Dutch drug developer ProQR Therapeutics is swinging for a $75 million Wall Street debut to fund its work on a treatment for cystic fibrosis, looking to compete with market leader Vertex Pharmaceuticals.
There's no mistaking Geoffrey Porges' opinion on Vertex's chances of success with its combo study of VX-809 and Kalydeco. After spending the weekend at the European Cystic Fibrosis Society meeting in Sweden, the prominent Bernstein analyst came away even more soured on the experimental CF drug's potential, with pivotal data for the combo looming on the horizon.
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 >>
Novartis notched an FDA approval on Friday for a dry-powder formulation of an antibiotic that fights a type of bacterial infection in patients with cystic fibrosis (CF).
Cambridge, MA-based Vertex Pharmaceuticals says it nailed solid mid-stage data for VX-787, an experimental influenza drug in a new class of medications that promises to prevent the virus from replicating. And the biotech added that it will be in hot pursuit of a new collaboration on the drug.
The evolution of hepatitis C treatment threatens to leave today's dominant companies with fossilized offerings. Vertex and Merck have the state-of-the-art approved drugs against the virus, but both companies are chasing after Gilead Sciences, Abbott Laboratories and others with programs that could be the first to win market approval with pill-only options. Read the full report >>
Vertex may be playing catch-up with the likes of Gilead and Abbott in the race to develop an all-oral hepatitis C combo, but the biotech is teaming with some major league players in this high-stakes game. Vertex announced this morning that it will pair up with J&J and GlaxoSmithKline on a pair of Phase II matchups.
A little more than a year after Vertex licensed in a pair of "nucs" from Alios BioPharma in a high-stakes bid to develop an oral, interferon-free approach to hepatitis C, Vertex is jettisoning one as a dud while racing ahead with the second into a mid-stage study slated to begin in a matter of weeks.
Hepatitis C drug developer Achillion Pharmaceuticals expects to haul in proceeds of a $41.8 million sale of common stock to QVT Financial. And the funding comes ahead of some important clinical trial results from the New Haven, CT-based developer's pipeline of hep C treatments.
Vertex shares took a nasty tumble this morning, freefalling 18% on the news that the company was forced to revise the data on its closely watched cystic fibrosis combo therapy after learning that the initial interim data report had been bungled.