October 26, 2025, the highly anticipated 10th China BioMed Innovation and Investment Conference (CBIIC) grandly opened in Nanjing. As a flagship session of the conference, the Clinical Trial Data Release Session has, for ten consecutive years, built a high-end exchange platform for investors, innovators and clinical experts, showcasing the cutting-edge developments of China’s pharmaceutical innovation to the world. It has received sustained attention and high acclaim from professionals in the investment community and the pharmaceutical industry.

Dr. CHEN Li, Chairman of the Drug Research and Development Specialty Committee of the China Pharmaceutical Innovation and Research Development Association (PhIRDA) and Chief Executive Officer of HUA Medicine, served as the moderator of this session.

Moderator: CHEN Li

During the Clinical Trial Data Release Session, nine drug candidates at key developmental stages were featured, with their core clinical data officially disclosed for the first time at the event.

01

Phase I-II Clinical Study of Fumalate Orbitezine Enteric-Coated Pellets Capsules (SM-1) Combined with Temozolomide in the Treatment of Recurrent High-Grade Gliomas

SM-1 is a Class I small-molecule antineoplastic new drug independently developed by Shenzhen Zhenxing Pharmaceutical Technology Co., Ltd. It targets Procaspase-3 and promotes its conversion to Caspase-3, a key executioner protein for apoptosis, thereby inducing tumor cell apoptosis.

The study results showed: Phase I clinical trial: 27 patients enrolled, 24 evaluable; 2 CR, 5 PR, 5 SD; ORR 29.2%, DCR 50%. Phase II clinical trial: 37 patients enrolled. 16 evaluable in the experimental group: 3 PR, 8 SD, ORR 18.8%, DCR 68.8%; 10 evaluable in the control group: 6 SD, ORR 0, DCR 60%.

LI Wenbin, Director of the Comprehensive Cancer Treatment Center, Beijing Tiantan Hospital, Capital Medical University

02

Global First-in-Class S1PR1 Agonist Proximod (LJR001) for Rheumatoid Arthritis: Phase IIb Clinical Trial

Proximod (LJR001) is the world’s first S1PR1 agonist advanced into Phase II (Phase IIb) clinical trials for the treatment of RA, jointly developed by Longivitron Biotech and the Institute of Materia Medica, Chinese Academy of Medical Sciences. It is mainly indicated for the inhibition of inflammatory and immune responses.

A total of 179 RA patients were enrolled in the trial and received treatment for 12 weeks. The proportion of patients achieving the American College of Rheumatology 20% improvement response criteria (ACR20) was significantly higher in the Proximod 5mg and 10mg groups than in the placebo group (66% and 77.8% vs 39.2%, P<0.01 and P<0.001, respectively). At the 4-week follow-up visit after drug discontinuation, the Proximod 5 mg and 10 mg groups maintained sustained efficacy, with a continuous increase in the proportion of patients achieving ACR50 response (31.7% and 37.9% vs 15.3%, P<0.01).

Most secondary clinical endpoints reached statistical significance. The Proximod 5 mg and 10 mg groups significantly improved the Clinical Disease Activity Index (CDAI) and Simplified Disease Activity Index (SDAI) of patients, as well as their quality of life. Notably, the drug exhibited distinct advantages in reducing the risk of thrombosis and lowering rheumatoid factor titers.

LI Chun, Chief Physician, Department of Rheumatology and Immunology, Peking University People’s Hospital

03

Phase I/II Clinical Study Report of Oroxylin A Tablets, a Class 1.1 Original Innovative Drug

Oroxylin A (CPU-118) is a monomeric compound derived from Scutellaria baicalensis with significant anti-tumor activity and protective effects on normal tissues including the liver, heart, and nervous system. Currently, Nanjing Qinling Pharmaceutical Technology Company Limited has achieved the total chemical synthesis of Oroxylin A with a purity of >99.7%, enabling industrial-scale production, and has been granted multiple national patents.

As a national Class 1.1 new drug, Oroxylin A has received clinical trial approval for the treatment of hepatocellular carcinoma (HCC) and pan-tumor indications. In the Phase Ib/IIa registrational clinical trial, a total of 37 patients with advanced solid tumors who had progressed after failure of standard therapy were enrolled, with 30 patients evaluable for efficacy. Among them, 2 patients achieved partial response (PR), 7 patients had stable disease (SD), resulting in a disease control rate (DCR) of 30%, and no treatment-related adverse events (TRAEs) of Grade >3 were observed. Notably, in 15 HCC patients receiving last-line therapy, 2 achieved PR and 3 had SD, with a DCR of 33.3%. These findings demonstrate that Oroxylin A monotherapy exhibits significant anti-tumor efficacy, particularly in HCC, warranting further confirmatory clinical studies including both monotherapy and combination regimens.

QIN Shukui, Dean of School of Basic Medicine and Clinical Pharmacy, China Pharmaceutical University

04

AI-Driven R&D Yields Breakthrough: EG-501 Shows Positive Clinical Data in Addressing Cognitive Impairment in NPSLE

The EG-501 project represents the first end-to-end closed-loop validation from drug discovery to positive clinical signals using Evergreen Therapeutics’ self-developed AI platform, providing a scalable and repeatable new model for drug R&D in the industry.

As the world’s first oral small-molecule drug to enter Phase II clinical trials for the treatment of cognitive impairment associated with neuropsychiatric systemic lupus erythematosus (NPSLE), EG-501 has successfully filled a critical medical gap in this field that has existed for decades. The Phase II clinical trial (multicenter, randomized, double-blind, placebo-controlled) completed in the United States achieved breakthrough positive results. The study was conducted at three leading U.S. academic centers, including Vanderbilt University Medical Center. Data showed that the EG-501 group achieved a statistically significant improvement in the total RBANS score (EG-501: +10.4; placebo: +4.2; p=0.032), with clear clinical significance.

LI Changqing, Co-founder, Chief Medical Officer of Evergreen Therapeutics

05

GENESEEQPRIME®：World’s First Tumor NGS Broad Panel Kit with Triple Certifications from NMPA, FDA, and CE

GENESEEQPRIME®, a pan-solid tumor NGS large-panel gene detection kit independently developed by Nanjing Geneseeq Technology Inc., has been approved by the U.S. FDA. It has become the world’s first and only NGS large-panel oncology gene detection product to simultaneously hold triple certifications from China NMPA, U.S. FDA and EU CE.

In performance verification, GENESEEQPRIME® was compared using multiple technical approaches to evaluate detection consistency across various variant types. The product demonstrated excellent detection accuracy for all variant types: compared with the reference kit, the positive percent agreement (PPA) for all point mutations and indels was 92.44%, and the negative percent agreement (NPA) reached 99.99%; for fusion mutations, PPA was 96.15% and NPA was 98.24%; for amplification mutations, PPA was as high as 100.00% and NPA was 99.49%. In addition, the Pearson correlation coefficient for tumor mutational burden (TMB) was 0.94; for microsatellite instability (MSI), PPA was 96.77% and NPA was 97.67%. The limit of detection (LOD) of the product has also been systematically verified with multiple gradients and large sample sizes, with a cumulative number of tests exceeding 2,000. Among them, the LOD for hotspot single-nucleotide variants (SNVs) was 2.14% VAF. The overall detection performance is comparable to leading similar products in the United States.

WANG Xiaonan, Founder and CTO of Nanjing Geneseeq Technology Inc.

06

First Domestic ERT Product-Gaurunning Clinical Research Data Introduction

Velaglucerase β for Injection (Gereining®) is the first domestinally developed enzyme replacement therapy (ERT) independently developed by CANbridge Pharmaceuticals and approved for marketing in China. Its launch has filled the technical gap in ERT product development among domestic pharmaceutical companies.

Key study results showed that after 9 months of treatment with CAN103 at 60 U/kg, the mean (standard error) percentage reduction in spleen volume from baseline was 35.32(3.02)%, with P<0.0001. Other key clinical endpoints also improved significantly: mean reduction in liver volume was 18.80(2.87)%; mean hemoglobin increased by 2.17(0.42) g/dL, and mean platelet count increased by 19.42(5.55)×10⁹/L. In addition, patients in the lowdose 30 U/kg group also achieved notable improvements across all parameters, suggesting a doseresponse relationship. Finally, cumulative data from up to 2 years of treatment indicated a favorable riskbenefit profile, supporting longterm use of the product in patients with Type I and Type Ⅲ Gaucher disease aged 12 years and older.

Joe SHEN, COO of CANbridge Pharmaceuticals Inc.

07

Development and Clinical Application of Oncolytic Adenovirus YH01 Injection

The oncolytic adenovirus YH01 Injection is a national Class 1.1 original innovative drug independently developed and produced by Beijing Inme Future Biopharmaceutical Technology Co., Ltd., a wholly-owned subsidiary of Suzhou Infinory Pharmaceuticals, LLC.

To date, 12 patients have been enrolled, all of whom are patients with refractory bladder cancer who have failed standard BCG therapy with no other effective treatment options available. Trial results showed that among the 10 patients who reached the efficacy evaluation period, 8 achieved complete response (CR, defined as 100% disappearance of tumor lesions), representing an overall complete response rate of 80% and a disease control rate of 100%, indicating that all evaluable patients achieved effective disease control. In addition, data showed a 9-month tumor recurrence rate of 0%, a result that ranks among the international leading levels for comparable marketed and investigational oncolytic virus products worldwide.

HUANG Yinghui, Chairman of the Board, Suzhou Infinory Pharmaceuticals, LLC

08

HTD1801: A Breakthrough China-Origin Therapeutic in Cardiovascular

-Kidney-Metabolic (CKM) Diseases

HighTide Therapeutics’core product, HTD1801, is a naturally derived oral anti-inflammatory metabolic modulator designed to address cardiovascular-kidney-metabolic (CKM) disorders. It achieves synergistic metabolic improvement and anti-inflammatory effects via a dual mechanism: activating AMP-activated protein kinase (AMPK) and inhibiting the NLR family pyrin domain-containing 3 (NLRP3) inflammasome.

Two Phase 3 trials in type 2 diabetes mellitus (T2DM) consistently demonstrated significant glucoselowering efficacy, along with improvements in lipid and inflammatory biomarkers. A pooled analysis revealed that in patients with mild renal impairment, HTD1801 significantly increased estimated glomerular filtration rate (eGFR), suggesting its potential for early renal intervention and functional recovery. Most currently approved drugs with renal benefits only slow eGFR decline and rarely reverse disease progression. Given its multidimensional clinical benefits, HTD1801 has the potential to become a foundational therapy in the CKM field.

Nadia GAO, Company Secretary & Deputy General Manager, HighTide Therapeutics.

09

Phase II Clinical Results of Asuptegravir (ASU), a New-generation HIV Integrase Inhibitor

Asuptegravir (ASU) is a novel HIV integrase inhibitor independently developed by Aidea Pharmaceutical Group. The Phase II clinical trial results of ASU were presented at this conference.

The trial results showed that in treatment-naive participants with HIV infection, monotherapy with ASU for 10 days achieved a mean reduction in viral load of 2.34 log10 copies/mL from baseline. Following continued treatment with the nucleoside backbone emtricitabine/tenofovir alafenamide(FTC/TAF) for an additional 18 days, more than 90% of participants achieved virologic suppression(HIV-RNA<50 copies/mL). Notably, across all dose groups, after 10 days of ASU monotherapy followed by 18 days of combination therapy with the nucleoside backbone, 100% of participants had HIV-RNA<200 copies/mL. The available study results demonstrate that ASU has a favorable safety profile, clear monotherapy efficacy, and when combined with FTC/TAF as a complete regimen, achieves high and rapid virologic suppression. In addition, the triple single-tablet regimen(STR) centered on asuptegravir plus FTC/TAF has been submitted for investigational new drug(IND) application and accepted for review. In the future, it is expected to provide a more convenient treatment option for people living with HIV.

FU Heliang, CSO and Chairman, Jiangsu Aidea Pharmaceutical Group Co., Ltd.

Clinical Trial Data Release Session

Over the past decade, the Clinical Trial Data Release Session has consistently served as a “launch platform” for cutting-edge achievements in the global pharmaceutical sector, as well as a “vital bridge” connecting capital, scientific research, and clinical practice. Here, participants have witnessed the full journey of multiple drug candidates—from laboratory research and clinical translation to regulatory approval and patient benefit. They have also seen the rise, breakthroughs, and growing global recognition of China’s innovative drugs on the world stage. To date, the session has showcased key clinical data for 87 innovative drugs to a global audience. These achievements have not only significantly expanded clinical treatment options but also delivered dual value to patients’ health and industrial investment potential. Looking ahead, we expect to leverage deeper collaboration between innovation and capital to continuously advance the high-quality development of domestic innovative drugs, enabling more China-originated R&D achievements to benefit patients worldwide.