Pharma Digest: Update on key developments in clinical trials
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Welcome to The Biotech Blueprint. As a scientist with a Ph.D. in biosciences, I’ve spent years learning about the intricacies of immunology, biology, and biochemistry and clinical trial research. My passion for understanding how scientific breakthroughs translate into medical advancements led me to explore the world of clinical trials and the dynamic biotech market. Here at The Biotech Blueprint, I’ll be sharing deep dives into clinical trials, market updates, financial analysis of biotech companies, and providing detailed overviews of publicly traded biotech firms. Stay updated with Pharma Digest, The Biotech Blueprint’s newsletter spotlighting recent exciting clinical advancements and news in the pharma industry.
The pharmaceutical industry has witnessed significant advancements in clinical trials in the last few months. This period has been marked by notable progress in late-stage clinical trials, regulatory approvals, and strategic partnerships aimed at accelerating drug development. This report provides an overview of recent developments, focusing on key pharmaceutical companies and their clinical trial advancements.
Key Developments in Clinical Trials
1. Rocket Pharmaceuticals’ Kresladi
Rocket Pharmaceuticals (RCKT) has been at the forefront of clinical trial advancements with its gene therapy, Kresladi, targeting leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder characterized by immunodeficiency resulting in recurrent infections. LAD-I is characterized by mutations in the CD18 receptor on cells of the immune system that cannot migrate to infection sites to kill invading pathogens. The therapy has shown promising results in Phase 2 trials in 2022, with all nine patients maintaining stable CD18 expression and no therapy-related serious adverse events (1). The FDA extended the priority review period for Kresladi by three months to June 2024 to review additional chemistry, manufacturing, and controls information. However, in July 2024, the FDA issued a Complete Response Letter requesting further data (2).
2. Verona Pharma’s Ensifentrine
Verona Pharma (VRNA) achieved a significant milestone with the FDA approval of its inhaled nonsteroidal nebulizer therapy, ensifentrine, for the maintenance treatment of chronic obstructive pulmonary disease on June 26, 2024 (1). The approval was based on the positive results from the Phase 3 ENHANCE-1 and ENHANCE-2 studies, which demonstrated significant improvements in lung function and a reduction in moderate to severe COPD exacerbations over a 24-week period.
3. Moderna’s Vaccine Programs
Moderna (MRNA) has made substantial progress in its vaccine programs, advancing multiple candidates to late-stage clinical trials. In late March 2024, the company announced that its next-generation COVID-19 vaccine candidate (mRNA-1283) successfully met its Phase 3 endpoints (3). Moderna is also conducting a Phase 2/3 trial for its RSV vaccine, mRNA-1345. Additionally, they have combination vaccine candidates targeting respiratory viruses with the highest disease burden. The Phase 3 trial for their flu and COVID-19 combination vaccine (mRNA-1083) for adults aged 50 and older is fully enrolled, with data expected in 2024. This combination vaccine received Fast Track designation from the FDA in May 2023. Moderna also anticipates a Phase 3 readout in 2024 for its cytomegalovirus vaccine.
The company further reported positive clinical trial data for three new vaccines targeting Epstein-Barr virus, Varicella-Zoster virus, and norovirus, advancing these programs toward Phase 3 development. These new vaccines represent exciting advancements in Moderna’s portfolio.
4. Gilead’s Seladelpar
Gilead Sciences (GILD) is anticipating the FDA’s decision on its drug seladelpar for the treatment of primary biliary cholangitis, with a Prescription Drug User Fee Act (PDUFA) date set for August 14, 2024 (1). Seladelpar has shown promise in clinical trials, and its approval could provide a new treatment option for patients with this chronic liver disease that diminishes liver function and overall quality of life.
5. AstraZeneca and Daiichi Sankyo’s Datopotamab Deruxtecan
AstraZeneca (AZN) and Daiichi Sankyo (DSNKY) are awaiting the FDA’s decision on their antibody-drug conjugate, datopotamab deruxtecan (Dato-DXd), for the treatment of triple-negative breast cancer, with a PDUFA date in the fourth quarter of 2024 (1). Dato-DXd consists of a monoclonal antibody that targets Trop-2, a protein commonly overexpressed in various cancers including breast, lung, and urothelial cancers. The antibody is linked to a potent cytotoxic drug deruxtecan, designed to be released inside the cancer cells, leading to targeted DNA damage and cell death. Dato-DXd has demonstrated significant efficacy in reducing the risk of progression or death in advanced non-small cell lung cancer and inoperable or metastatic hormone receptor (HR)-positive, HER2-low, or HER2-negative breast cancer across two Phase 3 studies, generating significant excitement in the oncology research community.
Trends Shaping the Pharmaceutical Landscape in 2024
Patient-Driven Trial Design
The trend towards patient-driven trial design is gaining momentum in 2024. This approach integrates multiple, study-specific efficacy and safety criteria into a single aggregated measure, estimating the net benefit of treatments and improving the clinical pertinence of trials (4). This strategy can also minimize sample sizes by an average of 20%, making trials more efficient and cost-effective.
Disruptive Technologies
Disruptive technologies such as generative AI, blockchain, and immersive technologies are being leveraged to create efficiencies along the value chain and across internal functions (5). For example, Bristol Myers Squibb (BMY) and Exscientia’s (EXAI) generative AI-designed molecule, EXS4318 (6), entered Phase 1 trials in just 11 months, significantly reducing drug development time. That’s 70% faster compared to the usual average of 4 years. EXS4318 is a potent, and selective inhibitor of protein kinase C-theta, a key driver of several autoimmune diseases, such as rheumatoid arthritis, psoriasis, inflammatory bowel diseases, asthma, type 1 diabetes or multiple sclerosis.
Revolution for Obesity Treatment
Obesity is linked to numerous chronic health conditions, and drugs designed to address obesity may soon become some of the most significant pharmaceuticals on the market. Originally developed for type 2 diabetes, GLP-1 receptor agonists also curb appetite and slow down the rate at which food leaves the stomach and enters the small intestine, moderating the rise in blood glucose levels after eating and promoting a feeling of fullness. Eli Lilly’s SURMOUNT-1 trial for the drug Mounjaro (tirzepatide) demonstrated substantial weight loss, with participants losing up to 22.5% of their body weight (7). This degree of weight reduction is unparalleled among current weight loss medications. Additionally, preliminary data suggest that Mounjaro may offer cardiovascular benefits, enhancing its value for patients at risk of cardiovascular diseases.
Inclusivity and Equity in Clinical Trials
Pharmaceutical companies are increasingly committed to inclusivity and equity in clinical trial recruitment. This involves diversifying trial participants based on race and location (urban vs. rural) through the use of AI for rapid patient population identification and community-centric approaches to address historical fears and mistrust (4).
Cost Optimization and Efficiency
Pharmaceutical companies are focusing on cost optimization and efficiency to create stability in the near term. This involves pruning pipelines, reallocating budgets, restructuring teams, and reducing headcount (5). Companies like Pfizer and Sanofi have announced ambitious cost-realignment strategies aimed at saving billions of dollars by 2024.
DISCLAIMER: This content is for informational purposes only. It should not be taken as legal, tax, investment, financial, or other advice. The views expressed here are my own and do not reflect the opinions of any company or institution.
DISCLOSURE: I have no business relationships with any company mentioned in this article.