March, 2024

article thumbnail

Amylyx ALS drug fails crucial study, putting company’s future in doubt

Bio Pharma Dive

The results have led Amylyx to pause promotion of Relyvrio and potentially pull it from the market in the coming weeks, a major blow to the company and ALS patients.

Drugs 342
article thumbnail

March 7, 2024: New Report Sets Out Posttrial Responsibilities in Pragmatic Clinical Trials

Rethinking Clinical Trials

Dr. Stephanie Morain In a new report from the NIH Pragmatic Trials Collaboratory, a team of bioethicists and implementation scientists argue for a “presumptive default” that the results of pragmatic clinical trials should be incorporated into healthcare delivery processes. This responsibility arises from a key rationale for conducting pragmatic trials: that they can facilitate uptake of their results by relevant decision-makers.

Insiders

Sign Up for our Newsletter

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

article thumbnail

What’s next for AAV gene therapies in 2024?

Pharmaceutical Technology

Upcoming regulatory events for pipeline AAV gene therapies, with a focus on neuromuscular and blood disorders, are setting high expectations for the space in 2024.

article thumbnail

BioNTech hit with 'notice of default' from NIH in COVID-19 vaccine royalty dispute

Fierce Pharma

Even as Germany's BioNTech deals with ongoing declines of its revenue and share price, it's facing another serious concern: U.S. | Even as Germany's BioNTech deals with ongoing declines of its revenue and share price, it's facing another serious concern: U.S. officials are pressing the company to pay royalties linked to the commercialization of its lucrative Pfizer-partnered COVID-19 vaccine.

article thumbnail

The New Age of Decentralized Clinical Trials

White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations

article thumbnail

FDA Approves Madrigal’s Rezdiffra as First MASH Therapy

BioSpace

Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) is the first-ever approved therapy for metabolic dysfunction-associated steatohepatitis—a decision experts say could signal a sea change in treatment of the disease.

article thumbnail

Nanoscope preps filing for retinitis pigmentosa gene therapy

pharmaphorum

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of underlying genetic mutations.

More Trending

article thumbnail

New wave of potential therapies to bring ‘paradigm shift’ in COPD treatment

BioPharma Reporter

The recent submission of clinical trial data for Sanofi/Regeneronâs Dupixent and Veronaâs Ensifentrine to the U.S. FDA indicates the entry of new therapies for chronic obstructive pulmonary disease (COPD) in 2024.

article thumbnail

Akums introduces Hydroxyurea oral suspension for sickle cell disease

Pharmaceutical Technology

Akums Drugs and Pharmaceuticals has introduced Hydroxyurea oral suspension, a room temperature-stable drug for treating sickle cell disease.

Drugs 306
article thumbnail

With Pfizer struggling in 2023, CEO Bourla hit with 35% pay cut to $21.6M

Fierce Pharma

Pfizer knew 2023 was going to be a challenging year of transition. But even the drugmaker was surprised by the plummeting demand for its COVID-19 products, missing badly on its 2023 guidance. | After a difficult year for Pfizer, in which its share price fell by 44%, CEO Albert Bourla’s compensation fell by 35% from $33 million in 2022 to $21.6 million in 2023.

article thumbnail

Epigenetic Editing Explodes on the Heels of Gene Editing Success

BioSpace

Ubiquitous potential, possible safety advantages and the recent growth of cell and gene therapy are driving investment in a different type of genetic editing.

article thumbnail

Roles and Responsibilities of Specialized Clinical Supply Experts

When selecting a clinical supply provider, consideration often focuses upon the manufacturing, packaging, storage and distribution capabilities available that will, at face-value, be sufficient to meet the needs of the sponsor and their trial. However, there are human-based and knowledge-driven factors that are often overlooked that go beyond these basic physical capabilities and are integral to the development and delivery of high performing clinical supply chains.

article thumbnail

Cannabinoids show promise in acute migraine clinical trial

pharmaphorum

Inhaled cannabinoids have been shown to perform better than placebo in providing pain relief for people suffering from acute migraine in a clinical trial

article thumbnail

Biotech stock fundings headed for best quarter in 3 years, Jefferies says

Bio Pharma Dive

Publicly traded companies raised nearly $10 billion in follow-on stock offerings in January and February, a financing surge that’s driving a “sector recovery,” the investment bank said.

320
320
article thumbnail

Antidote anecdotes: A conversation with an MS care partner

Antidote

MS is a chronic disease that is estimated to impact over a million Americans. The condition causes damage to the body’s central nervous system, leading to numbness, memory problems, sight issues, and difficulties with coordination.

105
105
article thumbnail

Oncology emerges as dominant therapy area for CRISPR technology

Pharmaceutical Technology

In late 2023, the Medicines and Healthcare products Regulatory Agency approved Vertex’s Casgevy, which became the inaugural clustered regularly interspaced short palindromic repeats (CRISPR)-based drug for the haematological indications beta thalassemia and sickle cell disease (SCD).

Medicine 278
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

BioNTech's shares tumble—again—amid continued COVID vaccine sales slide

Fierce Pharma

Four months ago, when BioNTech slashed its 2023 revenue projection from 5 billi | When BioNTech reported its fourth-quarter earnings Wednesday, it was another harsh reminder of the plummeting demand for COVID-19 vaccines. Sales came to 1.5 billion euros ($1.6 billion) for the fourth quarter and 3.8 billion euros ($4.1 billion) for 2023, coming up short of expectations.

Sales 132
article thumbnail

AstraZeneca’s Ultomiris Wins FDA Approval for Rare Autoimmune Disease

BioSpace

AstraZeneca’s Alexion on Monday secured the fourth indication for Ultomiris, which can now be used to treat the rare autoimmune condition neuromyelitis optica spectrum disorder.

article thumbnail

AI in Drug Discovery: Day One

pharmaphorum

There is no denying that artificial intelligence (AI) has captured the imagination of innovators working across the pharmaceutical sector. The drug discovery space is no different, with myriad applications promising to revolutionise the way that researchers and sponsors approach identifying and progressing new drug candidates.

Drugs 117
article thumbnail

FDA clears first-of-its-kind Duchenne drug for broad use

Bio Pharma Dive

The approval of Italfarmaco’s Duvyzat is the first for an oral, non-steroidal Duchenne drug and was issued for all patients at least six years of age, regardless of their genetic underpinnings.

Drugs 293
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

Pain is reduced by almost half with cannabis-based medicines – report finds

Outsourcing Pharma

On March 7, Celadon Pharmaceuticals plc, a leading UK-based pharmaceutical company specializing in cannabis-based medicines, unveiled promising findings from an early economic analysis of its chronic pain clinical trial, dubbed CANPAIN.

Medicine 124
article thumbnail

Bionomics to advance non-SSRI PTSD candidate to Phase III

Pharmaceutical Technology

Bionomics plans to meet with the FDA following positive topline data from the Phase IIb ATTUNE trial of the post-traumatic stress disorder (PTSD) drug BNC210.

Trials 262
article thumbnail

FDA approves first MASH drug: Madrigal's Rezdiffra breaks ground in notorious biopharma graveyard

Fierce Pharma

The decades-long wait for an effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) has ended, as the FDA has approved the first drug for the fatty liver disease. | The decades-long wait for an effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) has ended. The FDA has approved Madrigal's resmetirom under the brand name Rezdiffra as the first drug for the fatty liver disease.

article thumbnail

Boundless Bio Launches $100M IPO to Advance Cancer Therapy Candidates

BioSpace

Continuing 2024’s biotech initial public offering rally, Boundless Bio will debut Thursday on the Nasdaq with the proceeds used to advance its pipeline of extrachromosomal DNA cancer assets.

DNA 116
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

Navigating digital transformation in pharma: The rise of mobile messaging

pharmaphorum

Popular mobile messaging apps that offer end-to-end encryption for their users' communications means that the content of messages is only accessible to the sender and the recipient. However, like any software, they still have potential cybersecurity risks and compliance concerns that users should be aware of - especially in pharma.

article thumbnail

Madrigal, FDA approval in hand, outlines plan to sell MASH drug

Bio Pharma Dive

The company expects initial uptake of Rezdiffra, which costs $47,400 per year, to be slow as doctors and insurers establish the protocols needed to find patients eligible for treatment.

article thumbnail

Researchers suggest treatments for rare diseases could reduce burden of kidney disease

Pharma Times

Rare kidney diseases are a group of conditions which account for over 5% of people living with chronic kidney disease

Research 143
article thumbnail

AD/PD 2024: TauRx’s HMTM promising despite lacking primary endpoint data

Pharmaceutical Technology

Participants cannot remain blind to their treatment arm due to the properties of TauRx's drug, which causes urinary discoloration.

Drugs 299
article thumbnail

An Innovative & Creative Problem Solver Approach to Selling in the Medical Device Space

Speaker: Steve Goldstein, Sales Leader

Are you currently in sales, or involved in a business that depends on strong sales results? What about the extremely competitive world of medical device sales? What are some of the top challenges your customers face and how do you approach understanding what’s most important to them? Join Steve Goldstein, Sales Success Coach, Motivational Speaker and Medical Device Sales Leader from Gold Selling LLC., to discover critical strategies and approaches you can take to engage your customers, achieve g

article thumbnail

AstraZeneca pledges £650M in UK investments to boost vaccine capabilities, expand near HQ

Fierce Pharma

AstraZeneca CEO Pascal Soriot, who has been critical of the U.K.’s business environment for the life sciences industry, is directing a big chunk of money into the Big Pharma’s home country. | AstraZeneca CEO Pascal Soriot, who has been critical of the U.K.’s business environment for the life sciences industry, is directing £650 million into the Big Pharma’s home country.

article thumbnail

FDA Authorizes Invivyd’s Pemgarda to Prevent COVID-19 in Immunocompromised People

BioSpace

The regulator has allowed for emergency use of Invivyd’s monoclonal antibody Pemgarda as a COVID-19 pre-exposure prophylaxis for moderately or severely immunocompromised patients.

Antibody 130
article thumbnail

Employee experience in healthcare: The importance of EX for patient experience

pharmaphorum

Understanding the employee experience in healthcare is essential for improving the patient experience. Learn more about the importance of employee experience in healthcare and how it impacts patient care.

119
119
article thumbnail

After surprise trial failure, ALS doctors brace for one less treatment option

Bio Pharma Dive

Physicians are preparing for tough conversations with patients on Amylyx Pharmaceuticals’ drug, Relyvrio, while holding out hope it still may help some with the disease.

Doctors 306
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.