Med-Life Discoveries to start rare pediatric disease trial

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Med-Life Discoveries, LP (MLD) is a Canadian biopharmaceutical company dedicated to advancing a therapy to reduce symptoms for a devastating pediatric rare disease, Rhizomelic chondrodysplasia punctata (RCDP), which causes significant physical and neurological delays in children who inherit it. 

Med-Life Discoveries has received a no objection letter (NOL) from Health Canada to begin a phase 1 healthy adult trial for lead compound PPI-1011. This is a milestone on the path towards a potential therapy for children with RCDP.

Med-Life Discoveries’ focus is the development of plasmalogen therapeutics for complex diseases associated with plasmalogen deficiencies. PPI-1011 is a novel, first-in-class synthetic plasmalogen therapy being advanced for the treatment of RCDP. 

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 Med-Life Discoveries’ clinical trial

The NOL provides Med-Life Discoveries with regulatory approval to move forward with a clinical trial in Canada, which will enroll healthy adult volunteers later this month. 

This first-in-human phase 1 will evaluate the safety and tolerability of PPI-1011 and represents the first regulatory-approved trial for a pharmaceutical-grade plasmalogen drug of its kind. Med-Life Discoveries has been focused for the past six years on the development and advancement of a portfolio of plasmalogen-based compounds for RCDP and other neurodegenerative disorders with underlying compromised plasmalogen biosynthesis.

“There is a huge unmet need for legitimate, pharmaceutical-grade plasmalogen therapeutics for patients with RCDP, as well as other neurodegenerative diseases such as Alzheimer’s,” said Shawn Ritchie, chief executive officer and chief scientific officer at Med-Life Discoveries. 

“Receiving regulatory approval to run the phase 1 human study for a plasmalogen therapeutic is an exciting and long-awaited advance for patients and their families living with RCDP. For our team of scientists and researchers who are passionate and dedicated about solving the puzzle of RCDP and other plasmalogen deficiencies, it is a double win: an exciting milestone in MLD’s scientific research, but also a chance to improve the lives of children around the world living with this rare and devastating disease.”

Tara Smith, executive vice president of innovative therapies at Med-Life Discoveries, added: “We are elated to be the first company in the world to successfully complete formal preclinical safety studies for a synthetic plasmalogen drug candidate. We are proud to now bring this drug into the clinic to test its safety with approval of our regulator, Health Canada. While there is still a lot of work ahead of us, this human trial represents a major step towards a potential treatment for RCDP. There is strong support within the Rhizo community for a regulated, pharmaceutical approach like ours, as evidenced by the number of families enrolled in our on-going Natural History study. We thank all the Rhizo families – we couldn’t advance this treatment without your support.” 

“This is huge step in our journey to develop a medical therapy for our children and families affected by RCDP,” said Michael Bober, medical director of the Skeletal Dysplasia Program at Nemours Children’s Hospital, Delaware. 

Bober also serves as the principal investigator for the RCDP Natural History study. 

“We see a huge unmet need in the children that we treat in our clinic and that we are studying in the prospective Natural History trial. We very much hope that the phase 1 is successful so that we can then move towards an interventional trial.”

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