Nearly 50 Class 1 New Drugs Granted Clinical Trial Approval in China in February

Mar 03, 2024 08:29 CST Updated 08:29

Immunofoco

Developer of Novel Therapeutics for Solid Tumors

CHIGENOVO

Clinical Genetic Diagnosis, Prevention, and Gene Therapy Drug Development for Hereditary Eye Diseases

Immunotech Biopharm

Developer of Cellular Immunotherapy Products

Jinweike Biotechnology

AAV Gene Therapy New Drug Developer

▎Edited by the WuXi AppTec content team

According to the official website of the Center for Drug Evaluation (CDE) under China's National Medical Products Administration, in the just-concluded month of February, nearly 50 Class 1 new drugs received clinical trial implied permission for the first time in China, includingCell and Gene Therapy (CGT)、Antibody Drug、Antibody-Drug Conjugates (ADC)、Small Molecule Targeted TherapyDifferent types of drugs, etc.This article will focus on several CGT therapies approved for clinical trials in February (listed in no particular order), for readers' reference only.

For more information on Class 1 new drugs approved for clinical trials in China in February, pleaseScan the QR code below to obtain the PDF file of "Class 1 New Drug Granted First Implicit Clinical Trial Approval in China in February 2024."

CHIGENOVO: ZVS203e Injection

Mechanism of Action: In Vivo AAV Gene Editing Therapeutic Products

Indications: Retinitis Pigmentosa

ZVS203e is a Class 1 innovative ophthalmic gene-editing drug under research by CHIGENOVO.Using the third-generation artificial nuclease CRISPR/Cas9, targeting the mutatedRHOTargeted gene editing to treat diseases at their root cause by addressing pathogenic genes, achieving a one-time administration for lifelong cure.. The product has been approved for clinical use and is intended to treat patients carryingRHO-R135W（RHO c.403C>T）Patients with mutated Retinitis Pigmentosa (RP). RP is a group of progressive monogenic blinding eye diseases characterized by the progressive and selective loss of retinal photoreceptor cells and retinal pigment epithelial cells, among whichRHOGene mutations are most common in autosomal dominant RP.

ZVS203e was granted orphan drug designation by the U.S. FDA in July 2022 and initiated clinical trials at Peking University Third Hospital in September 2023.Investigator-Initiated Clinical Study（IIT) to complete the world's firstRHOInjection administration in RP patients with gene mutations showed good safety and efficacy in the first patient.

Immunofoco: IMC001 Injection

Mechanism of Action: EpCAM-Targeted Autologous CAR-T

Indications: Digestive System Tumors

IMC001 is a self-developed autologous CAR-T cell product targeting EpCAM by Immunofoco. EpCAM is highly expressed in various epithelial-derived tumor tissues, including gastrointestinal tumors, with an expression rate of over 90% in both colorectal cancer and gastric cancer.[3], and can serve as a therapeutic target for circulating tumor cells (CTC).IMC001 aims to address the challenges of metastasis and recurrence by targeting EpCAM, offering a new treatment option for advanced digestive system tumors.。

On February 7, Immunofoco announced that the product had been approved for clinical trials in China, intended for the treatment of advanced digestive system tumors with positive EpCAM expression, including but not limited to advanced stages.Gastric Cancer（GC）/Adenocarcinoma of the Esophagogastric Junction（GEJ）。February 23,ImmunofocoImmunofocoIt was announced that the product has once again received FDA approval for clinical research to treat the aforementioned indications. In the investigator-initiated trial (IIT) targeting advanced gastric cancer, researchers have identified the recommended dose of IMC001 that is both safe and effective; in this dose group, 2 out of 5 patients were confirmed to have achieved partial response (PR).Objective Response Rate (ORR) Reached 40%。

Immunotech Applied Science Limited: aT19 Injection

Mechanism of Action: CD19 Antigen-Presenting T Cells

Indications: Acute Lymphoblastic Leukemia

aT19 is a product under research by Immunotech Applied Science Limited.Autologous T cells genetically modified to forcibly express CD19Studies have shown that the recurrence after CD19-targeted CAR-T cell therapy for B-cell acute lymphoblastic leukemia (B-ALL) may be related to the insufficient persistence of CAR-T cells in vivo. The productAfter the administration of CAR-T-19 injection, it has the potential to reactivate CAR-T cells, restart their proliferation, and increase the potential of immune memory cells.。

The indication approved for clinical use in this approval is: Intended for patients aged 25 years or younger with relapsed/refractory B-ALL who have benefited clinically from CD19-targeted CAR-T cell therapy, to enhance treatment efficacy and reduce recurrence.Based on the preliminary IIT results, subjects who received sequential aT19 CAR-T cell therapyMRD-negative sustained remission has lasted for more than 3 years.No adverse reactions were reported during the study. The feasibility and efficacy of sequential CAR-T cell therapy with aT19 injection have been preliminarily validated.

Weiyuan Likang: WG1025

Mechanism of Action: Human CarrierCOL7A1Replication-defective HSV-1 vector

Indications: Dystrophic Epidermolysis Bullosa

WG1025 is a self-developed product by Beijing Weiyuan Likang Biological Technology Co., Ltd.Reusable, Topical Gene Therapy Biologic Drug. The active ingredient of this product is human-derivedCOL7A1A replication-defective type I herpes simplex virus (HSV-I) vector encoding type VII collagen (COL7) protein. WG1025 has been approved for clinical development.Dystrophic Epidermolysis Bullosa（DEB). DEB is a genetically inherited skin disease with severe clinical symptoms, caused byCOL7A1The lack of COL7 protein expression caused by gene mutations results in extremely fragile skin in patients.

DEB patients can apply WG1025 gel to the woundCOL7A1Gene delivery to patients' skin cells produces endogenous COL7 protein, which then forms anchoring fibrils that anchor the epidermis to the dermis, providing normal skin structural adhesion and promoting wound healing. An earlier IIT study conducted by WG1025 showed that after medication, subjects observed...More than 70% of wounds have continuous healing for over 3 months.Skin biopsy showed continuous distribution of COL7 protein between the epidermis and dermis in subjects after WG1025 treatment, confirming the safety and efficacy of WG1025.

Jinweike: JWK001 Injection

Mechanism of Action: AAV Gene Therapy Products

Indications: Neovascular Age-Related Macular Degeneration

JWK001 is a Class 1 new AAV gene therapy drug independently developed by Jinweike Biotechnology. The product has been approved for clinical development and is intended to be used forNeovascular Age-Related Macular Degeneration（nAMD）。Vascular endothelial growth factor (VEGF) has been demonstrated to play a crucial role in the formation of new blood vessels in nAMD.JWK001 adopts the "two-plasmid packaging system" to continuously and efficiently express anti-VEGF protein in retinal cells through an AAV vector carrying a newly self-designed anti-VEGF protein expression cassette. This achieves the goal of a single treatment with lifelong effectiveness.

According to the press release from Jinweike Biotechnology, the results from the ongoing IIT follow-up indicate that,JWK001 Injection demonstrates good safety and tolerability, with a low clinically effective dose. Patients require only a single administration without the need for adjuvant therapy., which can avoid the risks associated with traditional nAMD treatments, such as damage to ocular tissues from repeated intravitreal injections and poor patient compliance, offering significant convenience to patients.

In addition to the aforementioned drugs, other types of drugs, including ADCs, antibody drugs, and small-molecule targeted therapies, were approved for clinical trials in China for the first time in February.，Due to space limitations, this article will not go into details one by one. We hope that the subsequent clinical research of these new drugs will proceed smoothly, benefiting patients as soon as possible!

Scan the QR code below to access"New Class 1 Drug Granted Implicit Clinical Trial Approval in China for the First Time in February 2024"PDF file.

References:

[1] Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. Retrieved Feb 29, 2024, from https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c

[2] Official websites and press releases of various companies

[3] P Went ,et al.（2006） Frequent high-level expression of the immunotherapeutic target Ep-CAM in colon, stomach, prostate and lung cancers. Br J Cancer. doi: 10.1038/sj.bjc.6602924.PMID: 16404366

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