SAN DIEGO, March 10, 2022 – Pipeline Therapeutics, a clinical-stage biopharmaceutical company pioneering precision neuroregeneration, today reported positive clinical data from its recently completed Phase 1 clinical trial evaluating the safety and tolerability of PIPE-307 in healthy volunteers. PIPE-307, the company’s lead program for myelin restoration, is an oral, highly selective antagonist of the muscarinic M1 receptor that is being developed for the treatment of multiple sclerosis (MS). Currently, there are no approved medicines that support myelin restoration, which represents a significant unmet medical need in the treatment of MS.
The Phase 1 clinical results reported today comprised multiple dose cohorts of PIPE-307 in a total of 70 healthy volunteer subjects. PIPE-307 demonstrated linear pharmacokinetic (PK) data consistent with preclinical modeling and was generally well tolerated across all dose cohorts. Importantly, analysis of a battery of neuropsychological measures, including tests involving psychomotor, attention, learning, and executive function, were administered in the Phase 1 study and showed no significant PK or dose related effects on cognitive function.
“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. The results of the single- and multiple-ascending dose cohorts from the completed Phase 1 study demonstrate a favorable safety and PK profile, and we are pleased to have met our primary and secondary endpoints in this study,” said Stephen Huhn, M.D., Chief Medical Officer and Senior Vice President of Clinical Development of Pipeline Therapeutics. “These encouraging Phase 1 results give us great confidence to advance PIPE-307 into clinical studies with MS patients as soon as possible.”
Carmine Stengone, President and CEO of Pipeline, added, “PIPE-307 is our flagship program and we’re thrilled to have completed this first step in our approach to myelin restoration. Multiple sclerosis is a devastating neurodegenerative disease that impacts almost three million people worldwide, and therapies that are designed to restore neuronal damage, like PIPE-307, are desperately needed. Our clinical plans remain on track and we look forward to evaluating PIPE-307 in MS patients.”
The completed Phase 1 clinical trial of PIPE-307 was a randomized, double-blind, placebo-controlled trial in 70 healthy adult volunteers. The trial was conducted in three parts: (1) a Single Ascending Dose (SAD) study, (2) a Multiple Ascending Dose (MAD) study, and (3) a selected SAD cohort in a fed state to evaluate the effect of food on bioavailability of PIPE-307. The primary endpoint of the trial was to measure the safety and tolerability of PIPE-307, with a secondary endpoint measuring the effect of PIPE-307 on pharmacokinetics. This study was completed at a single center in Australia.
About PIPE-307 for Multiple Sclerosis
PIPE-307 is an oral, highly selective antagonist of the M1 muscarinic receptor in development for multiple sclerosis (MS). In preclinical studies, PIPE-307 promoted remyelination by the differentiation and maturation of oligodendrocyte precursor cells, or OPCs, into oligodendrocytes, leading to restored neuronal function in a preclinical animal model of inflammatory demyelination. Preclinical treatment with PIPE-307 also led to a restoration in visual evoked potentials, or VEPs, a measure of electrical conduction velocity used to measure clinical disease progression in MS. The Company expects to evaluate PIPE-307 in MS patients following the authorization of an Investigational New Drug (IND) by the US Food and Drug Administration.
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 CNS and neuro-otology conditions, and its flagship program, PIPE-307, has completed a Phase 1 clinical trial in healthy volunteers to support future studies in MS patients. For more information, please visit www.pipelinetherapeutics.com and engage with us on LinkedIn.
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