Company Overview - Ultragenyx (RARE) shares increased by 5.1% to $41.44 in the last trading session, with a notable trading volume [1] - The stock has also gained 5.1% over the past four weeks [1] Pipeline Progress - The rise in stock price is linked to positive investor sentiment regarding Ultragenyx's late-stage pipeline, particularly the regulatory application for FDA approval of UX111, a gene therapy for Sanfilippo syndrome type A, currently under Priority Review with a decision expected on August 18, 2025 [2] - Ultragenyx plans to submit a biologics license application for DTX401, an AAV8 gene therapy for glycogen storage disease type Ia, which could further enhance revenue if approved [2] Financial Expectations - The company is projected to report a quarterly loss of $1.27 per share, reflecting a year-over-year increase of 16.5%, with expected revenues of $161.61 million, up 9.9% from the previous year [3] - The consensus EPS estimate for the quarter has remained unchanged over the last 30 days, indicating that stock price movements may not sustain without trends in earnings estimate revisions [4] Industry Context - Ultragenyx operates within the Zacks Medical - Biomedical and Genetics industry, where another company, Fortress Biotech (FBIO), saw a 3.1% increase in its stock price, closing at $1.99, although FBIO has returned -7.2% over the past month [4] - Fortress Biotech's consensus EPS estimate for its upcoming report is -$0.31, which is a 57.5% improvement from the previous year, and it also holds a Zacks Rank of 3 (Hold) [5]

Key Takeaways The FDA granted Breakthrough Therapy designation to Ultragenyx's GTX-102 for Angelman syndrome. GTX-102 showed rapid, lasting improvements in AS patients during a phase I/II study over up to 3 years. RARE is enrolling for a phase III Aspire study and planning a broader phase II/III Aurora study for GTX-102.Ultragenyx Pharmaceuticals (RARE) announced that the FDA has granted the Breakthrough Therapy designation to its investigational antisense oligonucleotide therapy, GTX-102 (apazunersen), t ...

Core Viewpoint - Ultragenyx Pharmaceutical Inc. released its 2024 Impact Report, emphasizing its commitment to corporate responsibility and its mission to transform the lives of individuals affected by rare diseases [1][2] Group 1: Corporate Responsibility Approach - The company's corporate responsibility is structured around six key pillars: Innovation, Patients, People, Communities, Planet, and Governance, aligning with its vision to lead in rare disease medicine [2] - The 2024 Impact Report includes updates for all six pillars and references Sustainability Accounting Standards Board (SASB) and Global Reporting Initiative (GRI) indices [2] Group 2: Innovation - Ultragenyx is dedicated to developing transformative treatments for rare diseases where no options currently exist [3] Group 3: Patients - The company supports the rare disease community by developing novel therapies, sharing scientific expertise, ensuring access to screening and treatment, and collaborating with policymakers for impactful changes [4] Group 4: People - Ultragenyx focuses on maintaining an inclusive, safe, and healthy work environment, along with fair and equitable compensation practices [5] Group 5: Communities - The company is committed to initiatives that positively impact the rare disease community, including public health access, STEAM education, and support for at-risk local communities [6] Group 6: Achievements and Recognition - In 2024, Ultragenyx successfully transferred the manufacturing process of its DTX401 gene therapy and advanced several key clinical programs for rare diseases [7] - The company supported over 80 clinical and non-clinical investigator-sponsored trials globally and assisted more than 650 patients in 50 countries through various access programs since 2013 [7] - Ultragenyx received multiple accolades in 2024, including being named one of the 100 Most Sustainable Companies by Barron's and recognized as one of the Top Places to Work in the USA for the second consecutive year [11]

Corporate Presentation May 2025 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, plans with respect to commercializing our pro ...