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

According to the Center for Drug Evaluation of the National Medical Products Administration of China（CDE）According to incomplete statistics from the official website and publicly available information, 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. Indications focus on refractory solid tumors and metabolic diseases.

The following will introduce the first class 1 innovative drug 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 time.

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Event Time: September-October

SNC115 IIT Study in Patients with Relapsed or Refractory Small Cell Lung Cancer/Large Cell Neuroendocrine Carcinoma of the Lung Shows That Among 5 Efficacy-Evaluable Patients, Responses Varied 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 had PR. Currently, SNC115 is still under exploration for the safety and efficacy of higher doses.

ImStem's IMS001 Injection Approved for Clinical Trials, with Proposed Indications 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 address 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 U.S.ForWorld's First Clinical Trial of Systemic Administration of Embryonic Stem Cells in Humans。

HEC Pharmaceutical'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. It maintains significant efficacy against nucleoside drug-resistant strains and demonstrates superior in vivo and in vitro efficacy compared to clinical competitors. The company is concurrently developing a triple combination therapy of "siRNA+ASO+immunomodulator," which comprehensively suppresses HBV and HBsAg through multi-target synergistic effects.

SYH2070 Injection, Submitted by a Subsidiary of CSPC Group, Approved for Clinical Trials to Develop Treatment for Hyperlipidemia. According to the CSPC Group's press release, SYH2070 Injection is a product conjugated with N-acetylgalactosamine.（GalNAc）Achieving liver-targeted delivery of siRNA via subcutaneous administration to target angiopoietin-like protein 3（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, suitable for treating hypertriglyceridemia or mixed hyperlipidemia, and demonstrating the potential to effectively lower 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 effects, good safety, and high patient compliance. It has 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申报的注射用 3H-10000 获批临床，拟开发治疗晚期实体瘤。据该公司官网，3H-10000 是一款靶向 FGFR2b 的 ADC, 以高选择性的 FGFR2b 抗体作为载体，特异性地结合人 FGFR2b, 而不结合 FGFR 其它家族成员，具有治疗胃癌、鳞状非小细胞肺癌等多种实体肿瘤临床突破的潜力。目前，尚无该靶点产品上市或者进入验证性临床试验。

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