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1. Company Snapshot

1.a. Company Description

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Europe, and internationally.Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for treating long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia.The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III.


Ultragenyx Pharmaceutical Inc.has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer Healthcare LLC; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd.Ultragenyx Pharmaceutical Inc.


was incorporated in 2010 and is headquartered in Novato, California.

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1.b. Last Insights on RARE

Breaking News: Ultragenyx Pharmaceutical Inc. is facing a class action lawsuit, with several law firms announcing the opportunity for investors who suffered substantial losses to lead the lawsuit. The class period is between August 3, 2023, and December 26, 2025. Investors who purchased Ultragenyx common stock during this period have until April 6, 2026, to seek appointment as lead plaintiff. Law firms such as Robbins Geller Rudman & Dowd LLP, Pomerantz Law Firm, The Gross Law Firm, Faruqi & Faruqi, LLP, and ClaimsFiler are reminding investors of the pending lawsuit and lead plaintiff deadline.

1.c. Company Highlights

2. Ultragenyx's 2025 Financial Performance and 2026 Outlook

Ultragenyx reported total revenue of $673 million for 2025, representing a 20% year-over-year growth, driven by its established and growing commercial business. The company reported a net loss of $575 million or $5.83 per share. The actual EPS loss came out at -$1.29 relative to estimates at -$1.2. The company's cash, cash equivalents, and marketable securities stood at $738 million as of December 31, 2025. For 2026, Ultragenyx expects total revenue to be between $730 million and $760 million, representing 8% to 13% growth over 2025.

Publication Date: Feb -17

📋 Highlights
  • 2026 Key Inflection Points:: Potential approvals for MPS IIIA and GSD Ia, plus pivotal data readout in Angelman syndrome.
  • 2025 Financial Growth:: Total revenue reached $673M (20% YoY), despite a $575M net loss and $5.83/share loss.
  • 2026 Revenue Guidance:: Projected to grow 8–13% to $730–760M, with R&D/SG&A expenses flat to down low single digits.
  • Cash Reserves:: $738M in cash, cash equivalents, and marketable securities as of December 31, 2025.
  • 2027 Cost Reduction Plan:: R&D expenses expected to drop 38% ($280M decline) post-completion of Phase III studies.

Financial Highlights

The company's financial performance was highlighted by significant year-over-year revenue growth. According to Howard Horn, Chief Financial Officer, the company expects combined R&D and SG&A expenses to be flat to down low single digits versus 2025, indicating a controlled expense growth.

Valuation Metrics

To understand what's priced in, we can look at Ultragenyx's valuation metrics. The company's P/S Ratio stands at 2.94, while the EV/EBITDA ratio is -5.45. The ROE is -411.04%, and the ROIC is -57.72%. Analysts estimate next year's revenue growth at 34.8%, indicating a potential upside.

Pipeline and Regulatory Updates

Ultragenyx has a diversified pipeline with multiple programs in various stages. The company has resubmitted the UX111 BLA to FDA and is expecting a pivotal data readout in Angelman syndrome. Eric Crombez, Chief Medical Officer, highlighted the upcoming clinical and regulatory catalysts for 2026, including the DTX401 BLA submission and the GTX-102 Aspire Phase III data readout.

Pathway to Profitability

Emil Kakkis mentioned that the company expects continued growth from current products and contributions from upcoming launches on the revenue side. On the expense side, combined R&D and SG&A are expected to be flat to down low single digits in 2026 and down 15% or more in 2027, indicating a clear pathway to profitability.

Gene Therapy Investments

The company plans to continue its investment in gene therapy but does not plan to make it an exclusive focus. Ultragenyx has a diversified pipeline, including two gene therapies in the BLA stage, a Phase III gene therapy for ornithine transcarbamylase (OTC) deficiency, and a Phase II gene therapy for Wilson disease.

3. NewsRoom

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Ultragenyx Pharmaceutical Inc. Securities Class Action Filed; Lead Plaintiff Deadline April 6, 2026 – RGRD Law

14:31

Source: globenewswire.com

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Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)

13:30

Source: globenewswire.com

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Ultragenyx Pharmaceutical Inc. Sued for Securities Law Violations - Contact the DJS Law Group to Discuss Your Rights - RARE

09:08

Source: prnewswire.com

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RARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud Lawsuit with the Schall Law Firm

09:06

Source: prnewswire.com

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ROSEN, A TOP RANKED LAW FIRM, Encourages Ultragenyx Pharmaceutical Inc. Investors to Secure Counsel Before Important Deadline in Securities Class Action - RARE

02:36

Source: newsfilecorp.com

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ROSEN, A TOP RANKED LAW FIRM, Encourages Ultragenyx Pharmaceutical Inc. Investors to Secure Counsel Before Important Deadline in Securities Class Action - RARE

Feb -21

Source: newsfilecorp.com

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Ultragenyx Shareholder Alert: ClaimsFiler Reminds Investors With Losses In Excess Of $100,000 Of Lead Plaintiff Deadline In Class Action Lawsuit Against Ultragenyx Pharmaceutical Inc. - RARE

Feb -21

Source: prnewswire.com

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Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Feb -20

Source: globenewswire.com

4. Business Breakdown

4.a. Revenues by Country

4.b. Revenues by Segment

5. Expected revenues mid-term growth (10.12%)

6. Segments

Crysvita Royalty

Expected Growth: 10.43%

Crysvita Royalty's 10.43% growth is driven by increasing adoption in hypophosphatemia treatment, expanded geographic reach, and strong sales momentum. Additionally, Ultragenyx's strategic partnerships and investments in rare disease therapies contribute to the royalty's growth. Furthermore, the orphan drug designation and patent protection provide a competitive moat, ensuring sustained revenue growth.

Crysvita

Expected Growth: 10.43%

Crysvita's 10.43% growth is driven by increasing adoption in hypophosphatemia treatment, expansion into new markets, and strong demand from pediatric and adult patients. Additionally, Ultragenyx's strategic partnerships and investments in rare disease research contribute to the growth. The orphan drug designation and FDA approval for Crysvita also support its market expansion.

Dojolvi

Expected Growth: 9.27%

Dojolvi's 9.27% growth is driven by increasing adoption in long-chain fatty acid oxidation disorders treatment, expansion into new markets, and growing awareness of the disease among healthcare professionals. Additionally, Ultragenyx's strategic partnerships and investments in rare disease research contribute to the product's growth momentum.

Collaboration and License - Crysvita Collaboration Revenue in Profit-share Territory

Expected Growth: 10.27%

Strong demand for Crysvita in profit-share territory driven by increasing adoption in ultra-rare disease treatment, expanded patient access, and successful commercial execution by Ultragenyx. Additionally, growing awareness of hypophosphatemia treatment options and favorable reimbursement dynamics contribute to the 10.27% growth.

Mepsevii

Expected Growth: 9.27%

Mepsevii's 9.27% growth is driven by increasing adoption in the treatment of Sly Syndrome, a rare genetic disorder. Strong demand from patients and physicians, coupled with Ultragenyx's effective marketing strategies, have contributed to the growth. Additionally, the company's expansion into new markets and geographic regions has further boosted sales.

Evkeeza

Expected Growth: 9.27%

Evkeeza's 9.27% growth is driven by increasing adoption in the homozygous familial hypercholesterolemia (HoFH) market, expanded reimbursement coverage, and growing awareness of the treatment's benefits. Additionally, Ultragenyx's strategic partnerships and investments in rare disease research contribute to the growth.

Collaboration and License - Daiichi Sankyo

Expected Growth: 10.73%

Daiichi Sankyo's collaboration and license agreement with Ultragenyx Pharmaceutical Inc. drove 10.73% growth, fueled by joint development of novel therapies, expanded market access, and shared R&D costs. This partnership leverages Ultragenyx's expertise in rare genetic diseases and Daiichi Sankyo's global commercialization capabilities, accelerating innovation and revenue growth.

7. Detailed Products

Crysvita (burosumab)

A recombinant fully human monoclonal antibody that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23), a hormone that regulates phosphate metabolism.

Mepsevii (vestronidase alfa)

A recombinant human lysosomal enzyme that helps to break down and remove toxic substances that accumulate in the cells of patients with mucopolysaccharidosis VII (MPS VII).

Dojolvi (triheptanoin)

A synthetic triglyceride oil that provides a unique fatty acid profile to help manage long-chain fatty acid oxidation disorders.

UX007 (triheptanoin)

A synthetic triglyceride oil that provides a unique fatty acid profile to help manage long-chain fatty acid oxidation disorders.

8. Ultragenyx Pharmaceutical Inc.'s Porter Forces

Forces Ranking

Threat Of Substitutes

Ultragenyx Pharmaceutical Inc. operates in a niche market with limited substitutes, but the threat of substitutes is still present due to the ongoing research and development in the biotechnology industry.

Bargaining Power Of Customers

Ultragenyx Pharmaceutical Inc. has a diverse customer base, and no single customer has significant bargaining power, reducing the threat of customer bargaining power.

Bargaining Power Of Suppliers

Ultragenyx Pharmaceutical Inc. relies on a few key suppliers for raw materials and services, giving them some bargaining power, but the company's size and diversification mitigate this threat.

Threat Of New Entrants

The biotechnology industry is highly competitive, and new entrants can easily disrupt the market, posing a significant threat to Ultragenyx Pharmaceutical Inc.'s market share.

Intensity Of Rivalry

The biotechnology industry is highly competitive, with many established players and new entrants vying for market share, leading to a high intensity of rivalry.

9. SWOT Analysis

Strength

Weaknesses

Opportunities

Threats

10. Capital Structure

10.a. Balance Sheet

10.b. Weighted Average Cost of capital

Value
Debt Weight 13.55%
Debt Cost 7.31%
Equity Weight 86.45%
Equity Cost 7.31%
WACC 7.31%
Leverage 15.67%

11. Quality Control: Ultragenyx Pharmaceutical Inc. passed 2 out of 9 key points

12.a Historical Valuation

12.b Price/Earnings Ratio

12.c Margin Valuation

12.d Peers Valuation

Peers Group Analysis

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ACADIA Pharmaceuticals

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Value: 2.1

Growth: 9.0

Quality: 9.1

Yield: 0.0

Momentum: 9.0

Volatility: 4.3

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Arrowhead Pharmaceuticals

A-Score: 4.8/10

Value: 6.0

Growth: 8.6

Quality: 3.3

Yield: 0.0

Momentum: 9.0

Volatility: 2.0

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Axsome Therapeutics

A-Score: 4.8/10

Value: 6.0

Growth: 4.2

Quality: 3.8

Yield: 0.0

Momentum: 9.5

Volatility: 5.3

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TG Therapeutics

A-Score: 4.2/10

Value: 2.0

Growth: 8.1

Quality: 7.7

Yield: 0.0

Momentum: 4.5

Volatility: 3.0

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Ultragenyx Pharmaceutical

A-Score: 3.3/10

Value: 6.4

Growth: 4.7

Quality: 3.8

Yield: 0.0

Momentum: 1.0

Volatility: 3.7

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Intellia Therapeutics

A-Score: 3.0/10

Value: 7.3

Growth: 2.8

Quality: 4.6

Yield: 0.0

Momentum: 2.0

Volatility: 1.0

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Peers Metrics

12.e Scoring Insights

12.f DCF BETA

Parameters

Short Term Growth

Short term Time

Long-Term Growth

WACC

Target Price

22.88$

Current Price

22.88$

Potential

-0.00%

Expected Cash-Flows