Med-Life Discoveries to start trials of rare pediatric diseases

Med-Life Discoveries, LP (MLD) is a Canadian biopharmaceutical company dedicated to advancing therapies for alleviating symptoms of the devastating rare pediatric 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 the lead compound PPI-1011. This is a milestone towards a potential therapy for children with RCDP.

The focus of Med-Life Discoveries is the development of plasmalogen therapy for the complex diseases associated with plasmalogen deficiency. PPI-1011 is a novel, first-in-class synthetic plasmalogen therapy developed for the treatment of RCDP.

About RCDP

Rhizomelic chondrodysplasia punctata (RCDP) is a rare genetic pediatric disease caused by mutations in one of several genes that prevent the biosynthesis of a critical class of phospholipids called plasmalogens. Lack of these lipids in the body is the underlying metabolic cause of this disease. There is currently no treatment for RCDP.

Patients with RCDP often have skeletal dysplasia, cataracts, and severe growth and development delays. The children also exhibited severe neurological deficits, seizures, gastrointestinal and lung problems, which negatively impacted their quality of life. In addition, their life expectancy is drastically reduced, with many patients not living beyond 10 years. The overall survival rate and disease severity are directly related to the amount of plasmalogen in their body.

About the Med-Life Discoveries clinical trials

NOL gave Med-Life Discoveries regulatory approval to proceed with the clinical trial in Canada, which will enroll healthy adult volunteers later this month.

This first phase 1 in humans will evaluate the safety and tolerability of PPI-1011 and is the first regulatory-approved trial of a pharmaceutical-grade plasmalogen drug of its kind. Med-Life Discovery has been focused over the past six years on developing and advancing a portfolio of plasmalogen-based compounds for RCDP and other neurodegenerative disorders with compromised plasmalogen biosynthesis.

“There is a huge unmet need for legitimate pharmaceutical-grade plasmalogen therapy 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 conduct a phase 1 human study of plasmalogen therapy is an exciting and long-awaited advance for patients and their families living with RCDP. For our passionate and dedicated team of scientists and researchers dedicated to solving the puzzle of RCDP and other plasmalogen deficiency, this is a double win: an exciting milestone in MLD scientific research, but also an opportunity to improve the lives of children around the world living with the disease. this rare and devastating.”

Tara Smith, executive vice president of innovative therapies at Med-Life Discoveries, added: “We are excited to be the first company in the world to successfully complete a formal preclinical safety study for a synthetic plasmalogen drug candidate. We are proud to now bring this drug into the clinic to test it for safety with our regulatory approval, Health Canada. While there is still a lot of work ahead of us, this human trial is a major step toward 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 ongoing Natural History study. We thank all of the Rhizo family – we couldn’t have moved this care forward without your support.”

“This is a major step in our journey to develop medical therapies for the 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 principal investigator for the RCDP Natural History study.

“We see a huge unmet need in the children we care for in our clinic and who we study in a prospective Natural History trial. We really hope phase 1 is successful so we can move on to intervention trials.”

Source link

Related Articles

Back to top button