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EU approve Koselugo for children with neurofibromatosis & plexiform neurofibromas

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EU approve Koselugo for children with neurofibromatosis & plexiform neurofibromas

June 22, 2021: “AstraZenecaand MSD’s Koselugo (selumetinib) has been granted conditional approval in the EU for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above.

NF1 is a debilitating genetic condition affecting one in 3,000 individuals worldwide.

In 30-50% of people with NF1, tumours develop on the nerve sheaths (plexiform neurofibromas) and can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.

The approval by the European Commission was based on positive results from the SPRINT Stratum 1 Phase II trial sponsored by the National Institute of Health’s National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP).

This trial showed Koselugo reduced the size of inoperable tumours in children, reducing pain and improving quality of life.

This is the first approval of a medicine for NF1 PN in the EU and follows the positive recommendation by the Committee for Medicinal Products for Human Use of the European Medicines Agency in April 2021.

Safety and efficacy data from the SPRINT Phase II trial with longer follow up will be provided as one of the conditions of approval.

Brigitte C. Widemann, MD, Principal Investigator of the SPRINT trial and Chief, NCI Pediatric Oncology Branch, said: “For children with neurofibromatosis type 1, plexiform neurofibromas can grow and develop so significantly that, in some cases, it becomes debilitating.

In the SPRINT trial, selumetinib shrank NF1-associated PNs in 66% of patients and showed clinically meaningful improvements in PN-related symptoms.”

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: “As the first medicine approved in the EU for patients with neurofibromatosis type 1, Koselugo has the potential to transform the way plexiform neurofibromas are managed and treated.

The SPRINT data showed that Koselugo not only shrank tumours in some children, but also reduced pain and improved their quality of life.

This significant milestone was made possible thanks to our research partners, the National Cancer Institute, the Neurofibromatosis Therapeutic Acceleration Program, the Children’s Tumor Foundation, the patient community and every child, parent and doctor involved in the clinical trial.”

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: “Before this approval, surgery was the only treatment option for children in the EU with neurofibromatosis type 1 plexiform neurofibromas.

This approval marks a significant step forward in addressing the debilitating impact of these tumours.”

The SPRINT Stratum 1 Phase II trial showed Koselugo demonstrated an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in paediatric patients with NF1 PN when treated with Koselugo as twice-daily oral monotherapy.

ORR is defined as the percentage of patients with confirmed complete (disappearance of PN) or partial response (at least 20% reduction in tumour volume).8 Results were published in The New England Journal of Medicine.

Koselugo is approved in the US and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable PN. Further regulatory submissions are underway.

Clinical trials of Koselugo in adult patients with NF1 PN, including an alternative age-appropriate formulation for paediatric patients, are scheduled to begin this year.

NF1
NF1 is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with many symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called ‘café au lait’ spots).

In 30-50% of people, tumours develop on the nerve sheaths.

These PN can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bladder/bowel dysfunction and disfigurement, as well as having the potential to transform into malignant peripheral nerve sheath tumours.

PN begin developing during early childhood, with varying degrees of severity, and can reduce life expectancy by eight to 15 years.

SPRINT
The SPRINT Stratum 1 Phase II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with selumetinib monotherapy.

This trial sponsored by NCI CTEP was conducted under a Cooperative Research and Development Agreement between NCI and AstraZeneca with additional support from Neurofibromatosis Therapeutic Acceleration Program (NTAP).

Koselugo
Koselugo (selumetinib) is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2).

MEK1/2 proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. Both MEK and ERK are critical components of the RAS-regulated RAF-MEK-ERK pathway, which is often activated in different types of cancers.

Koselugo received US FDA Breakthrough Therapy Designation in April 2019, Rare Pediatric Disease Designation in December 2019 and US Orphan Drug Designation in February 2018.

Further orphan designations have been granted in the EU, Japan, Russia, Switzerland, South Korea, Taiwan and Australia.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/koselugo-approved-in-the-eu-for-children-with-neurofibromatosis-type-1-and-plexiform-neurofibromas.html

FDA Approves First Oral Blood Thinning Medication for Children

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FDA Approves First Oral Blood Thinning Medication for Children

June 21, 2021: The U.S. FDA approved Pradaxa (dabigatran etexilate) oral pellets to treat children 3 months to less than 12 years old with venous thromboembolism (a condition where blood clots form in the veins) directly after they have been treated with a blood thinner given by injection for at least five days.

The FDA also approved Pradaxa oral pellets to prevent recurrent clots among patients 3 months to less than 12 years old who completed treatment for their first venous thromboembolism.

In addition, Pradaxa was approved in capsule form to treat blood clots in patients eight years and older with venous thromboembolism directly after they have been treated with a blood thinner given by injection for at least five days, and to prevent recurrent clots in patients eight years and older who completed treatment for their first venous thromboembolism.

Pradaxa is the first FDA-approved blood thinning medication that children can take by mouth; the only other approved blood thinning medication for children is given by injection. Pradaxa was originally approved in 2010 to reduce the risk of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation.

“The FDA is committed to helping our youngest patients with serious medical conditions have treatments that are relatively easy to take,” said Ann Farrell, M.D., director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research.

“With today’s approval of Pradaxa, pediatric patients have another therapeutic option to treat and prevent potentially deadly blood clots.”

Blood clots can be a serious problem in children as well as adults.

Children are most at risk for blood clots if they have cancer, congenital heart disease, a central venous catheter, or are admitted to an intensive care unit.

Venous thromboembolism can lead to complications, including swelling and discomfort near the clot, chest pain, lung damage, and even death.

The safety and efficacy of Pradaxa for treating blood clots in patients younger than 18 was evaluated in one study of 267 pediatric patients. In this open-label study, patients were randomly assigned to receive either Pradaxa or standard of care.

The study compared the two groups for the number of patients who met the composite endpoint (a combination of multiple clinical endpoints), which meant that they had not died from a blood clot, their blood clots had completely resolved, and they had no additional blood clots.

Results showed that 81 (45.8%) of the 177 people taking Pradaxa met the composite endpoint compared to 38 (42.2%) of the 90 patients who received standard of care.

The safety of Pradaxa to prevent recurrent blood clots in the same pediatric population was evaluated in an open-label, single-arm study in 214 patients with a history of blood clots.

The primary endpoints of the study were recurrence of blood clots, major and minor bleeding events, and death (both overall and related to blood clots). The safety of Pradaxa with long-term use was similar to the previously discussed study.

Recurrence of blood clots occurred in three patients (1.4%), which was comparable to prior standard-of-care treatments.

The most common side effects of Pradaxa include digestive system symptoms and bleeding. Pradaxa can cause serious and fatal bleeding. Pradaxa is not recommended for patients with bioprosthetic heart valves or triple-positive antiphospholipid syndrome.

Pradaxa has a boxed warning cautioning that early treatment discontinuation may increase the risk of blood clots and that blood accumulation within parts of the spinal cord (spinal or epidural hematomas) in patients undergoing spinal procedures may cause serious side effects.

Pradaxa received a priority review designation for this indication.

The FDA granted the approval of Pradaxa to Boehringer Ingelheim Pharmaceuticals, Inc.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-oral-blood-thinning-medication-children

Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine

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Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine

JUNE 22, 2021: “Sanofi Pasteur, the vaccines global business unit of Sanofi and Translate Bio, a clinical-stage messenger RNA (mRNA) therapeutics company, have initiated a Phase 1 clinical trial evaluating an mRNA-based investigational vaccine against seasonal influenza.

The trial will evaluate the safety and immunogenicity of a monovalent flu vaccine candidate coding for the hemagglutinin protein of the A/H3N2 strain of the influenza virus.

Flu seasons that are dominated by A/H3N2 strain circulation activity tend to be more severe, especially among those considered at-risk such as older adults and younger children.

“The first clinical trial of a seasonal mRNA flu vaccine candidate is an exciting milestone in our quest for the next generation of influenza vaccines.

We’ve all witnessed the promise of mRNA technology during this pandemic and are now looking to extend that promise to select annual vaccines”, said Jean-François Toussaint, Global Head of Research and Development, Sanofi Pasteur. 

“In line with our global leadership and our 70-year history of protecting people with influenza vaccines, we will always focus on developing products that demonstrate protection beyond flu, as we believe it is critical to demonstrate protecting patients from hospitalizations due to cardiovascular events and pneumonia. We look forward to sharing initial results by year-end.”

“We are pleased to have this second mRNA vaccine program underway with our partner Sanofi Pasteur,” said Ronald Renaud, chief executive officer, Translate Bio.

“We believe that mRNA technology could have several advantages for a seasonal flu application including the potential ability to demonstrate robust immune responses based on preclinical data to date, enable antigen specificity within a short timeframe from seasonal virus strain selection, and deploy agile manufacturing capacity.

We look forward to evaluating the potential of these mRNA influenza vaccine candidates in this Phase 1 clinical trial.”

Sanofi and Translate Bio have developed and will evaluate two formulations of the vaccine (MRT5400 and MRT5401) in the Phase 1 influenza mRNA vaccine clinical trial. The two formulations differ in the lipid nanoparticle (LNP) that contains the mRNA.

The trial follows successful preclinical research which demonstrated promising safety and immunogenicity. Preclinical results were shared previously at the 8th annual mRNA Healthcare Conference in November 2020.

About the Phase 1 trial
The US-based Phase 1 clinical trial will assess the safety and immunogenicity (immune response) of the monovalent (single-strain) mRNA-based flu vaccine candidate in up to 280 participants.

The trial will evaluate several dose levels of both vaccine formulations given to healthy adults 18 – 49 years of age.

About the Sanofi Pasteur and Translate Bio collaboration
In June 2018, Translate Bio entered into a collaboration and exclusive license agreement with Sanofi Pasteur, the vaccines global business unit of Sanofi, to develop mRNA vaccines for up to five infectious disease pathogens.

The agreement was first expanded in March 2020 to include development of a novel mRNA vaccine for COVID-19.

In June 2020, the two companies built upon the existing agreement to pursue novel mRNA vaccines to broadly address current and future infectious diseases.

There are two ongoing mRNA vaccine clinical trials under the collaboration, one for COVID-19 and one for influenza.

This collaboration brings together Sanofi Pasteur’s leadership in vaccines and Translate Bio’s mRNA research and development expertise.

Under the agreement, the companies are jointly conducting research and development activities to advance mRNA infectious disease vaccine candidates and mRNA vaccine platform development during a research term of at least four years after the original signing in 2018.”

https://www.sanofi.com/en/media-room/press-releases/2021/2021-06-22-07-00-00-2250633

Bayer and Zydus announce continuity of their successful Joint Venture

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Bayer and Zydus announce continuity of their successful Joint Venture

June 21, 2021: Bayer and Cadila Healthcare Limited announced the extension of their Joint Venture by three years from June 2021.

The companies first entered into an agreement on January 28, 2011 to set up the Joint Venture – Bayer Zydus Pharma Private Limited for the sales and marketing of pharmaceutical products in India, with headquarters in Mumbai.

Over the last ten years, Bayer Zydus Pharma has been a successful partnership combining Bayer’s scientific expertise and commercialization of novel products backed by Cadila Healthcare’s strong India presence.

The Joint venture company focused on addressing the unmet healthcare needs of patients in India by providing best-in-class innovative health solutions.

During the term of the joint venture, the company also launched some of Bayer’s global innovative assets like Xarelto®, EyleaTM and Visanne® in India.

Bayer Zydus Pharma will continue to operate in core therapies including cardiovascular diseases, diabetes, women’s health, ophthalmology and oncology, with new products in the pipeline.

Key products in the company’s pharmaceutical portfolio include Xarelto®, Glucobay®, EyleaTM, Yaz®, Mirena® and Visanne® amongst others.

Mr. Manoj Saxena, Managing Director, Bayer Zydus Pharma, said, “The Joint Venture with our trusted partner Zydus Cadila has been successful over the last decade in driving scalable reach of our health solutions to patients across the country.

We endeavour to carry this momentum forward, harnessing the benefits of our partnership towards delivering innovation-led, patient-centric offerings and digital health tools in India.

By sustaining the gains we have made over the years, we aim to fulfill unmet patient needs, strengthen the Indian healthcare ecosystem and translate our vision of ‘Health for All, Hunger for None’ into reality.”

Speaking on the development, Managing Director of Cadila Healthcare Ltd., Dr. Sharvil Patel said, “We have always believed in the power of collaboration and working with partners to create win-win alliances which unlock value for patients and the healthcare system at large.

The spirit of partnership in this joint venture has all been about channeling the core strengths of both Zydus and Bayer for the benefit of the patients.

By maximizing the reach, enabling access to innovative products for the patients and leveraging expertise for better health outcomes, the JV has set a benchmark.

Moving ahead, we see the efforts intensifying as we address a common goal of contributing to a robust and sustainable healthcare system in India.”

Bayer has been present in India for 125 years in the area of Crop Science and Pharmaceuticals.

The pharmaceutical division’s extension of the joint venture with Cadila Healthcare testifies Bayer’s commitment to serve the healthcare needs of patients in India.

Through strategic and time-honoured alliances with local partners, Bayer Zydus Pharma will continue to offer new-generation therapies to address the nation’s prevailing and emerging health concerns.

https://www.bayer.in/en/news/bayer-and-zydus-announce-continuity-their-successful-joint-venture

UN statement on renewal of humanitarian lifeline to millions of people in north-west Syria

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UN statement on renewal of humanitarian lifeline to millions of people in north-west Syria

June 18, 2021: “Millions of people are pressed up against the border in an active war zone in north-west Syria and remain in need of humanitarian aid to survive.

The United Nations (UN) needs cross-border and cross-line access to reach those most in need.

We call for the renewal of Security Council authorization for cross-border operations from Turkey to north-west Syria.

A failure to do so would immediately stop UN delivery of food, COVID-19 vaccines, critical medical supplies, shelter, protection, clean water and sanitation, and other life-saving assistance to 3.4 million people, including 1 million children.

The UN continues engagement with all concerned parties to also allow cross-line convoys into the north-west.

They are critical for the expansion of the overall response, but even if deployed regularly they could not replicate the size and scope of the cross-border operation. There is simply no alternative.

A large-scale UN cross-border response for an additional 12 months remains essential to avert a humanitarian catastrophe in north-west Syria.”

https://www.who.int/news/item/18-06-2021-united-nations-statement-on-the-renewal-of-humanitarian-lifeline-to-millions-of-people-in-north-west-syria

AstraZeneca welcomes Court ruling on supply of its COVID-19 vaccine to Europe

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AstraZeneca welcomes Court ruling on supply of its COVID-19 vaccine to Europe

June 18, 2021: “AstraZeneca welcomed the ruling by the Court of First Instance in Brussels.

The European Commission had requested 120 million vaccine doses cumulatively by the end of June 2021, and a total of 300 million doses by the end of September 2021.

The judge ordered delivery of 80.2 million doses by 27 September 2021. To date, the Company has supplied more than 70 million doses to the European Union and will substantially exceed 80.2 million doses by the end of June 2021.

All other measures sought by the European Commission have been dismissed, and in particular the Court found that the European Commission has no exclusivity or right of priority over all other contracting parties.

The judgement also acknowledged that the difficulties experienced by AstraZeneca in this unprecedented situation had a substantial impact on the delay.

AstraZeneca now looks forward to renewed collaboration with the European Commission to help combat the pandemic in Europe.

The Company remains committed to broad and equitable distribution of the vaccine as laid out in the Advanced Purchase Agreement of August 2020.

In fewer than twelve months, AstraZeneca has worked extremely hard to develop an effective vaccine at no profit and is the second-largest supplier to the EU’s 27 member states.

The supply of the vaccine is estimated to have helped save tens of thousands of lives and to have significantly reduced hospitalisations.

Real World Evidence has consistently shown a greater than 90% reduction in severe disease and hospitalisations caused by COVID-19, while recent data from England and Scotland demonstrated 92% vaccine effectiveness against hospitalisation from the Delta (previously called “Indian”) variant.

Jeffrey Pott, General Counsel, said: “We are pleased with the Court’s order. AstraZeneca has fully complied with its agreement with the European Commission and we will continue to focus on the urgent task of supplying an effective vaccine, which we are delivering at no profit to help protect people in Europe and around the world from the deadliest pandemic in a generation.”

The Company continues to engage with governments, international organisations and collaborators around the world to ensure broad and equitable access to this well-tolerated and effective vaccine at no profit during the pandemic period.

Vaxzevria
Vaxzevria (ChAdOx1-S [Recombinant]), formerly AZD1222, was co-invented by the University of Oxford and its spin-out company, Vaccitech.

It uses a replication-deficient chimpanzee viral vector based on a weakened version of a common cold virus (adenovirus) that causes infections in chimpanzees and contains the genetic material of the SARS-CoV-2 virus spike protein.

After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2021/astrazeneca-welcomes-court-ruling-on-supply-of-its-covid-19-vaccine-to-europe.html

FDA Approves a Nasal Antihistamine for Non-prescription Use

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FDA Approves a Nasal Antihistamine for Non-prescription Use

June 17, 2021: “The U.S. FDA approved a nasal antihistamine for nonprescription use through a process called a partial prescription to nonprescription switch.

The FDA approved Astepro (azelastine hydrochloride nasal spray, 0.15%) for seasonal and perennial allergic rhinitis—commonly known as allergies—for adults and children six years of age and older. 

“Seasonal and perennial allergies affect millions of Americans every year, causing them to experience symptoms of nasal congestion, runny nose, sneezing and more,” said Theresa M. Michele, M.D., director of the office of nonprescription drugs in the FDA’s Center for Drug Evaluation and Research.

“Today’s approval provides individuals an option for a safe and effective nasal antihistamine without requiring the assistance of a healthcare provider.”

For a drug to switch from prescription to nonprescription status, the data provided must demonstrate that the drug is safe and effective for use in self-medication as directed in proposed labeling.

The manufacturer must show that consumers can understand how to use the drug safely and effectively without the supervision of a healthcare professional.

This approval is a first-in-class switch for a nasal antihistamine and is considered a partial switch because the 0.1% strength, which includes the perennial allergy indication for children 6 months to 6 years old and seasonal allergy indication for children 2 to 6 years old, will remain prescription based.

Azelastine can cause drowsiness. The label warns that consumers using this product should avoid alcoholic drinks and be careful when driving a motor vehicle or operating machinery.

Using azelastine nasal spray with alcohol, sedatives, or tranquilizers may increase drowsiness.

The FDA granted the approval of nonprescription Astepro to Bayer Healthcare LLC.”

https://www.fda.gov/news-events/press-announcements/fda-approves-nasal-antihistamine-nonprescription-use

Lilly Launches Think Talk Treat Migraine™ to Help Improve Diagnosis and Treatment of Migraine

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Lilly Launches Think Talk Treat Migraine™ to Help Improve Diagnosis and Treatment of Migraine

June 17, 2021: Based on results from a large population-based survey, it is estimated that approximately half of people with migraine hesitate to seek care for a variety of reasons, and even fewer receive a diagnosis and take the treatment they need.

To help address these important unmet needs in migraine care, Eli Lilly and Company launched Think Talk Treat Migraine™, a new initiative to empower people living with migraine and their primary care providers to recognize key symptoms of migraine and have better conversations about the disease that can lead to proper diagnosis and treatment.

The initiative is currently in pilot stage – available in four metropolitan areas – Hartford/New Haven (Connecticut), Raleigh/Durham (North Carolina), Indianapolis (Indiana) and Denver (Colorado) – and provides people living with migraine and their healthcare providers with simple resources to think about, talk about and treat migraine.

“People with significant disabling headache often do not recognize it as migraine and as a result, understandably do not seek care.

Further, among those who do seek care, there is often a gap in communication between them and their primary care provider, particularly about the impact migraine has on their life.

This can not only lead to fewer appropriate diagnoses, but also treatment recommendations that may not deliver the freedom from migraine pain they seek,” said Michael Cobas Meyer, MD, MBA, vice president of global medical affairs, Lilly Bio-Medicines.

“The goal of Think Talk Treat Migraine is to simplify the recognition and discussion of the disease from both the patient and the primary care provider perspectives, potentially leading to meaningful improvements in health outcomes through accurate diagnosis and appropriate, timely care.”

More people impacted by migraine seek care from a primary care provider than any other care setting, yet research shows that only a small proportion receive a migraine diagnosis and treatment recommendation during those initial discussions.

There can be many reasons for this including the short length of an office visit or the ability to effectively convey or recognize symptoms.

While 40% of people with migraine are eligible for preventive treatment, less than 20% receive it.

Further, only 28% of people impacted by migraine and eligible for acute treatment are seeking care, diagnosed with migraine and prescribed recommended acute medication.

“I’ve been living with migraine for most of my adult life and like many others who may be misdiagnosed or undiagnosed, I didn’t realize there was a name for what I was experiencing or that it was a neurological disease,” said Kelsey V., 27 years old from Colorado.

“It’s important that initial discussions about migraine with one’s health care provider are easier because having the right diagnosis and the right treatment can make all the difference.”

Think Talk Treat Migraine provides information, tools and resources specifically developed for people who may be living with migraine and primary care providers, including a Talk To Your Healthcare Provider Checklist, a new digital migraine management platform called VEGA™ and education about the ID Migraine® tool, which helps identify people living with migraine by asking three simple questions:

  • Has a headache limited your activities any day in the last month?
  • Are you nauseated or sick to your stomach when you have a headache?
  • Does light bother you when you have a headache?

Research has shown that if a person experiencing headaches answers yes to two of these questions, there is a greater than 90% chance they are living with migraine and should speak with their healthcare provider.

VEGA is a digital platform that offers a smartphone app to enable users to track important information about their migraine such as their ability to function, pain severity, symptoms, triggers and the medications they take to manage it.

With this information, a person can more effectively talk to their provider about the impact migraine has on their life, which may help inform an appropriate treatment plan. 

Think Talk Treat Migraine will also host webinars for primary care providers to help elevate awareness of migraine within the primary care setting.

Information provided during the webinars will focus on a streamlined approach to help identify migraine, information about how to assess the impact of migraine and insights about how the extent of impact a migraine has can inform an appropriate treatment plan.

“Despite its prevalence and the human and societal impact of migraine, this disease is often underestimated and misunderstood.

A variety of reasons, including stigma and lack of awareness, drive some people living with migraine to hide their pain and the effects of the disease.

This can lead to underdiagnoses and unnecessary pain and disability,” said Kevin Lenaburg, executive director, Coalition for Headache and Migraine Patients (CHAMP). 

“Lilly understands the impact that not getting an accurate diagnosis can have on people living with migraine and recognizes that it doesn’t have to be that way.

Think Talk Treat Migraine is fostering important conversations while providing the tools and resources to help make a meaningful difference for people with migraine and primary care providers alike.”

https://investor.lilly.com/news-releases/news-release-details/lilly-launches-think-talk-treat-migrainetm-help-improve

CureVac’s COVID-19 vaccine CVnCoV misses the mark in late-stage study

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CureVac’s COVID-19 vaccine CVnCoV misses the mark in late-stage study

June 16, 2021: “CureVac, a clinical-stage biopharmaceutical company developing a new class of transformative medicines based on messenger ribonucleic acid (“mRNA”) announced results of the second interim analysis of its international pivotal Phase 2b/3 study in approximatively 40,000 subjects (the HERALD study) of CureVac’s first-generation COVID-19 vaccine candidate, CVnCoV.

In the unprecedented context of at least 13 variants circulating within the study population subset assessed at this interim analysis, CVnCoV demonstrated an interim vaccine efficacy of 47% against COVID-19 disease of any severity and did not meet prespecified statistical success criteria.

Initial analyses suggest age and strain dependent efficacy. Available data were communicated with the European Medicines Agency (EMA).

The Data Safety Monitoring Board (DSMB) confirmed a favorable safety profile for CVnCoV.

The study is continuing to the final analysis and the totality of the data will be assessed for the most appropriate regulatory pathway.

In total, 134 Covid-19 cases were assessed in this interim analysis. Out of these cases, 124 were sequenced to identify the variant causing the infection.

The outcome confirms that only one single case was attributable to the original SARS-CoV-2 virus. More than half of the cases (57%) were caused by Variants of Concern.

Most of the remaining cases were caused by other less characterised variants such as Lambda or C.37, first identified in Peru (21%) and B.1.621, first identified in Colombia (7%).

In this context, the interim results suggest efficacy in younger participants but did not allow to conclude on efficacy in the age group above 60.

“While we were hoping for a stronger interim outcome, we recognize that demonstrating high efficacy in this unprecedented broad diversity of variants is challenging.

As we are continuing toward the final analysis with a minimum of 80 additional cases, the overall vaccine efficacy may change,” said Dr. Franz-Werner Haas, Chief Executive Officer of CureVac.

“In addition, the variant-rich environment underlines the importance of developing next-generation vaccines as new virus variants continue to emerge.”

The HERALD study, conducted by Curevac in conjunction with Bayer, enrolled approximately 40,000 participants in ten countries in Latin America and Europe.

The second interim analysis included 134 cases, occurring at least two weeks after administration of the second dose.

To identify strains causing COVID-19 infections within the trial, sequencing of virus variants has so far been performed on 474 COVID-19 cases, of which 124 fulfilled adjudication criteria and were included in the present efficacy analysis.

CureVac remains committed to COVID-19 vaccine development.

Beyond CVnCoV, the company develops in partnership with GSK second-generation COVID-19 vaccine candidates.

These candidates are based on new mRNA backbones and include potential variants in multivalent vaccine formats as well as combination vaccines for potential protection against multiple infectious diseases in one vaccine.

Preclinical data from the first vaccine candidate, CV2CoV, has recently been accepted for publication in Nature Communications.

CureVac and GSK expect to progress the second-generation vaccine candidate into clinical testing in the third quarter of 2021, with the goal of introducing the vaccine in 2022, subject to regulatory approval.”

https://www.curevac.com/en/2021/06/16/curevac-provides-update-on-phase-2b-3-trial-of-first-generation-covid-19-vaccine-candidate-cvncov/

Lilly Announces Head-to-Head Study Comparing Once-Monthly Emgality® with Every-Other-Day Nurtec® ODT for the Preventive Treatment of Migraine

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Lilly Announces Head-to-Head Study Comparing Once-Monthly Emgality® with Every-Other-Day Nurtec® ODT for the Preventive Treatment of Migraine

June 15, 2021: To advance the science of migraine treatment and aid the understanding of calcitonin gene-related peptide (CGRP) monoclonal antibodies (mAbs) compared to oral CGRP receptor antagonists in the prevention of migraine, Eli Lilly and Company  will be conducting a head-to-head study comparing once-monthly injectable Emgality® (galcanezumab-gnlm) with Nurtec® ODT (rimegepant), an orally disintegrating tablet patients take every other day.

CGRP is a protein in the brain thought to play a key role in migraine. Emgality binds to this protein, preventing it from attaching to the CGRP receptors, whereas Nurtec ODT blocks the receptor for this protein.

This study aims to answer important questions that will help clinicians and patients make more informed treatment decisions on the path to more migraine-free days.

The study, which is the first head-to-head clinical trial comparing two medications targeting CGRP, is a multi-site, randomized, double-blind, double-dummy, parallel-group Phase 4 study in patients who meet the International Classification of Headache Disorders (ICHD) criteria for a diagnosis of episodic migraine with or without aura.

There will be two treatment arms: Emgality 120 mg once-monthly injection, with an initial 240 mg loading dose, and Nurtec ODT 75 mg, taken every other day.

The study’s primary endpoint is 50% reduction in monthly migraine headache days. Enrollment is expected to begin later this year.

“Migraine is a painful, burdensome and complex neurologic disease. Every person’s experience is different. Providing patients with options and individualized treatment plans is vitally important,” said Ilya Yuffa, senior vice president and president, Lilly Bio-Medicines.

“We are confident in Emgality’s efficacy profile and that our head-to-head clinical trial against Nurtec ODT will yield valuable insights for patients and their healthcare providers.”

Emgality is the only CGRP therapy that includes ≥50%, ≥75% and 100% reduction in monthly migraine headache days for the duration of the treatment period for episodic migraine patients, in its FDA-approved labeling.

Emgality’s adherence and persistence findings were recently presented at AHS, and insights about interictal burden (impact of migraine between migraine attacks) were shared previously.

“The American Headache Society and the National Headache Foundation have endorsed several goals that raise the treatment outcomes bar for preventive migraine medications.

Patients tell us they want an easy and convenient migraine treatment that can help them be productive and free to focus on what matters most to them.

In order for patients to manage their own disease and have a sense of personal control, they need to find treatments that work for them that they can stay on.

Reducing the number of days patients experience migraine is possible and it’s important that patients and their HCPs talk about this as a goal,” said Dr. Merle Diamond, managing director of the Diamond Headache Clinic and longstanding board member of the National Headache Foundation.

“Undertaking this head-to-head study signals Lilly’s confidence in Emgality, a once-monthly injectable monoclonal antibody CGRP antagonist (CGRP mAb), as it compares to Nurtec ODT, a small molecule oral CGRP receptor antagonist (gepant), for the prevention of migraine.”

“This year, we are proud to help more than 700,000 Emgality patients. We encourage HCPs and patients to talk about the preventive treatment goal of freedom from migraine through reduced frequency of attacks, which can also result in greater quality of life and functional improvements. 

We believe people should expect more and get more from their migraine medications,” said Yuffa. “We’re looking forward to commencing the study later this year and sharing our results.”

About Emgality
Emgality is a monoclonal antibody that selectively binds to calcitonin gene-related peptide (CGRP) and was approved by the FDA in September 2018 for the preventive treatment of migraine in adults.

Emgality is the only CGRP monoclonal antibody with response rates in the episodic migraine headache population on ≥50%, ≥75% and 100% reduction from baseline in monthly migraine headache days over Months 1 to 6 included in its Full Prescribing Information.

In June 2019, Emgality was approved by the FDA for the treatment of episodic cluster headache in adults.

Indications and Usage for Emgality (galcanezumab-gnlm) 120 mg Injection
Emgality is a calcitonin gene-related peptide (CGRP) antagonist indicated in adults for the:

  • preventive treatment of migraine
  • treatment of episodic cluster headache

Important Safety Information for Emgality
Contraindications
Emgality is contraindicated in patients with serious hypersensitivity to galcanezumab-gnlm or to any of the excipients.

Warnings and Precautions
Hypersensitivity Reactions
Hypersensitivity reactions, including dyspnea, urticaria, and rash, have occurred with Emgality in clinical studies and the postmarketing setting.

Cases of anaphylaxis and angioedema have also been reported in the postmarketing setting.

If a serious or severe hypersensitivity reaction occurs, discontinue administration of Emgality and initiate appropriate therapy. Hypersensitivity reactions can occur days after administration and may be prolonged.

Adverse Reactions
The most common adverse reactions (incidence ≥2% and at least 2% greater than placebo) in Emgality clinical studies were injection site reactions.”

https://investor.lilly.com/news-releases/news-release-details/lilly-announces-head-head-study-comparing-once-monthly-emgalityr

Scientists develop ‘game-changing’ antibody test to detect new variants

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Scientists develop ‘game-changing’ antibody test to detect new variants

June 15, 2021: Antibody tests that can detect whether people have been exposed to new variants of Covid-19 have been developed by the University of Aberdeen, in collaboration with biotechnology group Vertebrate Antibodies Ltd and NHS Grampian.

The new tests can detect antibody responses to infection by SARS-CoV-2 virus with more than 98% accuracy and 100% specificity.

This is in contrast to currently available tests that are around 60-93% accurate and cannot differentiate unique variants.  

For the first time, the new tests can be used to estimate the prevalence of circulating variant strains in the community, including the variants first identified in Kent and in India, now known as the Alpha and Delta variants.

The tests can also assess the long-term immunity of an individual and whether immunity is vaccine-induced or is a result of previous exposure to the infection – information that is invaluable in helping to prevent the spread of infection.  

In addition to this, the tests can also provide information that can be used to estimate the duration of the immunity provided by the vaccine as well as the effectiveness of the vaccine on emerging variants.  

This is an improvement on the currently available tests that struggle to detect variants and give little or no information on the impact of virus mutations on vaccine performance.  

Professor Mirela Delibegovic from the University of Aberdeen and academic lead on the project explains: “Accurate antibody tests will become increasingly important in the management of the pandemic and this is a truly game-changing technology with the potential to dramatically change the trajectory of global recovery from the pandemic.” 

Professor Delibegovic worked alongside industry partners, Vertebrate Antibodies and colleagues in NHS Grampian to develop the new tests using the innovative antibody technology known as Epitogen. 

Funded by the Scottish Government Chief Scientist Office Rapid Response in COVID-19 (RARC-19) research programme, the team used artificial intelligence called EpitopePredikt, to identify specific elements, or ‘hot spots’ of the virus that trigger the body’s immune defence.

The researchers were then able to develop a new way to display these viral elements as they would appear naturally in the virus, using a biological platform they named EpitoGen Technology. 

This approach enhances the test’s performance which means only relevant viral elements are included to allow improved sensitivity.

Importantly, this approach is capable of incorporating emerging mutants into the tests thus enhancing the test detection rates.  

As well as Covid-19, the EpitoGen platform can be used for the development of highly sensitive and specific diagnostic tests for infectious and auto-immune diseases such as Type 1 Diabetes. 

Dr Abdo Alnabulsi, Chief Operating Officer of AiBIOLOGICS, who helped develop the technology said: “Our tests have been designed in line with the gold standard requirements for such tests and in our trials they have proven to be more accurate while giving more useful information than existing tests.” 

Dr Tiehui Wang, Director of Biologics at Vertebrate Antibodies Ltd, added: “We are extremely proud that our technologies have made such a contribution in a very challenging year.  

“The EpitoGen tests are the first of their kind and will play a significant role in combating the pandemic and pave the way for future diagnostics”. 

Professor Delibegovic adds: “As we move through the pandemic we are seeing the virus mutate into more transmissible variants such as the Delta variant whereby they impact negatively on vaccine performance and overall immunity.

Currently available tests cannot detect these variants.  As the virus mutates, existing antibody tests will become even less accurate hence the urgent need for a novel approach to incorporate mutant strains into the test – this is exactly what we have achieved. 

“Looking ahead, discussions are already underway to explore a possible roll-out of the tests to the NHS which we hope to see happen soon.” 

Dr Brittain-Long, Consultant in Infectious Diseases in NHS Grampian and a member of the research team adds: “This new testing platform adds crucial sensitivity and specificity to the current available serology tests and has the potential to monitor individual and population based immunity in a way that has not been possible before.  

“In my work I have experienced first-hand the detrimental effects this virus can have on people, and I am very excited to add another tool in the toolbox to fight this pandemic.” 

https://www.abdn.ac.uk/smmsn/news/15066/

Soaring e-waste affects the health of millions of children, WHO warns

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Soaring e-waste affects the health of millions of children, WHO warns

June 15, 2021: Effective and binding action is urgently required to protect the millions of children, adolescents and expectant mothers worldwide whose health is jeopardized by the informal processing of discarded electrical or electronic devices according to a new ground-breaking report from the World Health Organization: children and Digital Dumpsites.

“With mounting volumes of production and disposal, the world faces what one recent international forum described as a mounting “tsunami of e-waste”, putting lives and health at risk.” said Dr Tedros Adhanom Ghebreyesus, WHO Director-General.

“In the same way the world has rallied to protect the seas and their ecosystems from plastic and microplastic pollution, we need to rally to protect our most valuable resource –the health of our children – from the growing threat of e-waste.”

As many as 12.9 million women are working in the informal waste sector, which potentially exposes them to toxic e-waste and puts them and their unborn children at risk.

Meanwhile more than 18 million children and adolescents, some as young as 5 years of age, are actively engaged in the informal industrial sector, of which waste processing is a sub-sector.

Children are often engaged by parents or caregivers in e-waste recycling because their small hands are more dexterous than those of adults.

Other children live, go to school and play near e-waste recycling centres where high levels of toxic chemicals, mostly lead and mercury, can damage their intellectual abilities 

Children exposed to e-waste are particularly vulnerable to the toxic chemicals they contain due to their smaller size, less developed organs and rapid rate of growth and development.

They absorb more pollutants relative to their size and are less able to metabolize or eradicate toxic substances from their bodies.

Impact of e-waste on human health

Workers, aiming to recover valuable materials such as copper and gold, are at risk of exposure to over 1,000 harmful substances, including lead, mercury, nickel, brominated flame retardants and polycyclic aromatic hydrocarbons (PAHs).

For an expectant mother, exposure to toxic e-waste can affect the health and development of her unborn child for the rest of its life.

Potential adverse health effects include negative birth outcomes, such as stillbirth and premature births, as well as low birth weight and length. 

Exposure to lead from e-waste recycling activities has been associated with significantly reduced neonatal behavioural neurological assessment scores, increased rates of attention deficit/hyperactivity disorder (ADHD), behavioural problems, changes in child temperament, sensory integration difficulties, and reduced cognitive and language scores.

Other adverse child health impacts linked to e-waste include changes in lung function, respiratory and respiratory effects, DNA damage, impaired thyroid function and increased risk of some chronic diseases later in life, such as cancer and cardiovascular disease.

“A child who eats just one chicken egg from Agbogbloshie, a waste site in Ghana, will absorb 220 times the European Food Safety Authority daily limit for intake of chlorinated dioxins,” said Marie-Noel Brune Drisse, the lead WHO author on the report.

“Improper e-waste management is the cause.  This is a rising issue that many countries do not recognize yet as a health problem.

If they do not act now, its impacts will have a devastating health effect on children and lay a heavy burden on the health sector in the years to come.”

A rapidly escalating problem

E-waste volumes are surging globally. According to the Global E-waste Statistics Partnership (GESP), they grew by 21% in the five years up to 2019, when 53.6 million metric tonnes of e-waste were generated.  For perspective, last year’s e-waste weighed as much as 350 cruise ships placed end to end to form a line 125km long. This growth is projected to continue as the use of computers, mobile phones and other electronics continues to expand, alongside their rapid obsolescence. 

Only 17.4% of e-waste produced in 2019 reached formal management or recycling facilities, according to the most recent GESP estimates, the rest was illegally dumped, overwhelmingly in low- or middle-income countries, where it is recycled by informal workers.

Appropriate collection and recycling of e-waste is key to protect the environment and reduce climate emissions.

In 2019, the GESP found that the 17.4% of e-waste that was collected and appropriately recycled prevented as much as 15 million tonnes of carbon dioxide equivalents from being released into the environment.

Call to Action

Children and Digital Dumpsites calls for effective and binding action by exporters, importers and governments to ensure environmentally sound disposal of e-waste and the health and safety of workers, their families and communities; to monitor e-waste exposure and health outcomes; to facilitate better reuse of materials; and to encourage the manufacture of more durable electronic and electrical equipment.

It also calls on the health community to take action to reduce the adverse health effects from e-waste, by building health sector capacity to diagnose, monitor and prevent toxic exposure among children and women, raising awareness of the potential co-benefits of more responsible recycling, working with affected communities and advocating for better data and health research on the health risks faced by informal e-waste workers.

“Children and adolescents have the right to grow and learn in a healthy environment, and exposure to electrical and electronic waste and its many toxic components unquestionably impacts that right,” said Dr Maria Neira, Director, Department of Environment, Climate Change and Health, at the WHO.

“The health sector can play a role by providing leadership and advocacy, conducting research, influencing policy-makers, engaging communities, and reaching out to other sectors to demand that health concerns be made central to e-waste policies.”

https://www.who.int/news/item/15-06-2021-soaring-e-waste-affects-the-health-of-millions-of-children-who-warns

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