CymaBay Initiates IDEAL, a Phase 3 Placebo-Controlled, Randomized Study of Seladelpar in Patients with Primary Biliary Cholangitis and Incomplete Control of Alkaline Phosphatase

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Aug 10, 2023, 08:00 ET

Evaluates seladelpar 10 mg vs. placebo for alkaline phosphatase normalization at 52 weeks

Patients enroll with alkaline phosphatase levels between 1- and 1.67-times upper limit of normal

NEWARK, Calif., Aug. 10, 2023 /PRNewswire/ -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on developing and providing access to innovative therapies for patients with liver and other chronic diseases, today announced the initiation of its 52-week, placebo-controlled, randomized, Phase 3 study — "Intended to Determine the Effects of seladelpar on normalization of Alkaline phosphatase (ALP) Levels in subjects with Primary Biliary Cholangitis (PBC)" (IDEAL).

The IDEAL study aims to enroll 75 patients with PBC who have an incomplete response or intolerance to ursodeoxycholic acid (UDCA), in each case with ALP greater than the upper limit of normal (ULN) but less than 1.67xULN, and total bilirubin less than or equal to 2xULN. Patients will be randomly assigned using a 2:1 ratio to oral, once daily seladelpar 10 mg or placebo. The primary outcome measure is the normalization of ALP at 52 weeks.

Additional key outcomes evaluating efficacy include the percent change from baseline in ALP at 52 weeks and the level of self-reported pruritus (itch) at 6 months for patients with moderate to severe symptoms at baseline, assessed by a numerical rating scale (NRS) and recorded by electronic diary.

Charles McWherter, Ph.D., Chief Scientific Officer and President of Research and Development at CymaBay, stated, "CymaBay has a long-standing commitment to advancing patient care. We see the IDEAL study as an opportunity to evaluate the potential benefits of seladelpar in a population that has a persistent elevation of ALP, but whose ALP levels are not above the 1.67 times upper-limit-of-normal recommended by clinical guidelines or that are used for entering clinical trials of new second-line agents. We remain fully committed to providing all patients the opportunity to have their diseases and unmet needs addressed, and the IDEAL study is emblematic of this endeavor."

"Over the last few years there has been increasing interest in the medical community to understand how normalization of biochemical markers relates to risk of disease progression," said Dr. Cynthia Levy, MD, Professor of Medicine, University of Miami. "The IDEAL study is an important step forward in assessing the potential of a therapeutic intervention in normalizing biochemical markers of PBC in patients with incomplete control of ALP levels. Our hope is that the findings from this research will help advance scientific understanding of the role of ALP normalization in PBC, particularly when it comes to addressing some of the most distressing symptoms for patients."

About PBC
PBC is a rare, chronic inflammatory liver disease primarily affecting women (1 in 1,000) over the age of 40. PBC 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 causing increased levels of ALP and total bilirubin. The most common early symptoms of PBC are pruritis (itching) and fatigue, which can be debilitating for some patients. Progression of PBC is associated with an increased risk of liver cancer and liver-related mortality.

About Seladelpar
Seladelpar is a first-in-class oral, selective peroxisome proliferator-activated receptor (PPAR) delta agonist, or delpar, shown to regulate critical metabolic and liver disease pathways in indications with high unmet medical need. Preclinical and clinical data support its ability to regulate genes involved in bile acid synthesis, inflammation, fibrosis and lipid metabolism, storage, and transport.

About CymaBay
CymaBay Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on improving the lives of people with liver and other chronic diseases that have high unmet medical need through a pipeline of innovative therapies. Our deep understanding of the underlying mechanisms of liver inflammation and fibrosis, and the unique targets that play a role in their progression, have helped us receive breakthrough therapy designation (U.S. Food and Drug Administration), Priority Medicines status (European Medicines Agency) and orphan drug status (U.S. and Europe) for seladelpar, a first-in-class investigational treatment for people with PBC. Our evidence-based decision-making and commitment to the highest quality standards reflect our relentless dedication to the people, families, and communities we serve. To learn more, visit www.cymabay.com and follow us on X (formerly Twitter) and LinkedIn.

Cautionary Statements
Any statements made in this press release regarding the potential for seladelpar to treat PBC and potentially improve clinical symptoms of the disease, the potential benefits to patients and the ability of CymaBay to enroll and complete the IDEAL clinical trial are forward-looking statements that are subject to risks and uncertainties. Actual results and the timing of events regarding the further development of seladelpar could differ materially from those anticipated in such forward-looking statements as a result of risks and uncertainties, which include, without limitation, risks related to: the success, cost and timing of any of CymaBay's product development activities, including clinical trials; and effects observed in trials to date that may not be repeated in the future. Additional risks relating to CymaBay are contained in CymaBay's filings with the Securities and Exchange Commission, including without limitation its most recent Annual Report on Form 10-K, its Quarterly Reports on Form 10-Q and other documents subsequently filed with or furnished to the Securities and Exchange Commission. CymaBay disclaims any obligation to update these forward-looking statements except as required by law.

For additional information about CymaBay visit www.cymabay.com.

Public Relations Contact:

Lindsay Barnes
Evoke Kyne
(215) 410-9982
[email protected]

Investor Relations Contact:

Hans Vitzthum
LifeSci Advisors, LLC
(617) 430-7578
[email protected]

SOURCE CymaBay Therapeutics