clinical trials

First synthesized in 1912 by Merck, the empathogenic drug 3,4-Methylenedioxymethamphetamine (MDMA) is inching toward FDA approval following the positive results of a phase 3 study. The recently concluded phase 3 study, MAPP2, published in Nature Medicine, found that MDMA-assisted therapy significantly outperforms traditional talk therapy in reducing PTSD symptoms. Participants receiving MDMA-AT had an 86.5%…

Thanks to promising candidates such as efgartigimod, the Fc receptor (FcRn) inhibitor market is poised to reshape the autoimmune disease treatment landscape. Sales projections for the market top $10 billion, as Driehaus Capital Management estimated in 2020. The addressable U.S. patient base spans more than 228,500 individuals across various conditions including myasthenia gravis, warm autoimmune…

The biotech industry is facing a reckoning in 2023. To date, roughly 100 biopharmas have cut workers this year, matching the total number of layoffs in the sector in 2022. Many biotech startups have been hit hard. The wave of job cuts comes on the heels of a biotech boom following the COVID-19 pandemic, when…

Synthetic data in oncology is transforming how researchers and developers approach real-world evidence. They often need this evidence to test hypotheses, predict outcomes and develop algorithms. But privacy constraints and access related to patient data can create delays and lengthen project timelines. Oncology drug researchers and developers have recently begun using synthetic data in oncology…

Ketabon GmbH revealed positive top-line results from its phase 2 KET01-02 study investigating KET01, an oral slow-release formulation of oral ketamine, for treatment-resistant depression (TRD). Results were promising initially. Investigators noted improvements in depression severity as early as day 4, but data were not statistically significant over placebo at day 21. A look at Ketabon…

AI algorithms offer a myriad of advantages for clinical trials. AI techniques can, for instance, support patient enrollment and site selection, improve data quality and enhance patient outcomes. AI algorithms — combined with an effective digital infrastructure — can also help aggregate and manage clinical trial data in real time, as Deloitte has noted. Last…

Roughly nine out of ten clinical trials fail as a result of factors such as lack of efficacy and unmanageable toxicity, as the journal Pharma Excipients has noted. After seven years of development, Insilico Medicine has scored a breakthrough with its generative AI tool, inClinico. In particular, the tool demonstrated 79% accuracy in predicting the…

The journey to developing a successful drug, theoretically, may appear linear: you discover the right drug, find the suitable patient and administer it at the right time. The reality, however, often deviates from this straightforward path. Aligning these three variables remains notoriously difficult, often leading to elongated timelines strewn with failures, sometimes extending over a…

Cataracts are one of the most common ailments of an aging population, manifesting in the fifth or sixth decade of life. This condition, which leads to blurred or cloudy vision, is caused by proteins that break down in the lens of our eyes, forming an increasingly opaque mass. Although cataracts can cause blindness if untreated,…

The AI market is witnessing meteoric growth, with projections hinting at a potentially staggering increase over the next decade. Against this backdrop of rapid AI evolution, we recently spoke with Tehsin Syed, general manager of AWS Health. Syed shared that Amazon Web Services (AWS) is seeing growing interest from Big Pharma firms. “Nine of the…

In a sign of shifting attitudes, the FDA has released draft guidance to facilitate the development of psychedelic drugs. Historically, psychedelics have a long history of use in certain cultures, such as the indigenous tribes of the Amazon Basin and Native American communities, for millennia as sacraments.These substances were used not only recreationally but also…

As personalized medicine continues to progress, cell and gene therapy (CGT) development is poised to enable tremendous medical breakthroughs. Unlike traditional treatments which merely manage symptoms, CGT could treat the root biological cause of many diseases: the faulty genes. “The cell and gene therapy space is truly exciting. What this ultimately means for patients and…

Janssen continues to strengthen the case that its interleukin-23 inhibitor TREMFYA (guselkumab) offers promise to many patients with psoriatic arthritis (PsA). The company’s phase 3b COSMOS clinical trial involving 189 patients with active PsA and an inadequate response to one to two previous tumor necrosis factor (TNF) inhibitors, guselkumab showed sustained improvements in measures of…

The development of new drugs is undeniably a data-intensive endeavor. Despite impressive advances in AI over the past years, researchers often continue to grapple with crushing data volumes. This hurdle is particularly apparent in clinical trials, where crucial data is often stored in machine-unfriendly formats such as PDFs, PowerPoint or HTML or other formats. This article explores…

In an era of rapid AI progress, the quest to pioneer the first AI-developed drug candidates has led to an increasing number of these drug candidates entering clinical trials. One contender is ulotaront, an antipsychotic drug, that fared well in a phase 3 schizophrenia study published in NEJM in 2020. Sunovion discovered the drug in…

The year 2020 was a watershed moment for many reasons, but notably, it cast a light on the pervasive health and social inequities that have long marred the U.S. The COVID-19 pandemic hit diverse populations disproportionately hard, as Deloitte and others have noted. Additionally, the tragic deaths of George Floyd, Breonna Taylor and others provoked…

While scientific progress hinges on data integrity, plagiarism, data fabrication and image manipulation and other biomedical research scandals are ongoing concerns. An article in Science recently made waves by revealing startling conclusions from the research of the German neuropsychologist Bernhard Sabel, who developed a fake-paper detector. Publishing his findings in a preprint, Sabel concluded that there…

As enterprise AI adoption surges, the life science industry stands at a pivotal juncture. “We are truly at a tipping point,” remarked Najat Khan, chief data science officer at Janssen, during the 2023 Stanford Drug Discovery Symposium. “It’s amazing how quickly we’ve been able to make progress across the whole value chain in terms of…

The Estée Lauder family has donated $200 million to the Alzheimer’s Drug Discovery Foundation (ADDF), a nonprofit they founded in 1998 to support Alzheimer’s research. The gift is the largest ADDF has received. “We’ve deployed about $270 million so far for about 700 programs in 19 countries of drug discovery and development over the past…

AbbVie (NYSE:ABBV) and Janssen Pharmaceutical (NYSE:JNJ) have revealed their intent to voluntarily withdraw the accelerated approvals for Imbruvica (ibrutinib) for patients with mantle cell lymphoma (MCL) and marginal zone lymphoma (MZL) in the U.S.  Ibrutinib is a selective Bruton’s tyrosine kinase (BTK). The main reasons for the move relate to FDA’s request for additional studies…

Pharmaceutical companies face a long list of regulatory challenges ranging from patent expiry to bioequivalence and international harmonization. It’s not uncommon for drug makers to withdraw or discontinue drugs after failing to meet clinical requirements or endpoints, resulting in drug withdrawals. On average, life science companies pull roughly 4,500 drugs and devices from the market,…

Utilizing real-world data (RWD) to generate real-world evidence (RWE) is not new and pharmacoepidemiologists have long-standing experience designing and implementing studies utilizing RWE to satisfy regulatory post-marketing safety commitments and to evaluate the effectiveness of drugs. However, RWD and RWE can be used more largely to support the full life cycle of drug development, during…

Diversity in clinical trials is crucial for ensuring that new medical treatments are safe and effective for all populations. Last year, the FDA released draft guidance that aims to improve the enrollment of historically underrepresented populations in clinical trials. Demand is growing for more transparency and diversity in clinical trials, according to Erin Leckrone, senior…

Clinical trials and long-term follow-up studies (LTFUs) hinge on a whirlwind of data collection, where the volume and quality of patient data form the cornerstone of success. Erratic and patchy data can throw a monkey wrench into evaluating the potency and safety of cutting-edge drugs and devices. Such snags can drag approvals through the mud…

A gene therapy for spinal muscular atrophy (SMA) patients is making waves. Novartis (NYSE:NVS) revealed in a press release new long-term data highlighting the durability of Zolgensma (onasemnogene abeparvovec) up to 7.5 years after a single treatment. The data comes from two long-term follow-up studies, LT-001 and LT-002, which examined a range of patient populations…