Arikayce (liposomal amikacin for inhalation, also known as LAI) is an investigational drug being developed by the pharmaceutical company Insmed for the treatment of serious chronic lung infections, such as non-tuberculosis mycobacterium (NTM).
NTM is caused by bacteria found in the soil and water, and its prevalence is increasing worldwide. Common symptoms include fever, weight loss, blood in the sputum, cough, and fatigue. Research has shown that chronic lung diseases, such as COPD, are risk factors for NTM.
How Arikayce works
Arikayce’s active ingredient is amikacin, which belongs to the aminoglycoside group of antibiotics that are used to treat gram-negative bacterial infections. Amikacin is normally administered as an injection directly into the bloodstream or the muscles.
In Arikayce, amikacin is encapsulated in a liposome — a microscopic membrane shell that contains water — so that it can be inhaled, allowing the drug to be delivered directly to the site of infection.
In a bacterial infection, bacteria protect themselves by producing biofilms, which are negatively charged. Because opposite charges attract each other, when not encapsulated in liposome the positively charged amikacin would bind to the biofilms, trapping it before it could reach the bacteria. Liposome-encapsulated amikacin, however, is delivered directly to the lung where it is taken up by immune system cells called macrophages, where the NTM infection resides.
An efficient therapy-delivery system, Arikayce is still being developed and tested.
Arikyace in clinical trials
A randomized, double-blind and placebo-controlled Phase 2 study (NCT01315236) evaluated the effectiveness, safety, and tolerability of Arikayce in patients with NTM lung infection. Participants were randomized to either receive a once-daily dose of Arikyace (590 mg) or a placebo during an 84-day treatment period.
The primary endpoint was the change of mycobacterial density from baseline on a seven-point scale. Results showed that although there was no statistical significance in the primary endpoint, Arikayce was able to achieve culture conversion (three consecutive negative monthly sputum samples) in a shorter period compared to a placebo.
Trial participants had the option to enroll in an open-label, 84-day extension during which all of the patients received Arikayce plus standard care (an anti-mycobacterial drug) and were followed for 12 months. Eighty-nine patients started the trial and 59 completed it. Results showed that most participants continued to have negative sputum cultures in both the open-label and follow-up phases. Those taking Arikayce from the start continued to improve in the mean distance walked in six minutes, while those who switched from placebo to treatment in the open-label trial demonstrated a reduced rate of decline from baseline.
A second Phase 2 open-label extension study to test the safety, effectiveness, and tolerability of Arikayce taken once daily for 12 months is currently recruiting participants (NCT03038178). The study will assess the change in baseline sputum culture (a test of lung infection) and the change in a six-minute walking test as well as monitor for side effects.
Arikayce also is being evaluated in a global Phase 3 study (NCT02344004) with 350 patients with NTM lung infections. Participants were randomized to either receive Arikayce plus standard care or just standard care. The study is expected to end in July 2019.
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