▎WuXi AppTec Content Team Report

According to the data on the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration, more than ten Class 1 new drugs were approved for clinical trials in China for the first time this week. These new drugs are from Roche.（Roche）, MSD（MSD）、Sanofi（Sanofi）, Immunotech Applied Science Limited, Jinweike Biotechnology,Companies such as Gebo Biotech, involving cell and gene therapies (CGT), antibody-drug conjugates (ADC), protein degradation therapies, and more.Drug Type. This article will introduce some information about Class 1 new drugs for readers' reference only (the order is not significant).

Weiyuan Likang: WG1025

Mechanism of Action: Gene Therapy Drugs Using HSV-1 Vector

Indications: Dystrophic Epidermolysis Bullosa

WG1025 is a self-developed product by Beijing Weiyuan Likang Biological Technology Co., Ltd.Type I Herpes Simplex Virus（HSV-1) vector as a gene therapy delivery tool for Class 1 biological new drugs, with the active ingredient carrying the encoded Type VII collagen (COL7)COL7A1Replication-defective HSV-1 vector. This product has been approved for clinical trials and is proposed for 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.

Jinweike Biotechnology: JWK001 Injection

Mechanism of Action: AAV Gene Therapy Drugs

Indications: Neovascular Age-Related Macular Degeneration

JWK001 is a product independently developed by Jinweike Biotechnology using a "two-plasmid packaging system."Adeno-Associated Virus（AAV) Gene therapy new drug. It uses an AAV vector carrying a newly self-designed anti-VEGF protein expression cassette, which can continuously and efficiently express anti-VEGF proteins in retinal cells.Vascular Endothelial Growth Factor（VEGF) protein. VEGF has been confirmed inNeovascular Age-Related Macular Degeneration（nAMD) plays an important role in neovascularization. The product has been approved for clinical trials and is intended to be developed for the treatment of nAMD.

Immunotech Applied Science Limited:aT19 Injection

Mechanism of Action: Autologous T Cells Expressing CD19
Indications: Acute Lymphoblastic Leukemia

aT19 is an autologous T cell, genetically modified to forcibly express CD19, currently under development by Immunotech Applied Science Limited's subsidiary, Yongtai Ruikang. CAR-T cell therapy targeting CD19.B-cell Acute Lymphoblastic Leukemia（B-ALL) About 50% of relapsed patients are CD19-positive relapses. The aT19 injection, administered after the CAR-T-19 injection, has the potential to reactivate CAR-T cells, restart their proliferation, and increase the potential of immune memory cells. This product has been approved for clinical trials and is intended for development in patients aged 25 years or younger with relapsed/refractory B-ALL who have benefited clinically from prior CD19-targeted CAR-T cell therapy, aiming to enhance treatment efficacy and reduce relapse.

Sanofi: SAR443765 Injection

Mechanism of Action: IL-13/TSLP Bispecific Nanobody
Indications: Asthma

SAR443765 is a targeted therapy being developed by SanofiThymic Stromal Lymphopoietin（TSLP) andInterleukin-13（IL-13) bispecific nanobody.SanofiClinical data presented at the 2023 American Thoracic Society (ATS) annual meeting showed that SAR443765 demonstrated superior efficacy in a key asthma measurement compared to the use of TSLP or IL13 alone, indicating a synergistic effect. The product has been approved for clinical trials in China and is being developed for the treatment of moderate to severe asthma in adults.

Shanghai Institute of Biological Products: Injectable SIBP-A17

Mechanism of Action: ADC
Indications: Advanced malignant solid tumors

SIBP-A17 is an innovative product independently developed by the Shanghai Institute of Biological Products.Antibody-Drug Conjugates（ADC), with advantages such as a cleavable linker, strong payload activity, and an anti-tumor "bystander effect." Preclinical research data shows that SIBP-A17 has good therapeutic potential for breast cancer, gastric cancer, lung cancer, and pancreatic cancer; it exhibits good stability in animals, with significantly lower free small molecule levels compared to similar products, demonstrating better safety and tolerability. The product has been approved for clinical development intended forRefractory or relapsed advanced malignant solid tumors unresponsive to standard treatment。

Pufang Bio:PRO1107 for Injection

Mechanism of Action: ADC Targeting PTK7
Indications: Solid Tumors

PRO1107 is a product that Profound Bio is currently developing, whichProtein Tyrosine Kinase 7（PTK7) as the target, with MMAE as the payload, using LD343, a novel hydrophilic payload linker independently developed by Proforma Bio, and a DAR value of 8. PTK7 is a receptor protein present on the cell surface that plays an important role in tumor development and progression, with specific expression in tumors. PRO1107 has been approved for clinical development for solid tumor patients.

Merck:MK-7240 Injection

Mechanism of Action: Humanized Monoclonal Antibody Targeting TL1A
Indications: Ulcerative Colitis

MK-7240 is a product developed by Prometheus Biosciences targetingTumor Necrosis Factor (TNF)-like Ligand 1A（TL1A) humanized monoclonal antibody. In June 2023, Merck completed the acquisition of Prometheus for a total amount of $10.8 billion, thereby obtaining this immunology product. TL1A is a target associated with enteritis and fibrosis, playing a crucial role in immune regulation and inflammatory diseases. MK-7240 has been approved for clinical trials in China and is intended to be developed for...Moderate to Severe Active Ulcerative Colitis。

GluBio Pharmaceuticals: GLB-001 Capsule

Mechanism of Action: CK1α Molecular Glue Protein Degrader
Indications: Myeloid Malignancies

GLB-001 is a highly selective casein kinase 1α (CK1α) oral molecular glue protein degrader independently developed by GrailBio. CK1α is a type of enzyme encoded byCSNK1A1The serine/threonine protein kinase encoded by the gene has been confirmed to be crucial for the growth and survival of many different types of malignant hematological tumors and solid tumors. GLB-001 can interact with CRL4.^CRBN The substrate protein receptor CRBN in the E3 ubiquitin ligase complex binds to target and degrade CK1α, triggering the stabilization and accumulation of p53 and the activation of its downstream signaling pathways, leading to tumor cell cycle arrest and apoptosis, thereby exerting an inhibitory effect on tumor growth. GLB-001 has been approved for clinical development intended for...Myeloid Malignancies。

Livzon Pharmaceutical: JP-1366 Tablets

Mechanism of Action: Potassium-competitive Acid Blocker

Indications: Reflux Esophagitis

JP-1366 (zastaprzan) is an innovative drug developed by Onconic Therapeutics, a company based in South Korea.Potassium-competitive acid blocker（P-CAB), an application for market approval has been submitted in South Korea. In March 2023, Livzon Pharmaceutical reached a collaboration with Onconic, obtaining the authorization to develop, manufacture, and commercialize the product in mainland China, Hong Kong, Macau, and Taiwan. JP-1366 has been approved for clinical development in China for the treatment of reflux esophagitis.

HaiChuang Pharma: HP537 Tablets

Mechanism of Action: p300/CBP Small Molecule Inhibitor
Indications: Hematologic Malignancies

HP537 is independently developed by Haisco Pharmaceuticals.p300/CBP Inhibitor Small Molecule Anticancer Drugp300/CBP is involved in cell cycle progression and cell growth, differentiation, and development. It is highly expressed and activated in tumors and serves as a key regulatory factor for tumor cell growth. HP537 has been approved for clinical trials and is proposed for development.Hematologic Malignancies, including but not limited to patients with multiple myeloma (MM), non-Hodgkin lymphoma (NHL), acute myeloid leukemia (AML), and myelodysplastic syndromes (MDS).

Repare Corporation：Camonsertib Capsules

Mechanism of Action: ATR Inhibitor
Indications: Solid Tumors

Camonsertib is a selective oral ATR inhibitor developed by Repare.ATR, fully known as Ataxia Telangiectasia Mutated and Rad3-related protein, is one of the key enzymes in DNA damage repair.Research shows that inATMIn tumor cells with deficiencies, ATR inhibitors can induce synthetic lethality, leading toATMThe absence of tumor cell death does not affect normal cells.In addition, ATR inhibitors canCombined with PARP inhibitors to enhance treatment sensitivity.Camonsertib Approved for Clinical Trials in China, Intended for Development in Solid Tumors.

In addition to the aforementioned drugs, several other drugs have also been approved for clinical trials in China this week. Due to space limitations, this article will not introduce them one by one. It is hoped that the subsequent research of these drugs will proceed smoothly, benefiting a large number of patients as soon as possible.

References:

[1] Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. Retrieved Feb 24, 2024, fromhttps://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c

[2] Official websites of various companies and publicly available information