IP Library Granted Patent US 8,791,243
Granted Patent B2
US 8,791,243 · App. 12/811,049 · Granted Jul 29, 2014

Treatment and prophylaxis of amyloidosis

Inventors: Dale B. Schenk (Burlingame, CA); Peter A. Seubert (San Francisco, CA); Jonathan Wall (Knoxville, TN); Jose Saldanha (Middlesex, GB)
Assignee: Onclave Therapeutics Limited
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Quick Facts
Patent No.
US 8,791,243
App. No.
12/811,049
Granted
Jul 29, 2014
Kind
B2
Abstract

Methods useful for effecting prophylaxis or treatment of amyloidosis, including AA Amyloidosis and AL amyloidosis, by administering peptides comprising neoepitopes, such as AA fragments from a C-terminal region of AA, and antibodies specific for neoepitopes of aggregated amyloid proteins, for example, antibodies specific for the C-terminal region of AA fibrils. Antibodies for inhibition of formation and/or increasing clearance of amyloid deposits in a patient thus effecting prophylaxis or treating amyloid disease.

Claims (17)

1. A method of therapeutically treating a human patient having AL amyloidosis characterized by the presence of amyloid light chain-type (AL) protein fibrils comprising administering to the patient an effective dosage of an antibody or fragment thereof that specifically binds to an epitope comprising X 1 EDX 2 in an aggregated AL protein, wherein X 1 is H, T, S, P, A, L, C, Q, R, or E, and wherein X 2 is T, S, E, R, I, F, A, G, M, L, N, or P, to thereby treat AL amyloidosis.

2. The method of claim 1 , wherein the AL amyloidosis is associated with peripheral or autonomic neuropathy, carpal tunnel syndrome, macroglossia, restrictive cardiomyopathy, arthropathy of large joints, immune dyscrasia, myeloma, monoclonal gammopathy, occult dyscrasia, or a chronic inflammatory disease.

3. The method of claim 2 , wherein the AL amyloidosis is associated with a dyscrasia of the B lymphocyte lineage.

4. The method of claim 3 , wherein the dyscrasia is a malignancy.

5. The method of claim 4 , wherein the malignancy is multiple myeloma.

6. The method of claim 1 , wherein the antibody or fragment thereof specifically binds to an epitope comprising an amino acid sequence AEDS (SEQ ID NO: 13) in an aggregated AL protein.

7. The method of claim 1 , wherein the antibody or fragment thereof is a chimeric antibody, a human antibody, a humanized antibody, a single chain antibody, a tetrameric antibody, a tetravalent antibody, a multispecific antibody, a domain-specific antibody, a domain-deleted antibody, a fusion protein, a Fab fragment, a Fab' fragment, a F(ab') 2 fragment, a Fv fragment, or a ScFv fragment.

8. The method of claim 7 , wherein the antibody or fragment thereof is a chimeric or humanized antibody or fragment thereof.

9. The method of claim 1 , wherein the antibody or fragment thereof binds to monomeric AL protein with an affinity of less than about 10 7 M −1 .

10. A method of therapeutically treating a human patient having AL amyloidosis characterized by the presence of amyloid light chain-type (AL) protein fibrils comprising administering to the patient an effective dosage of an antibody raised to a peptide comprising GHEDT (SEQ ID NO: 3), or a fragment of the antibody, to thereby treat AL amyloidosis.

11. The method of claim 10 , wherein the antibody or fragment thereof is a chimeric antibody, a human antibody, a humanized antibody, a single chain antibody, a tetrameric antibody, a tetravalent antibody, a multispecific antibody, a domain-specific antibody, a domain-deleted antibody, a fusion protein, a Fab fragment, a Fab' fragment, a F(ab') 2 fragment, a Fv fragment, or a ScFv fragment.

12. The method of claim 11 , wherein the antibody or fragment thereof is a chimeric or humanized antibody or fragment thereof.

13. The method of claim 10 , wherein the antibody or fragment thereof binds to monomeric AL protein with an affinity of less than about 10 7 M −1 .

14. A method of therapeutically treating a human patient having AL amyloidosis characterized by the presence of amyloid light chain-type (AL) protein fibrils comprising administering to the patient an effective dosage of an antibody comprising a light chain variable region comprising three complementarity determining regions set forth as SEQ ID NOs: 168, 169, and 170, and a heavy chain variable region comprising three complementarity regions set forth as SEQ ID NOs: 171, 172, and 173.

15. A method of therapeutically treating a human patient having AL amyloidosis characterized by the presence of amyloid light chain-type (AL) protein fibrils comprising administering to the patient an effective dosage of an antibody, which is a humanized version of murine monoclonal antibody 2A4 produced by ATCC Accession Number PTA-9662.

16. A method of reducing risk or delaying onset of AL amyloidosis characterized by the presence of amyloid light chain-type protein fibrils in a human patient at risk of AL amyloidosis comprising administering to the patient an effective dosage of an antibody comprising a light chain variable region comprising three complementarity determining regions set forth as SEQ ID NOs: 168, 169, and 170, and a heavy chain variable region comprising three complementarity regions set forth as SEQ ID NOs: 171, 172, and 173.

17. A method of reducing risk or delaying onset of AL amyloidosis characterized by the presence of amyloid light chain-type protein fibrils in a human patient at risk of AL amyloidosis comprising administering to the patient an effective dosage of an antibody, which is a humanized version of murine monoclonal antibody 2A4 produced by ATCC Accession Number PTA-9662.

Assignments (6)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 13, 2018
From: PROTHENA THERAPEUTICS LIMITED
To: PROTHENA BIOSCIENCES LIMITED
Reel/Frame 046081/0329 →
CHANGE OF NAME Recorded Oct 26, 2015
From: ONCLAVE THERAPEUTICS
To: PROTHENA THERAPEUTICS LIMITED
Reel/Frame 036884/0947 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 28, 2012
From: ELAN PHARMACEUTICALS, INC.
To: ELAN PHARMA INTERNATIONAL LIMITED
Reel/Frame 028863/0177 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 28, 2012
From: ELAN PHARMA INTERNATIONAL LIMITED
To: ELAN SCIENCE ONE LIMITED
Reel/Frame 028863/0226 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 28, 2012
From: ELAN SCIENCE ONE LIMITED
To: NEOTOPE BIOSCIENCES LIMITED
Reel/Frame 028863/0251 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 28, 2012
From: NEOTOPE BIOSCIENCES LIMITED
To: ONCLAVE THERAPEUTICS
Reel/Frame 028863/0295 →
Continuity (3)
Provisional Application 61007544 · Dec 28, 2007
Provisional Application 61095932 · Sep 10, 2008
Related Publication 20110038790A1 · Feb 17, 2011