BELVIQ®: FDA accepts sNDA To Include Long Term Safety/Efficacy Data

WeightControl.com Interview with:

Dr. Lynn Kramer, MD FAANVP and Chief Clinical Officer & Chief Medical OfficeEisai Co., Ltd

Dr. Kramer

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).

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Risankizumab for Moderate to Severe Psoriasis: High Rates of Durable Clearance Through One Year

MedicalResearch.com Interview with:

Anne Robinson, Pharm DExecutive Scientific DirectorAbbVie

Dr. Robinson

Anne Robinson, Pharm D
Executive Scientific Director
AbbVie

MedicalResearch.com: What is the background for the risankizumab data presented at the American Academy of Dermatology 2019 Annual Meeting?

Response: Abstracts presented by AbbVie at the American Academy of Dermatology (AAD) 2019 Annual Meeting highlight additional data from the Phase 3 clinical trial program evaluating the safety and efficacy of risankizumab, an investigational interleukin-23 (IL-23) inhibitor. The registrational program for risankizumab evaluated more than 2,000 adult patients with moderate to severe plaque psoriasis across four pivotal studies. Continue reading

Oracea® Capsules + Soolantra Cream Effective for Inflammatory Rosacea

MedicalResearch.com Interview with:

Dr. James Q. Del Rosso, D.O., FAOCD, DermatologistResearch Director and Principal InvestigatorDel Rosso Dermatology Research Center, Las Vegas, NVGalderma Consultant

Dr. Del Rosso

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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Lilly Announces Phase 3 Results for Atopic Dermatitis Treatment

MedicalResearch.com Interview with:

Lotus Mallbris, MD PhD Vice President, Head of Global Immunology Drug Development Platform Team Leader at Lilly

Dr. Mallbris

Lotus Mallbris, MD PhD
Dermatologist and Vice President, Head of Global Immunology Drug Development
Platform Team Leader at Lilly

MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by atopic dermatitis? How common is this condition? 

Response:The BREEZE-AD1 and BREEZE-AD2 clinical trials are multicenter, randomized, double-blind, placebo-controlled, Phase 3 studies to evaluate the efficacy and safety of baricitinib monotherapy in adult patients with moderate to severe atopic dermatitis. These are two of five studies that will be part of the placebo-controlled data program intended to support global registrations.

Atopic dermatitis, a serious form of eczema, is a chronic, relapsing skin disease characterized by intense itching, dry skin and inflammation that can be present on any part of the body. It affects approximately 1-3 percent of adults worldwide.

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Single-Dose Tafenoquine to Prevent Malaria Relapse

MedicalResearch.com Interview with:
Malaria CDC imageGavin C.K.W. Koh, MB BChir MA PhD MCRP DTM&H

Department of Drug Discovery Unit for Diseases of the Developing World
GlaxoSmithKline | GSK

MedicalResearch.com: What is the background for this study?

Response: Malaria still remains one of the greatest global healthcare challenges so, as part of GSK’s efforts to fight diseases that disproportionately impact the poorest, we have been working on tafenoquine as a potential medicine for malaria for over 20 years.  In 2008, GSK entered into a collaboration with the not-for-profit product development partnership, Medicines for Malaria Venture (MMV), to develop tafenoquine as an anti-relapse medicine for patients infected with a particular species of malaria called Plasmodium vivax malaria.

  1. vivaxmalaria is estimated to cause around 7.5 million clinical infections every year. The disease may cause fever, chills, vomiting, malaise, headache and muscle pain, and in some cases, can lead to severe malaria and be fatal.

Unlike other malaria species such as P. falciparumP. vivax also has the ability to lie dormant in the liver from where it may periodically reactivate to cause relapses of P. vivax malaria. A single P. vivax infection may therefore give rise to multiple episodes of malaria, in the absence of a new mosquito bite. These relapses can occur weeks, months or even years after the initial infection. The dormant liver forms of the parasite cannot be treated with most other antimalarial treatments.

Another issue is that the only medicine currently available to stop the relapse is primaquine, a medicine approved in the 1950s, which must be given for 14 days. Given this length of treatment course, many people do not comply with the full course, which results in reduced effectiveness.

The aim of the DETECTIVE study was to look at the effectiveness of treatment in preventing relapse over six months with a 1-day course of tafenoquine, a 14-day course of primaquine, or placebo, with all patients also receiving a 3-day course of chloroquine, a medicine that is used to treat the initial infection.

MedicalResearch.com: What are the main findings?

Response: The DETECTIVE study met its primary endpoint. A significantly greater proportion of patients in the tafenoquine group did not have relapses compared to patients in the placebo group. A similar result was observed for the patients in the primaquine group compared to the placebo group. When considering the compliance issue of primaquine in the real-world setting, we saw that more than 95% of patients in the primaquine group took their treatment as instructed in the setting of a clinical study. From a safety perspective, adverse events from the study were consistent with the known safety profile of tafenoquine.

MedicalResearch.com: What should readers take away from your report?

Response: The positive results of the DETECTIVE study demonstrate the efficacy and safety of tafenoquine in an unprecedented single-dose for relapsing malaria.  These data supported the approval of the medicine by the US Food and Drug Administration and Australia Therapeutic Goods Administration in 2018 marking it as the first new medicine for the radical cure of P. vivax malaria in more than 60 years. Having the data published in the NEJM will help other P. vivax malaria endemic countries as they strive towards malaria elimination. 

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

Response: The DETECTIVE study was conducted in adult patients with P. vivax malaria and a significant part of the global burden of the disease is in children. GSK and MMV are therefore studying the use of tafenoquine in paediatric patients aged 6 months to 16 years (TEACH study).  In addition, since vivax malaria in Indonesia is frequently resistant to chloroquine, we are conducting another study that is looking at tafenoquine plus dihydroartemisinin–piperaquine as the blood schizonticide (INSPECTOR study) in patients with P. vivax malaria in Indonesia.

Disclosures: The DETECTIVE study was supported by GSK and MMV.

Citation:

Single-Dose Tafenoquine to Prevent Relapse of Plasmodium vivax Malaria
Marcus V.G. Lacerda, M.D., Alejandro Llanos-Cuentas, M.D., Srivicha Krudsood, M.D., Chanthap Lon, M.D., David L. Saunders, M.D., Rezika Mohammed, M.D., Daniel Yilma, M.D., Dhelio Batista Pereira, M.D., Fe E.J. Espino, M.D., Reginaldo Z. Mia, M.D., Raul Chuquiyauri, M.D., Fernando Val, Ph.D., et al.

January 17, 2019
N Engl J Med 2019; 380:215-228
DOI: 10.1056/NEJMoa1710775

Jan 18, 2019 @ 1:24 am 

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Marketers Spend At Least Six Times FDA Budget on Promotion of Medical Services

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.

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XARELTO® Associated With a Decreased Risk of Recurrent VTE

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

Paul Burton MD, PhD, FACC
Vice President, Medical Affairs
Internal Medicine
Janssen Scientific Affairs, LLC.

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: More than 900,000 Americans experience a venous thromboembolism (VTE) each year, with about one-third of these occurrences being fatal. Once a person experiences a VTE, they are at increased risk of a repeat occurrence. Guidelines currently recommend standard anticoagulant therapy with a Factor Xa inhibitor, like XARELTO® (rivaroxaban), for three months or longer. For those people who have had a VTE and stop anticoagulant therapy, as many as 10 percent of them will experience another VTE within one year and 20 percent within three years.

This study examined extended use of XARELTO® after the recommended three-month treatment period in patients who experienced an initial VTE, showing XARELTO® was associated with a decreased risk of recurrent VTE with no increase in major bleeding during this time period.  Continue reading

Healthcare Costs in Patients with Cancer Rise with Increasing Risk of Venous Thromboembolism

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

Paul Burton MD, PhD, FACC
Vice President, Medical Affairs
Internal Medicine
Janssen Scientific Affairs, LLC.

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Despite being largely preventable, venous thromboembolism (VTE) is the second leading cause of death in people with cancer. The risk of VTE is five times greater in people with cancer than those without cancer, and that risk is magnified in those receiving certain types of chemotherapy, in the newly diagnosed and in those with more advanced, metastatic disease. This 6,194-patient study examined economic burden associated with VTE, and found patients newly diagnosed with cancer who are at a higher risk of a VTE had significantly higher all-cause and VTE-related health care costs compared to patients with a lower risk of VTE. Continue reading

Lower Overall Costs with Rivaroxaban (XARELTO® ) vs Warfarin Among Morbidly Obese Patients with Venous Thromboembolism

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

Paul Burton MD, PhD, FACC
Vice President, Medical Affairs
Internal Medicine
Janssen Scientific Affairs, LLC.

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Treatment of venous thromboembolism (VTE) is complicated among morbidly obese patients. Current guidelines do not recommend use of Factor Xa inhibitors in these patients due to limited clinical data available. That’s why Janssen undertook this study to examine XARELTO® (rivaroxaban) in these patients. In this 5,780-patient retrospective study, results found patients treated with XARELTO® had a similar risk of recurrent VTE and major bleeding compared to those taking warfarin.

However, treatment with XARELTO® was associated with less all-cause health care resource utilization (HCRU) (e.g., inpatient hospitalizations and outpatient visits) and reduced total medical costs compared to warfarin. Of note, patients taking XARELTO® had an average $2,829 lower total medical costs per patient per year (PPPY) than those taking warfarin, which was mainly driven by lower hospitalization costs. Continue reading

How Do Patients With Multiple Myeloma Weight Treatment Options?

MedicalResearch.com Interview with:

Caroline McKay, PhD Real World Value & Evidence, Oncology Janssen Scientific Affairs

Dr. McKay

Caroline McKay, PhD
Real World Value & Evidence, Oncology
Janssen Scientific Affairs

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Considering patient preferences in treatment decision-making in oncology is growing in importance. While recent introduction of new treatments for multiple myeloma have improved survival and the possibility of sustaining longer remission periods, regimen options still vary with respect to efficacy, safety, and dosing.

Therefore, patients and providers must consider tradeoffs inherent in making treatment decisions that are growing in complexity. Despite this, there is a lack of research describing patient preferences within the context of currently available treatment regimens. To address this gap, this study examined how multiple myeloma patients evaluate, or weigh, treatment options. Key findings from the research are that treatment preferences do not appear to be static, but instead suggest that the relative importance of treatment attributes may change over time and treatment history.

Further, patients place higher importance on overall survival and progression-free survival than other treatment attributes, and may be willing to accept an increase in the risk of serious side effects and reduced convenience in exchange for greater efficacy; however, when efficacy is comparable, patients appear to place greater weight on dosing frequency than on the duration of treatment administration, i.e., more frequent dosing appears to be less preferable to patients than longer administration/infusion time.  Continue reading

ASH18: CENTAURUS Study Evaluates Dosing Schedule of DARZALEX® (daratumumab) for High Risk Smoldering Multiple Myeloma

MedicalResearch.com Interview with:

Peter Voorhees, MD Plasma Cell Disorders Program Department of Hematologic Oncology and Blood Disorders Levine Cancer Institute Atrium Health

Dr. Vorhees

Peter Voorhees, MD
Plasma Cell Disorders Program
Department of Hematologic Oncology and Blood Disorders
Levine Cancer Institute
Atrium Health

MedicalResearch.com: What is the background for this study?

Response: All multiple myeloma arises from its precursor conditions, monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM). Although the rate of progression to multiple myeloma for patients with MGUS is low (~5% over 5 years), patients with SMM have a ~50% likelihood of requiring therapy for their multiple myeloma within the first 5 years of diagnosis. For those at intermediate to high risk of disease progression, early intervention to delay progression of disease, thereby averting disease-related morbidities related and potentially changing the natural course of the disease, is highly desirable.

On the other hand, given the fact that these patients are by definition asymptomatic and would otherwise be monitored off treatment, it is critical that any intervention applied in this group of patients is well tolerated. Daratumumab is a highly attractive candidate in this particular space, because it has single agent activity in heavily-pretreated relapsed/refractory multiple myeloma and a favorable side effect profile relative to many other myeloma therapeutics. Additionally, given the importance of impaired immune surveillance in multiple myeloma, the immuno-stimulatory effects of daratumumab in the bone marrow microenvironment could potentially reawaken robust T cell responses to the disease.

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CLL: Overall Treatment Savings With Ibrutinib (Imbruvica) Despite Higher Prescription Costs

MedicalResearch.com Interview with:

Dr. Sundaram

Murali Sundaram, MBA, Ph.D.
Director of Real World Value and Evidence
Oncology, Janssen

MedicalResearch.com: What is the background for this study?

Response: Ibrutinib is a novel Bruton’s tyrosine kinase (BTK) inhibitor approved for the treatment of patients with newly diagnosed chronic lymphocytic leukemia (CLL).

Ibrutinib is administered orally while standard of care (CD20 monoclonal antibody-based chemoimmunotherapy [CIT]) is administered intravenously. This difference in route of administration impacts what type of benefit covers these treatments (i.e., pharmacy benefit for oral ibrutinib and medical benefit for intravenous CIT).

Previous studies evaluating the costs burden of patients treated with ibrutinib versus CIT did not include the full spectrum of real-world healthcare costs.

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FDA Grants Orphan Drug Designation For Eosinophilic Granulomatosis with Polyangiitis

MedicalResearch.com Interview with:

Mr. Tosh Butt Vice President, Respiratory AstraZeneca

Mr. Butt


Mr. Tosh Butt

Vice President, Respiratory
AstraZeneca

Mr. Butt discusses the recent announcement that the FDA has granted Orphan Drug Designation for Fasenra for the treatment of Eosinophilic Granulomatosis with Polyangiitis. 


MedicalResearch.com:
What is the background for this announcement? Can you tell us a little more about Eosinophilic Granulomatosis with Polyangiitis/Churg Strauss? How does it differ/resemble severe eosinophilic asthma?

  • The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for FASENRA™ (benralizumab) for the treatment of Eosinophilic Granulomatosis with Polyangiitis (EGPA). The ODD application was based on epidemiology demonstrating the rarity of the disease (<200k US patients) and a scientific rationale that FASENRA may benefit patients with this condition. The core role of eosinophilia in EGPA and FASENRA’s demonstrated eosinophil-depleting properties provided this rationale and suggest it may deliver benefit to patients with EGPA.
  • EGPA is a rare autoimmune disease that can cause damage to multiple organs and tissues. EGPA is characterized by inflammation of blood vessels and the presence of elevated levels of eosinophils, a type of white blood cell. All patients with EGPA have very high levels of eosinophils at some point in their disease. FASENRA induces rapid and near-complete depletion of eosinophils in the blood and has proven efficacy in severe eosinophilic asthma, which suggest it may deliver benefit to patients with EGPA. 

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Asthma: Biologic Benralizumab (FASENRA) Reduced Need For Rescue Medication

MedicalResearch.com Interview with:
“Asthma Inhaler” by NIAID is licensed under CC BY 2.0Sean O’Quinn MPH
Director, Patient Reported Outcomes
AstraZeneca 

MedicalResearch.com: What is the background for this study? How does benralizumab differ from traditional medications for asthma?

Response:  FASENRA™ (benralizumab 30mg for subcutaneous injection as add-on maintenance therapy in severe eosinophilic asthma for patients 12 years and older) has a strong clinical profile, including powerful efficacy against exacerbations and the ability to improve lung function. Benralizumab is a respiratory biologic that binds directly to the IL-5α receptor on eosinophils and attracts natural killer cells to induce rapid and near-complete depletion of eosinophils via apoptosis. (NOTE: The mechanism of action of FASENRA in asthma has not been definitively established.) Benralizumab is not indicated for treatment of other eosinophilic conditions or for relief of acute bronchospasm or status asthmaticus. The most common adverse reactions include headache and pharyngitis.

Dependence on rescue medications is indicative of poor asthma control. In the Phase III SIROCCO/CALIMA trials, patients with severe eosinophilic asthma had significantly reduced exacerbation frequency and improved lung function when treated with benralizumab 30mg Q8W (first three doses Q4W) vs. placebo.

Less was known about the effects of benralizumab on rescue medication usage—specifically daily total rescue medication use, daytime and nighttime rescue medication use, and nighttime awakenings requiring rescue medication use. The aim of this analysis was to understand the potential treatment effects of benralizumab on these parameters.  Continue reading

Dapagliflozin (FARXIGA): Reduction in Albuminuria Cannot Be Predicted by Clinical Characteristics

MedicalResearch.com Interview with:

Dr-Danilo Verge.png

Dr. Verge

Danilo Verge MD MBA
Vice President, CVRM Global Medical Affairs
AstraZeneca

MedicalResearch.com: What is the background for this study?

Response: Dapagliflozin, an SGLT2 inhibitor (sodium-glucose co-transporter 2), has been shown to improve glycemic control by decreasing glucose reabsorption in the kidneys and inducing urinary glucose clearance. SGLT2 inhibitors have also been shown to be effective in lowering albuminuria and stabilizing eGFR (estimated glomerular filtration rate). The effect of dapagliflozin on UACR (urine albumin-to-creatinine ratio) has been shown to vary among patients.

The objective of this post-hoc analysis, based on the pooled data from 11 randomized, placebo-controlled clinical trials, was to assess baseline characteristics and concurrent changes in cardiovascular (CV) risk markers associated with UACR response to dapagliflozin.

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Real-World Dosing of RAASi are Associated With Risk of Adverse Events in CKD

MedicalResearch.com Interview with:

Lei Qin

Lei Qin

Lei Qin MS
Director, Health Economics and Payer Analytics
AstraZeneca

MedicalResearch.com: What is the background for this study?

Response: Renin-angiotensin-aldosterone system inhibitors (RAASi) are guideline-recommended therapies for patients with chronic kidney disease (CKD), but are commonly prescribed at suboptimal doses, which has been associated with worsening clinical outcomes. The objective of our study was to estimate the real-world associations between RAASi dose and adverse clinical outcomes in patients prescribed RAASi therapies with new-onset CKD in the UK.

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LOKELMA (Sodium zirconium cyclosilicate) for Elevated Potassium: Results of the HARMONIZE GLOBAL Study

MedicalResearch.com Interview with:

Rahul Agrawal MD PhD VP, Global Medicines Leader AstraZeneca

Dr. Agrawal

Rahul Agrawal MD PhD
VP, Global Medicines Leader
AstraZeneca

MedicalResearch.com: What is the background for this study?  

About the study: HARMONIZE Global is a Phase III, randomized, multicenter, double-blind, placebo-controlled trial involving 267 patients with hyperkalemia (mean potassium levels greater than 5.0 mEq/L) in 47 study locations across the Asia Pacific region, which will support registration in Japan, Taiwan, Korea and Russia.

Study design: The trial design of HARMONIZE Global is similar to HARMONIZE (NCT02088073) but evaluated two doses of LOKELMATM (sodium zirconium cyclosilicate) instead of three, as well as patients in different geographical regions. Continue reading

FDA Approves Dupixent: Only Self-Administered Biologic for Moderate-to-Severe Asthma

MedicalResearch.com Interview with:
RegeneronNeil Graham,  M.B.B.S., M.D., M.P.H
VP of Immunology & Inflammation
Regeneron

MedicalResearch.com: What is the background for this announcement? 

Response: Patients with moderate-to-severe asthma often have uncontrolled, persistent symptoms despite standard-of-care therapy that may make them suitable for treatment with a biologic therapy. They live with coughing, wheezing and difficulty breathing, and are at risk of severe asthma attacks that may require emergency room visits or hospitalizations. [i],[ii] Oral corticosteroids can provide relief for severe, short-term symptoms. However, their chronic use is limited to the most severe patients due to the potential for serious side effects. [iii],[iv]

A particular type of inflammation contributes to the cause of uncontrolled symptoms in multiple inflammatory diseases such as asthma and atopic dermatitis.[v] Dupixent is a medicine that inhibits the overactive signaling of interleukin-4 (IL-4) and interleukin-13 (IL-13), two key proteins that contribute to this type of inflammation. This inhibits cytokine-induced inflammatory responses, including the release of proinflammatory cytokines, chemokines, nitric oxide, and IgE; however, the mechanism of action of Dupixent in asthma has not been definitively established. Continue reading

FDA Approves Single Dose XOFLUZA™ For Uncomplicated Flu

MedicalResearch.com Interview with:

Mark D. Eisner, MD, MPH Vice President, Product Development Immunology Infectious Disease and Ophthalmology Genentech 

Dr. Mark Eisner

Mark D. Eisner, MD, MPH
Vice PresidentProduct Development Immunology, Infectious Disease and Ophthalmology Genentech

Dr. Eisner discusses the announcement that the FDA has approved XOFLUZA™ (baloxavir marboxil) for the treatment of acute, uncomplicated influenza.

MedicalResearch.com: What is the background for this announcement?

Response: Each year, an estimated 3-11 percent of the U.S. population gets the flu, and it can be very serious, resulting in hospitalization or even death. Since 2010, the Centers for Disease Control and Prevention (CDC) estimates that the flu has resulted annually in 9.2 to 35.6 million illnesses, 140,000 to 900,000 hospitalizations and 12,000 to 80,000 deaths. The severity of last year’s flu season underscores the need for new medical options beyond currently available antivirals.

XOFLUZA was granted Priority Review in June 2018 based on results from the Phase III CAPSTONE-1 study of a single dose of XOFLUZA compared with placebo or oseltamivir 75 mg, twice daily for five days, in otherwise healthy people with the flu, as well as results from a placebo-controlled Phase II study in otherwise healthy people with the flu.

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Less Smoking Linked to Lower Antibody Levels in Modern Rheumatoid Arthritis Patients

MedicalResearch.com Interview with:

Pr Gilles Boire, M.D., M. ScService de rhumatologie Département de médecine Faculté de médecine et des sciences de la santé Université de Sherbrooke

Prof. Boire

Pr Gilles Boire, M.D., M. ScService de rhumatologie
Département de médecine
Faculté de médecine et des sciences de la santé
Université de Sherbrooke

MedicalResearch.com: What is the background for this study?

Response: Rheumatoid arthritis (RA) patients are heterogeneous at initial presentation, in response to treatments and according to their outcomes. No clinical features and very few biomarkers, except autoantibodies such as anti-Cyclic Citrullinated Peptides/Proteins (CCP), identify patients with divergent prognostic trajectories.

To help improve early prognostic classification, we initiated 20 years ago the single center longitudinal observational Early Undifferentiated PolyArthritis (EUPA) study of consecutive patients presenting with recent-onset inflammatory polyarthritis, 90% of which fulfill classification criteria for RA at baseline. Our registry includes 739 very early RA patients (median symptom duration 3.6 months), rapidly treated to joint remission (i.e. 0/66 swollen joint) and followed over 5 years. Each patient visit is linked to biosamples and to sequential radiographs scored according to the modified Sharp/van der Heijde method. As we had the clinical impression that clinical features of recruited patients were evolving, we compared patients from 3 periods (1998-2004; 2005-2010; 2011-2017).  Continue reading

Single Dose Baloxavir marboxil (Xofluza) Has Potential To Improve Treatment of High-Risk Flu

MedicalResearch.com Interview with:

Mark D. Eisner, MD, MPH Vice President, Product Development Immunology Infectious Disease and Ophthalmology Genentech 

Dr. Eisner

Mark D. Eisner, MD, MPH
Vice President, Product Development Immunology
Infectious Disease and Ophthalmology
Genentech 

MedicalResearch.com: What is the background for this study?

Response: CAPSTONE-2 is a Phase III multicenter, randomized, double-blind study that evaluated the efficacy and safety of a single dose of baloxavir marboxil compared with placebo and oseltamivir in people 12 years and older who are at a high risk of complications from the flu. The Centers for Disease Control and Prevention (CDC) defines people at high risk for serious flu complications to include adults 65 years of age or older, or those who have conditions such as asthma, chronic lung disease, morbid obesity or heart disease.

A total of 2,184 participants enrolled in the study and were randomly assigned to receive a single, oral dose of 40 mg or 80 mg of baloxavir marboxil (according to body weight), placebo or 75 mg of oseltamivir twice daily for five days. The primary objective of the study evaluated the efficacy of a single dose of baloxavir marboxil compared with placebo by measuring the time to improvement of influenza symptoms. Key secondary endpoints compared outcomes in baloxavir marboxil versus placebo or oseltamivir – these included time to resolution of fever, time to cessation of viral shedding, infectious virus detection in swabs of the nose and throat, prescription of antibiotics and influenza-related complications.

Genentech announced initial results from the study on July 16, 2018 but the full data was presented for the first time during a late-breaking oral presentation at the annual IDWeek meeting in San Francisco, CA on October 6, 2018.

Baloxavir marboxil is a first-in-class, single-dose investigational oral medicine with a novel proposed mechanism of action designed to target the influenza A and B viruses, including oseltamivir-resistant strains and avian strains (e.g. H7N9, H5N1). Baloxavir marboxil is the first potential influenza treatment to demonstrate a clinically meaningful benefit for people highly vulnerable to serious influenza complications in clinical trials.

The FDA accepted a New Drug Application (NDA) and granted Priority Review to baloxavir marboxil as a single-dose, oral treatment for acute, uncomplicated influenza in people 12 years and older. The NDA was based on results from the Phase III CAPSTONE-1 study of a single dose of baloxavir marboxil compared with placebo or oseltamivir 75 mg, twice daily for five days, in otherwise healthy people with the flu. Results from a placebo-controlled Phase II study in otherwise healthy people with the flu were included as supporting data in the NDA. The FDA is expected to make a decision on approval by December 24, 2018. Continue reading

Topical Minocycline Foam for Moderate-to-Severe Acne Meets Phase 3 Study Endpoints

MedicalResearch.com Interview with:
foamixDavid Domzalski
CEO

Foamix Pharmaceuticals

MedicalResearch.com: What is the background for this study?  How does FMX101 differ from other treatment for acne, ie benzoyl peroxide, topical clindamycin etc? 

Response: This study measures the safety and efficacy of a topical foam formulation of the antibiotic minocycline, for the treatment of moderate-to-severe acne.

Minocycline is one of the most commonly used products for the treatment of acne, but is currently only available in an oral dosage form.

Significant side effects are associated with oral minocycline, including GI upset, photosensitivity, headaches, dizziness, and other potential effects on the CNS.  In addition to the side effects associated with oral minocycline, many currently available topical acne medications contain ingredients which can be drying and irritating to the skin.  These side effects can be frustrating to patients and potentially impact overall compliance to their treatment regimen.  The study addresses important unmet needs in dermatology to determine whether providing patients with a topical dosage form of minocycline may have potential advantages over existing products.

In our first two Phase 3 clinical studies, >95% of facial local tolerability signs and symptoms were classified as “none” or “mild,” including dryness, erythema and itching.  Also, our topical minocycline foam, FMX101, is a natural triglyceride-based vehicle that does not contain ingredients that serve as  primary irritants or surfactants.  We believe that FMX101, if approved, would be the first topical minocycline available for the treatment of acne and provide a novel and much needed treatment option for patients who suffer from the physical and psycho-social effects of acne. Continue reading

New Antibiotic Combination IMI/REL Can Treat Resistant Infection With Less Kidney Toxicity

MedicalResearch.com Interview with:

Michelle Hoffman Brown Associate Principal Scientist at Merck Merck

Michelle Brown

Michelle Hoffman Brown
Associate Principal Scientist
Merck

MedicalResearch.com: What is the background for this study? What are the kidney risks of using colistin to treat carbapenem-resistant bacterial infections?

Response: Gram-negative pathogens are responsible for half of all healthcare-associated infections and their ability to resist traditional antibiotics makes them more dangerous for seriously ill patients in a healthcare setting. The need for new approaches to treat these pathogens is essential and this trial aimed to evaluate the efficacy and safety of imipenem/relebactam (IMI/REL) for the treatment of these challenging infections.

Nephrotoxicity is a common complication of colistin-based therapy and is the potential adverse experience of greatest concern to prescribing clinicians, limiting its use to treat carbapenem-resistant bacterial infections. Relebactam is a novel β-lactamase inhibitor that restores imipenem activity against many imipenem-non-susceptible strains of Gram-negative pathogens. In the Phase 3 RESTORE-IMI 1 study (NCT02452047), IMI/REL was shown to be as effective as, but better tolerated than, colistin plus imipenem, including as demonstrated by a lower incidence of treatment-emergent nephrotoxicity (prespecified secondary endpoint). This analysis looked at additional renal safety data from the RESTORE-IMI 1 trial.  Continue reading

Retrotope Expands Compassionate Use Access to RT001 for Amyotrophic Lateral Sclerosis

MedicalResearch.com Interview with:
RetrotopeRobert J Molinari, Ph.D.

President and CEO
Retrotope, Inc.

 

MedicalResearch.com: What is the background for this study?
How does RT001 differ from other treatments for neurodegenerative diseases? 

Response: Lipid peroxidation in critical cell and mitochondrial membranes represents a common pathway of cell death in many neurodegenerative diseases, regardless of the initiating trigger of original damage, e.g. aberrant gene expression, misfolded proteins such as tau and amyloid, environmental insults, etc. This hypothesis was supported by work showing that stabilizing lipids (using virtually indistinguishable isotopic variants of the original dietary molecules) was able to mitigate disease-related cell dysfunction, and reverse disease in a variety of animal models, and more recently, in human patients enrolled in clinical trials.

It has been known for decades that lipid peroxidation detritus of oxidized membrane fats are present in most all diseases of degeneration and aging, including Alzheimer’s disease, Parkinson’s disease, atherosclerosis, ALS, Huntington’s, and fatal inborn genetic errors resulting in neurodegenerative diseases (among others). The hypothesis that controlling such oxidation could provide therapeutic benefit was abandoned when numerous formal trials of classical antioxidants, e.g. Co-enzyme Q, Vitamin E, and others failed to provide meaningful benefit. We believed that the antioxidant mechanism used to attempt control of the membrane oxidation was flawed, but that the target itself was correct. Indeed, by using a new class of oral drugs that are lipids fortified against damage at the key susceptible bonds, we observed reduction in lipid peroxidation damage that halted, and even reversed neurodegenerative disease progression. Dosed in amounts and forms similar to omega 3 and 6 supplements, these drugs exhibited profound disease modification across a broad range of diseases in animal models, placebo controlled- and open label- human trials. Continue reading

How Much Do Clinical Trials For New Medications Really Cost?

MedicalResearch.com Interview with:

Thomas J Moore Senior Scientist Institute for Safe Medication Practices Lecturer, Department of Epidemiology and Biostatistics The George Washington University Milken Institute of Public Health Alexandria, VA 22314

Thomas J Moore

Thomas J Moore AB
Senior Scientist Institute for Safe Medication Practices
Lecturer, Department of Epidemiology and Biostatistics
The George Washington University
Milken Institute of Public Health
Alexandria, VA 22314

MedicalResearch.com: What is the background for this study? What are the main findings?

  • The study provides realistic cost estimates of pivotal clinical trials that establish drug benefits to support FDA approval of 59 new drugs released for marketing in 2015-2016.
  • The median estimated cost was just $19 million, with half of the 138 trials studied clustered between $12 million and $33 million.
  • The highest cost trials–with estimates up to $345 million–were for new drugs that were similar to drugs already available and already proven in treating serious illnesses. 

Continue reading