As BNET noted before, the poor gene therapy folks haven't had it easy. Their approach has yet to gain FDA approval, so there's no regulatory precedent. And toxicity issues -- including the much-publicized death of Jesse Gelsinger in 1999 -- have tarnished the technology's reputation. Neither of these factors warms the heart of potential partners.
Novartis is one of the few pharma firms with a deep interest in gene therapy. When Ciba-Geigy and Sandoz merged to form Novartis in 1996, both brought gene therapies to the table. But what's interesting is that most of the scant industry interest in gene therapy to date has been for oncology...and GenVec also has a Phase III gene therapy for pancreatic cancer on the partnering table.
The first interim analysis of GenVec's Phase III pancreatic cancer trial with TNFerade looked pretty good: a 25 percent reduction in risk of death (though not quite statistically significant) and 12-month overall survival of 39.9 percent vs. 22.5 percent for the control arm. As a reference, Tarceva (erlotinib) gained approval in pancreatic cancer by showing 12-month overall survival of 23.8 percent versus 19.4 percent for Gemzar.
The second interim analysis in the TNFerade study is only months away. And Novartis is no stranger to oncology: the pharma markets several cancer drugs including leukemia superstar Gleevec (imatinib).
So why did Novartis choose to license a preclinical hearing loss program rather than a Phase III pancreatic cancer program, when the two are based on the same adenovector technology?
I can think of two possible reasons: 1) Novartis didn't want TNFerade, or 2) Novartis did want TNFerade but would rather pay a scant $7 million upfront to test the waters in a preclinical program before committing to Phase III economics ahead of a risky data read-out. Or maybe Novartis just thought the hearing loss program was cool...but heck, you gotta wonder if somebody is eventually going to take the plunge and place a bigger gene therapy bet on TNFerade.