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Ultragenyx Pharmaceutical Inc (RARE)

(70% Positive) Ultragenyx Pharmaceutical Inc (RARE) Announces Enrollment Update for program Due to Patient Enrollment Issues, Regulatory Process, Manufacturing Considerations, Safety Review

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  • News bot Jan. 29, 2026, 8:46 p.m.

    📋 Ultragenyx Pharmaceutical Inc (RARE) - Clinical Trial Update

    Filing Date: 2026-01-12

    Accepted: 2026-01-12 07:45:24

    Event Type: Clinical Trial Update

    Event Details:

    Ultragenyx Pharmaceutical Inc (RARE) Announces Clinical Trial Update Ultragenyx Pharmaceutical Inc (RARE) provided an update on its clinical development programs. Clinical Development Highlights:
    • Drug Program: program, stage
    • Diseases/Conditions: Angelman syndrome NOVATO, Angelman syndrome
    • Clinical Stage: Phase 3, Phase 3 program
    • Collaboration: Ultragenyx Provides Financial and Business Updates
    • Updated Timeline: December 31, 2025, year end 2025
      • expected in the first half of 2026
      • expected in the second half of 2026

    🔬 Clinical Development Pipeline (Ultragenyx Pharmaceutical Inc):

    Product Type Development Stage Therapeutic Area Source
    DTX401 GENETIC Phase PHASE3 Glycogen Storage Disease Type IA ClinicalTrials.gov
    Reference Drug or Test Drug DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    No Investigational Product OTHER Phase PHASE3 Mucopolysaccharidosis IIIA ClinicalTrials.gov
    GTX-102 DRUG Phase PHASE2 Angelman Syndrome ClinicalTrials.gov
    UX701 GENETIC Phase PHASE1 Wilson Disease ClinicalTrials.gov
    setrusumab BIOLOGICAL Phase PHASE3 Osteogenesis Imperfecta ClinicalTrials.gov
    Crysvita (burosumab-twza) Treatment DRUG Phase PHASE4 Epidermal Nevus Syndrome ClinicalTrials.gov
    UX111 BIOLOGICAL Phase PHASE2 MPS IIIA ClinicalTrials.gov
    Adjuvant Immunomodulatory (IM) Therapy DRUG Phase PHASE3 Mucopolysaccharidosis IIIA ClinicalTrials.gov
    No intervention OTHER Preclinical Tumor-induced Osteomalacia (TIO) ClinicalTrials.gov
    Triheptanoin DRUG Phase PHASE3 Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) ClinicalTrials.gov
    Mepsevii DRUG Preclinical MPS VII ClinicalTrials.gov
    Venipuncture PROCEDURE Preclinical Mucopolysaccharidoses ClinicalTrials.gov
    CWP232291 DRUG Phase PHASE1 Acute Myeloid Leukemia ClinicalTrials.gov
    BAY2599023 (DTX201) DRUG Phase PHASE1 Hemophilia A ClinicalTrials.gov
    Adjuvant IM Therapy DRUG Phase PHASE2 MPS IIIA ClinicalTrials.gov
    Optimized Prophylactic IM Therapy DRUG Phase PHASE2 MPS IIIA ClinicalTrials.gov
    Prophylactic Immunomodulatory (IM) Therapy DRUG Phase PHASE2 MPS IIIA ClinicalTrials.gov
    Sodium Acetate DRUG Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Placebo for oral corticosteroids DRUG Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Oral Corticosteroids DRUG Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Placebo OTHER Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    DTX301 GENETIC Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Standard of Care (SOC) DRUG Phase PHASE1 Wilson Disease ClinicalTrials.gov
    HEALON DEVICE Approved Cataract Surgery ClinicalTrials.gov
    IXIUM TWIN DEVICE Approved Cataract Surgery ClinicalTrials.gov
    Bisphosphonate DRUG Phase PHASE3 Osteogenesis Imperfecta ClinicalTrials.gov
    Placebo for oral prednisolone DRUG Phase PHASE3 Glycogen Storage Disease Type IA ClinicalTrials.gov
    Oral prednisolone DRUG Phase PHASE3 Glycogen Storage Disease Type IA ClinicalTrials.gov
    Sham-LP PROCEDURE Phase PHASE3 Angelman Syndrome ClinicalTrials.gov
    GLUT1 DS DRUG Phase PHASE2 Glut1 Deficiency Syndrome ClinicalTrials.gov
    Sialic Acid Extended Release (SA-ER) Tablets DRUG Phase PHASE1 Hereditary Inclusion Body Myopathy (HIBM) ClinicalTrials.gov
    Sialic Acid Extended Release (SA-ER) Tables DRUG Phase PHASE1 Hereditary Inclusion Body Myopathy (HIBM) ClinicalTrials.gov
    Sialic Acid Extended Release (SA-ER) DRUG Phase PHASE2 GNE Myopathy ClinicalTrials.gov
    Vegetable Oil OTHER Phase PHASE2 Adult Polyglucosan Body Disease ClinicalTrials.gov
    Tridecanoic Acid DRUG Phase PHASE2 Rett Syndrome ClinicalTrials.gov
    SA-IR 500 mg DRUG Phase PHASE2 GNE Myopathy ClinicalTrials.gov
    SA-ER 500 mg DRUG Phase PHASE2 GNE Myopathy ClinicalTrials.gov
    POST Triheptanoin DRUG Phase PHASE1 Frontal Lobe Hypometabolism ClinicalTrials.gov
    UX003 DRUG Phase PHASE2 Sly Syndrome ClinicalTrials.gov
    Aceneuramic Acid Extended-Release DRUG Phase PHASE2 Hereditary Inclusion Body Myopathy ClinicalTrials.gov
    UX007 DRUG Phase PHASE2 Glucose Transporter Type 1 Deficiency Syndrome ClinicalTrials.gov
    DTX101 GENETIC Phase PHASE1 Hemophilia B ClinicalTrials.gov
    Placebo oil OTHER Phase PHASE2 Glycogen Storage Disease Type V ClinicalTrials.gov
    aceneuramic acid extended-release (Ace-ER) DRUG Phase PHASE3 Hereditary Inclusion Body Myopathy ClinicalTrials.gov
    Burosumab BIOLOGICAL Phase PHASE3 Hypophosphatemia ClinicalTrials.gov
    BPS804 DRUG Phase PHASE2 Osteogenesis Imperfecta ClinicalTrials.gov
    [6,6-2H2]glucose BIOLOGICAL Phase PHASE1 Glycogen Storage Disease Type IA ClinicalTrials.gov
    Placebo to 20mg/Kg BPS804 DRUG Phase PHASE2 Osteopenia ClinicalTrials.gov
    BPS804 20mg/Kg DRUG Phase PHASE2 Osteopenia ClinicalTrials.gov
    steroid regimen DRUG Phase PHASE1 GSD1 ClinicalTrials.gov
    Aceneuramic Acid Extended-Release Tablets DRUG Phase PHASE3 Hereditary Inclusion Body Myopathy ClinicalTrials.gov
    ABO-102 DRUG Phase PHASE1 MPS IIIA ClinicalTrials.gov
    zoledronic acid (optional) DRUG Phase PHASE2 Osteogenesis Imperfecta, Type I ClinicalTrials.gov
    Prophylactic Corticosteroid Taper Regimen DRUG Phase PHASE1 Ornithine Transcarbamylase (OTC) Deficiency ClinicalTrials.gov
    Reactive Corticosteroid Taper Regimen DRUG Phase PHASE1 Ornithine Transcarbamylase (OTC) Deficiency ClinicalTrials.gov
    scAAV8OTC GENETIC Phase PHASE1 Ornithine Transcarbamylase (OTC) Deficiency ClinicalTrials.gov
    Group 6: C2207(Period 1), JW0104(Period 2), JW0104 + C2207(Period 3) DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    Group 5: JW0104 + C2207(Period 1), C2207(Period 2), JW0104(Period 3) DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    Group 4: JW0104(Period 1), JW0104 + C2207(Period 2), C2207(Period 3) DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    Group 3: C2207(Period 1), JW0104 + C2207(Period 2), JW0104(Period 3) DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    Group 2: JW0104 + C2207(Period 1), JW0104(Period 2), C2207(Period 3) DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    Group 1: JW0104(Period 1), C2207(Period 2), JW0104 + C2207(Period 3) DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    Pitavastatin DRUG Phase PHASE4 Osteoporosis, Osteopenia ClinicalTrials.gov
    LEO 152020 placebo tablet DRUG Phase PHASE2 Atopic Dermatitis ClinicalTrials.gov
    LEO 152020 tablet DRUG Phase PHASE2 Atopic Dermatitis ClinicalTrials.gov
    Triheptanoin oil DRUG Phase PHASE2 Huntington Disease ClinicalTrials.gov
    LEO 152020 placebo DRUG Phase PHASE2 Cholinergic Urticaria ClinicalTrials.gov
    LEO 152020 DRUG Phase PHASE2 Cholinergic Urticaria ClinicalTrials.gov
    Moxifloxacin DRUG Phase PHASE1 Healthy Volunteers ClinicalTrials.gov
    Intra-articular injection of hyaluronic acid DEVICE Preclinical Gonarthrosis ClinicalTrials.gov
    H1 Blocker DRUG Phase PHASE1 Glycogen Storage Disease Type III ClinicalTrials.gov
    H2 Blocker DRUG Phase PHASE1 Glycogen Storage Disease Type III ClinicalTrials.gov
    Antipyretic DRUG Phase PHASE1 Glycogen Storage Disease Type III ClinicalTrials.gov
    UX053 BIOLOGICAL Phase PHASE1 Glycogen Storage Disease Type III ClinicalTrials.gov
    VA DRUG Phase PHASE3 Dyslipidemia ClinicalTrials.gov
    LivaloV DRUG Phase PHASE3 Dyslipidemia ClinicalTrials.gov
    LivaloVA DRUG Phase PHASE3 Dyslipidemia ClinicalTrials.gov
    ARTHRUM H 2% DEVICE Preclinical Knee Osteoarthritis ClinicalTrials.gov
    Test Drug(JW0302) or Reference Drug(C2206) DRUG Phase PHASE1 Healthy ClinicalTrials.gov
    ARTHRUM 2.5% DEVICE Preclinical Knee Osteoarthritis ClinicalTrials.gov
    Evinacumab DRUG Phase PHASE3 Homozygous Familial Hypercholesterolemia ClinicalTrials.gov

    💼 Business Developments:

    • Partnership
    • Acquisition: Not available
    • Licensing: Not available
    • Regulatory Approval
    • Executive Changes: Not available

    Structured Data:

    • Company Name: Ultragenyx Pharmaceutical Inc
    • Ticker Symbol: RARE

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