CLINICAL TRIALS PROFILE FOR ENZALUTAMIDE

All Clinical Trials for Enzalutamide

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00268476 ↗	Systemic Therapy in Advancing or Metastatic Prostate Cancer: Evaluation of Drug Efficacy	Recruiting	Medical Research Council	Phase 2/Phase 3	2005-07-08	The overall aim of this trial, which is called STAMPEDE, is to assess novel approaches for the treatment of men with prostate cancer who are starting long-term ADT for the first time, termed hormone-naïve prostate cancer. This trial aims to see if we can improve the way in which prostate cancer is currently managed, either by adding new treatments to the standard approach or by modifying the type of hormone therapy aiming to improve quality-of-life by reducing the side effects of treatment. Each new treatment approach is compared against a control arm receiving the current standard treatments. We aim to identify treatment strategies that enable men to live longer, or as long but with an improved quality-of-life, as well as offering value for money for the health service. Since opening to accrual in Oct-2005, the trial has tested many ways of treating prostate cancer and some results are now already known. More than 10,000 men will join the trial with answers becoming available throughout the trial. New patients joining the trial from Protocol version 17.0 onwards (activated in December 2018) may be eligible to join one of two treatment comparisons, metformin (treatment group K; the "metformin comparison") and transdermal oestradiol (treatment group L; the "transdermal oestradiol comparison"). A computer program will be used to allocate which treatment each participant receives, using a chance process. Summary of the research arms in STAMPEDE trial platform Summary of research treatment groups currently open to recruitment (June 2017) 1. Metformin (Arm K): This anti-diabetic medication is proposed to have both anti-cancer effects and may help prevent the adverse metabolic effects of long-term ADT. STAMPEDE will investigate whether adding metformin to the current standard-of-care for non-diabetic men can improve all-cause survival. 2. Transdermal oestradiol (Arm L): This is an alternative form of hormone treatment which has been shown to suppress testosterone as effectively as standard ADT and avoid some of the side-effects. It may also help to avoid the adverse metabolic effects and fatigue and therefore improve overall quality of life compared with standard forms of ADT. STAMPEDE will investigate whether transdermal oestradiol can treat the cancer as well as current standard forms of ADT. 3. Control group (Arm A): Patients allocated to this group receive the current standard-of-care ADT +/- RT +/- docetaxel.
NCT00510718 ↗	A Phase 1 Study of MDV3100 in Patients With Castration-Resistant (Hormone-Refractory) Prostate Cancer	Completed	Astellas Pharma Inc	Phase 1	2007-07-23	This is a multi-center open-label dose-escalation study of a novel compound (MDV3100) to treat patients with castration-resistant (hormone-refractory) prostate cancer. Additional patients will be enrolled in expanded cohorts at doses determined to be tolerable. Patients who tolerate the drug and do not progress will be allowed to continue to look for PSA response.
NCT00510718 ↗	A Phase 1 Study of MDV3100 in Patients With Castration-Resistant (Hormone-Refractory) Prostate Cancer	Completed	Medivation LLC, a wholly owned subsidiary of Pfizer Inc.	Phase 1	2007-07-23	This is a multi-center open-label dose-escalation study of a novel compound (MDV3100) to treat patients with castration-resistant (hormone-refractory) prostate cancer. Additional patients will be enrolled in expanded cohorts at doses determined to be tolerable. Patients who tolerate the drug and do not progress will be allowed to continue to look for PSA response.
NCT00510718 ↗	A Phase 1 Study of MDV3100 in Patients With Castration-Resistant (Hormone-Refractory) Prostate Cancer	Completed	Medivation, Inc.	Phase 1	2007-07-23	This is a multi-center open-label dose-escalation study of a novel compound (MDV3100) to treat patients with castration-resistant (hormone-refractory) prostate cancer. Additional patients will be enrolled in expanded cohorts at doses determined to be tolerable. Patients who tolerate the drug and do not progress will be allowed to continue to look for PSA response.
NCT00510718 ↗	A Phase 1 Study of MDV3100 in Patients With Castration-Resistant (Hormone-Refractory) Prostate Cancer	Completed	Pfizer	Phase 1	2007-07-23	This is a multi-center open-label dose-escalation study of a novel compound (MDV3100) to treat patients with castration-resistant (hormone-refractory) prostate cancer. Additional patients will be enrolled in expanded cohorts at doses determined to be tolerable. Patients who tolerate the drug and do not progress will be allowed to continue to look for PSA response.
NCT00974311 ↗	Safety and Efficacy Study of MDV3100 in Patients With Castration-Resistant Prostate Cancer Who Have Been Previously Treated With Docetaxel-based Chemotherapy	Completed	Astellas Pharma Inc	Phase 3	2009-09-30	This is a phase 3 study to compare the clinical benefit of MDV3100 versus placebo in patients with castration-resistant prostate cancer who have been previously treated with docetaxel-based chemotherapy.
NCT00974311 ↗	Safety and Efficacy Study of MDV3100 in Patients With Castration-Resistant Prostate Cancer Who Have Been Previously Treated With Docetaxel-based Chemotherapy	Completed	Medivation LLC, a wholly owned subsidiary of Pfizer Inc.	Phase 3	2009-09-30	This is a phase 3 study to compare the clinical benefit of MDV3100 versus placebo in patients with castration-resistant prostate cancer who have been previously treated with docetaxel-based chemotherapy.

Clinical Trial Conditions for Enzalutamide

Condition Name

Condition Name for Enzalutamide
Intervention	Trials
Prostate Cancer	112
Metastatic Castration-resistant Prostate Cancer	27
Castration-resistant Prostate Cancer	21
Metastatic Prostate Cancer	19

Condition MeSH

Condition MeSH for Enzalutamide
Intervention	Trials
Prostatic Neoplasms	294
Carcinoma	29
Adenocarcinoma	28
Breast Neoplasms	18

Clinical Trial Locations for Enzalutamide

Trials by Country

Trials by Country for Enzalutamide
Location	Trials
United Kingdom	199
Canada	177
France	152
Spain	139
Australia	126

Trials by US State

Trials by US State for Enzalutamide
Location	Trials
New York	90
California	89
Texas	67
Maryland	67
Michigan	59

Clinical Trial Progress for Enzalutamide

Clinical Trial Phase

Clinical Trial Phase for Enzalutamide
Clinical Trial Phase	Trials
Phase 4	14
Phase 3	42
Phase 2/Phase 3	5

Clinical Trial Status

Clinical Trial Status for Enzalutamide
Clinical Trial Phase	Trials
Recruiting	101
Completed	83
Active, not recruiting	75

Clinical Trial Sponsors for Enzalutamide

Sponsor Name

Sponsor Name for Enzalutamide
Sponsor	Trials
Medivation, Inc.	59
Astellas Pharma Inc	51
National Cancer Institute (NCI)	49

Sponsor Type

Sponsor Type for Enzalutamide
Sponsor	Trials
Industry	362
Other	358
NIH	51

Enzalutamide: A Comprehensive Update on Clinical Trials, Market Analysis, and Projections

Introduction to Enzalutamide

Enzalutamide, marketed under the brand name XTANDI, is a phenylimidazolidine derivative that acts as an anti-neoplastic agent. It is specifically designed to treat various stages of prostate cancer by inhibiting the activity of androgen receptors, which are crucial for the proliferation of prostate cancer cells[2].

Mechanism of Action

Enzalutamide is the first triple-acting androgen receptor antagonist. It works by inhibiting the activity of androgen receptors in prostate cancer cells, leading to a reduction in cell proliferation and a decrease in serum prostate-specific antigen (PSA) levels. This mechanism is particularly effective in addressing androgen receptor over-expression, a key factor in hormone resistance in prostate cancer[2].

Clinical Trials Update

EMBARK Trial

The Phase 3 EMBARK trial, conducted by Pfizer and Astellas, evaluated the efficacy of enzalutamide in combination with leuprolide in men with non-metastatic hormone-sensitive prostate cancer (nmHSPC) and high-risk biochemical recurrence. The trial met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in metastasis-free survival (MFS) for patients receiving enzalutamide plus leuprolide compared to those receiving placebo plus leuprolide or enzalutamide monotherapy[1].

ENZAMET Trial

The ENZAMET clinical trial, led by the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP), showed that adding enzalutamide to standard therapy (androgen deprivation therapy alone or combined with docetaxel) significantly improved overall survival for patients with advanced, hormone-sensitive prostate cancer. The trial results indicated that patients receiving enzalutamide were 30% less likely to die compared to those receiving standard treatment alone. These findings were presented at the ASCO 2022 Annual Meeting and highlighted the long-term benefits of enzalutamide treatment[4].

China ARCHES Study

In China, the ARCHES study, which is part of the global Phase 3 trials, also demonstrated consistent positive results for enzalutamide. This study was significant as it was the only Phase 3 clinical trial conducted specifically for Chinese patients, providing definitive clinical evidence for the treatment of advanced prostate cancer in this population. The approval of enzalutamide for the metastatic hormone-sensitive prostate cancer (mHSPC) indication in China further solidifies its role in comprehensive disease management[3].

Market Analysis

Global Sales

The global sales of XTANDI have shown a steady increase over the years. In 2020, the sales stood at USD 4,323.91 million, increasing to USD 4,756.66 million in 2021, and further to USD 4,883.96 million in 2022. By 2023, the sales had reached USD 5,192.90 million, reflecting a historical growth rate (CAGR) of 6.29% between 2020 and 2023[2].

Market Projections

The prostate cancer market, driven by the increasing prevalence of the disease and advancements in treatment options, is expected to grow significantly. The global prostate cancer market size was estimated at USD 11.87 billion in 2024 and is predicted to surpass USD 26.84 billion by 2034, with a CAGR of 8.50% during the forecast period. Androgen receptor-directed therapies, including enzalutamide and apalutamide, are expected to remain dominant, accounting for 58% of the total prostate cancer market by 2032[5].

Regional Market Dynamics

The Asia Pacific region is anticipated to be the fastest-growing market during the forecast period, driven by cost-effective treatments, technological advancements, and the rising incidence of prostate cancer. For instance, enzalutamide is significantly less expensive in Japan compared to the United States, making it a more accessible treatment option in this region[5].

Market Drivers and Challenges

Drivers

• Increasing Prevalence of Prostate Cancer: The rising number of prostate cancer cases globally is a significant driver for the market.
• Technological Advancements: Continuous innovations in treatment options, including vaccines, PARP inhibitors, kinase inhibitors, and PSMA-targeted radioligands, are driving market growth.
• Cost-Effective Treatments: The availability of cost-effective treatments, such as enzalutamide in certain regions, is increasing accessibility and demand[5].

Challenges

• Market Competition: The prostate cancer market is highly competitive, with multiple treatment options available.
• Regulatory Approvals: The process of obtaining regulatory approvals can be lengthy and challenging, affecting market entry and expansion.
• Economic Factors: Variations in pricing and reimbursement policies across different regions can impact market dynamics[5].

Expert Insights

Ahsan Arozullah, MD, MPH, Senior Vice President and Head of Oncology Development at Astellas, highlighted the significance of the China ARCHES study, stating that it provided definitive clinical evidence for Chinese clinicians in treating advanced prostate cancer. This underscores the importance of region-specific clinical trials in expanding treatment options[3].

Professor Ian Davis, Study Co-Chair and Board Chair of ANZUP, emphasized the collaborative nature of the ENZAMET trial and its impact on finding better treatment strategies for prostate cancer. The trial's results have been recognized with several awards, including the 2020 Trial of the Year Award[4].

Key Takeaways

• Enzalutamide has demonstrated significant clinical benefits in various stages of prostate cancer, including non-metastatic and metastatic hormone-sensitive prostate cancer.
• The drug has shown improved metastasis-free survival and overall survival in clinical trials such as the EMBARK and ENZAMET trials.
• Global sales of enzalutamide have been increasing steadily, with a projected growth in the prostate cancer market driven by technological advancements and increasing disease prevalence.
• Regional dynamics, particularly in the Asia Pacific, are expected to play a crucial role in the market's expansion due to cost-effective treatments and rising incidence rates.

FAQs

What is the mechanism of action of enzalutamide?

Enzalutamide acts as a triple-acting androgen receptor antagonist, inhibiting the activity of androgen receptors in prostate cancer cells, which reduces cell proliferation and decreases serum PSA levels[2].

What were the key findings of the EMBARK trial?

The EMBARK trial showed a statistically significant and clinically meaningful improvement in metastasis-free survival for patients with non-metastatic hormone-sensitive prostate cancer receiving enzalutamide plus leuprolide compared to other treatment arms[1].

How does the ENZAMET trial impact the treatment of prostate cancer?

The ENZAMET trial demonstrated that adding enzalutamide to standard therapy improves overall survival for patients with advanced, hormone-sensitive prostate cancer, reducing the risk of death by 30% compared to standard treatment alone[4].

What is the current market size and projected growth of the prostate cancer market?

The global prostate cancer market size was estimated at USD 11.87 billion in 2024 and is projected to reach USD 26.84 billion by 2034, with a CAGR of 8.50% during the forecast period[5].

Why is the Asia Pacific region expected to grow the fastest in the prostate cancer market?

The Asia Pacific region is expected to grow the fastest due to cost-effective treatments, technological advancements, and the rising incidence of prostate cancer, making treatments like enzalutamide more accessible[5].

Sources

1. Pfizer Inc. - Phase 3 Study Shows XTANDI® (enzalutamide) plus Leuprolide Significantly Improves Metastasis-Free Survival in Men with Non-Metastatic Prostate Cancer.
2. GlobalData - The Global Drug sales of Xtandi (2020 - 2026, USD Millions).
3. Astellas Pharma Inc. - China's National Medical Products Administration Approves XTANDI.
4. ANZUP - The updated results from the ENZAMET clinical trial show.
5. Precedence Research - Prostate Cancer Market Size To Hit USD 26.84 Billion By 2034.

Last updated: 2025-01-01