Retrophin Switches Name to Travere Therapeutics in Reflection of Evolving Pipeline

Eric Dube Travere CEO

Photo courtesy of Travere.

Retrophin announced today that it is changing its global corporate name to Travere Therapeutics. The new name is meant to reflect the company’s dedication to helping people with rare diseases, as well as its pipeline for potential first-in-class therapeutic candidates. Travere Therapeutics will also begin trading under the new ticker symbol, “TVTX” on the Nasdaq Global Select Market beginning on Nov. 19.

Retrophin had originally stood for "Re(place) (dys)trophin," according to former founder Martin Shkreli. However, Eric Dube, Ph.D., chief executive officer of Travere Therapeutics said the name was no longer relevant, as the company is not working on a treatment for DMD.

“Over the last several years we have emboldened a patient-inspired mindset in all aspects of our organization, and championed our mission of identifying, developing and delivering life changing therapies to people living with rare disease,” Dube said. “We developed this new name to reflect our commitment to forging new paths with patients to deliver hope and innovation, and to honor our remarkable evolution. As we enter an exciting period of our journey led by the promising future of our pipeline of potential first-in-class therapies, now is the right time to transition to Travere Therapeutics.”

Travere Therapeutics is currently working on therapeutic candidates for rare disorders in nephrology, hepatology and metabolism. At the moment, the company’s pipeline is being led by sparsentan, which is an investigational product candidate in Phase III clinical development. It has a duel mechanism of action combining endothelin receptor type A blockade with angiotensin receptor blockade.

Sparsentan, in particular, is being examined for the treatment of IgA nephropathy (IgAN). The company announced back in September that the first 280 patients had been enrolled in its Phase III PROTECT study, evaluating the safety and efficacy of the product. The goal of the trial was to evaluate the proteinuria efficacy endpoint in at least 280 patients after 36 weeks of treatment.

“With a lack of effective and durable treatment options available for people living with IgAN, many patients progress to end-stage renal disease requiring kidney transplant or dialysis,” said Noah Rosenberg, M.D., chief medical officer of what is now Travere Therapeutics, Inc. “We believe the continued progress of our PROTECT study reflects the unwavering dedication of patients and investigators to support the development of treatment options that could have the potential to delay transplant or dialysis.”

PROTECT is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled Phase III clinical trial that is expected to enroll a total of 380 patients with IgAN. Topline efficacy data from the 36-week proteinuria endpoint analysis are expected in the third quarter of 2021.

In order to further expand its pipeline, Travere Therapeutics, Inc. also acquired Orphan Technologies Limited earlier this month. Under the terms of the agreement, Travere Therapeutics, Inc. made an upfront payment of $90 million in cash at the closing of the transaction. The company will now have access to Orphan Technologies Limited’s product candidate OT-58 for the treatment of classical homocystinuria (HCU).

“We are excited to begin working with the HCU community to develop a deeper understanding of how we can continue to integrate their perspectives into the development of OT-58, and help address their unmet needs,” said Dube, at the time of the announcement. “We look forward to building upon the promising potential of OT-58 with the goal of developing and ultimately delivering the first disease modifying therapy for people living with HCU.”

Orphan Technologies shareholders remain eligible to receive up to $427 million in additional cash payments contingent upon the achievement of key milestones in the development and commercialization of OT-58.

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