CymaBay (CBAY) Stock Up on Positive Results From PBC Study
Shares of clinical-stage biopharmaceutical company CymaBay Therapeutics, Inc. CBAY gained after it announced positive top-line results from its late-stage RESPONSE study.
This phase III study evaluated the safety and efficacy of seladelpar, a potent, selective, orally active delpar, in development for the treatment of adult patients with primary biliary cholangitis (PBC).
The study randomized 193 PBC patients in a 2:1 ratio to once-daily seladelpar 10 mg or placebo.
A total of 61.7% of patients on seladelpar 10 mg met the primary composite endpoint related to serum alkaline phosphatase and bilirubin at 12 months versus 20.0% on placebo. The anti-cholestatic effect of seladelpar was supported by the normalization of alkaline phosphatase (ALP) at 12 months (key secondary endpoint) in 25.0% of patients on seladelpar versus zero on placebo.
A rare, chronic inflammatory liver disease, PBC, primarily affects women (1 in 1,000 women over the age of 40). It is characterized by impaired bile flow (known as cholestasis) and the accumulation of toxic bile acids in the liver, leading to inflammation and destruction of the bile ducts within the liver and increased levels of ALP and total bilirubin.
Seladelpar treatment compared with placebo also demonstrated a statistically significant reduction in pruritus or itch (a key secondary endpoint) after 6 months of treatment.
The registration trial achieved the primary and all key secondary endpoints and supports advancement to regulatory discussions and filing for regulatory approval with the FDA and other health authorities.
The safety profile was comparable between the placebo and seladelpar groups and was consistent with previous studies.
Shares of the company have gained 173.2% in the year so far against the industry’s decline of 30%.
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Seladelpar has been granted Breakthrough Therapy Designation and Orphan Drug Status by the FDA. The EMA has also granted Priority Medicines status and orphan drug status to the candidate.
CymaBay has also initiated a 52-week placebo-controlled, randomized, phase III study intended to determine the effects of seladelpar on the normalization of ALP levels in subjects with PBC.
The successful development and commercialization of seladelpar will be a significant boost for CymaBay.
We note that shares of CymaBay soared in late June after Ipsen and GENFIT announced topline data on elafibranor from the phase III ELATIVE study for PBC. While the trial met its primary composite endpoint, with 51% of patients on elafibranor 80 mg achieving a cholestasis response compared with 4% on placebo, seladelpar had reported better efficacy than elafibranor.
Assuming a tentative approval, seladelpar is likely to face competition from Intercept Pharmaceuticals’ ICPT lead drug, Ocaliva (obeticholic acid or OCA). The drug is approved for PBC and continues to witness strong growth. Intercept is also evaluating a combination of OCA and bezafibrate for the treatment of PBC.
Zacks Rank & Stocks to Consider
Currently, CBAY has a Zacks Rank #3 (Hold).
A couple of better-ranked stocks in the overall healthcare sector are Dynavax Technologies DVAX and Exelixis EXEL, each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Loss estimates for Dynavax for 2023 have narrowed to 24 cents from 56 cents in the past 60 days, while the earnings estimate for 2024 is currently pegged at 2 cents per share.
Shares of EXEL have gained 38% year to date. Earnings estimates for 2023 have risen by 9 cents to 98 cents.
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