Several months after rebranding, an old Martin Shkreli company touts promising pivotal data in a rare kidney disorder
If all goes well at the FDA, Calliditas could cross the finish line with its inflammatory kidney disease treatment Nefecon next month. But Travere Therapeutics isn’t far behind — and on Monday, the former Martin Shkreli company unveiled some promising pivotal data that may give Calliditas a run for its money.
After 36 weeks of treatment with Travere’s sparsentan, patients with a rare condition called IgA nephropathy saw a mean reduction in proteinuria (excessive protein in the urine) of 49.8% from baseline, the company said. That compares to a mean reduction of 15.1% in a group of patients who took Avapro, a current standard of care (p<0.0001).
“We continue to hear from the nephrology community that there is a clear need for a therapeutic option to meaningfully reduce proteinuria over and above widely used ACE and ARB treatments,” CMO Noah Rosenberg said on a call with investors.
IgAN is a condition that damages the filters, or glomeruli, inside the kidneys, causing the organs to lose their function. Travere’s Phase III study, dubbed PROTECT, enrolled 404 patients who experienced persistent proteinuria despite receiving ACE or ARB treatment. They were randomized to receive once-daily doses of either sparsentan or Avapro, an ARB treatment.
The interim results showed promise for the secondary endpoints, which measure estimated glomerular filtration rate (eGFR) after about one and two years, according to Travere.
“The Company believes that preliminary eGFR data available at the time of the interim analysis are indicative of a potential clinically meaningful treatment effect after two years of treatment,” it said in a statement, adding that topline results from the confirmatory endpoint analysis are coming in the second half of 2023.
Meanwhile, CEO Eric Dube plans on filing for an accelerated approval in the first half of next year.
Calliditas already has its foot in the door at the FDA, after submitting an NDA for Nefecon to treat IgAN back in March. Regulators set a Sept. 15 PDUFA date — and if approved, the drug would become the first approved treatment in the US for the rare kidney disorder.
But while it’s difficult to compare trials, it’s looking like sparsentan may have the upper hand in proteinuria reduction. In November, Calliditas said data from the first part of a Phase III study showed patients who took Nefecon had a 31% reduction in proteinuria after nine months, compared to a 5% reduction of those on placebo (p=0.005).
Back in February, Travere touted a Phase III win in another rare kidney disorder called focal segmental glomerulosclerosis. A full readout from that study is also expected in 2023, though Dube says there are enough data now to pursue accelerated approval.
Travere was founded as Retrophin by the “Pharma Bro” back in 2011, but has since gone to great lengths to sever its ties to Shkreli (including a name change back in November). The company gave Shkreli the boot back in 2014, shortly after he bought the rights to Thiola and jacked the price by 2,000%, according to an FTC complaint.
Upon rebranding the company, Dube told Endpoints News that the old name “doesn’t reflect who we are today, and the role that we want to play going into the future.” The new name is inspired by the Latin roots of the words “path” (tractus) and “truth” (ver).