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CymaBay Announces FDA Acceptance of NDA and Priority Review for Seladelpar for the Treatment of Primary Biliary Cholangitis

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CymaBay Therapeutics, Inc. (CBAY) announced that the FDA accepted its NDA for seladelpar, a treatment for PBC, with a target PDUFA date of August 14. Seladelpar has shown significant improvements in PBC-related markers and pruritus in Phase 3 studies.
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The acceptance of the New Drug Application (NDA) for seladelpar by the FDA signifies a critical milestone for CymaBay Therapeutics in the drug development process. The granting of Priority Review status accelerates the review timeline and implies a recognition of the drug's potential to address an unmet medical need. The target action date set by the Prescription Drug User Fee Act (PDUFA) provides a clear timeline for investors and the company, with a decision expected by August 14, 2024.

From a medical research perspective, the significance of seladelpar lies in its unique mechanism as a PPARδ agonist, which has shown promise in the management of primary biliary cholangitis (PBC), a condition with limited treatment options. The Phase 3 RESPONSE study's demonstration of statistically significant improvement in biochemical markers and pruritus measures is noteworthy, potentially positioning seladelpar as a second-line treatment for PBC patients who do not respond to or tolerate the current standard of care, ursodeoxycholic acid.

The financial implications of the FDA's decision to grant priority review for seladelpar are substantial for CymaBay Therapeutics. The market for PBC treatments is niche due to the rarity of the condition, but the lack of effective treatments for pruritus associated with PBC creates a potential market opportunity for CymaBay. The drug's approval could lead to a new revenue stream, enhancing the company's valuation and attractiveness to investors.

However, the financial success of seladelpar will depend on several factors, including the pricing strategy, market penetration and competitive landscape. The company's ability to effectively commercialize the drug post-approval will be critical. Investors should also consider the costs associated with the ongoing review process and potential marketing expenses, which could impact the company's short-term financials.

Understanding the market dynamics for PBC treatments is essential for assessing the potential impact of seladelpar's approval. The Breakthrough Therapy Designation previously granted to seladelpar and the lack of an advisory committee meeting suggest confidence in the drug's efficacy and safety profile. This could position CymaBay favorably in the eyes of healthcare providers and payers, potentially facilitating market access and adoption.

Market research would focus on the size of the target patient population, competitive products and the anticipated adoption curve for seladelpar. It would also analyze the potential for label expansion and the long-term market potential, considering the chronic nature of PBC and the ongoing need for effective management of the disease and its symptoms.

FDA has granted Priority Review with a target PDUFA date of August 14 -

NEWARK, Calif., Feb. 12, 2024 /PRNewswire/ -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for seladelpar, an investigational treatment for the management of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to ursodeoxycholic acid. The FDA has granted priority review and set a Prescription Drug User Fee Act (PDUFA) target action date of August 14, 2024 and notified the company that it is not currently planning to hold an advisory committee meeting to discuss the application.

PBC is a rare, chronic condition that can lead to inflammation and eventually liver cirrhosis. Disease progression is associated with an increase in liver-related complications, including mortality. People living with PBC can also experience symptoms that significantly impact their quality of life such as pruritus (itch) and fatigue. New treatment options seek to help prevent further disease progression by reducing inflammation and bile acids in the liver and help provide meaningful relief of pruritus.

Seladelpar is an investigational, potent, selective, orally active PPARδ agonist, or delpar, in development for PBC treatment. It is the only investigational agent to have demonstrated a statistically significant improvement in biochemical markers of disease progression and pre-specified measures of PBC-related pruritus in a Phase 3 study. The safety profile of seladelpar in the pivotal Phase 3 RESPONSE study, measured through rates of adverse events, was similar in those treated with seladelpar and placebo.

The NDA includes data from across the seladelpar PBC clinical development program in over 500 participants living with PBC, including from the placebo-controlled Phase 3 RESPONSE and ENHANCE studies, the long-term open-label ASSURE study, and prior Phase 2 studies. The FDA has previously granted seladelpar Breakthrough Therapy Designation (BTD) which enabled early submission of select non-clinical data through the rolling review process afforded by seladelpar's BTD status. FDA updated the Breakthrough Therapy Designation for seladelpar in October 2023 in recognition of clinical data that indicated 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.

"Acceptance of the seladelpar NDA for the treatment of PBC, is an important step forward in our ongoing mission to make this potential, novel, targeted treatment option available for people living with PBC," said Klara Dickinson, Chief Regulatory and Compliance Officer, CymaBay Therapeutics. "We are encouraged by the agency's decision to grant priority review for seladelpar, and its recognition of the significant need for new treatment options for people living with PBC. If approved, we believe seladelpar has the potential to raise the bar in second-line treatment of PBC by reducing markers of disease progression and improving pruritus, so we look forward to continued discussion with the agency throughout its review."

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 US). 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, ALT, and 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. 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 timing of potential FDA action on the seladelpar NDA, any potential approval or indication statement 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 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

Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/cymabay-announces-fda-acceptance-of-nda-and-priority-review-for-seladelpar-for-the-treatment-of-primary-biliary-cholangitis-302059522.html

SOURCE CymaBay Therapeutics

The target PDUFA date for seladelpar is August 14, 2024.

Seladelpar is a potent, selective, orally active PPARδ agonist, in development for PBC treatment.

The FDA granting priority review indicates the significant need for new treatment options for people living with PBC.

Clinical data showing a reduction in alkaline phosphatase (ALP) and improvement in pruritus in patients without cirrhosis or with compensated cirrhosis led to the FDA updating the Breakthrough Therapy Designation for seladelpar.

Klara Dickinson is the Chief Regulatory and Compliance Officer of CymaBay Therapeutics.
Cymabay Therapeutics Inc

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About CBAY

cymabay therapeutics is a clinical-stage biopharmaceutical company located in the san francisco bay area focused on developing therapies to treat metabolic diseases with high unmet medical need or serious rare and orphan diseases. we are committed to developing breakthrough medicines that improve the lives of patients and their families. cymabay was seeded with the assets from an earlier metabolic disease company in which more than $120m was invested to produce a robust pipeline.