The Company reports that its Phase 3 study of once-daily ARIKACE to treat Pseudomonas aeruginosa (Pa) in cystic fibrosis (CF) patients conducted at 70 sites in
The Company also announces that the
Phase 3 Clinical Trial Design for ARIKACE to Treat Pa in CF Patients
The Phase 3 trial was an open-label, multi-center, randomized study designed to assess the comparative safety and efficacy of ARIKACE and TOBI in CF patients with Pa. A total of 302 adult and pediatric CF patients with chronic Pa were randomized to receive 28-days of ARIKACE treatment delivered once-daily via an investigational eFlow® Nebulizer System, or TOBI delivered twice-daily via the PARI LC Plus® Nebulizer System over a 24-week treatment period.
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. The study was designed to demonstrate non-inferiority to TOBI at a 5% non-inferiority margin with 80% power agreed upon by the Company and the
The Phase 3 clinical trial of ARIKACE achieved its primary endpoint of non-inferiority to TOBI for relative change in FEV1 from baseline to end of study.
Overall, secondary endpoints showed comparability of once-daily ARIKACE compared with twice-daily TOBI consistent with the primary endpoint of the study. Complete trial data will be reviewed in greater detail and is expected to be released at a scientific conference later this year. The Company also intends to pursue publication of trial data in a peer-reviewed journal.
During all three treatment cycles, the safety profile of ARIKACE was comparable to TOBI, with adverse events consistent with those seen in similar studies and expected in a population of CF patients receiving inhaled antibiotics. There was no difference between arms in the reporting of serious adverse events and there were no unexpected adverse events.
"The Phase 3 study of ARIKACE achieved its primary endpoint and demonstrated that ARIKACE is an effective inhaled antibiotic. These results are consistent with our earlier studies of ARIKACE and we believe further strengthen the clinical data packages
"The study achieved its primary endpoint and suggests that once-daily ARIKACE treatment was comparable to twice-daily TOBI treatment, the current gold standard of care for cystic fibrosis patients with Pseudomonas aeruginosa lung infections," stated
"This early review of data is encouraging. ARIKACE has the potential to be the first once-daily treatment, as compared with the current treatment regimens that require multiple dosing each day. Once-daily treatment may significantly enhance patient convenience and compliance," stated
"Cystic fibrosis patients chronically infected with Pseudomonas aeruginosa are among the most heavily burdened patients when it comes to the number, frequency and types of treatments they need to take on a daily basis. The data from this study suggest that once-daily treatment with ARIKACE may provide a safe, effective and more convenient alternative to twice-daily TOBI treatment," stated
"We are very pleased with the results ARIKACE achieved in our Phase 3 trial and our attention now turns to preparing for regulatory filings with the EMA and
"We appreciate the support of the
Two-Year Extension Study
Eligible patients from this Phase 3 trial were given the option to participate in a two-year, open-label safety study. Approximately 75% of eligible patients have consented to participate in this study. The Company expects to use data from this study as part of the regulatory submissions to the European and Canadian authorities.
ARIKACE for the Treatment of NTM
"NTM lung infection is a disease that is being recognized and diagnosed with increasing frequency in the U.S. and worldwide. The risk for NTM lung disease is especially high in women as they age beyond the sixth decade of life. Pulmonary and Infectious Disease physicians are seeing these patients in ever growing numbers. While therapy can be reasonably effective for some patients, it routinely entails multiple, potentially toxic and expensive medications that must be given over long periods of time. Sadly, for many other patients, current therapies are inadequate with few alternative treatment strategies available," said Dr.
"Research in the area of NTM lung disease has been woefully underfunded with no major therapeutic advances in the last 20 years. The availability of a once-a-day product that can help reduce bacterial density in these patients would represent a major step forward in the treatment of this disease," added Dr. Griffith.
The Fast Track designation is intended to expedite the review of new drugs that have the potential to serve unmet medical needs in serious or life-threatening conditions. Under this designation
"QIDP designation for ARIKACE underscores the importance the
Pursuant to QIDP status, the Company expects to continue its dialogue with the
Conference Call and Webcast
A replay of the conference call will be accessible two hours after its completion through
About Cystic Fibrosis
According to the
About Non-Tuberculous Mycobacteria
NTM infections are acquired directly from the environment, where they are often present in soil and various water sources. The prevalence of NTM disease is reported to be increasing, and according to reports from the
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. PARI Pharma focuses on the development of aerosol delivery devices and inhalation drug development to advance aerosol therapies where drug and device can be optimized together. For more information, please visit www.paripharma.com.
* TOBI® is a Registered Trademark of
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