No Serious Adverse Events Related to Treatment with Nitric Oxide Reported
Compelling Preliminary Data on Key Secondary Endpoint of 6-Minute Walk
NEW YORK, Nov. 27, 2017 — AIT Therapeutics Inc. (AITB), a clinical-stage biopharmaceutical company focused on developing inhaled Nitric Oxide (NO) for the treatment of patients with serious lung infections and pulmonary hypertension, today announced that preliminary results from its NO-NTM Phase 2 study in Nontuberculous Mycobacteria (NTM) targeting patients suffering from Mycobacterium Abscessus Complex (MABSC) indicate that the primary endpoint of safety was met, with no NO-related serious adverse events (SAEs) observed. The safety data, along with positive results in other key parameters, drive AIT’s decision to move the program forward and plan for a potential pivotal trial to begin in the United States in the second half of 2018.
“We are thrilled with the data collected to date from this trial. 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,” said Steve Lisi, Chairman and Chief Executive Officer of AIT Therapeutics. “Patients suffering from MABSC 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 generator as a treatment option for NTM patients.”
All patients in the trial were refractory to the standard-of-care for MABSC and had underlying cystic fibrosis. Patients were treated with inhaled NO in addition to background antibiotic therapy at the physicians’ discretion. NO was delivered at a concentration of 160ppm for 30 minutes intermittently for 21 days (5 times per day for 14 days and then 3 times per day for a subsequent 7 days), after which no further NO treatments were provided. As per the protocol, all patients will be followed for 81 days. Nine patients were enrolled in the study and all have completed the 21-day dosing schedule. Data presented below are through Day 51 (30 days post completion of treatment) for the endpoints of 6-minute walk (6MW) and forced expiratory volume (FEV1). The primary endpoint of safety, as well as the endpoints of mycobacterium abscessus (M. abscessus) load in sputum and quality-of-life (QoL), assessed at Day 21, are discussed below. The Company anticipates having all data collected by the end of the first quarter of 2018. Key findings from the data collected to date include:
- The primary endpoint of safety was achieved: there were no NO-related SAEs reported over the 21-day treatment period, which included more than 800 inhalations.
- Based on unaudited preliminary results, one of nine patients achieved culture conversion at Day 21.
- QoL questionnaire SF-36 was used in the trial and all relevant data collected to date have trended positive.
- The mean change in distance versus baseline for the 6MW test at various timepoints is presented in Figure 1. Patients received therapy through Day 21, and the measurement at Day 51 occurred during the follow-up period post treatment.
- The mean % change in FEV1 versus baseline at various timepoints is presented in Figure 2. Patients received therapy through Day 21, and the measurement at Day 51 occurred during the follow-up period post treatment.
“These data are promising and exciting,” said Andrew Colin, M.D., Director, Division of Pediatric Pulmonology at the University of Miami, and the Principal Clinical Consultant of the NO-NTM study. “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 patients and their physicians as an addition to current treatment modalities.”
The Company plans to continue developing its inhaled NO therapy for this indication and expects to begin enrollment in a potential pivotal study in the U.S. in the second half of 2018. With the proprietary NO generator technology, the Company believes it has the ability to deliver inhaled NO to the patient at home, beyond the 21-day treatment period used in our current trial. Continuing treatment for an extended period of time, in addition to the standard of care, may improve upon the results shown above.
About Mycobacterium abscessus complex (MABSC) of the Lungs
MABSC is the most aggressive and difficult to treat form of non-tuberculous mycobacteria (NTM). There are currently no approved treatments for MABSC. Current standard-of-care is a cocktail of antibiotics that often proves to be ineffective with severe adverse effects. Most patients suffering from MABSC have an underlying disease which causes immune suppression, such as cystic fibrosis or chronic obstructive pulmonary disease (COPD). The median survival of those suffering from MABSC is less than 5 years.
Nitric oxide (NO) is a powerful molecule proven to play a critical role in a broad array of biological functions. In the airways, NO is believed to play a key role in the innate immune system at concentrations of approximately 200 ppm. In vitro studies suggest that NO possesses anti-microbial activity not only against common bacteria, both gram positive and gram negative, but also against other diverse organisms including mycobacteria, fungi, yeast, and parasites, and has the potential to eliminate their multi-drug resistant strains. At concentrations below 80 ppm, NO has proven to be an effective vasodilator in the pulmonary vasculature.
About AIT Therapeutics Inc.
AIT Therapeutics Inc. is a clinical-stage biopharmaceutical company using nitric oxide (NO) to treat serious lung infections and pulmonary hypertension. The Company is currently applying its therapeutic expertise to treat lower respiratory tract infections that are not effectively addressed with current standards of care. AIT Therapeutics is advancing its revolutionary NO respiratory targeted system in clinical trials for the treatment of bronchiolitis and for nontuberculous mycobacteria (NTM). For more information, visit www.AIT-Pharm.com.
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