Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) saw its shares make an incredible gain on Wednesday after the company announced results from its early and mid-stage trials in cystic fibrosis (CF). The stock actually pushed to an all-time high on the back of these results.
These are the first results to demonstrate the potential to treat the underlying cause of CF in patients who have a severe and difficult-to-treat type of the disease.
For some quick background, cystic fibrosis is a genetic defect that causes a buildup of mucus in the lungs which leads to chronic infection and progressive lung damage.
The company’s studies showed a boost in lung function by 9.6% or more, which shocked reviewers. According to an analyst at Jefferies, anything more than 2.5% would have been considered a success, but this was absolutely mind-blowing.
These most recent results are looking to target 90% of those affected by CF, as a majority of these patients do not have an approved treatment option for this disease.
Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex, commented:
These safety and efficacy data are clear and compelling, indicating significant potential benefit for people with CF from each of these three different triple combination regimens. We will be collecting and evaluating additional data from these and other studies and will make a decision on which regimen(s) to take forward into pivotal program(s), which we expect to begin in the first half of 2018.
Shares of Vertex were last trading up 21% at $160.05, with a consensus analyst price target of $129.74 and a 52-week range of $71.46 to $167.00.