Galmed Pharmaceuticals Reports Positive Preclinical Results for Aramchol in Treating Primary Sclerosing Cholangitis

Galmed Pharmaceuticals announced that Aramchol, an SCD1 inhibitor, significantly attenuates and prevents biliary fibrosis in mouse models of primary sclerosing cholangitis (PSC). Aramchol treatment leads to significant inhibition of TGFβ-induced hepatic fibrosis pathways and upregulates peroxisome proliferator activated receptor (PPAR) signaling. This provides a rationale for assessing Aramchol in further clinical studies in patients with fibrosis-driven liver cancers.

Galmed Pharmaceuticals (NASDAQ: GLMD) has announced significant results for Aramchol, their SCD1 inhibitor drug, in pre-clinical models of primary sclerosing cholangitis (PSC). The study demonstrated that Aramchol both prevented and reduced biliary fibrosis in PSC mouse models, with a 2-fold significant inhibition (p0.05) of TGFβ-induced hepatic fibrosis pathways. The drug showed dose-dependent reduction in fibrotic markers and hepatic stellate cell-activating genes in human and mouse cells [1].

These findings are particularly significant given the connection between PSC and cholangiocarcinoma (CCA), the second-most prevalent liver malignancy. PSC carries a 20% lifetime risk of developing CCA, which has an exceptionally poor prognosis with most patients surviving less than a year. The relationship between fibrosis and cancer is well-established, with 80-90% of hepatocellular carcinomas developing in fibrotic or cirrhotic livers [1].

Aramchol has already demonstrated significant improvement in liver fibrosis in previous human studies (ARREST Phase IIb trial and ARMOR Phase III extension), with an established safety profile. This creates a clear translational path toward testing in fibrosis-driven liver cancers. The company plans to advance Aramchol towards Phase 2/3 clinical studies focusing on GI oncology indications [1].

The research holds substantial clinical relevance, and Galmed's pre-clinical findings on Aramchol represent a significant scientific advancement in understanding the drug's mechanism as an SCD1 inhibitor. The data shows Aramchol both attenuates and prevents biliary fibrosis in PSC mouse models with dose-dependent reductions in fibrotic markers and hepatic stellate cell-activating genes. The 2-fold inhibition (p0.05) of TGFβ-induced hepatic fibrosis pathways while upregulating PPAR signaling is particularly noteworthy [2].

This development potentially repositions Aramchol from primarily a NASH treatment toward broader applications in PSC and GI oncological indications like cholangiocarcinoma and hepatocellular carcinoma. The company's stated intention to transition to Phase 2/3 studies in GI oncology represents a significant strategic expansion of their clinical development program, addressing conditions with substantial unmet medical needs [1, 2].

References:
[1] https://www.stocktitan.net/news/GLMD/galmed-reports-significant-anti-fibrotic-effects-of-aramchol-in-psc-8ul7j1w0cx4x.html
[2] https://www.prnewswire.com/news-releases/galmed-reports-significant-anti-fibrotic-effects-of-aramchol-in-psc-models-302453710.html