Transave Completes Successful Phase II Clinical Program for ARIKACE in the Treatment of Pseudomonas Lung Infections

Transave Completes Successful Phase II Clinical Program for ARIKACE in the
Treatment of Pseudomonas Lung Infections
Final Phase II results further validate ARIKACE(TM) efficacy and tolerability
in the treatment of CF patients and non-CF bronchiectasis patients

MONMOUTH JUNCTION, N.J., Oct. 19 /PRNewswire/ -- Transave Inc., today reported
additional positive clinical trial results on its lead investigational drug,
ARIKACE(TM) (liposomal amikacin for inhalation), an antibiotic that aims to
treat chronic lung infections. The latest results along with previously
reported data demonstrate significant clinical benefits and complete the
company's Phase II program in two indications - for the treatment of lung
infections due to the bacterium, Pseudomonas aeruginosa in cystic fibrosis
(CF) patients and non-CF bronchiectasis patients.

The new data from the Phase II clinical trial program in CF patients with
Pseudomonas lung infections indicate that ARIKACE, delivered at a dose of 560
mg once daily for 28 consecutive days, demonstrated superior clinical benefit
compared to placebo as measured by a significant and sustained improvement in
lung function, improvement in patient reported outcomes and reduction in
Pseudomonas density. In addition, ARIKACE was well-tolerated with overall
adverse events comparable to placebo. The Phase II program results were
presented Oct. 17 at the 23rd annual North American Cystic Fibrosis Conference
in Minneapolis, Minn., by JP Clancy, M.D., of the Children's Hospital of
Alabama and the University of Alabama at Birmingham. The Phase II results
were from a prospectively designed pooled analysis of two randomized,
double-blind and placebo-controlled studies of 105 patients at 33 European and
U.S. sites. Preliminary data from a third CF trial which is an on-going open
label study where ARIKACE is being dosed in multiple cycles were also
presented, showing continued improvement in lung function over repeat cycles.

"We now have consistent positive results from three placebo controlled and
three open label studies of ARIKACE in CF and non-CF bronchiectasis patients,"
said Tim Whitten, Transave's President and Chief Executive Officer. "To date,
studies suggest that ARIKACE has the potential to improve upon the standard of
care in the treatment of chronic Pseudomonas lung infections, and further
studies are merited in an effort to bring this medicine to market."

In the U.S., ARIKACE was administered once daily for 28 days at 70 mg, 140 mg
and 560 mg doses using a novel inhalation device, the eFlow® Electronic
Nebulizer (PARI Pharma GmbH). This study included member institutions of the
Cystic Fibrosis Foundation's Therapeutics Development Network. In Europe,
ARIKACE was administered once daily for 28 days at 280 mg and 560 mg dosages,
also using the eFlow® Electronic Nebulizer. Improved lung function was
dose-related with the higher 560 mg dose resulting in the greatest improvement
in lung function which was sustained for 28 days after treatment ended.
Adverse events reported were consistent with those expected in a population of
CF patients receiving inhaled medicine, and no differences between groups in
the overall rates of adverse events.

"Once-a-day ARIKACE, providing sustained improvement even after therapy ends,
suggests that it is possible to treat these stubborn Pseudomonas lung
infections while reducing the treatment burden that these patients currently
endure," said Dr. Clancy. "Further, the potential for ARIKACE to penetrate
the biofilm and achieve a sustained improvement in lung function would be an
important advance in this field."

Study results have consistently demonstrated significant reduction in
Pseudomonas bacterial density in both mucoid and non-mucoid strains of
Pseudomonas. Mucoid strains of Pseudomonas are associated with the presence
of biofilms. A biofilm is a protective gel-like barrier formed by colonies of
the common and often chronic Pseudomonas bacteria. The bacterial cells
encased within the biofilm are difficult to kill because many antibiotics
either stick to the surface of the biofilm and/or are cleared away before they
can act on the bacteria inside the biofilm. ARIKACE was designed with small
(0.3 micron), neutral liposomes that enable significant drug penetration into
the biofilm, which may be an important advantage for improving treatment of
lung infections due to Pseudomonas.

"ARIKACE development shows great promise for CF patients," said Robert J.
Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. "We have
supported its development in hopes that trials would demonstrate the potential
of ARIKACE to improve lung function and quality of life for CF patients."

Cystic Fibrosis Foundation Therapeutics, the nonprofit affiliate of the Cystic
Fibrosis Foundation, has provided a total award of $3.9 million to support the
development of ARIKACE. The Foundation is the leading organization devoted to
curing and controlling cystic fibrosis.

About ARIKACE(TM)
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), neutral liposomes that
enable penetration of the biofilm. ARIKACE can be delivered through
nebulization, which enables the small aerosol droplet size (1 to 5 microns) to
facilitate more effective distribution in the lungs. 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. ARIKACE has also been granted
orphan drug status by the FDA for the treatment of bronchiectasis in patients
with Pseudomonas or other susceptible pathogens.

About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure
for cystic fibrosis. The Foundation funds more CF research than any other
organization and nearly every CF drug available today was made possible
because of Foundation support. Based in Bethesda, Md., the Foundation also
supports and accredits a national care center network that has been recognized
by the National Institutes of Health as a model of care for a chronic disease.
For more information visit www.cff.org.

About PARI Pharma and the eFlow® Electronic Nebulizer
ARIKACE is delivered by an eFlow® Nebulizer System developed by PARI Pharma
GmbH. The eFlow® Nebulizer System 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 to 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. Combined with its silent mode of operation, small
size (it fits in the palm of the hand), light weight, and battery use,
products incorporating eFlow® Technology reduce the burden of taking daily,
inhaled treatments. The eFlow® Nebulizer System and eFlow® Technology are
proprietary to PARI Pharma and can be optimized to specific drug formulations.

About Transave, Inc.
Transave, Inc., is a biopharmaceutical company focused on the development of
innovative inhaled pharmaceuticals for the site-specific treatment of chronic
lung diseases. The company's major focus is on developing antibiotic therapy
delivered via proprietary advanced pulmonary liposome technology in areas of
high unmet need in lung diseases. The Transave team is dedicated to
leveraging its 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.

Jennifer Corrigan of Transave, Inc., +1-732-382-8898,
jenn.corrigan@comcast.net