Things are about to take a turn for good for the clinical trials in 2024. Waddling through the many challenges in last two years, the industry is now on the edge of a transformative shift fueled by moves like the Federal Reserve interest rate cuts in USA. While change is inevitable, let us focus our attention to the 10 clinical trials in 2024 that has the potential to be the gamechangers for the clinical research sector.
These clinical trials in 2024 are not just about science or research, they are important to find better ways to ensure our good health. Come, let’s dive in:
Roche – Lung Cancer
One of the major clinical trials in 2024, Roche’s Skyscraper-01 trial holds the spotlight in oncology. It focuses on TIGIT, a protein that’s become a prime target in recent years for cancer immunotherapy. This study assesses Roche’s drug tiragolumab alongside Tecentriq, compared to Tecentriq alone, in advanced lung cancer patients. Despite tiragolumab missing one main objective related to disease progression, hopes linger as the drug aims for its critical goal of extending patients’ lives.
In a surprising turn, an interim analysis, initially undisclosed by Roche, hinted at tiragolumab potentially prolonging lives. This revelation, discovered by Wall Street analysts, fueled optimism for TIGIT-targeting medicines, which have faced challenges in previous clinical testing. The final results, eagerly awaited in early 2024, hold the key to determining the value of this cancer immunotherapy class and the potential breakthrough in addressing advanced lung cancer.
Johnson & Johnson – Major Depressive Disorder
In a potential breakthrough for depression treatment, Johnson & Johnson’s experimental medication, aticaprant, is undergoing a late-stage trial with results expected as early as June. Targeting kappa opioid receptors (KORs), aticaprant aims to enhance symptoms for certain adults with major depressive disorder. Among the important clinical trials in 2024, J&J holds high expectations on it, foreseeing it as a key growth driver.
This novel approach has attracted attention beyond J&J, with Neumora Therapeutics and AbbVie also exploring KOR-targeting medicines. The competitive landscape underscores the significance of the upcoming results not only for J&J but for validating the entire KOR drug class. Analysts emphasize the potential for coexistence in the market, recognizing the substantial need for new depression treatments and the room for multiple solutions to address this critical health challenge.
Vertex Pharmaceuticals – Acute Pain
Known for cystic fibrosis treatments, Vertex Pharmaceuticals aims to diversify with one of its top clinical trials in 2024. The focus is on VX-548, a small molecule showing promise as a non-addictive painkiller. Despite positive signals, mid-stage study concerns exist, including missing efficacy data.
In early 2024, results from acute pain trials will be revealed, with potential financial implications exceeding $10 billion annually. This shift underscores Vertex’s pursuit beyond cystic fibrosis drugs, navigating challenges in pain medication development.
Alnylam Pharmaceuticals – Transthyretin Amyloidosis Cardiomyopathy
In 2023, Alnylam Pharmaceuticals faced a significant setback as the FDA rejected Onpattro’s application for transthyretin amyloidosis cardiomyopathy. This unexpected turn led to a strategic shift away from seeking approval for Onpattro in TTR cardiomyopathy. However, the company remains resilient, pinning its hopes on vutrisiran, a potential game-changer.
Among the anticipated clinical trials in 2024, Vutrisiran stands out with its subcutaneous injection method, offering a more convenient and steroid-free alternative for treating nerve-related TTR. Positioned as a contender in the Helios-B study, its success could not only secure approval for cardiomyopathy but also position it as a superior option in a competitive landscape.
Beam Therapeutics – Sickle Cell
Following the groundbreaking approval of Vertex and CRISPR Therapeutics’ Casgevy, the first CRISPR gene editing medicine for sickle cell disease, Beam Therapeutics is stepping into the arena with a second-generation approach. The company is conducting one of the most innovative clinical trials in 2024, with initial data expected in 2025, marking a crucial checkpoint for base editing and drawing attention to potential improvements in gene therapy.
Beam employs “base editing,” a derivative of CRISPR technology that allows precise modification of a single DNA “letter.” While Casgevy, though curative, poses risks, Beam’s innovative method aims to enhance the treatment for sickle cell patients by eliminating the painful crises associated with the disease.
Amylyx Pharmaceuticals – ALS
Amid the clinical trials in 2024, Amylyx Pharmaceuticals’ Relyvrio, dubbed a potential blockbuster, gained FDA clearance for ALS treatment based on a small yet impactful study. With approval secured, the larger confirmatory trial known as “Phoenix” is set to conclude in early March. Despite Relyvrio’s full approval, scrutiny persists due to FDA staff criticisms on data collection and analysis methods in the initial study. While regulatory approval allows Relyvrio to stay on the market without additional testing, the stakes are considerable for Amylyx.
Pfizer – Duchenne Muscular Dystrophy
In the quest for a gene therapy for Duchenne muscular dystrophy, Pfizer emerges as a contender to rival Sarepta Therapeutics, which secured FDA accelerated approval for Elevidys. While Elevidys targets young kids and faced challenges in confirming benefits, Pfizer takes a different approach with its Phase 3 trial. Unlike Sarepta, Pfizer opted for a thorough late-stage study, delaying an early look until 2024.
The decision reflects Pfizer’s strategy to leverage a larger and longer sample size, aiming for clearer treatment benefits and hoping to outpace Sarepta’s accomplishments. Pfizer’s cautious approach follows safety concerns that slowed gene therapy testing, including a participant’s death. If successful, Pfizer’s gene therapy could overcome earlier setbacks, potentially changing the trajectory of Duchenne muscular dystrophy treatment.
Sanofi – Multiple Sclerosis
Sanofi’s foray into multiple sclerosis treatment with tolebrutinib faces a pivotal moment as the drug nears its Phase 3 readout. Sanofi’s tolebrutinib, acquired through a significant deal in 2020, is a novel BTK inhibitor designed to efficiently penetrate the blood-brain barrier, a crucial feature for treating various forms of multiple sclerosis.
Despite Merck KGaA’s trial setback, Sanofi remains optimistic about tolebrutinib’s potential, listing it among the top clinical trials in 2024 and prospects for future blockbusters. The upcoming results in the middle of the year will determine the fate of Sanofi’s bold pursuit in revolutionizing multiple sclerosis treatment.
Moderna – Influenza, COVID-19
As the initial fervour for COVID-19 mRNA vaccines wanes, developers like Moderna are eyeing influenza as a new frontier. With shares dropping and revenue expectations tempered, Moderna seeks a resurgence by leveraging mRNA technology to swiftly adapt to prevalent strains of influenza each year.
Traditional flu shots boast only 40% to 60% effectiveness, leaving ample room for improvement. Moderna, with results from its Phase 3 trial expected in the first half of the year, appears to be leading the race in this innovative intersection of mRNA technology and preventive healthcare.
Gilead Sciences – Lung Cancer
Gilead Sciences’ $21 billion acquisition of Immunomedics in 2020 aimed to leverage the potential of Trodelvy, a breast cancer drug, in expanding its oncology portfolio. Gilead’s upcoming data release from a Trodelvy study, pitting it against chemotherapy for previously treated lung cancer patients, holds the key to tapping into a potentially lucrative market. The competitive scenario prompts questions about Trodelvy’s efficacy and its ability to rival established counterparts.
Conclusion:
In the clinical research field, these 10 clinical trials in 2024 are like signposts for potential changes. Whether it’s finding a painkiller that’s not addictive or trying out new therapies, each trial shows how the industry keeps going. As we see the results in the next few months, their impact will touch the lives of patients looking for better treatments. 2024 looks like a turning point, a time when science, new ideas, and healthier outcomes come together in real ways.
Stay tuned to IQA’s social media and upcoming blogs for more such interesting and timely information around life sciences and clinical trials in 2024. Visit www.inductivequotient.com to understand how we can help your trials succeed.