The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of of BRT-DA01 in patients with Parkinson’s disease.?. preliminary?efficacy?of

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Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing. We believe we are at a breakthrough moment in Alzheimer’s disease research.

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Codon

JANUARY 21, 2024

Today, refined versions of these human challenge studies have become standard practice in testing vaccines for vector-borne diseases (e.g., yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops. Why are challenge trials becoming more popular?

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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PPD

NOVEMBER 7, 2023

Food and Drug Administration (FDA) draft guidance, “Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials.” The issue of including diverse populations that represent disease prevalence and country and regional demographics is not new to industry.

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Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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The Pharma Data

SEPTEMBER 8, 2020

Phase III clinical trial programme initiated for investigational medicine fenebrutinib, designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS). Roche’s Chief Medical Officer and Head of Global Product Development. “In

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The Pharma Data

APRIL 7, 2021

Sutimlimab, a first-in-class investigational C1s inhibitor, has the potential to be the first approved treatment for hemolysis in adults with cold agglutinin disease, a serious and chronic autoimmune hemolytic anemia. Sutimlimab has the potential to address a major unmet medical need for people with cold agglutinin disease.”.

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Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Up until 2023 around 600 NK cell clinical trials have been reported and some 200 are clinically active and still recruiting patients.

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Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

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Quanticate

JUNE 16, 2020

The leading cause of death in patients with coronavirus disease 2019 (COVID-19), caused by the virus ‘severe acute respiratory syndrome coronavirus 2’ (SARS‑CoV‑2), is respiratory failure from acute respiratory distress syndrome. [1]

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The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . adults have been diagnosed with dry eye disease, a chronic, episodic, multifactorial disease. It also increases your risk for inflamed cornea, cornea eye disease, corneal ulcers and injuries, and eye infections. .

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The Pharma Data

AUGUST 22, 2021

The trial accrued 25 cases of symptomatic COVID-19 at the primary analysis.There were no cases of severe COVID-19 or COVID-19-related deaths in those treated with AZD7442. The trial included 5,197 participants in a 2:1 randomisation AZD7442 to placebo. The trial was conducted in 87 sites in the US, UK, Spain, France and Belgium.

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The Pharma Data

APRIL 3, 2021

The CoVIg-19 Plasma Alliance today announced that the Phase 3 Inpatient Treatment with Anti-Coronavirus Immunoglobulin (ITAC) clinical trial sponsored and funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), did not meet its endpoints. About the ITAC Trial.

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New Drug Approvals

SEPTEMBER 29, 2024

gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., gingivalis DNA was detected in postmortem cortices from people with AD and healthy controls, and in CSF of AD patients ( Jan 2019 news on Dominy et al., gingivalis is associated with cardiovascular disease.

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The Pharma Data

NOVEMBER 12, 2020

The DETOUR1 trial is the first-in-man trial of the FDA-designated Breakthrough Device, The Detour System, evaluating the safety and effectiveness of the novel procedure and device system for treating long, complex lesions in the superficial femoral artery (SFA). An illustration of a completed Detour Procedure (Graphic: Business Wire).

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The Pharma Data

AUGUST 7, 2020

The governing body claims this proposal could have positive effects on mitigating burdensome requirements for gene therapy clinical trials in the future. In this light, Pharma IQ’s weekly round-up focuses on advancing therapies and clinical trials to combat Covid-19.

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The Pharma Data

DECEMBER 5, 2020

.–(BUSINESS WIRE) December 5, 2020 — Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, today announced new clinical data from its Phase 2 trial evaluating SY-1425, its first-in-class selective retinoic acid receptor alpha (RAR?)

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The Pharma Data

NOVEMBER 12, 2020

12 November 2020 — The CALAVI Phase II trials for Calquence (acalabrutinib) in patients hospitalised with respiratory symptoms of COVID-19 did not meet the primary efficacy endpoint. No new safety signal for Calquence was observed in the trials. No new safety signal for Calquence was observed in the trials.

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The Pharma Data

JULY 22, 2021

NOTE: ADUHELM TM (aducanumab-avwa)injection 100 mg/mL solutionis indicated for the treatment of Alzheimer’s disease. Treatment with ADUHELM should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. Biogen Inc.

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The Pharma Data

SEPTEMBER 12, 2020

Food and Drug Administration to expand the enrollment of their Phase 3 pivotal COVID-19 vaccine trial to up to approximately 44,000 participants which also allows for the enrollment of new populations. The pivotal trial is event-based and there are many variables that will ultimately impact read-out timing.

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FDA Law Blog: Drug Discovery

OCTOBER 2, 2023

Just as the original 1962 efficacy standard makes scientific judgment central to a determination of substantial evidence, the 1997 amendment makes scientific judgment central to a determination of what constitutes sufficient confirmatory evidence to substantiate a single adequate and well-controlled trial.

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Broad Institute

JANUARY 30, 2024

Bhattacharyya wears many coats today, splitting his time between the Broad and the infectious disease ward at MGH, where a diagnostic method he developed at Broad is now being tested to see if it helps patients quickly receive the best antibiotics for their infections. million deaths attributed directly to resistant infections.

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The Pharma Data

JANUARY 17, 2021

n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life. n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We We’re the middleman,” he said.

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Drug Target Review

APRIL 26, 2023

Gene-editing methods allow researchers to modify DNA, resulting in alterations in physical characteristics such as eye colour and susceptibility to diseases. 1 RNA-delivery particles have the potential to revolutionise the treatment of lung diseases such as cystic fibrosis.

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The Pharma Data

SEPTEMBER 29, 2020

. Sosei Heptares has received a $5m milestone payment from its strategic alliance partner Pfizer after the first subject in a clinical trial was dosed with a new drug candidate from the collaboration between the two companies.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Now, FDA’s CBER is setting its sights on making ambitious strides toward tackling rare diseases.

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The Pharma Data

AUGUST 11, 2020

.–( BUSINESS WIRE )– Bristol Myers Squibb (NYSE: BMY) today announced the Phase 3 CheckMate -577 trial evaluating Opdivo (nivolumab) as an adjuvant therapy for patients with resected esophageal or gastroesophageal junction (GEJ) cancer met its primary endpoint of disease-free survival (DFS) at a pre-specified interim analysis.

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The Pharma Data

SEPTEMBER 3, 2020

Johnson & Johnson Announces that Janssen’s COVID-19 Investigational Vaccine Candidate Prevents Severe Clinical Disease in Pre-clinical Studies. Johnson & Johnson Announces that Janssen’s COVID-19 Investigational Vaccine Candidate Prevents Severe Clinical Disease in Pre-clinical Studies.

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The Pharma Data

DECEMBER 29, 2020

As in earlier outpatient trial, immune status when patients entered the trial was a strong predictor of viral load and clinical outcomes. The primary clinical objective of this initial analysis was to determine if there was sufficient efficacy in these patients to warrant continuing the trial (i.e., futility analysis).

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Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. Dr Rockley previously completed an MSc in Cancer Pharmacology and have experience in clinical research from an oncology clinical trial coordinator role. 2019 Mar 1;55:93–100.

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Drug Target Review

SEPTEMBER 21, 2023

Some report that they believe that if they don’t take the medication, they may develop onchocerciasis-related severe skin diseases and may go blind (river blindness) and would prefer a treatment that does not repeat every year. Need for adulticidal drugs More than 99 percent of countries worldwide has been affected by this disease.

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The Pharma Data

OCTOBER 1, 2021

Results from the Phase 3 POETYK PSO-1 and POETYK PSO-2 trials show that response rates for deucravacitinib continued to extend through Week 24 and were maintained through Week 52 in patients with moderate to severe plaque psoriasis. CESTidentified through 52 weeks of knowledge. million people within the us.

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The Pharma Data

SEPTEMBER 23, 2020

September 23, 2020 – Johnson & Johnson (NYSE: JNJ) (the Company) today announced the launch of its large-scale, pivotal, multi-country Phase 3 trial (ENSEMBLE) for its COVID-19 vaccine candidate, JNJ-78436735, being developed by its Janssen Pharmaceutical Companies. NEW BRUNSWICK, N.J., In the U.S.,

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The Pharma Data

SEPTEMBER 25, 2020

Johnson & Johnson Posts Interim Results from Phase 1/2a Clinical Trial of its Janssen COVID-19 Vaccine Candidate. Johnson & Johnson Posts Interim Results from Phase 1/2a Clinical Trial of its Janssen COVID-19 Vaccine Candidate. The full set of results will be published once the complete Phase 1/2a trial data are available.

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The Pharma Data

JANUARY 31, 2021

Clover plans to initiate a global Phase 2/3 efficacy trial of its protein-based S-Trimer COVID-19 vaccine candidate adjuvanted with Dynavax’s CpG 1018 plus alum in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021. EMERYVILLE, Calif. and CHENGDU, China , Feb.

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