# US 11,440,968 B2 · Humanized antibody against O-acetylated GD2 ganglioside (OAcGD2)

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- Number: US 11,440,968 B2
- Document ID: US11440968B2
- Status: Granted
- Kind: B2
- Application number: 16/317,972
- Filed: 2017-07-17
- Published: 2019-08-01
- Granted: 2022-09-13
- Earliest claimed: 2016-07-15
- Assignees: Ogd2 Pharma
- Applicants: Ogd2 Pharma
- Inventors: Mickaël Terme; Jean-Marc Le Doussal; Mylene Dorvillius; Brigitte Assouline
- Web page: https://patentgrep.com/patent/US11440968B2

## CPC

- C07K 16/3084: {against tumour-associated gangliosides}
- A61P 11/00: Drugs for disorders of the respiratory system
- A61P 13/12: of the kidneys
- A61P 15/00: Drugs for genital or sexual disorders (for disorders of sex hormones A61P5/24); Contraceptives
- A61P 17/00: Drugs for dermatological disorders
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61P 25/00: Drugs for disorders of the nervous system
- A61P 27/02: Ophthalmic agents
- A61P 35/00: Antineoplastic agents
- A61P 35/02: specific for leukemia
- G01N 33/5756: {involving tumour-associated glycolinkage [TAG]}
- C07K 2317/24: containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
- C07K 2317/56: variable (Fv) region, i.e. VH and/or VL
- C07K 2317/565: Complementarity determining region [CDR]
- C07K 2317/622: Single chain antibody (scFv)
- C07K 2317/73: Inducing cell death, e.g. apoptosis, necrosis or inhibition of cell proliferation
- C07K 2317/92: Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- G01N: INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES (measuring or testing apparatus or processes other than immunoassay, involving enzymes or microorganisms C12M, C12Q)

## Abstract

Disclosed is an antibody, functional fragment, and derivative thereof, which binds specifically to the OAcGD2 ganglioside, the antibody including i) a humanized light chain variable region (VL) polypeptide having the amino acid sequence SEQ id no 112; and ii) a humanized heavy chain variable region (VH) having the amino acid sequence SEQ id no 76; and its use in diagnostics and therapy.

## Claims

### Claim 1

An antibody, functional fragment, or derivative thereof, which binds specifically to the O-acetylated GD2 (OAcGD2) ganglioside, said antibody, functional fragment or derivative thereof comprising:
a) a humanized light chain variable region (VL) polypeptide having the amino acid sequence SEQ ID NO: 112; and
b) a humanized heavy chain variable region (VH) having the amino acid sequence SEQ ID NO: 76.

### Claim 2 (depends on 1)

The antibody of claim 1, wherein said antibody, functional fragment or derivative thereof comprises:
the light chain variable region (VL) having the amino acid sequence selected in the group comprising VL30BH (SEQ ID NO: 134) and VL28Bs01/A2 (SEQ ID NO: 135), and/or
the heavy chain variable region (VH) having the amino acid sequence selected in the group comprising VH72Bmax (SEQ ID NO: 131), VH49B (SEQ ID NO: 132), and VH49Bmax (SEQ ID NO: 133).

### Claim 3 (depends on 1)

The antibody of claim 1, wherein said antibody, functional fragment or derivative thereof comprises:
the light chain variable region (VL) having the amino acid sequence selected in the group comprising VL1 (SEQ ID NO: 8), VL2 (SEQ ID NO: 9), VL3 (SEQ ID NO: 10), VL4 (SEQ ID NO: 11), VL28BH (SEQ ID NO: 140), VL30BH (SEQ ID NO: 134) and VL28Bs01/A2 (SEQ ID NO: 135) and/or
the heavy chain variable region (VH) having the amino acid sequence selected in the group comprising VH1 (SEQ ID NO: 3), VH2 (SEQ ID NO: 4), and VH3 (SEQ ID NO: 5), VH72BCDR (SEQ ID NO: 136), VH72BH (SEQ ID NO: 137), VH49BCDR (SEQ ID NO: 138), VH49BH (SEQ ID NO: 139), VH72Bmax (SEQ ID NO: 131), VH49B (SEQ ID NO: 132); and VH49Bmax (SEQ ID NO: 133).

### Claim 4 (depends on 1)

The antibody of claim 1, wherein said antibody, functional fragment or derivative thereof, which binds specifically to the OAcGD2 ganglioside presents a Kd of less than 5×10−6M for said ganglioside at 25° C.

### Claim 5 (depends on 1)

The antibody of claim 1, wherein said antibody, functional fragment or derivative thereof, which binds specifically to the OAcGD2 ganglioside presents a binding affinity (Kd) of more than 5×10−5M at 25° C. for GD2 ganglioside.

### Claim 6 (depends on 1)

The antibody of claim 1, wherein the antibody functional fragments or derivatives thereof comprises at least both of said heavy and light chains variable regions.

### Claim 7 (depends on 1)

The antibody of claim 1, wherein the antibody functional fragments or derivatives thereof are the membrane anchored single-chain variable part of a chimeric antigen receptor (CAR).

### Claim 8 (depends on 7)

The antibody of claim 7, wherein said CAR comprises an ectodomain, a transmembrane domain and an endodomain.

### Claim 9 (depends on 1)

The antibody of claim 1, wherein said antibody is an immunoconjugate.

### Claim 10 (depends on 1)

The antibody of claim 1, wherein said functional fragments is selected in the group comprising ScFv fragment, Fab fragment, Fab′ fragment, F(ab′)2 fragment, Facb fragment, Fv and Fd fragments.

### Claim 11 (depends on 1)

A pharmaceutical composition comprising at least one antibody, functional fragment or derivative thereof according to claim 1, and a pharmaceutically acceptable carrier.

### Claim 12 (depends on 11)

The pharmaceutical composition as defined in claim 11 for use in a method of preventing and/or treating cancer expressing the OAcGD2 ganglioside in a subject.

### Claim 13 (depends on 12)

The pharmaceutical composition of claim 12, wherein said subject is a mammal.

### Claim 14 (depends on 12)

The pharmaceutical composition of claim 12 wherein said cancer expressing the OAcGD2 ganglioside is selected in the group comprising neuroblastoma, glioma, retinoblastoma, Ewing's family of tumors, sarcoma, small cell lung cancer, breast cancer, melanoma, metastatic renal carcinoma, head and neck cancer and hematological cancer.

### Claim 15 (depends on 1)

A method for diagnosing a cancer expressing OAcGD2 ganglioside in a subject comprising the step of contacting a biological sample of said subject with at least one antibody, functional fragment or derivative thereof as defined in claim 1, for determining an OAcGD2 ganglioside expression level in said biological sample, wherein a detectable OAcGD2 ganglioside expression level is indicative of such a cancer.

### Claim 16 (depends on 1)

A kit for diagnosing a cancer expressing the OAcGD2 ganglioside in a subject, which comprises at least one antibody, functional fragment or derivative thereof as defined in claim 1.

### Claim 17 (depends on 1)

The antibody of claim 1, wherein said antibody, functional fragment or derivative thereof, which binds specifically to the OAcGD2 ganglioside presents a Kd of equal to or less than 5×10−7 M, for said ganglioside at 25° C.

### Claim 18 (depends on 1)

The antibody of claim 1, wherein said antibody, functional fragment or derivative thereof, which binds specifically to the OAcGD2 ganglioside presents a Kd of equal to or less than 5×10−8M, for said ganglioside at 25° C.

### Claim 19 (depends on 1)

The antibody of claim 1, wherein said antibody, functional fragment or derivative thereof, which binds specifically to the OAcGD2 ganglioside presents a binding affinity (Kd) of more than 10−5 M at 25° C. for GD2 ganglioside.

### Claim 20 (depends on 13)

The pharmaceutical composition of claim 13, wherein the subject is a human.

### Claim 21 (depends on 1)

A method for treating a cancer expressing OAcGD2 in a subject in need thereof comprising administering to said subject an effective amount of at least one antibody, functional fragment or derivative thereof according to claim 1.