Corporate Deck
May 19, 2021
Legal Warning
Cautionary note regarding forward-lookingstatements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations and projections regarding our future operating results and financial performance, anticipated cost or expense reductions, plans with respect to commercializing our product and product candidates, our translational research program, expectations regarding our manufacturing capabilities, the expected timing of release of additional data for our product candidates, plans to initiate additional studies for product candidates and timing and design of these studies, plans regarding ongoing studies for existing programs, our liquidity position as of the most recent fiscal quarter end, expectations regarding the adequacy of clinical data to support marketing applications and approvals of product candidates, our intent to file, and potential timing and success of, marketing applications and other regulatory approvals, expectations regarding timing of receiving potential approval of product candidates, expectations regarding prevalence of patients, future regulatory interactions, and the value to be generated by our pipeline. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, our reliance on our third party partner, Kyowa Kirin Co., Ltd., for the supply of Crysvita, the effects from the COVID-19 pandemic on our clinical trial activities, business and operating results, smaller than anticipated market opportunities for our products and product candidates, manufacturing risks, competition from other therapies or products, uncertainties related to insurance coverage and reimbursement status of our newly approved products, our evolving integrated commercial organization, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of our regulatory filings the uncertainties inherent in the clinical drug development process, including the potential for substantial delays and risk that earlier study results may not be predictive of future study results, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the availability or commercial potential of our product and product candidates, and our ability to integrate acquired businesses, which are more fully described in our most recent Form 10-Q or Form 10-K under the caption "Risk Factors" and elsewhere in such reports. Any forward-looking statements made by us reflect our current views with respect to future events or to our future financial performance and involve known and unknown risks, uncertainties, and other factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by these forward-looking statements. Accordingly, our actual results may materially differ from our current expectations, estimates, and projections. Given these uncertainties, you should not place undue reliance on these forward-looking statements.
Any forward-looking statements made by us in this presentation speak only as of the date of this presentation and represent our estimates and assumptions only as of the date of this presentation. Except as required by law, we assume no obligation, and we disclaim any intent, to update these statements to reflect actual results.
This presentation concerns commercial products as well as discussion of investigational drugs that are under preclinical and/or clinical investigation and which have not yet been approved for marketing by the U.S. Food and Drug Administration (FDA). They are currently limited by Federal law to investigational use, and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated.
Ultragenyx, Ultragenyx Pharmaceutical, Ultragenyx Gene Therapy, Mepsevii, Dojolvi and our logo are our trademarks. Any other trademarks appearing in these slides are
2the property of their respective holders.
Building an Exceptional Rare Disease Company with Value Drivers Across Commercial, Clinical, and Platforms
Commercial
Expansion
Clinical
Development
Catalysts
Gene Therapy Manufacturing
- Continued Crysvita growth
- Strong Dojolvi launch for LC-FAOD
- Initiate four pivotal studies (including three gene therapy)
- Data catalysts and study initiations
- HeLa PCL: High quality, commercial scale
- Supports larger indications with reduced COGS
3
2017-2020: Completing Four Approvals and Launches
Refilling the pipeline through efficient business development
Pipeline Approvals
MPS VII | TIO | LC-FAOD | |
XLH | |||
2017 | 2018 | 2019 | 2020 | 2021+ | ||
Pipeline Build | ||||||
OTC AAV8 | GSDIa AAV8 | Wilson AAV | GENE THERAPY | |||
UX701 | Duchenne | |||||
DTX301 | DTX401 | CDKL5 Def. AAV | UX810 | |||
UX055 | ||||||
GSD III mRNA/LNP | Angelman-ASO | NUCLEIC ACID | ||||
UX053 | GTX102 | |||||
Osteogenesis MAb | ||||||
Imperfecta | ||||||
UX143 |
4
Three Therapeutic Areas Approaching $1 Billion of Product Revenue in the Next Five Years
COMMERCIAL CLINICAL
BONE ENDOCRINE | METABOLICS | |
Ph 3 | Ph 3 |
Ph 2/3
Ph | |
Ph 1/2 | 1/2/3 |
Setrusumab | UX053 | UX701 | DTX401 | DTX301 |
for OI | for GSDIII | for Wilson | for GSDIa | for OTC |
XLH | TIO | MPS VII | LC-FAOD | |
CNS / MUSCLE
Ph 1/2
GTX-102
for Angelman
5
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Ultragenyx Pharmaceutical Inc. published this content on 19 May 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 19 May 2021 12:49:05 UTC.