Author Interviews, Mental Health Research, Pharmaceutical Companies / 14.01.2024

MedicalResearch.com Interview with: Lauren Davis Lauren C. Davis, MBS Department of Medical Education Geisinger Commonwealth School of Medicine Scranton, PA 19409   MedicalResearch.com: What is the background for this study? Response: Financial conflicts of interest (COIs) resulting from ties between academia and industry have been under scrutiny for their potential to hinder the integrity of medical research. COIs can lead to implicit bias, compromise the research process, and erode public trust (1-6). The American Psychiatric Association’s Diagnostic and Statistical Manual of Mental Disorders (DSM), standardizes symptom criteria and codifies psychiatric disorders. This manual contributes to the approval of new drugs, extensions of patent exclusivity, and can influence payers and mental health professionals seeking third-party reimbursements. Given the implications of the DSM on public health, it is paramount that it is free of industry influence. Previous research has shown a high prevalence of industry ties among panel and task force members of the DSM-IV-TR and DSM-5, despite the implementation of a disclosure policy for the DSM-5 (7,8). This study (9) determined the extent and type of COIs received by panel and task-force members of the DSM-5-TR (2022) (10). As the DSM-5-TR did not disclose COI, we used the Center for Medicare and Medicaid Services Open Payments (OP) database (11) to quantify them. (more…)
Author Interviews, Moderna, NEJM, Pharmaceutical Companies, Respiratory, Vaccine Studies / 13.12.2023

MedicalResearch.com Interview with: Eleanor Wilson, M.D Moderna, 200 Technology Sq. Cambridge, MA 02139 MedicalResearch.com: What is the background for this study? What are the main findings and side effects (if any)? Response: The ConquerRSV trial is a randomized, double-blind, placebo-controlled study of approximately 37,000 adults 60 years or older in 22 countries. The primary efficacy endpoints were based on two definitions of RSV-associated lower respiratory tract disease (RSV-LRTD) defined as either two or more symptoms, or three or more symptoms of disease. Vaccine efficacy was 83.7% (95.88% confidence interval [CI], 66.0 to 92.2) against RSV-associated lower respiratory tract disease with at least two signs or symptoms and 82.4% (96.36% CI, 34.8 to 95.3) against the disease with at least three signs or symptoms. Most adverse reactions were mild to moderate in severity and included injection site pain, fatigue, headache, myalgia, and arthralgia. (more…)
Author Interviews, Eli Lilly, Gastrointestinal Disease / 03.11.2023

MedicalResearch.com Interview with: Lotus Mallbris, M.D., Ph.D., Senior Vice President of Immunology Development Eli Lilly MedicalResearch.com: Would you briefly describe the condition of Crohn's disease and who is most susceptible to this disease? Response: Crohn's disease is a form of inflammatory bowel disease (IBD) that can cause systemic inflammation manifested as abdominal pain, diarrhea, fever and weight loss. It can lead to intestinal obstruction, fibrosis and other complications. Approximately 900,000 patients in the U.S. and 1 million patients in Europe are currently suffering from Crohn’s disease, and 70% of those have moderate to severe disease. Although the majority of patients are started on conventional therapy such as corticosteroids and immunomodulators, many will unfortunately progress to having moderate to severe disease. Furthermore, current therapies to treat Crohn’s disease often fail to achieve remission for a majority of patients, and of the patients who do achieve remission, a substantial proportion lose it within the first year. (more…)
Author Interviews, Eli Lilly, Gastrointestinal Disease, NEJM / 02.11.2023

MedicalResearch.com Interview with: Marla C. Dubinsky, MD Professor of Pediatrics and Medicine Icahn School of Medicine at Mount Sinai Co- director, Susan and Leonard Feinstein IBD Clinical Center Mount Sinai Health System MedicalResearch.com: What is the background for this study? Would you briefly describe the condition of UC? Response: Lucent 1 and Lucent 2 were the induction and maintenance registration trials studying the efficacy and safety of mirikizumab in patients 18 years and older with moderate to severely active ulcerative colitis. Mirikizumab is a monoclonal antibody targeting the p19 subunit of IL23. Lucent-3 is the open label extension arm for those meeting inclusion criteria after completing Lucent 2. This study evaluated the long term efficacy and safety of mirikizumab in patients with ulcerative colitis who completed a total of 104 weeks of active mirikizumab treatment. Ulcerative colitis is a chronic incurable inflammatory condition of colon. Common symptoms include diarrhea, blood in the stool, abdominal cramping and bowel urgency. Bowel urgency is one of the most burdensome symptoms that a patient with you could experience. (more…)
Author Interviews, Dermatology, Eli Lilly, Immunotherapy / 18.05.2023

MedicalResearch.com Interview with: Lotus Mallbris, MD PhD Dermatologist andSenior Vice President Global Immunology Development and Medical Affairs  Lilly   MedicalResearch.com: What is the background for this study?  Would you briefly describe what is meant by atopic dermatitis and types treated in this study? Response: First, this study specifically evaluated lebrikizumab, a novel, investigational, monoclonal antibody that selectively binds to interleukin 13 (IL-13) with high-affinity and high potency. Inflammation due to over-activation of the IL-13 pathway plays a central role in the pathogenesis of moderate-to-severe atopic dermatitis, commonly called eczema. This secondary analysis focused on patients treated with lebrikizumab from the 16-week induction periods of the ADvocate 1 and ADvocate 2 studies and the ADhere study. In the trials, we assessed the presence or absence of face or hand dermatitis in patients with moderate-to-severe atopic dermatitis. If present at baseline, at 16 weeks, clinicians assessed the change from baseline on a scale of cleared, improved, no change, or worsened. Only patients with face and hand dermatitis were evaluated as part of the analysis. (more…)
Author Interviews, Genetic Research, Neurological Disorders, Novartis / 29.03.2023

MedicalResearch.com Interview with: Sitra Tauscher-Wisniewski, MD Vice President Clinical Development & Analytics Novartis Gene Therapies MedicalResearch.com: What is the background for this study? Would you briefly describe the condition of Spinal muscular atrophy (SMA)? Response: At the 2023 Muscular Dystrophy Association Conference, we presented new data from two of our  Long-Term Follow-Up (LTFU) studies, LT001 and LT002, which show the continued efficacy and durability of Zolgensma across a range of patient populations, with an overall benefit-risk profile that remains favorable. LT001 is a 15-year ongoing observational LTFU study following the Phase 1 START patients, who were the very first patients to receive our gene replacement therapy. LT-002 is a voluntary Phase 4 15-year ongoing follow-up safety and efficacy study of Zolgensma IV and investigational intrathecal (IT) OAV101 in patients previously treated in the Phase 3 IV studies (STR1VE-US, STR1VE-EU, STR1VE-AP, SPR1NT) and the Phase 1 IT study (STRONG). Spinal muscular atrophy (SMA) is a rare, devastating genetic disease that leads to progressive muscle weakness, paralysis, and when left untreated in one of its most severe forms (SMA Type 1), permanent ventilation or death in 90% of cases by age 2. It is caused by a lack of a functional survival motor neuron 1 (SMN1) gene, and in the most severe forms results in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. (more…)
Author Interviews, Dermatology, Eli Lilly, NEJM / 26.03.2023

MedicalResearch.com Interview with: Jonathan Silverberg, MD, PHD, MPH Professor Director of Clinical Research Director of Patch Testing George Washington University School of Medicine and Health Sciences Washington, DC MedicalResearch.com: What is the background for this study? What are the main findings? Response: Lebrikizumab was previously shown to be safe and effective as a treatment for moderate-severe atopic dermatitis in a phase 2 study. These Phase 3 randomized placebo-controlled trials are the largest studies to date of lebrikizumab in AD. They showed that lebrikizumab was safe and highly effective for the treatment of moderate-severe atopic dermatitis. These studies will hopefully support the approval of lebrikizumab in the United States later this year. (more…)
Author Interviews, Cost of Health Care, JAMA, Pharmaceutical Companies, Yale / 22.01.2023

MedicalResearch.com Interview with: Neeraj Patel Medical Student (MS-2), Yale School of Medicine New Haven, CT MedicalResearch.com: What is the background for this study? Response: Direct-to-consumer pharmaceutical advertising has been increasing in popularity for the past two decades or so, particularly via television. But it’s highly controversial. Only two high-income countries (the U.S. and New Zealand) widely permit this type of advertising for prescription drugs. Critics have pointed to a growing body of literature that suggests that direct-to-consumer advertising for prescription drugs can be misleading, lead to inappropriate prescribing, and inflate healthcare costs. Proponents have argued that it improves public health by promoting clinically beneficial prescribing. (more…)
Author Interviews, Biogen, NEJM, Rheumatology / 15.09.2022

MedicalResearch.com Interview with: Nathalie Franchimont, M.D., Ph.D. Senior Vice President, Head of Multiple Sclerosis and Immunology Head of the Multiple Sclerosis and Immunology Development Unit Biogen MedicalResearch.com: What is the background for this study? What are the main findings? Response: Systemic lupus erythematosus (SLE) is a chronic autoimmune disease that affects multiple organ systems. Rash and arthritis are among the most frequent manifestations of the disease and severe organ damage can also occur especially when organs like the kidney are affected. Litifilimab (known as BIIB059) is a monoclonal antibody being studied for the potential treatment of SLE and cutaneous lupus erythematosus (CLE). The Phase 2 LILAC study evaluated litifilimab versus placebo in two parts: Part A in participants who have SLE with active joint and skin manifestations; and Part B in participants with active CLE, including chronic and subacute subtypes, with or without other organ involvement. Results from the SLE portion of the study (Part A) show litifilimab met the study’s primary endpoint by significantly reducing total active joint count compared to placebo. Total active joint count was defined as the total number of tender or swollen joints. Litifilimab was generally well tolerated, with most reported adverse events (AEs) rated as mild or moderate. Note, this Phase 2 trial was not powered to assess secondary endpoints. Based on these positive Phase 2 results, Biogen is currently enrolling participants into the Phase 3 TOPAZ-1 and TOPAZ-2 studies, which will evaluate the efficacy and safety of litifilimab in participants with active SLE worldwide. Part B results from LILAC were published separately in NEJM on July 28, 2022 and expand the body of evidence supporting litifilimab as a potential first-in-class therapy for cutaneous lupus erythematosus in addition to SLE. (more…)
Author Interviews, Cost of Health Care, JAMA, Pharmaceutical Companies / 31.08.2022

MedicalResearch.com Interview with: Prof. Katharina Blankart, PhD Faculty of Economics and Business Administration University of Duisburg-Essen Essen, Germany MedicalResearch.com: What is the background for this study? Response: Given the high drug prices and policy discussions, we were interested whether the US may miss opportunities from medical innovation in availability of medicines compared to Germany. Since 2011, Germany has a unique way to determine value of new medicines after regulatory approval and to negotiate prices. We aimed to find out differences in availability of medicines in these two countries and timing of availability. We evaluated the differences in timing of availability and to characterize medicines not available to one of the two countries. (more…)
Author Interviews, Education, JAMA, Pharmaceutical Companies / 06.07.2022

MedicalResearch.com Interview with: SooYoung VanDeMark, MBS Geisinger Commonwealth School of Medicine Scranton, Pennsylvania MedicalResearch.com:  What is the background for this study?   Response: Health care providers utilize subscription-based, point-of-care databases such as DynaMed and UpToDate to provide clinical care guidance and remain current on the latest evidence-based findings. Both of these websites maintain this content through a cadre of physician contributors who write and edit articles for these sites. These physician contributors are required to self-report any conflicts of interest (COI) as outlined by the respective policies on each website. However, prior COI research into similarly self-regulated areas, such as medical and pharmacology textbooks, and clinical practice guidelines, has found both appreciable potential COI and inconsistencies between self-reported and industry mandated disclosures (1-3). This study (4) explored the accuracy of physician contributors to DynaMed and UpToDate by comparing their self-reported disclosure status with the financial remunerations they received from the healthcare industry (e.g., pharmaceutical companies) as reported to the U.S. Centers for Medicare and Medicaid Services’ Open Payments database. Physician contributors who reported “nothing to disclose” on their respective article topic but had an entry on Open Payments for having received money from industry, were classified as discordant and, thus, as having the potential for a COI. Additionally, total remuneration, gender, and payment category were investigated more in depth for each database. (more…)
Author Interviews, Cancer Research, Pharmaceutical Companies / 20.05.2022

MedicalResearch.com Interview with: Robert Wild, Ph.D Chief Scientific Officer Dracen Pharmaceuticals   MedicalResearch.com:  What is the background for the development of sirpiglenastat, i.e., would you briefly explain what is meant by glutamine antagonist? Response: Cancer cells consume and use glutamine for both energy generation and as a source of carbon and nitrogen for biomass accumulation. Many oncogenes and tumor suppressor genes drive large-scale metabolic reprogramming of tumors into glutamine addiction. These highly proliferating tumors create a hostile and immunosuppressive tumor microenvironment (TME), which is nutrient- depleted, acidic and hypoxic in nature. Sirpiglenastat (DRP-104), is a novel broad-acting glutamine antagonist that inhibits all 10 known glutamine metabolism enzymes. DRP-104 was designed to preferentially inhibit glutamine metabolism in tumors and associated TME and not in normal tissues, providing a large therapeutic window. DRP-104 demonstrates powerful direct apoptotic (cell death) properties and immune modulatory mechanisms through broad remodeling of the TME to infer DRP-104 impacts immune-metabolism. Inhibition of glutamine metabolism leads to:
  • Induction of apoptosis in glutamine-addicted tumor cells leading to substantial single-agent activity and tumor regressions
  • Rebalance of the TME that enhances immune cell infiltration and function
  • Differentiation and modulation of adaptive and innate immune cells toward a highly proliferative, activated and long-lived phenotype for a long-term durable response.
(more…)
Author Interviews, Boehringer Ingelheim, NEJM, Pulmonary Disease / 15.05.2022

MedicalResearch.com Interview with: Professor Luca Richeldi MD PhD Chair and Head, Division of Pulmonary Medicine Gemelli University Hospital - IRCCS Catholic University of the Sacred Heart Rome MedicalResearch.com:  What is the background for this study?  Would you briefly explain the condition of Idiopathic Pulmonary Fibrosis? Response: As you may know, Idiopathic pulmonary fibrosis (IPF) is a progressive, irreversible lung disease with high mortality. IPF is one of the more common forms of progressive fibrosing interstitial lung diseases and its symptoms of IPF include breathlessness during activity, a dry and persistent cough, chest discomfort, fatigue and weakness. IPF is considered a “rare” disease, but it affects more than 3 million people worldwide. Currently, there are two approved antifibrotic drugs that slow, but do not stop, the progression of fibrosis. Therefore, there is a need for additional treatments that can be used alone or with existing antifibrotic therapies. Pre-clinical research indicated that phosphodiesterase 4 (PDE4) inhibition is associated with anti-inflammatory and antifibrotic effects that may be beneficial in patients with idiopathic pulmonary fibrosis. In this Phase 2, double-blind, placebo-controlled trial, we investigated the efficacy and safety of BI 1015550, an oral preferential inhibitor of the PDE4B subtype, in patients with IPF. Patients were randomly assigned in a 2:1 ratio to receive BI 1015550 at a dose of 18 mg twice daily or placebo. (more…)
Pharmaceutical Companies / 21.03.2022

medications-pharmaceuticals-drugsMedicine is now more widely available than ever before, and shelves are filling up with different drugs that have all sorts of impacts on the human body. Recent events such as the covid pandemic have shown how quickly new medicines can be developed and distributed throughout the world. The manufacturing process has been refined in a big way, and swift changes continue to be made on this front. However, if you were wanting to find out a bit more about how medicine is made, there is a lot to say on this particular front. Everything starts off in the initial development phase before a drug goes through its research and development, passes a number of checks and balances, and is eventually released into the wider world as a whole. So, let’s go into a little bit more detail about how medicine is actually made. (more…)
Alzheimer's - Dementia, Author Interviews, Eli Lilly, NEJM / 16.03.2021

MedicalResearch.com Interview with: Stephen Salloway, M.D., M.S. Director of Neurology and the Memory and Aging Program, Butler Hospital Martin M. Zucker Professor of Psychiatry and Human Behavior Professor of Neurology, Alpert Medical School of Brown University Providence, RI 02906  MedicalResearch.com: What is the background for this study? Response: This 78 week phase 2 study tested donanemab in patients with early Alzheimer’s disease. Donanemab is a an anti-amyloid monoclonal antibody that targets the N3 pyroglutamate epitope.  MedicalResearch.com: What are the main findings? Response: The drug produced a substantial lowering of amyloid plaques and showed a slowing in cognitive decline. Key innovations included using PET scans to ensure all patients were amyloid positive and had a moderate level of tau build-up and switching from drug to placebo once the amyloid level was below the expected cut-off for Alzheimer’s disease. There were no new safety signals. The main side-effect was amyloid-related imaging abnormalities (ARIA) that have been seen with other anti-amyloid treatments. ARIA is managed with regular safety MRI scans.  Donanemab is now being tested in a larger phase 3 trial that could lead to regulatory approval. (more…)
Author Interviews, Brain Cancer - Brain Tumors, Cancer Research, Immunotherapy, Pharmaceutical Companies / 22.02.2021

MedicalResearch.com Interview with: https://www.inovio.com/Jeffrey Skolnik, MD Senior Vice President, Clinical Development INOVIO MedicalResearch.com: What is the background for this technology? Would you tell us a little about the brain tumor, Glioblastoma Multiforme? How common is it, whom does it primarily affect?  Response: Glioblastoma (GBM) is the most common malignant brain tumor, affecting more than 10 thousand people each year in the United States. Most people diagnosed with GBM are above the age of 60 years, although GBM can be diagnosed at any age, including in children and young adults. Despite decades of research, GBM remains almost universally fatal. GBM is a tumor of the glial cells of the brain, and current therapies are directed at removing tumor with surgery and killing residual tumor cells with radiation and chemotherapy. More recently, with the introduction of immunotherapies such as immune checkpoint inhibitors (ICI) for the treatment of cancer, clinical studies have tried to add this promising technology to the treatment of GBM. Unfortunately, despite success in other types of cancer, ICIs have not demonstrated any clinical benefit in treating GBM. Newer clinical studies aim at introducing a combination of newer therapies together to try to tackle this terrible disease, and INOVIO’s GBM-001 study is one such example of an innovative approach to treating GBM.    (more…)
Author Interviews, Infections, OBGYNE, Pharmaceutical Companies / 21.01.2021

MedicalResearch.com interview with: Dr. Stephen Brand, Chief Development Officer Mycovia Pharmaceuticals  Dr. Stephen Brand discusses the results of Mycovia’s three Phase 3 studies for recurrent vaginal yeast infections (RVVC )and what’s next for the company.  MedicalResearch.com: What is the background for these Phase 3 studies? Answer: Our Phase 3 clinical program for our oral therapy oteseconazole was comprised of three trials enrolling more than 870 patients at 176 sites across 11 different countries. Two of these trials, referred to as VIOLET were identical Phase 3 randomized, double-blind, placebo-controlled clinical trials to evaluate the safety of oteseconazole and its ability to prevent episodes of recurrent vulvovaginal candidiasis (RVVC), commonly referred to as chronic yeast infection. The trials took place over 48 weeks in subjects with an established disease history of at least three episodes of acute VVC in the past 12 months. More than 650 patients randomized at 125 sites across 11 countries. The VIOLET trials consisted of two parts: During the first part of the study which lasted two weeks after patients presented with an active VVC episode, patients were treated with three sequential 150mg doses of fluconazole. The second part consisted of 12 weeks, when the patient either took oteseconazole 150mg or a placebo once weekly (according to a random assignment), and then a 36-week follow-up period. In addition, subjects participating in the VIOLET trials in the U.S. who remained infection-free at their Week 48 visit were offered the opportunity to participate in an extension study and are being monitored for an additional 48 weeks to further define the long-term protection profile of oteseconazole. Eighty-five subjects are enrolled. The third Phase 3 study, called ultraVIOLET, was designed to complement and extend VIOLET as a 50-week randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of oteseconazole. In addition the study compared the effectiveness of oteseconazole compared to fluconazole, the current standard of care, to treat an acute VVC infection in the RVVC population. A total of 220 patients were randomized at 51 sites in the U.S. for the ultraVIOLET trial. The ultraVIOLET trial consisted of two parts: In the first part of the study RVVC subjects presenting with an active infection were randomized to receive either 2 days of dosing with oteseconazole or 3 sequential 150 mg doses of fluconazole (every 72 hours). The second part consisted of 11 weeks, when the patient took either oteseconazole or a placebo weekly (according to the random assignment from the first part of the study), and then a 37-week follow-up period. (more…)
Asthma, AstraZeneca, Author Interviews / 01.12.2020

MedicalResearch.com Interview with: Frank Trudo, MD MBA Vice President, US Medical, Respiratory & Immunology AstraZeneca  MedicalResearch.com: What is the background for this study? Response: PONENTE is a multicenter, open-label, single-arm, Phase IIIb trial to evaluate the efficacy and safety of reducing daily oral corticosteroids (OCS) use after initiation of 30 mg dose of FASENRA (benralizumab) administered subcutaneously in adult patients with severe eosinophilic asthma on high-dose inhaled corticosteroids plus long-acting beta2-agonist and long-term use of OCS therapy with or without additional asthma controller(s). The trial expands on OCS-sparing data previously seen in the ZONDA Phase III trial by using a faster steroid tapering schedule in patients who did not experience adrenal insufficiency to reduce OCS use from higher doses. Compared to published trials of other biologics, PONENTE has a personalized OCS tapering schedule that allows for more rapid OCS tapering from higher OCS doses, followed by an assessment of the adrenal function as part of decision-making to manage the risk of adrenal insufficiency. PONENTE also has a longer maintenance phase (approximately 24-32 weeks), allowing assessment of the durability of OCS reduction. FASENRA is a monoclonal antibody that binds directly to IL-5 receptor alpha on eosinophils and attracts natural killer cells to induce rapid and near-complete depletion of eosinophils via apoptosis (programmed cell death). MedicalResearch.com: How is it administered?  Response: FASENRA is injected under your skin (subcutaneously) one time every 4 weeks for the first 3 doses, and then every 8 weeks. In 2019, FASENRA was approved in the US for self-administration in a single dose prefilled autoinjector, the FASENRA pen. (more…)
AstraZeneca, Author Interviews, Cancer Research, ESMO, Lung Cancer, NEJM, Yale / 08.10.2020

MedicalResearch.com Interview with: Roy S. Herbst, M.D., Ph.D. Ensign Professor of Medicine (Medical Oncology) and Professor of Pharmacology Chief of Medical Oncology Yale Cancer Center and Smilow Cancer Hospital Associate Cancer Center Director for Translational Research Yale Cancer Center  MedicalResearch.com: What is the background for this study? How does osimertinib differ from prior versions of EGFR-TKI Inhibitors? o   ADAURA is a randomized, double-blinded, global and placebo-controlled Phase III trial in the adjuvant treatment of 682 patients with Stage IB, II, and IIIA EGFRm NSCLC following complete tumor resection and adjuvant chemotherapy as indicated. Patients were treated with osimertinib 80 mg once-daily oral tablets or placebo for three years or until disease recurrence. The primary endpoint is disease free survival (DFS) in Stage II and IIIA patients, and a key secondary endpoint is DFS in Stage IB, II and IIIA patients. Osimertinib is not currently approved in the adjuvant setting in any country. o   Osimertinib is a third-generation, irreversible EGFR-TKI with clinical activity against central nervous system metastases. The results of the Phase III ADAURA trial of osimertinib demonstrate for the first time in a global trial that an EGFR inhibitor can change the course of early-stage EGFR-mutated lung cancer for patients. o   ADAURA results were first presented in May during the American Society of Clinical Oncology ASCO20 Virtual Scientific Program. (more…)
Author Interviews, Eisai, Insomnia / 02.10.2020

MedicalResearch.com Interview with: Margaret Moline, PhD Executive Director, Neurology Business Group, Eisai, Inc Lemborexant International Program Lead and Global Medical Lead MedicalResearch.com: What is the background for this study? What are the main findings?
  • SUNRISE 2 was one of two pivotal Phase 3 studies evaluated in the U.S. Food and Drug Administration’s approval of DAYVIGO (lemborexant) CIV in December 2019.
  • SUNRISE 2 was a pivotal six-month placebo-controlled treatment trial with a 6-month active treatment period including adult patients age 18 or older who met DSM-5 criteria for insomnia disorder.
  • Patients were randomized to placebo (n=325), DAYVIGO 5 mg (n=323), or DAYVIGO 10 mg (n=323) once nightly for the first six months of the study (Treatment Period 1).
  • The primary efficacy endpoint was the mean change from baseline to end of treatment at six months for subjective sleep onset latency (sSOL; the estimated minutes from the time that the patient attempted to sleep until sleep onset).
  • Secondary efficacy endpoints were mean change from baseline to end of treatment at six months subjective sleep efficiency (sSE; the proportion of time spent asleep per time in bed) and subjective wake after sleep onset (sWASO; the minutes of wake from the onset of sleep until wake time). These endpoints were measured by sleep diary.
  • At Virtual SLEEP 2020, a post-hoc analysis of SUNRISE 2 was shared in an oral presentation, which looked specifically at the long-term efficacy and safety of lemborexant in elderly adults with insomnia disorder.
  • Insomnia disorder, a chronic condition with long-term consequences for health and well-being, is prevalent in older adults.
  • This analysis of the SUNRISE 2 data reflects new learnings on the sustained impact of DAYVIGO on sleep onset and sleep maintenance in an older patient population. 
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Author Interviews, Lipids, Regeneron / 26.08.2020

MedicalResearch.com Interview with: Professor F. J. Raal, FRCP, FCP(SA), Cert Endo, MMED, PhD Director, Carbohydrate & Lipid Metabolism Research Unit Professor & Head, Division of Endocrinology & Metabolism, Faculty of Health Sciences, University of the Witwatersrand  MedicalResearch.com: What is the background for this study? Response: The objective of this randomized phase 3 study was to evaluate the efficacy and safety of evinacumab in adult patients with homozygous familial hypercholesterolemia (HoFH), a condition that remains very difficult to treat. The primary endpoint was reduction of low-density lipoprotein cholesterol (LDL-C) from baseline with evinacumab compared to placebo at 24 weeks.  MedicalResearch.com: Would you briefly explain what is meant by homozygous familial hypercholesterolemia? How many individuals may be affected by this disorder? Response: HoFH, or homozygous familial hypercholesterolemia, is the most serious and more rare form of familial hypercholesterolemia (FH).  It is estimated that as many as 1 in 300,000 people worldwide and approximately 1,300 people in the U.S. are affected by HoFH. A person who has HoFH has inherited two FH genes, one from each parent. They therefore have LDL-C levels that are elevated 4-fold or greater from birth and are at high risk for premature atherosclerotic disease and cardiac events which can occur in childhood. While current treatment guidelines recommend early and intensive LDL-C lowering, people with HoFH tend to be less responsive (or unresponsive) to standard lipid-lowering therapies, including high-intensity statins and PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitors and often require lipid apheresis. (more…)
AstraZeneca, Author Interviews, Pulmonary Disease / 22.08.2020

MedicalResearch.com Interview with: Frank Trudo, MD MBA Vice President, US Medical Affairs Respiratory & Immunology AstraZeneca  MedicalResearch.com: What is the background for this study? Response: ETHOS was a randomized, double-blinded, 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. A subset of patients participated in the 4-hour pulmonary function test (PFT) sub-study, with the following primary endpoints: change from baseline in morning pre-dose trough FEV1 at Week 24 at (both doses of budesonide/glycopyrrolate/formoterol fumarate MDI versus glycopyrrolate/formoterol fumarate MDI), and FEV1 area under the curve from 0-4 hours at Week 24 (both doses of budesonide/glycopyrrolate/formoterol fumarate MDI vs budesonide/formoterol fumarate MDI).  (more…)
Asthma, AstraZeneca, Author Interviews / 22.08.2020

MedicalResearch.com Interview with: Frank Trudo, MD MBA Vice President, US Medical Affairs Respiratory & Immunology AstraZeneca     MedicalResearch.com: What is the background for this study?
  • Multiple pathways drive asthma. T2 inflammation-driven asthma is present in many patients with severe asthma and is typically characterized by elevated levels of T2 inflammatory biomarkers, including blood eosinophils, serum IgE and fractional exhaled nitric oxide.
    • Some patients with severe asthma do not present with increased T2 inflammation. However, currently available biologic therapies only target T2-driven inflammation.
  • The PATHWAY trial evaluated the efficacy and safety of three dose regimens of tezepelumab versus placebo as an add-on therapy in patients with a history of asthma exacerbations and uncontrolled asthma despite receiving inhaled corticosteroids/long-acting beta2-agonists with or without oral corticosteroids and additional asthma controllers. Overall, the trial showed tezepelumab significantly reduced annualized asthma exacerbation rates of 71%.
  • This post-hoc analysis presented virtually at ATS evaluated the effect of Tezepelumab treatment on asthma exacerbation rates on a seasonal and weekly basis.
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Author Interviews, Dermatology, FDA, Regeneron, Sanofi / 31.07.2020

MedicalResearch.com Interview with: Elizabeth Laws, PhD Vice President and Global Project Head for Dupilumab/Dupixent Sanofi Marcie Ruddy, MD, MA Strategic Program Direction, Immunology and Inflammation Regeneron  Dr. Laws and Dr. Ruddy discuss the FDA approval of a 300 mg single-dose pre-filled pen for Dupixent® (dupilumab) for all indications in patients aged 12 years and older.   MedicalResearch.com: What is the background for this announcement? What are the main indications for Dupixent? Response: Until now, Dupixent 300 mg dose was available only in pre-filled syringe for administration. The approval of the pre-filled pen provides an additional, easy-to-use option for patients to self-administer Dupixent. Dupixent is approved to treat patients aged 6 years and older with uncontrolled moderate-to-severe atopic dermatitis (AD) and can be used with or without topical treatments. Dupixent is also approved for use with other medicines for the maintenance treatment of uncontrolled moderate-to-severe eosinophilic or oral steroid dependent asthma in patients aged 12 years and older, and with other medicines for the maintenance treatment of uncontrolled chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults, respectively. The pre-filled pen is approved for use in patients prescribed Dupixent who are 12 years of age and older across current indications, at the 300 mg dose. (more…)
Author Interviews, Biogen, Rheumatology / 06.07.2020

MedicalResearch.com Interview with: Nathalie Franchimont, M.D., Ph.D. Vice President Multiple Sclerosis and Immunology Development Unit Biogen MedicalResearch.com: What is the background for this study? Response: BIIB059 is an investigational fully humanized IgG1 monoclonal antibody (mAb) targeting blood dendritic cell antigen 2 (BDCA2) expressed on plasmacytoid dendritic cells (pDCs), a protein present in specific cells within the immune system. An antibody against BDCA2 may potentially interrupt production of interferons, which are inflammatory molecules that are increased in patients with lupus and thought to contribute to disease activity. The LILAC study is two-part, Phase 2, randomized, double-blind trial investigating the efficacy and safety of BIIB059 in patients with cutaneous lupus erythematosus (CLE) and systemic lupus erythematosus (SLE). Data from the CLE portion of the study were recently presented at the European E-Congress of Rheumatology (EULAR) 2020, which was held virtually from June 3-6, 2020. Overall, study participants with CLE who received BIIB059 demonstrated statistically significant reduction of disease activity compared to those who received placebo, as assessed by the Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity (CLASI-A) score. The results were encouraging and it warrants continued evaluation of BIIB059 in patients with CLE. Cutaneous lupus erythematosus is a chronic autoimmune disease wherein the body’s immune system attacks healthy skin, often causing rashes and skin lesions which can be painful or itchy. There is substantial unmet medical need for people with lupus given the limited number of treatment options available to manage this difficult-to-treat and chronic disease. Ultimately, we are motivated by the possibility of bringing potential new treatment options to lupus patients in need. (more…)
AstraZeneca, Author Interviews, NEJM, Pulmonary Disease / 29.06.2020

MedicalResearch.com Interview with: Frank Trudo, MD MBA Vice President, US Medical Affairs Respiratory & Immunology AstraZeneca MedicalResearch.com: What is the background for this study? Response: ETHOS is a randomized, double-blinded, 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. Outcomes in the ETHOS trial included, as a primary endpoint, the rate of moderate or severe exacerbations. MedicalResearch.com: How does PT010 differ from other treatments for COPD?
  • Highly competitive: PT010’s Phase III clinical trial program demonstrates it has a highly competitive clinical profile in decreasing moderate or severe exacerbations. Severe exacerbations were defined as exacerbations leading to hospitalization or death.
  • All-cause mortality: In a secondary endpoint, PT010 showed a 46% reduction in the risk of all-cause mortality compared with glycopyrronium/formoterol fumarate. The data from ETHOS show that reducing risk of all-cause mortality is achievable for patients with this progressive disease and could transform treatment goals in COPD.
  • Two potential dose options: This is also the first time we have seen the benefit of closed triple-combination therapy at two ICS doses, which could transform care by allowing physicians to select the optimal dosing option for individual patients. 
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Author Interviews, Dermatology, Regeneron / 23.06.2020

MedicalResearch.com Interview with: Brad Shumel, MD Senior Director of Medical Affairs, Immunology Regeneron MedicalResearch.com: What is the background for this study? Response: Atopic dermatitis is a chronic inflammatory disease and one of the most common skin disorders in children. Severe atopic dermatitis is characterized by skin lesions that often cover a large body surface area and can include intense, persistent itch. Uncontrolled moderate-to-severe atopic dermatitis can have a physical, emotional and psychosocial impact on children, resulting in sleep deprivation, activity restriction, poor school performance, depression and anxiety that can have a greater impact on quality-of-life. The standard of care for this pediatric population has been topical corticosteroids. Children with severe atopic dermatitis who remain uncontrolled with topical therapies have limited treatment options. This Phase 3 trial was conducted to evaluate the safety and efficacy of dupilumab plus topical corticosteroids (TCS) compared with TCS alone in children with uncontrolled severe atopic dermatitis across two treatment arms – every four weeks and every two weeks (Q4W and Q2W). (more…)
Abbvie, Author Interviews, OBGYNE / 16.06.2020

MedicalResearch.com Interview with: https://www.abbvie.com/   Ayman Al-Hendy, M.D., Ph.D. Investigator for the ELARIS UF-2 clinical trials Professor of Gynecology Director of Translational Research University of Illinois at Chicago   Dr. Al-Hendy discusses the recent announcement that the FDA has approved  ORIAHNN™ for the management of heavy menstrual bleeding due to uterine fibroids in pre-menopausal women. MedicalResearch.com: What is the background for this approval? Uterine fibroids, commonly referred to as uterine leiomyomas, are the most common type of non-cancerous tumor known to impact women of reproductive age (30-50 years old). In fact, studies show that uterine fibroids can occur in up to 70 percent of European American women and over 80 percent of African American women by age 50. As a result of uterine fibroids, women can experience a range of symptoms, the most common being heavy menstrual bleeding (i.e. prolonged and/or frequent bleeding), which can lead to other health effects such as anemia, fatigue, pelvic pain, urinary frequency etc. Uterine fibroid treatment recommendations have historically been based on the size and location of the fibroid(s). When treating larger and more complicated fibroids, healthcare providers have typically believed that surgery is their best course of action, which has made uterine fibroids the leading reason for the hysterectomies performed in the U.S. The FDA approval of ORIAHNN was based on improving care for uterine fibroid sufferers who have had a negative impact on their quality of life due to disruptive symptoms. What makes the approval of ORIAHNN so exciting, is that women now have an oral therapy to directly address heavy menstrual bleeding due to uterine fibroids.  (more…)
Author Interviews, Dermatology, Pediatrics, Pharmaceutical Companies, Regeneron / 15.06.2020

MedicalResearch.com Interview with: Amy S Paller, MD Chair, Department of Dermatology Director, Skin Biology and Diseases Resource-Based Center Walter J. Hamlin Professor of Dermatology Professor of Dermatology and Pediatrics (Dermatology) Feinberg School of Medicine Northwestern University  Dr. Paller discusses the FDA approval of Dupixent® (dupilumab) for children aged 6 to 11 years with moderate-to-severe atopic dermatitis (eczema), whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.  MedicalResearch.com: What is the background for this announcement? Would you briefly discuss what is meant by atopic dermatitis and how it affects children? Response: “Atopic dermatitis, the most common form of eczema, is a chronic inflammatory disease that often appears as a rash on the skin. Moderate-to-severe atopic dermatitis is characterized by rashes that can potentially cover much of the body and can include intense, persistent itching, skin lesions and skin dryness, cracking, redness or darkness, crusting and oozing. Itch is one of the most burdensome symptoms for patients and can be debilitating. This recent FDA approval expands the use of Dupilumab in the U.S. to include children aged 6 to 11 years with uncontrolled moderate-to-severe atopic dermatitis, making it the only biologic medicine approved for this use in this population. Dupilumab is also approved in the U.S. to treat patients aged 12 years and older with moderate-to-severe atopic dermatitis. Moderate-to-severe atopic dermatitis can place a particularly substantial burden on young children aged 6 to 11 years and their families. Limited treatment options leave many of these children to cope with intense, unrelenting itch and skin lesions. Families of these children can spend countless hours helping them to manage their disease.” (more…)