Vertex $VRTX has come up with a stack of positive Phase III data points for their cystic fibrosis triple including the experimental VX-659. And its R&D group says they can ride these results all the way to an FDA approval.
Right now we’re just getting the primary endpoint readout, which is impressive. VX-659 is being credited with a 14% placebo-adjusted improvement from baseline in FEV1 for patients with one F508del mutation and one minimal function mutation, with a clear score on the p value (p<0.0001).
In a separate Phase III patients homozygous for the F508del mutation already taking Symdecko saw an average 10% improvement with VX-659 added on.
Now Vertex CMO Reshma Kewalramani says they will wait for the Phase III numbers on their other big add-on, VX-445, in Q1 before deciding on the regulatory strategy as they look to expand their hold on CF.
Of particular note: These new data points are right in line with Phase II results, offering some clear parallel support for their combination to take to regulators.
The data “live up to the hype,” noted Stifel’s Paul Matteis, highlighting the close comparison between the last two trials.
While details (as expected) are limited beyond the primary endpoint, from press release there’s nothing to nitpick (in our view) for what has been reaffirmed as a transformational drug regimen in “het-min” CF patients, a population that represents a multi-billion dollar opportunity with no targeted therapies approved today.
Michael Yee at Jefferies also joined the cheering section.
In homozygous which is a market already well penetrated by Orkambi and Symdeko, this triple of ‘659 is meant to significantly improve FEV efficacy from a current +4% to +14%, which is a huge improvement and benefit for patients and will eventually replace Orkambi/Symdeko over time and creates a huge barrier to entry given the overall efficacy/safety profile of ‘659 in this population.
Those views helped Vertex shares gain about 4% in early trading Tuesday.
Vertex has been surrounded by rivals looking to carve off a piece of the blockbuster market that the Boston biotech has been carving out for itself. In several cases, we’ve seen competitors stumble with mixed or poor data. Proteostasis has managed to escape that fate, with some positive early-stage numbers of its own. But Vertex has been dominant in CF for years now, and appears determined to stay well in the lead.
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