MLD Announces Completion of Dosing with Plasmalogen-precursor PPI-1011 in its Phase I Clinical Trial
Feb 6, 2024. SASKATOON, SASKATCHEWAN, CANADA. Med-Life Discoveries, LP (“MLD”, or the “Company”), a Canadian clinical-stage biopharmaceutical company developing innovative plasmalogen-based therapies for the treatment of Rhizomelic chondrodysplasia punctata (RCDP) and other neurological conditions, announced that all healthy adult participants of a Phase I clinical trial evaluating the safety and pharmacokinetics of its lead compound PPI-1011 have completed dosing. The company will now focus on preparing an FDA-IND application to advance the compound directly into a Phase III RCDP therapeutic registration trial.
The Phase I trial consisted of 56 total participants assigned to a single ascending dose arm to evaluate safety after single daily doses of between 10 and 100 mg/kg, followed by a 14-day in-clinic multiple ascending dose performed at daily doses of 75 and 100 mg/kg. PPI-1011 was very well-tolerated, with no serious AEs observed in any cohort throughout the study. Top-line results are being presented in Lisbon, Portugal on March 8th at the 2024 International AD/PD Conference.
Shawn Ritchie, MLD’s CEO & Chief Scientific Officer, stated, “The completion of this Phase I with PPI-1011 represents a monumental achievement, as it is the first formal human clinical safety study ever performed with a plasmalogen-targeted compound. We are now excited to move quickly into efficacy studies in RCDP and other neurological indications.”
Tara Smith, Executive VP of Innovative Therapies, added, “The RCDP community has made it clear to us that demonstrating the safety of synthetic plasmalogen treatments is critical. The Phase I trial was designed to extensively evaluate the safety of this first-in-class drug and while the study remains blinded, we can confirm that there were no adverse events in the single or multiple dose portion of the trial that would suggest a safety concern with PPI-1011. While we appreciate that there is still a long road ahead, these findings move us closer to evaluating PPI-1011 in the RCDP patient population. The focus now is to design the RCDP registration trial to maximize the possibility of demonstrating efficacy and successfully receiving the first approval for an RCDP-targeted treatment.”
About PPI-1011
MLD holds the world-wide rights to PPI-1011, a proprietary oral synthetic plasmalogen precursor that replenished plasmalogen levels throughout the body. Plasmalogens are membrane phospholipids containing a vinyl-ether bond that are involved in critical cellular processes including neurotransmission, cell signaling, oxidation, and myelination.
About RCDP
Rhizomelic chondrodysplasia punctata (RCDP) is a rare genetic pediatric disease that is caused by mutations in any one of several genes that prevents the biosynthesis of a critical class of phospholipids called plasmalogens. The lack of these lipids in the body is the underlying metabolic cause of the disease. There are currently no treatments for RCDP.
Patients with RCDP often have skeletal dysplasia, cataracts and profound growth and developmental delays. The children also exhibit severe neurological deficits, seizures, gastrointestinal and pulmonary issues, negatively impacting their quality of life. In addition, their life expectancy is dramatically reduced, with many patients not living beyond 10 years of age. Survival rates and overall disease severity are directly related to the amount of plasmalogens in their bodies.
About MLD
MLD is a biopharma company located in Saskatoon, Saskatchewan, Canada, which is developing a pipeline of plasmalogen-based drugs, including PPI-1011, for RCDP and other neurological indications. More information available at med-life.ca.