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之內容概不負責，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 容而產生或因倚賴該等內容而引致的任何損失承擔任何責任。 Grand Pharmaceutical Group Limited 遠大醫藥集團有限公司* ( 於百慕達註冊成立之有限公司 ) (股份代號: 00512) 自願性公告 曲前列尼爾注射液（20ml : 50mg）獲頒發藥品註冊證書 本公告乃遠大醫藥集團有限公司（「本公司」，連同其附屬公司統稱「本集團」）之 董事會（「董事會」）自願刊發。 董事會欣然公告，本集團用於治療肺動脈高壓（pulmonary arterial hypertension， 「PAH」，WHO 分類 1）的產品曲前列尼爾注射液的新規格（20ml : 50mg），近日獲 得中華人民共和國國家藥品監督管理局（中國藥監局）頒發藥品註冊證書，本產品 20ml:20mg 的規格已於二零二三年三月獲批上市。本產品爲醫保目錄產品，兩種規格 將有助於臨床醫生根據患者的情況提供更加精准的治療方案，這是本集團在心腦血管 急救領域罕見病方向的重大進展。 本集 ...

Financial Data and Key Metrics Changes - The company is preparing to file its New Drug Application (NDA) for bitopertin in October, with a potential PDUFA date between June and October of the following year, marking a significant milestone for the company [4][5] - The company reported a strong reduction in protoporphyrin-9 (PP9) levels with a P-value of less than 0.001, indicating clinically meaningful results from its phase 2 studies [9][10] Business Line Data and Key Metrics Changes - The lead program, bitopertin, is focused on treating erythropoietic protoporphyria, with the company ramping up efforts for its launch [4][5] - The second program is expected to present data on anemia of myelofibrosis and anemia of chronic kidney disease at upcoming conferences [5][6] Market Data and Key Metrics Changes - The company identified approximately 14,000 diagnosed patients in the U.S. for its lead indication, with enrollment in trials not being a problem due to strong patient advocacy [19][24] - The company plans to target the European and Japanese markets, with the genetic prevalence in Japan estimated to be four times higher than in the U.S. [39] Company Strategy and Development Direction - The company is focused on raising disease awareness and ensuring that patients and physicians are informed about available therapies, which is crucial given the historical lack of treatment options [23][24] - The company aims to build a self-sustaining entity driven by revenues from bitopertin to support its broader heme portfolio and explore larger indications [62] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for bitopertin, highlighting positive interactions with the FDA regarding the accelerated approval process [11][12] - The company is well-funded with $650 million on the balance sheet, providing a runway into 2028 to support ongoing trials and commercial preparations [62] Other Important Information - The company has orphan protection for bitopertin, which provides seven years of exclusivity in the U.S., along with method-of-use patents extending until 2042 [37] - The safety profile of bitopertin shows that dizziness occurs in over 50% of patients initially but resolves over time, indicating a manageable side effect [35] Q&A Session Summary Question: Can you recap the interactions with the FDA regarding the accelerated approval path for bitopertin? - The company had a series of meetings with the FDA, which were responsive to the rationale for using PP9 as a surrogate endpoint, leading to the decision to submit an NDA this fall [11][12] Question: What are the key differences between the Apollo and Aurora trials? - Apollo will focus on measuring the time patients can spend in light as a co-primary endpoint, while Aurora primarily assessed the reduction in PP9 levels [18][20] Question: How does the company plan to address the patient population for bitopertin? - The company segments the patient population based on engagement with healthcare providers, identifying about 6,000 engaged patients who regularly seek care [27][28] Question: What is the expected market opportunity for the anemia treatment in myelofibrosis? - The company estimates a market of about 22,000 patients for anemia in myelofibrosis, with potential pricing similar to other anemia treatments in related indications [45] Question: How does the company view its capital position and runway? - The company is well-funded and expects its current capital to support its operations and trials through 2028, aiming to become self-sustaining through revenues from bitopertin [62]

Mirum Pharmaceuticals (MIRM) 2025 Conference Summary Company Overview - **Company**: Mirum Pharmaceuticals - **Focus**: Rare disease therapeutics with three approved medicines - **Current Revenue Guidance**: $490 to $510 million, cash flow positive, financially independent [2][76] Key Products and Market Insights LIVMARLI - **Current Market Penetration**: Approximately 50% in the U.S. and mature European markets for Alagille syndrome [14] - **Patient Population**: Estimated 2,000 to 2,500 prevalent cases in the U.S., with 75% experiencing pruritus [18] - **Incidence Rate**: About 1 in 50,000 births [19] - **Newborn Screening**: Not necessary as most patients present with neonatal jaundice [16] SATEXLY - **Patient Identification Strategy**: Focused on identifying patients with CTX through a dedicated sales team and screening efforts [4] - **Diagnosis Rate**: Literature suggests only about 10% of patients are diagnosed [4] - **Newborn Screening Efforts**: Ongoing pilot projects to include CTX in newborn screening, with promising results from New York [9][10] PFIC (Progressive Familial Intrahepatic Cholestasis) - **Target Demographic**: Older children, adolescents, and adults [27] - **Patient Identification**: Focus on gastroenterologists and hepatologists to identify patients with idiopathic cholestasis [30] Financial Performance and Projections - **Revenue Recognition Model**: Revenue recognized upon family signing for LIVMARLI, reflecting demand sales [17] - **Takeda Partnership**: Significant revenue generation from Japan, with a scaling down of royalties expected over time [35][36] - **Future Profitability**: Potential for Mirum to become a very profitable company in the coming years, assuming successful product launches [76] Research and Development - **Pipeline**: Four clinical trials from three different product candidates, with three believed to be registration trials [2] - **Expanded Basket Study**: Aimed at treating patients with cholestatic pruritus beyond Alagille and PFIC, with an estimated 500 or more patients targeted [49][61] Strategic Planning and Risk Management - **Long-term Strategy**: Discussions on cash flow deployment and potential acquisitions are anticipated as profitability increases [78] - **Risk Appetite**: Expected to evolve with increased profitability, with a focus on validated mechanisms and lower biology risk [80][82] Core Competencies - **Strengths**: Late-stage clinical development, regulatory expertise, and strong commercial capabilities in rare diseases [85][88] Additional Insights - **Market Dynamics**: The company is optimistic about the potential for growth in the rare disease market, particularly with ongoing education and awareness efforts among healthcare providers [70][71] - **Patient Engagement**: Emphasis on building relationships with physicians and patient advocates to enhance product visibility and adoption [85][88]

Core Viewpoint - The article highlights the remarkable stock performance of Beihai Kangcheng, a small-cap biotech company, which has seen its share price increase by over 1700% this year, driven by the approval of its first self-developed rare disease drug and strategic investments [3][4]. Company Overview - Beihai Kangcheng's stock price has dramatically rebounded from a low of 0.315 HKD per share, with the highest price reaching 2.5 HKD per share after the approval of its innovative drug, Goryning (注射用维拉苷酶β) [4][5]. - The company was listed on the Hong Kong Stock Exchange on December 10, 2021, but faced significant challenges initially, including a nearly 27% drop on its first trading day [4][6]. Product Development - Goryning is the first self-developed class 1 innovative drug for rare diseases in China and has successfully passed the preliminary review for inclusion in the commercial insurance innovative drug directory [4][5]. - The drug's development timeline was notably short, taking only 6.5 years from project initiation to market approval, breaking the traditional "30 principle" of drug development [9][12]. Market Dynamics - The article discusses the changing landscape for rare disease drugs in China, with increasing recognition of the innovation capabilities of domestic biotech companies and supportive government policies [4][6]. - The company aims to significantly reduce the annual treatment cost for Goryning by at least 50%, making it more accessible to patients [11][12]. Challenges and Opportunities - Despite the recent success, the potential of rare disease drugs remains underestimated in the domestic market, with Beihai Kangcheng's market capitalization hovering around 1 billion HKD, despite having three approved products [13][14]. - The company is exploring international business development opportunities and aims to engage with multinational pharmaceutical companies to expand its market reach [14].

Summary of Beihai Kangcheng Conference Call Company Overview - **Company**: Beihai Kangcheng - **Focus**: Rare disease sector, with five products launched including Haier Si, Mai Rui Bei, and Wei La Gan En Zhi Bei Ta, covering mainland China, Taiwan, and Hong Kong-Macau regions [2][5][10] Strategic Partnerships - **Collaboration with Baiyang Pharmaceutical**: - Baiyang's exclusive CFO has joined and acquired nearly 15% equity, indicating a new development phase for the company [2][4] - This partnership is expected to influence future adjustments in medical insurance and commercial insurance directories [2][4][7] Product Development and Market Position - **Haier Si**: - The only enzyme replacement therapy globally for Mucopolysaccharidosis Type II, included in the initial selection list for innovative drug insurance payment [2][10] - Significant market potential with a high incidence rate in East Asian populations [10] - **Ge Rui Ning**: - First domestically developed enzyme replacement therapy for Gaucher disease types I and III, aiming to reduce annual treatment costs by at least 50% [2][12][13] - Plans for large-scale promotion starting in early 2026 and discussions for international market entry [2][17] - **CAN204**: - A gene therapy for Duchenne Muscular Dystrophy (DMD) that has passed animal testing and is in talks for international collaboration [2][21][22] Financial Goals and Projections - **Sales Targets**: - Aiming for sales of 200 million, 500 million, and even 1 billion RMB, with expectations of positive cash flow by 2026 [3][23][25] - **Commercialization Outlook**: - No specific sales forecasts for 2025 and 2026 yet, but significant growth is anticipated if innovative drug insurance payments are implemented [20] Market Trends and Global Strategy - **International Market Entry**: - The trend of Chinese innovative drugs entering global markets is irreversible, with a focus on rare diseases [6][9] - Plans to leverage partnerships for regulatory navigation and market entry [14][17] Research and Development - **R&D Focus**: - Continuous innovation in rare disease treatments, with a strategy to develop products with global market potential [9][21] - Emphasis on patient education, diagnostic technology, and updating treatment guidelines [18][19] Future Plans - **Ecosystem Development**: - Building a rare disease ecosystem in collaboration with CROs and CDMOs, and leveraging genetic testing technologies [24][26] - **Financial Management**: - Aiming for cash flow positivity by 2026, with a focus on balancing funding for new projects and operational sustainability [25][26] Conclusion - Beihai Kangcheng is positioned to capitalize on its strategic partnerships and innovative product pipeline in the rare disease sector, with ambitious sales targets and a clear path towards international market expansion and financial sustainability [2][3][6][17][25]

Core Insights - The National Healthcare Security Administration (NHSA) has released the initial review list for the "Commercial Insurance Innovative Drug Directory," marking a significant step in integrating commercial insurance with the national medical insurance system [2][3] - The list includes various high-value innovative drugs, such as CAR-T therapies and rare disease medications, which are expected to enhance market confidence and improve patient outcomes [2][6] Group 1: Commercial Insurance Directory - A total of 141 applications were received for the commercial insurance innovative drug directory, with 121 drug names passing the initial review [3] - The directory features expensive high-value innovative drugs, including CAR-T therapies, which have an average price in the million yuan range [3][4] - Some CAR-T products are being submitted for both basic medical insurance and commercial insurance, providing a second chance for reimbursement if they fail in the first round [4][5] Group 2: Rare Disease Medications - The commercial insurance directory includes 51 products for 52 rare diseases, potentially allowing patients to access previously unaffordable "orphan drugs" through commercial insurance [6] - The example of nusinersen for spinal muscular atrophy (SMA) illustrates the significant cost reduction from nearly 700,000 yuan to approximately 33,000 yuan after entering the insurance system [6] - The number of rare disease drugs in China is expected to increase, with 210 drug development pipelines projected for 2024, 38% of which are in Phase III clinical trials [6][7] Group 3: Market Dynamics and Future Outlook - The integration of commercial insurance with the national medical insurance system is seen as a flexible approach to facilitate access to high-value innovative drugs [7] - The expected results from national negotiations on drug pricing will be announced between October and November 2025, while the commercial insurance directory is set to be finalized by the end of September [7] - The evolving landscape indicates a narrowing gap between the availability and affordability of innovative treatments for patients [7]

Core Insights - Quoin Pharmaceuticals reported a net loss of $(6.28) per American Depositary Share (ADS) for Q2 2025, which was better than the consensus estimate of $(6.82) [1] - The company had zero revenue for the quarter, consistent with expectations, while operating expenses surged due to increased investment in its lead asset, QRX003 [1][5] - Despite the financial losses, the company made significant clinical and regulatory progress, including positive data for QRX003 in Netherton Syndrome and regulatory approvals [1][7] Financial Performance - Q2 2025 EPS (GAAP) was $(6.28), an improvement of 54.1% from $(13.68) in Q2 2024 [2] - Operating expenses reached $3.79 million, a 77.4% increase from $2.14 million in Q2 2024 [2] - Research and Development expenses were $2.05 million, reflecting a 247.5% increase from $0.59 million in Q2 2024 [2] - Cash, cash equivalents, and marketable securities at the end of the period were $7.79 million, down from $14.9 million as of December 31, 2024 [2][6] Clinical and Regulatory Developments - QRX003 showed positive six-month data in Netherton Syndrome, indicating improvements in disease severity and patient quality of life [7] - The U.S. FDA granted clearance for a second pivotal trial and Orphan Drug Designation from the European Medicines Agency, which can provide up to 10 years of market exclusivity [7] - QRX003 also received Rare Pediatric Disease Designation from the FDA, making it eligible for a Priority Review Voucher upon approval [7] Strategic Focus - Quoin Pharmaceuticals focuses on developing therapies for rare dermatological diseases, with QRX003 as its primary asset targeting Netherton Syndrome [3] - The company aims to complete recruitment for the second pivotal QRX003 study by the end of Q1 2026 and plans to submit a New Drug Application in late 2026 [10] - Ongoing collaborations with institutions support the pipeline beyond QRX003, and advocacy campaigns like NETHERTON NOW are expanding [4][8] Market Position - QRX003 is one of the few late-stage assets for Netherton Syndrome, with no approved therapies currently available [9] - Other biotech firms are developing products for Netherton Syndrome, but only Azitra is actively dosing subjects in clinical studies as of March 2025 [9] - Intellectual property protection remains a priority, with patents and licensing arrangements in place for core technologies [9]

Financial Data and Key Metrics Changes - Total revenues for the second quarter reached $128 million, representing a 64% increase compared to the same quarter last year [6][19] - The company raised its full-year revenue guidance for 2025 to a range of $490 million to $510 million, indicating nearly 50% top-line growth [7][13] Business Line Data and Key Metrics Changes - Net product sales for Lidmarley were approximately $128 million, driven by strong demand in both the U.S. and international markets [10][19] - In the U.S., Lidmarley generated about $57 million in net product sales, with increased patient awareness contributing to higher volumes [10][11] - Internationally, Lidmarley sales reached $31 million, supported by expanding reimbursement and strong demand [12][13] Market Data and Key Metrics Changes - The U.S. market for Lidmarley is seeing increased patient penetration, with approximately 70-75% persistence rates after one year for Alagille syndrome patients [25][59] - The company is experiencing growth in the PFIC market due to increased awareness and genetic testing, leading to more diagnoses [11][59] Company Strategy and Development Direction - The company's strategy focuses on commercial execution, scientific innovation, and financial discipline, with a commitment to delivering life-changing medicines for rare diseases [5][20] - The pipeline includes three late-stage studies expected to yield pivotal data over the next 24 months, particularly in primary sclerosing cholangitis [8][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's position and the opportunities ahead, highlighting the strong performance of its medicines and pipeline [20] - The company is optimistic about Lidmarley's potential to exceed $1 billion in revenue, driven by ongoing growth in Alagille syndrome and PFIC indications [14][62] Other Important Information - The company reported operating cash flow positivity for the quarter, with cash and investments totaling $322 million, reflecting a $29 million increase from the end of the previous year [19] Q&A Session Summary Question: What are the overall therapy persistence rates for Lidmarley? - Management indicated that persistence rates for Alagille syndrome patients are around 70-75% after one year, with lower attrition in subsequent years [25] Question: Can you provide details on the expected revenue distribution between Lidmarley and the bile acid business? - Management did not break down guidance by specific products but noted that trends observed year-to-date are expected to continue [33] Question: What are the main drivers of growth in Lidmarley sales? - Key drivers include increased awareness of PFIC and the introduction of a single tablet formulation, which has received positive feedback [39][41] Question: Are there any inventory impacts in the second quarter? - Inventory impacts are primarily relevant to Japan and Takeda, with no significant inventory issues in the U.S. or Europe [44] Question: What is the status of the Fragile X study? - The company has received clearance from the FDA and is on track to initiate the Phase II study by the end of the year [52] Question: How well penetrated is the company in the Alagille and PFIC markets? - The company estimates approximately 50% penetration in the Alagille syndrome market, with ongoing efforts to increase awareness and patient identification [59][60]

Group 1 - Pharming Group N.V. will participate in the Canaccord Genuity 45 Annual Growth Conference in Boston, USA, on August 12-13, 2025 [1] - CEO Fabrice Chouraqui will present on August 12 at 12:00pm EDT/18:00 CEST [1] - Investors can schedule one-to-one meetings with Pharming's management team through the Investor Relations team [1] Group 2 - Pharming Group N.V. is a global biopharmaceutical company focused on patients with rare, debilitating, and life-threatening diseases [2] - The company is involved in the commercialization and development of innovative medicines, including small molecules and biologics [2] - Pharming is headquartered in Leiden, the Netherlands, and operates in over 30 markets worldwide [2]

Core Insights - Agios Pharmaceuticals reported a notable revenue beat in Q2 2025, with GAAP net product revenue of $12.5 million for its lead product PYRUKYND, reflecting strong sales momentum despite increasing costs as the company prepares for major regulatory events and product launches [1][5] Financial Performance - EPS (GAAP) for Q2 2025 was $(1.93), a decline of 14.2% year-over-year from $(1.69) in Q2 2024 [2] - Revenue (GAAP) reached $12.5 million, up 45.4% from $8.6 million in Q2 2024 [2] - Net loss increased to $(112.0 million), a rise of 16.5% compared to $(96.1 million) in Q2 2024 [2][8] - R&D expenses rose to $91.9 million, an increase of 18.7% from $77.4 million in Q2 2024 [2] - SG&A expenses increased by 29.3% to $45.9 million, driven by preparations for regulatory approvals [2][6] Business Overview and Strategy - Agios specializes in therapies for rare blood disorders, focusing on high unmet needs, with PYRUKYND targeting pyruvate kinase deficiency [3] - The company aims to expand PYRUKYND's reach to other rare diseases, including thalassemia and sickle cell disease, through clinical trials and regulatory filings [3] Regulatory and Commercial Developments - The company is preparing for a key regulatory milestone with a PDUFA goal date of September 7, 2025, for PYRUKYND's approval in thalassemia [6][7] - Agios has established a European distribution partnership with Avanzanite Bioscience to launch PYRUKYND across Europe and the UK [6] - The FDA is currently reviewing the application for PYRUKYND in thalassemia, with late-stage trial results for sickle cell disease expected by year-end [7][9] Pipeline and Future Outlook - PYRUKYND product revenue grew 44.6% year-over-year, with unique patients enrolling for treatment reaching 248, a 6% increase from the prior quarter [5] - The company closed the quarter with $1.34 billion in cash and marketable securities, a decrease from year-end 2024 [8] - Management did not provide detailed financial guidance for fiscal 2025 but indicated that the current liquidity position supports plans for new launches and ongoing research investments [10]