Global First Embryonic Stem Cell-Derived Therapy Enters Clinic; Nearly 20 Companies Achieve Clinical Breakthroughs

According to the Center for Drug Evaluation of the National Medical Products Administration of China（CDE）Incomplete statistics from the official website and publicly available data show that last week(October 27 - November 2)Approximately 20 Class 1 innovative drugs are proposed to be included in the breakthrough therapy category/IND has been granted clinical tacit approval/IND application has been accepted, covering fields such as CAR-T, stem cells, small nucleic acids, ADCs, and bispecific antibodies. The indications focus on refractory solid tumors and metabolic diseases.

The following will introduce the first class of innovative drugs approved for clinical trials.

Simnova's SNC115 Injection Approved for Clinical Trials, Targeting Relapsed or Refractory Small Cell Lung Cancer and Relapsed or Refractory Other Neuroendocrine Cancers. SNC115 is a CAR-T targeting DLL3, featuring an innovative proprietary armored structure that demonstrated superior efficacy compared to similar CAR designs in preclinical studies. Simnova also possesses mature and stable CMC capabilities for cell preparation, significantly reducing the ex vivo expansion time of T cells and shortening patient waiting periods.

Product Trial

6-well/24-well/100ml Culture Flask

Application for New Product Trial

Event Time: September-October

IIT Study of SNC115 in Patients with Relapsed or Refractory Small Cell Lung Cancer/Large Cell Neuroendocrine Carcinoma of the Lung Shows That Among 5 Evaluable Patients, Responses Vary Across Dose Groups, with 1 Patient in DL1 Achieving Stable Disease（SD）; Three patients in DL2 were partial responses, respectively.（PR）1 case, stable disease(SD缩小)1 case, disease progression 1 case(DLL3 expression <1); One patient in DL3 achieved PR. Currently, SNC115 is still under exploration for the safety and efficacy of higher doses.

ImStem's IMS001 Injection Approved for Clinical Trials, Indicated for Relapsing-Remitting Multiple Sclerosis and Active Secondary Progressive Multiple Sclerosis. According to publicly available data, IMS001 is allogeneic mesenchymal stem cells derived from a human embryonic stem cell line. Preclinical trials have confirmed that IMS001 possesses immunomodulatory repair capabilities and the ability to cross the blood-brain barrier, which may offer significant advantages in treating neurological disorders, autoimmune diseases, and rare conditions. When treating diseases like multiple sclerosis, IMS001 may more effectively reduce disease relapses, continuous deterioration, and manage the condition.

In addition, IMS001 received FDA IND approval in the United States in 2020 for the treatment of multiple sclerosis, and subsequently completed the first patient dosing in the United States.ForWorld's First Clinical Trial of Systemic Administration of Embryonic Stem Cells in Humans。

HEC Pharm's self-developed siRNA therapy HECN30227 injection has been approved for clinical trials, intended for the development of treating chronic hepatitis B. HECN30227 is the company’s first siRNA developed based on its small nucleic acid technology platform, capable of simultaneously eliminating hepatitis B surface antigens derived from cccDNA and intDNA.（HBsAg）. The candidate drug adopts the company's uniquely designed HEC-GalNova(N-Acetylgalactosamine)Liver-targeting delivery system achieves precise and efficient liver delivery while significantly reducing off-target risks.

Preclinical data show that HECN30227 has pan-genotypic activity, effectively reducing HBsAg levels while maintaining significant efficacy against nucleoside drug-resistant strains. Its in vivo and in vitro efficacy also surpasses competing clinical products. The company is concurrently developing a triple combination therapy of "siRNA+ASO+immunomodulator," which aims to comprehensively suppress HBV and HBsAg through multi-target synergy.

SYH2070 Injection, submitted by a subsidiary of CSPC Group, has been approved for clinical trials, aiming to develop a treatment for hyperlipidemia. According to the CSPC Group's press release, SYH2070 Injection is a drug conjugated with N-acetylgalactosamine.（GalNAc）siRNA Achieving Liver-Targeted Delivery to Target Angiopoietin-Like Protein 3 via Subcutaneous Administration（ANGPTL3）, which can effectively reduce ANGPTL3 levels. By optimizing sequences and employing chemical modification strategies, this candidate product achieves longer-lasting gene silencing effects, with the potential to become an ultra-long-acting siRNA drug for reducing ANGPTL3. It is applicable for treating hypertriglyceridemia or mixed hyperlipidemia and has the potential to effectively reduce the risk of elevated remnant cholesterol levels.

Preclinical studies show that SYH2070 is superior to similar siRNA products in terms of drug activity and duration of efficacy, demonstrating the differentiated advantages of long-lasting drug effects, good safety, and high patient compliance, with high clinical development value. SYH2070 has previously been approved for clinical trials in the United States.

Orange Sail Pharmaceuticals' VBC101 has been approved for clinical trials, intended for the development of treatment for advanced malignant solid tumors. This is also the first IND approval Orange Sail Pharmaceuticals has received in China. VBC101 is an innovative EGFR/cMET bispecific antibody-drug conjugate (ADC), targeting key oncogenic factors in solid tumors. Its Phase I/II clinical trial for treating advanced solid tumors was registered on Clinicaltrials.gov on August 22 this year.

Simnova's Application for Injectable 3H-10000 Approved for Clinical Trials, Intended for Development to Treat Advanced Solid Tumors. According to the company’s official website, 3H-10000 is an ADC targeting FGFR2b, using a highly selective FGFR2b antibody as a carrier that specifically binds to human FGFR2b without binding to other FGFR family members. It shows potential for clinical breakthroughs in treating various solid tumors such as gastric cancer and squamous non-small cell lung cancer. Currently, no products targeting this site have been marketed or entered confirmatory clinical trials.

GenSci Pharmaceuticals' GenSci143 for injection has been approved for clinical trials, intended for the development of treatment for advanced solid tumors. Public information indicates that this is a bispecific antibody-drug conjugate (ADC), designed to induce tumor cell apoptosis by specifically recognizing and binding to tumor cells expressing B7H3 and/or PSMA, intended for the treatment of patients with locally advanced or metastatic solid tumors.

For more IND progress of new drugs, please refer to the table.

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