CLINICAL TRIALS PROFILE FOR MACITENTAN

All Clinical Trials for Macitentan

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00660179 ↗	Study of Macitentan (ACT-064992) on Morbidity and Mortality in Patients With Symptomatic Pulmonary Arterial Hypertension	Completed	Actelion	Phase 3	2008-05-01	The AC-055-302/SERAPHIN study will be an event-driven Phase III study, comparing two different doses of macitentan (ACT-064992) (3 and 10 mg) vs placebo in patients with symptomatic PAH. The main study objective is to demonstrate that macitentan (ACT-064992) prolongs time to the first morbidity or mortality event, and to evaluate the benefit/risk profile of macitentan (ACT-064992) in the treatment of patients with symptomatic PAH.
NCT00667823 ↗	Clinical Study to Assess the Long-term Safety and Tolerability of ACT 064992 in Patients With Symptomatic Pulmonary Arterial Hypertension	Completed	Actelion	Phase 3	2008-10-17	The main objective of the AC 055 303/SERAPHIN OL study, which will follow the AC 055 302/SERAPHIN study, will be to assess the long-term safety and tolerability of ACT 064992 in patients with symptomatic PAH.
NCT00903331 ↗	Macitentan Use in an Idiopathic Pulmonary Fibrosis Clinical Study	Completed	Actelion	Phase 2	2009-05-01	The AC-055B201/MUSIC study is a Phase II study, comparing one dose of ACT-064922 (macitentan) 10 mg with placebo in patients with idiopathic pulmonary fibrosis (IPF). The main study objective is to demonstrate that macitentan positively affects the forced vital capacity (FVC) in comparison with placebo in patients with idiopathic pulmonary fibrosis (IPF). The secondary objectives are to evaluate the effect of macitentan on the time to disease worsening or death in patients with IPF, and to evaluate the benefit/risk profile of macitentan in the treatment of patients with IPF.
NCT01346930 ↗	Safety and Tolerability Study of Macitentan in Patients With Idiopathic Pulmonary Fibrosis	Withdrawn	Actelion	Phase 2	2011-07-01	The MUSIC OL study is an open-label extension study, in which all eligible patients having completed the double-blind AC-055B201/MUSIC study as scheduled receive macitentan 10 mg once daily. The study objective is to assess the long-term safety and tolerability of macitentan in patients with idiopathic pulmonary fibrosis (IPF).
NCT01474109 ↗	Macitentan for the Treatment of Digital Ulcers in Systemic Sclerosis Patients	Completed	Actelion	Phase 3	2011-12-01	The DUAL-1 study is designed as a multicenter, double-blind two-period study with an initial fixed 16-week Period 1, followed by a Period 2 of variable duration. All patients completing Period 1 will continue on their original randomized treatment into Period 2, until the last randomized patient has completed Period 1. Patients will be randomized in a 1:1:1 ratio (macitentan 3mg: macitentan 10mg: placebo). The primary objective is to demonstrate the effect of macitentan on the reduction of the number of new digital ulcers in patients with systemic sclerosis and ongoing digital ulcers. Other objectives include: - the evaluation of the efficacy of macitentan on hand functionality and DU burden at Week 16 in SSc patients with ongoing DU disease. - the evaluation of the safety and tolerability of macitentan in these patients. - the evaluation of the efficacy of macitentan on time to first DU complication during the entire treatment period.
NCT01474122 ↗	Macitentan for the Treatment of Digital Ulcers in Systemic Sclerosis Patients	Terminated	Actelion	Phase 3	2011-12-01	The DUAL-2 study is designed as a multicenter, double-blind two-period study with an initial fixed 16-week Period 1, followed by a Period 2 of variable duration. All patients completing Period 1 continue on their original randomized treatment into Period 2, until the last randomized patient has completed Period 1. Patients are randomized in a 1:1:1 ratio (macitentan 3mg: macitentan 10mg: placebo). The primary objective is to demonstrate the effect of macitentan on the reduction of the number of new digital ulcers in patients with systemic sclerosis and ongoing digital ulcers (DU). Other objectives include: - the evaluation of the efficacy of macitentan on hand functionality and DU burden at Week 16 in SSc patients with ongoing DU disease. - the evaluation of the safety and tolerability of macitentan in these patients. - the evaluation of the efficacy of macitentan on time to first DU complication during the entire treatment period.
NCT01499251 ↗	Clinical Study on the Safety and Tolerability of Macitentan in Combination With Dose-dense Temozolomide in Patients With Recurrent Glioblastoma	Terminated	Actelion	Phase 1	2012-01-01	This is an open-label, single arm, Phase 1 study to assess the safety and tolerability of macitentan in combination with dose-dense temozolomide in adult patients with recurrent glioblastoma or gliosarcoma. The study is composed of three parts. A Phase 1 Dose Escalation Period with a traditional 3+3 design will determine the maximum tolerated dose of macitentan in combination with dose-dense temozolomide. A Phase 1b Period will expand the safety and tolerability data of two doses of macitentan and dose-dense temozolomide selected from the Dose Escalation Period and explore efficacy. An Ancillary Study will further evaluate the effects of macitentan on biomarkers in brain tumor tissue. The study is planned to have a minimum duration of 12 months. The study will end when all patients (excluding those prematurely withdrawn or lost to follow-up) in each part of the study have completed a visit at month 12 and 30 days of safety follow-up.

Clinical Trial Conditions for Macitentan

Condition Name

Condition Name for Macitentan
Intervention	Trials
Pulmonary Arterial Hypertension	17
Healthy	9
Pulmonary Hypertension	4
Chronic Thromboembolic Pulmonary Hypertension	4

Condition MeSH

Condition MeSH for Macitentan
Intervention	Trials
Hypertension	31
Pulmonary Arterial Hypertension	20
Familial Primary Pulmonary Hypertension	19
Hypertension, Pulmonary	12

Clinical Trial Locations for Macitentan

Trials by Country

Trials by Country for Macitentan
Location	Trials
United States	369
Canada	31
France	26
Germany	25
China	21

Trials by US State

Trials by US State for Macitentan
Location	Trials
Texas	22
Ohio	20
California	19
Massachusetts	17
Arizona	16

Clinical Trial Progress for Macitentan

Clinical Trial Phase

Clinical Trial Phase for Macitentan
Clinical Trial Phase	Trials
Phase 4	6
Phase 3	22
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for Macitentan
Clinical Trial Phase	Trials
Completed	26
Terminated	10
Recruiting	10

Clinical Trial Sponsors for Macitentan

Sponsor Name

Sponsor Name for Macitentan
Sponsor	Trials
Actelion	47
Medidata Solutions	2
University of California, Los Angeles	2

Sponsor Type

Sponsor Type for Macitentan
Sponsor	Trials
Industry	59
Other	30

Macitentan: Clinical Trials Update, Market Analysis, and Projections

Introduction to Macitentan

Macitentan is an endothelin receptor antagonist used primarily in the treatment of pulmonary arterial hypertension (PAH) and, in some cases, chronic thromboembolic pulmonary hypertension (CTEPH). It is marketed under the brand name OPSUMIT.

Clinical Trials Update

MACiTEPH Study

In a significant update, Janssen Pharmaceutical Companies announced the termination of the Phase 3 MACiTEPH study. This study was designed to evaluate the efficacy and safety of macitentan 75 mg in patients with CTEPH. The decision to stop the trial was based on a recommendation from the independent data monitoring committee following a pre-planned interim analysis, which indicated futility. No new safety signals were observed during the study[1].

UNISUS Study

Despite the termination of the MACiTEPH study, the UNISUS study, another Phase 3 clinical trial, is ongoing. The UNISUS study aims to demonstrate the superiority of macitentan 75 mg over the currently approved 10 mg dose in patients with PAH. This event-driven study is enrolling approximately 900 participants and focuses on the time to first clinical worsening events, including all-cause death, PAH-related hospitalization, and worsening of PAH requiring specific interventions[4].

Market Analysis

Global Market Overview

The global macitentan market is expected to grow significantly over the next few years. The market is segmented by type, application, and region.

• Type: The market is categorized into different purity levels of macitentan, including 99%, 98%, 97%, and 95% purity types. Each type has its own market share and growth prospects[2].
• Application: Macitentan is used primarily as an original drug and also as a generic drug. The original drug segment is expected to dominate the market due to its established brand and efficacy[2].

Regional Market Analysis

The global macitentan market is analyzed across various regions, including North America, Europe, Asia-Pacific, South America, and the Middle East and Africa.

• Asia-Pacific: This region is expected to show significant growth due to increasing healthcare expenditure and a growing patient population. Countries like China, Japan, and India are key contributors to this growth[2].
• North America and Europe: These regions are expected to maintain their market share due to well-established healthcare systems and high adoption rates of advanced treatments[2].

Market Size and Forecast

The global macitentan market size is projected to increase from 2023 to 2030, driven by increasing demand for effective treatments for PAH and CTEPH. The market is expected to grow in terms of both sales quantity and consumption value. The average price of macitentan is also anticipated to stabilize as the market becomes more competitive[2][5].

Market Projections

Growth Prospects

The macitentan market is expected to benefit from several growth drivers:

• Increasing Prevalence of PAH and CTEPH: The rising incidence of these conditions globally will drive the demand for effective treatments.
• Advancements in Clinical Trials: Ongoing and future clinical trials, such as the UNISUS study, may lead to expanded indications and improved efficacy profiles.
• Market Penetration in Emerging Markets: Growing healthcare infrastructure and increasing awareness in emerging markets will open new opportunities for macitentan[5].

Challenges and Barriers

Despite the growth prospects, the market faces several challenges:

• Competition from Other Therapies: The PAH treatment market is highly competitive, with several other drugs and therapies available.
• Regulatory Hurdles: Strict regulatory requirements and the need for extensive clinical trials can slow market entry and expansion.
• Pricing and Accessibility: High drug prices can limit accessibility, especially in developing regions[2][5].

Competitive Landscape

The macitentan market is characterized by a competitive landscape with several key players:

• Janssen Pharmaceutical Companies: As the primary developer and marketer of OPSUMIT, Janssen plays a significant role in the market.
• Other Manufacturers: Companies like Bio Crick are also involved in the production and distribution of macitentan, contributing to the market's competitive dynamics[2].

Product Development and Innovation

The macitentan market is driven by continuous innovation and product development:

• New Therapeutic Targets: Research into new therapeutic targets, such as metabolic dysfunction in PAH, is ongoing and may lead to future advancements[3].
• Dose Optimization: Studies like the UNISUS trial aim to optimize the dosing of macitentan to improve efficacy and safety profiles[4].

Key Takeaways

• Clinical Trials: The MACiTEPH study was terminated due to futility, while the UNISUS study continues to evaluate the superiority of macitentan 75 mg in PAH patients.
• Market Growth: The global macitentan market is projected to grow driven by increasing demand, advancements in clinical trials, and market penetration in emerging regions.
• Competitive Landscape: The market is competitive with key players like Janssen and other manufacturers contributing to its dynamics.
• Challenges: The market faces challenges such as competition from other therapies, regulatory hurdles, and pricing issues.

FAQs

What is the current status of the MACiTEPH study?

The MACiTEPH study was terminated due to futility based on a pre-planned interim analysis.

What is the primary objective of the UNISUS study?

The UNISUS study aims to demonstrate the superiority of macitentan 75 mg over the currently approved 10 mg dose in patients with PAH.

What are the key regions driving the growth of the macitentan market?

The Asia-Pacific region, along with North America and Europe, are expected to be key drivers of the macitentan market growth.

What are the main challenges facing the macitentan market?

The market faces challenges such as competition from other therapies, regulatory hurdles, and pricing issues that affect accessibility.

How is the competitive landscape of the macitentan market?

The market is competitive with Janssen Pharmaceutical Companies being a major player, along with other manufacturers like Bio Crick.

Sources

1. Janssen Pharmaceutical Companies of Johnson & Johnson. Janssen Announces Update to Phase 3 MACiTEPH Study Evaluating Macitentan 75 mg in Patients with Chronic Thromboembolic Pulmonary Hypertension (CTEPH). September 6, 2023.
2. Business Growth Reports. Detailed TOC of Global Macitentan Market 2024 by Manufacturers, Regions, Type and Application, Forecast to 2030.
3. European Respiratory Journal. Clinical trial design and new therapies for pulmonary arterial hypertension. 2023.
4. Janssen Science. OPSUMIT - Macitentan 75 mg in Treatment of Pulmonary Hypertension.
5. 360iResearch. Macitentan Tablets Market Size & Share 2025-2030.