Martin Shkreli’s old company touts PhIII win, guns for accelerated approval
Three months after rebranding to shed the last of its ties to convicted “Pharma Bro” Martin Shkreli, Travere Therapeutics is putting its best foot forward with an upbeat Phase III analysis and plans to seek accelerated approval in a rare kidney disorder.
Travere — formerly known as Retrophin — says its lead candidate sparsentan met the pre-specified interim endpoint in a study of 371 patients who have focal segmental glomerulosclerosis, a disorder where kidneys’ filtration breaks down and proteins leak into the urine. For many patients, the disorder leads to end-stage kidney disease.
In the ongoing Phase III DUPLEX study, patients were randomly given sparsentan or Avapro, the current standard of care. At 36 weeks, 42% of patients in the treatment arm achieved the FSGS partial remission of proteinuria endpoint, versus 26% in the Avapro group, according to Travere.
It’s still too soon to get a read on the confirmatory primary endpoint, which is the rate of change in estimated glomerular filtration rate (eGFR) over 108 weeks. But there are enough data to pursue accelerated approval, Travere added.
“For decades people living with FSGS have faced daily challenges in controlling proteinuria and a fear of progressing to transplant or dialysis because current treatment options are not enough,” CEO Eric Dube said in a statement. “As we move ahead, our organization will be focused on maintaining high quality in this ongoing study, and on continuing our engagement with regulators to enable submissions for accelerated approval with the available data set.”
DUPLEX is fully enrolled and on track for a readout on the confirmatory primary endpoint in the first half of 2023, according to the company. The drug is also being studied in Phase III for IgA nephropathy, with an interim analysis expected in Q3 of this year.
Travere has come a long way since it was founded by Shkreli in 2011. 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. After being ousted, Shkreli went on a “war path,” threatening to harm the company and the reputations of those in it, his successor Stephen Aselage testified, per a CNBC report.
The biotech filed a lawsuit against Shkreli in 2015, which the infamous biotech exec followed with a countersuit. In 2019, he tacked on another accusation against three board directors seeking $30 million in damages. Retrophin paid an undisclosed amount to Shkreli later that year to settle all of the disputes, and sever its ties with the “Pharma Bro,” who’s currently serving a 7-year sentence for defrauding investors of his hedge funds. Part of the case involved Shkreli’s use of Retrophin stock to pay off those investors.
The company changed its name to Travere in November, as the old name “doesn’t reflect who we are today, and the role that we want to play going into the future,” Dube told Endpoints News.
The new name is inspired by the Latin roots of the words “path” (tractus) and “truth” (ver).
The biotech’s pipeline also includes TVT-058, a Phase II candidate for classical homocystinuria that it snagged in its October buyout of Orphan Technologies. Travere agreed to put down up to $517 million for the low-profile company and its enzyme replacement therapy, including $427 million in milestones.