Full Press Release Details
AND TARGETED GENETICS INITIATE PHASE I CLINICAL TRIAL OF
IN PATIENTS WITH HEART FAILURE
Jolla, CA and Seattle,
17, 2007 - Celladon Corporation and Targeted Genetics Corporation (Nasdaq:
today announced the initiation of a Phase I clinical trial of
(AAV1/SERCA2a) in patients with cardiomyopathy and symptoms of heart failure.
an adeno-associated virus (AAV) vector to deliver the SERCA2a gene to heart
trial, titled "Calcium Up-Regulation by Percutaneous Administration of Gene
Therapy In Cardiac Disease (CUPID Trial)," is a Phase I/II, two-stage,
dose-escalation trial designed to evaluate the safety and feasibility of
single coronary artery infusion of four dose levels of an AAV1 vector expressing
the transgene for SERCA2a to subjects with ischemic or non-ischemic
cardiomyopathy and NYHA Class III/IV symptoms of heart failure. In Stage
the trial, 12 subjects will be enrolled and treated with one of four escalating
doses of open-label MYDICAR . In Stage 2 of the trial, 33 subjects will be
randomized to receive either MYDICAR
placebo. Initiation of this trial follows recent approvals by both the National
Institutes of Health Recombinant DNA Advisory Committee (RAC) without
requirement of an in-depth public review, and Office of Cellular, Tissue
Gene Therapies, Center for Biologics Evaluation and Research (CBER) branch
the Food and Drug Administration.
therapeutic candidate targets the SERCA2a pathway, an important regulator
myocardial contractility. Malfunction in this pathway is associated with
progressive heart failure," said Krisztina Zsebo, Ph.D., chief executive
of Celladon Corporation. "By bringing the levels of this enzyme back to normal
in the heart, we hope to both demonstrate the safety of MYDICAR and provide
evidence that MYDICAR has the potential to halt or reverse the progression of
disease in heart failure patients."
Genetics and Celladon have a collaboration agreement to develop
treatment of congestive heart failure. The collaboration combines Targeted
Genetics' expertise in the development, manufacture and clinical evaluation of
AAV-based therapies with Celladon's portfolio of genes with potential in the
treatment of congestive heart failure. "The initiation of this trial highlights
the enormous potential of our AAV delivery platform to enable novel treatments
for serious diseases," said H. Stewart Parker, president and chief executive
officer of Targeted Genetics. "It is our hope that Targeted Genetics' gene
delivery technology is proven to be the key that unlocks the potential of
pathway in treating a disease that affects millions of Americans."
an AAV1 vector to deliver the SERCA2a gene to the heart using standard cardiac
catheterization laboratory procedures and equipment. SERCA2a activity is
decreased in heart tissue obtained from heart failure patients. To date,
there have been compelling preclinical results suggesting that gene therapy
targeting calcium cycling within the heart may improve myocardial contractility
and reverse the progression of heart failure. Studies in large-animal models
heart failure demonstrated that administration of MYDICAR
significant improvements in heart function without any significant safety
failure is a serious condition in which the heart progressively loses its
ability to pump blood efficiently throughout the body. According to the National
Heart, Lung and Blood Institute, about 5 million people in the United States
alone have heart failure, and another 550,000 new cases are diagnosed each
Heart failure contributes to or causes about 300,000 deaths annually. The
disease is most common in people age 65 or older, women and African Americans.
The most common symptoms of heart failure are shortness of breath, feeling
tired, and swelling in the ankles, feet, legs, and sometimes the abdomen.
is no cure for heart failure.
Corporation is a molecular cardiology company based in La Jolla,
and founded by Roger J. Hajjar, M.D and Kenneth R. Chien M.D.,
Founding investors in Celladon include Enterprise Partners Venture Capital
Associates, recently joined by Johnson & Johnson Development Corporation. To
learn more about Celladon, visit Celladon's website at www.celladon.net.
Genetics Corporation is a biotechnology company committed to the development
innovative targeted molecular therapies for the prevention and treatment
acquired and inherited diseases with significant unmet medical need. Targeted
Genetics' proprietary Adeno-Associated Virus (AAV) technology platform allows it
to deliver genes that encode proteins to increase gene function or RNAi to
decrease or silence gene function. Targeted Genetics' product development
efforts target inflammatory arthritis, AIDS prophylaxis, congestive heart
failure and Huntington's disease. To learn more about Targeted Genetics,
Targeted Genetics' website at www.targetedgenetics.com.
Harbor Statement under the Private Securities Litigation Reform Act of
release contains forward-looking statements regarding the initiation and
of a clinical trial of Mydicar, the potential therapeutic benefit of Mydicar,
the potential of Targeted Genetics' gene delivery technology and other
statements about the plans, objectives, intentions and expectations of Targeted
Genetics. These forward-looking statements involve current expectations,
forecasts of future events and other statements that are not historical facts.
Inaccurate assumptions and known and unknown risks and uncertainties can
the accuracy of these forward-looking statements. Actual results or developments
may be materially different from the results projected or implied in these
forward-looking statements. Factors that could affect our actual results
include, but are not limited to, the risk of adverse clinical results as
moves into and advances in clinical trials, Targeted Genetics' ability to
obtain, maintain and protect its intellectual property, Celladon's ability to
recruit and enroll suitable trial participants, Targeted Genetics' dependence on
the efforts of third parties, the timing, nature and results of research
clinical trials, potential development of alternative technologies or more
effective processes by competitors, and, whether regulatory or institutional
approvals can be obtained and maintained, as well as other risk factors
described in Item 1A. Risk Factors in Targeted Genetics' report on Form 10-K for
the year ended December 31, 2006 as updated in its Form 10-Q for the quarter
ended March 31, 2007. You should not rely unduly on these forward-looking
statements, which apply only as of the date of this release. Targeted Genetics
undertakes no duty to publicly announce or report revisions to these statements
as new information becomes available that may change these