Financial Performance - Revenue increased by RMB 23.9 million or 30.3% to RMB 102.9 million for the year ended December 31, 2023, compared to RMB 79.0 million for the year ended December 31, 2022, primarily due to increased sales of HaiRuiSi® and MaiRuiBei®[4] - Loss for the year decreased by RMB 104.7 million or 21.7% to RMB 378.8 million for the year ended December 31, 2023, compared to RMB 483.5 million for the year ended December 31, 2022, attributed to increased revenue and reduced R&D and administrative expenses[4] - Adjusted loss for the year decreased by approximately RMB 97.8 million or 21.4% to RMB 358.9 million for the year ended December 31, 2023, from RMB 456.7 million for the year ended December 31, 2022[5] - Gross profit for the year ended December 31, 2023, was RMB 64.2 million, compared to RMB 48.9 million for the year ended December 31, 2022[6] - The group reported a net loss of RMB 378,837,000 for the year ended December 31, 2023, with a net debt of RMB 39,788,000 as of the same date[28] - Customer contract revenue for 2023 reached RMB 102,871,000, a 30.3% increase from RMB 78,972,000 in 2022[43] - Other income totaled RMB 12,659,000 in 2023, slightly down from RMB 12,883,000 in 2022[47] - The company reported a loss attributable to equity holders of RMB 378,837,000 for 2023, compared to a loss of RMB 483,475,000 in 2022, indicating a reduction in losses[61] Research and Development - R&D expenses decreased by RMB 54.0 million or 17.4% to RMB 257.2 million for the year ended December 31, 2023, from RMB 311.2 million for the year ended December 31, 2022, mainly due to reduced prepayments and milestone payments to partners, as well as decreased testing and clinical trial costs[4] - The company has developed a pipeline consisting of 14 drug assets, including 4 marketed products and 3 late-stage clinical candidates, targeting rare diseases and tumors with high unmet needs and significant market potential[75] - The company has received IND approval for CAN008 to treat GBM and completed patient recruitment for the Phase II clinical trial in March 2023[79] - The company is developing two gene therapy products using LogicBio Therapeutics' licensed AAV sL65 capsid for treatments of Pompe disease and Fabry disease[79] - The company has achieved positive topline results in the Phase Ib trial for CAN106, showing dose-dependent reductions in LDH and improvements in hemoglobin levels for PNH patients[77] - The company plans to report Phase II clinical trial data for CAN008 in GBM patients in the first half of 2024, with potential for priority review application in Greater China[145] - The Phase II clinical trial for CAN103 targeting Gaucher disease has completed patient recruitment, with plans to submit a new drug application in the second half of 2024[146] Financial Position and Liquidity - Cash and bank balances decreased to RMB 137.5 million as of December 31, 2023, from RMB 463.1 million as of December 31, 2022[18] - The company’s bank loans and other borrowings amounted to RMB 30.3 million as of December 31, 2023, down from RMB 37.6 million as of December 31, 2022[155] - The company has secured backup loans from several banks, with RMB 22 million already drawn down, and is in discussions to further withdraw bank loans[28] - The board believes that, considering the plans and measures in place, the group will have sufficient operating funds to meet its financial obligations for the next 12 months[30] - The company is actively negotiating with external parties to secure new financing sources or strategic capital investments to improve liquidity[28] - The company has implemented strict credit control measures to minimize credit risk associated with trade receivables[70] Market and Regulatory Environment - The global rare disease drug market grew from $109 billion in 2016 to $135.1 billion in 2020, with a projected compound annual growth rate (CAGR) of 11.0%, reaching $383.3 billion by 2030[80] - The Chinese rare disease drug market was approximately $1.3 billion in 2020, with projections to reach $25.9 billion by 2030, reflecting a CAGR of 34.5%[81] - The National Rare Disease Catalog in China has expanded to cover 207 rare diseases as of 2023, enhancing the regulatory environment for rare disease treatments[83] - The favorable regulatory environment in China, including streamlined approval processes and compensation policies, is expected to benefit the rare disease industry significantly[83] - The National Medical Products Administration of China has proposed a market exclusivity period of up to 12 months for the first approved pediatric new drug and up to 7 years for rare disease drugs under certain conditions[84] Operational Developments - The company appointed Dr. Jason West as Vice President and Head of Gene Therapy Research in May 2023, bringing expertise in gene therapy development and platform innovation[3] - The company has established major operational centers in Beijing and Shanghai, with a commercialization team in place to support product launches and market expansion[119] - The company has streamlined its workforce to 100 full-time employees, with over 70% having experience in multinational biopharmaceutical companies, enhancing its operational expertise in the rare disease sector[76] - The company is exploring production strategies for gene therapies to achieve high quality and capital efficiency, planning to utilize CDMO for further development[118] Product Approvals and Clinical Trials - The company has received marketing approval for MaiRuBei® (CAN108) in mainland China, Hong Kong, and Taiwan, becoming the first and only approved treatment for ALGS cholestatic pruritus in these regions[94] - MaiRuBei® has shown significant improvements in serum bile acids and itching in clinical trials, with over 200 children treated[93] - CAN106, a novel long-acting monoclonal antibody targeting complement C5, has demonstrated good pharmacokinetic and pharmacodynamic characteristics in clinical data[96] - CAN008 showed a 57% progression-free survival (PFS) rate at 12 months for the 400 mg dose, with a significant improvement in PFS from 4 months to 6 months in recurrent GBM patients[100] - CAN106 has received orphan drug designation from the FDA for the treatment of MG, providing benefits such as a 50% tax credit for eligible clinical trials and a 7-year market exclusivity period post-approval[104] - CAN008 has been granted orphan drug designation by the FDA and EMA, and is currently undergoing a Phase II trial in China for newly diagnosed GBM patients, with results expected in the first half of 2024[108] Cost Management - Cost control measures are being implemented, including prioritizing pipeline adjustments and reducing employee costs[28] - Selling and distribution expenses decreased by RMB 3.1 million to RMB 83.7 million, attributed to improved efficiency in sales activities[128] - The total employee cost for the reporting period was approximately RMB 126.9 million, down from RMB 158.6 million in 2022[199]

Financial Performance - R&D expenses decreased by approximately RMB 15.3 million, or 9.7%, to RMB 143.0 million for the six months ended June 30, 2023, compared to RMB 158.3 million for the same period in 2022[40]. - Adjusted loss for the six months ended June 30, 2023, decreased by RMB 24.5 million, or 10.7%, to RMB 204.4 million, compared to RMB 228.9 million for the same period in 2022[41]. - The company reported a loss of RMB 218.2 million for the six months ended June 30, 2023, a decrease of approximately RMB 30.8 million or 12.4% compared to RMB 249.0 million for the same period in 2022, primarily due to increased revenue and reduced sales, distribution, R&D, and administrative expenses[67]. - Revenue increased from RMB 34.7 million in the six months ending June 30, 2022, to RMB 43.1 million in the same period in 2023, primarily due to increased sales of HaiRuiSi® and MaiRuiBei®[95]. - The company's gross profit increased from RMB 22.2 million to RMB 26.7 million, with a gross margin of 62.0% for the six months ended June 30, 2023[138]. - Other income rose from RMB 6.4 million to RMB 8.5 million, mainly due to increased bank interest income[139]. - Administrative expenses decreased from RMB 55.6 million to RMB 48.2 million, attributed to lower employee costs and office expenses[123]. Market and Product Development - The Chinese rare disease drug market is projected to reach USD 25.9 billion by 2030, with a compound annual growth rate of 34.5%[46]. - The company is developing two gene therapy products using AAV sL65 capsid vectors for Fabry disease and Pompe disease[45]. - The company completed recruitment for a Phase 2 study of MaiRuiBei for biliary atresia, with topline data expected in the second half of 2023[33]. - The company has obtained global exclusive rights for the development, production, and commercialization of a second-generation gene therapy for SMA from the University of Massachusetts Chan Medical School[45]. - The company is actively involved in the development of therapies for rare diseases, which are characterized by unique features that create an efficient market for treatment development[45]. - The company plans to launch MaiRuiBei® (CAN108) nationwide in China in January 2024 and is negotiating for inclusion in the National Reimbursement Drug List (NRDL) in the second half of 2023[61]. - The company intends to submit a new drug application for MaiRuiBei® for patients with Progressive Familial Intrahepatic Cholestasis (PFIC) in the second half of 2023[62]. - CAN103 is the first ERT developed for Gaucher Disease (GD) to reach clinical stage in China[65]. - CAN008 is being developed as a first-line treatment for GBM patients, aiming to prevent tumor cell growth and restore immune function[85]. - The company anticipates the commercialization of CAN008 within the next three years, following regulatory approval[87]. Clinical Trials and Research - Preliminary positive topline data from the ongoing Phase 1b study of CAN106 for PNH patients in China shows effective blocking of complement C5 and good safety and tolerability[63]. - The independent data monitoring committee has completed a mid-term analysis of the Phase 2 study of CAN008 for newly diagnosed GBM patients in China, recommending continuation without changes to the current trial design[64]. - Preliminary positive results from the Phase 1b study of CAN106 for PNH treatment were reported in June 2023, demonstrating dose-dependent pharmacokinetics and maintaining free C5 levels below 0.5 µg/mL in all subjects[83]. - The company has received orphan drug designation from the FDA for CAN106 for the treatment of myasthenia gravis, which includes benefits such as tax credits and market exclusivity[84]. - The company has received orphan drug designation for CAN008 from the FDA and EMA, and it is classified as a first-class new drug in China, with ongoing Phase II trials for GBM[113]. - CAN103 is the first ERT in clinical trials for treating Gaucher disease in China, with the first patient dosed in an I/II trial[115]. - The company has completed part A of a multi-center Phase I/II clinical trial and commenced part B in Q1 2023[89]. Financial Position and Funding - Cash and bank balances as of June 30, 2023, were RMB 283.6 million, a decrease from RMB 463.1 million as of December 31, 2022, primarily due to cash outflows from operations[145]. - The current ratio as of June 30, 2023, was 103.6%, down from 181.6% as of December 31, 2022, attributed to a decrease in cash and an increase in trade payables[148]. - The debt-to-asset ratio as of June 30, 2023, was 6.1%, compared to 5.4% as of December 31, 2022, indicating a slight increase in leverage[149]. - The company may need to pursue further financing through public or private equity offerings, bond financing, and other sources to support ongoing business expansion and R&D efforts[146]. - The net proceeds from the global offering amounted to HKD 604.0 million after deducting underwriting fees, commissions, and estimated expenses[158]. - Approximately 45.4% of the proceeds will be allocated for the research and development of the core product candidate CAN008, including planned clinical trials and milestone costs[158]. - About 24.0% will be used to fund major products and candidates in the product pipeline[158]. - The remaining 16.8% of the net proceeds will be allocated for R&D and other general business purposes[165]. Corporate Governance and Shareholder Information - The company has a total of 39,337,839 unexercised share options under the pre-IPO equity incentive plan, representing approximately 9.27% of the total issued shares[188]. - As of June 30, 2023, there were 40,006,786 share options available for exercise under the pre-IPO equity incentive plan, accounting for about 9.43% of the total issued shares[192]. - Dr. Xue holds a beneficial interest in 733,050 shares and has been granted options for 9,481,420 shares under the pre-IPO equity incentive plan[194]. - As of June 30, 2023, no directors or senior executives held any interests or short positions in the company's shares that require disclosure under the Securities and Futures Ordinance[196]. - CTX Pharma Holdings Limited owns 26,042,380 shares, representing approximately 6.14% of the total shares[197]. - WuXi AppTec holds 40,346,960 shares, accounting for approximately 9.51% of the total shares[197]. - RA Capital Management, L.P. holds a total of 60,235,590 shares, which is approximately 14.19% of the total shares[197].

香港交易及結算所有限公司及香港聯合交易所有限公司對本公告的內容概不負責，對其準確性 或完整性亦不發表任何聲明，並明確表示，概不就因本公告全部或任何部分內容而產生或因依 賴該等內容而引致的任何損失承擔任何責任。 CANbridge Pharmaceuticals Inc. 北 海 康 成 製 藥 有 限 公 司 （於開曼群島註冊成立的有限公司） （股份代號：1228） 截至2023年6月30日止六個月 中期業績公告 北海康成製藥有限公司（「本公司」）董事會（「董事會」）欣然公佈本公司及其附屬 公司（「本集團」或「我們」）截至2023年6月30日止六個月（「報告期間」）的未經審 核簡明綜合業績，連同截至2022年6月30日止六個月的比較數字如下。 於本公告內，「北海康成」或「我們」指本公司及（如文義另有所指）本集團。本公 告所載若干金額及百分比數字已經約整，或約整至小數點後一位或兩位數。任何 表格、圖表或其他地方所列總數與金額總和之間的任何差異乃因約整所致。 業務摘要 海芮思®(CAN101)，一種用於治療黏多糖貯積症II型（MPS II，亦稱為亨特綜合 症）的酶替代療法（ERT）。黏多糖貯積症II型自2018年 ...

CANbridge Pharmaceuticals (01228) Update / Briefing June 27, 2023 07:00 AM ET Speaker0 Good day, ladies and gentlemen. Welcome to Cambridge's Omopuribart CAM One Zero Six phase one b preliminary data readout call. It's our great honor to have the following management with us here today. Doctor James Schreff, founder, chairman, and CEO of Cambridge Pharmaceuticals. Doctor Gerald Cox, chief development strategist and interim chief medical officer. Glen Hassan, chief financial officer, and Pauline Lee, senior ...

CANbridge Pharmaceuticals (01228) Update / Briefing June 07, 2023 12:00 PM ET Speaker0 Good day, ladies and gentlemen. Welcome to Cambridge's Live Marley Mainland China approval call. It's our great honor to have the following management with us here today. Doctor James Hsu, founder, chairman, and CEO of Cambridge Pharmaceuticals. Doctor. Gerald Cox, Chief Development Strategist Marcelo Tureski, Chief Commercial Officer and Glenn Hassan, Chief Financial Officer. Management team will give us a presentation o ...

北海康成製藥有限公司 CANbridge Pharmaceuticals Inc. （於開曼群島註冊成立的有限公司） 股份代號 Stock Code : 1228 ( Incorporated in the Cayman Islands with limited liability ) 年報 目錄 2 釋義 6 公司資料 8 財務摘要 10 主席報告 13 管理層討論及分析 31 董事及高級管理層履歷 38 董事會報告 77 企業管治報告 94 獨立核數師報告 99 綜合損益表 100 綜合全面收益表 101 綜合財務狀況表 103 綜合權益變動表 104 綜合現金流量表 106 財務報表附註 釋義 在本報告內，除文義另有所指外，以下詞彙具有下列涵義。該等詞彙及其定義未必與任何業內標準定義相符， 亦未必可直接與其他在本公司相同行業內經營的公司所採用的同類詞彙比較。 | 「股東週年大會」 | 指 | 本公司謹訂於2023年6月27日（星期二）上午九時正假座中國上海浦東新區 浦東大道535號上海裕景大飯店四樓雪松廳及在線鏈接（ http://meetings. | | --- | --- | --- | | | ...

CANbridge Pharmaceuticals (01228) H2 2022 Earnings Call March 31, 2023 12:00 PM ET Speaker0 Good morning, everyone. Welcome to Cambridge Pharmaceuticals twenty twenty two twelve Month Annual Results Conference Call. Today, it's a great honor to have Doctor. James Shre, Founder, Chairman and CEO of Cambridge Pharmaceuticals Doctor. Gerald Cox, Chief Medical Officer and Chief Development Strategist Glenn Hassan, Chief Financial Officer and Sophie Hsieh, Director of Investor Relations. Management team will giv ...

香港交易及結算所有限公司及香港聯合交易所有限公司對本公告的內容概不負責，對其準確性 或完整性亦不發表任何聲明，並明確表示，概不對因本公告全部或任何部分內容而產生或因倚 賴該等內容而引致的任何損失承擔任何責任。 CANbridge Pharmaceuticals Inc. 北 海 康 成 製 藥 有 限 公 司 （於開曼群島註冊成立的有限公司） （股份代號：1228） 截至2022年12月31日止年度的年度業績公告 北海康成製藥有限公司（「本公司」）董事會（「董事會」）欣然宣佈本公司及其附屬 公司（「本集團」或「我們」）截至2022年12月31日止年度（「報告期間」）的經審核綜 合年度業績，連同截至2021年12月31日止年度的比較數字如下。本集團於報告期 間的綜合財務報表已由審核委員會審閱，並經本公司核數師安永會計師事務所審 核。 於本公告內，「我們」指本公司及（如文義另有所指）本集團。本公告所載若干金額 及百分比數字已經約整，或約整至小數點後一位或兩位數。任何表格、圖表或其 他地方所列總數與金額總和之間的任何差異乃因約整所致。 業務摘要 本集團在其藥物管線及業務運營方面取得重大進展，包括以下里程碑及成就： ...

2O22 中期報告 北海康成製藥有限公司 CANbridge Pharmaceuticals Inc. （於開曼群島註冊成立的有限公司） 股份代號 : 1228 目錄 2 釋義 5 公司資料 7 業務摘要 10 財務摘要 11 管理層討論及分析 29 其他資料 46 獨立審閱報告 47 中期簡明綜合損益表 48 中期簡明綜合全面收益表 49 中期簡明綜合財務狀況表 51 中期簡明綜合權益變動表 52 中期簡明綜合現金流量表 54 中期簡明綜合財務資料附註 釋義 在本報告內，除文義另有所指外，以下詞彙具有下列涵義。該等詞彙及其定義未必與任何業內標準定義相符， 亦未必可直接與其他在本公司相同行業內經營的公司所採用的同類詞彙比較。 | --- | --- | --- | |------------------|-------|------------------------------------------------------------------------------------| | | | | | 「審核委員會」 | 指 | 董事會審核委員會 | | 「董事會」 | 指 | 本公司董事會 | | 「 ...

CANbridge Pharmaceuticals Inc. 北海康成製藥有限公司 （於開曼群島註冊成立的有限公司） （Incorporated in the Cayman Islands with limited liability） 股份代號 Stock Code : 1228 2021 年報 目錄 | --- | --- | |-------|----------------------| | | | | 2 | 釋義 | | 5 | 公司資料 | | 7 | 財務摘要 | | 9 | 主席報告 | | 11 | 管理層討論及分析 | | 25 | 董事及高級管理層履歷 | | 31 | 董事會報告 | | 57 | 企業管治報告 | | 75 | 獨立核數師報告 | | 80 | 綜合損益表 | | 81 | 綜合全面收益表 | | 82 | 綜合財務狀況表 | | 84 | 綜合權益變動表 | | 85 | 綜合現金流量表 | | 87 | 財務報表附註 | 釋義 在本報告內，除文義另有所指外，以下詞彙具有下列涵義。該等詞彙及其定義未必與任何業內標準定義相符， 亦未必可直接與其他在本公司相同行業內經 ...