Going for broke, Tricida is slamming the brakes on a pivotal trial early while planning a run at the FDA
Two years after a bitter setback at the FDA, with its cash reserves ebbing away, Tricida is executing a high-stakes gamble on winning a swift approval at the FDA. A win would put them back in play with a new drug to commercialize. A loss, as one Wall Street analyst recently assessed, would take the stock to zero.
Today Tricida $TCDA announced an “administrative stop” for its VALOR-CKD trial after watching 237 patients reach a primary endpoint event — renal death, end-stage renal disease, or greater than or equal to 40% reduction in estimated glomerular filtration rate — in the renal outcomes study for their metabolic acidosis and CKD drug veverimer. They’ll continue to track events into Q3 as they look for a top-line readout in the near term.
When they started out in the fourth quarter of 2018, Tricida researchers had set a target for 511 events, which the biotech says would stretch the study into 2024. But operating capital will be effectively tapped out by the early part of Q2 2023.
Execs say they got the FDA to sign off on the early trial stop after reviewing their financial situation with regulators along with study close-out procedures.
Phil Nadeau at Cowen recently assessed their chances, noting:
Assuming a true hazard ratio (HR) of 0.70, the trial would be 78% powered at 250 events. Switching from power to observed HR, VALOR-CKD could be successful at 250 events if the observed HR is 0.78. There is only a modest increase to 0.79 observed HR should 300 events be in the final analysis. This calculation on the observed HR required for significance is based on 300 simulations on trial outcomes.
A win, he added, wold spur a big rally — presumably setting up a raise — while a fail would amount to an extinction level event.
None of this will come as a big surprise to the biotech’s investors, as Tricida execs have been painfully clear about what they’re doing and why. CEO Gerrit Klaerner was equally transparent back in the summer of 2020, as regulators wound up to deliver a CRL for the drug.
This year, though, the industry has been roiled by a savage bear market for biotech stocks, and Tricida wants a few months of operating revenue in the bank when the biotech will make a fresh pitch.
“With regards to timing overall towards potential commercial launch, presuming we have the announcement of the VALOR study early in Q4, we would anticipate filing or resubmitting the NDA in Q2 of ‘23 and have a potential PDUFA date in Q4 of 2023,” Tricida CFO Geoff Parker said during their Q1 call a few days ago. “That’s a six-month review for a resubmission. And then roughly speaking, we would have a launch in Q1 of 2024. So presuming positive data on VALOR-CKD, we will put those plans into motion later this year.”