Core Viewpoint - The approval of the injection drug Velaglucerase beta (brand name: Gorynin) by the National Medical Products Administration (NMPA) marks a significant milestone as it is the first domestically developed enzyme replacement therapy for Gaucher disease in China, targeting adolescents and adults aged 12 and above with Type I and III Gaucher disease [1][2]. Group 1: Product Approval and Significance - Velaglucerase beta is the only locally developed enzyme replacement therapy for Gaucher disease in China, making it a unique addition to the treatment options available [1]. - The drug is noted for having the broadest range of indications among Gaucher disease treatments currently available [1]. Group 2: Disease Background and Treatment - Gaucher disease is a rare genetic lysosomal storage disorder, with clinical manifestations including hepatosplenomegaly, anemia, bone pain, and neurological complications, potentially leading to life-threatening conditions [2]. - The global prevalence of Gaucher disease is estimated at 0.7 to 1.75 cases per 100,000 people [2]. - Enzyme replacement therapy (ERT) has been the standard treatment for Gaucher disease for 30 years, showing significant efficacy in improving non-neurological symptoms and enhancing the quality of life for patients [2]. Group 3: Development and Technology - The development of Gorynin was facilitated by collaborative efforts with partners like WuXi Biologics, which helped reduce development costs and ensure the drug is safe and effective for patients [2]. - The drug's development utilized second-generation innovative technology, resulting in over a 110-fold increase in production yield and more than a 50% enhancement in enzyme activity, significantly lowering production costs [2]. - WuXi Biologics also developed a cell-based activity assay for quality control, addressing the unique challenges of testing enzyme-based drugs [2].

Core Viewpoint - The approval of domestic enzyme replacement therapy, Gorenin, for Gaucher disease patients in China is expected to significantly reduce treatment costs compared to imported alternatives [1][3][5]. Group 1: Product Overview - Gorenin is the first domestically developed enzyme replacement therapy for type I and III Gaucher disease patients aged 12 and above, fully replacing similar imported products [1][2]. - The drug has been approved for a comprehensive range of indications, covering both type I and III patients, including those who are intolerant or unresponsive to other treatments [2][5]. Group 2: Market Context - The annual treatment costs for imported therapies like imiglucerase can range from $100,000 to $300,000, with domestic adult patients potentially facing costs exceeding 1 million yuan [3][5]. - Approximately 3,000 Gaucher disease patients are reported in China, making it challenging for pharmaceutical companies to justify the investment in research and development for such a small patient population [5][6]. Group 3: Cost Management Strategies - WuXi Biologics, the CRDMO service provider, aims to control the development and production costs of rare disease drugs through innovative technology, allowing for competitive pricing [5][6]. - Gorenin is expected to reduce annual treatment costs for Gaucher disease patients by over 50% compared to imported drugs [3][5]. Group 4: Future Directions - The company plans to push for Gorenin's inclusion in medical insurance systems to ensure broader access for patients [6][7]. - There is a strategic focus on developing international markets for rare disease drugs, leveraging local clinical resources and competitive pricing strategies [7].

Group 1 - The approval of the drug "Gorainin" marks the first domestically developed enzyme replacement therapy for Gaucher disease in China, addressing a significant unmet medical need for patients who previously relied on imported treatments [1][2] - Gaucher disease is a rare genetic lysosomal storage disorder characterized by symptoms such as hepatosplenomegaly, anemia, bone pain, and neurological complications, with severe cases posing life-threatening risks [1] - The drug is specifically indicated for adolescents and adults aged 12 and above with Type I and Type III Gaucher disease, providing a targeted treatment option through intravenous infusion [1] Group 2 - The annual treatment cost for patients using imported drugs ranges from 1.5 million to 2.5 million RMB, making it unaffordable for many, highlighting the need for local production to improve accessibility [2] - The company aims to reduce the annual treatment cost of Gorainin by over 50% compared to imported alternatives, enhancing affordability for patients [2] - The drug's development involved collaboration with WuXi Biologics, utilizing innovative technology to significantly increase production yield and enzyme activity, ensuring better availability for patients [3][5] Group 3 - The drug is part of a pilot project for segmented production of biopharmaceuticals in China, which allows for flexible resource allocation and cost reduction in manufacturing [4][5] - The segmented production model has been successfully implemented for Gorainin, with the active ingredient produced by WuXi Biologics and the formulation handled by its subsidiary, demonstrating a new approach in the industry [5]

Core Viewpoint - The announcement highlights that Beihai Kangcheng-B has received approval from the National Medical Products Administration of China for the marketing of Velaglucerase beta (Goreining, CAN103) on May 15, 2025, for the treatment of Type I and Type III Gaucher disease [1] Group 1: Product Development - The company holds global exclusive rights for the development and commercialization of the product [1] - Goreining is the first domestically developed long-term enzyme replacement therapy for adolescents aged 12 and older and adults with Type I and Type III Gaucher disease [1] Group 2: Disease Overview - Gaucher disease is one of the most common lysosomal storage disorders caused by mutations in the glucocerebrosidase gene located on the long arm of chromosome 1q22 [1] - The disease affects both males and females equally, with clinical forms including Type 0 (perinatal lethal), Type I (chronic non-neuropathic), Type II (acute neuropathic), and Type III (chronic neuropathic), where patients with Type I and III can often live into adulthood [1] Group 3: Market Impact - The introduction of Goreining is expected to completely replace similar imported products, significantly enhancing the accessibility of medication for domestic patients [1]

Group 1 - The core point of the news is that Shiyao Group has signed an exclusive licensing agreement with Cipla USA, Inc. for the commercialization of Irinotecan liposome injection in the U.S., indicating significant commercial potential in the U.S. market [1] - The agreement includes an upfront payment of $15 million, potential milestone payments of up to $25 million for initial commercial sales and regulatory milestones, and up to $1.025 billion in additional commercial sales milestone payments, along with double-digit sales royalties based on annual net sales in the region [1] - This partnership represents a major breakthrough in the company's international strategy, enhancing its global market influence and investor confidence in future profitability [1] Group 2 - The first enzyme replacement therapy for Gaucher disease in China, β-CAN103, has been approved for long-term treatment in adolescents and adults aged 12 and above, filling a gap in the rare disease treatment field [2] - The therapy specifically supplements the enzyme glucocerebrosidase that is deficient in Gaucher disease patients, showcasing the company's research capabilities in rare diseases [2] - The market entry of β-CAN103 is expected to provide a new growth point for the company and enhance its competitiveness and market value in the biopharmaceutical sector [2] Group 3 - Xuanzhu Bio's CDK4/6 inhibitor, Pyrotinib tablets, have received approval for two indications, targeting HR+/HER2- advanced or metastatic breast cancer patients who have progressed after prior endocrine therapy and chemotherapy [3] - This approval marks a significant breakthrough in the company's oncology treatment portfolio, indicating substantial market potential [3] - The new drug approval is anticipated to enhance the company's competitiveness in innovative drugs and provide new performance growth points, boosting investor confidence in its research capabilities and future development [3] Group 4 - WuXi AppTec has entered into a licensing agreement with its major shareholder, Juno, granting non-exclusive rights to develop and commercialize cell therapy products using the JW sLVV production process and related technologies [4] - The total consideration for this agreement is capped at $10 million, which includes non-refundable upfront payments and additional payments [4] - This move not only generates revenue for the company but also strengthens its collaboration with a key shareholder, enhancing investor confidence in its future development [4] Group 5 - Hainan Haiyao has signed a strategic cooperation framework agreement with Malaysia's Aikang International Group, becoming the exclusive agent for Aikang's health products in China [6] - The company will be responsible for promoting, selling, and providing after-sales service for Aikang's high-quality health products in the Chinese market [6] - This partnership is a significant step in expanding the company's business footprint in the health sector, potentially increasing market share and profitability while enhancing investor confidence in its growth potential [6]

CANbridge Pharmaceuticals Corporate Presentation Aug 2023 Disclaimer THIS DOCUMENT OR THE INFORMATION CONTAINED HEREIN IS NOT INTENDED TO AND DOES NOT CONSTITUTE ANY OFFER OR INVITATION, SOLICITATION, COMMITMENT OR ADV ERTISEMENT OF ANY OFFER FOR SUBSCRIPTION, PURHCASE OR SALE OF ANY SECURITIES, NOR SHALL ANY PART OF THIS DOCUMENT FORM THE BASIS OF OR BE RELIED ON IN CONNECTION WITH ANY CONTRACT OR COMMITMENT WHATSOEVER. This document contains strictly conf idential and proprietary inf ormation in relation ...

Financial Performance - Revenue increased by RMB 225 million, a 184% year-over-year increase, primarily due to the commercialization of Nerlynx® in Taiwan in December 2020 and Hunterase® in mainland China in May 2021[134, 145] - R&D expenses decreased by RMB 1165 million, a 424% year-over-year decrease, mainly due to decreased payments to licensing partners[135, 145] - Cash balance increased by RMB 385 million, a 368% year-over-year increase, primarily attributed to the initial public offering in 2H 2021[133, 160] - Administrative expenses increased by RMB 27 million, a 50% year-over-year increase, primarily due to increased administrative employee costs and office expenses[145, 160] - The company's loss for the period was RMB 249 million in 1H 2022[146] Pipeline and Clinical Development - CANbridge identified 539 MPS II patients, and Hunterase has entered into 47 commercial insurance programs[37, 44, 135] - CAN108 NDA has been filed in mainland China and Taiwan for ALGS[41, 62] - The first patient was dosed in the Phase 1/2 trial for CAN103 in adult and adolescent patients with Gaucher disease in July 2022[47, 104] - CAN106 Phase 1 SAD study showed complete blockade of complement function, encouraging further studies in patients with PNH[70, 82] Strategic Initiatives - CANbridge opened a US-based Gene Therapy R&D center in Burlington, MA[47] - CANbridge formed a Complement Disease Scientific Advisory Board[47, 87]

CANbridge Pharmaceuticals FY2022 Annual Results Presentation March 2023 0 Disclaimer THIS DOCUMENT OR THE INFORMATION CONTAINED HEREIN IS NOT INTENDED TO AND DOES NOT CONSTITUTE ANY OFFER OR INVITATION, SOLICITATION, COMMITMENT OR ADVERTISEMENT OF ANY OFFER FOR SUBSCRIPTION, PURHCASE OR SALE OF ANY SECURITIES, NOR SHALL ANY PART OF THIS DOCUMENT FORM THE BASIS OF OR BE RELIED ON IN CONNECTION WITH ANY CONTRACT OR COMMITMENT WHATSOEVER. To be a Global Biopharmaceutical Company This document contains strictly ...

CANbridge Pharmaceuticals 2023 Interim Result Presentation Aug 2023 Disclaimer THIS DOCUMENT OR THE INFORMATION CONTAINED HEREIN IS NOT INTENDED TO AND DOES NOT CONSTITUTE ANY OFFER OR INVITATION, SOLICITATION, COMMITMENT OR ADV ERTISEMENT OF ANY OFFER FOR SUBSCRIPTION, PURHCASE OR SALE OF ANY SECURITIES, NOR SHALL ANY PART OF THIS DOCUMENT FORM THE BASIS OF OR BE RELIED ON IN CONNECTION WITH ANY CONTRACT OR COMMITMENT WHATSOEVER. This document contains strictly conf idential and proprietary inf ormation in ...

Financial Performance - CANbridge Pharmaceuticals reported a revenue of $50 million for the fiscal year ending December 31, 2024, representing a 25% increase compared to the previous year[8]. - Revenue for the year ended December 31, 2023, decreased by RMB 17.8 million or 17.3% to RMB 85.1 million for the year ending December 31, 2024, primarily due to the transition of the distribution of the rare disease drug, He Lai An®, in Hong Kong ending in the second half of 2023[12]. - The company reported a net loss of $8 million for the year, a decrease from a net loss of $12 million in the previous year, indicating improved operational efficiency[8]. - Loss for the year increased by approximately RMB 63.8 million or 16.8% from RMB 378.8 million for the year ended December 31, 2023, to RMB 442.6 million for the year ending December 31, 2024, primarily due to the write-off of right-of-use assets of RMB 88.0 million[12]. - Adjusted loss for the year decreased by RMB 12.0 million or 3.3% from RMB 358.9 million for the year ended December 31, 2023, to RMB 346.9 million for the year ending December 31, 2024[12]. - Gross profit decreased from RMB 64.2 million for the year ended December 31, 2023, to RMB 54.3 million for the year ended December 31, 2024, with a gross margin of 63.8% compared to 62.4% in the previous year[46]. - Other income and gains decreased from RMB 12.7 million for the year ended December 31, 2023, to RMB 7.9 million for the year ended December 31, 2024, primarily due to a reduction in interest income[47]. - Cash and bank balances decreased from RMB 137.5 million as of December 31, 2023, to RMB 10.5 million as of December 31, 2024, primarily due to net cash outflows from operations[58]. - The current ratio as of December 31, 2024, was 9.4%, a significant decrease from 64.0% as of December 31, 2023, primarily due to a reduction in cash and bank balances and an increase in trade payables[61]. - The debt-to-asset ratio as of December 31, 2024, was 26.0%, up from 7.7% as of December 31, 2023[62]. Revenue Guidance and Projections - For the upcoming fiscal year, CANbridge Pharmaceuticals has provided guidance for revenue growth of 20% to 25%, projecting revenues between $60 million and $62.5 million[8]. - The company is actively developing two new products expected to launch in Q3 2025, which are anticipated to contribute an additional $15 million in revenue[8]. - CANbridge Pharmaceuticals is expanding its market presence in Southeast Asia, targeting a 15% market share by the end of 2025[8]. Research and Development - CANbridge Pharmaceuticals plans to increase its R&D budget by 20% in 2025, focusing on innovative therapies for rare diseases[8]. - The company has established a partnership with a leading research institution to accelerate the development of its pipeline, with an expected investment of $3 million over the next two years[8]. - R&D expenses decreased by RMB 5.4 million or 2.1% from RMB 257.2 million for the year ended December 31, 2023, to RMB 251.8 million for the year ending December 31, 2024, mainly due to reductions in employee costs, licensing fees, and depreciation[12]. - The company is committed to advancing its rare disease R&D pipeline while ensuring financial stability amid challenging capital market conditions[15]. - The company has a comprehensive product line consisting of 8 drug assets targeting rare diseases and rare tumor indications, with significant unmet needs and market potential[18]. Product Development and Approvals - The approved rare disease treatment drug, Mai Rui Bei, has achieved significant commercial success in multiple regions and received additional regulatory approvals, reinforcing its leading position in treating ALGS and PFIC[14]. - The near-commercial drug CAN103 for treating Gaucher disease has had its new drug application accepted by the National Medical Products Administration of China, marking an important step in the company's mission against rare diseases[14]. - The company anticipates obtaining marketing approval for CAN103 (injectable Velaglucerase beta) in the first half of 2025, following successful registration checks[20]. - MaiRuiBei® is an oral, minimally absorbed IBAT reversible inhibitor under development for rare cholestatic liver diseases, with over 1,700 human subjects evaluated in clinical trials[29]. - The Phase III study for MaiRuiBei® in PFIC, the largest randomized placebo-controlled trial, included 93 patients across multiple genetic PFIC subtypes, showing significant improvements in itching, serum bile acids, and growth metrics[29]. Workforce and Operational Efficiency - The company plans to streamline its workforce from 67 to 50 full-time employees by mid-March 2025 to reduce operating costs[13]. - As of December 31, 2024, the company has streamlined its workforce to 67 full-time employees, with further reductions to 50 expected by mid-March 2025 to lower operational costs[19]. - The company has invested $5 million in new technology to improve its manufacturing processes, aiming for a 10% reduction in production costs[8]. Market and Industry Insights - The Chinese rare disease industry is expected to thrive under favorable policy support, with significant progress in simplifying approval processes and enhancing reimbursement policies[14]. - The Chinese rare disease drug market was approximately $1.3 billion in 2020, with projections to reach $25.9 billion by 2030, reflecting a compound annual growth rate of 34.5%[22]. - The updated National Rare Disease Catalog in China now includes 207 rare diseases, enhancing the regulatory environment for drug approvals[22]. - Approximately 80% of rare diseases are genetic, making gene therapy a promising treatment avenue for these conditions[23]. Corporate Governance and Management - Fangxin Li, appointed as a non-executive director, has been a senior investment manager at WuXi AppTec Singapore since April 2021, focusing on direct investments in the healthcare sector[77]. - James Arthur Geraghty has approximately 33 years of management experience in business development and strategy, previously serving as a senior vice president at Sanofi S.A.[80]. - Richard James Gregory has over 33 years of experience in R&D, previously serving as executive vice president and chief scientific officer at ImmunoGen Inc.[82]. - Chen Bingjun has over 28 years of experience in corporate finance and investment banking, currently serving as an independent non-executive director for multiple companies[84]. - Hu Lan was appointed as an independent non-executive director in February 2022, responsible for providing independent judgment to the board[85]. Shareholder and Equity Incentive Plans - The company has a maximum number of shares involved in the pre-IPO equity incentive plan of 54,549,230 shares, with 276,200 shares exercised and 9,178,072 shares forfeited during the reporting period[136]. - The board of directors consists of six members, including the executive director and CEO, Dr. Xue Qun[114]. - The company has established a compensation committee to review and approve matters related to the share plans in accordance with listing rules[168]. - The post-IPO share option plan allows for a maximum number of shares to be issued not exceeding 10% of the issued share capital as of June 27, 2024[172]. - The maximum number of stock options that can be granted to service providers under the revised post-IPO stock option plan is 4,248,383, which is approximately 1% of the total issued shares as of December 31, 2024[175]. Customer and Supplier Relationships - The largest customer accounts for 50.8% of the total revenue, while the top five customers account for 100% of total revenue[197]. - The largest supplier represents 57.0% of total procurement, with the top five suppliers making up 76.8% of total procurement[198]. - The company has established policies to monitor and manage trade receivables, ensuring no provisions are necessary for the settlements with the top five customers[200]. - Regular reviews of the recoverable amounts of individual trade receivables are conducted to minimize credit risk[200].