Targeted Genetics Announces Continued Clinical Study at University College London/Moorfields Eye Hospital

Targeted Genetics Announces Continued Clinical Study at University College London/Moorfields Eye Hospital for a Gene Therapeutic to Treat Leber's Congenital Amaurosis and Provides Business Update

SOURCE: Targeted Genetics Corporation

Jun 22, 2009 07:30 ETTargeted Genetics Announces Continued Clinical Study at University College London/Moorfields Eye Hospital for a Gene Therapeutic to Treat Leber's Congenital Amaurosis and Provides Business Update
Initial Phase 1/2 Findings Demonstrated Acceptable Safety and Improvement in Visual Function; Protocol Amended to Increase Dose and Include Younger Children

SEATTLE, WA--(Marketwire - June 22, 2009) - Targeted Genetics Corporation (NASDAQ: TGEN) today announced that its collaborator at the University College London/Moorfields Eye Hospital (UCL/M) has begun the next step in its Phase 1/2 dose escalation clinical trial to treat a form of Leber's Congenital Amaurosis caused by defects in the gene encoding the RPE65 protein. Leber's Congenital Amaurosis, or LCA, is an inherited disease that typically results in blindness. Under an amended protocol to its Phase 1 /2 dose escalation clinical trial, UCL/M is approved to include younger LCA patients in the trial and treat patients with higher doses of an adeno-associated virus (AAV) vector containing the RPE65 gene.

"We are very pleased to have received regulatory approval to treat younger children, who have more viable retina, as well as approval to use a higher dose, which increases the probability of a durable response," stated Professor Robin Ali, University College of London Institute of Ophthalmology and Moorfields Eye Hospital, who is leading the study. "We believe this amended study protocol optimizes risk/benefit for subjects and will further improve the quality of data acquired."

The higher dose of vector has been administered by subretinal injection at Moorfields Eye Hospital to a patient referred for inclusion in the trial by Dr. Ingeborgh van den Born of The Rotterdam Eye Hospital in The Netherlands.

The deterioration in visual function experienced by individuals with LCA results from a progressive retinal degeneration compounded by amblyopia caused by stimulus deprivation during early childhood. For these reasons the benefit of gene delivery therapy is expected to be greatest when applied at an early stage in the condition.

This revised protocol was reviewed and approved by the Gene Therapy Advisory Committee, the Medicines and Healthcare Products Regulatory Agency, and local ethics committees. Targeted Genetics' LCA program was initiated in 2005 when Targeted Genetics entered into a collaboration agreement to develop, manufacture and commercialize an AAV-RPE65 product candidate with University College London.

Results from the first stage of this open label, single center, Phase 1/2 clinical study were reported in April 2008 in three young adults between the ages of 23 and 17 years of age with early-onset severe retinal dystrophy. Data demonstrated that administration improved visual function as measured by visual field tests and improvement in subjective tests of visual mobility in one patient. The study resulted in no serious adverse events and findings supported further clinical studies. The study results were also published in the May 2008 issue of the New England Journal of Medicine. Completion of the current trial is anticipated in the second half of 2010.

The primary endpoint of this study is to determine whether AAV vector-mediated gene delivery to the retina is safe, and the secondary endpoint is to determine whether efficacy can be demonstrated in humans. Targeted Genetics, a leader in the development and manufacture of AAV-based product candidates, manufactured the vector that is being used in this trial.

"We believe gene therapeutics hold great promise as a new therapeutic modality that may have competitive advantages over proteins, antibodies and small molecules in certain indications and enable the treatment of conditions not previously treatable with current modalities. We believe that the continuation of this trial brings us closer to generating convincing data on the therapeutic benefit of delivering an AAV gene therapeutic," said B. G. Susan Robinson, President and Chief Executive Officer of Targeted Genetics. "Targeted Genetics' continued efforts in this area, however, depend on our successfully raising additional funding."

The Company also provided an update on its financing efforts to support its continued operations, product development efforts and manufacturing operations infrastructure. The Company has decided to meet product supply requirements using contract manufacturing organizations managed by the Company's key senior manufacturing staff and, as a result, will reduce its total staff count to 10 to 15 full-time equivalent employees by the end of July 2009. In parallel, the Company has continued to pursue a settlement of its Bothell lease obligations and has also entered into discussions to reduce or eliminate its other facility costs. As a result of anticipated cost savings resulting from these initiatives and additional expense reductions implemented by the Company, the Company believes its current financial resources and the cash it expects to receive from its collaborative partners will be adequate to fund its operations into August 2009. The Company continues to pursue additional capital through strategic transactions, licensing or selling technology, product development collaborations, and sales of stock. If the Company is not successful in raising additional funding sufficient to support ongoing operations, it will wind down its business or otherwise cease its operations.

About Leber's Congenital Amaurosis

Mutations in a number of genes, one of which is RPE65, disrupt the retinoid cycle that underlies human vision and cause a congenital retinopathy known as Leber's Congenital Amaurosis (LCA). LCA is a complex retinal disease in which visual loss is caused by a combination of dysfunction and degeneration of retinal photoreceptors. LCA is an inherited retinal degenerative disease characterized by severe impairment of vision from birth and total blindness by the third decade of life. There is no treatment currently available for LCA.

About UCL Institute of Ophthalmology and Moorfields Eye Hospital NIHR Biomedical Research Centre

The Centre was established in April 2007, funded by the Department of Health through the National Institute for Health Research (NIHR). It is one of 12 NHS-university partnerships that have been awarded Biomedical Research Centre status, following an international peer reviewed competition based on an outstanding international reputation for medical research and expertise, and experience of translating that research into the clinical setting.

About UCL Institute of Ophthalmology

UCL Institute of Ophthalmology is one of a number of specialized research centers linked to UCL (University College London) and is, together with Moorfields Eye Hospital, one of the leading centers for eye research. The Institute scored 5*A (the highest possible rating) in the last Research Assessment Exercise and is committed to a multi-disciplinary research portfolio that furthers an understanding of the eye and visual system, linked with clinical investigations targeted to specific problems in the prevention and treatment of eye disease. The combination of the Institute's research resource with the resources of Moorfields Eye Hospital, which has the largest ophthalmic patient population in the Western World, opens the way for advances at the forefront of vision research.

About The Rotterdam Eye Hospital

The Rotterdam Eye Hospital was founded in 1874 and is the only hospital in The Netherlands, specialized in eye health care. It receives tertiary referral cases from throughout the rest of The Netherlands and has its own research institute, The Rotterdam Ophthalmic Institute.

About Targeted Genetics Corporation

Targeted Genetics Corporation is a biotechnology company committed to the development of innovative therapies for the prevention and treatment of diseases with significant unmet medical need. A key area of focus for Targeted Genetics is applying its proprietary Adeno-Associated Virus (AAV) technology platform to deliver genetic constructs to increase gene function or silence gene function. Targeted Genetics' lead product development efforts target ocular and neurological indications, two therapeutic areas where AAV delivery may have competitive advantages over other therapeutic modalities. To learn more about Targeted Genetics, visit its website at

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:

This release contains forward-looking statements regarding the Company's liquidity and financial resources, its ability to fund ongoing and future operations, and its product development and business strategies, including statements regarding the LCA clinical trial, the potential advantages of AAV gene therapeutics, the Company's ability to continue its operations, raise additional capital or secure other financial resources in the near term, the extent of the Company's cash horizon, the possibility that the Company may cease its operations or otherwise wind up its business and other statements about the Company's plans, objectives, intentions and expectations. These 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 affect the accuracy of forward-looking statements. Factors that could affect actual future events or results include, but are not limited to, funding anticipated by the Company under product development collaborations and contracts, the Company's actual expenses, the Company's ability to raise capital when needed, the timing, nature and results of clinical trials, the risk that the Company will not be able to regain and maintain compliance with the standards for continued listing on the Nasdaq Capital Market within the required time periods and the risk that such non-compliance will cause the Company's securities to be delisted, potential development of alternative technologies or more effective products by competitors and the ability to obtain and maintain regulatory or institutional approvals, as well as other risk factors described in the section entitled "Item 1A. Risk Factors" in Part II, Item 1A of the Company's quarterly report on Form 10-Q for the period ended March 31, 2009, filed with the Securities and Exchange Commission on May 7, 2009, and in other filings with the SEC. You should not rely unduly on these forward-looking statements, which apply only as of the date of this release. The Company undertakes no duty to publicly announce or report revisions to these statements as new information becomes available that may change the Company's expectations.

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