Evaluate the Safety, Tolerability, Pharmacodynamics and Efficacy of CNP-106 in Subjects With Myasthenia Gravis

A Phase 1b/2a Double Blind, Placebo Controlled Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Efficacy of CNP-106 in Subjects Ages 18-75 With Generalized Myasthenia Gravis

Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106.

Study Overview

• Status: Active, not recruiting
• Conditions: Myasthenia Gravis; Generalized Myasthenia; AChR Myasthenia Gravis; MuSK MG
• Intervention / Treatment: Drug: CNP-106; Other: Placebo

Detailed Description

This is a Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106. The clinical study lasts 222-days (up to 42 days for Screening, 180 Study Days). Subjects ages 18-75 with generalized myasthenia gravis (MG) will be screened up to 42 days prior to enrollment into the clinical study.

• Study Type: Interventional
• Enrollment (Estimated): 54
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • Barrow Neurological Institute
  • California
    • Brea, California, United States, 92835
      • Infusion for Health
    • Orange, California, United States, 92868
      • University of California, Irvine
    • Sacramento, California, United States, 95817
      • University of California, Davis
  • Connecticut
    • New Haven, Connecticut, United States, 06516
      • Yale University
  • Florida
    • Miami, Florida, United States, 33173
      • Quantix Research, LLC
    • Miami, Florida, United States, 33173
      • Atlantis Research
    • Tampa, Florida, United States, 33612
      • University of South Florida
  • Illinois
    • Chicago, Illinois, United States, 60608
      • Insight Hospital and Medical Center
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center
  • Michigan
    • Dearborn, Michigan, United States, 48126
      • Insight Research Institute, Dearborn
  • Missouri
    • Columbia, Missouri, United States, 65211
      • University of Missouri, NextGen Precision Health
  • Ohio
    • Colombus, Ohio, United States, 43221
      • Ohio State University Wexner Medical Center
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Texas
    • Houston, Texas, United States, 77030
      • Nerve And Muscle Center Of Texas
    • Houston, Texas, United States, 77054
      • Prolato Clinical Research Center
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Subjects who are willing and able to provide Institutional Review Board (IRB) approved written informed consent and privacy language as per national regulations.
2. Men and non-pregnant women, ages 18-75 years inclusive.

3. Female subjects of childbearing potential must agree not to become pregnant during the clinical study, have a negative pregnancy test at Screening Visit, and agree to one of the following:

   • Use two highly effective forms of birth control starting at initial screening and continuing throughout the study duration.
   • Practice abstinence starting at initial screening and continuing throughout the study duration.
4. Subjects with a Myasthenia Gravis Foundation of America Clinical Classification Class II-IV.
5. Subjects positive for anti-AChR antibodies by radioimmunoassay (RIA) (LabCorp Central Laboratory Services Inc.).
6. Subjects with MG-ADL Score ≥6 at Screening and Baseline Visit with ≥50% of the score derived from non-ocular symptoms.
7. Subjects with QMG Score ≥11 at Screening and Baseline Visit.
8. For subjects on any medication used to treat the symptoms of MG (e.g. Corticosteroids, pyridostigmine), subjects must be on a stable dose for a minimum of 90 days prior to enrollment and must agree not to increase their dose throughout the study duration unless reviewed and approved by the medical monitor and the site investigator.
9. Female subjects who agree to not breastfeed starting at initial screening and throughout the study duration.
10. Female subjects who agree to not donate ova starting at initial screening and throughout the study duration.
11. Male subjects with a spouse or partner of childbearing potential, who themselves and their spouse or partner agree to practice an effective form of birth control as discussed with the study doctor or study staff starting at Screening and throughout the study duration.

Exclusion Criteria:

1. Subjects with a Myasthenia Gravis Foundation of America Clinical Classification Class I or V.
2. Subjects with a history of cerebrovascular accident in the past 12 months.
3. Subjects with MG-ADL Score <6 at Screen or Subjects with MG-ADL Score ≥6 at Screen with ˂50% of the score derived from non-ocular symptoms.
4. Subjects with QMG Score <11 at Screen.

5. Subjects may enter the study following the washout periods for medications as described below prior to screening and refrain from usage throughout the study:

   • Tacrolimus within 6 months
   • Azathioprine, cyclosporine, methotrexate, or mycophenolate mofetil within 90 days
   • Anti-FcRn therapies:
   • Cyclic Anti-FcRns (e.g., efgartigimod alfa, rozanolixizumab) within 50 days
   • Chronic Anti-FcRns (e.g., nipocalimab) within 80 days;
   • C5 complement inhibitor (e.g., eculizumab) within 90 days
   • Anti-CD20 (e.g., rituximab) within 6 months;
   • Other immunomodulatory drugs will be at the discretion of the medical monitor and study site investigator.
6. Subjects who have used immunoglobulins given SC or IV (SCIg or IVIg) or plasmapheresis/plasma exchange (PE) within 4 weeks before Screening.
7. Subjects who have had thymectomy or any other thymic surgery performed within 12 months prior to Screening.
8. Subjects with untreated thymic malignancy, carcinoma, or thymoma.

9. Subjects with a history of untreated tuberculosis, a positive PPD skin test, or a positive QuantiFERON test.

   • Subjects with documented evidence of satisfactory TB treatment can be considered after review with the medical monitor and documentation of treatment course.

10. Subjects who have received administration of any live vaccine (other than intranasal Influenza) within 28 days or subunit vaccine within 14 days prior to Screening or are planning to receive any vaccination throughout the study duration.
11. Subjects who have received any COVID-19 vaccine within 14 days prior to Screening. Subjects who have received the first dose of any COVID-19 vaccine may not screen for the study until 14 days following their last dose of the vaccine if applicable.
12. Subjects with laboratory test results at Screening or prior to study dosing that are outside the normal limits and considered by the investigator to be clinically significant. Note: Clinically significant laboratory test results at screening that are related to the condition (MG) are acceptable if all inclusion and no other exclusion criteria are met.
13. Subjects with positive test results for hepatitis B surface antigen (HbsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV) antigen/antibody as determined at Screening.
14. Subjects with a history of or currently active immune disorders other than MG (including autoimmune disease) unless the condition, after discussion with the medical monitor and study site investigator, has been deemed to be acceptable for the subject's participation in this clinical study.
15. Subjects with a history of or current active diseases other than myasthenia gravis requiring immunosuppressive drugs (including azathioprine, prednisone, prednisolone, budesonide, cyclosporine, tacrolimus, methotrexate, or mycophenolate mofetil) unless the condition, after discussion with the medical monitor and site investigator, has been deemed to be acceptable for the subject's participation in this clinical study.
16. Subjects with a clinical history of significant cardiovascular disease as determined by the Investigator.
17. Subjects with a complication or medical history of malignancy within the past 5 years which, in the investigator's opinion, makes the subject unsuitable for study participation.
18. Subjects with a history of mast cell activation disease.
19. Subjects who, in the investigator's opinion, will be unable to adhere to study procedures.
20. Subjects who have received an investigational therapy other than CNP-106 within 28 days or 5 half-lives, whichever is longer, prior to Screening.
21. Subjects with any known active condition which, in the investigator's opinion, makes the subject unsuitable for study participation.
22. Known sensitivity to any components of CNP-106 (PLGA, sucrose, mannitol, or sodium citrate).

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CNP-106; 200 mL intravenous infusion on Day 1 and Day 8: CNP-106	Drug: CNP-106; CNP-106 is comprised of an antigenic AChR Peptide Pool (~1 μg of each AChRα and AChRε peptide comprising AChR Peptide Pool Drug Substance per mg particles) dispersed within a negatively charged (-30 to -60 mV) polymer matrix of PLGA (Poly (DL-lactide-co-glycolide, 50:50 acid-end group)) particles (400-800 nm in size).
Placebo Comparator: Placebo; CNP-106 Placebo	Other: Placebo; CNP-106 Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs),	Frequency tables will be presented by treatment group for all AEs and SAEs by System Organ Class (SOC) and Preferred Term (PT). Frequency tables will also be produced by treatment group for AEs leading to COUR Pharmaceuticals Development Company, Inc. Confidential CNP-106-5.001 Protocol; IND 28774 Page 53 of 65 discontinuation from IP and study, by severity, and by causality. No formal statistical testing will be done.	Through study day 180

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in antigen specific CD4+ and CD8+ T cell levels in PBMC at Day 60, 90, and 180.	The mean change from Baseline to the endpoint within CNP-106 and Placebo treatment groups will be analyzed using ANOVA.	Through study day 180
Change from baseline in activated antigen specific CD4+ and CD8+ T cell levels in PBMC at Day 60, 90, and 180.	The mean change from Baseline to the endpoint within CNP-106 and Placebo treatment groups will be analyzed using ANOVA.	Through study day 180

Collaborators and Investigators

• Sponsor: COUR Pharmaceutical Development Company, Inc.
• Investigators: Study Chair: Paul Peloso, MD, COUR Pharmaceutical

Publications and helpful links

General Publications

• G Brew S, Frey M, P McCarthy D, Elhofy A, Nowak RJ. Antigen-specific immune therapy (CNP-106) for treatment of generalised myasthenia gravis: rationale and design of first-in-human randomised controlled trial. BMJ Neurol Open. 2024 Dec 18;6(2):e000836. doi: 10.1136/bmjno-2024-000836. eCollection 2024.

Study record dates

Study Major Dates

• Study Start (Actual): May 30, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): August 1, 2026

Study Registration Dates

• First Submitted: October 24, 2023
• First Submitted That Met QC Criteria: October 24, 2023
• First Posted (Actual): October 30, 2023

Study Record Updates

• Last Update Posted (Actual): June 9, 2026
• Last Update Submitted That Met QC Criteria: June 5, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Myasthenia Gravis
• Other Study ID Numbers: CNP-106-5.001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No