Pipeline Therapeutics is set to commence the Phase Ib/IIa clinical trial of its lead programme, PIPE-307, to treat relapsing-remitting multiple sclerosis (RRMS) patients.
The move comes after the company obtained clearance for its investigational new drug application from the US Food and Drug Administration (FDA) to launch the trial.
An oral, selective muscarinic M1 receptor antagonist, PIPE-307 is being developed for restoring myelin to treat MS.
The double-blind, randomised, placebo-controlled, dose-ranging trial will evaluate the safety and tolerability of two varying doses of PIPE-307 versus placebo in patients with RRMS.
It will also investigate the use of visual, radiological and neurophysiological assessments of remyelination.
To be carried out at multiple study centres in the US, the trial will enrol nearly 45 subjects to administer oral doses of the treatment daily for more than three months.
Pipeline Therapeutics president and CEO Carmine Stengone said: “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.”
According to findings from the concluded Phase I studies in healthy subjects, PIPE-307 was found to possess linear pharmacokinetic (PK) data in line with preclinical modelling. It was also found to be well tolerated in various dose cohorts.
The latest approval comes after the company reported positive data from a Phase I trial of PIPE-307 in healthy subjects for potential MS treatment.
The oral therapy showed to have favourable safety and PK profile and met the primary and secondary endpoints of the trial.