Financial Data and Key Metrics Changes - As of March 31, 2025, the company's cash, cash equivalents, and marketable securities totaled approximately $144.8 million [23] - Revenues for Q1 2025 were zero, compared to approximately $4.9 million for Q1 2024, with the previous revenue being from a $40 million distribution agreement fully recognized by the end of 2024 [23][24] - Operating expenses for Q1 2025 were approximately $16.2 million for R&D, up from $10.1 million in Q1 2024, and general and administrative expenses were approximately $3.1 million, compared to $1.8 million in Q1 2024 [24] - The net loss for Q1 2025 was approximately $24.4 million, compared to a net loss of approximately $9.8 million for Q1 2024 [25] Business Line Data and Key Metrics Changes - The company is focused on the BLA for daramycin, aimed at treating Duchenne muscular dystrophy (DMD) cardiomyopathy, with a strong emphasis on the safety and efficacy data supporting the application [5][8] - The company has been providing daramycin to all open-label extension patients for over three years, with nearly all HOPE-three patients now in open-label extension [15] Market Data and Key Metrics Changes - The company is negotiating with Nippon Shinyaku for the distribution of deramycin in Europe, with the negotiation period extended through the end of Q2 2025 [18] - The company is also exploring opportunities for its technology in other global markets [18] Company Strategy and Development Direction - The company aims to transition from a translational medicine company to a commercial stage entity, actively working with NS Pharma on launch readiness in the U.S. [12] - The company plans to have over 100 patients transition from clinical to commercial product following potential BLA approval [14] - The company is enhancing its medical leadership to guide physicians through the prescribing process for daramycin [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength of the data supporting the BLA and the unmet need in treating DMD cardiomyopathy, which could lead to FDA approval [5][6] - Management noted that the FDA has not indicated any substantive issues with the application, providing confidence in the ongoing review process [30] - The company has a cash balance of approximately $145 million, with a runway extending into 2027 without additional cash infusions [20] Other Important Information - The company is developing its Stealth Exosome Platform technology as part of a next-generation drug delivery platform, although this program has taken a backseat to daramycin [19] - The company is also working on a vaccine candidate for COVID-19 prevention under Project NextGen, with Phase 1 trials set to start in Q3 2025 [20] Q&A Session Summary Question: Has the site inspection in San Diego occurred? - The site inspection is scheduled for the upcoming quarter, and management feels prepared for it [28] Question: What is the status of negotiations with Nippon Shinyaku for Europe? - The company is actively negotiating and evaluating opportunities for commercialization in Europe, while also preparing to work directly with European authorities [37] Question: What are the key drivers of proof for the efficacy data? - The statistical significance of cardiac MRI data is a key driver, showing very little chance that the data is due to chance [46] Question: What is the plan if the FDA issues a CRL for efficacy? - The company would submit data from the HOPE-three trial for skeletal muscle dysfunction if a CRL is issued [55] Question: What is the plan for the PRV voucher? - The current plan is to sell the PRV voucher to strengthen the balance sheet [72]

Financial Data and Key Metrics Changes - The company reported net sales of $4 million for Q1 2025, with a revenue guidance for the full year of $35 million to $45 million from T Cellra sales [4][5][50] - The average turnaround time from apheresis to release was 27 days, beating the target of 30 days [6] - The company achieved a 100% manufacturing success rate from its U.S. T Cell manufacturing center [6][31] Business Line Data and Key Metrics Changes - A total of 21 patients have been treated with T Cellra in 2025, with 13 in Q1 and 8 in early Q2 [4] - The company invoiced 14 T Cellra treatments in 2025 to date, with 6 in Q1 [5] - The company expects to have approximately 30 authorized treatment centers (ATCs) operational by the end of 2025, which is a year earlier than initially anticipated [4][5] Market Data and Key Metrics Changes - The company has seen effective patient access to T Cellra with no patient denials to date [6] - The annual incidence rate for eligible patients diagnosed with synovial sarcoma is approximately 1,000, which aligns with the company's expectations [68] Company Strategy and Development Direction - The company is focused on the successful launch of T Cellra and is preparing for the launch of Letocell, anticipated in 2026 [8] - The company is reviewing strategic options with Cowen as advisors to explore opportunities that benefit patients and shareholders [9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving the revenue guidance based on the number of patients treated and the operational efficiency of ATCs [50] - The company has had three formal meetings with the FDA in the last six months, indicating a positive regulatory environment [28] Other Important Information - The company has implemented cost reduction actions and a restructuring plan that impacted Q1 spending [46] - The gross margin in Q1 was around 78%, with expectations of normalizing to about 70% in the long run [66] Q&A Session Summary Question: Clarification on apheresis patients invoiced - Management confirmed that most apheresis patients invoiced in Q1 were from the prior quarter, with expectations for invoicing the majority in the coming month [10][11] Question: Trends in patient referrals and screening - Management expects incremental growth quarter on quarter without specific seasonalization, driven by increased awareness and onboarding of ATCs [16][18] Question: Impact of regulatory changes - Management noted that the FDA remains engaged and has not indicated any issues with the regulatory process [28] Question: Key learnings from the early launch - The onboarding of treatment centers has occurred faster than anticipated, and the manufacturing success rate has been exceptional [29][31] Question: Financing and liquidity concerns - The company paid down $25 million of financing obligations to manage its balance sheet and leverage [42][45] - The company has less than twelve months of cash runway, and detailed cash guidance was deemed inappropriate due to various influencing factors [46][47]

撰文丨王聪 编辑丨王多鱼 排版丨水成文 嵌合抗原受体巨噬细胞 （CAR-M） 疗法在实体瘤治疗中展现出巨大潜力；然而，CAR-M 在免疫抑制性肿 瘤微环境中的表型再驯化限制了其抗肿瘤免疫能力。 2025 年 4 月 29 日，山东大学 姜新义 教授、 史本康 教授、 荆卫强 等人在 Nature 子刊 Nature Cancer 上发 表了题为： An in situ engineered chimeric IL-2 receptor potentiates the tumoricidal activity of proinflammatory CAR macrophages in renal cell carcinoma 的研究论文。 该研究开发了一种新型 原位 CAR-M 细胞疗法 ，通过原位工程化的嵌合 IL-2 受体增强 CAR-M 细胞的的肿 瘤杀伤活力，用于肾细胞癌的治疗。 论文链接 ： 在这项最新研究中，研究团队报告了一种原位工程化的嵌合白介素（IL）-2 信号受体 （CSR） ，用于可控 地调节嵌合抗原受体巨噬细胞 （CAR-M） 的促炎表型，增强其持续的抗肿瘤免疫能力。 具体而言，研究团队定 ...

Investment Rating - The report maintains a "Buy" rating for King’s Ray Biotechnology [3][9]. Core Views - The company is experiencing positive development across its three main segments, with an expected improvement in profitability. The joint venture Legend Biotech is ramping up production, which is anticipated to contribute profits to the parent company in 2026 [3][9]. Financial Summary - Revenue for 2024 is projected at $595 million, reflecting a 6.1% increase. The group net profit is expected to reach approximately $2.96 billion, marking a significant turnaround from losses, primarily due to a one-time investment gain from the merger with Legend Biotech [9]. - The adjusted net profit from continuing operations is stable at about $59.8 million, up by 2.9% [9]. - Revenue breakdown includes: - Life Sciences segment: $455 million (+10.2%), with an adjusted gross margin of 52% (+5.9 percentage points) [9]. - CDMO segment: $95 million (-13.2%), with an adjusted gross margin of $14 million [9]. - Baisjie segment: $54 million (+24.6%), with an adjusted gross margin of 36.1% (+2.8 percentage points) [9]. - The CAR-T therapy sales are projected to reach nearly $1 billion, with significant new capacity coming online [9]. Growth Catalysts - The CAR-T therapy is expected to exceed sales expectations, and a recovery in investment and financing is anticipated [9]. - The approval of the second-line MM in the U.S. is expected to drive sales growth, with CARVYKTI projected to generate $963 million in revenue for 2024, a 92.6% increase [9].