Nitric Oxide Delivered to First Patient Enrolled in Phase 2 Trial for Serious Respiratory Infection

Nitric Oxide Delivered to First Patient Enrolled in Phase 2 Trial for Serious Respiratory Infection

The first patient enrolled in a Phase 2 trial for patients with a serious infection — Mycobacterium abscessus complex (MABSC) — has successfully received the first dose of nitric oxide (NO), according to AIT Therapeutics, the study’s sponsor.

The trial expects to enroll 10 patients with nontuberculous mycobacteria (NTM) with MABSC. This condition affects many patients with respiratory conditions, including those with bronchiectasis.

“We are excited to advance this potentially life-changing therapy for NTM patients with the dosing of the first patient in our trial,” Steve Lisi, CEO of AIT Therapeutics, said in a press release. “MABSC is a difficult to treat infection with a high unmet medical need.”

Intensive nitric oxide treatment for three weeks may prevent the need for “long term, toxic and costly antibiotic therapy, significantly benefiting patients suffering with this disease,” Lisi said.

NTM infection is a “rare and serious condition causing debilitating pulmonary disease associated with increased morbidity and mortality,” according to AIT Therapeutics. NTM is a public health concern worldwide because of its multi-drug antibiotic resistance, the company states.

“MABSC comprises a group of rapidly growing, multi-drug resistant bacteria that are responsible for a wide spectrum of pulmonary diseases predominantly in vulnerable hosts with underlying structural lung disease such as cystic fibrosis, bronchiectasis, COPD, and lung cancer,” said Andrew Colin, MD, professor of cystic fibrosis and pediatric pulmonology at the Miller School of Medicine at the University of Miami.

“The conventional anti-microbial therapies are disappointing and new treatment modalities are urgently needed,” added Colin, also director of the pediatric CF program at the Miller School of Medicine. “At this time, nitric oxide shows great promise.”

The Phase 2 trial was initiated after a previous study showed that inhaling NO gas was safe and effective in several respiratory diseases, including CF, bronchiolitis, and NTM. Specifically, NO was shown to effectively eliminate several pathogens (bacteria, viruses, fungi) from infected lungs, and also may decrease the ability of bacteria to acquire resistance.

Enrolled patients will inhale NO for 30 minutes at a concentration of 160 ppm (parts per million). The treatment will be conducted over 21 days, with NO being administered three to five times a day.

The study’s primary goal is to confirm the safety of NO therapy, which will be achieved by measuring the rate of NO-related serious adverse events during the treatment period. Patients also will perform a six-minute walk test and undergo sputum analysis for Mycobacterium abscessus (secondary goals). The trial’s estimated completion date is April 2018, with a data collection date of December 2017.

AIT Therapeutics is involved in additional clinical trials, including a Phase 3 trial in children who are hospitalized with bronchiolitis.