Small cap CymaBay Therapeutics has taken a look at its ongoing midstage test for its liver med seladelpar, and says it’s happy with what it sees.
The interim results from its ongoing low-dose phase 2 study of seladelpar in patients with primary biliary cholangitis (PBC), a rare form of liver disease, were positive, according to the biotech’s statement.
In the first part of the study, patients at high-risk of disease progression, who don’t respond well to standard PBC treatment ursodeoxycholic acid (UDCA), were given 5mg or 10mg of seladelpar once a day.
A planned mid-way look at the 24 patients in the two dose groups showed that, after 12 weeks of treatment, a significant alkaline phosphatase (AP) reduction from baseline of 39% and 45% for the two groups, respectively.
“On seladelpar, 45% of patients in the 5mg and 82% of patients in the 10mg dose groups had AP values less than 1.67 times the upper limit of normal (ULN). AP is an established surrogate marker of disease progression in PBC, and reaching a level of less than 1.67 x ULN is a key component in the composite endpoint used for regulatory approval,” the biotech said.
Alongside lowering of AP, patients in both dose groups also saw decreases in other liver markers of cholestasis, and also improved metabolic and inflammatory markers with patients experiencing decreases in low-density lipoprotein-C and high sensitivity C-reactive protein.
CymaBay did not, however, give a detailed breakdown of the data for these in its release. It added that it hadn’t seen any serious safety issues either, but again did not given full details.
But the company said that, after showing these figures to the FDA, the agency says seladelpar treatment can now be given beyond six months.
Pol Boudes, CMO of CymaBay, said: “These interim results support the potent anti-cholestatic and anti-inflammatory effects of seladelpar. We are particularly excited about the FDA’s decision to allow dosing of seladelpar beyond six months enabling us to turn our attention towards planning the phase 3 program.”
Last year, Intercept Pharmaceuticals got European and US nods for its PBC drug Ocaliva (obeticholic acid)
Intercept’s drug works as a semisynthetic bile acid and activates the farnesoid X receptor (FXR), which controls the production of bile. By activating this receptor, Ocaliva is expected to reduce the production of bile in the liver, thus reducing the exposure of the liver to toxic levels of bile acids.
This is a fairly narrow indication in terms of patient population and Intercept, along with Shire, Gilead, BMS, Genfit and a host other biopharmas are all hoping to gain the first approval for a broader liver disease label, namely the treatment of NASH, or fatty liver disease.
CymaBay was up 12% in early premarket trading on the news this morning.