Pipeline Testing PIPE-307 Remyelinating Compound in Healthy Volunteers

Pipeline Therapeutics has initiated a Phase 1 clinical trial to investigate PIPE-307, its lead treatment candidate for multiple sclerosis (MS), in healthy volunteers.

The trial (NCT04725175) will assess the compound’s safety, tolerability, and pharmacokinetics, or how the drug moves through the body. Up to 72 people, ages 18 to 55, will be enrolled at the trial’s single testing center in Western Australia. Recruitment is ongoing; more information can be found here.

Results from this study are expected to inform the design and optimal dose for a future Phase 2 trial in MS patients.

“Evaluating PIPE-307 in healthy volunteers is a critical step in our effort to identify the appropriate dose selection for the follow-on Phase 2 study, to be conducted in patients with multiple sclerosis,” Stephen Huhn, MD, Pipeline’s chief medical officer and senior vice president, clinical development, said in a press release.

PIPE-307 is designed to help regrow myelin, a fatty substance that surrounds nerve cells like a sheath and is important for these cells to efficiently transmit electrical signals.

Myelin is damaged in MS patients, leading to nerve damage and chronic disability. Yet, effective treatments that induce myelin regrowth, or remyelination, to counteract disease progression in MS patients are lacking.

“While immune-modulatory approaches reduce relapse rate, there is currently no effective restorative therapy on the market that targets remyelination in order to address the progressive disability associated with multiple sclerosis,” Huhn said, underscoring remyelination’s significance as an unmet medical need in MS.

PIPE-307 works by inactivating the muscarinic receptor type 1, which stimulates oligodendrocyte progenitor cells to grow into mature, myelin-producing oligodendrocyte cells. Of note, oligodendrocytes are the myelinating cells of the central nervous system, comprised of the brain and spinal cord.

The ongoing clinical trial will be conducted in three parts. In part one, some 48 participants will receive a single ascending dose of oral PIPE-307 or a placebo over six weeks. Part 2 will assign 24 participants to either multiple ascending doses of PIPE-307 or a placebo over seven weeks.

Finally, Part 3 will evaluate how food affects the bioavailability of PIPE-307 — meaning how much ingested medicine reaches the blood and is actively used for therapeutic effect — among approximately eight participants from the single ascending dose section. This part of the study will last for six weeks.

Researchers will assess the therapy’s safety through periodic electrocardiograms, blood laboratory analyses, vital sign measurements, physical examinations, and monitoring for adverse side effects.

Pipeline expects to announce results from this study in the second half of the year.

Earlier this year, Pipeline raised $80 million in support of its neurodegenerative development programs. In addition to PIPE-307, other candidate therapies include compounds targeting remyelination and neuroinflammation, axonal (or nerve fiber) repair, and sensorineural hearing loss.