Travere Therapeutics’ kidney disease strategy has hit a snag. A pivotal phase 3 trial of sparsentan missed its primary endpoint, but the biotech is undeterred, outlining plans to talk to the FDA about a path to market based on other aspects of the data set.
The randomized clinical trial compared the dual endothelin angiotensin receptor antagonist to irbesartan, a drug sold by Sanofi as Avapro, in patients with focal segmental glomerulosclerosis (FSGS). After 108 weeks, Travere saw a “favorable difference” on the estimated glomerular filtration rate (eGFR) total, but the difference fell short of statistical significance.
Failure on the U.S. primary endpoint, eGFR total slope, and the European primary endpoint, eGFR chronic slope, sent shares in Travere down 24% to $17.10 in premarket trading. Travere took positives from the data set, though, such as a reduction in proteinuria, a sign of kidney disease, and wants to talk to the FDA.
“We believe there are strong rationale to engage with regulators to understand if there is a path to enabling sparsentan to have a role in treating FSGS,” Travere CEO Eric Dube, Ph.D., said on a conference call with investors to discuss the data. “In particular, we believe that the trends on eGFR slope and the sustained proteinuria reduction, paired with the well-tolerated safety profile observed in the study, give us reasons to present the totality of the sparsentan data and explore a potential path to registration.”
William Rote, Ph.D., senior vice president of R&D at Travere, provided further details on the call. The biotech wants to put together some subgroup analyses before it talks to the agency, suggesting the meeting will happen in the summer and minutes will be available one month later.
Rote, while noting that he cannot speak for the FDA, went on to outline why Travere thinks the agency may be open to approving sparsentan in FSGS. According to Rote, the FDA has “commented in the past that this is a difficult disease to study” and maintained that “this is a rare disease that has a very high unmet medical need.”