CLINICAL TRIALS PROFILE FOR EVEROLIMUS

505(b)(2) Clinical Trials for everolimus

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT00373815 ↗	Everolimus in Combination With Cyclosporine A and Prednisolone for the Treatment of Graft Versus Host Disease	Terminated	University Hospital Tuebingen	Phase 1	2006-09-01	The present protocol is a dose-finding and toxicity study in preparation of a randomised study comparing current standard treatment CSA/prednisolone with the new combination CSA/prednisolone/everolimus.
New Indication	NCT01175096 ↗	Safety and Tolerability Profile of RAD001 Daily in Chinese Patients With Advanced Pulmonary Neuroendocrine Tumor	Unknown status	Novartis	Phase 1/Phase 2	2010-07-01	RAD001 continues to be investigated as an anticancer agent on new indications such as neuroendocrine tumors (incl. carcinoid), breast cancer, liver cancer, gastric cancer and lymphoma based on its potential to act: - directly on the tumor cells by inhibiting tumor cell growth and proliferation - indirectly by inhibiting angiogenesis leading to reduced tumor vascularity (via potent inhibition of tumor cell HIF-1 activity and VEGF production and VEGF-induced proliferation of endothelial cells) A role for RAD001 in combination with Sandostatin LAR® Depot in the treatment of advanced carcinoid tumor is suggested by data on the regulatory role of mTOR in cell growth and protein translation and the finding that somatostatin-induced growth arrest is mediated in part by inhibition of the PI3K pathway (Charland, et al. 2001). The present study is designed to collect safety/tolerability data and evidences for efficacy of RAD001 in the medically highly unmet indication of advanced pulmonary neuroendocrine tumor in Chinese patients.
New Indication	NCT01175096 ↗	Safety and Tolerability Profile of RAD001 Daily in Chinese Patients With Advanced Pulmonary Neuroendocrine Tumor	Unknown status	Guangdong General Hospital	Phase 1/Phase 2	2010-07-01	RAD001 continues to be investigated as an anticancer agent on new indications such as neuroendocrine tumors (incl. carcinoid), breast cancer, liver cancer, gastric cancer and lymphoma based on its potential to act: - directly on the tumor cells by inhibiting tumor cell growth and proliferation - indirectly by inhibiting angiogenesis leading to reduced tumor vascularity (via potent inhibition of tumor cell HIF-1 activity and VEGF production and VEGF-induced proliferation of endothelial cells) A role for RAD001 in combination with Sandostatin LAR® Depot in the treatment of advanced carcinoid tumor is suggested by data on the regulatory role of mTOR in cell growth and protein translation and the finding that somatostatin-induced growth arrest is mediated in part by inhibition of the PI3K pathway (Charland, et al. 2001). The present study is designed to collect safety/tolerability data and evidences for efficacy of RAD001 in the medically highly unmet indication of advanced pulmonary neuroendocrine tumor in Chinese patients.
New Indication	NCT01175096 ↗	Safety and Tolerability Profile of RAD001 Daily in Chinese Patients With Advanced Pulmonary Neuroendocrine Tumor	Unknown status	Guangdong Provincial People's Hospital	Phase 1/Phase 2	2010-07-01	RAD001 continues to be investigated as an anticancer agent on new indications such as neuroendocrine tumors (incl. carcinoid), breast cancer, liver cancer, gastric cancer and lymphoma based on its potential to act: - directly on the tumor cells by inhibiting tumor cell growth and proliferation - indirectly by inhibiting angiogenesis leading to reduced tumor vascularity (via potent inhibition of tumor cell HIF-1 activity and VEGF production and VEGF-induced proliferation of endothelial cells) A role for RAD001 in combination with Sandostatin LAR® Depot in the treatment of advanced carcinoid tumor is suggested by data on the regulatory role of mTOR in cell growth and protein translation and the finding that somatostatin-induced growth arrest is mediated in part by inhibition of the PI3K pathway (Charland, et al. 2001). The present study is designed to collect safety/tolerability data and evidences for efficacy of RAD001 in the medically highly unmet indication of advanced pulmonary neuroendocrine tumor in Chinese patients.
New Combination	NCT02520063 ↗	Preoperative Combination of Letrozole, Everolimus, and TRC105 in Postmenopausal Hormone-Receptor Positive and Her2 Negative Breast Cancer	Active, not recruiting	Novartis Pharmaceuticals	Phase 1/Phase 2	2016-02-01	This study will test how well a new combination of three drugs (Letrozole, Everolimus, and TRC105) is tolerated and how well it works in Stage 2 and 3 breast cancer when given prior to definitive surgery. Letrozole blocks the estrogen receptor expressed by many breast cancers while everolimus blocks signals that drive cancer cells to grow. TRC105 is an investigational drug that blocks the formation and growth of blood vessels that feed the cancer and promote its growth. The goal of this study is to investigate the safety and efficacy of this multitargeted approach in breast cancer.
New Combination	NCT02520063 ↗	Preoperative Combination of Letrozole, Everolimus, and TRC105 in Postmenopausal Hormone-Receptor Positive and Her2 Negative Breast Cancer	Active, not recruiting	Tracon Pharmaceuticals Inc.	Phase 1/Phase 2	2016-02-01	This study will test how well a new combination of three drugs (Letrozole, Everolimus, and TRC105) is tolerated and how well it works in Stage 2 and 3 breast cancer when given prior to definitive surgery. Letrozole blocks the estrogen receptor expressed by many breast cancers while everolimus blocks signals that drive cancer cells to grow. TRC105 is an investigational drug that blocks the formation and growth of blood vessels that feed the cancer and promote its growth. The goal of this study is to investigate the safety and efficacy of this multitargeted approach in breast cancer.

All Clinical Trials for everolimus

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00081874 ↗	RAD001 in Relapsed or Refractory AML, ALL, CML in Blastic-Phase, Agnogenic Myeloid Metaplasia, CLL, T-Cell Leukemia, or Mantle Cell Lymphoma	Completed	Novartis	Phase 1/Phase 2	2004-04-01	The goal of this clinical research study is to find the highest safe dose of RAD001 that can be given as a treatment for leukemia, mantle cell lymphoma, or myelofibrosis. Another goal is to learn how effective the dose that is found is as a treatment.
NCT00081874 ↗	RAD001 in Relapsed or Refractory AML, ALL, CML in Blastic-Phase, Agnogenic Myeloid Metaplasia, CLL, T-Cell Leukemia, or Mantle Cell Lymphoma	Completed	M.D. Anderson Cancer Center	Phase 1/Phase 2	2004-04-01	The goal of this clinical research study is to find the highest safe dose of RAD001 that can be given as a treatment for leukemia, mantle cell lymphoma, or myelofibrosis. Another goal is to learn how effective the dose that is found is as a treatment.
NCT00085566 ↗	Everolimus and Gefitinib in Treating Patients With Progressive Glioblastoma Multiforme or Progressive Metastatic Prostate Cancer	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2004-03-01	RATIONALE: Everolimus may stop the growth of tumor cells by stopping blood flow to the tumor. Gefitinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Combining everolimus with gefitinib may kill more tumor cells. PURPOSE: This phase I/II trial is studying the side effects and best dose of everolimus when given together with gefitinib and to see how well they work in treating patients with progressive glioblastoma multiforme or (progressive metastatic prostate cancer closed to accrual 10/19/06).
NCT00085566 ↗	Everolimus and Gefitinib in Treating Patients With Progressive Glioblastoma Multiforme or Progressive Metastatic Prostate Cancer	Completed	Memorial Sloan Kettering Cancer Center	Phase 1/Phase 2	2004-03-01	RATIONALE: Everolimus may stop the growth of tumor cells by stopping blood flow to the tumor. Gefitinib may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Combining everolimus with gefitinib may kill more tumor cells. PURPOSE: This phase I/II trial is studying the side effects and best dose of everolimus when given together with gefitinib and to see how well they work in treating patients with progressive glioblastoma multiforme or (progressive metastatic prostate cancer closed to accrual 10/19/06).
NCT00093639 ↗	Everolimus and Imatinib Mesylate in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia Who Are Not in Complete Cytogenetic Remission After Previous Imatinib Mesylate	Completed	Novartis Pharmaceuticals	Phase 1/Phase 2	2004-08-01	RATIONALE: Drugs used in chemotherapy, such as everolimus, work in different ways to stop cancer cells from dividing so they stop growing or die. Imatinib mesylate may stop the growth of cancer cells by blocking the enzymes necessary for their growth. Combining everolimus with imatinib mesylate may be effective in killing cancer cells that have become resistant to imatinib mesylate. PURPOSE: This phase I/II trial is studying the side effects and best dose of everolimus when given together with imatinib mesylate and to see how well they work in treating patients with chronic phase chronic myelogenous leukemia who are not in complete cytogenetic remission after previous imatinib mesylate.
NCT00096486 ↗	Gefitinib and Everolimus in Treating Patients With Stage IIIB or Stage IV or Recurrent Non-Small Cell Lung Cancer	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	2004-05-01	RATIONALE: Gefitinib and everolimus may stop the growth of tumor cells by blocking the enzymes necessary for their growth. Giving gefitinib together with everolimus may kill more tumor cells. PURPOSE: This phase I/II trial is studying the side effects, best way to give, and best dose of giving gefitinib with everolimus and to see how well it works in treating patients with stage IIIB or stage IV or recurrent non-small cell lung cancer.

Clinical Trial Conditions for everolimus

Condition Name

Condition Name for everolimus
Intervention	Trials
Breast Cancer	55
Neuroendocrine Tumors	23
Renal Cell Carcinoma	21
Renal Transplantation	20

Condition MeSH

Condition MeSH for everolimus
Intervention	Trials
Breast Neoplasms	111
Carcinoma	96
Carcinoma, Renal Cell	86
Neoplasms	56

Clinical Trial Locations for everolimus

Trials by Country

Trials by Country for everolimus
Location	Trials
Italy	335
Spain	212
China	82
Brazil	73
Netherlands	73

Trials by US State

Trials by US State for everolimus
Location	Trials
Texas	142
California	126
New York	119
Massachusetts	98
Florida	95

Clinical Trial Progress for everolimus

Clinical Trial Phase

Clinical Trial Phase for everolimus
Clinical Trial Phase	Trials
Phase 4	115
Phase 3	96
Phase 2/Phase 3	11

Clinical Trial Status

Clinical Trial Status for everolimus
Clinical Trial Phase	Trials
Completed	412
Terminated	98
Unknown status	78

Clinical Trial Sponsors for everolimus

Sponsor Name

Sponsor Name for everolimus
Sponsor	Trials
Novartis Pharmaceuticals	205
Novartis	140
National Cancer Institute (NCI)	74

Sponsor Type

Sponsor Type for everolimus
Sponsor	Trials
Other	806
Industry	511
NIH	90

Everolimus Market Analysis and Financial Projection

Everolimus: Clinical Trials, Market Analysis, and Projections

Introduction to Everolimus

Everolimus, known by brand names such as Afinitor and Zortress, is a potent mTOR (mechanistic target of rapamycin) inhibitor. It is widely used in the treatment of various types of cancer and as an immunosuppressant to prevent organ rejection in transplant patients. Here, we will delve into the latest clinical trials, market analysis, and projections for this versatile drug.

Clinical Trials Update

EVEREST Trial: Adjuvant Everolimus for Renal Cell Carcinoma

A significant recent development in the clinical use of everolimus comes from the EVEREST trial. This phase III trial, conducted by the SWOG Cancer Research Network, investigated the use of adjuvant everolimus in patients with clear cell renal cell carcinoma (ccRCC) at very high risk of recurrence after nephrectomy. The results showed that patients receiving adjuvant everolimus had a statistically significant improvement in recurrence-free survival compared to those receiving a placebo. This improvement was observed in both the intent-to-treat and per-protocol cohorts, despite higher rates of grade ≥ 3 adverse events in the everolimus group[1].

Phase II Clinical Trial in Pan-Cancer Cohort

Another clinical trial of note is a phase II study conducted at the Dana-Farber Cancer Institute and Memorial Sloan Kettering Cancer Center. This trial evaluated the efficacy of everolimus in patients with advanced solid tumors harboring TSC1, TSC2, or MTOR mutations. The study found a low response rate to everolimus, with objective evidence of tumor burden reduction primarily in patients with tumors having PEComa pathologic features or the specified genetic mutations[4].

Market Analysis

Market Size and Growth

The global everolimus market is experiencing significant growth driven by several factors. The increasing incidence of cancer worldwide, particularly renal cell carcinoma, breast cancer, and pancreatic neuroendocrine tumors, is a major driver. Everolimus's effectiveness in treating these cancers and its use in conjunction with other targeted therapies have enhanced its market reach[3].

Applications and Segmentation

Everolimus is segmented into several applications, including oncology, organ transplantation, and gastrointestinal disorders. In oncology, it is used to treat various cancers by inhibiting cell growth and proliferation. In organ transplantation, it prevents organ rejection in liver and kidney transplant patients. The market is also segmented by type, with tablets available in 2.5 mg, 5 mg, 7.5 mg, and 10 mg formulations[2][5].

Geographical Distribution

The market for everolimus is distributed across several regions, with North America and Europe being significant contributors due to their advanced healthcare infrastructure and high prevalence of cancer. The Asia-Pacific region is also experiencing rapid growth, driven by increasing cancer rates and improving healthcare facilities. Latin America and the Middle East & Africa are emerging markets, though they face challenges such as economic constraints and limited access to advanced medications[3].

Key Market Players

Major players in the everolimus market include Novartis Inc., Biocon Inc., Breckenridge Pharmaceutical Inc., Teva Pharmaceuticals USA, Inc., Viatris Inc., and Glenmark Pharmaceuticals Limited. These companies are driving market growth through their extensive product portfolios and strategic initiatives[2][3][5].

Market Projections

Future Growth Drivers

Several factors are expected to drive the growth of the everolimus market in the coming years:

• Rising Cancer Incidence: The increasing global incidence of cancer will continue to drive the demand for effective therapies like everolimus.
• Expanding Applications: Ongoing research into new applications of everolimus, such as in personalized medicine and novel cancer types, will enhance its market potential.
• Growing Transplant Procedures: The rise in organ transplant surgeries worldwide will increase the demand for immunosuppressive medications like everolimus.
• Technological Developments: Advances in drug delivery methods and formulations are expected to improve the efficacy and safety profile of everolimus, making it more appealing to patients and healthcare professionals[3].

Challenges and Limitations

Despite the positive outlook, the everolimus market faces several challenges:

• Adverse Effects: Everolimus can have serious side effects, which may restrict its use or lead to treatment discontinuation.
• Generic Competition: The potential for generic versions of everolimus, should the patent expire, could reduce the profit margins of the original manufacturers.
• Clinical Trial Failures: The failure of clinical trials or the emergence of new competitors could impact the market share of everolimus[3].

Key Takeaways

• Everolimus has shown significant promise in clinical trials, particularly in the EVEREST trial for adjuvant treatment in high-risk renal cell carcinoma.
• The global market for everolimus is driven by increasing cancer incidence, expanding applications, and growing organ transplant procedures.
• Major players like Novartis and Biocon are key drivers of market growth.
• Despite growth drivers, the market faces challenges such as adverse effects, generic competition, and clinical trial failures.

FAQs

What is the primary use of everolimus in oncology?

Everolimus is primarily used in oncology to treat various cancers, including renal cell carcinoma, breast cancer, and pancreatic neuroendocrine tumors, by inhibiting cell growth and proliferation.

How is everolimus used in organ transplantation?

Everolimus is used as an immunosuppressant to prevent organ rejection in liver and kidney transplant patients, helping to maintain the stability and function of the transplanted organs.

What are the key drivers of the everolimus market?

The key drivers include the rising incidence of cancer, expanding applications in personalized medicine and novel cancer types, and the growth in organ transplant procedures worldwide.

What are the potential challenges facing the everolimus market?

Challenges include adverse effects, the potential for generic competition if the patent expires, and the risk of clinical trial failures.

Which regions are significant contributors to the everolimus market?

North America and Europe are significant contributors due to their advanced healthcare infrastructure and high prevalence of cancer. The Asia-Pacific region is also experiencing rapid growth.

Sources

1. EVEREST Trial Adjuvant Everolimus for High-Risk Clear Cell Renal Cell Carcinoma - ASCO Post, October 2023.
2. Global Everolimus Tablet Market - Allied Market Research.
3. Everolimus Market Size, & Forecast - Verified Market Research.
4. Phase II Clinical Trial of Everolimus in a Pan-Cancer Cohort of Patients with Advanced Solid Tumors - AACR Journals.
5. Global Everolimus Tablet Market Research Report 2024 - Valuates Reports.