Recent Updates
Recently added Catalysts
RARE Positive Sentiment

Ultragenyx to Present Pipeline Update at Analyst Day on Monday, October 16, 2023

Key Takeaway: Ultragenyx Pharmaceutical Inc. will hold an Analyst Day on October 16, 2023, to present updates on its development pipeline. The agenda will feature discussions on UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome. Notable experts, including principal investigators from prominent universities, will participate in panel discussions. A live webcast will be available for those interested in following the updates on their therapeutic advancements.
Price reaction · baseline $34.24 (2023-10-04 close) · hit after-hours · 1 other RARE headline(s) in the window, move may be shared
day 0 close · peak
+2.1%

Market Sentiment Analysis

POSITIVE FACTORS

  • Upcoming Analyst Day will provide insights into development pipeline.
  • Discussion on innovative therapies for rare genetic diseases.
  • External expert panels will enhance credibility of presentations.

BiopharmaWatch Analysis

From our catalyst data and publicly available data · not financial advice
Best trade, last catalyst
+43%
120-day peak, hindsight
Typical move
2.9%
average across 6 past catalysts
Cash runway
~13 mo
Low dilution risk
Lead asset
BPS804
Phase 2 · Hypophosphatasia

Full Press Release Details

NOVATO, Calif., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, will host an Analyst Day at 8:30 a.m. ET on Monday, October 16, 2023. Ultragenyx leaders and external experts will provide an update on the company’s development pipeline.
The agenda will include updates on UX143 for osteogenesis imperfecta (OI), GTX-102 for Angelman syndrome (AS), and the company’s gene therapy programs and platform. Two external investigator panels will be featured:
Panel discussion on setrusumab (UX143) for the treatment of osteogenesis imperfects (OI)
Thomas Carpenter, M.D., Professor of Pediatrics (Endocrinology) and of Orthopaedics and Rehabilitation, Yale School of Medicine and Medical Director, Yale Center for Clinical Investigation's Hospital Research Unit, and principal investigator for UX143 Phase 3 program
Gary Gottesman, M.D., Professor of Pediatrics and Medicine, Washington University School of Medicine in St. Louis, and principal investigator for UX143 Phase 3 program
Heather Byers, M.D., senior medical director at Ultragenyx
Panel discussion on GTX-102 for the treatment of Angelman syndrome (AS)
Elizabeth Berry-Kravis, M.D., Ph.D., Professor, Department of Pediatrics, RUSH Medical College, and principal investigator for GTX-102 Phase 1/2 study
Kemi Olugemo, M.D., F.A.A.N., vice president of global clinical development at Ultragenyx
A live video webcast of the program will be available at https://www.webcaster4.com/Webcast/Page/359/49192. An archived version of the remarks will also be available through the Ultragenyx website.
About Ultragenyx Pharmaceutical Inc.
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 4, 2023 and its subsequent periodic reports filed with the Securities and Exchange Commission.
In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s investor relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/)
Contacts – Ultragenyx Pharmaceutical, Inc.

Frequently Asked Questions

When is Ultragenyx's Analyst Day planned?

Ultragenyx's Analyst Day is scheduled for 8:30 a.m. ET on October 16, 2023.

What topics will be discussed at the Analyst Day?

The agenda includes updates on UX143 for osteogenesis imperfecta and GTX-102 for Angelman syndrome.

Who are the external experts participating in the panels?

Notable experts include Thomas Carpenter, Gary Gottesman, Elizabeth Berry-Kravis, and Kemi Olugemo.

How can I watch the Analyst Day live?

A live video webcast will be available on the Ultragenyx website.

What is Ultragenyx focused on developing?

Ultragenyx develops novel therapies for rare and ultrarare genetic diseases.

Last updated: Oct 5, 2023