A Phase 1/2a Study of Oral PRO-1562 in Healthy Subjects and Patients With Relapsing Multiple Sclerosis

A Randomized, Double-Blind, Placebo-Controlled Phase 1/2a Trial of PRO-1562 in Healthy Adults and Adults With Multiple Sclerosis and Chronic Optic Neuropathy

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Multiple Sclerosis
• Intervention / Treatment: Drug: PRO-1562 Oral Capsules; Drug: Placebo Oral Capsule

Detailed Description

Phase 1 is a randomized, double-blind, placebo-controlled, single-ascending dose (SAD) trial of PRO-1562 in healthy adult participants at a single clinical research unit to characterize the safety and pharmacokinetics of a range of oral doses of PRO-1562. Phase 2a is a randomized, double-blind, placebo-controlled, multi-center, 6-month trial of PRO-1562 in adult patients with relapsing multiple sclerosis who also have chronic optic neuropathy. Patients will remain on their stable regimen of an approved MS disease modifying therapy during the trial. Oral PRO-1562 or placebo will be dosed once a month at scheduled clinic visits. Participants will be given the option to continue on their assigned study treatment after completing the 6-month treatment period until the last patient completes the 6-month treatment period.

• Study Type: Interventional
• Enrollment (Estimated): 160
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Chief Medical Officer; Phone Number: 858-306-2085; Email: pfrohna@progentos.com

Study Locations

• Australia
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • CMAX Clinical Research
      • Contact: Study Director; Phone Number: 61 1800 150 433; Email: cmax@cmax.com.au

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Phase 1 Inclusion Criteria:

1. Able and willing to provide written informed consent
2. Body weight ≥ 48 kg (105.6 lbs) and body mass index (BMI) ≥ 18.5 and ≤ 30 kg/m2
3. 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
4. No clinically-significant laboratory abnormalities
5. Willing and able to use highly-effective forms of contraception for at least 30 days after the end of study visit.

Phase 1 Exclusion Criteria:

1. Ongoing medical condition requiring systemic therapy.
2. Ongoing infection
3. mRNA or live vaccine during the past 60 days
4. History of significant hypersensitivity, intolerance, or allergy to any drug/medication
5. Participation in another clinical trial with an investigational agent within 30 days prior to Check-in or 5 half-lives (if known) of the investigational drug's PK, PD, or biological activity (if known), whichever is longer
6. Positive alcohol breath test or urine screen for drugs of abuse.
7. Use or intent to use natural products or nutritional/dietary supplements (including St. John's wort), vitamins, minerals, and phytotherapeutic /herbal/plant-derived preparations within or received within 14 days or 5 half-lives prior to Check-in, whichever is longer.
8. Poor peripheral venous access.
9. Receipt of blood products within 2 months prior to Check-in.
10. Donation of blood or blood products during the 4 weeks prior to Check-in.
11. Participants who in the opinion of the Principal Investigator (or designee), should not participate in this study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Single Group Assignment
• Masking: Quadruple

Number of Arms

10

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Phase 1, PRO-1562 Dose level 1; PRO-1562 oral capsule	Drug: PRO-1562 Oral Capsules; Oral, CNS-penetrant small molecule designed to induce remyelination
Experimental: Phase 1, PRO-1562 Dose level 2; PRO-1562 oral capsule	Drug: PRO-1562 Oral Capsules; Oral, CNS-penetrant small molecule designed to induce remyelination
Experimental: Phase 1, PRO-1562 Dose level 3; PRO-1562 oral capsule	Drug: PRO-1562 Oral Capsules; Oral, CNS-penetrant small molecule designed to induce remyelination
Experimental: Phase 1, PRO-1562 Dose level 4; PRO-1562 oral capsules	Drug: PRO-1562 Oral Capsules; Oral, CNS-penetrant small molecule designed to induce remyelination
Experimental: Phase 1, PRO-1562 Dose level 5; PRO-1562 oral capsule	Drug: PRO-1562 Oral Capsules; Oral, CNS-penetrant small molecule designed to induce remyelination
Experimental: Phase 1, PRO-1562 Dose level 6; PRO-1562 oral capsule	Drug: PRO-1562 Oral Capsules; Oral, CNS-penetrant small molecule designed to induce remyelination
Placebo Comparator: Phase 1, Placebo; Placebo oral capsule	Drug: Placebo Oral Capsule; Placebo filled capsules that look like the active intervention
Experimental: Phase 2, PRO-1562 Oral Capsules Dose level 1; PRO-1562 low dose level selected from Phase 1 (oral capsule)	Drug: PRO-1562 Oral Capsules; Oral, CNS-penetrant small molecule designed to induce remyelination
Experimental: Phase 2, PRO-1562 Oral Capsules Dose level 2; PRO-1562 high dose level selected from Phase 1 (oral capsule)	Drug: PRO-1562 Oral Capsules; Oral, CNS-penetrant small molecule designed to induce remyelination
Placebo Comparator: Phase 2, Placebo; Placebo oral capsule	Drug: Placebo Oral Capsule; Placebo filled capsules that look like the active intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	From enrollment until 7 days post dose
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	From enrollment until the end of 6 months of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	From enrollment until 7 days post dose.
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	From enrollment to the end of 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	From enrollment until the end of 6-months of treatment

Collaborators and Investigators

• Sponsor: Progentos Therapeutics
• Investigators: Study Chair: Paul A Frohna, MD, PhD, PharmD, Progentos Therapeutics

Study record dates

Study Major Dates

• Study Start (Estimated): October 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): February 1, 2029

Study Registration Dates

• First Submitted: September 8, 2026
• First Submitted That Met QC Criteria: September 11, 2026
• First Posted (Actual): September 17, 2026

Study Record Updates

• Last Update Posted (Actual): September 17, 2026
• Last Update Submitted That Met QC Criteria: September 11, 2026
• Last Verified: September 1, 2026

More Information

Terms related to this study

• Keywords: relapsing multiple sclerosis; Remyelination; chronic optic neuropathy
• Additional Relevant MeSH Terms: Nervous System Diseases; Autoimmune Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Multiple Sclerosis
• Other Study ID Numbers: PRO-1562-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No