嘉因生物冲刺港股：净亏损扩大至9433万元 核心产品临床成功率存疑

Core Business and Business Model - Exegenesis Bio Inc. focuses on gene therapy and oligonucleotide drug development, established in 2019, with headquarters in the Cayman Islands and operational entities in Hangzhou and Guangzhou, China. The company utilizes two core technology platforms: AAVarta and SODA, targeting rare and common diseases for innovative therapy development [1] - The company has 11 candidate products in development, including treatments for spinal muscular atrophy (SMA), wet age-related macular degeneration (wAMD), and Fabry disease. The core product EXG001-307 (for SMA type 1) is in Phase III clinical trials, while EXG102-031 (for wAMD) and EXG202 (for retinal neovascular diseases) are in Phase I/II [1] Revenue and Financial Performance - As of the first nine months of 2025, the company reported revenue of 1.299 million RMB, primarily from R&D services for overseas clients, indicating a lack of commercialized products and a revenue scale significantly below industry averages [2][3] - The net loss for the first nine months of 2025 reached 94.33 million RMB, continuing a trend of increasing losses primarily due to high R&D expenditures, which accounted for 87.3% of total operating expenses [2] - The gross margin for the first nine months of 2025 was only 10%, reflecting a weak profitability of the current business model, which relies heavily on R&D services rather than product sales [2] Customer and Supplier Concentration - The company’s revenue is entirely dependent on a single overseas client, resulting in a customer concentration of 100%, which poses a significant risk to business continuity [3][7] - The company has a high dependency on suppliers, with the top five suppliers accounting for 47.6% of procurement in the first nine months of 2025, indicating potential supply chain risks [8] Financial Challenges - As of September 2025, the company faced severe financial challenges, with net current liabilities of 1.632 billion RMB and a cash burn rate of approximately 70 million RMB per month, suggesting that existing cash reserves can only sustain operations for about 3.7 months [5][6] Competitive Landscape - Compared to peers, Exegenesis Bio's R&D efficiency is lower, with 145 million RMB spent in 2024 to advance only one Phase III and two Phase I/II products, while competitors have achieved more significant milestones with higher investments [6] - In the SMA treatment market, competition is intense, with Novartis's Zolgensma already approved globally, and domestic competitors advancing more rapidly in clinical trials [6][10] Management and Governance - The company is controlled by a founding team holding 31.16% of voting rights, which may lead to a lack of checks and balances in decision-making due to concentrated ownership [9] - The management team has extensive experience in gene therapy, but there is a lack of transparency regarding their compensation and incentive mechanisms [9] Regulatory and Commercialization Risks - The company faces multiple risks related to clinical development, regulatory approval, and commercialization, particularly with the core product's clinical data showing inferior efficacy compared to competitors [10][11][12] - As a biotechnology company under Chapter 18A, it must meet ongoing R&D requirements post-listing, with potential delisting risks if core product development fails [11] - The company has not yet established a sales team, raising concerns about its commercialization capabilities in a competitive market [12]