Dive Brief:
- Shares of Ocera Therapeutics rose March 8 after the company announced new data from a Phase 2b study of its lead drug OCR-002 in hepatic encephalopathy (HE), a debilitating neurocognitive complication of liver cirrhosis or liver failure.
- Top-line results from the same trial announced in January had shown the drug failed to meet its primary and secondary endpoint, precipitating a 70% nosedive in the value of Ocera stock.
- Despite the failure, Ocera still hopes to push OCR-002 into Phase 3 testing and plans to meet with the Food and Drug Administration in the third quarter to inform future development of the compound. Wednesday's more detailed results, which showed a correlation between reduction of ammonia and clinical improvement, could help Ocera in those discussions.
Dive Insight:
Presenting at the Cowen and Company 37th Annual Healthcare Conference Wednesday, Ocera did its best to rebuild confidence in its sole clinical candidate.
While the drug failed to show a statistically significant benefit on its main goal this past January, company CEO Linda Grais noted the drug's effect on reducing ammonia levels as a positive signal for moving forward.
"With greater confidence, we believe the most relevant efficacy considerations likely include earlier timing of drug administration, measuring efficacy sooner after drug administration, and administering the appropriate and tolerable dose regimen of OCR-002," Grais said.
According to the company, OCR-002 triggered a dose-proportional reduction in ammonia levels faster than placebo.
Current treatments for HE are inadequate for meeting patient need, Ocera said. Lactulose, an inexpensive laxative used for decades to treat HE, is hard to administer, causes diarrhea and has poor patient compliance, according to Ocera. Valeant's Xifaxan (rifaximin), a non-absorbed antibiotic that kills gut bacteria, is only approved for maintenance, not acute treatment of HE, and doesn’t directly clear circulating ammonia.
Ocera believes OCR-002 has an equal or better risk-benefit ratio than Xifaxan, which is sometimes used in hospitals and acute-care settings despite its narrower indication for reducing risk of HE recurrence.
In the U.S., 30 million to 35 million people have liver disease and 5.5 million are at risk for chronic HE, which would be a sizable commercial opportunity if Ocera can find success with OCR-002. The stock tick-up Wednesday indicated investors haven't given up hope, but Ocera will have its work cut out for it to prove the drug's value.