Lung Rx, Inc., a wholly-owned subsidiary of
United Therapeutics Corporation (Nasdaq: UTHR), announced that its Canadian
affiliate, Northern Therapeutics, Inc., has commenced the first-ever human
trial of a novel, cell-mediated gene therapy for the treatment of pulmonary
arterial hypertension. The first patient in the trial received therapy at
St. Michael's Hospital in Toronto on November 7, 2006, and the procedure
was well tolerated.
The trial, called PHACeT (Pulmonary Hypertension: Assessment of Cell
Therapy), is the first to assess the use of progenitor cells combined with
gene therapy for the treatment of a cardiac or vascular condition. PHACeT
is an open-label, dose-ranging Phase IIa trial that will enroll up to 18
patients with pulmonary arterial hypertension from across Canada in a
careful dose- escalation protocol. In the novel clinical therapy used in
the trial, engineered stem cell-like "endothelial progenitor cells" are
harvested from the patient's own blood. These cells are then engineered
using a DNA vector containing the gene for endothelial NO-synthase (eNOS),
an enzyme needed for the production of nitric oxide. People with pulmonary
arterial hypertension exhibit low production of nitric oxide, which is a
vasodilator that also plays a key role in the growth and repair of blood
cells. The engineered progenitor cells are then injected into the lung to
repair and regenerate damaged blood vessels, potentially reversing the
progress of the disease. The trial's primary endpoint is safety and
tolerability, but data will also be collected to establish the potential
efficacy of this innovative treatment. Expected to be completed within 18
months, the PHACeT trial is being conducted at two centers.
"There is a tremendous unmet need for effective therapies to treat this
devastating disease, and using cell-based gene therapy to regrow and repair
blood vessels in the lungs is a new approach to treatment," said Duncan
Stewart, M.D., Chief Scientific Officer of Northern Therapeutics. "We
anticipate that this trial's results will lay the foundation for further
research and bring us closer to a therapy that reverses pulmonary arterial
hypertension, instead of merely addressing its symptoms as current
treatments do."
Northern Therapeutics was co-founded in 2000 by Lung Rx, Inc., which
holds an exclusive option and right of first refusal to acquire
commercialization rights to market products developed by Northern
Therapeutics outside of Canada. As previously reported, Lung Rx entered
into an exclusive license agreement with Northern Therapeutics in October
2006 to obtain the United States developmental and commercial rights to
Northern Therapeutics' cell- based gene transfer technology for the
treatment of pulmonary arterial hypertension. Lung Rx owns approximately 68
percent of Northern Therapeutics.
"I applaud the three exciting first-evers being accomplished here,"
said Martine Rothblatt, Ph.D., Chairman and CEO of Lung Rx and United
Therapeutics. "The first-ever use of gene therapy for patients with
pulmonary arterial hypertension, the first-ever clinical use of endothelial
progenitor cells to target the repair of damaged blood vessels in the lung,
and the first-ever use of combined gene and cell therapy for patients with
a heart or blood vessel disorder. We are proud to have financed these
pioneering accomplishments."
TRIUMPH Enrollment Update
Lung Rx also announced today that approximately 150 patients have been
enrolled in its TRIUMPH-1 trial, a 200-patient 12-week placebo-controlled
trial of inhaled treprostinil in pulmonary arterial hypertension. As Lung
Rx has decided not to perform an interim assessment of the study results,
TRIUMPH-1 is expected to conclude when 200 evaluable patients have
completed the study.
About United Therapeutics
United Therapeutics is a biotechnology company focused on the
development and commercialization of unique products for patients with
chronic and life- threatening cardiovascular, cancer, and infectious
diseases.
Forward-Looking Statements
In addition to historical information, this press release contains
forward-looking statements about expectations and intentions regarding the
timing of completion of the PHACeT study, the impact of the PHACeT study
results, and enrollment in the TRIUMPH-1 trial that are based on United
Therapeutics' current beliefs and expectations as to future outcomes. These
forward-looking statements are subject to risks and uncertainties, such as
those described in United Therapeutics' periodic reports filed with the
Securities and Exchange Commission, which may cause actual results to
differ materially from anticipated results. Consequently, such
forward-looking statements are qualified by the cautionary statements,
cautionary language and risk factors set forth in United Therapeutics'
periodic reports and documents filed with the Securities and Exchange
Commission, including the company's most recent Form 10-K and Form 10-Q.
United Therapeutics is providing this information as of December 12, 2006,
and undertakes no obligation to publicly update or revise the information
contained in this press release whether as a result of new information,
future events or any other reason.
United Therapeutics Corporation
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