The Phase 2 clinical study is a randomized, placebo-controlled study of ARIKACE in adult patients with recalcitrant NTM lung disease. Eligibility for the study required patients to have been on the
The primary efficacy endpoint in the study is the semi-quantitative measurement of the change in mycobacterial density on a seven-point scale from baseline (day one) to the end of the randomized portion of the trial on day 84. Mycobacterial density is a measurement currently used in clinical practice to assess the progress or decline of those patients with recalcitrant NTM. Following the randomized portion of the study, all eligible patients may choose to receive ARIKACE once daily for an additional 84 days in an open-label design.
Patients in the trial are stratified for either Mycobacterium avium complex (MAC) infections or Mycobacterium abscessus infections. These pathogens collectively account for approximately 85% percent of all patients with NTM lung disease in the U.S. Additionally, stratification is performed based on patients with cystic fibrosis versus those who do not have cystic fibrosis.
Certain secondary, tertiary and exploratory endpoints being measured include, but are not limited to, time to sputum conversion, change in clinical signs and symptoms, change in patient related outcomes/quality of life and safety.
The Company has completed recruitment with 90 patients in the trial as required per protocol. The Company has concluded that the study was sufficiently powered to achieve statistical significance with 80% power to demonstrate a one-step change in the seven-step scale measuring bacterial density, as specified in the protocol.
"We are very pleased to have completed enrollment in this important clinical trial of ARIKACE to treat recalcitrant NTM lung infections," stated
"Completing enrollment of this trial is a major accomplishment for
"We entered this study with compelling in vitro and in vivo data showing ARIKACE had good activity against the NTM pathogens. In addition, we demonstrated in our Phase 3 clinical study of ARIKACE in cystic fibrosis patients that our liposomal inhalation technology safely and effectively delivers the drug to the site of infection and reduces bacterial burden," Lewis added.
"Importantly, recent U.S. regulatory designations underscore the importance the
About Nontuberculous Mycobacteria (NTM)
Nontuberculous mycobacteria (NTM) are organisms found in the soil and water that can cause serious lung disease in susceptible individuals, for which there are currently limited effective treatments. The prevalence of NTM disease is reported to be increasing, and according to reports from the
NTM lung disease is often a chronic condition that can lead to progressive inflammation and lung damage, characterized by bronchiectasis and cavitary disease. NTM infections often require lengthy hospital stays for medical management. Treatment usually involves lengthy multi-drug regimens that can be poorly tolerated and with limited effectiveness, especially in patients with severe disease or in those who have failed prior treatment attempts. According to a company sponsored patient chart study conducted by
ARIKACE is a form of the antibiotic amikacin, which is enclosed in nanocapsules of lipid called liposomes. This advanced pulmonary liposome technology prolongs the release of amikacin in the lungs while minimizing systemic exposure. The treatment uses biocompatible lipids endogenous to the lung that are formulated into small (0.3 micron), charge-neutral liposomes. ARIKACE is administered once-daily using an optimized, investigational eFlow® Nebulizer System manufactured by
ARIKACE has Orphan Drug, QIDP and Fast Track designations from the
About eFlow® Technology and PARI Pharma
ARIKACE is delivered by an investigational eFlow® Nebulizer System developed by PARI Pharma and optimized specifically for ARIKACE. The optimized device uses eFlow Technology to enable highly efficient aerosolization of medication including liposomal formulations via a vibrating, perforated membrane that includes thousands of laser-drilled holes. Compared with other nebulization technologies, eFlow Technology produces aerosols with a very high density of active drug, a precisely defined droplet size and a high proportion of respirable droplets delivered in the shortest possible period of time. eFlow Technology is not an ultrasonic nebulizer technology and is not a general purpose electronic aerosol generator nebulizer technology. Combined with its quiet mode of operation, small size, light weight and battery use, eFlow Technology reduces the burden of taking daily, inhaled treatments.
This release contains forward-looking statements. Words, and variations of words, such as "intend," "expect," "will," "anticipate," "believe," "continue," "propose" and similar expressions are intended to identify forward-looking statements. Investors are cautioned that such statements in this release, including statements relating to the status, results and timing of clinical trials and clinical data, the anticipated benefits of