Pipeline Therapeutics, a clinical-stage biopharmaceutical firm specializing in precision neuroregeneration, has announced the initiation of dosing for the Phase 1 clinical trial of PIPE-791 in healthy volunteers. The novel small molecule antagonist targets the lysophosphatidic acid 1 receptor (LPA1), setting the stage for a new era in treating CNS indications.

Carmine Stengone, President and CEO of Pipeline Therapeutics, emphasized the importance of this trial, stating, “Initiating dosing in this first in human study with PIPE-791 is a significant milestone for Pipeline that brings us one step closer to transforming the treatment of CNS indications by addressing both remyelination and neuroinflammation. PIPE-791 is our lead, wholly owned program, and we look forward to evaluating its ability to inhibit the LPA1 receptor to bring benefit to people with MS and other neurological and systemic diseases.”

Last month, the US Food and Drug Administration (FDA) cleared Pipeline Therapeutics to launch the Phase 1 clinical trial of PIPE-791. The randomized, double-blind, placebo-controlled dose-ranging study aims to enrol roughly 80 healthy volunteers, and it marks a pivotal phase in the development of PIPE-791 for remyelination and neuroinflammation treatment.

PIPE-791, recognized as a potent and selective brain-penetrant small molecule antagonist of the LPA1 receptor, has exhibited promising results in in vitro and in vivo models. It has also been tolerated in IND-enabling studies. Lysophosphatidic acid (LPA), a pro-inflammatory lipid, is upregulated in MS patients’ plasma, peripheral blood mononuclear cells, and cerebrospinal fluid. LPA’s activation of the LPA1 receptor leads to a localized neuroinflammatory response that hampers remyelination.

The capabilities of PIPE-791 have been highlighted through in vitro evidence of oligodendrocyte differentiation, myelination, and protection from cytokine-induced oligodendrocyte cell death. Pre-clinical in vivo models have further revealed PIPE-791’s potential in enhancing central nervous system LPA1 receptor occupancy, increasing remyelination, and inhibiting neuroinflammation.

This advancement in Pipeline Therapeutics’ clinical work on PIPE-791 signifies a meaningful progression in the field of neuroregeneration and represents a crucial effort to address complex neurological conditions like MS. The Phase 1 clinical trial stands as a testimony to the company’s unwavering commitment to pioneering innovative solutions that may ultimately benefit individuals afflicted with various neurological and systemic diseases.

  Carmine Stengone, Central nervous system, PIPE-791, Pipeline Therapeutics, USA