The Pharma Data

SEPTEMBER 14, 2020

. AstraZeneca has confirmed that clinical trials assessing its Oxford University partnered coronavirus vaccine AZD1222 have resumed in the UK, following a green light by the Medicines Health Regulatory Authority (MHRA). Source link.

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pharmaphorum

MARCH 3, 2021

Novavax hopes its COVID-19 vaccine could be filed in the US in the second quarter, following a potential approval from the UK regulator in the coming weeks. The timetable for approval from the FDA depends on whether the regulator accepts data from the company’s trial in the UK, which could set up a potential Emergency Use Authorisation.

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The Pharma Data

DECEMBER 3, 2020

New Tau Protein is Abnormal in Very Early Alzheimer’s Disease. Investigators with the University of Gothenburg identified new forms of tau protein that become abnormal in the very early stages of Alzheimer’s disease, even before the development of cognitive problems. “The Every week there are numerous scientific studies published.

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XTalks

APRIL 3, 2024

The RBCs in circulation are marked by proteins in the complement system for removal by the spleen and liver. Voydeya’s FDA approval was based on positive results from the pivotal ALPHA Phase III trial. In the trial, the most common adverse events were headache, nausea, arthralgia (joint stiffness) and diarrhea.

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pharmaphorum

DECEMBER 22, 2021

Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient. In an update posted today the pharma giant said “we do not yet have complete information and are actively working with the trial site investigator to understand what happened.”

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XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness. It is injected into a patient’s muscle tissue.

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Gene Therapy Gene Protein FDA Approval 98

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. The post bluebird seeks gene therapy trial restart after cancer scare appeared first on.

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Drug Discovery World

JANUARY 11, 2024

The team, led by University College London (UCL), King’s College London and Moderna scientists, found that mRNA could be used to correct a rare genetic liver disease known as argininosuccinic aciduria in a mouse model. mRNA contains instructions that direct the cells to make proteins.

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Research Gene Therapy RNA Gene 59

XTalks

SEPTEMBER 17, 2021

After asking for a placebo-controlled Phase III study for the drug in April that upset the ALS Association, the FDA dialed back on the request this week, agreeing to accept only Phase II trial data for evaluation of Amylyx’s drug application. Accelerated FDA Approval Modeled on Aduhelm. People with ALS and their loved ones cannot wait.”.

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XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012.

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Drug Discovery World

NOVEMBER 27, 2023

The European Commission has extended use of the therapy to include children with CF aged two through five years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “As The combined actions of ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways.

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XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Regulators may ask at some point to provide that documentation, so it’s important to track that across the study.

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Clinical Trials Clinical Trials Trials FDA Approval 98

The Pharma Data

NOVEMBER 22, 2020

As the Pfizer and BioNTech COVID-19 vaccine goes to regulators and the Moderna vaccine approaches the end of its Phase III trials, AstraZeneca and the University of Oxford announced high-level results from an interim analysis of their COVID-19 vaccine, AZD1222. Trials are also being run in the U.S.,

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Drug Discovery World

APRIL 5, 2023

The drug, which is now called NXP800 and has already entered a Phase I clinical trial, was discovered by scientists at The Institute of Cancer Research, London. It targets a pathway that cancer cells are highly dependent upon, involving the heat shock factor 1 protein (HSF1).

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pharmaphorum

SEPTEMBER 17, 2020

US medical experts are reportedly concerned that a neurological side effect picked up in AstraZeneca’s closely-watched COVID-19 vaccine trial could compromise the whole project, as the FDA weighs whether to give the go ahead for US studies to resume. Feature image courtesy of Rocky Mountain Laboratories/NIH.

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XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

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pharmaphorum

DECEMBER 1, 2020

Pfizer and BioNTech have filed their COVID-19 vaccine with the European medicines regulator, paving the way for a potential approval before the end of the year. The companies initially filed the nonclinical data and other technical data, followed by emerging clinical trial results.

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Drug Discovery World

JANUARY 23, 2023

Armed with this understanding, the researchers further demonstrated that the genetic inhibition of DGAT1 suppresses radioresistance. Additionally, the clinical trial period will be considerably shorter than that required for new drug development.

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Drug Discovery World

OCTOBER 18, 2023

4 Unfortunately, many AAV-based gene therapies fail during clinical trials, despite them appearing safe and efficacious in preclinical studies. For example, researchers may find that an AAV vector-based gene therapy displays excellent transduction efficiency in a preclinical model, only to see the efficiency drop in clinical trials.

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The Pharma Data

NOVEMBER 25, 2021

A combination of two targeted cancer drugs showed unprecedented, “clinically meaningful” activity in patients with highly malignant brain tumors that carried a rare genetic mutation, according to a clinical trial report by investigators from Dana-Farber Cancer Institute.

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XTalks

DECEMBER 12, 2023

The world of neuroscience trials is rapidly evolving, presenting new challenges and opportunities for researchers and clinicians. The worldwide market for neurology clinical trials stood at a valuation of $5.24 There is currently a heightened emphasis on imaging and liquid biomarkers in neuroscience clinical trials.

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pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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Drug Discovery World

MAY 24, 2023

G protein-coupled receptors (GPCRs) are a large family of proteins that regulate many processes in the body and are the target of one third of clinically used drugs. The researchers genetically modified mice to express the human NK1 receptor.

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XTalks

JANUARY 26, 2023

The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinical trials. Diagnosis is confirmed by a differential complete blood count and genetic testing.

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Drug Discovery World

SEPTEMBER 5, 2022

Tumours (yellow) pervade the lungs of mice genetically prone to small cell lung cancer (left) on this computed tomography scan. Phase I trial on the horizon. The team discovered that RNF113A is regulated by a protein called SMYD3 that is highly expressed in small cell lung cancer and some other cancers.

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Protein Drugs Medicine Research 52

XTalks

SEPTEMBER 2, 2021

The AlzoSure Predict prognostic biomarker Alzheimer’s blood test detects levels of an unfolded conformational variant of the p53 protein in the blood using a proprietary antibody called U-p53AZ. p53 in Alzheimer’s. It is known as the “guardian of the genome” given its central role in the DNA damage response. Diadem’s Alzheimer’s Blood Test.

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pharmaphorum

OCTOBER 6, 2020

The announcement comes just days after the regulator said its CHMP scientific committee had begun to look at the first batch of data from AZ’s rival. In the case of a rolling review, the EMA evaluates trial data as they become available before a complete filing is submitted.

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Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP). A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.

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pharmaphorum

NOVEMBER 9, 2020

The companies said they will be ready to file the vaccine with the FDA in the third week of November once they have enough safety data to meet the regulator’s requirements. Top-line results come from a phase 3 trial of the candidate known as BNT162b2 and an evaluation of 94 cases from the trial of 43,538 patients.

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Vaccination Vaccine Clinical Trials Clinical Trials 98

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. “We are committed to understand and mitigate any risk of cancer causation.”

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pharmaphorum

NOVEMBER 18, 2021

RNAi uses small RNA molecules to bind to messenger RNA, which is responsible for synthesising proteins, and either destroy it or recruit other cellular factors that regulate its activity. Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.

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Drug Discovery World

JULY 5, 2023

For example, in CAR-T-cell therapy, scientists can take T-cells from a patient and ex vivo (outside the body) genetically engineer them to effectively target and kill cancerous cells, then infuse them back in to the patient. Of course, if a patient’s tumour does not have this protein, then this type of treatment won’t be effective.

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Research Protein Scientist Immune Response 98

XTalks

MARCH 10, 2023

It is caused by a mutation in the FXN gene , which is responsible for the production of the protein frataxin. Omaveloxolone Clinical Trials Skyclarys is an oral medication that contains omaveloxolone, an activator of the nuclear factor-erythroid 2 related factor 2 (Nrf2) pathway.

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pharmaphorum

DECEMBER 21, 2020

But the tragic events of 2020 have meant these companies have become household names as their trailblazing mRNA vaccines became the first to be approved by regulators against the COVID-19 scourge. In this case that is the “spike” protein seen on the surface of the coronavirus that it uses to invade host cells.

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The Pharma Data

MARCH 16, 2021

This NDA is based on data from the Phase 2 Study-004 trial, in which belzutifan showed a confirmed overall response rate of 36.1% (n=22/61) (95% CI: 24.2-49.4) About the Phase 2 Study-004 Trial. Proteins known as hypoxia-inducible factors, including HIF-2?, If not properly regulated, the accumulation of HIF-2?

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XTalks

MARCH 13, 2024

XTALKS WEBINAR: Addressing Ophthalmology Clinical Trial Design and Effective Data Monitoring Committee Strategies On-Demand: Thursday, February 8, 2024 Register for this free webinar gain insights into statistical data strategies for ophthalmology clinical trials and learn best practices for establishing and reporting ophthalmic data.

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XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. The disease is caused by mutations on the TSC1 and TSC2 genes, which produce the proteins hamartin and tuberin, respectively. In the study, 148 patients out of a total of 224 received Epidiolex, and changes in seizure frequency from baseline were measured.

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