Epirium Bio Presented Late-Breaking Preclinical Data of MF-300 (15-PGDH Enzyme Inhibitor) at the Gerontological Society of America Annual Scientific Meeting 2024

MF-300 is a first-in-class, orally administered, 15-PGDH enzyme inhibitor shown to restore muscle quality and strength in aged mice

SAN DIEGO–(BUSINESS WIRE)–Epirium Bio, Inc. (Epirium), a biopharmaceutical company advancing medicines for neuromuscular and fibrotic diseases, presented preclinical data for its lead candidate, MF-300, at the Gerontological Society of America’s (GSA) annual meeting being held November 13-16, 2024, in Seattle, WA.

MF-300 is a first-in-class, orally administered, 15-hydroxyprostaglandin dehydrogenase (15-PGDH) enzyme inhibitor in development for the treatment of age-induced muscle weakness, or sarcopenia. According to the U.S. Food and Drug Administration’s (FDA) Patient-Focused Drug Development Initiative report on sarcopenia, up to a third of Americans over the age of 60 are affected by sarcopenia, which increases their risk for falls, fractures and mortality. There are no FDA-approved therapies for sarcopenia, a disease that disproportionately affects fast-twitch muscle fibers, and is characterized by decreased muscle quality and muscle mass.

Epirium’s study, presented at GSA, tested whether MF-300 could improve muscle force in aged mice. After 12 weeks of oral administration at its target dose, MF-300 significantly increased absolute and specific force (i.e., force per unit of muscle) of isometric plantar flexion in aged animals compared to vehicle. The study also demonstrated that MF-300 significantly increased specific force and contraction rate in clinically relevant fast-twitch muscle. Together, these data show that MF-300 has the potential to reverse the decline in muscle quality (i.e., the loss of specific muscle force) that occurs with aging.

“The capacity of muscle to generate force is a better indicator of muscle quality, health, overall functional capacity, and survival than the quantity of muscle mass. MF-300’s novel mechanism of action on intrinsic muscle properties has the potential to improve functional outcomes and allow people to live physically independent lives longer,” said Dr. Scott Delp, Professor of Bioengineering, Stanford University Schools of Medicine and Engineering.

Details of the poster presentation include:

Title: MF-300 (15-PGDH Enzyme Inhibitor) Reverses Age-Related Muscle Weakness in Mice by Restoring Muscle Quality

Presenter: Micah Webster, Ph.D.

Poster Number: 1914819

The presentation is available in the “Posters and Publications” section of Epirium’s website, www.epirium.com.

About MF-300

MF-300 is an orally bioavailable small molecule that reversibly binds to the Prostaglandin E2 (PGE2)-binding site of 15-hydroxyprostaglandin dehydrogenase (15-PGDH), an enzyme that converts PGE2 to an inactive metabolite. MF-300 target engagement inhibits 15-PGDH activity, stabilizing and increasing levels of PGE2 in a cell-based assay and in vivo in skeletal muscle in preclinical studies.

In humans and rodents, 15-PGDH gene expression is elevated in muscle coincident with the onset of age-induced muscle weakness. PGE2, a lipid signaling molecule with multiple beneficial effects on the motor unit, including enhanced muscle quality and improved function of the neuromuscular junction, is reduced in skeletal muscle of aged mice due to increased activity of 15-PGDH. Inhibiting 15-PGDH in aged muscle may be a strategy to increase physiologic levels of PGE2 to improve muscle quality and function.

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.

Contacts

Email: info@epirium.com

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

ARMGO Pharma is a privately held biopharmaceutical company dedicated to applying original, targeted science to the discovery and development of novel small-molecule therapeutics to treat debilitating cardiac, musculoskeletal, and neurological disorders. The company’s proprietary drugs, known as Rycals®, are a new class of oral agents that repair calcium leaks through the Ryanodine Receptor (RyR).

ARMGO Pharma - developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

ARM210 is a potential disease modifying therapy for genetic diseases, which targets the Ryanodine Receptor calcium channel (RyR), an intracellular calcium-release channel that becomes leaky in these and other diseases. Intracellular calcium leaks via mutant RyR1 channels impair muscle contraction leading to muscle weakness and loss of function, and activate toxic pathways that damage muscle, causing the symptoms in RYR1-RM.

ARM210 is a small molecule that binds to leaky RyR channels and repairs the leak, as demonstrated in vitro in muscle biopsies from RYR1-RM patients. The unique mechanism of action of ARM210 makes it an ideal potential disease modifying therapy for RYR1-RM. Muscle biopsies of the patients in this trial have been previously shown to respond biochemically to ARM210 in vitro. This trial will evaluate the safety and explore the biochemical effect of oral administration of ARM210 in these same patients.

ARMGO Pharma has been awarded an exclusive, worldwide license from Columbia University for its RyR technology based on the research of founding scientist Andrew R. Marks, M.D. In December 2021, ARMGO Pharma raises a $35 million Series B  funding round to progress clinical studies of lead molecule ARM210 in cardiac and skeletal muscle diseases.

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

More info : www.armgo.com

Keywords : ARMGO Pharma , small molecule , therapeutics , RyR technology , ARM210 , cardiovascular , musculoskeletal disorders , neurological disorders , CNS , neuroscience , Rycals , Ryanodine Receptor , RyR , Ryanodine Receptor modulators

Biophytis – therapeutics to slow down the degenerative processes associated with aging

Biophytis is a clinical-stage biotechnology company specialized in the development of therapeutics that are aimed at slowing the degenerative processes associated with aging and improving functional outcomes for patients suffering from age-related diseases, including severe respiratoy failure in patients suffering from COVID-19.

Biophytis - therapeutics to slow down the degenerative processes associated with aging

Biophytis SA, founded in 2006, develops drug candidates targeting diseases of aging. Using its technology and know-how, Biophytis has begun clinical development of innovative therapeutics to restore the muscular and visual functions in diseases with significant unmet medical needs. Specifically, the company is advancing two proprietary drug candidates into mid-stage clinical testing this year: Sarconeos (BIO101) to treat sarcopenic obesity and Macuneos (BIO201) to treat dry age-related macular degeneration (AMD). The business model of BIOPHYTIS is to ensure the conduct of the project until clinical activity in the patient is proven, then to license the technologies in order to continue the development in partnership with a pharmaceutical laboratory. The company was founded in partnership with researchers at the UPMC (Pierre and Marie Curie University) and collaborates with scientists at the Institute of Myology, and the Vision Institute

Its lead drug candidate, Sarconeos (BIO101), is an orally administered small molecule in development for the treatment of neuromuscular diseases, including sarcopenia and Duchenne muscular dystrophy (DMD). Our second drug candidate, Macuneos (BIO201), is an orally administered small molecule in development for the treatment of retinal diseases, including dry AMD and Stargardt disease.

Biophytis also has preclinical efficacy data for Sarconeos (BIO101) in Spinal Muscular Atrophy (SMA), a rare neuromuscular disease. Spinal Muscular Atrophy is a rare progressive neurodegenerative disease that robs an individual of the ability to walk, eat and breathe. SMA is the leading genetic cause of death in infants. Symptoms may appear in the first 6 months of life (type 1, the most severe and common), in infancy (types 2 and 3) or in adulthood (type 4, the least common form). SMA affects 1 in 11,000 births in the United States each year, and about 1 in 50 Americans is a genetic carrier.

More info : www.biophytis.com

Keywords for Biophytis :

neuromuscular diseases, dry age-related macular degeneration, AMD, ophthalmology, infectious diseases, COVID19, Asthma, COPD, small molecule, sarcopenia, Stargardt disease, Duchenne muscular dystrophy , DMD, preclinical stage pharma company, clinical stage pharma company , Spinal Muscular Atrophy , rare diseases , aging