|
||
| |||||||
|
tgAAVCF: Will The Latest Failure Be
The Straw That Breaks Investor’s Backs?
Products grouped under the DNA/RNA market umbrella include antisense oligonucleotide therapies, gene therapies, RNA interference products, aptamers and ribozymes. Product developers operating in this market are post-genomic companies whose products and platform technologies provide the most direct route to commercializing the human genome. However the technologies within this market are at different stages of evolution, with gene therapies and antisense products representing the most advanced products. Gene therapies represent the most actively studied DNA/RNA programs, despite the lack of success of bringing a gene therapy to the market in the US or Europe. According to the Journal of Gene Medicine, gene therapy trial approvals rose steadily through the 1990s to peak in 1999. However, with the death of an American teenager in 1999 in a gene therapy clinical trial, gene therapy trial approvals have fallen steadily since then, Belsey says. “Indeed only one gene therapy has so far been launched worldwide—SiBiono’s Gendicine, used to treat cancer—and only six out of the remaining 110 active gene therapy products are thought to be likely to launch by 2010.”
The 111 active gene therapy development programs are being developed for a very broad range of indications, with oncology representing the leading therapeutic focus. However genetic disorders (e.g. Cystic Fibrosis and hemophilia) account for a proportionally larger percentage of active DNA/RNA programs, compared to total pharmaceutical/biotech product development. This is due primarily to the suitability of using technology platforms such as gene therapy for treating such indications.
Of the gene therapies in
development to treat genetic disorders, Targeted Genetics’s tgAAVCF is the most
advanced product and was in development to treat cystic fibrosis (CF), Belsey
says. “CF is a monogenic autosomal hereditary recessive disorder and the disease
is primarily manifested as CF lung disease. CF gene therapy using products such
as tgAAVCF targets the mutated gene with the aim of replacing it with a normal
copy. Datamonitor estimates that there are approximately 65,000 CF patients in
the seven major markets**.” tgAAVCF utilizes Targeted Genetics’s proprietary adeno-associated virus vector (AAV) technology in an aerosol delivery using a nebulizer. Of the 111 gene therapies, the third most prevalent vector are AAVs, behind adenoviral vectors and non-viral/naked vectors. Leading players in the AAV field include Avigen and Targeted Genetics, who have consolidated their position in this market by building a strong intellectual property position, Belsey says. “AAV vectors have several advantages relative to other gene therapies, including long transgene expression and high transfection efficacy.” “Early-stage clinical trial data indicated that tgAAVCF was broadly distributed throughout the lung and that the product had a good safety profile. Phase II data presented in 2003 indicated that there was an improvement in overall lung function after 30 days of administration,” he says. Based on these promising clinical trial results, Targeted Genetics gained funding from a collaboration with Cystic Fibrosis Foundation Therapeutics, which allowed it to carry out a Phase II trial. Preliminary results from this trial were published on March 17 2005, when Targeted Genetics announced that its product had failed to meet the primary endpoint in a Phase II clinical trial for cystic fibrosis. tgAAVCF failed to significantly improve lung function in patients with mild to moderate CF 30 days after administration compared to those receiving a placebo. Following the failure of tgAAVCF, Targeted Genetics announced that they were not going to continue with developing this product, which triggered a steep fall in the share price of the company. What next for gene therapy & DNA/RNA market? Significant governmental and private equity funding has been pumped into gene therapy development, yet due to delivery, safety and efficacy issues, there is still considerable concern over the very small number of gene therapies set to launch over the forecast period, Belsey says. “The DNA/RNA market is highly innovative, but remains largely unproven and the market as a whole must demonstrate that it is possible to successfully develop and launch DNA/RNA products representing a range of technologies to attract valuable partnerships with Pharma/Biotech and secure significant funding revenues.” “With the recent failure of tgAAVCF- the sector’s most advanced product- confidence in investment in gene therapy companies has taken another tumble. However Datamonitor believes that if investors can tough out the temperamental share price fluctuations and the negative trial data, the high level of innovation, scientific expertise and investment will result in the successful launch of a range of highly efficacious products with the potential to treat diseases with a high unmet need.” “The successful development and approval of Pfizer/Eyetech’s aptamer Macugen (pegaptanib) has demonstrated that this is possible. This has given gene therapy companies and other companies operating in the DNA/RNA market hope, but these companies must now turn their scientific expertise into reality to help restore confidence to the market,” Belsey says. Ends
Notes for editors
To order these reports contact peter.barfoot@bioportfolio.com or telephone +44 1300 321501 or +1 415 680 2472 and a representative will get back to you. You can also order on line at: http://www.bioportfolio.com/cgi-bin/acatalog/search.html
|
|
| ||||||||