Ultragenyx Sells European Crysvita Rights to Novo Nordisk for $300M

TL;DR

UGYX sells EU Crysvita rights to NVO for $300M upfront, focusing on US/Canada.

AI Summary

Ultragenyx Pharmaceutical Inc. announced on July 17, 2024, that it has entered into a definitive agreement to sell its European commercialization rights for the drug Crysvita to Novo Nordisk for an upfront payment of $300 million. The agreement also includes potential milestone payments and royalties, with Ultragenyx retaining rights in the U.S. and Canada. This transaction is expected to close in the third quarter of 2024.

Why It Matters

This strategic divestiture allows Ultragenyx to focus on its pipeline and U.S. commercialization efforts while securing significant upfront capital. For Novo Nordisk, it expands their rare disease portfolio in Europe.

Risk Assessment

Risk Level: medium — The deal is subject to customary closing conditions and regulatory approvals, and the actual value could change based on future milestone payments and royalties.

Key Numbers

• $300M — Upfront Payment (Ultragenyx receives this amount from Novo Nordisk for European Crysvita rights.)

Key Players & Entities

• Ultragenyx Pharmaceutical Inc. (company) — Seller
• Crysvita (drug) — Product
• Novo Nordisk (company) — Buyer
• $300 million (dollar_amount) — Upfront payment
• July 17, 2024 (date) — Announcement date
• third quarter of 2024 (date) — Expected closing period

FAQ

Key Financial Figures

• $0.001 — nge on which registered Common Stock, $0.001 par value RARE Nasdaq Global Select

Filing Documents

• rare-20240717.htm (8-K) — 48KB
• 0000950170-24-084254.txt ( ) — 158KB
• rare-20240717.xsd (EX-101.SCH) — 23KB
• rare-20240717_htm.xml (XML) — 4KB

01 Other Events

Item 8.01 Other Events. On July 17, 2024, Ultragenyx Pharmaceutical Inc. (the "Company") announced the successful completion of an end-of-Phase 2 ("EoP2") meeting with the U.S. Food and Drug Administration ("FDA"), supporting its Phase 3 study plans for GTX-102, an antisense oligonucleotide for Angelman syndrome. Phase 3 design and next steps The EoP2 meeting focused on discussion of the Company's interim Phase 1/2 data and resulted in alignment with the FDA on the Phase 3 study design and endpoints. The pivotal Phase 3, will be a global, randomized, double-blind, sham-controlled trial and will include a 48-week primary efficacy analysis period enrolling approximately 120 patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The primary endpoint will be improvement in cognition assessed by Bayley-4 cognitive raw score. Control patients completing the study will be eligible to roll over onto treatment after the double-blind period is over. Previously disclosed results from the Phase 1/2 study showed that UBE3A gene deletion patients treated with GTX-102 experienced rapid, progressive and clinically significant improvement in cognition, as assessed by Bayley-4, that was far greater than the minimal change observed in Natural History data (1) in deletion patients. UBE3A gene deletion patients are at the severe end of the clinical spectrum, with lower Bayley scores at baseline, and demonstrate a much slower rate of skill attainment compared to, for example, UBE3A missense mutation patients, who demonstrate higher Bayley cognition improvement in Natural History data (2) . In the Phase 1/2 study, GTX-102 treated patients also demonstrated meaningful improvements in other domains of communication, motor function, sleep problems, and behavior. The Phase 3 study will include the key secondary endpoint of the Multi-domain Responder Index (MDRI) across all five domains of cognition, receptive communication, behav

SIGNATURES

SIGNATURES Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized. Ultragenyx Pharmaceutical Inc. Date: July 17, 2024 By: /s/ Howard Horn Howard Horn Executive Vice President, Chief Financial Officer, Corporate Strategy

View Full Filing

View this 8-K filing on SEC EDGAR