Epirium Bio Announces First Participants Dosed in Phase 1 Clinical Trial in Healthy Volunteers, Evaluating MF-300, a First-In-Class, Oral 15-PGDH Enzyme Inhibitor, for the Treatment of Sarcopenia

Company expects to share preliminary Phase 1 clinical data in the second half of 2025

SAN DIEGO--(BUSINESS WIRE)--Epirium Bio, Inc. (Epirium), a biopharmaceutical company advancing medicines for neuromuscular and fibrotic diseases, today announced that it has initiated dosing of healthy volunteers in its Phase 1 dose-escalation clinical trial of MF-300, an investigational, first-in-class, orally administered,15-hydroxyprostaglandin dehydrogenase (15-PGDH) enzyme inhibitor in development for the treatment of sarcopenia, or age-induced muscle weakness.

It is estimated that up to a third of Americans over the age of 60 are affected by sarcopenia, a disease that increases the risk of falls, fractures, disability and all-cause mortality. Despite sarcopenia’s widespread prevalence and serious health implications, there are currently no FDA-approved therapies available to treat sarcopenia, highlighting the significant unmet medical need for this disease.

“Dosing our first participants with MF-300, is a significant achievement and marks an important milestone for Epirium in our successful transition to a clinical-stage company developing an oral therapy for the over 20 million elderly Americans estimated to suffer from sarcopenia,” said Alex Casdin, Chief Executive Officer of Epirium. “I’m deeply grateful to our team and the study’s participants. The results of this Phase 1 trial will provide Epirium with important insights into the safety and pharmacological profile of MF-300, as well as valuable target engagement data. These results have the potential to represent a key step forward in advancing MF-300 as a first-in-class oral therapy for patients with sarcopenia.”

About the MF-300 Phase 1 Clinical Trial

The Phase 1 clinical trial is a two-part, randomized, double-blind, placebo controlled, dose-escalation study designed to assess the safety, pharmacokinetics, and pharmacodynamics of single (Part 1) and multiple ascending doses (Part 2) of MF-300. The Company anticipates reporting topline results in the second half of 2025.

About MF-300

MF-300 is an investigational, orally bioavailable small molecule that reversibly binds to the prostaglandin E2 (PGE2) binding site of 15-hydroxyprostaglandin dehydrogenase (15-PGDH), inhibiting 15-PGDH activity and increasing physiologic levels of PGE2 in skeletal muscle in preclinical studies and raising PGE2 levels in cell-based assays. PGE2 plays a crucial role in promoting aged muscle strength by improving underlying muscle quality (i.e., muscle strength independent of muscle mass) as well as function of the neuromuscular junction in preclinical studies of aged mice. 15-PGDH, an enzyme that converts PGE2 to an inactive metabolite, is upregulated at the level of gene expression in muscle of aged humans and rodents coinciding with age-induced muscle weakness. Inhibiting 15-PGDH in aged muscle may be a strategy to increase physiologic levels of PGE2 to improve muscle quality and function in sarcopenia.

About Epirium Bio

Epirium, a biopharmaceutical company based in San Diego, California, has identified and established an IP-protected platform of orally bioavailable small molecules that constitute a new class of therapeutics with the potential to improve function in neuromuscular diseases, including sarcopenia and spinal muscular atrophy. Epirium has generated preclinical data in a broader scope of indications with significant unmet medical need, including fibrosis, which Epirium’s development pipeline has the potential to address.

To learn more about Epirium, please visit www.epirium.com and follow us on LinkedIn.