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Ultragenyx Pharmaceutical Inc. (RARE)

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical firm dedicated to discovering, developing, and commercializing innovative treatments for rare and ultra-rare genetic conditions. Its operations span North America, Europe, and other international markets. The company's portfolio of marketed biologic products addresses several serious diseases. This includes Crysvita (burosumab), an antibody that targets fibroblast growth factor 23, used to treat X-linked hypophosphatemia and tumor-induced osteomalacia. Mepsevii offers enzyme replacement therapy for both pediatric and adult patients suffering from Mucopolysaccharidosis VII. Dojolvi is available for individuals with long-chain fatty acid oxidation disorders, while Evkeeza (evinacumab) provides a treatment option for homozygous familial hypercholesterolemia. Ultragenyx also boasts a robust pipeline of investigational therapies. Notable candidates include DTX401, an adeno-associated virus 8 (AAV8) gene therapy for glycogen storage disease type Ia, and DTX301, another AAV8 gene therapy aimed at ornithine transcarbamylase deficiency. Other promising developments feature UX143, a human monoclonal antibody for osteogenesis imperfecta; GTX-102, an antisense oligonucleotide designed for Angelman syndrome; UX701, targeting Wilson disease; and UX053, in development for glycogen storage disease type III. To advance its research and development efforts, Ultragenyx Pharmaceutical Inc. maintains strategic collaboration and licensing agreements with numerous partners, such as Kyowa Kirin Co., Ltd., Saint Louis University, REGENXBIO Inc., Bayer Healthcare LLC, GeneTx, Mereo, University of Pennsylvania, Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc., and Daiichi Sankyo Co., Ltd. The company was founded in 2010 and operates from its headquarters in Novato, California.

Sector: Pharmaceutical Preparations

Industry: Biotechnology

Ultragenyx Pharmaceutical Inc. logo

Ultragenyx Pharmaceutical Inc.

NASDAQ:RAREHealthcare
-4.05%$16.10
AH$16.10+0.00%vs close
Mkt Cap$1.59B
Float93.53M
O/S98.59M
Vol
$ Vol
RVOL
Inst Own-
Earnings
Dilution
Ultragenyx Pharmaceutical Inc. logoNASDAQ:RARE$16.10-4.05%
Phase Distribution
63 lead-sponsored trials·18 active·27 completed
Phase 11
Phase 1/29
Phase 216
Phase 2/32
Phase 313
Not applicable22
(28 more as collaborator)
Status Overview
Not yet recruiting1
Recruiting5
Active, not recruiting13
Completed27
Stopped15
Expanded access2

Trials need funding. See this company's dilution risk and cash position.

Clinical Trials
Sponsor role
Status
Phase
  • Phase 3
    Completed

    A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

    UX003

    MPS 7, Sly Syndrome +2

    Enrollment
    12
    Start
    Dec 2014
    Primary Completion
    May 2016
    Completion
    May 2016
    NCT02230566
  • Phase 3
    Completed

    Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

    aceneuramic acid extended-release (Ace-ER), Placebo

    Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles +2

    Enrollment
    89
    Start
    May 2015
    Primary Completion
    Jun 2017
    Completion
    Jun 2017
    NCT02377921
  • Phase 3
    Completed

    A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

    UX003

    Sly Syndrome, MPS VII +2

    Enrollment
    12
    Start
    Nov 2015
    Primary Completion
    Jan 2019
    Completion
    Jan 2019
    NCT02432144
  • Phase 3
    Terminated

    Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

    Aceneuramic Acid Extended-Release Tablets

    Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles +3

    Enrollment
    143
    Start
    May 2016
    Primary Completion
    Jan 2018
    Completion
    Jan 2018
    NCT02736188
  • Phase 3
    Terminated

    Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

    UX007, Placebo

    Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

    Enrollment
    44
    Start
    Apr 2017
    Primary Completion
    Oct 2019
    Completion
    Oct 2019
    NCT02960217
  • Phase 3
    Enrolling by invitation

    Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

    Adjuvant Immunomodulatory (IM) Therapy

    Mucopolysaccharidosis IIIA, MPS IIIA +2

    Enrollment
    41
    Start
    Sep 2020
    Primary Completion
    Aug 2027est.
    Completion
    Aug 2027est.
    NCT04360265
  • Phase 3
    Completed

    A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

    Oral prednisolone, Placebo for oral prednisolone

    Glycogen Storage Disease Type IA

    Enrollment
    52
    Start
    Nov 2021
    Primary Completion
    Feb 2024
    Completion
    Feb 2026
    NCT05139316
  • Phase 3
    Active, not recruiting

    Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

    Oral Corticosteroids, Placebo for oral corticosteroids

    OTC Deficiency

    Enrollment
    37
    Start
    Oct 2022
    Primary Completion
    Sep 2027est.
    Completion
    Mar 2031est.
    NCT05345171
  • Phase 3
    Active, not recruiting

    Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

    Bisphosphonate, Setrusumab

    Osteogenesis Imperfecta

    Enrollment
    69
    Start
    Jun 2023
    Primary Completion
    Oct 2025
    Completion
    Apr 2027est.
    NCT05768854
  • Phase 3
    Active, not recruiting

    A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

    Triheptanoin

    Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

    Enrollment
    69
    Start
    Feb 2023
    Primary Completion
    Aug 2027est.
    Completion
    Aug 2027est.
    NCT05933200
  • Phase 3
    Active, not recruiting

    Long-term Extension of GTX-102 in Angelman Syndrome

    GTX-102

    Angelman Syndrome

    Enrollment
    255
    Start
    Jul 2024
    Primary Completion
    Feb 2029est.
    Completion
    Feb 2029est.
    NCT06415344
  • Phase 3
    Active, not recruiting

    Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

    GTX-102

    Angelman Syndrome

    Enrollment
    129
    Start
    Dec 2024
    Primary Completion
    Jul 2026
    Completion
    Nov 2027est.
    NCT06617429
  • Phase 3
    Active, not recruiting

    Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta

    setrusumab

    Osteogenesis Imperfecta

    Enrollment
    6
    Start
    Oct 2024
    Primary Completion
    Jan 2027est.
    Completion
    Jan 2028est.
    NCT06636071
  • Phase 2/3
    Recruiting

    Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

    UX111, Prophylactic Immunomodulatory (IM) Therapy

    MPS IIIA, Sanfilippo Syndrome +2

    Enrollment
    36
    Start
    Apr 2016
    Primary Completion
    Mar 2029est.
    Completion
    Mar 2029est.
    NCT02716246
  • Phase 2/3
    Active, not recruiting

    Setrusumab vs Placebo for Osteogenesis Imperfecta

    Setrusumab

    Osteogenesis Imperfecta

    Enrollment
    183
    Start
    Feb 2022
    Primary Completion
    Oct 2025
    Completion
    Apr 2027est.
    NCT05125809
  • Phase 2
    Completed

    Safety and Efficacy of Multiple Dosing Regimens of BPS804 in Post Menopausal Women With Low Bone Mineral Density

    BPS804 20mg/Kg, Placebo to 20mg/Kg BPS804

    Osteopenia, Osteoporosis

    Enrollment
    44
    Start
    Jul 2011
    Primary Completion
    Oct 2013
    Completion
    Oct 2013
    NCT01406548
  • Phase 2
    Completed

    Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)

    BPS804

    Hypophosphatasia

    Enrollment
    8
    Start
    Jul 2011
    Primary Completion
    Sep 2012
    Completion
    Sep 2012
    NCT01406977
  • Phase 2
    Completed

    Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta

    BPS804

    Osteogenesis Imperfecta

    Enrollment
    10
    Start
    Jun 2011
    Primary Completion
    Dec 2012
    Completion
    Dec 2012
    NCT01417091
  • Phase 2
    Completed

    A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

    Sialic Acid Extended Release (SA-ER), Placebo

    GNE Myopathy, Hereditary Inclusion Body Myopathy

    Enrollment
    46
    Start
    May 2012
    Primary Completion
    Nov 2013
    Completion
    Nov 2013
    NCT01517880
  • Phase 2
    Withdrawn

    Study of Safety and Tolerability of BPS804 in Patients With Late-stage Chronic Kidney Disease

    BPS804, Placebo

    Chronic-kidney Disease Stage 5D on Stable Hemodialysis

    Enrollment
    0
    Start
    Aug 2013
    Primary Completion
    Apr 2014est.
    Completion
    Apr 2014est.
    NCT01806610
  • Phase 2
    Completed

    An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

    SA-ER 500 mg, SA-IR 500 mg

    GNE Myopathy, Hereditary Inclusion Body Myopathy (HIBM)

    Enrollment
    59
    Start
    Jun 2013
    Primary Completion
    Feb 2017
    Completion
    Feb 2017
    NCT01830972
  • Phase 2
    Completed

    A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

    UX007

    Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency +3

    Enrollment
    29
    Start
    Feb 2014
    Primary Completion
    Aug 2016
    Completion
    Aug 2016
    NCT01886378
  • Phase 2
    Completed

    Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

    UX007, Placebo

    Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

    Enrollment
    36
    Start
    Feb 2014
    Primary Completion
    Sep 2017
    Completion
    Sep 2017
    NCT01993186
  • Phase 2
    Completed

    Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies

    UX007

    Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency +3

    Enrollment
    94
    Start
    Dec 2014
    Primary Completion
    Dec 2020
    Completion
    Dec 2020
    NCT02214160
  • Phase 2
    Completed

    Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

    UX003

    Sly Syndrome, MPS VII +2

    Enrollment
    8
    Start
    Jul 2015
    Primary Completion
    Mar 2019
    Completion
    Mar 2019
    NCT02418455

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