RARE Ultragenyx Pharmaceutical Inc.

+1.56  (+3%)
Previous Close 58.26
Open 58.34
Price To Book 4.57
Market Cap 3025672809
Shares 50,579,619
Volume 418,893
Short Ratio
Av. Daily Volume 574,886

Drug Information

Drug catalyst information is displayed when you hover over the stage bar graph.

Phase 1/2 high dose Cohort 3 to be initiated 2018 with data due mid-2019.
Ornithine Transcarbamylase (OTC) Deficiency
Approved under priority review November 15, 2017.
MEPSEVII (vestronidase alfa)
Mucopolysaccharidosis 7 (MPS 7)
Phase 3 data released October 26, 2018 - endpoints not met.
UX007 (triheptanoin)
Glut1 DS patients with the movement disorder phenotype
Phase 3 data released August 22, 2017 - endpoints not met.
Aceneuramic acid extended release (Ace-ER)
GNE Myopathy
Approval announced April 17, 2018.
Crysvita (Burosumab)
X-Linked Hypophosphatemia (XLH)
Data released September 2018.
KRN23 Burosumab
Tumor-induced osteomalacia (TIO)
NDA to be filed mid-2019.
UX007 (triheptanoin)
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
sBLA filing for children due 2H 2019.
KRN23 Burosumab
X-Linked Hypophosphatemia (XLH)
Phase 1/2 positive low dose cohort data released January 4, 2019. Data from second cohort due mid-2019.

Latest News

  1. Edited Transcript of RARE earnings conference call or presentation 19-Feb-19 10:00pm GMT
  2. Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia
  3. Report: Exploring Fundamental Drivers Behind Canopy Growth, Adobe, Ultragenyx Pharmaceutical, Humana, Ship Finance International, and NexGen Energy — New Horizons, Emerging Trends, and Upcoming Developments
  4. Here's Why This Small Biotech Stock Just Hit A Four-Month High
  5. Ultragenyx (RARE) Posts Narrower-Than-Expected Loss in Q4
  6. Ultragenyx Pharmaceutical Inc (RARE) Q4 2018 Earnings Conference Call Transcript
  7. Ultragenyx: 4Q Earnings Snapshot
  8. Ultragenyx Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Update
  9. Ultragenyx Pharmaceutical, Inc. to Host Earnings Call
  10. Ultragenyx and Kyowa Kirin Announce Positive 64-Week Results for Crysvita® (burosumab) from Phase 3 Study in Children with X-linked Hypophosphatemia (XLH)
  11. Will Ultragenyx (RARE) Report Negative Q4 Earnings? What You Should Know
  12. Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2018 Financial Results and Corporate Update
  13. See what the IHS Markit Score report has to say about Ultragenyx Pharmaceutical Inc.
  14. Is Ultragenyx Pharmaceutical Inc.’s (NASDAQ:RARE) Liquidity Good Enough?
  15. Ultragenyx Appoints Shehnaaz Suliman, M.D., to Board of Directors
  16. Ultragenyx's Fatty Acid Disorder Candidate Succeeds in Study
  17. Ultragenyx stock up 2.9% after safety and efficacy trial reaffirms Phase 2 data
  18. Ultragenyx Announces Positive Topline Data from Ongoing Long-Term Extension Study of UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders
  19. J&J (JNJ) to Commence Pharma Q4 Earnings: What to Expect