clinical trials Archives - Page 3 of 11

GLP-1 drug tirzepatide shines in SURMOUNT-3 trial with weight loss of 26.6%

In the phase 3 SURMOUNT-3 trial, tirzepatide recipients saw some of the most impressive weight loss results among trials of GLP-1 drugs, including most notably semaglutide. In the study, participants’ total mean weight loss was 26.6% over 84 weeks following a 12-week intensive lifestyle intervention and subsequent tirzepatide treatment. In all, participants who received tirzepatide lost an additional 18.4% of their body weight compared to a weight gain of 2.5% in the placebo group.

In SURMOUNT-3, some 87.5% of participants who received tirzepatide achieved an additional weight reduction of 5% or more, compared to 16.5% of those in the placebo group.

While it is difficult to compare data from separate studies, the highest weight loss percentage for tizepatide, 26.6%, was 53% higher than the highest percentage, 17.4%, for semaglutide (OASIS 1).

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Mirikizumab shows continued promise for ulcerative colitis after regulatory hiccup

Lilly has announced that its interleukin-23 blocker mirikizumab demonstrated promise in patients with moderately to severely active ulcerative colitis (UC) in the ongoing open-label LUCENT-3 extension study. The trial evaluated two-year efficacy and safety of the monoclonal antibody in patients who had previously undergone UC treatments, including biologics, that were ineffective, stopped working, or were intolerable.

In April, the company announced that it had received a complete response letter for mirikizumab for UC as a result of issues FDA identified related to the proposed manufacturing of the antibody. The agency noted no concerns about the clinical data package, safety, or label for the therapy.

After revealing the most recent LUCENT-3 data, Lilly plans on filing a marketing application for mirikizumab in Crohn’s disease to FDA. It intends to file paperwork with other regulatory agencies in 2024.

Mirikizumab efficacy in LUCENT-3

The LUCEN…

Guselkumab shows durable benefits in Crohn’s disease in LTE of phase 2 study

The interleukin-23 blocker guselkumab (Tremfya) continues to show promise in treating Crohn’s disease (CD). First winning FDA approval for plaque psoriasis in 2017, guselkumab recently demonstrated robust efficacy and a consistent safety profile in the long-term extension of the GALAXI Phase 2 study for CD.

Some 54.1% of patients receiving guselkumab achieved clinical remission by the three-year mark, compared to 46.0% for those treated with Stelara (ustekinumab), the company’s interleukin-12 and -23 antagonist that won FDA approval for CD in 2016. Patient-reported outcomes were also promising in the GALAXI trial, with 51.4% of guselkumab-treated patients achieving PRO-2 remission, a patient-reported outcome measurement used in inflammatory bowel disease (IBD) studies, compared to 39.7% for ustekinumab. From an endoscopic perspective, 34.7% of patients on guselkumab showcased a positive response, compared with 19.4% on ustekinumab.

An overview of GALAXI LTE …

Alzheimer’s at an inflection point as drug and diagnostics breakthroughs emerge

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Alzheimer’s disease research appears to be hitting its stride, thanks to recent therapeutic advances in drug development and the emergence of biomarkers to detect the condition. “All the pieces of the puzzle of precision medicine, which is already quite common in oncology, are now in place,” said Hartmuth Kolb, vice president, neuroscience biomarkers and R&D global imaging at Johnson & Johnson Innovative Medicine.

There’s urgency to stem the tide of the disease, which not only can be heart-wrenching for patients and caregivers, but also its financial burden. The cost of caring for individuals living with Alzheimer’s or other dementias in the U.S. could hit $345 billion in 2023, according to the Alzheimer’s Association, marking a $24 billion increase over the prior year.

Aiming to stop the cascade

FDA has approved two amyloid-targeting antibodies, lecanemab and …

A century after its synthesis, MDMA could be headed for FDA approval for PTSD

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% response rate (≥10-point reduction in PTSD symptoms), compared to a 69.0% response rate among participants receiving therapy with placebo. Equally impressive, 71.2% of the MDMA-AT participants no longer met the PTSD criteria post-treatment, in contrast to the 47.6% in the placebo with therapy group. At the close of the trial, 46.2% of the MDMA-AT participants met the remission criteria in contrast to the 21.4% in the placebo with therapy group.

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The multi-billion dollar promise of efgartigimod and the broader FcRn inhibitor market

Simplified schematic illustration showing the proposed mechanism of action of FcRn inhibitors. The left side illustrates how IgG molecules enter cells and interact with the neonatal Fc receptors (FcRn), and the right side depicts the impact of FcRn inhibitors on this process. Image by Benff, licensed under [CC BY-SA 4.0], via Wikimedia Commons. Modified from D.D. Patel, J.B. Bussel’s work.

The Fc receptor (FcRn) inhibitor market, led by promising candidates such as efgartigimod, promises to reshape the autoimmune disease treatment landscape. Sales projections for the market top $10 billion, as Driehaus Capital Management estimates. The addressable U.S. patient base spans more than 228,500 individuals across various conditions including myasthenia gravis, warm autoimmune hemolytic anemia (wAIHA) and idiopathic thrombocytopenia purpura (ITP), and more. As the estimated annual treatment cost per patient for targeted…

30 biotech startups making waves

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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 new biopharmas proliferated.

Against this turbulent backdrop, a select group of biotech startups have managed to sustain growth. We’ve selected 30 firms that have collectively raised nearly $8 billion. A fair share of these companies are focused on AI techniques to accelerate drug discovery while others have homed in on niche areas like gene therapies, RNA modulation and epigenetic therapies.

1. Tempus Mission: Applying AI for cancer care and beyond. With a valuation ranging in billions and high-profile partnerships, Tempus has forged high-profile partnerships as it extends i…

How synthetic data accelerates oncology research and drug development

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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 to get around the privacy constraints and access issues related to patient data that create delays and lengthen project timelines.

Conceptually, synthetic data in oncology is about taking private patient information and enabling researchers to access the data without compromising privacy, offering a significant tool for current oncology research processes.

Traditional vs. new data approaches in oncology research

Traditionally, oncology researchers and drug developers have relied on t…

At Day 21, low-dose ketamine KET01 shows no statistical edge over placebo.

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 GmbH’s KET01-02 study in treatment-resistant depression

The trial studied KET01 doses of 120 mg and 240 mg per day. It involved 122 individuals who were outpatients with current major depressive episodes not responding to at least two standard antidepressants. Participants received KET01 once daily in addition to a traditional antidepressant treatment over the three-week period. In particular, the 240 mg/day dose showed clinically relevant improvements with statistical significance versus placebo on days 4 and 7. The drug, however, did not maintain significance at day 21.

K…

A checklist for unlocking the promise of AI in clinical trials

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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 week, a startup revealed an AI system that can accurately predict clinical trial outcomes.

Yet for organizations to fully realize AI’s promise is not simple. The task requires oversight, transparency and diverse collaboration. Core considerations include educating users to build trust in AI tools and ensuring the clinical precision of medical-grade AI algorithms. From unraveling the ‘black box’ of algorithms to safeguarding patient privacy, this article provides a checklist to help organizations responsibly incorporat…

New HHS initiatives put spotlight on long COVID

The U.S. Department of Health and Human Services (HHS) recently announced two major initiatives to shed light on long COVID, a condition affecting millions. First, the agency plans to establish the Office of Long COVID Research and Practice. HHS is also set to launch new long COVID-19 clinical trials through its RECOVER initiative. Jointly, the long COVID drug development efforts highlight the need for therapies and create openings for pharmaceutical companies to drive research and drug development related to the condition.

HHS has earmarked $1.15 billion for the RECOVER Initiative to explore novel therapies for the condition estimated to have affected between 7.7 and 23 million Americans. To date, the RECOVER Initiative has enrolled more than 24,000 participants in observational studies on long COVID. New clinical trials, beginning enrollment in summer 2023, will test experimental therapies across five focus areas to identify therapies that can alleviate symptoms and…

In data we trust: AI’s growing influence on drug development

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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 decade with costs often in the billion-dollar range.

In recent years, the use of AI in drug discovery and development has grown swiftly, marking a significant shift in how we understand, discover and develop new drugs. The technology promises to chip away at timelines and save the industry billions of dollars eventually. But those promises aren’t exactly new. Even before ChatGPT became popular, many drug developers were working with high-end algorithms to “try to really hone in on databases,” said Andrew…