Esperion Nominates ESP-2001 as Preclinical Development Candidate for Treatment of Primary Sclerosing Cholangitis

Rhea-AI Summary

Esperion (NASDAQ: ESPR) nominated ESP-2001, a highly-specific allosteric ACLY inhibitor, as its preclinical development candidate for primary sclerosing cholangitis (PSC) on October 16, 2025. The company will begin IND-enabling studies with a goal to file an IND with the FDA in 2026 to start first-in-human trials. Preclinical models reportedly showed consistent reductions in markers of liver and bile duct injury, inflammation, and fibrosis. Esperion retains exclusive global development and commercialization rights and cites an estimated diagnosed PSC prevalence of ~76,000 in the U.S. and Europe and a potential market opportunity of over $1 billion annually. ESP-2001 may be eligible for Orphan Drug, Fast Track, and PRIME designations.

Positive

• IND filing goal set for 2026
• Preclinical models showed reduced liver and bile duct injury markers
• Company retains exclusive global rights to ESP-2001
• Estimated diagnosed PSC population of ~76,000 (U.S. and Europe)
• Company cites a potential market of over $1 billion annually

Negative

• Preclinical stage only; no human data yet
• Regulatory designations listed as potential, not awarded

– Highly-Specific Allosteric ACLY Inhibitor Designed to Target Liver and Bile Duct Injury, Inflammation, and Fibrosis Associated with Primary Sclerosing Cholangitis (PSC) and Related Diseases –

– ESP-2001 Has the Potential to Meaningfully Impact the Progression of PSC, a Disease with No Approved Treatments –

ANN ARBOR, Mich., Oct. 16, 2025 (GLOBE NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced the nomination of ESP-2001, the Company’s highly-specific allosteric ATP citrate lyase (ACLY) inhibitor, as preclinical development candidate for the treatment of primary sclerosing cholangitis (PSC). With this candidate selection, the Company will begin Investigational New Drug (IND)-enabling studies with a goal to file an IND with the U.S. Food and Drug Administration (FDA) to initiate first-in-human clinical studies in 2026.

“The nomination of ESP-2001 marks a pivotal moment in Esperion’s evolution as we expand our therapeutic focus beyond cardiovascular disease,” said Sheldon Koenig, President and Chief Executive Officer of Esperion. “PSC is a devastating condition with no approved treatments, and our preclinical data suggest ESP-2001 has the potential to meaningfully impact disease progression through multiple mechanisms. We are proud to advance a candidate that reflects the capabilities of our next generation ACLY inhibitor program and our commitment to addressing areas of high unmet need.”

With the nomination of ESP-2001, Esperion builds on its leadership in the discovery and development of ACLY therapies for multiple life-threatening diseases. ESP-2001 was discovered leveraging Esperion’s deep expertise in ACLY therapy and utilizing Evotec’s integrated drug discovery platform. Following its identification through integration of high-throughput, virtual, and structure-based screening approaches, ESP-2001 was optimized for novel ACLY-dependent activities associated with PSC pathogenesis. In multiple preclinical models, ESP-2001 consistently reduced markers of liver and bile duct injury, inflammation, and fibrosis.

Esperion is continuing its partnership with Evotec to leverage their INDiGO platform, an integrated, clinical-enabling solution designed to derisk and accelerate the development of small molecule drug candidates from candidate nomination to IND submission.

PSC is a rare, progressive, debilitating autoimmune liver disease characterized by chronic inflammation and scarring of the bile ducts, which can lead to cirrhosis, liver failure, transplant, and death. PSC also increases the risk of various carcinomas and is frequently associated with inflammatory bowel disease, particularly ulcerative colitis. PSC remains an area of great unmet medical need, with no FDA-approved therapies.

With an estimated prevalence of approximately 76,000 diagnosed PSC patients across the U.S. and Europe, and with no approved treatment options, ESP-2001 – a wholly owned asset for which Esperion retains exclusive global development and commercialization rights – represents a potential blockbuster market opportunity of over $1 billion annually. In addition, ESP-2001 has potential eligibility for Orphan Drug and Fast Track designations from the U.S. FDA and PRIME designation from the European Medicines Agency.

About Primary Sclerosing Cholangitis
Primary Sclerosing Cholangitis (PSC) is a rare, progressive, cholestatic, immune‐mediated disease of the bile ducts. Bile ducts carry the digestive liquid bile from the liver to the small intestine. In PSC, inflammation and injury cause scarring, structuring, and concentric, obliterative fibrosis within the bile ducts. These processes make the bile ducts hard and narrow leading to biliary cirrhosis, portal hypertension, and eventually hepatic failure in a majority of patients.

PSC also increases the risk of carcinomas and is frequently associated with inflammatory bowel disease, particularly ulcerative colitis. The cause of PSC remains unknown and there is no proven medical or interventional therapy to halt the progression of disease. A liver transplant is the only known long-term treatment option for PSC, however, up to 30% of patients who receive a liver transplant for PSC experience recurrence.

About Esperion Therapeutics
Esperion Therapeutics, Inc. is a commercial stage biopharmaceutical company focused on bringing new medicines to market that address unmet needs of patients and healthcare professionals. The Company developed and is commercializing the only U.S. Food and Drug Administration (FDA) approved oral, once-daily, non-statin medicines for patients who are at risk for cardiovascular disease and are struggling with elevated low density lipoprotein cholesterol (LDL-C). These medications are supported by the nearly 14,000 patient CLEAR Cardiovascular Outcomes Trial. Esperion continues to build on its success with its next generation program which is focused on developing ATP citrate lyase inhibitors (ACLYi). New insights into the structure and function of ACLYi fully enables rational drug design and the opportunity to develop highly potent and specific inhibitors with allosteric mechanisms.

Esperion continues to evolve into a leading global biopharmaceutical company through commercial execution, international partnerships and collaborations and advancement of its pre-clinical pipeline. For more information, visit esperion.com and follow Esperion on LinkedIn and X.

Forward-Looking Statements
This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the federal securities laws, including statements regarding marketing strategy and commercialization plans, current and planned operational expenses, expected profitability, future operations, commercial products, clinical development, including the timing, designs and plans for the CLEAR Outcomes study and its results, plans for potential future product candidates, financial condition and outlook, including expected cash runway and profitability, and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “suggest,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions. Any express or implied statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements involve risks and uncertainties that could cause Esperion’s actual results to differ significantly from those projected, including, without limitation, the net sales, profitability, and growth of Esperion’s commercial products, clinical activities and results, supply chain, commercial development and launch plans, the outcomes and anticipated benefits of legal proceedings and settlements, and the risks detailed in Esperion’s filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Esperion disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

Esperion Contact Information:
Investors:
Alina Venezia
investorrelations@esperion.com
(734) 887-3903

Media:
Tiffany Aldrich
corporateteam@esperion.com
(616) 443-8438

FAQ

What did Esperion (ESPR) announce about ESP-2001 on October 16, 2025?

Esperion nominated ESP-2001 as its preclinical candidate for PSC and will begin IND-enabling studies aiming to file an IND in 2026.

What stage of development is ESP-2001 (ESPR) currently in?

ESP-2001 is at the preclinical stage with IND-enabling studies planned before a targeted 2026 IND filing.

How large is the PSC patient population cited by Esperion for ESP-2001 (ESPR)?

Esperion cites an estimated diagnosed PSC population of approximately 76,000 across the U.S. and Europe.

What commercial opportunity does Esperion (ESPR) attribute to ESP-2001?

The company states ESP-2001 represents a potential market opportunity of over $1 billion annually.

Does Esperion (ESPR) own global rights to ESP-2001?

Yes; Esperion retains exclusive global development and commercialization rights to ESP-2001.

What efficacy signals did preclinical studies show for ESP-2001 (ESPR)?

In multiple preclinical models, ESP-2001 reportedly reduced markers of liver and bile duct injury, inflammation, and fibrosis.