Infoseek: The News Channel

Targeted Genetics Announces Initiation of Phase I Clinical Trials of Aerosolized Gene Therapy to Treat Cystic Fibrosis
07:13 a.m. Dec 08, 1998 Eastern

SEATTLE, Dec. 8 /PRNewswire/ -- Targeted Genetics Corporation today announced that it has initiated a Phase I clinical trial for an aerosolized version of the company's proprietary gene therapy product for the treatment of cystic fibrosis (CF), tgAAV-CF. Development of this product is being conducted with Medeva PLC (NYSE: MDV) under a joint development and supply agreement as announced on November 24, 1998.

Targeted Genetics is conducting the multi-center study in CF patients at Stanford University Medical Center and in collaboration with the Cystic Fibrosis Foundation through the recently established Therapeutics Development Center Network (TDN). This network, which is managed by the TDN Coordinating Center in Seattle and led by Bonnie Ramsey, MD, comprises seven specialized clinical research centers in the United States whose primary objective is to develop and conduct early-phase clinical trials of novel therapies for the treatment of CF. This Phase I study will be conducted at TDN centers at Children's Hospital and Regional Medical Center and the University of Washington in Seattle and at Children's Hospital in Boston, MA.

"The TDN was established to increase the pace of developing new treatments for CF," said Robert J. Beall, Ph.D., President and Chief Executive Officer of the Cystic Fibrosis Foundation. "We believe that tgAAV-CF is a promising approach that could lead to correcting the underlying cause of the disease, rather than only ameliorating its symptoms. By combining the expertise of Targeted Genetics in gene therapy, the clinical expertise of CF investigators, and the development and regulatory capabilities of Medeva, we will move ahead quickly."

The trial, a multi-center, dose-escalation, open-label study, will evaluate the safety of an aerosolized version of tgAAV-CF in up to 12 adult patients with mild to moderate CF lung disease. The company believes that an aerosolized formulation will provide widespread delivery of tgAAV-CF to the airways of the human lung and has the potential to alleviate the pulmonary symptoms associated with CF. Ultimately, the company expects its development partner Medeva to take the aerosolized formulation of tgAAV-CF through the regulatory process for approval as a commercial product for the treatment of CF.

"We are pleased and honored to be working closely with Stanford and the TDN in the continued development of tgAAV-CF," said H. Stewart Parker, President and Chief Executive Officer of Targeted Genetics. "This product, tgAAV-CF, is the first gene therapeutic for CF to be supported by the TDN. The Foundation's wealth of information and expertise has been extremely valuable in moving tgAAV-CF into the clinic and we look forward to working with the clinicians at the TDN toward the development of a new treatment for this serious disease."

Joint Development Agreement with Medeva PLC Background

In November of 1998, Targeted Genetics and Medeva PLC announced the formation of a collaboration to develop and commercialize tgAAV-CF. Under the terms of the agreement, Targeted Genetics could receive up to $54 million, including $12 million of loans. The consideration being paid by Medeva to Targeted Genetics comprises: a $5 million technology license fee; clinical and regulatory milestone payments totaling $19 million; a loan of $2 million; investment in Targeted Genetics equity totaling $3 million; and up to a maximum of $15 million in development funding for this project over a three year period. Under certain conditions, Medeva will also make available to Targeted Genetics a loan of $10 million toward the building of a GMP manufacturing facility for the production of tgAAV-CF. Of the above amounts, a total of $8.5 million was paid upon the signing of the agreement. Medeva will cover the costs of clinical and regulatory activities related to the development of the product for which it is responsible under the agreement.

Clinical trials of tgAAV-CF in the United States will be managed by Targeted Genetics through the completion of Phase I studies. Targeted Genetics may, under certain conditions, manage Phase II clinical trials in the United States. Medeva will manage all other trials and will be responsible for worldwide registration of tgAAV-CF. Upon registration, Medeva will be responsible for worldwide marketing of the product. A joint development committee, comprised of three individuals from each company, will be formed to oversee the formation and execution of the tgAAV-CF clinical development plan. Assuming successful development and registration of the product, Medeva will purchase tgAAV-CF from Targeted Genetics under a long-term supply agreement.

CF Background

CF results from the loss of function of the CFTR gene. Normally, the protein encoded by this gene is found in the respiratory passageways, pancreas, salivary and sweat glands, where it helps to regulate the cellular concentration of chloride ions and water. In CF patients, the loss of this gene's function causes the production and build-up in the lungs and sinuses of thick mucus, which is readily colonized by bacteria. The thickness of the mucus prevents clearance of bacteria and reduces the efficacy of antibiotics. Repeated infections produce scarring in the lung, which ultimately results in loss of respiratory function. It is estimated that 60,000 people worldwide have cystic fibrosis including approximately 24,000 people in the United States. The median age of survival for patients with CF is approximately 31 years.

Targeted Genetics Corporation develops gene and cell therapy products for the treatment of certain acquired and inherited diseases. The Company has three lead product development programs targeting cystic fibrosis, cancer, and infectious disease, as well as an extensive technology platform.

NOTE: This release contains forward-looking statements relating to the Company's products under development, technologies and future operating results that are subject to certain risks and uncertainties that could cause actual results to differ materially from those projected. The words "believes," "expects," "intends," "anticipates," variations of such words, and similar expressions identify forward-looking statements, but their absence does not mean that the statement is not forward-looking. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could affect the Company's actual results include the need for additional capital, the early stage of product development, uncertainties related to clinical trials, and uncertainties related to patent position. Reference is made to the Company's latest Annual Report on Form 10-K filed with the SEC for a more detailed description of such factors. Readers are cautioned not to place an undue reliance on these forward-looking statements, which speak only as of the date of this release. The Company undertakes no obligation to update publicly any forward-looking statements to reflect new information, events or circumstances after the date of this release or to reflect the occurrence of unanticipated events. SOURCE Targeted Genetics Corporation

Volver a Medios de Comunicación