GSK and CEPI Joint Collaboration for Development of Coronavirus Vaccine
On 3rd February 2020, the Coalition for Epidemic Preparedness Innovations (CEPI) and GlaxoSmithKline (GSK) have announced a new collaboration aimed at helping the global effort to develop a vaccine for the 2019 novel coronavirus, renamed COVID-19 by the World Health Organisation (WHO) as of 11th February 2020. As part of the collaboration GSK will make its established pandemic vaccine adjuvant platform technology available for development of an effective vaccine against the virus. The use of an adjuvant is of importance in a pandemic situation since it can reduce the number of antigens required per dose, allowing more vaccine doses to be produced and made available to more people.
Thomas Breuer, Chief Medical Officer, GSK Vaccines, stated: “As a leader in science and innovation, we believe we can help to contribute to the fight against 2019-nCOV with one of our advanced vaccine adjuvant systems. Our adjuvant technology has previously been used successfully in the pandemic flu setting. It enables using only small quantities of the vaccine antigen which allows the production of more doses of the vaccine – a crucial advantage in a pandemic.”
CEPI will coordinate the engagements between GSK and CEPI funded entities who want to test their vaccine platform with GSK’s adjuvant technology to develop effective vaccines against COVID-19. The first agreement has been signed between GSK and the University of Queensland, Australia, which entered a partnering agreement with CEPI on January 2019 to develop a “molecular clamp” vaccine platform, intended for targeted and rapid vaccine production against multiple viral pathogens. CEPI has extended the funding to work on a COVID-19 virus vaccine candidate, and access to the GSK adjuvant technology will now support early stage research.
Gilead’s Remdesivir to Enter Human Clinical Trials for Coronavirus Treatment in China
Gilead Sciences has entered a partnership with the Chinese Health Authorities to conduct a randomised Phase 3, double-blind, placebo-controlled multicentre study to evaluate the efficacy and safety of remdesivir in hospitalized adult patients with mild and moderate COVID-19 infections. The study is expected to be completed on 27th April, set to enrol 270 patients with mild and moderate pneumonia caused by the coronavirus and be carried out in the China-Japan Friendship Hospital in Beijing.
The drug was originally developed for an intravenous treatment for Ebola, but it also showed potential against coronavirus and Nipah virus infections. Remdesivir is yet to be approved anywhere globally and has not been proved to be safe or effective for any use. A paper in The New England Journal of Medicine published 31st January suggested there was a possibility of using the drug candidate to help contain the deadly COVID-19. On 26th January, a 35-year-old patient in the United States (US) with the infection received remdesivir on day seven of illness and their clinical condition was seen to have improved on day eight.
Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences stated: “Remdesivir is not yet licensed or approved anywhere globally and has not been demonstrated to be safe or effective for any use. At the request of treating physicians, and with the support of local regulatory agencies, who have weighed the risks and benefits of providing an experimental drug with no data in 2019-nCoV, Gilead has provided remdesivir for use in a small number of patients with 2019-nCoV for emergency treatment in the absence of any approved treatment options. Gilead is working with health authorities in China to establish a randomised, controlled trial to determine whether remdesivir can safely and effectively be used to treat 2019-nCoV. We are also expediting appropriate laboratory testing of remdesivir against 2019-nCoV samples.”
The European Medicines Agency (EMA) Announces Support for Research of Treatment for COVID-19
On 4th February 2020, The European Medicines Agency (EMA) released a statement on the outbreak of COVID-19, the agency is taking concrete actions to accelerate the development and availability of medicinal products for the treatment and prevention of the virus. There are currently no commercially available medicinal products that are authorised to detect, treat or prevent infections.
Guido Rasi, EMA Executive Director stated: “EMA has activated its plan for managing emerging health threats. The new coronavirus has been declared a public health emergency of international concern by the World Health Organization, and we are drawing on the strong expertise of the European medicines network to provide fast-track scientific advice and give prompt feedback on any proposed medicine developments.”
The agency is looking into potential antivirals or vaccines to treat or prevent COVID-19 infections and is analysing all available information from developers’ drug pipelines. The regulatory body is ready to support drug developers with all available regulatory tools to advance and expedite the advancement of effective measures to fight and prevent the spread of this virus.
Developers working on medicinal products or vaccines that could be used for treatment or prevention of novel coronavirus infections are encouraged to contact the agency and discuss their strategy for evidence generation as soon as possible. They can contact EMA by sending an email to 2019-nCoV@ema.europa.eu for initial discussions with EU regulators.
BioMotiv and Bristol-Myers Squibb (BMS) Launch Anteros Pharmaceuticals
On 4th February 2020, BMS and drug development accelerator BioMotiv announced that they have launched Anteros Pharmaceuticals, a biotechnology company focused on developing a new class of drugs for fibrotic and other inflammatory diseases. The new company is backed by intellectual property (IP) originally developed at Yale University, and licensed by BMS which later assigned it to Anteros. BMS will also support Anteros with data, and reagents for a series of small molecules targeting an undisclosed mechanism, and BioMotiv working in close partnership with Yale, will handle research and development activities. Once Anteros nominates a pre-clinical candidate, BMS has an option to acquire the company from BioMotiv under pre-agreed terms.
Rupert Vessey, Executive Vice President, Research & Early Development, Bristol-Myers Squibb stated: “Bristol-Myers Squibb is committed to partnering with innovative collaborators like BioMotiv, which expand our reach and capabilities in early stage research. These types of partnerships also enable us to progress the transformational research being performed at leading academic institutions such as Yale and effectively translate those discoveries into new therapies for patients. We look forward to our continued collaboration with BioMotiv and to the scientific advancements driven by Anteros.”
On September 2019 BMS and BioMotiv established a partnership to form and providing funding to companies for the development of drugs to treat diseases with an unmet medical need, Anteros Pharmaceuticals is the first company launched since the agreement was put into place.
Aimmune Therapeutics Secures Funding from Nestlé Health Science
On 5th February 2020, Aimmune Therapeutics Inc., a US based biopharmaceutical company developing and commercialising treatments for potentially life-threatening food allergies, announced that Nestlé Health Science will make an additional investment of $200 million, the latest funding bringing Nestlé’s total investment in Aimmune to $473 million. This announcement follows Food and Drug Administration (FDA)’s approval of Palforzia® on 31st January, indicated for mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanuts. The drug, manufactured in powdered form from peanuts, can be taken in initial dose escalation, up-dosing and maintenance phases.
Jayson Dallas, M.D., President and CEO of Aimmune Therapeutics stated: “Nestlé Health Science has been an important equity investor and strategic partner to Aimmune since we entered into our original collaboration in 2016, and we are grateful for their ongoing commitment toward our mutual goal of developing innovative therapies for food allergies. This additional capital strengthens our financial position as we prepare to launch Palforzia®, the first FDA-approved medicine for peanut allergy in the U.S. In addition, this capital will help fund the continued advancement of our pipeline, including conducting clinical trials of AIMab7195 which we recently in-licensed from Xencor.”
Sanofi’s Investigational Multiple Sclerosis Drug Meets Primary Endpoint in Phase 2 Trial
On 6th February 2020, Sanofi announced that its Phase 2b trial evaluating its investigational Bruton’s tyrosine kinase (BTK) inhibitor, named SAR442168, for the treatment of relapsing multiple sclerosis (RMS) had achieved its primary endpoint. In the trial, SAR442168 significantly reduced multiple sclerosis’ (MS) disease activity as measured by brain lesions using magnetic resonance imaging (MRI). Safety results were consistent with the previously reported Phase 1 study. SAR442168 is an investigational, oral, brain-penetrant, selective small-molecule inhibitor of BTK that had previously shown BTK binding as well as cerebrospinal fluid exposure in Phase 1 studies. The efficacy and safety of SA442168 has not yet been reviewed by any regulatory authority.
John Reed, M.D., Ph.D., Sanofi’s Global Head of Research and Development stated: “The vast majority of people living with multiple sclerosis still endure disability during the course of their disease. We believe our BTK inhibitor has the potential to transform how MS is treated. This molecule may be the first B-cell-targeted MS therapy that not only inhibits the peripheral immune system, but also crosses the blood-brain barrier to suppress immune cells that have migrated into the brain, while also modulating the brain-resident microglia cells that have been implicated in MS progression. Building on Sanofi’s heritage in multiple sclerosis, we are encouraged by these clinical results and look forward to rapidly advancing our brain-penetrant BTK inhibitor into pivotal clinical trials.”
Given the results, the company plans to start four Phase 3 trials investigating the effects of SAR442168 on MS relapse rates, disability progression, and underlying central nervous system damage to be initiated later this year.
Medicines and Healthcare Products Regulatory Agency (MHRA) Grants Licence Update for Ipsen’s Dysport®
On 6th February 2020, Ipsen, a global biopharmaceutical company focused on innovation and specialty care, announced that the MHRA had granted a licence update to Dysport® for the symptomatic treatment of focal spasticity of upper limbs in paediatric cerebral palsy patients, two years of age and older. Spasticity refers to abnormal and involuntary muscle stiffness, or overactive muscular contractions, with cerebral palsy being the leading cause of childhood disability affecting function and development, and the most frequent cause of spasticity in children. Approximately 17 million people are affected by cerebral palsy worldwide, 1 in 400 babies in the United Kingdom (UK) are born with the condition.
Dysport® is an injectable form of a botulinum neurotoxin type A product and supplied as lyophilized powder, works by inhibiting the effective transmission of nerve impulse, reducing muscular contractions. MHRA’s decision comes from results of a Phase 3 randomised, double-blind, low dose controlled, multicentre study evaluating Dysport® in a group of 210 children aged 2 to 17 for upper limb spasticity due to cerebral palsy and measured by the Modified Ashworth Scale (MAS), a standard scale for assessing muscle resistance associated with spasticity. Patients with a Grade 2 or higher MAS at the primary targeted muscle groups (PTMG), were injected into the PTMG with doses of Dysport® at 8,16 or 2 Units/kg. Primary endpoint of the study was mean change in MAS grade from baseline to Treatment 1 at week 6 in PTMG. Results from the study showed that treatment with Dysport® lead to statistically significant improvements from baseline in MAS in the PTMG at week 6. The safety profile of Dysport® was also assessed and noted as being consistent with that seen in the approved indications for paediatric cerebral palsy lower limb spasticity after repeated injections and no new safety concerns were found.
Asad Mohsin Ali, UK & Ireland General Manager, Ipsen stated: “Today’s approval is an important advancement for children in the UK living with cerebral palsy, who can now benefit from long-lasting symptom relief between their botulinum toxin A injections. As a father myself, I am proud that Ipsen is the first company to have obtained this approval that may help children live as normal a life as possible.”
Company Led Drug Alert: Glenmark Recalls Iohexol Solution Batches
On 6th February 2020, the MHRA announced that Glenmark Pharmaceuticals is recalling specific batches of Iohexol solution for injection (350 mg/ml and 300 mg/ml) as a precautionary measure.
The recall is due to an out of specification result from the ongoing stability studies. The out of specification result for all batches of Iohexol distributed in UK is up to 4% over the upper specifications limits for content (105%). The affected batches are Iohexol 350 mg/ml solution for injection PL 25258/0247 and Iohexol 300 mg/ml solution for injection PL 25258/0246. The company is conducting their recall at the wholesaler and pharmacy level, according to the MHRA all remaining stock of these batches should be quarantined and returned to the original supplier for credit.
FDA Approves Marketing of First AI-Guided Image Acquisition System
On 7th February 2020, the FDA announced that it had authorised marketing of software Caption Guidance from medical artificial intelligence (AI) firm Caption Health, that allows medical professionals without specialised training to perform cardiac ultrasound. Caption Guidance is an accessory to compatible diagnostic ultrasound systems and uses AI to provide real-time guidance and help the user capture images of a patient’s heart that are of acceptable diagnostic quality, empowering healthcare providers and those without prior ultrasound experience. The software is indicated for use in ultrasound examination of the heart, known as two-dimensional transthoracic echocardiography (2D-TTE), for adult patients, specifically in the acquisition of standard views of the heart from different angles which are used in the diagnosis of various cardiac conditions.
Robert Ochs, Ph.D., deputy director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Centre for Devices and Radiological Health stated: “Echocardiograms are one of the most widely-used diagnostic tools in the diagnosis and treatment of heart disease. Today’s marketing authorization enables medical professionals who may not be experts in ultrasonography, such as a registered nurse in a family care clinic or others, to use this tool. This is especially important because it demonstrates the potential for AI and machine learning technologies to increase access to safe and effective cardiac diagnostics that can be life-saving for patients.”
Caption Guidance was developed using machine learning to train the software to differentiate between acceptable and unacceptable image quality, forming the basis of an interactive AI user interface that provides prescriptive guidance to users on how to maneuverer the ultrasound probe to acquire standard echocardiographic images and video clips of diagnostic quality. The AI interface provides real-time feedback on potential image quality, can auto-capture video clips, and automatically saves the best video clip acquired from a particular view.
The Caption Guidance software currently can be used with a specific FDA-cleared diagnostic ultrasound system produced by Teratech Corporation, with the potential to be used with other ultrasound imaging systems that have technical specifications consistent with the range of ultrasound systems used as part of the development and testing.
Roche’s and Lily’s Investigational Drugs in DIAN-TU-001 Study Fail to Achieve Primary Endpoint
On 10th February 2020, it was announced that an international clinical trial evaluating Lily’s solanezumab and Roche’s gantenerumab on whether two investigational drugs could slow memory loss and cognitive decline in people in the early stages of a rare, inherited form of Alzheimer’s disease (AD) has yielded disappointing results an initial analysis of the data has shown.
A Phase 2/3 randomised, double-blind, placebo-controlled study sponsored by Washington University School of Medicine in St. Louis, US in the Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU) testing two investigational therapies compared to placebo to determine if either of these treatments could slow the rate of cognitive decline and improve disease-related biomarkers in people with a rare, inherited, early-onset form of AD did not meet its primary endpoint. The study followed 194 participants for up to seven years; all participants come from families that carry a genetic mutation that causes early-onset Alzheimer’s dementia.
Participants were randomly assigned to receive solanezumab, gantenerumab or placebo. Family members who did not have the Alzheimer’s mutations were also included as a comparison. As part of the trial doses of the investigational drugs were increased during the study to try to enhance potential beneficial effects. The primary outcome measure for the study, the DIAN Multivariate Cognitive Endpoint, is a novel outcome measure designed to assess cognitive performance in people with AD. Initial analysis indicated that neither drug met the primary outcome of the study, which was a slowing of cognitive decline as measured by multiple tests of thinking and memory. Both investigational drugs are designed to target and neutralise amyloid beta in the brain through different mechanisms and are being evaluated in other, more common forms of Alzheimer’s disease.
Trial’s data will be presented at the Advances in Alzheimer’s and Parkinson’s Therapies Focus Meeting on 2nd April 2020, followed by presentations at the Alzheimer’s Association International Conference in Amsterdam in July. Despite the study’s disappointing results, DIAN-TU introduced a ground-breaking collaboration among academic institutions, pharmaceutical companies, patient-advocacy groups, the National Institutes of Health (NIH) and private supporters. The public-private partnership and researcher network created to conduct the trial can be useful for future studies of investigational Alzheimer’s drugs.
FDA Grants Priority Review to Novartis’s Capmatinib
On 11th February 2020, Novartis announced that the FDA had accepted and granted Priority Review to capmatinib’s (INC280) New Drug Application (NDA). Capmatinib is designed to inhibit a heterodimer receptor tyrosine kinase called mesenchymal–epithelial transition (MET) being evaluated for use in patients with non-small cell lung cancer (NSCLC), advanced or metastatic MET exon 14 skipping (METex14) mutated NSCLC in first-line and also previously treated patients. If approved, capmatinib will be the first therapy to specifically target METex14 mutated advanced lung cancer, a type of lung cancer with a particularly poor prognostic which currently lacks approved treatments.
The NDA submission includes results from the Phase 2 GEOMETRY mono-1 clinical trial, which showed 40.6% overall response rate among previously treated patients and 67.9% among treatment-naïve participants based on the Blinded Independent Review Committee (BIRC) assessment. It also showed that capmatinib provided durable responses among all patients: median duration of response was 11.14 months in treatment-naïve patients and 9.72 months (in previously treated patients. The most common drug-related adverse events noted were peripheral oedema, and nausea.
John Tsai, M.D., Head of Global Drug Development and Chief Medical Officer, Novartis stated: “We are extremely encouraged by the FDA’s Priority Review designation for capmatinib, a MET inhibitor that may be a major treatment advance for patients with this particularly aggressive form of lung cancer. Results of the GEOMETRY mono-1 trial clearly identify METex14 as an oncogenic driver and we are inspired to bring capmatinib, potentially the first METex14 targeted therapy, to patients and to reimagine medicine and outcomes for people with lung cancer.”
WHO Announces Official Name of Disease Caused by 2019 Novel Coronavirus
On 11th February 2020, WHO announced that the official name of the disease caused by the 2019 novel coronavirus is COVID-19.
A statement was released by the WHO explain the reasoning behind the name choice, Dr Tedros Adhanom Ghebreyesus, Director-General of the WHO stated : “Under agreed guidelines between WHO, the World Organisation for Animal Health and the Food and Agriculture Organization of the United Nations, we had to find a name that did not refer to a geographical location, an animal, an individual or group of people, and which is also pronounceable and related to the disease. Having a name matters to prevent the use of other names that can be inaccurate or stigmatizing. It also gives us a standard format to use for any future coronavirus outbreaks.”
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