2025: A Year of Promise in Pharma and Biotech — Key Trials and Drug Candidates to Watch

As the new year begins, the life sciences sector is buzzing with anticipation about the clinical trials and drug candidates that could reshape the landscape of medicine in 2025. With advancements in technology, new biologics, and novel drug delivery systems, 2025 promises to be a year of breakthrough discoveries. In this article, we’ll highlight some of the most promising clinical trials and emerging therapies to keep an eye on, as well as recap some of the key developments from 2024 that set the stage for what’s to come.

Key Drug Development Advancements in 2024

Before we look ahead to 2025, let’s first acknowledge some of the landmark moments in drug development from 2024. This year saw substantial progress in several therapeutic areas, including oncology, neurology, and gene therapy:

1. mRNA Therapeutics Beyond COVID-19: Following the success of mRNA COVID-19 vaccines, 2024 marked a pivotal year for mRNA technology, with several candidates entering late-stage trials for infectious diseases like flu and RSV (Respiratory Syncytial Virus). Pfizer and Moderna are leading the charge in developing mRNA vaccines for a variety of conditions, including cancer, autoimmune diseases, and rare genetic disorders.

2. Gene Editing Advancements: CRISPR and other gene-editing technologies gained significant traction in 2024, with companies like CRISPR Therapeutics and Editas Medicine advancing clinical trials targeting genetic disorders such as sickle cell disease and beta-thalassemia. Early results have shown promise in permanently correcting gene mutations with minimal side effects.

3. Immuno-Oncology Breakthroughs: The immuno-oncology space continued to evolve, with multiple trials showing significant progress in treating cancers that were once considered resistant to immune therapies. The approval of Roche’s Vabysmo and early results from Bristol-Myers Squibb's LAG-3 inhibitor in melanoma and other cancers have raised hopes for novel immune checkpoint inhibitors.

4. Neurodegenerative Disease Innovation: In Alzheimer's and Parkinson’s disease, 2024 saw promising data from Biogen’s Leqembi (lecanemab) and Eli Lilly’s Donanemab. These therapies represent a step forward in targeting amyloid plaques, though the debate over efficacy and safety continues to shape the regulatory landscape.

   
   

Top Clinical Trials and Drug Candidates to Watch in 2025

As we look forward to 2025, the industry is turning its attention to several promising drug candidates and clinical trials that could change the way we treat a wide range of diseases. Below are the trials and candidates we’re most excited about:

1. AstraZeneca's Enhertu (Trastuzumab Deruxtecan) in HER2-Low Breast Cancer

AstraZeneca's HER2-targeted antibody-drug conjugate (ADC), Enhertu, has already shown remarkable efficacy in HER2-positive breast cancer. But in 2025, the focus will shift to HER2-low breast cancer, which represents a significant portion of breast cancer patients. Phase III trials are already underway, and data from these studies could revolutionize treatment for a broader group of patients, potentially establishing Enhertu as a cornerstone of breast cancer therapy.

2. Novartis’s Kymriah (Tisagenlecleucel) in Solid Tumors

Kymriah, an FDA-approved CAR T-cell therapy, has already made significant waves in blood cancers like leukemia and lymphoma. However, its extension into solid tumors could be a game changer. Phase II and III trials are testing Kymriah’s efficacy against non-Hodgkin lymphoma, glioblastoma, and other solid malignancies. Success here could make Kymriah a revolutionary tool in treating cancers that have traditionally been much harder to treat with immunotherapy.

3. Vertex Pharmaceuticals’ CRISPR-Based Cystic Fibrosis Treatment

2025 could be the year that CRISPR finally delivers on its promise of curative treatments for genetic diseases. Vertex Pharmaceuticals, in collaboration with CRISPR Therapeutics, is progressing with a trial that uses gene editing to correct the underlying genetic defect in cystic fibrosis (CF). This trial has the potential to not only provide a one-time cure for CF but also to open the door to gene-editing solutions for a host of other genetic disorders.

4. AbbVie’s Rinvoq (Upadacitinib) for Rheumatoid Arthritis

AbbVie’s Rinvoq has already established itself as a promising treatment for inflammatory diseases, but in 2025, the focus will be on its use in rheumatoid arthritis (RA) and other autoimmune conditions. The Phase III trials examining Rinvoq’s potential in RA patients who are non-responders to traditional biologics could position it as a leading therapy in the autoimmune disease market, particularly if it can demonstrate a faster onset of action and improved efficacy over older treatments.

5. Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy (DMD)

Sarepta’s SRP-9001, a gene therapy targeting Duchenne muscular dystrophy (DMD), is one of the most anticipated therapies in the gene therapy space. Phase III trials are expected to deliver data in 2025, and early results have already shown promise in helping DMD patients retain muscle function and slow disease progression. If successful, this therapy could offer a transformative treatment option for DMD patients, an area where few effective treatments currently exist.

6. Neurocrine Biosciences’ Ingrezza (Valbenazine) for Tardive Dyskinesia (TD)

Neurocrine Biosciences’ Ingrezza has already been a major player in treating tardive dyskinesia, a side effect of antipsychotic medications. However, 2025 will see the expansion of Ingrezza into new indications like Huntington’s disease and Parkinson’s disease-related movement disorders. If clinical trials are successful, this could position Ingrezza as a cornerstone therapy for a broad range of neurodegenerative disorders.

7. Pfizer’s Oral OX40 Agonist for Autoimmune Diseases

Pfizer is working on a first-in-class oral OX40 agonist, PF-06831807, which has shown promise in early trials for autoimmune diseases such as lupus and psoriasis. This candidate works by stimulating the OX40 receptor on T-cells, enhancing the body’s immune system while potentially reducing inflammation. If ongoing trials in 2025 confirm its safety and efficacy, this could be a major breakthrough in autoimmune disease therapy.

Looking Ahead: Optimism and Challenges

With the potential for several game-changing therapies to enter the market, industry anticipation around 2025 is high. However, it’s important to recognize that with every new therapeutic advance, there are also challenges. Regulatory hurdles, safety concerns, and the need for robust real-world data will play significant roles in determining whether these drug candidates reach the patients who need them. Moreover, issues like pricing, accessibility, and long-term efficacy will remain critical factors in the adoption of new treatments.

Political and geopolitical factors will continue to shape drug development and clinical trial approvals this year, with regulatory divergence, trade policies, and international relations playing key roles. Political stability in major markets like the U.S., EU, and China can influence the speed and ease of drug approvals, while tensions between countries—such as trade wars or sanctions—may delay access to critical medicines or disrupt global supply chains. Additionally, political pressure to control drug prices in publicly funded healthcare systems could affect the commercial viability of new therapies. With increasing global interdependence, multinational clinical trials and international collaborations will also be affected by shifting regulations, requiring drug developers to navigate complex, often divergent regulatory landscapes to ensure broader approval and market access.

Nevertheless, the sheer breadth of innovation on the horizon means that 2025 could be a banner year for the pharma and biotech industries. Whether it’s harnessing the power of gene editing, revolutionizing cancer treatment, or advancing immunotherapy, the future of medicine looks brighter than ever.

At BioFocus, we’re committed to tracking these developments closely, providing our readers with up-to-date insights and analysis on the drugs and trials shaping the future of healthcare. Stay tuned as we continue to monitor the cutting-edge advances in the biotech and pharma worlds throughout the year.