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TRANSAVE ANNOUNCES
POSITIVE PHASE II RESULTS FOR ONCE-DAILY
ARIKACE™ IN THE TREATMENT OF CYSTIC FIBROSIS
PATIENTS WHO HAVE PSEUDOMONAS LUNG INFECTIONS
Transave, Inc., reported positive results from a
Phase II clinical trial on its lead
investigational drug, Arikace™ (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, placebocontrolled
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® 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 dosedependent
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 µm), neutrallycharged 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 openlabel
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.
About Arikace™ (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
µm), 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 µm) 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 States by the FDA, and has
received an orphan drug designation in Europe by
the European Medicines Agency for the treatment
of Pseudomonas infections in patients with CF.
About PARI Pharma and the eFlow®
Electronic Nebulizer
Arikace is delivered by a novel inhalation
device, the eFlow® 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
http://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.
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