Granted Patent B2

US 12,558,398 · App. 17/615,494 · Granted Feb 24, 2026

Inventors: Petra Duda (Charlestown, MA); Ramin Farzaneh-Far (Brookline, MA); Alonso Ricardo (Winchester, MA); Camil Sayegh (Belmont, MA); Nanqun Zhu (Lexington, MA); Douangsone D. Vadysirisack (Boston, MA); Olivier Boyer (Rouen, FR); Laurent Drouot (Blainville-Crevon, FR)

Assignee: UCB Holdings, Inc.

A61K38/12A61K35/16A61P21/00C07K16/2887G01N33/564A61M5/329C07K2317/24

Abstract

Claims (31)

1 . A method of treating Immune-Mediated Necrotizing Myopathy (IMNM), the method comprising administering a C5 inhibitor to a subject having IMNM.

2 . The method of claim 1 , wherein the subject has an IMNM subtype that is anti-signal recognition particle (Anti-SRP) subtype.

3 . The method of claim 1 , wherein the subject has an IMNM subtype that is Anti-HMG-CoA reductase (Anti-HMGCR) subtype or antibody negative subtype.

4 . The method of claim 1 , wherein one or more IMNM-related symptoms are reduced or eliminated in the subject and the one or more IMNM-related symptoms comprise one or more of muscle weakness, chronic muscle inflammation, muscle atrophy and fatty replacement, elevated serum creatine kinase (CK), dysphagia, neck weakness, myalgia, muscle fiber necrosis, and interstitial lung disease.

5 . The method of claim 1 , wherein the C5 inhibitor is administered in combination with a therapeutic agent and/or therapeutic regimen that comprises one or more of rituximab, a steroid, an immunosuppressive therapy (IST), and an intravenous immunoglobulin therapy.

6 . The method of claim 1 , wherein the C5 inhibitor comprises a peptide, optionally wherein the peptide is a macrocyclic peptide.

7 . The method of claim 1 , wherein the C5 inhibitor is zilucoplan.

8 . The method of claim 1 , wherein CK levels of the subject are reduced after 8 weeks of complement inhibitor treatment.

9 . The method of claim 1 , wherein improvement in a secondary endpoint is observed after treatment with the C5 inhibitor, wherein the secondary endpoint comprises an improved score from an assessment comprising one or more of Triple Timed Up and Go (3TUG) Testing, Abbreviated Manual Muscle Testing in 8 Groups (MMT8), Physician Global Activity Visual Analogue Scale (VAS) assessment, and Patent Global Activity VAS assessment.

10 . The method of claim 7 , wherein zilucoplan is administered at a dose of from about 0.1 mg/kg to about 0.3 mg/kg.

11 . The method of claim 7 , wherein zilucoplan is administered daily.

12 . The method of claim 7 , wherein zilucoplan administration comprises self-administration.

13 . The method of claim 7 , wherein zilucoplan is administered by subcutaneous injection, optionally wherein the subcutaneous injection comprises the use of a prefilled syringe, the use of a self-administration device, and/or the use of a 29-gauge needle.

14 . The method of claim 13 , wherein the subcutaneous injection comprises the use of self-administration device, wherein the self-administration device comprises an auto injection device comprising a prefilled glass syringe comprising a fill volume of from about 0.2 ml to about 2 ml.

15 . The method of claim 14 , wherein zilucoplan is administered as a pharmaceutical composition comprising an aqueous solution.

16 . The method of claim 7 , wherein the subject is screened prior to zilucoplan administration, wherein the screening comprises:

assessment of CK level;

verification that subject age is between 18 and 75 years old;

assessment of anti-HMGCR or anti-SRP antibody levels;

manual muscle testing in at least one proximal limb muscle group;

assessment of previous IMNM diagnosis;

assessment of subject corticosteroid treatment history;

assessment of subject immunosuppressive therapy treatment history; and/or

a serum pregnancy test and/or a urine pregnancy test.

17 . The method of claim 1 , wherein the subject is evaluated or monitored for an IMNM characteristic, wherein the IMNM characteristic comprises an assessment made by one or more of CK level analysis, 3TUG test, Proximal MMT, Physician Global Activity VAS assessment, Patient Global Activity VAS assessment, HAQ, MDAAT score analysis, ACR/EULAR Response Criteria assessment, and FACIT-Fatigue scale assessment.

18 . The method of claim 17 , wherein treated subject CK levels are reduced at or before 8 weeks of treatment.

19 . The method of claim 1 , wherein the subject is evaluated for improvement in one or more of muscle strength, muscle function, and muscle tissue necrosis.

20 . The method of claim 19 , wherein the C5 inhibitor is zilucoplan and the neuromuscular inflammatory indication is IMNM.

21 . The method of claim 1 , the method comprising:

administering zilucoplan to the subject; and

evaluating subject muscle strength, wherein subject muscle strength is evaluated by measuring contraction of a subject muscle.

Assignments (6)

Priority Claims (1)

Continuity (5)

References Cited (352)