Pipeline Therapeutics Receives IND Clearance from FDA to Initiate Phase 1b/2a Study of PIPE-307 in Patients with Relapsing-Remitting Multiple Sclerosis

SAN DIEGO--(BUSINESS WIRE)-- 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 1b/2a clinical trial of PIPE-307 in patients with relapsing-remitting multiple sclerosis (RRMS). Currently, there are no approved medicines that support myelin restoration, which represents a significant unmet medical need in the treatment of multiple sclerosis (MS).

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 MS. The Company recently completed multiple Phase 1 studies of PIPE-307 in healthy volunteers, including a single and multiple-ascending dose study to evaluate safety and pharmacokinetics, and a positron emission tomography (PET) study to determine M1 receptor occupancy and clearance in the brain after a single dose of PIPE-307. Results from the PET study demonstrate that doses tested in the Phase 1 study achieve a level of uptake in the human brain that have been associated with remyelination observed in preclinical studies.

“FDA clearance to initiate our Phase 1b/2a clinical study of PIPE-307 is a major regulatory milestone for Pipeline, as it allows us to proceed with the evaluation of PIPE-307 in RRMS patients,” said Carmine Stengone, President and CEO of Pipeline. “Today, approved medicines for MS patients are focused on immune modulation but do not address the fundamental MS disease pathology that leads to long-term decline in neurological function – chronic demyelination. Our objective with PIPE-307 is to develop the first effective treatment that can restore myelin and improve outcomes for patients.”

Stephen Huhn, M.D., Chief Medical Officer and Senior Vice President of Clinical Development of Pipeline, added, “We are excited to initiate the Phase 1b/2a study of PIPE-307 in RRMS patients, which follows on the heels of positive data from our Phase 1 studies. The Phase 1b/2a study is designed to primarily assess the safety and tolerability of two different doses of PIPE-307 compared to placebo in RRMS patients, and to explore the use of neurophysiological, visual and radiological measures of remyelination. We remain on track to commence patient dosing in mid-2022.”

The Phase 1b/2a clinical trial of PIPE-307 will be conducted as a randomized, double-blind, placebo-controlled dose-ranging study in multiple sites across the US. The study will enroll approximately 45 patients who will undergo daily oral dosing over three months. The study is expected to start enrolling patients in mid-2022.

About PIPE-307 for Multiple Sclerosis

PIPE-307 is an oral, highly selective antagonist of the M1 muscarinic receptor commencing clinical studies in MS patients. The Company recently completed multiple Phase 1 studies of PIPE-307 in healthy volunteers, including a single- and multiple-ascending dose study to evaluate safety and pharmacokinetics, and a PET study to determine the occupancy of brain M1 after a single dose of PIPE-307 in healthy volunteers. In these studies, 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. Results from the Phase 1 PET study demonstrated that doses tested in the Phase 1 SAD/MAD study achieve a level of uptake in the human brain that have been associated with remyelination observed in preclinical studies.

In March 2022, the Company received clearance from the U.S. Food and Drug Administration to proceed with a Phase 1b/2a study of PIPE-307 in RRMS patients.

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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