Drug Discovery and Development Archives - Page 72 of 158

An intelligent approach to data cleaning

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The collection of good quality data from clinical trials is essential to data analysis to produce robust results that meet the precise requirements for regulatory need. Data from clinical trials are increasingly complex, related to involved protocols, the geography of trial sites, increasing data streams and technological advances. Therefore, studies must be set up to be efficient, offer support and training to trial sites and ensure that the right data are collected correctly.

Data Management teams have historically reviewed data once source document verification (SDV) has taken place by the Clinical Monitors on an ongoing basis. Data issues can be actioned early in the trial, and corrective action put in place. This activity has been a very manual process, thorough and time-consuming and has left less time to focus on insightful data analysis.

Collaboration: Data science and da…

CureVac and GSK announce positive preclinical data for bivalent next-gen mRNA vaccine candidate

Tübingen, Germany–based CureVac N.V. (Nasdaq:CVAC) and its partner GSK (NYSE:GSK) have announced positive preclinical data about their second-generation COVID-19 vaccine candidate that combines mRNAs for the beta and delta variants.

The companies conducted the preclinical research in conjunction with the Friedrich-Loeffler-Institut, Germany.

The research pitted the bivalent candidate against monovalent COVID-19 vaccine candidates.

The bivalent vaccine generated a robust immune response in animals, resulting in a significant reduction of viral load, high neutralizing antibody titers and strong T cell responses.

In a test focused on rats, the bivalent vaccine candidate resulted in a two-fold increase in neutralizing antibody titers against the omicron variant compared to the delta variant.

The researchers also tested the vaccine candidate in transgenic mice expressing the human ACE2 receptor.

The companies have published a manuscript o…

Trikafta wins Canadian approval for Trikafta in children with cystic fibrosis

Vertex Pharmaceuticals Inc. (Nasdaq: VRTX) has won marketing authorization for the expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children with cystic fibrosis who are between the ages of 6 and 11 years old.

The latest approval covers children with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The approval allows an additional dosage strength of Trikafta tablets consisting of elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg, and ivacaftor 75 mg.

According to the company, the new approval will provide a treatment option to roughly 500 children.

“This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF,” said Dr. Reshma Kewalramani, chief executive officer and president at Vertex. “We will now work closely with all provinces and territories to secure access for eligible patients as quickly …

AstraZeneca’s Evusheld offered strong protection against COVID-19 in Phase 3 study

In the Phase 3 Provent trial, Evusheld (AZD442) cut the risk of developing symptomatic COVID-19 by 77% in the primary analysis focused on patients at high risk for severe COVID-19. The monoclonal antibody cocktail, including tixagevimab and cilgavimab cut the risk by 83% in the six-month follow-up analysis compared to placebo.

The drug is currently authorized for emergency use for pre-exposure prophylaxis of COVID-19.

Evusheld won a EUA in December 2021.

The adults enrolled in the trial had an inadequate response to COVID-19 vaccination, an elevated risk of exposure to SARS-CoV-2 or both. Subjects were either obese, immunocompromised, had pre-existing health conditions such as heart failure or chronic kidney disease, or were 60 years of age or older.

After receiving a single 300 mg intramuscular dose of Evusheld, concentrations of the drug remained elevated for six months after administration, according to a pharmacokinetic analysis.

Th…

Otonomy shares positive top-line results from hearing loss trial

The neurotology biopharma Otonomy (Nasdaq:OTIC) has announced that the brain-derived neurotrophic factor agonist OTO-413 resulted in clinically meaningful improvement in a Phase 2a trial focused on subjects with hearing loss.

In the randomized, double-blind, placebo-controlled study, subjects received a single intratympanic injection of 0.3 mg of OTO-413.

Subjects in the study had 30 evaluable subjects with up to moderately-severe hearing loss.

A sustained exposure formulation of brain-derived neurotrophic factor (BDNF) bested placebo in multiple speech-in-noise (SIN) hearing tests.

The trial also administered the Patient Global Impression of Change (PGIC) on days 57 and 85 of the study.

A total of 40% (8 of 20) of OTO-413 recipients had a clinically-meaningful improvement in at least one of the three SIN tests. Conversely, two out of 10 placebo recipients achieved a comparable level of positive change.

Three of the patients in the d…

Amazon Web Services is powering medtech innovation: Its chief medical officer explains

Dr. Taha Kass-Hout is the chief medical officer and director of machine learning at Amazon Web Services [Photo courtesy of Amazon]

It doesn’t get any bigger than Amazon in the world of cloud computing.

The Amazon Web Services cloud computing business at Seattle-based Amazon.com (Nasdaq: AMZN) is the largest player in the industry, with control of about a third of the market and a significant lead over cloud competitors Microsoft and Google.

Dr. Taha Kass-Hout, the chief medical officer and director of machine learning at AWS, spoke with Medical Design & Outsourcing as part of an ongoing series of conversations about cloud computing’s contributions to medtech and the potential ahead.

In the following interview, Kass-Hout explains how Moderna was born in the cloud and how the company has used cloud computing to scale mRNA technology.

Get the full story from our sister site, Medical Design …

Takeda wins approval to distribute Novavax’s COVID-19 vaccine in Japan

Takeda (NYSE:TAK) has announced that it has won manufacturing and marketing approval from the Japan Ministry of Health, Labour and Welfare (MHLW) for Nuvaxovid, a novel COVID-19 vaccine.

Nuvaxovid (NVX-CoV2373) was initially developed by Novavax (Nasdaq:NVAX).

The approval covers the use of the vaccine as both a primary and booster immunization in adults at least 18 years old.

Nuvaxovid is a recombinant protein-based vaccine.

Sanofi (Nasdaq:SNY) and GSK (NYSE:GSK) are also jointly developing a similar COVID-19 vaccine. Sanofi and GSK also announced their intent to file international regulatory submissions for their recombinant protein-based COVID-19 vaccine candidate.

Osaka, Japan–based Takeda won regulatory approval after submitting efficacy and safety data from a Phase 1/2 study in Japan and data from two pivotal Phase 3 trials in the UK, U.S. and Mexico. The company also submitted data from Phase 1/2 studies in Australia and Phase 2 data…

Genomics 2.0: Trusted research environments to manage 500M genomes

Introduction

Image courtesy of Pixabay

The study of genetics dates to the mid-19th century, from the works of Gregor Mendel, but it wasn’t until the second half of the 20th century that the field of genetics made great strides. The completion of the Human Genome Project and other significant technological advances were the driving factors for this significant impact. In light of these advances, more than 1,800 disease genes have been discovered, and more than 2000 genetic tests have become available to the public.

Advances in technology such as next-generation sequencing (NGS) have allowed scientists to perform experiments at a rate that was never possible before. NGS is a DNA sequencing technology that allows the whole genome of an individual to be sequenced within one day, producing a large amount of clinical health data. Unfortunately, maintaining small data sets in a centralized location for analysis (Ge…

Moderna says bivalent COVID-19 boosters could offer more robust protection against variants

Moderna (Nasdaq:MRNA) announced that its first bivalent booster vaccine candidate, mRNA-1273.211, offered superior protection against SARS-CoV-2 variants of concern compared with its first-gen mRNA-1273 vaccine.

A 50-µg booster of the mRNA-1273.211 vaccine offered protection against the beta, delta and omicron variants for six months and appeared to provide similar tolerability to the FDA-approved mRNA-1273 vaccine.

The company continues to develop a vaccine known as mRNA-1273.214 with more omicron-specific mutations. That vaccine candidate is now in a Phase 2/3 clinical trial. The company expects data from that trial to be available in the second quarter.

Moderna has published data related to the mRNA-1273.211 candidate as a preprint study in Research Square.

The company continues to bet that its bivalent booster candidate, mRNA-1273.214, will find use as a booster in autumn 2022 in the Northern Hemisphere.

In any event, the mRNA-1273.211 …

Black Diamond Therapeutics doses first patient in Phase 1 study focused on non-small cell lung cancer NSCLC and glioblastoma

Black Diamond Therapeutics (Nasdaq: BDTX) has announced that it has dosed the first patient in the Phase 1 study focused on BDTX-1535.

BDTX-1535 is a MasterKey inhibitor of epidermal growth factor receptor (EGFR) to potentially treat non-small cell lung cancer (NSCLC) and glioblastoma (GBM).

Cambridge, Massachusetts–based Black Diamond believes BDTX-1535 holds significant potential for EGFR-mutant NSCLC and GBM.

“This is the second MasterKey inhibitor derived from our MAP drug discovery engine; we are incredibly excited about BDTX-1535’s advancement into the clinic, and we look forward to providing a clinical update in the second half of 2023,” said David M. Epstein, CEO of Black Diamond Therapeutics.

“Despite recent successes in targeting EGFR-mutated NSCLC, there is still a need for better therapeutics for patients with disease progression following first-line EGFR inhibitors,” said Dr. Melissa Johnson, director of lung cancer research for Sara…

Alzheimer’s group files FOIA request related to CMS decision on amyloid-targeting antibodies

UsAgainstAlzheimer’s, a nonprofit organization committed to stopping Alzheimer’s disease, is hoping to get more information related to CMS’s national coverage determination related to coverage for Aduhelm (aducanumab) from Biogen (Nasdaq:BIIB) and other monoclonal antibodies targeting amyloid to treat Alzheimer’s disease.

The organization has filed a Freedom of Information Act (FOIA) request for information about the decision.

UsAgainstAlzheimer’s is seeking information describing the potentially harmful impact of refusing to cover such monoclonal antibodies outside of clinical trials.

CMS announced their national coverage determination for Aduhelm and other similar future monoclonal antibodies last week.

UsAgainstAlzheimer’s concluded that CMS has denied access to millions of people in the U.S. living with Alzheimer’s.

“The National Coverage Determination is historic – and not in a good way. CMS has covered every FDA-approved drug, but now…

Sanofi announces publication of early study of rilzabrutinib for immune thrombocytopenia

Sanofi (Nasdaq:SNY) has announced that the New England Journal of Medicine (NEJM) has published positive data from the Phase 1/2 dose-finding study testing the safety, pharmacokinetics and clinical activity of rilzabrutinib for immune thrombocytopenia.

Immune thrombocytopenia is associated with bruising and bleeding. Fewer than 200,000 Americans are diagnosed with the condition each year.

Rilzabrutinib is an experimental oral Bruton’s tyrosine kinase (BTK) inhibitor. The drug candidate has been the focus of about 20 clinical trials to date, with indications ranging from asthma to urticaria.

The study, published in NEJM, focused on adults with heavily pre-treated immune thrombocytopenia (ITP), concluded that rilzabrutinib therapy led to a rapid and durable increase in platelet count. Rilzabrutinib also appeared to have a good safety profile.

The drug candidate may increase platelet counts in patients with immune thrombocytopenia. As the NEJM articl…