Vertex Pharmaceuticals' piecemeal approach to the world's cystic fibrosis sufferers took a hit as Kalydeco failed to meet its primary endpoint in a Phase III trial on patients with one form of the disease, sending the company's shares down as much as 6% premarket on Thursday morning before an eventual rebound.
In drug development, everything's a gamble, if you're doing something new and shooting at a big target. But there has to be a reasonable assumption that if safety issues aren't being glossed over and the efficacy data hold up, these top drugs can change standards of care and grab market share. So here's my pick of the likely big winners >>
While pedestrian investors wait on press releases, conference calls and trial results before acting on biotech stocks, some of the world's largest hedge funds have figured out how to speed up the process of publicity: Ask the FDA.
Biotech companies have set out to keep U.S. tax credits pinned to costs of developing drugs for rare or "orphan" diseases, seeking to escape the mammoth budgetary ax of Congress on Capitol Hill.
The FDA placed a partial clinical hold on U.S. development of a 200 mg dose of VX-135 after three patients in a European study showed signs of liver toxicity after taking 400 mg doses of the compound, Vertex spokesman Zach Barber confirmed via email.
Johnson & Johnson and Vertex Pharmaceuticals gave the thumbs-up to FDA's new breakthrough drug program, designed to speed development of promising therapies.
It's a great time to helm a large biotech company, generally. Big biotech players overall posted major growth last year, and their CEOs got richer in the process. Most got fat raises while others saw their bundles of pay pale in comparison to 2011 figures. We surveyed the total compensation packages of the 10 largest biotech companies based on market capitalization as of early May, sleuthing mostly proxy statements for the financial details on pay for some of the most powerful people in the industry. Check it out >>
The R&D numbers for the top 10 biotechs may only amount to a fraction of what you'll find in Big Pharma. But unlike the giants, which are trying to keep a lid on multibillion-dollar budgets, you'll find a much faster crowd when you turn your gaze to the biotechs.
Vertex Pharmaceuticals jolted Wall Street with a fresh batch of midstage data on one of its combo therapies against cystic fibrosis.
EvaluatePharma researchers totted up sales for the last 5 years' worth of analysts' blockbuster picks--and found plenty of bad bets.