IP Library › Granted Patent US 12661400
Granted Patent B2
US 12661400 · App. 18/431,294 · Granted Jun 23, 2026

Methods of treating myopathies with anti-pro/latent myostatin antibodies

Inventors: Michelle Straub (Yarmouth, ME); Dong Yun Lee (Boston, MA); William K. McConaughy (Boston, MA); Katherine Jane Turner (Acton, MA); Nagesh K. Mahanthappa (Cambridge, MA); Justin W. Jackson (Cambridge, MA)
Assignee: Scholar Rock, Inc.
A61K39/3955A61P19/08A61P21/00A61P21/06A61K2039/505C07K14/475C07K16/22C07K2317/21C07K2317/30C07K2317/33C07K2317/76C07K2317/92C07K2317/94
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Quick Facts
Patent No.
US 12661400
App. No.
18/431,294
Granted
Jun 23, 2026
Kind
B2
Abstract

Aspects of the present disclosure relate to antibodies that specifically bind proMyostatin and/or latent Myostatin and uses thereof.

Claims (55)

1 . A method of treating a subject having, or at risk of developing, Duchenne's muscular dystrophy, facioscapulohumeral muscular dystrophy, or Becker's muscular dystrophy, the method comprising administering to the subject an effective amount of an antibody or antigen-binding fragment thereof that specifically binds to pro/latent myostatin, and wherein the antibody or antigen-binding fragment thereof comprises six complementarity determining regions (CDRs): CDRH1, CDRH2, CDRH3, CDRL1, CDRL2, and CDRL3, wherein:

a) CDRH1 comprises the amino acid sequence of SEQ ID NO: 1,

CDRH2 comprises the amino acid sequence of SEQ ID NO: 4,

CDRH3 comprises the amino acid sequence of SEQ ID NO: 10,

CDRL1 comprises the amino acid sequence of SEQ ID NO: 12,

CDRL2 comprises the amino acid sequence of SEQ ID NO: 18, and

CDRL3 comprises the amino acid sequence of SEQ ID NO: 22, wherein the CDR sequences are numbered according to Kabat numbering system; or

b) CDRH1 comprises the amino acid sequence of SEQ ID NO: 2,

CDRH2 comprises the amino acid sequence of SEQ ID NO: 5,

CDRH3 comprises the amino acid sequence of SEQ ID NO: 10,

CDRL1 comprises the amino acid sequence of SEQ ID NO: 13,

CDRL2 comprises the amino acid sequence of SEQ ID NO: 19, and

CDRL3 consists of the amino acid sequence of SEQ ID NO: 22, wherein the CDR sequences are numbered according to IMGT numbering system.

2 . The method of claim 1 , wherein the effective amount is sufficient to prevent muscle loss or muscle atrophy.

3 . The method of claim 1 , wherein the effective amount is sufficient to increase muscle mass and/or muscle function.

4 . The method of claim 1 , wherein the antibody or antigen-binding fragment binds to pro/latent myostatin with a higher affinity at a neutral pH than at an acidic pH.

5 . The method of claim 1 , wherein the antibody or antigen-binding fragment inhibits activation of mature myostatin by tolloid protease with an IC50 of less than 1 μM.

6 . The method of claim 1 , wherein the antibody or antigen-binding fragment comprises an IgG 4 constant domain.

7 . The method of claim 1 , wherein the antibody or antigen-binding fragment binds neonatal Fc receptor (FcRn) with a lower affinity at neutral pH as compared to an acidic pH.

8 . A method of treating a subject having, or at risk of developing, amyotrophic lateral sclerosis (ALS), the method comprising administering to the subject an effective amount of an antibody or antigen-binding fragment thereof that specifically binds to pro/latent myostatin, and wherein the antibody or antigen-binding fragment thereof comprises six complementarity determining regions (CDRs): CDRH1, CDRH2, CDRH3, CDRL1, CDRL2, and CDRL3, wherein:

a) CDRH1 comprises the amino acid sequence of SEQ ID NO: 1,

CDRH2 comprises the amino acid sequence of SEQ ID NO: 4,

CDRH3 comprises the amino acid sequence of SEQ ID NO: 10,

CDRL1 comprises the amino acid sequence of SEQ ID NO: 12,

CDRL2 comprises the amino acid sequence of SEQ ID NO: 18, and

CDRL3 comprises the amino acid sequence of SEQ ID NO: 22, wherein the CDR sequences are numbered according to Kabat numbering system; or

b) CDRH1 comprises the amino acid sequence of SEQ ID NO: 2,

CDRH2 comprises the amino acid sequence of SEQ ID NO: 5,

CDRH3 comprises the amino acid sequence of SEQ ID NO: 10,

CDRL1 comprises the amino acid sequence of SEQ ID NO: 13,

CDRL2 comprises the amino acid sequence of SEQ ID NO: 19, and

CDRL3 consists of the amino acid sequence of SEQ ID NO: 22, wherein the CDR sequences are numbered according to IMGT numbering system.

9 . The method of claim 8 , wherein the effective amount is sufficient to prevent muscle loss or muscle atrophy.

10 . The method of claim 8 , wherein the effective amount is sufficient to increase muscle mass and/or muscle function.

11 . The method of claim 8 , wherein the antibody or antigen-binding fragment binds to pro/latent myostatin with a higher affinity at a neutral pH than at an acidic pH.

12 . The method of claim 8 , wherein the antibody or antigen-binding fragment inhibits activation of mature myostatin by tolloid protease with an IC50 of less than 1 μM.

13 . The method of claim 8 , wherein the antibody or antigen-binding fragment comprises an IgG 4 constant domain.

14 . The method of claim 8 , wherein the antibody or antigen-binding fragment binds FcRn with a lower affinity at neutral pH as compared to an acidic pH.

15 . A method of treating a subject having osteogenesis imperfecta, the method comprising administering to the subject an effective amount of an antibody or antigen-binding fragment thereof that specifically binds to pro/latent myostatin, and wherein the antibody or antigen-binding fragment thereof comprises six complementarity determining regions (CDRs): CDRH1, CDRH2, CDRH3, CDRL1, CDRL2, and CDRL3, wherein:

a) CDRH1 comprises the amino acid sequence of SEQ ID NO: 1,

CDRH2 comprises the amino acid sequence of SEQ ID NO: 4,

CDRH3 comprises the amino acid sequence of SEQ ID NO: 10,

CDRL1 comprises the amino acid sequence of SEQ ID NO: 12,

CDRL2 comprises the amino acid sequence of SEQ ID NO: 18, and

CDRL3 comprises the amino acid sequence of SEQ ID NO: 22, wherein the CDR sequences are numbered according to Kabat numbering system; or

b) CDRH1 comprises the amino acid sequence of SEQ ID NO: 2,

CDRH2 comprises the amino acid sequence of SEQ ID NO: 5,

CDRH3 comprises the amino acid sequence of SEQ ID NO: 10,

CDRL1 comprises the amino acid sequence of SEQ ID NO: 13,

CDRL2 comprises the amino acid sequence of SEQ ID NO: 19, and

CDRL3 consists of the amino acid sequence of SEQ ID NO: 22, wherein the CDR sequences are numbered according to IMGT numbering system.

16 . The method of claim 15 , wherein the antibody or antigen-binding fragment binds to pro/latent myostatin with a higher affinity at a neutral pH than at an acidic pH.

17 . The method of claim 15 , wherein the antibody or antigen-binding fragment inhibits activation of mature myostatin by tolloid protease with an IC50 of less than 1 μM.

18 . The method of claim 15 , wherein the antibody or antigen-binding fragment comprises an IgG 4 constant domain.

19 . The method of claim 15 , wherein the antibody or antigen-binding fragment binds FcRn with a lower affinity at neutral pH as compared to an acidic pH.