Vertex Pharmaceutical‘s drug Kalydeco is currently prescribed to the vast majority of eligible cystic fibrosis patients in the U.S. and Europe. Investors are now waiting for the results of a late-stage trial due out in the middle of this year for signs of the company’s future potential for growth.
According to the news website Bio Flash, the Boston-based global biotechnology last Wednesday announced total revenues of $1.2 billion for 2013, which represents a 20 percent drop from 2012. Less than a third of of revenues came from Kalydeco, but investors believe that the drug, as well as others in development to treat cystic fibrosis, will be critical in the coming years, as Vertex seeks to get the drug approved in more areas of the world, and in a wider patient population in the areas where it is already approved.
“Our strategy is to reach as many people with CF as possible with our medicines and to enhance the benefit for those that we treat,” said Vertex CEO Jeffrey Leiden in a conference call with investors Wednesday, quoted by Bio Flash. “This will be a very important year for patients, for Vertex and for our investors.”
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Kalydeco is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator and is taken orally. It is administered to help the CFTR protein function more normally once it reaches the cell surface, and it helps to hydrate and clear mucus from the airways so the patient can breathe better. The drug was approved in January 2012 in the U.S. to treat a rare form of cystic fibrosis in patients ages 6 years and older who have a gene mutation called G551D. In the U.S., that represents 1,200 patients, about 4 percent of the 30,000 patients nationwide with the deadly disease.
Besides Europe, Kalydeco has already been approved in Australia and Canada, but is pending approval in both areas for public reimbursement before can be made available to most of the 300 patients in those two countries who would benefit from it.
By the middle of this year, Vertex will report its results from two phase 3 trials of the drug in combination with another development, VX-809, which will probably determine Kalydeco’s market. Earlier this week, Vertex announced the results from one of those Phase 3 clinical trials in patients with the R117H mutation of cystic fibrosis aged 6 and older.
The company reported that the study did not demonstrate enough improvement in lung function to meet the primary endpoint. However, participants ages 18 and older did show significant improvement, and Vertex plans on discussing the results with the U.S. Food and Drug Administration (FDA).
The trial aims to test both drug’s effectiveness in treating the most common form of the disease, the so-called F508del homozygous population, which accounts for 40 percent of total CF patients. Leiden said that such approval would mean an additional 28,000 patients or more for the company’s drugs.
Bio Flash says that the company expects its annual revenue to be essentially cut in half in 2014. In Wednesday’s announcement, the company said it expects revenues of $570 million to $600 million in the coming year, with $470 to $500 million of that coming from Kalydeco. The other main source of revenue, its drug for hepatitis C, Incivek, accounted for nearly half of Vertex’s total revenues in 2013, but is expected to plummet in the next year as new drugs for the disease with fewer side effects are expected to hit the market. Vertex said in October it would stop investing in future growth of Incivek, and laid off 370 employees — or 15 percent of its staff — as a result.
AbbVie is currently enrolling participants in a long-term clinical trial to test a new investigational Cystic Fibrosis therapy targeted. Click on the company’s icon below visit the clinical trials page and get more information on where the trials are being held in the U.S.:
DISCLAIMER: BioNews Texas is a publishing company that occasionally focuses on the clinical trials industry. The information provided in this article is designed to help educate patients on clinical trials that may be of interest to them, based on the topic of the story, and to help patients contact the centers conducting the research. BioNews Texas is neither promoting this research nor involved in conducting any of these trials. Some study summaries have been edited for clarity purposes to make them easier to understand.