Courier CStone's potential "best-in-class" ROR1 antibody conjugate drug is approved by the US FDA to enter the clinic

▎This article is transferred from the official WeChat account of CStone Pharmaceuticals (Suzhou, China, January 3, 2022) CStone Pharmaceuticals (Hong Kong Stock Exchange code: 2616), a company that focuses on research, development and commercialization of innovative tumor immunotherapy and precision treatment drugs The leading biopharmaceutical company today announced that its CS5001 clinical trial application (IND) has been approved by the U.
S.
Food and Drug Administration (FDA)
.

This is a potentially best-in-class antibody-conjugated drug (ADC) in the world, targeting receptor tyrosine kinase-like orphan receptor 1, ROR1
.

CS5001 will enter the clinic as one of the fastest-growing ROR1 ADCs in the world, marking another important milestone in the realization of CStone's pipeline 2.
0 strategy
.

ROR1 is a typical tumor embryo protein, which is low or not expressed in adult tissues, but is highly expressed in a variety of tumors, including various types of leukemia, non-Hodgkin’s lymphoma, breast cancer, lung cancer and ovarian cancer Therefore, it is a very potential ADC target
.

CS5001 is an ADC targeting ROR1 with many differentiated features, including proprietary site-specific coupling, tumor-selective cleavable linker and prodrug technology
.

Pre-clinical research data indicate that CS5001 exhibits strong selective cytotoxicity in a variety of tumor cell lines expressing ROR1, and exhibits significant in vivo anti-tumor activity in both blood and solid tumor xenograft mouse models
.

Dr.
Xie Yizhao, Chief Scientific Officer of CStone Pharmaceuticals, said: "We are very pleased to see that CS5001 has been approved for clinical use in the United States in 2021
.

The previous CS5001's data in preclinical pharmacology and pharmacodynamics is encouraging, demonstrating its therapeutic potential in a variety of hematological diseases and malignant solid tumors
.

The first human trial to be launched is to evaluate the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of CS5001 in advanced B-cell lymphoma and solid tumors
.

We will do our best to advance this clinical trial of CS5001.
At the same time, we have submitted an Australian clinical trial application and plan to submit a clinical trial application in China in the near future
.

"Regarding CS5001 (ROR1 ADC) In October 2020, CStone Pharmaceuticals and LegoChem Biosciences, Inc.
(LCB) reached a licensing agreement on the development and commercialization of CS5001
.

CS5001 was originally a joint venture between LCB and ABL bio, a leading Korean biotechnology company.
Synthesis
.

According to the terms of the agreement, CStone Pharmaceuticals has obtained exclusive authorization to lead the development and commercialization of CS5001 in other regions of the world outside of Korea
.

CS5001 is a receptor tyrosine kinase-like receptor 1 (receptor tyrosine kinase-like).
Orphan receptor 1, ROR1) as the target antibody-conjugated drug (ADC)
.

CS5001 has a unique design, using tumor-specific activated pyrrolobenzodiazepine (PBD) protoxin payload (Payload) and linker ( linker)
.
After
CS5001 reaches the tumor and is endocytosed by tumor cells, its linker in the lysosome is cleaved by a specific enzyme highly expressed in tumor cells to release the PBD protoxin, and then the PBD protoxin is in the tumor cells.
Activate, thereby killing tumor cells
.

This "dual control" mechanism of linker plus protoxin effectively reduces the toxicity problems associated with traditional PBD loads and obtains a larger safety window
.

CS5001 has been proven to have complete tumor suppressor effects in several preclinical cancer models, and exhibits good serum half-life and pharmacokinetic characteristics
.

These will be transformed into a wide range of therapeutic indicators for a variety of solid and hematological malignancies
.

In addition, CS5001 uses directional coupling technology to obtain a precise drug-to-antibody ratio (DAR), which facilitates the realization of homogeneous production and large-scale production
.

About CStone Pharmaceuticals CStone Pharmaceuticals (HKSE code: 2616) is a biopharmaceutical company that focuses on research, development and commercialization of innovative tumor immunotherapy and precision treatment drugs to meet the ardent medical needs of cancer patients in China and around the world
.

Established at the end of 2015, CStone Pharmaceuticals has assembled a world-class management team with extensive experience in new drug development, clinical research and commercial operations
.

With tumor immunotherapy combination therapy as the core, the company has established a rich product pipeline consisting of 15 tumor drug candidates
.

So far, CStone Pharmaceuticals has obtained five new drug approvals in the Greater China region, three in Mainland China, one in Hong Kong, and one in Taiwan
.

Many late-stage drug candidates are in critical clinical trials or registration stages
.

CStone's vision is to become a world-renowned biopharmaceutical company, leading the way to overcome cancer
.

For more information, please visit
.

Forward-looking statements The forward-looking statements made in this article only relate to events or information as of the date when the statement is made in this article
.

Except as required by law, we have no obligation to update or publicly revise any forward-looking statements and unexpected events after the date of forward-looking statements, regardless of whether new information, future events or other circumstances appear
.

Please read this article carefully and understand that our actual future results or performance may differ materially from expectations
.

All statements in this article are made on the date of publication of this article and may change due to future developments
.

Disclaimer: WuXi AppTec's content team focuses on introducing global biomedical health research progress
.

This article is for the purpose of information exchange only.
The opinions expressed in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views in the article
.

This article is not a treatment recommendation either
.

If you need guidance on the treatment plan, please go to a regular hospital for treatment
.