Arikayce (also known as liposomal amikacin for inhalation, or LAI) is being developed by Insmed as a possible inhalation treatment for serious lung conditions, such as bronchiectasis. Arikayce consists of liposomes that contain amikacin, an antibiotic used to treat severe or serious bacterial infections, such as non-tuberculosis mycobacterium pulmonary disease (NTM-PD).
How Arikayce works
The active ingredient of Arikayce is amikacin. This is an aminoglycoside antibiotic that works by interrupting bacterial protein synthesis after binding to the bacteria. Amikacin is used to treat infections caused by aerobic, gram-negative bacteria and mycobacteria, the bacteria that cause tuberculosis, which can lead to serious lung complications in people with bronchiectasis.
Normal inhaled amikacin can be inefficient in treating NTM-PD, as the bacteria can produce a negatively-charged substance that can attract and trap the positively charged amikacin before it can reach the bacteria.
Arikayce uses liposomes to bypass this. Liposomes are microscopic phospholipid spheres that contain water, and in Arikayce, the water-soluble amikacin can be contained at high concentrations inside the liposome. Insmed’s liposomal technology is designed to deliver amikacin directly to the site of infection in the lungs for immediate treatment and to maintain the antibiotic locally in the lungs over time.
In recent years, NTM-PD has come to represent a high risk in terms of its incidence, prevalence, hospitalization rate, and mortality rate, and people with chronic pulmonary conditions like bronchiectasis are particularly susceptible. NTM-PD symptoms often overlap with those of the underlying disease, so that NTM-PD is often diagnosed at a later stage. Treatments that might address this disease, especially at earlier stages, are of growing interest.
Arikayce in clinical trials
A Phase 2 study (NCT01315236) assessed the effectiveness, safety, and tolerability of Arikayce, versus placebo, in about 90 people with chronic NTM-PD who failed to respond to standard antibiotics. Treated patients were given 590 mg of the inhalation therapy, once daily, for 84 days. Trial results showed that Arikayce is efficient in treating people with Mycobacterium avium complex infection, but that the treatment was not superior to placebo for those patients with Mycobacterium abcessus infection.
Participants completing the Phase 2 trial had the option of continuing Arikayce in an open-label extension study. The 59 patients who completed the extension study received Arikayce for 84-days plus standard care (an anti-mycobacterial regimen), and were monitored for 12 months. The results supported those of the Phase 2 study. Participants who achieved negative sputum cultures continued to be free of infection in both the open-label and follow-up phases. Patients who were assigned Arikayce in the Phase 2 study showed a continued improvement 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.
A second open-label extension study is recruiting participants (NCT03038178). The Phase 2 study aims to test the safety, effectiveness, and tolerability of Arikayce taken once daily for 12 months. The primary goal of the study is the change in baseline sputum culture (a test for lung infection). The patients will be monitored for adverse events due to Arikayce and other measures, such as the change in six-minute walk test, will be assessed.
Insmed has an ongoing Phase 3 study (NCT02344004) called CONVERT evaluating Arikayce in about 350 people with drug-resistant NTM-PD, and expects to release top-line data in the second half of 2017. The trial’s primary goal is the proportion of patients who achieve different cultures of bacteria after six months of treatment with Arikayce plus a standard regimen of anti-mycobacterial medications, compared to those treated with the standard regimen alone.
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