Monotherapy

▶HIV Infection

Azvudine is a pyrimidine nucleoside drug with broad-spectrum antiviral activity.Azvudine was conditionally approved by the NMPA in July 2021 as anational Class 1 innovative drug for treating HIV-1 infected adults with high viral load.

According to Frost & Sullivan, azvudine is a dual-targeted oral nucleoside drug for the treatment of HIV that acts as both an NRTI and a Vif co-protein inhibitor (NRTIs refer to nucleoside reverse transcriptase inhibitors, a class of first-line antiretroviral therapy (ART) drugs commonly used for the treatment of HIV infections, while Vif co-protein inhibitors protect a human enzyme with innate antiviral activity from the effects of HIV). Azvudine can be used in combination with antiretroviral drugs of different regimens as the backbone of multiple two- or three-arm ART regimens. It provides a treatment option with safety and antiviral activity observed in clinical studies to date for HIV-infected patients, including those who are intolerant to or have limited treatment options under existing antiretroviral regimens.

In addition, azvudine has been listed as one of the National Science and Technology Major Projects (Major New Drug Innovation) in China. Azvudine was included in the 2021 and 2024 China AIDS Treatment Guidelines after it was approved by the NMPA.

▶ COVID-19  

Azvudine is the first NMPA-approved oral direct-acting antiviral treatment for COVID-19 developed by a Chinese company, which was conditionally approved for the treatment of common COVID-19 in adults in July 2022.

As an RNA-dependent RNA polymerase (RdRp) inhibitor, azvudine can effectively suppress the replication of SARS-CoV-2, thereby terminating viral RNA chain elongation and virus replication.

Moreover, azvudine and its triphosphate are largely concentrated in PBMCs and the thymus suggesting an immune-targeting nature of azvudine on top of its antiviral effect. The inhibition of SARS-CoV-2 replication in the thymus might protect the host immune system from viral attack and promote host T cell immunity against viruses.

In the Phase Ⅲ clinical trials in China and Russia, azvudine effectively reduced viral load in patients with a baseline viral load above a certain threshold and alleviated clinical symptoms for COVID-19 patients.In April 2023, azvudine became the first domestically developed small-molecule oral medication for COVID-19 included in the NRDL.As of December 31, 2025, Azvudine for the COVID-19 indication has been widely commercialized domestically, covering an aggregate of over 50,000 medical institutions nationwide.

▶ The Blood Cancer Drug

We are also developing our Core Product, azvudine, for the treatment of myeloma, lymphoma and acute leukemia. Azvudine has a dual anti-tumor mechanism, i.e.: Azvudine can inhibit cancer cell proliferation by terminating DNA strand elongation and interfering with various enzymes involved in the synthesis of nucleic acids in cancer cells.Azvudine can also act as an immunomodulator, reducing the over-clustering of myeloid-derived suppressor cells (MDSCs) in tumor microenvironment and promoting the infiltration and expansion of CD8+ T, CD4+ T cells and natural killer (NK) cells, thereby exerting the tumor inhibition effect.

Genuine Biotech submitted the IND application for the Phase II clinical trial of azvudine monotherapy for the treatment of blood cancers, obtained the IND approval in December 2025.

▶Immunological Non-Responders (INRs) in HIV-infected Patients

Genuine Biotech is also developing azvudine for the treatment of INRs—HIV-infected patients who have received ART for more than four years, maintained plasma viral load below the detection limit (i.e.<50 copies/mL) for over three years, yet continue to exhibit CD4+ T-cell counts persistently below 350 cells/µL, after excluding other causes of chronic CD4+ T-cell depletion.

A single-center, single-arm exploratory investigator-initiated trial (“IIT”) was conducted in China to evaluate the efficacy of azvudine for the treatment of INRs in HIV-infected patients.These findings demonstrate that azvudine-based triple antiretroviral regimen have the potential to effectively elevate CD4+ T-cell counts, restore CD4/CD8 immune balance, and promote immune reconstitution in HIV-infected INRs, supporting its promising clinical utility.

Genuine Biotech has jointly applied with Peking Union Medical College Hospital of the Chinese Academy of Medical Sciences （中国医学科学院北京协和医院） for the R&D of such indication to be designated as a National Major Science and Technology Project of 2025 （2025年国家科技重大专项）， and got approved by theMinistry of Science and Technology of the PRC.

Combination Therapy

Combination therapy has been increasingly explored in cancer treatment. Genuine Biotech has adopted a scientific guiding strategy that is centered on our Core Product, azvudine, and are developing an Azvudine+ Platform, aiming to further explore the clinical and commercial potential of azvudine.

▶Azvudine + Anti-PD-1

PD-1 is a type of receptor expressed on the surface of immune cells, including T cells. Cancer cells can upregulate the expression of PD-L1, which binds to PD-1 on T cells, thereby inhibiting the proliferation and activation of CD4+ T cells and CD8+ T cells and suppressing T cell responses to cancer cells. PD-1 inhibitors block the interaction between PD-1 and PD-L1 and restore T cell activity, leading to enhanced recognition and remission of cancer cells.

Azvudine can also be used as an immunomodulator, effectively restoring and enhancing the anti-tumor killing function of T cells. By reshaping the tumor immune microenvironment, it transforms "cold tumors" with dormant immune responses into "hot tumors" with active immune cell infiltration, thereby exerting an antitumor effect. The tumor-suppressive effect of this mechanism is related to the expression of MDSCs in the tumor microenvironment, and the effect is better in solid tumors with more MDSC infiltration, such as liver cancer, colorectal cancer and NSCLC.

Genuine Biotech has entered into a collaboration agreement with Innovent Biologics (Suzhou) Co., Ltd. (“Innovent”)， a principal subsidiary of Innovent Biologics, Inc., a company listed on the Main Board of the Stock Exchange (01801.HK), under which Innovent has agreed to supply for nil consideration a sufficient quantity of SINTILIMAB, a commercialized anti-PD-1 agent owned by Innovent, for the Phase I clinical trial of azvudine + anti-PD-1 combination therapy for the treatment of cancer indications in accordance with the research and development plan approved by both parties. We obtained the IND approval of azvudine + anti-PD-1 for the treatment of liver cancer and colorectal cancer in February 2026.

▶Azvudine + Dosimertinib

As a nucleoside analogue, azvudine can induce tumor cells to release damage-associated molecular patterns (DAMPs), which interact with immune cells and could enhance APCs’ ability of antigen presenting to improve the ability of T cells to kill and damage tumor cells. In addition, azvudine reduces myeloid-derived suppressor cells in the tumor microenvironment and promotes the infiltration and expansion of natural killer (NK) cells and T cells, thereby augmenting the immune cell-mediated cytotoxicity against tumor cells. Azvudine and dosimertinib’s complementary mechanisms have the potential to lead to synergistic effect in the treatment of NSCLC.

We obtained the IND approval of azvudine + dosimertinib for the treatment of NSCLC in September 2025 and initiated a Phase I/IIa trial in November 2025.

▶Azvudine + CTX

In our preclinical studies, compared to the untreated control group, both the azvudine and the CTX monotherapy groups showed significantly inhibited tumor growth, with a TGI (tumor growth inhibition) rate of 84.46% and 81.29% after two weeks, respectively, at the dosage of 1mg/kg once a day (QD) and 30 mg/kg once a week (QW),respectively. The combination of azvudine and CTX showed encouraging effect, achieving 100% tumor remission in all models in the combination therapy group after two weeks at the same dose levels. In this group, during the post-treatment observation period (from day 14 to day 60), the tumors in two models remained without recurrence, and the median survival time was 54.5 days,increased by 289.3% compared to the untreated control group.

▶Azvudine/CL-197 All-oral Long-acting Composite Tablet

Genuine Biotech is developing a composite tablet comprising azvudine and CL-197. Combination antiretroviral therapy (cART) is a treatment approach that has been widely adopted in clinical practice for HIV infection which can suppress viral replication and reduce the risk of drug resistance associated with monotherapy.

The azvudine/CL-197 can realize combined action on three targets, thus the composite tablet is by itself a cocktail therapy for the treatment of HIV infection while being expected to reduce the risk of drug resistance based on the preclinical study data.Genuine Biotech believes it has the potential to be administered orally on a weekly basis with long-acting effects and strengthen patient adherence.At present,Genuine Biotech has completed the preclinical research on azvudine/CL-197 dual-drug long-acting oral treatment for HIV.

Genuine Biotech is developing CL-197, another new NRTI that can inhibit reverse transcription by mimicking endogenous purine nucleotides, and has potential long-term effects in the treatment of HIV infection. Pharmacokinetic studies in an oral gavage animal model showed that the half-life of CL-197 in PBMC was about 168 hours, confirming its potential for long-term efficacy and possibly improving medication compliance. CL-197 demonstrated promising results with respect to safety, tolerability and PK characteristics in its Phase I clinical trial.Genuine Biotechcommenced the Phase IIa trial in November 2025.

Dosimertinib is a potent, selective and orally administered epidermal growth factor receptor (EGFR)-targeting drug candidate, for the treatment of advanced EGFR mutation-positive non-small cell lung cancer (NSCLC), one of the most prevalent types of lung cancer in China. Dosimertinib is designed to address the medical needs of advanced NSCLC

patients harboring EGFR mutations that are resistant to previous generations of targeted drugs.Preclinical pharmacokinetic studies also showed higher tissue exposure in lung and brain tissues, demonstrating dosimertinib has an advantage in the treatment of lung cancer and brain metastases.

We have completed the Phase I clinical trial, enrolled 23 patients have observed an overall good safety profile and good dose-related efficacy.The amendment of the Phase II clinical trial protocol was approved by the CDE in May 2025, with its first patient enrolled in June 2025.

ZSSW-136 is a novel inhibitor of topoisomerase I (TOPO1) enzymes which participate in the overwinding or underwinding of DNA and are particularly vulnerable to TOPO1 inhibitors during their cleavage reaction.

However, CPT-based drugs face the problems of primary and post-treatment drug resistance. Utilizing an AI-Computer Assisted Drug Design (CADD) method we developed in-house, we successfully replaced one of the five rings for the core nucleus of CPT, which led to the discovery of a new generation of innovative TOPO1 inhibitors and a novel ADC payload platform with strong global IP protection positions.

Genuine Biotech has discovered a PCC molecule, ZSSW-136, that exhibits broad-spectrum antitumor activity and can effectively inhibit dozens of cancer cells at nano-molar concentrations.Genuine Biotech has discovered hundreds of novel molecules, from which we have selected several payload compound candidates with potential payload properties. More importantly, our payload molecules have a completely new parent nucleus structure, thus potentially addressing the drug resistance issue of commonly used payloads.

ZS-1004 is a PSMA—targeting dual-payload antibody—drug conjugate (ADC) composed of a proprietary humanized monoclonal antibody, a highly hydrophilic linker, and an optimized dual-payload configuration with controlled overall drug loading and a tuned payload ratio designed to improve tolerability. Upon binding to PSMA on tumor cells, ZS-1004 is rapidly internalized and undergoes lysosomal processing to release two cytotoxic payloads—a microtubule inhibitor and a topoisomerase I inhibitor—thereby enabling coordinated inhibition of tumor cell proliferation and induction of tumor cell death.

ZS-1004 exhibits a highly homogeneous conjugate profile, strong plasma stability, and favorable PK properties in preclinical studies. Following binding to PSMA, ZS-1004 undergoes efficient internalization and payload release, resulting in potent tumor cell killing. In the 22Rv1 cell-derived xenograft (CDX) mouse model, a single intravenous administration of ZS-1004 at 2 mg/kg produced potent and durable tumor growth inhibition through Day 27 post-dose.

We are developing ZS-2004, a GRPR—targeting antagonist as a theranostic radioligand for molecular imaging and targeted radionuclide therapy in GRPR-expressing malignancies, including

lung, breast, and prostate cancers. By leveraging GRPR overexpression in certain tumor types,ZS-2004 is designed to enable tumor visualization for diagnostic imaging and to deliver -particle radiation from lutetium-177 to tumor cells, inducing DNA damage that may lead to tumor cell death.

Preclinical studies have evaluated the pharmacokinetics, biodistribution, efficacy, and safety of ZS-2004. In vivo PET/SPECT imaging in a PC-3 mouse tumor model demonstrated clear tumor accumulation with favorable tumor-to-nontarget (T/NT) ratios, supporting robust tumor visualization. Biodistribution studies further confirmed preferential uptake in tumors with limited retention in normal tissues and organs. In pharmacology studies, ZS-2004 produced potent tumor growth inhibition in a PC-3 cell-derived xenograft (CDX) mouse model (TGI = 96.9% on Day 52,Q2W × 3). In nonclinical safety assessments, ZS-2004 was well tolerated. In mouse models, no significant systemic toxicity was observed at doses up to 98-fold higher than the human equivalent dose (HED) of the anticipated maximum clinical therapeutic dose.

MTB-1806 is a small-molecule drug candidate under development for AIS,Genuine Biotech has commenced preclinical studies for MTB-1806. So far MTB-1806 has demonstrated its efficacy in pharmacodynamic studies, where it effectively reduced the infarction (the death of brain tissue) volume and the brain swelling volume and improved the neurobehavioral score.Additionally, under lower drug dosing regimens (10 mpk and 15 mpk), MTB-1806 was evaluated in a rat model of global ischemia-reperfusion injury, which is commonly associated with ischemic stroke. The study results indicated that MTB-1806 was associated with reductions in cerebral edema and neurological deficit scores in this preclinical model.