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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, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. 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 the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; 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; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701, an adeno-associated AAV9 gene therapy which is in Phase 2 clinical trial for the treatment of Wilson liver disease. The company has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Regeneron; and Abeona. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
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A unique technical analysis driven score developed by Kalkine showing momentum in stock prices based on select technical indicators identified.
Ultragenyx Pharmaceutical develops and commercial therapies for rare genetic and metabolic diseases, with a focus on biologics and gene therapies for orphan indications. Long-term growth may be supported by expanding regulatory approvals and late-stage pipeline assets targeting high unmet needs and limited competition that can support premium pricing. The company appears positioned to convert clinical progress into durable revenue streams given its specialized focus and commercial experience in rare-disease markets.
Material risks include clinical and regulatory setbacks, dependency on a small number of products, pricing and reimbursement pressures, and manufacturing or commercialization execution challenges.
Note: The summary and risk score provided above is created by AI and should not be construed as a recommendation of Kalkine.
| Breakdown | 2025-12-31 | 2024-12-31 | 2023-12-31 | 2022-12-31 | 2021-12-31 |
|---|---|---|---|---|---|
| Type | yearly | yearly | yearly | yearly | yearly |
| Date | 2025-12-31 | 2024-12-31 | 2023-12-31 | 2022-12-31 | 2021-12-31 |
| Income before tax | -571.00M | -567.59M | -608.46M | -701.73M | -452.98M |
| Net income | -575.00M | -569.18M | -606.64M | -707.42M | -454.02M |
| Selling general administrative | 349.00M | 321.61M | 309.80M | 278.14M | 219.98M |
| Gross profit | 564.00M | 483.50M | 389.04M | 335.01M | 335.40M |
| Reconciled depreciation | 35.00M | 35.54M | 26.01M | 18.22M | 13.24M |
| Ebit | -509.00M | -504.55M | -542.46M | -648.92M | -381.74M |
| Ebitda | -474.00M | -469.00M | -516.45M | -639.41M | -381.50M |
| Depreciation and amortization | 35.00M | 35.54M | 26.01M | 9.51M | 0.24M |
| Non operating income net other | - | - | - | 9.51M | 0.24M |
| Operating income | -535.00M | -535.97M | -569.21M | -648.92M | -381.74M |
| Other operating expenses | 1208.00M | 1096.20M | 1003.46M | 1012.25M | 733.14M |
| Interest expense | 62.00M | 63.04M | 66.00M | 43.02M | 29.42M |
| Tax provision | 4.00M | 1.60M | -1.82M | 5.70M | 1.04M |
| Interest income | 25.00M | 36.51M | 26.69M | 11.07M | 1.93M |
| Net interest income | -37.00M | -26.54M | -39.32M | -31.94M | -29.42M |
| Income tax expense | 4.00M | 1.60M | -1.82M | 5.70M | 1.04M |
| Total revenue | 673.00M | 560.23M | 434.25M | 363.33M | 351.41M |
| Total operating expenses | 1099.00M | 1019.48M | 958.25M | 983.93M | 717.13M |
| Cost of revenue | 109.00M | 76.73M | 45.21M | 28.32M | 16.01M |
| Total other income expense net | -36.00M | -31.61M | -39.26M | -52.81M | -71.24M |
| Net income from continuing ops | -575.00M | -569.18M | -606.64M | -707.42M | -454.02M |
| Net income applicable to common shares | - | - | - | -707.42M | -454.02M |
| Breakdown | 2025-12-31 | 2024-12-31 | 2023-12-31 | 2022-12-31 | 2021-12-31 |
|---|---|---|---|---|---|
| Type | yearly | yearly | yearly | yearly | yearly |
| Date | 2025-12-31 | 2024-12-31 | 2023-12-31 | 2022-12-31 | 2021-12-31 |
| Total assets | 1532.00M | 1503.46M | 1491.01M | 1545.44M | 1522.40M |
| Intangible assets | 176.00M | 178.31M | 166.27M | 160.10M | 130.79M |
| Other current assets | 47.00M | 40.29M | -777.11M | 68.93M | -187.19M |
| Total liab | 1605.00M | 1241.16M | 1215.60M | 1192.95M | 599.84M |
| Total stockholder equity | -80.00M | 255.30M | 275.41M | 352.49M | 922.56M |
| Deferred long term liab | - | - | - | 31.67M | 33.31M |
| Other current liab | 272.00M | 295.10M | 29.24M | 204.68M | 145.56M |
| Common stock | - | 0.09M | 0.08M | 0.07M | 0.07M |
| Capital stock | 0.00M | 0.09M | 0.08M | 0.07M | 0.07M |
| Retained earnings | -4532.00M | -3956.84M | -3387.66M | -2781.02M | -2073.60M |
| Other liab | - | - | - | 911.93M | 387.56M |
| Good will | 44.00M | 44.41M | 44.41M | 44.41M | 44.41M |
| Other assets | - | - | - | 16.85M | 20.56M |
| Cash | 434.00M | 173.73M | 213.58M | 132.94M | 307.58M |
| Total current liabilities | 384.00M | 344.15M | 280.44M | 261.21M | 181.37M |
| Current deferred revenue | - | - | 196.49M | 1.48M | 7.61M |
| Net debt | 842.00M | -133.39M | -170.41M | -101.35M | -265.61M |
| Short term debt | 81.00M | 10.30M | 12.60M | 11.78M | 11.07M |
| Short long term debt total | 1276.00M | 40.34M | 43.17M | 31.59M | 41.97M |
| Other stockholder equity | 4451.00M | 4212.69M | 3662.35M | 3140.02M | 2997.50M |
| Property plant equipment | - | - | - | 259.73M | 141.25M |
| Total current assets | 951.00M | 817.12M | 732.18M | 883.90M | 856.60M |
| Long term investments | 57.00M | 135.00M | 199.90M | 154.50M | 258.93M |
| Net tangible assets | - | - | - | 147.98M | 758.40M |
| Short term investments | 259.00M | 436.30M | 363.62M | 614.82M | 432.61M |
| Net receivables | 159.00M | 121.80M | 73.39M | 40.45M | 28.43M |
| Inventory | 52.00M | 45.01M | 33.97M | 26.77M | 16.23M |
| Accounts payable | 31.00M | 38.76M | 42.11M | 43.27M | 17.14M |
| Accumulated other comprehensive income | 1.00M | -0.64M | 0.65M | -6.57M | -1.40M |
| Common stock total equity | - | - | - | 0.07M | 0.07M |
| Retained earnings total equity | - | - | - | -2781.02M | -2073.60M |
| Non currrent assets other | 35.00M | 62.68M | 51.73M | 16.85M | 20.56M |
| Non current assets total | 581.00M | 686.33M | 758.83M | 661.54M | 665.79M |
| Capital lease obligations | 36.00M | 40.34M | 43.17M | 31.59M | 41.97M |
| Breakdown | 2025-12-31 | 2024-12-31 | 2023-12-31 | 2022-12-31 | 2021-12-31 |
|---|---|---|---|---|---|
| Type | yearly | yearly | yearly | yearly | yearly |
| Date | 2025-12-31 | 2024-12-31 | 2023-12-31 | 2022-12-31 | 2021-12-31 |
| Investments | 258.00M | -17.77M | 103.42M | -291.65M | -201.18M |
| Change to liabilities | - | - | - | 78.21M | -25.18M |
| Total cashflows from investing activities | 236.00M | -17.77M | 168.00M | -291.65M | -195.37M |
| Total cash from financing activities | 478.00M | 399.24M | 388.14M | 501.21M | 118.55M |
| Change to operating activities | - | - | - | 3.80M | -29.51M |
| Net income | -575.00M | -569.18M | -606.64M | -707.42M | -454.02M |
| Change in cash | 253.47M | -35.24M | 81.80M | -171.98M | -416.71M |
| Begin period cash flow | 184.16M | 219.40M | 137.60M | 309.58M | 726.29M |
| End period cash flow | 436.00M | 184.16M | 219.40M | 137.60M | 309.58M |
| Total cash from operating activities | -466.00M | -414.19M | -474.81M | -380.46M | -338.69M |
| Issuance of capital stock | 86.00M | 380.98M | 53.30M | 0.00M | 78.94M |
| Depreciation | 35.00M | 35.54M | 26.01M | 18.22M | 13.24M |
| Other cashflows from investing activities | -16.00M | -14.94M | -7.55M | -95.78M | 78.90M |
| Change to inventory | -6.00M | -11.21M | -6.93M | -9.70M | -3.12M |
| Change to account receivables | -31.00M | -33.60M | -22.78M | -12.07M | -5.43M |
| Sale purchase of stock | - | - | - | 10.81M | 40.10M |
| Other cashflows from financing activities | 478.00M | 18.26M | 8.39M | 490.39M | 39.61M |
| Change to netincome | - | - | - | 74.80M | 0.23M |
| Capital expenditures | 6.00M | 7.49M | 46.77M | 146.12M | 73.09M |
| Change receivables | - | - | - | -12.07M | -5.43M |
| Cash flows other operating | - | - | - | 253.00M | 103.80M |
| Cash and cash equivalents changes | - | - | - | -170.91M | -415.51M |
| Change in working capital | -23.00M | 13.92M | -15.10M | 60.23M | -63.24M |
| Stock based compensation | 153.00M | 158.03M | 135.21M | 130.38M | 104.95M |
| Other non cash items | -56.00M | -52.50M | -12.67M | 119.76M | 11.71M |
| Free cash flow | -472.00M | -421.68M | -521.57M | -526.59M | -411.79M |
Sector: Healthcare Industry: Biotechnology
|
Stock Name
|
Change
(USD) |
Price
(USD) |
Trailing
PE (x) |
Forward
PE (x) |
Price Sales
TTM (x) |
Price to Book Value (x)
|
Enterprise Value
to Revenue (x) |
Enterprise Value
to EBITDA (x) |
|---|---|---|---|---|---|---|---|---|
| RARE Ultragenyx |
-0.42 2.44% |
16.78 | - | - | 1.80 | 19.68 | 0.97 | -6.46 |
| NVO Novo Nordisk A/S |
0.85 2.22% |
39.14 | 41.58 | 31.15 | 2.13 | 32.99 | 2.11 | 4.70 |
| VRTX Vertex Pharmaceuticals Inc |
10.31 1.95% |
540.00 | 30.22 | 24.27 | 10.36 | 6.50 | 9.99 | 24.66 |
| REGN Regeneron Pharmaceuticals Inc |
5.50 0.68% |
818.77 | 29.07 | 20.12 | 7.57 | 3.82 | 6.95 | 19.42 |
| ARGNF argenx SE |
- -% |
790.15 | 45.77 | 31.25 | 13.84 | 7.12 | 11.38 | 36.06 |
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, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. 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 the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; 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; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701, an adeno-associated AAV9 gene therapy which is in Phase 2 clinical trial for the treatment of Wilson liver disease. The company has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Regeneron; and Abeona. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
| Name | Title | Year Born |
|---|---|---|
| Dr. Emil D. Kakkis M.D., Ph.D. | Founder, Pres, CEO & Director | 1960 |
| Mr. John Richard Pinion II | Chief Quality Operations Officer & Exec. VP of Translational Sciences | 1966 |
| Dr. Camille L. Bedrosian M.D. | Chief Medical Officer & Exec. VP | 1953 |
| Mr. Erik Harris | Exec. VP & Chief Commercial Officer | 1970 |
| Mr. Theodore A. Huizenga | Sr. VP, Corp. Controller & Principal Accounting Officer | 1971 |
| Mr. Dennis Karl Huang | Chief Technical Operations Officer & Exec. VP | 1965 |
| Ms. Danielle Keatley | Sr. Director of Investor Relations & Corp. Communications | NA |
| Ms. Karah Herdman Parschauer J.D. | Chief Legal Officer & Exec. VP | 1978 |
| Mr. Ernie W. Meyer | Chief HR Officer & Exec. VP | 1964 |
| Mr. Thomas R. Kassberg | Chief Bus. Officer & Exec. VP | 1960 |
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