Esperion unveils novel PSC treatment candidates

ANN ARBOR, Mich. - Esperion Therapeutics (NASDAQ:ESPR) announced today its latest research on development candidates targeting primary sclerosing cholangitis (PSC), a rare liver disease. The announcement was made ahead of an R&D Day event in New York City, where the company will present its findings and host a live webcast at 9:00 a.m. ET.

The company’s research focuses on allosteric inhibitors of ATP citrate lyase (ACLY), an enzyme implicated in liver inflammation and fibrosis. Esperion’s CEO, Sheldon Koenig, emphasized the potential of these inhibitors, particularly ESP-1336, as first-in-class candidates for addressing PSC, which currently lacks FDA-approved therapies for its progression.

According to David E. Cohen, MD, PhD, from Brigham and Women’s Hospital, these inhibitors may offer a new treatment approach by targeting acetyl-CoA levels and disrupting disease-driving pathways in PSC.

Esperion’s R&D Day will also feature guest speakers from Harvard Medical School and PSC Partners, highlighting the science underpinning the company’s ACLY inhibition discovery program. The program aims to develop more potent and selective inhibitors for PSC, informed by multi-omics studies and preclinical disease modeling.

Preclinical models have shown the candidates to reduce liver injury, inflammation, and fibrosis. With an estimated market opportunity exceeding $1 billion annually and a patient population of around 76,000 in the U.S. and Europe as of 2024, Esperion is pursuing Orphan Drug and Fast Track designations from the FDA. Despite maintaining a healthy gross profit margin of 65.44% and a current ratio of 1.37, InvestingPro analysis indicates the company faces near-term profitability challenges. InvestingPro subscribers have access to 8 additional key insights about Esperion’s financial health and market position.

PSC is characterized by inflammation and scarring of bile ducts, leading to cirrhosis and liver failure. Treatment options are limited, with liver transplant being the only long-term solution, though recurrence can occur in up to 30% of cases.

Esperion, known for its FDA-approved non-statin LDL-C lowering medications, is expanding its portfolio to address unmet needs in liver and kidney diseases through its next-generation ACLY inhibitor program. With the stock currently appearing undervalued according to InvestingPro Fair Value calculations, investors seeking detailed analysis can access comprehensive Pro Research Reports covering Esperion and 1,400+ other top stocks, providing actionable intelligence for informed investment decisions.

The press release did not provide specific details on the timeline for clinical trials or commercial availability of the new PSC treatment candidates. The information in this article is based on a press release statement from Esperion Therapeutics.

In other recent news, Esperion Therapeutics announced the resignation of Eric Warren as Chief Commercial Officer, effective April 18, 2025. This marks a significant change in the company’s executive team, although Esperion clarified that Warren’s departure is not due to any disagreements with company operations. Additionally, Esperion has appointed Robert E. Hoffman as an independent director and future Chairperson of the Audit Committee, with his tenure starting on May 29, 2025. Meanwhile, JMP Securities reaffirmed a Market Outperform rating for Esperion, setting a price target of $4.00, highlighting the potential benefits of label expansion for bempedoic acid. The company is set to begin Phase 3 clinical trials for its cholesterol-lowering drug, bempedoic acid, targeting pediatric patients with familial hypercholesterolemia. These trials are expected to start within the current year, following the FDA’s approval of Esperion’s regulatory path. The trials will assess the efficacy and safety of the drug over a one-year period, potentially extending patent protection until June 2031. Investors are closely monitoring these developments, as they could significantly impact Esperion’s market position and financial outlook.

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