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ESPR Aims to Diversify With Rare Liver Disease Candidate, Stock Up

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Key Takeaways

  • Esperion named ESP-2001 its new preclinical candidate for primary sclerosing cholangitis.
  • ESP-2001 was discovered with Evotec and has shown liver injury reduction in preclinical studies.
  • Esperion will hold exclusive global rights to develop and commercialize ESP-2001.

Esperion Therapeutics (ESPR - Free Report) announced that it has nominated ESP-2001, a highly specific allosteric ATP citrate lyase (“ACLY”) inhibitor, as its new preclinical development candidate for the treatment of primary sclerosing cholangitis (“PSC”).

Currently, there are no approved treatments for PSC, a rare and debilitating autoimmune liver disease.

The company plans to initiate investigational new drug (“IND”)-enabling studies for ESP-2001 and submit an IND application to the FDA, with the goal of starting clinical studies in 2026.

Shares of Esperion were up 5.2% yesterday following the announcement of the news.

Notably, ESP-2001 was discovered through the collaboration between Esperion and Evotec (EVO - Free Report) . The collaboration leveraged ESPR’s deep expertise in ACLY therapy with Evotec’s integrated drug discovery platform.

The nomination of ESP-2001 as a preclinical candidate for PSC triggered an undisclosed payment for Evotec.

Per management, ESP-2001 has consistently reduced markers of liver and bile duct injury, inflammation and fibrosis in various preclinical studies, underlining its potential to meaningfully impact the progression of PSC.

ESP-2001 could qualify for Orphan Drug and Fast Track designations from the FDA, as well as PRIME designation from the European Medicines Agency.

ESPR's Price Performance

Esperion stock has risen 20.5% so far this year compared with the industry’s growth of 8.3%.

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ESPR Aims to Diversify Beyond Cardiovascular Diseases

Esperion has two FDA-approved drugs in its commercial portfolio, Nexletol (bempedoic acid) and Nexlizet, which are approved for treating elevated LDL-C (bad cholesterol) and for cardiovascular risk reduction. Nexlizet is a combination of bempedoic acid and ezetimibe.

These two oral drugs are marketed as Nilemdo and Nustendi in ex-U.S. markets (excluding Japan, where the company has a collaboration with Otsuka Pharmaceuticals) in partnership with Daiichi Sankyo. The company records royalties on sales of its drugs in ex-U.S. markets.

Esperion records revenues from product sales and collaboration revenues, which include royalties on sales of Nilemdo/Nustendi by collaboration partners.

Net product sales of Nexletol and Nexlizet in the United States grew 42% in the first half of 2025 to $75.2 million, reflecting increased prescription growth volumes.

If successfully developed, ESP-2001 can help Esperion expand and diversify beyond cardiovascular diseases into the lucrative liver disorder space. The company retains exclusive global development and commercialization rights to ESP-2001, targeting a potential blockbuster market opportunity of over $1 billion annually.

ESPR's Zacks Rank & Stocks to Consider

Esperion currently carries a Zacks Rank #3 (Hold).

Some top-ranked stocks in the biotech sector are Chemomab Therapeutics (CMMB - Free Report) and ANI Pharmaceuticals (ANIP - Free Report) , each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 60 days, estimates for Chemomab Therapeutics’ 2025 loss per share have narrowed from $2.40 to 60 cents. Loss per share estimates for 2026 have narrowed from $2.80 to $1.00 during the same period. CMMB stock has plunged 55.1% year to date.

Chemomab Therapeutics’ earnings beat estimates in three of the trailing four quarters, while meeting the same on the remaining occasion, with an average surprise of 26.25%.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2025 earnings per share have risen from $7.25 to $7.29. During the same period, earnings per share for 2026 have increased from $7.74 to $7.79. Year to date, ANIP’s shares have rallied 66.5%.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with an average surprise of 22.66%.

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