The driving application for growth in 2009 will be the progression of gene therapy trials. Such genomics technologies will be the Next Big Thing in drug development; however, gene therapy will facilitate, not supplant biopartnering as the defining dynamic of the new century.
Genomics represents the catalyst most capable of not only producing a level of never-before-seen drug development, but offers the best option to save many of the mired clinical candidates in need of "something new" to tow them out of their state of perpetually suspended ineffectiveness. The technology has shown potential in developing curative drugs, as well as facilitating the completion cycles of existing candidates that are stuck on issues related to therapeutic site delivery or manipulation of abnormal cellular activity.
Successful breakthroughs in even a portion of the many promising genetic therapeutics in mostly early- and mid-stage development will depend on the biopartnering collaborations that are behind most of the significant projects ongoing in the field.
RNAi developers such as biotechs Alnylam Pharmaceuticals Inc. and Sirna Therapeutics Inc. have deeply aligned themselves with big pharma heavyweights to make concerted efforts to get products of their own to the market or to use their patented technologies to get the stalled trials of their partners a second chance at success.
Alnylam has partnering agreements with big pharma mainstays Roche, GlaxoSmithKline plc, Biogen Idec Inc. and Takeda Pharmaceutical Co. Ltd., while Sirna, with no drugs in its pipeline anywhere near commercialization, was acquired by Merck & Co. Inc. in a December 2006 deal worth $1.1 billion.
In January 2009, Ark Therapeutics plc reported that its marketing authorization application for Cerepro, a gene-based therapy for operable malignant glioma, filed with the European Medicines Regulatory Agency cleared the validation stage and will proceed to a formal review via the centralized procedure, further portending the imminent arrival of gene therapy technology en masse onto the market. The drug is prescribed for brain cancer and has been granted orphan drug status by the European Committee for Orphan Medicinal Products and by the FDA's Office of Orphan Products Development.
In September 2008 the FDA refused to accept Introgen Therapeutics Inc.'s biologics license application for the gene therapy Advexin in the treatment of head and neck cancer. The company says that Advexin remains a priority, however. The drug is under review in Europe, and Introgen is appealing the FDA's decision not to review the BLA.
As soon as the first one of these gene therapy drugs gets to market in the U.S., in particular, it will signal the beginning of unprecedented developments in drug development and there will likely be an investor rush to align with genomics developers to capitalize on an eventual ground-breaking market phenomenon opportunity.
Biopartnering in the genomics space is projected to carry a trend that will surpass the current level of dealmaking and potentially cure pharma's clinical blues and bring the novel therapeutics in biotech pipelines within reach of establishing unseen approval rates.