Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX016, a Sialic Acid Prodrug for the Treatment of GNE Myopathy
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
Check Earnings Report
US:NASDAQ Investor Relations:
ir.ultragenyx.com/investor-relations
Company Research
Source: GlobeNewswire
NOVATO, Calif., March 30, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX016, an investigational small molecule prodrug of sialic acid (SA) being evaluated as a substrate replacement therapy for GNE myopathy (GNEM). GNEM is a rare, severely debilitating, inherited neuromuscular disorder caused by mutations in the GNE gene that lead to deficient SA production. The UX016 program is externally funded by a patient group through clinical proof-of-concept, including a Phase 1/2 study expected to begin in the second half of 2026. “People living with GNE myopathy face a profound and progressive loss of muscle function that affects every aspect of daily life, yet there are currently no approved treatment options to slow or alter the course of the disease,” said Coleman Kennedy, chief executive officer of the Neuromuscular Disease Foundatio
Show less
Read more
Impact Snapshot
Event Time:
RARE
Last Price
Price Change
Price Change%
Volume Shares
Max Up
Max Down
%
Volume Ratio
Performance Comparison
Updated
|
Event Day
Stock performance from the time of news release until the following 4pm ET market close
|
Last price at news event
|
Since Event
Multi-day stock performance from the time of the news release
|
|---|---|---|
|
EVENT DAY
Stock performance from the time of news release until the following 4pm ET market close
|
LAST
PRICE AT NEWS EVENT |
SINCE EVENT
Multi-day stock performance from the time of the news release
|
| Last Price | ||
| VWAP | ||
|
High:
|
Max Up |
High:
|
|
Low:
|
Max Down |
Low:
|
|
%
|
Post news range |
%
|
|
|
Price change |
|
|
|
Price Change Percentage |
|
|
|
S&P 500 (SPX) |
|
|
%
|
Volume ratio |
%
|
| Volume | ||
| Ticks | ||
|
|
Avg transaction size |
|
Don't Miss Out On The Next BIG Stock Move
Event day
Event Day Chart will not be displayed beyond 90-day period of the event
Performance since event
Time And Velocity Analysis
Be the first to know
Opt in for alerts from News Quantifed
Sign Up Today
Opt-in for
RARE alerts
Learn More With NQ
Reading the Impact Report
Help Me Understand This Data
Help Me Understand This Data
How NQ Works
What is news-based trading?
And why is it so successful?
And why is it so successful?
Movers & Shakers
The biggest gainers and losers and WHY it moved
RARE alerts
High impacting Ultragenyx Pharmaceutical Inc. news events
Weekly update
A roundup of the hottest topics
RARE
News
- RARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud Lawsuit with the Schall Law FirmGlobeNewswire
- Ultragenyx Pharmaceutical Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RAREPR Newswire
- RARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud Lawsuit with the Schall Law FirmPR Newswire
- INVESTOR ALERT: Ultragenyx Pharmaceutical Inc. (RARE) Investors with Substantial Losses Have Opportunity to Lead Securities Class Action Lawsuit - RGRD Law [TheStreet.com]TheStreet.com
- INVESTOR ALERT: Ultragenyx Pharmaceutical Inc. (RARE) Investors with Substantial Losses Have Opportunity to Lead Securities Class Action Lawsuit - RGRD LawGlobeNewswire
RARE
Earnings
- 2/12/26 - Miss
RARE
Sec Filings
- 3/30/26 - Form 8-K
- 3/27/26 - Form ARS
- 3/27/26 - Form DEFA14A
- RARE's page on the SEC website