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 ...

2024 Highlights Include Proposed Strategic Business Combination and Progress at Flagship Peak ProjectKey Takeaways Tactical Resources’ proposed business combination with Plum Acquisition Corp. III sets the stage for a Nasdaq listing and the opportunity to raise significant growth capital.The Peak Project’s strategic readiness positions Tactical Resources to support U.S. critical mineral needs amid China’s export restrictions.Advancements in Phase 1 technical work and corporate alignment with U.S. defense ob ...

Financial Performance and Guidance - Total revenue for 2024 is estimated to be $555 million to $560 million, exceeding the updated guidance range and representing approximately 29% growth versus 2023 [7] - Crysvita revenue for 2024 is estimated to be $405 million to $410 million, representing approximately 24% growth versus 2023 [7] - Dojolvi revenue for 2024 is estimated to be $87 million to $89 million, representing approximately 25% growth versus 2023 [7] - 2025 total revenue is expected to be between $640 million and $670 million, with a growth of approximately 14-20% compared to 2024 [1][6] - Cash, cash equivalents, and available-for-sale investments were approximately $745 million as of December 31, 2024 [1] Clinical Trials and Pipeline Updates - Phase 3 Orbit and Cosmic clinical trials for setrusumab in pediatric and young adult patients with osteogenesis imperfecta (OI) are ongoing, with the second interim analysis expected in mid-2025 and a potential final analysis in Q4 2025 [2][8] - UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) has a Biologics License Application (BLA) submitted, with a PDUFA decision and launch expected in the second half of 2025 [3] - UX701 AAV gene therapy for Wilson Disease is in Phase 1/2/3 study, with Cohort 4 enrollment expected to complete in the second half of 2025 [5][15] - GTX-102 Phase 3 Aspire study for Angelman syndrome is enrolling, with completion expected in the second half of 2025 [13] - DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa) is expected to file a BLA in mid-2025 [14] - DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency is in Phase 3 study, with enrollment completion expected in early 2025 [16] Strategic Outlook and Milestones - The company anticipates important pivotal Phase 3 results in osteogenesis imperfecta and completion of enrollment in the Phase 3 trial for Angelman syndrome in 2025 [11] - The company is preparing for the potential launch of its first gene therapy for Sanfilippo syndrome and filing a BLA for its second gene therapy for Glycogen Storage Disease Type Ia in 2025 [11] - The company expects to potentially launch three to four new therapies over the next couple of years, accumulating a total of eight to nine approved products over a 10-year period [11] Corporate Overview - Ultragenyx is a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases [17] - The company has a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need [17] - The company is led by a management team experienced in the development and commercialization of rare disease therapeutics [18]

Ultragenyx Pharmaceutical (RARE) announced that the European Commission (EC) has expanded the eligible patient population for one of its marketed products, Evkeeza (evinacumab), an ANGPTL3 inhibitor. Per the latest approval, Evkeeza is now indicated as an adjunct to diet and other lipid-lowering therapies to treat children aged six months and older with homozygous familial hypercholesterolemia (HoFH). The EC’s approval makes Evkeeza the first HoFH medicine indicated for this pediatric population in the EU.H ...

NOVATO, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 AM PT. The live and archived webcast of the presentation will b ...