Arikayce (liposomal amikacin for inhalation, also known as LAI) is an investigational drug being developed by Insmed Pharmaceutical for the treatment of serious chronic lung infections, such as non-tuberculosis mycobacterium.

Non-tuberculosis mycobacterium (NTM) is caused by a bacteria found in the soil and water, and its prevalence is increasing worldwide. Common symptoms include fever, weight loss, blood in the sputum, cough or 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 blood stream or 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 mucus and biofilms, which are negatively charged compounds. As opposite charges attract each other, amikacin, which is positively charged, binds to mucus and biofilms.

Insmed’s liposomal technology, which is still being developed and tested, is designed to deliver high levels of amikacin to the infection site to be taken up by immune system cells called macrophages, resulting in an efficient drug delivery system.

Studies of ARIKAYCE for COPD

A randomized, double-blind and placebo-controlled Phase 2 study (NCT01315236) evaluated the efficacy, safety, and tolerability of Arikayce in patients with NTM lung infection. Participants were randomized to either receive a once-daily dose of Arikayce (590 mg) or a placebo during an 84-day treatment period.

The primary endpoint was the change from baseline of mycobacterial density 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 placebo.

Trial participants had the option to enroll in an  open-label, 84-day extension study (NCT03038178), during which all received Arikayce plus standard care (an anti-mycobacterial regimen), and were followed for 12 months. Seventy-five patients started and 59 completed the extension trial.  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 the six-minute walk test, while those who switched from placebo to treatment in the open-label trial  demonstrated a reduced rate of decline from baseline.

Arikayce is currently being evaluated in a global Phase 3 study (NCT02344004) in about 350 people with NTM lung infections, with participants randomized to either receive Arikayce plus standard care or just standard care. This study is expected to end in July 2019.

Other details

LAI is also being studied as a treatment for cystic fibrosis patients with chronic pseudomonas aeruginosa infection. In a Phase 3 study (NCT01315678), the efficacy and safety of Arikace, as the drug is labeled for CF patients, were compared to that of Tobi (an inhaled antibiotic). Results showed that the drug is generally safe and well-tolerated, with no adverse events observed. Participants taking Arikayce improved their respiratory system compared to Tobi.

Insmed announced that liposomal amikacin for inhalation was issued a U.S. patent in February 2017.

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