PPD

OCTOBER 9, 2023

“Patient-centric” is used frequently to describe today’s clinical trial and health care landscape. The industry applauds that point of view and strives to include a patient-centric approach in all aspects of clinical trial designs. Patient centricity has become more than a key consideration.

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PPD

JUNE 24, 2024

In the realm of vaccine development, mega trials — studies enrolling 5,000 subjects or more — have been instrumental in the fight against many pathogens, including influenza, rotavirus, malaria, RSV and most recently in the rapid development of vaccines against COVID-19.

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PPD

AUGUST 5, 2024

Though they do positively impact patients’ quality of life and hemodynamic parameters, these treatments — prostacyclin analogs and receptor agonists, phosphodiesterase 5 inhibitors, endothelin-receptor antagonists, and cGMP activators — have shown limited beneficial effects on survival and disease progression.

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Clinical Trials Trials Clinical Research Disease 98

FDA Law Blog: Biosimilars

APRIL 13, 2025

LYTGOBI was granted accelerated approval for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or rearrangements. As a single-arm trial (i.e., Join us as we break down what went wrong this time. What Happened?

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Drugs FDA Trials Treatment 52

On Medicine

SEPTEMBER 12, 2023

Considering such findings, the United Nations Office on Drugs and Crime (UNODC) combined the TRT intervention with parenting strategies designed to enhance the carer’s capacity to support children through life events such as disasters, conflicts, and displacement. TRT + Parenting was trialled in Lebanon with Syrian families.

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FDA Law Blog: Drug Discovery

MARCH 7, 2024

Clissold — The trio of CDER, CBER, and CDRH released a new draft guidance titled “ Use of Data Monitoring Committees in Clinical Trials ” that revises the 2006 guidance “Establishment and Operation of Clinical Trial Data Monitoring Committees” and, when final, will replace the 2006 guidance.

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Clinical Trials Trials FDA Regulations 105

Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases. Visit broad.io/L2C2023

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis.

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FDA Approval Treatment FDA DNA 96

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.

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DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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PPD

JULY 29, 2024

AI also has the potential to incorporate real-world data (RWD) obtained from electronic health records (EHRs), medical claims or other data sources to inform the design and optimization strategy of clinical trials.

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Clinical Research Clinical Trials Research Trials 98

Conversations in Drug Development Trends

AUGUST 10, 2023

As one of the more recent fields to emerge in the clinical space, psychedelics have gained significant traction as a potential treatment for various neuropsychiatric diseases. Psychedelic Research at Worldwide Since 2017, Worldwide Clinical Trials have supported a myriad of sponsors leading the way in psychedelic research programs.

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Science Clinical Trials Trials Clinical Research 98

Broad Institute

DECEMBER 18, 2023

Brain disorders are difficult to study and many drug candidates have failed in clinical trials, causing pharmaceutical companies to reduce their investments or even exit the field entirely. But a new path for bringing treatments to patients is starting to emerge. We all recognize the vast, unmet medical need.

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PPD

SEPTEMBER 16, 2024

This can be converted to a clinical trial simulator, which can model a clinical trial before the trial design is finalized and initiated. AI approaches are optimizing trial designs With a significant increase in the number of regulatory submissions to the U.S.

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Conversations in Drug Development Trends

JULY 8, 2024

By: Lisle Kingery, PhD, Therapeutic Strategy Lead, Neuroscience With depression rates at an all-time high and limited new treatments available, depression clinical trials are more important than ever. Treatment Treatment can take various forms, including therapy, medication, a combination of both, or a clinical trial.

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Clinical Trials Trials Treatment Therapies 52

Quanticate

AUGUST 2, 2024

Real-world evidence (RWE) is changing clinical drug development, bridging the gap between controlled clinical trial environments and the complexities of everyday patient experiences.

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Drug Development Clinical Trials Drugs Trials 104

PPD

OCTOBER 17, 2024

In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches. There has also been an increase in government and regulatory support for CGT trials, in conjunction with an increase in investments for these products to get to market.

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The Pharma Data

DECEMBER 30, 2020

Long-term safety profile consistent with previously completed controlled trials. Treatment with AXS-07, rapidly, substantially, and durably relieved migraine pain and associated symptoms in this trial. Axsome remains on track to submit an NDA for AXS-07 in the acute treatment of migraine in the first quarter of 2021.

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Trials Treatment Clinical Trials Therapies 52

Chemical Biology and Drug Design

MAY 11, 2023

Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects. Therefore, this compound is considered a safe and effective therapeutic choice for neurodegenerative illnesses like Parkinson's disease.

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Vial

JUNE 24, 2024

Contract research organizations (CROs) and pharmaceutical companies can leverage these cutting-edge technologies to streamline clinical trials and introduce automation in drug discovery. As clinical trials grow in complexity, the volume of data being gathered and utilized for these studies is expanding.

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Clinical Trials Trials Clinical Research Treatment 52

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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The Pharma Data

JANUARY 3, 2021

United Kingdom Medicines and Healthcare Products Regulatory Agency authorized Clinical Trial Application. With these important regulatory clearances for our first-in-human clinical trial for INZ-701 in subjects with ENPP1 deficiency, we have transitioned from a research-stage to a clinical-stage company. “We

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Advarra

JUNE 20, 2023

Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community. More targeted therapies may change which patients and cancers may benefit, since a given treatment may only impact very specific biomarkers and genetic profiles.

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Trials Clinical Trials FDA Approval Therapies 52

The Pharma Data

DECEMBER 30, 2020

Axsome Therapeutics reported positive data from the long-term, open-label Phase III MOVEMENT trial of AXS-07 for the treatment of acute migraine. The MOVEMENT trial evaluated long-term safety of the drug for up to 12 months in patients with migraine attacks. The most common adverse events were nausea, dizziness, and vomiting.

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Trials Drugs Treatment 52

The Pharma Data

JANUARY 17, 2021

Aruvant has chosen Lonza to help develop and manufacture ARU-1801 for its upcoming pivotal trial. As we advance our gene therapy through clinical trials, this partnership signifies a critical milestone in the development pathway.” Once complete, Lonza will deliver cGMP material supply for the pivotal clinical trial.

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Conversations in Drug Development Trends

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

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Clinical Trials Trials Disease Drug Development 52

The Pharma Data

JANUARY 4, 2021

05, 2021 (GLOBE NEWSWIRE) — Polyphor AG (SIX: POLN) announced today that Gökhan Batur, Chief Executive Officer, will take part in a panel discussion at the LifeSci Partners 10th Annual Healthcare Corporate Access Event on Thursday, January 7 from 8:00AM Eastern Standard Time. Investors can pre-register for the event here.

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The Pharma Data

SEPTEMBER 6, 2021

The primary efficacy outcome was met showing a statistically significant reduction in the 24-hour cough count (average hourly cough frequency based on 24-hour sound recordings) of up to 27% for eliapixant 75 mg twice daily over placebo after 12 weeks of treatment.

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Trials Clinical Trials Treatment International 52

The Pharma Data

AUGUST 30, 2021

ALXN1840 demonstrated approximately three times greater copper mobilisation from tissues than standard-of-care treatments. The trial enrolled 214 patients, including treatment-naïve participants and those who have been on SoC therapy for an average of ten or more years.

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Disease Trials Therapies Treatment 52

The Pharma Data

JANUARY 13, 2021

Food and Drug Administration (FDA) has designated as a Fast Track development program the investigation of Brilacidin as a potential treatment for COVID-19. A molecular screening study of 11,552 compounds also supports Brilacidin as a promising novel coronavirus treatment. WAKEFIELD, Mass.,

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Clinical Trials Trials Treatment Pharmaceuticals 52

Advarra

OCTOBER 1, 2024

Managing clinical trial budgets efficiently is necessary for the success and sustainability of clinical research sites. Effective budget management not only ensures trials are financially viable but also maximizes return on investment (ROI). the impact and value of the data produced).

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Clinical Trials Trials Compliance Laboratories 52

Conversations in Drug Development Trends

DECEMBER 19, 2024

At the recent World Orphan Drug Congresses (WODC) in Europe and the United States, Worldwide Clinical Trials Derek Ansel , Vice President, Therapeutic Strategy Lead, Rare Disease, facilitated roundtable discussions to explore the operational challenges and ethical barriers surrounding genetic testing.

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Clinical Trials Regulations Drugs Trials 52

The Pharma Data

DECEMBER 29, 2020

Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy. galactosidase-A product candidate under development for the treatment of Fabry disease. Twenty of twenty-two patients completed the 12-month treatment duration. CARMIEL, Israel and BOSTON , Dec. nM and 13.81

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Clinical Trials Disease Treatment Trials 52

Drug Target Review

AUGUST 6, 2024

Autoimmune diseases : Rheumatoid Arthritis : ADCs targeting specific immune cells or inflammatory mediators can provide more precise treatment options with potentially fewer side effects. Obesity : By targeting adipose tissue or specific metabolic pathways, ADCs could offer new treatments for obesity and related metabolic disorders.

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The Pharma Data

AUGUST 16, 2021

Novartis today announced positive results from two Phase III clinical trials assessing Beovu ® (brolucizumab) 6 mg versus aflibercept 2 mg in patients with diabetic macular edema (DME). Both trials demonstrated an overall well-tolerated safety profile. for Beovu vs. 1.7% for Beovu vs. 1.7% for Beovu vs. 1.7%

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Trials Clinical Trials Treatment Disease 52

Advarra

FEBRUARY 24, 2023

But what does “working” really mean when efficacy means something subjective, like quality of life, reduced psychiatric events, or less symptoms? In most cases, the results you are really looking for and trying to prove in a trial can be complex. All key questions to consider and address before the therapy even leaves the lab.

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Clinical Trials Trials FDA Therapies 52

The Pharma Data

MARCH 8, 2021

Of 202 treated participants, no safety signals have been identified and of the 4 serious adverse events reported, none were considered to be study drug related. At a time where there is unmet need for antiviral treatments against SARS-CoV-2, we are encouraged by these preliminary data.”.

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