Study Finds LOKELMA™ (sodium zirconium cyclosilicate) Reduces Elevated Potassium in Hemodialysis Patients

MedicalResearch.com Interview with:

Steven Fishbane MD Chief, Division of Kidney Disease and Hypertension Vice President, Northwell Health for Network Dialysis Services, Northwell Health Professor of Medicine Donald and Barbara Zucker School of Medicine at Hofstra/Northwell Department of Medicine, Zucker School of Medicine at Hofstra/Northwell, Great Neck, New York

Dr. Fishbane

Steven Fishbane MD
Chief, Division of Kidney Disease and Hypertension
Vice President, Northwell Health for Network Dialysis Services, Northwell Health
Professor of Medicine
Donald and Barbara Zucker School of Medicine at Hofstra/Northwell
Department of Medicine, Zucker School of Medicine at Hofstra/Northwell,
Great Neck, New York 


MedicalResearch.com: What is the background for this study?

Response: Patients on hemodialysis have a great frequency of hyperkalemia. The hemodialysis treatment removes some potassium but not enough to get rid of this problem. Available medications to bind potassium have not been tested among these patients.

The purpose of the study was to see if sodium zirconium cyclosilicate could be used as a potassium binder to reduce the risk of hyperkalemia in patients on a hemodialysis.

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Synthetic Cannabidiol Explored As Potential Topical Antibiotic

MedicalResearch.com Interview with:

Dr Mark Blaskovich PhD Institute for Molecular Bioscience's Centre for Superbug Solutions The University of Queensland In collaboration with Botanix Pharmaceuticals Ltd

Dr. Blaskovich

Dr Mark Blaskovich PhD
Institute for Molecular Bioscience’s Centre for Superbug Solutions
The University of Queensland
In collaboration with Botanix Pharmaceuticals Ltd 

MedicalResearch.com: What is the background for this study?  

Response: Botanix is a company that has been developing topical formulations of CBD for treatment of skin diseases such as atopic dermatitis and acne, based on its reported anti-inflammatory properties. However, these diseases are also associated with bacterial infection, so they were interested in looking at potential antimicrobial activity, as there are some previous literature reports suggesting it is active. They contacted us to do some more in-depth investigations.

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Real World Study of Subcutaneous Abatacept (ORENCIA®) For Rheumatoid Arthritis

MedicalResearch.com Interview with:

Sean Connolly, Ph.D. Director of Non-Registrational Data Generation Study Director for ASCORE Bristol-Myers Squibb

Dr. Connolly

Sean Connolly, Ph.D.
Director of Non-Registrational Data Generation
Study Director for ASCORE
Bristol-Myers Squibb

MedicalResearch.com: What is the background for this study?

Response: ASCORE is a two-year, prospective multicenter study to observe retention and response rates of moderate-to-severe rheumatoid arthritis (RA) patients receiving ORENCIA® (abatacept), administered subcutaneously via a pre-filled syringe, in routine clinical practice. Findings shared at the Annual European Congress of Rheumatology (EULAR 2019) are the results from the first 12 months.

An important objective of our development program is to understand how well we can replicate findings from our clinical trials among a real-world patient population. In the case of ASCORE, which looked at approximately 3,000 patients, both bio-naïve and patients receiving later-line therapies, these data add to the body of research that may help inform physicians treating patients with RA.

Patients participating in ASCORE were divided into two distinct cohorts at the outset of the study: bio-naïve and patients previously administered one or more biologic agents. The primary endpoint is to estimate the rentention rate of patients in each cohort over a 24-month period. Furthermore, ASCORE examines the patient populations across ten countries to understand factors including: how ORENCIA is prescribed, characteristics of patients from each country (socio-demographic data, medical history, co-morbidities, etc.), and population health statistics within each country. This sub-analysis is factored into patient response to treatment across both cohorts, which may help physicians better understand how and why certain populations demonstrate a specific retention rate. Continue reading

FARXIGA (dapagliflozin) Reduced Kidney Function Decline in Type II Diabetes

MedicalResearch.com Interview with:

Naeem Khan MD Vice President at AstraZeneca

Dr. Khan

Naeem Khan MD
Vice President at AstraZeneca 

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: A pre-specified exploratory analysis of renal data from the DECLARE-TIMI 58 trial, the largest SGLT-2 inhibitor (SGLT-2i) cardiovascular outcomes trial (CVOT) conducted to date, showed that FARXIGA (dapagliflozin) reduced the composite of kidney function decline, end-stage renal disease (ESRD) or renal death by 47% in patients with type 2 diabetes (T2D).

Additionally, FARXIGA reduced the relative risk of a cardio-renal composite of kidney function decline, ESRD, or renal or cardiovascular (CV) death by 24% compared to placebo.

The analysis evaluated 17,160 patients with type 2 diabetes and predominantly preserved renal function, irrespective of underlying atherosclerotic CV disease (ASCVD).

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Lowered Price of Repatha® (evolocumab) Translates to Cost Effectiveness for Range of Heart Conditions in Some High Risk Patients

MedicalResearch.com Interview with:

Gregg C. Fonarow, MD, FACC, FAHA Eliot Corday Professor of Cardiovascular Medicine and Science Director, Ahmanson-UCLA Cardiomyopathy Center Co-Chief of Clinical Cardiology, UCLA Division of Cardiology Co-Director, UCLA Preventative Cardiology Program David Geffen School of Medicine at UCLA Los Angeles, CA, 90095-1679

Dr. Gregg Fonarow

Gregg C. Fonarow, MD, FACC, FAHA
Eliot Corday Professor of Cardiovascular Medicine and Science
Director, Ahmanson-UCLA Cardiomyopathy Center
Co-Chief of Clinical Cardiology, UCLA Division of Cardiology
Co-Director, UCLA Preventative Cardiology Program
David Geffen School of Medicine at UCLA
Los Angeles, CA 

MedicalResearch.com: What is the background for this study?

Response: Last year, Amgen made the PCSK-9 inhibitor evolocumab available at a reduced list price of $5,850 per year This 60% reduction was aimed at improving patient access by lowering patient copays, especially for Medicare beneficiaries.

Additionally, the treatment landscape for PCSK9 inhibitors was further defined in 2018 when the American College of Cardiology/American Heart Association Multisociety Clinical Guideline on the Management of Blood Cholesterol recommended PCSK9 inhibitors for, among other patient populations, patients with very high-risk (VHR) ASCVD whose low-density lipoprotein cholesterol levels remain at 70 mg/dL or more  despite a heart-healthy lifestyle and treatment with standard background therapy.

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Accumulation of Metabolite M-2 Predicts Overall Survival of Chemorefractory Metastatic Colorectal Cancer Patients Treated with Regorafenib (Stivarga®)

MedicalResearch.com Interview with:
Joseph Germino, M.D., PhD
Vice President US Medical Affairs Oncology
Bayer Healthcare Pharmaceuticals
Whippany, N.J. 07981

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Regorafenib is an oral multi-kinase inhibitor that potently blocks multiple protein kinases involved in tumor angiogenesis (VEGFR1, -2, -3, TIE2), oncogenesis (KIT, RET, RAF-1, BRAF), metastasis (VEGFR3, PDGFR, FGFR) and tumor immunity (CSF1R).

This prospective pharmacokinetic (PK) ancillary study is part of a prospective phase II study evaluating treatment response with regorafenib in patients with chemorefractory metastatic colorectal cancer (mCRC) called TEXAN, which aimed to investigate correlations between overall survival (OS) and regorafenib, or its enterohepatic cycle-dependent active metabolites M-2 and M-5 concentrations.

As measured by LC-MS/MS, the main findings showed that regorafenib, M-2 and M-5 were respectively 1.99 (1.03-2.73), 1.44 (0.89-2.49) and 1.61 (0.79-2.37) mg/L during the first cycle at day 15 (C1D15) and 1.90 (1.10-2.76), 1.29 (0.77-2.24) and 1.17 (0.45-2.42) mg/L at during the second cycle at day 15 (C2D15).  Continue reading

Children with High Risk AML: Intensification of Induction II Chemotherapy and Liberalization of Stem Cell Donor Source does not Improve Outcomes

MedicalResearch.com Interview with:
Joseph Germino, M.D., PhD
Vice President US Medical Affairs Oncology
Bayer Healthcare Pharmaceuticals
Whippany, N.J. 07981

MedicalResearch.com: What is the background for this study?

Response: Sorafenib (Nexavar®) is an oral anticancer therapy approved in more than 100 countries worldwide. It is approved for the treatment of patients with advanced hepatocellular carcinoma (HCC); advanced renal cell carcinoma (RCC) who have failed prior interferon-alpha or interleukin-2 based therapy or are considered unsuitable for such therapy; progressive, locally advanced or metastatic differentiated thyroid carcinoma (papillary/follicular/Hürthle cell), that is refractory to radioactive iodine (RAI).

The AAML 1031 is a recently completed Phase III clinical trial evaluating the use of bortezomib and sorafenib in patients 30 years or younger with newly diagnosed acute myeloid leukemia (AML).

At the 2019 ASCO Annual meeting, results of a report from the AAML1031 trial, which assessed whether intensification of Induction II chemotherapy (ADE or AraC/ Mitoxantrone) and liberalized stem cell transplant (SCT) donor source criteria improved clinical outcomes in patients with residual AML.  Continue reading

Many ER Patients with Elevated Potassium, End up Being Admitted After Discharge

MedicalResearch.com Interview with:
AstraZenecaJill Davis, MS
Director, Health Economics and Outcomes Research
AstraZeneca 

MedicalResearch.com: What is the background for this study? Who is most at risk for hyperkalemia post discharge?

Response: In the United States, an estimated 30 million people suffer from chronic kidney disease (CKD), about 3.7 million of which have hyperkalemia (elevated potassium level). Hyperkalemia (HK) can be chronic, so it’s important that those who have been diagnosed with hyperkalemia previously or have CKD have their potassium levels monitored by their healthcare provider. Additionally, although HK is estimated to be prevalent in more than 66,000 emergency department (ED) visits annually, there is limited knowledge about the management of patients with HK in the ED setting and post-discharge. We decided to focus our study to better understand and compare the ED management and post-discharge outcomes among patients with varying levels of hyperkalemia

To conduct this study, we analyzed the electronic medical record data from the Research Action for Health Network (2012-2018) of 6,222 adult patients with a randomly selected HK-related ED visit. We concluded that improved management of HK patients in the ED and post-discharge period is needed to reduce the recurrence of hyperkalemia.

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Larotrectinib (VITRAKVI® ): Efficacy and Safety in Pediatric TRK Fusion Cancer Patients

MedicalResearch.com Interview with:

Douglas S. Hawkins, M.D. Hematology/Oncology Division Chief and Professor Pediatrics at Seattle Children's Hospital University of Washington School of Medicine

Dr. Hawkins

Douglas S. Hawkins, M.D.
Hematology/Oncology Division Chief and Professor
Pediatrics at Seattle Children’s Hospital
University of Washington School of Medicine

MedicalResearch.com: What is the background for this study?

Response: TRK fusion cancer is caused by a rare genomic alteration called a neurotrophic receptor tyrosine kinase (NTRK) gene fusion.

Larotrectinib is a central nervous system (CNS) active, oral and highly selective TRK inhibitor used for the treatment of adult and pediatric patients with solid tumors that have a rare genomic alteration called an NTRK gene fusion. Larotrectinib was approved at the end of 2018 in the U.S. under the brand name VITRAKVI®, with European and worldwide regulatory submissions underway.

At ASCO 2019, we will be presenting results from a new analysis specifically looking at the efficacy and safety of larotrectinib in pediatric patients (n=34) included in the expanded dataset from both adults and children across 24 tumor types, which was presented first at the European Society for Medical Oncology (ESMO) 2019 Annual Meeting.  Continue reading

CLL: Ibrutinib (IMBRUVICA®) Demonstrated Durable Responses for High Risk Patients

MedicalResearch.com Interview with:

Paul M. Barr, M.D. Associate Professor of Medicine and Director of the Clinical Trials Office Director of the Clinical Trials Office Wilmot Cancer Institute

Dr. Barr

Paul M. Barr, M.D.
Associate Professor of Medicine and Director of the Clinical Trials Office
Director of the Clinical Trials Office
Wilmot Cancer Institute 

MedicalResearch.com: What is the background for this study?  

Response: When the study was designed, chronic lymphocytic leukemia (CLL)  treatment options were largely limited to chemotherapy and monoclonal antibodies.

Ibrutinib had shown promise in early studies. The intent was to compare ibrutinib to a standard of care treatment option at that time, of atumumab, in patients with relapsed or refractory disease. The goal of the current analysis is to evaluate the durability of ibrutinib and report the long-term safety results.

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Novartis Piqray® Approved for Patients with a PIK3CA Mutation in HR+/HER2- Advanced Breast Cancer

MedicalResearch.com Interview with:

Fabrice André, MD, PhD Research director and head of INSERM Unit U981 Professor in the Department of Medical Oncology Institut Gustave Roussy in Villejuif, France Global SOLAR-1 Principal Investigator.

Dr. Fabrice André

Fabrice André, MD, PhD
Research director and head of INSERM Unit U981
Professor in the Department of Medical Oncology
Institut Gustave Roussy in Villejuif, France
Global SOLAR-1 Principal Investigator.

MedicalResearch.com: What is the background for this study? How does Piqray®  differ from other treatments for this type of advanced breast cancer? 

  • The US Food and Drug Administration (FDA) approved Piqray® (alpelisib, formerly BYL719) in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-), PIK3CA-mutated, advanced or metastatic breast cancer, as detected by an FDA-approved test after disease progression following an endocrine-based regimen.
  • Piqray is the first and only combination treatment with fulvestrant specifically for postmenopausal women, and men, with HR+/HER2- advanced or metastatic breast cancer with a PIK3CA mutation following progression on or after an endocrine-based regimen, bringing a biomarker-driven therapy option to this population for the first time.
  • Advanced breast cancer is incurable, and patients with all types need more treatment options. With this approval, physicians can now use an FDA-approved test to determine if their patients’ HR+/HER2- advanced breast cancer has a PIK3CA mutation and may be eligible for treatment with Piqray plus fulvestrant combination therapy. 

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Fewer Oncologists Have Financial Ties to Pharmaceutical Companies

MedicalResearch.com Interview with:
Deborah C. Marshall, MD
Icahn School of Medicine at Mount Sinai

MedicalResearch.com: What is the background for this study? What are the main findings? 

Response: Open Payments has brought sweeping change to medicine by introducing transparency to physician relationships with industry. We have seen its impact on oncology through the recent media attention to high-profile physicians in oncology scrutinized for their failure to disclose industry relationships and through the resulting changes to conflict of interest policies of clinical, professional and research organizations in recent months.

We wanted to better understand the impact of Open Payments on individual physician behavior due to the important ethical and policy implications.  We have a cohort of oncology physicians that we followed from the inception of Open Payments to see whether the implementation and increasing awareness of Open Payments have resulted in fewer physicians engaging with industry and has shifted payments towards those considered more appropriate.

The study is important because we evaluate trends at the physician-level to explore the impact of Open Payments on how physicians interact with industry, which is difficult to measure. 

MedicalResearch.com: What should readers take away from your report?

Response: The most important finding is that oncology physician interactions with industry are decreasing, which we interpret as being due to the effect of Open Payments.  Notably, we do not see large shifts in the types of payments yet, which suggests that transparency alone may not be enough to significantly alter behavior.  Moreover, while there has been a decrease in oncology physicians interacting with industry, the number and value of these interactions has not shifted greatly, which should reassure those who were concerned that this type of transparency program would have a negative impact on beneficial industry interactions.

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: We are likely going to see the continued impact of Open Payments over time as the downstream effects of transparency become apparent, which warrants ongoing attention to help guide future policy-making.  Engaging stakeholders in these discussions, as well as investigating the impact of industry relationships on how physicians are providing care, conducting and reporting research, and educating future doctors are relevant areas of further research.  Also, there is increasing financial interest in oncology so addressing the risk associated with financial interactions with industry and conflicts of interest are more important than ever. 

Citation: 2019 ASCO Annual Meeting  June 1 2019

Trends in financial relationships between industry and individual medical oncologists in the United States from 2014 to 2017: A cohort study.

Author(s): Deborah Catherine Marshall, Elizabeth Stieglitz Tarras, Kenneth Rosenzweig, Deborah Korenstein, Susan Chimonas; Icahn School of Medicine at Mount Sinai, New York, NY; New York University School of Medicine, New York, NY; Memorial Sloan Kettering Cancer Center, New York, NY

https://abstracts.asco.org/239/AbstView_239_258191.html 

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TItan Study: Apalutamide (ERLEADA®) plus Androgen Deprivation Therapy Improved Survival in Some Metastatic Prostate Cancer Patients

MedicalResearch.com Interview with:

Dr. Kim Chi. MDProfessor of MedicineMedical Oncologist and Medical Director at BC Cancer – VancouverUniversity of British Columbia,Principal Investigator of the TITAN Study.

Dr. Kim Chi

Dr. Kim Chi. MD
Professor of Medicine
Medical Oncologist and Medical Director at BC Cancer – Vancouver
University of British Columbia,
Principal Investigator of the TITAN Study.

MedicalResearch.com: What is the background for this study?

Response: For more than 70 years, androgen deprivation therapy (ADT) has been the standard of care therapy for patients with metastatic prostate cancer. The Phase 3 TITAN study looked at adding apalutamide (®®®®) to ADT compared with placebo plus ADT in a broad group of patients with metastatic castration-sensitive prostate cancer (mCSPC), regardless of disease volume or prior docetaxel treatment history.

Metastatic castration-sensitive prostate cancer is prostate cancer that still responds to androgen deprivation therapy and has spread to other parts of the body. Patients with mCSPC tend to have a poor prognosis, with a median overall survival (OS) of less than five years, underscoring the need for new treatment options. The dual primary endpoints of this study were overall survival and radiographic progression-free survival (rPFS).  Continue reading

Myrbetriq® (mirabegron) Improved BPH Urinary Symptoms in Men Already on Tamsulosin (Flomax)

MedicalResearch.com Interview with:

Steven A. Kaplan, M.D., FACS Professor of Urology Director, The Men's Health Program Icahn School of Medicine at Mount Sinai

Dr. Kaplan

Steven A. Kaplan, M.D., FACS
Professor of Urology
Director, The Men’s Health Program
Icahn School of Medicine at Mount Sinai

MedicalResearch.com: What is the background for this study?

Response: PLUS is the first large-scale trial conducted in North America and Europe specifically designed to study the effects of mirabegron in controlling residual symptoms of urinary urgency and frequency in men with benign prostatic hyperplasia (BPH) using common agents such as tamsulosin (Flomax).

We explored whether mirabegron (Myrbetriq), an agent approved for the treatment of overactive bladder (OAB), improved patient outcomes when added to tamsulosin. This was a randomized, double-blind, placebo-controlled, multi-center study enrolling 715 male patients 40 years of age and older.

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Systemic Sclerosis (scleroderma): Nintedanib Slowed Loss of Pulmonary Function

MedicalResearch.com Interview with:

Donald Zoz, M.D.,Senior associate directorClinical Development & Medical AffairsBoehringer Ingelheim Pharmaceuticals, Inc.

Dr. Zoz

Donald Zoz, M.D.,
Senior associate director
Clinical Development & Medical Affairs
Boehringer Ingelheim Pharmaceuticals, Inc.

MedicalResearch.com: What is the background for this study? How does nintedanib differ from other treatments for SSc-ILD? What are the main findings? 

Response: SENSCIS is a Phase III double-blind, randomized placebo-controlled trial that included 576 patients in 32 countries. It is the largest trial to have been conducted in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD). The primary endpoint was the annual rate of decline in forced vital capacity (FVC) over 52 weeks. At the end of the 52-week trial, patients receiving nintedanib had an adjusted annual rate of decline in FVC (mL/year) of -52.4 with nintedanib versus -93.3 with placebo (absolute difference 41.0mL/year [95% CI 2.9, 79.0]; p=0.04). This corresponds to a relative difference of 44% reduction in lung function decline.

There are currently no approved treatments for SSc-ILD., BI conducted the SENSCIS study to evaluate in SSc-ILD patients the impact of nintedanib. Nintedanib, a selective tyrosine kinase inhibitor, is an antifibrotic agent.

Results of the study, which were published in The New England Journal of Medicine and presented at the American Thoracic Society (ATS) International Conference, showed that nintedanib slowed the loss of pulmonary function by 44% in patients with SSc-ILD relative to placebo, as measured by FVC over 52 weeks. 

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Cariprazine Demonstrated Efficacy in Bipolar Depression and Concurrent Manic Symptoms

MedicalResearch.com Interview with:

Dr. Stephen Stahl MD PhDProfessor of Psychiatry University of California San Diego

Dr. Stahl

Dr. Stephen Stahl MD PhD
Professor of Psychiatry
University of California San Diego 

MedicalResearch.com: What is the background for this study? How does cariprazine differ from other medications for bipolar depression? 

MedicalResearch.com: It is important to note that cariprazine, a dopamine D3-preferring D3/D2 receptor and serotonin 5-HT1A receptor partial agonist, is approved for the treatment of schizophrenia (1.5-6 mg/d) and bipolar mania (3-6 mg/d) in adults. It is not yet approved for depressive episodes related to bipolar I disorder (bipolar depression).

In these data that focus on the investigational use for the treatment of bipolar depression, cariprazine has demonstrated efficacy vs placebo (PBO) in 3 phase 2/3 studies of patients with bipolar depression (NCT01396447, NCT02670538, NCT02670551). These analyses investigated the efficacy of cariprazine in patients with bipolar depression and concurrent manic symptoms (mixed features).  Continue reading

FDA Approves EYLEA (aflibercept) To Treat All Stages of Diabetic Retinopathy, With Two Dosing Options

MedicalResearch.com Interview with:

Blausen.com staff (2014). "Medical gallery of Blausen Medical 2014". WikiJournal of Medicine 1 (2). DOI:10.15347/wjm/2014.010. ISSN 2002-4436. Illustration depicting diabetic retinopathy

Illustration depicting diabetic retinopathy


Robert L. Vitti, MD, MBA
Vice President and Head, Ophthalmology
Regeneron Pharmaceuticals

Dr. Vitti discusses the recent announcement that the FDA has approved EYLEA to treat all stages of diabetic retinopathy.

MedicalResearch.com: Can you provide additional background on this approval? Would you briefly explain diabetic retinopathy and it’s impact on patients?

Response: The FDA has approved EYLEA (aflibercept) Injection to treat all stages of diabetic retinopathy (DR). DR is the leading cause of blindness among working-aged American adults. Approximately 8 million people live with DR, a complication of diabetes characterized by damage to the blood vessels in the retina (per 2010 data).

The disease generally starts as non-proliferative diabetic retinopathy (NPDR) and often has no warning signs or symptoms. Over time, NPDR often progresses to proliferative diabetic retinopathy (PDR), a stage in which abnormal blood vessels grow on the surface of the retina and into the vitreous cavity, potentially causing severe vision loss.

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Angion Receives Support to Develop Drug Candidate for a Scarring Form of Kidney Disease, FSGS

MedicalResearch.com Interview with:

Dr. Jay Venkatesan MDPresident and CEO of Angion

Dr. Venkatesan

Dr. Jay Venkatesan MD
President and CEO of Angion

Dr. Venkatesan discusses the recent announcement that ANGION, has received DOD funding for the study of ANG-3070, in treatment of CKD caused by focal segmental glomerulosclerosis, 

MedicalResearch.com: What is the background for this announcement? Would you tell us a little about focal segmental glomerulosclerosis (FSGS)? How does ANG-3070 work to prevent kidney scarring?

Response: Angion has received a follow-on grant from the Department of Defense (DoD) for $4.76 million in support of the development of ANG-3070, our drug candidate for a form of chronic kidney disease known as focal segmental glomerulosclerosis (FSGS). This funding will allow us to expand our proof-of-concept data for ANG-3070 as a potential anti-fibrotic agent for slowing the progression of FSGS.

FSGS is a serious kidney disorder characterized by progressive scarring of the glomeruli, the filtering units of the kidney. There are approximately 80,000 cases of FSGS in the U.S. and Europe, involving both children and young adults. If uncontrolled, FSGS can lead to kidney failure, which may lead to the need for dialysis or a kidney transplant. No therapies exist that treat the underlying cause of FSGS. Therapies such as corticosteroids, immunosuppressants or diuretics  are used, but are mainly supportive and a large proportion of patients progress to end-stage renal disease over a 5-10 year period of time.

ANG-3070 is an oral small molecule that selectively inhibits molecular pathways associated with scarring or fibrosis in the kidney and other organs. Our current preclinical study in collaboration with NEPTUNE aims to identify the “signalosome,” or human disease and drug response profile based on the genes, networks and pathways that correlate with the therapeutic activity of ANG-3070 in FSGS. Ultimately, this collaboration will allow us to  develop a precision medicine approach to identify and treat patients in whom ANG-3070 is most likely to block the pathways causing FSGS. 

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ASPECT-NP: Randomized, Double-Blind, Phase III Trial Comparing Efficacy & Safety of Ceftolozane/Tazobactam vs Meropenem in Ventilated Nosocomial Pneumonia

MedicalResearch.com Interview with:

Dr. Elizabeth Rhee MD Director, Infectious Disease Clinical Research at Merck

Dr. Rhee

Elizabeth Rhee, MD
Executive Director, Infectious Disease Clinical Research
Merck Research Laboratories

MedicalResearch.com: What is the background for this study? Would you briefly explain the condition of ventilated nosocomial pneumonias?

Dr. Rhee: Nosocomial pneumonia (NP) is a lung infection that occurs during a hospital stay. NP is often serious, and is associated with high mortality. It is one of the most common health-care associated infections in both the U.S. and Europe, accounting for over 20% of such cases. Gram-negative bacteria, mainly Pseudomonas aeruginosa (PSA) and Enterobacteriaceae, are frequent causes of nosocomial pneumonia. Limited options currently exist for the management of NP caused by Gram-negative pathogens. This is concerning because rates of resistance to Gram-negative bacteria are growing, and they are becoming increasingly difficult to treat.

Forms of nosocomial pneumonia include hospital-acquired pneumonia (HAP), ventilator-associated pneumonia (VAP), and ventilated HAP. High rates of death (ranging from 20% to more than 50%) are especially associated with ventilated HAP. Pseudomonas aeruginosa, a Gram-negative bacterium, is the most common cause of HAP/VAP in both the U.S. and Europe. Patients with NP are often critically ill, requiring ventilator support and time in intensive care, and it was important to look at this population as we explore new options for the treatment of NP.

Ceftolozane/tazobactam (C/T) is an antipseudomonal cephalosporin/beta-lactamase inhibitor combination with broad in vitro activity against Gram-negative pathogens, including multi-drug resistant (MDR) P. aeruginosa and many extended-spectrum beta-lactamase (ESBL) producers. It is FDA approved for complicated intra-abdominal and urinary tract infections in adults at 1.5g (1g ceftolozane/0.5g tazobactam) q8h. C/T is currently being studied at an investigational new dose of 3g (2g/1g) q8h, for the treatment of ventilated nosocomial pneumonia, in the ASPECT-NP Phase 3 trial.

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Verubecestat Failed to Slow Progression of Early Alzheimer’s Disease

MedicalResearch.com Interview with:
Michael F. Egan, MDVice President,  NeuroscienceGlobal Clinical DevelopmentMerck Research LaboratoriesNorth Wales, PAMichael F. Egan, MD
Vice President,  Neuroscience
Global Clinical Development
Merck Research Laboratories
North Wales, PA 

MedicalResearch.com: What is the background for this study?  

Response: Alzheimer’s disease (AD) appears to be due to the gradual accumulation of amyloid over many years (the “amyloid hypothesis”). At some point, it is thought that amyloid triggers abnormalities in tau, which then forms deposits within neurons and leads to progressive neurodegeneration.

Amyloid is made up of  a small, sticky peptide, Abeta, which is produced when the enzyme BACE cleaves a large protein called APP.  In our trial, we tested whether a potent BACE inhibitor, verubecestat, could slow disease progression in subjects with early AD (or prodromal AD) by blocking formation of Abeta.  A previous trial in subjects with dementia due to AD failed to find evidence of efficacy.

One possible reason for this failure is that subjects had too much amyloid in their brain already.

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Genentech Submits New Drug Application to FDA for Venclexta Plus Gazyva for Untreated CLL with Co-Existing Medical Conditions

MedicalResearch.com Interview with:

Nancy Valente, M.D.VP of Global Hematology DevelopmentGenentech

Dr. Valente

Nancy Valente, M.D.
VP of Global Hematology Development
Genentech

Dr. Valenta discusses the announcement of the submission by Genentech of a supplemental New Drug Application to the FDA for Venclexta plus Gazyva for people with previously untreated chronic lymphocytic leukemia  with co-existing medical conditions.

MedicalResearch.com: What is the background for this study?
What are the main findings of the Phase III CLL14 study? 

Response: We completed the submission of a supplemental New Drug Application (sNDA) to the FDA for Venclexta® (venetoclax) in combination with Gazyva® (obinutuzumab) in people with previously untreated chronic lymphocytic leukemia (CLL) and co-existing medical conditions. CLL is the most common form of adult leukemia and more than 20,000 new cases will be diagnosed in the U.S. this year.

The sNDA is based on data from the Phase III CLL14 study, which evaluated fixed-duration Venclexta in combination with Gazyva in people with previously untreated CLL. Results showed this chemotherapy-free combination can help people with previously untreated CLL live significantly longer without their disease worsening (progression-free survival; PFS) compared to standard-of-care Gazyva plus chlorambucil.

The FDA is reviewing our application under the Real-Time Oncology Review (RTOR) pilot program, which is exploring a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.  Continue reading

BELVIQ®: FDA accepts sNDA To Include Long Term Safety/Efficacy Data

WeightControl.com Interview with:

Dr. Lynn Kramer, MD FAANVP and Chief Clinical Officer & Chief Medical OfficeEisai Co., Ltd

Dr. Kramer

Dr. Lynn Kramer, MD FAAN
VP and Chief Clinical Officer & Chief Medical Office
Eisai Co., Ltd

WeightControl.com: What is the background for this announcement?

Response: On February 25th, Eisai announced that the U.S. Food and Drug Administration (FDA) accepted its supplemental New Drug Application to potentially update the label for BELVIQ® (lorcaserin HCI) CIV 10 mg twice-daily/BELVIQ XR (lorcaserin HCI) CIV once daily to include long-term efficacy and safety data from CAMELLIA-TIMI 61, a clinical trial of BELVIQ in 12,000 overweight and obese patients with cardiovascular (CV) disease and/or multiple CV risk factors such as type 2 diabetes mellitus (T2DM).

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Risankizumab for Moderate to Severe Psoriasis: High Rates of Durable Clearance Through One Year

MedicalResearch.com Interview with:

Anne Robinson, Pharm DExecutive Scientific DirectorAbbVie

Dr. Robinson

Anne Robinson, Pharm D
Executive Scientific Director
AbbVie

MedicalResearch.com: What is the background for the risankizumab data presented at the American Academy of Dermatology 2019 Annual Meeting?

Response: Abstracts presented by AbbVie at the American Academy of Dermatology (AAD) 2019 Annual Meeting highlight additional data from the Phase 3 clinical trial program evaluating the safety and efficacy of risankizumab, an investigational interleukin-23 (IL-23) inhibitor. The registrational program for risankizumab evaluated more than 2,000 adult patients with moderate to severe plaque psoriasis across four pivotal studies. Continue reading

Oracea® Capsules + Soolantra Cream Effective for Inflammatory Rosacea

MedicalResearch.com Interview with:

Dr. James Q. Del Rosso, D.O., FAOCD, DermatologistResearch Director and Principal InvestigatorDel Rosso Dermatology Research Center, Las Vegas, NVGalderma Consultant

Dr. Del Rosso

Dr. James Q. Del Rosso, D.O., FAOCD, Dermatologist
Research Director and Principal Investigator
Del Rosso Dermatology Research Center, Las Vegas, NV
Galderma Consultant

MedicalResearch.com: What is the background for this study? What are the main findings?

  • The ANSWER study, a 12-week, randomized, multicenter, Phase 4, Phase 3b in Canada and Europe clinical trial, is the first study of its kind to compare the efficacy and safety of combination therapy with Oracea® (doxycycline, USP) 40 mg Capsules + Soolantra® (ivermectin) Cream, 1% versus Soolantra® (ivermectin) Cream, 1% monotherapy in 273 adults with severe papulopustular rosacea (IGA 4) at clinical trial sites in the United States, Canada and Europe (Czech Republic, Poland, Hungary and Germany).
  • Results showed the combination therapy with Oracea Capsules + Soolantra Cream was well tolerated and effective with a faster onset of action than Soolantra Cream given as monotherapy. Key highlights of the study include:
  • The mean reduction in percentage of inflammatory lesions from baseline to Week 12 was significant with combination therapy compared to monotherapy (80.29% vs. 73.56%, respectively; p=0.032).
  • 5 times as many patients taking combination therapy achieved 100% clearance of inflammatory lesions by Week 12 compared with monotherapy (17.8% vs. 7.2%, respectively; p=0.006).
  • Over 2 times as many patients taking combination therapy achieved 100% clear (IGA 0) by Week 12 compared with monotherapy (11.9% vs. 5.1%, respectively; p=0.043).
  • Combination therapy was generally well tolerated and no discontinuation of treatments due to side effects.

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Lilly Announces Phase 3 Results for Atopic Dermatitis Treatment

MedicalResearch.com Interview with:

Lotus Mallbris, MD PhD Vice President, Head of Global Immunology Drug Development Platform Team Leader at Lilly

Dr. Mallbris

Lotus Mallbris, MD PhD
Dermatologist and Vice President, Head of Global Immunology Drug Development
Platform Team Leader at Lilly

MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by atopic dermatitis? How common is this condition? 

Response:The BREEZE-AD1 and BREEZE-AD2 clinical trials are multicenter, randomized, double-blind, placebo-controlled, Phase 3 studies to evaluate the efficacy and safety of baricitinib monotherapy in adult patients with moderate to severe atopic dermatitis. These are two of five studies that will be part of the placebo-controlled data program intended to support global registrations.

Atopic dermatitis, a serious form of eczema, is a chronic, relapsing skin disease characterized by intense itching, dry skin and inflammation that can be present on any part of the body. It affects approximately 1-3 percent of adults worldwide.

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Single-Dose Tafenoquine to Prevent Malaria Relapse

MedicalResearch.com Interview with:
Malaria CDC imageGavin C.K.W. Koh, MB BChir MA PhD MCRP DTM&H

Department of Drug Discovery Unit for Diseases of the Developing World
GlaxoSmithKline | GSK

MedicalResearch.com: What is the background for this study?

Response: Malaria still remains one of the greatest global healthcare challenges so, as part of GSK’s efforts to fight diseases that disproportionately impact the poorest, we have been working on tafenoquine as a potential medicine for malaria for over 20 years.  In 2008, GSK entered into a collaboration with the not-for-profit product development partnership, Medicines for Malaria Venture (MMV), to develop tafenoquine as an anti-relapse medicine for patients infected with a particular species of malaria called Plasmodium vivax malaria.

  1. vivaxmalaria is estimated to cause around 7.5 million clinical infections every year. The disease may cause fever, chills, vomiting, malaise, headache and muscle pain, and in some cases, can lead to severe malaria and be fatal.

Unlike other malaria species such as P. falciparumP. vivax also has the ability to lie dormant in the liver from where it may periodically reactivate to cause relapses of P. vivax malaria. A single P. vivax infection may therefore give rise to multiple episodes of malaria, in the absence of a new mosquito bite. These relapses can occur weeks, months or even years after the initial infection. The dormant liver forms of the parasite cannot be treated with most other antimalarial treatments.

Another issue is that the only medicine currently available to stop the relapse is primaquine, a medicine approved in the 1950s, which must be given for 14 days. Given this length of treatment course, many people do not comply with the full course, which results in reduced effectiveness.

The aim of the DETECTIVE study was to look at the effectiveness of treatment in preventing relapse over six months with a 1-day course of tafenoquine, a 14-day course of primaquine, or placebo, with all patients also receiving a 3-day course of chloroquine, a medicine that is used to treat the initial infection.

MedicalResearch.com: What are the main findings?

Response: The DETECTIVE study met its primary endpoint. A significantly greater proportion of patients in the tafenoquine group did not have relapses compared to patients in the placebo group. A similar result was observed for the patients in the primaquine group compared to the placebo group. When considering the compliance issue of primaquine in the real-world setting, we saw that more than 95% of patients in the primaquine group took their treatment as instructed in the setting of a clinical study. From a safety perspective, adverse events from the study were consistent with the known safety profile of tafenoquine.

MedicalResearch.com: What should readers take away from your report?

Response: The positive results of the DETECTIVE study demonstrate the efficacy and safety of tafenoquine in an unprecedented single-dose for relapsing malaria.  These data supported the approval of the medicine by the US Food and Drug Administration and Australia Therapeutic Goods Administration in 2018 marking it as the first new medicine for the radical cure of P. vivax malaria in more than 60 years. Having the data published in the NEJM will help other P. vivax malaria endemic countries as they strive towards malaria elimination. 

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: The DETECTIVE study was conducted in adult patients with P. vivax malaria and a significant part of the global burden of the disease is in children. GSK and MMV are therefore studying the use of tafenoquine in paediatric patients aged 6 months to 16 years (TEACH study).  In addition, since vivax malaria in Indonesia is frequently resistant to chloroquine, we are conducting another study that is looking at tafenoquine plus dihydroartemisinin–piperaquine as the blood schizonticide (INSPECTOR study) in patients with P. vivax malaria in Indonesia.

Disclosures: The DETECTIVE study was supported by GSK and MMV.

Citation:

Single-Dose Tafenoquine to Prevent Relapse of Plasmodium vivax Malaria
Marcus V.G. Lacerda, M.D., Alejandro Llanos-Cuentas, M.D., Srivicha Krudsood, M.D., Chanthap Lon, M.D., David L. Saunders, M.D., Rezika Mohammed, M.D., Daniel Yilma, M.D., Dhelio Batista Pereira, M.D., Fe E.J. Espino, M.D., Reginaldo Z. Mia, M.D., Raul Chuquiyauri, M.D., Fernando Val, Ph.D., et al.

January 17, 2019
N Engl J Med 2019; 380:215-228
DOI: 10.1056/NEJMoa1710775

[last-modified] 

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Marketers Spend At Least Six Times FDA Budget on Promotion of Medical Services

MedicalResearch.com Interview with:
Steven Woloshin, MD, MS Professor Co-director of the Center for Medicine and Media The Dartmouth InstituteSteven Woloshin, MD, MS
Professor
Co-director of the Center for Medicine and Media
The Dartmouth Institute

MedicalResearch.com: What is the background for this study? What are the main findings? What influence does medical marketing have on medical care and drug prices?

Response: There are published studies looking at promotional spending mostly for drugs (DTC and professional).  This paper is unique because it is such a broad look including not just drugs but also marketing of disease (in “awareness campaigns”), health services and laboratory tests.

What is new here is the size and scope of marketing.  For context, $29.9 billion spent on promoting prescription drugs, disease awareness campaigns, health services, and laboratory tests corresponds approximately to $1000 per American.    For context, FDA’s total budget is around $5 billion – and NIH’s total budget is about $30 billion.

This figure is up from $17.7 billion in 1997, with the most rapid increase in DTC promotion of prescription drugs and health services.   Pharmaceutical marketing to professionals (detailing visits and samples) accounted for most spending and remained high despite policies to limit industry influence.

$30 billion is of an underestimate (egg, we did not include monies spent on professional marketing (detailing) of laboratory tests, health services or devices, the value of drug coupons/discounts/rebates, company marketing budgets, lobbying or campaign contributions).

Further it is just the tip of the iceberg – marketing works so promotional spending is an important driver of why medical care is so expensive:  it leads to more – and more expensive – tests and treatments.

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XARELTO® Associated With a Decreased Risk of Recurrent VTE

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

Paul Burton MD, PhD, FACC
Vice President, Medical Affairs
Internal Medicine
Janssen Scientific Affairs, LLC.

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: More than 900,000 Americans experience a venous thromboembolism (VTE) each year, with about one-third of these occurrences being fatal. Once a person experiences a VTE, they are at increased risk of a repeat occurrence. Guidelines currently recommend standard anticoagulant therapy with a Factor Xa inhibitor, like XARELTO® (rivaroxaban), for three months or longer. For those people who have had a VTE and stop anticoagulant therapy, as many as 10 percent of them will experience another VTE within one year and 20 percent within three years.

This study examined extended use of XARELTO® after the recommended three-month treatment period in patients who experienced an initial VTE, showing XARELTO® was associated with a decreased risk of recurrent VTE with no increase in major bleeding during this time period.  Continue reading

Healthcare Costs in Patients with Cancer Rise with Increasing Risk of Venous Thromboembolism

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

Paul Burton MD, PhD, FACC
Vice President, Medical Affairs
Internal Medicine
Janssen Scientific Affairs, LLC.

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Despite being largely preventable, venous thromboembolism (VTE) is the second leading cause of death in people with cancer. The risk of VTE is five times greater in people with cancer than those without cancer, and that risk is magnified in those receiving certain types of chemotherapy, in the newly diagnosed and in those with more advanced, metastatic disease. This 6,194-patient study examined economic burden associated with VTE, and found patients newly diagnosed with cancer who are at a higher risk of a VTE had significantly higher all-cause and VTE-related health care costs compared to patients with a lower risk of VTE. Continue reading

Lower Overall Costs with Rivaroxaban (XARELTO® ) vs Warfarin Among Morbidly Obese Patients with Venous Thromboembolism

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

Paul Burton MD, PhD, FACC
Vice President, Medical Affairs
Internal Medicine
Janssen Scientific Affairs, LLC.

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Treatment of venous thromboembolism (VTE) is complicated among morbidly obese patients. Current guidelines do not recommend use of Factor Xa inhibitors in these patients due to limited clinical data available. That’s why Janssen undertook this study to examine XARELTO® (rivaroxaban) in these patients. In this 5,780-patient retrospective study, results found patients treated with XARELTO® had a similar risk of recurrent VTE and major bleeding compared to those taking warfarin.

However, treatment with XARELTO® was associated with less all-cause health care resource utilization (HCRU) (e.g., inpatient hospitalizations and outpatient visits) and reduced total medical costs compared to warfarin. Of note, patients taking XARELTO® had an average $2,829 lower total medical costs per patient per year (PPPY) than those taking warfarin, which was mainly driven by lower hospitalization costs. Continue reading