Category: Health

The Emerging Biotech Sector: A Bright Spot for Investors

Posted on by slightbourne@noblecapitalmarkets.com

The biotechnology sector has seen a flurry of activity recently, with major drugmakers looking to acquire smaller biotech firms to boost their pipelines. According to a recent Bloomberg News report, French pharmaceutical giant Sanofi is in talks to acquire Mirati Therapeutics, a clinical-stage biotech focused on developing novel cancer treatments.

While negotiations are still ongoing, this potential deal highlights the enormous value being seen in emerging biotech firms like Mirati that are pioneering cutting-edge medicines. Mirati’s lead drug candidate is a treatment for non-small cell lung cancer, one of the most common and deadly forms of cancer.

Other pharma giants have also inked deals to tap into biotech innovation. Earlier this month, cancer drug developer Immunome merged with Morphimmune. And Eli Lilly recently completed its acquisition of POINT Biopharma for $1.4 billion.

These mergers and acquisitions underscore the biotech sector’s immense growth prospects. With novel approaches to treating cancer, genetic diseases, and other unmet medical needs, biotechs have become hotbeds for innovation. And major drugmakers are increasingly looking to tap into these scientific advancements through strategic deals.

For investors, the busy M&A environment highlights the potential windfalls in identifying and investing in promising biotech firms early on. While risky, buying shares in companies with disruptive technologies before they are on Big Pharma’s radar can result in exponential returns.

With science rapidly advancing, the coming decades are likely to see huge leaps in biomedical innovations. The recent wave of pharma-biotech mergers shows large drugmakers recognize the future potential of biotech. Savvy investors who spot these opportunities stand to profit as more biotech firms are snatched up or grow into full-fledged pharmaceutical leaders themselves.

Take look at other emerging biotechnology companies by taking a look at Noble Capital Market’s Senior Research Analyst Robert LeBoyer’s coverage list.

The Promise of Biotech

Biotechnology represents an exciting new frontier in drug development. Unlike traditional pharmaceuticals derived from chemical compounds, biotech drugs utilize living organisms and biological molecules to treat disease.

Some key innovations driving growth in biotech include:

  • Gene therapy – Altering genes to treat genetic conditions. Gene editing tools like CRISPR have made gene therapy more precise and scalable.
  • Cell therapy – Using modified human cells as treatments, such as CAR T-cell therapy for blood cancers.
  • RNA interference – Silencing specific genes by blocking mRNA translation through RNAi mechanisms.
  • Monoclonal antibodies – Targeted antibodies cloned from immune cells are blockbuster therapies for cancer, autoimmunity, and more.
  • Vaccines – Novel vaccine platforms like mRNA vaccines are enabling rapid development of new vaccines.

These advanced technologies promise to revolutionize medicine and usher in an era of personalized, precision medicine. The possibilities span from regenerative medicine to reversing aging. For investors, biotech represents enormous growth potential.

Biotech Deal Frenzy

Given the vast promise of biotech, it is no surprise that pharmaceutical giants have been scooping up biotech firms at a fierce clip. Small promising biotechs are prime targets for acquisition.

Big Pharma companies like Sanofi, Merck, and Bristol-Myers need to refill drying pipelines. Revenue-generating biotech drugs can also help offset losses from older medications losing patent protection.

For the acquiring company, buying a biotech with an innovative drug candidate eliminates the risks and costs of developing a new medicine from scratch. And the more established resources and expertise of a pharma giant can help push novel therapies through late-stage trials and regulatory approvals.

Meanwhile, being acquired provides biotech startups with an influx of funds and resources to continue advancing their pipeline. It also offers a lucrative exit for early investors.

As the healthcare needs of the global population increase, larger pharmaceutical companies will likely continue acquiring emerging biotechs to remain competitive. This M&A frenzy shows no signs of slowing down.

Investment Opportunities

For investors, the busy biotech M&A environment provides exciting opportunities to profit. Here are some tips:

  • Seek out early-stage biotechs with promising technologies before they become acquisition targets. The ideal scenario is investing pre-IPO.
  • Focus on firms with advanced clinical pipelines addressing urgent unmet needs like cancer, rare diseases, neurodegeneration, etc.
  • Evaluate the strength of a biotech’s intellectual property and licensing agreements for its core technology.
  • Assess the management team’s experience in drug development and commercialization.
  • Consider biotechs focused on next-gen platforms like gene editing, cell therapy, RNAi, etc which are garnering lots of interest.
  • Be prepared to hold for longer time horizons and have a higher risk tolerance. Clinical trials have high failure rates.

For investors comfortable with risk, the payoffs for spotting the next potential biotech star could be immense as entire new markets for cutting-edge medicines continue to emerge.

Release – GeoVax Receives Notice of Allowance for HIV Vaccine Patent

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

ATLANTA, GA, October 5, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance for Patent Application No. 17/409,574 titled “Multivalent HIV Vaccine Boost Compositions and Methods of Use.” The allowed claims generally cover a priming vaccination with a DNA vector encoding multiple HIV antigens in virus-like particles (VLPs), followed by a boost vaccination with GeoVax’s vector platform for expressing HIV-1 antigens in VLPs utilizing an MVA viral vector.

David Dodd, GeoVax President and CEO, commented, “Our development priorities continue to be our next-generation COVID-19 vaccine, currently in Phase 2 clinical trials, and our cancer immunotherapy program, with Gedeptin® as our lead product in a Phase 1/2 clinical trial for advanced head and neck cancer. While we are focusing on these two products in the near-term, our long-term vision includes developing vaccines against other global public health threats such as HIV. Although not currently under active development, our HIV program forms an important part of the dataset underpinning all of our MVA-based development programs. The patent protection associated with the use of this vaccine is an important part of this product platform.”

Mr. Dodd continued, “Earlier this year, data were presented from a clinical study of a combinational HIV therapy that included GeoVax’s HIV vaccine candidate, MVA62B. The goal of the trial was to reduce or eliminate viral replication in the absence of antiviral medications in HIV-positive individuals (a “functional cure”). The data indicated very high levels of immunogenicity of the treatment, particularly the induction of T cell immunity, even though HIV infection compromises the immune system. We are pleased that GeoVax’s MVA-vectored HIV vaccine was selected to be a part of the experimental combination therapy and contributed to the positive findings. MVA62B was also previously tested in multiple clinical trials as a component of vaccine regimens designed to induce immune responses that prevent HIV infection. The team here at GeoVax is committed to help end the HIV epidemic worldwide and recognizes that the consistent results of MVA62B reflected in clinical trials to date may offer promise towards achieving this goal. We remain committed to supporting corporate and/or academic collaborations or partnering of this potentially critically important vaccine.”

About GeoVax

GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel therapies and vaccines for solid tumor cancers and many of the world’s most threatening infectious diseases. The company’s lead program in oncology is a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, presently in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax’s lead infectious disease candidate is GEO-CM04S1, a next-generation COVID-19 vaccine targeting high-risk immunocompromised patient populations. Currently in three Phase 2 clinical trials, GEO-CM04S1 is being evaluated as a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, and as a booster vaccine in patients with chronic lymphocytic leukemia (CLL). In addition, GEO-CM04S1 is in a Phase 2 clinical trial evaluating the vaccine as a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. GeoVax has a leadership team who have driven significant value creation across multiple life science companies over the past several decades. For more information, visit our website: www.geovax.com.

Forward-Looking Statements

This release contains forward-looking statements regarding GeoVax’s business plans. The words “believe,” “look forward to,” “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 and financial trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax is able to obtain acceptable results from ongoing or future clinical trials of its investigational products, GeoVax’s immuno-oncology products and preventative vaccines can provoke the desired responses, and those products or vaccines can be used effectively, GeoVax’s viral vector technology adequately amplifies immune responses to cancer antigens, GeoVax can develop and manufacture its immuno-oncology products and preventative vaccines with the desired characteristics in a timely manner, GeoVax’s immuno-oncology products and preventative vaccines will be safe for human use, GeoVax’s vaccines will effectively prevent targeted infections in humans, GeoVax’s immuno-oncology products and preventative vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete development, there is development of competitive products that may be more effective or easier to use than GeoVax’s products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control.

Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. 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. 

Company Contact: Investor Relations Contact: Media Contact:
info@geovax.com      paige.kelly@sternir.com      sr@roberts-communications.com
678-384-7220 212-698-8699 202-779-0929

Kyowa Kirin Bets on Gene Therapy With $477M Orchard Therapeutics Acquisition

Posted on by slightbourne@noblecapitalmarkets.com

Japan-based pharma Kyowa Kirin has agreed to acquire gene therapy specialist Orchard Therapeutics in a deal worth up to $477.6 million. The buyout aims to strengthen Kyowa Kirin’s emerging presence in the high-potential genetic medicine field.

Under the terms, Kyowa Kirin will pay $16 per Orchard ADS in cash upfront, representing a 144% premium to Orchard’s recent share price. Orchard shareholders will also receive a contingent value right worth an additional $1 per ADS if certain regulatory milestones are met.

The total potential payout values the deal at $477.6 million. Kyowa Kirin expects the acquisition to close in Q1 2024 pending approvals.

Orchard focuses on developing therapies using genetically modified hematopoietic stem cells (HSCs) taken from patients themselves. Its treatments aim to correct the underlying genetic cause of diseases in a single administration.

The company’s lead asset is Libmeldy, approved in Europe for treating a rare metabolic disorder called MLD. It also has two other programs for pediatric neurological conditions in late-stage testing.

Beyond the commercial and near-term pipeline assets, Kyowa Kirin gains Orchard’s HSC gene therapy platform. This technology can be leveraged to develop new treatments for diseases in Kyowa Kirin’s wheelhouse like oncology, autoimmune disorders, and others.

Kyowa Kirin has made gene and cell therapy a priority as part of its vision to deliver transformative new medicines. Orchard’s proven development capabilities and leadership position in HSC gene therapy make it an ideal fit for this strategy.

The high premium paid reflects Orchard’s status as a pioneer in the burgeoning field of genetic medicine. The deal provides Kyowa Kirin immediate scale and expertise in leveraging gene therapy.

Kyowa Kirin also gains commercial infrastructure to support the global launch of Libmeldy. The FDA is currently reviewing Libmeldy for approval in the U.S. with a decision date in March 2024.

Orchard’s two other clinical programs in development also address rare pediatric neurological disorders with immense unmet need. Additional earlier stage preclinical assets add further upside to the pipeline.

The deal continues biotech industry consolidation as large players acquire innovators to reinforce their drug development pipelines. The competition among pharmas for gene therapy assets has intensified as the field matures.

For Orchard investors, the buyout represents a significant premium after a long stretch of the stock languishing. But with cash running low, the company faced challenges transitioning its pipeline programs to commercial status alone.

The deal provides ample resources to continue advancing Orchard’s mission of tackling rare genetic diseases. Kyowa Kirin expects to hit $1 billion in sales from the MLD treatment alone if approved in the U.S.

Gene therapy has disrupted drug development over the past decade with its potential to deliver curative, lifelong treatment through a single administration. As technology improves, dealmaking and R&D in the space continues gaining steam.

Kyowa Kirin is the latest pharma to bet big on gene therapy’s possibilities. If it can successfully harness Orchard’s specialized platform and assets, the deal may pave the way to developing life-changing genetic medicines while delivering solid returns to shareholders.

Standard BioTools and SomaLogic to Merge, Creating $1B Life Sciences Tools Leader

Posted on by slightbourne@noblecapitalmarkets.com

Standard BioTools and SomaLogic have announced plans to unite through an all-stock merger aimed at creating a diversified life sciences tools platform with over $1 billion in equity value. The deal brings together technologies, expertise and customer bases across genomics, proteomics and other omics fields.

Standard BioTools provides genomic analysis tools catering to academic and clinical research settings. SomaLogic specializes in proteomics technology that profiles proteins for biopharmaceutical drug discovery. Their complementary offerings provide scale, synergies, and cross-selling opportunities.

Under the merger agreement, SomaLogic shareholders will receive 1.11 shares of Standard BioTools stock for each SomaLogic share they own. This values SomaLogic at over $370 million based on recent Standard BioTools share prices.

The combined company expects to generate $80 million in cost synergies by 2026 through optimization of its integrated operations. It will also hold over $500 million in cash to fund growth initiatives and new product development.

Standard BioTools CEO Michael Egholm touted SomaLogic’s proteomics capabilities as an ideal fit to accelerate his company’s strategy in the over $100 billion life science tools industry. The deal diversifies Standard BioTools’ portfolio beyond genomics while leveraging its global commercial infrastructure.

SomaLogic provides proteomic analysis that reveals functional expressions of genes, filling a key gap left by genomics. Its SOMAscan platform uses aptamer-based technology to measure thousands of proteins in biological samples.

Take a moment to take a look other biotech companies by looking at Noble Capital Markets’ Senior Research Analyst Robert LeBoyer’s coverage list.

The technology has become an industry leader in enabling biopharma researchers to identify and validate new drug targets. SomaLogic has relationships with nine of the ten largest pharma companies along with partnerships like its recently launched proteogenomics offering with Illumina.

Standard BioTools plans to tap into these biopharma relationships to cross-sell its genomic analysis tools. Meanwhile, SomaLogic can leverage Standard BioTools’ strong presence selling to academic labs. The combined customer base spans nearly all major end markets.

SomaLogic interim CEO Adam Taich called the merger an opportunity to better serve translational and clinical research customers while creating shareholder value. The healthy $500 million cash position provides ample capital to fund the commercial ramp.

Standard BioTools increased its 2023 revenue outlook to $100-105 million following the merger news. SomaLogic maintained its full-year guidance of $80-84 million. Together, the combined entity expects to generate over $180 million this year.

The boards of both companies have unanimously approved the transaction. Major shareholders holding around 16% of Standard BioTools stock and 1% of SomaLogic have also committed support through voting agreements.

The deal is expected to close in the first quarter of 2024 after securing shareholder and antitrust regulatory approvals. The combined company will operate under the Standard BioTools name and stock ticker, with dual headquarters in South San Francisco and Boulder, Colorado.

Standard BioTools has undergone major changes after a period of underperformance, divesting its sequencing business earlier this year. The merger with SomaLogic continues its strategic shift toward life science research tools.

Together, the companies aim to accelerate development of new diagnostics and precision medicines through their multi-omics technology. Providing genomics, proteomics and other readouts on disease samples provides deeper insights to researchers.

With scale, synergies, ample resources, and multi-pronged revenue opportunities, the combined Standard BioTools and SomaLogic expects to occupy a strengthened position in the competitive life science tools space. Their integration marks the continued consolidation in the industry amid rising demand for omics-based research capabilities.

Release – ZyVersa Therapeutics Announces New Peer-Reviewed Publication Reinforcing the Rationale for Inhibiting ASC Specks with IC 100 to Attenuate Spread of Inflammation into Surrounding Tissues

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ZVSA

Oct 4, 2023

PDF Version

  • The paper, published in Pharmaceuticals, demonstrates that pyrin inflammasome-mediated inflammation induced by traumatic brain injury (TBI) contributes to cardiovascular co-morbidities through systemic release of proinflammatory mediators that activate AIM2 inflammasomes in the heart leading to damaging inflammation.
  • Marks the third publication in 2023 supporting the potential need to attenuate the spread of inflammation to surrounding tissues to minimize the potential for co-morbidities in certain diseases.
  • ZyVersa is developing Inflammasome ASC Inhibitor IC 100 to inhibit multiple types of inflammasomes and their associated ASC specks that trigger damaging inflammation and its spread to surrounding tissues.

WESTON, Fla., Oct. 04, 2023 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of inflammatory and renal diseases, announces that world renowned inflammasome researchers from the University of Miami Miller School of Medicine and inventors of Inflammasome ASC Inhibitor IC 100 have published a scientific paper in the peer-reviewed journal, Pharmaceuticals, highlighting how inflammation in the brain mediated by traumatic injury can trigger inflammation in the heart.

In the paper titled, “Neural–Cardiac Inflammasome Axis after Traumatic Brain Injury,” the authors conducted a study in animal models of TBI and evaluated serum from patients following TBI and from healthy controls. Data indicate that pyrin inflammasome-mediated inflammation induced by TBI contributes to cardiovascular co-morbidities through systemic release of proinflammatory mediators that activate AIM2 inflammasomes in the heart leading to damaging inflammation. The role of inflammasome activation in the development of TBI-induced cardiovascular co-morbidities was substantiated by increased ASC speck formation in the brains and hearts of TBI mouse models.

“A heightened systemic inflammatory response is often induced after TBI, but the underlying pathomechanisms that contribute to co-morbidities remain poorly understood. Here, we investigated whether extracellular vesicles (EVs) containing inflammasome proteins are released after severe controlled cortical impact in C57BL/6 mice and cause activation of inflammasomes in the heart that result in tissue damage,” said Dr. Robert W. Keane, Professor, Physiology and Biophysics, Neurological Surgery and Microbiology, and Immunology, University of Miami Miller School of Medicine. “TBI resulted in the release of EVs into the serum, which contain a cargo of inflammasome-, complement- and cardiovascular-related signaling proteins, and adoptive transfer of EVs from TBI patients resulted in inflammasome activation in cardiovascular cells.”

“Importantly, here, we also found an increase in ASC specks in the brain and the heart of TBI mice, suggesting that these prion-like ASC structures play a significant role in the inflammatory pathogenesis observed after TBI in the brain and systemically. Moreover, this finding emphasizes the potential benefit of therapies targeting ASC specks after CNS injury,” stated Dr. Juan Pablo de Rivero Vaccari, Associate Professor, Department of Neurological Surgery, University of Miami Miller School of Medicine. To review the paper, Click Here.

“This research published in the Journal, Pharmaceuticals, along with recent research published in the Journal of Clinical Investigation and Translational Research reinforces the importance of attenuating extracellular propagation of IL-1β to minimize induction and perpetuation of inflammation in surrounding tissues and organs,” commented Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO, and President. “ZyVersa’s Inflammasome ASC inhibitor IC 100 is designed to inhibit formation of multiple types of inflammasomes to attenuate initiation of the inflammatory cascade, and to inhibit their associated ASC specks to reduce perpetuation of damaging inflammation.”

To review a white paper summarizing the mechanism of action and preclinical data for IC 100, Click Here.

About Inflammasome ASC Inhibitor IC 100

IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 was designed to attenuate both initiation and perpetuation of the inflammatory response. It does so by binding to a specific region of the ASC component of multiple types of inflammasomes, including NLRP1, NLRP2, NLRP3, NLRC4, AIM2, Pyrin. Intracellularly, IC 100 binds to ASC monomers, inhibiting inflammasome formation, thereby blocking activation of IL-1β early in the inflammatory cascade. IC 100 also binds to ASC in ASC Specks, both intracellularly and extracellularly, further blocking activation of IL-1β and the perpetuation of the inflammatory response that is pathogenic in inflammatory diseases. Because active cytokines amplify adaptive immunity through various mechanisms, IC 100, by attenuating cytokine activation, also attenuates the adaptive immune response.

About ZyVersa Therapeutics, Inc.

ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced, proprietary technologies to develop first-in-class drugs for patients with renal and inflammatory diseases who have significant unmet medical needs. The Company is currently advancing a therapeutic development pipeline with multiple programs built around its two proprietary technologies – Cholesterol Efflux Mediator™ VAR 200 for treatment of kidney diseases, and Inflammasome ASC Inhibitor IC 100, targeting damaging inflammation associated with numerous CNS and other inflammatory diseases. For more information, please visit www.zyversa.com.

Cautionary Statement Regarding Forward-Looking Statements

Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing.

New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.

This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.

Corporate and IR Contact:
Karen Cashmere
Chief Commercial Officer
kcashmere@zyversa.com
786-251-9641        

Media Contacts
Tiberend Strategic Advisors, Inc.
Casey McDonald
cmcdonald@tiberend.com
646-577-8520

Dave Schemelia
dschemelia@tiberend.com
609-468-9325

Release – Tonix Pharmaceuticals Announces Issuance of EU Patent Covering Intranasal Potentiated Oxytocin (TNX-1900) for the Treatment of Pain

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

October 04, 2023 7:00am EDT

New patent expected to expire in 2036

Proof of concept data from Phase 2 study for prevention of migraine headache expected in early December 2023

CHATHAM, N.J., Oct. 04, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, announced today that the European Patent Office (EPO) issued European Patent No. 3242676 to the Company on October 4, 2023. The patent, entitled “Magnesium-Containing Oxytocin Formulations and Methods of Use”, claims methods and compositions for treating pain, including migraine headaches, using intranasal magnesium-containing oxytocin formulations. This patent, excluding possible patent term extensions, is expected to provide Tonix with market exclusivity in the member states of the European Patent Office until 2036. The U.S. Patent and Trademark Office (USPTO) has previously issued U.S. Patents No. 9,629,894 and 11,389,473 which are expected to provide market exclusivity in the U.S. until 2036.

In August 2023, Tonix announced it completed enrollment in its proof-of-concept Phase 2 PREVENTION study of TNX-1900 (intranasal potentiated oxytocin) for the prevention of migraine headache in chronic migraineurs with a total of 88 patients enrolled. The Company expects topline results from this trial in early December 2023.

“We believe this European patent combined with the U.S. issuance provides a strong patent position for TNX-1900 as we move forward in clinical development for the treatment of chronic migraine,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “With a differentiated formulation including magnesium, which Tonix has shown to potentiate the action of oxytocin at oxytocin receptors in animal models, we believe TNX-1900 has the potential to help those chronic migraine sufferers by engaging and stimulating oxytocin receptors in the trigeminal ganglia. By expanding our patent coverage in Europe, we are ensuring the IP protection of this unique approach.”

In addition to Tonix’s patents that protect certain uses including treating migraine, Tonix has licensed patents from INSERM, the French national health and medical research institute, that claim methods of treating feeding disorders with onset during neonatal development, specifically Prader Willi syndrome and non-organic failure to thrive (NOFTT), using oxytocin (EP 2575853, US8853158 and US9125862) and licensed patents from University of Geneva that claim methods of treating insulin resistance using oxytocin (US9101569 and EP2571511).

About Migraine

Migraine is a neurological condition that manifests in throbbing headache, often on one side of the head, that lasts at least four hours. It can also be accompanied by nausea, vomiting, visual disturbances, and sensitivity to bright light, strong smells, and loud noises.1 Epidemiological studies indicate that globally, approximately 1.2 billion individuals suffer from migraines annually.2 In the U.S., approximately 39 million Americans suffer from migraines and among these individuals, approximately four million experience chronic migraines (15 or more headache days per month).2

About TNX-1900

TNX-1900 (intranasal potentiated oxytocin) is a proprietary formulation of oxytocin in development as a candidate for prophylaxis of chronic migraine and for the treatment of craniofacial pain, insulin resistance and related conditions. In 2020, TNX-1900 was acquired from Trigemina, Inc. who had licensed the technology underlying the composition and method from Stanford University. TNX-1900 is a drug-device combination product, based on an intranasal actuator device that delivers oxytocin into the nose. Oxytocin is a naturally occurring human hormone that acts as a neurotransmitter in the brain. Oxytocin has no recognized addiction potential. It has been observed that low oxytocin levels in the body can lead to an increase in migraine headache frequency, and that increased oxytocin levels can relieve migraine headaches. Certain other chronic pain conditions are also associated with decreased oxytocin levels. Migraine attacks are caused, in part, by the activity of pain-sensing trigeminal nerve cells which, when activated, release of the calcitonin gene-related peptide (CGRP) which binds to receptors on other nerve cells and starts a cascade of events that is believed to result in headache. Oxytocin, when delivered via the nasal route, concentrates in the trigeminal system3 resulting in binding of oxytocin to receptors on neurons in the trigeminal system, inhibiting transmission of pain signals and releasing of the CGRP.4 Blocking CGRP release is a distinct mechanism compared with CGRP antagonist and anti-CGRP antibody drugs, which block the binding of CGRP to its receptor. With TNX-1900, the addition of magnesium to the oxytocin formula enhances oxytocin receptor binding5, its effects on trigeminal neurons, and its craniofacial analgesic effects in animal models.7 Intranasal oxytocin has been shown to be well tolerated in several clinical trials in both adults and children.6 Targeted nasal delivery results in low systemic exposure and lower risk of non-nervous system, off-target effects, which could potentially occur with systemic CGRP antagonists such as anti-CGRP antibodies.8 For example, CGRP has roles in dilating blood vessels in response to ischemia, including in the heart. We believe nasally targeted delivery of oxytocin could translate into selective blockade of CGRP release in the trigeminal ganglion and not throughout the body, which could be a potential safety advantage over systemic CGRP inhibition. In addition, daily dosing is more quickly reversible, in contrast to monthly or quarterly dosing, as is the case with anti-CGRP antibodies, giving physicians and their patients greater control.

  1. https://www.mayoclinic.org/diseases-conditions/migraine-headache/symptoms-causes/syc-20360201
  2. Burch et al., Migraine: Epidemiology, Burden, and Comorbidity, Neurol Clin 37 (2019) 631–649.
  3. Yeomans DC, et al. Transl Psychiatry. 2021. 11(1):388.
  4. Tzabazis A, et al. Cephalalgia. 2016. 36(10):943-50.
  5. Antoni FA and Chadio SE. Biochem J. 1989. 257(2):611-4.
  6. Yeomans, DC et al. 2017. US patent US2017368095
  7. Cai Q, et al., Psychiatry Clin Neurosci. 2018. Mar;72(3):140-151.
  8. MaassenVanDenBrink A, et al. Trends Pharmacol Sci. 2016. 37(9):779-788

Tonix Pharmaceuticals Holding Corp.*

Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg under a transition services agreement with Upsher-Smith Laboratories, LLC from whom the products were acquired on June 30, 2023. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia, having completed enrollment of a potentially confirmatory Phase 3 study in the third quarter of 2023, with topline data expected in late December 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 proof-of-concept study has been completed, and topline results were reported in the third quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily oral formulation being developed as a treatment for major depressive disorder (MDD), that completed enrollment in a Phase 2 proof-of-concept study in the third quarter of 2023, with topline results expected in early November of 2023. TNX-4300 (estianeptine) is a single isomer version of TNX-601, small molecule oral therapeutic in preclinical development to treat MDD, Alzheimer’s disease and Parkinson’s disease. Relative to tianeptine, estianeptine lacks activity on the µ-opioid receptor while maintaining activity in the rat Novel Object Recognition test in vivo and the ability to activate PPAR-β/δ and neuroplasticity in tissue culture. TNX-1900 (intranasal potentiated oxytocin), is in development for preventing headaches in chronic migraine, and has completed enrollment in a Phase 2 proof-of-concept study with topline data expected in early December 2023. TNX-1900 is also being studied in binge eating disorder, pediatric obesity and social anxiety disorder by academic collaborators under investigator-initiated INDs. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the fourth quarter of 2023. Tonix’s rare disease development portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology development portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 was initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals.

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

Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. Intravail is a registered trademark of Aegis Therapeutics, LLC, a wholly owned subsidiary of Neurelis, Inc. All other marks are property of their respective owners.

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; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Investor Contact

Jessica Morris
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 904-8182

Peter Vozzo
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Media Contact

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
(919) 360-3039

Source: Tonix Pharmaceuticals Holding Corp.

Released October 4, 2023

Release – Ocugen To Present at The 2023 Cell & Gene Meeting On The Mesa

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

October 4, 2023

PDF Version

MALVERN, Pa., Oct. 04, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen will present at the 2023 Cell & Gene Meeting on the Mesa being held October 10-12, 2023 in Carlsbad, CA at the Park Hyatt Aviara Resort.

Dr. Musunuri will provide an overview of Ocugen’s clinical development programs, including the business strategy across its unique gene and cell therapy platforms, anticipated milestones, and more detail about the Company’s recent positive Phase 1/2 OCU400 data results.

Details of the presentation are as follows:

Event:2023 Cell & Gene Meeting on the Mesa
Date & Time:October 11, 2023 at 5:15 PM, PDT
Location:Park Hyatt Aviara Resort, Carlsbad, CA, Aseptic Technologies Ballroom

“The Cell & Gene Meeting on the Mesa convenes leading industry professionals confronting major scientific, logistic, policy, and economic questions in cell and gene therapy,” said Dr. Musunuri. “I am pleased to join this impressive group and share the latest gene therapy news from Ocugen—compelling positive results from our Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA)—as well as our progress toward initiating the Phase 3 trial for the Company’s novel cell therapy product candidate for cartilage repair in the second half of 2024.”

Virtual attendance is available, which includes a livestream of Ocugen’s presentation and on-demand viewing of all conference sessions. Please visit https://meetingonthemesa.com for full information including registration.

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 patients’ 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 Twitter and LinkedIn.

Forward-Looking Statements 
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, 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. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release. 

Contact: 
Tiffany Hamilton 
Head of Communications 
Tiffany.Hamilton@ocugen.com 

PDS Biotechnology Corp. (PDSB) – Strong Phase 2 Survival Data, But Stock Sells Off Anyway

Posted on by cpereira@noblefcm.com


Wednesday, October 04, 2023

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of molecularly targeted cancer and infectious disease immunotherapies based on the Company’s proprietary Versamune® and Infectimune™ T-cell activating technology platforms. Our Versamune®-based products have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. The Company’s pipeline products address various cancers including HPV16-associated cancers (anal, cervical, head and neck, penile, vaginal, vulvar) and breast, colon, lung, prostate and ovarian cancers.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

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

PDS Biotech Updated Phase 2 Survival Data. PDS Biotech updated its Phase 2 VERSATILE-002 data presented at the ACSO conference in May 2023. This trial is testing the combination of PDS0101 and pembrolizumab (Keytruda, from Merck) in recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC). The data presented at ASCO in May included patients followed up to 12 months, while the update followed patients up to 24 months. 

Survival Of 74% Is A Significant Improvement. Patients that were followed for two years had an overall survival rate of 74% compared with published studies showing less than 30% survival. Overall survival at 12 months at 80% compares with published results of 30% to 50%. Tumor shrinkage was seen in 60% (31/52) and confirmed by second MRI scan (overall response rate, ORR) in 27% (14/52 patients). Treatment related adverse events (TRAEs) were limited to Grade 3 in 13% (8/55) patients, and none had Grade 4 or Grade 5. We see these data as significantly improved over pembrolizumab alone or with chemotherapy.


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Lilly Makes $1.4 Billion Bet on Radioactive Cancer Drugs with POINT Biopharma Acquisition

Posted on by slightbourne@noblecapitalmarkets.com

Pharmaceutical giant Eli Lilly is expanding its cancer treatment portfolio into a promising new area by acquiring POINT Biopharma, a company developing radioactive drugs that precisely target tumors, for $1.4 billion.

POINT specializes in radioligand therapies, an emerging approach to cancer treatment that uses radioactive particles linked to molecules that bind to receptors on cancer cells. This enables the radiation to selectively kill tumors while limiting damage to healthy tissue.

Lilly is paying $12.50 per share in cash for POINT, an 87% premium over the stock’s latest closing price. The deal will give Lilly control of POINT’s pipeline of radioligand therapy candidates, which includes two late-stage experimental drugs.

One drug, PNT20021, targets prostate cancer tumors by binding to a protein called PSMA. Study data expected later this year will show whether it extends the lives of men with metastatic castration-resistant prostate cancer.

The other late-stage drug, PNT20031, homes in on neuroendocrine tumor cells via their somatostatin receptors. It may provide a new option for patients with advanced gastroenteropancreatic neuroendocrine tumors.

Take a look at PDS Biotechnology, a clinical stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies.

Beyond these lead programs, POINT has several earlier stage radioligands in development for cancers of the breast, lung, and brain. Lilly gains full access to progress these toward human testing.

The deal also gives Lilly two specialized facilities Point has built to produce and research radioligands. Manufacturing the drugs involves linking medical radioisotopes like actinium-225 to the targeting molecules, which requires nuclear expertise.

Jacob Van Naarden, head of Lilly’s oncology division, touted the promise of radioligands to safely destroy cancer while avoiding the side effects of traditional chemo. “We are excited by the potential of this emerging modality,” he said.

Lilly has been growing its cancer treatment business in recent years through deals for other firms’ drug candidates and technologies. The POINT acquisition similarly expands Lilly’s footprint into an area well-suited for precision medicine.

The U.S. Food and Drug Administration has already approved over a half dozen radioligand therapies from Lilly competitors like Novartis. Their success is driving a surge of investment and deal-making in the radiopharmaceutical field.

But analyst Geoffrey Porges of SVB Securities thinks Lilly overpaid for POINT. “We believe the valuation fails to reflect the very high risks inherent in drug development,” he wrote in a note to investors.

Porges added that Lilly may need to invest over $2 billion more to fully develop POINT’s pipeline over the next 5-7 years, with no certainty the drugs will pan out.

Lilly expects the acquisition to close by the end of 2023 after gaining required antitrust and regulatory approvals. The majority of POINT shareholders also must tender their shares as part of the agreement.

The deal marks Lilly’s second major oncology purchase in 2022. It paid $1.1 billion earlier in the year access to cancer drug candidates from China’s Zymeworks. With POINT, Lilly is now positioned as a leader across multiple next-wave approaches in the high-stakes race to develop better cancer treatments.

Bruker Pays $108 Million to Acquire Single-Cell Biology Firm PhenomeX

Posted on by slightbourne@noblecapitalmarkets.com

Life science tools provider Bruker Corporation is expanding into the hot field of single-cell analysis through the acquisition of PhenomeX in an all-cash deal valued at around $108 million. Bruker aims to complement its existing microscopy and proteomics businesses by adding PhenomeX’s platforms for studying gene and protein expression in individual cells.

PhenomeX itself was formed just this year through the merger of two prior companies, Berkeley Lights and IsoPlexis. The combined entity offers instruments, software, and reagents to examine the genotype and phenotype of single cells in high throughput.

This enables researchers to link specific cellular traits to underlying genetics and molecular characteristics. It provides a valuable window into cell function relevant to disease as well as development of new therapies.

Bruker cited antibody discovery, cell line engineering, and cell and gene therapy as prime application areas that are fueling rapid growth in the single-cell analysis market. The company pointed to the over 400 systems PhenomeX already has installed at customers as evidence of strong demand.

PhenomeX’s key technology is its Beacon Optofluidic System, which can assess the behavior of tens of thousands of individual cells in parallel while keeping them alive for further genomic analysis. The IsoLight and IsoSpark systems complement this by measuring protein expression in each cell.

Bruker views the acquisition as a launching point into the single-cell biology space, which it called a natural adjacency to its existing fluorescence microscopy offerings. The company has begun expanding beyond its core analytical instrumentation business into faster-growth life science markets.

But some analysts questioned the hefty valuation Bruker paid, amounting to a 90% premium over PhenomeX’s stock price prior to the merger announcement of its predecessor companies. While the technology is cutting-edge, PhenomeX remains an early-stage business posting minimal revenue.

Bruker may need to invest significantly more over years to fully commercialize PhenomeX’s products and turn it into a growth contributor. But the opportunity to lead a new market niche ultimately justified the price.

Take a moment to take a look at other biotech companies by looking at Noble Capital Markets Senior Research Analyst Robert LeBoyer’s coverage list.

Single-cell analysis has become one of the hottest areas within the life sciences amid the precision medicine revolution. Researchers are realizing individual cells often differ genetically and behave differently even within the same tissue sample. Analyzing them as a group obscures key insights.

By examining each cell’s genotype and phenotypic traits individually, links can be drawn to implicate specific genes in cellular functions relevant to diseases like cancer. This enables more targeted drug discovery.

The market is still in its early days but is on a steep growth trajectory as biopharma companies incorporate single-cell capabilities into their R&D. Bruker already enjoys strong relationships through its existing product lines and now gains a foothold to capitalize.

Meanwhile, gaining Bruker’s global commercial infrastructure and resources provides a major boost to PhenomeX, which was formed through the merger of two startups. Having an established industry leader backing its technology and lowering R&D risks strengthens its prospects.

For Bruker investors, the purchase expands the company’s total addressable market by billions in the coming years. While not yet profitable, PhenomeX offers a bridge into a complementary fast-growing segment aligned with Bruker’s strengths.

The deal is the largest in Bruker’s recent spate of acquisitions to reshape itself beyond analytical tools into a more diversified life sciences player. It mirrors consolidation in the broader market as major instrumentation firms seek more exposure to biopharma and cell-based research markets.

If Bruker can successfully integrate and scale up PhenomeX’s platform, the $108 million price tag may prove to be a bargain entry point into the booming single-cell analysis space.

Release – PDS Biotech Announces Interim 24-Month Survival Rate of 74% in Immune Checkpoint Inhibitor Naïve Head and Neck Cancer Patients Treated with PDS0101 in Combination with KEYTRUDA® (pembrolizumab)

Posted on by aweinsoff@channelchek.com

Research News and Market Data on PDSB

  • Interim VERSATILE-002 data for the first-line treatment of recurrent or metastatic head and neck cancer patients who were immune checkpoint inhibitor (ICI) naïve; published 24-month survival rate of less than 30% for approved ICI1.
  • Received feedback from US Food and Drug Administration (FDA) on the Company’s amended Investigational New Drug (IND) application allowing initiation of VERSATILE-003 Phase 3 trial in the fourth quarter of 2023.
  • 12-month overall survival (OS) rate of 56% in ICI refractory patients; published median 12-month OS rate of 17% with no salvage chemotherapy following tumor progression on ICI (ICI Refractory)2*.
  • Well tolerated with no patients having Grade 4 or 5 combination treatment-related adverse events.
  • Interim VERSATILE-002 data and VERSATILE-003 Phase 3 trial design to be discussed during Key Opinion Leader (KOL) Roundtable today at 8:00 a.m. EDT.

PRINCETON, N.J., Oct. 03, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (PDS Biotech or the Company), a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer immunotherapies and infectious disease vaccines based on the Company’s proprietary T cell activating platforms, today announced updated interim data based on an August 2nd cut off from the VERSATILE-002 Phase 2 clinical trial evaluating PDS0101 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab), in patients with unresectable, recurrent, or metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC). VERSATILE-002 is investigating two patient populations whose cancer has returned or spread – ICI naïve and ICI refractory. The ICI naïve group had not responded to standard-of-care treatments but had not yet been treated with an ICI. The ICI refractory group included patients who had not responded to multiple prior treatments, including ICI therapy. Data presented at ASCO was based on a January 13th cut off.

“The updated interim data from our VERSATILE-002 clinical trial further validates the potential of PDS0101 when combined with KEYTRUDA® to address the urgent need for more effective therapies that are well tolerated and allow advanced recurrent and metastatic HPV16-positive head and neck cancer patients to live significantly longer lives than current approaches,” said Frank Bedu-Addo, PhD, President and Chief Executive Officer of PDS Biotech. “Following feedback from the FDA, we look forward to evaluating this promising combination treatment in the VERSATILE-003 Phase 3 clinical trial, which we expect to initiate in the fourth quarter of 2023. VERSATILE-003 will investigate the efficacy and safety of PDS0101 combined with KEYTRUDA® compared to KEYTRUDA® monotherapy in ICI-naïve patients with recurrent or metastatic HPV16-positive HNSCC. The primary endpoint for the study will be overall survival.”

VERSATILE-002: ICI Naïve

Highlights of the interim data from the ICI naïve cohort include:

  • 24-month overall survival (OS) rate is 74%; published 24-month survival rate of less than 30% for approved ICI1.
  • 12-month OS rate is 80%; published results of 30-50% with approved ICIs1.
  • Tumor shrinkage seen in 60% (31/52) of patients.
  • Confirmed overall response rate (ORR) is 27% (14/52) to date.
  • Median progression-free survival (PFS) is 8.1 months to date; published results of 2-3 months PFS with approved ICIs1.
  • 13% (8/62) of patients experienced Grade 3 treatment-related adverse events (TRAE) and 0% (0/62) experienced Grade 4 or 5 TRAE; published results report 13-17% Grade 3-5 TRAE with approved ICI monotherapy1.
  • 60% (33/55) of patients have CPS score of 1-19 (who generally have a weaker response to KEYTRUDA®), and 40% (22/55) have CPS score >20 (who generally have a higher response to KEYTRUDA®).

VERSATILE-002: ICI Refractory

The goal of this ICI refractory cohort was to confirm and to better understand the role of PDS0101 in prolonging the survival of advanced HPV16-positive head and neck cancer patients who received PDS0101 in combination with KEYTRUDA®. This analysis is also intended to provide insight to the contribution of PDS0101 to overall survival in the National Cancer Institute-led study evaluating the combination of PDS0101, PDS0301 (antibody conjugated IL12), and an ICI.

Highlights of the interim data from the ICI refractory cohort include:

  • The 12-month OS rate is 56%. The published median 12-month OS rate is 17% with no salvage chemotherapy following tumor progression on ICI (ICI Refractory)2*.
  • 0% (0/21) confirmed ORR suggests that PDS0101’s impact on survival does not appear to be dependent on tumor shrinkage.
  • 4% (1/25) of patients experienced Grade 3 TRAE and 0% (0/21) patients experienced Grade 4 and 5 TRAE.

“We are pleased with the OS results and knowledge gained from the ICI refractory cohort of VERSATILE-002. In agreement with our Data Monitoring Committee (DMC), we will not progress to stage 2 of this cohort in VERSATILE-002. As previously announced, we have no plans to further develop this combination for ICI refractory patients,” said Lauren V. Wood, MD, Chief Medical Officer of PDS Biotech. “PDS0101 appears to immunologically alter the patient’s tumor microenvironment to promote survival. This important data will help inform our development plans for PDS0101.”

Key Opinion Leader Roundtable
Today, Tuesday, October 3, 2023, from 8:00 – 9:00 AM EDT, the Company will host a Key Opinion Leader (KOL) Roundtable on Addressing Current and Future Treatments for Recurrent/Metastatic Human Papillomavirus (HPV)-Positive Head and Neck Squamous Cell Carcinoma (HNSCC) and the Potential Application of PDS0101, including a discussion of the interim data from the VERSATILE-002 trial.

A live webcast of the event will be available online in the Investor Relations section of the Company’s website at https://www.pdsbiotech.com/index.php/investors. A replay will be available for 90 days following the webcast.

1*Ferris R.L., Nivolumab for Recurrent Squamous-Cell Carcinoma of the Head and Neck; N Engl J Med 2016; 375:1856-1867; Burtness B et al., Pembrolizumab alone or with chemotherapy versus cetuximab with chemotherapy for recurrent or metastatic squamous cell carcinoma of the head and neck (KEYNOTE- 048): a randomized, open-label phase 3 study; Lancet 2019; 394(10212):1915-1928. *No control or comparative studies have been conducted between immune checkpoint inhibitors and PDS0101.
https://www.opdivo.com/head-and-neck-cancer
https://www.keytruda.com/head-and-neck-cancer/keytruda-clinical-trials/
2Bila M, Van Dessel J, Smeets M, Vander Poorten V, Nuyts S, Meulemans J, Clement PM. A Retrospective Analysis of a Cohort of Patients Treated With Immune Checkpoint Blockade in Recurrent/Metastatic Head and Neck Cancer. Front Oncol. 2022 Jan 27;12:761428. doi: 10.3389/fonc.2022.761428. PMID: 35155226; PMCID: PMC8828639. *No controlled or comparative studies have been conducted between PDS0101 and no salvage chemotherapy.

About PDS0101
PDS0101, PDS Biotech’s lead candidate, is a novel investigational human papillomavirus (HPV)-targeted immunotherapy that stimulates a potent targeted T cell attack against HPV-positive cancers. PDS0101 is given by subcutaneous injection alone or in combination with other immunotherapies and cancer treatments. In a Phase 1 study of PDS0101 in monotherapy, the treatment demonstrated the ability to generate multifunctional HPV16-targeted CD8 and CD4 T cells with minimal toxicity. Interim data suggests PDS0101 generates clinically active immune responses, and the combination of PDS0101 with other treatments can demonstrate significant disease control by reducing or shrinking tumors, delaying disease progression and/or prolonging survival. The combination of PDS0101 with other treatments does not appear to compound the toxicity of other agents.

About VERSATILE-002
VERSATILE-002 is a single-arm Phase 2 trial evaluating the safety and efficacy of PDS0101, an HPV16-targeted investigational T cell-activating immunotherapy that leverages PDS Biotech’s proprietary Versamune® technology, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab).The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve and ICI-refractory patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC) and was granted Fast Track designation by the Food and Drug Administration in June 2022.

About VERSATILE-003
The VERSATILE-003 Phase 3 clinical trial is a randomized, active comparator-controlled study designed to investigate the safety and efficacy of PDS0101 combined with KEYTRUDA® compared to KEYTRUDA® monotherapy in ICI-naïve patients with recurrent or metastatic HPV16-positive HNSCC. The primary efficacy endpoint for VERSATILE-003 is overall survival. The Phase 3 study is expected to involve approximately 90-100 clinical sites globally.

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®, Versamune® plus PDS0301, and Infectimune® T cell-activating platforms. We believe our targeted immunotherapies 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 and shrink 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 and will be advancing into a Phase 3 clinical trial in combination with KEYTRUDA® for the treatment of recurrent/metastatic HPV16-positive head and neck cancer in 2023. 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 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; 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. KEYTRUDA® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, N.J., USA.

Investor Contact:
Rich Cockrell
CG Capital
Phone: +1 (404) 736-3838
Email: pdsb@cg.capital

Media Contact:
Gina Cestari
6 Degrees
Phone: +1 (917) 797-7904
Email: gcestari@6degreespr.com

Investors Take Interest in MAIA Biotechnology as FDA Clears Path for Cancer Drug Trial

Posted on by slightbourne@noblecapitalmarkets.com

Shares of MAIA Biotechnology were trading higher Tuesday after the company announced that the FDA has cleared its application to test its experimental cancer therapy THIO in patients in the United States.

MAIA Biotech is developing THIO as a novel immunotherapy approach for advanced non-small cell lung cancer (NSCLC). With FDA clearance of its Investigational New Drug (IND) application, the company can now include U.S. cancer patients in its ongoing mid-stage trial evaluating THIO’s safety and efficacy.

This regulatory win is driving investor enthusiasm and higher trading volume for MAIA stock today.

About MAIA Biotech and THIO

MAIA Biotech is a clinical-stage immunotherapy company aiming to improve cancer treatment by targeting telomeres. Telomeres play an important role in cancer cell survival and resistance to standard therapies.

The company’s lead therapy THIO represents a first-in-class telomere-targeting agent for NSCLC. Early preclinical research indicates THIO can induce cancer cell death and stimulate anti-tumor immune responses.

MAIA is positioning THIO as a second or third line of treatment for NSCLC patients who have stopped responding to initial immunotherapy. The company sees THIO’s novel approach as a way to improve outcomes in this hard-to-treat population.

Phase 2 Trial Details

THIO is currently being tested in a Phase 2 clinical trial involving sites across Europe, Asia Pacific, and now with FDA clearance, the United States.

The trial is evaluating THIO in combination with the PD-1 checkpoint inhibitor Libtayo in advanced NSCLC patients. Researchers want to see if giving THIO first to “prime” the immune system, followed by Libtayo, can enhance and prolong the immune response against cancer cells.

The primary goal is assessing THIO’s safety and antitumor activity based on overall response rates. Secondary goals include evaluating biomarkers and overall survival. The trial expects to enroll approximately 90 patients total.

Next Steps for MAIA

While still early stage, the FDA clearance represents an important milestone for MAIA as it works to expand THIO’s potential reach. Being able to include U.S. sites should support faster enrollment and generate data from a larger, more diverse patient population.

Positive Phase 2 results would support advancing to a pivotal Phase 3 study, which the company hopes could lead to regulatory approval. MAIA sees a multi-billion dollar market opportunity in later-line NSCLC treatment.

The company is also exploring THIO’s potential in other cancer types like melanoma, prostate cancer, and multiple myeloma.

Why Investors are Excited

FDA clearance of THIO’s IND removes a key regulatory hurdle for MAIA. Being able to test the therapy in the major U.S. market is critical for the smaller biotech company.

Today’s stock move reflects investors’ increased confidence in THIO’s outlook and MAIA’s ability to execute on development plans. If the Phase 2 trial goes well, it would further validate THIO’s novel approach and cancer-fighting potential.

While still highly speculative given the early stage, MAIA represents an intriguing immunotherapy play for investors interested in emerging approaches for hard-to-treat cancers. The company’s focus on telomere biology and unique combination strategy with Libtayo differentiate it from other biotechs.

MAIA stock could continue to be volatile in the months ahead as Phase 2 data approaches. But the FDA clearance has put a spotlight on this previously lesser known name. For investors open to some risk, MAIA may be a cancer immunotherapy stock to have on the radar.

Take a look at more research on MAIA Biotechnology by Noble Capital Markets Senior Research Analyst Robert LeBoyer.

Biotech Poised for a Powerful Comeback

Posted on by slightbourne@noblecapitalmarkets.com

After a fallow period, signs point to the biotech sector regaining its prior momentum. Several factors indicate a pending return to rapid growth and prolific innovation for biotech companies. This prospective resurgence could replicate the boom years of the 1980s and 1990s.

The first driver is scientific advancements that open new possibilities. CRISPR gene editing has revolutionized biotech, allowing cheaper and easier manipulation of genetic code. This enables creation of novel treatments and cures previously out of reach. Other breakthroughs like mRNA vaccines have proven the ability to rapidly develop radically new therapeutic approaches.

Vast amounts of genomic data generated in recent decades have also unlocked new understandings of biologically rooted diseases. By identifying key genetic drivers, drug targets can be validated to produce higher success rates in clinical trials. Failed drug candidates have historically been a major drag on biotech.

Substantial investment capital is also lining up behind biotech again after the sector fell out of favor. While biotech IPOs dropped sharply in 2022, venture funding actually rose to its second highest level ever at $32 billion. Investor appetite remains strong, especially for companies with promising new platforms.

Large cash piles among pharmaceutical giants could further bolster biotech. Big pharma companies have routinely turned to buying biotech firms to fill product pipelines. With major players like Pfizer and Merck holding over $25 billion in deployable cash reserves, expect more dealmaking ahead.

Take a look at several emerging growth biotech companies by looking at Noble Capital Market’s Senior Research Analyst Robert LeBoyer’s coverage list.

Regulatory incentives additionally sweeten the proposition of getting back into biotech. The FDA is actively supporting development in areas like gene therapy, rare diseases, and certain cancers through its pilot programs and priority reviews. This guidance can derisk investments.

The maturing ecosystem around biotech also fuels its potential rebound. Experienced veteran executives can now be tapped to steer startups. Clustering in hubs like Boston and San Francisco persists to provide concentration of talent, capital, and resources.

While risks like high failure rates remain, the ingredients are aligning for biotech’s next generation. Comparisons to the internet boom of the late 1990s resonate, with biotech representing a similar pivotal platform shift. The world’s demographics also underpin demand for new therapies – aging populations in developed nations will drive need.

This fertile environment parallels periods that produced prior biotech booms. In the late 1970s and 80s, the industry arose virtually from scratch around pioneering companies like Genentech and Amgen. The advent of genetic engineering allowed creation of the first biologic drugs.

Another surge came through the 90s as enabling technologies like high throughput screening scaled up the drug discovery process. The mapping of the human genome unleashed another wave of possibilities.

Today’s scientific advances pose an even greater leap, allowing drug development and treatment paradigms hardly imaginable just decades ago. The bounds of human understanding keep expanding.

The stars are aligning for biotech 2.0, an evolution building on past successes but catalyzed by new potentials. It promises to usher in an era of curative therapies, genomic precision, and accelerated innovation. The post-pandemic landscape offers the ideal springboard for this biotech revival.

With therapeutic bottlenecks getting cleared, investor interest rekindling, and confidence restored, expect biotech to reclaim its prior growth trajectory. The lessons of past booms were well learned, leaving companies better positioned to capitalize. Weapons to fight disease grow more powerful by the month.

The public expects and demands new treatments for pressing needs, from cancer to neurological conditions. Demographics favor biotech’s prospects as populations age. An energized ecosystem stands ready to nourish exciting science from lab to market.

The pieces are in place for biotech liftoff. As science unlocks new horizons, investor insight aligns with public health demands, fueling momentum. Biotech’s foundational role in driving modern medical progress is poised to only accelerate. The cycle of innovation spins up again.