Ultragenyx Pharmaceutical Inc. (RARE) | Investment Case

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.

Show Full description

1.b. Last Insights on RARE

Breaking News: Ultragenyx Pharmaceutical Inc. has been presenting at recent conferences, including the Evercore 8th Annual Healthcare Conference and the Citi Annual Global Healthcare Conference 2025. The company has not released a recent earnings report in the provided data. Analyst recommendations are not available in the given information. Management's presentations may provide insights into the company's strategy and outlook. Investors may look for updates on the company's product pipeline and development progress. Further information on Ultragenyx Pharmaceutical Inc. may be found through their official reports and public statements.

1.c. Company Highlights

Last Price	34.34
Change Percentage	-5.22%
Open	36.05
Previous Close	36.23
Market Cap ( Millions)	3307
Volume	1438461
Year High	46.5
Year Low	25.81
M A 50	33.51
M A 200	33.72

FCF Yield	-13.65%
Dividend Yield	0.00%
ROE	-414.17%
Debt / Equity	132.57%
Net Debt / EBIDTA	38.69%
Price To Book	374.07
Price Earnings Ratio	-5.91
Price To FCF	-7.33
Price To sales	5.24
EV / EBITDA	-6.33

2. Ultragenyx's Q3 2025 Earnings: A Closer Look

The company's financial performance for the third quarter of 2025 was marked by significant revenue growth, with total revenue projected to be between $640 million and $670 million, representing a 14% to 20% increase from the previous year. The actual EPS came out at -$1.81, missing estimates of -$1.23. The revenue growth is a testament to the company's successful commercial product launches and the strength of its global commercial organization. According to Erik Harris, the commercial team generated 50 new start forms, leading to approximately 50 more patients on reimbursed therapy, bringing the total to around 875 patients on commercial product.

Publication Date: Nov -08

                                    📋 Highlights
                                    $400M Non-Dilutive Financing:
                                                Secured $400 million in royalty financing from OMERS, deferring payments until January 2028.
Revenue Growth Projection:
                                                Forecast 14-20% annual revenue growth for existing products, targeting $640M–$670M in 2025.
GTX-102 Clinical Milestone:
                                                129 patients enrolled in the pivotal Aspire study for Angelman syndrome, with Phase II/III Aurora trial initiated.
UX111 & DTX401 Regulatory Updates:
                                                Adjusted FDA submission timelines: UX401 first, then UX111; ongoing longer-term data review for MPS IIIA.

                                

Financial Strength and Capital Structure

The company bolstered its financial position through a $400 million non-dilutive capital raise from OMERS via the sale of a portion of its Crysvita royalties. This financing will not require payments until January 2028 and is capped, providing Ultragenyx with a strengthened balance sheet to support its multiple product launches and growth initiatives. With a current 'P/S Ratio' of 4.68 and an 'EV/EBITDA' of -5.61, the market seems to be pricing in a mix of growth and risk.

Pipeline Progress and Catalysts

Ultragenyx is making significant progress across its late-stage pipeline programs. The GTX-102 program for Angelman syndrome is advancing, with the pivotal Aspire study fully enrolled and expected to read out data in the second half of 2026. The company is also making headway with UX111 for MPS IIIA and DTX401 for Glycogen Storage Disease Type Ia, having had constructive interactions with the FDA. For setrusumab in osteogenesis imperfecta, the company is on track to share top-line data from the Orbit and Cosmic studies in December or January, which could be a significant catalyst for the stock.

Growth Prospects and Valuation

Analysts estimate next year's revenue growth at 21.7%, indicating a strong trajectory for the company. The current 'P/E Ratio' of -5.27 and 'ROE (%)' of -414.17 highlight the challenges in valuing a company with negative earnings, but the growth prospects and pipeline advancements are positives. The 'P/B Ratio' of 333.66 is extremely high, suggesting that the market is pricing in significant future growth or that the book value does not accurately reflect the company's intangible assets and future potential.

Operational Highlights and Future Directions

3. NewsRoom

Fisher Asset Management LLC Has $6.75 Million Stock Position in Ultragenyx Pharmaceutical Inc. $RARE

Dec -05

Source: defenseworld.net

Ultragenyx Pharmaceutical Inc. (RARE) Presents at Evercore 8th Annual Healthcare Conference Transcript

Dec -03

Source: seekingalpha.com

Ultragenyx Pharmaceutical Inc. (RARE) Presents at Citi Annual Global Healthcare Conference 2025 Transcript

Dec -03

Source: seekingalpha.com

CENTURY LITHIUM ACHIEVES HIGH RECOVERY OF RARE EARTH AND CRITICAL ELEMENTS FROM PRIMARY LEACH SOLUTIONS

Dec -02

Source: prnewswire.com

Campbell & CO Investment Adviser LLC Makes New $309,000 Investment in Ultragenyx Pharmaceutical Inc. $RARE

Nov -18

Source: defenseworld.net

Ultragenyx Q3 Earnings and Sales Miss Estimates, Increase Y/Y

Nov -05

Source: zacks.com

Ultragenyx Pharmaceutical Inc. (RARE) Q3 2025 Earnings Call Transcript

Nov -05

Source: seekingalpha.com

Here's What Key Metrics Tell Us About Ultragenyx (RARE) Q3 Earnings

Nov -04

Source: zacks.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

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

Strong Liquidity

Low Deb

Strong Interest Coverage

Healthy Cash Flow

Operating Efficiency

Strong Return On Equity

Consistent Revenue Growth

Consistent Operating Cash Flow Growth

Margin Improvement

12.a Historical Valuation

12.b Price/Earnings Ratio

12.c Margin Valuation

12.d Peers Valuation

Peers Group Analysis

ACADIA Pharmaceuticals

A-Score: 5.6/10

Value: 3.0

Growth: 9.0

Quality: 9.1

Yield: 0.0

Momentum: 9.0

Volatility: 3.3

1-Year Total Return ->

TG Therapeutics

A-Score: 4.8/10

Value: 2.0

Growth: 8.2

Quality: 7.7

Yield: 0.0

Momentum: 8.5

Volatility: 2.7

1-Year Total Return ->

Axsome Therapeutics

A-Score: 4.6/10

Value: 6.0

Growth: 4.1

Quality: 3.6

Yield: 0.0

Momentum: 9.0

Volatility: 5.0

1-Year Total Return ->

Arrowhead Pharmaceuticals

A-Score: 3.9/10

Value: 6.2

Growth: 1.1

Quality: 4.2

Yield: 0.0

Momentum: 10.0

Volatility: 2.0

1-Year Total Return ->

Intellia Therapeutics

A-Score: 3.4/10

Value: 7.0

Growth: 2.8

Quality: 4.8

Yield: 0.0

Momentum: 4.5

Volatility: 1.0

1-Year Total Return ->

Ultragenyx Pharmaceutical

A-Score: 3.0/10

Value: 6.4

Growth: 4.7

Quality: 3.5

Yield: 0.0

Momentum: 0.0

Volatility: 3.7

1-Year Total Return ->

Peers Metrics

12.e Scoring ^Insights

12.f DCF ^BETA

Parameters

Short Term Growth

19.19%

Short term Time

10.0

Long-Term Growth

2.00%

WACC

7.31%

Ultragenyx Pharmaceutical Inc. - US90400D1081

1. Company Snapshot

1.a. Company Description

1.b. Last Insights on RARE

1.c. Company Highlights

Average Growth

Medium Volatility

Fairly Valued

Earnings Miss

Revenues Miss

Average Quality

Stable Momentum

Average Yield

Earnings Beats

Revenues Beats

Strong Momentum

Weak Momentum

2. Ultragenyx's Q3 2025 Earnings: A Closer Look

Financial Strength and Capital Structure

Pipeline Progress and Catalysts

Growth Prospects and Valuation

Operational Highlights and Future Directions

3. NewsRoom

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

Expected Growth: 10.43%

Dojolvi

Expected Growth: 9.27%

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

Expected Growth: 10.27%

Mepsevii

Expected Growth: 9.27%

Evkeeza

Expected Growth: 9.27%

Collaboration and License - Daiichi Sankyo

Expected Growth: 10.73%

7. Detailed Products

Crysvita (burosumab)

Mepsevii (vestronidase alfa)

Dojolvi (triheptanoin)

UX007 (triheptanoin)

8. Ultragenyx Pharmaceutical Inc.'s Porter Forces

Forces Ranking

Threat Of Substitutes

Bargaining Power Of Customers

Bargaining Power Of Suppliers

Threat Of New Entrants

Intensity Of Rivalry

9. SWOT Analysis

Strength

Weaknesses

Opportunities

Threats

10. Capital Structure

10.a. Balance Sheet

10.b. Weighted Average Cost of capital

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

Peers Metrics

12.e Scoring Insights

12.f DCF BETA

Parameters

Short Term Growth

Short term Time

Long-Term Growth

WACC

Target Price

34.34$

Current Price

34.34$

12.e Scoring ^Insights

12.f DCF ^BETA