Tag: Biotechnology

Ocugen (OCGN) – OCU500 Nasal COVID-19 Vaccine Chosen By NIH To Start Clinical Trials

Posted on by cpereira@noblefcm.com

Wednesday, October 11, 2023

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

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

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

Good News For A Program Where No News Was Expected. Ocugen announced that its OCU500 nasal vaccine for COVID-19 has been chosen to start Phase 1 clinical trials run by the National Institute for Allergy and Infectious Disease, a division of the National Institutes of Health. We see this as a positive development for the vaccine program since Ocugen had no plans to develop OCU500 without obtaining non-dilutive funding. We have no revenues for the vaccine programs in our models, and we see the selection by NIAID for Phase 1 clinical trials as upside for the company.

We See The Selection By NIAID As A Technology Validation. OCU500 was developed to stimulate immunity in the nasal passages and lungs where the virus enters the body and the infection begins. This is intended to prevent both the initial infection and transmission of the virus to others. We see the OCU500 selection as a validation to the Ocugen inhaled vaccine technology, which currently includes OCU500 for COVID-19, OCU510 for influenza, and OCU520 for both COVID-19 and influenza. The carrier technology can be used to develop other vaccines as well.


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. 

Release – Ocugen Mucosal Vaccine Candidate Ocu500 Selected By NIH/NIAID Project Nextgen For Inclusion In Clinical Trials

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

October 10, 2023

PDF Version

  • NIAID is conducting early phase clinical trials on select next generation vaccine candidates with the intent to identify the most effective platforms and delivery routes
  • OCU500 will be tested as both inhaled and intranasal vaccine candidates
  • Clinical trials scheduled to start in early 2024

MALVERN, Pa., Oct. 10, 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 the National Institute of Allergy and Infectious Diseases (NIAID), a part of the National Institutes of Health, will conduct a trial comparing the administration of Ocugen’s mucosal vaccine candidate, OCU500, via two different mucosal routes, inhalation into the lungs and as a nasal spray.

Ocugen is developing a novel anti-viral mucosal vaccine platform initially targeting COVID-19 and influenza (flu). The intent is to provide protection against severe disease, increase durability and prevent transmission of viral threats. OCU500 is based on a novel chimpanzee adenovirus-vectored (ChAd) technology. Earlier clinical studies to prevent COVID-19 employing a similar vector administered via inhalation demonstrated increased mucosal antibodies, systemic antibodies, and durable immune response up to one year using one fifth (1/5) of the dose compared to the same vaccine given via intramuscular administration. Additionally, Ocugen believes that this vaccine can be rapidly scaled-up as new variants emerge.

“We believe our novel mucosal vaccine platform technology has the potential to prevent infection and spread of COVID-19, and improve durability for an annualized vaccine similar to flu,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “This is the first vaccine candidate using our inhaled platform technology, which we hope to expand in order to address multiple respiratory threats, including flu. We have benefited from a strong collaborative relationship with NIAID and BARDA since the start of Project NextGen and we look forward to participating in this initiative.”

NIAID will be conducting clinical trials to evaluate several early stage vaccine candidates. The study involving Ocugen’s vaccine will be administered via both intranasal and inhaled routes and is designed to help answer an important question – does an inhaled COVID-19 vaccine provide greater immune response than the same vaccine administered through a nasal spray. Upon completion of the trial NIAID and Ocugen will assess the results and determine next steps for OCU500.

Project NextGen is a $5 billion multi-government agency initiative to develop the next generation of vaccines and therapeutics to combat the spread of COVID-19. NIAID, with funding from Project NextGen, will cover the full cost of the clinical trials, including operations and related analysis. Ocugen will be responsible for providing clinical trial materials and upon completion will have full right of reference to the findings, which Ocugen believes will provide clinical evidence to support the further development of the Company’s lead mucosal vaccine candidate.

The announced collaboration comes at a time when COVID-19 infection rates are rising with the emergence of new variants. Durability of existing vaccines continues to be of concern with antibody protection waning several months following vaccination while vaccine compliance rates have declined since the initial wave. According to a recent Harris poll, 66% of Americans would prefer to have more vaccine options. The poll also found that 52% of Americans would be more open to getting an intranasal or inhaled, versus injectable COVID-19 vaccine.

Ocugen looks forward to this important next step in the development of its novel mucosal vaccine platform and further supporting the Company’s commitment to advancing public health.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, 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 

Haemonetics Expands Hospital Portfolio Through $253 Million Acquisition of OpSens

Posted on by slightbourne@noblecapitalmarkets.com

Medical technology firm Haemonetics Corporation recently announced a definitive agreement to acquire OpSens, Inc. in an all-cash deal valued at approximately $253 million. OpSens is a medical device company specializing in innovative fiber optic sensor technology for interventional cardiology applications. This strategic acquisition allows Haemonetics to expand its hospital business into the high-growth interventional cardiology market estimated at $1 billion.

Haemonetics, based in Boston, offers a suite of products for blood and plasma collection, the surgical suite, and hospital transfusion services. With the addition of OpSens’ sensor-guided guidewire and pressure guidewire products for transcatheter aortic valve replacement (TAVR) and percutaneous coronary intervention (PCI), Haemonetics bolsters its portfolio with clinically validated technology to improve patient outcomes.

OpSens’ core offerings include the SavvyWire, the first sensor-guided guidewire for TAVR procedures which enables shorter hospital stays, and the OptoWire, a pressure guidewire used to aid coronary artery disease diagnosis by measuring key parameters like fractional flow reserve (FFR). OpSens leverages proprietary optical technology across its sensor solutions for medical devices and critical industrial applications.

According to Stewart Strong, President of Global Hospital at Haemonetics, this acquisition expands Haemonetics’ leadership in interventional cardiology while providing a foundation for additional growth. By combining OpSens’ innovative technology with Haemonetics’ commercial infrastructure and hospital relationships, there is tremendous potential to increase adoption and improve patient care globally.

Strategically, Haemonetics gains several advantages from the purchase:

  • Access to a $1 billion total addressable market in interventional cardiology, a specialty area witnessing increasing procedure volume. OpSens’ competitive, clinically validated offerings are well-positioned for long-term growth.
  • The ability to accelerate OpSens product adoption leveraging Haemonetics’ existing commercial footprint and depth of penetration in U.S. hospitals for its VASCADE vascular closure portfolio.
  • Expanded product breadth and enhanced diversification into adjacent applications like industrial sensors. OpSens technology can be leveraged across Haemonetics’ hospital business and new markets.
  • Opportunities for continued R&D, clinical study efforts, and other business development activities to augment internal product development. Haemonetics aims to expand its hospital division via organic and inorganic investments.

Financially, Haemonetics expects the deal will be immediately accretive to revenue growth. On an adjusted basis, earnings per share is also expected to be accretive right away. Due to one-time integration costs, GAAP earnings per share may be slightly dilutive in the first full fiscal year before turning accretive.

Haemonetics will finance the transaction through existing cash balances and its revolving credit facility. This will result in a manageable rise in the company’s net debt to EBITDA ratio to around 2.1x. The purchase is anticipated to close by January 2024, subject to customary approvals.

In summary, the acquisition of OpSens for $253 million in cash strengthens Haemonetics’ position in the attractive interventional cardiology space while providing new technologies, commercial synergies, and earnings accretion over the long-term. It signals a bold move to supplement organic growth with value-enhancing strategic M&A, as Haemonetics looks to deliver innovation and drive better patient outcomes through continued expansion.

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

Release – GeoVax Receives Notice of Allowance for Marburg Vaccine Patent

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

ATLANTA, GA, October 9, 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. 17/584,231 titled “Replication-Deficient Modified Vaccinia Ankara (MVA) Expressing Marburg Virus Glycoprotein (GP) and Matrix Protein (VP40).” The allowed claims generally cover GeoVax’s vector platform for expressing Marburg virus antigens in virus-like particles (VLPs) utilizing an MVA viral vector.

A recent presentation of data from nonhuman primate studies demonstrated that immunization with GeoVax’s vaccine candidate, GEO-MM01, conferred 80% survival in cynomolgus macaques following a lethal dose of Marburg virus. Vaccination protected from viremia, weight loss and death following challenge with a lethal Marburg virus dose. Evaluation of immune responses following vaccination demonstrated the presence of both neutralizing antibodies and functional T cells, indicating a breadth of responses that combine for optimal protection. GeoVax is currently evaluating study designs to assess the potential for administering different dose levels of the vaccine and different routes of vaccine delivery to optimize utility and efficacy.

David Dodd, GeoVax President and CEO, commented, “While our focus and development priorities continue to be our next-generation COVID-19 vaccine and cancer immunotherapy programs, developing vaccines against lethal hemorrhagic fever viruses represents our commitment to addressing highly fatal endemic threats throughout the world. Our team is committed to supporting the successful advancement of such a vaccine, as we recognize the critically important medical and biodefense need, reflected by the inclusion of Marburg virus in the FDA Priority Review Voucher program. This patent allowance adds to our growing portfolio of wholly owned, co-owned, and in-licensed intellectual property, now standing at over 115 granted or pending patent applications spread over 24 patent families.”

About Marburg Virus

Marburg virus (MARV) is a hemorrhagic fever virus of the Filoviridae family, which also includes Ebola virus, and causes severe human disease with up to a 90% fatality rate. The Marburg virus is transmitted to people from fruit bats, and human-to-human transmission occurs through direct contact with bodily fluids, or contaminated surfaces and materials. MARV is rated by the World Health Organization (WHO) as a Risk Group 4 Pathogen. In the United States, the NIH/National Institute of Allergy and Infectious Diseases ranks it as a Category A Priority Pathogen and the Centers for Disease Control and Prevention lists it as a Category A Bioterrorism Agent. MARV typically appears in sporadic outbreaks throughout Africa and the virus continues to pose potential public health and biodefense threats. There are currently no licensed vaccines or therapeutics against the diseases caused by MARV.

About the GV-MVA-VLPTM Platform

GeoVax’s GV-MVA-VLPTM vaccine platform utilizes modified vaccinia Ankara (MVA), a large virus capable of carrying several vaccine antigens, that expresses proteins that assemble into virus-like particles (VLP) immunogens in the person receiving the vaccine. The production of VLPs in the person being vaccinated can mimic the virus production that occurs in a natural infection, stimulating both the humoral and cellular arms of the immune system to recognize, prevent, and control the target infection. The MVA-VLP derived vaccines can elicit durable immune responses in the host similar to a live-attenuated virus, while providing the safety characteristics of a replication-defective vector.

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

Bristol Myers Squibb to Acquire Mirati Therapeutics for $4.8 Billion

Posted on by slightbourne@noblecapitalmarkets.com

Pharma giant Bristol Myers Squibb (BMY) announced today that it will acquire clinical-stage biotech Mirati Therapeutics (MRTX) for $58 per share in an all-cash deal totaling $4.8 billion. Mirati stockholders will also receive a contingent value right worth up to $12 per share, bringing the total potential deal value to $5.8 billion.

The acquisition will expand Bristol Myers Squibb’s oncology portfolio and pipeline. Mirati’s lead asset is KRAZATI (adagrasib), the first and only FDA-approved drug targeting the KRAS G12C mutation. KRAS mutations occur in about 13% of non-small cell lung cancers (NSCLC) and are linked to poor prognosis.

KRAZATI was granted accelerated approval in October 2022 as a second-line treatment for KRAS G12C-mutated NSCLC. It is also being tested in combination with a PD-1 inhibitor as a potential first-line NSCLC therapy. Beyond lung cancer, KRAZATI has shown promise in colorectal and pancreatic cancers.

“With multiple targeted oncology assets including KRAZATI, Mirati is another important step forward in our efforts to grow our diversified oncology portfolio,” said Bristol Myers CEO Giovanni Caforio. The company aims to leverage its global commercial infrastructure to maximize KRAZATI’s reach.

Mirati’s earlier-stage pipeline includes MRTX1719, an innovative PRMT5 inhibitor, as well as several KRAS-targeted agents. MRTX1719 could be the first targeted therapy for MTAP-deleted tumors, which represent about 10% of cancers.

“Bristol Myers Squibb’s global scale, resources and commitment to innovation will enable Mirati’s therapeutics to benefit more patients, faster,” said Mirati CEO Charles Baum.

Strategic Fit

Lung cancer is the most common cancer and leading cause of cancer death globally. The addition of KRAZATI establishes Bristol Myers as a leader in developing targeted lung cancer therapies. Mirati also expands Bristol Myers’ presence in colorectal and pancreatic cancers.

The acquisition builds on Bristol Myers’ recent deals for Turning Point Therapeutics and Eisai’s oncology business. As patents expire for the pharma giant’s top-selling cancer immunotherapy Opdivo, it aims to refill its oncology pipeline.

“With a strong strategic fit, great science and clear value creation opportunities for our shareholders, the Mirati transaction is aligned with our business development goals,” said Caforio.

Broader Biopharma Implications

The blockbuster Mirati acquisition also has significant implications for the broader biotech and biopharma sector. As large pharmas look to replenish pipelines, M&A activity has intensified. The deal shows that promising clinical-stage biotechs with innovative oncology pipelines continue to be attractive buyout targets.

Analysts note the 52% buyout premium Bristol Myers paid as a sign of their urgency to tap into Mirati’s next-gen oncology science. For startup biotechs pursuing novel approaches in high-value areas like oncology, it underscores the possibility of commanding large premium buyouts from “big pharma” acquirers.

However, smaller players also face the risk of being squeezed out as consolidation accelerates. The Mirati deal exemplifies the scaling up required to compete in cutting-edge areas like targeted cancer therapies. Smaller biotechs could find it increasingly difficult to independently develop and commercialize new drugs in the future.

That said, smaller biotechs may also benefit from big pharma’s growing appetite for M&A. The premiums being offered for innovative science and pipelines create lucrative exit opportunities for startups. And the influx of capital from buyouts can fund the next generation of biotech innovation.

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

Deal Terms

Under the definitive agreement, Bristol Myers will pay $58 per share for Mirati’s outstanding common stock. This represents a 52% premium over Mirati’s 30-day volume-weighted average price. Including Mirati’s $1.1 billion cash balance, the total equity value comes to $4.8 billion.

Each Mirati shareholder will also receive a CVR worth up to $12 per share. This contingent value right payment is triggered if Mirati’s MRTX1719 is approved within 7 years as a NSCLC therapy after two or fewer systemic treatments. The CVR adds up to $1 billion in potential additional value.

The transaction is expected to close in the first half of 2024, pending approval from regulators and Mirati shareholders. Bristol Myers anticipates the deal will be dilutive to its non-GAAP earnings through 2025 as it integrates Mirati. It plans to finance the acquisition through cash and debt offerings.

Caforio stated: “With a strong strategic fit, great science and clear value creation opportunities for our shareholders, the Mirati transaction is aligned with our business development goals.” The deal furthers Bristol Myers Squibb’s transformation into a leading oncology-focused biopharma.

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.

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 – 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 

Cancer Drug Developer Immunome To Merge With Morphimmune in Quest For Targeted Therapies

Posted on by slightbourne@noblecapitalmarkets.com

Immunome, a clinical-stage biotech developing novel antibody drugs for cancer, plans to merge with private peer Morphimmune in an all-stock deal. The combined company will unite complementary technology platforms with the goal of creating best-in-class targeted oncology therapies.

Morphimmune brings its proprietary Targeted Effector platform designed to selectively deliver anti-cancer payloads directly to tumor cells. Immunome contributes its human memory B cell interrogation platform that can identify novel antibodies against disease-associated antigens.

The merged entity, which will operate under the Immunome name, intends to submit 3 IND applications within 18 months after the transaction closes. The deal is expected to be completed by the end of 2023.

Leading the charge as new Immunome CEO will be current Morphimmune chief Clay Siegall, an industry veteran who previously founded and led Seattle Genetics for over two decades. Siegall built Seagen into a multi-billion cancer drug company on the back of its antibody-drug conjugate (ADC) technology.

His experience commercializing ADCs, which similarly target treatments directly to tumors, is highly relevant. Siegall called the merger “the first step in establishing a preeminent oncology company.”

The transaction will also bring in $125 million via a concurrent private placement from healthcare institutional investors. Participants include Enavate Sciences, EcoR1 Capital, Redmile Group, and Janus Henderson.

The fresh funding will support advancement of Immunome’s lead asset, a novel IL-38 targeting antibody. It originates from the company’s memory B cell interrogation platform, which sorts through patient blood samples to uncover new therapeutic candidates.

From Morphimmune, a potent TLR7 agonist and radioligand therapy are currently in preclinical testing. The TLR7 program stimulates the immune system against cancer cells when targeted via Morphimmune’s effector platform. The radioligand directly delivers cell-killing radiation.

Siegall highlighted the productive synergy between Morphimmune’s delivery technology and Immunome’s antibody generation engine. The combined company will be able to pursue a wider array of novel targets across multiple therapeutic modalities.

For investors, the merger and additional capital provide Immunome with a deeper pipeline and strengthened financial footing. The $125 million infusion should fund operations well into 2024 even with increased R&D activity.

The more diversified targeted therapy portfolio also helps mitigate risk, with programs based on different mechanisms of action. This provides more shots on goal for achieving clinical success and advancing partnership opportunities.

However, Immunome stock initially fell on news of the deal, indicating some investors were unimpressed by the initial progress made since its August 2020 IPO. The cash position was also becoming strained, likely necessitating the additional financing.

But the opportunity to start fresh under industry ace Siegall may give the story new appeal. His track record of building shareholder value and delivering oncology drugs could reinvigorate Immunome.

The merger puts all the pieces in place to become a fully integrated cancer therapy player. Immunome now has platform technology, industry expertise, development capabilities and a strengthened balance sheet.

Execution will be key, but Siegall’s involvement is about as good as it gets in terms of leadership. For long-term investors, Immunome may offer an intriguing backdoor into the vision of one of biotech’s most accomplished CEOs.

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