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J&J Drug for Rare Infant Blood Disorder Achieves Main Goal of Phase 2 Test

by Bailey Amber
written by Bailey Amber

Johnson & Johnson drug nipocalimab is in late-stage clinical testing in a rare neuromuscular disorder, and if it stays on track could become the third-to-market drug in its class for that indication. But the pharmaceutical giant now has encouraging preliminary data from a separate test in a rare blood disorder affecting fetuses, helping to build the therapy’s case in a life-threatening disease with no approved therapies.

The data announced Monday are from a Phase 2 clinical trial in severe hemolytic disease of the fetus and newborn (HDFN). Stemming from the incompatibility of the infant’s blood type and the mother’s blood type, this disease leads to the mother’s blood crossing the placenta and attacking fetal red blood cells. The resulting anemia can become fatal to the fetus.

Nipocalimab is intended to reduce levels of antibodies implicated in several antibody-driven diseases. The drug is itself an antibody designed to selectively bind to the Fc receptor (FcRN) of immunoglobulin G (IgG), the most common antibody circulating in the blood. J&J’s Janssen Pharmaceutical Companies division said Monday that Phase 2 results showed most of the pregnant patients who received the experimental drug and achieved a live birth did not need an intrauterine transfusion throughout the entire pregnancy, which was the main goal of the study. The number of patients achieving this goal was not specified.

Katie Abouzahr, vice president, autoantibody portfolio development leader at Janssen Research & Development, said in a prepared statement that the full Phase 2 results will be presented at an upcoming scientific medical meeting. She added that the company plans to advance the drug to a pivotal Phase 3 study in this indication.

Nipocalimab joined the J&J pipeline in 2020 via the pharma giant’s $6.5 billion acquisition of Momenta Pharmaceuticals, a biotech that developed drugs by analyzing the interaction of antibodies and receptors that modulate immune responses. Nipocalimab was Momenta’s lead asset, having reached pivotal testing in warm autoimmune hemolytic anemia (WAIHA), a rare disorder in which antibodies destroy healthy red blood cells.

Momenta had envisioned nipocalimab as applicable to multiple autoimmune conditions. J&J embraced that potential and is currently testing the drug in nine clinical trials—six in immunology and three in neuroscience. Those studies span diseases characterized by alloantibodies, which are produced following exposure to a foreign antigen, such as the maternal antibodies that lead to HDFN; diseases characterized by autoantibodies produced in response to the body’s own tissues; and rheumatological disorders such as rheumatoid arthritis and lupus.

Under J&J, nipocalimab has reached pivotal testing in myasthenia gravis, in which autoantibodies interfere with the communication between nerves and muscles. In 2021, FDA-approval of Argenx’s Vyvgart in myasthenia gravis made it the first FcRn-targeting drug to pass the agency’s regulatory bar. The biotech is also developing the drug for other rare antibody-driven disorders. UCB is on Vyvgart’s heels with rozanolixizumab, an FcRn-blocking drug that was submitted for FDA review last month.

The pivotal study of nipocalimab in myasthenia gravis is expected to produce data in 2024, J&J Chairman and CEO Joaquin Duato said during the company’s presentation last month at the J.P. Morgan Healthcare Conference. The most advanced immunology clinical test of the FcRn-targeting drug is the pivotal test in WAIHA. That clinical trial may have a data readout this year, Duato said. A Phase 2 test in rheumatoid arthritis is also expected to yield data this year, he added.

Photo: Niels Wenstedt/BSR Agency, via Getty Images

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What Will the Pharma Industry Face in the Year Ahead?

by Bailey Amber
written by Bailey Amber

Life science organizations faced down an array of daunting challenges during the last three years: a global health crisis, supply chain upheaval, and uncertain economic climate, just to name a few. But if we can say the dust is beginning to settle, we can also look to the future with the knowledge that uncertainty will persist – and that we should be prepared. How can companies prepare for an unpredictable year ahead?

Many pharma and medical device companies will look inward, with the goal of standardizing and modernizing even highly regulated activities. They’ll lean into new approaches for specific challenges, and apply purpose-built technology across drug and device development. Here’s a closer look at some important industry trends for 2023.

Tech maturation matters

Pharmaceutical companies continue to forge ahead in their quest to create a truly end-to-end insights management function. Teams understand that insight generation and analysis are vital to the success of new drug development – but may lack the willingness to devote significant business resources to solving the problem.

Happily, even in a notoriously conservative and risk-averse industry, leaders are coming around to the idea that they can solve the problem with technology. Next year, pharma companies will begin putting both minds and money behind the push to put insights management into the spotlight as a strategic business pillar.

One way they’ll do this is by using artificial intelligence to support – not replace – talented humans who don’t have enough time to manually comb through reams of patient data, medical records, and other important sources of information. For pharmaceutical teams, 2023 will be the year of understanding that AI can be a benevolent partner rather than an intimidating threat.

What’s the danger of ignoring AI? Teams that don’t understand AI applications run the risk of missing out on key insights, and all the opportunities those can provide. This can have a particularly meaningful impact on applications like precision medicine, where developing the best treatment pathway often requires analyzing information about different aspects of the patient experience.

While much of this information is obtained from structured data from electronic medical records, there is also valuable information contained in the unstructured text of physician notes, referral forms, and medical charts. Developing precision therapies also requires input from global experts who won’t necessarily turn up in the usual publishing and speaking circuits. This is an ideal case for technologies that are specific to life science, where gaps in knowledge can interfere with the expedient development of precision and targeted therapies.

Adding agility where it counts

Like other industries, pharmaceutical and medical device companies are eager to return to the days of in-person meetings and busy show floors. But as folks return to racking up frequent flyer miles, an old problem is rearing its head: at in-person events, where do the insights go?

How are important observations collected and shared, and how will that information make its way into the mix with data from other channels, like virtual meetings and social platforms? As organizations balance traditional and tech-enabled ways of working, technology will stay in the picture to add process and consistency.

What does this look like in practice? Teams will gladly return to the muscle memory of an in-person medical congress, but it will be far less chaotic: they’ll use social listening to understand trending topics ahead of and during the event, adding currency to their real-time conversations. They might share same-day observations in a virtual venue and achieve alignment on important discussions before they pack to go home. And once the event is over, the conversations can continue online, with sentiment analysis tools to potentially shorten the time from insight to action by weeks, or even months.

Increasing the all-important agility factor simply equips life science organizations to have more choice in engaging truly global audiences, eliminating traditional roadblocks like travel time and expense, disparate geographical locations, and different preferred languages. Having experienced first-hand what it’s like to work in a world where travel – even as far as an in-town office or clinic – is impossible, the importance of this flexibility can’t be overstated.

As pandemic-induced limitations fell away, many old habits returned. Some, like traveling and meeting in person, were welcomed. Others, such as congress chaos and deluges of data, have been less well-received. The year ahead will be about figuring out how to move forward with flexibility and preparedness when – not if – the next challenge arises.

Photo: Feodora Chiosea, Getty Images

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By Eliminating Requirement for Animal Testing, FDA Modernization Act Allows Faster, More Cost-effective Drug Development

by Bailey Amber
written by Bailey Amber

Congress passed the FDA Modernization Act 2.0 last week removing the requirement to use animal testing in drug development. This will allow drug companies the option to use alternative safety-testing models when making new drug submissions.

This is important because the Federal Food, Drug and Cosmetics Act of 1938, which is still in force, mandates animal testing for every new drug development protocol. Advocates of non-animal experimentation have argued that the cost of animal research is high—in dollars, in time, and in delays in approvals of beneficial drugs for human use. They also maintain that some drugs that were deemed safe by animal studies went on to cause harm in human subjects in clinical testing, although this last position is poorly quantified.

Once the FDA Modernization Act 2.0 is enacted, a transitional moment will follow. The section of the House bill that allows for alternatives to animal use to be incorporated into pre-clinical testing outlines approaches most likely to predict human response based on scientific evidence. These include cell-based assays; organ chips and microphysiological systems; and sophisticated computer modeling. Several of these approaches allow drug developers to incorporate safety assessment alternatives that are robust enough to convince regulators that a program has been adequately de-risked.

The bill comes at a pivotal time; modern toxicity testing has been moving away from a reliance on animal studies. One substitute has been mechanism-based testing strategies, such as cell-based assays. These are already being used as research tools to support the interpretation of in vitro toxicity data, as well as the design of in vitro experiments, and considerable progress has been achieved in making assays available and deployable in a user-friendly form.

So-called organ chips and microphysiological systems began to be used more frequently during Covid-19 drug and vaccine testing, which reportedly helped researchers to better understand how Covid-19 interacted with human organs and elicited an immune response. Using human cells and engineered structures, these approaches create an environment that mimics or models the function of organs, and they may have application in testing drug efficacy in genetically diverse human populations using human genetic material.

The average cost of bringing a drug to market is about $2 billion, of which more than half is spent on clinical trials. But before trials are conducted, researchers are increasingly employing computer modeling, a technology that has been expanding for several decades and which provides several benefits.

Sophisticated modeling that uses in-silico, computer-based testing with virtual patients, biosimulation is fast and relatively inexpensive and reveals rich information about how a drug would perform and how to best design a trial before the drug is ever tested in patients. Biosimulation also offers the flexibility of computer-based testing, allowing developers to optimize trial design and dosing for different patient populations.

By easing regulatory requirements for animal testing, the Act allows scientists to use innovative, leading-edge technologies more fully in future drug development strategies.  These alternatives are not yet the complete answer, but by increasingly taking a “totality of evidence” approach where the combination of multiple data points can be used to assess whether a confidence threshold is reached in relation to the safety of a drug at a particular dose, regulators are allowing developers to accelerate the process.

Now that Congress has passed the FDA Modernization Act, drug companies should consider how to take advantage of the translational tools outlined above, which can be an important part of the drug developer’s arsenal. Depending on how they are used, they can result in researchers more cost-effectively developing the lifesaving drugs and vaccines that patients need, faster.

Photo: Rawf8, Getty Images

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FogPharma’s $178M Funding Leads Way as 7 Biotechs Raise Cash for Cancer Drugs

by Bailey Amber
written by Bailey Amber

Cancer continues to be a big draw for biotech investors, and cancer research is well represented in the past week’s financing activity. Seven biotech companies announced financing rounds to support a range of tumor-targeting therapeutic approaches that include small molecules, oncolytic viruses, and peptide drugs.

Peptides are the focus of FogPharma, which raised the largest financing round of the Thanksgiving holiday-shortened week. The Cambridge, Massachusetts-based biotech is developing a new class of peptide drugs that address therapeutic targets deemed “undruggable.” FogPharma is led by CEO Greg Verdine, a former Harvard professor who has become a biotech entrepreneur. Verdine is also chief executive of LifeMine Therapeutics, a GSK-partnered startup that analyzes fungal genes to discover new drugs.

Arch Venture Partners and Invus are among the financial backers of LifeMine, and those firms also participated in FogPharma’s new $178 million round of funding. The Series D financing comes as the biotech prepares for its first clinical trial. The company says polypeptide drugs from its Helicon platform combine the targeting abilities of antibodies with the features of small molecule drugs: broad tissue distribution, intracellular target engagement, and oral dosing. Lead program FOG-001 is designed to block TCF-blocking beta-catenin to address a dysregulated signaling pathway found in an estimated 20% of all human cancers.

In preclinical research, FogPharma says FOG-001 stopped tumor growth and led to tumor regression. The company plans to submit an investigational new drug application and start Phase 1 testing by mid-2023. The new capital will also support development of FogPharma’s preclinical pipeline, which addresses other biologically validated but elusive cancer drug targets such as TEAD, NRAS, Pan-KRAS, and Cyclin E1.

Here’s a look at the other biotech financings for the past week:

—Nearly three months after Roche reached a deal to acquire cancer drug developer Good Therapeutics in a $250 million deal, a spinout from the biotech called Bonum Therapeutics has raised $93 million in Series A financing. Seattle-based Bonum is developing cytokine cancer therapies for cancer therapies. These drugs will be conditionally activated, meaning that they will activate only when the antibody sensor component of the therapy binds to its target, which is intended to reduce toxicity.

The technology that is the basis for Bonum’s drugs was validated by Good. Good’s financial backers, including Rivervest Venture Partners, Roche Venture Fund, Digitalis Ventures, 3×5 Partners, and Codon Capital, teamed up again for Bonum’s Series A financing, which added a new investor, Vivo Capital.

—Clinical-stage CG Oncology closed a $120 million Series E financing. The Irvine, California-based biotech’s lead drug candidate, CG0070, is an oncolytic virus that has reached Phase 3 testing as a monotherapy for non-muscle invasive bladder cancer that does not respond to Bacillus Calmette-Guerin, the most common intravesical immunotherapy used for treating early-stage bladder cancer. A Phase 2 study is also underway testing CG0070 in combination with Merck immunotherapy Keytruda. CG Oncology said it will use the new capital to advance its lead programs toward FDA review and broaden its drug pipeline to address other unmet needs in urologic cancer.

—CatalYM closed a €50 million Series C financing to expand Phase 2 clinical testing of its lead program, which is in development for treating solid tumors. The antibody drug candidate, visugromab, is engineered to neutralize Growth Differentiation Factor-15 (GDF-15), a tumor-produced protein that regulates immune cell activation and stops immune cells from infiltrating tumor tissue. Visugromab’s solid tumor test is evaluating the drug in combination with a type of immunotherapy that blocks the checkpoint protein PD-1. Preliminary data are expected in early 2023. Munich, Germany-based CatalYM’s new round of financing was co-led by Brandon Capital and Jeito Capital.

—Casma Therapeutics closed $46 million in Series C financing to bring its lead program for MYD88 mutant lymphoma through the preclinical research that will support an investigational new drug application. The Cambridge, Massachusetts-based company develops therapies that leverage autophagy, a mechanism for recycling old or damaged cellular components. A similar approach called targeted protein degradation focuses only on proteins and peptides. But autophagy can address larger cellular components such as organelles.

—Rezo Therapeutics, a University of California at San Francisco spinout that is developing new cancer drugs, launched with $78 million. The technology of the San Francisco-based company identifies how mutations rewire cancer-driving networks, using that insight to uncover tumor-specific drug targets. This tech platform comes from UCSF’s Quantitative Biosciences Institute. Rezo’s Series A financing was led by SR One, a16z Bio + Health, and Norwest Venture Partners.

—Opna Bio, a startup developing cancer therapies acquired from Plexxikon, unveiled $38 million in Series A financing. The company’s co-founders include Douglas Hanahan, a distinguished scholar in the Lausanne Branch of the Ludwig Institute for Cancer Research and emeritus professor at the Swiss Federal Institute of Technology Lausanne. Opna’s launch and financing comes as research from Hanahan’s EPFL laboratory was published in the journal Science describing the role of fragile X mental retardation protein (FMPL) as an immuno-oncology target. Opna has licensed this FMPL technology. Longitude Capital and Northpond Ventures led the Series A round of Opna, which maintains operations in Lausanne, Switzerland, and South San Francisco.

—In non-cancer biotech funding news, MBX Biosciences raised $115 million to develop therapies in a new class of peptide drugs, including a lead program in early-stage clinical development for hypoparathyroidism. Carmel, Indiana-based MBX says its precision endocrine peptides, or PEPS, overcome limitations of traditional peptide drugs. In addition to supporting lead PEP product candidate MBX 2109, MBX said the new capital will support its preclinical drug pipeline. The Series B round of funding was led by Wellington Management.

—Bain Capital Life Sciences led a $107 million investment in Jnana Therapeutics as the biotech continues Phase 1 testing that could demonstrate clinical proof of concept for its lead program, a potential treatment for phenylketonuria. The inherited metabolic disorder leads to a deficiency of phenylalanine hydroxylase, an enzyme required to break down an amino acid called phenylalanine. Jnana’s drug, JNT-517, is small molecule designed to block phenylalanine reabsorption in the kidney, which in turn reduces blood levels of phenylalanine.

Separate from Jnana’s Series C financing, the biotech announced a second collaboration with Roche focused on the discovery of small molecule drugs for cancer, immune-mediated diseases, and neurological disorders. The Swiss pharmaceutical giant is paying Jnana $50 million up front; milestone payments could reach up to $2 billion.

Image by nopparit, Getty Images

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Why and How To Achieve Effective Data Management Strategies Early in the Biopharma Lifecycle

by Bailey Amber
written by Bailey Amber

therapeutics

With more sophisticated therapeutics comes larger and more complex data sets. The complexity is heightened with the advancements in personalized medicine as it introduces new data points at apheresis (the extraction and infusion of patient blood, cells, tissue and/or regenerative medicinal compounds) and during enhancements of cells and genes.

While data integrity is critical to building confidence in the supply chain and product quality, as well as meeting FDA compliance obligations, many organizations remain highly reliant on spreadsheets, manual data entry, paper records and email. This creates numerous opportunities for error and can result in FDA warning letters, fines or recalls. While data “capture” may start early in biopharmaceutical R&D, oftentimes, a variety of disparate IT systems are installed without a view towards data coherence throughout process development and clinical and commercial manufacturing.

These challenges are compounded by the universal reliance on external partners for significant process development and manufacturing operations.

To attenuate the risks of delayed, incomplete and inconsistent data, biopharma companies must establish a solid data management approach early in product development. Especially for startups that may not have a lot of IT experience or staff, this can be daunting.

The following items should be prioritized in order to better address and mitigate enterprise risks around data integrity and reliability:

  • Creation of a digital data backbone throughout the product and process lifecycle and across internal and external teams, sites and partners
  • Interdepartmental review of Quality and Supply Agreements with CDMOs [contract development and manufacturing organizations] to ensure data visibility, IP ownership and process oversight

Establish a single digital data backbone early

There are new business demands for information to be processed faster. Building a digital data backbone early supports key activities further downstream – late-stage process development, scale-up and tech transfer, and manufacturing where quality assurance and compliance requirements come into the picture.

New digital data systems retain or establish the context and relative importance of data collected from the IT infrastructure. By implementing a cloud-based data backbone, data can be gathered and organized in a central platform without compromising context. It can scale as product and IT infrastructure matures, and remains relevant as it integrates with systems like LIMS [laboratory informatics managements systems], historians, MES [manufacturing execution systems] and eBRs [electronics batch records software], to serve as your single verifiable source of truth for data critical to monitoring process control and conducting analysis and reporting.

With the increasing demand for accelerated tech transfer, FDA filings and commercialization, creating a data backbone early generates significant time and cost benefits: fewer PPQ [process performance qualification] runs, right first time tech transfer, streamlined investigations and production and earlier batch release.

Though a cloud-based data management solution is the first step, companies must also be vigilant when partnering with manufacturers.

Data visibility in quality and supply agreements

With the acceleration of new drug and therapy development, complex manufacturing requirements, and associated capital investments, the growth in outsourcing is predicted to continue for the foreseeable future.

Despite outsourcing manufacturing, the drug owner (sponsor) remains liable for meeting the FDA’s standards for product quality, demonstrating control over the contract manufacturer and the drug manufacturing process, and establishing an inscrutable, high-integrity process, product and quality data set. The near-universal reliance on contract manufacturers, and the FDA’s focus on data integrity issues in drug manufacturing, have generated unprecedented scrutiny into manufacturing operations by the FDA, strategic acquirers and the SEC. As the supply chain continues to expand in complexity, process development and manufacturing, data management is an area that demands new approaches/innovation.

While data integrity challenges can lead to quality and operational issues, they can also create legal risks, such as loss of manufacturing intellectual property and failure to demonstrate control over the CDMO, which can affect the company’s enterprise value.

Although these challenges affect large and small companies alike, data visibility is a key pain point for small biopharma companies, as most are 100% reliant on CDMOs but often lack the expertise and/or negotiation power against well-established CDMOs.

Despite mandates from the FDA for managing their CDMOs – and the manufacturing processes, drug owners struggle to meet these requirements, being physically remote and often lacking IT systems designed for data sharing between owner and contract partners. Failure to meet this requirement can result in the issuance of FDA warning letters. In fact, approximately 50% of all FDA warning letters in 2019 were related to data integrity issues.

Supply agreements must anticipate data needs and emphasize data visibility and ownership of critical information, including process control parameters.

Fortunately, an increasing number of CDMOs realize the compliance burden on their drug sponsors and that the future of biopharma is dependent upon collaboration and visibility in their manufacturing workflows. With state-of-art data management solutions and collaboration with CDMOs, biopharma companies can become more confident in their product quality and more prepared to satisfy strict compliance requirements.

Cloud-based data management solutions help the industry meet its business and compliance challenges. These platforms need to replace traditional data management methods and workflows for biopharma companies and CDMOs that seek competitive advantages.

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Autoimmune Biotech Zenas Gets $118M to Take Antibody Drug Into Phase 3

by Bailey Amber
written by Bailey Amber

Zenas BioPharma is preparing to take its lead drug candidate into a global Phase 3 test and it now has $118 million to finance the clinical research.

The Zenas drug, obexelimab, is being developed as a treatment for IgG4-RD, a chronic inflammatory disease that affects multiple organs. First-line treatment includes steroids, but these drugs can have toxic effects and relapse is common.

Obexelimab is intended to tamp down the activity of B cells, a type of immune cell. The drug is a bispecific antibody. One part of the drug binds to CD19, a protein on the surface of B cells. The other part of obexelimab binds to another immune cell target called FcYRIIB. According to the company, binding to both targets simultaneously mimics a natural antigen-antibody complex that down regulates B cell activity. This approach has potential applications in many autoimmune disorders.

Zenas acquired global rights to obexelimab from Xencor last year, issuing equity to that company as an upfront payment. According to the deal terms, Xencor could receive up to $480 million in milestone payments depending on Zenas’s progress with the drug.

In addition to supporting obexelimab, Zenas said the new capital, a Series B financing, will also go toward advancing other programs into clinical development in 2023. Enavate Sciences led the latest investment in Waltham, Massachusetts-based Zenas. Other new investors in the company include Longitude Capital, Vivo Capital, Rock Springs Capital, Perceptive Advisors, Agent Capital, Pivotal bioVenture Partners and Superstring Capital. Earlier investors Fairmount, Wellington Management, Tellus BioVentures, Quan Venture Fund, and Xencor also participated in the financing

Zenas’s $118 million financing was the biggest biotech financing of this week. Here’s a look at other companies that raised money:

—Bringing computational techniques to the study of secreted proteins, Juvena Therapeutics is developing new drugs for chronic disorders and diseases of aging. Juvena’s lead program is in preclinical development for the muscle disorder myotonic dystrophy type 1 and the Redwood City, California-based startup has raised $41 million to support that drug and others in the pipeline. Mubadala Capital and Horizons Ventures led Juvena’s Series A financing.

—Sensorium Therapeutics emerged with $30 million in funding to develop psychoactive molecules to address mental health disorders. Founded by scientists from Massachusetts General Hospital and Harvard Medical School, Sensorium’s research has produced a lead program, SENS-01, for anxiety and depression. The Boston-based biotech said it expects to begin preclinical research that could support an investigational new drug application in 2023, laying the groundwork to reach clinical testing by early 2024. Santé Ventures led Sensorium’s Series A financing.

—New investors pumped money into Lipidio Pharmaceuticals, extending the biotech’s Series A round of financing to more than $20 million. San Diego-based Lipidio is developing drugs for metabolic and skin disorders. Lead drug candidate GDD3898 is in development for treating acne, sebaceous hyperplasia, obesity, and Prader-Willi syndrome. The company said it expects to complete three early-stage studies of the drug by the end of this year, laying the groundwork for data analysis and a Series B financing planned for the first quarter of 2023.

—Blood transfusion technology company Hemanext raised $18 million. The Lexington, Massachusetts-based company has CE mark certification in Europe for a red blood cell processing and storage system and is working to expand its presence into additional European markets in 2023. Hemanext described the latest financing as the first close of its Series B round. The company says it has raised about $130 million total to date.

—Neuroscience company NRG Therapeutics closed £16 million in financing to continue development of small molecule drugs that penetrate the brain to potentially slow or halt neurodegeneration. The drugs of the Stevenage, England-based company target mitochondria, the powerhouses of cells. The Series A round of financing was led by Omega Funds. NRG plans to use the capital to advance its molecules through the preclinical research needed to support an investigational new drug application.

Picture: Feodora Chiosea, Getty Images

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AstraZeneca & Sanofi Win European Nod for RSV-Preventing Drug for Infants

by Bailey Amber
written by Bailey Amber

Respiratory syncytial virus’s early surge this year has some healthcare officials concerned that this season could be a rough one. Infants, who are among the most vulnerable to infection from this pathogen, now have a new protective measure. The European Commission on Friday granted marketing authorization to an AstraZeneca and Sanofi drug that protects newborns from RSV infection in their first months of life.

The drug, an antibody called nirsevimab, is engineered to bind to the RSV’s fusion protein, preventing the virus from using that protein to enter cells. Partners AstraZeneca and Sanofi will market the new drug under the name “Beyfortus.”

The AstraZeneca and Sanofi drug protects differently than a vaccine. A vaccine triggers the immune system to produce an antibody, offering what is called active immunization. This type of immunity can take weeks to develop. Beyfortus is the protective antibody engineered in a lab. Administered directly to the infant, this passive immunity provides immediate protection.

AstraZeneca had previously commercialized a different RSV drug, Synagis. That antibody was developed to provide premature babies with protective antibodies that they lack. But Synagis has a half-life of about 20 days. It’s dosed as an intramuscular injection given before the RSV season starts, then once monthly during the RSV season.

AstraZeneca has described Beyfortus as a next-generation RSV antibody. It is engineered to have a long-half life—long enough to last the entire RSV season. That longer-life is intended to protect the baby during the first six months of life, the time when an infant is most vulnerable to RSV infection. Beyfortus can also address more babies as the product’s label covers infants broadly, not just those who are born prematurely.

The European Commission decision is based on clinical trial results showing that compared to a placebo, a single dose of Beyfortus met the main goal of reducing the incidence of medically attended lower respiratory tract infections during the RSV season. The companies reported that the drug’s safety was similar to a placebo. Also, clinical trial results showed that Beyfortus’s safety and tolerability were comparable to Synagis.

The Beyfortus vaccine alliance between AstraZeneca and Sanofi dates to 2017, when the antibody was still in mid-stage clinical testing. Sanofi paid its new partner €120 million up front; another €495 million was tied to the achievement of milestones. According to the deal terms, AstraZeneca took the lead on development and manufacturing activities. Sanofi will lead commercialization of the product. The partners share equally in development costs and profits from sales.

AstraZeneca and Sanofi aren’t the only companies trying to improve the way infants are protected from RSV. Earlier this week, Pfizer reported positive Phase 3 data for its RSV vaccine for infants, called RSVpreF. That vaccine offers a different form of passive immunization. This maternal vaccine is administered to a healthy mother, whose immune system produces antibodies that pass through the placenta and to the baby. Pfizer said it plans to seek FDA approval by the end of this year, followed by regulatory submissions with other global authorities in coming months.

Photo: sinonimas, Getty Images

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CVS, Walgreens will pay $10 billion total to settle opioid-related lawsuits

by Bailey Amber
written by Bailey Amber

CVS Health and Walgreens both announced today they would pay nearly $5 billion each in a settlement to resolve all opioid-related litigation and claims against the companies. 

The final settlement depends on the number of states, counties, and Native American tribes that agree to join, but if the non-monetary terms are finalized, CVS Health will pay $4.9 billion over the next 10 years and Walgreens will pay approximately $4.95 billion in remediation payments to be paid out over 15 years, according to separate news releases from each company.  

“We are pleased to resolve these longstanding claims and putting them behind us is in the best interest of all parties, as well as our customers, colleagues and shareholders,” said Thomas Moriarty, Chief Policy Officer and General Counsel, CVS Health in a news release. “We are committed to working with states, municipalities and tribes, and will continue our own important initiatives to help reduce the illegitimate use of prescription opioids.” 

Walgreens also released a statement. “We believe this is in the best interest of the company and our stakeholders at this time, and allows our pharmacists, dedicated healthcare professionals who live and work in the communities they serve, to continue playing a critical role in providing education and resources to help combat opioid misuse and abuse,” the company said in a news release. 

In the news release from Walgreens, the company said it expects the financial settlement would resolve all opioid claims against the company. 

Reuters reported that Walmart would also pay to settle opioid-litigation claims, but when asked by MedCity News to confirm this, the company said they had no comment. 

There are more than 3,000 cases that are part of the multidistrict litigation concerning the opioid epidemic. So far, most drugmakers and distributors have reached settlements with counties that brought cases against them. In a unique ruling in August in which a judge sided against large pharmacies, an Ohio district court judge ordered CVS Health, Walgreens and Walmart to pay $650 million to two Ohio counties for their part in the opioid epidemic. 

CVS Health conducted a call disclosing the company’s third-quarter earnings on Wednesday following the news release about the settlement. The company reported earnings of $81.2 billion for the third quarter, up 10% compared to the third quarter of 2021. The company reported higher losses than last year, in part due to the $5.2 billion in opioid-related litigation, according to a news release. 

When asked about the likelihood of states signing onto the settlement agreement, CVS Health CEO Karen Lynch said she has a “high degree of confidence” because state attorneys general were at the table in the past few months during mediation conversations.

CVS Health and Walgreens did not respond to a request for comment. 

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Pfizer RSV Vaccine Hits Trial Goal for Infants; Year-End FDA Filing Planned

by Bailey Amber
written by Bailey Amber

Pfizer is one step closer to a successful vaccine that prevents severe respiratory syncytial virus infection in infants—infection that can become deadly in young and developing lungs. The pharmaceutical giant reported Tuesday that its vaccine candidate met a key goal in a pivotal study, paving the way for plans to seek regulatory approvals by the end of this year.

The Pfizer RSV vaccine, called RSVpreF, is based on a prefusion form of RSV F, a protein that the virus uses to enter cells. Using a stabilized version of that protein, the vaccine is intended to elicit antibodies that confer protection. For infants, the vaccine works through a practice called maternal immunization. The vaccine is administered to a healthy mother during pregnancy. Antibodies produced by the mother’s immune system pass through the placenta to reach the baby. Following birth, those antibodies provide temporary protection while the infant’s immune system matures. This time is key for an RSV vaccine because the first six months of life are when infants are most vulnerable to RSV infection.

The double-blind Phase 3 study enrolled about 7,400 pregnant participants in 18 countries. Those participants were randomly assigned to receive a single dose of the RSV vaccine or a placebo, then followed for six months after delivery. Follow-up in infants continued for at least one year to assess the safety and efficacy of the vaccine.

According to Pfizer, an independent interim analysis of the trial data found that through the infant’s first three months of life, the observed efficacy against severe medically attended lower respiratory tract illness was 81.8%. At the six-month follow-up mark, Pfizer reported “substantial efficacy” of 69.4%, which was enough to meet one of the main goals of the study.

The study did not achieve a secondary goal of assessing efficacy against all causes of medically attended lower respiratory tract illness. On that measure, Pfizer reported “clinically meaningful efficacy” of 57.1% from birth through the first three months of life. At six months, that efficacy measure was 51.3%. The pre-planned safety reviews conducted regularly throughout the study indicate the vaccine is well-tolerated with no safety concerns for both the vaccinated mothers and their newborns.

Pfizer said that it has stopped enrollment in the study at the recommendation of the independent data monitoring committee and in consultation with the FDA. Based on these positive results, the company said it plans to submit a biologics license application with the FDA by the end of this year, followed by applications with other agencies in the coming months. Pfizer said it also plans to submit the clinical trial results data for peer-review in a scientific journal.

The RSV vaccine results in infants come two months after Pfizer reported efficacy results for RSVpreF in adults age 60 and older. Based on those efficacy results, the pharma company said it planned to seek regulatory approval in this patient population in the fall. GSK is among the other companies developing an RSV vaccine for older adults. Last month, the British pharmaceutical giant reported positive Phase 3 data for that vaccine candidate, RSVPref3.

Photo: metinkiyak, Getty images

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Self Care

Report: Cancer deaths are down but pancreatic cancer is growing, racial disparities persist

by Bailey Amber
written by Bailey Amber

Cancer death rates are coming down for adults and children, and the declines are steepest in some of the most commonly diagnosed types of cancers. However, pancreatic cancer stands out as one of the few cancer types whose incidence and death rates are on the rise.

The findings come from the Annual Report to the Nation on the Status of Cancer, a document compiled by the National Cancer Institute, the Centers for Disease Control and Prevention, the American Cancer Society, and the North American Association of Central Cancer Registries. Those entities have collaborated on the annual report since 1998. The most recent data for this year’s report are from 2019; the findings are based data from before the COVID-19 pandemic. The report was published in the journal Cancer.

Let’s start with the declines. From 2015 to 2019, cancer death rates in men decreased by 2.3% per year, according to the report. In women, death rates decreased by 1.9% per year. The recent reductions observed in cancer deaths was driven by declines in lung cancer mortality, the report said. Lung cancer and melanoma together saw the declines in both men and women.

Declines in lung cancer incidence and deaths were attributed to continuous declines in smoking as well as better treatments, specifically advances in targeted therapies and immunotherapies for non-small cell lung cancer (NSCLC). AstraZeneca’s Iressa became the first targeted therapy approved for NSCLC in 2003 leading the way for other targeted therapies to pass regulatory muster, followed by the class of cancer immunotherapies called checkpoint inhibitors.

Non-treatment variables that may contribute to the declines in lung cancer deaths include improved access to care following the expansion of Medicaid to a broader group of low-income adults starting in 2014, the report said. Another factor is the increase in screening, which in turn leads to earlier diagnosis. Despite this progress, the report notes that lung cancer remains the most common cause of cancer death across racial and ethnic groups and the more than 30 million adults who smoke cigarettes.

In adolescents and young adults, defined as ages 15 to 39, the report states cancer incidence rates increased by less than 1% per year from 2014 to 2018. The most common type of cancer in this age group was female breast cancer; next was thyroid cancer.

In some cancers, the death rates are climbing. In men, the report shows an increase in death rates for cancers of the pancreas, brain, and bones and joints. In women, death rates are increasing for cancers of the pancreas and uterus. Pancreatic cancer accounts for just 3% of new cancer diagnoses. Despite the rareness of this type of cancer, it leads to 8% of cancer deaths, making it the fourth-leading cause of cancer deaths for males and females, the report said.

From 2001 to 2018, the incidence of pancreatic cancer increased by 1% per year in men and women; death rates increased by 0.2% per year, according to the report. Two of the most common subtypes of pancreatic cancer are neuroendocrine tumors and adenocarcinomas. Both types increased in men and women from 2001 to 2018.

Despite those increases, the analysis also found survival improvements according to subtype. In pancreatic neuroendocrine tumors the report found that one-year survival increased from 65.9% in 2001 to 84.2% in 2017. For those diagnosed with pancreatic adenocarcinomas, one-year survival rose from 24% to 36.7% in the same time span. Survival also increased at the five-year mark. These survival improvements may be due to improvements in therapy. However, no improvement was seen for other types of pancreatic tumors, which were mostly diagnosed in older adults.

Racial disparities persist for some types of cancer. While the incidence rates for bladder cancer declined in Whites, Blacks, Asian/Pacific Islanders, and Hispanics, the report found rates increased in American Indian and Alaska Native people. The incidence of uterine cancer was similar in Black and White women, but the death rates from this type of cancer are nearly twice as high in Black women. For breast cancer, the death rate for Blacks is 40% higher than it is for Whites.

“The highest breast cancer death rates seen among Black females are partially caused by the significant barriers to providing access to timely, high-quality medical care, which require addressing multiple dimensions of disparity across the continuum of cancer care,” the report said.

Photo: nopparit, Getty Images 

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