- A Food and Drug Administration advisory panel on Thursday roundly rejected Reata Pharmaceuticals' experimental drug for a subset of patients with chronic kidney disease, putting the future of the medicine in doubt.
- The FDA asked the expert committee whether Reata's research showed that the drug, bardoxolone, slows kidney disease progression in patients with Alport syndrome and that its benefits that outweigh its risks. All 13 panel members voted no.
- While the FDA doesn't have to follow the advice of its outside experts, it usually does. And the agency made its doubts about Reata's drug clear, telling panel members in briefing documents published ahead of the meeting that it didn't believe bardoxolone had shown a clinical benefit and had potential safety concerns.
The company's shares plummeted by more than 40% in early Thursday trading after taking a hit earlier in the week when the FDA released the documents that outlined its concerns with Reata's data. The shares, now trading at about $30, had traded above $150 in June.
Chronic kidney disease patients with Alport syndrome currently have no approved therapy, and Reata isn't ready to give up on the drug. The company said it will keep talking with the FDA and will provide additional information and data before the agency's decision date of Feb. 25.
Reata is studying bardoxolone in other patient populations and may be able to use some of that data in its efforts to change the FDA's mind. Still, analysts were skeptical that the drug has any future in the U.S.
Baird analyst Brian Skorney called the advisory meeting a "train wreck" for Reata and said salvaging the application without another multiyear study is a long shot. He said he sees almost no chance of approval based on the Phase 3 study the company used in its application.
The FDA agreed with Reata that its Phase 3 study met the prespecified goals, but review team members said they weren't convinced the findings translated into a meaningful benefit for patients in slowing the loss of function in kidneys and keeping them from failing.
Drugmakers are in touch with the FDA during the development process, and Reata told analysts that it followed the agency's guidance for researching bardoxolone. It's possible that the "goal posts" were moved at some point during the process. Still, the episode suggests a breakdown in communication between Reata and the FDA, Skorney said.
It's "impossible to have confidence that the company isn't misreading regulatory cues here," Skorney wrote in a note to investors. That raises concerns about the company's other main product in development, omaveloxolone for Friedreich's ataxia, he said.