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.

Clinical Trial Conditions for Enzalutamide

Condition Name

Condition Name for Enzalutamide
Intervention	Trials
Prostate Cancer	120
Metastatic Castration-resistant Prostate Cancer	36
Metastatic Prostate Cancer	23
Castration-Resistant Prostate Carcinoma	22

Condition MeSH

Condition MeSH for Enzalutamide
Intervention	Trials
Prostatic Neoplasms	323
Adenocarcinoma	30
Carcinoma	29
Breast Neoplasms	19

Clinical Trial Locations for Enzalutamide

Trials by Country

Trials by Country for Enzalutamide
Location	Trials
United Kingdom	212
Canada	194
France	185
Spain	167
Japan	161

Trials by US State

Trials by US State for Enzalutamide
Location	Trials
New York	98
California	97
Texas	75
Maryland	72
Michigan	64

Clinical Trial Progress for Enzalutamide

Clinical Trial Phase

Clinical Trial Phase for Enzalutamide
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	11
PHASE2	17

Clinical Trial Status

Clinical Trial Status for Enzalutamide
Clinical Trial Phase	Trials
Recruiting	133
Completed	84
Active, not recruiting	75

Clinical Trial Sponsors for Enzalutamide

Sponsor Name

Sponsor Name for Enzalutamide
Sponsor	Trials
Medivation, Inc.	59
National Cancer Institute (NCI)	55
Astellas Pharma Inc	51

Sponsor Type

Sponsor Type for Enzalutamide
Sponsor	Trials
Industry	392
Other	385
NIH	57

Enzalutamide: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

Introduction

Enzalutamide, marketed as Xtandi, is an oral androgen receptor inhibitor primarily approved for treating metastatic castration-resistant prostate cancer (mCRPC). Since its FDA approval in 2012, it has established itself as a critical therapy in prostate cancer management, with ongoing clinical trials expanding its indications. This comprehensive analysis covers recent clinical trial updates, market dynamics, competitive positioning, and future forecasts for enzalutamide.

---

Clinical Trials Update

Recent Developments and Ongoing Trials

Since its initial approval, enzalutamide's clinical development has been extensive, with multiple Phase III trials demonstrating its efficacy and safety.

• PREVAIL Trial (2014): This pivotal study confirmed enzalutamide's efficacy in chemotherapy-naïve mCRPC patients, showing significant improvements in overall survival (OS) and radiographic progression-free survival (rPFS) compared to placebo. The trial led to FDA approval for this indication.

• ARCHES Trial (2020): Focused on hormone-sensitive metastatic prostate cancer, this Phase III trial evaluated enzalutamide combined with androgen deprivation therapy (ADT). Results showed a 42% reduction in radiographic progression risk and significant improvement in progression-free survival (PFS). Based on these findings, enzalutamide received approval for hormone-sensitive disease.

• ENZAMET Trial (2021): This study compared enzalutamide with standard non-steroidal anti-androgens. Data indicated increased progression-free survival and overall survival with enzalutamide, reinforcing its role across different disease stages.

Emerging Trials and Additional Indications

Clinical trials are exploring enzalutamide's efficacy beyond prostate cancer:

• Non-metastatic Castration-Resistant Prostate Cancer (nmCRPC): Trials like PROSPER demonstrated that enzalutamide prolongs metastasis-free survival, leading to expanded indications.

• Potential in Breast Cancer: Early-phase studies assess enzalutamide's role in androgen receptor-positive triple-negative breast cancer (TNBC). Although preliminary, results are promising, warranting further investigation.

• Combination Regimens: Ongoing trials examine enzalutamide in combination with immunotherapies (e.g., PD-1 inhibitors), chemotherapies, and other targeted agents to enhance therapeutic outcomes.

Safety Profile Updates

Recent data reaffirm enzalutamide's manageable safety profile, with common adverse effects including fatigue, hypertension, hot flashes, and less frequently, seizures. Long-term safety data emphasize ongoing monitoring, especially in combination therapies.

---

Market Analysis

Current Market Landscape

Global Market Valuation

The enzalutamide market was valued at approximately $2.8 billion in 2022. It is projected to grow at a CAGR of 8-10% over the next five years, driven by expanding indications, improved survival rates, and increased global adoption.

Geographic Distribution

• North America dominates the market owing to high prostate cancer prevalence and established healthcare infrastructure.
• Europe follows closely, with widespread adoption facilitated by regulatory approvals.
• Emerging Markets (Asia-Pacific, Latin America, Middle East) are expected to exhibit robust growth owing to increased healthcare access and awareness.

Market Drivers

• Expanded Indications: Efficacy in hormone-sensitive and non-metastatic settings broadens patient eligibility.
• Competitive Edge: Compared to alternatives like abiraterone, enzalutamide offers oral administration, fewer dietary restrictions, and favorable safety profiles.
• Technological Advancements: Improved diagnostic tools lead to earlier detection and treatment, increasing demand.
• Rising prostate cancer Incidence: Estimated to reach 1.4 million new cases globally in 2023 [1], ensuring sustained demand.

Market Challenges

• Patent Expiry and Generic Competition: Enzalutamide's patent protection is set to expire around 2029, risking market share erosion by generics.
• Pricing Pressures: Healthcare systems are increasingly emphasizing cost-effective treatments, possibly impacting profitability.
• Side Effect Management: While generally safe, adverse events might influence prescribing behaviors.

Competitive Landscape

• Other androgen receptor inhibitors: Apalutamide (Erleada) and darolutamide (Nubeqa) directly compete, offering similar efficacy with different safety profiles.
• Chemotherapy and other targeted therapies: Used in later lines of therapy, shaping a complex treatment ecosystem.

---

Future Market Projections

Forecasts (2023–2030)

The market for enzalutamide is expected to experience steady growth, reaching an estimated $5.5–$6.0 billion by 2030. The key factors include:

• Broadened Labeling: Anticipated expansion into earlier disease stages and additional cancers.
• Innovative Combinations: Trials demonstrate promising results when combining enzalutamide with immunotherapies and targeted agents, potentially enhancing outcomes and coverage.
• Global Expansion: Increasing access in emerging markets, supported by local manufacturing and licensing agreements.

Influence of Emerging Therapies

• Gene and Precision Medicine: Evolving understandings of tumor biology may lead to combination treatments that could either complement or compete with enzalutamide.
• Biosimilars: Patent expirations will catalyze market entry of biosimilars, potentially reducing costs but also altering competitive dynamics.

Regulatory and Healthcare Policy Impact

• Regulatory agencies’ support for earlier-stage prostate cancer treatments is expected to facilitate broader usage.
• Price negotiations and healthcare reforms may influence market share and pricing strategies.

---

Key Takeaways

• Clinical Leadership: Enzalutamide remains a benchmark in mCRPC treatment, with ongoing trials expanding its indications and improving survival outcomes.
• Market Expansion: The global prostate cancer burden, combined with evolving treatment guidelines, positions enzalutamide for continued market growth, especially in emerging markets.
• Competitive Dynamics: The presence of similar androgen receptor inhibitors necessitates differentiation through efficacy, safety, cost, and combination strategies.
• Intellectual Property: Patent expiry around 2029 presents both challenges and opportunities; early planning in biosimilars and second-generation formulations is critical.
• Future Outlook: Technological and therapeutic advances, including precision medicine and combination regimens, will shape enzalutamide’s role and market trajectory over the next decade.

---

Frequently Asked Questions

1. When will enzalutamide's patent expire, and what are the implications?
Patent protection is expected to expire around 2029. This may lead to the introduction of biosimilars, increasing competition, and potentially reducing prices. Companies are therefore exploring extended patent protections and formulation patents to maintain market share.

2. Are there ongoing trials investigating enzalutamide in combination therapies?
Yes. Recent and ongoing studies evaluate its combination with immunotherapies, chemotherapies, and targeted agents to enhance efficacy in prostate and other cancers, potentially extending its therapeutic applications.

3. How does enzalutamide compare to other androgen receptor inhibitors?
Enzalutamide generally offers similar efficacy to apalutamide and darolutamide but has a distinct safety profile; for instance, darolutamide is associated with fewer central nervous system side effects. Choice depends on individual patient considerations and tolerability.

4. What are the primary safety concerns associated with enzalutamide?
Adverse events include fatigue, hypertension, hot flashes, and rare occurrences of seizures. Long-term safety data supports its manageable safety profile, but clinicians should monitor neurological and cardiovascular health during treatment.

5. What is the outlook for enzalutamide’s use in non-prostate cancers?
Preliminary research in androgen receptor-positive triple-negative breast cancer indicates potential, but this remains investigational. Further clinical trials are necessary before regulatory approval and widespread use can be anticipated.

---

References

[1] International Agency for Research on Cancer (IARC), Global Cancer Statistics 2023.