IP Library › Granted Patent US 12,661,400
Granted Patent B2
US 12,661,400 · 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 12,661,400
App. No.
18/431,294
Filed
Feb 2, 2024
Granted
Jun 23, 2026
Kind
B2
Art Unit
1674
USPC
435/7.1
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.

Assignments (3)
SECURITY INTEREST Recorded Mar 3, 2026
From: SCHOLAR ROCK, INC.
To: LSI FINANCING LLC, AS ADMINISTRATIVE AGENT
Reel/Frame 075015/0854 →
CHANGE OF ASSIGNEE ADDRESS Recorded Oct 31, 2025
From: SCHOLAR ROCK, INC.
To: SCHOLAR ROCK, INC.
Reel/Frame 073225/0055 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 17, 2025
From: STRAUB, MICHELLE; LEE, DONG YUN; MCCONAUGHY, WILLIAM K.; TURNER, KATHERINE JANE; MAHANTHAPPA, NAGESH K.; JACKSON, JUSTIN W.
To: SCHOLAR ROCK, INC.
Reel/Frame 072286/0487 →
Continuity (8)
Continuation 17446897 · Sep 3, 2021
Continuation 16374854 · Apr 4, 2019
Continuation 15524651
Provisional Application 62219094 · Sep 15, 2015
Provisional Application 62187348 · Jul 1, 2015
Provisional Application 62100361 · Jan 6, 2015
Provisional Application 62076230 · Nov 6, 2014
Related Publication 20240374718A1 · Nov 14, 2024
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