Company Overview - CANbridge Pharmaceuticals aims to be a global biopharmaceutical company with a foundation in China, delivering life-changing therapies to patients[7] - The company has raised a total capital of $357 million since 2015 through multiple funding rounds and an IPO[12, 13] - CANbridge has a commercialization team of over 40 experienced professionals in Greater China[30] Pipeline and Products - CANbridge has 2 marketed rare disease products and 4 late-stage clinical programs[19] - The company anticipates 3 registration filings/approvals in the next 12 months for LIVMARLI in MPS II ALGS in Mainland China, ALGS in Hong Kong, and ALGS in Taiwan[19] - Hunterase has entered into 109 cities' commercial insurance program ("Huiminbao") as of June 30, 2023, covering a population of 586 million in China, with 72% of Hunterase treated patients covered by commercial insurance[32, 63] - Livmarli achieved $751 million in net product sales in the first full fiscal year of its U S launch[69] Market and Financials - The global rare disease drug market was $259 billion in 2020 and is projected to reach $383 billion by 2030[40] - China's contribution to the global rare disease market is currently less than 1%[40] - Orphan drugs achieved combined sales of $173 billion globally in 2022[43] - The company identified 739 MPS II patients[61] - The company's revenue increased by RMB 84 million year-over-year due to increased sales of Hunterase and Livmarli[163]

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]