Sat.Nov 25, 2023 - Fri.Dec 01, 2023

article thumbnail

Pharma benefited from basing business overseas. An international tax effort could spur a rethink.

Bio Pharma Dive

U.S. tax law changes enacted six years ago slashed large pharma companies' rates and saved them billions. Now, a push for an international floor could disrupt their R&D accounting.

article thumbnail

Acelyrin claims errors by CRO Fortrea led to late-stage trial downfall

Pharmaceutical Technology

A Phase IIb/III trial studying the company’s immunotherapy izokibep, failed to achieve statistical significance for its primary endpoint

Trials 243
Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

By: Amy Raymond, PMP, PhD, Senior Director, Therapeutic Strategy Lead, Cellular & Genetic Medicines On November 16, the British regulatory body, Medicines and Healthcare products Regulatory Agency (MHRA) approved exagamglogene autotemcel (exa-cel) to treat severe sickle cell disease for patients ages 12 and up, becoming the first gene editing treatment to receive regulatory approval.

article thumbnail

November 30, 2023: HCSRN Calls for Abstracts and Panel Presentations for 2024 Annual Conference

Rethinking Clinical Trials

The Health Care Systems Research Network (HCSRN) is accepting abstract submissions and panel presentation submissions for its 2024 Annual Conference until December 11, 2023. This year’s meeting will be held in Milwaukee, Wisconsin, from April 9 to 11, 2024. The HCSRN is a 20-member research network focused on supporting research institutes aligned with healthcare delivery systems.

article thumbnail

The New Age of Decentralized Clinical Trials

This new white paper defines and details the impact of Decentralized Clinical Trials on the Pharmaceutical industry and how the impact can be measured along with steps companies can take to ensure adoption.

article thumbnail

GSK cancer drug Blenrep gets surprise trial win

Bio Pharma Dive

One year after the U.K. drugmaker withdrew the multiple myeloma drug from the U.S. market because of negative data, new study results might crack open the door to a relaunch.

Drugs 280
article thumbnail

Aro Biotherapeutics raises funds for Pompe disease treatment

Pharmaceutical Technology

Aro Biotherapeutics has raised $41.5m in a Series B financing round to advance the development of ABX1100 for Pompe disease.

More Trending

article thumbnail

November 28, 2023: Workshop Summary Now Available From ‘Getting the Right Evidence to Decision-Makers Faster’

Rethinking Clinical Trials

The workshop summary is now available from the NIH Pragmatic Trials Collaboratory’s recent workshop, “Getting the Right Evidence to Decision-Makers Faster.” The 2-day workshop explored the critical cycle of evidence generation by researchers to decision-making by healthcare system leaders to implement the findings of pragmatic clinical trials conducted within healthcare systems.

article thumbnail

FDA names chief scientist Bumpus as Woodcock’s successor

Bio Pharma Dive

Bumpus, a former Johns Hopkins professor, named “creating a new model” for the FDA’s Office of Regulatory Affairs as one of her priorities when she steps into the role.

Scientist 246
article thumbnail

VPAS is dead, long live VPAG—could the UK’s new clawback revision save the day?    

Pharmaceutical Technology

After months of negotiations, the UK's DHSC and the Association of the British Pharmaceutical Industry emerged with a plan on to replace VPAS.

Marketing 221
article thumbnail

FDA investigates 'serious risk' of secondary cancer following CAR-T treatment

Fierce Pharma

In a blow to CAR-T therapies, the FDA is investigating a “serious risk” of patients developing new cancers after treatment with these highly efficacious oncology drugs. | In a blow to CAR-T therapies, the FDA is investigating a “serious risk” of patients developing new cancers after treatment with these highly efficacious oncology drugs.

Drugs 143
article thumbnail

Commercial Drug Sourcing for Clinical Trials

The sourcing of commercial drugs for use in clinical trials either as a comparator, rescue or co-medication can be a complex process. Sponsors can face a number of challenges when trying to obtain these critical supplies for their studies, including addressing product lead times, availability, expiry limitations, safety and cost, among others. Choosing the right partner with global sourcing, regulatory, quality and clinical packaging expertise can provide the necessary guidance to help sponsors

article thumbnail

November 29, 2023: Special Biostatistics Series Continues With Guidelines for Stepped-Wedge Trials

Rethinking Clinical Trials

In this Friday’s PCT Grand Rounds, Jim Hughes of the University of Washington will continue our special series, Advances in the Design and Analysis of Pragmatic Clinical Trials, with his presentation, “Guidelines for Design and Analysis of Stepped-Wedge Trials.” The session will be held on Friday, December 1, at 1:00 pm eastern. Hughes is a professor emeritus of biostatistics at the University of Washington.

Trials 147
article thumbnail

BioMarin secures hemophilia gene therapy coverage in Germany

Bio Pharma Dive

Drawn-out negotiations led to a lower price than initially expected, but analysts called the agreement a step forward for the biotech company.

article thumbnail

FDA accepts Orexo’s NDA for opioid overdose medication

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has accepted to review Orexo’s new drug application (NDA) for opioid overdose medication, OX124.

Drugs 233
article thumbnail

Pfizer takes Poland to court over alleged missed COVID-19 vaccine payments

Fierce Pharma

As the pandemic eased earlier this year, officials in Poland became vocal critics of the financial obligations laid down by Pfizer’s large vaccine supply pact with the European Union. | As the pandemic eased earlier this year, officials in Poland became vocal critics of the financial obligations laid down by Pfizer’s large vaccine supply pact with the European Union.

article thumbnail

How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

article thumbnail

November 27, 2023: NIH Announces Funding Opportunity for Implementation Studies of Substance Use Treatment and Prevention in Populations That Experience Health Disparities

Rethinking Clinical Trials

The National Institutes of Health’s Office of Disease Prevention (ODP) issued a new funding opportunity to support implementation studies in treatment and prevention for alcohol, tobacco, and other drugs use and misuse in adult populations that experience health disparities. ODP and participating NIH Institutes and Centers are inviting applications for research projects that test innovative approaches to implementing screening, brief intervention, and referral to treatment or prevention fo

article thumbnail

Roivant’s Matt Gline on raising capital and biotech’s outlook in 2024

Bio Pharma Dive

Fresh off a $7 billion deal with Roche, the chief executive spoke with BioPharma Dive about this year’s “discordant combination” of major biotech achievements alongside market headwinds.

Marketing 187
article thumbnail

FDA fast-tracks Genelux’s oncolytic virus-based ovarian cancer therapy

Pharmaceutical Technology

Olvi-Vec is currently being evaluated in a Phase III trial as a treatment for platinum- resistant/refractory ovarian cancer.

Trials 246
article thumbnail

Amgen expands pact with Amazon to usher drug manufacturing into the AI era

Fierce Pharma

Seeking to identify ways to improve the discovery and production of medicines, Amgen is expanding its partnership with tech giant Amazon. | Amgen is growing its decade-old collaboration with Amazon Web Services—a widely used cloud platform—to create generative artificial intelligence that the companies aim to use to increase the manufacturing throughput of pharmaceuticals.

article thumbnail

What the FDA's New Dosage Guidance Means for the Future of Clinical Research

Speaker: Dr. Ben Locwin - Biopharmaceutical Executive & Healthcare Futurist

What will the future hold for clinical research? A recent draft from the FDA provides valuable insight. In "Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases," the FDA notes that "targeted therapies demonstrate different dose-response relationships compared to cytotoxic chemotherapy, such that doses below the Maximum Tolerated Dose (MTD) may have similar efficacy to the MTD but with fewer toxicities.

article thumbnail

BMS Expands Cardio Partnership with Avidity in Potential $2.3B Deal

BioSpace

Building on a previous 2021 collaboration, Bristol Myers Squibb is paying $100 million upfront for the development of five cardiovascular targets utilizing Avidity Biosciences’ antibody oligonucleotide conjugates.

Antibody 124
article thumbnail

FDA investigating cancer risk linked to CAR-T cell therapy

Bio Pharma Dive

The agency said the benefit of approved treatments like Gilead’s Yescarta still outweighs any such risk, but the alert could slow drugmaker efforts to develop the treatments for wider use.

article thumbnail

Where do anti-tumor necrosis factor like cytokine agents go now?

Pharmaceutical Technology

In the past few months, significant interest within the inflammatory bowel disease (IBD) space has been directed towards the therapeutic target, tumour necrosis factor-like cytokine 1A (TL1A).

130
130
article thumbnail

Biden admin urges Supreme Court to nix mifepristone restrictions

Fierce Pharma

Following similar campaigns mounted by industry players and biopharma trade groups, the United States' Solicitor General has joined a chorus of voices calling on the Supreme Court to overturn an ap | Following similar campaigns mounted by industry players and biopharma trade groups, the United States' Solicitor General has joined a chorus of voices calling on the Supreme Court to overturn an appeals court’s August ruling on the abortion pill mifepristone.

128
128
article thumbnail

2022 Research: The Rapid Rise of Ocean Freight Visibility

A research study conducted by The Journal of Commerce and FourKites surveyed hundreds of international shippers, exploring how their usage of global supply chain visibility technology has evolved since the onset of global disruptions caused by COVID-19. For international shippers, ocean freight visibility has evolved from optional to essential and satisfaction with visibility varies greatly depending on how it is obtained and delivered.

article thumbnail

Karuna Secures FDA Decision Date for Schizophrenia Candidate KarXT

BioSpace

The regulator accepted Karuna Therapeutics' NDA for KarXT and set a PDUFA date of September 26, 2024. If approved, it would be the first new mechanism of action to treat schizophrenia in decades, the company contends.

article thumbnail

AbbVie joins ADC dealmaking with $10B ImmunoGen buyout

Bio Pharma Dive

The acquisition of ImmunoGen gives AbbVie an approved medicine for ovarian cancer, and continues a pharma spending spree on antibody-drug conjugates.

Antibody 154
article thumbnail

Cyclin Dependent Kinase 4 drugs in development, 2023

Pharmaceutical Technology

GlobalData’s report assesses the drugs in the Cyclin Dependent Kinase 4 pipeline by therapy areas, indications, stages, MoA, RoA, molecule type and the key players in the development pipeline.

Drugs 130
article thumbnail

Scottish stem cell pro RoslinCT inks deal to produce Vertex and CRISPR's Casgevy

Fierce Pharma

Following the historic approval of the world’s first CRISPR-based gene-editing therapy in the United Kingdom this month, Vertex Pharmaceuticals has tapped its long-time manufacturing partner Roslin | RoslinCT, the CDMO arm of the Roslin Institute, is set to manufacture Vertex and CRISPR Therapeutics’ exa-cel “across the world” as part of Vertex’s global production network.

article thumbnail

Making the Case for Case Management for Cell and Gene Therapies

As demand for advanced therapies increases, so does the need for more specialized supply chain support, as these products have strict transportation and handling requirements. Deviations from the intended supply chain can delay timelines and affect these high value products, which can be detrimental to budgets, but more importantly can have dire effects on the patients whose lives depend on receiving these critical doses safely and on time.

article thumbnail

Legalized medical cannabis five years on - Releaf discusses how far the industry hasn't come

BioPharma Reporter

It is five years this month since a ground-breaking change in UK healthcare history happened â with doctors being able to prescribe cannabis-based medicines for the first time.

Medicine 123
article thumbnail

Regeneron, Sanofi to seek new Dupixent approval in US after COPD success

Bio Pharma Dive

Results from the second of two Phase 3 trials testing the blockbuster drug in the respiratory condition showed treatment could reduce attacks and improve lung function.

Drugs 154
article thumbnail

Bromodomain Containing Protein 4 drugs in development, 2023

Pharmaceutical Technology

GlobalData’s report assesses the drugs in the Bromodomain Containing Protein 4 pipeline by therapy areas, indications, stages, MoA, RoA, molecule type and the key players in the development pipeline.

Protein 130
article thumbnail

SpringWorks Wins First FDA Approval for Non-Cancerous Desmoid Tumors

BioSpace

The Ogsiveo tablets are the first approval therapy for the rare subtype of soft tissue sarcomas, which can lead to severe pain and disability and previously were treated primarily through surgery.

article thumbnail

Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.