Key Drugs to Watch in Oncology, Hematology, and Neurology for 2025

Each year, dozens of new drugs are launched and enter the market, offering countless patients new therapeutic options for a multitude of common diseases and conditions, such as in hemophilia management, oncology, or treatment of neurological conditions. In 2025, multiple drugs are poised to significantly transform their indicated disease states in the coming year and beyond.¹

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Analysts created the list by evaluating each drug using factors including expected approval or launch dates, a drug’s regulatory status, notable clinical trial results, and other aspects that make these medications important to keep an eye on throughout 2025. Below, we’ll dive into drugs in the oncology, hematology, and neurology spaces expected to make an impact in 2025 and highlight the associated therapeutic potential, relevant clinical trial data, and implications for pharmacists and treatment providers.¹

Xanomeline and trospium chloride (Cobenfy, KarXT; Karuna Therapeutics)

A novel combination of xanomeline and trospium chloride to make a dual M1/M4 muscarinic acetylcholine receptor agonist, KarXT is a twice daily oral treatment for adults with schizophrenia and in development for patients with inadequate response to schizophrenia and psychosis related to Alzheimer disease (AD). It received FDA approval in September 2024 for the treatment of adults with schizophrenia, becoming the first in a new class by selectively targeting the M1 and M4 receptors in the brain, while not blocking the dopamine D2 receptors.^1,2

Many existing schizophrenia treatments do target D2 receptor signaling, which makes it difficult for every patient to find an effective treatment. In the EMERGENT clinical trial program, KarXT demonstrated improvements in total Positive and Negative Syndrome Scale score in patients with schizophrenia, with treatment effects observed for some patients as early as 2 weeks. Its potential success in reducing psychosis related to AD makes this new treatment one to watch throughout 2025.¹

Fitusiran (Alnylam Pharmaceuticals, Sanofi)

Fitusiran is an antithrombin-targeting small interfering RNA (siRNA) designed for subcutaneous administration, monthly or bimonthly, for prophylactic treatment of hemophilia A or B regardless of inhibitor status. Fitusiran is part of a generation of novel RNA interference therapies, which provide the ability to selectively down-regulate or silence specific genes to better target complicated diseases.^1,3

Clinical trial data from completed phase 3 studies indicate that fitusiran leads to meaningful reductions in annualized bleeding rate (ABR) in patients with hemophilia A or B. In ATLAS-A/B, a trial of 120 participants with hemophilia A or B without inhibitors, once-monthly administration of fitusiran led to an 89.9% reduction in ABR compared to those receiving an on-demand factor concentrate. Furthermore, ATLAS-INH demonstrated superiority of fitusiran over an on-demand bypass agent, showing a 90.8% reduction in ABR over 9 months.¹

Fitusiran is expected to launch in March 2025, and is poised to provide a new, effective, and convenient option for patients with hemophilia A or B.

Tarlatamab (Imdelltra; Amgen)

Granted FDA approval in May 2024, tarlatamab is poised to revolutionize the small cell lung cancer (SCLC) space. Tarlatamab is approved as a biweekly intravenous (IV) infusion for adults with extensive-stage SCLC with disease progression or following platinum-based chemotherapy. By binding to DLL3 tumor cells and CD3 T-cells, tarlatamab can eliminate DLL3-expressing SCLC cells in the body.^1,5

Tarlatamab utilizes bispecific T cell engager molecules developed by Amgen to engage in its mechanism of action. Given the typically poor prognosis associated with SCLC and the lack of treatment options beyond chemotherapy for patients, tarlatamab’s approval following significantly positive clinical trial results from the DeLLphi-301 study puts it on a path to becoming the standard of care for previously treated ES-SCLC in 2025.¹

Vepdegestrant (ARV-471; Arvinas, Pfizer)

Orally administered treatment for adults with estrogen receptor (ER)-positive (ER+)/HER2-negtative locally advanced or metastatic breast cancer, vepdegestrant could make a large impact in the ER+/HER2- breast cancer space. It could become the first PROteolysis Targeting Chimera (PROTAC) protein degrader to be granted regulatory approval.¹

The treatment is designed to degrade the ER protein, with early trial results indicating that the PROTAC-based mechanism of action behind vepdegestrant makes protein degradation effective and complete compared with oral selective estrogen receptor degraders (SERDs).¹

Phase 3 trials examining vepdegestrant’s safety and efficacy are currently ongoing, but results from phase 1b and phase 2 of the VERITAC clinical trial have been reported. In phase 1b, vepdegestrant demonstrated efficacy in patients with at least 1 prior line of endocrine therapy and no more than 2 lines of chemotherapy in the metastatic setting. Furthermore, the phase 2 cohort expansion of VERITAC showed the medicine was effective and led to strong progression-free survival in patients who had received CDK4/6 inhibitors in the metastatic setting. Continued development of vepdegestrant will allow for further analysis in other patient populations as it prepares to launch in 2025.¹

Zanzalintinib (XL092; Exelixis)

A tyrosine kinase (TK) inhibitor that targets vascular endothelial growth factor (VEGF), zanzalintinib is a once daily, orally administration drug currently under investigation in non-clear-cell renal cell carcinoma (nccRCC), colorectal cancer (CRC), and squamous cell carcinoma of the head and neck (SCCHN), with an expected launch in the US of 2026. According to Clarivate experts, it is designed to target the activity of receptor TKs that feature in tumor angiogenesis, metastasis, and immunosuppression.¹

Zanzalintinib is currently being examined in a series of phase 3 trials in combination with other immune checkpoint inhibitors. The drug has potential to offer a new treatment option specifically for the non-clear-cell histology if approved, easing the disease burden for patients in this population who may struggle to find an effective treatment. Zanzalintinib’s half-life supports administration once daily, which could lead to more favorable tolerability and better adherence among prescribed patients.¹

REFERENCES

1. Clarivate. Drugs to Watch in 2025. Accessed January 10, 2025. Full report available online at https://clarivate.com/drugs-to-watch/.

2. Gallagher A. FDA approves Cobenfy, previously KarXT, for treatment of schizophrenia. Pharmacy Times. Published September 27, 2024. Accessed January 9, 2025. https://www.pharmacytimes.com/view/fda-approves-cobenfy-previously-karxt-for-treatment-of-schizophrenia

3. Dymala K. The promise of RNA interference (RNAi) therapies: Revolutionizing medicine. Pharmacy Times. Published May 9, 2024. Accessed January 9, 2025. https://www.pharmacytimes.com/view/the-promise-of-rna-interference-rnai-therapies-revolutionizing-medicine

4. Gallagher A. FDA accepts application for 5-in-1 meningococcal vaccine candidate. Pharmacy Times. Published April 16, 2024. Accessed January 9, 2025. https://www.pharmacytimes.com/view/fda-accepts-application-for-5-in-1-meningococcal-vaccine-candidate

5. Ferruggia K. The FDA grants accelerated approval to tarlatamab-dlle in small cell lung cancer. Pharmacy Times. Published May 17, 2024. Accessed January 9, 2025. https://www.pharmacytimes.com/view/the-fda-grants-accelerated-approval-to-tarlatamab-dlle-in-small-cell-lung-cancer