Multiple ascending dose study of PPI-1011 begins dosing
Med-Life Discoveries, LP (“MLD”), is Canadian clinical stage biopharmaceutical company advancing therapies for the treatment of plasmalogen deficiencies, including Rhizomelic chondrodysplasia punctata (RCDP). MLD is excited to announce that dosing has begun in the first cohort of participants in the multiple ascending dose (MAD) portion of its Phase 1 trial of PPI-1011. The MAD study will evaluate the safety, tolerability, and pharmacokinetics of PPI-1011 at two dose levels in healthy volunteers when administered the compound for 14 consecutive days.
Initiation of the MAD study builds on the successful completion of a single ascending dose study in which five ascending doses levels of PPI-1011 were evaluated. While analysis of this study remain on-going, all reported AEs were mild in nature, with no serious adverse events (SAE) reported.
Overall the study represents the first regulatory-approved trial for a pharmaceutical-grade plasmalogen product. MLD is developing its synthetic plasmalogen-based compounds for the treatment of RCDP and other neurodegenerative disease which display compromised plasmalogen biosynthesis.
“RCDP is the result of a chronic plasmalogen deficiency and therefore treatments aimed at addressing this deficiency will need to be administered long term, “ said Dr. Shawn Ritchie, Chief Executive Officer and Chief Scientific Officer of MLD. “The MAD study represents a critical component of the clinical program of PPI-1011, confirming that repeated administration of PPI-1011 at relatively high-doses is well-tolerated.”
The results of both the single and multiple dose clinical studies, together with numerous nonclinical safety studies, will be used to support an Investigational New Drug (IND) application to the FDA to allow for the initiation of an intervention study in RCDP patients. MLD’s priority in the coming year will be the design of this intervention study which will evaluate the safety, tolerability, pharmacokinetics, and clinical efficacy of PPI-1011 in patients with RCDP. The study design will draw heavily on the data gathered as part of its on-going Natural History Study of RCDP taking place at Nemours Children’s Hospital in Wilmington, Delaware.
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.