CymaBay Submits New Drug Application to FDA for Seladelpar for the Treatment of Primary Biliary Cholangitis

- NDA is supported by data evaluating seladelpar efficacy and safety in over 500 patients -

- Seladelpar is the first New Drug Application from CymaBay in its ongoing mission to help people living with PBC -

NEWARK, Calif., Dec. 15, 2023 /PRNewswire/ -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for investigational treatment, seladelpar, for the management of primary biliary cholangitis, including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to ursodeoxycholic acid (UDCA). CymaBay has requested a Priority Review of the NDA that, if accepted, would mean the FDA would plan to complete its review within six months of accepting the application.

Seladelpar has been granted Breakthrough Therapy Designation by the FDA and is the only potent, selective, orally active delpar, or PPARδ agonist, with Phase 3 trial results demonstrating a statistically significant improvement in markers of cholestasis related to risk of progression and PBC-related pruritus. The comprehensive NDA application is supported by robust data evaluating the efficacy and tolerability profile of seladelpar in more than 500 participants with PBC in the placebo-controlled Phase 3 RESPONSE and ENHANCE studies, the long-term open-label ASSURE study, as well as in prior Phase 2 studies.

Breakthrough Therapy Designation is granted by the FDA to investigational agents intended to treat a serious condition and preliminary clinical evidence indicates that it may demonstrate substantial improvement over available therapy on a clinically significant endpoint. FDA updated the Breakthrough Therapy Designation for seladelpar in October 2023 in recognition of data that indicates seladelpar may provide meaningful improvement over existing therapy based on a reduction in alkaline phosphatase (ALP) and improvement in pruritus in patients without cirrhosis or with compensated cirrhosis.

“People living with PBC need new treatment options to reduce the risk of disease progression and the daily impact of their disease from debilitating symptoms like pruritus. Today’s announcement of our submission of the NDA for seladelpar is an important milestone in our ongoing work to bring forward new innovative therapies with the potential to help people living with PBC,” said Klara Dickinson, Chief Regulatory and Compliance Officer, CymaBay Therapeutics. “We would like to extend our thanks to the participants, their families, and all the investigators who have taken part in studies of seladelpar to date, which together have enabled the clinical development and submission of seladelpar for FDA approval. We now look forward to working with the FDA to secure validation of the application and throughout its review.”

CymaBay intends to file marketing authorization applications to the European Medicines Agency (EMA) and U.K. Medicines and Healthcare products Regulatory Agency (MHRA) in the first half of 2024. Seladelpar has received Priority Medicines (PRIME) status from the EMA, as part of the program to optimize development plans and speed up evaluation of priority medicines so they can reach patients earlier.

About PBC
PBC is a rare, chronic inflammatory liver disease primarily affecting women (1 in 1,000 women over the age of 40 or about 130,000 total people in the U.S.). 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, alanine aminotransferase (ALT), and gamma-glutamyl transferase (GGT), enzymes found primarily in the liver, as well as total bilirubin. The most common early symptoms of PBC are pruritus (itching) and fatigue, which can be debilitating for some patients. Progression of PBC is associated with an increased risk of liver-related mortality.

About Seladelpar
Seladelpar, an investigational treatment for people with PBC, is a first-in-class oral, selective peroxisome proliferator-activated receptor delta (PPARδ) 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 (FDA), Priority Medicines status (EMA), 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 potential FDA acceptance of the seladelpar NDA, its subsequent review and the timing thereof, the potential for seladelpar to treat PBC and potentially improve ALP levels, clinical symptoms or outcomes of the disease, the future EMA and MHRA filing plans of CymaBay and the timing thereof 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:

Theresa Dolge
Evoke Kyne
(609) 915-2156
Theresa.Dolge@evokegroup.com

Investor Relations Contact:

PJ Kelleher
LifeSci Advisors, LLC
(617) 430-7579
pkelleher@LifeSciAdvisors.com

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SOURCE CymaBay Therapeutics