Although we don't believe in timing the market or panicking over market movements, we do like to keep an eye on big changes -- just in case they're material to our investing thesis.
What: Shares of the Dutch gene-therapy company uniQure (QURE 0.54%) climbed by more than 50% today on extremely high volume after the company announced that Bristol-Myers Squibb (BMY 0.77%) agreed to invest $50 million upfront in the smaller company, giving it a 5% stake. Before the end of the year, Bristol will also acquire another 5% of uniQure, giving it a 10% stake overall.
Bristol's interest in uniQure reportedly centers around the company's gene-therapy platform aimed primarily at treating cardiovascular diseases. If this novel technology works, uniQure would be in line to receive $254 million in milestone payments for its lead product candidate S100A1, and up to $217 million for each additional therapy.
So what: Bluebird bio's recent breakthrough with its lead gene-therapy for a rare blood disorder has given this much-maligned technology a rebirth of sorts. And with powerful new vectors and a far deeper understanding of the genetic underpinnings of disease in hand, big pharma apparently believes now is the time to give gene therapy another shot, evinced, in part, by Bristol's lucrative deal with uniQure.
Now what: This next generation of cardiovascular gene therapies will get their first major test later this month when Celladon (EIGR 0.47%)releases the topline data from its ongoing midstage trial dubbed CUPID-2. This study is the largest gene therapy trial ever conducted for cardiovascular disease, and may act as a referendum on uniQure's related technology.(EIGR 0.47%)
What's intriguing about today's news is that Bristol is the latest big pharma to become an early stakeholder in this potentially game-changing technology. Pfizer, Johnson & Johnson, and Novartis, for example, previously snapped up large chunks of Celladon prior to the company's IPO. All told, Bristol's agreement with uniQure suggests that big pharma is gaining confidence that this new wave of gene therapies will indeed bear fruit and possibly usher in a new era of cardiovascular care.
