Dreamstime
A month ago, analysts cast the biotech
in the role of victim to a notionally capricious U.S. Food and Drug Administration, after the FDA made a last-minute decision to convene an advisory committee to review its application for a drug called roxadustat.
The decision came 13 months after
(ticker: FGEN) had first applied for approval for roxadustat. FibroGen shares fell 24.7% as the company’s CEO,
Enrique Conterno,
complained at an investor conference that the FDA had previously told it multiple times that there would be no need for an advisory committee hearing.
Now, FibroGen has made another startling announcement about roxadustat. This one puts the company in a much less sympathetic light, and renders the advisory committee hearing the least of its troubles.
In its statement late Tuesday, FibroGen (ticker: FGEN) said that safety data it had touted for years from a Phase 3 trial of roxadustat had “included post-hoc changes to the stratification factors.”
In other words, the categorization of subgroups in the study had been adjusted after the trial was complete, something the company had not disclosed to investors. That data had been presented as though it were based on pre-specified stratification factors. The actual data based on the pre-specified stratification factors aren’t radically different from what FibroGen originally presented, but are different enough that it could have implications for the drug’s approval and use.
The data compared the safety profile of roxadustat, for which the company is seeking approval to treat anemia in chronic kidney disease, with that of epoetin-alfa, the current standard of care. “We continue to have confidence in roxadustat’s benefit risk profile,” said Conterno in a statement.
Yet in a note out Wednesday, Cowen analyst
Yaron Werber
said that the new analysis “shifts roxadustat’s safety profile to non-inferior vs [epoetin-alfa]” in the subgroup of patients who had begun dialysis within four months, referred to as incident dialysis patients. “This removes one of the main differentiators for the drug.”
Jefferies analyst Michael Yee, in a separate Wednesday note, wrote: “Bottom line is while overall the stats don’t have a huge change and the [company] believes conclusions still support an FDA approval – we believe the fact that Incident Dialysis is no longer ‘statistically’ superior – is a material change to the profile and one of the key prior advantages for [patients].”
FibroGen shares were down 28.6% in premarket trading on Wednesday. Mizuho analyst Difei Yang on Wednesday downgraded the stock to Neutral from Buy, and cut her target for the stock price to $29, from $72. FibroGen was trading at $24.65 early Wednesday.
FibroGen didn’t immediately respond to a request for comment on the downgrade.
“As members of senior management were preparing for the upcoming FDA Advisory Committee meeting, we became aware that the primary cardiovascular safety analyses included post-hoc changes to the stratification factors,” Conterno said in the Tuesday statement. “While all of the analyses set forth below, including the differences in the stratification factors, were included in the [new drug application], we promptly decided to clarify this issue with the FDA and communicate with the scientific and investment communities.”
The release goes on to provide safety data on the drug with and without the after-the fact adjustments.
“We have since begun a comprehensive internal review to ensure this doesn’t occur in the future. This review is in its early stages,” Conterno said on an investor call late Tuesday. “I don’t have any additional details to share at this time.”
Investors are likely to still have questions. In a note out early Wednesday, SVB Leerink analyst Dr. Geoffrey Porges called the incident a “major ‘oops.’”
The incorrect analysis, Porges noted, “had previously been disclosed in multiple press releases and appeared in numerous conference posters and presentations.”
FibroGen also said on Tuesday that the FDA advisory committee meeting on roxadustat had been tentatively scheduled for July 15.
Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com
