Ultragenyx-JPM-202401.pdf

1、42nd AnnualJ.P.Morgan Healthcare ConferenceJanuary 2024Emil D.Kakkis,M.D.,Ph.D.CEO and President Confidential and ProprietaryForward looking statementsCautionary note regarding forward-looking statements:This presentation contains forward-looking statements,including,but not limited to,statements re

2、garding 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 capabi

3、lities,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

4、,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 pr

5、oduct 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 ach

6、ievements 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,fluctuations in buying or distribution patterns from distribut