Regulatory

Vertex’s rapid-fire PhIII program hits a roadblock at the FDA. Will regulators force rival Galapagos to slow down too?

Vertex’s R&D team doesn’t make many mistakes. So during yesterday’s Q1 call, a group of analysts were quick to pick up on a rare stumble that just might make it more vulnerable to a rival like Galapagos.

Geoffrey Porges, Leerink

While noting plans for launching a new Phase III effort with a combination drug using VX-445 with Symdeko, following up as planned, investigators $VRTX also conceded that the FDA sees its deuterated version of Kalydeco — VX-561 — as a novel agent that needs some additional dose-ranging trial work before regulators will allow a move into Phase III.

Vertex bought ‘561 from Concert a little more than a year ago for $160 million in cash and another $90 million in milestones, taking over the Phase II trial.

Geoffrey Porges at Leerink sees a delay on VX-561 of at least a year, forcing the shift of focus to the VX-445 combo now as a kind of Plan B.

This means that the company is now running two roughly equivalent horses in the same expensive race, and while the risk mitigation from this strategy is laudable, it does seem more expensive, in terms of trial costs, time and revenue foregone, than is necessary. Vertex may still develop a Vx561 containing once-daily triple combination, but such a product might not come to market now until 2022 or 2023. The FDA’s stance does provide some reassurance about Vertex’s competitive position – the FDA is clearly signaling that there are no short-cuts (at least in the US) to traditional good drug development practices in this indication.

That “competitive position” he’s referring to deals with Galapagos $GLPG, which launched an exploratory study called FALCON for its triple earlier this week. And what the FDA finds good for Vertex, they’ll likely find good for Galapagos, where the dose-ranging standards may also likely to slow things down. Notes Michael Yee from Jefferies:

Thus since GLPG has all new NCE’s they will need to do lots of dose-finding work requiring lots more Phase IIs than the “quick” studies they’re doing now…so while GLPG might have “data” for FALCON study H2-18 and more data in 2019 — these are simple studies and will need thorough work per FDA read-through.

Brian Skorney

Baird’s Brian Skorney also took a shot at Galapagos.

(W)e believe it shows clear regulatory precedent for potential competitors who think they can move a combination of three new chemical entities into late stage development without demonstrating the clinical attributes and liabilities of each component (we’re looking at you Galapagos).


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