CLINICAL TRIALS PROFILE FOR ARIKAYCE KIT

All Clinical Trials for Arikayce Kit

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00558844 ↗	Safety/Tolerability Study of Arikayce™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa	Completed	Insmed Incorporated	Phase 1/Phase 2	2008-01-01	This is a study to determine the safety and tolerability of 28 days of daily dosing of 560 mg of Arikayce™ versus placebo and daily dosing of 70 mg and 140 mg of Arikayce™ versus placebo in patients who have Cystic fibrosis (CF) and chronic infection due to pseudomonas aeruginosa.
NCT00775138 ↗	Safety and Tolerability Study of 2 Dose Level of Arikayce™ in Patients With Bronchiectasis and Chronic Infection Due to Pseudomonas Aeruginosa.	Completed	Insmed Incorporated	Phase 2	2008-06-24	This is a study to determine the safety and tolerability of 28 days of daily dosing of two doses (280 mg and 560 mg) of Arikayce™ versus placebo in patients who have bronchiectasis and chronic infection due to Pseudomonas infection.
NCT00777296 ↗	Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE™)	Completed	Insmed Incorporated	Phase 1/Phase 2	2007-02-22	A major factor in the respiratory health of cystic fibrosis (CF) subjects is acquisition of chronic Pseudomonas aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of CF subjects in the U.S. are infected. Liposomal Amikacin for Inhalation (Arikace™) is a sterile aqueous liposomal suspension consisting of amikacin sulfate encapsulated in liposomes. This formulation of amikacin maximizes the achievable dose and delivery to the lungs of subjects infected via a nebulizer. Because liposome particles are small enough to penetrate and diffuse through sputum into the bacterial biofilm, they deposit drug in close proximity to the bacterial colonies, thus improving the bioavailability of amikacin at the infection site. The clinically achievable doses of amikacin in the LAI formulation can effectively increase the half-life of the drug in the lungs, and decrease the potential for systemic toxicity. LAI offers several advantages over current therapies in treating CF subjects with chronic infection caused by P. aeruginosa.
NCT01315236 ↗	Liposomal Amikacin for Inhalation (LAI) for Nontuberculous Mycobacteria	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	2012-04-19	The purpose of this study is to evaluate the efficacy, safety and tolerability of 84 days of daily dosing of 590 mg of LAI versus placebo in patients with treatment refractory NTM lung disease. The first part of the study is the 84-day double-blind phase to evaluate the primary and secondary endpoints.

Clinical Trial Conditions for Arikayce Kit

Condition Name

Condition Name for Arikayce Kit
Intervention	Trials
Cystic Fibrosis	4
Bronchiectasis	1
Mycobacterium Infections, Nontuberculous	1
Pseudomonas Aeruginosa Infection	1

Condition MeSH

Condition MeSH for Arikayce Kit
Intervention	Trials
Cystic Fibrosis	5
Fibrosis	4
Infections	4
Pseudomonas Infections	3

Clinical Trial Locations for Arikayce Kit

Trials by Country

Trials by Country for Arikayce Kit
Location	Trials
United States	36
Canada	5
Hungary	5
Serbia	5
Poland	5

Trials by US State

Trials by US State for Arikayce Kit
Location	Trials
Pennsylvania	3
Maryland	2
Florida	2
Colorado	2
California	2

Clinical Trial Progress for Arikayce Kit

Clinical Trial Phase

Clinical Trial Phase for Arikayce Kit
Clinical Trial Phase	Trials
Phase 3	2
Phase 2	3
Phase 1/Phase 2	2

Clinical Trial Status

Clinical Trial Status for Arikayce Kit
Clinical Trial Phase	Trials
Completed	7

Clinical Trial Sponsors for Arikayce Kit

Sponsor Name

Sponsor Name for Arikayce Kit
Sponsor	Trials
Insmed Incorporated	7
National Institute of Allergy and Infectious Diseases (NIAID)	1

Sponsor Type

Sponsor Type for Arikayce Kit
Sponsor	Trials
Industry	7
NIH	1

Arikayce: Clinical Trials Update, Market Analysis, and Projections

Introduction to Arikayce

Arikayce, also known as amikacin liposome inhalation suspension, is a groundbreaking treatment for Mycobacterium avium complex (MAC) lung disease. Developed by Insmed, it was granted accelerated approval by the FDA in September 2018 for adult patients with limited or no alternative treatment options. Here, we will delve into the latest clinical trials, market analysis, and future projections for Arikayce.

Clinical Trials Update

ARISE Study

The ARISE study is a pivotal Phase 3 clinical trial that evaluated the efficacy and safety of Arikayce in patients with MAC lung disease. Here are the key findings:

• Patient Enrollment and Treatment: The study involved 99 patients randomized 1:1 to receive either Arikayce plus a macrolide-based background regimen or a placebo plus the same background regimen for six months, followed by one month off treatment[2][4].
• Outcomes: Patients treated with Arikayce showed significant improvements in quality of life (QOL) as measured by the QOL-B instrument. Specifically, 43.8% of Arikayce-treated patients achieved an improvement in QOL-B respiratory score above the meaningful within-subject score difference, compared to 33.3% in the comparator arm[2][4].
• Statistical Trends: Although the study was not powered to show a statistically significant difference between treatment arms, a strong trend toward significance was observed for improvement from baseline at Month 7[2].

ENCORE Trial

The ENCORE trial is an ongoing Phase 3b study designed to fulfill the FDA's post-marketing requirement for full approval of Arikayce and to support a supplemental new drug application.

• Study Design: This randomized, double-blind, placebo-controlled trial evaluates the efficacy and safety of Arikayce in patients with newly diagnosed or recurrent MAC lung disease who have not started antibiotics. Patients are randomized 1:1 to receive Arikayce plus a background regimen or a placebo plus the same regimen once daily for 12 months, followed by a three-month assessment period for the durability of culture conversion[2][4].
• Endpoints: The primary endpoint is the change from baseline to Month 13 in respiratory symptom score, while the key secondary endpoint is the proportion of patients achieving durable culture conversion at Month 15[2][4].

Safety and Adverse Reactions

Arikayce has been associated with several respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease. These reactions have led to hospitalizations in some cases[2][4][5].

Market Analysis

Current Market Performance

As of the first quarter of 2024, Arikayce generated total revenue of $75.5 million, reflecting a 16% annual growth over the same period in 2023. This growth indicates strong market acceptance and increasing adoption of the treatment[3].

Market Opportunity

The market opportunity for Arikayce is substantial, particularly in regions with high concentrations of NTM patients.

• U.S. Market: Estimated to have 80,000 to 90,000 diagnosed MAC patients, with 12,000 to 17,000 being refractory cases. The U.S. launch has seen strong initial uptake, with over 1,300 unique prescribers and positive reimbursement trends[5].
• EU and Japan: The EU5 (France, Germany, Italy, Spain, and the UK) has approximately 14,000 diagnosed NTM patients, with 1,400 being refractory MAC cases. Japan has an estimated 125,000 to 145,000 diagnosed NTM patients, with 15,000 to 18,000 being refractory MAC cases[5].

Commercial Approach and Expansion

Insmed is focusing on several key strategies to expand the market reach of Arikayce:

• U.S. Market: Continued commercial efforts are focused on key geographies with high patient concentrations. The company has seen positive physician feedback and a supportive payer landscape, with 55% of sales coming from Medicare, 36% from commercial payers, and 9% from other sources[5].
• Global Expansion: Plans are underway to expand Arikayce into the EU and Japan, leveraging the positive data from clinical trials to secure regulatory approvals and market access[5].

Future Projections

Clinical and Regulatory Milestones

• ENCORE Trial Completion: The successful completion of the ENCORE trial is crucial for achieving full FDA approval and potentially expanding the label to include all patients with MAC lung infections[2][4].
• Post-Marketing Requirements: Fulfilling the FDA's post-marketing requirements will further solidify Arikayce's position in the market and pave the way for broader indications[4].

Market Growth

Given the strong clinical data and growing market acceptance, Arikayce is poised for continued growth.

• Revenue Projections: With a current annual growth rate of 16%, Arikayce is expected to continue generating significant revenue. The expansion into new markets and potential label expansion could further accelerate this growth[3].
• Market Penetration: As more healthcare providers become familiar with Arikayce and its benefits, the treatment is likely to become a standard option for MAC lung disease, increasing its market penetration[5].

Key Takeaways

• Clinical Success: Arikayce has shown promising results in clinical trials, particularly in improving quality of life and achieving culture conversion in MAC lung disease patients.
• Market Growth: Strong revenue growth and positive market trends indicate a bright future for Arikayce.
• Safety Considerations: While Arikayce is associated with certain respiratory adverse reactions, its benefits outweigh the risks for many patients.
• Global Expansion: Insmed is actively working to expand Arikayce into new markets, which could significantly increase its global reach.

FAQs

What is Arikayce used for?

Arikayce is used for the treatment of Mycobacterium avium complex (MAC) lung disease in adult patients who have limited or no alternative treatment options.

What are the key findings from the ARISE study?

The ARISE study showed that patients treated with Arikayce had significant improvements in quality of life and respiratory symptoms compared to the placebo group.

What are the potential risks associated with Arikayce?

Arikayce is associated with an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease.

What is the current market performance of Arikayce?

As of the first quarter of 2024, Arikayce generated $75.5 million in revenue, reflecting a 16% annual growth.

What are the future projections for Arikayce?

Arikayce is expected to continue growing in revenue, with potential label expansion and global market penetration driving future growth.

Sources

1. Insmed Announces Positive Topline Results from Phase 3 ARISE Study of ARIKAYCE in Patients with NTM Lung Disease Caused by MAC. Investor Relations - Insmed.
2. Insmed Announces Positive Topline Results from Phase 3 ARISE Study of ARIKAYCE in Patients with NTM Lung Disease Caused by MAC. PR Newswire.
3. Insmed Reports First-Quarter 2024 Financial Results and Provides Business Update. Investor Relations - Insmed.
4. Insmed Presents Positive Patient-Reported Outcomes and Microbiologic Data from Phase 3 ARISE Study of ARIKAYCE in Patients with NTM Lung Disease Caused by MAC at American Thoracic Society. PR Newswire.
5. Investor Presentation. Insmed Investor Relations.