The preliminary results applied to the formulation’s use against nontuberculous mycobacteria, which are collectively known as the mycobacterium abscessus complex.
AIT said the NO-NTM Abscessus study showed that the formulation can improve patients’ quality of life, that it is safe, and that patients tolerate it well.
The findings prompted AIT to plan a pivotal trial in the United States in the second half of 2018. A pivotal trial is one that’s intended to provide evidence for a drug’s regulatory approval.
Nitric oxide is a critical player in a broad array of biological processes. When applied at specific doses directly to the airways it can boost the immune cells there, improving their ability to fight bacteria, fungi, yeast and parasites.
It can also help counter the multi-drug-resistant strains of bacteria that have appeared in the past few decades.
Information on three CF patients treated with AIT’s nitric oxide under the U.S. Food and Drug Administration’s compassionate use program showed that 21 days of treatment either eradicated nontuberculous mycobacteria or significantly reduced it. The compassionate use program allows people with life-threatening illnesses to obtain access to experimental treatments that have yet to be approved.
Nine cystic fibrosis patients received AIT’s nitric oxide formulation in the NO-NTM Abscessus trial. Researchers administered it for 21 days. In the first 14, patients received nitric oxide in five sessions of 30 minutes each. In the last seven days, they had three sessions a day.
In addition to nitric oxide, patients received antibiotics at doctors’ discretion.
To assess the therapy’s safety, researchers followed patients during the 21 days of treatment and up to 60 days afterward.
A month after treatment, patients’ respiratory function had improved by 3.7 percent, researchers said. The yardstick they used was a test known as forced expiratory volume in one second, or FEV1. This is the amount of air a person can exhale in a second after taking a deep breath.
Nitric oxide also improved patients’ distance in the 6-Minute Walk Test, a measure of exercise capacity.
Analysis of patients’ lung fluid at day 21 showed that nitric oxide eliminated M. abscessus in one of the nine patients.
“We are thrilled with the data collected to date from this trial,” Steve Lisi, AIT Therapeutics’ chairman and CEO, said in a press release. “The clear evidence of safety, coupled with positive data across several clinical outcome measures, which resulted in meaningful health benefits to patients, is a significant step forward in developing treatment options for patients suffering from this debilitating disease.
“Patients suffering from MABSC [the bacteria in the trial] have limited options, and everyone at AIT is honored to have this opportunity to continue on the path to provide our proprietary system with our NO [nitric oxide] generator as a treatment option,” Lisi added.
AIT expects to have complete trial results in the first quarter of 2018.
“These data are promising and exciting,” said Dr. Andrew Colin, director of the division of Pediatric Pulmonology at the University of Miami, the principal consultant for the trial. “Current treatments for MABSC have very limited efficacy and significant adverse effects. AIT’s system provides clinically meaningful benefits to the patient with no additional safety concerns.
“In my opinion, continued development of this therapy, with treatment over a longer duration, would be welcomed by NTM [nontuberculous mycobacteria] patients and their physicians as an addition to current treatment” regimens, Colin concluded.