Here's Why Investors are Already Anticipating Vertex Trial Data Coming This Month
March 16, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor
Investors are waiting with bated breath for new Phase IIB trial data from Vertex Pharmaceuticals due later this month for its experimental cystic fibrosis drug VX-661, as hopes remain it could be a breakthrough treatment with blockbuster potential, the Boston Business Journal reported.
Although the U.S. Food and Drug Administration (FDA) has not yet approved the drug, the 40-patient trial currently being conducted is looking at the efficacy of two different dose levels of VX-661 in conjunction with Vertex's already-approved drug, Kalydeco, against a placebo. Vertex has said it will release the results by the end of March—and that based on its initial undisclosed findings, it has already started four separate Phase III trials of VX-66.
“The key question — one which has led to months of speculation — is exactly how effective it will turn out to be. However, what might seem like a simple measure of improved lung function is complicated by the many different gene variations which lead to CF, and the fact that different drugs can address certain ones but not others,” wrote BBJ reporter Don Seiffert on Friday.
“The Phase IIb data expected in coming days, for instance, is in patients with two copies of what's called the F508del gene. That's exactly the same population (about 22,000 worldwide) being targeted by Vertex's drug now awaiting FDA approval, called lumacaftor,” he said.
That drug combined with Kalydeco improved lung function by around 3 percent in its recent Phase III trial, a number biotech analysts have said could be boosted to as much as 6 percent by VX-661, raising hopes that it will be a blockbuster treatment for patients and Vertex alike.
“But ultimately, VX-661 is going to be tested in Phase 3 trials in patients with different types of gene mutations, including in those with one copy of the F508del gene who have residual ability to produce the protein which is deficient in CF patients,” wrote Seiffert. “That represents about 3,000 patients. Another, higher-risk trial will be in patients with one copy of the F508del gene who have minimal ability to produce the protein. And while that trial has a higher likelihood of failure, it has a bigger payoff in the form of another 17,000 patients who could be treated.”
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