A panel of FDA advisers voted unanimously, 13 to 0, that Reata Pharmaceuticals' chronic kidney disease (CKD) med was not effective.
The committee said bardoxolone did not prove effective in slowing the progression of CKD in patients with Alport syndrome and that its benefits did not outweigh its risks based on data Reata submitted.
Trading of the company's share was halted Wednesday due to the Cardiovascular and Renal Drugs Advisory Committee meeting. The biotech ended last week at $78.83 apiece and closed Tuesday at $54.42 per share. The advisors' recommendation sent shares tanking 38% to $33.64 a piece as of 10:17 a.m. Eastern time Thursday.
The FDA is slated to decide the drug's fate Feb. 25, 2022. If given the green light, Reata's drug would be the first approved treatment for Alport syndrome. But the likelihood of approval for the rare indication "has come down substantially," Jefferies analysts wrote in a note after the day-long adcomm meeting.
Reata said it plans to provide additional information and data leading up to decision day, including potential data from non-Alport studies. The Jefferies analysts warned, though, that it's "unclear" how new data from such studies would be able to convince the "more conservative" Cardiovascular and Renal unit at FDA.
The rare genetic disease can lead to progressive loss of kidney function and kidney failure as well as deafness and potential loss of vision, the FDA said.
While bardoxolone achieved its primary and key secondary goals in a phase 3 trial, dubbed CARDINAL, the agency's review team questioned whether the drug actually reduced disease progression.
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The pharma's data don't show the drug is effective at slowing the loss of kidney function or reducing the risk of buildup to kidney failure, the regulator's review team wrote in a briefing document released Monday. The company also didn't submit data from an animal model of Alport syndrome "or other adequate and well-controlled clinical trials in AS or CKD" that prove the treatment's ability to tamper kidney function loss.
In the public hearing session, one parent of a patient asked the advisers to not "discount the human aspect."
The Plano, Texas, biotech submitted its paperwork for approval in February. Prior to that filing, Reata met with the FDA in January and September 2020 to see about a potential accelerated approval. Based on an interim analysis of the phase 3 trial, the regulator "did not agree with the proposed approach," and Reata ditched the accelerated path.
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Reata delivered year one data from the CARDINAL study in November 2019, a month after AbbVie ended a nine-year partnership on the drug with the biotech. Reata paid AbbVie $300 million to regain the rights to bardoxolone, and the biotech's CEO claimed AbbVie had not been active in the partnership in recent years.
The drug has a long development history. Reata linked up with AbbVie in the early 2010s with deals totaling more than $800 million. A data monitoring committee found that patients with CKD in a phase 3 study had a higher rate of heart-related side effects in 2012. In response, Reata halted the program, eliminated half its workforce and returned to the drawing board.
Then it moved the into a midstage study in a different indication in 2014. Since then, the company has tested the drug in multiple types of rare CKD besides Alport syndrome.
The drug is also in a 550-patient pivotal trial in patients with autosomal dominant polycystic kidney disease and in a 70-patient midstage study in people with CKD at meaningful risk of progression to end-stage kidney disease.
Editor's note: This story was updated Thursday, Dec. 9, at 10:18 a.m. Eastern time to include analyst commentary.