Author Interviews, COVID -19 Coronavirus, Pharmaceutical Companies, Vaccine Studies / 17.03.2020

MedicalResearch.com Interview with: [caption id="attachment_53541" align="alignleft" width="180"]Nathalie Charland PhD Senior Director, Scientific and Medical Affairs Medicago  Dr. Machelska[/caption] Nathalie Charland PhD Senior Director, Scientific and Medical Affairs Medicago  MedicalResearch.com: What is the background for this study? What are the main findings? Response: We started to work on solutions as soon as we were able to obtain the appropriate genetic information for the new COVID-19. Medicago is committed to advancing therapeutics against life-threatening diseases worldwide. 
Author Interviews, Cost of Health Care, JAMA, Pharmaceutical Companies / 04.03.2020

MedicalResearch.com Interview with: [caption id="attachment_53363" align="alignleft" width="200"]Olivier Wouters, Ph.D. Assistant Professor of Health Policy Department of Health Policy (COW 2.06) London School of Economics and Political Science Dr. Wouters[/caption] Olivier Wouters, Ph.D. Assistant Professor of Health Policy Department of Health Policy (COW 2.06) London School of Economics and Political Science MedicalResearch.com: What is the background for this study? What are the main findings? Response: Drug companies often point to high research and development costs as justification for the rising prices of new medicines. Yet most prior analyses of research and development costs have been based on confidential data voluntarily supplied by drug companies to researchers with financial ties to the industry. Independent teams have not been able to verify those findings.
AHA Journals, Author Interviews, Pharmaceutical Companies, Stroke / 21.02.2020

MedicalResearch.com Interview with: [caption id="attachment_53245" align="alignleft" width="143"]Michael Tymianski, CM, MD, PhD, FRCSC, FAHA Head, Division of Neurosurgery, University Health Network Medical Director, Neurovascular Therapeutics Program, University Health Network Professor, Departments of Surgery and Physiology, University of Toronto Senior Scientist, Toronto Western Hospital Research Institute Director, Neuroprotection Laboratory, Toronto Western Hospital President and CEO, NoNO Inc Dr. Tymianski[/caption] Michael Tymianski, CM, MD, PhD, FRCSC, FAHA Head, Division of Neurosurgery, University Health Network Medical Director, Neurovascular Therapeutics Program, University Health Network Professor, Departments of Surgery and Physiology, University of Toronto Senior Scientist, Toronto Western Hospital Research Institute Director, Neuroprotection Laboratory, Toronto Western Hospital President and CEO, NoNO Inc MedicalResearch.com: What is the background for this study? How is alteplase related to and affect nerinetide? Response: Cerebral neuroprotection for acute ischemic stroke (AIS) is defined as a therapy aimed at enhancing the brain’s resilience to ischemia to improve the clinical outcome of affected individuals. Although traditionally aimed at the salvage of neurons, this term may be equally applicable to all the cellular constituents of the brain, including cells of cerebral blood vessels, neurons, and glia. Pharmacological neuroprotection (hereafter referred to as neuroprotection) would be achieved by drugs targeting one or more critical components of the ischemic cascade that lead to ischemic damage. The feasibility of neuroprotection has a strong basis in animal experiments, but research for several decades has failed to translate neuroprotective treatments from animals to humans. The disappointing results of all controlled clinical neuroprotection trials for AIS have cast doubts as to whether neuroprotection in humans is biologically possible and, given the complexities of human stroke syndromes, whether it is a clinically practicable therapy for patients experiencing AIS in the community. In the case of neuroprotection trials for acute ischemic stroke, all to date have failed to demonstrate a clinical benefit of the study agent. Our review of studies since the year 2000 shows that many were not conducted in accordance with the animal studies that supported efficacy. They enrolled a heterogeneous subject population with varying (small and large) vessel occlusions and without knowledge of the degree of completed infarctions. Most had not implemented a strategy to ensure that the treatment effect size was maximized, and all in-hospital trials enrolled in treatment windows that exceeded 4 hours, at which an important proportion of enrolled subjects cannot respond to treatment because they no longer have salvageable brain. The ESCAPE-NA1 addressed past deficiencies of AIS trials. It was based on a sound scientific foundation including extensive animal studies, and capitalized on the designs that led to success in AIS trials of endovascular thrombectomy. ESCAPE-NA1 enrolled patients proven by these past trials to have salvageable brain at the time that the treatment was given, and tested the drug in an ischemia-reperfusion scenario in which it was anticipated to be most effective. The enrollment was over 12 hours, but only including patients who had medical imaging suggestive that they still had salvageable brain. Alteplase is an agent that activates the protease plasmin in the bloodstream. Plasmin cleaves peptides at certain spots within their structure, and it is a known biological fact that plasmin is able to cleave nerinetide. What was not known at the time of the trial was the degree to which this would reduce nerinetide plasma leves in humans, and the impact that this would have on the therapeutic effects of nerinetide. This is why we conducted a very large trial in which the participants’ enrollment was stratified according to whether or not they received alteplase. This ensured that there was good balance within each stratum, thereby enabling us to make more robust conclusions.
Author Interviews, Endocrinology, Pharmaceutical Companies, Testosterone / 18.02.2020

MedicalResearch.com Interview with: [caption id="attachment_53201" align="alignleft" width="200"]Robert E. Dudley, Ph.D. Chairman, Chief Executive Officer and President Clarus Therapeutics Dr. Dudley[/caption] Robert E. Dudley, Ph.D. Chairman, Chief Executive Officer and President Clarus Therapeutics Dr. Dudley discusses the recent announcement that Clarus Therapeutics, Inc. has launched  JATENZO® (testosterone undecanoate) capsules for the treatment of appropriate men with testosterone deficiency (hypogonadism): MedicalResearch.com: What is the background for this announcement? Response: JATENZO® is the first and only oral softgel testosterone undecanoate and the first oral testosterone product approved by the U.S. FDA in more than 60 years. JATENZO is indicated for testosterone replacement therapy in adult males for conditions associated with a deficiency or absence of endogenous testosterone. The launch of JATENZO means that physicians and men living with testosterone deficiency due to genetic or structural abnormalities finally have a safe and effective oral testosterone replacement therapy. We are proud to commercially launch this unique oral formulation to healthcare providers and the appropriate patients who they treat. JATENZO is now available at pharmacies across the country.
ASCO, Author Interviews, Bayer, Cancer Research / 13.02.2020

MedicalResearch.com Interview with: [caption id="attachment_53162" align="alignleft" width="140"]Dr. Hong Dr. Hong[/caption] Dr. David S. Hong MD Deputy Chair Department of Investigational Cancer Therapeutics Division of Cancer MedicineThe University of Texas MD Anderson Cancer Center Houston, TX    MedicalResearch.com: What is the background for this study? What are the main findings?
  • A rare genomic alteration called a neurotrophic receptor tyrosine kinase (NTRK) gene fusion is a primary oncogenic driver that causes TRK fusion cancer, which has been found in a variety of common tumor types, including GI cancers such as colon, cholangiocarcinoma, pancreatic, and appendiceal cancers. In patients with gastrointestinal (GI) cancer, including pancreatic cancer and colorectal cancer, NTRK gene fusions are estimated to have a frequency of ~0.3%.
  • Larotrectinib is an oral and highly selective TRK inhibitor used for the treatment of adult and pediatric patients with solid tumors that have an NTRK gene fusion. Under the brand name Vitrakvi®, it is the first and only approved TRK inhibitor exclusively designed to treat tumors with an NTRK gene fusion with approval in the US in 2018 and other worldwide markets in 2019.
  • At ASCO GI 2020, we presented results of a new analysis of the efficacy and safety of larotrectinib specifically in patients with TRK fusion with gastrointestinal cancers, which is an often underdiagnosed patient group. The subset included 14 adult patients with GI tumor types with NTRK gene fusions, including colon, cholangiocarcinoma, pancreas, appendix and hepatic; of the eight patients with colon cancer, seven were microsite instability (MSI)-high.
  • In this subset of patients, the overall response rate (ORR) was 43%. Additionally, median overall survival was 33.4 months at 19 months of follow-up (range 2.8–36.5), median progression-free free survival (PFS) was 5.3 months (range 2.2-9.0) and median time to response was 1.8 months (range 1.7-2.1). In colon cancer patients, the ORR was 50% and the median PFS ranged from 1.5+ to 16.7+ months. 
ASCO, Author Interviews, Bayer, Cancer Research / 11.02.2020

MedicalResearch.com Interview with: bayer-pharmaceuticalsDr. Kirhan Ozgurdal Global Medical Affairs Physician Oncology, Bayer MedicalResearch.com: What is the background for this study? What are the main findings?
  • Regorafenib is an oral multi-kinase inhibitor that potently blocks multiple protein kinases involved in tumor angiogenesis, oncogenesis, metastasis and tumor immunity. It is approved for the treatment of patients with hepatocellular carcinoma (HCC) who have previously been treated with sorafenib. The safety and effectiveness of regorafenib is being evaluated in patients with unresectable hepatocellular carcinoma (uHCC),a liver tumor not eligible for curative treatment approaches such as surgery, given the extent of disease.
  • Following the Phase 3 RESORCE trial, which showed that regorafenib significantly improves overall survival versus placebo in patients with uHCC who progressed on prior sorafenib therapy, we conducted an interim analysis (the first 500 of 1000 patients) of the global REFINE observational trial to evaluate the safety and effectiveness of regorafenib in uHCC in the real-world setting.
  • The REFINE study shows a more varied patient population than the Phase 3 RESORCE trial, including a higher proportion of patients with ECOG performance status ≥1, and a higher proportion with Child–Pugh B liver function.
  • The incidence of regorafenib-related grade ≥3 treatment-emergent adverse events were lower than that reported in the RESORCE trial, possibly indicating improved adverse event management with the use of regorafenib in clinical practice.
  • The median overall survival was longer than that reported in RESORCE, but the proportion of censored patients was high in this interim analysis; the median progression free survival was similar to that reported in RESORCE.
Author Interviews, Dartmouth, JAMA, Pharmaceutical Companies, Primary Care / 27.01.2020

MedicalResearch.com Interview with: [caption id="attachment_52969" align="alignleft" width="147"]Steven Woloshin, MD, MS Professor of Medicine and Community and Family Medicine Professor, The Dartmouth Institute for Health Policy and Clinical Practice Prof. Woloshin[/caption] Steven Woloshin, MD, MS Professor of Medicine and Community and Family Medicine Professor, The Dartmouth Institute for Health Policy and Clinical Practice MedicalResearch.com: What is the background for this study? Response: Industry spends more on detailing visits and free samples than any other form of prescription drug marketing.  There is good evidence that these activities can lead to more use of expensive new drugs over equally effective cheaper options.  Given these concerns there have been efforts by some hospitalls and practices to restrict these forms of marketing. We asked physicians in group practices delivering primary care about how often pharmaceutical reps visit their practice and whether they have a free sample closet. 
Abbvie, Author Interviews, NEJM, OBGYNE / 23.01.2020

MedicalResearch.com Interview with: [caption id="attachment_52895" align="alignleft" width="150"]William D Schlaff MD Chair, Department of Obstetrics & Gynecology Jefferson University Dr. Schlaff[/caption] William D Schlaff  MD Chair, Department of Obstetrics & Gynecology Jefferson University MedicalResearch.com: What is the background for this study? Response: Symptomatic uterine fibroids are the most common indication for hysterectomy in the US.  Heavy bleeding is the most common and troublesome symptom.  The primary treatment for this problem is surgery—either hysterectomy or (less commonly) myomectomy.  Medical treatment which reduces the bleeding related to fibroids without surgery is a valuable treatment for many women.  Existing medications include, most commonly GnRH agonists.  These are injectable medications that are given every 1 or 3 months (depending on the formulation) and have been shown to reduce bleeding related to fibroids.  They work by initially stimulating the ovaries to increase estrogen levels for 10-14 days before suppressing estrogen and thereby reducing bleeding.  Even though the medication is given every 1 or 3 months, the effect of the medication can last quite a bit longer; in cases of adverse response, the medication cannot be immediately stopped.  The medication reported in this trial, Elagolix, is a GnRH antagonist given by mouth twice daily and resulting in suppression of estrogen secretion within a matter of hours.  The effect of this medication wears off much more rapidly than the depot formulations described and can be stopped in the uncommon cases of adverse side effects. 
Abbvie, Author Interviews, Rheumatology / 27.11.2019

MedicalResearch.com Interview with: [caption id="attachment_52304" align="alignleft" width="200"]Aileen Lorenzo Pangan MD Executive Medical Director AbbVie Dr. Pangan[/caption] Aileen Lorenzo Pangan MD Executive Medical Director AbbVie MedicalResearch.com: What is the background for this study? Response: Our ongoing commitment to advancing the standard of care for patients with rheumatic diseases is illustrated by our growing portfolio and the thirty-eight abstracts we presented at this year's ACR/ARP Annual Meeting, including results from five studies of RINVOQ in rheumatoid arthritis (RA) and primary results from our study in ankylosing spondylitis (AS).
Author Interviews, Cancer Research, Genetic Research, Pharmaceutical Companies / 26.11.2019

MedicalResearch.com Interview with: Ambry GeneticsRachid Karam, MD PhD Director, Ambry Translational Genomics Lab Ambry Genetics MedicalResearch.com: What is the background for this study? Response: Standard DNA testing for hereditary cancer risk excludes large portions of DNA, thereby missing some mutations. In addition, DNA testing can produce inconclusive results and fail to determine that an error in our DNA increases cancer risk. These limitations impact patients and their families because doctors may not have the information needed to recommend appropriate preventive, early detection, or therapeutic steps. Additionally, relatives may not be referred for genetic testing and obtain the care they would otherwise have gotten if they had learned they had mutations. The study looked at how the addition of RNA genetic testing to standard DNA testing for hereditary cancer risk was able to increase diagnostic yield. The study looked at the first 2,500 patients that received Ambry Genetics +RNAinsight™, paired RNA and DNA genetic testing for hereditary cancer risk. The data from this study showed that the addition of RNA genetic testing to DNA testing (1) identified new mutations that would have been missed with DNA testing alone, and (2) clarified inconclusive results as disease-causing.
Author Interviews, Biogen / 20.11.2019

MedicalResearch.com Interview with: [caption id="attachment_52241" align="alignleft" width="199"]Aaron Deykin MD Vice President, Late Stage Clinical Development Biogen  Dr. Deykin[/caption] Aaron Deykin MD Vice President, Late Stage Clinical Development Biogen  MedicalResearch.com: What data support the U.S. Food and Drug Administration’s (FDA) approval of VUMERITY™ (diroximel fumarate)? Response: The FDA approval of VUMERITY was based on a New Drug Application (NDA) submitted under the 505(b)(2) filing pathway. It included data from pharmacokinetic bridging studies comparing VUMERITY and TECFIDERA® (dimethyl fumarate) to establish bioequivalence, and relied, in part, on the FDA’s findings of safety and efficacy for TECFIDERA. The NDA submission also included interim exposure and safety findings from EVOLVE-MS-1, an ongoing, Phase 3, single-arm, open-label, two-year safety study evaluating VUMERITY in patients with relapsing-remitting multiple sclerosis (MS). Interim results from EVOLVE-MS-1 at the time of NDA submission included a low overall rate of VUMERITY treatment discontinuation due to adverse events (6.3 percent), and a rate of less than one percent of patients who discontinued VUMERITY treatment due to gastrointestinal (GI) adverse events. Additional exploratory efficacy endpoints in the ongoing EVOLVE-MS-1 study showed changes in clinical and radiological measures compared to baseline.
Author Interviews, Cancer Research, Genetic Research, Pharmaceutical Companies / 23.10.2019

MedicalResearch.com Interview with: Ambry GeneticsRachid Karam, MD, PhD Ambry Genetics Aliso Viejo, California MedicalResearch.com: What is the background for this study? Response: DNA genetic testing is a powerful tool used to tailor medical care based on an individual’s cancer risk. However, even medical grade DNA genetic testing can produce inconclusive results, finding a change in our DNA to be a variant of unknown significance (a VUS) and failing to determine whether it increases cancer risk. When this happens, healthcare providers might not have the information needed to recommend appropriate preventive and early detection steps, or certain cancer treatments, and relatives may not be referred for genetic testing for their own care. In this study, investigators from Ambry, Dana-Farber Cancer Institute, Cedars-Sinai Medical Center, Rutgers Cancer Institute, and University of Kansas Cancer Center demonstrated that performing both DNA and RNA genetic testing reduces inconclusive results enabling clinicians to offer cancer screening and treatment resources to the right patients.
Author Interviews, Heart Disease, Pharmaceutical Companies, Pulmonary Disease / 21.10.2019

MedicalResearch.com Interview with: [caption id="attachment_51892" align="alignleft" width="85"]Olivier Sitbon, MD, PhD Université Paris–Sud Dr. Sitbon[/caption] Olivier Sitbon, MD, PhD Université Paris–Sud  MedicalResearch.com: What is the background for this study? How does this treatment competition differ from other treatments for PAH?  Response:  Pulmonary arterial hypertension (PAH) is a complex, progressive and potentially fatal disease with no cure. Over the past decades, advances in understanding the pathophysiology of PAH have led to major prognostic improvement and developments of new treatment guidelines and therapies. Current treatment guidelines recommend initial combination therapy for these patients to target multiple PAH-associated pathways in parallel. OPTIMA was a prospective, multicenter, single-arm, open-label, Phase IV trial designed to evaluate the efficacy, safety and tolerability of initial oral combination therapy with macitentan and tadalafil in patients with newly diagnosed PAH. Treatment with macitentan 10 mg once-daily and tadalafil 20 mg once-daily was initiated on the same day. After 8±3 days, tadalafil dose was increased to 40 mg once-daily. Safety and tolerability findings were consistent with previous clinical trials that supported the approval and use of macitentan 10 mg once-daily. Efficacy outcomes were assessed at Week 16 and safety continued to be monitored until study closure. The results from the OPTIMA analysis suggest that initial treatment with macitentan in combination with tadalafil is associated with hemodynamic improvement in newly diagnosed patients with pulmonary arterial hypertension
Author Interviews, Boehringer Ingelheim, Pulmonary Disease / 01.10.2019

MedicalResearch.com Interview with: Dr. Donald ZozDonald Zoz, M.D. Director, Clinical Development and Medical Affairs Respiratory Specialty Care Boehringer Ingelheim Pharmaceuticals, Inc. MedicalResearch.com: What is the background for this announcement? Would you briefly explain what is meant by systemic sclerosis and how it affects lung function?  Response: In September, the U.S. Food and Drug Administration (FDA) approved Ofev as the first and only therapy to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD). The FDA granted priority review and Fast-Track designation earlier this year. Interstitial lung disease (ILD) is characterized by thickening and scarring of lung tissue and is the leading cause of death among people with systemic sclerosis (SSc), also known as scleroderma. In fact, approximately 25 percent of SSc patients develop significant lung involvement within three years of diagnosis. Prior to this approval, there were no options for this patient population, making this an exciting announcement for the community. 
AstraZeneca, Author Interviews, Pulmonary Disease / 17.09.2019

MedicalResearch.com Interview from: AstraZeneca Spokesperson MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by COPD? How common is severe COPD? Response: COPD, or chronic obstructive pulmonary disease, is a progressive disease associated mainly with tobacco smoking, air pollution or occupational exposure, which can cause obstruction of airflow in the lungs making it hard to breathe. In the United States, COPD is the fourth leading cause of death. Its prevalence in adults 18 years of age and older is 6.5 percent. An estimated 16 million people are currently diagnosed with COPD, and millions more are believed to have it but do not know it. ETHOS is a randomized, double-blind, multi-center, parallel-group, 52-week trial to assess the efficacy and safety of PT010 in symptomatic patients with moderate to very severe COPD and a history of exacerbation(s) in the previous year. The trial compared two doses given twice daily of PT010 (320/14.4/9.6mcg and 160/14.4/9.6mcg) with glycopyrrolate/formoterol fumarate (14.4/9.6mcg) and PT009 (320/9.6mcg), all using AEROSPHERETM Delivery Technology in a pressurized metered-dose inhaler (pMDI). Outcomes in the ETHOS trial included, as a primary endpoint, the rate of moderate or severe exacerbations. The Phase III ETHOS trial builds on the Phase III KRONOS data which together show PT010’s ability to reduce exacerbation risk in a broad range of patients with COPD, irrespective of whether they have had an exacerbation in the previous twelve months.
AstraZeneca, Author Interviews, Brigham & Women's - Harvard, Diabetes, Heart Disease / 10.09.2019

MedicalResearch.com Interview with: [caption id="attachment_51229" align="alignleft" width="200"]Dr. David Berg MD Senior Fellow in Cardiovascular Medicine and Critical Care Medicine Brigham and Women’s Hospital Postdoctoral Research Fellow with the TIMI Study Group. Dr. David Berg[/caption] Dr. David Berg MD Senior Fellow in Cardiovascular Medicine and Critical Care Medicine Brigham and Women’s Hospital Postdoctoral Research Fellow with the TIMI Study Group. MedicalResearch.com: What is the background for this study? Response: Heart failure is a frequent and important complication of type 2 diabetes mellitus (T2DM), but there are limited tools for identifying which patients with T2DM are at the highest risk of developing heart failure. In this study, we developed and validated the TIMI Risk Score for Heart Failure in Diabetes [TRS-HF(DM)], a novel clinical risk score that identifies patients with T2DM who are at heightened risk for hospitalization due to heart failure. Fortunately, the risk score also identifies patients who have the greatest absolute reduction in the risk of hospitalization for heart failure with a new class of glucose-lowering therapies called sodium-glucose cotransporter-2 (SGLT2) inhibitors. 
Allergies, Author Interviews, Pediatrics, Pharmaceutical Companies / 21.08.2019

MedicalResearch.com Interview with: [caption id="attachment_51005" align="alignleft" width="159"]Todd Green MD Vice President of Medical Affairs North America DBV Technologies Dr. Todd Green[/caption] Todd Green MD Vice President of Medical Affairs North America DBV Technologies https://www.dbv-technologies.com   Dr. Green discusses the recent announcement that DBV Technologies is submitting a BLA for Viaskin Peanut to the FDA.   MedicalResearch.com: What is the background for this announcement? How common is peanut allergy in children?  DBV Technologies is a global clinical-stage biopharmaceutical company whose mission is to improve the lives of patients with food allergies and other immunological diseases through our investigational epicutaneous immunotherapy technology platform. For more than 15 years, we’ve been striving to deliver transformative treatments for patients suffering with the burden and life-threatening risk of food allergies. On August 7, 2019 DBV announced the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration for Viaskin® Peanut for the treatment of peanut-allergic children ages 4 to 11 years. This submission addressed the additional data needed on manufacturing procedures and quality controls which were communicated by the FDA in December 2018, when DBV voluntarily withdrew its prior BLA submission for Viaskin Peanut. Peanut allergy is one of the most common food allergies and can cause severe, potentially fatal allergic reactions, including anaphylaxis. In the United States, nearly one million children suffer from a peanut allergy.[1] Fear of life-threatening reactions triggered by accidental peanut exposure during everyday activities may lead to significantly increased anxiety and decreased quality of life for patients and their families.[2,3,4] Currently, avoidance and readiness to manage accidental exposure reactions remain the standard of care. At DBV, we are committed to finding treatments that will help address the urgent unmet medical need of those suffering from food allergies, including peanut allergy, and our mission is to improve the lives of those patients and their families.
Author Interviews, Bayer, FDA, Prostate Cancer / 16.08.2019

MedicalResearch.com Interview with: [caption id="attachment_50898" align="alignleft" width="133"]Neal D. Shore, MD, FACS Director, CPI, Carolina Urologic Research Center Atlantic Urology Clinics Myrtle Beach, South Carolina Dr. Shore[/caption] Neal D. Shore, MD, FACS Director, CPI, Carolina Urologic Research Center Atlantic Urology Clinics Myrtle Beach, South Carolina Neal D. Shore, MD, FACS is the Medical Director for the Carolina Urologic Research Center. He practices with Atlantic Urology Clinics in Myrtle Beach, South Carolina. Dr. Shore discusses the recent announcement that the FDA has approved Nubeqa®(darolutamide),  for the treatment of patients with non-metastatic castration-resistant prostate cancer..  MedicalResearch.com: What is the background for this study? How does NUBEQA®(darolutamide) differ from other treatments for nmCRPC?  Response: In 2017, patients did not have an approved therapy for non-metastatic castration-resistant prostate cancer, or nmCRPC. If untreated, patients with this diagnosis will go on to develop metastases, or progression of the cancer throughout the body. NUBEQA® (darolutamide) became the third and most recently approved treatment for nmCRPC, demonstrating a benefit of metastasis-free survival, or MFS. NUBEQA is different due to its adverse event and safety profile reported in the Phase III ARAMIS trial. In that study, there were no significant findings of falls and fractures as well as other adverse events reported from the earlier Phase III trials. 
Author Interviews, Heart Disease, Pharmaceutical Companies, Stem Cells, Technology / 12.08.2019

MedicalResearch.com Interview with: [caption id="attachment_50687" align="alignleft" width="125"]Misti Ushio, Ph.D. Chief Executive Office Dr. Ushio[/caption] Misti Ushio, Ph.D. Chief Executive Officer [caption id="attachment_50688" align="alignleft" width="125"]Michael Graziano, PhD Chief Scientific Officer TARA Biosystems Dr. Graziano[/caption] Michael Graziano, PhD Chief Scientific Officer TARA Biosystem MedicalResearch.com: What is the background for this study? Response: Almost half of all drug recalls are due to cardiac toxicity that was not picked up during early screens. These human cardiac liabilities can go undetected because historically it has been challenging to predict how human hearts will respond to potentially cardiotoxic drugs despite rigorous testing in both animals and in vitro systems throughout drug development. Traditional in vitro systems and animal models do not translate well to humans, and human donor tissue availability is limited for in vitro testing. There is great potential for human-induced pluripotent stem cell cardiomyocytes (iPSC-CMs) to bridge this human translation gap, but it’s been a challenge to train these cells to recapitulate pharmacology seen in mature human heart cells. This stems from the fact that existing experimental models utilize immature human iPSC-CMs which lack relevant physiological hallmarks of adult human cardiac muscle and therefore fail to predict drug responses seen in the clinic.
Author Interviews, Cost of Health Care, JAMA, NYU/NYMC, Ophthalmology, Pharmaceutical Companies / 02.08.2019

MedicalResearch.com Interview with: [caption id="attachment_50532" align="alignleft" width="150"] Dr. Thiel[/caption] Cassandra L. Thiel, PhD Assistant Professor NYU Langone School of Medicine Department of Population Health NYU Wagner Graduate School of Public Service NYU Tandon School of Engineering MedicalResearch.com: What is the background for this study? Response: Most healthcare professionals and researchers are aware that the healthcare sector makes up about 18% of the US Gross Domestic Product. What many do not realize is that all of that economic activity results in sizable resource consumption and environmental emissions. The healthcare industry is responsible for 10% of the US’s greenhouse gas (GHG) emissions and 9% of air pollutants.1 Sustainability in healthcare is a developing field of research and practice, and my lab offers data and information by quantifying resource use and emissions of healthcare delivery. We started looking at cataract surgery a few years ago, in part because operating rooms (ORs) typically represent the largest portion of spending and garbage generation in a hospital.2,3 Cataract surgeries are interesting because they are one of the most common surgeries performed in the world. In the US, we spend $6.8 billion on them each year. Any changes we can make to individual cases would have much larger, global impacts. I studied cataract surgeries at a world-renowned, high-volume eye surgery center in India and helped validate that clinical care could be designed in a way that was effective, cost-efficient, and resource efficient. Compared to the same procedure in the UK, this surgery center generates only 5% of the carbon emissions (with the same outcomes).2 This site’s standard policy is to multi-dose their eye drops, or use them on multiple patients until the bottle was empty. As such, the site generated very little waste. Returning to the US, I observed cataract cases and heard the complaints of OR staff that they had to throw out many partially used or unused pharmaceuticals. In reviewing the literature, we could not find a study that quantified how much we were throwing away and what it cost us to do so. We, therefore, set up a study to look at this particular issue.
AstraZeneca, Author Interviews, Kidney Disease, Mineral Metabolism / 08.07.2019

MedicalResearch.com Interview with: [caption id="attachment_50153" align="alignleft" width="142"]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[/caption] 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.
Author Interviews, Infections, Pharmaceutical Companies / 08.07.2019

MedicalResearch.com Interview with: [caption id="attachment_50138" align="alignleft" width="150"]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[/caption] 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.
Author Interviews, Bristol Myers Squibb, Rheumatology / 26.06.2019

MedicalResearch.com Interview with: [caption id="attachment_49997" align="alignleft" width="200"]Sean Connolly, Ph.D. Director of Non-Registrational Data Generation Study Director for ASCORE Bristol-Myers Squibb Dr. Connolly[/caption] 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.
AstraZeneca, Author Interviews, Diabetes, Kidney Disease / 24.06.2019

MedicalResearch.com Interview with: [caption id="attachment_49957" align="alignleft" width="200"]Naeem Khan MD Vice President at AstraZeneca Dr. Khan[/caption] 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).
Amgen, Author Interviews, Cost of Health Care, JAMA, Lipids, UCLA / 24.06.2019

MedicalResearch.com Interview with: [caption id="attachment_25325" align="alignleft" width="160"]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[/caption] 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.
Author Interviews, Bayer, Biomarkers, Colon Cancer / 05.06.2019

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). 
ASCO, Author Interviews, Bayer, Leukemia, Pediatrics / 05.06.2019

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. 
AstraZeneca, Kidney Disease / 04.06.2019

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.
ASCO, Author Interviews, Bayer, Cancer Research, Pediatrics / 03.06.2019

MedicalResearch.com Interview with: [caption id="attachment_49569" align="alignleft" width="173"]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[/caption] 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. 
ASCO, Author Interviews, Cancer Research, J&J-Janssen, Leukemia / 03.06.2019

MedicalResearch.com Interview with: [caption id="attachment_49526" align="alignleft" width="144"]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[/caption] 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.