Company Highlights - Cidara Therapeutics Inc. (CDTX) reached a 52-week high of $87.19 as it advances its development plan for CD388, a non-vaccine preventive treatment for seasonal influenza, with enrollment in a Phase 3 study expected to start by September 2025 targeting 6,000 participants [1] - Maze Therapeutics Inc. (MAZE) saw its stock rise to a 52-week high of $27.09 following positive Phase 1 clinical results for MZE782, an oral therapy for Phenylketonuria and chronic kidney disease, with plans to enter Phase 2 studies in 2026 [3][4] - Medpace Holdings Inc. (MEDP) is set to report Q3 2025 financial results on October 22, 2025, and has raised its full-year guidance twice, indicating strong operational momentum, with the stock hitting a 52-week high of $506.55 [5][6] - Monopar Therapeutics Inc. (MNPR) rebounded from potential Nasdaq delisting to a 52-week high of $73.75, planning to seek approval for ALXN1840 for Wilson disease in early 2026, supported by long-term clinical data [7][8] - Prenetics Global Limited (PRE) projects 2025 revenue between $85 million and $100 million, a significant increase from $30.6 million in 2024, with its new wellness brand IM8 Health on track for $100 million in annual recurring revenue [9][10] - Protagonist Therapeutics Inc. (PTGX) is nearing a major milestone with Icotrokinra for psoriasis, having submitted a regulatory application to the FDA and EMA in July 2025, with the stock reaching a 52-week high of $66.70 [11][12] - uniQure N.V. (QURE) announced positive results from its Phase I/II study of AMT-130 for Huntington's disease, planning to submit a Biologics License Application in Q1 2026, with the stock soaring over 200% to a 52-week high of $51.21 [13][14] - Rezolute Inc. (RZLT) surged to a 52-week high of $9.02 ahead of topline results from its pivotal Phase III trial for hypoglycemia treatment, expected in December [15][16] - United Therapeutics Corp. (UTHR) reached a 52-week high of $442.01 as anticipation builds for data from the TETON-2 study of nebulized Tyvaso in idiopathic pulmonary fibrosis, with positive results reported earlier [17][18]

Market Overview - U.S. stocks experienced a decline, with the Nasdaq Composite falling over 100 points on Wednesday. The Dow decreased by 0.35% to 46,129.26, the NASDAQ fell by 0.47% to 22,468.39, and the S&P 500 dropped by 0.40% to 6,630.54 [1] - Energy shares saw an increase of 1.9% on Wednesday, while materials stocks fell by 1.5% on Tuesday [1] Commodity Market - In commodity trading, oil prices rose by 2.5% to $64.97, while gold prices decreased by 1.3% to $3,767.30. Silver fell by 1.1% to $44.105, and copper increased by 3.8% to $4.8220 [4] European Market - European shares were mostly higher, with the eurozone's STOXX 600 rising by 0.01%, Spain's IBEX 35 Index increasing by 0.2%, London's FTSE 100 up by 0.29%, Germany's DAX 40 rising by 0.23%, and France's CAC 40 falling by 0.57% [5] Asian Market - Asian markets closed mostly higher, with Japan's Nikkei 225 gaining 0.30%, Hong Kong's Hang Seng surging by 1.37%, China's Shanghai Composite rising by 0.83%, while India's BSE Sensex fell by 0.47% [6] Company News - uniQure N.V. shares surged by 235% to $45.79 after announcing a $175 million non-dilutive senior secured term loan facility with Hercules Capital [8] - SHF Holdings, Inc. saw its shares increase by 120% to $7.18 following a $150 million common stock purchase agreement with CREO [8] - Lithium Americas Corp. shares rose by 97% to $6.05 after reports that Trump officials are seeking an equity stake in the company as part of a $2.26 billion loan renegotiation for the Thacker Pass lithium project [8] - WORK Medical Technology Group LTD shares plummeted by 88% to $0.1081 after partnering with Hong Kong Web3.0 Standardization Association for blockchain and RWA innovations [8] - Firefly Aerospace Inc. shares decreased by 33% to $0.7142, and Zhengye Biotechnology Holding Limited shares fell by 28% to $5.01 [8] Economic Indicators - Sales of new single-family homes in the U.S. increased by 20.5% from the previous month to an annualized rate of 800,000 units in August [10] - Building permits in the U.S. declined by 2.3% to an annualized rate of 1.330 million in August [10] - The volume of mortgage applications rose by 0.6% from the previous week in the week ending September 19 [10]

Market Overview - U.S. equities experienced a pullback on September 24, 2025, after a multi-day rally, with major indexes drifting lower as investors took profits and digested comments from Federal Reserve Chair Jerome Powell regarding elevated asset valuations [1][3] - The S&P 500 was down 0.3% to close at 6,656.92, following a 0.6% decline on Tuesday, while the Nasdaq Composite fell 0.4% to 22,573.47, and the Dow Jones Industrial Average decreased by 0.2% to 46,292.78 [2] Sector Performance - The Energy sector rose by 2% on September 24, supported by a 2% increase in West Texas Intermediate crude oil futures, which reached $64.75 per barrel [4] - The Information Technology and Materials sectors were among the worst performers, both down approximately 1% in afternoon trading, reflecting a broader pullback in growth-oriented assets [5] Company News - Micron Technology (MU) saw a decline of roughly 4% despite reporting record quarterly sales, attributed to profit-taking after a substantial year-to-date gain of 97.7% [13] - Alibaba (BABA) shares surged nearly 9% after announcing plans to increase its AI infrastructure budget beyond $53 billion, indicating strong investor interest in AI investments [13] - Freeport-McMoRan (FCX) plummeted over 10% after lowering its third-quarter sales outlook for copper and gold, alongside a tragic incident in Indonesia [13] - Boeing Co. (BA) gained 2% following an $8 billion agreement to deliver 22, 787 Dreamliners to Uzbekistan Airways [13] - uniQure (QURE) experienced a remarkable surge of 241% after releasing positive results from its Huntington's gene therapy [13] - Nike (NKE) shares are trading just under $71, with analysts anticipating significant price increases ahead of its upcoming quarterly update [13] - Adobe (ADBE) shares fell after a downgrade from Morgan Stanley to 'Equal-Weight' [13] Upcoming Market Catalysts - Investors are monitoring the upcoming release of the U.S. core Personal Consumption Expenditures (PCE) price index on September 26, which is a key inflation gauge [7] - Flash Purchasing Managers' Index (PMI) surveys and revised U.S. GDP numbers are also on the economic calendar, providing insights into economic growth and inflation trends [7][9]

PresentationGood day, and welcome to the top line results for AMT-130 in Huntington's disease. [Operator Instructions] As a reminder, this call may be recorded. I would now like to turn the call over to Chiara Russo, Senior Director, Investor Relations.Chiara Russo Thank you. This morning, uniQure announced pivotal data on patients treated with our investigational gene therapy, AMT-130 in our ongoing Phase I/II clinical trials in Huntington's disease, taking place in the U.S., EU and the U.K. This 3-year u ...

Uniqure (NASDAQ: QURE) shares opened nearly 200% up on Wednesday – marking one of the most dramatic single-day moves in biotech this year. The surge follows a trifecta of bullish development: a breakt... ...

智通财经APP获悉，周三，uniQure(QURE.US)暴涨超186%，创四年新高，报39.11美元。消息面上，9 月24日，该公司宣布与Hercules Capital达成1.75亿美元非稀释性高级担保定期贷款协议，包括现有5000 万美元债务再融资，额外1.25亿美元融资可提取，利息率降至9.70%，显著改善财务灵活性。此外，该 公司公布AMT-130在亨廷顿病1/2期关键研究中达到主要终点，高剂量组36个月数据显示疾病进展减缓 75%cUHDRS测量，同时关键次要终点TFC测量显示疾病进展减缓60%。 ...

周三，uniQure(QURE.US)暴涨超186%，创四年新高，报39.11美元。消息面上，9月24日，该公司宣布 与Hercules Capital达成1.75亿美元非稀释性高级担保定期贷款协议，包括现有5000万美元债务再融资， 额外1.25亿美元融资可提取，利息率降至9.70%，显著改善财务灵活性。此外，该公司公布AMT-130在 亨廷顿病1/2期关键研究中达到主要终点，高剂量组36个月数据显示疾病进展减缓75%cUHDRS测量，同 时关键次要终点TFC测量显示疾病进展减缓60%。 ...

uniQure N.V. QURE on Wednesday released topline data from the pivotal Phase 1/2 study of AMT-130 for Huntington’s disease.Huntington’s disease is an inherited condition in which nerve cells in the brain break down over time. The disease affects a person’s movements, thinking ability and mental health.The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating ...

Summary of uniQure's AMT-130 Conference Call Company and Industry - **Company**: uniQure - **Industry**: Gene therapy for Huntington's disease Key Points and Arguments Clinical Trial Results - **Pivotal Data Announcement**: uniQure announced pivotal data from the Phase I/II clinical trials of AMT-130 for Huntington's disease, showing a statistically significant 75% slowing of disease progression at 36 months as measured by the Composite Unified Huntington's Disease Rating Scale (CUHDRS) [4][18] - **Total Functional Capacity (TFC)**: The study also demonstrated a 60% reduction in disease progression on the TFC, a key measure of a patient's ability to live independently [14][19] - **Neurofilament Light Chain (NFL)**: CSF neurofilament light chain levels were below baseline at 36 months, indicating a reduction in neurodegeneration [16][19] Regulatory and Commercial Plans - **Biologics License Application (BLA)**: uniQure plans to submit a BLA for AMT-130 in the first quarter of 2026, with a request for priority review [5][18] - **FDA Engagement**: A pre-BLA meeting with the FDA is scheduled to discuss the data and the content of the BLA [18][34] Patient Population and Market Opportunity - **At-Risk Population**: Approximately 200,000 Americans are at risk for Huntington's disease, with about 100,000 being genetically identifiable [26] - **Diagnosed Patients**: There are around 40,000 symptomatic HD patients in the U.S., with about 20,000 currently diagnosed [26][27] - **Initial Treatable Patients**: An estimated 6,000 patients could be considered initially treatable at launch [27] Treatment Characteristics - **Durable Treatment**: AMT-130 is designed as a durable, once-administered treatment, which is crucial for a slowly progressing disease [6] - **Targeted Delivery**: The treatment allows for precision-based delivery to affected brain regions, maximizing therapeutic concentrations and minimizing systemic exposure [7] - **Mechanism of Action**: AMT-130 targets the first exon of the Huntington gene, suppressing both the full-length mutant protein and toxic splice isoforms [7] Safety Profile - **Well Tolerated**: AMT-130 has shown a manageable safety profile, with no new treatment-related serious adverse events reported since December 2022 [17][19] - **Common Adverse Events**: Most adverse events were related to study procedures, such as headache and procedural pain [17] Expert Perspectives - **Clinical Impact**: Dr. Sarah Tabrizi emphasized the potential of AMT-130 to significantly impact the quality of life for patients and families affected by Huntington's disease [24] - **Long-term Benefits**: The slowing of disease progression could allow patients to maintain independence and work longer, potentially preventing symptoms from occurring [41] Future Considerations - **Payer Engagement**: Early engagement with payers is critical to educate them on the value of AMT-130 and its impact on patients' lives [51] - **Labeling and Eligibility**: Discussions are ongoing regarding the potential labeling of AMT-130, including whether it will be restricted to symptomatic patients [80] Additional Important Content - **Statistical Analysis**: The study's primary analysis showed a 75% reduction in disease progression with a p-value of 0.003, indicating strong statistical significance [18] - **Longitudinal Data**: The data from the Enroll-HD study provided a robust external control for comparison, enhancing the credibility of the results [55] This summary encapsulates the critical findings and strategic plans discussed during the conference call regarding AMT-130 and its implications for Huntington's disease treatment.

Pivotal Phase I/II AMT-130 Huntington's Disease Update September 24, 2025 Disclaimer This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to," "may ...