Corporate Presentation February 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 ou ...

Financial Data and Key Metrics Changes - For 2024, total revenue was reported at $560 million, representing a 29% growth over 2023 [28] - The net loss for the year was $569 million, equating to $6.29 per share [31] - Cash, cash equivalents, and marketable securities stood at $745 million as of December 31 [31] - The company expects reduced net cash used in operations in 2025 compared to 2024 [31] Business Line Data and Key Metrics Changes - CRYSVITA contributed $410 million to total revenue, with a 25% growth over 2023 [28] - DOJOLVI generated $88 million, reflecting a 25% growth over 2023 [29] - EVKEEZA contributed $32 million, with demand building following launches outside the U.S. [29] - MEPSEVII generated $30 million, continuing to treat patients in an ultra-rare indication [29] Market Data and Key Metrics Changes - Latin America and Turkey saw a 78% growth in CRYSVITA revenue over 2023 [28] - The U.S. market for CRYSVITA showed strong underlying demand, contributing significantly to revenue [18] - The EMEA region for DOJOLVI saw over 250 patients treated across 14 countries, with strong demand noted [21] Company Strategy and Development Direction - The company aims for full-year GAAP profitability by 2027, supported by multiple product launches globally [9] - The focus is on advancing late-stage programs in serious genetic conditions, with six programs expected to progress [6][7] - The company is preparing for the potential launch of two or three new products in the coming year [25] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming year, anticipating strong global revenue growth supported by product launches [9] - The company highlighted the importance of early treatment for patients, particularly in the context of newborn screening [12] - Management noted the FDA's increasing focus on rare diseases and the potential for accelerated development pathways [15] Other Important Information - The company has filed for accelerated approval of UX111 for Sanfilippo syndrome based on significant clinical data [10] - The Phase 3 studies for UX143 and GTX-102 are progressing well, with expectations for strong data readouts [36][38] Q&A Session Summary Question: Confidence in the second interim read for OI study - Management expressed greater confidence in the second interim analysis due to the design and patient treatment duration [53] Question: Risks if the second interim does not hit - Management indicated that high variation in fracture rates could be a reason for not hitting the second interim [58] Question: Filing timeline after Orbit study results - If the Orbit study hits, the filing will be expedited, likely occurring within a quarter [62] Question: Characteristics of patients with fractures in Phase 2 - Management noted that fractures occurred in various types, including type 1 patients, indicating no specific pattern [108] Question: Duration of therapy for OI - Management believes chronic treatment is necessary for OI patients, contrary to the 12-month cap seen in other conditions [134] Question: Sham control trial for Angelman syndrome - The decision for a sham control was influenced by ethical considerations and regulatory feedback [140]

Core Insights - Ultragenyx reported revenue of $164.88 million for the quarter ended December 2024, reflecting a 29.4% increase year-over-year [1] - The company's EPS was -$1.39, an improvement from -$1.52 in the same quarter last year [1] - Revenue exceeded the Zacks Consensus Estimate of $163.23 million by 1.01%, while EPS fell short of the consensus estimate of -$1.24 by 12.10% [1] Revenue Breakdown - Product sales for Dojolvi reached $31.10 million, surpassing the estimated $27.23 million, marking a 33.6% increase year-over-year [4] - Evkeeza generated $10.37 million, slightly below the $10.65 million estimate, but showed a significant year-over-year increase of 393.5% [4] - Mepsevii sales were reported at $7.98 million, below the average estimate of $8.91 million [4] - Total product sales amounted to $71.87 million, compared to the average estimate of $83 million, representing a 39.1% year-over-year increase [4] - Crysvita sales were $22.42 million, significantly lower than the estimated $35.76 million [4] - Non-cash collaboration royalty revenue was reported at $93.01 million, exceeding the average estimate of $71.60 million [4] Stock Performance - Ultragenyx shares have returned +6% over the past month, outperforming the Zacks S&P 500 composite's +3.9% change [3] - The stock currently holds a Zacks Rank 3 (Hold), indicating expected performance in line with the broader market in the near term [3]

Core Viewpoint - Ultragenyx reported a quarterly loss of $1.39 per share, which was worse than the Zacks Consensus Estimate of a loss of $1.24, but an improvement from a loss of $1.52 per share a year ago [1] Financial Performance - The company posted revenues of $164.88 million for the quarter ended December 2024, exceeding the Zacks Consensus Estimate by 1.01% and showing an increase from $127.39 million year-over-year [2] - Over the last four quarters, Ultragenyx has surpassed consensus EPS estimates two times and topped consensus revenue estimates three times [2] Stock Performance - Ultragenyx shares have increased approximately 3.2% since the beginning of the year, compared to a 2.9% gain in the S&P 500 [3] Future Outlook - The company's earnings outlook will be crucial for determining future stock performance, with current consensus EPS estimates at -$1.36 for the coming quarter and -$4.98 for the current fiscal year [4][7] - The estimate revisions trend for Ultragenyx is currently mixed, resulting in a Zacks Rank 3 (Hold), indicating expected performance in line with the market [6] Industry Context - The Medical - Biomedical and Genetics industry, to which Ultragenyx belongs, is currently ranked in the top 27% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event reported): January 12, 2025 Ultragenyx Pharmaceutical Inc. (Exact name of Registrant as Specified in Its Charter) Delaware 001-36276 27-2546083 (State or Other Jurisdiction of Incorporation) 60 Leveroni Court Novato, California 94949 (Address of Principal Executive Offices) (Zip Code) (IRS Employer Identi ...

2024 Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million 2025 Financial Guidance: Total Revenue between $640 million to $670 million, Crysvita revenue of $460 million to $480 million, and Dojolvi revenue of $90 million to $100 million NOVATO, Calif., Feb. 13, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare a ...

NOVATO, Calif., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, February 13, 2025, to discuss its financial results and corporate update for the quarter and the year ending December 31, 2024. The live and replayed webcast of the call will be available thr ...

Ultragenyx Pharmaceutical (RARE) reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA).Per the data readout from the phase I/II/III Transpher A study, treatment with the candidate resulted in a statistically significant improvement in Bayley-III raw scores for cognition, receptive communication and expressive communication in MPS IIIA patients. Compared to natural history data from untreated patients ...

Private Placement Details - Tactical Resources Corp completed a non-brokered private placement of unsecured convertible debentures with a principal amount of $500,000 [1] - The debentures have a maturity date of January 21, 2027 and can be converted into units of the company at a conversion price of $0.20 per unit [2] - Each unit consists of one common share and one share purchase warrant, with each warrant exercisable into an additional share at $0.20 for three years [2] - The debentures bear interest at 10% per annum, with all interest payable on the maturity date [3] - The company paid a cash finder's fee of $4,564 in connection with the private placement [5] Conversion and Restrictions - The debentures and warrants are subject to blocker provisions that restrict conversion or exercise if it would result in the securityholder holding 10% or more of issued shares (10% blocker) or 20% or more of issued shares (20% blocker) [4] - Securities issued in the private placement are subject to a statutory resale restriction for four months plus one day from the closing date [5] Use of Proceeds and Business Combination - Proceeds from the private placement will be used for general working capital and transaction expenses related to the proposed business combination with Plum Acquisition Corp III [6] - The proposed business combination is expected to close in the first half of 2025 and will provide growth capital for the Peak Project and future development initiatives [13] - At closing, Tactical Resources' existing shareholders will convert 100% of their ownership stakes into a newly-formed public company [13] Company Overview - Tactical Resources is a mineral exploration and development company focused on U S-made rare earth elements used in semiconductors, electric vehicles, advanced robotics, and national defense [9] - The company is developing innovative metallurgical processing techniques to unlock rare earth elements development potential [10] - The company's flagship asset, the Peak Project, contains rights to acquire rare earth elements-enriched tailings and stockpiled materials from the Sierra Blanca Quarry [11] - The Peak Project is strategically located southeast of El Paso, Texas, with extensive infrastructure and initial quarry operating permits already in place [11] Insider Participation - Two insiders of the company participated in the private placement, acquiring debentures with a total aggregate principal amount of $15,000 [7] - The private placement may constitute a related party transaction but is exempt from formal valuation and minority approval requirements [7]

In conjunction with the 2025 J.P. Morgan Healthcare Conference, the analyst hosted an investor lunch with Ultragenyx Pharmaceutical Inc RARE.The analyst keeps an Overweight rating on Ultragenyx with a price target of $102 and says the company is uniquely positioned in the long term with an expansive and diverse pipeline of orphan disease assets and highly regarded management. JP Morgan sees multiple value-creating catalysts over the next six to 18 months.Ultragenyx’s recent meeting focused on setrusumab and ...