PIPE-307, an investigational myelin-restoring medication being developed by Pipeline Therapeutics for the treatment of multiple sclerosis (MS), appears to be safe and well tolerated in healthy adults.
The results are from a Phase 1 clinical trial (NCT04725175) that evaluated the safety and tolerability of PIPE-307 in 70 healthy volunteers, ages 18 to 55, at a center in Australia. It also looked at the medication’s pharmacokinetics, or how the medication moves through the body, which was found to be consistent with what was seen in preclinical models.
“These encouraging Phase 1 results give us great confidence to advance PIPE-307 into clinical studies with MS patients as soon as possible,” Stephen Huhn, MD, said in a press release. Huhn is chief medical officer and senior vice president of clinical development with Pipeline.
The company expects to move into clinical testing in MS patients after the U.S. Food and Drug Administration gives the green light on an investigational new drug application for the medication.
PIPE-307 is an antagonist of the M1 muscarinic receptor. This means that it blocks this type of receptor, which helps oligodendrocyte precursor cells transition into oligodendrocytes. Oligodendrocytes are the myelinating cells of the central nervous system (the brain and spinal cord) that make myelin, a fatty substance that forms a sheath around nerves to protect them and allow these cells to efficiently transmit electrical signals.
PIPE-307 is expected to work by restoring myelin, which becomes damaged in MS. This damage makes it difficult for electrical impulses to be carried to and from the brain along the nerves, causing the symptoms of the disease. PIPE-307 is anticipated to counteract these symptoms.
“We believe that PIPE-307, as a first-in-class myelin restoration therapeutic, represents a differentiated and clinically-validated approach for the treatment of MS,” Huhn said.
Click to Subscribe for the MS Beacon eNewsletter and MS educational events
|
