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…)
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…)
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…)
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…)
AACR, Author Interviews, Biomarkers, Cancer Research, MD Anderson, Pharmaceutical Companies / 09.05.2020

MedicalResearch.com Interview with: David S Hong, M.D MD Anderson Department of Investigational Cancer Therapeutics Division of Cancer Medicine University of Texas MedicalResearch.com: What is the background for this study? Response: Larotrectinib is a first-in-class, CNS active, oral TRK inhibitor exclusively designed to treat tumors with an NTRK gene fusion and does not have secondary targets. In previous presentations and published in The Lancet Oncology, larotrectinib demonstrated robust tumor-agnostic efficacy in an integrated dataset of 159 adult and pediatric patients with TRK fusion cancer across three clinical trials (Feb 2019 data cut-off date). In these studies, the objective response rate (ORR), according to investigator assessment, was 79% (95% confidence interval [CI], 72 – 85%), with a complete response rate of 16%. In this analysis presented at AACR 2020, we sought to evaluate the outcomes in patients from the integrated data set based on different baseline characteristics, including prior lines of therapy and Eastern Cooperative Oncology Group (ECOG) performance status. ECOG measures how the disease impacts a patient. ECOG describes a patient’s level of functioning with a numbering scale (0-5) so physicians can uniformly describe a patient’s ability to care for themselves, daily activity and physical activity (selfcare, walking, working, etc). (more…)
Author Interviews, Biogen, Neurological Disorders, Pharmaceutical Companies / 30.04.2020

MedicalResearch.com Interview with: Toby Ferguson, M.D., Ph.D. Vice President, Head Neuromuscular Development Unit Biogen MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by SMA, who is primarily affected and incidence? Response: Spinal muscular atrophy (SMA) is a rare, genetic, neuromuscular disease characterized by a loss of motor neurons in the spinal cord and lower brain stem that can result in severe, progressive muscle atrophy and weakness. Approximately one in 10,000 live births have a diagnosis of SMA. It is a leading genetic cause of infant mortality; however, people of all ages are impacted by the disease. More than three years ago, SPINRAZA (nusinersen) became the first FDA-approved treatment option for SMA. The DEVOTE study, which recently treated its first patient, is designed to evaluate the safety and potential for even greater efficacy of SPINRAZA when administered at a higher dose than currently approved for the treatment of SMA. The Phase 2/3 randomized, controlled, dose-escalating study will be conducted at approximately 50 sites around the world and aims to enroll individuals of all ages with SMA. (more…)
Author Interviews, COVID -19 Coronavirus, Pharmaceutical Companies / 02.04.2020

MedicalResearch.com Interview with: Dr. Larry Schlesinger MD Professor, President and CEO Texas Biomed MedicalResearch.com: What is the background and mission of Texas Biomed? Response: Texas Biomedical Research Institute (Texas Biomed) is a not-for-profit, independent research institute with a strong history of pioneering, biomedical breakthroughs that have contributed to the world of science and human health for nearly 80 years. The Texas Biomed mission is to pioneer and share scientific breakthroughs that protect you, your families and our global community from the threat of infectious diseases. Texas Biomed is capitalizing on its strengths – outstanding collaborative scientists and unique assets and resources. Texas Biomed is home to the nation’s only privately-owned BSL4 facility, five fully outfitted BSL3 facilities with the latest technologies and the Southwest National Primate Research Center (SNPRC). The Institute focuses on a core understanding of the basic biology of infectious diseases, animal model development, and studies to move therapies and vaccines to human clinical trials. The Institute’s independent, nonprofit business model moves science from the bench to clinical trials faster and with less bureaucracy. (more…)
Author Interviews, Bristol Myers Squibb, Cancer Research, Pharmaceutical Companies / 19.03.2020

MedicalResearch.com Interview with: https://www.cgen.com/ Anat Cohen-Dayag, Ph.D. President and CEO Compugen MedicalResearch.com: What is the background for this announcement? Would you discuss Compugen’s underlying cancer hypothesis regarding the targeting of multiple checkpoint pathways to enhance tumor response? Response: Cancer immunotherapy has revolutionized the landscape for cancer treatments by providing new drug options leading to lasting benefits for patients. Yet, response rates vary greatly across different cancer indications, leaving a significant unmet medical need for many patients and a continuing challenge to discover new biological pathways that can serve for the development of new cancer immunotherapies for non-responsive and refractory patients. Using a computational approach which is designed to discover new biological pathways and drug targets, we identified PVRIG as a novel immune checkpoint and a newly discovered inhibitory pathway in the DNAM axis. Our hypothesis is that PVRIG and TIGIT (another inhibitory pathway discovered by us and others) are two parallel and complementary inhibitory pathways in the DNAM axis and that in certain tumor types and patient populations, there may be a need to block both PVRIG and TIGIT in order to enhance anti-tumor immune responses. Moreover, reported molecular intersections between the DNAM axis and the PD-1 pathway, the most prevalent pathway targeted by approved immunotherapies, suggest that there is a linkage between these three pathways. As such, our PVRIG inhibitor may work in synergy with PD-1 and TIGIT inhibitors, suggesting that various drug combinations may be required to address these three pathways based on their dominance in different cancer patients and cancer indications. With this recently announced Phase 1/2 triple combination study, we will be directly testing our hypothesis of an intersection between the three parallel immune checkpoint pathways – PVRIG, TIGIT and PD-1 – and that the simultaneous blockade of these pathways has the potential to synergistically enhance anti-tumor immune response and expand the reach of cancer immunotherapy to patients non-responsive or refractory to approved immunotherapies.  (more…)
Author Interviews, COVID -19 Coronavirus, Pharmaceutical Companies, Vaccine Studies / 17.03.2020

MedicalResearch.com Interview with: Nathalie Charland PhD Senior Director, Scientific and Medical Affairs Medicago  MedicalResearch.com: What is the background for this study? What are the main findings? Response: We started to work on solutions as soon as we were able to obtain the appropriate genetic information for the new COVID-19. Medicago is committed to advancing therapeutics against life-threatening diseases worldwide.  (more…)
Author Interviews, Cost of Health Care, JAMA, Pharmaceutical Companies / 04.03.2020

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

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

MedicalResearch.com Interview with: Robert E. Dudley, Ph.D. Chairman, Chief Executive Officer and President Clarus Therapeutics Dr. Dudley discusses the recent announcement that Clarus Therapeutics, Inc. has launched  JATENZO® (testosterone undecanoate) capsules for the treatment of appropriate men with testosterone deficiency (hypogonadism): MedicalResearch.com: What is the background for this announcement? Response: JATENZO® is the first and only oral softgel testosterone undecanoate and the first oral testosterone product approved by the U.S. FDA in more than 60 years. JATENZO is indicated for testosterone replacement therapy in adult males for conditions associated with a deficiency or absence of endogenous testosterone. The launch of JATENZO means that physicians and men living with testosterone deficiency due to genetic or structural abnormalities finally have a safe and effective oral testosterone replacement therapy. We are proud to commercially launch this unique oral formulation to healthcare providers and the appropriate patients who they treat. JATENZO is now available at pharmacies across the country. (more…)
Author Interviews, Dartmouth, JAMA, Pharmaceutical Companies, Primary Care / 27.01.2020

MedicalResearch.com Interview with: Steven Woloshin, MD, MS Professor of Medicine and Community and Family Medicine Professor, The Dartmouth Institute for Health Policy and Clinical Practice MedicalResearch.com: What is the background for this study? Response: Industry spends more on detailing visits and free samples than any other form of prescription drug marketing.  There is good evidence that these activities can lead to more use of expensive new drugs over equally effective cheaper options.  Given these concerns there have been efforts by some hospitalls and practices to restrict these forms of marketing. We asked physicians in group practices delivering primary care about how often pharmaceutical reps visit their practice and whether they have a free sample closet.  (more…)
Author Interviews, Cancer Research, Genetic Research, Pharmaceutical Companies / 26.11.2019

MedicalResearch.com Interview with: Ambry GeneticsRachid Karam, MD PhD Director, Ambry Translational Genomics Lab Ambry Genetics MedicalResearch.com: What is the background for this study? Response: Standard DNA testing for hereditary cancer risk excludes large portions of DNA, thereby missing some mutations. In addition, DNA testing can produce inconclusive results and fail to determine that an error in our DNA increases cancer risk. These limitations impact patients and their families because doctors may not have the information needed to recommend appropriate preventive, early detection, or therapeutic steps. Additionally, relatives may not be referred for genetic testing and obtain the care they would otherwise have gotten if they had learned they had mutations. The study looked at how the addition of RNA genetic testing to standard DNA testing for hereditary cancer risk was able to increase diagnostic yield. The study looked at the first 2,500 patients that received Ambry Genetics +RNAinsight™, paired RNA and DNA genetic testing for hereditary cancer risk. The data from this study showed that the addition of RNA genetic testing to DNA testing (1) identified new mutations that would have been missed with DNA testing alone, and (2) clarified inconclusive results as disease-causing. (more…)
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…)
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, 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…)
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, 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…)
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, Diabetes, Pharmaceutical Companies, Weight Research / 12.03.2019

WeightControl.com Interview with: Dr. Lynn Kramer, MD FAAN VP and Chief Clinical Officer & Chief Medical Office Eisai Co., Ltd WeightControl.com: What is the background for this announcement? Response: On February 25th, Eisai announced that the U.S. Food and Drug Administration (FDA) accepted its supplemental New Drug Application to potentially update the label for BELVIQ® (lorcaserin HCI) CIV 10 mg twice-daily/BELVIQ XR (lorcaserin HCI) CIV once daily to include long-term efficacy and safety data from CAMELLIA-TIMI 61, a clinical trial of BELVIQ in 12,000 overweight and obese patients with cardiovascular (CV) disease and/or multiple CV risk factors such as type 2 diabetes mellitus (T2DM). (more…)
Author Interviews, Dermatology, Pharmaceutical Companies / 05.03.2019

MedicalResearch.com Interview with: Dr. James Q. Del Rosso, D.O., FAOCD, Dermatologist Research Director and Principal Investigator Del Rosso Dermatology Research Center, Las Vegas, NV Galderma Consultant MedicalResearch.com: What is the background for this study? What are the main findings?
  • The ANSWER study, a 12-week, randomized, multicenter, Phase 4, Phase 3b in Canada and Europe clinical trial, is the first study of its kind to compare the efficacy and safety of combination therapy with Oracea® (doxycycline, USP) 40 mg Capsules + Soolantra® (ivermectin) Cream, 1% versus Soolantra® (ivermectin) Cream, 1% monotherapy in 273 adults with severe papulopustular rosacea (IGA 4) at clinical trial sites in the United States, Canada and Europe (Czech Republic, Poland, Hungary and Germany).
  • Results showed the combination therapy with Oracea Capsules + Soolantra Cream was well tolerated and effective with a faster onset of action than Soolantra Cream given as monotherapy. Key highlights of the study include:
  • The mean reduction in percentage of inflammatory lesions from baseline to Week 12 was significant with combination therapy compared to monotherapy (80.29% vs. 73.56%, respectively; p=0.032).
  • 5 times as many patients taking combination therapy achieved 100% clearance of inflammatory lesions by Week 12 compared with monotherapy (17.8% vs. 7.2%, respectively; p=0.006).
  • Over 2 times as many patients taking combination therapy achieved 100% clear (IGA 0) by Week 12 compared with monotherapy (11.9% vs. 5.1%, respectively; p=0.043).
  • Combination therapy was generally well tolerated and no discontinuation of treatments due to side effects.
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Author Interviews, Cost of Health Care, Dartmouth, JAMA, Pharmaceutical Companies / 09.01.2019

MedicalResearch.com Interview with: Steven Woloshin, MD, MS Professor Co-director of the Center for Medicine and Media The Dartmouth InstituteSteven Woloshin, MD, MS Professor Co-director of the Center for Medicine and Media The Dartmouth Institute MedicalResearch.com: What is the background for this study? What are the main findings? What influence does medical marketing have on medical care and drug prices? Response: There are published studies looking at promotional spending mostly for drugs (DTC and professional).  This paper is unique because it is such a broad look including not just drugs but also marketing of disease (in "awareness campaigns"), health services and laboratory tests. What is new here is the size and scope of marketing.  For context, $29.9 billion spent on promoting prescription drugs, disease awareness campaigns, health services, and laboratory tests corresponds approximately to $1000 per American.    For context, FDA's total budget is around $5 billion – and NIH's total budget is about $30 billion. This figure is up from $17.7 billion in 1997, with the most rapid increase in DTC promotion of prescription drugs and health services.   Pharmaceutical marketing to professionals (detailing visits and samples) accounted for most spending and remained high despite policies to limit industry influence. $30 billion is of an underestimate (egg, we did not include monies spent on professional marketing (detailing) of laboratory tests, health services or devices, the value of drug coupons/discounts/rebates, company marketing budgets, lobbying or campaign contributions). Further it is just the tip of the iceberg – marketing works so promotional spending is an important driver of why medical care is so expensive:  it leads to more – and more expensive - tests and treatments. (more…)