logo
Plus   Neg
Share
Email
Comment

Vertex Reports Final Data From Phase 2 Combination Study Of VX-809 And KALYDECO

Vertex Pharmaceuticals Inc. (VRTX) announced final data from a Phase 2 study of VX-809 and KALYDECO (ivacaftor) that exhibited statistically significant improvements in lung function among adults with cystic fibrosis who have two copies of the most common mutation in the cystic fibrosis transmembrane conductance regulator gene, F508del.

The study randomized homozygous F508del patients to three treatment groups that assessed increasing doses of VX-809 (200mg, 400mg or 600mg; QD) alone for 28 days, followed by VX-809 in combination with KALYDECO (250mg; q12h) from day 28 to 56. When patients received the two medicines in combination (day 28 to 56), there was a statistically significant improvement in lung function (percent predicted forced expiratory volume in one second, FEV1) in each of the homozygous treatment groups compared to placebo, noted the company.

The greatest improvements in lung function were observed in patients who received 600mg of VX-809, the highest dose evaluated in the study, in combination with KALYDECO. Vertex stated that these data support its plans to initiate a pivotal program in early 2013, which is expected to evaluate VX-809 (600mg) in combination with KALYDECO (250mg) in homozygous patients, pending discussions with regulatory agencies.

Most adverse events observed during the 56-day study were mild to moderate in severity across all treatment groups and similar between treatment and placebo groups.

Besides, the study included an exploratory treatment group that looked at a subset of heterozygous patients who have one copy of the F508del mutation and a second mutation that is not expected to respond to KALYDECO dosed as monotherapy. This group of patients received VX-809 (600mg) and KALYDECO on the same dosing schedule as homozygous patients. Heterozygous patients who were treated with the combination experienced a mean absolute improvement in lung function compared to placebo from day 28 to 56.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
French drug giant Sanofi SA Monday announced an agreement to buy US-based hemophilia drugmaker Bioverativ Inc. (BIVV) for around $11.6 billion. The deal expands Sanofi's presence in specialty care and strengthens leadership in rare diseases. Sanofi shares were losing around 4 percent in the early morning trading in Paris. Swiss banking giant UBS Group AG reported Monday a hefty net loss in its fourth quarter hurt by a charge related to US tax law changes. Pre-tax profit, however, increased from last year with improved operating income and lower expenses. Further, the company announced an increase in dividend, as well as a share buyback of up to 2 billion Swiss francs. Angry protesters are planning more than 50 demonstrations on Friday at several Tim Hortons locations across Canada after some franchise owners in Ontario slashed workers' benefits as well as paid breaks in response to the Ontario government's minimum wage hike. The protests are being organized by advocacy group Leadnow and the Fight for $15 & Fairness Campaign.
comments powered by Disqus
Follow RTT