NOVATO, Calif., May 17, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 17,180 restricted stock units of the company's common stock to ten newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Induce ...

Financial Data and Key Metrics Changes - The company reported total revenue of $109 million for Q1 2024, with Crysvita contributing $83 million, including $40 million from North America, $36 million from Latin America and Turkey, and $6 million from Europe [28][29] - The net loss for the first quarter was $171 million, or $2.03 per share, with total operating expenses of $274 million [30][31] - The company reaffirmed its 2024 revenue guidance, expecting total revenue between $500 million and $530 million, representing 15% to 22% growth compared to 2023 [32] Business Line Data and Key Metrics Changes - Crysvita's demand in the U.S. remained strong, with approximately 60% of start forms coming from adult patients, indicating potential for continued growth [20][21] - Dojolvi revenue for Q1 was $16 million, while Mepsevii revenue was $7 million, showing steady performance across product lines [30] - The company added over 30 start forms for Dojolvi in the U.S., resulting in over 485 reimbursed patients since launch [24] Market Data and Key Metrics Changes - In Latin America, the company added approximately 50 new patients to Crysvita, totaling over 550 patients on reimbursed therapy since launch, with Brazil driving more than 60% of revenue [22] - The company is seeing increasing requests for Dojolvi in the EMEA region, with approximately 200 patients treated across 12 countries [25] - Evkeeza is expected to launch in more EMEA countries in 2024, with nearly 100 patients treated in EMEA as of Q1 2024 [26][27] Company Strategy and Development Direction - The company is focused on advancing its clinical pipeline, with significant progress expected in the GTX-102 program for Angelman syndrome and DTX401 for glycogen storage disease type 1a [41][42] - The company plans to initiate Phase III studies for GTX-102 around the end of the year, with a global randomized placebo-controlled study expected to enroll approximately 100 to 120 patients [35] - The company is also preparing for the launch of Evkeeza in Japan and securing reimbursement agreements in Canada [26][27] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the underlying demand for their products and reiterated guidance for revenue growth in 2024 [28][32] - The management highlighted the urgency of treatments for conditions like GSD1a, emphasizing the strong desire for patients to receive therapy [48] - There is optimism regarding the potential of GTX-102 to transform treatment for Angelman syndrome, with positive feedback from key opinion leaders [34][56] Other Important Information - The company is transitioning to a royalty model for Crysvita in North America, which is expected to increase revenue as the royalty rate rises [29] - The company has a strong cash position with $569 million in cash, cash equivalents, and marketable securities as of March 31, 2024 [30] Q&A Session Summary Question: What is required for the GSD1a program to reach a positive study? - The primary endpoint is the continuous variable of cornstarch utilization, comparing the decrease in cornstarch utilization between treated and placebo groups [54] Question: What are the most misunderstood points regarding GTX-102? - There is difficulty in appreciating the significance of changes in the Bayley score, and misconceptions about the safety profile related to lower extremity weakness [56] Question: Will the Orbit and COSMIC studies read out at the same time? - Both studies are expected to read out around the same time, with plans to file for the full age range based on the data [60] Question: What will be the primary endpoint for the GTX-102 Phase III trial? - The primary endpoint is likely to be the Bayley cognition score, with other domains included as secondary endpoints [62] Question: How will the company assess changes in global copper metabolism for Wilson's disease? - The company will measure free copper in urine and ceruloplasmin oxidase activity to assess copper metabolism [78][81]

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 March 31, 2024 OR (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification No.) 60 Leveroni Court Novato, California 94949 (Address of principal executive offices) (Zip Code) (415) 483-8800 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES E ...

Exhibit 99.1 Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa ir@ultragenyx.com Ultragenyx Reports First Quarter 2024 Financial Results and Corporate Update First quarter total revenue of $109 million, Crysvita® revenue of $83 million and Dojolvi® revenue of $16 million Reaffirmed 2024 expected total revenue guidance between $500 million to $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million Presented positive interim Phase 1/2 data ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-K ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 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 jurisdiction of inc ...

Call End: Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) Q4 2023 Earnings Conference Call February 15, 2024, 05:00 PM ET Company Participants Joshua Higa - Vice President of Investor Relations Emil Kakkis - Chief Executive Officer & President Erik Harris - Chief Commercial Officer Howard Horn - Chief Financial Officer Eric Crombez - Chief Medical Officer Conference Call Participants Gena Wang - Barclays Maurice Raycroft - Jefferies Anupam Rama - JPMorgan Tazeen Ahmad - Bank of America Dae Gon Ha - Stifel Me ...

Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) Q3 2023 Earnings Conference Call November 2, 2023 5:00 PM ET Company Participants Joshua Higa - Vice President of Investor Relations Emil Kakkis - Chief Executive Officer & President Erik Harris - Chief Commercial Officer Howard Horn - Chief Financial Officer Eric Crombez - Chief Medical Officer Conference Call Participants Anupam Rama - JPMorgan Dae Gon Ha - Stifel Michael Riad - Morgan Stanley Tazeen Ahmad - Bank of America Kristen Kluska - Cantor Fitzgerald M ...

ultrageny% Cautionary note regarding forward-looking statements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations and projections regarding our future operating results and financial performance, anticipated cost or expense reductions, plans with respect to commercializing our product and product candidates, our translational research program, expectations regarding our manufacturing capabilities, the expected timing of release of ad ...

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 September 30, 2023 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) (State or other jurisdiction of i ...

Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) Q2 2023 Earnings Conference Call August 3, 2023 5:00 PM ET Company Participants Joshua Higa - Director, IR & Corporate Communications Emil Kakkis - Founder, President, CEO & Director Eric Crombez - Chief Medical Officer & EVP Aaron Olsen - SVP, Corporate Strategy & Finance Erik Harris - EVP & Chief Commercial Officer Conference Call Participants Gena Wang - Barclays Bank Liisa Bayko - Evercore ISI Maurice Raycroft - Jefferies Tommie Reerink - Goldman Sachs Group ...