EPM301
EPM301 for PWS-Related Symptoms
epm Therapeutics is pioneering EPM301, a patent-protected, orally available small molecule that has the potential to address some of the most challenging symptoms of Prader-Willi syndrome (PWS). Our goal is to deliver a treatment that works with safety and efficacy and fits conveniently into the daily lives of those living with PWS and their caregivers.
EPM301 is a unique therapy. Our development program points towards a convenient option ideal for pediatric, adolescent, and adult patients. Animal models predict EPM301 may address multiple PWS symptoms by targeting hyperphagia (excessive hunger), anxiety, and sleep disturbances in an easy to administer oral treatment.
- https://pubmed.ncbi.nlm.nih.gov/35628417/ (hyperphagia)
- https://pubmed.ncbi.nlm.nih.gov/29057454/ (anxiety)
- https://pubmed.ncbi.nlm.nih.gov/29860002/ (mood stabilizer)
- https://pubmed.ncbi.nlm.nih.gov/31838151/ (sleep disturbances)
Our mission is to deliver a safe, effective, and easy-to-administer treatment that improves quality of life for patients and their families.
The Science Behind EPM301
EPM301, Cannabidiol Acid Methyl Ester (CAME), is recognized as a New Chemical Entity (NCE) by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
epm Therapeutics holds strong intellectual property protection for its oral and topical administration.
For EPM301 drug delivery we are developing an oral soft gel capsule that is designed for ease of administration across a broad age range of patients, with a particular focus on low calorie, no dyes, and minimal preservative load.
Results from PWS-Relevant Models
The Magel2-null mouse model is a gold-standard research model for PWS. In studies with EPM301, we were able to secure these published results:
Hyperphagia and Metabolic Improvements
- Reduced Hyperphagia: EPM301 significantly decreased excessive eating—like giving the brain’s "hunger switch" a reset.
- https://pubmed.ncbi.nlm.nih.gov/35628417/
- Lowered Body Weight: Treated group gained less fat despite having access to calorie-rich food.
- Blocked Weight Gain: Test group stayed at a healthier weight compared to untreated mice.
- Improved Body Composition: Increased lean muscle mass and reduced fat accumulation, reducing core weakness and obesity-related risks.
Behavioral & Emotional Stabilization
- Anxiety Reduction: Observed calming effects, with decreased stress-related behaviors in preclinical models.
- https://pubmed.ncbi.nlm.nih.gov/29057454/
- Mood Stabilization: Potential antidepressant-like effects, suggesting broader neuroregulatory benefits beyond appetite control.
- https://pubmed.ncbi.nlm.nih.gov/29860002/
Sleep/Wake Cycle Regulation
- Improved Sleep Patterns: Normalized circadian rhythms, leading to more restorative sleep and daytime alertness.
- https://pubmed.ncbi.nlm.nih.gov/31838151/
Distinguishing EPM301 from Cannabinoids (CBD)
Almost everyone has heard of CBD and how it has been suggested as a possible treatment for many different conditions. We understand with all the information out there, some based in real science and the majority not, it can be a challenge to understand the potential.
EPM301 is a cannabinoid chemically known as cannabidiol acid methyl ester (CAME). Cannabidiol acids were originally discovered, isolated, then stabilized for research in the labs of Raphael Mechoulam at the Hebrew University in Jerusalem. Dr. Mechoulam recognized, investigated, and documented cannabidiol acids’ unique pharmacokinetic and efficacy profile when compared to CBD. epm Therapeutics is continuing Dr. Mechoulam’s vision to define EPM301’s unique qualities as we develop it as a drug product for the treatment of symptoms common in Prader-Willi syndrome (PWS). In addition to hyperphagia (uncontrolled appetite), we are investigating EPM301 for its potential improvement in the sleep/wake cycle (narcolepsy) and behavioral (anxiety) symptoms in PWS patients.
We are confident that our in-vitro and animal model testing data to date provides a solid foundation from which to support our clinical program. Our team is focused on bringing EPM301 into the clinic and documenting its safety and efficacy toward an NDA filing and approval to market.
Please follow us for updated information or email us at letstalk@epmtherapeutics.com.
The Road Ahead
epm Therapeutics is committed to advancing EPM301 as a transformative therapy for PWS patients. EPM301 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the FDA and Orphan Drug Designation by the EU EMA. This offers us expedience in the drug development process and the review for approval to market.
Join Us On The Journey Toward An Improved Treatment
The path forward is clear, but we can’t do it alone. For families, clinicians, caregivers, advocates and investors who are ready to redefine PWS care—please join us. epm Therapeutics is committed to fast-tracking this potential breakthrough therapy for patients and caregivers currently with limited adequate treatments. Let's communicate and keep working toward an improved tomorrow.