Category: Health

Release – Tonix Pharmaceuticals Announces Presentation of Licensed Antiviral Drug Technology at the 2nd Wnt & β-catenin Targeted Drug Development Conference

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Research News and Market Data on TNXP

January 26, 2023 4:15pm ESTDownload as PDF

Oral Presentation Describes Activity of Wnt/β-catenin Signaling Pathway Inhibitors Against SARS-CoV-2 in Cell Culture and in an Animal Model

CHATHAM, N.J., Jan. 26, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that Tom Hobman, Ph.D., Professor of Cell Biology, University of Alberta, presented data from his laboratory at The University of Alberta during a presentation at the 2nd Wnt & β-catenin Targeted Drug Development Conference held in Boston, Mass., on January 26, 2023. The oral presentation titled, “Targeting the Wnt/β -catenin pathway as a broad-spectrum antiviral strategy,” includes research sponsored by Tonix Pharmaceuticals focused on the development and testing of Wnt/β-catenin signaling pathway inhibitors as broad-spectrum antivirals against SARS-CoV-2 and other emerging viruses. Tonix has previously announced that it exercised an option to license the antiviral technology platform. A copy of the presentation is available on the Tonix Pharmaceuticals corporate website at www.tonixpharma.com.

“Antiviral therapeutics are needed to mitigate the effects of SARS-CoV-2 and future coronavirus outbreaks, and Professor Hobman’s work is designed to facilitate the identification and testing of novel broad-spectrum antiviral drugs,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Professor Hobman presented data showing that inhibition of Wnt/β-catenin pathway induces peroxisomes and enhances interferon response during viral infection, significantly reducing SARS-CoV-2 replication in vitro and in vivo.”

“For future pandemics, the scientific community must be ready with an arsenal of easily self-administered drugs that can be tested in rapid, efficient clinical trials immediately after the causative viral agent is identified,” said Professor Tom Hobman. “The research collaboration between Tonix and The University of Alberta is focused on the development and testing of Wnt/β-catenin signaling pathway inhibitors as broad-spectrum antivirals against SARS-CoV-2 and other emerging viruses.”

Tonix Pharmaceuticals Holding Corp.*

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022 and interim data expected in the second quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix initiated a Phase 2 study in Long COVID in the third quarter of 2022 and expects interim data in the third quarter of 2023. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the first quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is expected to enter the clinic with a Phase 2 study in the first quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily formulation of tianeptine being developed as a potential treatment for major depressive disorder (MDD) with a Phase 2 study expected to be initiated in the first quarter of 2023. Tonix’s rare disease portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the first half of 2023. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox, TNX-801, a next-generation vaccine to prevent COVID-19, TNX-1850, a platform to make fully human monoclonal antibodies to treat COVID-19, TNX-3600, and humanized anti-SARS-CoV-2 monoclonal antibodies, TNX-3800, recently licensed from Curia. TNX-801, Tonix’s vaccine in development to prevent smallpox and monkeypox, also serves as the live virus vaccine platform or recombinant pox vaccine (RPV) platform for other infectious diseases. A Phase 1 study of TNX-801 is expected to be initiated in Kenya in the second half of 2023.

*All of Tonix’s product candidates are investigational new drugs or biologics and have not been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
[email protected]
(862) 904-8182

Olipriya Das, Ph.D. (media)
Russo Partners
[email protected]
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
[email protected]
(443) 213-0505

Source: Tonix Pharmaceuticals Holding Corp.

Released January 26, 2023

Item 9 Labs (INLB) – Reports 4Q and Full Year 2022 Results

Posted on by [email protected]


Thursday, January 26, 2023

Item 9 Labs Corp. (OTCQX: INLB) is a vertically integrated cannabis operator and dispensary franchisor delivering premium products from its large-scale cultivation and production facilities in the United States. The award-winning Item 9 Labs brand specializes in best-in-class products and user experience across several cannabis categories. The company also offers a unique dispensary franchise model through the national Unity Rd. retail brand. Easing barriers to entry, the franchise provides an opportunity for both new and existing dispensary owners to leverage the knowledge, resources, and ongoing support needed to thrive in their state compliantly and successfully. Item 9 Labs brings the best industry practices to markets nationwide through distinctive retail experience, cultivation capabilities, and product innovation. The veteran management team combines a diverse skill set with deep experience in the cannabis sector, franchising, and the capital markets to lead a new generation of public cannabis companies that provide transparency, consistency, and well-being. Headquartered in Arizona, the company is currently expanding its operations space by up to 640,000-plus square feet on its 50-acre site, one of the largest properties in Arizona zoned to grow and cultivate flower. For additional information, visit https://investors.item9labscorp.com/.

Joe Gomes, Managing Director – Generalist Analyst, 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.

4Q22 Results. Item 9 Labs filed its 10K last week for the fiscal year ending September 30, 2022. While the Company did not press release the results, we backed into the 4Q22 results which shows revenue of $4 million and a $15 million operating loss, driven by a $409,000 impairment charge and a $9.5 million loss on assets held for sale. Item 9 Labs recorded a net loss for the quarter of $18.4 million, or a loss of $0.19 per share. We had forecast revenue of $5.5 million, a $2.2 million operating loss, and a net loss of $3.9 million, or a loss of $0.04 per share.

Still Winning Awards. Item 9 Labs continues to pile up awards for its products, which we believe will not only help the Company through the current challenging market but also positions the Company for above average growth as the cannabis market rebounds. BDSA recognized Item 9 Labs as one of the top 10 cannabis brands in Arizona overall, along with top five placements across multiple vape categories.


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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. 

Release – PDS Biotech Announces Participation in the Channelchek Takeaway Series

Posted on by [email protected]

Research News and Market Data on PDSB

FLORHAM PARK, N.J., Jan. 26, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, today announced its participation in the Channelchek Takeaway Series from the J.P. Morgan Healthcare Conference to be broadcast Thursday, January 26, starting at 10:00 a.m., EST. The event will include an overview of PDS Biotech’s business and clinical development strategy from Frank K. Bedu-Addo, Ph.D., President and CEO, followed by a question and answer session with Noble Capital Markets’ Senior Analyst Robert LeBoyer.

Details regarding the event are as follows:

Event:Channelchek Takeaway Series
Date:January 26, 2023
Time:10:00 a.m., EST
Registration:https://us02web.zoom.us/webinar/register/WN_8AW1yjzKSIeqE1satmhSrQ

The J.P. Morgan Healthcare Conference is considered to be the most important healthcare investment symposium in the industry, connecting global industry leaders, emerging fast-growth companies, innovative technology creators, and members of the investment community. Noble Capital Markets’ equity analysts and investment bankers attended the conference and sat down with various c-suite executives. For the Channelchek Takeaway Series, Noble’s analysts are unpacking what they learned at the conference and talking to a selection of c-suite executives in the healthcare space.

The event will be broadcast starting at 10:00 a.m., EST on Thursday, January 26. Investors can attend the Channelchek Takeaway Series virtually at no cost. Registration details are available on Channelchek.

About PDS Biotechnology

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune®, PDS0301, and Infectimune™ T cell-activating platforms. We believe our targeted Versamune® and PDS0301 based candidates have the potential to overcome the limitations of current immunotherapy approaches through the activation of the right type, quantity and potency of T cells. To date, our lead Versamune® clinical candidate, PDS0101, has demonstrated the ability to reduce tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV16-associated cancers in multiple Phase 2 clinical trials. Our Infectimune™ based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

Forward Looking Statements

This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance,” “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune® and Infectimune™ based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune® and Infectimune™ based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Versamune® is a registered trademark and Infectimune™ is a trademark of PDS Biotechnology.

Investor Contacts:
Deanne Randolph
PDS Biotech
Phone: +1 (908) 517-3613
Email: [email protected]

Rich Cockrell
CG Capital
Phone: +1 (404) 736-3838
Email: [email protected]

Media Contacts:
Tiberend Strategic Advisors, Inc.
Dave Schemelia
Phone: +1 (609) 468-9325
[email protected]

Bill Borden 
Phone: +1 (732) 910-1620
[email protected]

Release – Onconova Therapeutics Announces Participation in The Channelchek Takeaway Series

Posted on by [email protected]

Research News and Market Data on ONTX

NEWTOWN, Pa., Jan. 26, 2023 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced their participation in the Channelchek Takeaway Series from the J.P.Morgan Healthcare Conference, to be broadcast Thursday, January 26, starting at 10:00 EST.

The J.P. Morgan Healthcare Conference is considered to be the most important healthcare investment symposium in the industry, connecting global industry leaders, emerging fast-growth companies, innovative technology creators, and members of the investment community. Noble Capital Markets’ equity analysts and investment bankers attended the conference and sat down with various c-suite executives. For the Channelchek Takeaway Series, Noble’s analysts are unpacking what they learned at the conference and talking to a selection of c-suite executives in the healthcare space.

Steven M. Fruchtman, MD, President and Chief Executive Officer of Onconova, provides a corporate overview, then takes questions from Noble’s Senior Analyst Robert LeBoyer.

The event will be broadcast starting at 10:00 am EST on Thursday, January 26. Investors can attend the Channelchek Takeaway Series virtually at no cost. Registration details are available on Channelchek.

About Onconova Therapeutics, Inc.

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose escalation and expansion studies. These trials are currently underway in the United States and China. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also planning a combination trial of narazaciclib with estrogen blockade in advanced endometrial cancer, as well as its clinical study in additional indications.

Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies, including a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, and a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa.

For more information, please visit www.onconova.com.

About Noble Capital Markets

Noble Capital Markets, Inc. was incorporated in 1984 as a full-service SEC / FINRA registered broker-dealer, dedicated exclusively to serving underfollowed small / microcap companies through investment banking, wealth management, trading & execution, and equity research activities. Over the past 37 years, Noble has raised billions of dollars for these companies and published more than 45,000 equity research reports. www.noblecapitalmarkets.com email: [email protected]

About Channelchek

Channelchek (.com) is a comprehensive investor-centric portal – featuring more than 6,000 emerging growth companies – that provides advanced market data, independent research, balanced news, video webcasts, exclusive c-suite interviews, and access to virtual road shows. The site is available to the public at every level without cost or obligation. Research on Channelchek is provided by Noble Capital Markets, Inc., an SEC / FINRA registered broker-dealer since 1984. www.channelchek.com email: [email protected]

Company Contact:
Mark Guerin
Onconova Therapeutics, Inc.
267-759-3680
[email protected]
https://www.onconova.com/contact/

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
[email protected]

Release – Cocrystal Pharma Announces Participation in Virtual Investor Events

Posted on by [email protected]

JANUARY 26, 2023

BOTHELL, Wash., Jan. 26, 2023 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) announces the participation of James Martin, co-CEO and CFO, in the following virtual investment community events:

  • Noble Capital Markets’ J.P. Morgan Healthcare Conference Takeaways hosted by ChannelChek featuring an interview with Noble Capital Market’s managing director and senior biotechnology analyst Robert LeBoyer discussing management’s perspective on the market environment for biotechnology companies and Cocrystal’s opportunity. The Takeaways event is available beginning today, January 26, at 11:00 a.m. Eastern time. Register here for the event.
  • Noble Capital Markets C-Suite Interview Series hosted by ChannelChek featuring an interview with Mr. LeBoyer with an update on Cocrystal’s influenza and COVID-19 antiviral programs and upcoming clinical and regulatory milestones. The interview will be available by February 1 on the ChannelChek platform by registering here.
  • Sequire’s Virtual Biotechnology Conference featuring a company presentation on Thursday, February 2 at 11:30 a.m. Eastern time Eastern time. Register here for the presentation.

About ChannelChek
Channelchek.com is a comprehensive investor-centric portal featuring more than 6,000 emerging growth companies that provides advanced market data, independent research, balanced news, video webcasts, exclusive c-suite interviews, and access to virtual road shows. The site is available to the public. Research on Channelchek is provided by Noble Capital Markets, Inc., an SEC / FINRA registered broker-dealer since 1984. More information is available at www.channelchek.com.

About Sequire
Sequire is a premier investor intelligence and communications platform where companies can track their investors’ behaviors and trends and use those insights to engage current and potential investors across marketing channels. More information is available at srax.com and mysequire.com.

About Cocrystal Pharma, Inc.
Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), hepatitis C viruses and noroviruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.

Investor Contact:
LHA Investor Relations
Jody Cain
310-691-7100
[email protected]

Media Contact:
JQA Partners
Jules Abraham
917-885-7378
[email protected]

# # #

Source: Cocrystal Pharma, Inc.

Released January 26, 2023

Baudax Bio (BXRX) – Baudax Bio Announces Positive Interim Analysis Of BX1000 Phase II Trial

Posted on by cpatches


Wednesday, January 25, 2023

Baudax Bio is a pharmaceutical company focused on innovative products for acute care settings. ANJESO is the first and only 24-hour, intravenous (IV) COX-2 preferential non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. In addition to ANJESO, Baudax Bio has a pipeline of other innovative pharmaceutical assets including two novel neuromuscular blocking agents (NMBs) and a proprietary chemical reversal agent specific to these NMBs. For more information, please visit www.baudaxbio.com.

Gregory Aurand, Senior Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Phase II Trial For Patients Undergoing Elective Surgery.  The BX1000 neuromuscular blocker is an intermediate-duration agent (~45 minutes) enrolling 80 patients in a Phase II trial. The randomized, double-blind, active-controlled IV-administered clinical trial compares three different doses of BX1000 (0.15 mg/kg IV, 0.25 mg/kg IV, 0.35 mg/kg IV) to a standard dose (0.6 mg/kg IV) of rocuronium, a standard of care blocking agent. (ClinicalTrials.gov Identifier: NCT05687253)

Primary Endpoints And Secondary Endpoints.  The primary endpoints assess the time frame needed to reach intubation conditions (time frame is within 2 minutes of administration) and also assess, using a standardized scale (Poor, Good, Excellent), the proportion of patients meeting Good or Excellent conditions. Secondary endpoints assess the safety and tolerability profile of BX1000.


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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. 

Axcella Therapeutics (AXLA) – Long COVID Phase 2b/3 Trial Changes The AXA1125 Outlook

Posted on by [email protected]


Wednesday, January 25, 2023

Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using compositions of endogenous metabolic modulators (EMMs). The company’s product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to restore cellular homeostasis in multiple key biological pathways and improve cellular energetic efficiency. Axcella’s pipeline includes lead therapeutic candidates in Phase 2 development for the treatment of Long COVID and non-alcoholic steatohepatitis (NASH), and the reduction in risk of overt hepatic encephalopathy (OHE) recurrence. The company’s unique model allows for the evaluation of its EMM compositions through non-IND clinical studies or IND clinical trials. For more information, please visit www.axcellatx.com.

Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Regulators Agree With Axcella’s Plan For A Phase 2b/3 Clinical Trial. Axcella Heath announced that US and UK regulators have agreed to a plan for a global Phase 2b/3 study of AXA1125 in Long COVID-19 patients. An IND has been submitted to the FDA for trials in the US, and agreement from the UK regulators has been received.  The Phase 2b/3 trial will include other countries to support worldwide approvals.  We see this as an important milestone for AXA1125 and the future of the company.

The Trial Announcement Reduces Uncertainty Around AXA1125.  As discussed in our Research Note on August 3, results from the Phase 2a trial in Long COVID showed a significant improvement in the physical fatigue, mental fatigue, and the 6-minute walk test. In December 2022, the company restructured to preserve cash, raising uncertainty about further development for the drug. The announcement of the Phase 2b/3 IND submission provides a development plan, allowing investors to assess timeframes and progress toward the market.


Get the Full Report

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. 

Cancer Cells Response to Surroundings

Posted on by [email protected]
Image Credit: NIH Image Gallery (Flickr)

How Cancer Cells Move and Metastasize is Influenced by the Fluids Surrounding Them – Understanding How Tumors Migrate Can Help Stop Their Spread

Cell migration, or how cells move in the body, is essential to both normal body function and disease progression. Cell movement is what allows body parts to grow in the right place during early development, wounds to heal and tumors to become metastatic.

Over the last century, how researchers understood cell migration was limited to the effects of biochemical signals, or chemotaxis, that direct a cell to move from one place to another. For example, a type of immune cell called a neutrophil migrates toward areas in the body that have a higher concentration of a protein called IL-8, which increases during infection.

In the past two or three decades, however, scientists have started to recognize the importance of the mechanical, or physical, factors that play a role in cell migration. For example, human mammary epithelial cells – the cells lining the milk ducts in the breast – migrate toward areas of increasing stiffness when placed on a surface with a stiffness gradient.

This article was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts. It represents the research-based findings and thoughts of, Yizeng Li, Assistant Professor of Biomedical Engineering, Binghamton University, State University of New York.

And now, instead of focusing on just the effect of the “solid” environment of cells, researchers are turning toward their “fluid” environment. As a theoretician trained in applied mathematics, I use mathematical models to understand the physics behind cell biology. My colleagues Sean X. Sun and Konstantinos Konstantopoulos and I were among the pioneering scientists who discovered how water and hydraulic pressure influence cell migration through theoretical models and lab experiments. In our recently published research, we found that human breast cancer cell migration is enhanced by the flow and viscosity of the fluids surrounding them, clarifying one of the factors influencing how tumors metastasize.

How Fluids Affect Cell Migration

Cells in the human body are constantly exposed to fluids of different physical properties. Water is one such fluid that can direct cell migration. For example, we found that how water flows across the membranes of breast cancer cells influences how they move and metastasize. This is because the amount of water traveling in and out of a cell causes it to shrink or swell, inducing movement by translocating different parts of the cell.

The viscosity, or thickness, of body fluids varies from organ to organ, and from health to disease, and this can also affect cell migration. For example, the fluid between cancer cells in tumors is more viscous than the fluid between normal cells in healthy tissues. When we compared how quickly breast cancer cells move in confined channels filled with fluid of normal viscosity versus fluid of high viscosity, we found that cells in high viscosity channels counterintuitively sped up by a significant 40%. This discovery was unexpected because the fundamental laws of physics tell us that inert particles should slow down in high viscosity fluids due to increased resistance.

We wanted to figure out the mechanism behind this surprising result. So we identified what molecules were involved in this process, discovering a cascade of events that allow high viscosity environments to enhance cell motility.

We found that high viscosity fluids first promote the growth of protein filaments called actin, which open channels in the cell’s membrane and increase water intake. The cell expands from the water, activating another channel that takes in calcium ions. These calcium ions activate another type of protein filament called myosin that induces the cell to move. This cascade of events induces cells to change their structure and generate more force to overcome the resistance imposed by high viscosity fluid, meaning the cells aren’t inert at all.

We also discovered that cells retained “memory” after exposure to a high viscosity medium. This meant that if we put cells in a high viscosity medium for several days and then returned them to a normal viscosity medium, they would still move at a faster speed. How cells retain this memory is still an open question.

We then wondered whether our findings on viscous memory would remain true in animals, not just in Petri dishes. So we exposed human breast cancer cells to a high viscosity medium for six days, then placed them in a normal viscosity medium. We then injected the cells into chicken embryos and mice.

Our results were consistent: Cells pre-exposed to a high viscosity medium had an increased ability to leak into surrounding tissues and metastasize compared to cells that were not pre-exposed. This result demonstrates that the viscosity of the fluids in a cell’s surrounding environment is a mechanobiological cue that promotes cancer cells to metastasize.

Implications for Cancer Treatment

Cancer patients usually don’t die from the original source of the tumor, but from its spread to other parts of the body.

When cancer cells travel through the body, they move into spaces that will have varying fluid viscosity. Understanding how fluid viscosity affects the movement of tumor cells could help researchers figure out ways to better treat and detect cancer before it metastasizes.

The next step is to build imaging and analysis techniques to precisely examine how cells from various types of lab animals respond to changes in fluid viscosity. Identifying the molecules that regulate how cells respond to changes in viscosity could help researchers identify potential drug targets to reduce the spread of cancer.

A Good Place to Start to Evaluate Specific Biotech Companies?  

Posted on by [email protected]
Image Credit: Andrea Piacquadio (Pexels)

Exploring and Discovering Biotech Stocks

When it comes to hand-selecting companies for investment, a critical ingredient for success is information. This ingredient becomes even more critical with biotech companies. Each year, many companies have been involved in medicine, medical equipment, genetics, and wellness that take off and provide investors with double or triple-digit gains. During the same years, there are stocks in the sector that, on the surface seem to have just as much going for them, yet a diligent peak below the surface demonstrates their success is less probable.

The ability to get below the surface is one reason the JPMorgan Health Symposium draws between 8,000 and 10,000 attendees each year. Attending is an expensive commitment, but firsthand information, insights from others that are in-the-know, and exposure to scientific paths, trends and research that barely existed a few years earlier, can pay off.

If you were not among the 8,000 counted as attending at the 2023 JP Morgan Health Symposium, you’ll want to know, Noble Capital Markets, teamed with Channelchek to provide a video recap with insights and key takeaways on some of the biotech trends that may be worth exploring. This takeaway, coupled with select company presentations and questions from two top equity analysts in the field is sure to build on your current health sector knowledge.  Go Here For More Information (and free access).  

Trends Worth Exploring

Molecular diagnostics, involves taking DNA or RNA which is our unique and easily obtainable genetic code, and analyzing the sequences for red flags. These markers can pinpoint the chance for emergence of specific diseases. This field has expanded rapidly in recent years, with some products now being used regularly. But the potential is for far more to be developed and approved for use. This provides for tremendous profit potential.

Alternative pain relief, non-opioid and non-NAISD pain medications for chronic sufferers, could benefit millions who suffer eah day. The potential runs the gamut from chronic headaches or back pain to situations where one is recovering from surgery, sports injuries, or accidents. Millions of prescriptions are written each year for pain medications. This has, in part fed into the opioid crisis in the U.S. It has prompted an almost emergency-level need for replacing older addictive medications with effective alternatives.  There are a number of companies making gains in this area of great need.

Gene therapy is a technique to treat or cure disease by modifying one’s genes. In many cases, the hope is that it leads to a permanent cure. New gene therapies are being developed for a wide swath of ailments including life-threatening disease. It is expected to be in many cases the next generation of cure. The methods for gene therapy include replacing a disease-causing gene with a healthy copy, or inactivating the disease-causing gene. In other cases a modified gene may be introduced to help treat the disease. The research and development include cancers, infectious disease, organ failures, and autoimmune problems. Many of these companies will be opening the door to welcome life improvements for the some people, and curing what are now incurable diseases for others.

Drilling Down at the Company Level

It may feel uncomfortable to suggest that investing in and backing the right companies that resolve health issues can be profitable to you. But, the truth is, without investments and interest in stock ownership, tomorrow’s miracle drugs would never come to exist.

Watch the Takeaway from the JP Morgan conference with an eye toward what the company presenters deem important, and then listen to the analysts that also drill deeper beyond concept and stage of development, they discuss finances, which for many less experienced biotech investors, isn’t focused on enough. The companies selected for the Noble Capital Markets Takeaway all fall within one the fields mentioned above.  Register Here.

Possible Side-Effects

The J.P. Morgan Healthcare Symposium was held in mid-January. It is one of life science’s largest and most frenzied sharing of information related to the industry. Not everyone gets to go. We’re enthusiastic to be bringing you a slice of the excitement in hopes that you deepen your understanding of not just these companies, but what to look for in others as well.

Paul Hoffman

Managing Editor, Channelchek

Source

https://www.jpmorgan.com/solutions/cib/insights/health-care-conference

https://www.sfchronicle.com/sf/article/JPMorgan-health-care-conference-brings-8-000-to-17706261.php#:~:text=The%20JPMorgan%20health%20care%20conference%2C%20the%20largest%20industry,the%20first%20time%20since%202020%2C%20drawing%208%2C000%20attendees.

Release – Baudax Bio Announces Positive Outcome of Interim Analysis of Phase II Randomized Trial for BX1000

Posted on by [email protected]

Research News and Market Data on BXRX

January 24, 2023 8:00am EST

MALVERN, Pa., Jan. 24, 2023 (GLOBE NEWSWIRE) — Baudax Bio, Inc. (NASDAQ:BXRX) a pharmaceutical company focused on innovative products for hospital and related settings, today announced the successful outcome of its first interim analysis in a Phase II trial of BX1000 for neuromuscular blockade (NMB) in patients undergoing elective surgery.

“We are encouraged by the results of the first interim analysis of the BX1000 Phase II surgery trial,” said Gerri Henwood, Baudax Bio’s President and Chief Executive Officer. “We believe the use of BX1000, combined with our reversal agent, BX3000, could make for precise control of timing under neuromuscular paralysis for surgical patients, which could result in time and cost savings for patients and hospitals alike. We look forward to completing enrollment in the study in Q1 and sharing topline results for the study in April 2023.”

This randomized, double-blind, active-controlled clinical trial comparing three different doses of BX1000 to a standard dose of rocuronium is planned to enroll a total of 80 adult patients undergoing elective surgery utilizing total intravenous anesthesia. The primary efficacy endpoint is the proportion of patients meeting criteria for Good or Excellent intubating conditions using a standardized scale. Additionally, the trial is evaluating the safety and tolerability profile of BX1000 and rocuronium in this patient population.

This pre-planned interim analysis evaluated the intubating conditions for each patient after administration of study drug in a blinded fashion. In the 20-patient cohort, 5 patients per group received one of the study medications. All 20 patients were observed to have met the criteria for Good or Excellent intubating conditions at 60 seconds. Nineteen of the subjects were successfully intubated following the assessment at 60 seconds, and the one remaining subject following the assessment at 90 seconds. Study treatments were generally well tolerated with no occurrence of severe or serious adverse events.

This blinded interim analysis did not result in the decision to drop any of the four study groups nor any decision to adjust planned study enrollment numbers.

About Baudax Bio’s Neuromuscular Blocking Agents (NMBs)

Baudax Bio holds exclusive global rights to two novel NMBs, BX1000, an intermediate duration, clinical stage blocking agent, and BX2000, an ultra-short duration, clinical stage blocking agent, as well as a proprietary chemical reversal agent, BX3000, undergoing nonclinical studies intended to support an IND filing in 2023. BX3000 is a specific reversal agent that rapidly reverses BX1000 and BX2000. All three agents are licensed from Cornell University. We believe these agents allow for a very rapid induction of neuromuscular blockade for surgical settings, followed by a rapid reversal of the neuromuscular blockade. These novel agents have the potential to meaningfully reduce time to onset of blocking and of reversal of blockade, reducing time in operating rooms or post-acute care settings, resulting in potential clinical and cost advantages, as well as time-related valuable cost savings for hospitals and ambulatory surgical centers.

About Baudax Bio

Baudax Bio is a pharmaceutical company focused on innovative products for hospital and related settings. The Company has a pipeline of innovative pharmaceutical assets including two clinical-stage, novel neuromuscular blocking (NMBs) agents, one in a Phase II study and an additional unique NMB in a dose escalation Phase I study, as well as a proprietary chemical reversal agent specific to these NMBs. Baudax Bio has received approval for and marketed ANJESO®, the first and only 24-hour, intravenous (IV) COX-2 preferential non-opioid, non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. For more information, please visit www.baudaxbio.com.

Forward-Looking Statements

This press release contains forward-looking statements that involve risks and uncertainties. Such forward-looking statements reflect Baudax Bio’s expectations about its future performance and opportunities that involve substantial risks and uncertainties. When used herein, the words “anticipate,” “believe,” “estimate,” “may,” “upcoming,” “plan,” “target,” “goal,” “intend” and “expect” and similar expressions, as they relate to Baudax Bio or its management, are intended to identify such forward-looking statements. These forward-looking statements, which include statements relating to the development of each of BX1000 and BX3000, are based on information available to Baudax Bio as of the date of this press release and are subject to a number of risks, uncertainties, and other factors that could cause actual results to differ materially from those expressed in, or implied by, these forward-looking statements. These risks and uncertainties include, among other things, risks related to market, economic and other conditions, the ongoing economic and social consequences of the COVID-19 pandemic, Baudax Bio’s ability to advance its current product candidate pipeline through pre-clinical studies and clinical trials, Baudax Bio’s ability to raise future financing for continued development of its product candidates such as BX1000, BX2000 and BX3000, Baudax Bio’s ability to pay its debt and satisfy conditions necessary to access future tranches of debt, Baudax Bio’s ability to comply with the financial and other covenants under its credit facility, Baudax Bio’s ability to manage costs and execute on its operational and budget plans, Baudax Bio’s ability to achieve its financial goals; Baudax Bio’s ability to comply with all listing requirements of the Nasdaq Capital Market; and Baudax Bio’s ability to obtain, maintain and successfully enforce adequate patent and other intellectual property protection. These forward-looking statements should be considered together with the risks and uncertainties that may affect Baudax Bio’s business and future results included in Baudax Bio’s filings with the Securities and Exchange Commission at www.sec.gov. These forward-looking statements are based on information currently available to Baudax Bio, and Baudax Bio assumes no obligation to update any forward-looking statements except as required by applicable law.

CONTACT:

Investor Relations Contact:

Argot Partners
Sam Martin / Kaela Ilami
(212) 600-1902
[email protected]

Media Contact:

Argot Partners
David Rosen
(212) 600-1902
[email protected]

Source: Baudax Bio, Inc.

Released January 24, 2023

Release – Tonix Pharmaceuticals Announces Publication of Paper in Antiviral Research Highlighting the Company’s Development of Monoclonal Antibody Therapeutics for Monkeypox and Smallpox

Posted on by [email protected]

Research News and Market Data on TNXP

January 24, 2023 7:00am EST

Data Represent Research and Development Work Being Conducted at Tonix’s Infectious Disease R&D Center (RDC) in Frederick, Md.

CHATHAM, N.J., Jan. 24, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced the publication of a paper entitled, “Development of a rapid image-based high-content imaging screening assay to evaluate therapeutic antibodies against the monkeypox virus,” in the journal Antiviral Research. The publication describes the development and optimization of two high-content image-based assays that were employed to screen for potential therapeutic antibodies against the monkeypox virus using surrogate poxviruses such as vaccinia virus. The article highlights Tonix’s TNX-3400 platform, which includes antibodies to potentially prevent or treat monkeypox and smallpox. The article can be accessed online at https://pubmed.ncbi.nlm.nih.gov/36592670/.

“These data represent the first wave of research and development conducted at our Infectious Disease R&D Center (RDC) in Frederick, Md,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “The RDC greatly enhances our ability to advance development of our pipeline of vaccines and therapeutics for infectious diseases. The TNX-3400 platform has promise for preventative and therapeutic monoclonal antibodies to treat monkeypox and smallpox.”

“The article describes how we optimized and standardized two high-content high-throughput image-based assays. The first assay was a neutralizing assay which detected viral proteins of vaccinia virus and used the assay to screen a large library of antibodies made against pox viruses. The second assay specifically detected phenotype changes in cells, called syncytia, after infection and protection by therapeutics.” said Sina Bavari, Ph.D., Executive Vice President of Tonix Pharmaceuticals and site director of the RDC. A critical component in assessing antibodies during pandemics requires the development of rapid but detailed methods to detect and quantitate the neutralization activity. “The neutralizing assay identified several antibodies with the capacity to protect against vaccinia virus infection. We believe this technology will allow us to identify combination therapies for monkeypox and smallpox viruses which was difficult to achieve before. Furthermore, our data suggest that applying this technology has the potential to increase the throughput of screening novel antivirals to shorten the discovery time for antivirals.”

About TNX-3400

TNX-3400 is the term for series of monoclonal antibodies which bind to key components of pox viruses such as monkeypox and smallpox and protect cells and tissues from infection with these pandemic causing viruses. These antibodies are being developed as broad-spectrum antipox virus and to potentially prevent or treat monkeypox and smallpox infection.

Tonix Pharmaceuticals Holding Corp.*

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022 and interim data expected in the second quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix initiated a Phase 2 study in Long COVID in the third quarter of 2022 and expects interim data in the third quarter of 2023. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the first quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is expected to enter the clinic with a Phase 2 study in the first quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily formulation of tianeptine being developed as a potential treatment for major depressive disorder (MDD) with a Phase 2 study expected to be initiated in the first quarter of 2023. Tonix’s rare disease portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the first half of 2023. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox, TNX-801, a next-generation vaccine to prevent COVID-19, TNX-1850, a platform to make fully human monoclonal antibodies to treat COVID-19, TNX-3600, and humanized anti-SARS-CoV-2 monoclonal antibodies, TNX-3800, recently licensed from Curia. TNX-801, Tonix’s vaccine in development to prevent smallpox and monkeypox, also serves as the live virus vaccine platform or recombinant pox vaccine (RPV) platform for other infectious diseases. A Phase 1 study of TNX-801 is expected to be initiated in Kenya in the second half of 2023.

*All of Tonix’s product candidates are investigational new drugs or biologics and have not been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
[email protected]
(862) 904-8182

Olipriya Das, Ph.D. (media)
Russo Partners
[email protected]
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
[email protected]
(443) 213-0505

Source: Tonix Pharmaceuticals Holding Corp.

Released January 24, 2023

What MRI and fMRI Scans of Programmers’ Brains Reveal

Posted on by [email protected]
Image Credit: Alex Shipps (Canva)

This is Your Brain – This is Your Brain on Code

Steve Nadis | MIT CSAIL

Functional magnetic resonance imaging (fMRI), which measures changes in blood flow throughout the brain, has been used over the past couple of decades for a variety of applications, including “functional anatomy” — a way of determining which brain areas are switched on when a person carries out a particular task. fMRI has been used to look at people’s brains while they’re doing all sorts of things — working out math problems, learning foreign languages, playing chess, improvising on the piano, doing crossword puzzles, and even watching TV shows like “Curb Your Enthusiasm.”

One pursuit that’s received little attention is computer programming — both the chore of writing code and the equally confounding task of trying to understand a piece of already-written code. “Given the importance that computer programs have assumed in our everyday lives,” says Shashank Srikant, a PhD student in MIT’s Computer Science and Artificial Intelligence Laboratory (CSAIL), “that’s surely worth looking into. So many people are dealing with code these days — reading, writing, designing, debugging — but no one really knows what’s going on in their heads when that happens.” Fortunately, he has made some “headway” in that direction in a paper — written with MIT colleagues Benjamin Lipkin (the paper’s other lead author, along with Srikant), Anna Ivanova, Evelina Fedorenko, and Una-May O’Reilly — that was presented earlier this month at the Neural Information Processing Systems Conference held in New Orleans.

The new paper built on a 2020 study, written by many of the same authors, which used fMRI to monitor the brains of programmers as they “comprehended” small pieces, or snippets, of code. (Comprehension, in this case, means looking at a snippet and correctly determining the result of the computation performed by the snippet.) The 2020 work showed that code comprehension did not consistently activate the language system, brain regions that handle language processing, explains Fedorenko, a brain and cognitive sciences (BCS) professor and a coauthor of the earlier study. “Instead, the multiple demand network — a brain system that is linked to general reasoning and supports domains like mathematical and logical thinking — was strongly active.” The current work, which also utilizes MRI scans of programmers, takes “a deeper dive,” she says, seeking to obtain more fine-grained information.

Whereas the previous study looked at 20 to 30 people to determine which brain systems, on average, are relied upon to comprehend code, the new research looks at the brain activity of individual programmers as they process specific elements of a computer program. Suppose, for instance, that there’s a one-line piece of code that involves word manipulation and a separate piece of code that entails a mathematical operation. “Can I go from the activity we see in the brains, the actual brain signals, to try to reverse-engineer and figure out what, specifically, the programmer was looking at?” Srikant asks. “This would reveal what information pertaining to programs is uniquely encoded in our brains.” To neuroscientists, he notes, a physical property is considered “encoded” if they can infer that property by looking at someone’s brain signals.

Take, for instance, a loop — an instruction within a program to repeat a specific operation until the desired result is achieved — or a branch, a different type of programming instruction than can cause the computer to switch from one operation to another. Based on the patterns of brain activity that were observed, the group could tell whether someone was evaluating a piece of code involving a loop or a branch. The researchers could also tell whether the code related to words or mathematical symbols, and whether someone was reading actual code or merely a written description of that code.

That addressed a first question that an investigator might ask as to whether something is, in fact, encoded. If the answer is yes, the next question might be: where is it encoded? In the above-cited cases — loops or branches, words or math, code or a description thereof — brain activation levels were found to be comparable in both the language system and the multiple demand network.

A noticeable difference was observed, however, when it came to code properties related to what’s called dynamic analysis.

Programs can have “static” properties — such as the number of numerals in a sequence — that do not change over time. “But programs can also have a dynamic aspect, such as the number of times a loop runs,” Srikant says. “I can’t always read a piece of code and know, in advance, what the run time of that program will be.” The MIT researchers found that for dynamic analysis, information is encoded much better in the multiple demand network than it is in the language processing center. That finding was one clue in their quest to see how code comprehension is distributed throughout the brain — which parts are involved and which ones assume a bigger role in certain aspects of that task.

The team carried out a second set of experiments, which incorporated machine learning models called neural networks that were specifically trained on computer programs. These models have been successful, in recent years, in helping programmers complete pieces of code. What the group wanted to find out was whether the brain signals seen in their study when participants were examining pieces of code resembled the patterns of activation observed when neural networks analyzed the same piece of code. And the answer they arrived at was a qualified yes.

“If you put a piece of code into the neural network, it produces a list of numbers that tells you, in some way, what the program is all about,” Srikant says. Brain scans of people studying computer programs similarly produce a list of numbers. When a program is dominated by branching, for example, “you see a distinct pattern of brain activity,” he adds, “and you see a similar pattern when the machine learning model tries to understand that same snippet.”

Mariya Toneva of the Max Planck Institute for Software Systems considers findings like this “particularly exciting. They raise the possibility of using computational models of code to better understand what happens in our brains as we read programs,” she says.

The MIT scientists are definitely intrigued by the connections they’ve uncovered, which shed light on how discrete pieces of computer programs are encoded in the brain. But they don’t yet know what these recently-gleaned insights can tell us about how people carry out more elaborate plans in the real world. Completing tasks of this sort — such as going to the movies, which requires checking showtimes, arranging for transportation, purchasing tickets, and so forth — could not be handled by a single unit of code and just a single algorithm. Successful execution of such a plan would instead require “composition” — stringing together various snippets and algorithms into a sensible sequence that leads to something new, just like assembling individual bars of music in order to make a song or even a symphony. Creating models of code composition, says O’Reilly, a principal research scientist at CSAIL, “is beyond our grasp at the moment.”

Lipkin, a BCS PhD student, considers this the next logical step — figuring out how to “combine simple operations to build complex programs and use those strategies to effectively address general reasoning tasks.” He further believes that some of the progress toward that goal achieved by the team so far owes to its interdisciplinary makeup. “We were able to draw from individual experiences with program analysis and neural signal processing, as well as combined work on machine learning and natural language processing,” Lipkin says. “These types of collaborations are becoming increasingly common as neuro- and computer scientists join forces on the quest towards understanding and building general intelligence.”

Reprinted with permission from MIT News” ( http://news.mit.edu/ )

Release – Axcella Announces Regulatory Path to Registration of AXA1125 for Long COVID Fatigue

Posted on by [email protected]

Research News and Market Data on AXLA

January 23, 2023 at 4:15 PM EST

MHRA guidance aligns on key measurements for a registration trial, including primary endpoint and trial design elements

IND for phase 2b/3 trial submitted to the FDA

Axcella to host a conference call Tuesday, January 24 at 8:00 a.m. ET; To register, click here

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jan. 23, 2023– Axcella Therapeutics (Nasdaq: AXLA), a clinical-stage biotechnology company pioneering novel approaches to treating complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, announced a regulatory path to registration of AXA1125 in the treatment of Long COVID Fatigue. The company reported that it had received regulatory guidance from The Medicines and Healthcare products Regulatory Agency (MHRA), the U.K.’s regulatory agency, supporting a single trial that could serve as the registration trial for patients with Long COVID fatigue, and aligning on key measurements, including primary endpoint and trial design. Axcella will be meeting with the MHRA in the near term to discuss the Innovative Licensing and Access Pathway (ILAP) application. The company further reported submission of an Investigational New Drug (IND) Application to the U.S. Food and Drug Administration (FDA) for a Phase 2b/3 trial.

These efforts follow submission of materials to both regulatory agencies including results from the Phase 2a randomized, double-blind, placebo-controlled investigation to evaluate the efficacy and safety of AXA1125 in patients with fatigue related to Long COVID. Notably, in this study subjects who received AXA1125 experienced clinically and statistically significant improvement in mental (p=0.0097) and physical (p=0.0097) fatigue scores compared to placebo subjects.

Long COVID is a persistent and growing long-term part of the pandemic, affecting an estimated one hundred million patients worldwide, with fatigue as the most commonly reported symptom. Recent estimates indicate that 15-20% of Americans with COVID have persisting health issues,i up to four million Americans are out of work due to Long COVID symptoms, and that Long COVID has contributed to approximately $1 trillion in lost earnings and $544 billion in increased medical spending.ii

“At Axcella, we are gratified and validated that there is a clear path to advance the Long COVID program into a potential registration trial with a leading regulator,” said Bill Hinshaw, CEO of Axcella. “This treatment could help millions of people around the world and there are no other agents that have demonstrated impact on the level of fatigue in a controlled trial. Many stakeholders have been eagerly anticipating a regulatory path in this new, important, and widespread disease and it is exciting to have this milestone achieved. With additional resources or collaboration, this program has the potential to advance and quickly reach patients in need.”

“We were pleased to have such constructive engagement with the MHRA, who have consistently recognized the urgent needs of patients and the healthcare system in the U.K. and have taken a rigorous and engaged, forward-looking approach to addressing Long COVID,” said Margaret Koziel, M.D., Chief Medical Officer of Axcella. “The tenor of the MHRA response and the results of our trial informed our recently completed FDA IND submission for our phase 2b/3 Long COVID trial. AXA1125 is the most advanced clinical-stage program for this devastating disease and we look forward to the opportunity to conduct a global trial that has the potential to rapidly enroll and submit for approval as the leading program in the field.”

“The statistically significant improvement in reported mental and physical fatigue among study participants receiving AXA1125 is a very encouraging finding for Long COVID patients, who often experience extreme and constant fatigue throughout their days,” added Betty Raman, M.D., Associate Professor of Cardiovascular Medicine at the Radcliffe Department of Medicine, University of Oxford, who led the phase 2a study.

“Given the devastating health and economic impact that Long COVID is having on millions of patients and their families, there is an urgent need for new treatments developed specifically for this population,” said Oved Amitay, Chief Executive Officer of Solve M.E., a non-profit organization that serves as a catalyst for critical research into diagnostics, treatments, and cures for myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), Long COVID and other post-infection diseases. “We are encouraged by data from Axcella’s phase 2 study in Long COVID chronic fatigue patients and the hope that AXA1125 provides to these patients, and by the opportunity to advance the program to a pivotal clinical trial.” Amitay, who also is the co-founder of the Long COVID Alliance, a network of patient-advocates, scientists, disease experts, and drug developers focused on educating policy makers and accelerating research into post-viral illnesses, added, “I urge the US government to use all the available mechanisms, including the RECOVER study infrastructure, to advance a clinical study of Axcella’s investigational drug in the U.S. as rapidly as possible. Patients are waiting.”

Investor Conference Call Information

Axcella will host a live conference call and webcast at 8:00 a.m. ET on Tuesday, January 24, 2023. To access the live conference call, please dial 844-808-7139 (domestic) or 412-902-0127 (international) and refer to “Axcella Health.” A webcast of the call will also be available under “Events and Presentations” in the Investors section of the Axcella Health website at https://ir.axcellatx.com/. The archived webcast will be available on Axcella’s website approximately two hours after the conference call and will be available for 90 days following the call.

About Axcella Therapeutics (Nasdaq: AXLA)

Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using compositions of endogenous metabolic modulators (EMMs). The company’s product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to reset multiple biological pathways, improve cellular energetics, and restore homeostasis. Axcella’s pipeline includes lead therapeutic candidates for the treatment of Long COVID, NASH, and the reduction in risk of OHE recurrence. The company’s unique model allows for the evaluation of its EMM compositions through non-IND clinical studies or IND clinical trials. For more information, please visit www.axcellatx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the potential utility of AXA1125 as a treatment of Long COVID and the Company’s anticipated regulatory pathway for AXA1125 and the timing and potential success thereof. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the potential impact of COVID-19 on the company’s ability to conduct and complete its ongoing or planned clinical studies and clinical trials in a timely manner or at all due to patient or principal investigator recruitment or availability challenges, clinical trial site shutdowns or other interruptions and potential limitations on the quality, completeness and interpretability of data the company is able to collect in its clinical trials of AXA1125, other potential impacts of COVID-19 on the company’s business and financial results, including with respect to its ability to raise additional capital and operational disruptions or delays, changes in law, regulations, or interpretations and enforcement of regulatory guidance, whether data readouts support the company’s clinical trial plans and timing, clinical trial design and target indications for AXA1125, the clinical development and safety profile of AXA1125 and its therapeutic potential, whether and when, if at all, the company’s product candidates will receive approval from the FDA or other comparable regulatory authorities, potential competition from other biopharma companies in the company’s target indications, and other risks identified in the company’s SEC filings, including Axcella’s Annual Report on Form 10-K, Quarterly Report on Form 10-Q and subsequent filings with the SEC. The company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Axcella disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent the company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. The company explicitly disclaims any obligation to update any forward-looking statements.

i Bull-Otterson L, Baca S, Saydah S, et al. Post–COVID Conditions Among Adult COVID-19 Survivors Aged 18–64 and ≥65 Years — United States, March 2020–November 2021. MMWR Morb Mortal Wkly Rep 2022;71:713–717. DOI: http://dx.doi.org/10.15585/mmwr.mm7121e1external icon
ii Bach K. New data shows long Covid is keeping as many as 4 million people out of work. Brookings Institute. August 24, 2022. https://www.brookings.edu/research/new-data-shows-long-covid-is-keeping-as-many-as-4-million-people-out-of-work/#footnote-3

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Source: Axcella Therapeutics