Swiss pharma giants Novartis and Roche consistently rank among the top pharma companies in terms of revenue. In 2024, the two companies are also leading in total international patent volume. Meanwhile, smaller firms like Incyte and Vertex Pharma are punching above their weight in market impact. For instance, Incyte, with a market cap of just…
The return on engagement: How sustained community outreach is improving clinical trial representation
Abstract Despite a wealth of case studies and recent FDA guidance highlighting the advantages of community-level engagement in clinical trials, local clinics and research sites with access, trust, and experience in treating diverse patient populations are frequently overlooked and under-resourced in the drug development process. Lloryn Hubbard and Zoé Felicié, patient diversity experts at the PPD Clinical…
FDA rejection of MDMA and journal retractions jolt psychedelic therapy research
In a double-blow to psychedelic therapy, the FDA has rejected an application for MDMA-assisted treatment of PTSD, while a prominent medical journal has retracted three key papers on the subject. The agency, which had granted Breakthrough Therapy Designation to MDMA in 2017, signaled it may be open to changing its mind if presented with data…
ChatRWD outperforms tech giants in medical question-answering
When it comes to medical AI, the biggest names aren’t necessarily delivering the best results. While tech giants race to build ever-larger language models, a new preprint reveals that when it comes to clinical accuracy and physician trust, a smaller player is outperforming the industry heavyweights. Putting large language model-based systems to the test Researchers…
Preparing for the golden age of small molecule oncology drugs
Small-molecule drugs have been a mainstay of medicine for over a century, including many of the most impactful pharmaceuticals in human history, such as aspirin and penicillin. Recently, though, biologics—or large-molecule drugs—have become more popular. In fact, in 2016, eight out of 10 of the best-selling drugs globally were biologics. However, small-molecule drugs are experiencing…
Boehringer Ingelheim launches phase 1 trial for triple-action obesity drug
Boehringer Ingelheim has partnered with Gubra, a specialized biotech company and preclinical CRO that specializes in peptide-based drug discovery for metabolic diseases, to launch phase 1 clinical trials for BI 3034701. This drug candidate is a long-acting peptide designed to target three different receptors associated with weight loss, offering a potential new avenue for tackling…
How drug developers can escape from the clinical trial phase maze
It’s something of a truism that about nine out of ten drug candidates in clinical trials fail to reach the market. While some amount of guess-work is unavoidable in drug development, Dr. Ali Pashazadeh, co-founder and CEO of Treehill Partners sees faulty decision-making and poor communication as chief factors contributing to high failure rate. While…
Pharma’s cultural barriers could blunt AI progress despite pressures for more efficient drug approvals.
The FDA’s Center for Drug Evaluation and Research (CDER) recently unveiled the launch of the CDER Center for Clinical Trial Innovation (C3TI), which aims to drive clinical trial innovation. When asked to assess the potential impact of the center, Charles Fisher, founder and CEO of Unlearn, said: “The truth is, I don’t know. I’m sort…
Does biopharma R&D spending translate to results? It’s complicated
A recent review of financial data from 25 major pharmaceutical companies reveals an inconsistent relationship between R&D spending, revenue, and stock returns. The analysis, which included industry giants such as Merck, Pfizer, Johnson & Johnson, and AbbVie, among others, aimed to uncover potential correlations between these key performance indicators. The companies with the highest highest…
Real-world evidence use is expanding. What’s behind the change?
In the rapidly evolving landscape of drug development, real-world evidence (RWE) is making significant strides, finding its way into the early stages of the process. No longer confined to just the peri- and post-approval phases, RWE is now being tapped for external control arms (ECAs), aiding in health technology assessment (HTA), and informing payer discussions.…
Unleashing a new frontier: The power of germline clinico-genomic data to drive therapeutic development
Over the past decade, the use of deeper sources of real-world data across all stages of the drug development life cycle has become increasingly important to guide disease understanding, trial designs, clinical guidelines, regulatory submissions and post-market studies. The advent of these deeper sources was prompted by the HITECH Act, which had the effect of…
How digital pathology can help drug developers address the forked road in oncology and beyond
Digital pathology is disrupting the drug development process, replacing traditional microscopy rooted in Victorian Era approaches with high-resolution images and AI-powered analysis tools. In a recent editorial webinar, industry experts Nathan Buchbinder (chief strategy officer at Proscia), Dr. John Cochran (chief medical officer and chief pathologist at Q² Solutions, an IQVIA subsidiary), and Dr. Monika…
Inside the AI-powered Roche-PathAI companion diagnostics collaboration
PathAI and Roche Tissue Diagnostics (RTD) have inked an exclusive collaboration to develop AI-enabled companion diagnostics that builds on their initial partnership announced in October 2021. To date, PathAI and Roche have commercially launched four algorithms through the partnership. The new partnership will provide biopharma sponsors with integrated technology for developing companion diagnostics incorporating AI-based…
Unlearn CEO: Digital twins could slash clinical trial patient enrollment by 25% or more
The startup Unlearn embodies several trendy AI characteristics. Generative AI company? Check. San Francisco headquarters? Check. Aims to disrupt drug development (specifically, clinical trials) with AI? Check. Prominent AI leadership? Check. Mira Murati, the high-profile CTO of OpenAI, joined Unlearn’s board of directors in 2023. But the company’s pedigree is unique. The firm was founded…
In 2023, Roche and Novartis led the pack in drug pipeline scale
When reviewing R&D spending trends for 2023, Merck & Co. is a clear outlier given its decision to count its $10.3 billion Prometheus acquisition as an R&D charge. In all, the company committed more than half of its revenue to R&D. But Swiss giants Roche and Novartis remain frontrunners in terms of their pipeline of…
Could LSD change the game in anxiety treatment?
A once-controversial psychedelic substance could potentially be a promising treatment for generalized anxiety disorder (GAD). That’s the view of Dr. Rakesh Jain, a psychiatrist with extensive experience in clinical practice, research, and education, affiliated with Texas Tech University School of Medicine. Jain expressed optimism in LSD-based therapy while acknowledging the challenges inherent in such a…
Are Big Pharma giants getting the right ROI on their R&D investments? A visual exploration
While annual reports show broadly similar R&D strategies among Merck & Co., Pfizer, Johnson & Johnson and AbbVie, their 2020–2023 financial metrics reveal a concerning trend. Merck & Co. may be the new top dog of Big Pharma, but the firm’s 1.4% revenue growth in 2023 represents a significant slowdown. Pfizer’s 41.7% revenue decline from…
Moving the needle on diversity in clinical trials: Where do we go from here?
Enhancing patient diversity in clinical trials has become a key priority in drug development. The main concern is that critical data that includes underrepresented patient populations is being left out as many clinical trials do not reflect all populations that may eventually take a therapy. These underrepresented groups consist of women, including those who are…
From gatekeeper to strategist: The evolution of the CISO role in drug development
There’s an old joke about chief information security officers (CISOs) being gatekeepers of new technologies and initiatives – the infamous “Department of No.” Imagine a bouncer who, strangely, doesn’t let anyone in, saying the club is already too full, even when it’s clearly empty. But that image is outdated — especially in risk-focused industries like…
Off with the training wheels: AI-based patient characterization can improve clinical trial performance without large data sets
Only 12% of new drug candidates that enter phase 1 clinical development ultimately receive FDA approval. This dismal success rate leaves millions of patients with unmet medical needs and drives up the costs for the small number of drugs that make it to market. More frustratingly, it leaves untold numbers of potentially transformative therapies back-burnered…
Rice Biotech Launch Pad plans to make Houston a top-tier biotech hub
Houston boasts many world-class assets that have made it a formidable player on the global stage. From the world’s largest medical complex to mission control for the cosmos, few other cities can compete with its diverse strengths. Houston is also home to the prestigious Rice University, renowned for its leading science and engineering programs. Houston,…
Could an ‘extreme’ hibernating ground squirrel unlock new obesity treatments?
In late 2023, Eli Lilly, whose stock is now up close to 80% over the past year, inked a deal with the Emeryville, California–based Fauna Bio potentially worth $494 million that focuses on the discovery of novel drug targets for treating obesity. In 2020, Fauna entered into an obesity-focused collaboration with Novo Nordisk, Lilly’s primary…
Biotech bounces back at JPM 2024 on optimism, breakthroughs and calculated bets, but uncertainties persist
At the dawn of 2024, there’s a sense of renewed optimism in the biotech sector despite recent sector-specific challenges. This week, the JP Morgan Health Care Conference witnessed strong deal-making activity. For instance, Merck agreed to acquire cancer drug developer Harpoon Therapeutics for roughly $680 million, highlighting continued interest in oncology cancer therapies. Meanwhile, Novartis…
Core trends in 2023 FDA drug approvals: Oncology, neurology and hematology dominate
2023 was a big year for hematology, neurology and oncology, with the medical specialties seeing the most FDA approvals. In terms of sponsors, Pfizer had the most approvals with six total, followed by UCB and Chiesi, each with three apiece. When looking at commercial prospects, AstraZeneca’s respiratory syncytial virus antibody Beyfortus could be the biggest…
How a J&J exec found her calling in autoantibody drug development
Dr. Katie Abouzahr’s career, which began in the wards of the UK’s National Health Service (NHS) before extending into management consulting, paved the way for her leadership of autoantibody and maternal fetal medicine programs at Johnson & Johnson. “It’s not a typical pharma executive’s straight line path,” she acknowledged. “Careers can often be jungle gyms…