pharmaphorum

NOVEMBER 4, 2020

It may not be appropriate to use the word ‘failure’ when summarising the utility of clinical trials in the search for a curative or preventative treatment for disease. Most of the trials are underpowered to demonstrate efficacy on mortality and long-term consequences of the disease.

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Clinical Trials Clinical Trials Trials Vaccination 105

AuroBlog - Aurous Healthcare Clinical Trials blog

MAY 18, 2023

In particular, researchers noticed a reduction in the interleukin-1-beta (IL-1β) protein, which helps to regulate the body’s […] It seems the chemical compound can stop some of the damage done to the brain that’s usually associated with the disease.

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Protein Regulation Research Clinical Trials 200

pharmaphorum

OCTOBER 2, 2020

European regulators have started a first ‘rolling review’ of a COVID-19 vaccine, which is being developed by AstraZeneca in collaboration with the University of Oxford. In a separate announcement, AstraZeneca said that a trial of the vaccine has resumed in Japan after a safety scare in the UK.

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Regulation Vaccination Vaccine Protein 105

BioPharma Reporter

DECEMBER 11, 2020

Sanofi and GSK announced a delay on Friday in their adjuvanted recombinant protein-based COVID-19 vaccine program to improve immune response in the elderly, saying clinical trials showed an insufficient immune response in older people.

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Immune Response Vaccination Vaccine Trials 105

Drug Discovery World

SEPTEMBER 20, 2023

The collaboration is designed to bolster the company’s offerings in the fields of research, population health, and regulated clinical trials. Olink Explore HT is the company's latest solution for high-throughput proteomics, allowing scientists to accurately measure over 5,300 proteins [.] Not yet a DDW member?

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Genome Genomics Research Clinical Trials 59

Pharmaceutical Technology

JUNE 22, 2023

The TARPEYO delayed-release capsule is a corticosteroid indicated for reducing proteinuria to treat primary immunoglobulin A nephropathy (IgAN) in adult patients at risk of quick disease progression, generally having a urine protein-to-creatinine ratio greater than or equal to 1.5g/g.

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Clinical Trials Clinical Trials Branding Protein 130

Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. One strategy proposed the use of synthetic mRNA or modified mRNA analogs, which can enhance the stability and protein expression of mRNA.

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Vaccination Vaccine Protein Antibody 52

Pharmaceutical Technology

JUNE 9, 2023

Rocket Pharmaceuticals stated that the fast-track designation enables increased communication with the regulator across the development cycle of the gene therapy. In April 2023, the FDA approved the investigational new drug (IND) application for RP-A601 to begin a Phase I clinical trial.

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Gene Therapy Gene FDA Approval Trials 147

Pharmaceutical Technology

MAY 8, 2023

The small molecule Bruton’s tyrosine kinase (BTK) inhibitor Brukinsa has been specially designed for delivering sustained BTK protein inhibition by optimising bioavailability, half-life, and selectivity. In April this year, the regulator converted these conditional approvals to regular approvals for R/R WM and R/R CLL/SLL.

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Bioavailability Clinical Trials Clinical Trials Protein 130

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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Vaccination Vaccine Trials Immune Response 52

The Pharma Data

JANUARY 17, 2021

The clinical trial to evaluate the safety and efficacy of the plasma-derived medicine will have doctors Oriol Mitjà and Bonaventura Clotet as the principal researchers and is expected to begin in early 2021. BARCELONA, Spain , Jan. 18, 2021 /PRNewswire/ — Grifols (MCE: GRF, MCE: GRF.P,

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Clinical Trials Clinical Trials Trials Containment 52

The Pharma Data

MARCH 23, 2022

Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. The researchers discovered that Ebola virus polymerase hijacks a cellular protein called GSPT1. To treat Ebola virus infections, researchers are taking a close look at a key piece of the virus: polymerase.

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Protein Genome Genomics Research 52

pharmaphorum

JUNE 30, 2021

Just a few months after starting clinical trials of its nasal spray vaccine for COVID-19, US biotech Altimmune is abandoning the project, saying that it generated weaker than expected immune responses in a phase 1 trial. . The post Altimmune ditches nasal COVID vaccine on weak trial data appeared first on.

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Vaccination Vaccine Trials Immune Response 98

pharmaphorum

MARCH 19, 2021

DeepVerge has announced trial data showing its COVID-19 Microtox BT breath test can deliver accurate results in under 60 seconds, with a potential roll-out later this year if further tests support it. A femtogram is 0.000000000000000001 kilograms.

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Contamination Clinical Trials Clinical Trials Protein 145

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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Trials Drugs FDA Approval Clinical Trials 98

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. 3 The world’s confidence in the future of these therapies is also clear, as the global AAV vector manufacturing market has a projected compound annual growth rate (CAGR) of 22.5%

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Gene Therapy Gene Clinical Trials Clinical Trials 64

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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Protein Research Regulation Gene 52

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. Pfizer has been talking about having interim data from the trial in the third quarter of next year.

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Gene Therapy Gene Trials Clinical Trials 58

pharmaphorum

JANUARY 5, 2022

A COVID-19 vaccine that could work against multiple variants of the coronavirus – developed by US biotech Gritstone bio – has generated encouraging immune response data in its first clinical trial. “With new variants – like Omicron and B.1.640.2

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Protein Vaccination Vaccine Immune Response 126

pharmaphorum

OCTOBER 4, 2021

Discover the Intricacies of Inflammasome Regulation, Leverage Inflammasome Proteins as Biomarkers & Accelerate Novel Inflammasome Therapeutics into Proof-Of-Concept Clinical Trials & Beyond. Download the full event guide for the speaker faculty and session details.

Protein 52

Protein Clinical Trials Clinical Trials Regulation 52

Pharmaceutical Technology

JANUARY 25, 2023

Development of PROTACs (PROteolysis TArgeting Chimeras) has advanced fairly rapidly as excitement grows over their potential to selectively degrade proteins that are involved in various diseases, including cancer. PROTACs are a type of heterobifunctional degrader that offers multiple advantages over traditional small molecules.

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Protein Bioavailability Development Scientist 130

The Pharma Data

DECEMBER 28, 2020

Cyclophilins, of which there are 17 known isoforms in humans, play a central role in protein folding and contribute to numerous biological effects including inflammation and fibrosis, to name a few. The AMBITION trial is the first placebo-controlled study of CRV431 in NASH patients with evidence of moderate-to-severe fibrosis.

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Clinical Trials Clinical Trials Trials Development 40

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 caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells.

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Gene Therapy Gene Protein FDA Approval 98

The Pharma Data

MARCH 30, 2021

Basel, March 30, 2021 — Novartis has obtained exclusive worldwide rights to develop and commercialize therapeutic applications for a library of Fibroblast Activation Protein (FAP) targeting agents including FAPI-46 and FAPI-74, through an assignment agreement with iTheranostics, Inc., an affiliate of SOFIE Biosciences, Inc.

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Protein Licensing Licensing Containment 52

Drug Discovery World

NOVEMBER 15, 2023

The small molecule, which binds to an inner region of a calcium channel to indirectly regulate it, outperformed gabapentin without troublesome side effects, providing a promising candidate for treating pain. A first-in-class small molecule Protein CRMP2 is a key regulator of the Cav2.2

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Scientist Protein Regulation Clinical Trials 52

Drug Discovery World

JULY 19, 2023

Lilly will work with the FDA and other global regulators to achieve the fastest path to traditional approvals. Participants in TRAILBLAZER-ALZ 2 were stratified by their level of the brain protein tau, a predictive biomarker for Alzheimer’s disease progression.

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Trials Clinical Trials Clinical Trials Research 52

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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Protein Gene Medicine Gene Therapy 52

pharmaphorum

AUGUST 10, 2020

Biogen’s Alzheimer’s drug aducanumab could be on the market in the next few months after the FDA accepted a clinical trial dossier and granted a fast Priority Review. The FDA has set a decision date for March 27 next year, and Biogen said that the regulator could make a ruling before then.

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Drugs Clinical Trials Clinical Trials Trials 105

Pharmaceutical Technology

FEBRUARY 26, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. It is composed of IL-2 and a pMHC complexed with a dominant peptide derived from the from the Wilms’ Tumor protein. The company also provides clinical trails and research services.

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Drugs Clinical Trials Clinical Trials Development 100

Pharmaceutical Technology

FEBRUARY 26, 2023

Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. It is composed of IL-2 and a pMHC complexed with a dominant peptide derived from the from the Wilms’ Tumor protein. The company also provides clinical trails and research services.

Drugs 100

Drugs Clinical Trials Clinical Trials Development 100

Drug Discovery World

JANUARY 31, 2024

Alpelisib is a protein-targeted medication that is used for treating breast cancer and overgrowth disorders in children. The medication regulates cell growth, proliferation and development and insulin signalling. This will radically change the way these patients are managed and could benefit thousands of children around the world.”

Drugs 52

Drugs Insulin Medicine Clinical Trials 52

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. Verifying the biomarker’s clinical validity for use as a surrogate endpoint in rare disease research is another hurdle which is generally a longer-term goal.

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Trials Gene Hormones Clinical Trials 98

Drug Discovery World

AUGUST 3, 2023

It becomes the ninth Covid-19 vaccine to be authorised by the UK’s independent medicines regulator. Bimervax combines a part of the SARS-CoV-2 virus spike protein with an adjuvant. The clinical trial has been authorised by the Spanish Agency for Medicines and Health Products (AEMPS).

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Vaccination Vaccine Immune Response Medicine 52

XTalks

SEPTEMBER 16, 2022

Approval was based on results from their Phase III clinical trials which demonstrated the superior efficacy and acceptable safety profile of Sotyktu compared to placebo and a comparator therapy. Tyrosine kinases are enzymes that regulate signaling pathways with downstream effects on cell growth, differentiation and death.

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FDA Approval Clinical Trials Clinical Trials Trials 52

XTalks

JANUARY 8, 2024

The FDA’s endorsement stems from results obtained from the company’s Phase III NefIgArd clinical trial. This accumulation triggers inflammation and damages kidney tissues, leading to the leakage of blood and protein into the urine. IgAN, a rare kidney disorder, is marked by the accumulation of IgA deposits within the kidneys.

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FDA Approval Clinical Trials Clinical Trials Nurses 64

Drug Discovery World

JANUARY 11, 2024

Argininosuccinic aciduria is an inherited metabolic disorder that affects how the body breaks down protein – potentially leading to high levels of ammonia in the blood. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

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Research Gene Therapy RNA Gene 59

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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Drugs Scientist Clinical Trials Clinical Trials 52