CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that independent director Stan V. Smith, Ph.D. made an individual purchase of 100,000 shares of MAIA’s common stock, and warrants to purchase 100,000 shares, of MAIA’s common stock for an aggregate purchase price of $225,900 as part of the Company’s recent private placement of common stock and warrants announced by the Company on October 28, 2024, and which closed on November 1, 2024.
“As one of our original investors, Stan has participated in nearly every private placement financing round since our Company’s inception. We are grateful for his consistent participation in our financings and for his long-time service on our Board,” said Vlad Vitoc, M.D., Chairman and CEO of MAIA.
Stan Smith, Ph.D. commented, “As a private investor in MAIA, I believe the science behind the THIO franchise, coupling telomere targeting and immunogenicity, can be disruptive in the treatment markets for multiple difficult-to-treat cancer types.”
Dr. Smith is president of Smith Economics Group, Ltd. in Chicago, providing economic and financial consulting nationwide. Trained at the University of Chicago and specializing in litigation economics, Dr. Smith co-authored the first textbook on the subject of economic damages. Dr. Smith has served as an adjunct professor at the University of Chicago and at DePaul University College of Law where he created the first course in the United States in forensic economics.
Additional details on the private placement can be found in the Company’s Current Report on Form 8-K filed with the Securities and Exchange Commission on October 29, 2024, at www.sec.gov.
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.
MALVERN, Pa., Nov. 07, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that on November 6, 2024, the Company entered into a new $30 million credit facility with Avenue Venture Opportunities Fund, L.P., a fund of Avenue Capital Group. Proceeds from the facility are intended for general corporate purposes, capital expenditures, working capital, and general and administrative expenses.
The credit facility, which has a term of 4 years, provided $30 million fully funded on the closing date.
“We are pleased to enter into this relationship with Avenue Capital Group that provides what we believe is a shareholder-friendly financing for the Company,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-founder of Ocugen. “This additional working capital will support the clinical development of our three, first-in-class modifier gene therapies and provide adequate funding to near completion of the OCU400 Phase 3 liMeliGhT clinical trial and prepare for the BLA and MAA submissions.”
This most recent financing is part of Ocugen’s diversified strategy to fund the business and appropriately allocate resources across the portfolio.
“We are pleased to partner with Ocugen with this financing as the Company drives its next chapter of growth, based on its novel scientific platforms and dedication to fighting blindness diseases,” said Chad Norman, Senior Portfolio Manager, Avenue Capital.
With net proceeds from this facility and current cash, cash equivalents, and restricted cash, the Company’s expected cash runway extends into the first quarter of 2026.
Chardan and Titan Partners Group, a division of American Capital Partners, acted as financial advisors to Ocugen on the transaction.
About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, 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.
About Avenue Venture Opportunities The Avenue Venture Debt Funds seek to provide creative financing solutions to high-growth, venture capital-backed technology and life science companies, focusing generally on companies within the underserved segment of the market created by the widening financing gap between commercial banks and larger debt funds. The Avenue Venture Debt funds are part of the larger group of funds of Avenue Capital Group. For additional information on Avenue Capital Group, which is a global investment firm with assets under management of approximately $12.2 billion, visit www.avenuecapital.com.
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, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs;expectations regarding the satisfaction of closing conditions, timing of the funding and the anticipated use of proceeds; Ocugen’s financial condition and expected cash runway into the first quarter of 2026; and statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, 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 annual and 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.
CHICAGO, Nov. 07, 2024 (GLOBE NEWSWIRE) — GoHealth, Inc. (NASDAQ: GOCO) (“GoHealth” or the “Company”), a leading health insurance marketplace and Medicare-focused digital health company, today announced financial results for the three and nine months ended September 30, 2024.
Third Quarter Highlights
Third quarter 2024 net revenues of $118.3 million, a $13.7 million decrease compared to $132.0 million in the prior year period.
Third quarter 2024 Submissions were 166,195, a 2.9% increase compared to 161,550 Submissions in the prior year period, with strong contributions from GoHealth’s internal captive agents, partially offset by a decline in Submissions from the external GoPartner Solutions (“GPS”) agents.
Third quarter 2024 net income of $15.4 million, an improvement of $71.6 million compared to a net loss of $56.2 million in the prior year period.
Third quarter 2024 Adjusted EBITDA(1) of negative $12.1 million, a decrease of $0.6 million compared to negative $11.5 million in the prior year period.
Third quarter 2024 trailing twelve months (“TTM”) positive cash flow from operations of $35.1 million, an increase of $38.3 million compared to TTM negative cash flow from operations of $3.2 million in the prior year period.
Refinanced Credit Facility, establishing new five-year term with new lender group.
Completed the strategic acquisition of e-TeleQuote Insurance, Inc. (“e-TeleQuote”), adding approximately $90.5 million in contract assets and $22.5 million in cash (inclusive of the Company’s initial $5.0 million investment), and recording a $77.4 million gain on bargain purchase, reinforcing GoHealth’s aspirations to expand its market leadership and operational capacity.
Achieved an 11.0% improvement in Direct Operating Cost per Submission(2) in the third quarter of 2024 compared to the prior year period, through advancements in artificial intelligence (“AI”), automation, and marketing efficiencies, along with targeted operational improvements.
Appointed Brendan Shanahan as Chief Financial Officer, who brings over 30 years of healthcare and financial strategy expertise to GoHealth.
“Our third-quarter results underscore the strength of our ongoing transformation into a Medicare engagement company. We’ve helped over 650,000 consumers navigate Medicare options through tools like PlanFit CheckUp, and with the addition of e-TeleQuote’s 400 agents, we’re prepared to efficiently serve the demand surge we are seeing in this year’s Medicare Annual Enrollment Period (“AEP”). We believe these developments reinforce GoHealth’s leadership in the eBroker space, positioning us for sustained growth and profitability,” said Vijay Kotte, CEO of GoHealth.
“The acquisition of e-TeleQuote not only added $90.5 million in contract assets and $22.5 million in cash (inclusive of our initial $5.0 million investment), but also provided a substantial gain on bargain purchase of $77.4 million, boosting our financial position,” Kotte continued. “This strategic move expanded our agent capacity ahead of a pivotal AEP, which we believe will enable us to capitalize on market demand. As the competitive landscape shifts, GoHealth aims to stand out, ready to support the millions of consumers facing benefit reductions or coverage losses.”
“GoHealth’s third-quarter results reflect disciplined cost management, as we reduced our Direct Operating Cost per Submission(2) by 11.0%, despite broader market challenges. These efficiency gains and a focus on high-quality lead generation underscore our commitment to driving profitable growth, especially as we progress through AEP,” said Brendan Shanahan, CFO of GoHealth. “With over $77 million gained through the e-TeleQuote acquisition, we believe we’re positioned with the liquidity and flexibility needed to drive long-term value, even as we expand our agent base during this critical AEP.”
“This AEP, we’re seeing unprecedented disruption, with over two million consumers losing coverage and more than six million experiencing reduced benefits. We believe GoHealth’s investments in AI-driven technology, an expanded agent network, and our focus on consumer engagement uniquely position us to lead through these market changes, delivering both growth and improved customer experience,” said Kotte.
(1)
Adjusted EBITDA is a non-GAAP measure. For a definition of Adjusted EBITDA and a reconciliation to the most comparable GAAP measure, please see below.
(2)
Direct Operating Cost per Submission is an operating metric. For a definition of Direct Operating Cost per Submission and an explanation of its calculation, please see below.
Conference Call Details
The Company will host a conference call today, Thursday, November 7, 2024 at 8:00 a.m. (ET) to discuss its financial results. A live audio webcast of the conference call will be available via GoHealth’s Investor Relations website, https://investors.gohealth.com/. A replay of the call will be available via webcast for on-demand listening shortly after the completion of the call.
About GoHealth, Inc.
GoHealth is a leading health insurance marketplace and Medicare-focused digital health company whose purpose is to compassionately ensure consumers’ peace of mind when making healthcare decisions so they can focus on living life. For many of these consumers, enrolling in a health insurance plan is confusing and difficult, and seemingly small differences between health plans may lead to significant out-of-pocket costs or lack of access to critical providers and medicines. GoHealth’s proprietary technology platform leverages modern machine-learning algorithms, powered by over two decades of insurance purchasing behavior, to reimagine the process of matching a health plan to a consumer’s specific needs. Its unbiased, technology-driven marketplace coupled with highly skilled licensed agents has facilitated the enrollment of millions of consumers in Medicare plans since GoHealth’s inception. For more information, visit https://www.gohealth.com.
Key Points: – Avid Bioservices will be acquired by GHO Capital and Ampersand Capital in a $1.1 billion deal, with a 13.8% per-share premium. – Acquisition to enhance Avid’s biologics CDMO services with expanded resources for development and manufacturing. – Expected to close in Q1 2025, enabling Avid to operate privately and accelerate its service offerings for the biotechnology sector.
Avid Bioservices, Inc. (NASDAQ: CDMO), a major biologics contract development and manufacturing organization (CDMO), announced its acquisition by GHO Capital Partners LLP and Ampersand Capital Partners for approximately $1.1 billion. The all-cash transaction positions Avid for substantial growth, with the backing of experienced healthcare-focused investors to expand its development and manufacturing capabilities in the biotechnology sector. This acquisition marks a strategic move for GHO and Ampersand, leveraging Avid’s expertise to strengthen their portfolios in the life sciences industry.
Under the agreement terms, GHO and Ampersand will acquire Avid’s outstanding shares for $12.50 each in cash, reflecting a 13.8% premium over Avid’s closing share price and a 21.9% premium over its 20-day average. This values the transaction at $1.1 billion, enhancing Avid’s growth potential within a private company framework where it can develop its offerings with the support of dedicated capital and expanded industry networks.
Alan MacKay and Mike Mortimer, Managing Partners of GHO, expressed their excitement to work with Avid’s team to realize the company’s full potential, calling Avid “an ideal addition” that exemplifies their mission to improve healthcare access through efficient manufacturing and high-quality innovation. The deal will enable Avid to maintain its focus on serving the biotechnology and pharmaceutical sectors, helping it meet growing demand for complex biologics at clinical and commercial stages. Additionally, the acquisition aligns with GHO and Ampersand’s broader healthcare strategies, aiming to optimize Avid’s impact in global markets.
Nick Green, President and CEO of Avid, noted that partnering with GHO and Ampersand comes at an opportune time, as Avid has been strategically expanding to meet a broader range of customer needs. He emphasized that this partnership will further Avid’s impact, positioning it to better serve biopharma innovators by utilizing GHO and Ampersand’s resources. “After years of investment and expansion, now is the right time to move forward as a private company,” Green stated, expressing confidence that this move will significantly enhance Avid’s capabilities.
The acquisition is expected to support Avid’s future projects, including cell line development, CGMP clinical and commercial manufacturing, analytical testing, and expanded services for early-stage programs. This additional support allows Avid to optimize customer offerings, broaden its expertise, and develop new industry capabilities that align with its dedication to quality and regulatory standards.
The acquisition, unanimously approved by Avid’s board of directors, is expected to close in Q1 2025, pending customary approvals. Avid’s shares will no longer be publicly traded after the acquisition, and the company will continue to operate under its current name and brand identity, with its headquarters remaining in Tustin, California. Financial and legal advising for Avid are being handled by Moelis & Company LLC and Cooley LLP, respectively, while GHO and Ampersand are advised by William Blair & Company and Ropes & Gray LLP.
This partnership reflects GHO and Ampersand’s strategic investment approach, enabling Avid to strengthen its leadership in biologics manufacturing and develop solutions that respond to high-growth demand in the industry.
Key Points: – Sonnet BioTherapeutics priced a $5 million public offering to fund research and trials. – The offering sparked a significant increase in Sonnet’s trading volume. – Sonnet advances its FHAB platform with promising cancer treatments like SON-1411 and SON-1010.
Sonnet BioTherapeutics (NASDAQ: SONN), a clinical-stage biotech company specializing in oncology-focused immunotherapies, has priced a $5.0 million underwritten public offering. The offering includes 1,111,111 shares of common stock, each sold with one common warrant for the purchase of an additional two shares, at a combined price of $4.50 per share. This offering is set to close on or around November 7, 2024, and is expected to generate gross proceeds of approximately $5.0 million before underwriting discounts and commissions.
The proceeds from this offering are intended to fund Sonnet’s ongoing research and development, clinical trials, working capital, and liability repayments, advancing the company’s mission to develop novel biologic therapies for cancer treatment. While the offering is an exciting opportunity for the company to secure necessary funding, it also brings with it potential risks, including the possibility of shareholder dilution through the issuance of new shares and warrants.
Notably, Sonnet’s share price has seen increased volatility today, with trading volume significantly surging. This rise in trading activity follows the announcement of the offering and its anticipated closure. As is often the case with at-the-market offerings under Nasdaq rules, the pricing of the shares could pressure the stock value in the short term. However, investors may also be reacting positively to the financial backing that will enable Sonnet to accelerate the clinical development of its promising drug pipeline.
Sonnet is known for its proprietary FHAB (Fully Human Albumin Binding) platform, which enables the development of biologic drugs designed to target tumor and lymphatic tissues more efficiently. This technology utilizes a human single-chain antibody fragment (scFv) to hitch a ride on human serum albumin, guiding the drug directly to the target tissue for improved therapeutic effectiveness. The FHAB platform is adaptable, enabling the creation of a wide range of therapeutic candidates, including cytokines, peptides, antibodies, and vaccines.
One of Sonnet’s leading therapeutic candidates is SON-1411, a novel bifunctional fusion protein designed to enhance the efficacy of the immune response against cancer. SON-1411 combines IL-18BPR (a receptor that binds IL-18) with IL-12 and is linked to the FHAB platform. This innovative approach is aimed at overcoming limitations observed in previous IL-18-based therapies, which suffered from poor efficacy due to the presence of IL-18 binding protein (IL-18BP) in the tumor microenvironment. By modifying the IL-18 domain, SON-1411 seeks to bypass this issue and enhance the therapeutic potential of IL-18 in cancer treatment.
In addition to SON-1411, Sonnet is also advancing SON-1010, an IL-12-FHAB fusion protein, through clinical trials for solid tumors and ovarian cancer. The company is evaluating SON-1010 in collaboration with Roche, in combination with the immune checkpoint inhibitor atezolizumab, for the treatment of platinum-resistant ovarian cancer. Moreover, Sonnet is working on SON-1210, a combination of IL-12-FHAB and IL-15, for the treatment of solid tumors like pancreatic cancer.
Despite the potential dilution concerns stemming from the offering, the announcement underscores the company’s strategic commitment to advancing its promising drug candidates. As Sonnet BioTherapeutics progresses through clinical trials and secures additional funding, the surge in trading volume today suggests strong market interest in the company’s future prospects. The funding from the offering will be crucial in supporting Sonnet’s clinical trials and advancing the company’s vision of delivering targeted, effective treatments for cancer patients.
PRINCETON, N.J., Nov. 06, 2024 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines, today announced that the Company will host a conference call to report financial results for the quarter ended September 30, 2024, and provide a clinical program update on Thursday, November 14, 2024, at 8:30 a.m. Eastern Time.
Conference Call Details
Date: November 14, 2024 Time: 8:30 a.m. Eastern Time Dial-in: 1-877-704-4453 or 1-201-389-0920 Webcast Registration: Click Here Call MeTM Registration: Click Here (Available 15 minutes prior to call)
About PDS Biotechnology PDS Biotechnology is a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines. The Company plans to initiate a pivotal clinical trial in 2024 to advance its lead program in advanced HPV16-positive head and neck squamous cell cancers. PDS Biotech’s lead investigational targeted immunotherapy Versamune® HPV is being developed in combination with a standard-of-care immune checkpoint inhibitor, and also in a triple combination including PDS01ADC, an IL-12 fused antibody drug conjugate (ADC), and a standard-of-care immune checkpoint inhibitor. We believe that proof-of-concept long-term data have shown positive survival results and tumor shrinkage with these combinations and indicate favorable tolerability.
We believe that the novel investigational mechanisms of action of Versamune® HPV and the combination of Versamune® HPV and PDS01ADC have resulted in compelling results with potential to successfully disrupt a tumor’s internal defenses, while also generating potent, targeted killer T-cells to attack the tumor. We also believe that data from more than 350 patients, as well as ongoing clinical trials across multiple tumor types and standard treatment regimens, have validated the potential for both platforms and point to potential broad utility.
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 Versamune® HPV, PDS01ADC, 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 Versamune® HPV, PDS01ADC 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 the Company’s 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; the Company’s ability to continue as a going concern; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control. 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 other risks, uncertainties, and other factors described under “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in the documents we file with the U.S. Securities and Exchange Commission. 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® and Infectimune® are registered trademarks of PDS Biotechnology Corporation.
Late breaking abstract to provide new updates from THIO-101 Phase 2 clinical trial in non-small cell lung cancer (NSCLC)
Poster to highlight long-term therapeutic benefits of THIO sequenced with cemiplimab beyond treatment cessation
CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that a late-breaking abstract (LBA) detailing new updates from its Phase 2 THIO-101 clinical trial was selected for oral and poster presentation at the 2024 Annual Meeting of the Society for Immunotherapy of Cancer (SITC), being held November 6-10, 2024, in Houston, Texas. The updates will include new data on efficacy and safety from its clinical trial evaluating THIO sequenced with Regeneron’s immune checkpoint inhibitor (CPI) cemiplimab (Libtayo®) in patients with advanced non-small cell lung cancer (NSCLC) who have failed two or more standard-of-care therapy regimens.
“We are honored to have our THIO-101 data recognized by SITC in a late-breaking abstract, a category reserved for research that has the potential to change medical practices. We believe that our latest data is compelling and further supports the ability of THIO to produce cancer cell specific immune memory and to remain active against cancer cells after extended periods of time,” said Vlad Vitoc, M.D., Chairman and CEO of MAIA. “Our findings to date are particularly significant for advanced-stage patients resistant to CPI and chemotherapy treatments who are in desperate need of new treatment options. In our opinion, the combination of THIO with a CPI is showing promise as a durable and effective NSCLC treatment.”
Presentation details:
Title:
Telomere-Targeting Agent THIO in Sequential Combination with Cemiplimab Demonstrates Long Term Therapeutic Benefits Beyond Treatment Cessation — A Phase 2 Trial in Advanced Immune Checkpoint Inhibitor Resistant Non-Small Cell Lung Cancer Patients
According to SITC, a late-breaking abstract (LBA) submission is solely for abstracts with late-breaking data from interventional clinical trials in humans. The reference does not refer to abstracts that are submitted “late,” as in after submission deadlines.
As of August 1, 2024, 16 patients in the THIO-101 trial had survival follow-up surpassing 12 months, including 9 in third line treatment (3L). Interim median survival follow-up in 3L was 10.6 months. THIO’s substantial survival benefit in third line NSCLC surpasses current standard-of-care overall survival of 5.8 months.1
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 Regeneron’s PD-1 inhibitor 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 THIO followed by cemiplimab (Libtayo®) 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.
_____________________________ 1 Girard N, et al. J Thorac Onc 2009;12:1544-1549.
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.
Interim Update From Phase 2 Trial Scheduled For SITC. MAIA is scheduled to present a data update from the ongoing THIO-001 trial at a Late-Breaker session of the STIC (Society For The Immunotherapy of Cancer) Annual meeting on Saturday, November 9. The presentation could include data on endpoints and measures of efficacy such as disease control rate, overall survival, and median survival with additional patients and longer treatment times.
Trial Treats Patients With THIO and A Checkpoint Inhibitor. The THIO-001 trial enrolled advanced non-small cell lung cancer (NSCLC) patients that had progressive disease and no longer responded after two or more standard-of-care therapy regimens. Patients were treated with the combination of 3-week cycles of THIO and the standard dose of cemiplimab (350 mg Libtayo, an anti-PD-1 checkpoint inhibitor from Regeneron). Patient enrollment was completed in February 2024.
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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.
Proposed clinical study in Kenya intended to test the safety, tolerability, and immunogenicity of TNX-801, a vaccine being developed to prevent mpox (formerly called monkeypox)
The World Health Organization (WHO) declared mpox a public health emergency of international concern (PHEIC)1-4: cases of the new Clade Ib mpox detected in Sweden, Thailand, Singapore, India, Germany and England
In preclinical data, TNX-801, Tonix’s attenuated live-virus vaccine candidate, demonstrated efficacy as a vaccination against mpox in animal models
CHATHAM, N.J., Nov. 04, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that it has entered into a sponsored research agreement with the Kenya Medical Research Institute (KEMRI) to design, plan and seek regulatory approval for a Phase I clinical study in Kenya to test the safety, tolerability, and immunogenicity of TNX-801 (horsepox, live virus) as a vaccine to prevent mpox and smallpox. Tonix will be the sponsor and KEMRI will lead the execution of the proposed clinical trial.
“We are excited to advance development of TNX-801 under this research agreement with KEMRI,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “There is an urgent need for the worldwide availability of a single dose mpox vaccine with durable protection. TNX-801 has demonstrated encouraging preclinical data and was recently shown to align with the World Health Organization’s (WHO’s) newly issued preferred target product profile (TPP) for mpox vaccines. Further, TNX-801 can be scaled up for manufacturing, distribution and storage without a costly supply chain. We believe TNX-801 has the potential to address and help interrupt the spread of mpox worldwide.”
In September 2024, Tonix announced that the preferred target product profile (TPP), released at the WHO sponsored Mpox Research and Innovation Scientific Conference, aligns with the potential characteristics of TNX-801. Key elements of the WHO draft TPP include single-dose, durable protection, administration without special equipment, and stability at ambient temperature. Other potential beneficial characteristics include the ability to limit forward transmission, use in case-contact vaccination strategies and suitability for use in immunocompromised individuals.
In August 2024, Tonix announced a collaboration with Bilthoven Biologics (Bbio) to develop GMP manufacturing processes for TNX-801. Bbio is part of the world’s largest vaccine manufacturer, the Cyrus Poonawalla Group, which also includes the Serum Institute of India.
About TNX-801*
TNX-801 is a live minimally-replicating attenuated horsepox vaccine that is believed to provide immune protection against mpox in animals with better tolerability than 20th century vaccinia viruses. Vaccinia vaccines from the 20th century are descendants of Edward Jenner’s circa 1800 that have become more virulent in connection with losing regulatory elements in their genomes. Given the modern understanding that Jenner’s circa 1800 vaccine would be called “horsepox” today, TNX-801 was designed to be similar to Jenner’s vaccine. After a single dose vaccination, TNX-801 protected animals against lethal challenge with intratracheal Clade Ia monkeypox virus.5 In this experiment, TNX-801 vaccination prevented clinical disease and skin lesions and also decreased shedding in the mouth and lungs. The findings are consistent with mucosal immunity and suggest the ability to block forward transmission. In addition, TNX-801 has decreased virulence in immunocompromised animals relative to 20th Century vaccinia viruses.6 Based on animal studies, TNX-801 combines immune protection with improved tolerability compared to live-virus vaccinia vaccines. TNX-801 is administered with a single dose which has advantages over two-dose regimens. The focus on single-dose vaccines confirms early recommendations by the Bipartisan Commission on Biodefense, 7 and the U.S. National Academies of Science.7,8 The National Academies of Science (NAS) report highlights the difficulty of a ring vaccination strategy with even a two-dose regimen.7 TNX-1800 is an engineered version of horsepox that expresses the spike protein of SARS-CoV-2. 9,10 The U.S. National Institutes of Health (NIH) selected Tonix’s COVID-19 vaccine, TNX-1800 for Project NextGen.
About Mpox* On August 14, 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a PHEIC, the second such declaration in the past two years called in response to an mpox outbreak.1 The current outbreak is caused by Clade Ib monkeypox virus while the 2022 outbreak was caused by Clade IIb monkeypox virus. The 2022 global mpox outbreak, which is ongoing, has affected over 90,000 persons in countries where mpox had previously not been endemic, including Europe and the US. The rapid spread of Clade IIb strain mpox in 2022 underscores the pandemic potential of mpox. Unlike Clade IIb mpox, the Clade Ib strain of mpox appears to be spreading in Africa, particularly those neighboring the Democratic Republic of the Congo, including Burundi, Rwanda, and Uganda, but now affecting 16 countries. According to the U.S. Centers for Disease Control and Prevention (CDC), and other experts, there is a significant risk that the Clade Ib monkeypox may appear in the U.S.2,3
Tonix Pharmaceuticals Holding Corp.* Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to progress TNX-102 SL, a product candidate for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation and its development is supported by a grant from the U.S. National Institude of Drug Abuse and Addiction. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix has a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
* Tonix’s product development candidates are investigational new drugs or biologics; their efficacy and safety have not been established 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.
1WHO Press Release August 14, 2024. “WHO Director-General declares mpox outbrfeak a public health emergency of international concern”. URL: www.who.int/news/item/14-08-2024-who-director-general-declares-mpox-outbreak-a-public-health-emergency-of-international-concern (accessed 8-15-24) 2McQuiston JH, et al. U.S. Preparedness and Response to Increasing Clade I Mpox Cases in the Democratic Republic of the Congo. 2024, MMWR Morbi Mortal Wkly Rep: United States. p. 435-440 3CDC. 2022-2023 Mpox: US Map and Case Count. https://www.cdc.gov/poxvirus/mpox/response/2022/us-map.html 4World Health Organization SAGE meeting highlights on updated mpox vaccine recommendations. 2024, March 5Noyce RS, et al. Viruses. 2023 Jan 26;15(2):356. Doi: 10.3390/v15020356. PMID: 36851570; PMCID: PMC9965234 6Trefry, SV et al. bioRxiv 2023.10.25.564033; doi: https://doi.org/10.1101/2023.10.25.564033 7Bipartisan Commission on Biodefense. Box the Pox: Reducing the risk of Smallpox and Other Ortho poxviruses, Washington:2024 8U.S. National Academies of Science. Future State of Smallpox Medical Countermeasures. Washington:2024 9Awasthi M, et al. Viruses. 2023 Oct 21;15(10):2131. Doi: 10.3390/v15102131. PMID: 37896908; PMCID: PMC10612059. 10Awasthi M et al Vaccines (Basel). 2023 Nov 2;11(11):1682. Doi: 10.3390/vaccines11111682.PMID: 38006014
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 Toni’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, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, and periodic reports filed with the SEC on or after the date thereof. All of Toni’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.
New data show tolerability and no evidence of spreading to blood or tissues even at high doses of Tonix’s attenuated live-virus, minimally replicating vaccine candidate TNX-801 in immunocompromised animals
TNX-801, has characteristics that align closely with the World Health Organization’s (WHO) preferred target product profile (TPP) criteria for mpox vaccines
WHO-declared public health emergency of international concern (PHEIC)1-4: Mpox cases of the new clade Ib mpox detected in Sweden, Thailand, Singapore, India, Germany and England
TNX-801 vaccination demonstrated efficacy in protecting animals from lethal challenge with clade I monkeypox and is in development as an mpox vaccine
CHATHAM, N.J., Nov. 01, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company with marketed products and a pipeline of development candidates, presented data in an oral presentation at the World Vaccine Congress-Europe 2024, held October 28-31, 2024 in Barcelona, Spain. A copy of the Company’s presentation is available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com following the conference.
The presentation titled, “A Novel, Single-dose, Live, Attenuated, Minimally Replicating Mpox Vaccine”, highlighted positive preclinical efficacy data, demonstrating tolerability in immunocompromised animals and showed that TNX-801 is unable to spread in blood or tissues in these animals, even at an approximately 100-fold higher dose than 20th century vaccinia vaccines.
TNX-801 is an attenuated live-virus vaccine based on synthesized horsepox that has been shown to provide single-dose immune protection against a monkeypox challenge with better tolerability than 20th century vaccinia live-virus vaccines in animals. TNX-801 has previously been shown to protect animals against lethal challenge with intratracheal clade I monkeypox virus.1 After a single dose vaccination, TNX-801 prevented clinical disease and lesions and also decreased shedding in the mouth and lungs of animals challenged with clade I monkeypox.1 These findings are consistent with TNX-801 inducing mucosal immunity and suggest TNX-801 has the ability to block forward transmission. An outbreak of clade I mpox has recently been declared a Public Health Emergency of International Concern (PHEIC) by the World Health Organization (WHO).2,3 Starting from an outbreak in the Democratic Republic of the Congo, clade I mpox has spread to sixteen Central African Countries and cases have been reported in Sweden, Thailand, Singapore, India, Germany and England. According to the U.S. Centers for Disease Control and Prevention (CDC), and other experts, there is a significant risk that clade I strain may appear in the U.S.4
“Data continue to support TNX-801’s strong tolerability and efficacy profiles by continually displaying protective immunity to animals with single-dose administration,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “Synthetic biology is an important technology for vaccine development as viral diseases continue to rapidly evolve. The new data demonstrate that TNX-801 is highly attenuated relative to 20th century vaccinia vaccines in immunocompromised animals.5 With TNX-801’s target profile, favorable shipping and storing requirements and our manufacturing collaboration agreements, we believe TNX-801 is in a strong position to make an impact towards preventing mpox and control mpox epidemics.”
In September 2024, Tonix announced that the World Health Organization’s (WHO’s) preferred target product profile (TPP), released at the WHO sponsored Mpox Research and Innovation Scientific Conference, aligns with the characteristics of TNX-801. Key elements of the WHO draft TPP include single-dose, durable protection, administration without special equipment, and stability at ambient temperature. Other potential beneficial characteristics include the ability to limit forward transmission, use in case-contact vaccination strategies and suitability for use in immunocompromised individuals.
In August 2024, Tonix announced a collaboration with Bilthoven Biologics (Bbio) to develop GMP manufacturing processes for its mpox vaccine. Bbio is part of the world’s largest vaccine manufacturer, the Cyrus Poonawalla Group, which also includes the Serum Institute of India.
About TNX-801* TNX-801 is a live replicating attenuated vaccine based on horsepox that is believed to provide immune protection with better tolerability than 20th century vaccinia viruses. As previously disclosed, TNX-801 protected animals against lethal challenge with intratracheal clade I monkeypox virus.1 After a single dose vaccination, TNX-801 prevented clinical disease and lesions and also decreased shedding in the mouth and lungs of non-human primates.1 The Findings are consistent with mucosal immunity and suggest the ability to block forward transmission, similar to Dr. Edward Jenner’s vaccinia vaccine, which eradicated smallpox and kept mpox out of the human population. On August 26, 2024, Tonix announced a collaboration to develop GMP manufacturing processes for its mpox vaccine with Bilthoven Biologics (Bbio), part of the world’s largest vaccine manufacturer, the Cyrus Poonawalla Group, which also includes the Serum Institute of India.
On the horsepox platform, Tonix is developing TNX-1800 (horsepox expressing SARS-CoV-2 spike protein) for protecting against COVID-19. TNX-1800 is an engineered version of horsepox that expresses the spike protein of SARS-CoV-2. In preclinical studies of TNX-1800 highlighted in the presentation, TNX-1800 was tested for immunogenicity and efficacy of TNX-1800 in nonhuman primates following a SARS CoV-2 challenge. 6,7 TNX-1800 vaccination results in a neutralizing antibody response that was associated with significant reduction in virus replication/shedding in the respiratory tract and tolerability. 2,3 TNX-1800 was selected by the NIH’s, Project NextGen for inclusion in clinical trials as part of a select group of next generation COVID-19 vaccine candidates with the intent to identify promising vaccine platforms. NIH plans to conduct a Phase 1 trial of TNX-1800 and cover the full cost of the study, while Tonix provides the vaccine candidate.
About Mpox* On August 14, 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a public health emergency of international concern, the second such declaration in the past two years called in response to an mpox outbreak. The current outbreak was caused by clade I monkeypox virus, while the 2022 outbreak was clade II monkeypox virus. The global mpox outbreak, which commenced in 2022 has affected over 90,000 persons in countries where mpox had previously not been endemic, including Europe and the US. The spread of clade IIb strain mpox in 2022 underscores the pandemic potential of mpox. Unlike clade IIb mpox, the clade I strain of mpox appears to be spreading to countries neighboring the Democratic Republic of the Congo. According to the U.S. Centers for Disease Control and Prevention (CDC), and other experts, there is a significant risk that the deadlier clade I strain may appear in the U.S.4
Tonix Pharmaceuticals Holding Corp.* Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to progress TNX-102 SL, a product candidate for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation and its development is supported by a grant from the U.S. National Institude of Drug Abuse and Addiction. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
*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.
1Noyce RS, et al. Viruses. 2023 Jan 26;15(2):356. Doi: 10.3390/v15020356. PMID: 36851570; PMCID: PMC9965234 2WHO Press Release August 14, 2024. “WHO Director-General declares mpox outbrfeak a public health emergency of international concern”. URL: www.who.int/news/item/14-08-2024-who-director-general-declares-mpox-outbreak-a-public-health-emergency-of-international-concern (accessed 8-15-24) 3McQuiston JH, et al. U.S. Preparedness and Response to Increasing Clade I Mpox Cases in the Democratic Republic of the Congo. 2024, MMWR Morbi Mortal Wkly Rep: United States. p. 435-440 4CDC. 2022-2023 Mpox: US Map and Case Count. 5Trefry, SV et al. bioRxiv 2023.10.25.564033; doi: https://doi.org/10.1101/2023.10.25.564033 6Awasthi M et al Vaccines (Basel). 2023 11(11):1682. doi: 10.3390/vaccines11111682.PMID: 38006014 7Awasthi M, et al. Viruses. 2023 15(10):2131. doi: 10.3390/v15102131. PMID: 37896908; PMCID: PMC10612059.
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, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, 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.
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.
Islet Cell Transplant Patients Achieve Insulin Independence. A clinical study using tegoprubart as an immunosuppressant in islet cell transplantation was presented at a medical meeting on Tuesday, October 29, 2024. The first two subjects receiving islet cell transplants with tegoprubart as an immunosuppressant were able to regulate their blood glucose and achieve insulin independence. We see this as a significant advance that could enable islet cell transplantation to become a treatment for diabetes.
Tegoprubart Prevented The Toxicities That Have Led To Failure. Previous attempts to transplant healthy islet cells to restore insulin production have been unsuccessful. This is partly due to side effects of tacrolimus, the immunosuppression drug that is effective but toxic to the kidney and islet cells. This trial used tegoprubart instead of tacrolimus, resulting in islet cells surviving, engrafting, and producing insulin at effective levels.
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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.
The Company’s antiviral candidate for seasonal and pandemic influenza shows in vitro activity against the avian influenza A PB2 protein, whereas current influenza vaccines offer no protection against pandemic avian influenza
BOTHELL, Wash., Oct. 31, 2024 (GLOBE NEWSWIRE) — As the fall seasonal influenza and COVID season gets underway in the Northern Hemisphere, Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) highlights the ability of the Company’s innovative structure-based drug discovery platform technology to discover and develop novel broad-spectrum antivirals therapeutics to treat a wide range of viral diseases, including newly emerging pandemic strains such as recent H5N1 avian influenza identified in the US.
“As our nation enters the fall flu and COVID season and learns of the emerging highly pathogenic avian H5N1 influenza A strain in dairy cattle and humans, the need for more effective antivirals is clear. Current flu vaccines are developed for seasonal influenza strains, not for pandemic avian influenza strains. Also, treatment-emergent resistance to approved antivirals and transmission of resistant viruses has been a challenging issue,” said Sam Lee, PhD, President and co-CEO of Cocrystal. “For example, the widespread oseltamivir (Tamiflu®) resistance of the pandemic avian influenza strains could create a serous public health situation. Clearly, there continues to be an unmet need for therapeutics with a high barrier to resistance.
“We believe our approach makes it possible to develop highly effective therapeutics for noroviruses, coronaviruses and influenza A because we target the highly conserved, essential function of viral enzymes, regardless of whether the strain is seasonal or pandemic,” he added. “We recently revealed the high-resolution cocrystal structure of the avian influenza PB2 protein complexed with CC-42344, further confirming that our PB2 inhibitor CC-42344 binds to its highly conserved PB2 region, indicating activity against this strain.”
Cocrystal’s platform utilizes Nobel Prize-winning technology to develop a new class of direct-acting antivirals that work against enzymes that are essential for viral replication. The Company is evaluating its oral CC-42344 in a Phase 2a study in healthy subjects infected with a seasonal influenza A strain. Topline safety and tolerability results from this trial are expected by the end of 2024, and preparations are underway for an Investigational New Drug (IND) application to conduct a late-stage clinical study with CC-42344 in the U.S.
The Company is also conducting a Phase 1 study with its oral protease inhibitor CDI-988, the first pan-viral drug candidate in clinical evaluation for both noroviruses and coronaviruses. Topline safety and tolerability results of the multiple-ascending cohorts are expected late this year or early next year. There is no approved vaccine or antiviral for norovirus. Norovirus is highly contagious and is the most common cause of acute gastroenteritis, which has gained notoriety for outbreaks in closed quarters such as on cruise ships and in nursing homes. According to the Centers for Disease Control and Prevention (CDC), an estimated 685 million cases and an estimated 200,000 deaths are attributed to norovirus each year worldwide, with an estimated societal cost of approximately $60 billion.
“We view the next viral pandemic as a question of timing as seasonal viruses like flu and COVID continue to evolve,” said James Martin, CFO and co-CEO of Cocrystal. “Our proprietary technology platform allows us to efficiently discover and develop potent, broad-spectrum, effective antiviral drug candidates for pandemic and seasonal outbreaks relatively quickly and far less costly than traditional approaches to drug development. As a small company, Cocrystal is highly efficient in utilizing our groundbreaking technology to develop differentiated antivirals for high-value indications with the goal of improving people’s lives.”
Avian Influenza A multistate outbreak of highly pathogenic avian influenza in dairy cows was initially reported in March 2024. This is the first time that avian flu viruses were found in cows, with outbreaks now confirmed in herds in 14 states. In April 2024 the CDC confirmed an avian flu infection in a person exposed to dairy cows that were presumed to be infected with the virus. This is believed to be the first instance of likely mammal to human spread of this virus. In September 2024 the CDC reported the first human case of avian influenza without a known occupational exposure to sick or infected animals. As of October 30, 2024, the CDC has reported 36 human cases of this highly pathogenic avian influenza A in the U.S. during 2024.
The CDC analyzed blood collected from people of all ages in all 10 Health & Human Services regions during the 2022-2023 and 2021-2022 flu seasons. These samples were challenged with the avian flu subtype H5N1 virus to determine whether there was an antibody reaction. Data from this study suggest that there is extremely low to no population immunity to clade 2.3.4.4b A (H5N1) viruses in the U.S. Antibody levels remained low regardless of whether or not participants received a seasonal flu vaccination, meaning that seasonal flu vaccination did not produce antibodies to avian flu H5N1 viruses.
Antiviral Influenza Candidate CC-42344 CC-42344 is Cocrystal’s novel, broad-spectrum investigational antiviral candidate for the treatment of pandemic and seasonal influenza A. CC-42344 inhibits the first step in influenza A’s viral replication by binding to a highly conserved PB2 site of the influenza polymerase complex that is essential to replication and was discovered using Cocrystal’s proprietary structure-based drug discovery platform technology.
Cocrystal is evaluating safety, viral and clinical measures of oral CC-42344 in healthy volunteers who are challenged with influenza A in a Phase 2a human challenge study underway in the United Kingdom. CC-42344 was advanced into this study following favorable safety and tolerability results reported in a Phase 1 study in healthy volunteers conducted in Australia. 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 pharmacokinetic and safety profiles.
Cocrystal used its structure-based platform to determine the high resolution X-ray crystal structure of the recent avian influenza A (H5N1) PB2 protein, and confirmed activity of CC-42344 in vitro (NIH GeneBank ID:influenza A/Texas/37/2024(H5N1). The crystal structure of the avian influenza A (H5N1) PB2 protein showed new mutations located outside the PB2 active site. Subsequent studies showed that CC-42344 binds to the active site of the avian influenza A (H5N1) PB2 protein as previously demonstrated with the pandemic and seasonal influenza A PB2.
Structure-Based Platform Technology Cocrystal’s proprietary structural biology, along with its expertise in enzymology and medicinal chemistry, enable its development of novel antiviral agents. The Company’s platform provides a three-dimensional structure of inhibitor complexes at near-atomic resolution, providing immediate insight to guide Structure Activity Relationships. This helps to identify novel binding sites and allows for a rapid turnaround of structural information through highly automated X-ray data processing and refinement. The goal of this technology is to facilitate the development of best-in-class antiviral therapies that have fast onset of action and/or shortened treatment time, are safe, well tolerated and easy to administer, are effective against all viral subtypes that cause disease and have a high barrier to viral resistance.
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), noroviruses, and hepatitis C viruses. 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.
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 regarding the potential efficacy of Cocrystal’s product candidates against certain viruses, ongoing research and development efforts including the expected timing of clinical studies and topline results for such product candidates, and the potential market for such product candidates. 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, risks relating to our ability to obtain regulatory authority for and proceed with clinical trials including the recruiting of volunteers for such studies by our clinical research organizations and vendors, the results of such studies, our collaboration partners’ technology and software performing as expected, general risks arising from clinical studies, receipt of regulatory approvals, regulatory changes, and potential development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government, and potential mutations in a virus we are targeting that may result in variants that are resistant to a product candidate we develop. 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, 2023. 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.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
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Eledon Announces Islet Cell Transplant Patients Achieve Insulin Independence. A clinical study using tegoprubart as an immunosuppressant in islet cell transplantation was presented at a medical meeting on Tuesday, October 29, 2024. The first two subjects treated with tegoprubart as an immunosuppressant were able to control blood glucose within the normal range and achieve insulin independence. We see this as a significant advance that could allow islet cell transplantation to allow diabetics to become insulin independent.
Tegoprubart Prevented The Toxicities That Have Led To Failure. Previous attempts to transplant healthy islet cells to restore insulin production have been unsuccessful. This is partly due to the side effects of tacrolimus, the standard of care for immunosuppression that is effective but toxic to the kidney and islet cells. In this trial, tegoprubart was used instead of tacrolimus, with results showing the islet cells were able to survive, engraft, and produce insulin and without the toxicities associated with tacrolimus.
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