PPD

JANUARY 2, 2025

Its ability to maneuver persistent drug development challenges, like patient recruitment, trial complexity and rising costs, will ultimately determine its success. The biotech industry is a dynamic sector, rapidly evolving and poised for continued growth. FSP outsourcing is growing faster than FSO.

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The Pharma Data

JUNE 27, 2025

The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options.

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The Pharma Data

JUNE 13, 2025

Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies. Among patients who had previously undergone two or more lines of therapy (n = 12), the ORR was 92% and the CRR was 75%.

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New Drug Approvals

APRIL 11, 2025

2] History The efficacy and safety of fitusiran were assessed in two multicenter, randomized clinical trials which enrolled a total of 177 adult and pediatric male participants with either hemophilia A or hemophilia B. [2] 2] [3] Names Fitusiran is the international nonproprietary name. [4] Fitusiran 1711.0g/mol, Fitusiran 1711.0g/mol,

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PPD

DECEMBER 16, 2024

Cost and complexity go hand-in-hand The rising costs and growing complexity in clinical trials are deeply linked, with patient recruitment, extended timelines and meeting regulatory demands emerging as some of the key drivers. Nearly half (49%) of clinical trial sponsors surveyed identified rising costs as their foremost concern in 2024.

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PPD

JUNE 7, 2023

The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations. As therapies for the treatment of NASH in adult patients go this year to the U.S.

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PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

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The Pharma Data

JUNE 20, 2025

FDA Approves Dupixent as the First and Only Targeted Therapy for Adults with Bullous Pemphigoid In a groundbreaking development for patients suffering from a rare and debilitating autoimmune skin disease, the U.S. This has underscored the urgent need for non-immunosuppressive, targeted therapies with more favorable safety profiles.

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PPD

OCTOBER 31, 2024

From adapting to complex new trial designs to embracing cutting-edge technologies, staying ahead requires a deep understanding of the current landscape. Drug development The rising cost of clinical trials is the top challenge this year, due to increasingly complex protocol designs and difficult patient recruitment.

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Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. flash (powering the free Google App tier) and Claude Sonnet 3.5.

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Drug Discovery Today

NOVEMBER 10, 2022

A team of surgeons and scientists from the UK, Sweden and Canada, funded by Rinri Therapeutics, has confirmed secure surgical access to the central core of the human cochlea The research, published in Scientific Reports, is critical to the first in-human trials of new cell, gene and drug therapies for the inner ear, and will assist with treatment for (..)

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The Premier Consulting Blog

JANUARY 14, 2025

As the oncology landscape continues to evolve, the OCE aims to improve access to safe, effective, and cutting-edge therapies through streamlined regulatory processes and patient-centered approaches. These include Fast Track Designation, Breakthrough Therapy Designation, Priority Review Designation, and the Accelerated Approval Pathway (AAP).

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Conversations in Drug Development Trends

OCTOBER 29, 2024

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Previously, organizations had limited influence on the direction of basic research, clinical trial designs, and data ownership.

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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

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Conversations in Drug Development Trends

APRIL 3, 2024

By: Sue Batchelor, Executive Director, Oncology Project Management Radiopharmaceuticals represent a cutting-edge frontier in oncology treatment, offering the promise of highly targeted therapy with the potential to revolutionize cancer care. Are you aware of the challenges you must address for a successful radiopharmaceutical trial?

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The Pharma Data

JULY 3, 2025

The results, recently published in the International Journal of Molecular Sciences , come from a collaborative effort between researchers at Erlangen University Hospital and Berlin-based biotech firm AudioCure. The potential of AC102 to treat both hearing loss and tinnitus sets it apart from other investigational therapies.

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New Drug Approvals

JUNE 29, 2025

The resulting mixture was stirred at an internal temperature of about 65° C. was further added thereto, and then the resulting mixture was stirred at an internal temperature of 79° C. was added dropwise at an internal temperature of 22° C. The resulting mixture was stirred at an internal temperature of 19° C. for 4 hours.

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The Pharma Data

JUNE 18, 2025

Biogen Launches Global Phase 3 Pediatric Trial of Omaveloxolone for Friedreich Ataxia Biogen has officially launched its BRAVE study a pivotal, global Phase 3 clinical trial aimed at evaluating omaveloxolone in pediatric patients with Friedreich ataxia (FA). Head of the Neuromuscular Development Unit at Biogen.

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The Pharma Data

JUNE 22, 2025

The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C.,

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The Pharma Data

JUNE 22, 2025

In the trial, participants were directly switched to Mim8 without a washout period. Strong Safety Profile Reinforces Mim8’s Potential Mim8’s safety profile was a central focus of the FRONTIER5 trial. Throughout the 26-week study period, the investigational therapy was well-tolerated.

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The Pharma Data

JUNE 16, 2025

a principal investigator for the TRANSCEND FL study and a lymphoma and cell therapy specialist at Memorial Sloan Kettering Cancer Center in New York. This high durability highlights the sustained Benefit provided by liso-cel in a population typically faced with poor prognosis after multiple lines of standard therapy. months, and 24.5

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PPD

FEBRUARY 10, 2025

By harnessing the full range of innovative technologies and taking advantage of an FSP partners extensive skills and experience, sponsors are able to bring their therapies to market more quickly and within budget even in the face of complicated global regulations and widely fluctuating workloads.

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Drug Target Review

OCTOBER 30, 2024

Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first. Organoids will play increasingly crucial roles not only in research and diagnostics, but also in therapies to restore and enhance the human body.

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The Pharma Data

JUNE 24, 2025

The strategic funding arrangement is designed to support the continued advancement and global commercialization of Revolution Medicines’ RAS(ON) inhibitor portfolio, including its lead candidate daraxonrasib, as the company scales its operations independently across the international oncology market.

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SCIENMAG: Medicine & Health

JUNE 15, 2023

A new therapy is on the horizon for patients with metastatic colorectal cancer who have run out of treatment options. Credit: Vanderbilt University Medical Center A new therapy is on the horizon for patients with metastatic colorectal cancer who have run out of treatment options.

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Drug Target Review

DECEMBER 11, 2024

By employing advanced analytical techniques like high-resolution mass spectrometry (HRMS) and carefully calibrating systems with optimal internal standards (IS), we effectively mitigate matrix effects and provide reliable preclinical toxicology data. These solutions must contain analytes and internal standards at known concentrations.

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Conversations in Drug Development Trends

MAY 1, 2024

This process can be daunting, but understanding how to manage feedback effectively is crucial for developing and ultimately gaining approval for new therapies, especially in oncology clinical trials. Each nation has varying definitions of what qualifies as a pivotal study.

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The Pharma Data

JUNE 18, 2025

The study successfully met its primary endpoint, showing that significantly fewer patients treated with atogepant discontinued therapy due to AEs compared to those on topiramate. This underscores the need for more effective and tolerable options, as current therapies often fall short of achieving satisfactory outcomes or long-term adherence.

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New Drug Approvals

APRIL 2, 2025

The efficacy of Journavx was evaluated in two randomized, double-blind, placebo- and active-controlled trials of acute surgical pain, one following abdominoplasty and the other following bunionectomy. Both trials demonstrated a statistically significant superior reduction in pain with Journavx compared to placebo. under nitrogen.

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Advarra

MARCH 6, 2025

For more than 35 years, Advarra has been committed to protecting the rights and welfare of clinical trial participants while helping to improve healthcare outcomes, advancing medical knowledge, and bringing innovative, life-extending treatments to market that benefit millions of patients worldwide.

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The Pharma Data

JUNE 16, 2025

FDA Approves ANDEMBRY (garadacimab-gxii) The First Once-Monthly Prophylactic HAE Therapy Targeting Factor XIIa CSL a leading biotechnology company with a strong track record of developing innovative medicines for patients with rare and serious disorders, today announced that the U.S. when directly comparing ANDEMBRY to placebo.

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The Pharma Data

JUNE 6, 2025

Sanofi Highlights Robust Pipeline of Innovations in Rare Blood Disorders at ISTH 2025 Sanofi is poised to take center stage at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), set to be held in Washington, D.C., In addition, two poster presentations will offer deeper insights into the LUNA 3 trial.

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The Pharma Data

JUNE 24, 2025

To date, more than 70 joint research projects have been initiated, and over 10 co-authored publications have appeared in top-tier international academic journals. Oncology remains a core priority, reflecting the rising global burden of cancer and the need for more effective, targeted therapies.

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The Pharma Data

JUNE 19, 2025

Grifols’ BT524 Fibrinogen Therapy Shows Promise in Phase 3 Trial Published in The Lancet’s eClinicalMedicine Grifols , a globally recognized healthcare company and a leader in the production of plasma-derived therapies, has marked a significant advancement in the treatment of acquired fibrinogen deficiency (AFD).

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Drug Patent Watch

DECEMBER 9, 2024

This includes: Preclinical studies Clinical trials Regulatory approval Manufacturing and marketing Throughout this process, pharmaceutical companies seek to protect their investments through patents.

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The Pharma Data

DECEMBER 11, 2020

11, 2020 — Results from a long-term study of a gene therapy technique to prevent inherited mitochondrial disease show promise, researchers say. But they added a key caveat: The study found varying levels of mtDNA from the mother that replicated and built up within some internal organs. FRIDAY, Dec. in the small intestine.

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The Pharma Data

JULY 23, 2021

(Nasdaq: BIIB) today announced it will share multiple oral and poster presentations from its Alzheimer’s disease clinical development portfolio at the Alzheimer’s Association International Conference (AAIC), which will be held in Denver, Colorado and online July 26-30, 2021.

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