Data Presented at the European Cystic Fibrosis Society Conference MONMOUTH JUNCTION, N.J., June 13
MONMOUTH JUNCTION, N.J., June 13 /PRNewswire/ -- Transave, Inc., today
reported positive results from a Phase II clinical trial on its lead
investigational drug, Arikace(TM) (liposomal amikacin for inhalation). The
compound is being developed for the treatment of cystic fibrosis (CF) patients
who have lung infections due to the bacterium Pseudomonas aeruginosa. The
Phase II data indicated that Arikace, delivered once daily for 28 consecutive
days, produced a significant improvement in lung function, was well-tolerated,
and had a side-effect profile comparable to placebo. The Phase II trial was a
randomized, double-blind, placebo-controlled study of 64 patients from 15
centers in Europe. Results were presented today at the 31st European Cystic
Fibrosis Society (ECFS) Conference in Prague, Czech Republic, by Lieven
Dupont, MD, Associate Professor of Respiratory Medicine at the Katholieke
Universiteit, Leuven, Belgium, and co-lead investigator of the study.
Arikace is a novel molecular entity comprised of the antibiotic amikacin,
which is enclosed in nanocapsules of lipids called liposomes. Arikace was
administered once daily for 28 days at 280 mg and 560 mg dosages, using a
novel inhalation device, the eFlow(R) Electronic Nebulizer (PARI Pharma GmbH).
The intent-to-treat analysis for efficacy demonstrated that Arikace, when
administered once daily either at 280 mg or 560 mg for 28 days, resulted in
clinically significant improvement in lung function at the end of treatment.
This improvement was dose-dependent and was sustained at 28 days after
completion of dosing, which was day 56 of the study. Pulmonary function (FEV1)
increased significantly among patients receiving the 560 mg dose of Arikace,
with a sustained treatment effect of 224 ml (p=0.004) and 17.6% (p=0.009)
increase compared to placebo, at day 56.
Arikace was well tolerated, with no differences observed in the overall
rates of adverse events or drug-related adverse events between groups. Fewer
serious adverse events, pulmonary exacerbations, and hospitalizations were
observed in patients receiving Arikace compared to placebo. Additionally, the
time to receiving anti-pseudomonal rescue treatment was prolonged for the
patients in the Arikace arm, as compared to those in the placebo arm, which
further confirms the clinical benefit of Arikace.
"The magnitude and sustained improvement in lung function provided by
Arikace administered once-daily may contribute to advances in treatment
options for cystic fibrosis patients who have Pseudomonas lung infections,"
said Renu Gupta, MD, Transave's Executive Vice President for Development and
Chief Medical Officer. Dr. Gupta indicated that the company will seek to
confirm the positive Phase II results in a Phase III trial. "This formulation
of liposomal amikacin was specifically designed for sustained release and
penetration of the mucus and biofilm in the lungs, and we believe it will
provide benefit to patients with cystic fibrosis. These results support the
potential value of delivering amikacin through Transave's next-generation
liposomal technology."
The biofilm is a gel-like matrix in the lungs formed by colonies of the
common and often chronic bacterium Pseudomonas aeruginosa, which creates a
protective barrier for the bacteria. This barrier often prevents patients with
CF from clearing infections, even under aggressive antibiotic treatment.
Arikace was designed with small (0.3 micrometers), neutrally-charged liposomes
that enable penetration of the biofilm, which may be an important advantage
for improving treatment of lung infections due to Pseudomonas. At both the 280
mg and 560 mg doses, patients achieved a reduction in the density of
Pseudomonas that was greater than the reduction achieved by placebo. Patients
receiving the 560 mg dose achieved a 2.2 log reduction in bacterial density.
"This potential new therapy for those with chronic Pseudomonas infections
is very promising. We are excited to see improvement in pulmonary outcome
measures in patients taking Arikace," said Robert J. Beall, PhD, President and
CEO of the Cystic Fibrosis Foundation. "We are particularly pleased that an
award from our Therapeutics Development Program has enabled this promising
result for a novel drug delivery system."
Cystic Fibrosis Foundation Therapeutics, the nonprofit affiliate of the
Cystic Fibrosis Foundation, provided a $1.7 million award to support the
development of Arikace The Foundation is the leading organization devoted to
curing and controlling cystic fibrosis.
"A once-daily drug that is safe and well tolerated may offer significant
advantages to patients by reducing patient treatment burden and helping to
improve adherence to treatment," said Dr. Dupont. "Many cystic fibrosis
patients are living longer in a state of chronic infection. The ability to
deliver a drug once a day that may penetrate the CF biofilm and achieve a
sustained improvement in lung function would be an important advance in this
field.
Based on these results, the Data Safety Monitoring Board has recommended
an open-label extension of the study, in which patients would be treated with
a 560 mg dose of Arikace for three additional 28-day on-treatment and 28-day
off-treatment cycles.
The data presented today at the 31st European Cystic Fibrosis Society
Conference are currently available on the company's website:
(http://www.transaveinc.com/NewsEvents.aspx?category=Articles&archive=false).
About Arikace(TM) (liposomal amikacin for inhalation)
Arikace is a form of the antibiotic amikacin, which is enclosed in
nanocapsules of lipid called liposomes. This proprietary next-generation
liposomal delivery 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 micrometers),
neutrally-charged liposomes that enable penetration of the biofilm and are
highly efficient, with a very low lipid-to-drug ratio (0.65). Arikace can be
delivered through nebulization, which enables the small aerosol droplet size
(~3.0 micrometers) to facilitate more effective distribution in the lungs. In
addition to clinical studies in CF patients with Pseudomonas lung infections,
clinical development has been initiated in non-CF bronchiectasis patients with
Pseudomonas lung infections. Arikace has been granted orphan drug status in
the United Statesby the FDA, and has received an orphan drug designation in
Europeby the European Medicines Agency for the treatment of Pseudomonas
infections in patients with CF.
About PARI Pharma and the eFlow(R) Electronic Nebulizer
Arikace is delivered by a novel inhalation device, the eFlow(R) Electronic
Nebulizer, developed by PARI Pharma GmbH. eFlow is a quiet, portable nebulizer
that enables efficient aerosolization of liquid medications, including
liposomal formulations such as Arikace, via a vibrating perforated membrane.
Based on its 100-year history of working with aerosols, PARI Pharma is
dedicated to advancing inhalation therapies by developing innovative delivery
platforms and new pharmaceutical formulations that work together to improve
patient care.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the leading organization devoted to
curing and controlling cystic fibrosis. Headquartered in Bethesda, MD, the
Foundation funds CF research, has 80 chapter and branch offices throughout the
country, and supports and accredits a nationwide network of 115 CF Care
Centers, which provide vital treatments and other CF resources to CF patients
and their families. For more information, visit www.cff.org.
About Transave, Inc.
Transave, Inc., is a biopharmaceutical company focused on the development
of innovative inhaled pharmaceuticals for the site-specific treatment of
serious lung diseases. The company's major focus is on developing antibiotic
therapy delivered via proprietary next-generation liposomal technology in
areas of high unmet need in respiratory diseases. The Transave team is
dedicated to leveraging its advanced liposomal development and
commercialization expertise, along with its intellectual property, to bring
life-extending and life-enhancing medicines to patients. For more information
about Transave's technology and development programs, visit
http://www.transaveinc.com/.
SOURCE Transave, Inc.
