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Bayer Pharmaceuticals accelerates innovations for patients

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Bayer Pharmaceuticals accelerates innovations for patients

March 24,2023: “At its annual Pharma Media Day 2023, Bayer presented the latest progress in the ongoing transformation of its pharmaceutical business focusing on accelerating innovative healthcare solutions for the benefits of patients and delivering long-term, sustainable business growth.

“The transformation of our Pharma business is progressing well. With bold investments in industry-leading technology platform companies, we have bolstered our R&D capabilities and strengthened our early development pipeline,” said Stefan Oelrich, Member of the Board of Management, Bayer AG and President of Bayer’s Pharmaceuticals Division. “Going forward, we will focus on realizing the full potential of our projected blockbuster candidates Nubeqa™, Kerendia™, asundexian and elinzanetant.

We will specifically allocate resources to grow our oncology and cardiology businesses in the U.S. The implementation of our strategy aims to deliver new medicines to patients faster and drive sustainable growth across our key areas of focus.”

During this year’s Pharma Media Day 2023, Bayer showcased how its investments in strategic technology platforms accelerate its medical and digital innovations and will advance the company’s leadership ambitions in cardiology, oncology, and radiology.

In addition, Bayer provided insights into its innovation strategy, comprising enhanced research and development (R&D) capabilities in key areas.

“Patient needs, precision therapies to address disease complexity as well as advances in science and technology are evolving rapidly.

Therefore, we are evolving our R&D strategy and operating model to focus on differentiated, high-value breakthrough medicines,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development.

“Moving forward, we will focus our early research activities on core strategic areas: oncology, cardiovascular, neurology & rare diseases and immunology, where we believe we can make the biggest possible difference for the patients we serve.”

Delivering significant growth through a leadership position in oncology

The company has doubled its portfolio of oncology treatments over the past five years. With Nubeqa (darolutamide) as a key growth driver it is gearing up to become a leader in prostate cancer, the second most commonly diagnosed cancer in men.

Nubeqa has the potential to become the standard of care for prostate cancer patients from the early- to the late-stage of this disease.

Going forward, growth of Nubeqa will be driven by recent approvals for the treatment of metastatic hormone-sensitive prostate cancer (mHSPC) in the U.S., EU, Japan, and China.

In parallel, the robust Phase III clinical development program with darolutamide aims to broaden its use from the metastatic into the earlier disease stages, e.g. in patients with hormone-sensitive prostate cancer with high-risk of biochemical recurrence and as an adjuvant treatment for patients with localized prostate cancer with very high risk of recurrence.

The company has projected a peak sales potential for Nubeqa of more than three billion euros.

“With Nubeqa’s potential to set a new standard of care in prostate cancer, its compelling clinical program across the spectrum of prostate cancer and our growing early pipeline, we are well placed to deliver on our commitment to bring innovative medicines to cancer patients,” said Christine Roth, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of the Oncology Strategic Business Unit.

“Bayer has clear ambitions to become a top oncology company. We are investing in innovative treatments and modalities that have the potential to be transformative for a broad range of cancer patients, who currently have little or no treatment options.”

Bayer is advancing its oncology R&D efforts in three scientific areas that have the potential to address unmet needs in cancer patients.

This includes targeted radiopharmaceuticals (specifically targeted alpha therapies), next-generation immuno-oncology and precision molecular oncology approaches.

In these areas, the company is advancing truly first-in-class, highly innovative projects in its portfolio. Nearly one third of new molecular entities within Bayer’s development pipeline is dedicated to fighting cancer, spanning all phases of clinical development.

With recent major investments, Bayer has fortified its oncology innovation engine substantially.

The new $140 million Bayer Research and Innovation Center (BRIC) facility, located in the center of thriving Boston-Cambridge, Massachusetts, is home to Bayer’s precision molecular oncology research group, fostering collaborations in this outstanding U.S. innovation hub, including with the Broad Institute of MIT and Harvard.

Vividion Therapeutics, which Bayer acquired for $1.5 billion, comprises a leading chemoproteomics platform capable of identifying previously unknown binding pockets in undruggable targets to generate first-in-class novel drug candidates in indications of high unmet medical need.

This technology and the unique approach of an innovative arms-length operating model have the potential to accelerate the timelines to get assets into the clinic.

For the first two programs, which are addressing high profile cancer targets NRF2 and STAT3, Vividion is expected to file the Investigational New Drug Application (IND) in the U.S. in 2023.

Developing the next generation of cardiovascular therapies

The short- to medium-term growth in the area of cardiovascular (CV) diseases will be driven mainly by Kerendia (finerenone), a non-steroidal, selective mineralocorticoid receptor antagonist, representing a valuable treatment option for patients with cardio-renal disease.

Kerendia is already approved in major markets in chronic kidney disease (CKD) associated with type 2 diabetes.

Bayer has exclusive worldwide marketing rights for this product including the U.S.

The ongoing development program to expand Kerendia into additional indications comprises three Phase III studies: FINEARTS-HF, which investigates the compound in patients with symptomatic heart failure with preserved ejection fraction; FIND-CKD, with the goal to make the medicine available also to patients with non-diabetic CKD etiologies including hypertension and chronic glomerulonephritis (inflammation of the kidneys); and FIONA, which investigates finerenone in pediatric patients with CKD and severely increased proteinuria.

Kerendia is estimated to generate peak sales of more than three billion euros.

Bayer’s investigational treatment of cardiovascular diseases asundexian is currently in Phase III clinical development.

Asundexian is an oral Factor XIa (FXIa) small molecule inhibitor being investigated in patients with atrial fibrillation (AF) as well as in patients with an acute non-cardioembolic ischemic stroke or high-risk transient ischemic attack (TIA), a temporary period of symptoms similar to those of a stroke.

The respective Phase III clinical trial program, OCEANIC, which has recently started enrolling patients, is one of the largest conducted in the company’s history. Asundexian, which belongs to a new class of drugs, has a projected peak sales potential of more than five billion euros.

“Cardiovascular diseases can have a significant impact on patients’ lives. We are advancing highly innovative projects in our portfolio to address patients’ high unmet medical needs and shape the future in cardiology,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development.

“The number of innovative approaches and novel modalities in our portfolio will triple in future. With those we will be able to target pathologies and disease mechanisms that were not druggable before.”

Building on its strong late-stage projects, the company has set a clear directive towards precision cardiology, exploring innovative approaches across a diverse range of modalities, with the aim of pushing the boundaries of innovation for patients with cardiovascular diseases with high unmet needs and potentially reversing life-altering conditions.

Complementing in-house expertise with external collaborations remains a vital part of Bayer’s strategy to expand its R&D expertise and develop robust cardiovascular therapies, with the goal of addressing high unmet medical needs across broad indications, including specialty cardiology and rare indications.

Through Asklepios BioPharmaceutical Inc. (AskBio), Bayer is developing a gene therapy as a potential new approach for treating congestive heart failure.

With the acquisition of AskBio, Bayer gained access to an industry-leading gene therapy platform with demonstrated applicability and world-class manufacturing facilities.

Beyond congestive heart failure, AskBio is also currently advancing four clinical-stage and a number of preclinical candidates addressing medical need indications across a diverse set of diseases such as Parkinson’s disease, Pompe disease and Huntington’s disease.

Bayer’s advancements in cardiovascular disease are enabled by digitalized, decentralized and more diverse clinical trials, as well as partnerships on industry-leading sources of data, such as the Accelerating Medicines Partnership in Heart Failure and the Broad Institute’s Precision Cardiology Laboratory.

These initiatives are helping to accelerate the discovery of new molecules and bring new treatments to patients that need them.

Leveraging digital innovation to support diagnosis and drug discovery

Bayer is implementing its innovation strategy in radiology, with a key focus on digital medical imaging and artificial intelligence (AI). The medical imaging AI market is the fastest growing segment within the global radiology industry.

The estimated compounded annual growth rate is more than 26 percent reaching $1.36 billion in sales by 2026. 

A cornerstone of Bayer’s ambition is its recently launched platform Calantic™ Digital Solutions, which delivers access to digital applications, including those powered by AI.

It provides an ecosystem of third-party and Bayer products to deliver disease-oriented solutions for radiologists and their teams. The platform is designed to support healthcare professionals at various stages of their work, from diagnosis through treatment of their patients.

To bolster its position in the medical imaging AI market, Bayer recently acquired the global strategic imaging AI platform and solutions provider Blackford Analysis Ltd, which will operate under Bayer’s well established “arm’s length” operating model.

Utilizing Blackford’s and Bayer’s combined expertise in healthcare technology, this acquisition fosters Bayer’s engagement to drive digital innovation in healthcare, with the aim to deliver true value to radiologists and their teams for the benefit of their patients.

“Building on a deep medical expertise across a multitude of diagnostic and therapeutic areas, Bayer is a leader in key radiology segments with a comprehensive portfolio that generated close to 2 billion euros sales in 2022,” said Gerd Krueger, President Radiology, Bayer.

“Expanding our offerings into the field of AI further advances our strong position in this market. It follows our mission to deliver disease-oriented solutions for patients and their physicians.

The transformative expansion of our radiology business is a key element of Bayer’s accelerated digital transformation, which strategically leverages data science and advanced analytics across the value chain.”

Bayer is investing to leverage the power of data and rapidly evolving technologies such as artificial intelligence throughout the entire pharma value chain, from discovery to production.

The company is partnering with technology leaders such as Google Cloud and biotech leaders such as Recursion Pharmaceuticals, Inc. to access new areas of innovation.

In addition, Bayer has built up extensive in-house expertise to transform its development, manufacturing and medical operations to better serve the needs of patients, healthcare professionals and healthcare systems with relevant innovative solutions. Bayer works on leveraging all the possibilities of the technologies while fully integrating all regulatory and bioethical requirements.”

https://www.bayer.com/media/en-us/bayer-pharmaceuticals-accelerates-innovations-for-patients/

Macomics and Ono Pharmaceutical partner to discover Cancer therapy

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Macomics and Ono Pharmaceutical partner to discover Cancer therapy

March 23, 2023: “Macomics Ltd, a leader in macrophage drug discovery, announces that it has entered into a worldwide drug discovery collaboration agreement with Ono Pharmaceutical Co., Ltd. (Osaka, Japan; President and CEO: Gyo Sagara; “Ono”) to develop new immuno-oncology antibody drugs against a novel macrophage target of interest in cancer.

Under the terms of the agreement, Macomics will identify and characterise antibody candidates against the novel target of interest using its ENIGMACTM macrophage drug discovery platform.

Ono will have an exclusive option to license global rights to the candidates for further development and commercialization.

Macomics will receive an up-front payment, R&D funding, and success-based milestone payments, as well as tiered royalties based on global net sales.

Macomics is exploiting the potential of macrophage-based approaches to develop novel precision medicines to target disease specific macrophage biology.

Macomics’ ENIGMAC macrophage drug discovery platform integrates large volume human data sets, custom cell models, and proprietary human macrophage genome editing capability to discover novel targets and unlock disease specific target biology.

Macrophages (TAMs) are often the most abundant immune cell many types of cancer and modulating TAMs can enhance the body’s ability to fight cancer.

Toichi Takino, Senior Executive Officer / Executive Director, Discovery & Research of Ono said:

“Targeting macrophages in immune-oncology is emerging as an exciting area with significant opportunity to deliver novel therapeutics to improve cancer outcomes and to change the lives of patients with cancer. Macomics has demonstrated the power of its macrophage platform and drug discovery approach and we are delighted to partner with them on taking this novel target discovery program forwards.”

Stephen Myatt, CEO of Macomics said:

“This global collaboration with Ono is testament to our strong program portfolio, and the unique enablement offered by our ENIGMAC discovery platform, and our world class R&D team. Ono is a leader in immuno-oncology and we are delighted to have a partner in Ono who brings the complementary skills necessary to succeed in this therapeutic area.”

https://macomics.com/2023/03/23/macomics-and-ono-pharmaceutical-partner-to-discover-and-develop-macrophage-targeting-antibody-therapy/

Debiopharm announces launch of galuci study for its Caix-Targeted radiopharmaceutical program

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Debiopharm announces launch of galuci study for its Caix-Targeted radiopharmaceutical program

MARCH 22, 2023: ” Debiopharm, a Swiss-based, global biopharmaceutical company, aiming to establish tomorrow’s standard-of-care to cure cancer and infectious diseases announced the first patient dosed of their first-in-human, phase 1/2 study, GaLuCiTM.

The first patient was screened and dosed at the Australian-based Peter MacCallum Cancer Centre.

This multicenter international trial, evaluating a radioligand theranostic pair will be carried out in three stages: Part A to confirm the safety and reliability of Debio 0328 in detecting CAIX-expressing solid tumors, Part B to assess escalating doses of the therapeutic agent, Debio 0228 in patients, whose tumors show high uptake of Debio 0328 and finally, based on the recommended dose from part B, Part C will further assess safety and preliminary efficacy in selected tumor types.

Currently, Debio 0228/0328 is the only peptide-based theranostic pair targeting CAIX in clinical development, with pan-tumor potential, and developed first for patients with advanced cancers such as renal, pancreatic, and colorectal.

It leverages a theranostic approach to identify and deliver radiation to diseased tissues, allowing the imaging-based pre-identification of patients who have the target proteins necessary to respond to the targeted radioligand.

“The results of the GaLuCiTM trial are highly anticipated considering the therapeutic potential of Debio 0228 as observed in preclinical models.

Using this theranostic pair could pave the way for personalized nuclear medicine, enabling administration of the lutetium coupled radioligand only to patients who are more likely to respond to the therapy.” explained Angela Zubel, Chief Development Officer at Debiopharm.

“We always have immense gratitude for our patients who participate in first time in human trials, but in this case, we are particularly thankful for our patient who agreed to be the first person in the world to have their kidney cancer imaged with Debio 0328 on the GaLuCiTM trial. 

We hope this is the beginning of the theranostics era in kidney cancer!” expressed Dr. Ben Tran, Lead Genito-urinary medical oncologist, Peter MacCallum Cancer Centre. 

“We are excited about this first-in-human study as it is a novel approach for advanced kidney cancer patients,” said Darren R. Feldman, MD, Associate Attending Physician, Genitourinary Oncology Service at Memorial Sloan Kettering Cancer Center. 

“Precision nuclear medicine applied to CA9 could benefit advanced cancer patients who still experience a high unmet medical need. This theranostic pair allows targeted radiation delivery to the cancer cells bearing CAIX, which is largely expressed, over 85%, in clear cell renal cell carcinoma.”  

https://www.debiopharm.com/drug-development/press-releases/debiopharm-announces-launch-of-the-phase-1-2-galucitm-study-for-its-caix-targeted-radiopharmaceutical-program/

AZ’s Eplontersen shpows benefit for hereditary transthyretin-mediated amyloid polyneuropathy through 66 weeks

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AZ’s Eplontersen shpows benefit for hereditary transthyretin-mediated amyloid polyneuropathy through 66 weeks

March 27, 2023: “Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed eplontersen met its co-primary endpoints through 66 weeks.

The results were consistent with the positive 35-week findings announced in June 2022.

At 66 weeks, patients treated with eplontersen continued to demonstrate a statistically significant and clinically meaningful change from baseline versus an external placebo group on the co-primary endpoints of modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN).

The trial also met its third co-primary endpoint demonstrating a statistically significant reduction in serum TTR concentration versus an external placebo group. TTR reductions were consistent with those reported at week 35.

Eplontersen continued to demonstrate a safety and tolerability profile consistent with that observed at 35 weeks.

Sami Khella, M.D., Chief, Department of Neurology, Penn Presbyterian Medical Center and Professor of Clinical Neurology, University of Pennsylvania School of Medicine, said: “The positive results from the 66-week analysis of the Phase III NEURO-TTRansform trial show that eplontersen provided consistent and sustained transthyretin protein reduction and that a substantial number of patients improved in measures of both neuropathy progression and quality of life.

This builds on the favourable 35-week results, which first demonstrated eplontersen’s potential to significantly improve outcomes in this underserved population.”

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “These results further underscore eplontersen’s potential to be a best-in-class treatment across all forms of transthyretin-mediated amyloidosis, including polyneuropathy and cardiomyopathy which can lead to heart failure.

With limited treatment options currently available, there is an urgent unmet medical need for new therapies and earlier, accurate diagnosis across the different types of this systemic, progressive and fatal condition.”

Eugene Schneider, M.D., Executive Vice President and Chief Clinical Development Officer for Ionis, said: “These latest results from our NEURO-TTRansform study represent an important step towards delivering a potential new therapy for ATTRv-PN patients living with this debilitating and fatal disease. We are encouraged by the sustained benefit demonstrated by eplontersen and what a self-administered treatment could mean for patients and families affected by ATTRv-PN.

Together, with our partner AstraZeneca, we look forward to sharing detailed results from this study at the upcoming American Academy of Neurology Annual Meeting.”

ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.

As part of a global development and commercialisation agreement, AstraZeneca and Ionis are seeking regulatory approval for eplontersen for the treatment of ATTRv-PN in the US and plan to seek regulatory approval in Europe and other parts of the world.

Earlier this month, the US Food and Drug Administration accepted a New Drug Application for eplontersen for the treatment of ATTRv-PN.

Eplontersen was granted Orphan Drug Designation in the US.

Eplontersen is currently being evaluated in the Phase III CARDIO-TTRansform trial for transthyretin amyloid cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three to five years from disease onset.

The results from both the 35 and 66-week analyses of the trial will be presented as an Emerging Science presentation at the American Academy of Neurology Annual Meeting in April.

Notes

TTR Amyloidosis
ATTR cardiomyopathy and polyneuropathy are progressive systemic diseases caused by aging or genetic mutations, resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium and peripheral nerves, respectively.

In patients with ATTR, both hereditary and wild type (non-hereditary), TTR protein builds up as fibrils in tissues, such as the peripheral nerves and heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow.

The presence of TTR fibrils interferes with the normal functions of these tissues.

As the TTR protein fibrils accumulate, more tissue damage occurs and the disease worsens, resulting in poor QoL and eventually death.8 Worldwide, there are an estimated 300,000 – 500,000 patients with ATTR-CM9 and about 40,000 patients with ATTRv-PN.

NEURO-TTRansform
NEURO-TTRansform is a global, open-label, randomised trial evaluating the efficacy and safety of eplontersen in patients with ATTRv-PN.

The trial has enrolled adult patients with ATTRv-PN Stage 1 or Stage 2 and will be compared to the external placebo group from the TEGSEDI® (inotersen) NEURO-TTR registrational trial that Ionis completed in 2017.

The final analysis comparing eplontersen to external placebo was completed at week 66 and all patients will be followed on treatment until week 85, when they will have the option to transition into an open-label extension study.

The 66-week analysis evaluated percent change from baseline in serum TTR concentration, changes in the mNIS+7 and Norfolk-QOL-DN in the eplontersen group versus an external placebo group.

The mNIS+7 uses highly standardised, quantitative and referenced assessments to quantify muscle weakness, muscle stretch reflexes, sensory loss and autonomic impairment.

The Norfolk QoL-DN is a patient-reported questionnaire capturing neuropathy-related QoL.

Eplontersen
Eplontersen is a ligand-conjugated antisense (LICA) investigational medicine designed to reduce the production of transthyretin, or TTR protein, to treat all types of ATTR, a systemic, progressive and fatal disease.”

https://www.astrazeneca.com/media-centre/press-releases/2023/eplontersen-demonstrated-sustained-benefit-in-phase-iii-trial.html

Novartis Kisqali® Phase III NATALEE trial meets primary endpoint at interim analysis for breast cancer

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Novartis Kisqali® Phase III NATALEE trial meets primary endpoint at interim analysis for breast cancer

March 27, 2023: “Novartis announced positive topline results from an interim analysis of NATALEE, a Phase III trial evaluating Kisqali® (ribociclib) plus endocrine therapy (ET) in a broad population of patients with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) early breast cancer (EBC) at risk of recurrence.

The Independent Data Monitoring Committee recommended stopping the trial early as the primary endpoint of invasive disease-free survival (iDFS) has been met.

Kisqali plus ET significantly reduced the risk of disease recurrence, compared to standard adjuvant ET alone, with consistent benefit in patients with stage II and stage III EBC regardless of nodal involvement.

“While most patients are diagnosed and treated early with the aim to cure breast cancer, the risk of cancer returning, often as metastatic disease, peaks within three years after diagnosis, but never goes away completely,” said Dennis J. Slamon, MD, Director of Clinical/Translational Research, University of California, Los Angeles Jonsson Comprehensive Cancer Center and Chairman and Executive Director of Translational Research In Oncology (TRIO) and NATALEE trial lead investigator.

“There is a critical need for new, well-tolerated options that keep patients cancer-free without disrupting quality of life.

The NATALEE trial, where ribociclib was given for three years plus ET, was designed with these unmet needs in mind, and it is extremely encouraging that this study met its primary endpoint.” 

Per the NATALEE study protocol, patient follow-up will continue to evaluate long-term outcomes, including overall survival1.

“The positive topline results from NATALEE represent a major milestone in our ambition to expand the benefits of Kisqali to patients with earlier stages of breast cancer, building on the heritage of this effective treatment in HR+/HER2- metastatic breast cancer,” said Shreeram Aradhye, M.D., President, Global Drug Development and Chief Medical Officer, Novartis.

“These data have the potential to be paradigm-shifting for patients at risk of recurrence, including those with no nodal involvement, who have limited well-tolerated options to prevent recurrence.

Our teams are working on submissions to health authorities around the world with the hope to bring Kisqali to many more patients diagnosed with breast cancer.”

These findings build on the legacy of Kisqali in metastatic breast cancer (MBC), where it has consistently demonstrated overall survival benefit while preserving or improving quality of life across three Phase III trials.

Updates to the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for breast cancer, released in January 2023, recommend ribociclib (Kisqali) as the only Category 1 preferred CDK4/6 inhibitor for first-line treatment of patients with HR+/HER2- MBC when combined with an aromatase inhibitor (AI).

About NATALEE
NATALEE is a global Phase III multi-center, randomized, open-label trial to evaluate the efficacy and safety of Kisqali with ET as adjuvant treatment versus ET alone in patients with HR+/HER2- EBC, being conducted in collaboration with Translational Research In Oncology (TRIO).

The primary endpoint of NATALEE is iDFS as defined by the Standardized Definitions for Efficacy End Points (STEEP) criteria; secondary endpoints include safety, quality of life, and overall survival, among others. iDFS is a composite endpoint in EBC adjuvant trials, which incorporates locoregional relapse, ipsilateral and contralateral invasive breast cancer, distant recurrence, and types of new cancer events or death from any cause.

Approximately 5,100 adult patients with HR+/HER2- EBC across 20 countries were randomized in the trial, including patients with tumor stages IIA (select patients), IIB or III, regardless of nodal involvement. NATALEE explored a lower starting dose (400 mg) of Kisqali than the dose approved for treatment in MBC (600 mg) with the goal to minimize disruptions to patient quality of life without compromising efficacy1.

About Early Breast Cancer
More than 90% of patients diagnosed with breast cancer have EBC. Approximately 30-60% of people with HR+/HER2- stage II and III EBC treated with ET only remain at risk of breast cancer recurrence.

The risk of recurrence peaks within the first three years after initial diagnosis and continues over decades. For many of these patients, there are currently no targeted therapeutic options outside of the standard chemotherapy and ET.

About Kisqali® (ribociclib) 
Kisqali has consistently demonstrated overall survival benefit while preserving or improving quality of life across three Phase III trials. Updates to the NCCN Guidelines® for breast cancer, released in January 2023, recommend ribociclib (Kisqali) as the only Category 1 preferred CDK4/6 inhibitor for first-line treatment of patients with HR+/HER2- MBC when combined with an AI.

Additionally, Kisqali has the highest rating of any CDK4/6 inhibitor on the ESMO Magnitude of Clinical Benefit Scale, achieving a score of five out of five for first-line premenopausal patients with HR+/HER2- advanced breast cancer.

Further, Kisqali in combination with either letrozole or fulvestrant has uniquely, among other CDK4/6 inhibitors, received a score of four out of five for postmenopausal patients with HR+/HER2- advanced breast cancer treated in the first line19.
        
Kisqali has been approved in 99 countries worldwide, including by the United States Food and Drug Administration (FDA) and the European Commission.

In the U.S., Kisqali is approved for the treatment of adult patients with HR+/HER2- advanced or metastatic breast cancer in combination with an AI as initial ET or fulvestrant as initial ET or following disease progression on ET in postmenopausal women or in men. In the EU, Kisqali is approved for the treatment of women with HR+/HER2- advanced or metastatic breast cancer in combination with either an AI or fulvestrant as initial ET or following disease progression. In pre- or perimenopausal women, the ET should be combined with a luteinizing hormone-releasing hormone agonist.

https://www.novartis.com/news/media-releases/novartis-kisqali-phase-iii-natalee-trial-meets-primary-endpoint-interim-analysis-demonstrating-clinically-meaningful-benefit-broad-population-patients-early-breast-cancer

FDA Issues Draft Guidance Aimed at Improving Oncology Clinical Trials for Accelerated Approval

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FDA Issues Draft Guidance Aimed at Improving Oncology Clinical Trials for Accelerated Approval

March 24, 2023: “The U.S. FDA issued draft guidance, Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics, regarding clinical trial design considerations to support accelerated approval applications.

The accelerated approval pathway is commonly used for approval of oncology drugs in part due to the serious and life-threatening nature of cancer and because of available intermediate clinical endpoints likely to predict clinical benefit.

“The FDA’s accelerated approval program has provided patients with cancer earlier access to novel treatments that can be practice changing,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence.

“Today’s draft guidance provides recommendations to sponsors for designing clinical trials to support accelerated approval. Building quality and efficiency into the design of oncology clinical trials is a crucial component in providing maximum benefit to those living with cancer.”

The draft guidance discusses the design of clinical trials, and ways to improve the data available at the time of accelerated approval and reduce clinical uncertainty for patients by initiating postmarketing confirmatory studies in a timely manner.

Specifically, the draft guidance addresses the design, conduct and analysis of data through two randomized clinical trial approaches – conducting two separate randomized controlled clinical trials or using one trial for both accelerated approval and to verify clinical benefit.

The draft guidance also provides considerations for sponsors to determine the adequacy of single-arm studies to support an application.
  
For drugs granted accelerated approval, postmarketing confirmatory trials have been required to verify and describe the anticipated clinical benefit.

The draft guidance discusses a potential advantage of randomized clinical trials–compared to single-arm trials–by highlighting that use of the one-trial approach, in appropriate cases, may not require separate clinical trials because longer term follow-up in the same trial could fulfill a postmarketing requirement to verify clinical benefit.

Moreover, confirmatory trials that are in progress at the time of accelerated approval are more likely to result in a timely verification of clinical benefit, therefore minimizing the period of uncertainty for patients.

In a commentary in the New England Journal of Medicine in 2022External Link Disclaimer, Oncology Center of Excellence staff outlined the concepts included and expanded upon in the draft guidance.

The Oncology Center of Excellence has also launched Project Confirm, as an initiative that promotes the transparency of outcomes related to accelerated approval for oncology indications and  fosters discussion and research on the accelerated approval program.

The project developed a searchable database with information on the status of all oncology accelerated approvals, a model that was then adopted by FDA’s Center for Drug Evaluation and Research for all accelerated approvals.”

https://www.fda.gov/news-events/press-announcements/fda-issues-draft-guidance-aimed-improving-oncology-clinical-trials-accelerated-approval

Sandoz receives US FDA approval for biosimilar Hyrimoz® high-concentration formulation

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Sandoz receives US FDA approval for biosimilar Hyrimoz® high-concentration formulation

March 21, 2023: “Sandoz, a global leader in generic pharmaceuticals and biosimilars, today announced that the US Food and Drug Administration (FDA) approved a citrate-free high-concentration formulation (HCF) of its biosimilar Hyrimoz® (adalimumab-adaz) injection.

The adalimumab citrate-free HCF (100 mg/mL) is approved to treat seven indications covered by the reference medicine, Humira®* (adalimumab), including rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis and plaque psoriasis.1

Sandoz intends to launch the Hyrimoz citrate-free HCF in the US on July 1, 2023.

“As one of the first adalimumab high-concentration formulation biosimilars approved in the US, Hyrimoz HCF has the potential to expand access for millions of people who face the realities of living with a serious inflammatory disease and to enhance the patient experience,” said Keren Haruvi, President, Sandoz Inc., Head of North America.

“Sandoz has more than two decades of experience researching, developing and bringing biosimilars to markets across the globe. We are excited to continue this leadership by providing patients with another treatment option to help manage their chronic conditions.”

The FDA approval was based on a Phase I pharmacokinetics (PK) bridging study comparing the FDA-approved adalimumab 50 mg/mL to the citrate-free 100 mg/mL (HCF).

This study met all of the primary objectives, demonstrating comparable PK and showing similar safety and immunogenicity of the adalimumab 50 mg/mL and adalimumab HCF.

“Biosimilars are extensively studied, FDA-approved treatments,” said Steve Taylor, president and chief executive officer, Arthritis Foundation. “There are millions of patients affected by chronic inflammatory conditions that drastically impact their everyday lives.

Given the high burden of disease for these conditions, biosimilars are one potential solution for healthcare providers and patients to consider, to ensure patients can take and stay on their medicines to help manage their disease and health outcomes.”

The FDA approval of Hyrimoz HCF builds on the already approved and well-established Sandoz global biosimilar portfolio in immunology. Sandoz has nearly 120 million days of patient experience with Hyrimoz across 40 countries.

When it launches, Hyrimoz HCF will represent the first launch of a Sandoz biosimilar in the US market in this specific disease space.

Sandoz is committed to helping millions of patients sustainably and affordably access critical and potentially life-changing biologic medicines across a range of areas including immunology, oncology, supportive care and endocrinology.

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

Since launching the first biosimilar in the US in 2015, Sandoz has proven biosimilars create early and expanded patient access to life-altering medicines while increasing healthcare savings and creating competition that fuels innovation and development of new and enhanced treatments in areas of unmet need.

About Hyrimoz® (adalimumab-adaz)
Adalimumab, the active ingredient in Hyrimoz, is an inhibitor of tumor necrosis factor (TNF), a protein that is overproduced in certain autoimmune conditions — including rheumatoid arthritis, plaque psoriasis, Crohn’s disease and ulcerative colitis — causing inflammation and tissue destruction in joints, mucosa or skin.

In some cases of autoimmune disease, the immune system damages the body’s own tissues. Hyrimoz targets and blocks the protein that contributes to disease symptoms.”

https://www.novartis.com/news/media-releases/sandoz-receives-us-fda-approval-biosimilar-hyrimoz-adalimumab-adaz-high-concentration-formulation

Calquence granted first regulatory approval in China for adults with previously treated mantle cell lymphoma

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Calquence granted first regulatory approval in China for adults with previously treated mantle cell lymphoma

March 23, 2023: “AstraZeneca’s Calquence (acalabrutinib), a next generation, selective Bruton’s tyrosine kinase (BTK) inhibitor, has been conditionally approved in China for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.

This is the first approved indication for Calquence in China.

The conditional approval by the National Medical Products Administration (NMPA) was based on positive results from two clinical trials, including the ACE-LY-004 global Phase II trial in adults with relapsed or refractory MCL and a Phase I/ II trial in Chinese patients with relapsed or refractory MCL and other B-cell malignancies.

Continued approval for this indication may be contingent upon verification of ongoing randomised controlled confirmatory trials.

MCL is typically an aggressive, rare form of non-Hodgkin lymphoma (NHL) that accounts for between 2-6% of all patients diagnosed with NHL in China. Patients are generally diagnosed around 60 years of age, often at later stages of the disease.

Jun Zhu, Chief Physician, Department of Lymphatic Oncology, Peking University Cancer Hospital, Beijing, said: “Mantle cell lymphoma progresses rapidly and responds poorly to conventional treatment such as immunochemotherapy. 

Before the emergence of BTK inhibitors, there were few satisfactory treatment options for patients. 

The next-generation BTK inhibitor Calquence has higher target selectivity, fewer side effects, and a higher response rate compared to currently available treatments. This approval of Calquence in China can provide a new treatment option which can better benefit patients with this disease.”

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “This approval for Calquence offers people living with mantle cell lymphoma in China an effective and tolerable new treatment option to help control their disease.

As the first approval in China for Calquence, it is also an exciting step forward for AstraZeneca in blood cancers, enabling us to help more patients across the globe gain access to innovative treatments.”

Results from the ACE-LY-004 Phase II trial showed at a median follow up of 15.2 months, investigator-assessed overall response rate (ORR) with Calquence was 80.6% (95% confidence interval [CI] 72.6-87.2), with a complete response (CR) achieved in 39.5% of patients with relapsed or refractory MCL (95% CI 30.9-48.7).

Longer-term follow-up data showed at 38.1 months, patients treated with Calquence remained progression-free for a median of 22 months, with median overall survival (OS) of 59.2 months (95% CI 36.5-NE).

Additionally, results from a Phase I/II trial conducted in China showed Calquence achieved a 82.4% ORR, with a CR achieved in 35.3% of patients with MCL based on a blinded independent central-review (BICR) analysis (95% CI 65.5-93.2). 

Calquence reduced the risk of disease progression or death by 51.5% (95% CI 33.3-67.0) at 12 months, with an estimated duration of response (DOR) of 65.5% (95% CI 66.6-93.3). The median DOR was not reached.2

The safety and tolerability of Calquence in these trials was consistent with that observed in previous clinical trials.

Calquence is approved for the treatment of chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL) in the US and Japan and is approved for the treatment of CLL in the EU and in several other countries worldwide in the treatment-naïve and relapsed or refractory settings. 

Calquence is also approved in the US and several other countries for the treatment of adult patients with MCL who have received at least one prior therapy. 

Calquence is not currently approved for the treatment of MCL in Japan or the EU.”

https://www.astrazeneca.com/media-centre/press-releases/2023/calquence-granted-first-regulatory-approval-in-china-for-adults-with-previously-treated-mantle-cell-lymphoma.html

Bayer’s Nubeqa™ approved for additional prostate cancer indication in China

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Bayer’s Nubeqa™ approved for additional prostate cancer indication in China

March20,2023: “The Chinese National Medical Products Administration (NMPA) has approved the oral androgen receptor inhibitor (ARi) Nubeqa™ (darolutamide) in combination with docetaxel for the treatment of patients with metastatic hormone-sensitive prostate cancer (mHSPC).

Nubeqa is already approved in China for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease.

“Prostate cancer cases in China have increased significantly in recent years. Compounding that, nearly a third of patients who are newly diagnosed will have metastatic disease.

We are therefore delighted that patients in China will now have a new treatment option for metastatic hormone-sensitive prostate cancer that delays disease progression, extends survival and maintains quality of life”, said Christine Roth, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of the Oncology SBU at Bayer.

“Bayer is committed to improving health outcomes for people living with prostate cancerand that as many eligible patients as possible gain access to Nubeqa.”

The approval is based on the positive results from the Phase III ARASENS trial, which demonstrated that darolutamide plus androgen deprivation therapy (ADT) in combination with docetaxel significantly reduced the risk of death by 32.5% compared to ADT with docetaxel, in patients with metastatic hormone-sensitive prostate cancer (mHSPC).

Additionally, the darolutamide combination showed consistent benefits across clinically relevant secondary endpoints, with the overall incidence of treatment-emergent adverse events being similar between treatment arms. These results were published in The New England Journal of Medicine.

Darolutamide is being investigated in a broad development program with three additional ongoing or planned large clinical studies, to evaluate its potential across prostate cancer patients from early- to late-stage disease.

This includes the ARANOTE Phase III trial evaluating darolutamide plus androgen deprivation therapy (ADT) versus ADT alone for mHSPC.

Nubeqa has been developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company.

About the ARASENS Trial
The ARASENS trial is the only randomized, Phase III, multi-center, double-blind, trial which was prospectively designed to compare the use of a second-generation oral androgen receptor inhibitor (ARi), darolutamide, plus ADT in combination with docetaxel to ADT plus docetaxel (a guideline recommended standard-of-care) in metastatic hormone-sensitive prostate cancer (mHSPC).

A total of 1,306 newly diagnosed patients were randomized in a 1:1 ratio to receive 600 mg of darolutamide twice a day or matching placebo, plus ADT in combination with docetaxel.

The primary endpoint of this trial was overall survival (OS). Secondary endpoints included time to castration-resistant prostate cancer (CRPC), time to pain progression, time to first symptomatic skeletal event (SSE), time to initiation of subsequent anticancer therapy, all evaluated at 12‐week intervals, as well as adverse events (AEs) as a measure of safety and tolerability. Results from this trial were published in the New England Journal of Medicine.

A plain language summary publication of these data was published in Future Oncology. The ARASENS trial demonstrated that darolutamide plus ADT in combination with docetaxel significantly reduced the risk of death by 32.5% compared to ADT with docetaxel alone.

Improvements in the secondary endpoints supported the benefit observed in the primary endpoint, overall survival.1

About Metastatic Hormone-Sensitive Prostate Cancer
Prostate cancer is the second most commonly diagnosed malignancy in men worldwide. In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, and about 375,000 died from the disease worldwide.

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy.

Upon relapse, when the disease will metastasize or spread, or if the disease is newly diagnosed, but has already spread, the disease is hormone-sensitive and androgen deprivation therapy (ADT) is the cornerstone of treatment.

Current treatment options for men with metastatic hormone-sensitive prostate cancer (mHSPC) include hormone therapy, such as ADT, androgen receptor pathway inhibitors plus ADT or a combination of ADT and docetaxel.

Despite these treatments, a large proportion of men with mHSPC will eventually experience progression to metastatic castration-resistant prostate cancer (mCRPC), a condition with high morbidity and limited survival.


About Nubeqa™ (darolutamide)
Darolutamide is an oral androgen receptor inhibitor (ARi) with a distinct chemical structure that binds to the receptor with high affinity and exhibits strong antagonistic activity, thereby inhibiting the receptor function and the growth of prostate cancer cells.

The low potential for blood-brain barrier penetration for darolutamide is supported by preclinical models and neuroimaging data in healthy humans.

This is supported by the overall low incidence of central nervous system (CNS)-related adverse events (AEs) compared to placebo as seen in the ARAMIS Phase III trial and the improved verbal learning and memory observed in the darolutamide arm of the Phase II ODENZA trial.

Nubeqa is approved in more than 80 countries around the world for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC).

It is also approved for the treatment of patients with metastatic hormone-sensitive prostate cancer (mHSPC) in a number of markets including the U.S., Japan, EU and China. Filings in other regions are underway or planned.

Bayer believes that the peak sales potential for Nubeqa may exceed €3 billion.

The compound is also being investigated in further studies across various stages of prostate cancer, including in the ARANOTE Phase III trial evaluating darolutamide plus androgen deprivation therapy (ADT) versus ADT alone for metastatic hormone-sensitive prostate cancer (mHSPC), as well as the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP) led international Phase III co-operative group DASL-HiCaP (ANZUP1801) trial evaluating darolutamide as an adjuvant treatment for localized prostate cancer with very high risk of recurrence. Information about these trials can be found at www.clinicaltrials.gov.

In addition, a study to explore the potential of darolutamide in the early setting for patients who have experienced a rise in their prostate specific antigen (PSA) levels following surgery or radiation, is also planned.

About Prostate Cancer at Bayer
Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments.

The company has the passion and determination to develop new medicines that help improve and extend the lives of people living with cancer.

Prostate cancer is the second most commonly diagnosed cancer in men3 and a key area of focus for Bayer.

The company’s franchise includes two products on the market (Nubeqa™ and Xofigo™) and several compounds in development, including a unique approach of advancing targeted alpha therapies.

Bayer is focused on addressing the unique needs of prostate cancer patients, providing treatments that extend their lives throughout the different stages of the disease and allowing them to continue their everyday activities, so that they can live longer, better lives.”

https://www.bayer.com/media/en-us/nubeqa-darolutamide-approved-for-additional-prostate-cancer-indication-in-china/

FDA Denies Marketing of Two Vuse Solo Menthol E-Cigarette Products

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FDA Denies Marketing of Two Vuse Solo Menthol E-Cigarette Products

March 17, 2023: “The U.S. FDA issued marketing denial orders (MDOs) for two menthol e-cigarette products currently marketed by R.J. Reynolds Vapor Company under the Vuse Solo brand.

The currently marketed products include the Vuse Replacement Cartridge Menthol 4.8% G1 and the Vuse Replacement Cartridge Menthol 4.8% G2.

The company must not market or distribute these products in the U.S. or they risk FDA enforcement action.

The company may resubmit applications or submit new applications to address the deficiencies for the products that are subject to these MDOs. 

The FDA evaluates premarket tobacco product applications (PMTAs) based on a public health standard that considers the risks and benefits of the product on the population as a whole.

After reviewing the company’s PMTAs, the FDA determined that the applications lacked sufficient evidence to demonstrate that permitting the marketing of the products would be appropriate for the protection of the public health, which is the applicable standard legally required by the 2009 Family Smoking Prevention and Tobacco Control Act.

Specifically, evidence submitted by the applicant did not demonstrate that its menthol-flavored e-cigarettes provide an added benefit for adult smokers relative to tobacco-flavored e-cigarettes.

“The FDA is a data driven agency and science remains the cornerstone of our tobacco product regulatory activities,” said Brian King, Ph.D., M.P.H., director of the FDA’s Center for Tobacco Products.

“The science has guided – and will always guide – the FDA’s decision making on premarket tobacco product applications, including today’s marketing denial orders.”   

Existing evidence, including data from the 2022 National Youth Tobacco Survey (NYTS), shows that non-tobacco-flavored e-cigarettes, including menthol-flavored e-cigarettes, have a known and substantial risk with regard to youth appeal, uptake and use.

In contrast, data indicate tobacco-flavored e-cigarettes do not have the same appeal to youth and therefore do not pose the same degree of risk.

Given these existing differences in youth risk, applicants need to provide robust evidence to demonstrate that using their menthol-flavored e-cigarette products are likely to promote complete switching or are likely to significantly reduce combustible cigarette use in adult smokers beyond that facilitated by tobacco-flavored e-cigarettes.

NYTS data also found Vuse to be the second most common brand youth e-cigarette users reported “usually” using.

These products cannot be legally introduced into interstate commerce in the U.S. without risking FDA enforcement.

In addition to ensuring that the manufacturer complies with this order, as with unauthorized products generally, the FDA intends to ensure compliance by distributors and retailers.

Retailers should contact R.J. Reynolds Vapor Company with any questions about products in their inventory. 

Today’s actions are just one of many the FDA has taken to ensure any tobacco products that are marketed in the U.S. undergo science-based review and receive marketing authorizations by the agency.

To date, the agency has received applications for more than 26 million deemed products and has made determinations on 99% of these applications.

To date, the FDA has authorized 23 tobacco-flavored e-cigarette products and devices, which are the only e-cigarettes that currently may be lawfully sold or distributed in the U.S.; this includes the Vuse Solo e-cigarette device and two accompanying tobacco-flavored e-liquid cartridges.

The FDA has also denied marketing applications for millions of products that did not meet the requirements in the law, including ten non-tobacco-flavored e-liquid cartridges under the Vuse Solo brand, Vuse Vibe Tank Menthol 3.0% and the Vuse Ciro Cartridge Menthol 1.5%.”

https://www.fda.gov/news-events/press-announcements/fda-denies-marketing-two-vuse-solo-menthol-e-cigarette-products

Pfizer’s XTANDI® plus Leuprolide Significantly Improves Metastasis-Free Survival in Men with Non-Metastatic Prostate Cancer

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Pfizer’s XTANDI® plus Leuprolide Significantly Improves Metastasis-Free Survival in Men with Non-Metastatic Prostate Cancer

March 16, 2023: “Pfizer Inc. and Astellas Pharma Inc. announced positive topline results from the Phase 3 EMBARK trial evaluating XTANDI® (enzalutamide) in men with non-metastatic hormone-sensitive prostate cancer (nmHSPC; also known as non-metastatic castration-sensitive prostate cancer or nmCSPC) with high-risk biochemical recurrence (BCR).

Patients enrolled in the trial were randomized to one of three study arms: XTANDI plus leuprolide, placebo plus leuprolide, or XTANDI monotherapy.

The study met its primary endpoint with a statistically significant and clinically meaningful improvement in metastasis-free survival (MFS) for patients treated with XTANDI plus leuprolide versus placebo plus leuprolide.

At the time of the analysis, a positive trend in the key secondary endpoint of overall survival (OS) was also observed, but these data were not yet mature. Patients in the trial will be followed for a subsequent final OS analysis.

The study also met a key secondary endpoint with a statistically significant and clinically meaningful improvement in MFS for patients treated with XTANDI monotherapy versus placebo plus leuprolide.

Additional key secondary endpoints reached statistical significance, including time to prostate-specific antigen (PSA) progression and time to first use of new antineoplastic therapy.

Other secondary endpoints are being analyzed. No new safety signals have been observed to date in the preliminary safety analysis, which is consistent with the established safety profile of XTANDI.

“As the only novel hormone therapy approved for three disease states of prostate cancer in the U.S., XTANDI has impacted hundreds of thousands of men,” said Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology and Rare Disease, Pfizer Global Product Development.

“The topline findings from EMBARK are highly encouraging and we look forward to engaging with health authorities to potentially bring XTANDI to men with non-metastatic hormone-sensitive prostate cancer with high-risk biochemical recurrence.”

“While current treatment options for localized prostate cancer are intended to be curative, some men remain at higher risk for biochemical recurrence following primary treatment, which may result in metastases,” said Ahsan Arozullah, M.D., MPH, Senior Vice President and Head of Development Therapeutic Areas, Astellas.

“The EMBARK trial is the first study to demonstrate a statistically significant improvement in MFS using the combination of XTANDI plus leuprolide in men with this stage of disease.”

Detailed results from EMBARK will be presented at a future medical meeting.

These data will also be discussed with regulatory authorities, including the U.S. Food and Drug Administration (FDA), to support a potential regulatory submission for XTANDI in this indication.

About EMBARK
The Phase 3, randomized, double-blind, placebo-controlled, multi-national trial enrolled 1,068 patients with non-metastatic hormone-sensitive prostate cancer (nmHSPC; also known as non-metastatic castration-sensitive prostate cancer or nmCSPC) with high-risk biochemical recurrence (BCR) at sites in the United States, Canada, Europe, South America, and the Asia-Pacific region.

Patients who were considered high-risk BCR had a prostate-specific antigen (PSA) doubling time ≤ 9 months, serum testosterone ≥ 150 ng/dL (5.2 nmol/L), and screening PSA by the central laboratory ≥ 1 ng/mL if they had a radical prostatectomy (with or without radiotherapy) as primary treatment for prostate cancer or at least 2 ng/mL above the nadir if they had radiotherapy only as primary treatment for prostate cancer.

Patients in the EMBARK trial were randomized to receive enzalutamide 160 mg daily plus leuprolide, enzalutamide 160 mg as a monotherapy, or placebo plus leuprolide. 

The primary endpoint of the trial was metastasis-free survival (MFS) for enzalutamide plus leuprolide and placebo plus leuprolide.

MFS is defined as the duration of time in months between randomization and the earliest objective evidence of radiographic progression by central imaging or death.

For more information on the EMBARK (NCT02319837) trial go to www.clinicaltrials.gov. 

XTANDI has not been approved for the treatment of patients with nmHSPC with high-risk BCR.

About Non-Metastatic Hormone-Sensitive Prostate Cancer with High-Risk Biochemical Recurrence  
Non-metastatic hormone- (or castration-) sensitive prostate cancer (nmHSPC or nmCSPC) means there is no detectable evidence of the cancer spreading to distant parts of the body (metastases) with conventional radiological methods (CT/MRI) and the cancer still responds to medical or surgical treatment to lower testosterone levels.

Of men who have undergone definitive prostate cancer treatment, including radical prostatectomy, radiotherapy, or both, an estimated 20-40% will experience a biochemical recurrence (BCR) within 10 years.

About 9 out of 10 men with high-risk BCR will develop metastatic disease, and 1 in 3 will die as a result of the recurrence.

The EMBARK trial focused on men with high-risk BCR. Per the EMBARK protocol, patients with nmHSPC with high-risk BCR are those initially treated by radical prostatectomy or radiotherapy, or both, with a PSA doubling time ≤ 9 months.

High-risk BCR patients with a PSA doubling time of ≤ 9 months have a higher risk of metastases and death.

About XTANDI® (enzalutamide)
XTANDI (enzalutamide) is an androgen receptor signaling inhibitor.

The recommended dosage of XTANDI is 160 mg (capsules or tablets) administered orally once daily with or without food.

XTANDI is a standard of care that has received regulatory approvals for use in men with mHSPC, mCRPC, and nmCRPC in the United States and for one or more of these indications in more than 100 countries, including the European Union and Japan. More than 720,000 patients have been treated with XTANDI globally.”

https://www.pfizer.com/news/press-release/press-release-detail/phase-3-study-shows-xtandir-enzalutamide-plus-leuprolide

FDA Denies Marketing of Two Vuse Solo Menthol E-Cigarette Products

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FDA Denies Marketing of Two Vuse Solo Menthol E-Cigarette Products

March 17, 2023: “The U.S. Food and Drug Administration issued marketing denial orders (MDOs) for two menthol e-cigarette products currently marketed by R.J. Reynolds Vapor Company under the Vuse Solo brand.

The currently marketed products include the Vuse Replacement Cartridge Menthol 4.8% G1 and the Vuse Replacement Cartridge Menthol 4.8% G2.

The company must not market or distribute these products in the U.S. or they risk FDA enforcement action. The company may resubmit applications or submit new applications to address the deficiencies for the products that are subject to these MDOs. 

The FDA evaluates premarket tobacco product applications (PMTAs) based on a public health standard that considers the risks and benefits of the product on the population as a whole.

After reviewing the company’s PMTAs, the FDA determined that the applications lacked sufficient evidence to demonstrate that permitting the marketing of the products would be appropriate for the protection of the public health, which is the applicable standard legally required by the 2009 Family Smoking Prevention and Tobacco Control Act.

Specifically, evidence submitted by the applicant did not demonstrate that its menthol-flavored e-cigarettes provide an added benefit for adult smokers relative to tobacco-flavored e-cigarettes.

“The FDA is a data driven agency and science remains the cornerstone of our tobacco product regulatory activities,” said Brian King, Ph.D., M.P.H., director of the FDA’s Center for Tobacco Products.

“The science has guided – and will always guide – the FDA’s decision making on premarket tobacco product applications, including today’s marketing denial orders.”   

Existing evidence, including data from the 2022 National Youth Tobacco Survey (NYTS), shows that non-tobacco-flavored e-cigarettes, including menthol-flavored e-cigarettes, have a known and substantial risk with regard to youth appeal, uptake and use.

In contrast, data indicate tobacco-flavored e-cigarettes do not have the same appeal to youth and therefore do not pose the same degree of risk.

Given these existing differences in youth risk, applicants need to provide robust evidence to demonstrate that using their menthol-flavored e-cigarette products are likely to promote complete switching or are likely to significantly reduce combustible cigarette use in adult smokers beyond that facilitated by tobacco-flavored e-cigarettes. NYTS data also found Vuse to be the second most common brand youth e-cigarette users reported “usually” using.

These products cannot be legally introduced into interstate commerce in the U.S. without risking FDA enforcement.

In addition to ensuring that the manufacturer complies with this order, as with unauthorized products generally, the FDA intends to ensure compliance by distributors and retailers.

Retailers should contact R.J. Reynolds Vapor Company with any questions about products in their inventory. 

Today’s actions are just one of many the FDA has taken to ensure any tobacco products that are marketed in the U.S. undergo science-based review and receive marketing authorizations by the agency.

To date, the agency has received applications for more than 26 million deemed products and has made determinations on 99% of these applications.

To date, the FDA has authorized 23 tobacco-flavored e-cigarette products and devices, which are the only e-cigarettes that currently may be lawfully sold or distributed in the U.S.; this includes the Vuse Solo e-cigarette device and two accompanying tobacco-flavored e-liquid cartridges.

The FDA has also denied marketing applications for millions of products that did not meet the requirements in the law, including ten non-tobacco-flavored e-liquid cartridges under the Vuse Solo brand, Vuse Vibe Tank Menthol 3.0% and the Vuse Ciro Cartridge Menthol 1.5%.”

https://www.fda.gov/news-events/press-announcements/fda-denies-marketing-two-vuse-solo-menthol-e-cigarette-products

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