Summary of Ultragenyx Pharmaceutical FY Conference Call Company Overview - **Company**: Ultragenyx Pharmaceutical (NasdaqGS:RARE) - **Focus**: Development and commercialization of drugs for patients with rare diseases, particularly Osteogenesis Imperfecta (OI) and Angelman syndrome [2][26] Key Points Product Pipeline and Development - **Current Products**: Success with Crysvita, supported by Mepsevii, Evkeeza, and Dojolvi [2] - **Upcoming Data**: Anticipation for phase three data readouts, particularly for Osteogenesis Imperfecta [2] - **Setrusumab**: Expected to show significant results in both Orbit and Cosmic studies, with a focus on the probability of success [5][6] Financial Strategy - **Royalty Financing**: Initiated due to the delay in potential revenue from the Sanfilippo program, allowing for a stronger balance sheet without diluting stock [3] Clinical Trials - **Orbit and Cosmic Studies**: Both studies are designed to be reported together, with confidence in their success [4] - **Effect Size Expectations**: A treatment effect size of 30% to 40% is considered clinically meaningful, with a powered expectation of at least 50% [6][7] - **Rescue Treatment Protocol**: Ethical considerations in allowing patients to receive rescue treatment if they experience high fracture rates [9][10] Commercial Considerations - **Pricing Strategy**: Pricing for Osteogenesis Imperfecta will be informed by the successful launch of Crysvita [11] - **Duration of Treatment**: Setrusumab is expected to be a lifelong treatment due to its mechanism of action [12] Patient Demographics - **Trial Composition**: Orbit phase three includes approximately 50% type 3 and 4 patients, with a commercial mix of about 60% type 1 and 38% type 3 and 4 patients [14][15] Confidence in Data - **Confidence Level**: High confidence in the upcoming data readout for Setrusumab, supported by consistent results in earlier phases [16] Angelman Syndrome Program - **Phase 1/2 Study**: Enrollment of 74 patients, with ongoing discussions about data release prior to the next phase [17] - **Safety Monitoring**: Close monitoring of lower extremity weakness, with mild cases reported and resolved quickly [18][19] - **Dropout Rates**: Expected to remain low due to the nature of the study and the potential benefits for patients [19] Developmental Outcomes - **Cognitive Improvement**: Unique potential to improve cognition and communication in patients with Angelman syndrome, rather than just preventing deterioration [22] - **Primary Endpoint Focus**: Cognition is the primary endpoint, followed by communication and motor skills [25] Future Outlook - **Gene Therapy Programs**: Continued focus on gene therapy programs, with upcoming PDUFA dates for Sanfilippo and GSD1A expected to lead to successful launches [26]

Company Overview - Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing novel products for rare and ultra-rare genetic diseases across various regions including North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific [4] Financial Performance - In Q3 2025, Ultragenyx reported total revenue of $160 million, reflecting a 15% year-over-year growth [2] - The company is projected to achieve full-year revenue between $640 million and $670 million, indicating a growth of 14% to 20% compared to 2024 [2] - Revenue from Crysvita accounted for $112 million in Q3, with full-year guidance expected between $460 million and $480 million, representing a 12% to 17% growth over 2024 [2] - AJOVY revenue for the quarter was $24 million, while Mepsevii revenue was $7 million [2] - The company reported a net loss of $180 million, or $1.81 per share, for Q3 [3] Analyst Insights - Barclays analyst Gena Wang lowered the price target for Ultragenyx from $81 to $50 but maintained an Overweight rating, citing analysis of the setrusumab COSMIC study while still indicating over a 70% chance of a positive result [1][3] - Despite the lowered price target, the company is advancing multiple late-stage clinical programs with significant commercial potential, including UX 143 (setrusumab) and GTX-102 [3]

Core Insights - Ultragenyx Pharmaceutical reported a third-quarter 2025 loss of $1.81 per share, which was wider than the Zacks Consensus Estimate of a loss of $1.23, and compared to a loss of $1.40 per share in the same quarter last year [1][6] - Total revenues for the quarter were $159.9 million, reflecting a 15% year-over-year increase, but fell short of the Zacks Consensus Estimate of $168 million [1][6] Revenue Breakdown - Crysvita generated total revenues of $111.9 million, up 14% year over year, with contributions of $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe [4] - Mepsevii product revenues declined by 27% year over year to $7 million, while Dojolvi revenues increased by 14% to $24.3 million due to new patient demand [5] - Evkeeza recorded sales of $16.7 million, up 57% as the drug continues to be launched in territories outside the United States [5][6] Operating Expenses - Operating expenses for the quarter were $330.8 million, a 22% increase year over year, driven by higher investments in late-stage pipeline programs and marketing costs [8] - Research and development expenses were $216.2 million (up 27%), selling, general and administrative expenses were $86.6 million (up 8%), and cost of sales was $28 million (up 33%) [8] Financial Guidance - The company expects total revenues in 2025 to be between $640 million and $670 million, representing growth of approximately 14-20% compared to 2024 [10] - Crysvita revenues are projected to be in the range of $460-$480 million (up 12-17% year over year), while Dojolvi revenues are expected between $90 million and $100 million (up 2-14% year over year) [10] Pipeline Updates - Ultragenyx faced a setback with the FDA issuing a complete response letter for its biologics license application for UX111, requesting more information related to manufacturing [12] - The company plans to resubmit the BLA for UX111 early in 2026 [13] - GTX-102 received Breakthrough Therapy designation from the FDA for treating Angelman syndrome, with data expected in the second half of 2026 [14] - The company has begun the rolling submission of a BLA for its investigational gene therapy DTX401, expected to be completed in Q4 2025 [15]

Core Insights - Ultragenyx reported revenue of $159.93 million for the quarter ended September 2025, reflecting a year-over-year increase of 14.7% [1] - The company's EPS was -$1.81, a decline from -$1.40 in the same quarter last year, and fell short of the consensus estimate of -$1.23 by 47.15% [1][3] - The revenue fell short of the Zacks Consensus Estimate of $167.55 million, resulting in a surprise of -4.55% [1] Revenue Breakdown - Dojolvi generated $24.28 million in product sales, below the average estimate of $25.96 million, marking a year-over-year increase of 13.6% [4] - Evkeeza achieved $16.72 million in product sales, exceeding the estimated $13.24 million, representing a significant year-over-year growth of 56.9% [4] - Mepsevii reported $7 million in product sales, which was below the average estimate of $8.82 million, indicating a year-over-year decline of 27.2% [4] - Total Crysvita revenue was $111.94 million, falling short of the average estimate of $119.46 million [4] - Overall product sales reached $94.99 million, surpassing the average estimate of $89.79 million, with a year-over-year increase of 23% [4] - Royalty revenue was $64.94 million, below the estimated $78.87 million, but showed a year-over-year increase of 4.3% [4] Stock Performance - Ultragenyx shares have returned +10.9% over the past month, outperforming the Zacks S&P 500 composite's +2.1% change [3] - The stock currently holds a Zacks Rank 3 (Hold), suggesting it may perform in line with the broader market in the near term [3]

Financial Data and Key Metrics Changes - In Q3 2025, Ultragenyx reported total revenue of $160 million, representing a 15% increase compared to Q3 2024 and an 18% increase for the first nine months of 2025 over the same period in 2024 [14][15] - The net loss for the quarter was $180 million, equating to $1.81 per share [15] - As of September 30, 2025, the company had $447 million in cash, cash equivalents, and securities, bolstered by a recent $400 million financing [16] Business Line Data and Key Metrics Changes - Crysvita generated $112 million in revenue during Q3 2025, with contributions of $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe [15] - Dojolvi contributed $24 million, maintaining steady growth, while Evkeeza and Mepsevii contributed $17 million and $7 million, respectively [15] - The company expects total revenue for 2025 to be between $640 million and $670 million, indicating a growth of 14% to 20% over 2024 [16][17] Market Data and Key Metrics Changes - In Latin America, approximately 875 patients are now on Crysvita therapy, with 50 new start forms generated in Q3 2025 [8] - Dojolvi has seen approximately 700 new start forms since its launch in 2020, leading to around 625 patients on reimbursed therapy, with a split of 65% pediatric and 35% adult patients [9] - Evkeeza has approximately 310 patients across 17 countries in the EMEA region, with 120 new patients added since the beginning of the year [10] Company Strategy and Development Direction - The company is focused on maximizing opportunities from its four commercial products and has two BLA submissions in progress for ultra-rare diseases [4] - Ultragenyx aims to maintain financial discipline while preparing for upcoming product launches and pivotal data readouts [14] - The company is positioned for growth with a strong balance sheet and plans to achieve profitability by 2027 [16][48] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming pivotal data readouts for GTX-102 and UX143, which are expected to be transformative for patients with Angelman syndrome and osteogenesis imperfecta [5][23] - The management team highlighted the importance of maintaining cash burn and prioritizing investments as they approach significant clinical milestones [5][16] - The company anticipates continued double-digit revenue growth from existing products and contributions from new launches [48] Other Important Information - The company received $400 million in non-dilutive capital through the sale of a portion of Crysvita royalties, with payments deferred until January 2028 [4][13] - The management team emphasized the unique value of Crysvita and its potential to deliver significant long-term value [14] Q&A Session Summary Question: Data update on Orbit and Cosmic studies - Management confirmed that data from both studies will be reported together, expected in December or January [27] Question: Open label extension from phase II for OI - Management stated that they have not released new quantitative data but are confident in the phase III expectations, aiming for a 40% to 70% reduction in fractures [31][32] Question: Clarification on UX111 and DTX401 submissions - Management explained that the timing of submissions was adjusted due to the need for additional reports, but both filings remain closely aligned [36] Question: Impact of OMERS transaction on financing needs - Management indicated that the $400 million raised will help address investor concerns regarding financing needs in 2026 and support upcoming product launches [42][48] Question: Length of treatment for Setrusumab - Management expressed confidence that Setrusumab will require chronic treatment to maintain bone health, suggesting bisphosphonates may become obsolete [70][73] Question: Loss of exclusivity for Setrusumab - Management highlighted the importance of orphan designation for exclusivity and mentioned ongoing patent protections that extend beyond 2030 [92]

Financial Data and Key Metrics Changes - In Q3 2025, the company reported total revenue of $160 million, representing a 15% increase compared to Q3 2024 and an 18% increase for the first nine months of 2025 over the same period in 2024 [13][14] - The net loss for the quarter was $180 million, equating to $1.81 per share [15] - As of September 30, 2025, the company had $447 million in cash, cash equivalents, and securities, bolstered by a recent $400 million financing [16] Business Line Data and Key Metrics Changes - Crysvita generated $112 million in revenue during Q3 2025, with contributions of $57 million from North America, $47 million from Latin America and Turkey, and $8 million from Europe [14] - Dojolvi contributed $24 million, maintaining steady growth, while Evkeeza and Mepsevii contributed $17 million and $7 million, respectively [14] - The company expects total revenue for 2025 to be between $640 million and $670 million, indicating a growth of 14% to 20% over 2024 [16] Market Data and Key Metrics Changes - In Latin America, the company added approximately 50 new patients on reimbursed therapy for Crysvita, bringing the total to around 875 patients [8] - In the EMEA region, Evkeeza has approximately 310 patients receiving treatment across 17 countries, with 120 new patients added since the beginning of the year [10] Company Strategy and Development Direction - The company is focused on maximizing opportunities from its four commercial products and has two BLA submissions in progress for ultra-rare diseases [3] - A recent $400 million financing through the sale of a portion of Crysvita royalties is aimed at strengthening the balance sheet and supporting upcoming pivotal data readouts [3][12] - The company plans to maintain financial discipline while preparing for the launch of new products and managing cash burn [4][12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming pivotal data readouts for GTX-102 and UX143, which are expected to be transformative for patients with Angelman syndrome and osteogenesis imperfecta [4][23] - The company reaffirmed its path to profitability by 2027, supported by expected double-digit growth from existing products and new launches [12][48] Other Important Information - The company is preparing for a rolling BLA submission for DTX401, with expectations to complete this filing in December [23] - Management highlighted the importance of patient support programs in ensuring access to treatments, especially in a competitive landscape for Angelman syndrome therapies [64] Q&A Session Summary Question: Data update on Orbit and Cosmic studies - Management confirmed that data from both studies will be reported together, expected in December or January [26][27] Question: Open label extension from phase II for OI - Management stated that while no new quantitative data is available, they are confident in the phase III expectations, anticipating a fracture reduction of 40% to 70% [30][32] Question: Clarification on UX111 and DTX401 submissions - Management explained that the timing of submissions was adjusted due to the need for additional reports, but both filings remain closely timed [36][38] Question: Impact of OMERS transaction on financing needs - Management indicated that the recent financing strengthens the balance sheet and supports the path to profitability in 2027 [44][48] Question: Length of treatment for Setrusumab - Management expressed confidence that Setrusumab will require chronic treatment to maintain bone health, suggesting bisphosphonates may become obsolete [72][74] Question: Loss of exclusivity for Setrusumab - Management highlighted orphan designation and additional IP protections that extend exclusivity beyond 2030 [98][99]

Core Insights - Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is recognized as a promising biotech stock with significant potential [1] - Bank of America Securities analyst Tazeen Ahmad has maintained a Buy rating on Ultragenyx and set a price target of $79.00 [1] Financial Performance - For fiscal Q2 2025, Ultragenyx reported total revenue of $166 million, with Crysvita® generating $120 million and Dojolvi® contributing $23 million [2] - The company reaffirmed its 2025 revenue guidance, now expecting total revenue in the range of $640 million to $670 million [2] Product Portfolio - Ultragenyx focuses on developing, acquiring, and commercializing novel products aimed at treating genetic diseases [2] - The company's product portfolio includes Crysvita, Mepsevii, Dojolvi, and Evkeeza [2]

Group 1 - Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is recognized as a promising biotech stock, with Assenagon Asset Management increasing its holdings by 439.0% in Q2, acquiring 947,865 shares for a total investment of $42,314,000, representing about 1.23% ownership [1] - The company has received Breakthrough Therapy designation for GTX-102 in Angelman syndrome and is positioned to advance UX143, which is expected to be a transformative treatment for osteogenesis imperfecta in both pediatric and adult patients [2] - Strategic collaborations, including a recent FDA approval for Regeneron's Evkeeza, indicate a positive outlook for Ultragenyx, despite a recent 26% drop in stock price due to a failed treatment approval [3] Group 2 - Ultragenyx is a California-based biopharmaceutical company focused on discovering, developing, and commercializing treatments for rare and ultra-rare genetic diseases, with key products including Crysvita, Mepsevii, Dojolvi, and Evkeeza [4]

Group 1 - Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is considered a strong buy stock by Wall Street, with a Buy rating and a price target of $105.00 set by Cantor Fitzgerald [1] - The company reported total revenue of $166 million for fiscal Q2 2025, with Crysvita generating $120 million and Dojolvi contributing $23 million [2] - Ultragenyx reaffirmed its revenue guidance for 2025, expecting total revenue in the range of $640 million to $670 million [2] Group 2 - Ultragenyx develops, acquires, and commercializes products for genetic diseases, with a portfolio that includes Crysvita, Mepsevii, Dojolvi, and Evkeeza [3] - In 2024, the company expanded its business through four products across five indications globally [3]

Company Overview - Ultragenyx has been dedicated to developing new treatments for rare diseases for 15 years, focusing on areas with high unmet medical needs [2] - The company has a diverse platform approach, including enzyme replacement therapy with Mepsevii, small molecule therapy with Dojolvi, and gene therapy programs [2] Product Pipeline - The foundational product, Mepsevii, is an enzyme replacement therapy, followed by Dojolvi as a small molecule treatment [2] - Recent advancements include the Angelman program utilizing antisense oligonucleotides (ASO) and an antibody program for osteogenesis imperfecta (OI) [2]