Phase 1 will test single oral doses of PRO-1562 in healthy adult subjects to characterize the safety and the amount of drug that is absorbed into the body. Some subjects will have lumbar punctures performed to measure the amount of drug that crosses the blood-brain barrier since that is the site of drug activity.
Phase 2a will be a 6-month trial to test the ability of once monthly dosing of PRO-1562 to promote remyelination of the CNS and provide clinical benefit to adult patients with relapsing multiple sclerosis (RMS) who also have long-term vision problems diagnosed as chronic optic neuropathy. The benefits of remyelination will be measured using tests of visual acuity, speed of optic nerve conduction signals, and clinical assessments of cognitive and motor function. PRO-1562 will be added on to a stable regimen of MS disease modifying therapy.
Able and willing to provide written informed consent
Body weight ≥ 48 kg (105.6 lbs) and body mass index (BMI) ≥ 18.5 and ≤ 30 kg/m2
Must be in good health and without clinically significant abnormalities by review of medical and surgical history, physical examination, vital signs measurement, and 12-lead ECG
No clinically-significant laboratory abnormalities
Ongoing medical condition requiring systemic therapy.
Ongoing infection
mRNA or live vaccine during the past 60 days
History of significant hypersensitivity, intolerance, or allergy to any drug/medication
Single group
PRO-1562 Oral Capsules
DrugOral, CNS-penetrant small molecule designed to induce remyelination
Placebo Oral Capsule
DrugPlacebo filled capsules that look like the active intervention
10
Treatment groupsSee each treatment group below.
Phase 1 Incidence of ≥Grade 2 treatment-emergent adverse events
Incidence of treatment-emergent adverse events including clinically significant laboratory abnormalities of ≥Grade 2 in healthy participants
Phase 2 Change from baseline P100 VEP latency
The change from baseline on visual evoked potential (VEP) in affected eye of RMS patients after 6 months of study treatment
Area under the concentration-time curve of PRO-1562 (Phase 1)
Concentration over time analysis to determine overall exposure to PRO-1562 by dose group
Brain myelin content, change from baseline
MRI measurement of brain myelin content in multiple brain regions will be determined as baseline and after 6 months of treatment
Safety Phase 2 Incidence of ≥Grade 2 treatment-emergent adverse events
Incidence of treatment emergent adverse events including clinically significant laboratory abnormalities of ≥Grade 2 in RMS patients
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