May 20, 2019, 4:02 p.m. EDT

CymaBay Therapeutics Presents Data in Patients with Primary Biliary Cholangitis at DDW 2019

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NEWARK, May 20, 2019 (GLOBE NEWSWIRE via COMTEX) -- CymaBay Therapeutics, Inc. /zigman2/quotes/201917619/composite CBAY -1.92% , a biopharmaceutical company focused on developing and providing access to innovative therapies for patients with liver and other chronic diseases, today announced results related to its seladelpar clinical development program for the treatment of primary biliary cholangitis (PBC). These data were presented today at Digestive Disease Week (DDW) in San Diego, California. Seladelpar is a potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist currently in development for PBC and nonalcoholic steatohepatitis (NASH).

A poster presentation titled "Capturing the experience and impact of itch in patients with primary biliary cholangitis (PBC)," highlighted results from a qualitative study designed to understand the experience and impact of itch on PBC patients and to examine the validity of three existing pruritus patient-reported outcome (PRO) measures (Itch Numeric Rating Scale (NRS), 5-D Itch Scale, and the Itch Visual Analogue Scale (VAS)). A total of 12 PBC patients with a score greater-than or equal to4 on the Itch NRS (0-10) were interviewed which involved completing the three PRO measures and describing their PBC itch experience. All participants reported receiving medication to manage their PBC. Most participants described their PBC severity as "moderate," "moderate and severe," or "severe." Fifty percent of participants reported experiencing PBC-related itch a few times a day and generally described their itch as feeling like something crawling under the skin, having an involuntary reaction to scratch, or a tingling or creepy sensation that often wasn't relieved with scratching. The most commonly reported impacts from itch were on sleeping, daily activities, and social functioning from scratching in public or having sores on skin. Participants considered the Itch NRS, 5-D Itch, and Itch VAS relevant instruments for assessing itch and found that a 3- to 5-point reduction in the Itch NRS score (>66.7%) and a 5- to 55-point (average -30 points) reduction in the Itch VAS (100%) reflect a meaningful change in treatment effectiveness.

"While 'itch' may sound like a benign disease symptom, these new study results show that it can have significant negative effects on PBC patients," said Cathy Mumford, a PBC patient and member of the Executive Committee of PBCers Organization, an advocacy group offering support and education to PBC patients and their families and raising funds to support PBC research and discover a cure for the disease. "As I know from my own experience and the experiences of the patients we serve, evaluating the potential of new treatment options to improve aspects of patient quality of life, such as itch, are critical to improving the long-term management of PBC."

"Itch (pruritus) has been one of the most common complaints amongst PBC patients, occurring in 20% to 70% of PBC patients," said Dr. Pol Boudes, Chief Medical Officer of CymaBay Therapeutics. "We are honored to collaborate with the PBCers Organization, the Canadian PBC Society, and Evidera and to share data that highlight the experience and relevance of itch from the patient perspective. We recognize that effectively managing PBC-related itch is important for improving quality of life for PBC patients, and are evaluating the effects of seladelpar on this important symptom in ENHANCE, our ongoing global Phase 3 registration study of seladelpar in PBC patients."

Encore Data Presentations

Data previously presented at The International Liver Congress(TM) in April 2019 were also presented in two posters at DDW:

"Seladelpar for the treatment of primary biliary cholangitis: experience with 25 cirrhotic patients"

This poster reported the results of a subset of patients with compensated cirrhosis (Child-Pugh A) from an ongoing Phase 2 study designed to assess the safety and efficacy of seladelpar at a daily dose of 5 mg or 10 mg in PBC patients who had an inadequate response (alkaline phosphatase [AP] greater-than or equal to 1.67 x upper limit of normal [ULN]) or an intolerance to ursodiol and a total bilirubin less-than or equal to 2 mg/dL. Cirrhosis was diagnosed using liver biopsy, liver elastography, or liver imaging. Patients initiated on 5 mg could be dose-escalated to 10 mg after 12 weeks of treatment if it was tolerated and AP threshold criterion was not met (5/10 mg group). The primary outcome was percent change from baseline in AP. Secondary outcome measures included ALT, total bilirubin, and pruritus using the visual analogue scale (VAS). At 52 weeks in patients with cirrhosis, mean relative decreases in AP were -36% and -43% in the 5/10 mg and 10 mg group, respectively. Treatment with seladelpar also demonstrated robust anti-inflammatory activity with a decrease in ALT comparable to what was observed in non-cirrhotic patients. Total bilirubin remained stable throughout 52 weeks. Seladelpar was well tolerated and appeared safe. Three patients with cirrhosis experienced an SAE, all unrelated to seladelpar. Total bilirubin, platelets, albumin, and INR remained stable. No liver decompensation events were observed. There was no transaminase safety signal, and importantly, seladelpar treatment was not associated with drug-induced pruritus or hepatotoxicity.

"Pharmacokinetics, safety, and tolerability of seladelpar in subjects with hepatic impairment"

Data in this poster demonstrated that single dose oral administration of seladelpar was well tolerated and appeared safe in subjects with varying degrees of hepatic impairment (Child-Pugh A-C) and thus provided important information on seladelpar pharmacokinetic exposure and implications for dosing in this challenging population.

CymaBay's presentations from Digestive Disease Week(R) 2019 can be found at: .

About PBC

Primary biliary cholangitis (PBC) is a serious and potentially life-threatening autoimmune disease of the liver characterized by impaired bile flow (cholestasis) and accumulation of toxic bile acids. There is an accompanying inflammation and destruction of the intrahepatic bile ducts, which can progress to fibrosis, cirrhosis and liver failure. Other clinical symptoms of PBC include fatigue and pruritus, which can be quite disabling in some patients. PBC is primarily a disease of women: 1 in 1000 women over the age of 40 lives with PBC.

About Seladelpar

Seladelpar is a potent, selective, orally active PPARδ agonist that is in development for the treatment of the liver diseases PBC and NASH. For PBC, seladelpar has received an orphan designation from the US Food and Drug Administration (FDA) and the European Medicine Agency (EMA). Seladelpar also received Breakthrough Therapy Designation from the FDA and PRIority MEdicine status from the EMA for PBC.


ENHANCE (NCT03602560) is a 52-week, placebo-controlled, randomized, Phase 3 study to evaluate the safety and efficacy of seladelpar. It will be conducted in more than 20 countries over five continents (North America, South America, Europe, Australasia and Asia). Approximately 240 PBC patients will be randomized to seladelpar 10 mg/day, seladelpar 5/10 mg/day (starting treatment at 5 mg with the possibility to escalate dose to 10 mg after 6 months), or placebo. Patients must experience an inadequate response to UDCA (defined as a serum alkaline phosphatase level greater-than or equal to 1.67 x the upper limit of normal after at least 12 months of treatment) or an intolerance to UDCA to be eligible for the study. Patients who are inadequate responders to UDCA will continue their treatment during the study, and UDCA will be provided free of charge. The primary outcome measure is the responder rate after 52 weeks. A responder is defined as a patient who achieves an alkaline phosphatase level < 1.67 x the upper limit of normal with at least a 15% decrease from baseline and has a normal level of total bilirubin. Additional key outcomes of efficacy will compare the rate of normalization of alkaline phosphatase at 52 weeks and the level of pruritus at 6-months assessed by a numerical rating scale recorded with an electronic diary. Additional information can be found at . After completing the study, patients will be offered to continue treatment in an open label extension study. Patients on placebo will be offered to start seladelpar in the extension study.

About CymaBay

US : U.S.: Nasdaq
$ 4.59
-0.09 -1.92%
Volume: 1.43M
Oct. 17, 2019 4:00p
P/E Ratio
Dividend Yield
Market Cap
$321.52 million
Rev. per Employee
1 2
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