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EC approves label update for AstraZeneca’s Lokelma

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EC approves label update for AstraZeneca’s Lokelma
May 04, 2020: “The European Commission (EC) has approved a dosing and administration label update in the EU for AstraZeneca’s Lokelma (sodium zirconium cyclosilicate) to include patients with hyperkalaemia on stable haemodialysis.

The approval by the EC was based on data from the Phase IIIb DIALIZE trial, the first ever randomised, placebo-controlled trial to evaluate a potassium binder in patients on stable haemodialysis.

The Phase IIIb, multicentre, double-blinded trial investigated the efficacy and safety of Lokelma versus placebo in 196 patients on haemodialysis with hyperkalemia.

Patients were randomised to receive Lokelma or placebo once daily on non-dialysis days for a treatment period of eight weeks.

This included a four-week dose adjustment phase (starting at 5g and titrated weekly in 5g increments up to a maximum of 15g) and a four-week evaluation phase on stable dose.

The trial showed sustained potassium control pre-dialysis for more patients receiving Lokelma, compared with placebo. 41% of patients receiving Lokelma maintained pre-dialysis potassium levels on at least three out of four dialysis treatments after the long interdialytic interval and did not require urgent rescue therapy.

This compared with 1% of patients receiving placebo, making it a statistically significant and clinically meaningful improvement.

The safety profile of Lokelma observed in DIALIZE was consistent with previous trials.

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, said: “More than 300,000 patients with end-stage renal disease in Europe are undergoing haemodialysis and are at risk of experiencing high potassium levels.

This label update provides important guidance to manage hyperkalaemia, a condition which can be life-threatening in these patients if left untreated.”

Lokelma is a highly selective, oral potassium-removing agent currently approved in the US, EU, Canada, Hong Kong, China, Russia and Japan for the treatment of hyperkalemia.

Recently, the Food and Drug Administration (FDA) approved a label update in the US to include a dosing regimen specifically to treat hyperkalaemia in patients with end-stage renal disease on chronic haemodialysis.”

https://pharmafield.co.uk/pharma_news/ec-approves-label-update-for-astrazenecas-lokelma/

Roche’s COVID-19 antibody test receives FDA Emergency Use Authorization and is available in markets accepting the CE mark

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Roche’s COVID-19 antibody test receives FDA Emergency Use Authorization and is available in markets accepting the CE mark

May 03, 2020: “Roche announced that the U.S. Food and Drug Administration (FDA) has issued an Emergency Use Authorization (EUA) for its new Elecsys® Anti-SARS-CoV-2 antibody test.

The test is designed to help determine if a patient has been exposed to the SARS-CoV-2 virus and if the patient has developed antibodies against SARS-CoV-2.

Roche has already started shipping the new antibody test to leading laboratories globally and will ramp up production capacity to high double-digit millions per month to serve healthcare systems in countries accepting the CE mark as well as the U.S.

“Thanks to the enormous efforts of our dedicated colleagues we are now able to deliver a high-quality antibody test in high quantities, so we can support healthcare systems around the world with an important tool to better manage the COVID-19 health crisis,” said Severin Schwan, CEO Roche Group.

”I am in particular pleased about the high specificity and sensitivity of our test, which is crucial to support health care systems around the world with a reliable tool to better manage the COVID-19 health crisis.”

“Our best scientists have worked 24/7 over the last few weeks and months to develop a highly reliable antibody test to help fight this pandemic,” said Thomas Schinecker, CEO Roche Diagnostics.

“Roche is committed to helping laboratories deliver fast, accurate, and reliable results to healthcare professionals and their patients.”

Roche’s SARS-CoV2 antibody test, which has a specificity greater than 99.8% and 100% sensitivity (14 Days post-PCR confirmation), can help assess patients’ immune response to the virus.

As more is understood about immunity to SARS-CoV-2, the test may help to assess who has built up immunity to the virus.

With extensive global manufacturing capabilities, Roche will be able to deliver high double-digit millions of tests per month.

Hospitals and reference laboratories can run the test on Roche’s cobas e analysers, which are widely available around the world.

For countries with specific regulatory requirements, local approval timelines apply. In addition there may be other country-specific regulations, such as import requirements, which will determine when the test becomes available locally.

Roche will work closely with the respective regional representatives to ensure we appropriately support local registration efforts.


An antibody test, also called a serology test, is used to determine whether a person might have gained immunity against a pathogen or not.

The human body makes antibodies in response to many illnesses. In the current situation of the COVID-19 pandemic, antibody tests need to be able to specifically detect antibodies against SARS-CoV-2 with no cross-reactivity to other similar coronaviruses, which could generate a false positive result and thus wrongly indicate potential immunity.

A false positive result happens when a person receives a positive test result, when they should have received a negative result.

False positives are particularly critical when we do not know how many people in a given population have had COVID-19. As of 24 April 2020, no study has evaluated whether the presence of antibodies to SARS-CoV-2 confers immunity to subsequent infection by this virus in humans.


Elecsys® Anti-SARS-CoV-2 is an immunoassay for the in-vitro qualitative detection of antibodies (including IgG) to Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) in human serum and plasma.

Through a blood sample, the test, which is based on an in-solution double-antigen sandwich format, can detect antibodies to the new coronavirus causing COVID-19, which could signal whether a person has already been infected and potentially developed immunity to the virus.

Based on the measurement of a total of 5272 samples, the Elecsys® Anti-SARS-CoV-2 assay has 99.81% specificity and shows no cross-reactivity to the four human coronaviruses causing common cold.

This means it can lower the chance of false positives due to the detection of similar antibodies that may be present in an individual, but are specific for coronaviruses other than SARS-CoV-2.

Elecsys® Anti-SARS-CoV-2 detected antibodies with 100% sensitivity in samples taken 14 days after a PCR-confirmed infection. The importance of specificity and sensitivity of a particular test will be dependent on its purpose and disease prevalence within a given population.

Hospitals and reference laboratories can run the test on Roche’s cobas e analysers, which are widely available around the world.

These fully automated systems can provide SARS-CoV-2 test results in approximately 18 minutes for one single test, with a test throughput of up to 300 tests/hour, depending on the analyser.“

https://www.roche.com/media/releases/med-cor-2020-05-03.htm

CDC launches national viral genomics consortium to better map SARS-CoV-2 transmission

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CDC launches national viral genomics consortium to better map SARS-CoV-2 transmission
May 1, 2020: CDC has kicked off the SARS-CoV-2 Sequencing for Public Health Emergency Response, Epidemiology and Surveillance (SPHERES) consortium, which will greatly expand the use of whole genome sequencing (WGS) of the COVID-19 virus.

This national network of sequencing laboratories will speed the release of SARS-CoV-2 sequence data into the public domain.

SPHERES will provide consistent, real-time sequence data to the public health response teams investigating cases and clusters of COVID-19 across the country.

It will help them better understand how the virus is spreading, both nationally and in their local communities. Better data, in turn, will help public health officials interrupt chains of transmission, prevent new cases of illness, and protect and save lives.

“The U.S. is the world’s leader in advanced rapid genome sequencing. This coordinated effort across our public, private, clinical, and academic public health laboratories will play a vital role in understanding the transmission, evolution, and treatment of SARS-CoV-2.

I am confident that our finest, most skilled minds are working together to help us save lives today and tomorrow,” said CDC Director Robert Redfield, M.D.

Tracking the COVID-19 virus as it evolves

Genomic sequence data can give unprecedented insight into the biology of SARS-CoV-2, the virus that causes COVID-19, and help define the changing landscape of the pandemic.

By sequencing viruses from across the United States, CDC and other public health authorities can monitor important changes in the virus and use this information to guide contact tracing, public health mitigation efforts, and infection control strategies.

The SPHERES consortium is an ambitious effort to coordinate SARS-CoV-2 genome sequencing nationally, organizing dozens of smaller, individual efforts into a single, distributed network of laboratories, institutions and corporations.

The consortium combines the expertise, technology, and resources of 40 state and local public health departments, several large clinical laboratories, and over two dozen collaborating institutions across the federal government, academia, and the private sector.

SPHERES will establish best practices and consensus data standards, accelerate open data sharing, and establish a pool of resources and expertise to help bring cutting-edge technology to the national COVID-19 response.

SPHERES data open, shared

Consortium members share a commitment to rapid open sequence sharing.

They plan to submit all useful sequence data into public repositories at the National Library of Medicine’s National Center for Biotechnology Information (NLM/NCBI), the Global Initiative on Sharing Avian Influenza Data (GISAID), and other public sequence repositories.

This will help ensure that that viral sequence data from across the United States is rapidly available for public health decision making and freely accessible to researchers everywhere.

Consortium members include:

  • Federal agencies and laboratories
  • Centers for Disease Control and Prevention, Office of Advanced Molecular Detections
  • Argonne National Laboratory
  • National Institute of Allergy and Infectious Diseases, Office of Genomics and Advanced Technology
  • National Institute of Standards and Technology
  • National Library of Medicine’s National Center for Biotechnology Information
  • Walter Reed Army Institute of Research
  • State/local public health laboratories
  • Arizona
  • California
  • Delaware
  • District of Columbia
  • Florida
  • Hawaii
  • Massachusetts
  • Maine
  • Maryland
  • Michigan
  • Minnesota
  • North Carolina
  • New Mexico
  • North Dakota
  • Nevada
  • New York
  • Utah
  • Virginia
  • Washington
  • Wisconsin
  • Wyoming
  • Academic Institutions
  • Baylor University
  • Cornell University
  • Fred Hutchinson Cancer Research Center
  • Mount Sinai School of Medicine
  • New York University
  • Northern Arizona University
  • University of Buffalo
  • University of California, Berkeley
  • University of California, Davis
  • University of California, Irvine
  • University of California, Los Angeles
  • University of California, San Francisco
  • University of California, Santa Cruz
  • University of Chicago
  • University of Maryland
  • University of Minnesota
  • University of Nebraska
  • University of New Mexico
  • University of Washington
  • Yale University
  • https://www.cdc.gov/media/releases/2020/p0501-SARS-CoV-2-transmission-map.html

Coronavirus (COVID-19) Update: Daily Roundup May 1, 2020

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Coronavirus (COVID-19) Update: Daily Roundup May 1, 2020

May 01, 2020: “The U.S. Food and Drug Administration today announced the following actions taken in its ongoing response effort to the COVID-19 pandemic:

  • Today, the FDA issued an Emergency Use Authorization (EUA) for the investigational antiviral drug remdesivir.

    The EUAExternal Link Disclaimer allows for remdesivir to be distributed in the U.S. and administered by health care providers, as appropriate, to treat suspected or laboratory-confirmed COVID-19 in adults and children hospitalized with severe disease.

    Severe disease is defined as patients with low blood oxygen levels or needing oxygen therapy or more intensive breathing support such as a mechanical ventilator.

    Based on evaluation of the emergency use authorization criteria and the scientific evidence available, it was determined that it is reasonable to believe that remdesivir may be effective in treating COVID-19, and that, given there are no adequate, approved, or available alternative treatments, the known and potential benefits to treat this serious or life-threatening virus currently outweigh the known and potential risks of the drug’s use.
  • Today, the FDA updated its guidance on convalescent plasma and associated web page. The updated guidance provides clarification for investigators on how to submit investigational applications for COVID-19 convalescent plasma.

    In addition, the guidance includes updated information regarding potential donors. Previously, the FDA’s guidance noted that to qualify, individuals should have complete resolution of symptoms for 28 days or resolution for 14 and a negative diagnostic test.

    The revised guidance recommends that individuals  have complete resolution of symptoms for at least 14 days prior to donation A negative lab test for COVID-19 disease is not necessary to qualify for donation.

    The revised guidance also clarifies that FDA does not recommend storing a retention sample from the convalescent plasma donation for single patient emergency INDs.
  • Given the many questions people have about grocery shopping safety, the FDA has posted a video, 12 Tips for Grocery Shopping During the PandemicExternal Link Disclaimer, to advise consumers.
  • The FDA has also posted an updated COVID-19 Response At-A-Glance Summary. It contains updates on major agency activities as well as some important facts and figures.
  • To help address shortages of continuous renal replacement therapy (CRRT) products during the COVID-19 public health emergency, today the FDA issued an EUA to Fresenius Medical Care for emergency use of the multiFiltrate PRO System and multiBic/multiPlus Solutions.

    CRRT is a type of dialysis therapy used to filter and clean the blood when the kidneys are damaged or are not functioning normally.

    The Fresenius multiFiltrate PRO System and multiBic/multiPlus Solutions have been authorized to provide CRRT to treat patients in an acute care environment during the COVID-19 public health emergency.
  • Diagnostics update to date:
    • During the COVID-19 pandemic, the FDA has worked with more than 380 test developers who have said they will be submitting EUA requests to the FDA for tests that detect the virus.
    • To date, the FDA has issued 54 individual EUAs for test kit manufacturers and laboratories.

      In addition, 23 authorized tests have been added to the EUA letter of authorization for high complexity molecular-based laboratory developed tests (LDTs).
    • The FDA has been notified that more than 235 laboratories have begun testing under the policies set forth in our COVID-19 Policy for Diagnostic Tests for Coronavirus Disease-2019 during the Public Health Emergency Guidance.
    • The FDA also continues to keep its COVID-19 Diagnostics FAQ up to date.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.”

https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-daily-roundup-may-1-2020

Gilead’s Investigational Antiviral Remdesivir Receives U.S. Food and Drug Administration Emergency Use Authorization for the Treatment of COVID-19

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Gilead’s Investigational Antiviral Remdesivir Receives U.S. Food and Drug Administration Emergency Use Authorization for the Treatment of COVID-19

May 1, 2020: Gilead Sciences, Inc. announced that the U.S FDA has granted emergency use authorization (EUA) for the investigational antiviral remdesivir to treat COVID-19.

The EUA will facilitate broader use of remdesivir to treat hospitalized patients with severe COVID-19 disease, enabling access to remdesivir at additional hospitals across the country.

Allocation of the currently limited available supply of remdesivir will be made based on guiding principles that aim to maximize access for appropriate patients in urgent need of treatment, with direction from and in collaboration with the government.

Remdesivir is authorized for the treatment of hospitalized patients with severe COVID-19 disease.

The optimal duration of treatment is still being studied in ongoing clinical trials. Under the EUA, both 5-day and 10-day treatment durations are suggested, based on the severity of disease.

The authorization is temporary and does not take the place of the formal new drug application submission, review and approval process.

The EUA allows for the distribution and emergency use of remdesivir only for the treatment of COVID-19; remdesivir remains an investigational drug and has not been approved by FDA.

The U.S. government will coordinate the donation and distribution of remdesivir to hospitals in cities most heavily impacted by COVID-19.

Given the severity of illness of patients appropriate for remdesivir treatment and the limited availability of drug supply, hospitals with intensive care units and other hospitals that the government deems most in need will receive priority in the distribution of remdesivir.

Gilead is working with the U.S. government on the logistics of remdesivir distribution and will provide more information when the company begins shipping the drug under the EUA.

“This EUA opens the way for us to provide emergency use of remdesivir to more patients with severe symptoms of COVID-19,” said Daniel O’Day, Chairman and Chief Executive Officer of Gilead Sciences.

“We will continue to work with partners across the globe to increase our supply of remdesivir while advancing our ongoing clinical trials to supplement our understanding of the drug’s profile.

We are working to meet the needs of patients, their families and healthcare workers around the world with the greatest sense of urgency and responsibility.”

The EUA is based on available data from two global clinical trials – the National Institute for Allergy and Infectious Diseases’ placebo-controlled Phase 3 study in patients with moderate to severe symptoms of COVID-19, including those who were critically ill, and Gilead’s global Phase 3 study evaluating 5-day and 10-day dosing durations of remdesivir in patients with severe disease.

Multiple additional clinical trials are ongoing to generate more data on the safety and efficacy of remdesivir as a treatment for COVID-19.

Remdesivir must be administered intravenously.

The optimal dosing and duration of remdesivir for the treatment of COVID-19 is still unknown. Under this EUA, the 10-day dosing duration is suggested for patients requiring invasive mechanical ventilation and/or extracorporeal membrane oxygenation (ECMO), and the 5-day dosing duration is suggested for patients not requiring invasive mechanical ventilation and/or ECMO.

If a patient on the 5-day dosing duration does not demonstrate clinical improvement after five days, treatment may be extended for up to five additional days (10 days total).

As previously announced, Gilead has donated the entirety of its existing supply of finished and unfinished product to help address the urgent medical needs posed by this pandemic around the world.

Assuming a 10-day treatment course, Gilead’s donation of 1.5 million individual doses of remdesivir equates to more than 140,000 treatment courses that will be provided at no cost to treat patients following potential emergency authorizations and regulatory approvals, including this EUA.

Gilead will continue to support clinical trials, and expanded access and compassionate use programs for remdesivir. In addition, Gilead will evaluate global allocation of supply on an ongoing basis using multiple, independent data sources to track the incidence and severity of the outbreak.

An EUA is intended to provide availability of a medicine during an emergency; an EUA is not the equivalent of an FDA approval. See below for important information about remdesivir.

Scale-Up of Remdesivir Supply

Gilead has aggressively implemented a multipronged approach to scale up production and rapidly build supply of the investigational antiviral remdesivir.

The company has invested significant capital, at risk, to meet the supply needs for clinical trials and emergency treatment programs, and to prepare for even greater demand following regulatory authorizations, should these occur.

Through process refinements, Gilead has substantially shortened the manufacturing lead time from raw materials through to finished product.

Gilead has also supplemented internal manufacturing with significant additional capacity from multiple partners in North America, Europe and Asia.

The company has set a goal of producing at least 500,000 treatment courses by October, 1 million treatment courses by December 2020 and millions more in 2021, if required. These goals were based on a 10-day treatment course.

Gilead now anticipates being able to cover significantly more patients based on the SIMPLE study results, which demonstrated similar efficacy with 5-day and 10-day dosing durations in patients with severe disease.

Looking ahead, Gilead is building a geographically diverse consortium of leading pharmaceutical and chemical manufacturing companies to help reach and exceed manufacturing goals, and go above and beyond what any company could do individually. More details about these efforts are available here.

Important Information about Remdesivir

Remdesivir (GS-5734™) is authorized for use under a EUA only for the treatment of patients with suspected or laboratory-confirmed SARS-CoV-2 infection and severe COVID-19 disease. SARS-CoV-2 is the coronavirus that causes COVID-19 disease.

Severe disease is defined as patients with oxygen saturation (SpO2) ≤ 94% on room air or requiring supplemental oxygen or requiring mechanical ventilation or requiring extracorporeal membrane oxygenation (ECMO).

Remdesivir is authorized for adult or pediatric patients who are admitted to a hospital and for whom use of an IV agent is clinically appropriate, as remdesivir must be administered intravenously.

Remdesivir is an investigational drug that has not been approved by the FDA for any use. It is not yet known if remdesivir is safe and effective for the treatment of COVID-19.

There are limited clinical data available for remdesivir. Serious and unexpected adverse events may occur that have not been previously reported with remdesivir use.

Warnings: In clinical studies with remdesivir, infusion-related reactions and liver transaminase elevations have been observed.

Remdesivir should not be used in patients who are hypersensitive to any ingredient of remdesivir.

If signs and symptoms of a clinically significant infusion reaction occur, immediately discontinue administration of remdesivir and initiate appropriate treatment.

Patients should have appropriate clinical and laboratory monitoring to aid in early detection of any potential adverse events.

Monitor renal and hepatic function prior to initiating and daily during therapy with remdesivir; additionally, monitor serum chemistries and hematology daily during therapy.

The decision to continue or discontinue remdesivir therapy after development of an adverse event should be made based on the clinical risk benefit assessment for the individual patient.

Healthcare providers and/or their designee are responsible for mandatory FDA MedWatch reporting of all medication errors and serious adverse events or deaths occurring during remdesivir treatment and considered to be potentially attributable to remdesivir.

These events must be reported within 7 calendar days from the onset of the event. MedWatch adverse event reports can be submitted to FDA online at www.fda.gov/medwatch or by calling 1-800-FDA-1088.

This is not all of the important information for remdesivir. The FDA has authorized distribution of this medicine with accompanying Fact Sheets, which can be accessed at www.gilead.com/remdesivir.

Healthcare providers should review the Fact Sheet for Healthcare Providers for more information on the authorized use of remdesivir and mandatory requirements of the EUA.

The distribution of remdesivir has been authorized only for the treatment of COVID-19. It is not authorized for the treatment of any other viruses or pathogens.

Information from the FDA about the FDA-authorized emergency use of remdesivir is available at https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization.

https://www.gilead.com/news-and-press/press-room/press-releases/2020/5/gileads-investigational-antiviral-remdesivir-receives-us-food-and-drug-administration-emergency-use-authorization-for-the-treatment-of-covid19

Chimerix Announces Initiation of a Phase 2/3 Study of DSTAT in Acute Lung Injury for Patients with Severe COVID-19

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Chimerix Announces Initiation of a Phase 2/3 Study of DSTAT in Acute Lung Injury for Patients with Severe COVID-19

April 29, 2020: “Chimerix, a biopharmaceutical company focused on accelerating the development of medicines to treat cancer and other serious diseases announced the Company’s initiation of a Phase 2/3 study of dociparstat sodium (DSTAT) in COVID-19 patients with acute lung injury (ALI). 

DSTAT is a glycosaminoglycan derivative of heparin with robust anti-inflammatory properties, including the potential to address underlying causes of coagulation disorders with substantially reduced risk of bleeding complications compared to commercially available forms of heparin.

“Given the severity of the COVID-19 pandemic, we have evaluated many potential targets to address the clinical manifestations associated with severe COVID-19,” said Joseph Lasky, M.D., Professor of Medicine, Pulmonary and Critical Care Section Chief, John W. Deming, M.D. Endowed Chair in Internal Medicine at Tulane University Medical School.

“Based on the literature, we believe DSTAT has the potential to reduce the excessive inflammation, immune cell infiltration and hypercoagulation associated with poor outcomes in patients with severe COVID-19 infection.”

“DSTAT is well-suited to unlock the anti-inflammatory properties of heparin as it may be dosed at much higher levels than any available form of heparin without triggering bleeding complications,” said Mike Sherman, Chief Executive Officer of Chimerix.

“We had planned to evaluate DSTAT in several indications of high unmet need, including ALI from different causes.

The pandemic intensified our focus on ALI associated with COVID-19.

Our team has worked closely with critical care physicians treating COVID-19 patients and with the U.S. Food and Drug Administration (FDA) to develop a Phase 2/3 protocol to determine if DSTAT can reduce the need for mechanical ventilation and improve the rate of survival in patients with severe COVID-19 infection.”

Phase 2/3 Study Design
The study is a 1:1 randomized, double-blind, placebo-controlled, Phase 2/3 trial to determine the safety and efficacy of DSTAT in adults with severe COVID-19 who are at high risk of respiratory failure.

 Eligible subjects will be those with confirmed COVID-19 who require hospitalization and supplemental oxygen therapy.

The primary endpoint of the study is the proportion of subjects who survive and do not require mechanical ventilation through day 28.

Additional endpoints include time to improvement as assessed by the National Institute of Allergy and Infectious Disease ordinal scale, time to hospital discharge, time to resolution of fever, number of ventilator-free days, all-cause mortality, and changes in key biomarkers (e.g. interleukin-6 (IL-6), tumor necrosis factor-α (TNF-α), high mobility group box 1 (HMGB1), C-reactive protein and d-dimer). 

The Phase 2 portion of the study will enroll 24 subjects to confirm the maximum safe dose and will then expand by an additional 50 patients (74 total) at the selected dose.

A formal analysis of all endpoints, including supportive biomarkers will be performed at the conclusion of the phase 2 portion of the study. Contingent upon positive results, the Phase 3 portion of the study will enroll approximately 450 subjects.

Clinical Rationale for DSTAT in COVID-19 Patients with ALI

The clinical manifestations of COVID-19 range from mild, self-limited respiratory tract illness to severe alveolar damage and progressive respiratory failure, multiple organ failure, and death.

Mortality in COVID-19 is associated with severe pulmonary disease and coagulation disorders such as disseminated intravascular coagulation (DIC).  

The mechanistic rationale supporting DSTAT’s potential in ALI patients with COVID-19 is two-fold:

  • Potential to decrease inflammation/immune cell infiltration in COVID-19 patients with ALI:
    • A primary anti-inflammatory effect of DSTAT is mediated by inhibition of HMGB1 activity.
      HMGB1 induces downstream proinflammatory cytokines, including but not limited to, IL-6, TNF-α, monocyte chemoattractant protein-1 (MCP-1) and macrophage inflammatory protein-1α (MIP-1α), all of which are elevated in COVID-19.
       
    • Infiltration of monocytes and other immune cells into inflamed lung tissue is a key pathogenic driver of ALI.
       DSTAT reduces lung infiltration by immune cells in ALI, likely by inhibition of MCP-1 and other ligands involved in migration of monocytes, neutrophils and other effector cells that promote hyperinflammation in the lungs.
       
  • Potential to alleviate the underlying causes of coagulation disorders by inhibiting HMGB1 and platelet factor 4 (PF4) activities:
    • Two recent studies have identified a high neutrophil/lymphocyte ratio and low platelet counts as clinically relevant indicators of disease severity and mortality in COVID-19.

      Neutrophils are early responders to infection capable of extruding granular and nuclear contents to produce neutrophil extracellular traps (NETs). NETs may be beneficial (e.g., by trapping pathogens); however, excessive neutrophils and NET release can be pathogenic.

       HMGB1 promotes NETs which may drive hypercoagulation by providing a substrate for platelet aggregation and upregulating tissue factor on endothelial cells.

       Activated platelets in turn release PF4, which further exacerbates inflammation.
      DSTAT’s inhibition of inflammatory drivers of coagulation (e.g., PF4 and HMGB1) has the potential to prevent and treat  coagulation disorders observed in COVID-19.

    • In a recent Phase 2 Acute Myeloid Leukemia (AML) study DSTAT was well tolerated with adverse events similar across DSTAT and control groups.  DSTAT is an investigational agent, not yet licensed or approved for use. 

Conference Call and Webcast

Chimerix will host a conference call and live audio webcast today at 8:30 a.m. ET. To access the live conference call, please dial 877-354-4056 (domestic) or 678-809-1043 (international) at least five minutes prior to the start time and refer to conference ID 8263766.

A live audio webcast of the call will also be available on the Investors section of Chimerix’s website, www.chimerix.com. An archived webcast will be available on the Chimerix website approximately two hours after the event.“

https://ir.chimerix.com/news-releases/news-release-details/chimerix-announces-initiation-phase-23-study-dstat-acute-lung

Novartis receives CHMP positive opinion for Enerzair® Breezhaler® (QVM149), a potential first-in-class inhaled LABA/LAMA/ICS combination for uncontrolled asthma

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Novartis receives CHMP positive opinion for Enerzair® Breezhaler® (QVM149), a potential first-in-class inhaled LABA/LAMA/ICS combination for uncontrolled asthma

May 01, 2020: “Novartis announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of Enerzair® Breezhaler® (QVM149; indacaterol acetate, glycopyrronium bromide and mometasone furoate [IND/GLY/MF]) as a maintenance treatment of asthma in adult patients not adequately controlled with a maintenance combination of a long‑acting beta2‑agonist (LABA) and a high dose of an inhaled corticosteroid (ICS) who experienced one or more asthma exacerbations in the previous year.

If approved, this will be the first once-daily LABA/long-acting muscarinic antagonist (LAMA)/ICS fixed-dose combination for these patients.

The European Commission (EC) reviews the CHMP recommendation and usually delivers its final decision in approximately two months.

“This is an important development for patients with uncontrolled asthma because IND/GLY/MF has been shown to improve lung function and symptoms versus a LABA/ICS standard-of-care,” said Professor Huib Kerstjens, Head, Department of Pulmonology at the University Medical Center Groningen.

“In secondary analyses of the Phase III IRIDIUM study statistically significant reductions in moderate-to-severe and severe asthma exacerbation rates were observed with high-dose IND/GLY/MF compared to high-dose salmeterol xinafoate/fluticasone propionate.”

IND/GLY/MF will be administered via the dose-confirming Breezhaler® device, which enables once-daily inhalation using a single inhaler.

If approved, IND/GLY/MF will be the first asthma treatment in the EU that can be prescribed together with a digital companion; a Propeller Health sensor and app custom-built for the Breezhaler® device.

This companion will provide patients with inhalation confirmation, medication reminders and access to objective data that can be shared with their physician in order to help them make better therapeutic decisions.

“Today’s news is a key milestone in our journey to reimagine asthma care by bringing innovative medicines and a digital companion to patients with uncontrolled asthma,” said Linda Armstrong, MD, Respiratory Development Unit Head, Novartis Pharmaceuticals. “Once-daily IND/GLY/MF has the potential to improve asthma control for patients whose lives are still impacted by their disease, despite existing inhaled therapies. Additionally, we are pleased to bring an innovative sensor and app companion supplied with IND/GLY/MF to patients to help support enhanced adherence.”

The CHMP recommendation is based on robust efficacy and safety data from over 3,000 patients with asthma from the Phase III IRIDIUM study, in which IND/GLY/MF demonstrated statistically significant improvements in lung function compared with indacaterol acetate/mometasone furoate (IND/MF).

In the IRIDIUM study, the key secondary endpoint was the improvement in Asthma Control Questionnaire score (ACQ-7) for IND/GLY/MF versus IND/MF. Both treatments delivered clinically meaningful improvements in this measure of symptoms from baseline at Week 26, but the key secondary endpoint was not met.

Among other secondary analyses, IRIDIUM explored asthma exacerbation rates, where statistically significant reductions were observed in moderate-to-severe and severe asthma exacerbation rates with IND/GLY/MF compared with an established LABA/ICS standard-of-care (twice-daily salmeterol xinafoate/fluticasone propionate). Safety findings were consistent with the known safety profiles of the monocomponents.

Last month the CHMP also recommended the approval of Atectura® Breezhaler® (IND/MF) as a maintenance treatment of asthma for adults and adolescents 12 years of age and older not adequately controlled with inhaled corticosteroids and inhaled short-acting beta-agonists.

Novartis is committed to bringing IND/GLY/MF and IND/MF to patients, with additional regulatory filings currently underway in multiple countries, including Switzerland, Japan and Canada.

In keeping with the Novartis commitment to reduce the environmental impact of our asthma combinations, IND/GLY/MF and IND/MF will both be available in the Breezhaler® device which is hydrofluoro alkane/chlorofluorocarbon (HFA/CFC)-free.

Asthma affects an estimated 358 million people worldwide and can cause a significant personal, health and financial burden when not adequately controlled.

Despite current therapy, over 40% of patients with asthma at Global Initiative for Asthma (GINA) Step 3, and over 45% at GINA Steps 4 and 5 remain uncontrolled.

 Patients with uncontrolled asthma may downplay or underestimate the severity of their disease and are at a higher risk of exacerbation, hospitalization or death.

Barriers, such as treatment mismatch, safety issues with an oral corticosteroid and ineligibility for biologics, have created an unmet medical need in asthma.

Enerzair® Breezhaler® (QVM149; IND/GLY/MF) is intended as a maintenance treatment of asthma in adult patients not adequately controlled with a maintenance combination of a long‑acting beta2‑agonist and a high dose of an inhaled corticosteroid who experienced one or more asthma exacerbations in the previous year.

This formulation combines the bronchodilation of indacaterol acetate (a LABA) and the antimuscarinic effects of glycopyrronium bromide (a LAMA) with mometasone furoate (high-dose ICS) in a precise once-daily formulation, delivered via the dose-confirming Breezhaler® device.

Glycopyrronium bromide certain use and formulation intellectual property were exclusively licensed to Novartis in April 2005 by Sosei Heptares and Vectura. Mometasone furoate is exclusively licensed to Novartis from a subsidiary of Merck & Co., Inc, Kenilworth, NJ, USA, for use in IND/GLY/MF (worldwide excluding the U.S.).

The sensor for the Breezhaler® device was developed by Propeller Health and is a CE marked Medical Device, designed and licensed to Novartis exclusively for use with the Breezhaler® inhaler worldwide.

The sensor includes a microchip, a microphone, Bluetooth capabilities, an antenna and a battery.

The sensor does not alter the drug delivery characteristics of the Breezhaler® inhaler itself but produces a recording of each administered dose. Based on the patient’s recorded medication usage, personalized content is presented within the app to help the patient better self-manage their asthma.

Atectura® Breezhaler® (QMF149; IND/MF) is the combination of indacaterol acetate and mometasone furoate intended as a maintenance treatment of asthma in adults and adolescents 12 years of age and older not adequately controlled with ICS and inhaled short-acting beta-agonists.

IND/MF combines the bronchodilation of indacaterol acetate (a LABA) with the anti-inflammatory mometasone furoate (an ICS) in a precise once-daily formulation, delivered via the dose-confirming Breezhaler® device. Mometasone furoate is exclusively licensed to Novartis from a subsidiary of Merck & Co., Inc, Kenilworth, NJ, USA, for use in IND/MF.

The PLATINUM program, containing over 7,500 patients worldwide, is the Novartis Phase III clinical development program supporting the development of IND/GLY/MF and IND/MF.

It includes four studies: the QUARTZ study, which compared a low-dose of IND/MF with MF alone; the PALLADIUM study, which compared IND/MF with MF and salmeterol xinafoate/fluticasone propionate (Sal/Flu); the IRIDIUM study, which compared IND/GLY/MF with IND/MF and Sal/Flu; and the ARGON study, which compared IND/GLY/MF with a combination of Sal/Flu and tiotropium.

IRIDIUM was a Phase III, multicenter, randomized, double-blind, parallel-group study, designed to compare the efficacy and safety of IND/GLY/MF with IND/MF in patients with asthma.

The purpose of the trial was to evaluate the efficacy and safety of two different doses of IND/GLY/MF (High: 150/50/160 μg and Medium:150/50/80 μg), versus two respective IND/MF doses (High: 150/320 μg and Medium: 150/160 μg) in patients with uncontrolled asthma, as determined by pulmonary function testing and effects on asthma control.

All patients were required to be symptomatic at screening despite being on treatment with medium or high stable doses of LABA/ICS. Approximately 3,092 male and female adult patients with asthma were randomized 1:1:1:1:1 (approximately 618 patients in each of the treatment groups) to receive either:

  • IND/GLY/MF 150/50/80 μg (once-daily)
  • IND/GLY/MF 150/50/160 μg (once-daily)
  • IND/MF 150/160 μg (once-daily)
  • IND/MF 150/320 μg (once-daily)
  • Sal/Flu 50/500 μg (twice-daily, via Accuhaler®)

The primary objective of this study was to demonstrate the superiority of both high-dose IND/GLY/MF versus high-dose IND/MF and medium-dose IND/GLY/MF versus medium-dose IND/MF, all delivered once-daily, in improving trough FEV1 (volume of air that can be forced out in the first second of expiration approximately 24 hours post-administration of study drug) after 26 weeks of treatment in patients with asthma.

The key secondary objective was to demonstrate the superiority of both doses of IND/GLY/MF  versus respective doses of IND/MF, in improving Asthma Control Questionnaire (ACQ-7) score after 26 weeks of treatment in patients with asthma.

Other secondary analyses also included reduction of exacerbation rate, comparing high-dose IND/GLY/MF with high-dose IND/MF and medium-dose IND/GLY/MF with medium-dose IND/MF. Secondary analyses included efficacy comparisons for both doses of IND/GLY/MF compared with Sal/Flu (50/500 μg).

The study showed that IND/GLY/MF was superior to IND/MF in improving trough FEV1 after 26 weeks, meeting the primary endpoint. The key secondary endpoint, improvement in ACQ-7 score for IND/GLY/MF versus IND/MF was not met but tested treatments showed clinically meaningful improvements in this measure of symptoms from baseline at Week 26. Among other secondary analyses, a substantial reduction was observed in moderate-to-severe and severe asthma exacerbation rates with IND/GLY/MF compared with twice-daily Sal/Flu (50/500 µg).

IND/GLY/MF was generally well tolerated, and safety was comparable across treatment arms.

PALLADIUM (NCT02554786) was a multicenter, randomized, 52-week treatment, double-blind, triple-dummy, parallel-group study, designed to assess the efficacy and safety of IND/MF compared with MF alone in patients with asthma.
                                                          
PALLADIUM included 2,216 male and female patients (including 107 adolescents, aged ≥12 to <18 years old) with medium or high-dose ICS or low-dose ICS/LABA use 3 months prior to screening, a pre-bronchodilator FEV1 of ≥50% and less than 80% of the predicted normal value for the patient and an Asthma Control Questionnaire (ACQ-7) score of greater than 1.5.

Patients were randomized 1:1:1:1:1 to receive either high-dose IND/MF (150/320 μg) once-daily delivered via Breezhaler® (n=445); medium-dose IND/MF (150/160 μg) once-daily delivered via Breezhaler® (n=439); MF 400 μg once-daily delivered via Twisthaler® (n=444); MF 800 μg administered as 400 μg twice-daily delivered via Twisthaler® (n=442); or salmeterol xinafoate/fluticasone propionate (Sal/Flu) 50/500 μg twice-daily delivered via Accuhaler® (n=446).

The study showed that medium and high doses of IND/MF demonstrated significant improvements compared with MF in trough FEV1 at Week 26 (Medium: 0.211 L; p<0.001) (High: 0.132 L; p<0.001).

The key secondary endpoint of improvement in ACQ-7 at Week 26 was also met for combined doses of IND/MF compared with combined doses of MF (-0.209; p<0.001). These positive results were also observed at Week 52.

Analyses of other lung function endpoints showed greater improvements for IND/MF compared with MF in both morning and evening Peak Expiratory Flow (PEF).

Reductions in daily rescue medication use and exacerbation rates were also observed. Patients demonstrated a 12% increase in FEV1 and 200 mL within 30 minutes following administration of 400 µg salbutamol/360 µg albuterol (or equivalent dose) at the first visit or from historical data.

In the secondary analyses of comparison to Sal/Flu, high-dose IND/MF showed improvements in trough FEV1 (0.048 L; p=0.040) at 52 weeks.

In asthma control, high-dose IND/MF and Sal/Flu were comparable with a difference in ACQ-7 score of 0.010 (p=0.824). Improvements were observed in both morning and evening PEF (Morning: 13.8 L/min; p<0.001) (Evening: 9.1 L/min; p=0.002), and percentage of rescue medication-free days over 52 weeks (4.3; p=0.034) in patients treated with high-dose IND/MF versus Sal/Flu. High-dose IND/MF also showed a faster onset of action over Sal/Flu as demonstrated by FEV1 measurement at 5 minutes on Day 1 (0.055 L; p<0.001).

IND/MF was generally well-tolerated, and safety was comparable across treatment arms.

The QUARTZ Study (NCT02892344) was a Phase III, multicenter, randomized, 12-week treatment, double-blind study, designed to assess the efficacy and safety of IND/MF (150/80μg) compared with MF (200 μg) delivered via the Twisthaler® device in adult and adolescent patients with asthma.

All patients were required to be on a stable low dose of ICS (with or without LABA) for at least one month prior to entering the run-in period.

During the run-in period, all patients received open-label fluticasone propionate 100 μg twice-daily delivered via Accuhaler®.

Patients meeting the eligibility criteria at the end of the run-in period were equally randomized (1:1) to one of the two treatment groups IND/MF 150/80 μg once-daily delivered via the dose-confirming Breezhaler® device, or MF 200 μg once-daily, delivered via Twisthaler®.

802 male and female patients (including 64 adolescents, aged ≥ 12 to < 18 years old) were randomized to receive IND/MF (n=398) or MF (n=404). The mean age was 45.6 years with a mean asthma duration of 14.0 years.

Mean pre-bronchodilator FEV1 (% predicted of normal) was 75.1% at baseline and the mean FEV1 reversibility after inhalation of short acting beta agonist was 20.7%. The majority of patients were treated with low-dose ICS (42.9%) or low-dose LABA/ICS (56.0%) prior to the study.

Spirometry was performed at the end of the treatment period. The primary objective was to demonstrate the superiority of IND/MF 150/80 μg once-daily (in the evening) compared with MF 200 μg once-daily in terms of trough FEV1 after 12 weeks of treatment in adults and adolescents.

The key secondary objective was to demonstrate the superiority of IND/MF 150/80 μg to MF 200 μg (once-daily) in terms of the Asthma Control Questionnaire (ACQ-7) after 12 weeks of treatment.

IND/MF demonstrated statistically significant improvements in trough FEV1 compared with MF after 12 weeks of treatment in adult and adolescent patients with inadequately controlled asthma (least squares [LS] mean treatment difference: 0.182 L, 95% CI: 0.148, 0.217; p<0.001).

In addition, clinically meaningful lung function benefit for IND/MF was supported by improvements in evening PEF of 26.1 L/min compared with MF alone (95% CI, 21.0, 31.2).

IND/MF also demonstrated statistically significant improvements in asthma control compared with MF, as measured by ACQ-7 after 12 weeks of treatment (LS mean treatment difference: -0.218, 95% CI: -0.293, -0.143; p<0.001).

In addition, a clinically meaningful improvement in ACQ-7 was supported by a responder analysis, in which the IND/MF group had a greater proportion of responders (improvement in ACQ-7 >=0.5) compared with the MF group (74.7% vs 64.9%, respectively (odd ratio: 1.69, 95% CI: 1.23, 2.33).

IND//MF was generally well tolerated, and safety was comparable across treatment arms.

ARGON (NCT03158311) is a Phase IIIb, multicenter, randomized, 24-week, parallel-group, non-inferiority, open-label (blinded for the two IND/GLY/MF tested strengths), active-controlled study comparing the efficacy and safety of IND/GLY/MF with a free combination of salmeterol xinafoate/fluticasone propionate (Sal/Flu) plus tiotropium in patients with uncontrolled asthma.

The ARGON study has completed in the field and its Clinical Study Report is currently being finalized.

The purpose of this trial was to demonstrate that the efficacy of two strengths of the fixed-dose combination product IND/GLY/MF (High: 150/50/160 μg and Medium: 150/50/80 μg) is non-inferior to the efficacy of the free combination of Sal/Flu (50/500 μg) plus tiotropium (5 μg) in patients with uncontrolled asthma.

All patients were symptomatic at screening despite treatment with medium or high stable doses of LABA/ICS as defined by the Asthma Control Questionnaire (ACQ-7) score ≥ 1.5.

Approximately 1,251 male and female patients with uncontrolled asthma (aged 18 and above) were randomized 1:1:1 (approximately 417 patients in each of the treatment groups) to receive either:

  • IND/GLY/MF 150/50/80 μg (once-daily)
  • IND/GLY/MF 150/50/160 μg (once-daily)
  • Open label Sal/Flu 50/500 μg (twice-daily) delivered via Diskus® plus tiotropium delivered via Respimat®

The primary objective of this study was to demonstrate non-inferiority of both high-dose IND/GLY/MF and low-dose IND/GLY/MF to comparator Sal/Flu plus tiotropium after 24 weeks of treatment based on Asthma Quality of Life Questionnaire.

Secondary objectives included:

  • To evaluate the efficacy of high-dose IND/GLY/MF and medium-dose IND/GLY/MF compared with Sal/Flu plus tiotropium based on trough FEV1 after 24 weeks of treatment
  • To evaluate the efficacy of high-dose IND/GLY/MF and medium-dose IND/GLY/MF compared with Sal/Flu plus tiotropium based on Asthma Quality of Life Questionnaire over 24 weeks of treatment.
  • To evaluate the efficacy of high-dose IND/GLY/MF and medium-dose IND/GLY/MF compared with Sal/Flu plus tiotropium based on ACQ-7 over 24 weeks of treatment.
  • To evaluate the efficacy of high-dose IND/GLY/MF and medium-dose IND/GLY/MF compared with Sal/Flu plus tiotropium based on lung function over 24 weeks of treatment.”

https://www.novartis.com/news/media-releases/novartis-receives-chmp-positive-opinion-enerzair-breezhaler-qvm149-potential-first-class-inhaled-labalamaics-combination-uncontrolled-asthma

Novartis Cosentyx® gains fourth indication in EU with first-in-class approval in axial spondyloarthritis spectrum

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Novartis Cosentyx® gains fourth indication in EU with first-in-class approval in axial spondyloarthritis spectrum

April 29, 2020: “Novartis, a leader in rheumatology and immuno-dermatology, announced the European Commission (EC) has approved Cosentyx® (secukinumab) for the treatment of adult patients with active non-radiographic axial spondyloarthritis (nr-axSpA).

“This approval of Cosentyx for non-radiographic axial spondyloarthritis means clinicians across Europe now have an effective new treatment option to help patients gain relief from the burden of this painful, debilitating disease and achieve a better quality of life both at home and at work,” said Atul Deodhar, MD, Professor of Medicine and Medical Director of Rheumatology Clinics at Oregon Health & Science University, USA, and an investigator in the PREVENT clinical trial.

The approval is based on data from the Phase III PREVENT study, in which Cosentyx met the primary endpoint. In the study, 41.5% of nr-axSpA patients treated with Cosentyx 150 mg showing a significant and clinically meaningful reduction in disease activity versus placebo (41.5% vs 29.2%: p<0.05), as measured by at least a 40% improvement in ASAS40 at week 16, with improvements continued through week 52.

Statistically significant improvements in secondary endpoints were also demonstrated, including pain, disease burden and health-related quality of life. PREVENT is the largest ever study of a biologic in patients with nr-axSpA.

“Whether a patient has nr-axSpA or AS, the condition has a significant impact on their everyday life.

We, therefore, welcome the news that Cosentyx has gained approval for the treatment of this form of axial spondyloarthritis because it enables patients to realize relief from their symptoms earlier in the spectrum of disease,” said Eric Hughes, Global Development Unit Head, Immunology, Hepatology & Dermatology at Novartis.

“This is a firm demonstration of our commitment to reimagine medicine for patients and a step forward in our plans to expand Cosentyx across ten indications over the next ten years.”

Novartis is working closely with all stakeholders to ensure that eligible European patients can start benefitting from Cosentyx as quickly as possible.

Novartis has also submitted Cosentyx for review by the US Food and Drug Administration (FDA) and the Japan Pharmaceuticals and Medical Devices Agency (PMDA) for the treatment of adults with nr-axSpA.

AxSpA is a spectrum of the long-term inflammatory disease characterized by chronic inflammatory back pain.

The axSpA spectrum includes AS, in which joint damage is generally visible on x-ray, and nr-axSpA, in which joint damage is not visible on x-ray.

Both parts of the disease spectrum have a comparable symptom burden, including nocturnal waking caused by pain, spinal pain, morning stiffness, fatigue and functional disability.

If left untreated, axSpA impairs activity, leads to lost work time and has a significant impact on quality of life, including family relationships.

Cosentyx is the first and only fully-human biologic that directly inhibits IL-17A, a cornerstone cytokine involved in the inflammation and development of PsO, PsA and AS

Cosentyx is backed by robust clinical evidence, including five-year data across three indications of PsO, PsA and AS, as well as data from real world evidence.

These data strengthen the unique position of Cosentyx as a rapid and long-lasting comprehensive treatment across axSpA, PsA and psoriatic disease, with more than 300,000 patients treated worldwide with Cosentyx since launch.

PREVENT is an ongoing two-year randomized, double-blind, placebo-controlled Phase III study (with a two-year extension phase) to investigate the efficacy and safety of Cosentyx, in patients with active nr-axSpA.

The study enrolled 555 male and female adult patients with active nr-axSpA (with onset before 45 years of age, spinal pain rated as >=40/100 on a visual analog scale (VAS) and Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) >=4) and who had been taking at least two different non-steroidal anti-inflammatory drugs (NSAIDs) at the highest dose up to 4 weeks prior to study start.

Patients may have previously taken a TNF inhibitor (not more than one) but had an inadequate response.

Of the 555 patients enrolled in the study, 501 (90.3%) were biologic naïve. Patients were allocated to one of three treatment groups:

Cosentyx 150 mg subcutaneously with loading dose (induction: 150 mg secukinumab subcutaneously weekly for 4 weeks, then maintenance with 150 mg secukinumab monthly); Cosentyx 150 mg no loading dose (150 mg secukinumab subcutaneously monthly), or placebo (induction of subcutaneously weekly for 4 weeks, followed by maintenance of once-monthly)

The primary endpoints are the proportion of patients achieving an ASAS40 response with Cosentyx 150 mg at Weeks 16 and 52 in TNF-naive patients.

Secondary endpoints include among others change in BASDAI over time and change in the Ankylosing Spondylitis Disease Activity Score with CRP (ASDAS-CRP).”

https://www.novartis.com/news/media-releases/novartis-cosentyx-gains-fourth-indication-eu-first-class-approval-axial-spondyloarthritis-spectrum

AstraZeneca and Oxford University announce landmark agreement for COVID-19 vaccine

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AstraZeneca and Oxford University announce landmark agreement for COVID-19 vaccine

April 30, 2020: “AstraZeneca and the University of Oxford announced an agreement for the global development and distribution of the University’s potential recombinant adenovirus vaccine aimed at preventing COVID-19 infection from SARS-CoV-2.

The collaboration aims to bring to patients the potential vaccine known as ChAdOx1 nCoV-19, being developed by the Jenner Institute and Oxford Vaccine Group, at the University of Oxford.

Under the agreement, AstraZeneca would be responsible for development and worldwide manufacturing and distribution of the vaccine.

Pascal Soriot, Chief Executive Officer, AstraZeneca, said: “As COVID-19 continues its grip on the world, the need for a vaccine to defeat the virus is urgent.

This collaboration brings together the University of Oxford’s world-class expertise in vaccinology and AstraZeneca’s global development, manufacturing and distribution capabilities.

Our hope is that, by joining forces, we can accelerate the globalisation of a vaccine to combat the virus and protect people from the deadliest pandemic in a generation.”

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “The University of Oxford and AstraZeneca have a longstanding relationship to advance basic research and we are hugely excited to be working with them on advancing a vaccine to prevent COVID-19 around the world. We are looking forward to working with the University of Oxford and innovative companies such as Vaccitech, as part of our new partnership.”  

Alok Sharma, UK Business Secretary, said: “This collaboration between Oxford University and AstraZeneca is a vital step that could help rapidly advance the manufacture of a coronavirus vaccine.

It will also ensure that, should the vaccine being developed by Oxford University’s Jenner Institute work, it will be available as early as possible, helping to protect thousands of lives from this disease.”

Professor Sir John Bell, Regius Professor of Medicine at Oxford University, said: “Our partnership with AstraZeneca will be a major force in the struggle against pandemics for many years to come.

We believe that together we will be in a strong position to start immunising against coronavirus once we have an effective approved vaccine.

Sadly, the risk of new pandemics will always be with us and the new research centre will enhance the world’s preparedness and our speed of reaction the next time we face such a challenge.”

Professor Louise Richardson, Vice-Chancellor of Oxford University, said: “Like my colleagues all across Oxford, I am deeply proud of the work of our extraordinarily talented team of academics in the Jenner Institute and the Oxford Vaccine Group.

They represent the best tradition of research, teaching and contributing to the world around us, that has been the driving mission of the University of Oxford for centuries. Like people all across the country, we are wishing them success in developing an effective vaccine.

If they are successful, our partnership with AstraZeneca will ensure that the British people and people across the world, especially in low and middle income countries, will be protected from this terrible virus as quickly as possible.”

The potential vaccine entered Phase I clinical trials last week to study safety and efficacy in healthy volunteers aged 18 to 55 years, across five trial centres in Southern England.

Data from the Phase I trial could be available next month. Advancement to late-stage trials should take place by the middle of this year.

ChAdOx1 nCoV-19

Developed at the University of Oxford’s Jenner Institute, and working with the Oxford Vaccine Group, ChAdOx1 nCoV-19 uses a viral vector based on a weakened version of the common cold (adenovirus) containing the genetic material of SARS-CoV-2 spike protein.

After vaccination, the surface spike protein is produced, which primes the immune system to attack COVID-19 if it later infects the body.

The recombinant adenovirus vector (ChAdOx1) was chosen to generate a strong immune response from a single dose and it is not replicating, so cannot cause an ongoing infection in the vaccinated individual.

Vaccines made from the ChAdOx1 virus have been given to more than 320 people to date and have been shown to be safe and well tolerated, although they can cause temporary side effects such as a temperature, flu-like symptoms, headache or sore arm.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2020/astrazeneca-and-oxford-university-announce-landmark-agreement-for-covid-19-vaccine.html

AI Nurse set to transform standards of care for heart failure patients in China

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AI Nurse set to transform standards of care for heart failure patients in China

April 30, 2020: “A fast-paced Novartis joint development project with one of the world’s largest technology companies has created an innovative, AI-enabled digital nurse to support heart failure patients in China.

Co-developed with China’s internet multinational Tencent, together with leading Chinese medical organizations, AI Nurse launched on April 30 and will serve as a patient needs-led intelligent platform supporting patients, physicians and nurses to better manage heart disease.

This program is the first of its kind in China to build up a digital chronic disease management model together with hospital and physician visits, which makes it possible for heart failure patients to do more self-monitoring and management.

The aim is to provide a caring, convenient assistant, accessible 24/7, to improve users’ capacity in self-initiating healthcare actions. Overall, the goal is to reduce re-hospitalization and improve other outcomes in a country where heart disease is on the increase.

This new AI-enabled solution engages with patients in a familar and caring digital setting – easily accessible within a social media platform used for everyday living by more than 1.1 billion of China’s 1.4 billion population.

Growing need

In China, morbidity and mortality are on the rise, as the population ages. Prescription adherence is poor once patients leave hospitals without community support, and many patients with heart failure are re-hospitalized several times within a year of their initial cardiovascular event.

As the leading cause of death in China, accounting for two out of every five deaths, chronic heart disease is a key issue in the country’s national ‘Healthy China 2030’ plan, which outlines health priorities, science and technology innovation as strategic platforms.

The Novartis partnership with Tencent is a bold move to maximize the potential of digital transformation in engaging with patients and healthcare professionals to better manage chronic diseases.

We are going big on data and digital to prepare for disruptive healthcare scenarios in the future, reimagining medicine with the power of data science and digital technology.

“To transform how medicine is practised, you have to do something really deep – and this is a bold move that will really make a difference,” says Dr David Fan, executive director of Tencent’s Medical AI Lab. “85% of the disease spectrum is chronic, and that’s a huge burden on patients, their loved ones, the healthcare system and society in general.

We are demonstrating that a digital platform can transform chronic disease management. Our long-term vision from this collaboration with Novartis is to change how medicine is practiced, to benefit as many patients and their loved ones as possible.”

Potential:

This new AI Nurse platform could benefit 16 million patients with heart disease in China. Sitting within Tencent’s WeChat social media platform, used by the Chinese for everyday lifestyle needs such as booking a taxi and ordering food, AI Nurse is designed to be user-friendly and easy to access.

The new heart failure management platform covers daily health indicator monitoring and health condition assessment through voice and image recognition and interaction, including a voice-enabled chatbot facility and a link back to hospital physicians. It also provides personalized education materials, schedules follow-up appointments and enables repeat prescription ordering.

Fast pace

Since the agreement with Tencent was finalized in October 2019, development of the platform has moved at speed.

A prototype of AI Nurse – a mini app for heart failure patients – was demonstrated in just a week from contract signing. China’s top cardiovascular specialists welcomed the innovation when it was presented at the China International Import Expo in November 2019.

A month later, the physician interface became available for testing; both versions incorporated detailed user feedback and insight.

After trials at three Chinese hospitals in January, 17 additional features were developed, plus a third interface, for nurses to support ongoing interactions with patients after hospital discharge in a country with no established primary care structure.

Disease-specific, not drug-specific

The new digital healthcare resource applies to any patient with heart failure in China, regardless of whether he or she is prescribed Novartis drugs or those from other pharmaceutical companies.

“We have lots of collaborations with many different sectors, but this is a flagship project for us because of how many patients are in scope.

Novartis has put a lot of emphasis on digital and they are leading in this field to develop a service that will benefit a whole patient population, not just those on its own products.” says Tencent AI Nurse Product Director Mike Tang.

According to Christian Hein, Novartis Executive Director Digital Products, AI Nurse could ultimately widen its reach and inspire future innovation.“

https://www.novartis.com/news/ai-nurse-set-transform-standards-care-heart-failure-patients-china

    Almac Discovery to Collaborate with MSD on selected DUB therapeutic targets

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    Almac Discovery to Collaborate with MSD on selected DUB therapeutic targets

    April 29, 2020: “Almac Discovery announced a research collaboration with MSD focusing on the generation of novel small molecule inhibitors against specified Deubiquitinase (DUB) targets for the treatment of a range of neurodegenerative and other diseases.

    Almac Discovery possesses a demonstrated track-record and a wealth of experience and expertise in the field of DUBs and utilises its unique, purpose-built, fully comprehensive assay and drug finding platform -Ubi-Plex® – to identify and develop novel inhibitors against a number of therapeutically relevant DUBs.

    Whilst this collaboration will focus on neurodegenerative diseases, the Ubi-Plex platform also offers the opportunity to generate novel, innovative candidate drugs targeting unmet need in a range of therapeutic areas including oncology, viral, inflammatory and metabolic disorders.

    Dr Stephen Barr, Managing Director & President, Almac Discovery commented: “We are delighted to have signed our latest research collaboration agreement with such an experienced and renowned leader in research.

    Both MSD and Almac are aligned in our missions to develop treatments for illnesses that represent areas of significant unmet medical need.”

    Almac Discovery and MSD will initially collaborate in a two-year joint research programme with the objective to identify and progress novel, potent and selective small molecule inhibitors.

    MSD will be responsible for conducting lead optimisation, preclinical and clinical development and commercialisation.

    “Establishing external collaborations is an integral part of our strategy to drive medical advances against some of the most challenging diseases facing ageing populations,” said Dr Fiona Marshall, Vice President, Head of Neuroscience Discovery and Head of Discovery Science at MSD UK.

    “We look forward to working with scientists at Almac to evaluate the potential of novel small molecule deubiquitinase inhibitors.”

    Prof. Tim Harrison, Vice President Drug Discovery, added “The Almac Discovery team are looking forward to working with MSD to prosecute this exciting target class, with a view to developing much needed new treatments for neurodegenerative diseases.”

    Under the terms of the agreement Almac Discovery will receive an upfront licence fee and research funding from MSD, the financial details of which are not disclosed.

    Almac Discovery is also eligible for payments based on the achievement of predetermined research, development and commercial milestones, as well as tiered royalties on potential commercial sales of products resulting from the collaboration.”

    https://www.almacgroup.com/news/almac-discovery-to-collaborate-with-msd-on-selected-dub-therapeutic-targets/

    NICE joins international collaboratives to respond to the COVID-19 pandemic

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    NICE joins international collaboratives to respond to the COVID-19 pandemic

    April 30, 2020: ‘The National Institute for Health and Care Excellence (NICE) says it is supporting a growing number of international collaborations striving to share knowledge and identify new treatments in response to the COVID-19 pandemic.

    Initiatives such as the Evidence Collaborative for COVID-19 established by the World Health Organisation, the International Network of Agencies for Health Technology Assessment, and the European Network for Health Technology Assessment are working to identify evidence on the prevention, diagnosis and treatment of COVID-19.

    NICE is sharing its work on COVID-19 with these partners, and is also helping select priority areas for research through the Cochrane collaboration’s rapid reviews on COVID-19.

    The collaborations will also help NICE ensure that its rapid guidelines – which are being made available for access without the normal licencing fee – are developed and updated to internationally recognised standards of best practice.

    “Having our guidelines all in one place, in an easily accessible format, means we are able to share our knowledge and recommendations with health care professionals around the world, and learn from each other’s findings,” said Paul Chrisp, director for the Centre for Guideline.

    “NICE will continue to support, contribute to and learn from international efforts to coordinate the development and maintenance of high-quality evidence-based guidelines on COVID-19.”

    NICE Scientific Advice is also providing free fast-track advice for researchers who are developing novel diagnostics or therapeutics for COVID-19, while NICE International is also continuing to provide an advisory service to international organisations, ministries and government agencies to support the use of evidence-based decision making in health and social care systems.”

    https://www.nice.org.uk/news/article/nice-joins-international-collaboratives-to-respond-to-the-covid-19-pandemic

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