Author Interviews, Breast Cancer, Chemotherapy, JAMA, Novartis / 26.03.2018

MedicalResearch.com Interview with: Melanie E. Royce, MD, PhD Division of Hematology/Oncology University of New Mexico Comprehensive Cancer Center Albuquerque MedicalResearch.com: What is the background for this study? What are the main findings? Response: BOLERO-4 is an open label, single-arm, Phase II study that evaluates the combination of everolimus plus letrozole as a first-line treatment for hormone receptor (HR)-positive/human epidermal growth factor receptor (HER2)-negative advanced breast cancer patients, as well as the use of everolimus plus exemestane beyond initial progression. Results of the BOLERO-4 trial published in JAMA Oncology showed that everolimus in combination with endocrine therapy is an effective first-line treatment option for postmenopausal women with HR+/HER2- advanced breast cancer. A total of 202 patients received everolimus in combination with letrozole as first-line treatment between March 7, 2013 and December 17, 2014. Median progression-free survival (PFS) in the first-line setting was 22.0 months (95% CI 18.1-25.1) with an overall response rate of 45% (95% CI 38.1-52.2) and clinical benefit rate of 74% (95% CI 67.7-80.1). A total of 152 (75%) discontinued treatment, primarily due to disease progression (51%) or adverse events (16%). Data from a smaller number of patients in BOLERO-4 also show limited efficacy with continued everolimus, combined with exemestane, following disease progression. Second-line treatment was ongoing in 16 (32%) patients, while 34 (68%) had discontinued. The most frequent reason for second-line treatment discontinuation was disease progression (56%). In the second-line setting, median PFS was 3.7 months (95% CI 1.9-7.4) with an overall response of 6% (95% CI 1.3-16.5) and clinical benefit rate of 28% (95% CI 16.2-42.5). Safety findings from BOLERO-4 are consistent with previous studies of Afinitor in advanced breast cancer. The most common (≥ 20% incidence) first-line all-grade adverse events were stomatitis (69%), weight loss (44%), nausea (37%) and anemia (35%). Most were ‘low grade’ in severity (grade 1 or 2) and generally well managed. Safety findings show the most common (≥ 10% incidence) second-line adverse events were stomatitis (20%) and weight loss (20%). Lower rates of stomatitis in second-line were noted.  (more…)
Abuse and Neglect, Author Interviews, Neurology, Pharmaceutical Companies / 23.03.2018

MedicalResearch.com Interview with: Dr. Joseph Horrigan MD Pediatric neuropsychiatrist Chief medical officer   MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by myotonic dystrophy? What are the manifestations of this disease? Response: This was the first pharmaceutical intervention study conducted in adolescents and adults with congenital and juvenile onset DM1. Myotonic dystrophy type 1 (DM1) is a disorder that impacts multiple body systems following a trinucleotide expansion repeat of the DMPK gene on chromosome 19. Children with Congenital DM1 present at birth with respiratory insufficiency, talipes equinovarus (clubfoot), feeding difficulties and hypotonia. There is a risk mortality rate in the first year of life. As children grow, they are at risk for intellectual impairment, autistic features, gastrointestinal symptoms, motor delay and a variety of muscle-based symptoms. (more…)
Author Interviews, Opiods, Pharmaceutical Companies / 23.03.2018

MedicalResearch.com Interview with: Vishal Bala Senior Quantitative Data Analyst CareDash MedicalResearch.com: What is the background for this study? What are the main findings? Response: Prior research into physicians and their relationships with the pharmaceutical industry has typically retained a narrow scope, focusing on how payments may be associated with prescription habits (sometimes limited to specific regions) for specific categories of drugs. For example, Modi et al. 2017 and Bandari et al. 2017 explored these connections in the context of some urologic drugs specifically. Research conducted by ProPublica in 2016 studied the connection between industry payments and physician prescriptions across some of the largest medical specialties, but was only able to look at “brand-name” vs. “generic” categories and were limited by overlapping timeframes for payments and prescriptions. CareDash took this analysis further by using Open Payments and Medicare Part D data to investigate the relationship between payments made by individual companies for specific drugs and the prescribing habits of the recipient physicians for those drugs. CareDash’s main findings are that healthcare providers who received payments for a drug from a pharmaceutical company are 5 times more likely to be high prescribers for that drug than those physicians who did not receive a payment. Physicians are 5.3 times more likely to prescribe a drug than their peers after they have received a payment for that drug from the manufacturer. When physicians already prescribe a drug significantly more often than their peers, they are 5.6 times more likely to later receive payment for that drug from the drug's manufacturer. Looking at the opioid drug class specifically, CareDash found that physicians receiving payment on behalf of an opioid were 14.5 times more likely to prescribe that opioid over alternatives. (more…)
Author Interviews, Flu - Influenza, Infections, Mental Health Research, Primary Care, Roche / 14.03.2018

MedicalResearch.com Interview with: James W. Antoon, MD, PhD, FAAP Assistant Professor of Clinical Pediatrics University of Illinois at Chicago Associate Medical Director, Pediatric Inpatient Unit Children's Hospital, University of Illinois Hospital & Health Sciences System Chicago, IL 60612  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Oseltamivir, commonly known as Tamiflu, is the only commercially available medication FDA approved to treat the flu.  Since the 2009 H1N1 flu epidemic pediatric prescriptions for Tamiflu have soared.  In the United States, about 40% of Tamiflu prescriptions are given to children less than 16 years of age.  Following reports of abnormal behavior, such as hallucinations, self-injury and suicide attempts in adolescents on Tamiflu, the FDA placed a new warning about these neuropsychiatric symptoms on the drug label.  Whenever the FDA puts out label warning about a drug, doctors and the public take notice. Whether Tamiflu truly causes these side effects is unclear.  For this study we chose to focus on the most consequential of those reports: suicide. The potential link between a drug and suicide is a particularly difficult topic to study for a number of reasons. There are things that happen together or at the same time that can influence someone to attempt suicide and it is very difficult to know which thing is actually having an affect. In our study, other things that can influence suicide are socioeconomic status, mental health, trauma, abuse, among others.  Separating the effects of these confounders can be difficult. It is also possible that the disease itself, which in this case is the flu, causes the effect of suicide. Finally, and luckily, suicide is rare. Our database had 12 million children per year and over five year 21,000 attempted suicide. Of those, only 251 were taking Tamiflu. To get past these issues, we took advantage of a growing drug safety research collaboration between the Departments of Pediatrics and Pharmacy at our institution.  Previous studies have compared those on Tamiflu to those not on Tamiflu to see if there are more side effects in the Tamiflu group.  Our team utilized a novel study method called a case-crossover design. What’s different about this study is that we used each patient as their own comparison.  In other words, we compared each patient to themselves rather than a different group of people.  We essentially studied how patients behaved when the Tamiflu was in their system compared to other l periods where they were not on Tamiflu.  This allowed use to account for the personal differences noted above like mental health and socioeconomic status.   We also compared those children with flu who got Tamiflu and those with flu who did not get Tamiflu to see if the infection itself could be associated with increased suicide. After accounting for all these variables, we did not find any an association between Tamiflu exposure and suicide. Our findings suggest that Tamiflu does NOT increase the risk of suicide in children or teenagers. (more…)
Author Interviews, GSK, Herpes Viruses, Infections, Vaccine Studies / 10.03.2018

MedicalResearch.com Interview with: Anthony. L. Cunningham, MD The Westmead Institute for Medical Research Westmead, NSW University of Sydney, Sydney,   MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study examines the reasons why the HZ subunit vaccine candidate (HZ/su vaccine) consisting of a single viral protein, varicella-zoster glycoprotein E, and and adjuvant (immunostimulant) combination AS01B is so effective as a vaccine to prevent shingles (>90%), especially in those over the age of 70 years and 80 years, as published in recent trials i.e. it combats the declining immunity in the aging which usually restricts vaccine efficacy to under 60% in these age groups.  (more…)
Author Interviews, Multiple Sclerosis, Ophthalmology, Pharmaceutical Companies / 08.03.2018

MedicalResearch.com Interview with: Sarah A. Morrow MD, MS, FRCPC Associate Professor of Neurology Department of Clinical Neurological Sciences University of Western Ontario (Western) MedicalResearch.com: What is the background for this study? Response: Acute demyelinating optic neuritis, which presents with loss of vision and painful eye movements, is common in multiple sclerosis (MS) occurring 50% of persons with MS. High dose (≥ 1g) corticosteroids administered through an IV became the standard of practice after the landmark Optic Neuritis Treatment Trial as IV administration. However, in that study the IV dose of corticosteroids was much higher (1 gram daily) than the oral dose (1 mg/kg). Thus, it is not clear if IV administration is still better if equivalent doses are used orally. Oral administration is much more convenient for patients and less expensive, and previous studies showed that it is preferred by patients. In this study, we asked the following question: are high dose (≥ 1000mg) IV corticosteroids superior to equivalent doses of oral corticosteroids for the acute treatment of optic neuritis? We randomly assigned fifty-five cases of acute optic neuritis to 1000mg IV methylprednisolone or 1250mg oral prednisone daily for three days and compared recovery of their vision over the next 6 months.  (more…)
Author Interviews, Dermatology, Eli Lilly, Sexual Health / 07.03.2018

MedicalResearch.com Interview with: Dr. Jennifer Cather MD Medical Director at Modern Dermatology and Modern Research Associate Dallas, Texas  MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Genital psoriasis can be an uncomfortable and burdensome condition that many people living with moderate-to-severe plaque psoriasis experience. Due to the significant impact, Lilly conducted a 12-week Phase 3b clinical trial with patients with moderate-to-severe genital psoriasis treated with ixekizumab, which found that patients had a greater decrease in the impact of their condition on sexual activity compared to placebo as early as one week. Specifically, trial patients were randomized to receive ixekizumab (80 mg every two weeks, following a 160-mg starting dose) or placebo and researchers measured pre-specified patient-reported outcomes, including the Genital Psoriasis Sexual Impact Scale (GPSIS), which is composed of the Sexual Activity Avoidance (Avoidance) and Impact of Sexual Activity on Genital Psoriasis Symptoms (Impact) subscales. Patient-reported outcomes were also measured by the Sexual Frequency Questionnaire (SFQ) item 2, evaluating the impact of genital psoriasis on the frequency of sexual activity, and the Dermatology Life Quality Index (DLQI) item 9, evaluating the impact of skin symptoms on sexual difficulties. At 12 weeks, patients reported the following outcomes:
  • DLQI Item 9 0/1: 92.0 percent of patients treated with ixekizumab compared to 56.8 percent of patients treated with placebo reported no (0) or little (1) sexual difficulties caused by skin symptoms.
  • SFQ Item 2 0/1:  78.4 percent of patients treated with ixekizumab compared to 21.4 percent of patients treated with placebo (reported the frequency of sexual activity was either never (0) or rarely (1) limited by genital psoriasis.
  • GPSIS-Avoidance 1/2:  76.7 percent of patients treated with ixekizumab compared to 25.7 percent of patients treated with placebo reported never (1) or rarely (2) avoiding sexual activity due to genital psoriasis.
  • GPSIS-Impact 1/2:  85.7 percent of patients treated with ixekizumab compared to 52.9 percent of patients treated with placebo reported worsening of genital psoriasis symptoms during or after sexual activity was very low/none at all (1) or low (2). 
(more…)
Author Interviews, Boehringer Ingelheim, Dermatology, Pulmonary Disease, Rheumatology / 05.03.2018

MedicalResearch.com Interview with: Donald Zoz, MD Senior Associate Director Clinical Development & Medical Affairs IPF/ILD Boehringer Ingelheim Pharmaceuticals, Inc. MedicalResearch.com: What is the background for this platform? Would you briefly explain what is meant by scleroderma? How does it affect a person's skin and ability to function? Whom does this disease primarily affect? Response: “More Than Scleroderma™: The Inside Story” is Boehringer Ingelheim’s new global initiative highlighting real-life, inspirational stories of people living with the rare disease scleroderma. The new effort, created with support from the Scleroderma Foundation in the U.S., aims to raise awareness of the disease, dispel misperceptions and provide important resources to support and guide those on their journey with scleroderma. The initiative’s website http://www.morethanscleroderma.com/us/ features a powerful and inspiring collection of diverse photographs and video profiles of 10 people across the U.S. living with scleroderma and sharing their ‘inside story.’ Each tells their unique and moving experience with scleroderma through diagnosis to learning to live with the disease and manage it. Many less fortunate people in developing countries have to deal with this on a daily basis and only have the support of Orphan Drug Distributors and charities and I'm so thankful that people like this are available to support these people! Scleroderma, also known as systemic sclerosis, is a rare disease characterized by thickening and scarring of the skin, lungs and other organs. Scleroderma affects fewer than 200,000 people in the U.S. and typically affects women in the prime of their lives, between the ages of 25 and 55 taking a marked toll just as they are building their careers and bearing the responsibility of caring for their family. Nearly all people with scleroderma (more than 90%) will develop some skin symptoms including skin thickening, tightened skin around the joints, small red spots on the face and hands and hard lumps on pressure points and joints. Most people with the disease will also develop some degree of lung scarring, or interstitial lung disease (ILD). When the disease's signature thickening and scarring develops in vital organs, such as the lungs, there are potentially debilitating and life-threatening consequences. (more…)
Author Interviews, Autism, Pharmaceutical Companies, Roche, Vanderbilt / 16.02.2018

MedicalResearch.com Interview with: Dr Kevin Sanders, MD Principle Medical Director-Product Development Neuroscience Assistant Professor, Departments of Psychiatry and Pediatrics Vanderbilt University  MedicalResearch.com: What is the background for this announcement?  Response: The FDA has granted Roche Breakthrough Therapy Designation for its investigational oral medicine balovaptan (previously known as RG7314), a vasopressin 1a (V1a) receptor antagonist for individuals with autism spectrum disorder (ASD). FDA Breakthrough Therapy Designation for balovaptan is primarily based on efficacy findings in the VANILLA (Vasopressin ANtagonist to Improve sociaL communication in Autism) study, a Phase II trial of balovaptan in adults with ASD. Trial results were first presented at the International Congress for Autism Research (IMFAR) in May 2017. Treatment effects were observed on the Vineland-II (secondary endpoint) and also demonstrated that balovaptan was safe and well tolerated by the subjects in the study. The Vineland-II is a scale that measures socialization, communication and daily living skills. This data was presented to the FDA and is part of the basis of the Breakthrough Designation.  (more…)
Author Interviews, Hematology, Novo Nordisk, Surgical Research / 15.02.2018

MedicalResearch.com Interview with: Stephanie Seremetis, M.D. Corporate Vice President and Chief Medical Officer Biopharmaceuticals at Novo Nordisk MedicalResearch.com: What is the background for this announcement? Response: We’re proud and excited to make Rebinyn® (Coagulation Factor IX (Recombinant), GlycoPEGylated) available as a new extended half-life treatment for hemophilia B management. Rebinyn® is an injectable medicine used to treat and control bleeding in adults and children with hemophilia B. It can be used to treat bleeds when they occur and to manage bleeding during surgery. Rebinyn® is not used for routine prophylaxis or for immune tolerance induction in patients with hemophilia B. Hemophilia B is a serious, chronic, inherited bleeding disease that affects about 5,000 people in the U.S. People living with hemophilia B have low levels of clotting Factor IX protein in the blood, often resulting in prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs.  (more…)
Author Interviews, Cancer Research, Novartis, Pancreatic / 05.02.2018

MedicalResearch.com Interview with: Lynn Matrisian, PhD, MBA Chief science Officer Pancreatic Cancer Action Network MedicalResearch.com: Would you tell us a little about PNETs? How common is this type of pancreatic tumor? How does Lutathera differ from other treatments for this tumor?  Response: Pancreatic neuroendocrine tumors (PNETs) make up about 6 percent of all pancreatic cancer diagnoses. They are less common and slower growing than the more common type of pancreatic cancer, adenocarcinoma, and have a better prognosis. Lutathera® is a peptide receptor radionuclide therapy (PRRT) that was approved for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including PNETs, that express somatostatin receptors. The drug is a somatostatin analog that is conjugated to a radionuclide (177Lu) to selectively deliver radiotherapy to the cancer cells. Other treatment options for PNETs include surgery (partial or complete removal of the tumor), chemotherapy (typically in combination) or radiation therapy (conventional as well as PRRT). Patients may also receive targeted therapies. Sutent® blocks platelet-derived growth factor receptors (PDGFRs) α and β, stem-cell factor receptor (c-kit) and vascular endothelial growth factor receptor (VEGFR)-2 and VEGFR-3, leading to inhibition of cell growth and angiogenesis. Afinitor® behaves as a rapamycin analog, blocking the mammalian target of rapamycin (mTOR) signaling pathway. Prior to Lutathera’s approval, there were two non-PRRT somatostatin analogs approved for PNET patients. These drugs were initially intended to mitigate some of the symptoms of the disease, but they were also found to slow the cancer cells’ growth. The approved somatostatin analogs are lanreotide and octreotide.  (more…)
Abbvie, Author Interviews, Hepatitis - Liver Disease, NEJM, Pharmaceutical Companies / 29.01.2018

MedicalResearch.com Interview with: Stefan Zeuzem, M.D. Professor of Medicine Chief Internal Medicine Goethe University Hospital Frankfurt, Germany MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Chronic hepatitis C virus (HCV) infection is a major global public health problem with more than 71 million people infected worldwide, and can result in significant morbidity and mortality, including liver cirrhosis, hepatocellular carcinoma, and death.1 This publication describes the efficacy and safety results from two Phase 3 clinical trials, ENDURANCE-1 and ENDURANCE-3, in patients with chronic HCV genotypes (GT) 1 or 3 infection who were treated with an all-oral, once-daily combination regimen of direct-acting antiviral agents (DAA) glecaprevir (GLE) at 300 mg and pibrentasvir (PIB) at 120 mg. The findings from ENDURANCE-1 trial show that the GLE/PIB combination regimen (G/P) given for 8 weeks to HCV GT1 chronically infected non-cirrhotic treatment-naïve or treatment-experienced (with sofosbuvir and/or interferon with ribavirin) patients was safe and well-tolerated, achieved high efficacy with a sustained virologic response at post-treatment week 12 (SVR12) rate >99% and was non-inferior to 12-week treatment with G/P. The trial also included subjects who were co-infected with human immunodeficiency virus (HIV), and all of these subjects achieved SVR12 while maintaining HIV suppression throughout the study. ENDURANCE-3 trial results show that the G/P regimen given for 8 weeks to HCV GT3 chronically infected non-cirrhotic treatment-naïve patients was safe and well-tolerated, achieved high efficacy in this historically difficult to cure GT with an SVR12 rate >94%, and was non-inferior to 12-week treatment with G/P, which in turn was non-inferior to the treatment with 12-week DAA regimen of sofosbivir and daclatasvir.  (more…)
Allergan, Author Interviews, JAMA, Ophthalmology / 05.01.2018

MedicalResearch.com Interview with: Steven Woloshin, MD MS Professor of The Dartmouth Institute Professor of Medicine Professor of Community and Family Medicine The Center for Medicine in the Media Dartmouth Institute for Health Policy and Clinical Practice Lebanon, New Hampshire MedicalResearch.com: What is the background for this study? What are the main findings? Response: There has been a lot of debate about the legal maneuvers (ie, transferring patents to the Mohawk Indians) Allergan has employed to delay marketing of generic alternatives to Restasis (cyclosporine ophthalmic emulsion 0.05%).   But there is a more fundamental question that has received little attention:  Does Restasis work?  It is not approved in the European Union, Australia or New Zealand where registration applications were "withdrawn prior to approval due to insufficient evidence of efficacy" in 2001.   Although Canada approved Restasis, its national health technology assessment unit, unconvinced of meaningful benefit, recommended Canada not pay for it - according to our research, no Canadian provincial or federal drug plan currently does.   Nevertheless, Americans have spent $8.8 billion in total sales between 2009 and 2015 on Restasis, including over $2.9 billion in public monies through Medicare Part D. (more…)
Author Interviews, Infections, Pfizer, Vaccine Studies / 22.12.2017

MedicalResearch.com Interview with: Judith Absalon, M.D., M.P.H Senior Director, Vaccines Clinical Research Pfizer Pharmaceuticals MedicalResearch.com: What is the background for these two studies? Response: Invasive serogroup B meningococcal disease (MenB) is uncommon, yet serious, is unpredictable and can strike at any age, including healthy teenagers and young adults, with potentially long-lasting and devastating consequences, including death. The data from these two Phase 3 studies, one in adolescents (Study 1009) and one young adults (Study 1016), highlight that Trumenba can help protect teens and young adults against meningococcal group B disease. Additionally, these two large Phase 3 studies confirmed the results of earlier studies and supported the transition from Accelerated to Traditional Approval in the US; were pivotal for approvals in Europe, Australia, and Canada earlier this year; and add to the growing portfolio of research for TRUMENBA. (more…)
Allergan, Author Interviews, Bipolar Disorder, Brigham & Women's - Harvard / 20.12.2017

MedicalResearch.com Interview with: allerganGary Sachs, MD Associate Clinical Professor of Psychiatry Harvard Medical School  MedicalResearch.com: What is the background for this data milestone? Response: Bipolar disorder affects about 5.7 million adults in the United States.  It is a common, often disabling condition in which abnormal mood states impair a person’s ability to carry out everyday tasks. Bipolar disorder touches nearly every family and community in America, because periods of illness, a patient’s symptoms often impact their family, their friends, and their community. There are a limited number of products approved to treat bipolar depression and even fewer products that have been studied and approved to treat the full spectrum of bipolar disorder, from mania through depression. Having another product proven to treat the full range of bipolar disorder would be a welcome addition to the treatment options currently available to the psychiatry community and patients. (more…)
Author Interviews, Genetic Research, Pharmaceutical Companies / 17.12.2017

MedicalResearch.com Interview with: Alexander S Hauser, PhD student MRC Laboratory of Molecular Biology Cambridge UK Department of Drug Design and Pharmacology, University of Copenhagen Copenhagen, Denmark MedicalResearch.com: What is the background for this study? What are the main findings? Response: The prevalence and impact of genetic variation among all human G protein coupled receptors (GPCRs) that are targeted by FDA-approved drugs remain unknown. In this study, we present a comprehensive analysis and map of the pharmacogenomics landscape of GPCR drug targets. The key highlights are: - GPCRs targeted by drugs show extensive genetic variation in the human population - Variation occurs in functional sites and may result in altered drug response - Understanding GPCR genetic variation may help reduce global healthcare expenses (more…)
Author Interviews, Brigham & Women's - Harvard, Infections, Leukemia, Merck, Transplantation / 12.12.2017

MedicalResearch.com Interview with: Francisco M. Marty, M.D Associate Professor, Harvard Medical School Dana–Farber Cancer Institute and Brigham and Women’s Hospital MedicalResearch.com: What is the background for this study? What are the main findings? Response: Cytomegalovirus (CMV) is the most common infection in patients who undergo allogeneic hematopoietic-cell transplantation (bone marrow transplantation with cells from donors different than the patient). Up until now, we had no antiviral agent that could be used for prophylaxis (prevention) of CMV post-transplant because of the side effects of drugs available to date (ganciclovir, valganciclovir, foscarnet, cidofovir). This trial confirmed that letermovir was highly effective in preventing CMV infection when used in the first 100 days after allogeneic HCT, was associated with minimal side effects of concern and was also associated with lower all-cause mortality by Week 24 post-HCT. (more…)
Author Interviews, Heart Disease, Lipids, Sanofi / 29.11.2017

MedicalResearch.com Interview with: Dr. Jay Edelberg MD, PhD VP Head of CV Development and Head Global CV Medical Affairs Sanofi  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Patients with heterozygous familial hypercholesterolemia (HeFH) are often not able to achieve their target low-density lipoprotein cholesterol (LDL-C) levels, and some may require lipoprotein apheresis (LA) to lower their “bad cholesterol.” Apheresis is a procedure similar to kidney dialysis, where bad cholesterol is mechanically removed from the blood. It is an invasive, expensive, and time-consuming treatment for patients, as well as physicians. The Phase III ESCAPE clinical study looked at the potential effect of LA on total Praluent, free and total PCSK9 concentrations, as well as the combined pharmacodynamics effect of total Praluent on LDL-C-lowering. Praluent levels remained unaffected by apheresis, and Praluent consistently suppressed free PCSK9 levels in patients with HeFH, regardless of LA treatment. This analysis further confirms clinical ESCAPE data that Praluent can be used in conjunction with LA and may reduce or potentially eliminate the need for LA in some patients. (more…)
Author Interviews, Pharmaceutical Companies, Technology / 29.11.2017

MedicalResearch.com Interview with: HITLab Mr. David Moore MBA Senior Vice President, Commercial Novo Nordisk Mr. David Moore presented the keynote presentation “Breaking Through Barriers in Innovation” at the 4th Annual HITLAB Innovators Summit, NYC November 28, 2017.  MedicalResearch.com: How did you become interested in incorporating technological advances into the sales and marketing end of pharmaceutical delivery? Response: Part of our message today is that we understand that there is an opportunity to improve outcomes if we have a better focus on collaboration and integration through engagement with the patient rather than through medications alone. We continue to innovate in diabetes pharmaceutical care but are also focusing more on meeting the patient in their journey through everyday life. The opportunity to connect, collaborate and integrate is what digital health is all about. As manufacturers, we are still early in the integration process. When we think about technological changes from the patient’s perspective, health care has become much different. The patient no longer just goes to the doctor’s office for a conversation or opinion, with the doctor keeping all the notes. Now the patient has access to a broad range of medical information and usually their own medical records. There is an element of needing things to be user-friendly. As with any consumer product or service with choices, if the choice isn’t user-friendly, the consumer won’t use it. We are looking for ways to make the delivery of pharmacologic products user-friendly to both the patient and provider. (more…)
Author Interviews, Lipids, Sanofi / 29.11.2017

MedicalResearch.com Interview with: Dr. Jay Edelberg MD, PhD VP Head of CV Development and Head Global CV Medical Affairs Sanofi MedicalResearch.com: What is the background for this study? What are the main findings? Response: Clinical trials of lipid-lowering therapies (LLTs), including statins, often report variations in treatment response regarding effects on low density-lipoprotein cholesterol (LDL-C) levels, although LDL-C reductions are fairly consistent between trials. Praluent is generally well tolerated, however hyporesponsiveness exists in few patients. Potential causes for variation in patient responsiveness to Praluent include lack of receipt of active study drug, changes in concurrent LLTs, inaccurate or unrepresentative baseline lipid levels, concurrent acute-phase illness, and biological nonresponsiveness. This analysis evaluated patients pooled from 10 ODYSSEY trials to assess characteristics of patients with hyporesponsiveness to Praluent, defined as <15% LDL-C reduction from baseline at all analyzed time points. Overall, only 1% of patients (n=33) had <15% LDL-C reduction at all time points. Prolonged hyporesponsiveness to Praluent was rarely associated with Praluent antidrug antibodies. Of the 33 patients with <15% LDL-C reduction at all study timepoints, 27 had undetectable or missing alirocumab levels, absence of pharmacokinetics analyses, or early treatment discontinuation. (more…)
Author Interviews, Heart Disease, Pharmaceutical Companies / 28.11.2017

MedicalResearch.com Interview with: https://www.verseon.com/ Anirban Datta, PhD Director Discovery Biology Verseon Corporation MedicalResearch.com: What is the background for this study? Response: Today’s anticoagulant market is dominated by the NOACs. These oral anticoagulants require less constant monitoring and have reduced drug and food interactions compared to their predecessors, warfarin and heparin. However, there is still a significant bleeding risk associated with the NOACs. This is particularly problematic when they are co-dosed with antiplatelet drugs. While life-long therapy combining an oral anticoagulant with one or two antiplatelet drugs is desired for the many patients suffering from both non-valvular atrial fibrillation and coronary artery disease, current treatment guidelines limit such therapy to a maximum of six months to a year due to safety concerns. At Verseon, we are developing a novel class of precision anticoagulants that combine efficacy comparable to the NOACs with a significantly reduced bleeding risk in preclinical testing. We believe that this profile can have a positive impact on the lives of the many patients in need of long-term anticoagulation-antiplatelet combination therapy. We are currently advancing two development candidates toward clinical trials in 2018. VE-1902, our first development candidate, is scheduled to enter phase I in the first half of the year. (more…)
Author Interviews, Boehringer Ingelheim, Surgical Research / 27.11.2017

MedicalResearch.com Interview with: Thomas Seck, M.D., vice president Clinical Development and Medical Affairs Primary Care Boehringer Ingelheim Pharmaceuticals, Inc. MedicalResearch.com: What is the background for this study? What are the main findings? Response: This is a new subanalysis of the phase III RE-VERSE AD™ study, which evaluated the safety and efficacy of idarucizumab, marketed in the U.S. as Praxbind®, in reversing the anticoagulant effect of Pradaxa® (dabigatran etexilate mesylate). This data assessed idarucizumab in a subset of patients requiring an urgent procedure or emergency surgery. The analysis found that idarucizumab rapidly and completely reversed the anticoagulant effect of dabigatran in approximately 98 percent of patients based on dTT. The median time between administration of idarucizumab and start of surgery was 1.7 hours for patients requiring abdominal procedures, 1.9 hours for orthopedic procedures, 1.4 hours for vascular procedures, 1.3 hours for drainage procedures and 1.2 hours for catheter procedures. Among these patients, periprocedural homeostasis was assessed as normal in more than 92 percent of patients, across all surgery types. (more…)
Author Interviews, Cancer Research, Pharmaceutical Companies / 20.11.2017

MedicalResearch.com Interview with: Dr. Koustubh Ranade, PhD Vice President of Research & Development Translational Medicine MedImmune MedicalResearch.com: What is the background for this study? What are the main findings? Response: In a healthy person, abnormal cells including cancer cells are typically detected and destroyed by the immune system in response to danger signals activated by the abnormal cells. However, some solid tumors avoid triggering danger signals, and thus the immune system cannot recognize and destroy cancer cells, permitting tumor growth. To help activate the patient’s immune system to fight these “hidden” cancer cells, MedImmune scientists have developed MEDI9197, a TLR 7/8 agonist, to trigger the needed danger signals. Our latest data from the Phase 1 study of MEDI9197 demonstrated that through intratumor injection, the therapy binds to TLR7 and TLR8 receptors and activates dendritic cells, which call in other immune cells to fight the tumor. (more…)
Author Interviews, Dermatology, J&J-Janssen, Pharmaceutical Companies, Rheumatology / 13.11.2017

MedicalResearch.com Interview with: Atul A. Deodhar, MD, MRCP, FACP, FACR Professor of Medicine Division of Arthritis & Rheumatic Diseases Medical Director, Rheumatology Clinics Medical Director, Immunology Infusion Center Oregon Health & Science University (OHSU)  MedicalResearch.com: What is the background for this study? What are the main findings? Response: The Phase 2, randomized, double-blind, placebo–controlled, multicenter trial was designed to evaluate the efficacy and safety of guselkumab (Tremfya®) compared with placebo in adults with active psoriatic arthritis, despite having received treatment with standard-of-care therapies, including anti-tumor necrosis factor (TNF)-alpha agents. In an observed analysis presented at ACR 2017, more than 70 percent of patients receiving guselkumab achieved at least a 20 percent improvement in signs and symptoms of disease (ACR 20) at week 56.  Findings also showed that improvements in tender and swollen joints, skin clearance, pain and physical function, and patient-reported quality of life outcomes reported at week 24, were maintained through week 56 in patients receiving guselkumab maintenance therapy (subcutaneous injections every eight weeks).  (more…)
Author Interviews, Gastrointestinal Disease, Pharmaceutical Companies, University of Michigan / 23.10.2017

MedicalResearch.com Interview with: William D. Chey, M.D., F.A.C.G. Timothy T. Nostrant Professor of Gastroenterology & Nutrition Director, Digestive Disorders Nutrition & Lifestyle Program Michigan Medicine Ann Arbor, Michigan  MedicalResearch.com: What is the background for this study? Response: Functional Dyspepsia (FD) has been characterized as recurring indigestion with no known organic cause and is an area of high unmet medical need. This medical condition, which is non-life threatening, can have a significant impact on an individual’s quality of life. It remains poorly recognized and presents a significant management challenge for providers and patients. Gastrointestinal symptoms can include epigastric pain or discomfort, inability to finish a normal-sized meal, heaviness, pressure, nausea, bloating and belching. Currently, there are no FDA-approved drugs for FD. Off-label medications are used to treat the condition and patient dissatisfaction remains high.[1] In a real-world, observational study, called FDACT™ (Functional Dyspepsia Adherence and Compliance Trial), we analyzed information on the frequency of FD symptoms, daily consumption of capsules, onset of action, improvement in FD symptoms, quality of life and patient satisfaction among 600 patients who took FDgard®, a nonprescription medical food specially formulated for the dietary management of FD. (more…)
Author Interviews, Cancer Research, Eli Lilly, Lung Cancer / 20.10.2017

MedicalResearch.com Interview with: Martin Reck, MD, PhD Head of the Department of Thoracic Oncology Head of the Clinical Trial Department Department of Thoracic Oncology at the Lung Clinic Grosshansdorf  MedicalResearch.com: What is the background for this study? What are the main findings? Response: There is an urgent medical need to improve outcomes in pretreated patients with advanced non-small cell lung cancer (NSCLC), in particular those with fast progressing tumors. The Phase 3 REVEL study, which included patients with nonsquamous and squamous forms of NSCLC, demonstrated improved overall survival (OS), progression‐free survival (PFS), and objective response rate (ORR) – independent of histology. This analysis confirmed efficacy - with improvement of ORR, PFS and OS - in poor prognosis patients with fast progressing tumors (after 9, 12 or 18 weeks) without additional toxicity or impact on Quality of Life compared to the intent-to-treat (ITT) population results of REVEL. (more…)
AstraZeneca, Author Interviews, Boehringer Ingelheim, Diabetes, Eli Lilly, J&J-Janssen, Merck, Pharmacology / 18.10.2017

MedicalResearch.com Interview with: Melanie J Davies CBE MB ChB MD FRCP FRCGP Professor of Diabetes Medicine NIHR Senior Investigator Emeritus Diabetes Research Centre Leicester Diabetes Centre – Bloom University of Leicester MedicalResearch.com: What is the background for this study? What are the main findings? Response:  This was the first study to test the effectiveness of an oral GLP-1 in patients with type 2 diabetes. The main findings were that compared to both placebo and a GLP-1, Semaglutide, delivered by sub-cutaneous injection weekly, the oral therapy delivered once a day produced better results than placebo and similar results to injectable GLP-1 with regard to reductions in HbA1c and weight loss. (more…)
Author Interviews, Cost of Health Care, Infections, Merck, Stem Cells, Transplantation / 12.10.2017

MedicalResearch.com Interview with: Dr. Jonathan Schelfhout, PhD Director, Outcomes Research Merck & Co. Inc. North Wales, PA MedicalResearch.com: What is the background for this study? What are the main findings? Response: The cost of hematopoietic stem cell transplantation has received increased attention after it was identified as a top 10 contributor to increasing healthcare costs in an AHRQ 2016 report. Many recent studies have explored the cost of HSCT but additional research is needed on the costly complications that can follow the transplant procedure. This research is particularly relevant for inpatient decision makers, as most transplant centers receive one bundled payment for the transplant and the treatment of any complications over the first 100 days. (more…)
Author Interviews, Boehringer Ingelheim, Clots - Coagulation, Stroke / 05.10.2017

MedicalResearch.com Interview with: Craig Anderson | MD PhD FRACP Executive Director Professor of Neurology and Epidemiology, Faculty of Medicine, UNSW Sydney Neurologist, Neurology Department, Royal Prince Alfred Hospital The George Institute for Global Health at Peking University Health Science Center Haidian District | Beijing, 100088 P.R. China MedicalResearch.com: What is the background for this study? What are the main findings? Response:  There is much controversy over the benefits of a lower dose of intravenous alteplase, particularly in Asia, after the Japanese regulatory authorities approved a dose of 0.6 mg/kg 10 years ago compared to the US FDA and other regulatory authorities approving 0.9 mg/kg 20 years ago.  The investigator inititiated and conducted ENCHANTED trial aimed to determine the effectiveness and safety of these two doses in an international multicentre pragmatic open design. The main results did not confirm the low-dose to be statistically ‘non-inferior’ partly due to the primary outcome measure chosen and partly due to the statistical approach, but it did confirm that the lower dose was safer with less risk of the major complication of this treatment, that of major bleeding in the brain.  However, it would appear that this safety effect was offset by some reduce efficacy in terms of functional recovery. The aim of this secondary analysis of the trial data was to examine in more detail the differences between low and standard dose alteplase according to the participants’ age, ethnicity (Asian vs non-Asian) and severity of neurological deficit at the time of treatment.  We did this because the popular belief is that a lower dose might be preferred in older people, and Asians, because of the potential for more likelihood of bleeding, and preferentially to use the standard dose in those with more severe strokes potentially due to greater ‘clot burden’ from a blocked artery to the brain. The results showed that the main findings on the outcome of surviving free of disability were the same according to age, ethnicity and stroke severity – that is, there was no preferential dose in any of these groups.  Similarly, the safety benefit of low dose alteplase on brain haemorrhage, did not clearly translate into clinical disability outcomes in any of the patient groups studied. (more…)