The Two-Year Open-label Extension Study
Eligible patients from the open-label, multicenter, randomized Phase 3 CLEAR-108 trial (as described below) were given the option to participate in a two-year, open-label, multi-cycle extension study designed to evaluate the long-term safety and tolerability of ARIKACE in CF patients. 206 patients enrolled in this study, representing 77% of the patients that completed the randomized portion of the Phase 3 trial, and received at least one dose of study drug. The data presented in the attached charts and discussed below includes 98 patients who had completed one year in the CLEAR-110 study by
These data demonstrate that patients receiving ARIKACE for six cycles (12 months) in the extension study showed mean increase in relative change in FEV1 which is sustained during both on-treatment and off-treatment months. Overall, ARIKACE was well-tolerated during the six cycles administered over this 12-month period, with adverse events being consistent with those expected in a population of CF patients receiving inhaled medicines.
"As we announced on
"We are especially pleased that these data demonstrated that patients taking ARIKACE actually saw their FEV1 levels remain above baseline on average. In addition we continue to see reduction of bacterial density and consistency in our overall safety profile. Further, we believe that once-daily administration of ARIKACE will support patient convenience and compliance," said
"I would like to recognize Dr. Gupta and our clinical team for designing and conducting such an important trial. This is the first trial to investigate the safety and efficacy of inhaled antibiotics in CF patients for a period of up to two years and it speaks to Insmed's ongoing commitment to understanding the safe and effective use of this drug in this target population," added
Recap of ARIKACE Data from Phase 3 Clinical Trial to Treat Pa in CF Patients
The Phase 3 trial was designed to assess the comparative safety and efficacy of ARIKACE and
The primary endpoint was relative change in FEV1 measured after three treatment cycles, with each cycle consisting of 28 days "on" treatment and 28 days "off" treatment. Secondary endpoints measured were relative changes in FEV1 at other time points, time to and number of pulmonary exacerbations, time to antibiotic rescue treatment, change in density of Pa in sputum, respiratory hospitalizations and changes in Patient Reported Outcomes assessing Quality of Life.
The Phase 3 clinical trial of ARIKACE achieved its primary endpoint of non-inferiority to
About Cystic Fibrosis
CF is an inherited chronic disease that affects roughly 70,000 children and adults worldwide, including 30,000 children and adults in the US (Cystic Fibrosis Foundation Patient Registry, 2011) and 35,000 patients in
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. ARIKACEis administered once-daily using an optimized, investigational eFlow® Nebulizer System manufactured by
ARIKACE has been granted orphan drug designation in the U.S. by 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 that are made pursuant to provisions of Section 21E of the Securities Exchange Act of 1934. 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