Summary of Amicus Therapeutics (FOLD) FY Conference Call - June 10, 2025 Company Overview - **Company**: Amicus Therapeutics - **Core Business**: Development of therapies for rare diseases, primarily focusing on Galafold for Fabry disease and Pombility for Pompe disease - **Sales Projections**: Expected to surpass $500 million in sales for Galafold and $100 million for Pombility in 2025 [3][4] Key Points and Arguments Financial Performance and Growth - **Galafold**: - Projected to achieve $1 billion in peak sales with a growth rate of 10% to 15% in 2025 [4] - Currently in a $2 billion global market, expected to grow to $3 billion over the next decade [8][9] - **Pompe Disease Therapies**: - Pombility and Upholda projected to achieve $100 million in sales with a growth rate of 50% to 65% [4] - Anticipated to reach $1 billion in sales in the future [4] - **New Asset - DMX200**: - Licensed from Dimerix for FSGS, a rare kidney disease, with potential blockbuster status [5] - Expected to contribute to significant revenue growth by 2028 [5] Market Position and Competitive Landscape - **Macro Environment**: - Company believes it is well-positioned to navigate macroeconomic challenges affecting the sector [7][8] - Emphasizes the undervaluation of Galafold and the potential for increased diagnoses in the Fabry disease space [8][9] - **Launch Dynamics**: - Initial launch delays due to COVID-19 impacted market entry for Pompe therapies [37] - Positive trends observed in the U.S. market with increasing patient switches from competitors [39][41] Regulatory and Operational Considerations - **Impact of Tariffs and Regulations**: - Minimal impact from tariffs due to low cost of goods for Galafold, which is manufactured in Switzerland [16][18] - Company is adapting to regulatory changes and believes it is insulated from potential negative impacts [20][22] - **FDA Dynamics**: - New leadership at the FDA is expected to support rare disease regulatory reforms, potentially benefiting Amicus [23][24] Guidance and Future Outlook - **Profitability Expectations**: - Guidance for GAAP profitability in the second half of 2025, with revenue growth projected at 15% to 22% [25][26] - Operating expenses expected to remain flat or increase slightly, allowing for positive free cash flow [27][29] - **Long-term Strategy**: - Focus on maintaining financial discipline while expanding the product portfolio [6][12] - Plans to leverage cash flow for new development opportunities in rare diseases [80] Additional Important Insights - **Market Dynamics**: - The company is seeing strong uptake in initial launch countries, with market shares reaching 20-30% in key markets [47] - New countries like the Netherlands are expected to significantly contribute to revenue through patient switches [50][52] - **Patient Diagnosis Trends**: - Increased diagnosis rates for Fabry disease due to lower costs of genetic testing and newborn screenings [64][66] - Potential for significant patient population growth as awareness and testing improve [67][70] Conclusion Amicus Therapeutics is positioned for strong growth in the rare disease market, with a focus on financial discipline and strategic expansion. The company anticipates achieving profitability in the near term while navigating macroeconomic challenges and regulatory changes. The potential for increased patient diagnoses and successful product launches underpins a positive outlook for future revenue growth.

Core Viewpoint - The innovative drug Tofersen injection for treating SOD1-ALS has officially launched in China, marking a significant advancement in the treatment of this rare and fatal neurological disease [1][2]. Group 1: Drug and Treatment Details - Tofersen injection is the first disease-modifying treatment for SOD1-ALS, providing a new hope for patients suffering from this condition [1][2]. - The drug is an antisense oligonucleotide (ASO) that reduces the synthesis of the toxic SOD1 protein, thereby alleviating damage to motor neurons and slowing disease progression [2][3]. - The drug was approved in September 2022 for adult patients with SOD1 gene mutations, addressing a significant unmet need in the treatment of ALS [2][3]. Group 2: Patient and Clinical Insights - The average onset age for SOD1-ALS patients is around 50 years, with a typical presentation of lower limb onset and simultaneous damage to upper and lower motor neurons [3]. - There is an urgent demand for targeted therapies among patients due to the fatal nature of the disease [3]. - The importance of genetic testing for all ALS patients, including those with SOD1 mutations, is emphasized for accurate diagnosis and treatment [2][3]. Group 3: Advocacy and Future Directions - Experts call for the standardization of genetic testing and treatment protocols to ensure timely access to this breakthrough therapy for eligible patients [3]. - There is a collective appeal from advocacy groups to raise awareness and improve access to innovative therapies for rare disease populations [3].

Core Insights - 23andMe, a well-known genetic testing company, is experiencing a dramatic turn of events after filing for bankruptcy three months ago, with co-founder Anne Wojcicki proposing a $305 million acquisition bid to restart the bidding process for the company's assets, including customer genetic data [1][3] Company Overview - 23andMe filed for bankruptcy in March 2025 due to declining consumer demand and a data breach incident in 2023 that exposed sensitive genetic and personal information of millions of customers, leading to significant operational challenges [1][3] - The company’s stock price is currently at $3.965, with a market capitalization reduced to $106 million [3] - In the bankruptcy auction phase, Regeneron Pharmaceuticals initially offered $256 million and was selected as the primary bidder, but Wojcicki's non-profit organization, TTAM Research Institute, later made a competing bid [3] Financial Context - Following its merger with VG Acquisition Corp in June 2021, 23andMe raised $509 million in cash and $250 million in PIPE financing, which highlights the company's previous financial strength before its recent struggles [3] Leadership and Influence - Anne Wojcicki, a successful entrepreneur and former wife of Google co-founder Sergey Brin, has been a prominent figure in Silicon Valley, showcasing resilience by continuing to pursue ownership of 23andMe despite the company's challenges [5][6]

Group 1 - The case of Kathleen Folbigg highlights the intersection of genetics and the judicial system, where rare genetic mutations played a crucial role in overturning a wrongful conviction for infanticide [18][21][24] - The initial conviction was based on the "Meadow's Law," which suggested that the probability of multiple sudden infant deaths in one family was extremely low, leading to the presumption of foul play [2][4][5] - Advances in genetic testing, particularly whole genome sequencing (WGS) and whole exome sequencing (WES), have revolutionized the ability to identify genetic factors contributing to unexplained deaths, shifting the focus from statistical assumptions to molecular evidence [10][22][23] Group 2 - The identification of the CALM2-G114R mutation in Kathleen and her daughters provided a genetic explanation for the sudden deaths, linking them to a hereditary cardiac arrhythmia [11][15][17] - The case underscores the need for integrating genetic testing into forensic investigations, particularly in cases of sudden unexplained deaths, to avoid misjudgments based on indirect evidence [21][22] - The evolution of forensic genetics, including the use of next-generation sequencing (NGS), is transforming the landscape of criminal investigations, allowing for more precise identification of genetic markers associated with diseases [22][23]

Group 1: Company Listings - Furuitek (Zhejiang) Intelligent Technology Co., Ltd. has submitted a listing application to the Hong Kong Stock Exchange, with joint sponsors including CITIC Securities, CICC, Huatai International, and HSBC [1] - Weir Shares plans to issue overseas listed shares (H-shares) and list on the Hong Kong Stock Exchange to accelerate its international strategy and enhance overseas financing capabilities [2] Group 2: Financial Reports - Miniso reported a total revenue of 4.43 billion yuan for Q1 2025, a year-on-year increase of 19%, with a gross margin of 44.2%, the highest for a first quarter [14] - Tongcheng Travel achieved revenue of 4.377 billion yuan in Q1 2025, a year-on-year growth of 13.2%, with adjusted EBITDA reaching 1.159 billion yuan, up 41.3% [15] Group 3: Market Developments - The National Healthcare Security Administration announced price regulations for radiological examination services, with 22 provinces implementing specific pricing plans [4] - Nike plans to raise prices on various products, including footwear and apparel, by $2 to $10 due to the impact of the tariff war [5] Group 4: Corporate Actions - ByteDance issued a compliance reminder to business partners, emphasizing a zero-tolerance policy towards bribery and corruption [7] - Microsoft has officially withdrawn its antitrust lawsuit against its acquisition of Activision Blizzard, marking the end of nearly two years of legal disputes [9]

Core Insights - Lung cancer is the most prevalent and deadly malignancy in China, with approximately 1.0606 million new cases and 733,300 deaths reported in 2022, accounting for 22.0% and 28.5% of all cancer cases respectively [1][3] - Advances in genetic testing and targeted therapies have improved survival rates for patients with rare targets like ALK and ROS1, transforming lung cancer into a manageable chronic disease for some patients [1][2] - There is a pressing need to enhance awareness and understanding of rare targets among clinicians and to ensure equitable access to medical resources for comprehensive lung cancer diagnosis and treatment [1][5] Group 1: Disease Burden and Treatment Advances - Lung cancer accounts for 80%-85% of all cases of non-small cell lung cancer (NSCLC) in China, with ROS1 fusion positivity occurring in about 2% of patients, most of whom are diagnosed at advanced stages [3][4] - The introduction of targeted therapies has shifted the treatment paradigm from chemotherapy to personalized molecular treatments, with FDA-approved therapies targeting mutations such as EGFR, ALK, and ROS1 [4][7] - Research indicates that with appropriate targeted treatment, patients with rare targets can significantly extend their survival, with some achieving over ten years [1][2] Group 2: Challenges in Diagnosis and Treatment - The actual rate of genetic testing for ROS1 fusion is significantly lower than recommended, influenced by regional differences in physician awareness and patient access to testing [5][6] - Economic burdens and the high costs of testing contribute to the underutilization of targeted therapies, with many patients opting for chemotherapy due to delays in obtaining test results [6][8] - There is a need for improved detection methods and faster turnaround times for test results to enhance patient outcomes [6][10] Group 3: Standardization and Education - Standardization and quality control in cancer diagnosis and treatment are critical, with ongoing efforts to improve the skills of clinicians in rural areas through education and training programs [9][10] - The establishment of efficient testing platforms is essential to manage the increasing number of lung cancer patients, requiring collaboration among manufacturers, enterprises, and government [10]

Core Insights - The announcement of former President Biden's prostate cancer diagnosis has heightened public awareness of prostate cancer and its implications for health management [1][2] - Prostate cancer incidence is rapidly increasing in China, particularly among older populations, with significant implications for healthcare strategies and treatment options [2][3] Group 1: Prostate Cancer Trends - Prostate cancer has become the most common malignant tumor among men in Europe and the U.S., with a notable increase in cases in China [2] - In Zhejiang Province, the ranking of prostate cancer among male cancers has risen from fourth to third place in 2023 [2] - The incidence of prostate cancer is highly correlated with age, particularly in men over 70, and is influenced by genetic factors and lifestyle choices [2] Group 2: Early Detection and Diagnosis - Early-stage prostate cancer often presents no obvious symptoms, making PSA screening crucial for early detection [2] - The early diagnosis rate for prostate cancer in China was previously below 30%, but has improved significantly with increased PSA screening, reducing late-stage diagnosis rates from over 50% to around 30% [2] Group 3: Treatment Innovations - Heavy ion therapy offers a new treatment option for localized prostate cancer, providing precise targeting of tumors with minimal damage to surrounding tissues [3] - The five-year survival rate for low-risk patients treated with heavy ion therapy exceeds 90%, and for high-risk patients, tumor control rates improve by 15%-20% compared to photon therapy [3] - Nuclear medicine treatments are being researched to target bone metastases effectively, utilizing radioactive isotopes to minimize damage to normal tissues [4] Group 4: Future Directions - The integration of advanced technologies such as gene testing and AI imaging is expected to enhance screening and treatment efficacy for prostate cancer [4] - The establishment of comprehensive treatment centers for heavy ion therapy and accelerated nuclear drug development in Zhejiang Province is anticipated to significantly improve the five-year survival rates for prostate cancer patients [4] - A shift from passive treatment to proactive health management for men is essential for effective prostate cancer control and treatment [4]

Core Insights - NF1 is increasingly gaining attention in the medical community due to its high incidence in newborns and severe disease burden [1][2] - Early diagnosis and treatment are crucial for NF1 patients, with a focus on multidisciplinary collaboration for comprehensive management [1][3] Group 1: Disease Characteristics - NF1 is an autosomal dominant genetic disorder caused by mutations in the NF1 gene, affecting multiple systems and leading to significant disease burden [1] - Key clinical features include multiple café-au-lait spots, which are important for identification, along with other symptoms like freckles, iris hamartomas, skeletal deformities, and cognitive dysfunction [1][2] Group 2: Patient Statistics and Risks - Approximately 30% to 50% of NF1 patients develop plexiform neurofibromas (PN), with many children showing symptoms at birth [2] - Tumor growth is uncontrolled, with a growth rate of 35.1% per year in children aged 3-5 years, and 8% to 13% of tumors may undergo malignant transformation, with a five-year survival rate of less than 50% post-malignancy [2] Group 3: Diagnosis and Treatment - Diagnosis is primarily based on clinical symptoms, with genetic testing and imaging techniques like MRI used when symptoms are not apparent [2][3] - Treatment varies based on tumor location and impact on the patient, with options including laser treatment for superficial lesions and surgical removal for deep tumors, which pose significant surgical risks [3] Group 4: Management Challenges - Long-term follow-up and standardized management are essential for improving the quality of life for NF1 patients, facing challenges such as difficulties in drug reimbursement and insufficient pediatric medication availability [3] - Multidisciplinary involvement is necessary for effective diagnosis and treatment, requiring collaboration among various specialties including pediatrics, ophthalmology, dermatology, oncology, and surgery [3]

2025 年 05 月 13 日 投资要点 分析师：孟之绪 执业编号：S0300524080001 电话： 邮箱：mengzhixu@ykzq.com 证券研究报告 | 策略点评 投资策略研究 粤开市场日报-20250513 今日关注 指数涨跌情况：今日 A 股主要宽基指数涨少跌多。截止收盘，沪指涨 0.17%， 收报 3374.87 点；深证成指跌 0.13%，收报 10288.08 点；创业板指跌 0.12%， 收报 2062.26 点；科创 50 跌 0.15%，收报 1009.68 点。总体上全天个股涨少 跌多，Wind 数据显示，全市场 1940 只个股上涨，3233 只个股下跌，237 只个 股收平。沪深两市今日成交额合计 12915 亿元，较上个交易日缩量约 169 亿 元。 行业涨跌情况：今日申万一级行业涨多跌少，银行、美容护理、医药生物、 交通运输和煤炭行业领涨，涨幅分别为 1.52%、1.18%、0.90%、0.72%和 0.62%， 国防军工、计算机、机械设备、电子和通信行业领跌，跌幅分别为 3.07%、0.80%、 0.66%、0.64%和 0.61%。 板块涨跌情况：今日涨幅居前概念 ...

Core Insights - Berry Genomics (000710) reported a revenue of 1.078 billion yuan for 2024, representing a year-on-year decline of 6.36% [1] - The net loss attributable to shareholders was 192 million yuan, compared to a net loss of 427 million yuan in the same period last year [1] - The basic earnings per share were -0.5443 yuan per share [1] - The company announced plans not to distribute cash dividends, issue bonus shares, or increase capital through reserves [1]