CymaBay flashes positive results from the trial they have to relaunch
Two weeks after the FDA lifted its clinical hold on their lead drug, CymaBay said it showed positive results in an aborted Phase III trial.
The drug, a small molecule known as seladelpar, had been in development for three different liver conditions before an independent review of a NASH study last year showed that it might actually be damaging patient’s liver cells. The FDA slapped a clinical hold across all three trials, only lifting it last month when an FDA review determined that the drug hadn’t caused liver damage.
That hold meant Sujal Shah-led CymaBay couldn’t complete an intended 52-week trial in primary biliary cholangitis (PBC), a chronic disorder that slowly destroys a patient’s bile ducts. Still, the biotech said Monday that the 12-week and 26-week data they collected before the trial was halted for the drug were positive. That data covered 167 patients out of the 265 patients the trial was originally scheduled to enroll.
“We couldn’t be happier with what we’ve seen in this data set, despite the fact that this Phase III study was halted early,” Shah told Endpoints News.
After 12 weeks, CymaBay said, 78% on the high dose of seladelpar had achieved the primary endpoint — a composite metric of liver function. That compared with 12.5% on placebo, leading to a p value less than 0.0001.
On a secondary endpoint, 27.3% of patients on high dose seladelpar achieved normal levels of a liver enzyme known as ALP, which is often used as a marker of disease. Zero patients on placebo achieved that endpoint.
Shah argued the results look better than what Intercept saw in its Phase III study for Ocaliva, which was approved for PBC in 2016, although CymaBay did not put the therapies head-to-head in the same trial, making comparisons difficult.
Unfortunately for CymaBay, the clinical hold means the data can’t support an application for approval, as the trial was originally intended to do. Instead, the results simply burnish the company’s decision, announced last month, to restart the study, hoping this time to gather the 52-week data they need to support an NDA. Enrollment for the new trial should begin at the start of next year, Shah said. He added they’ll eventually look to restart trials in primary sclerosing cholangitis as well.
CymaBay’s stock was up 20%, or $0.74, on the news.
Before the NASH implosion, CymaBay was one of a handful of companies that gathered investor interest for its potential to treat the fatty liver condition. Their failure, though, was one of a string to hit the field over the last 18 months.
The company hasn’t left NASH entirely, Shah said, but they have put it on the backburner. He said that although the company is well financed, they’re constrained by a small staff – they went from around 60 employees to 20 after the trial failure – and they believe that a study will ultimately be treated by combination therapies. So they’ll wait until a partner presents itself.
“I think the data we had from our Phase II study, particularly on NASH resolution and fibrosis, is quite compelling,” Shah said.