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. (more…)
Author Interviews, Heart Disease, Pharmaceutical Companies, Pulmonary Disease / 21.10.2019

MedicalResearch.com Interview with: 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 (more…)
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.  (more…)
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. (more…)
AstraZeneca, Author Interviews, Brigham & Women's - Harvard, Diabetes, Heart Disease / 10.09.2019

MedicalResearch.com Interview with: 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.  (more…)
Allergies, Author Interviews, Pediatrics, Pharmaceutical Companies / 21.08.2019

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Bayer, FDA, Prostate Cancer / 16.08.2019

MedicalResearch.com Interview with: 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.  (more…)
Author Interviews, Heart Disease, Pharmaceutical Companies, Stem Cells, Technology / 12.08.2019

MedicalResearch.com Interview with: Misti Ushio, Ph.D. Chief Executive Officer 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. (more…)
Author Interviews, Cost of Health Care, JAMA, NYU, Ophthalmology, Pharmaceutical Companies / 02.08.2019

MedicalResearch.com Interview with: 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. (more…)
AstraZeneca, Author Interviews, Kidney Disease, Mineral Metabolism / 08.07.2019

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  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. (more…)
Author Interviews, Infections, Pharmaceutical Companies / 08.07.2019

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  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. (more…)
Author Interviews, Bristol Myers Squibb, Rheumatology / 26.06.2019

MedicalResearch.com Interview with: 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. (more…)
AstraZeneca, Author Interviews, Diabetes, Kidney Disease / 24.06.2019

MedicalResearch.com Interview with: 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). (more…)
Amgen, Author Interviews, Cost of Health Care, JAMA, Lipids, UCLA / 24.06.2019

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  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. (more…)
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).  (more…)
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.  (more…)
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. (more…)
ASCO, Author Interviews, Bayer, Cancer Research, Pediatrics / 03.06.2019

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 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.  (more…)
ASCO, Author Interviews, Cancer Research, J&J-Janssen, Leukemia / 03.06.2019

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  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. (more…)
Author Interviews, Breast Cancer, Cancer Research, Genetic Research, Novartis / 03.06.2019

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. 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. 
(more…)
ASCO, Author Interviews, Cancer Research, J&J-Janssen, NEJM, Prostate Cancer / 01.06.2019

MedicalResearch.com Interview with: 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).  (more…)
Author Interviews, Pharmaceutical Companies, Prostate, Urology / 31.05.2019

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 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. (more…)
Author Interviews, Boehringer Ingelheim, NEJM, Pharmaceutical Companies, Pulmonary Disease / 30.05.2019

MedicalResearch.com Interview with: 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.  (more…)
Allergan, Author Interviews, Bipolar Disorder, Mental Health Research / 23.05.2019

MedicalResearch.com Interview with: 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).  (more…)
Author Interviews, Diabetes, Ophthalmology, Regeneron / 17.05.2019

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Kidney Disease, Pharmaceutical Companies / 26.04.2019

MedicalResearch.com Interview with: 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.  (more…)
Author Interviews, Hospital Acquired, Infections, Merck / 16.04.2019

MedicalResearch.com Interview with: 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. (more…)
Alzheimer's - Dementia, Author Interviews, Merck, NEJM / 10.04.2019

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. (more…)
Author Interviews, Cancer Research, Genentech, Leukemia / 25.03.2019

MedicalResearch.com Interview with: 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.  (more…)