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Pipeline Therapeutics Receives IND Clearance from FDA to Initiate Phase 1 Study of PIPE-791

-Company on track to begin enrolling healthy volunteers in the second half of 2023-

SAN DIEGO, June 8, 2023 – Pipeline Therapeutics, a clinical-stage biopharmaceutical company pioneering precision neuroregeneration, today announced that the U.S. Food and Drug Administration (FDA) cleared the company to initiate its Phase 1 clinical trial of PIPE-791 in healthy volunteers.  The Company’s wholly owned program, PIPE-791, is a potent, selective and brain-penetrant small molecule antagonist of the lysophosphatidic acid 1 receptor (LPA1). LPA1 is a critical element in bioactive phospholipid signaling, and is a powerful mediator of both nervous system and systemic inflammatory conditions. Specifically, LPA1 inhibition has important therapeutic potential through augmentation of remyelination and mitigation of neuro-inflammation.  Multiple sclerosis (MS) is a leading indication for PIPE-791, as there are no currently approved medicines designed to prevent neuronal loss and mitigate clinical disability in MS patients .

“The FDA clearance of PIPE-791’s IND marks another significant milestone for Pipeline and demonstrates our team’s continued focus and commitment to bringing precision therapies to patients with neurological diseases,” said Carmine Stengone, President and CEO of Pipeline.  “PIPE-791 is our second clinical program and our first to address both remyelination and neuroinflammation.  We look forward to evaluating our first-in-class approach to LPA1 inhibition with PIPE-791, with the goal to deliver meaningful benefit to individuals with MS , as well as other neurological and systemic diseases.”

Stephen Huhn, M.D., Chief Medical Officer and Senior Vice President of Clinical Development of Pipeline, added, “We are excited to initiate the Phase 1 study of PIPE-791, which is designed to assess the safety and tolerability of PIPE-791 compared to placebo in healthy volunteers. We expect to commence dosing in the second half of 2023.”

The Phase 1 clinical trial of PIPE-791 will be conducted as a randomized, double-blind, placebo-controlled dose-ranging study, and enroll approximately 80 healthy volunteer subjects.  

About PIPE-791

The Company’s wholly owned program, PIPE-791, is advancing toward clinical development for remyelination and neuroinflammation. 

PIPE-791 is a potent, selective, brain-penetrant small molecule antagonist of the LPA1 receptor that has demonstrated activity in in vitro and in vivo models and was tolerated in IND-enabling studies.  Lysophosphatidic acid (LPA) is a pro-inflammatory lipid, upregulated in the plasma, peripheral blood mononuclear cells and cerebrospinal fluid of MS patients. LPA activates the G-protein coupled LPA1 receptor on both oligodendrocyte precursor cells (OPCs) and microglia resulting in a local neuroinflammatory response limiting remyelination.

PIPE-791 has demonstrated in vitro evidence of oligodendrocyte differentiation, myelination, and protection from cytokine-induced oligodendrocyte cell death. In pre-clinical in vivo models, PIPE-791 has demonstrated evidence of central nervous system LPA1 receptor occupancy, increased remyelination, and inhibition of neuroinflammation.

About Pipeline Therapeutics

Pipeline Therapeutics is a clinical-stage biopharmaceutical company pioneering the development and commercialization of first-in-class therapies for precision neuroregeneration including myelin restoration, synaptogenesis and axonal repair.  The Company has a broad pipeline of programs to address multiple CNS disorders.  In April 2023, Pipeline announced that it entered into a global license and development agreement with Janssen Pharmaceutica NV (Janssen) for its lead program, PIPE-307, a highly selective M1 antagonist, in central nervous system disorders.  The Company’s second program, PIPE-791, an LPA1 receptor antagonist, has received FDA clearance of its IND and is advancing into Phase 1 testing.

For more information, please visit www.pipelinetherapeutics.com and engage with us on LinkedIn.

Company Contact:
Peter Slover

Investor Contact:
Amy Conrad