Pipeline Therapeutics announced positive results from a phase 1 clinical trial of PIPE-307, an investigational oral myelin restoration therapy for patients with multiple sclerosis (MS). A news release from the company described PIPE-307 as a highly selective antagonist of the muscarinic M1 receptor.

“Currently, there are no approved medicines that support myelin restoration, which represents a significant unmet medical need in the treatment of MS,” the company noted. “These encouraging Phase 1 results give us great confidence to advance PIPE-307 into clinical studies with MS patients as soon as possible.”

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In a preclinical animal model of inflammatory demyelination, PIPE-307 promoted the differentiation and maturation of oligodendrocyte precursor cells, leading to remyelination and restored neuronal functioning. In addition, PIPE-307 restored visual evoked potentials, an important measure of clinical disease progression in patients with MS.

The company’s phase 1 clinical trial was a randomized, double-blind, placebo-controlled study that evaluated the safety and tolerability of PIPE-307 in 70 healthy volunteers across multiple dose cohorts. The cohorts included a single ascending dose study, a multiple ascending dose study, and a single ascending dose study with fed participants to assess the effect of food on the drug’s bioavailability.

According to the company, the trial results demonstrated that the drug was generally well-tolerated, with no significant pharmacokinetic (PK) or dose-related effects on cognitive function observed in any of the patient cohorts. The company hopes to receive authorization for an Investigational New Drug (IND) application from the US Food and Drug Administration (FDA) then advance PIPE-307 into trials with MS patients as soon as possible.

Reference

Pipeline Therapeutics reports positive phase 1 clinical results for PIPE-307, a neuroregenerative therapeutic for the treatment of multiple sclerosis. News release. Pipeline Therapeutics; March 10, 2022.