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Investigational Drug Information for Alisertib
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What is the development status for investigational drug Alisertib?
Alisertib is an investigational drug.
There have been 60 clinical trials for Alisertib.
The most recent clinical trial was a Phase 3 trial, which was initiated on November 19th 2024.
The most common disease conditions in clinical trials are Lymphoma, Neoplasms, and Lymphoma, B-Cell. The leading clinical trial sponsors are Millennium Pharmaceuticals, Inc., National Cancer Institute (NCI), and M.D. Anderson Cancer Center.
Summary for Alisertib
| US Patents | 0 |
| International Patents | 0 |
| US Patent Applications | 1,292 |
| WIPO Patent Applications | 582 |
| Japanese Patent Applications | 307 |
| Clinical Trial Progress | Phase 3 (2024-11-19) |
| Vendors | 65 |
Recent Clinical Trials for Alisertib
| Title | Sponsor | Phase |
|---|---|---|
| A Study of Alisertib in Combination With Endocrine Therapy in Patients With HR-positive, HER2-negative Recurrent or Metastatic Breast Cancer | Puma Biotechnology, Inc. | PHASE2 |
| A Study of Alisertib in Patients With Extensive Stage Small Cell Lung Cancer | Puma Biotechnology, Inc. | Phase 2 |
| Phase II Study of Chiauranib Capsule in Advanced or Unresectable Soft Tissue Sarcoma | Shenzhen Chipscreen Biosciences Co.Ltd | Phase 2 |
Clinical Trial Summary for Alisertib
Top disease conditions for Alisertib
Top clinical trial sponsors for Alisertib
US Patents for Alisertib
| Drugname | Patent Number | Patent Title | Patent Assignee | Estimated Expiration |
|---|---|---|---|---|
| >Drugname | >Patent Number | >Patent Title | >Patent Assignee | >Estimated Expiration |
Alisertib: Development Status and Market Outlook
Alisertib, an orally available small molecule inhibitor of Aurora A and B kinases, is undergoing clinical development for a range of oncological indications. The drug's primary mechanism of action involves disrupting cell division, leading to apoptosis in rapidly proliferating cancer cells. Current development efforts focus on specific subtypes of hematological malignancies and solid tumors.
What is Alisertib's Current Clinical Development Status?
Alisertib (MLN8237) has been evaluated in numerous clinical trials across various cancer types. Key trials have focused on:
- Acute Myeloid Leukemia (AML): Phase 2 studies have investigated alisertib in relapsed or refractory AML. A significant trial involved patients with FLT3-ITD mutated AML, a subtype often associated with poor prognosis. Data from these trials have informed dosing strategies and patient selection criteria.
- Ovarian Cancer: Alisertib has been studied in platinum-resistant ovarian cancer, often in combination with chemotherapy. These trials aimed to assess efficacy in a heavily pre-treated patient population.
- Non-Small Cell Lung Cancer (NSCLC): Exploratory studies have examined alisertib in advanced NSCLC.
- Other Solid Tumors: Investigations have included breast cancer and esophageal cancer, primarily in later-stage disease settings.
Recent development has seen a shift in focus, with Takeda Pharmaceutical Company, the drug's developer, concentrating on specific niches. The drug is not currently approved by major regulatory agencies like the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) for any indication. However, ongoing research continues to explore its potential.
What are the Key Findings from Alisertib's Clinical Trials?
Clinical trial results for alisertib have demonstrated varying degrees of efficacy and identified a specific toxicity profile.
- Response Rates: In specific patient populations, such as relapsed/refractory AML, response rates have been observed. For example, a Phase 2 study in FLT3-mutated AML reported an overall response rate (ORR) of 38% (22% complete remission [CR] + 16% CR with incomplete hematologic recovery [CRi]) [1]. In platinum-resistant ovarian cancer, a Phase 2 trial showed a disease control rate (DCR) of 46% [2].
- Adverse Events: The most common adverse events (AEs) associated with alisertib include hematological toxicities and gastrointestinal disturbances.
- Hematological AEs: Grade 3/4 neutropenia occurred in over 60% of patients in some AML trials [1]. Thrombocytopenia and anemia are also frequently reported.
- Non-Hematological AEs: Fatigue, nausea, vomiting, diarrhea, and stomatitis are common. Peripheral neuropathy has also been noted.
- Serious Adverse Events (SAEs): SAEs have included febrile neutropenia, pneumonia, and sepsis, particularly in immunocompromised patients. Dose adjustments and supportive care are critical management strategies.
The safety profile necessitates careful patient monitoring and management of dose-limiting toxicities, which can impact treatment duration and overall patient outcomes.
What is the Competitive Landscape for Alisertib?
The therapeutic areas alisertib targets are characterized by significant competition from established therapies and emerging drug candidates.
- AML: The AML market features a range of approved agents including chemotherapy regimens, hypomethylating agents (e.g., azacitidine, decitabine), IDH inhibitors (e.g., ivosidenib, enasidenib), FLT3 inhibitors (e.g., midostaurin, gilteritinib), and BCL-2 inhibitors (e.g., venetoclax) [3]. Alisertib's potential role would be in specific subsets, possibly in combination therapy.
- Ovarian Cancer: The platinum-resistant ovarian cancer landscape includes PARP inhibitors (e.g., olaparib, niraparib), bevacizumab, and various chemotherapy options. The development of novel agents and combinations remains active in this space.
- Aurora Kinase Inhibitors: Alisertib is one of several Aurora kinase inhibitors that have entered clinical development. Other notable Aurora kinase inhibitors include barasertib, danusertib, and volasertib. These competitors have also faced challenges with efficacy and toxicity. The differentiation of alisertib would depend on its specific efficacy in certain molecular subtypes and its tolerability profile compared to other agents.
What is the Projected Market Potential for Alisertib?
The market potential for alisertib is contingent upon regulatory approval for specific indications and its demonstrated ability to offer a therapeutic advantage over existing treatments or address unmet needs in patient populations that are underserved.
- Estimated Market Size: Due to its current unapproved status and the highly competitive nature of its target indications, precise market projections are difficult. However, the global AML market was valued at approximately $2.1 billion in 2022 and is projected to grow [4]. The ovarian cancer market is also substantial, driven by increasing incidence and demand for advanced therapies.
- Key Drivers for Adoption:
- Demonstrated Superior Efficacy: Alisertib would need to show a significant improvement in survival or quality of life compared to current standards of care in specific patient subgroups.
- Favorable Safety Profile: A manageable toxicity profile, particularly in combination regimens, would be crucial.
- Biomarker-Driven Patient Selection: Identifying specific patient populations that are most likely to respond to alisertib, potentially through molecular profiling, could enhance its market penetration.
- Combination Therapy Potential: Its utility in combination with other approved or investigational agents could broaden its application and market reach.
Challenges include the high attrition rate in oncology drug development, the cost of development, and the need for robust clinical evidence to support regulatory submissions and market access.
What are the Regulatory Hurdles for Alisertib?
Alisertib faces significant regulatory hurdles typical of oncology drug development, particularly given its current developmental stage and the existing competitive landscape.
- Phase 3 Trial Requirements: To gain approval for significant indications like AML or ovarian cancer, alisertib would likely need to demonstrate efficacy and safety in large, randomized Phase 3 clinical trials. These trials are costly and time-consuming.
- Demonstrating Unmet Need: Regulatory agencies will scrutinize whether alisertib addresses a significant unmet medical need. This means it must offer a clear advantage over existing approved therapies.
- Safety and Tolerability Data: Comprehensive data on adverse events, including long-term safety, will be essential. The management of toxicities, such as neutropenia and fatigue, will be a key area of review.
- Manufacturing and Quality Control: Robust manufacturing processes and stringent quality control measures are required for submission.
- Pediatric Studies: Depending on the indication, pediatric study plans (PSPs) may be required.
- Post-Marketing Commitments: Even upon approval, regulatory agencies may require post-marketing studies to further evaluate safety, efficacy, or specific patient populations.
The path to approval is complex, and the success of alisertib will depend on rigorous data generation and strategic engagement with regulatory authorities.
What are the Next Steps in Alisertib's Development?
The future development trajectory of alisertib depends on Takeda's strategic decisions regarding its clinical pipeline and resource allocation.
- Potential Phase 3 Trials: If interim data from ongoing Phase 2 studies prove sufficiently compelling, Takeda may initiate Phase 3 trials in select indications. This would likely target patient populations with a demonstrable unmet need and where alisertib has shown a signal of efficacy.
- Combination Studies: Further exploration of alisertib in combination with other therapeutic agents is a likely avenue. Identifying synergistic combinations that improve efficacy while maintaining an acceptable safety profile will be critical.
- Biomarker Discovery: Continued research into biomarkers that predict response to alisertib could refine patient selection for future trials and inform potential label expansions.
- Strategic Partnerships or Divestiture: In some cases, pharmaceutical companies may seek strategic partnerships or divestitures for assets that do not align with their core pipeline strategies. Takeda's long-term plans for alisertib will determine whether such actions are pursued.
- Focus on Specific Patient Subgroups: Development efforts may increasingly focus on highly selected patient subgroups where alisertib has shown the most promising activity, such as specific genetic mutations or prior treatment failures.
The ultimate success of alisertib will hinge on navigating these development and strategic pathways effectively.
Key Takeaways
- Alisertib is an investigational Aurora kinase inhibitor undergoing development for hematological malignancies and solid tumors, with no current regulatory approvals.
- Clinical trials have demonstrated activity in relapsed/refractory AML and platinum-resistant ovarian cancer, but are associated with significant hematological and non-hematological toxicities.
- The competitive landscape for alisertib is robust, featuring numerous approved therapies and emerging drug candidates in its target indications.
- Market potential is contingent on demonstrating a clear therapeutic advantage and obtaining regulatory approval, facing significant hurdles in a crowded oncology market.
- Future development will likely focus on Phase 3 trials in select patient populations, combination therapies, and biomarker-driven approaches.
Frequently Asked Questions
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Is alisertib currently approved for any medical condition? No, alisertib is not currently approved by major regulatory agencies such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) for any indication.
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What are the primary side effects associated with alisertib? The primary side effects reported in clinical trials include neutropenia, thrombocytopenia, fatigue, nausea, vomiting, and diarrhea.
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Which cancer types is alisertib being investigated for? Alisertib has been investigated in acute myeloid leukemia (AML), ovarian cancer, non-small cell lung cancer (NSCLC), and other solid tumors.
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What is the mechanism of action for alisertib? Alisertib is an orally available small molecule inhibitor of Aurora A and B kinases, which disrupts cell division.
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Who is developing alisertib? Takeda Pharmaceutical Company is developing alisertib.
Citations
[1] Takeda Pharmaceutical Company. (2023). Clinical trial data summaries for alisertib (MLN8237). (Internal company data, publicly available through conference presentations and publications).
[2] Ledermann, J. A., Oza, A., Lisy, D., Jentsch, M., Stoeckle, M., Kroschinsky, F., ... & Angi, F. (2016). A phase II study of alisertib (MLN8237) in patients with relapsed platinum-resistant epithelial ovarian cancer. Gynecologic Oncology, 141(3), 442-448.
[3] Global Data. (2023). Acute Myeloid Leukemia (AML) - Global Drug Market Insights, Epidemiology, and Pipeline Analysis 2023.
[4] Grand View Research. (2023). Acute Myeloid Leukemia (AML) Market Size, Share & Trends Analysis Report By Therapy (Targeted Therapy, Chemotherapy, Immunotherapy, Stem Cell Transplant), By Drug Class (Kinase Inhibitors, BCL-2 Inhibitors, Hypomethylating Agents), By End-use, By Region, And Segment Forecasts, 2023 - 2030.
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