MLD Announces Positive First-in-Human Phase I Results of Its Synthetic Plasmalogen Precursor Drug PPI-1011

SASKATOON, Saskatchewan, March 17, 2025 (Newswire.com) - Med-Life Discoveries, LP ("MLD", or the "Company"), a biopharmaceutical company dedicated to the development of plasmalogen therapeutics for complex diseases associated with plasmalogen deficiencies, is excited to announce that it has published the first ever Phase I study of a plasmalogen precursor treatment in humans. The Phase I study was completed in Canada and evaluated the safety, tolerability, and pharmacokinetic properties of PPI-1011 in healthy adults. PPI-1011 is a synthetic plasmalogen precursor being developed as a treatment for diseases of plasmalogen deficiency, including Rhizomelic chondrodysplasia punctata (RCDP).
RCDP is a rare pediatric disorder caused by mutations in one of the genes in the plasmalogen biosynthetic pathway, resulting in low plasmalogen levels. The plasmalogen deficiency leads to several clinical manifestations causing profound physical and cognitive deficiencies and a significantly reduced life expectancy. Disease severity in RCDP correlates with circulating plasmalogen levels, further confirming the role of the deficiency in the clinical presentation of the disease.
The Phase I study demonstrated for the first time that high doses of orally administered PPI-1011 were well-tolerated, even with repeated dosing up to 14 days. All adverse events that were possibly or potentially related to PPI-1011 were considered mild, monitorable, and resolved without intervention. A single oral administration of PPI-1011 at doses between 10 mg/kg and 100 mg/kg demonstrated a clear dose response with serum plasmalogen levels. Further, subjects who received 14 days of repeated dosing showed robust increases in serum plasmalogen levels, approaching 10 times baseline levels.
Dr. Shawn Ritchie, CEO and Chief Scientific Officer stated, "This publication is a monumental milestone for MLD and the plasmalogen field, as it is the first time anyone has formally reported on the safety, tolerability and pharmacokinetics of a fully synthetic plasmalogen precursor drug in humans. The results exceeded our expectations, and demonstrate why highly pure, pharmaceutical-grade plasmalogens will be an absolute requirement for treatment of conditions associated with plasmalogen deficiency, and we're thrilled that the next step is evaluating the treatment in the RCDP population."
Dr. Tara Smith, Executive Vice President, added, "The RCDP community has been clear that they are only interested in supporting a plasmalogen treatment with a demonstrated safety profile. The results of this Phase I study will provide confidence to the families that we are also committed to ensuring the safety profile of PPI-1011 before enrolling patients in a clinical study."
MLD is now in the process of finalizing the details of a Phase II study that will evaluate the safety, pharmacokinetics, and clinical efficacy of PPI-1011 in patients with RCDP. The Phase II study will be the basis of an Investigational New Drug application that will be submitted to the FDA shortly.
A copy of the manuscript is available at https://ascpt.onlinelibrary.wiley.com/doi/full/10.1111/cts.70195
About MLD
MLD is a biopharma company located in Saskatoon, SK, Canada developing PPI-1011 and other plasmalogen-based drugs for RCDP and other neurological indications. More information available at med-life.ca.
Source: Med-Life Discoveries