PPD

MARCH 11, 2025

Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.

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Conversations in Drug Development Trends

AUGUST 23, 2023

Patients are the backbone of clinical trials, playing an essential role in the drug development process. This engagement is often less understood and is underutilized by sponsors, meaning a significant element of the trial and drug experience is missed during sponsor engagement with the FDA.

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Conversations in Drug Development Trends

NOVEMBER 21, 2023

Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. As a provider of end-to-end clinical development solutions, we plan to leverage our expertise to refine drug-disease matches identified by Every Cure’s use of AI and data analysis.

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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

JANUARY 21, 2025

Regulatory bodies such as the FDA oversee clinical trials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. Any delays or missteps in bioanalysis during a Phase I trial can derail the trajectory of a promising drug.

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The Pharma Data

DECEMBER 14, 2020

The trial, which will explore the pharmacokinetics and safety of ATX01 in healthy volunteers, is due to start in January 2021. AlgoTherapeutix recently raised a 12M€ Series A that will fund the Phase 1 and 2 clinical development of ATX01. CIPN is a leading cause for modification or interruption of chemotherapy.

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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.

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PPD

NOVEMBER 21, 2023

To keep a pulse on this rapidly changing industry, the PPD clinical research business of Thermo Fisher Scientific surveyed 150 leaders in the biopharma and biotech industries for the second consecutive year. Differences in size, resources and agility have led the two groups to take different approaches to clinical development.

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PPD

NOVEMBER 9, 2023

Instead of the standard approach where studies are designed to address a primary focus, a parallel study method — a format that incorporates multiple endpoints and objectives, modifications, treatment arms and dose selections into a single protocol — is being rapidly adopted.

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SugarCone Biotech

MARCH 2, 2025

Bicara has focused ficerafusp alfa clinical development on head and neck squamous cell carcinoma (HNSCC), adding pembrolizumab to separately block the PD-1 pathway. No chemotherapy regimen is used in the trial. Another clinical program that has making headlines in HNSCC is petosemtamab, an anti-EGFR x LGR5 bispecific antibody.

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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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ASPET

FEBRUARY 26, 2024

FXR-targeted drugs have shown promise in late-stage clinical trials for non-alcoholic steatohepatitis. Cilofexor is one of few remaining FXR agonists in clinical development. The farnesoid X receptor (FXR) is a nuclear receptor that controls bile acid, lipid, and cholesterol metabolism.

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H1 Blog

JUNE 7, 2023

Clinical trial success is a key factor for pharma companies when designing and recruiting patients into trials. This data can also provide guidance in terms of diversifying clinical trials to include different types of population demographics such as age, gender, race, ethnicity, or geographic location.

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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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Conversations in Drug Development Trends

DECEMBER 5, 2023

Author: Lona Sheeran, SVP, Clinical Operations, Early Phase At this year’s Clinical Trials Nexus, I had the privilege of representing Worldwide Clinical Trials as the sole CRO on a panel discussion: “Reversing the Conversation: What the Clinical Trial Industry Really Wants from its Service Providers.”

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DrugBank

JUNE 6, 2024

In the rapidly evolving landscape of oncology drug development, artificial intelligence (AI) has emerged as a revolutionary force, altering not just the pace but the very methodology of discovery and development.   What sets RADR ® apart is its robust data-driven approach.

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Cytel

JUNE 18, 2024

External Control Arms (ECAs) provide comparative evidence when recruiting patients is difficult or unethical in randomized controlled trials. ECAs have significant potential to save resources and accelerate access to innovative treatment.

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The Premier Consulting Blog

OCTOBER 21, 2024

However, accelerated approval comes with a condition: sponsors must conduct confirmatory trials to verify the drug’s anticipated clinical benefits using robust outcome measures. Concerns have arisen over delays—sometimes spanning over 7–8 years—that may expose patients to risks before confirmatory trials are completed.

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Conversations in Drug Development Trends

MAY 14, 2024

As the strategists for Worldwide Clinical Trials’ rare disease sponsors, we design customized solutions that make a difference in achieving successful outcomes. This philosophy is encapsulated in our belief: “If you’ve seen one trial for a rare indication, you’ve seen one trial.”

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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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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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The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. Kimberly Smith, M.D., About the long-acting regimen of cabotegravir and rilpivirine.

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Advarra

JUNE 20, 2023

Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54 Typical clinical development timelines for anticancer drugs average an estimated 6.7

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Cytel

JANUARY 23, 2023

This has aligned with the broader curation of a number of new dose-escalation and efficacy designs for clinical development in oncology. A nuanced understanding of these considerations enables sponsors to ask more complex questions like:

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The Pharma Data

AUGUST 31, 2021

In addition, as part of the label update, this indication has been revised to be for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for any platinum-containing chemotherapy. Source link: [link].

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The Pharma Data

DECEMBER 21, 2020

The trial will be conducted at 10 sites across Germany and led by principal investigator Prof. The FOCUS trial is Ultimovacs’ fourth Phase II clinical trial with UV1 and comes in addition to the collaboration Phase II trial announced in May 2020. “We Mascha Binder, M.D.,

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The Pharma Data

JANUARY 13, 2021

Food and Drug Administration (FDA) has granted Orphan Drug Designation for HPN217 for the treatment of multiple myeloma. Harpoon has four drug product candidates in clinical development for the treatment of solid and hematologic malignancies based on its proprietary TriTAC platform. “I NYSE: ABBV).

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The Pharma Data

MARCH 8, 2021

We are very pleased to share our initial Phase 2 infectivity data at this important conference, which remains at the forefront for critical clinical scientific information in infectious diseases,” shared Dr. Wendy Painter, Chief Medical Officer of Ridgeback Biotherapeutics. “At About Molnupiravir.

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KIF1A

SEPTEMBER 30, 2023

Read the Article Both of these studies reflect a global effort to understand the genetic underpinnings of disorders so people can find the right treatments sooner. In addition to benefiting awarded sponsors, this pilot program will help create best practices of communication with the FDA during rare disease therapeutic development.

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The Pharma Data

JANUARY 13, 2021

14, 2021 (GLOBE NEWSWIRE) — Innovation Pharma (Formerly known as Cellceutix) (OTCQB:IPIX) (“the Company”), a clinical stage biopharmaceutical company, is pleased to announce that the U.S. A molecular screening study of 11,552 compounds also supports Brilacidin as a promising novel coronavirus treatment. WAKEFIELD, Mass.,

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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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The Pharma Data

APRIL 4, 2022

The high rate of unvaccinated people will continue to put a strain on hospitals and healthcare systems around the world, furthering the need for effective treatments for patients hospitalised with COVID-19,” said Levi Garraway, M.D., Chief Medical Officer and Head of Global Product Development, Roche. Actemra/RoActemra is not U.S.

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The Pharma Data

AUGUST 24, 2020

It will investigate the safety, measure key biomarkers and assess surrogate markers of efficacy in its AGT103-T treatment. The treatment is being researched by scientists collaborating from American Gene Technologies, the Laboratory of Immunoregulation and the National Institute of Allergy and Infectious Diseases.

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The Pharma Data

NOVEMBER 29, 2020

Prior to joining Gannex, Dr. Palmer was Head of Liver Disease Clinical Development at Takeda Pharmaceuticals. She has also held senior leadership positions at Shire Pharmaceuticals and Kadmon Corporation, where she was in charge of the global development of NASH and other liver disease programs. ” About Ascletis.

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Broad Institute

NOVEMBER 2, 2023

Bayer’s established collaboration with the Broad Institute has already resulted in three clinical oncology candidates over the past decade. We look forward to continuing our work with renowned Broad scientists to advance additional innovative cancer targets into clinical development.”

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Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer.

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The Pharma Data

MAY 8, 2021

Sandoz, a Novartis division, today announced progress in the late-stage clinical development program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. Aflibercept is a key treatment in ophthalmology.

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