Orphan disease drug developers such as Vertex Pharmaceuticals operate differently from big pharma: they often take one or two “best shots” at diseases where there are substantial unmet needs, pour all of their resources into developing a winning molecule that actually works, and either succeed or fail based on the clinical trial results. In the case of Gilead’s Sovaldi for Hepatitis C, the company has managed to create what is being touted as a true cure for the disease. Similarly, Vertex hopes to be able to achieve similar results in the cystic fibrosis sphere with its experimental drug Lumacaftor, which is also known as VX-809. The viability of the drug developer’s CF therapy hangs in the balance of two major clinical trials, which are set up wrap up in the summer of 2014. In the meantime, the cystic fibrosis community waits.
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For starters, Vertex has already proven that it can craft effective CF therapies. Take, for example, its first foray into CF, Kalydeco, which functions as a CFTR potentiator and viable treatment for some cystic fibrosis patients. The problem with Kalydeco is not whether it works or not — at issue is the fact that it only works with about 10% of the CF patient population. On the ledger sheet, the drug is successful, since the company can command premium prices for it. But in terms of developing a pervasive cystic fibrosis treatment that could potentially change the outcomes for hundreds of thousands of suffers both now in the future, Vertex needs to effectively pair Kalydeco with CFTR correctors, which would in effect augment the treatment so that it could be effective in treating the entire CF patient population. This is where Lumacaftor comes in.
As a CFTR corrector, Vertex hopes that Lumacaftor can augment Kalydeco to expand its efficacy, which is what is currently being tested in Phase III studies, dubbed the TRAFFIC and TRANSPORT trials, which are targeting CF patients who are homozygous for the F508del mutation. The F508del mutation accounts for about 50% of the CF patient population, which means that if the drug pairing works well, then it will offer a true breakthrough treatment option for cystic fibrosis.
Investors have bristled at some of the bumps in the road for Lumacaftor, however, the news to this point has been more circumstantial than conclusive. In an earlier study of Lumacaftor, which was a “monotherapy” study that only tested the drug without the pairing of Kalydeco, the results were not positive. Yet, considering that Vertex’s gambit on Lumacaftor has chiefly been to combine it with Kalydeco to reach more of the patient population, its solo performance does not necessarily impact what TRAFFIC and TRANSPORT will tell us in mid-2014.
For cystic fibrosis patients and their families, many of whom follow CF research news closely, the ups and downs associated with drug development for the disease can constitute a roller coaster ride for many. However, Vertex’s achievements in the CF have been largely encouraging, and considering the company is primarily focused on its cystic fibrosis therapies, the CF patient population can rest assured that by summer we will have a good idea of whether or not Lumacaftor can deliver on expanding the effectiveness of Kalydeco.