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Ultragenyx Pharmaceutical (NasdaqGS:RARE) Earnings Call Presentation
2026-02-12 12:00
Corporate Presentation February 2026 Confidential and Proprietary Forward looking statements Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations, estimates, assumptions, and projections regarding our future operating results and financial performance, including our expectations for profitability in 2027, anticipated cost or expense management, including the company's expectations rel ...
Aligos Therapeutics (ALGS) FY Conference Transcript
2025-09-05 12:00
Summary of Aligos Therapeutics Conference Call Company Overview - **Company**: Aligos Therapeutics - **Industry**: Biotechnology, focusing on liver and viral diseases - **Mission**: To improve patient outcomes by developing best-in-class therapies for chronic hepatitis B virus (HBV) infection and metabolic dysfunction-associated steatohepatitis (MASH) [1][34] Key Drug: ALG-000184 - **Type**: Capsid assembly modulator for HBV - **Discovery**: Originated from research by Professor Raymond Schinazi at Emory University, known for developing several antiviral drugs [3][4] - **Mechanism of Action**: - Binds to HBV core protein, preventing encapsulation of pregenomic RNA, thus blocking DNA production [4][16] - Evokes a secondary mechanism that reduces CCC DNA, a long-lived viral reservoir [6][17] - **Pharmacokinetics**: - Improved from 5% to 80% oral bioavailability [5] - Demonstrated significant reductions in HBV DNA and surface antigens in clinical studies [6][7] Clinical Data and Efficacy - **Phase 1B Study**: - 96-week data showed no emergence of drug resistance, allowing for potential monotherapy [10][11] - Achieved 100% of E antigen negative patients below 10 international units of HBV DNA at week 48, compared to historical data of around 20% for standard treatments [21][22] - **Endpoints**: - Primary endpoint for monotherapy is chronic suppression of HBV DNA [18][19] - Importance of achieving below 10 international units for better long-term outcomes, including reduced liver cancer progression [20][22] Future Development: B Supreme Study - **Design**: Ongoing phase 2 study comparing ALG-000184 with TDF in both E positive and E negative patients [24][25] - **Goals**: - Superiority in HBV DNA endpoints and antigen reductions [27][28] - Paired biopsies to quantify integration events and CCC DNA levels [28] Competitive Landscape - **Positioning**: ALG-000184 aims to become the standard of care for chronic HBV suppression and a backbone for functional cure regimens [30][31] - **Antisense Oligonucleotide Program**: Aligos is developing its own ASO program to complement ALG-000184, enhancing potential treatment options [31] Other Drug: ALG-055009 for MASH - **Mechanism**: Targets metabolic dysfunction in liver disease, showing significant fat reduction in phase 2A studies [36][37] - **Combination Potential**: Compatible with GLP-1 therapies, enhancing fat reduction and weight loss [38][39] Upcoming Milestones - **2025-2027 Timeline**: - Final data from the 96-week study of ALG-000184 to be presented at AASLD [41] - Interim readout from the B Supreme study in 2026 [41] - Partnership announcement for ALG-055009 expected early next year [42] Conclusion - Aligos Therapeutics is positioned to address significant unmet needs in the treatment of HBV and MASH, with promising clinical data and a robust pipeline aimed at improving patient outcomes in these areas [40][43]
Stoke Therapeutics(STOK) - 2025 Q2 - Earnings Call Transcript
2025-08-12 21:30
Financial Data and Key Metrics Changes - The company ended the second quarter with $355 million in cash, cash equivalents, and marketable securities, expected to fund operations through mid-2028 [42] - Total revenue for the quarter was $13.8 million, driven by collaborations with ACADIA and Biogen, with expectations for revenue from Biogen to increase [43] - The net loss for the quarter was $23.5 million, or $0.40 per share, slightly improved from the prior year despite a $6.9 million year-over-year increase in operating expenses [43] Business Line Data and Key Metrics Changes - The Phase III EMPORER study for Dravet syndrome is underway, with the first patient dosed and strong enrollment anticipated due to high awareness and urgent patient need [5][21] - The company has advanced STK002 into Phase I clinical development for autosomal dominant optic atrophy (ADOA), indicating a growing pipeline [7][35] Market Data and Key Metrics Changes - The collaboration with Biogen enhances the company's ability to deliver zurivanersen globally and strengthens its balance sheet [8] - The company estimates approximately 13,000 patients currently living with ADOA across key geographies, indicating a significant market opportunity [38] Company Strategy and Development Direction - The key priority remains the development of zurivanersen for Dravet syndrome, aiming to deliver a disease-modifying medicine [4] - The company is focused on establishing internal capabilities and enhancing leadership to support growth and value creation [9] - The strategic collaboration with Biogen is expected to provide global expertise in commercializing high-value disease-modifying medicines [8] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term potential of zurivanersen to modify the course of Dravet syndrome, supported by positive data from ongoing studies [7][31] - The company is committed to advancing zurivanersen to patients as quickly as possible, leveraging breakthrough therapy designation from the FDA [50][52] Other Important Information - The company has seen a favorable safety profile for zurivanersen, with no clinical manifestations associated with elevated CSF protein levels observed in patients [32][94] - The Vineland-three assessment is being used to measure changes in cognition and behavior in patients with Dravet syndrome, with significant improvements noted over time [28][30] Q&A Session Summary Question: Can you help us understand the potential for accelerated approval for zurivanersen? - Management confirmed that zurivanersen has breakthrough designation and they are collecting further data to discuss with the FDA in the second half of the year [50][52] Question: What is the magnitude of cognition and behavior improvements in the Vineland-three data? - Management noted that changes in cognition and behavior are clinically meaningful, with caregivers identifying even small improvements as significant [62][63] Question: Can you explain the data used to inform the powering assumptions for the Phase III EMPOR study? - Management indicated that data from previous studies and natural history studies were used to inform the powering assumptions, ensuring robust statistical significance [70][72] Question: Are there trends in seizure reduction and neurodevelopmental benefits among different age groups? - Management acknowledged variability in responses but emphasized the importance of treating younger patients to potentially change the course of their development [86][87] Question: Can you discuss the higher incidence of CSF protein elevations in the OLE study? - Management clarified that elevated CSF protein levels are a laboratory finding and have not been associated with clinical effects, supporting the safety profile for moving into Phase III [92][94] Question: What prompted the decision to explore STK-two in ADOA? - Management explained that a thorough evaluation of the opportunity in ADOA, including promising nonhuman primate data, led to the decision to pursue clinical studies [98][100]
Ultragenyx's Q2 Loss Narrower Than Expected, Revenues Rise Y/Y
ZACKS· 2025-08-06 16:45
Core Insights - Ultragenyx Pharmaceutical reported a second-quarter 2025 loss of $1.17 per share, which is an improvement from a loss of $1.52 per share in the same quarter last year and better than the Zacks Consensus Estimate of a loss of $1.27 [1][5] - Total revenues for the quarter reached $166.5 million, reflecting a 13% year-over-year increase, driven primarily by higher product sales, and surpassing the Zacks Consensus Estimate of $162 million [1][5] Revenue Breakdown - Crysvita generated total revenues of $120.4 million, up 6% year over year, with contributions of $79 million from North America, $35 million from Latin America and Turkey, and $7 million from Europe [3] - Mepsevii product revenues increased by 35% year over year to $8.3 million, while Dojolvi revenues rose 20% to $23.2 million due to new patient demand [4] - Evkeeza recorded sales of $14.6 million in the first quarter, showing significant growth as the drug continues to be launched in territories outside the United States [4] Financial Guidance - The company reaffirmed its 2025 financial guidance, expecting total revenues between $640 million and $670 million, which represents a growth of approximately 14-20% compared to 2024 [9] - Crysvita revenues are anticipated to be in the range of $460-$480 million, reflecting a year-over-year increase of 12-17%, while Dojolvi revenues are expected to be between $90 million and $100 million, up 2-14% year over year [9] Operating Expenses - Operating expenses for the quarter were $274.4 million, a 4% increase year over year, attributed to higher investments in late-stage pipeline programs and marketing costs for approved drugs [7] - Research and development expenses were $164.7 million (up 2%), selling, general and administrative expenses were $86.6 million (up 7%), and cost of sales was $23 million (up 8%) [7] Pipeline Updates - The FDA issued a complete response letter for Ultragenyx's biologics license application for UX111, requesting additional information related to manufacturing processes, which the company plans to address promptly [11][12] - The company is also developing GTX-102 for Angelman syndrome, which received Breakthrough Therapy designation, with data expected in the second half of 2026 [14] - Ultragenyx plans to submit a BLA for DTX401, a gene therapy for glycogen storage disease type Ia, in the fourth quarter of 2025 [15]