Core Insights - The FDA has accepted Ultragenyx Pharmaceutical Inc.'s Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A, with a Priority Review status and a PDUFA action date of August 18, 2025 [1][2] - The acceptance of the BLA is a significant step towards providing the first-ever treatment for Sanfilippo syndrome type A, with the company prepared to launch the therapy upon successful approval [2] - UX111 is a novel in vivo gene therapy designed to address the underlying SGSH enzyme deficiency, which leads to the accumulation of heparan sulfate in the brain, causing neurodegeneration [3][4] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing therapies for serious rare and ultrarare genetic diseases, with a diverse portfolio aimed at addressing high unmet medical needs [5] - The company has a management team experienced in the development and commercialization of rare disease therapeutics, emphasizing efficient drug development processes [6] Product Details - UX111 is currently in Phase 1/2/3 development and is administered as a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional SGSH gene [3] - The therapy has received multiple designations, including Regenerative Medicine Advanced Therapy and Orphan Drug designations in the U.S., and PRIME designation in the EU [3] Disease Context - Sanfilippo syndrome type A is a rare, fatal lysosomal storage disease affecting approximately 3,000 to 5,000 patients, characterized by rapid neurodegeneration and a median life expectancy of 15 years [4] - The disease is caused by biallelic pathogenic variants in the SGSH gene, leading to a deficiency in the SGSH enzyme responsible for breaking down heparan sulfate [4]

Core Viewpoint - Ultragenyx Pharmaceutical reported a wider loss per share in Q4 2024 compared to the previous year and the consensus estimate, despite a significant increase in total revenues driven by higher product sales [1][2][8]. Financial Performance - The total revenues for Q4 2024 were $164.9 million, reflecting a 29% year-over-year increase and surpassing the Zacks Consensus Estimate of $163 million [2]. - The net loss per share for Q4 2024 was $1.39, wider than the consensus estimate of a loss of $1.24 and compared to a loss of $1.52 in the same quarter last year [1][8]. - For the full year 2024, total revenues reached $560.2 million, up 29% from $434.2 million in 2023, exceeding the Zacks Consensus Estimate of $558.6 million [8]. Product Performance - Crysvita generated $115.4 million in revenues for Q4 2024, a 23% increase year-over-year, with notable growth in Latin America and Turkey [4]. - Dojolvi's revenues were $31.1 million, up 34% due to strong new patient demand, while Mepsevii's revenues remained flat at $8 million [5]. - Evkeeza recorded sales of $10.4 million in Q4 2024 [5]. Operating Expenses - Operating expenses for Q4 2024 totaled $287.2 million, a 15% increase year-over-year, driven by investments in late-stage pipeline programs and marketing costs [6]. - Research and development expenses were $187.8 million, up 17%, while selling, general and administrative expenses were $82.5 million, up 7% [6]. Cash Position - As of December 31, 2024, cash, cash equivalents, and marketable debt securities amounted to $745 million, down from $825 million as of September 30, 2024 [7]. Future Guidance - For 2025, Ultragenyx expects total revenues between $640 million and $670 million, indicating a growth of approximately 14-20% compared to 2024 [9]. - Crysvita revenues are projected to be between $460 million and $480 million, while Dojolvi revenues are expected to range from $90 million to $100 million [9]. Pipeline Developments - Ultragenyx is developing UX143 for osteogenesis imperfecta in collaboration with Mereo BioPharma, with updates expected in mid-2025 [12]. - The company is also evaluating UX701 for Wilson disease and plans to submit a biologics license application for DTX401 in mid-2025 [13]. - In December 2024, dosing began in the pivotal phase III Aspire study for GTX-102, targeting Angelman syndrome [14]. - A biologics license application for UX111 to treat Sanfilippo syndrome type A was submitted in December 2024, with a decision expected in the second half of 2025 [15].

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]

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]