Rozanolixizumab for MOG-AD

(cosMOG Trial)

Recruiting at 84 trial locations

Overseen By UCB Cares

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: UCB Biopharma SRL

Disqualifiers: Multiple sclerosis, NMOSD, Active infection, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

104 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a medication called rozanolixizumab to see if it can safely and effectively treat adults with a nervous system condition called MOG-AD. The medication aims to reduce harmful antibodies that cause the disease.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

What makes the drug Rozanolixizumab unique for treating MOG-AD?

Rozanolixizumab is unique because it is specifically designed to target and reduce the activity of certain immune system components that contribute to autoimmune diseases, which may offer a novel approach for treating MOG-AD, a condition with limited standard treatment options. ^{1 2 3 4 5}

Research Team

UCB Cares

Principal Investigator

001 844 599 2273

Eligibility Criteria

This trial is for adults aged 18-89 with a confirmed diagnosis of MOG-AD, who have had at least one relapse in the past year and tested positive for MOG antibodies recently. They must be stable health-wise and not have other autoimmune diseases or primary immunodeficiency.

Inclusion Criteria

Participant must be clinically stable at the time of the Screening Visit and during the Screening Period

I am between 18 and 89 years old.

I have been diagnosed with MOG Antibody Disease.

Exclusion Criteria

I tested positive for aquaporin-4 antibodies.

Participant has a serum total IgG level ≤ 5.5g/L

I do not have any serious ongoing infections.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-Blind Treatment

Participants receive either rozanolixizumab or placebo in a double-blind manner

up to approximately 132 weeks

Open-Label Extension

Participants receive rozanolixizumab in an open-label manner

up to 52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Rozanolixizumab

Trial Overview The study tests Rozanolixizumab's effectiveness and safety against a placebo in treating MOG-AD. Participants will randomly receive either the actual drug or a placebo to compare outcomes.

Participant Groups

Group I: Rozanolixizumab Arm Experimental Treatment 1 Intervention

Participants randomized into this arm will receive rozanolixizumab at pre-specified timepoints.

Group II: Placebo Arm Placebo Group 1 Intervention

Participants randomized into this arm will receive placebo at pre-specified timepoints to maintain the blinding.

Rozanolixizumab is already approved in United States, European Union, Japan for the following indications:

Approved in United States as Rystiggo for:

• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Approved in European Union as Rystiggo for:

• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Approved in Japan as Rystiggo for:

• Generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

Who Is Running the Clinical Trial?

UCB Biopharma SRL

Lead Sponsor

Trials

118

Recruited

23,200+

Jean-Christophe Tellier

UCB Biopharma SRL

Chief Executive Officer since 2015

MD from University of Reims Champagne-Ardenne, Rheumatology specialization from University of Paris V, Executive business programs at Harvard and INSEAD

Dr. Iris Loew-Friedrich

UCB Biopharma SRL

Chief Medical Officer since 2014

MD from University of Leuven, PhD in Medical Sciences from University of Leuven

Findings from Research

Researchers developed new RORγt inverse agonists that effectively inhibit the production of pro-inflammatory Th17 cytokines, which are linked to autoimmune diseases, by designing compounds with improved potency and physicochemical properties.

Two specific compounds, identified as 10 and 33, demonstrated strong pharmacological activity in both biochemical assays and a rodent model, successfully reducing IL-17 cytokine production after oral administration at a dose of 15 mg/kg.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Synthesis and Biological Evaluation of New Triazolo- and Imidazolopyridine RORγt Inverse Agonists. Hintermann, S., Guntermann, C., Mattes, H., et al.[2018]

A new series of biaryl amides were developed as RORγt inhibitors, showing promising oral bioavailability and the ability to penetrate the central nervous system (CNS).

Compounds 9a and 9g exhibited strong in vivo efficacy in a mouse model of experimental autoimmune encephalomyelitis (EAE), demonstrating effective results with once daily oral dosing.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Discovery of Biaryl Amides as Potent, Orally Bioavailable, and CNS Penetrant RORγt Inhibitors. Wang, Y., Cai, W., Cheng, Y., et al.[2020]

A new compound, identified as an RORγt agonist, was developed through structure-based drug design and showed a favorable pharmacokinetic profile in a mouse tumor model.

Despite its promising design and pharmacokinetics, the compound did not demonstrate any significant efficacy in reducing tumor growth at the tested doses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Substituted benzyloxytricyclic compounds as retinoic acid-related orphan receptor gamma t (RORγt) agonists. Harikrishnan, LS., Gill, P., Kamau, MG., et al.[2023]

Researchers developed a new lead compound, MG 2778, as an inverse agonist for the RORγt receptor, which is important for treating immune diseases, and created a small library of modified compounds to test their effectiveness.

Among the tested compounds, 19a showed the highest activity in reducing RORγt activity at low micromolar concentrations, although all compounds were less potent than the reference compound ursolic acid, indicating potential for further development.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Targeting RORs nuclear receptors by novel synthetic steroidal inverse agonists for autoimmune disorders. Dal Prà, M., Carta, D., Szabadkai, G., et al.[2018]

A new series of tetrahydroisoquinoline derivatives were developed as potential oral treatments for autoimmune diseases driven by Th17 cells, with compound 15 showing promising results as an inverse agonist for the RORγt receptor.

Compound 15 demonstrated a strong ability to inhibit IL-17A production in mice, indicating its potential efficacy in treating Th17-driven autoimmune conditions, along with an improved pharmacokinetic profile for better absorption and effectiveness.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Discovery of orally efficacious RORγt inverse agonists. Part 2: Design, synthesis, and biological evaluation of novel tetrahydroisoquinoline derivatives. Kono, M., Oda, T., Tawada, M., et al.[2018]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Synthesis and Biological Evaluation of New Triazolo- and Imidazolopyridine RORγt Inverse Agonists. [2018]

2. United States pubmed.ncbi.nlm.nih.gov

Discovery of Biaryl Amides as Potent, Orally Bioavailable, and CNS Penetrant RORγt Inhibitors. [2020]

3. England pubmed.ncbi.nlm.nih.gov

Substituted benzyloxytricyclic compounds as retinoic acid-related orphan receptor gamma t (RORγt) agonists. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Targeting RORs nuclear receptors by novel synthetic steroidal inverse agonists for autoimmune disorders. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Discovery of orally efficacious RORγt inverse agonists. Part 2: Design, synthesis, and biological evaluation of novel tetrahydroisoquinoline derivatives. [2018]