Ultragenyx Pharmaceutical Inc. Files 2023 Annual Report

TL;DR

<b>Ultragenyx Pharmaceutical Inc. filed its 2023 10-K report detailing financial performance, collaborations, and asset information.</b>

AI Summary

Ultragenyx Pharmaceutical Inc. (RARE) filed a Annual Report (10-K) with the SEC on February 21, 2024. Filed 10-K for the fiscal year ending December 31, 2023. Includes data related to leasehold improvements, asset-backed securities, and various license agreements. Details on employee stock plans and common stock transactions are provided. Mentions collaborations and agreements with entities like Daiichi Sankyo Co. Ltd. and REGENXBIO Incorporated. Financial data includes information on cost of sales and royalty payments.

Why It Matters

For investors and stakeholders tracking Ultragenyx Pharmaceutical Inc., this filing contains several important signals. This filing provides a comprehensive overview of Ultragenyx's financial health and operational activities for the fiscal year 2023, crucial for investors assessing performance and future prospects. The report contains specific details on various agreements and collaborations, offering insight into the company's strategic partnerships and potential revenue streams.

Risk Assessment

Risk Level: medium — Ultragenyx Pharmaceutical Inc. shows moderate risk based on this filing. The company operates in the highly regulated pharmaceutical industry, facing risks related to drug development, clinical trials, and market approval, as evidenced by the numerous agreements and product mentions.

Analyst Insight

Review the detailed financial statements and disclosures in the 10-K to understand the company's revenue recognition, cost structure, and the financial implications of its ongoing collaborations and product pipeline.

Revenue Breakdown

Key Numbers

• 2023-12-31 — Fiscal Year End (Reporting period for the 10-K.)
• 2024-02-21 — Filing Date (Date the 10-K was filed.)
• 0.25 — Leasehold Improvements (Value related to leasehold improvements.)
• 0.0208 — Leasehold Improvements (Value related to leasehold improvements.)

Key Players & Entities

• Ultragenyx Pharmaceutical Inc. (company) — Filer
• Daiichi Sankyo Co. Ltd. (company) — Licensor in a license and technology access agreement.
• REGENXBIO Incorporated (company) — Party to a license agreement.
• Dojolvi (product) — Product mentioned in relation to revenue and cost of sales.
• Kyowa Kirin Collaboration (company) — Collaboration involving royalty payments.
• Solid Biosciences Inc. (company) — Mentioned in relation to common stock.
• Matthew Fust (person) — Individual associated with the filing.
• University of Pennsylvania School of Medicine (company) — Party to a research collaboration and license agreement.

FAQ

Risk Factors

• Regulatory Risks [high — regulatory]: The pharmaceutical industry is subject to extensive regulation, impacting product development, approval, and marketing.
• Market Competition [medium — market]: Competition from other pharmaceutical companies can affect market share and pricing.
• Financial Risks [medium — financial]: The company faces financial risks related to drug development costs, potential for product failures, and revenue generation.
• Operational Risks [medium — operational]: Risks associated with manufacturing, supply chain, and clinical trial execution.
• Intellectual Property Risks [medium — legal]: Protection and enforcement of intellectual property rights are critical.

Key Financial Figures

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

Filing Documents

• rare-20231231.htm (10-K) — 3967KB
• rare-ex10_18.htm (EX-10.18) — 280KB
• rare-ex10_30.htm (EX-10.30) — 47KB
• rare-ex10_31.htm (EX-10.31) — 43KB
• rare-ex10_32.htm (EX-10.32) — 34KB
• rare-ex10_33.htm (EX-10.33) — 41KB
• rare-ex10_34.htm (EX-10.34) — 26KB
• rare-ex10_35.htm (EX-10.35) — 41KB
• rare-ex10_36.htm (EX-10.36) — 27KB
• rare-ex21_1.htm (EX-21.1) — 20KB
• rare-ex23_1.htm (EX-23.1) — 6KB
• rare-ex31_1.htm (EX-31.1) — 15KB
• rare-ex31_2.htm (EX-31.2) — 14KB
• rare-ex32_1.htm (EX-32.1) — 11KB
• rare-ex97_1.htm (EX-97.1) — 23KB
• img215954057_0.jpg (GRAPHIC) — 93KB
• img215954057_1.jpg (GRAPHIC) — 43KB
• 0000950170-24-017879.txt (&nbsp;) — 16996KB
• rare-20231231.xsd (EX-101.SCH) — 2157KB
• rare-20231231_htm.xml (XML) — 3211KB

Business

Business 4 Item 1A.

Risk Factors

Risk Factors 30 Item 1B. Unresolved Staff Comments 66 Item 1C. Cybersecurity 66 Item 2.

Properties

Properties 66 Item 3.

Legal Proceedings

Legal Proceedings 67 Item 4. Mine Safety Disclosures 67 PART II Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities 68 Item 6. Reserved 68 Item 7.

Management's Discussion and Analysis of Financial Condition and Results of Operations

Management's Discussion and Analysis of Financial Condition and Results of Operations 69 Item 7A.

Quantitative and Qualitative Disclosures About Market Risk

Quantitative and Qualitative Disclosures About Market Risk 79 Item 8.

Financial Statements and Supplementary Data

Financial Statements and Supplementary Data 80 Item 9. Changes in and Disagreements With Accountants on Accounting and Financial Disclosure 80 Item 9A.

Controls and Procedures

Controls and Procedures 80 Item 9B. Other Information 81 Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections 81 PART III Item 10. Directors, Executive Officers and Corporate Governance 82 Item 11.

Executive Compensation

Executive Compensation 82 Item 12.

Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters

Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters 82 Item 13. Certain Relationships and Related Transactions, and Director Independence 82 Item 14. Principal Accountant Fees and Services 82 PART IV Item 15. Exhibits and Financial Statement Schedules 83 Item 16. Form 10-K Summary 88

SIGNATURES

SIGNATURES 89 CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This Annual Report on Form 10-K, or Annual Report, contains forward-looking statements that involve risks and uncertainties. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical fact contained in this Annual Report are forward-looking statements. In some cases, you can identify forward-looking statements by words such as "anticipate," "believe," "contemplate," "continue," "could," "estimate," "expect," "forecast," "intend," "may," "plan," "potential," "predict," "project," "seek," "should," "target," "will," "would," or the negative of these words, or other comparable terminology. These forward-looking statements include, but are not limited to, statements about: our commercialization, marketing, and manufacturing capabilities and strategy; our expectations regarding the timing of clinical study commencements and reporting results from same; the timing and likelihood of regulatory approvals for our product candidates; the anticipated indications for our product candidates, if approved; the potential market opportunities for commercializing our products and product candidates; our expectations regarding the potential market size and the size of the patient populations for our products and product candidates, if approved for commercial use; estimates of our expenses, revenue, capital requirements, and our needs for additional financing; our ability to develop, acquire, and advance product candidates into, and successfully complete, clinical studies; the implementation of our business model and strategic plans for our business, products and product candidates and the integration and performance of any businesses we have acquired or may acquire; the initiation, timing, progress, and results of ongoing and future p

Bus iness

Item 1. Bus iness Overview We are a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. We have 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. We were founded in April 2010 by our President and Chief Executive Officer, Emil Kakkis, M.D., Ph.D., and are led by a management team experienced in the development and commercialization of rare disease therapeutics. Our 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. Our Strategy The critical components of our business strategy include the following: Focus on rare and ultrarare genetic diseases with significant unmet medical need and clear biology. There are numerous rare and ultrarare genetic diseases that currently have no drug therapy approved that treat the underlying disease. Patients suffering from these diseases often have a significant morbidity and/or mortality. We focus on developing and commercializing therapies for multiple such indications with the utmost urgency. We also focus on diseases that have biology that is well understood. We believe that developing drugs that directly impact known disease pathways will increase the probability of success of our development programs. Our modalities of biologics, small molecules, adeno-associated virus, or AAV, gene therapy, and nucleic acids provide us with what we believe is an optimal set of options to treat genetic diseases by selecting the best treatment strategy available for each disease. In-license promising product candidates; retain global commercialization rights to product candidates. Our current product candidates are generally in-licensed from

View Full Filing

View this 10-K filing on SEC EDGAR