Epirium Bio Inc., a clinical-stage biopharmaceutical company, has completed dosing in its first-in-human Phase 1 clinical trial evaluating MF-300, an investigational oral treatment for sarcopenia. The San Diego-based company announced that preliminary data from the trial showed a favorable safety profile, with all adverse events considered mild to moderate and no severe or serious adverse events reported.
The randomized, double-blind, placebo-controlled single and multiple-ascending dose (SAD and MAD) trial was designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of MF-300 in healthy adults. Notably, there were no early discontinuations from the study, and no stopping criteria were met during the trial conduct.
"We are pleased to have completed dosing, and anticipate sharing the results later this Quarter," said Alex Casdin, Chief Executive Officer of Epirium. "We look forward to gaining valuable insights from this first-in-human investigational study of MF-300 ahead of our Phase 2 safety and efficacy trial in patients with sarcopenia, which is projected to commence enrollment mid-2026."
Novel Mechanism Targets Age-Related Muscle Weakness
MF-300 represents a first-in-class approach to treating sarcopenia through its unique mechanism of action. The investigational compound is an orally bioavailable small molecule that reversibly occupies the prostaglandin E2 (PGE2) binding site of 15-hydroxyprostaglandin dehydrogenase (15-PGDH). This enzyme metabolically degrades PGE2 and is transcriptionally upregulated in aged muscle.
Preclinical data demonstrate that PGE2 plays a crucial role in promoting aged muscle strength by improving muscle quality—defined as muscle strength independent of muscle mass—as well as function of the neuromuscular junction. In preclinical studies, oral administration of MF-300 increased physiologic levels of PGE2 in skeletal muscle in rats and increased muscle force while improving muscle quality in aged mice.
The therapeutic strategy of inhibiting 15-PGDH in aged muscle aims to increase physiologic levels of PGE2 to improve muscle quality and function in sarcopenia patients.
Addressing Significant Unmet Medical Need
Sarcopenia represents a substantial healthcare challenge, with the U.S. Food and Drug Administration (FDA) estimating that up to a third of Americans over the age of 60 are affected by this condition. The disease increases the risk of falls, fractures, disability and all-cause mortality, creating serious health implications for the aging population.
Despite sarcopenia's widespread prevalence and serious health consequences, there are currently no FDA-approved therapies available to treat the condition, highlighting the significant unmet medical need that MF-300 could potentially address.
Development Pipeline and Future Plans
Epirium has established an IP-protected platform of orally bioavailable small molecules that constitute a new class of therapeutics with potential applications in neuromuscular diseases, including sarcopenia and spinal muscular atrophy. The company has generated preclinical data across a broader scope of indications with significant unmet medical need, including fibrosis.
The completion of Phase 1 dosing marks a critical milestone in MF-300's development pathway. With topline results expected later this quarter, Epirium is positioning for the next phase of clinical development, with Phase 2 safety and efficacy trials in sarcopenia patients projected to begin enrollment by mid-2026.
