September 05, 2023
2 min read
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Key takeaways:
- Nearly half of patients reported improvements to quality of life compared with placebo.
- Nontuberculous mycobacteria are widely found in water systems in residential, office and health care facilities.
A drug that treats nontuberculous mycobacterial lung infections caused by Mycobacterium avium complex performed well among newly diagnosed patients who had not started antibiotics, its manufacturer said.
In 2018, Arikayce became the first treatment approved under the FDA’s Limited Population Pathway for Antibacterial and Antifungal Drugs, which streamlines the development of drugs meant to treat serious but rare infections. It was approved for patients who cannot be treated with conventional M. avium therapies.
Arikayce resulted in an improved quality of life for people with newly diagnosed or recurrent nontuberculous mycobacterial lung infections. Image: Adobe Stock
Insmed, the company that makes Arikayce (amikacin liposome inhalation suspension), said in a press release that it plans to work with regulators to accelerate the approval of Arikayce for newly infected patients.
The company said the ongoing phase 3b ENCORE trial will continue to enroll participants into 2024, and that topline data from the trial are expected in 2025.
According to results announced Tuesday by Insmed, Arikayce resulted in an improved quality of life for people with newly diagnosed or recurrent nontuberculous mycobacterial (NTM) lung infections caused by M. avium complex based on patient-reported outcomes.
NTM are common in the environment and can form biofilms that survive in water systems in residential, office and health care facilities, and are resist to chlorination, according to the CDC.
People with underlying lung disease or who are immunosuppressed are most at risk for NTM infection, although it is typically not transmitted from person to person.
Just under 90,000 people in the United States have NTM, according to the American Lung Association, with treatment generally requiring several antibiotics for multiple years. Experts have said lifestyle changes such as exercise and diet improvements can help alleviate symptoms.
“The ARISE study represents a clear and unambiguous win for the entire NTM community,” Insmed Chief Medical Officer Martina Flammer, MD, MBA, said in the press release. “We are thrilled that these results validate a [patient-reported outcome] tool in NTM lung disease, but also show that patients treated with an Arikayce-based regimen felt better versus patients in the comparator arm.”
The phase 3 ARISE trial enrolled 99 people who were randomly assigned in a 1:1 ratio to receive either Arikayce plus macrolide-based background regimen or placebo plus macrolide-based background regimen for 6 months, followed by 1 month off treatment.
Among patients in the treatment group, as measured by a Quality of Life instrument, 43.8% of people achieved an improvement in quality of life compared with 33.3% of people in the placebo group, according to Insmed.
The company said a strong trend toward improvement was seen from baseline at month 7, and people in the treatment group achieved higher culture conversion rates at month 7 compared with patients in the placebo group (78.8% vs. 47.1%), although the trial was not powered to show a statistically significant difference between treatment arms.
“We look forward to discussing these excellent results from this well-executed study in the near future with regulators,” Insmed’s Chief Development Officer Kevin Mange, MD, MSCE, said in the release.
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