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Ultragenyx Pharmaceutical Inc.
NASDAQ: RARE Healthcare Pharma 🔎 Screen
$1.4B
Market Cap
P/E
PEG
-128.3%
ROCE
N/M
ROE
-17.15
D/E
-79.5%
OPM
-60.9%
% from 52W High
12
α RS
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About

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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⭐ Superinvestors Holding RARE
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Manager Shares Value % of Fund Period
Steve Cohen Point72 Asset Management 893.3K $18.7M 0.02% Mar 2026
Jim Simons Renaissance Technologies LLC 561.7K $11.8M 0.02% Mar 2026

SEC Form 13F data. 45-day lag from quarter end.

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🎙 Management Tone Confident Specific ↑ Improving 4 quarters Full tone analysis in Intelligence →
📊 MIXED Ultragenyx guides $730M-$760M revenue for 2026, PDUFAs in Aug/Sep, Angelman phase III readout in H2.
Revenue & Profitability
First quarter 2026 total revenue was $136 million. CRYSVITA contributed $93 million, DOJOLVI $18 million, EVKEEZA $18 million, and MEPSEVII $7 million. The net loss for the quarter was $185 million, or $1.84 per share. Total operating expenses were $305 million, including $30 million in stock-based compensation and $30 million in restructuring costs. The company had $534 million in cash and marketable securities as of March 31, 2026. Full-year 2026 revenue guidance was reaffirmed at $730-$760 million, representing 8%-13% growth over 2025.
Outlook
Management expressed confidence in continued double-digit revenue growth from the base business, driven by strong underlying demand for rare disease therapies. They noted that the rare disease market requires specialized patient-finding and reimbursement navigation, and they expect the upcoming launches of DTX401 and UX111 to contribute to growth. No specific macro headwinds were discussed.
Growth Drivers
Key growth levers include: CRYSVITA's steady demand across regions (950+ patients in Latin America), DOJOLVI's expansion (more than 675 patients in North America, launch in Japan pending), EVKEEZA's 64% year-over-year revenue growth to $18 million, and the planned launches of DTX401 (PDUFA August 23, 2026) and UX111 (PDUFA September 19, 2026). The Angelman syndrome program (GTX-102) represents a major upside opportunity if phase III data are positive. The company also expects to monetize two Priority Review Vouchers (PRVs) associated with the gene therapies.
Balance Sheet & CapEx
Not discussed in detail in this earnings call. The company mentioned $38 million in payments related to UX143 manufacturing activities in Q1 2026, indicating ongoing investment in manufacturing inventory for gene therapies. No specific CapEx guidance was provided.
Margins
Management expects 2026 combined R&D and SG&A expenses to be flat to down low single digits versus 2025, and a decrease of at least 15% in 2027 versus 2025. No explicit gross margin or operating margin figures were provided. The company is on a path to profitability in 2027, implying improving operating leverage.
Key Risks
Key risks flagged include: the phase I/II results for GTX-102 may not be predictive of phase III outcomes; variability in CRYSVITA ordering patterns in Brazil could cause quarterly fluctuations; discontinuations in the Angelman long-term extension study were due to patient burden; and the company may not achieve all regulatory approvals or commercial launch success. Analysts also raised concerns about baseline imbalances in the Aspire study and the impact of caregiver input on cognitive scores.
Generated by AI · Q1 2026 results · Not investment advice
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✓ 📞 Earnings Call Transcripts (5 quarters) submit a missing quarter
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📞 Earnings Call Transcripts (5)
Q2 2026 Q2 2026 2026-08-04
Record Q2 revenue of $214M was driven by strong global demand for key products and robust commercial execution. Two gene therapy BLAs are under FDA review with launches anticipated in late 2026, and profitability is targeted for 2027, supported by disciplined expense management and PRV monetization.
Q1 2026 Q1 2026 2026-05-05
Q1 2026 revenue reached $136M, with strong growth in EVKEEZA and steady performance in CRYSVITA and DOJOLVI. 2026 revenue guidance is $730–$760M, with profitability targeted for 2027 and multiple new gene therapy launches anticipated.
Q4 2025 Q4 2025 2026-02-12
Delivered 20% revenue growth in 2025, surpassing guidance, with strong commercial execution and expansion across four products. Strategic restructuring aims for profitability by 2027, supported by cost reductions, new launches, and PRV monetization. Regulatory and market risks remain.
Q3 2025 Q3 2025 2025-11-04
Q3 2025 revenue grew 15% year-over-year to $160M, with strong performance across all commercial products and a $400M royalty sale bolstering liquidity. Pivotal data readouts for key late-stage programs are on track, and 2025 revenue guidance of $640M–$670M is reaffirmed.
Q2 2025 Q2 2025 2025-08-05
Q2 2025 revenue grew 13% year-over-year to $166M, with strong performance from CRYSVITA and DULJOVI. Pipeline advanced with key milestones: GTX-102 received FDA breakthrough therapy designation and completed Phase III enrollment, while UX143 and DTX-401 remain on track for major data and regulatory events.
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Financial Model
Projections are built from each company's audited annual financials (Income Statement, Balance Sheet, Cash Flow) over the last 5 fiscal years. Forward assumptions — revenue growth %, EBITDA margin, D&A (USD millions), interest expense, tax rate, and capex — are AI-generated using historical context and refreshed twice a year: after the December results season and after the September/Q4 results season.

DCF Valuation
Fair Value = Σ(FCFt / (1+WACC)t) + Terminal Value. Terminal Value uses the Gordon Growth Model: FCF5 × (1+g) / (WACC−g). Default WACC: 10% (US risk-free ~4.5%, equity risk premium ~5.5%). Default terminal growth: 3% (long-run US nominal GDP proxy).

CAGR Tracker
Expected 5-year CAGR = (DCF Fair Value / Current Price)1/5 − 1. Assumes fair value is reached in exactly 5 years — a mechanical estimate only.

Data Sources & Limitations
Financial statements sourced from public filings. Prices updated daily. Forward assumptions are AI-generated. All monetary values in USD millions. Non-US ADR companies may have currency conversion inaccuracies. Models are point-in-time and do not update intra-quarter or account for M&A, macro shocks, or extraordinary items.

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Investment Risk:
Investing in securities, including US equities and ETFs, involves inherent risks including the potential loss of principal. All investments are subject to market fluctuations, economic conditions, regulatory changes, and other factors that may affect their value. Past performance is not indicative of future results. This analysis is provided for informational and educational purposes only and should not be construed as investment advice under any circumstances.

No Investment Recommendation:
This analysis does not constitute, nor should it be interpreted as, an offer, solicitation, or recommendation to buy, sell, or hold any securities or financial products. Investors are strongly advised to conduct their own independent research and due diligence and to consult with a licensed financial advisor or an SEC-registered investment adviser before making any investment decisions, taking into account their individual financial situation, risk tolerance, and investment objectives.

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Information Sources:
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