Core Insights - Ultragenyx Pharmaceutical reported promising findings from its pivotal study of UX111, a gene therapy for Sanfilippo syndrome type A (MPS IIIA) [1][2][8] Clinical Findings - The phase I/II/III Transpher A study showed statistically significant improvements in cognitive and communication skills in MPS IIIA patients treated with UX111, alongside a notable reduction in cerebrospinal fluid (CSF) heparan sulfate levels [2][3] - Treatment with UX111 resulted in a median CSF-HS reduction of 65% across all treated patients and 66% in the modified intention-to-treat (mITT) group, with an average follow-up duration of 34 months [3] - All 10 patients excluded from the mITT group due to older age or advanced disease retained meaningful functional abilities, which is significant as such functions typically deteriorate in late childhood and early adolescence [5] Safety Profile - All doses of UX111 were reported to be generally safe and well-tolerated, with treatment-related adverse events mostly mild to moderate in severity [6] Regulatory Developments - Ultragenyx submitted a biologics license application (BLA) to the FDA for UX111 under the accelerated approval pathway, with a final decision expected in the second half of 2025 [8][9] - If approved, UX111 will be the first FDA-approved therapy for MPS IIIA in the United States, affecting approximately 3,000 to 5,000 patients [9] Pipeline Overview - Ultragenyx has several other gene therapy candidates in its pipeline, including UX143 for osteogenesis imperfecta and UX701 for Wilson disease, with various stages of development and regulatory designations [11][12][13] Market Performance - In the past three months, shares of Ultragenyx (RARE) have declined by 10%, compared to an 8.7% decline in the industry [4]

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 ...

Core Insights - Tactical Resources Corp. is advancing its flagship Peak Project and pursuing a proposed business combination with Plum Acquisition Corp. III to enhance its position in the rare earth elements (REEs) market [2][3][4] Business Combination - The proposed business combination with Plum, announced in August 2024, aims to facilitate a Nasdaq listing and provide capital to accelerate production at the Peak Project [3][10] - The transaction is expected to close in the first half of 2025, converting existing shareholders into a newly-formed public company [10] Peak Project Developments - The Peak Project, located in Texas, is positioned as a critical contributor to the U.S. REE supply chain, with operational permits secured and access to REE-enriched tailings [5][9] - Tactical Resources has made significant progress in its Phase 1 technical work, including bench-scale and vat-leach testing, which will inform future production planning [7][9] Strategic Importance - The geopolitical landscape, particularly China's export restrictions on critical minerals, underscores the need for a reliable U.S.-based REE supply chain [6][8] - Tactical Resources aligns its operations with U.S. national defense goals, as REEs are essential for defense applications [8][9] Future Outlook - The company aims to leverage its 2024 achievements to become a leading U.S. producer of REE materials, capitalizing on market opportunities and delivering long-term shareholder value [11]

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 ...

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 ...

Core News Summary - The European Commission (EC) has extended the approval of Evkeeza (evinacumab) for treating children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH), making it the first and only medicine approved in the EU for this age group [1][2] - Evkeeza is an ANGPTL3 inhibitor that reduces LDL-C levels and is now indicated for pediatric patients as young as 6-months old, addressing a critical unmet need in managing HoFH [1][5] Efficacy and Safety - Model-based extrapolation analysis predicts that pediatric patients aged 6-months to less than 5 years will experience a similar or higher magnitude of LDL-C reduction at week 24 compared to adults when receiving a 15 mg/kg dose every 4 weeks [2] - Compassionate use data for five patients aged 1-4 years showed clinically meaningful LDL-C reductions consistent with older patients, with no new safety concerns identified [2] Market and Availability - Evkeeza is now reimbursed and commercially available in the UK, US, Canada, Italy, Japan, the Netherlands, Spain, and Luxembourg, with early access schemes in 13 additional countries including Austria and France [2] - The treatment was initially approved for adults and adolescents aged 12+ in June 2021, with the indication extended to children aged 5-11 in December 2023 [2] Disease Background - HoFH is an ultrarare genetic disorder affecting 1 in 300,000 people globally and approximately 1,600 in the EU, characterized by dangerously high LDL-C levels (>400 mg/dL) and early cardiovascular risks [3][4] Product Mechanism - Evkeeza works by blocking the ANGPTL3 protein, which regulates cholesterol levels, and is administered via monthly infusions [5] Development and Commercialization - Evkeeza was discovered and developed by Regeneron Pharmaceuticals, Inc, with Ultragenyx responsible for its development and commercialization outside the US [7] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing novel therapies for rare and ultrarare genetic diseases, with a diverse portfolio addressing high unmet medical needs [12][13]

Ultragenyx Pharmaceutical (RARE) announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway.Earlier this year, the FDA aligned with Ultragenyx, after a thorough evaluation, on the use of cerebral spinal fluid (CSF) heparan sulfate (HS) as a well-characterized biomarker to support an accelerated approval pathway for mucopolysaccharidoses ...