Ultragenyx Pharmaceutical Inc.

Listed Company

RARECIK 0001515673NasdaqLarge AcceleratedPharmaceutical PreparationsDelawareFY ends Dec 31

Period

FY 2025

Revenue

$673.00M

Net Income

$-575.00M

Total Assets

$1.53B

Equity

$-80.00M

Shares Out

96.63M

Op. Cash Flow

$-466.00M

Business

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing and commercializing novel therapies for serious rare and ultrarare genetic diseases. It discovers, in‑licenses, develops and commercializes biologics, small molecules, AAV gene therapies and nucleic acid product candidates, and currently markets approved products including Crysvita, Mepsevii, Dojolvi and Evkeeza. Its operations encompass clinical development, regulatory strategy, commercial functions and manufacturing capabilities (including a gene therapy manufacturing facility) and extensive licensing and collaboration arrangements. The company commercializes products across North America, the EU/UK, Latin America, Turkey, Asia and select international markets through its own commercial teams, partners and third‑party distributors.

Summary from filing dated 2025-02-19

Financials

Consolidated Statement of Income

Year Ended · In millions, except per-share amounts

Concept	FY 2025 2025-12-31	FY 2024 2024-12-31	FY 2023 2023-12-31	FY 2022 2022-12-31	FY 2021 2021-12-31
Total revenues	673	560	434	363	351
Cost of sales	109	77	45	28	16
Research and development	750	698	648	706	497
Selling, general and administrative	349	321	310	278	220
Total operating expenses	1,208	1,096	1,003	1,012	733
Loss from operations	−535	−536	−569	−649	−382
Interest income	25	37	27	11	2
Non-cash interest expense on liabilities for sales of future royalties	−62	−63	−66	−43	−29
Other income (expense)	1	−5	−1	−2	−2
Loss before income taxes	−571	−567	−609	−702	−453
(Provision for) benefit from income taxes	−4	−2	2	−6	−1
Net loss	−575	−569	−607	−707	−454
Net loss per share, basic	−5.83	−6.29	−8.25	−10.12	−6.7
Net loss per share, diluted	−5.83	−6.29	−8.25	−10.12	−6.7
Shares used in computing net loss per share, Basic	99	91	74	70	68
Shares used in computing net loss per share, Diluted	99	91	74	70	68

Consolidated Balance Sheet

As of · In millions, except per-share amounts

Concept	FY 2025 2025-12-31	FY 2024 2024-12-31	FY 2023 2023-12-31	FY 2022 2022-12-31	FY 2021 2021-12-31
Cash and cash equivalents	421	174	214	133	308
Marketable securities	259	436	364	615	433
Accounts receivable, net	158	122	73	40	28
Inventory	52	45	34	27	16
Prepaid expenses and other assets	61	40	48	69	72
Total current assets	951	817	732	884	857
Property, plant, and equipment, net	244	266	291	260	141
Marketable securities	57	135	200	149	259
Intangible assets, net	176	178	166	160	131
Goodwill	44	44	44	44	44
Other assets	60	63	58	48	21
Total assets	1,532	1,503	1,491	1,545	1,522
Accounts payable	31	39	42	43	17
Accrued liabilities	265	241	196	205	146
Lease liabilities	12	10	13	—	—
Liabilities for sales of future royalties	69	50	29	0	—
Other liabilities	7	4	0	—	—
Total current liabilities	384	344	280	261	181
Lease liabilities	24	30	31	—	—
Deferred tax liabilities	30	30	30	32	33
Liabilities for sales of future royalties	1,147	820	862	875	352
Other liabilities	20	17	12	5	1
Total liabilities	1,605	1,241	1,216	1,193	600
Commitments and contingencies (Note 15)	—	—	—	—	—
Noncontrolling interest	7	7	0	—	—
Preferred stock, par value of $0.001 per share-25.0 shares authorized; nil outstanding in 2025 and in 2024	0	0	0	0	0
Common stock, par value of $0.001 per share-250.0 shares authorized; outstanding-96.6 in 2025 and 92.5 in 2024	0	0	82,000	70,000	69,000
Treasury stock, at cost, 0.2 shares in 2025 and 0.1 shares in 2024	−8	−4	−432,000	0	—
Deferred compensation obligation	8	4	432,000	0	—
Additional paid-in capital	4,451	4,213	3,662	3,140	2,997
Accumulated other comprehensive income (loss)	1	−1	647,000	−7	−1
Accumulated deficit	−4,532	−3,957	−3,388	−2,781	−2,074
Total stockholders' equity (deficit)	−80	255	275	352	923
Total liabilities, noncontrolling interest and stockholders' equity (deficit)	1,532	1,503	1,491	1,545	1,522

Consolidated Statement of Cash Flows

Year Ended · In millions, except per-share amounts

Concept	FY 2025 2025-12-31	FY 2024 2024-12-31	FY 2023 2023-12-31	FY 2022 2022-12-31	FY 2021 2021-12-31
Stock-based compensation	153	158	135	130	105
Amortization of discount on marketable securities, net	−4	−13	−13	3	7
Depreciation and amortization	35	36	26	18	13
Non-cash royalty revenue	−112	−101	−69	—	—
Other	−2	−2	3	−230,000	235,000
Accounts receivable	−31	−34	−23	−12	−5
Inventory	−6	−11	−7	−10	−3
Prepaid expenses and other assets	−10	12	15	4	—
Accounts payable, accrued, and other liabilities	24	47	−1	80	32
Net cash used in operating activities	−466	−414	−475	−380	−339
Purchase of property, plant, and equipment	−6	−7	−44	−116	−73
Purchase of marketable securities	−186	−409	−526	−615	−1,012
Proceeds from sale of marketable securities	0	3	51	84	93
Proceeds from maturities of marketable securities	444	410	696	451	718
Payments for intangible asset	−15	−13	−3	−30	0
Other	−1	−2	−6	−844,000	−942,000
Net cash provided by (used in) investing activities	236	−18	168	−292	−195
Proceeds from the sale of future royalties, net	392	0	0	491	0
Proceeds from the issuance of common stock and pre-funded warrants in connection with underwritten public offerings, net	0	381	326	0	0
Proceeds from the issuance of common stock in connection with at-the-market offering, net	80	0	53	0	79
Proceeds from the issuance of common stock under equity plans, net	6	11	8	11	40
Proceeds from issuance of equity from noncontrolling interest	0	7	0	0	—
Other	0	0	1	−555,000	−492,000
Net cash provided by financing activities	478	399	388	501	119
Effect of exchange rate changes on cash	4	−2	0	−1	−1
Net increase (decrease) in cash, cash equivalents, and restricted cash	252	−35	81	−172	−417
Acquired lease liabilities arising from obtaining right-of-use assets and property, plant, and equipment	6	10	22	1	3
Non-cash interest expense on liabilities for sales of future royalties capitalized during the year into ending property, plant and equipment	0	0	9	11	5
Costs of intangible assets included in accounts payable, accrued and other liabilities	5	15	10	18	19

Filings

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