Ultragenyx Analyst Day Presentation
Key licenses & intellectual property – Commercial products
Product
CRYSVITAⓇ
(XLH, TIO)
MEPSEVII®
(MPS 7)
License
Kyowa Kirin Co. (KKC)
St. Louis University (Know-How)
N/A (IP Owned by Ultragenyx)
Baylor Research Institute (BRI)
DOJOLVIⓇ
(LC-FAOD)
N/A (IP Owned by Ultragenyx)
Product
License
EVKEEZA®
(HOFH)
Regeneron
Includes granted U.S. patent term extension
2Includes projected U.S. patent term extension
³Includes projected extension via supplementary protection certificates (SPCs)
20
Confidential and Proprietary
US Intellectual Property Rights/Royalties
• Anti-FGF23 antibodies and use for treatment of XLH and TIO (2023-2032)¹
Q2W dosing for treatment of FGF23-associated hypophosphatemic disorders (2035)
See discussion of KKC license and collaboration in annual report for royalty summary
Low single-digit royalty until expiration of orphan drug exclusivity
Recombinant human GUS (rhGUS) and use for treatment of MPS7 (2035)
Compositions comprising triheptanoin (2025-2029)²
Mid single-digit royalty
Ultrapure triheptanoin and use in treatment of FAOD (Pending; 2034)
EU Intellectual Property Rights/Royalties + Milestones
Evkeeza antibody and use for treatment of HOFH (2036)³
Evkeeza antibody in combination with other agents for treatment of HOFH (Pending: 2037)
Stabilized formulations of Evkeeza (Pending: 2041)
Regeneron supplies product and charges Ultragenyx a transfer price from the low 20% range up to 40% on net sales
Ultragenyx to pay up to $63M in potential regulatory and sales milestones
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