PresentationGreat. Thank you, everyone, for joining. I'm Max Skor, biotech analysts with Morgan Stanley. And I'm happy to be hosting this session with Ultragenyx, Eric Crombez, Chief Medical Officer. Thank you very much for joining us today. And I just wanted to briefly touch on important disclosures. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales repre ...

Key Takeaways RARE's DTX401 showed a lasting 61% mean reduction in daily cornstarch needs at week 96 in GSDIa patients.Patients reported fewer hypoglycemic events, better fasting tolerance, and higher quality of life.RARE has begun rolling BLA submission for DTX401, with completion expected later this year.Ultragenyx Pharmaceutical (RARE) reported positive longer-term data from a late-stage study of its AAV gene therapy, DTX401, for the treatment of glycogen storage disease type Ia (GSDIa). GSDIa is a rare, ...

At Week 96 participants experienced even greater reductions in daily cornstarch intake while maintaining low levels of hypoglycemia, improved levels of euglycemia and improved fasting tolerance Phase 3 results are further supported by early data from open-label Japanese cohort (n=3) where all participants were able to eliminate daily cornstarch while maintaining or improving glycemic control DTX401 was well tolerated with an acceptable safety profile NOVATO, Calif., Sept. 08, 2025 (GLOBE NEWSWIRE) -- Today, ...

Question-and-Answer SessionReally appreciate the time. So to kick it off, I'll ask you the typical fireside chat question, which is just to please give us a brief overview of everything going on at the company.Howard HornExecutive VP of Corporate Strategy & CFO Sure. I'll kick off, and then I'll hand to Eric. First of all, thank you again for having us. Good morning, everybody. Ultragenyx is a rare disease company, focused on transformative treatments where none have existed in the past. We're at an interes ...

Core Insights - Ultragenyx Pharmaceutical Inc. is focused on developing and commercializing therapies for serious rare and ultra-rare genetic diseases [5][6] - The company will participate in three upcoming investor conferences, providing opportunities for engagement with investors [1][2][3][4] Conference Participation - At the Cantor Global Healthcare Conference on September 4, 2025, Eric Crombez, M.D., and Howard Horn will participate in a fireside chat and host one-on-one meetings [2] - Eric Crombez will also participate in a fireside chat and host one-on-one meetings at the Morgan Stanley 23rd Annual Global Healthcare Conference on September 9, 2025 [3] - The Bank of America Global Healthcare Conference on September 23, 2025, will feature Eric Crombez in a fireside chat and one-on-one meetings, with webcasts available on the company's website [4] Company Overview - Ultragenyx has a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical needs [5] - The management team is experienced in the development and commercialization of rare disease therapeutics, focusing on efficient drug development [6]

Company expects to complete BLA submission in the fourth quarter of 2025 NOVATO, Calif., Aug. 18, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the initiation of a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval for DTX401 AAV gene therapy as a treatment for Glycogen Storage Disease Type Ia (GSDIa). The company has submitted the non-clinical and clinical modules to the FDA and plans to comple ...

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]

Core Insights - Ultragenyx reported revenue of $166.5 million for the quarter ended June 2025, reflecting a 13.2% increase year-over-year and a surprise of +2.94% over the Zacks Consensus Estimate of $161.74 million [1] - The company's EPS was -$1.17, an improvement from -$1.52 in the same quarter last year, with an EPS surprise of +7.87% compared to the consensus estimate of -$1.27 [1] Revenue Breakdown - Dojolvi product sales reached $23.21 million, exceeding the average estimate of $22.38 million by analysts, marking a +19.9% change from the previous year [4] - Evkeeza product sales were $14.57 million, significantly surpassing the estimated $11.08 million, representing an impressive +85.5% increase year-over-year [4] - Mepsevii product sales totaled $8.31 million, slightly below the average estimate of $8.64 million [4] - Total Crysvita revenue was $120.41 million, exceeding the average estimate of $118.04 million by analysts [4] - Overall product sales amounted to $80.82 million, which was below the estimated $84.66 million, but still showed a +9.5% change compared to the previous year [4] - Royalty revenue reached $85.68 million, surpassing the average estimate of $77.43 million, reflecting a +17% increase year-over-year [4] Stock Performance - Ultragenyx shares have declined by -28.2% over the past month, contrasting with a +1% change in the Zacks S&P 500 composite [3] - The stock currently holds a Zacks Rank 4 (Sell), indicating potential underperformance relative to the broader market in the near term [3]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to . Commission File No. 001-36276 ULTRAGENYX PHARMACEUTICAL INC. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other ju ...

Financial Data and Key Metrics Changes - In Q2 2025, total revenue was reported at $166 million, representing a 13% increase over Q2 2024 and a 20% increase year-to-date [20] - The net loss for the quarter was $115 million, or $1.17 per share, with cash, cash equivalents, and marketable securities totaling $539 million as of June 30, 2025 [21][22] - The company expects 2025 total revenue to be between $640 million and $670 million, indicating a growth of 14% to 20% over 2024 [22] Business Line Data and Key Metrics Changes - Crysvita contributed $120 million in Q2 2025, with $79 million from North America, $35 million from Latin America and Turkey, and $7 million from Europe [20] - Dajolvi generated $23 million, while Akiza and Mepsevii contributed $15 million and $8 million, respectively, reflecting steady growth trajectories for these products [20] - The commercial teams reported double-digit revenue growth, with total revenue across the first two quarters reaching $306 million, a 20% increase compared to the previous year [12] Market Data and Key Metrics Changes - In Latin America, approximately 825 patients are now on Crysvita, with positive feedback from healthcare providers leading to increased prescriptions [14] - In the U.S. and Canada, Crysvita revenue is expected to continue growing as new pediatric and adult patients are identified [15] - The EMEA region has seen approximately 280 patients treated with DERJOVY under named patient sales, with strong demand noted in France and other countries [17] Company Strategy and Development Direction - The company aims to achieve GAAP profitability by 2027 while focusing on revenue growth and managing expenses [22][30] - Ultragenyx is advancing its clinical pipeline with five Phase III programs fully enrolled or at the BLA submission stage, including UX143 for osteogenesis imperfecta and GTX102 for Angelman syndrome [6][10] - The company is committed to navigating pricing negotiations and expanding its market presence in various regions, particularly in Latin America and EMEA [19] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing clinical studies and the potential for transformative treatments, particularly for UX143 and GTX102 [9][10] - The company is optimistic about the upcoming data readouts and the overall strength of its product portfolio despite recent regulatory challenges [30] - Management highlighted the importance of maintaining a strong cash position while preparing for future commercial launches [22][30] Other Important Information - The company received breakthrough therapy designation for GTX102 from the FDA, indicating substantial improvement over existing therapies [10] - The Phase III ASPIRE study for GTX102 completed enrollment ahead of schedule, with results expected in 2026 [11] - The company is actively working with the FDA to address observations from a complete response letter for UX111, aiming for a timely resubmission [26][97] Q&A Session Summary Question: Recent FDA interactions and their impact - Management reported productive interactions with the FDA since the complete response letter, expressing confidence in the ongoing discussions [35] Question: Differences in dosing regimens for GTX102 - The company explained that the LNA chemistry used in GTX102 is more potent, allowing for lower dosing regimens compared to other drugs [38][39] Question: COSMIC trial rationale and assumptions - The COSMIC trial aims to evaluate young patients on bisphosphonates, with the goal of demonstrating a significant improvement in fracture rates [44][46] Question: Expected clinical benefit from the Orbit study - Management indicated that even if the fracture data is slightly under expectations, other supportive data could still make a strong case for FDA approval [62] Question: Additional clinical data for UX111 resubmission - The FDA requested updated clinical endpoint data and biomarker data for the resubmission of UX111, which the company is prepared to provide [57][58] Question: Financial management and cost control - The company is prioritizing cost control measures, including delaying certain expenses, to ensure a path to profitability by 2027 [90][91]