The Pharma Data

JANUARY 15, 2021

In the DESTINY-Gastric01 trial, patients (n=126) in the Enhertu treatment arm had a 41% reduction in the risk of death versus patients (n=62) treated with chemotherapy (based on a hazard ratio [HR] of 0.59; 95% confidence interval [CI] 0.39-0.88; Regular approval by the U.S. months [95% CI 9.6-14.3] months [95% CI 6.9-10.7] with chemotherapy.

Treatment 52

Treatment Trials Disease Therapies 52

The Pharma Data

JANUARY 13, 2021

we determined the crystal structure of CD40-ligand 2 , developed a humanized version of our antibody (hu5c8, ruplizumab, or Antova®) and tested it in human trials for preventing organ transplant rejection and autoimmunity. CD40-ligand is a protein expressed on the surface of activated T lymphocytes that mediates T cell helper function.

Pharmaceuticals 52

Pharmaceuticals Small Molecule Vaccine Protein Expression 52

FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Where vector presence is detected, transgene mRNA and/or protein expression levels should also be measured. Section #3: Human Trials Finally, the guidance provides a quick overview of clinical study recommendations. For gene therapy products, the use of quantitative and sensitive assays such as qPCR are recommended.

Therapies 59

Therapies FDA Production Disease 59

The Pharma Data

OCTOBER 28, 2020

In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons.

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Protein Expression Therapies Treatment Disease 40

The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. This guidance provides clarity on the path to registration for AVXS-101 IT.

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Clinical Development Therapies Clinical Trials Treatment 52

The Pharma Data

APRIL 8, 2021

Roche will present data from five studies from the EVRYSDI clinical development programme, which was designed to represent a broad spectrum of people living with SMA. Spinal Muscular Atrophy (SMA). Multiple Sclerosis (MS). Neuromyelitis Optica Spectrum Disorder (NMOSD). Roche will present five sets of data on adults living with NMOSD.

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Clinical Trials Trials Disease Therapies 52

Conversations in Drug Development Trends

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.

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Treatment Clinical Trials Small Molecule Therapies 81

The Pharma Data

SEPTEMBER 2, 2020

Initiation of Phase IIIb OCREVUS higher dose clinical trial programme and Phase IV study evaluating OCREVUS in minority populations. Roche is also deeply committed to addressing barriers to clinical trial participation and advancing inclusive research. Many people with NMOSD remain misdiagnosed and untreated.

Clinical Trials 52

Clinical Trials Disease Treatment Therapies 52

The Pharma Data

AUGUST 9, 2021

Positive high-level results from the head-to-head DESTINY-Breast03 Phase III trial showed that Enhertu (trastuzumab deruxtecan), the AstraZeneca and Daiichi Sankyo Company, Limited (Daiichi Sankyo) HER2-directed antibody drug conjugate (ADC), demonstrated superiority over trastuzumab emtansine (T-DM1).

Trials 52

Trials Treatment Clinical Trials Protein Expression 52

The Pharma Data

OCTOBER 26, 2020

The purpose of this exploratory Phase 1b/2 trial was to evaluate the safety and efficacy of the combination of TG4001 and an immune checkpoint inhibitor in a heterogeneous group of patients with aggressive, recurrent and/or metastatic HPV16-positive cancers. Key findings of the trial: .

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Vaccine Trials Treatment Virus 40