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Pfizer Invites Public to Listen to Webcast of Pfizer Discussion at Healthcare Conference

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Pfizer Invites Public to Listen to Webcast of Pfizer Discussion at Healthcare Conference

February 09, 2023: “Pfizer Inc. invites investors and the general public to listen to a webcast of a discussion with Andy Schmeltz, SVP Commercial Strategy & Innovation, and Navin Katyal, U.S. Commercial & Global Business Lead for mRNA Portfolio at the SVB Securities Global Biopharma Conference on Thursday, February 16, 2023 at 10:00 a.m. Eastern Standard Time.

To listen to the webcast, visit our web site at www.pfizer.com/investors. Information on accessing and registering for the webcast will be available at www.pfizer.com/investors beginning today.

The transcript and webcast replay of the discussion will be made available on our web site at www.pfizer.com/investors within 24 hours after the end of the live discussion and will be accessible for at least 90 days.

About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives.

We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines.

Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time.

Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world.

For more than 170 years, we have worked to make a difference for all who rely on us.”

https://www.pfizer.com/news/press-release/press-release-detail/pfizer-invites-public-listen-webcast-pfizer-discussion-14

GSK announces results from 17-year retrospective study on US clinical trial diversity

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GSK announces results from 17-year retrospective study on US clinical trial diversity

February 06, 2023: “GSK plc announced results from a retrospective study on clinical trial diversity, investigating the historical representation of US-based participants in GSK clinical trials.

The study looked at clinical trial demographic data from 495 GSK and ViiV clinical trials involving US-based participants from the period 2002 to 2019.

The results published today in Clinical Trials: Journal of the Society of Clinical Trials demonstrate that using real world diseases epidemiology data, rather than the traditional benchmark of US Census Bureau race and ethnicity data, would ensure clinical trial enrolment reflects the populations affected by different diseases.

The results will inform how GSK and potentially other clinical trial sponsors can better design research to represent the diversity of real-world patient populations, given that some diseases disproportionately impact certain racial and ethnic groups.

The safety and efficacy of medicines and vaccines can differ based on genetic or environmental factors, and appropriate representation in clinical research is critical for advancing our understanding of new medicines and vaccines to ensure they have the biggest impact on patients.

Christopher Corsico, Senior Vice President, Development, GSK said: “GSK is committed to ensuring our clinical trials reflect the diverse demographics of the patients impacted by the disease under study, given the disproportional impact some diseases may have on specific patient groups.

While we have more to do at GSK, today’s publication offers important new data and analyses for sponsors to consider when planning their clinical programmes.

To make meaningful progress on diverse participation in clinical trials, we need meaningful collaboration with regulators, patients, academia, other biopharma companies and the wider healthcare ecosystem so that together we can achieve a shared goal of better health outcomes for all.”

US Census Bureau data do not necessarily reflect the proportion of the population by ethnicity that may be impacted by a specific disease.

GSK’s study confirmed that in four disease areas (asthma, COPD, HIV and influenza) census data differed from the epidemiological data.

For example, while US Census Bureau data indicates 13.4% of the total US population is Black/ African American, 17% of the US population with asthma, 7.1% of those with COPD, 55.3% of those with HIV and 23.7% of those with influenza are Black/African American.

In addition, US Census Bureau data indicates 18.5% of the total US population is Hispanic/ Latinx while the proportion of the US population who have each disease who are Hispanic/Latinx is 14.4% for asthma; 6.5% for COPD; 35.7% for HIV and 10.4% for influenza.

The study also showed that GSK trial enrolment for each condition differed by race and ethnicity.

For example, enrolment in clinical trials of Black/ African Americans for asthma (22.6%) exceeded both census (13.4%) and epidemiologic (17%) levels.

In HIV trials, enrolment of Black/ African Americans at 35.1% markedly exceeded census levels of 13.4% but underrepresented epidemiologic levels of 55.3%.

GSK is committed to applying the insights from the study results in ongoing efforts to improve diversity in clinical trial enrolment. At the end of 2022, 100% of GSK’s Phase III trials had a diversity plan in place to enrol the groups most affected by the disease being studied, as based on epidemiology data.

GSK is working with patient advocacy groups and academic organisations to tackle the barriers that negatively impact clinical trial turnout of certain patient populations, such as access to transportation, language differences, lack of trust and awareness.

One way GSK is addressing these barriers is by rolling out global cultural competency training to around 15,000 clinical trial staff members so they can engage with diverse communities and advocacy groups to build trust, enhance disease awareness and provide appropriately tailored information.

Further research will support industry efforts to improve representation in clinical trials.

To build on the US-focused analysis published today, GSK is currently evaluating retrospective clinical trial demographic data for past participants who were enrolled in other countries around the world.

Regional, demographic and therapeutic area analyses of the full global data set are planned to further expand on the evidence base for improving diversity benchmarking in future clinical research.”

https://www.gsk.com/en-gb/media/press-releases/gsk-announces-results-from-17-year-retrospective-study-on-us-clinical-trial-diversity/

GSK announces results from 17-year retrospective study on US clinical trial diversity

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GSK announces results from 17-year retrospective study on US clinical trial diversity

February 06, 2023: “GSK plc announced results from a retrospective study on clinical trial diversity, investigating the historical representation of US-based participants in GSK clinical trials.

The study looked at clinical trial demographic data from 495 GSK and ViiV clinical trials involving US-based participants from the period 2002 to 2019.

The results published today in Clinical Trials: Journal of the Society of Clinical Trials demonstrate that using real world diseases epidemiology data, rather than the traditional benchmark of US Census Bureau race and ethnicity data, would ensure clinical trial enrolment reflects the populations affected by different diseases.

The results will inform how GSK and potentially other clinical trial sponsors can better design research to represent the diversity of real-world patient populations, given that some diseases disproportionately impact certain racial and ethnic groups.

The safety and efficacy of medicines and vaccines can differ based on genetic or environmental factors, and appropriate representation in clinical research is critical for advancing our understanding of new medicines and vaccines to ensure they have the biggest impact on patients.

Christopher Corsico, Senior Vice President, Development, GSK said: “GSK is committed to ensuring our clinical trials reflect the diverse demographics of the patients impacted by the disease under study, given the disproportional impact some diseases may have on specific patient groups.

While we have more to do at GSK, today’s publication offers important new data and analyses for sponsors to consider when planning their clinical programmes.

To make meaningful progress on diverse participation in clinical trials, we need meaningful collaboration with regulators, patients, academia, other biopharma companies and the wider healthcare ecosystem so that together we can achieve a shared goal of better health outcomes for all.”

US Census Bureau data do not necessarily reflect the proportion of the population by ethnicity that may be impacted by a specific disease.

GSK’s study confirmed that in four disease areas (asthma, COPD, HIV and influenza) census data differed from the epidemiological data.

For example, US Census Bureau data indicates 13.4% of the population is Black/ African American while the prevalence of asthma among this group in the US is 17%; COPD is 7.1%; HIV is 55.3%; and influenza is 23.7%.

In addition, US Census Bureau data indicates 18.5% of the population is Hispanic/ Latinx while the prevalence of different diseases among this group varies (asthma 14.4%; COPD 6.5%, HIV 35.7% and influenza 10.4%).

The study also showed that GSK trial enrolment for each condition differed by race and ethnicity.

For example, enrolment in clinical trials of Black/ African Americans for asthma (22.6%) exceeded both census (13.4%) and epidemiologic (17%) levels.

In HIV trials, enrolment of Black/ African Americans at 35.1% markedly exceeded census levels of 13.4% but underrepresented epidemiologic levels of 55.3%.

GSK is committed to applying the insights from the study results in ongoing efforts to improve diversity in clinical trial enrolment.

At the end of 2022, 100% of GSK’s Phase III trials had a diversity plan in place to enrol the groups most affected by the disease being studied, as based on epidemiology data.

GSK is working with patient advocacy groups and academic organisations to tackle the barriers that negatively impact clinical trial turnout of certain patient populations, such as access to transportation, language differences, lack of trust and awareness.

One way GSK is addressing these barriers is by rolling out global cultural competency training to around 15,000 clinical trial staff members so they can engage with diverse communities and advocacy groups to build trust, enhance disease awareness and provide appropriately tailored information.

Further research will support industry efforts to improve representation in clinical trials.

To build on the US-focused analysis published today, GSK is currently evaluating retrospective clinical trial demographic data for past participants who were enrolled in other countries around the world.

Regional, demographic and therapeutic area analyses of the full global data set are planned to further expand on the evidence base for improving diversity benchmarking in future clinical research.”

https://www.gsk.com/en-gb/media/press-releases/gsk-announces-results-from-17-year-retrospective-study-on-us-clinical-trial-diversity/

Forxiga approved in EU for treatment of symptomatic chronic heart failure

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Forxiga approved in EU for treatment of symptomatic chronic heart failure

February 2023: “Forxiga (dapagliflozin) has been approved in the European Union to extend the indication for heart failure (HF) with reduced ejection fraction (HFrEF) to cover patients across the full spectrum of left ventricular ejection fraction (LVEF), including HF with mildly reduced and preserved ejection fraction (HFmrEF, HFpEF).

The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use in December 2022 and was based on the positive results from the DELIVER Phase III trial.

Results from the prespecified pooled analysis of DELIVER and DAPA-HF Phase III trials also established Forxiga as the first HF medication to demonstrate mortality benefit across the full ejection fraction range.

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “This broader indication for Forxiga for the treatment of symptomatic chronic heart failure across the full ejection fraction range will help more patients to benefit from this well-tolerated and guideline-directed treatment.

We are redefining treatment of cardiorenal diseases with Forxiga’s demonstration of life-saving benefits, underscoring AstraZeneca’s commitment to provide innovative solutions that can help address the complexities of heart failure across the spectrum of the disease.”

HF is a chronic, long-term condition that worsens over time and affects about 15 million people in Europe.

Approximately half of HF patients die within five years of diagnosis and patients with HFmrEF and HFpEF are not only at greater risk of death and hospitalisations but experience an especially high burden of symptoms and physical limitations, and a poor quality of life.

HFmrEF and HFpEF are also severely underdiagnosed as signs and symptoms are often nonspecific and overlapping with other clinical conditions.

These conditions are frequently complicated by multiple interrelated diseases, specifically coronary heart disease, obesity, diabetes, long-standing hypertension, and chronic kidney disease (CKD), highlighting the importance of risk management for patients with this complex syndrome.

Forxiga (known as Farxiga in the US) is approved for the treatment of patients with type-2 diabetes (T2D), HFrEF and CKD in more than 100 countries around the world including the US, the EU, China and Japan.It has most recently received regulatory approvals in Great Britain, Japan and Turkey to extend the HF indication to include patients across the full spectrum of LVEF.

The HF indication extension application is currently under review in the US and other countries.”

https://www.astrazeneca.com/media-centre/press-releases/2023/forxiga-approved-in-the-eu-for-the-treatment-of-symptomatic-chronic-heart-failure.html

Sandoz BLA for proposed biosimilar denosumab accepted by US FDA

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Sandoz BLA for proposed biosimilar denosumab accepted by US FDA

February 06, 2023: “Sandoz, a global leader in off-patent (generic and biosimilar) medicines announced that the US Food and Drug Administration has accepted its Biologics License Application (BLA) for proposed biosimilar denosumab.

The application includes all indications covered by the reference medicines Prolia® (denosumab)* and Xgeva® (denosumab)* for treating a variety of conditions, including osteoporosis in postmenopausal women and in men at increased risk of fractures, treatment-induced bone loss, prevention of skeletal related complications in cancer that has spread to the bone, giant cell tumor of the bone, and treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy.
        
“In addition to being an important medicine for cancer of the bone, denosumab is critical in the treatment of osteoporosis and potential prevention of osteoporosis-related fractures that so many women over 50 are at risk of,” said Keren Haruvi, President, Sandoz Inc. and Head of North America.

“We are proud to be among the first to submit a BLA for a denosumab biosimilar as, if approved, it could increase patient access to an affordable, high-quality, potentially disease-modifying treatment across the US, while also delivering savings for healthcare systems.”

In the US alone, more than 10 million adults over age 50 are estimated to have osteoporosis, of whom more than 80% are women.

It is predicted that one in two of these women and one in four men will have an osteoporosis-related fracture in their lifetimes.

Osteoporosis-related fractures may lead to diminished quality of life, disability, and even death.

The BLA includes a comprehensive analytical and clinical data package, including data from the Phase I/III ROSALIA study.

Results confirmed that the proposed biosimilar denosumab matches the reference medicine in terms of pharmacokinetics, pharmacodynamics, efficacy, safety and immunogenicity in women with postmenopausal osteoporosis; and contributes to demonstration of similarity, which is the basis for use in all indications.

Sandoz biosimilars help patients, in areas including immunology, oncology, supportive care and endocrinology, access critical and potentially life-changing medicines sustainably and affordably.

Sandoz has a leading global portfolio with eight marketed biosimilars and a further 15-plus in various stages of development.

About denosumab
Denosumab is a human monoclonal antibody designed to bind to the RANKL protein, an activator of osteoclasts (cells involved in breaking down bone tissue).

By binding to and inhibiting RANKL, denosumab decreases the production and activity of osteoclasts, resulting in a reduction of bone loss, and subsequently the likelihood of fractures and other serious bone conditions.”

https://www.novartis.com/news/media-releases/sandoz-biologics-license-application-proposed-biosimilar-denosumab-accepted-us-fda

Tezspire approved for self-administration in US with a new pre-filled pen

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Tezspire approved for self-administration in US with a new pre-filled pen

February 02, 2023: “AstraZeneca and Amgen’s Tezspire (tezepelumab) has been approved in the US for self-administration in a pre-filled, single-use pen for patients aged 12 years and older with severe asthma. 

Tezspire is the only biologic approved for severe asthma with no phenotype (e.g. eosinophilic or allergic) or biomarker limitation within its approved label.

The approval by the US Food and Drug Administration (FDA) was based on results from the PATHFINDER clinical trial programme, which included results from the PATH-BRIDGE Phase I trial and the PATH-HOME Phase III trial.

The majority (92%) of healthcare providers, patients and caregivers were able to successfully administer Tezspire both in the clinic and at home throughout the PATH-HOME trial.

The improvements in asthma control and the safety profile of Tezspire observed in the PATH-HOME trial were consistent with previous clinical trials.

Kenneth Mendez, President and CEO of the Asthma and Allergy Foundation of America, said: “Severe asthma continues to be a very complex condition to manage, so we welcome the Tezspire pre-filledpen as an option that will empower patients and healthcare providers with increased choice.

We believe self-administration alternatives can play an important role in patients’ lives and address unmet needs for those living with severe asthma.”

Ruud Dobber, Executive Vice President and President, BioPharmaceuticals Business Unit, AstraZeneca, said: “Tezspire is the first and only biologic approved for patients with severe asthma with no phenotype or biomarker limitation within its approved label.

With the approval of the pre-filledpenin the US, we can now offer greater flexibility to patients and physicians with the option to administer Tezspire at home or in the clinic.”

Tezspire self-administration and the Tezspire pre-filled pen are also approved in the European Union (EU) and are under regulatory review in several other countries around the world. 

Tezspire is currently approved for the treatment of severe asthma in the US, EU, Japan and other countries.

Notes

Tezspire pre-filledpen
Tezspire will be available as a fixed-dose 210mg subcutaneous injection via a pre-filled, single-use auto-injector (the Tezspire pre-filled pen) or via a pre-filled, single-use syringe (the Tezspire pre-filled syringe). Both are administered every four weeks.

The Tezspire pre-filledpenenables patients and caregivers to self-administer the medicine at home or in clinic via a simple process. The device is fitted with a safety guard and viewing window and has audible clicks at the start and end of the injection to guide patients.

Clinical trials
PATH-HOME was a Phase III multi-centre, open-label, parallel-group trial designed to assess patient, caregiver and healthcare provider-reported functionality and performance of a single-use, pre-filled syringe (PFS) or auto-injector (AI) with a fixed 210mg dose of Tezspire administered subcutaneously every four weeksin a clinic and in an at-home setting in 216 patients aged 12 years and older with severe asthma.

The majority (92%) of healthcare providers, patients and caregivers were able to successfully administer the Tezspire pre-filled penboth in the clinic and at home throughout the trial.

At-home administration of the Tezspire pre-filled pen at weeks 12 and 16 was successful in 97% of the patients or caregivers (102/105).

The trial also demonstrated for the first time that adolescents can successfully administer Tezspire using the two devices.

The very low proportion of device malfunctions (0.9% of PFS and 0.8% of AIs) provides support that the instructions for use provided to healthcare providers, patients and caregivers is adequate for successful subcutaneous administration of Tezspire both in the clinic and at home.

PATH-BRIDGE was a single-centre, randomised, open-label, parallel-group Phase I trial in healthy people to compare the pharmacokinetic (PK) exposure following a single 210mg dose of Tezspire by using a vial-and-syringe (V-S), PFS or pre-filled AI device.

Tezspire PK exposure was comparable following subcutaneous administration via V-S, PFS or AI.

In addition, injection site-pain was low in severity and injection-site reactions were uncommon in all device groups.

In addition to PATH-BRIDGE and PATH-HOME, the PATHFINDER clinical trial programme included the pivotal NAVIGATOR Phase III trial in which Tezspire demonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.

NAVIGATOR was the first Phase III trial to show benefit in severe asthma irrespective of eosinophils by targeting thymic stromal lymphopoietin (TSLP).

These results support the FDA Breakthrough Therapy Designation granted to Tezspire in September 2018 for patients with severe asthma, without an eosinophilic phenotype.

In July 2021, Tezspire was the first and only biologic to be granted Priority Review in the US for the treatment of asthma by the FDA.

Tezspire
Tezspire (tezepelumab) is being developed by AstraZeneca in collaboration with Amgen as a first-in-class human monoclonal antibody that inhibits the action of TSLP, a key epithelial cytokine that sits at the top of multiple inflammatory cascades and is critical in the initiation and persistence of allergic, eosinophilic and other types of airway inflammation associated with severe asthma, including airway hyperresponsiveness.

TSLP is released in response to multiple triggers associated with asthma exacerbations, including allergens, viruses and other airborne particles.

Expression of TSLP is increased in the airways of patients with asthma and has been correlated with disease severity.

Blocking TSLP may prevent the release of pro-inflammatory cytokines by immune cells, resulting in the prevention of asthma exacerbations and improved asthma control.

Tezspire acts at the top of the inflammation cascade and has the potential to help address a broad population of severe asthma patients irrespective of biomarker levels.

Tezspire is approved in the US, EU, Japan and other countries for the treatment of severe asthma.

Tezspire is also in development for other potential indications including chronic obstructive pulmonary disease (COPD), chronic rhinosinusitis with nasal polyps, chronic spontaneous urticaria and eosinophilic esophagitis (EoE).

In October 2021, tezepelumab was granted Orphan Drug Designation by the FDA for the treatment of EoE.

https://www.astrazeneca.com/media-centre/press-releases/2023/tezspire-approved-for-self-administration-in-the-us-with-a-new-pre-filled-pen.html

FDA Approves First Oral Treatment for Anemia Caused by Chronic Kidney Disease

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FDA Approves First Oral Treatment for Anemia Caused by Chronic Kidney Disease

February 01, 2023: “The U.S. FDA approved Jesduvroq tablets (daprodustat) as the first oral treatment for anemia (decreased number of red blood cells) caused by chronic kidney disease for adults who have been receiving dialysis for at least four months. Jesduvroq is not approved for patients who are not on dialysis.

Other FDA-approved treatments for this condition are injected into the blood or under the skin. 

“With an oral drug option in addition to the FDA-approved injection options, adults with chronic kidney disease on dialysis now have multiple ways to treat their anemia,” said Ann Farrell, M.D., director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research.

“This approval demonstrates the FDA’s commitment to helping bring a range of therapeutic options to patients with chronic diseases. Patients can consult with their healthcare providers to select the option that is most appropriate.” 

More than a half million adults in the U.S. have chronic kidney disease requiring dialysis (a treatment that filters the blood and removes excess fluid from the blood).

Kidneys produce a hormone called erythropoietin, which signals the body to make red blood cells.

In a person with chronic kidney disease on dialysis, the kidneys cannot produce enough erythropoietin, leading to reduced numbers of red blood cells. 

Jesduvroq increases erythropoietin levels. The effectiveness of Jesduvroq was established in a randomized study of 2,964 adults receiving dialysis. In this study, adults received either oral Jesduvroq or injected recombinant human erythropoietin (a standard of care treatment for patients with anemia due to chronic kidney disease).

Jesduvroq raised and maintained the hemoglobin (the protein in red blood cells that carries oxygen and is a common measure of anemia) within the target range of 10-11 grams/deciliter, similar to that of the recombinant human erythropoietin. 

Jesduvroq has a boxed warning for an increased risk of thrombotic vascular (blood clotting) events including death, heart attack, stroke, and blood clots in the lungs, legs, or dialysis access site.

Jesduvroq’s warnings and precautions include a risk of hospitalization for heart failure, worsening increase of blood pressure, and stomach erosions and gastrointestinal bleeding. 

Jesduvroq is not approved for patients with anemia due to chronic kidney disease who are not on dialysis because its safety has not been established in that population.

The most common side effects of Jesduvroq include high blood pressure, thrombotic vascular events, abdominal pain, dizziness and allergic reactions. 

Patients should not use Jesduvroq if they also take certain drugs that cause increased levels of Jesduvroq or if they have uncontrolled high blood pressure. 

The FDA granted the approval to GlaxoSmithKline LLC.

https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-treatment-anemia-caused-chronic-kidney-disease-adults-dialysis

Dupixent approved as first and only targeted medicine indicated for eosinophilic esophagitis

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 Jan 30, 2023. The European Commission has expanded the marketing authorization for Dupixent® (dupilumab) in the European Union (EU) to treat eosinophilic esophagitis (EoE) in adults and adolescents 12 years and older, weighing at least 40 kg, who are inadequately controlled by, are intolerant to, or who are not candidates for conventional medicinal therapy.

EoE is a chronic, progressive inflammatory disease that damages the esophagus and prevents it from working properly.

With this approval, Dupixent is the first and only targeted medicine specifically indicated to treat EoE in Europe and the U.S.

Naimish Patel, M.D.  
Head of Global Development, Immunology and Inflammation at Sanofi  
“The impact of EoE on a patient’s daily life cannot be overstated – the narrowing and scarring of the esophagus can make something as simple as eating a painful and distressing experience, and may lead to choking and food impaction. 

With this latest approval for Dupixent, adults and adolescents in the EU suffering from the chronic and often debilitating symptoms of EoE now have the first and only targeted treatment option clinically proven to reduce both esophageal inflammation and damage, as well as improve swallowing ability, pain and health-related quality of life.”

George D. Yancopoulos, M.D., Ph.D. 
President and Chief Scientific Officer at Regeneron 
“This latest approval establishes Dupixent as the only targeted medicine specifically indicated for eosinophilic esophagitis in the European Union.

Dupixent is also the only biologic shown in pivotal trials to help patients achieve histological remission, reduce difficulty swallowing and improve health-related quality of life – all of which are crucial to reducing the burden of this debilitating disease. 

Since its first approval, Dupixent has redefined the treatment of certain chronic diseases with underlying type 2 inflammation and is now indicated for five conditions in the European Union.

We remain committed to investigating Dupixent’s potential in additional diseases in which IL-4 and IL-13 may play a key role.”

The EC decision is supported by 52-week data from a Phase 3 trial consisting of three parts (Part A, B and C).

Part A and Part B investigated Dupixent 300 mg weekly (Part A n=42; Part B n=80) compared to placebo (Part A n=39; Part B n=79) for 24 weeks. Part C (n=188) observed patients who had continued on or switched to Dupixent from Parts A and B for an additional 28 weeks.

Dupixent patients in Parts A and B, respectively, experienced:

  • An approximately 10 times higher rate of histological disease remission (60% and 59%), a co-primary endpoint, compared to placebo (5% and 6%).
  • A 69% and 64% reduction in disease symptoms compared to 32% and 41% with placebo. Disease symptoms were measured using the Dysphagia Symptom Questionnaire (DSQ), on which Dupixent patients experienced a 21.9- and 23.8-point clinically meaningful improvement compared to a 9.6- and 13.9-point improvement for placebo, a co-primary endpoint. Swallowing improvement was observed as early as four weeks.
  • A greater than seven-fold reduction in abnormal endoscopic findings from baseline (-3.2 and -4.5 points) compared to placebo (-0.3 and -0.6 points).
  • Nominally significant improvements in swallowing-related pain and health-related quality of life, as well as less frequent non-swallowing symptoms.

Histological disease remission, swallowing improvement and reduction in abnormal endoscopic findings were consistent with the overall population in patients who were uncontrolled, or not responsive to or not eligible for swallowed topical corticosteroids. Longer term efficacy in Part C was similar to results observed in Parts A and B.

The safety results of the trial were generally consistent with the known safety profile of Dupixent in its approved indications.

The most common side effects across indications include injection site reactions, conjunctivitis, conjunctivitis allergic, arthralgia, oral herpes and eosinophilia. Adverse events more commonly observed in EoE patients treated with Dupixent (n=122) compared to placebo (n=117) included infections (32% vs. 25%).

An additional adverse reaction of injection site bruising was reported in the EoE trial. The safety profile through 52 weeks was generally consistent with the safety profile observed at 24 weeks.

About Eosinophilic Esophagitis

EoE is a chronic, progressive inflammatory disease that damages the esophagus and prevents it from working properly.

The results seen with Dupixent in adults and adolescents with EoE demonstrate that interleukin-4 (IL-4) and interleukin-13 (IL-13) are key and central drivers of the type 2 inflammation underlying this disease. For people with EoE, swallowing even small amounts of food can be a painful and worrisome choking experience.

They are often left to contend with the frustration and anxiety of a constantly evolving list of foods to avoid, a poor quality of life and a higher risk of depression.

In cases where EoE causes the esophagus to narrow, forced and potentially painful dilation (physical expansion) of the esophagus may be needed.

In severe cases, a feeding tube may be the only option to ensure proper caloric intake and adequate nutrition. In the EU, about 50,000 adults and adolescents live with severe uncontrolled EoE.

About the Dupixent Eosinophilic Esophagitis Trial

The three-part Phase 3 randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of Dupixent in patients aged 12 years and older with EoE.

All patients had previously not responded to proton pump inhibitors, and, across Parts A and B, 74% of patients were previously treated with swallowed topical corticosteroids.

At 24 weeks, the co-primary endpoints in Parts A and B assessed patient-reported measures of difficulty swallowing (change from baseline in the DSQ on a 0-84 scale) and esophageal inflammation (proportion of patients achieving histological disease remission, defined as peak esophageal intraepithelial eosinophil count of ≤6 eos/hpf).

Additional endpoints included abnormal endoscopic findings (EoE Endoscopic Reference Score [EoE-EREFS] on a 0-18 scale), swallowing-related pain (DSQ pain score), health-related quality of life (EoE Impact Questionnaire [EoE-IQ]) and frequency of other non-dysphagia symptoms (EoE Symptom Questionnaire [EoE-SQ]).

About Dupixent

Dupixent is an injection administered under the skin (subcutaneous injection) at different injection sites. In the EU for adolescents and adults with EoE, Dupixent is administered at 300 mg every week.

It is available as both a pre-filled pen and pre-filled syringe at the 300 mg dose.

Dupixent is intended for use under the guidance of a healthcare professional and can be given in a clinic or at home by self-administration after training by a healthcare professional.

Dupixent is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant.

The Dupixent development program has shown significant clinical benefit and a decrease in type 2 inflammation in Phase 3 trials, establishing that IL-4 and IL-13 are key and central drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases.

These diseases include approved indications for Dupixent, such as atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis (CRSwNP), prurigo nodularis and EoE.

Dupixent has received regulatory approvals in one or more countries around the world for use in certain patients with atopic dermatitis, asthma, CRSwNP, EoE or prurigo nodularis in different age populations.

Dupixent is currently approved for one or more of these indications in more than 60 countries, including in Europe, the U.S. and Japan. More than 500,000 patients have been treated with Dupixent globally.

Dupilumab Development Program

Dupilumab is being jointly developed by Sanofi and Regeneron under a global collaboration agreement.

To date, dupilumab has been studied across more than 60 clinical trials involving more than 10,000 patients with various chronic diseases driven in part by type 2 inflammation.

In addition to the currently approved indications, Sanofi and Regeneron are studying dupilumab in a broad range of diseases driven by type 2 inflammation or other allergic processes in Phase 3 trials, including pediatric EoE, hand and foot atopic dermatitis, chronic inducible urticaria-cold, chronic spontaneous urticaria, chronic pruritus of unknown origin, chronic obstructive pulmonary disease with evidence of type 2 inflammation, chronic rhinosinusitis without nasal polyposis, allergic fungal rhinosinusitis, allergic bronchopulmonary aspergillosis and bullous pemphigoid.

These potential uses of dupilumab are currently under clinical investigation, and the safety and efficacy in these conditions have not been fully evaluated by any regulatory authority.

Novartis continues to grow with further core margin expansion and achieves important innovation milestones

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Novartis continues to grow with further core margin expansion and achieves important innovation milestones

February 1, 2023: “Commenting on 2022 results, Vas Narasimhan, CEO of Novartis, said: “Novartis is on track to become a pure-play innovative medicines company, uniquely positioned to leverage its global scale and R&D platforms.

Our six multi-billion brands now represent 32% of our Innovative Medicines sales and are growing 26%. Pluvicto and Scemblix had very strong launch performances, and the Leqvio launch continues to progress.

Pivotal Ph3 readouts for two iptacopan studies and Pluvicto in earlier lines of therapy provide strong confidence for near to mid-term growth. Looking ahead, we have a catalyst rich pipeline with 15 pivotal readouts in the mid-term.

We expect to continue to deliver improved financials and strengthen Novartis ESG foundations, on our journey to become most trusted and valued medicines company in the world”.

Key figures1

 Q4 2022Q4 2021% changeFY 2022FY 2021% change
 USD mUSD mUSDccUSD mUSD mUSDcc
Net sales12 69013 229-4350 54551 626-24
Operating income1 9492 562-24-149 19711 689-21-13
Net income1 46616 306-91-906 95524 018-71-67
EPS (USD)0.697.29-91-893.1910.71-70-66
Free cash flow3 5523 02717 11 94513 282-10 
Core operating income4 0303 81961516 66516 58808
Core net income3 2513 13541413 35214 094-53
Core EPS (USD)1.521.409196.126.29-36

1 Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 50 of the Condensed Financial Report. Unless otherwise noted, all growth rates in this Release refer to same period in prior year. 

A table showing the Q4 2022 and FY 2022 key figures excluding Roche can be found on page 8 and a reconciliation of 2021 IFRS results and non-IFRS measures core results to exclude the impacts of the 2021 divestment of our Roche investment can be found on page 58 of the Condensed Financial Report. 

As per December 31, 2022.   Please see detailed guidance assumptions on page 6.  Potential USD sales.

Strategy Update

Our focus

During 2022, Novartis unveiled a new focused strategy with our transformation into a “pure-play” Innovative Medicines business. We have a clear focus on five core therapeutic areas (cardiovascular, immunology, neuroscience, solid tumors and hematology), with multiple significant in-market and pipeline assets in each of these areas, that address high disease burden and have substantial growth potential.

In addition to two established technology platforms (chemistry and biotherapeutics), three emerging platforms (gene & cell therapy, radioligand therapy, and xRNA) are being prioritized for continued investment into new R&D capabilities and manufacturing scale. Geographically, we are focused on growing in our priority geographies – the US, China, Germany and Japan.

Our priorities

  1. Accelerate growth: Renewed attention to deliver high-value medicines (NMEs) and focus on launch excellence, with a rich pipeline across our core therapeutic areas.
  2. Deliver returns: Continuing to embed operational excellence and deliver improved financials. Novartis remains disciplined and shareholder-focused in our approach to capital allocation, with substantial cash generation and a strong capital structure supporting continued flexibility.
  3. Strengthening foundations: Unleashing the power of our people, scaling data science and technology and continuing to build trust with society.

Sandoz planned spin-off

The planned spin-off remains on track for H2 2023. Completion of the transaction is subject to certain conditions, including consultation with works councils and employee representatives (as required), general market conditions, tax rulings and opinions, final Board of Directors endorsement and shareholder approval in line with Swiss corporate law.

The transaction is expected to be tax neutral to Novartis.

Financials

Fourth quarter

Net sales were USD 12.7 billion (-4%, +3% cc) in the fourth quarter driven by volume growth of 10 percentage points, partly offset by price erosion of 3 percentage points and the negative impact from generic competition of 4 percentage points.

Operating income was USD 1.9 billion (-24%, -14% cc), mainly due to higher restructuring costs (USD 0.6 billion), primarily related to the implementation of the previously announced streamlined organizational model.

Net income was USD 1.5 billion (-91%, -90% cc), impacted by Roche income in the prior year of USD 14.6 billion. Excluding the impact of Roche income, net income grew +2% (cc). EPS was USD 0.69 (-91%, -89% cc). Excluding the impact of Roche income, EPS grew +7% (cc).

Core operating income was USD 4.0 billion (+6%, +15% cc) driven by higher sales and productivity, including initial savings from the previously announced streamlined organizational model. Core operating income margin was 31.8% of net sales, increasing by 2.9 percentage points (+3.5 percentage points cc).

Core net income was USD 3.3 billion (+4%, +14% cc), mainly driven by growth in core operating income, partly offset by the loss of Roche core income. Excluding the impact of Roche core income, core net income grew +17% (cc). Core EPS was USD 1.52 (+9%, +19% cc), benefiting from lower weighted average number of shares outstanding. Excluding the impact of Roche core income, core EPS grew +23% (cc).

Free cash flow amounted to USD 3.6 billion (+17% USD), mainly driven by higher net cash flows from operating activities and lower purchases of intangible assets.

Innovative Medicines net sales were USD 10.4 billion (-3%, +3% cc) with volume contributing 11 percentage points to growth, mainly driven by continued strong performance from Entresto, Kesimpta, Pluvicto and Kisqali. Generic competition had a negative impact of 5 percentage points, mainly due to Gilenya, Exjade and Afinitor.

Pricing had a negative impact of 3 percentage points, including approximately 1 percentage point impact from a revenue deduction true-up for Cosentyx in the US, which was related to prior quarters in 2022.

Sales growth for the quarter was also negatively impacted by the prior year reclassification of contract manufacturing from other revenues to sales.

Excluding the contract manufacturing reclassification impact, sales would have grown +4% (cc). Sales in the US were USD 4.2 billion (+7%) and in the rest of the world USD 6.2 billion (-9%, +1% cc).”

https://www.novartis.com/news/media-releases/novartis-continues-grow-further-core-margin-expansion-and-achieves-important-innovation-milestones

FDA Proposes Redesign of Human Foods Program to Enhance Coordinated Prevention and Response Activities

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FDA Proposes Redesign of Human Foods Program to Enhance Coordinated Prevention and Response Activities

January 31, 2023: “As the oldest comprehensive consumer protection agency in the country, for more than a century the U.S. FDA has been responsible for ensuring the safety of food products consumed by hundreds of millions of Americans each day while also advancing nutrition.

It is our mission to ensure that our programs are organized in a manner that will protect and promote public health.

The agency has carefully reviewed the findings and recommendations from an external evaluationExternal Link Disclaimer conducted by an expert panel of the Reagan-Udall Foundation that I requested and a separate internal review of the agency’s infant formula supply chain response completed last year.

The findings and recommendations from these reviews identified issues surrounding culture, structure, resources, and authorities.

They also noted several areas of need, including modernizing data systems, providing more resources and authorities, improving emergency response systems, and building a more robust regulatory program.

Today I am announcing a new, transformative vision for the FDA Human Foods Program. I am also announcing a transformative vision for the Office of Regulatory Affairs (ORA, the FDA’s field-based operations) to support the FDA organization as a whole.

The proposed structures for both groups will have clear priorities that are focused on protecting and promoting a safe, nutritious U.S. food supply that more quickly adapts to an ever-changing and evolving environment.

I believe this proposed approach addresses the recommendations outlined in both reports, and takes into consideration feedback from stakeholders, as well as the voices of employees working in the Human Foods Program who had an opportunity to share input through numerous interactive and listening sessions over the past month.

Creating a Human Foods Program under a single leader who reports directly to the Commissioner unifies and elevates the program while removing redundancies, enabling the agency to oversee human food in a more effective and efficient way.

Under this plan, the functions of the Center for Food Safety and Applied Nutrition (CFSAN), Office of Food Policy and Response (OFPR), as well as certain functions of ORA will be unified into a newly envisioned organization called the Human Foods Program.

The FDA will conduct a competitive national search for a Deputy Commissioner for Human Foods, who will oversee the Program.

The person in this position will report directly to me and will be charged with leading a unified Human Foods Program that keeps the foods we regulate safe and nutritious, while ensuring the agency remains on the cutting edge of the latest advancements in science, technology, and nutrition.

The Deputy Commissioner will have decision-making authority over policy, strategy, and regulatory program activities within the Human Foods Program, as well as resource allocation and risk-prioritization.

Other key elements of the proposed new Human Foods Program organization include:

  • Creation of a Center for Excellence in Nutrition that prioritizes the agency’s ongoing efforts to help American consumers make more informed food choices, including by working with industry to offer healthier, more nutritious food products.
    The FDA proposes to establish an Office of Critical Foods, as directed by the 2023 Consolidated Appropriations Act, within this center.  
  • Establishment of an Office of Integrated Food Safety System Partnerships that will focus on elevating, coordinating and integrating our food safety and response activities with state and local regulatory partners to more effectively meet the vision of an Integrated Food Safety System as envisioned in the FDA Food Safety Modernization Act of 2011.
    This newly proposed structure will ensure greater collaboration and support of state-level inspectional activities.
    We know that we cannot be everywhere, at all times, and our relationships with our state and local regulatory partners will be more important than ever going forward. 

To help support the agency’s scientifically grounded decision-making activities, a Human Foods Advisory Committee will be established. Advisory Committees are commonly used to obtain independent expert advice on various issues.

The Human Foods Advisory Committee will consist of external experts to advise on challenging and emerging issues in food safety, nutrition and innovative food technologies.

Finally, there will be an emphasis on strengthening our enterprise information technology and analytical capabilities to fulfill the promise described in the New Era of Smarter Food Safety and support the improvement in workflow that will accompany these changes.

This area of focus will support the work of the Human Foods Program by enabling more facile communication, more efficient operations and enhanced empirical risk algorithms to guide the priorities of the program and the work in the field.

As part of this proposed new vision, ORA’s operating structure will be transformed into an enterprise-wide organization that supports the Human Foods Program and all other FDA regulatory programs (e.g., agency centers) by focusing on its critical activities.

This realignment will allow ORA to be singularly focused on excellence in its core mission – inspections, laboratory testing, import, and investigative operations. This will optimize ORA’s operations in line with the FDA’s public health and prevention-oriented goals.

Certain other functions of ORA will be aligned in other parts of the FDA to create an overall stronger agency.

While the FDA’s Center for Veterinary Medicine (CVM) will continue to operate as a stand-alone center, the relevant food safety activities will be closely coordinated between the CVM Center Director and Deputy Commissioner for Human Foods.

This proposed structure will allow CVM to support the Human Foods Program where its activities are relevant to human food safety. 

The FDA has recently formed an Implementation and Change Management Group that will be charged with developing a detailed plan to ensure the successful execution of this vision.  

Consumers can be confident in the safety of the food they eat each day in part thanks to the work of the FDA’s dedicated workforce.

Our ability to continue our work means consistently evolving and adapting with the constantly changing, complex industries we regulate and the emergence of new technologies.

As a next step, the FDA will need to develop the vision announced today into a concrete reorganizational proposal in close coordination and communication with internal and external stakeholders while ensuring we meet our labor obligations.

While details of this proposal continue to be developed, CFSAN, ORA, and OFPR will continue to operate under their current structures, with my direct oversight.

I look forward to providing additional public updates by the end of February on our progress, organizational design and timeline.”

https://www.fda.gov/news-events/press-announcements/fda-proposes-redesign-human-foods-program-enhance-coordinated-prevention-and-response-activities

Sandoz announces agreement to acquire leading antifungal agent Mycamine® from Astellas

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Sandoz announces agreement to acquire leading antifungal agent Mycamine® from Astellas

January 24, 2023: “Sandoz, the global leader in off-patent (generic and biosimilar) medicines, has signed an agreement to acquire worldwide product rights for leading systemic antifungal agent Mycamine® (micafungin sodium, Funguard® in Japan) from Astellas.

Closing is anticipated in the course of H1, 2023, subject to standard conditions and regulatory approvals.

Astellas reported Mycamine® sales of JPY 18.9 billion (USD 135 million) for the year ending March 31, 2022.

The announcement comes after Sandoz successfully completed the acquisition of GSK’s global cephalosporins portfolio in October 2021.

Sandoz CEO Richard Saynor said: “Acquiring this leading and respected global brand will significantly reinforce the Sandoz global hospital offering, as well as complement our existing global leadership position in generic antibiotics.”

He added: “This will also be an important addition to our growing portfolio of anti-infective therapies aimed at combatting the spread of antimicrobial resistance, by providing the right drug to the right patient at the right time.”

Sandoz Anti-Infectives global medical lead Nicholas Adomakoh said: “Modern medicine is increasingly characterized by the need for complex interventions involving highly vulnerable patients.

This welcome and timely portfolio addition will allow us to respond even better to the needs of patients and clinicians across the healthcare continuum.”

Mycamine®   is a leading global echinocandin, one of three major classes of antifungal agents, with a global patient base of well over two million.

It   is a therapy of choice in hospitals and intensive care units worldwide, a proven prophylactic in hematology and oncology patients, and widely used in organ transplants.

Mycamine®   is indicated for treatment of invasive candidiasis and espophageal candidiasis, which are currently both on the rise with a higher occurrence of associated hospital outbreaks, as well as prevention of candida and aspergillus infections in patients undergoing hematopoietic stem cell transplantation.”

https://www.novartis.com/news/media-releases/sandoz-announces-agreement-acquire-leading-antifungal-agent-mycamine-from-astellas-reinforcing-hospital-offering-and-leading-anti-infectives-portfolio

Bayer acquires Blackford Analysis Ltd. bolstering the company’s position in digital medical imaging

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Bayer acquires Blackford Analysis Ltd. bolstering the company’s position in digital medical imaging

January 18, 2023: ‘Bayer announced the acquisition of the global strategic imaging AI platform and solutions provider Blackford Analysis Ltd.

The acquisition is part of Bayer’s strategy to drive innovation in radiology, including the development and adoption of AI within the clinical workflow, with the goal to ultimately improve patient care and advance Bayer’s position in digital medical imaging.

“Adding Blackford and its AI technology to our radiology portfolio secures Bayer an excellent position in the fastest growing segment within the overall global radiology industry,” said Stefan Oelrich, Member of the Board of Management, Bayer AG and President of Bayer’s Pharmaceutical Division.

“This acquisition complements our comprehensive radiology portfolio and nourishes our engagement to drive innovation in digital health.

We are pleased to join forces with Blackford and their exceptional team to optimally utilize our combined expertise in healthcare technology with the aim to deliver true value to radiologists and their teams for the benefit of their patients.”

Blackford, which has a presence in the United Kingdom and the United States, provides infrastructure and access to a rich clinical application (ClinApp) ecosystem focused on imaging and analytics.

The acquisition follows a development and license agreement between both companies in 2020 that laid the foundation for Bayer’s recently launched medical imaging platform, Calantic™ Digital Solutions.

Building on technology from Blackford and adding additional workflow and analytics components, Calantic Digital Solutions delivers access to applications, including those enabled by AI, for medical imaging.

Blackford will continue to operate as an independent organization on an arm’s length basis to preserve its entrepreneurial culture as an essential pillar for nurturing successful innovation.

The company will remain accountable to advance its technology, channel partnerships and ClinApp portfolio while benefiting from the experience, infrastructure and reach of Bayer as a global pharmaceutical company.

The acquisition is expected to close later this year, pending the satisfaction of customary closing conditions.

“Blackford exists to improve the lives of patients and populations by unlocking the adoption and benefits of medical imaging AI. 

We investigated many routes to scale our business to deliver our mission and were delighted by Bayer’s invitation to deepen our partnership, whilst continuing to operate independently based on Bayer’s well-established arm’s length model,” said Ben Panter, Chief Executive Officer, Blackford Analysis.

“Combining our knowledge and experience as one of the leading platform providers in the industry with Bayer’s advanced radiology portfolio will enable us to provide solutions to deliver ongoing clinical value to radiologists and their teams.”

The overall global medical imaging AI field with sales of more than USD 400 million in 2021 is expected to continue growing dynamically, with an estimated compounded annual growth rate of more than 26 percent (2020 to 2026) reaching USD 1.36 billion by 2026. Innovation powered by AI is needed more than ever.

Aging populations and changing lifestyles are leading to an increase in chronic conditions, such as cardiovascular disease and cancer.

Consequently, the demand for medical imaging to detect diseases, guide treatment decisions and support therapy planning is growing. AI comes with the value proposition to aid diagnosis and increase the throughput of radiological examinations.

About Radiology at Bayer
As a true life-science company with a heritage of over 100 years in Radiology, Bayer is committed to providing excellence, from innovative products to high-quality services to support efficient and optimized patient care.

Bayer is offering a comprehensive portfolio of contrast media for computed tomography (CT), X-Ray, and magnetic resonance imaging (MRI), devices for their precise administration, informatics solutions as well as a medical imaging platform delivering access to applications, including those enabled by AI. 

Altogether, the Radiology products from Bayer generated €1.8 billion sales in 2021.

Following the company’s ambition to outperform the radiology segment’s average annual growth of 5% until 2030, Bayer is strongly committed to research and development, which includes using artificial intelligence and driving innovation in medical imaging.

Each of these offerings provide effective tools to support radiologists in their mission to deliver treatment critical answers and a clear direction – from diagnosis to care.

About Blackford Analysis
Blackford provides tailored tools and services to unlock the value of imaging AI, drive efficiencies and improve patient outcomes.

For over a decade, Blackford has worked in partnership with leading healthcare organizations and ground-breaking technology providers to integrate best-fit AI into clinical workflows to realize ongoing value.

Partnering with Blackford provides healthcare organizations access to a wide portfolio of AI solutions via a single tried-and-tested platform.

The collaboration with Bayer has its roots in 2019, when Blackford was among the selected start-ups of Bayer’s G4A Digital Health Partnerships Program that year.”

https://www.bayer.com/media/en-us/bayer-acquires-blackford-analysis-ltd-bolstering-the-companys-position-in-digital-medical-imaging/

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