Drug Discovery and Development Archives - Page 10 of 158

Evidence on GLP-1 drugs and muscle loss remains murky

Recent research on glucagon-like peptide-1 (GLP-1) drugs for weight loss indicates these medications may lead to significant reductions in lean body mass, including muscle. The STEP 1 and SUSTAIN 8 of semaglutide trials found 39–40% of weight lost on the GLP-1 drugs was lean mass. A 2021 meta-analysis of 18 randomized controlled studies showed significant drops in fat-free mass with GLP-1 drugs, including oral and subcutaneous semaglutide and older GLP-1 drugs such as lixisenatide, exenatide, and liraglutide. The meta-analysis focused on both GLP-1 receptor agonists and sodium-glucose cotransporter (SGLT-2) inhibitors.

Sizing up the GLP-1 impact on lean mass: Mixed signals

While some data suggests that GLP-1 drugs are associated with muscle loss, there may be more to the story than meets the eye. In STEP 1 and SUSTAIN 8, the proportion of lean mass to total body mass still increased overall, pointing to an overall improvement in body composition. And data from the …

Tirzepatide beats semaglutide 3-to-1 for weight loss goals in real-world data

A new real-world study (pre-print) is the first to directly compare weight loss outcomes between the popular diabetes medications Lilly’s Mounjaro (tirzepatide) and Novo Nordisk’s Ozempic (semaglutide). The results show Mounjaro users are significantly more likely to achieve meaningful weight loss.

Analyzing data from more than 40,000 patients from a large U.S. health database, the researchers found those taking Mounjaro were nearly three times more likely to lose 10% of their body weight compared to Ozempic users. Additionally, they were also almost twice as likely to lose 5% of their weight, and three times more likely to lose 15%.

Weight Loss Thresholds Comparison google.charts.load('current', {'packages':['corechart']}); google.charts.setOnLoadCallback(drawChart); function drawChart() { var data = google.visualization.arrayToDataTable([ ['Drug', '≥5% Weight Loss', '≥10…

Organon reinforces commitment to women’s health beyond reproductive health

The fact that women’s health has long been underprioritized is common knowledge. Organon, with roots going back to 1923 and having sharpened its focus on women’s health post its 2021 spinoff from Merck & Co., aims to address significant gaps in treatment and care. “Our commitment to women’s health is not just a part of our corporate strategy; it’s at the core of who we are as a company,” said Dr. Juan Camilo Arjona Ferreira, chief medical officer at the company, in a recent interview.

Organon sees women’s health as encompassing more than just reproductive issues. “It’s a continuum that spans a woman’s entire life,” said Arjona, a gynecologist by training. “There’s plenty of opportunities across the board because women’s health has been understudied and underappreciated.”

Organon’s approach to women’s health challenges

Dr. Juan Camilo Arjo…

Nvidia-Genentech AI drug discovery alliance unites computing brawn with biological brains

NVIDIA BioNeMo AI molecular modeling software can uncover complex biochemical interactions through AI-driven molecular modeling techniques. [Image courtesy of NVIDIA]

Technically, graphics processing and AI hardware powerhouse Nvidia is also a drug discovery company. It may not discover drugs in-house, but it has developed BioNeMo, a comprehensive generative AI platform for drug discovery and Clara, a collection of healthcare frameworks, applications, and tools, including for biopharma. Nvidia partners include Amgen, AstraZeneca, GSK and Insilico Medicine.

Similarly, biotech pioneer and Roche subsidiary Genentech is also an AI company. It has experience in applying machine learning to an array of disease areas, and has extensive biological and molecular datasets and research capabilities. Its initiatives include alliance with firms such as Recursion Pharmaceuticals and Reverie Labs that focus on using AI for nov…

Nipocalimab shows promise in RA subgroups in phase 2a IRIS-RA study

Johnson & Johnson’s nipocalimab, which works by targeting the neonatal Fc receptor (FcRn), has the potential to treat an array of autoimmune conditions. But the antibody recently hit a snag in the first-ever clinical study of an FcRn inhibitor in rheumatoid arthritis (RA), missing its primary endpoint. The development has sparked debate within the rheumatology community. “Now, there are some highly respected physicians who’ve said, ‘This is a failed trial,’” said Dr. Peter Taylor, the Norman Collisson Professor of Musculoskeletal Sciences at the University of Oxford. “I think that’s an incorrect interpretation.”

Johnson & Johnson is also pursuing a host of other autoimmune indications for nipocalimab. The FDA has granted it Fast Track designation for hemolytic disease of the fetus and newborn (HDFN) and orphan drug status for HDFN while EMA granted it orphan medicinal product designation for HDFN.

Breaking down the data from IRIS-RA

Johnson …

From Silicon Valley to the lab of tomorrow: Synfini’s leap to large chemistry models

Conceptual visualization of a molecular structure, not representative of a specific molecule. [Image courtesy of Adobe Stock]

During the COVID-19 pandemic in late 2020, the storied Silicon Valley institution SRI International secured a $4.3 million DARPA contract to develop a tool for generating therapeutic small molecules to combat biological threats. Not just known for innovations like the computer mouse and Siri, SRI International is also responsible for Synfini, a multimodal chemistry model akin to large language models like ChatGPT. In September 2023, the company spun out Synfini as an independent entity.

“The core group and project started about seven years ago at SRI International, and began as part of the DARPA Make-It program,” said Peter Madrid, co-founder and head of scientific development at Synfini. DARPA’s Make-It initiative aimed to automate small molecule discovery and synthesis. Thanks to the su…

Vaccine mega-trials: Rare behemoths in the vaccine trial landscape

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Abstract

The vast majority of vaccines are prophylactic in nature. As a result, the demonstration of their efficacy paradoxically requires the infectious disease to occur among non-diseased study participants randomized between investigational vaccine and appropriate control groups. The statistics of vaccine efficacy (VE) calculation are nearly entirely and solely based on the number of observed incident disease cases during follow-up. For certain diseases, the sample sizes needed to achieve the required number of observations may reach 5,000 subjects or many more, requiring vaccine mega-trials to be conducted. In this article, the author provides an overview of mega vaccine trials conducted over the last 20 years.

Introduction

Prophylactic vaccines generally are intended for healthy or medically stable populations. Administration to hundreds of thousands or even millions of people in a few yea…

Cybin sees near future for psychedelic therapy after promising interim phase 2 data

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With promising interim phase 2 data in hand, Cybin believes psychedelic therapy will become a reality in the “not too distant future,” according to CEO Doug Drysdale.

As recently as the 1990s, it would be difficult to imagine that a psychedelic drug would potentially be a clinical option for a mood disorder like depression. But before long, data from multiple phase 3 trials will be available on psilocybin derivatives for various mood disorders.

Interim phase 2 CYB003 data as a ray of hope for treating mood disorders

Toronto, Canada–based Cybin recently unveiled upbeat interim phase 2 data related to CYB003, a proprietary deuterated psilocybin analog, in patients with moderate to severe major depressive disorder (MDD). In the randomized double-blind placebo-controlled study, CYB003 demonstrated a rapid and robust reduction in depression symptoms just three weeks …

Anticipating IND submission: Ensuring your drug is ready for preclinical toxicology studies

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Drug development is often a vast and intricate journey. Each phase signifies an advancement in the process, always with an eye toward patient safety and efficacy. But before any therapeutic finds itself on the bedside tables of hopeful patients, it faces a formidable challenge: preclinical toxicology testing. As the gateway to clinical trials, this early testing is more than just a safety assessment. A robust examination establishes a drug’s potential efficacy, possible adverse reactions, and activity within the human body.

Preclinical toxicology is when a drug’s strengths and vulnerabilities are laid bare for all to see. Imagine the invaluable insights drawn from organ-specific tests that reveal hidden adverse reactions or genotoxicity evaluations that shine a light on the risks of cellular mutations.

Such revelations can influence everything from dosing decisi…

Unlocking the secrets of cellular immunogenicity: A deep dive in ELISpot assays

A colorized scanning electron micrograph of a T cell from NIAID.

Next generation therapies and vaccine research have recently dominated headlines and scientific discovery. This research and its ability to treat previously untreatable conditions has rightfully excited the scientific and medical communities worldwide. But the complexities involved require a nuanced approach to ensure the treatment is safe and effective. Integrating cell culture into cellular immunity testing can be challenging, which is why the bioanalytical community has sought options beyond traditional immunoassays. Enter ELISpot assays.

ELISpot assays boast unprecedented sensitivity and specificity. They could legitimately shape the future of cellular immunogenicity testing. As scientists push the boundaries of drug discovery and development, they will inevitably turn to the best tools available. ELISpot assays, with their unique capabilit…

Digital dreams and realities clash pharma and biotech in 2023

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The tale of digital pharma and biotech in 2023 is one of two realities. In one corner, you have AI and digital-focused startups and sometimes executives at Big Pharma companies with grand AI ambitions proclaiming the power of the technology. But on the other side of the pharma-AI coin is a more cautious crowd. Here, industry veterans and pragmatic managers wrestle with the realities of integrating AI into the tightly regulated and often legacy infrastructure that has guided drug development for decades.

Ambitious plans versus on-the-ground realities

It is true that the vast majority of pharma and biotech companies are working on crafting a strategy for AI tools. Only 1% of the participants in CRB’s Horizons Life Sciences 2023 report said they did not have such a strategy. Three-quarters, 76%, said such a strategy would be implemented within the next two years while 21% said they planned on doing so…

The future of MDD treatment: A comparative table highlighting the emergence of fast-acting therapies

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The treatment landscape for major depressive disorder (MDD) continues to diversify, and in the coming years, psychedelic options may be available, including COMPASS Pathways COMP360 and the deuterated psilocybin analog CYB003 from Cybin. CYB003 demonstrated a significant -14.08 point reduction in MADRS score, a 53.3% response rate, and a 20% remission rate at a 12mg dose in interim results from its phase 2 study.

The potential of faster-acting more effective therapies

While it is difficult to compare the results from disparate clinical trials, some broad patterns emerge when viewing data from current treatment options and drugs now in clinical development for MDD (see table below). In contrast, newer treatment options, especially psychedelic therapies such as CYB003 and COMP360, demonstrate potentially faster onset and greater magnitude of symptom reduction.

The promise of NMDA and dopamine rec…