# US 2021/0333281 A1 · Biomarkers for a therapy comprising a sorafenib compound

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- Number: US 2021/0333281 A1
- Document ID: US20210333281A1
- Status: Published application
- Kind: A1
- Application number: 17/282,218
- Filed: 2019-10-02
- Published: 2021-10-28
- Earliest claimed: 2018-10-05
- Assignees: Eisai R&D Management Co., Ltd.
- Applicants: Eisai R&D Management Co., Ltd.
- Inventors: Yasuhiro Funahashi; Yukinori Minoshima; Michio Kanekiyo; Saori Miyano; Taisuke Hoshi
- Web page: https://patentgrep.com/patent/US20210333281A1

## CPC

- G01N 33/575: for cancer
- G01N 33/57525: of the liver or pancreas
- A61K 31/47: Quinolines; Isoquinolines
- A61P 35/00: Antineoplastic agents
- C12Q 1/6886: for cancer (immunoassay for cancer G01N33/575)
- C12Q 2600/106: Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
- C12Q 2600/158: Expression markers
- G01N 2333/50: Fibroblast growth factors [FGF]
- 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)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- C12Q: MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS (immunoassay G01N33/53); COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES

## Abstract

Biomarkers are provided that predict whether a subject having a hepatocellular carcinoma is responsive to a therapy comprising soratenib or a pharmaceutically acceptable salt thereof. The biomarkers, compositions, and methods described herein are useful in selecting appropriate treatment modalities for and treating a subject having, suspected of having, or at risk of developing a hepatocellular carcinoma.

## Claims

### Claim 1

A method of predicting the response of a human subject having, suspected of having, or at risk of developing a hepatocellular carcinoma to a therapy comprising sorafenib or a pharmaceutically acceptable salt thereof, the method comprising measuring or having measured a baseline expression level of fibroblast growth factor 21 (FGF21) in a biological sample obtained from the human subject,
wherein a high baseline expression level of FGF21, as compared to a control, is predictive that the human subject is not responsive to the therapy comprising sorafenib or a pharmaceutically acceptable salt thereof.

### Claim 2 (depends on 1)

The method of claim 1, further comprising administering to the human subject a therapy comprising an anti-tumor agent other than sorafenib or a pharmaceutically acceptable salt thereof.

### Claim 3

A method of treating a human subject having, suspected of having, or at risk of developing a hepatocellular carcinoma, comprising administering to the human subject a therapy comprising an anti-tumor agent other than sorafenib or a pharmaceutically acceptable salt thereof, wherein the human subject has a baseline expression level of FGF21, in a biological sample obtained from the human subject, that is higher than a control.

### Claim 4

A method of treating a human subject having, suspected of having, or at risk of developing a hepatocellular carcinoma, comprising:
measuring or having measured in a biological sample obtained from the human subject a baseline expression level of FGF21 that is high as compared to a control; and
administering to the human subject a therapy comprising an anti-tumor agent other than sorafenib or a pharmaceutically acceptable salt thereof.

### Claim 5 (depends on 1)

The method of claim 1, wherein the biological sample is a blood sample, a serum sample, or a plasma sample.

### Claim 6 (depends on 1)

The method of claim 1, wherein the baseline expression level of FGF21 is measured by measuring the amount of mRNA encoding FGF21 in the biological sample.

### Claim 7 (depends on 1)

The method of claim 1, wherein the baseline expression level of FGF21 is measured by measuring the amount of FGF21 protein in the biological sample.

### Claim 8 (depends on 1)

The method of claim 1, wherein the sorafenib or a pharmaceutically acceptable salt thereof is sorafenib tosylate.

### Claim 9 (depends on 1)

The method of claim 1, wherein the hepatocellular carcinoma is unresectable hepatocellular carcinoma.

### Claim 10 (depends on 2)

The method of claim 2, wherein the anti-tumor agent other than sorafenib or a pharmaceutically acceptable salt thereof is lenvatinib or a pharmaceutically acceptable salt thereof.

### Claim 11 (depends on 2)

The method of claim 2, wherein the anti-tumor agent other than sorafenib or a pharmaceutically acceptable salt thereof is lenvatinib mesylate.