.S. are living with fibromyalgia, the majority of whom are women
CHATHAM, N.J., Dec. 20, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that the Phase 3 RESILIENT study evaluating TNX-102 SL (cyclobenzaprine HCl sublingual tablets) met its pre-specified primary endpoint in the second of two positive Phase 3 clinical trials, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia (Table 1). Statistically significant and clinically meaningful results were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function. Additionally, as it relates to improving daily pain, treatment with TNX-102 SL showed a robust and clinically meaningful analgesic effect size of 0.38, with rapid onset of action, separating from placebo for each week of the study. TNX-102 SL was well tolerated with an adverse event profile comparable to prior studies, and no new safety signals were observed. Tonix plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2024 for TNX-102 SL for the management of fibromyalgia. An estimated 6 million to 12 million U.S. adults are living with fibromyalgia, the majority of whom are women.
TNX-102 SL is a tablet formulation containing 2.8 mg cyclobenzaprine HCl and is a novel, centrally-acting, non-opioid analgesic, designed to be taken once daily at bedtime for the management of fibromyalgia. RESILIENT was a 14-week randomized, double-blind, placebo-controlled trial of TNX-102 SL 5.6 mg, in which 457 participants with fibromyalgia were randomized in a 1:1 ratio to TNX-102 SL or placebo across 33 sites in the U.S. All participants received one 2.8 mg tablet of TNX-102 SL (2.8 mg) or placebo for the first 2 weeks, which was increased to two 2.8 mg tablets of TNX-102 SL (5.6 mg) or placebo for the remaining 12 weeks.
In December 2020, Tonix reported positive results from the first Phase 3 RELIEF study of TNX-102 SL 5.6 mg for the management of fibromyalgia. The RELIEF study met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.010) in participants with fibromyalgia, and showing activity in key secondary endpoints.
“We believe that the positive results of RESILIENT and RELIEF show that fibromyalgia can be successfully treated by TNX-102 SL 5.6 mg and may provide the opportunity for Tonix to have the first FDA-approved drug for fibromyalgia in more than a decade,” said Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals. “We are now an important step closer to bringing a new, first-line treatment to fibromyalgia patients that offers broad symptom relief and favorable tolerability for chronic use and adherence. We believe that we are well positioned to submit an NDA to the FDA under the 505(b)(2) regulatory approval pathway in the second half of 2024, and are on track to supply the U.S. market upon FDA approval.”
Table 1. Results of Primary and Secondary Endpoints for the Phase 3 RESILIENT Study of TNX-102 SL
Outcome Measure at Week 14
Intent-to-Treat Analysis1
P-value
Primary Endpoint
Daily Pain Diary, NRS
Mean Change from Baseline2
0.00005*
Key Secondary Endpoints
Non-specific
Patient Global Impression of Change
Proportion “Much” or “Very Much Improved”3
<0.001*
Fibromyalgia Syndrome-Related
FIQ-R Symptom Domain
Mean Change from Baseline
<0.001*
FIQ-R Function Domain
Mean Change from Baseline
0.001*
PROMIS Sleep Disturbance
Mean Change from Baseline
<0.001*
PROMIS Fatigue
Mean Change from Baseline
<0.001*
Daily Sleep Quality Diary, NRS
Mean Change from Baseline
<0.001*
Abbreviations: FIQ-R = Fibromyalgia Impact Questionnaire – Revised; NRS = Numeric Rating Scale; PROMIS = Patient-Reported Outcomes Measurement Information System
*Statistically significant; to control for overall type 1 error, a pre-specified, serial gatekeeping procedure was utilized.
1Analysis by mixed model repeated measures with multiple imputation unless otherwise indicated.
2Primary endpoint analysis for FDA approvals of Cymbalta®and Lyrica® in fibromyalgia.
3Pearson’s chi-squared test responder analysis, with missing data considered non-responders
“These data are terrific news for patients with fibromyalgia,” said Daniel J. Clauw, M.D., Professor of Anesthesiology, Medicine and Psychiatry at the University of Michigan. “Despite approved medications, there remains a need for new treatment options to better address the quality of life impacts many fibromyalgia patients experience on a chronic basis. TNX-102 SL is a non-opioid, centrally-acting analgesic, the active ingredient of which has a known, favorable safety profile from decades of use. The fact that cyclobenzaprine was also beneficial in many other key symptom domains, including sleep quality, sleep disturbance and fatigue, will be appreciated by fibromyalgia patients that struggle with not just pain but multiple other symptoms.”
“These positive data from RESILIENT and previously with RELIEF, with remarkable separation from placebo on pain, sleep, and fatigue, add support to TNX-102 SL’s proposed mechanism of improving sleep quality to improve the syndromal effects of fibromyalgia,” commented Gregory Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals. “The sublingual formulation of TNX-102 SL, which uses our proprietary Protectic® and Angstro® technologies, is integral to our treatment paradigm. These technologies enable transmucosal delivery of cyclobenzaprine with distinctive pharmacokinetic properties that include rapid absorption after dosing and bypass of first-pass hepatic metabolism. I would like to thank the RESILIENT study participants and their families and caregivers, as well as the investigators and their hard-working staff who all made this a highly successful trial.”
Summary of Topline Results of the RESILIENT Study
The RESILIENT study achieved statistical significance on the pre-specified primary efficacy endpoint: change from baseline in the weekly average of daily diary pain severity numerical rating scale (NRS) scores for TNX-102 SL 5.6 mg (LS mean [SE]: -1.8 [0.12] units) versus placebo (-1.2 [0.12] units), analyzed by mixed model repeated measures with multiple imputation (LS mean [SE] difference: -0.7 [0.16] units, p=0.00005, Table 1). In addition, all pre-specified sensitivity analyses of the primary endpoint were statistically significant (p<0.001). Figure 1 shows reduction in pain across all weeks of the 14-week study, with nominal p<0.01 for every week. Note the rapid onset of action with separation from placebo at Week 1 was sustained throughout all weeks of dosing.
Abbreviations: LS = least squares; NRS = numerical rating scale; SE = standard error
The statistically significant improvement in pain is further substantiated when diary pain was analyzed by another standard statistical approach, a 30 percent responder analysis, with 45.9% on active and 27.1% on placebo having a 30 percent or greater reduction in pain (Pearson Chi-Squared Test; difference in proportions [95% CI]: 18.8% [10.1%, 27.4%]; nominal p<0.001).
TNX-102 SL showed statistical significance (p≤0.001) on all six pre-specified key secondary efficacy outcome measures (Table 1).
Consistent with the proposed mechanism that TNX-102 SL acts in fibromyalgia through improving sleep quality, TNX-102 SL showed statistically significant improvement of sleep by two main measures. For the daily diary sleep quality ratings, improvement in sleep quality for TNX-102 SL (-1.8 [0.12] units) was significantly greater than that of placebo (-1.2 [0.12] units; LS mean [SE] difference from placebo: -0.6 [0.17] units; p<0.001). For the PROMIS Sleep Disturbance instrument, TNX-102 SL also demonstrated significantly greater improvement over placebo on T-scores (LS mean [SE] difference from placebo: -4.2 [0.79] units; p<0.001). Fatigue is another cardinal symptom of fibromyalgia and has a major impact on quality of life. TNX-102 SL showed significant improvement over placebo on the PROMIS Fatigue instrument T-scores (-3.0 [0.77] units; p<0.001).
The Fibromyalgia Impact Questionnaire – Revised (FIQ-R) is a 21-item self-rated instrument that assesses level of function, overall impact, and symptoms due to fibromyalgia, and the symptoms and function domains were key secondary endpoints in RESILENT. At Week 14 on the FIQ-R Symptoms domain, there was significantly greater improvement with TNX-102 SL than with placebo (LS mean [SE] difference from placebo: -7.7 [1.62], p<0.001). Similarly, TNX-102 SL resulted in greater improvement on FIQ-R Function (LS mean [SE] difference from placebo: -5.4 [1.66], p=0.001). Although not a key secondary efficacy endpoint, TNX-102 SL also separated from placebo on the FIQ-R Impact domain (nominal p=0.001). These results, along with the robust effects on improving sleep and fatigue, suggests broad symptomatic coverage of the syndrome of fibromyalgia.
Safety Results of the Phase 3 RESILIENT Study
In the RESILIENT study, TNX-102 SL was well tolerated and consistent with prior trials, with no new safety signals observed. Among participants randomized to the TNX-102 SL and placebo arms, 81.0% and 79.2%, respectively, completed the 14-week dosing period. As expected based on prior TNX-102 SL studies, administration site reactions were the most commonly reported adverse events and were higher in the TNX-102 SL treatment group (Table 2). Hypoaesthesia oral and paraesthesia oral, or tongue and mouth numbness or tingling, product taste abnormal (typically a bitter aftertaste upon dosing), and tongue discomfort were local effects nearly always temporally related to dose administration and transiently expressed (<60 minutes) in most occurrences. The only treatment-emergent adverse events that occurred at a rate of 3.0% or greater in either arm were these four oral adverse events, along with COVID-19, somnolence, and headache (Table 2). Adverse events resulted in premature study discontinuation in 6.1% of those who received TNX-102 SL compared with 3.5% of placebo recipients. There were a total of seven serious adverse events in five patients, five of which were experienced by three patients in the placebo arm, and two of which were in the TNX-102 SL arm. Of the two in the TNX-102 SL arm, one was renal cancer, deemed unrelated to study drug, and the other was acute pancreatitis with onset 14 days after dosing was completed and reported as possibly related to study drug.
Table 2. Treatment-Emergent Adverse Events at a Rate of 3% or Greater in Either Treatment Arm
TNX-102 SL (N=231)
Placebo (N=226)
Total (N=457)*
Administration Site Reactions
N
%
N
%
N
%
Hypoaesthesia oral
55
23.8%
1
0.4%
56
12.3%
Product taste abnormal
27
11.7%
2
0.9%
29
6.3%
Paraesthesia oral
16
6.9%
2
0.9%
18
3.9%
Tongue discomfort
16
6.9%
0
0.0%
16
3.5%
Systemic
Adverse Events
N
%
N
%
N
%
COVID-19
10
4.3%
7
3.1%
17
3.7%
Somnolence
7
3.0%
3
1.3%
10
2.2%
Headache
7
3.0%
4
1.8%
11
2.4%
*Safety Population
The Changes in Sexual Functioning Questionnaire short form (CSFQ-14) served as a safety measure for assessing potential adverse effects on sexual functioning. In females, the total score on the CSFQ-14 at Week 14 improved (indicating better sexual functioning) in the TNX-102 SL group compared with placebo (nominal p=0.010 by analysis of covariance). This potentially indicates an important tolerability advantage over pharmacotherapeutics which potently inhibit reuptake of serotonin. The low percentage of males in the safety population (<5%) did not allow meaningful analysis of the CSFQ-14 data.
About the Phase 3 RESILIENT Study
The RESILIENT study is a double-blind, randomized, placebo-controlled trial designed to evaluate the efficacy and safety of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) in the management of fibromyalgia. The two-arm trial randomized 457 participants in the U.S. across 33 sites. The first two weeks of treatment consist of a run-in period in which participants start on TNX-102 SL 2.8 mg (1 tablet) or placebo. Thereafter, all participants increase their dose to TNX-102 SL 5.6 mg (2 x 2.8 mg tablets) or two placebo tablets for the remaining 12 weeks. The primary endpoint is the daily diary pain severity score change (TNX-102 SL 5.6 mg vs. placebo) from baseline to Week 14 (using the weekly averages of the daily numerical rating scale scores), analyzed by mixed model repeated measures with multiple imputation.
For more information, see ClinicalTrials.gov Identifier: NCT05273749.
About Fibromyalgia
Fibromyalgia is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. Fibromyalgia afflicts an estimated 6 million to 12 million adults in the U.S., the majority of whom are women. Symptoms of fibromyalgia include chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products.
About TNX-102 SL
TNX-102 SL is a patented sublingual tablet formulation of cyclobenzaprine hydrochloride which provides rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional agent with potent binding and antagonist activities at the 5-HT2A-serotonergic, α1-adrenergic, H1-histaminergic, and M1-muscarinic cholinergic receptors, TNX-102 SL is in development as a daily bedtime treatment for fibromyalgia, fibromyalgia-type Long COVID (formally known as post-acute sequelae of COVID-19 [PASC]), alcohol use disorder and agitation in Alzheimer’s disease. Dr. Harvey Moldofsky, Professor Emeritus of Psychiatry and Medicine at the University of Toronto, founding Director of the University of Toronto Center for Sleep and Chronobiology, first recognized the central role of non-restorative sleep in the pathogenesis of fibromyalgia1,2. Our program is based on the subsequent pioneering work of Dr. Iredell W. Iglehart III, who recognized that a sleep-focused cyclobenzaprine treatment protocol had the potential to target non-restorative sleep and lead to improvement of fibromyalgia at the syndromal level3. Teams led by Giorgio Reiner at APR Applied Pharma Research S.A., a wholly-owned subsidiary of Relief Therapeutics Holding AG, and Professor Marino Nebuloni and Patrizia Colombo at Redox Analytical Science Srl invented and developed these underlying technologies in collaboration with Tonix. The United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation claimed in the patent are important elements of Tonix’s proprietary TNX-102 SL composition. These patents are expected to provide TNX-102 SL, upon NDA approval, with U.S. market exclusivity until 2034/2035. In addition, Tonix has pending but not issued U.S. patent applications directed to the transmucosal absorption of CBP-HCl, with U.S. market exclusivity expected until 2033, for treating major depressive disorder in fibromyalgia, with U.S. market exclusivity expected until 2032, and for treating pain in fibromyalgia with U.S. market exclusivity expected until 2041.
1Moldofsky H et al, Psychosom Med 1975;37:341-51. 2Moldofsky H and Scarisbrick P. Psychosom Med 1976;38:35-44. 3Iglehart IW. 2003; US Patent 6,541,523.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg under a transition services agreement with Upsher-Smith Laboratories, LLC from whom the products were acquired on June 30, 2023. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), has completed two positive Phase 3 studies for the management of fibromyalgia. Tonix intends to meet with the FDA and submit an NDA for the approval of TNX-102 SL for the management of fibromyalgia in the second half of 2024. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition, and topline results were reported in the third quarter of 2023. TNX-1900 (intranasal potentiated oxytocin) is being studied in binge eating disorder, pediatric obesity, bone health in autism and social anxiety disorder by academic collaborators under investigator-initiated INDs. 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 2024 Tonix’s rare disease development portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology development portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 was initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases, including TNX-1800, in development as a vaccine to protect against COVID-19. During the fourth quarter of 2023, TNX-1800 was selected by the U.S. National Institutes of Health (NIH), National Institute of Allergy and Infectious Diseases (NIAID) Project NextGen for inclusion in Phase 1 clinical trials. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals.
*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.
Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
About Redox – Analytical Science Srl
Redox is an independent CRO company headquartered in Monza- Italy with R&D activities and customer analytical support to pharmaceutical companies for more than 30 years. From more than 25 years the analytical activities have been certified by national and international agencies (European Medicines Agency, the Italian Medicines Agency (AIFA), FDA, and etc). One of the main activities is the development of new drug products in order to improve the pharmaceutical actions and at the same time improve the stability and reducing the cost of the new drug substance. Several unique and sophisticated analytical techniques and equipment are used in support to research and development strategies with the focus to reach the best and effective pharmaceutical formulation in a short time frame. More than 30 professional people are dedicated to our efforts and many projects are ongoing in collaboration with the pharmaceutical industry as well as with Italian and international Universities.
Further information about Redox can be found at www.labredox.com.
About APR Applied Pharma Research S.A., a wholly-owned subsidiary of Relief Therapeutics Holding AG
Relief Therapeutics is a commercial-stage biopharmaceutical company committed to advancing treatment paradigms and delivering improvements in efficacy, safety, and convenience to benefit the lives of patients living with select specialty and rare diseases. Relief Therapeutics’ portfolio offers a balanced mix of marketed, revenue-generating products, our proprietary, globally patented Physiomimic™ and TEHCLO™ platform technologies and a targeted clinical development pipeline consisting of risk-mitigated assets focused in three core therapeutic areas: rare metabolic disorders, rare skin diseases and rare respiratory diseases. In addition, Relief Therapeutics is commercializing several legacy products via licensing and distribution partners. Relief Therapeutics’ mission is to provide therapeutic relief to those suffering from rare diseases and is being advanced by an international team of well-established, experienced biopharma industry leaders with extensive research, development and rare disease expertise. Relief Therapeutics is headquartered in Geneva, with additional offices in Balerna, Switzerland, Offenbach am Main, Germany and Monza, Italy. Relief Therapeutics is listed on the SIX Swiss Exchange under the symbol RLF.
Further information about APR can be found at www.relieftherapeutics.com or by following Relief Therapeutics on LinkedIn and Twitter.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
The pharmaceutical industry experienced seismic shifts in 2023 through breakthrough innovations, changing disease priorities, and new regulations. Several landmark events promise to transform medicine, captivate investors, and save or improve lives.
The most transformational breakthrough came in Alzheimer’s disease. After decades of failure, the FDA approved Leqembi from Eisai and Biogen – the first ever drug to slow progression of the memory-robbing disease. With Alzheimer’s costing the U.S. $321 billion annually, this long-awaited milestone offers new hope to patients and caregivers.
Equally remarkable was the meteoric rise of anti-obesity medications. Once stagnant, the weight loss market exploded with Novo Nordisk’s Wegovy and Eli Lilly’s Mounjaro. These injections suppress appetite while also lowering blood sugar and weight in diabetics. Sales hit billions within the first year. The obesity epidemic affliction impacts over 40% of American adults. Novo Nordisk and Lilly now boast half-trillion-plus market valuations.
Additional firsts included respiratory syncytial virus (RSV) shots from Pfizer and GlaxoSmithKline for older adults. RSV leads to thousands of hospitalizations and deaths yearly in seniors. These vaccines gained quick uptake since their recent launch.
Furthermore, gene editing stepped into the mainstream. The revolutionary technology promises to correct disease-causing mutations. In a watershed moment, the FDA approved the first-ever gene editing therapy from Bluebird and Vertex. It treats sickle cell disease, a painful inherited blood disorder impacting 100,000 Americans. More approvals seem imminent as gene editing solidifies itself at medicine’s cutting edge.
Shifting Disease Priorities
With waning COVID-19 cases, demand evaporated for vaccines and treatments. Juggernauts Pfizer and Moderna confronted sharply declining sales, inventory piles, and plummeting share prices. After prioritizing infectious disease, the world shifted focus to chronic illnesses like obesity, Alzheimer’s, diabetes, and cancer.
Novartis recently listed Alzheimer’s and obesity among its top five growth drivers. Many firms now chase weight loss billions, with Amgen, Pfizer, and Lilly reporting positive clinical trial results in 2023. Obesity has become pharma’s hottest investment theme. Meanwhile, Alzheimer’s treatments from Roche, Biogen, Lilly and Eisai should continue advancing through pipelines.
Gene editing and genomics represent additional high-growth areas as companies unlock genetics’ role in disease. Vertex intends to file its second cutting-edge therapy in 2023 for blood disorders. CRISPR pioneer Intellia began mid-stage sickle cell and beta thalassemia trials. In cancer, GRAIL’s blood test screens for early detection, while Moderna publishes positive data on personalized vaccines. Increased R&D funding and medicine partnerships with gene editing/genomics firms seem likely.
Controversy Over Drug Pricing Regulations
Controlling escalating prescription costs has become a contentious political issue. Over half of Americans take prescription medicines, with one-quarter facing difficulties affording them. To expand access, the Inflation Reduction Act enables Medicare to negotiate prices for certain high-cost drugs starting in 2026.
But negotiations face vigorous resistance from pharma who invested billions developing treatments. With less revenue, they argue that funding for future innovations could dramatically fall. A third of affected companies even sued to halt the program’s launch. All signed agreements to participate after the initial drug selection, but negotiations don’t formally begin until 2024. It remains uncertain if meaningful savings can emerge without detriment to future medical progress.
Investment Implications of Pharma Firsts
Despite drug pricing disputes, 2023’s breakthroughs highlight pharma’s prescribing power for investors. Unmet needs still abound across oncology, immunology, rare diseases and neuroscience. With obesity prevalence doubling since the 1990s, its remedies from Novo Nordisk and Lilly should continue realizing substantial revenues. Even Alzheimer’s treatments represent multibillion-dollar opportunities if benefit is demonstrated.
Gene editing, genomics, and precision medicine’s potential to transform therapeutic landscapes brims with possibility. Approved gene therapies already exceed $2 million per patient. As insurers eventually broaden coverage, early innovators like Bluebird, Vertex, and many others seem poised for sustainable growth. Meanwhile, liquid biopsy leaders Exact Sciences and PDS Biotech might one day screen entire populations for cancers.
Despite political rhetoric over high costs, investors ultimately care about innovation that changes patient lives. With pharma endlessly discovering new medical frontiers, its life-saving products should keep enhancing portfolio health for decades to come.
Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
OCU400 Received RMAT Designation. The FDA has designated OCU400 as a Regenerative Medicine Advanced Therapy (RMAT) for treating retinitis pigmentosa associated with RHO mutations. RMAT designation is intended to speed development of regenerative medicines, and is awarded to therapies that have potential treat, reverse, or modify a life-threatening disease. We see this as a validation of Ocugen’s clinical trial data. Additional data submissions to expand the designation to other gene mutations in RP and Leber congenital amaurosis (LCA) are planned.
Phase 3 Trial Should Benefit From RMAT Designation. RMAT designation includes the benefits of Fast Track and Breakthrough designations, with increased guidance from the FDA. It also allows the use of surrogate markers as endpoints in clinical trials, and accelerated review after the application is filed. Ocugen is currently finalizing the design of the Phase 3 trial for OCU400. We expect the trial to begin in early 2024 with data available in 2H24.
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MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
MAIA Selects THIO Middle Dose Level For Future Studies. MAIA announced that it has selected the 180mg dose of THIO to continue the Phase 2 THIO-101 trial in non-small lung cancer (NSCLC). The THIO-101 trial tested three THIO dose levels (60mg, 180mg, or 360mg) followed by cemiplimab (Libtayo), an anti-PD-1 checkpoint inhibitor from Regeneron. The 180mg dose level showed a better safety profile with better measures of efficacy. All future patients in the trial will be treated with the 180mg dose.
Trial Shows The Best THIO Disease Control Data To Date. MAIA also stated that the trial shows all dose levels exceed the disease control rate thresholds in stage 1 of the trial. Disease control rates in the first 8 out of 9 patients exceeded the goal of disease control in 8 out of 19 patients per arm. The 180mg dose had the best preliminary response rate compared with the 60mg and 360mg arms. We see strong results as consistent with prior data presentations showing large survival improvements for THIO treated patients.
Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.
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.
The E.W. Scripps Company (NASDAQ: SSP) is a diversified media company focused on creating a better-informed world. As one of the nation’s largest local TV broadcasters, Scripps serves communities with quality, objective local journalism and operates a portfolio of 61 stations in 41 markets. The Scripps Networks reach nearly every American through the national news outlets Court TV and Newsy and popular entertainment brands ION, Bounce, Defy TV, Grit, ION Mystery, Laff and TrueReal. Scripps is the nation’s largest holder of broadcast spectrum. Scripps runs an award-winning investigative reporting newsroom in Washington, D.C., and is the longtime steward of the Scripps National Spelling Bee. Founded in 1878, Scripps has held for decades to the motto, “Give light and the people will find their own way.”
Michael Kupinski, Director of Research, Equity Research Analyst, Digital, Media & Technology , Noble Capital Markets, Inc.
Jacob Mutchler, Research Associate, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Noblecon19. On December 4th, management presented at Noblecon19 at Florida Atlantic University (FAU) in Boca Raton, Florida, to the investment community. The fireside chat presentation conducted by Lisa Knutson, Chief Operating Officer, highlighted the dynamic value proposition of Scripps Sports, favorable retransmission trends, implications of the Disney/Charter deal and green shoots in advertising. A replay of the presentation can be viewed here: https://www.channelchek.com/videos/e-w-scripps-noblecon19-replay.
Favorable sports model. Scripps Sports employs a unique model that offers sports teams wider viewership than the traditional Regional Sports Networks (RSNs) model that is now failing. Management indicated that its two NHL sports licenses for the Arizona Coyotes and the Vegas Golden Knights will account for a 4% point increase in its core advertising in Q4 and 3% for full year 2024.
Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.
This 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.
Insurance brokerage and consulting powerhouse Aon (AON) unveiled a definitive agreement on December 20th to acquire middle-market peer NFP in an all-cash $13.4 billion deal. NFP focuses on property and casualty brokerage, benefits consulting, wealth management and retirement plan advisory specifically for mid-sized clients.
The landmark transaction allows Aon to aggressively expand into the lucrative mid-corporation segment amid an economic landscape stoking demand for recession-resistant insurance policies. With NFP expecting 2022 revenues nearing $2.2 billion and a roster of over 7,700 client organizations, the bolt-on acquisition provides Aon a launching pad towards deepening its presence among growth-oriented middle-market enterprises.
Tap Exploding Market for Mid-Sized Firms
Several tailwinds have powered extraordinary growth within insurance brokerages catering to mid-cap corporations. As middle-market companies strive for enhanced risk management oversight amid volatile conditions, they increasingly seek broker partners delivering customized guidance on property/casualty and employee benefits policies.
NFP’s singular mid-market focus perfectly aligns with this surging addressable market. The brokerage brings specialized consulting capabilities around financial, health, and retirement offerings that resonate powerfully among mid-sized organizations. After closing in mid-2024, NFP’s offerings significantly broaden and diversify Aon’s middle-market resources.
The opportunistic move also builds on Aon’s existing relationship with mid-market insurance access point Businessolver. By consolidating NSM Insurance and now NFP, Aon assembles an unrivaled mid-corporation product portfolio spanning risk management, human resources, payroll, and compliance functionality.
Betting on Consistent Insurance Demand
Aon’s bold acquisition reflects confidence that commercial insurance spending will continue rising despite recessionary warnings. Employer-sponsored health plans, property policies, casualty coverage, and other risk transfer solutions retain fundamental necessity for corporations of all sizes. With mid-sized companies facing substantial human capital and operational exposures, brokerages like NFP and Aon constitute trusted partners for navigating complex risk landscapes.
The sector’s recession resilience and anti-cyclical behaviors produce reliable revenues amid broader economic uncertainty. Aon has witnessed only one year of revenue declines over the past decade. The industry giant averaged yearly sales growth of 8.4% since 2013.
Strategic Growth Play
From a financial perspective, NFP dramatically strengthens Aon’s growth trajectory. Adding the brokerage’s high-single-digit annual revenue gains provides immediate scale. In an investor presentation, management projected total company sales expansion of 8% in 2024 and 14% in 2025 post-acquisition. Significant cross-selling opportunities and global expansion of NFP’s capabilities should spur ongoing upside.
Aon expects to realize $150 million in cost synergies by 2025. The combination presents chances to eliminate redundant corporate structures and leverage joint capabilities in technology, data analytics and digitization to drive efficiency gains. Ensuing margin expansion would magnify bottom-line profit growth produced by the increased revenues.
Although the transaction costs require $7 billion in new debt, NFP is projected to start contributing towards deleveraging by 2025. While 2024 margins may compress initially, management reinforced commitment towards long-term margin expansion. From 2013-2021, Aon’s margins grew from 16.4% to record 35.7% levels.
Risks and Costs
Despite projected profitability gains, Aon’s stock dropped nearly 8% on the announcement as shareholders weigh risks around significant integration costs and execution challenges. Management forecasts $400 million in one-time transaction and integration expenses associated with consolidating the sizable acquisitions.
There are additionally risks tied to client retention. As occurred with some Willis Towers Watson customers after Aon’s failed merger attempt in 2021, certain NFP accounts may reevaluate relationships depending on changes in account management or service model adjustments.
Overall, however, investor reception remains positive. The deal continues an active era defined by transformative combinations as large brokers fight for differentiation. Aon has now spent nearly $30 billion on M&A to distinguish its portfolio. Adding NFP crucially now arms the brokerage giant to increasingly capitalize on lucrative mid-market tailwinds in coming years.
Are you looking to supercharge your investment portfolio? In the ever-evolving world of finance, finding undervalued stocks can be your ticket to potential wealth and financial security. But how do you identify these hidden gems in the vast stock market?
In this guide, we’re going to dive deep into the art of discovering undervalued stocks that have the potential to yield substantial returns. Whether you’re an income-seeking investor eyeing undervalued dividend stocks, a tech enthusiast interested in undervalued tech stocks, or someone with an appetite for growth stocks, we’ve got you covered.
But how do you spot those hidden gems in a stock market containing over 7,000 possibilities? It takes knowing what to look for – the right metrics, indicators, and overall characteristics. Whether you’re an income investor, growth investor, trader, or speculator – finding and investing in undervalued stocks aligns with most investing philosophies. The logic is simple – buy low, sell high. When you purchase quality stocks trading at a discount, your margin for profit substantially rises.
By the end of this guide, you’ll have the knowledge and tools to identify undervalued stocks that align with your investment goals. Whether you’re a seasoned investor or just starting, this guide will equip you to make informed decisions in the world of undervalued stocks. Read on to discover your next financial opportunity.
What are Undervalued Stocks?
Undervalued stocks are stocks trading below their inherent worth or true value. But estimating a stock’s intrinsic value involves looking beyond its current market price. By using valuation metrics, financial modeling, and qualitative assessments, investors determine when the market misprices a stock relative to its potential.
The market regularly misvalues stocks by either overvaluing or undervaluing them. This disconnect between price and value stems from economic conditions, investor sentiment, and company specifics.
Economy-wide or sector-specific downturns indiscriminately pressure stock prices down across industries. Near-term operating headwinds or weak quarterly results can also sink stocks regardless of long-term prospects.
Additionally, negative market psychology and prevailing pessimism frequently drag stocks below fair value. The key is blocking out noise and objectively assessing a business’s fundamental health.
Investors favor underpriced stocks because it provides a margin of safety. The gap between price and projected value presents potential upside as undervalued stocks mean revert towards full valuation.
Think of undervalued stocks as companies facing temporary issues, their true long-range trajectories still intact. Identifying and investing in them before the crowd catches on provides huge value creation through future price appreciation.
Valuation Metrics Signaling Undervalued Stocks
As Peter Lynch emphasized – price is what you pay, value is what you get. Finding stocks valued less than what they’re intrinsically worth is the cornerstone of value investing.
Several key valuation metrics demonstrate when stocks trade at bargain prices:
Price-to-Earnings Ratio: The P/E ratio measures a company’s current share price relative to its earnings per share. A low P/E ratio signals an undervalued stock since investors assign little value relative to profits. Compare P/Es within sectors to find discounted stocks with upside to mean ratios.
Price-to-Book Ratio: The P/B calculates whether a stock sells for less than its book value or net assets. A P/B below 3.0 signals a potential value stock while ratios under 1.0 indicate deep value. Compare book values over asset values to confirm if fire-sale prices exist.
Price-to-Sales Ratio: For higher growth early stage companies that reinvest profits into expansion, the P/S ratio substitutes sales for earnings. Compare ratios amongst industry peers to find stocks with solid revenue trading at discounts.
Dividend Yield: Undervalued dividend stocks feature higher yields than industry averages and historical ranges. Yields signal what income return you receive upfront while awaiting share price increases.
Future Cash Flow Analysis: Discounted cash flow models estimate intrinsic value based on projected future cash flows. Compare these model prices to current prices to quantify discounts-to-value.
Technical Analysis – Momentum and Trend Reversals
While valuation rankings highlight intrinsically cheap stocks, technical analysis examines price action and trends to confirm upside potential.
Technicians employ stock charts and technical indicators like moving averages to reveal investor psychology and emerging momentum. Upside breakouts, oversold readings that trigger reversals, and upside volume surges provide buy signals on beaten-down stocks.
Oversold RSI levels signal capitulation selling exhaustion from which stock rebound as selling pressure recedes. Positive divergences with price carving higher lows while indicators like RSI or On-Balance Volume trend higher hints upside coming.
Technicians also analyze previous support levels and trendlines where stocks find buying interest to re-enter. Combining discounted valuations with constructive chart patterns and indicators gives higher conviction around upside.
Qualitative Analysis – Beyond The Numbers
Even attractively priced stocks need proper qualitative vetting to ensure their businesses remain healthy. Analyze softer aspects like:
Industry Trends: Favorable secular shifts provide tailwinds regardless of economic cycles. Disruptive innovation and new high growth markets often mask temporary business challenges.
Management Quality: Study executive backgrounds, performance incentives, capital allocation plans, and past navigations of crises. Skilled leaders offset risks especially on battered stocks.
Competitive Advantages: Analyze what differentiation prevents customer losses and erosion from rivals. Network effects, intellectual property, scale cost advantages and brand equity strengthen leading positions.
Growth Drivers: Robust pipelines, new product launches, expansion possibilities and M&A opportunities indicate upside not quantified in current earnings.
Types of Undervalued Stocks
While all stocks can trade below fair values, certain categories routinely present coiled springs.
Undervalued Dividend Stocks: Mature low-volatility companies often face skepticism despite consistent dividends and buybacks. Compare payout ratios and yields to reveal mispriced income stocks.
Undervalued Tech Stocks: Rapid innovation leaves many tech companies misunderstood and their disruptive threats underestimated. When growth hits temporary snags, investors quickly extrapolate doomsday scenarios.
Undervalued Growth Stocks: Growth favorites correction 50% or more during economic and liquidity shifts as prosperous long-term outlooks get ignored in panic selling.
Strategies to Find the Most Undervalued Stocks Now
Finding even one undervalued stock with upside can transform portfolio returns. But several proven strategies efficiently uncover today’s biggest disconnects between price and potential.
Deep Value Investing: Iconic investors like Warren Buffett target extreme discounts to book value, earnings power and cash flow generation. Deep value investing works best buying companies with staying power over market dips.
Contrarian Investing: Buying out-of-favor, unloved stocks that short sellers target demands resolve but reaps huge rewards. The best opportunities surface when stocks face industry upheaval or company uncertainty despite solid cores.
Growth at a Reasonable Price: Find quality growth companies hitting air pockets from temporary setbacks not deterioration. Seeking growth stalwarts trading at discounts to historical multiples provides upside with less downside.
Screening Tools and Stock Scanners: Input valuation metrics, fundamentals criteria and technical filters into screeners to generate stock idea lists objectively matching deep value criteria. Scan within industries and market caps for mispricings relative to comparable groups.
Risks When Investing in Undervalued Stocks
While undervalued stocks present significant upside potential, they also carry increased risks in some cases. Here are the major hazards when targeting deeply discounted stocks:
Financial Distress Risk – Some cheap stocks are outright value traps en route to bankruptcy and liquidation. Analyze debt levels, cash burn rates, credit ratings and avenues to raise capital to avoid terminal value declines.
Lack of Catalysts – Certain stocks trade at low valuations indefinitely without catalysts to unlock value. Change often needs to come externally through management changes, activist investors, private equity interest or strategic mergers.
Opportunity Cost – Capital gets tied up in stocks other assets outperform during extended downturns. Consider position sizing each undervalued stock to limit opportunity costs.
Emotional Risk – Buying stocks amidst bad news and continuing price declines tests conviction. Volatility may heighten before gains accrue.
While higher risk, historically the excess returns justify bargain hunting during fearful periods provided you stick to quality stocks.
The key lies in fundamental analysis separating temporary problems from permanent ones when identifying mispriced companies to mitigate risks associated with undervalued stocks.
DISCLAIMER: Undervalued, emerging growth stocks may represent a greater risk to the investor. This guide is for informational purposes only and does not constitute investment advice. Any investment decisions should be made with a licensed investment advisor.
Finding undervalued stocks stacks odds dramatically in your favor to reap life-changing wealth. But calculated approaches win over blind stock picking while circumventing value traps. Follow the playbooks of legendary investors who built fortunes identifying future blue chips trading at bargain prices at the time.
Arm yourself with the metrics, models, resources and strategies outlined to pinpoint the market’s mispriced stocks today. Consistently applying a framework for finding, researching and buying undervalued stocks builds a dividend machine primed to deliver outsized returns as investments revert to true values.
The journey begins by creating your free Channelchek account to tap into institutional-grade equity research and screeners identifying discounted opportunities across global markets.
Beloved Pizza Chain Expands Texas Footprint in Richmond Suburb
LOS ANGELES, Dec. 19, 2023 (GLOBE NEWSWIRE) — FAT (Fresh. Authentic. Tasty.) Brands Inc., parent company of Round Table Pizza and 17 other restaurant concepts, announces that it has officially opened its first Round Table Pizza location in greater Houston. Located in Richmond, Texas, the new restaurant is operated by franchisee Paul Tran, an oil industry veteran and retiree, and will be a welcome addition to the West Houston suburbs.
Since its founding, Round Table Pizza has been recognized as “Pizza Royalty™” for its homemade dough, signature three-cheese blend, and gold-standard ingredients topped to the edge. Customers can enjoy the chain’s proprietary handmade pizzas, salads, baked-to-perfection Garlic Parmesan Twists, classic and boneless wings, and more.
“Establishing a presence in Houston represents a pivotal step in Round Table Pizza’s domestic growth strategy, especially as we continue building upon our presence in Texas,” says Taylor Wiederhorn, Chief Development Officer of FAT Brands. “Round Table Pizza has seen great success in the San Antonio and Dallas markets, and we believe the Richmond community will enjoy what we have to offer.”
Round Table Pizza in Richmond will be located at 8323 Farm to Market Rd 723, Richmond, TX 77406. It is open Sunday through Thursday, 11 a.m. to 9 p.m., and Fridays and Saturdays, 11 a.m. to 10 p.m. For more information on Round Table Pizza, please visit www.roundtablepizza.com.
About FAT (Fresh. Authentic. Tasty.) Brands
FAT Brands (NASDAQ: FAT) is a leading global franchising company that strategically acquires, markets, and develops fast casual, quick-service, casual dining, and polished casual dining concepts around the world. The Company currently owns 18 restaurant brands: Round Table Pizza, Fatburger, Marble Slab Creamery, Johnny Rockets, Fazoli’s, Twin Peaks, Great American Cookies, Hot Dog on a Stick, Buffalo’s Cafe & Express, Hurricane Grill & Wings, Pretzelmaker, Elevation Burger, Smokey Bones, Native Grill & Wings, Yalla Mediterranean and Ponderosa and Bonanza Steakhouses, and franchises and owns over 2,300 units worldwide. For more information on FAT Brands, please visit www.fatbrands.com.
About Round Table Pizza
Inspired by the honor, valor, and revelry of the Knights of the Round Table, Round Table Pizza’s® superior pizza and commitment to quality and authenticity have earned the reputation of “Pizza Royalty™” for over 60 years. With more than 410 restaurants across the United States, Round Table celebrates community, family, and making merry. For more information, visit www.roundtablepizza.com.
RMAT designation intended to help expedite development of new regenerative medicines
MALVERN, Pa., Dec. 19, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the FDA has granted RMAT designation to Ocugen’s investigational product OCU400 for the treatment of retinitis pigmentosa (RP) associated with RHO mutations.
“RMAT designation is a significant accomplishment for the OCU400 clinical development program, as it validates the potential for our game-changing gene therapy approach to fulfill an unmet medical need for people who are facing blindness due to RP,” said Arun Upadhyay, PhD, Chief Scientific Officer and Head of R&D at Ocugen. “FDA’s decision also reinforces the sense of urgency to bring a therapeutic option to these patients.”
RMAT designation for OCU400 was based on preliminary clinical data supporting the maintenance and improvement of visual acuity and function in RP patients in the OCU400 -101 Phase 1/2 clinical trial as measured by Best Corrected Visual Acuity (BCVA), Low Luminance Visual Acuity (LLVA), and Multi-Luminance Mobility Test (MLMT).
RMAT designation is part of the 21st Century Cures Act. The program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition. Receiving RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with the FDA. Ocugen is working with the FDA to finalize the Phase 3 protocol necessary to advance the clinical development for OCU400 to support an application for marketing authorization.
Current data support the gene-agnostic mechanism of action for OCU400, which suggests that it may be able to provide treatment benefit to a broader group of RP and Leber congenital amaurosis (LCA) patients. Ocugen intends to submit additional efficacy and safety data for OCU400 in RP and LCA patients to the FDA in the future to potentially expand this RMAT designation to broader RP and LCA patient populations.
RHO mutations affect more than 10,000 of the 110,000 people in the United States diagnosed with RP. In the latest clinical study update from the Phase 1/2 trial of OCU400, 86% (6/7) of RHO mutation subjects experienced either stabilization or improvement in MLMT scores from baseline, among which 29% (2/7) demonstrated 3 Lux luminance level improvement. There are currently no treatment options available for RP patients with RHO gene mutations.
OCU400 represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions, such as homeostasis, reproduction, development, and metabolism to potentially improve retinal health and function.
About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.
Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, the expected benefits of RMAT designation, and anticipated regulatory interactions, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contact: Tiffany Hamilton Head of Communications IR@ocugen.com
Selected dose shows unprecedented disease control and overall response rates in a NSCLC clinical trial
CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA” or the “Company”), a clinical-stage biopharmaceutical company developing telomere-targeting immunotherapies for cancer, today announced dose selection for THIO-101, a Phase 2 clinical trial evaluating its lead asset, THIO, in sequential combination with Regeneron’s anti-PD-1 cemiplimab (Libtayo®) in patients with advanced non-small cell lung cancer (NSCLC).
During the dose-finding stage of THIO-101, patients were administered either 60mg, 180mg, or 360mg of THIO per cycle, followed by 350mg of cemiplimab (Libtayo®). The selected dose, 180mg/cycle, presented better safety profile and outperformed the other doses in the key measures of efficacy for NSCLC trials. Subsequently, all future trial participants will be treated with THIO 180mg/cycle.
“All THIO dose levels tested exceeded the disease control rate (DCR) thresholds in Stage 1 of the THIO-101 Phase 2 trial. We observed disease control in the first 8 to 9 patients with a post baseline scan in each arm, beating our goal of disease control in 8 out of 19 patients per arm. Among the three studied doses, the 180mg dose showed stronger DCR and preliminary response rates compared to other doses,” said Vlad Vitoc, M.D., MAIA’s Chairman and Chief Executive Officer.
“These results are particularly impressive in this pool of patients who were heavily pre-treated and resistant to prior treatments with immune checkpoint inhibitors, a group that does not yet have standard of care treatment. We are highly encouraged by the unprecedented clinical data generated thus far in our Phase 2 trial, and as we move forward, we plan to pursue accelerated approval for THIO in the U.S. for the treatment of patients with advanced NSCLC. We believe THIO’s DCRs and ORRs in second line treatment suggest the drug’s potential to define the standard of care for this NSCLC patient population.”
THIO is the only direct telomere targeting agent currently undergoing clinical development in the field of cancer drug discovery and treatment.
About THIO
THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.
About THIO-101, a Phase 2 Clinical Trial
THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. It is the first trial designed to evaluate THIO’s anti-tumor activity when followed by PD-(L)1 inhibition. The trial is testing the hypothesis that low doses of THIO administered prior to cemiplimab (Libtayo®) will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of THIO administered as an anticancer compound and a priming immune activator (2) to assess the clinical efficacy of THIO using Overall Response Rate (ORR) as the primary clinical endpoint. Treatment with cemiplimab (Libtayo®) followed by THIO has been generally well-tolerated to date in a heavily pre-treated population. For more information on this Phase II trial, please visit ClinicalTrials.gov using the identifier NCT05208944.
About MAIA Biotechnology, Inc.
MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Forward Looking Statements
MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.
Great Lakes Dredge & Dock Corporation is the largest provider of dredging services in the United States. In addition, Great Lakes is fully engaged in expanding its core business into the rapidly developing offshore wind energy industry. The Company has a long history of performing significant international projects. The Company employs experienced civil, ocean and mechanical engineering staff in its estimating, production and project management functions. In its over 131-year history, the Company has never failed to complete a marine project. Great Lakes owns and operates the largest and most diverse fleet in the U.S. dredging industry, comprised of approximately 200 specialized vessels. Great Lakes has a disciplined training program for engineers that ensures experienced-based performance as they advance through Company operations. The Company’s Incident-and Injury-Free® (IIF®) safety management program is integrated into all aspects of the Company’s culture. The Company’s commitment to the IIF® culture promotes a work environment where employee safety is paramount.
Joe Gomes, Managing Director, Equity Research Analyst, Generalist , Noble Capital Markets, Inc.
Joshua Zoepfel, Research Associate, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Continued Momentum. Great Lakes Dredge & Dock received some additional awards, according to the Department of Defense daily contract release. While value of one of the awards is more nuanced than the DOD release statement, combined, the two recent awards total some $66 million over a three-year period. Great Lakes continues to build momentum in securing new business, which will bode well for 2024 and beyond.
Jacksonville. Yesterday, the U.S. Army Corps of Engineers awarded Great Lakes a $32.4 million firm-fixed-price contract for shore protection and beach renourishment. Work will be performed in Jacksonville, Florida, with an estimated completion date of October 13, 2024. Fiscal 2023 civil construction funds in the amount of $32.4 million were obligated at the time of the award.
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This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).
*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
Cumulus Media (NASDAQ: CMLS) is an audio-first media company delivering premium content to over a quarter billion people every month — wherever and whenever they want it. Cumulus Media engages listeners with high-quality local programming through 406 owned-and-operated radio stations across 86 markets; delivers nationally-syndicated sports, news, talk, and entertainment programming from iconic brands including the NFL, the NCAA, the Masters, CNN, the AP, the Academy of Country Music Awards, and many other world-class partners across more than 9,500 affiliated stations through Westwood One, the largest audio network in America; and inspires listeners through the Cumulus Podcast Network, its rapidly growing network of original podcasts that are smart, entertaining and thought-provoking. Cumulus Media provides advertisers with personal connections, local impact and national reach through broadcast and on-demand digital, mobile, social, and voice-activated platforms, as well as integrated digital marketing services, powerful influencers, full-service audio solutions, industry-leading research and insights, and live event experiences. Cumulus Media is the only audio media company to provide marketers with local and national advertising performance guarantees. For more information visit www.cumulusmedia.com.
Michael Kupinski, Director of Research, Equity Research Analyst, Digital, Media & Technology , Noble Capital Markets, Inc.
Jacob Mutchler, Research Associate, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Noblecon19. On December 4th, management presented at Noblecon19 at Florida Atlantic University (FAU) in Boca Raton, Florida, to the investment community. The presentation conducted by Frank Lopez-Balboa, CFO, highlighted the company’s digital growth initiatives, debt reduction focus and provided insight into the current advertising environment. A replay of the presentation can be viewed here: https://www.channelchek.com/videos/cumulus-media-noblecon19-replay.
Digital growth initiatives. Management highlighted its digital growth strategy and key drivers for podcasting and digital marketing services (DMS) growth. Importantly, 50% of digital revenues are derived from national adverting, thus an improvement in the national advertising environment would positively impact digital revenue. Notably, the company has increased its focus on growing its hyper-local Digital Marketing Services business, a development we view favorably.
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This 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.
