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    Earnings call· Jun 2026(Q2 FY26)

    Ultragenyx Pharmaceutical Q2 FY26 earnings call RARE

    Aug 4, 2026 Source

    Executive summary

    Ultragenyx Q2 FY26 — Strong Commercial Performance and Key Pipeline Catalysts Ahead

    Ultragenyx delivered strong Q2 FY26 results, driven by robust commercial execution across its product portfolio, leading to record quarterly revenue. The company is actively preparing for the potential launches of two gene therapies, DTX401 and UX111, following their upcoming PDUFA dates, and anticipates top-line Phase III data for GTX-102 in Angelman syndrome. Despite a setback in the setrusumab program, management reaffirmed its full-year guidance and remains confident in its path to profitability by 2027, supported by continued growth and disciplined expense management.

    Highlights

    5

    • Achieved highest quarterly revenue in company history of $214 million.

    • Evkeeza revenue grew 50% over Q2 FY25 to $21 million.

    • Crysvita added 50 new patients in Latin America, bringing the regional total to 1,000 patients.

    • Dojolvi generated approximately 30 new start forms in North America, with ~675 patients on reimbursed therapy.

    • Reaffirmed full-year revenue and expense guidance, with a clear path to profitability in 2027.

    Concerns

    3

    • Phase III Orbit and Cosmic studies for setrusumab (UX143) did not hit statistical significance for primary endpoints of annualized fracture reductions.

    • The MHRA (UK) indicated a new randomized study may be needed for setrusumab approval.

    • Crysvita revenue experienced quarter-to-quarter variability due to ordering patterns, though underlying demand remains strong.

    Guidance & targets

    4
    CategoryTargetConfidence
    Full-year revenue
    Reaffirmed
    high materiality
    High
    Combined R&D and SG&A operating expenses
    Flat to down low single digits
    medium materiality
    High
    Combined R&D and SG&A operating expenses
    Decrease of at least 15%
    medium materiality
    High
    Profitability
    Achieve profitability
    high materiality
    High

    Segment performance

    4
    SegmentRevenueYoYQoQMargin
    Crysvita
    Strong performance in Latin America supported by patient growth and timing of regional orders. Underlying demand continues to grow steadily despite quarter-to-quarter variability.
    North America Revenue: $94 millionLatin America and Türkiye Revenue: $54 millionEurope Revenue: $8 millionNew patients on commercial therapy (Latin America): 50Total patients on commercial therapy (Latin America): 1,000Patients treated (Türkiye Named Patient Program): >100
    		$156 million———
    Dojolvi
    Continued trend of steady growth. Notable growth in MENA region and strong start in Japan following National Health Insurance Drug Price List listing.
    New start forms (North America): 30Patients on reimbursed therapy (North America): ~675Patients treated (Europe via early access/named patient programs): ~300Marketing authorization approval: Kuwait
    		$27 million———
    Evkeeza
    Important and growing contributor to revenue base, with demand building following launches in territories outside the United States.
    Patients treated (outside US): >500Countries with patients treated: 25
    		$21 million50%——
    Mepsevii
    Continues to treat patients in this ultra-rare indication.
    		$10 million———

    Operational metrics

    12
    Total revenue
    $214 million
    Q2 FY26

    Highest quarterly revenue in company history.

    Total operating expenses
    $289 million
    Q2 FY26
    Cost of sales
    $34 million
    Q2 FY26

    Included in total operating expenses.

    Combined R&D and SG&A expenses
    $255 million
    Q2 FY26

    Included in total operating expenses.

    Noncash stock-based compensation
    $34 million
    Q2 FY26

    Included in total operating expenses.

    Net loss
    $92 million
    Q2 FY26
    Net loss per share
    $0.90
    Q2 FY26
    Cash, cash equivalents, and marketable securities
    $436 million
    as of June 30, 2026
    Net cash used in operations
    $97 millionsignificant decrease from Q1 FY26
    Q2 FY26

    Consistent with expectations.

    GSDIa addressable patient population
    ~6,000
    current
    Sanfilippo syndrome addressable patient population
    ~3,000-5,000
    current
    Sanfilippo syndrome incidence rate
    1 in 100,000
    annual

    Approximately 30 to 40 new kids diagnosed per year.

    Industry KPIs

    3
    MetricValueDetails
    Pipeline read out calendarMultiple readouts
    Regulatory approvals filings2 PDUFA dates
    Clinical trial efficacy safety dataBayley-4 cognitive raw score; Multi-Domain Responder Index (MDRI)

    Risks & headwinds

    2
    Setrusumab (UX143) Phase III studies missed primary endpointPast (results disclosed)

    Neither the Orbit nor Cosmic study hit statistical significance for the primary endpoints of annualized fracture reductions.

    Mitigation: Engaging in discussions with regulatory agencies (FDA, MHRA) to define what additional clinical data would be needed to support a potential BLA, including the possibility of a new randomized study in the UK.

    Quarter-to-quarter revenue variability for CrysvitaOngoing

    Ordering patterns will continue to create some quarter-to-quarter variability.

    Mitigation: Confidence that the underlying demand for Crysvita will continue to grow steadily.

    What to watch in Q3 FY26

    5

    GTX-102 (Angelman) Phase III Aspire data readout

    September or October 2026
    CurrentDatabase cleaning and locking in process
    TargetTop-line results

    Why it matters

    This is a major catalyst for the company and the Angelman community, potentially leading to the first late-stage clinical data in this disease.

    For a global study like this, the process can take months, and we expect to unblind and share top-line results with you in the September or October timeframe.

    Q&A highlights

    5

    Clarification on FDA sign-off for single endpoint sufficiency and whether endpoints are powered for clinically meaningful results.

    Emil Kakkis confirmed FDA agreement on alpha sharing for the co-primary endpoints (Bayley-4 cognitive and MDRI), meaning either can be positive for success. He stated a 5-6 point change in Bayley is clinically meaningful, and MDRI inherently scores clinically meaningful results due to its design thresholds.

    “We had our discussion with the FDA on the sharing of primary alpha, and that was agreed to in our end of Phase II discussions and follow up discussions on the 2 endpoints. So sharing of alpha means that either endpoint can be positive in their analysis.”

    asked by Kristen Kluska · answered by Emil Kakkis

    2 min read5 chapters

    Detailed Narrative

    01

    Commercial Performance and Global Infrastructure Strength

    Ultragenyx reported its highest quarterly revenue of $214 million in Q2 FY26, driven by strong commercial execution across its product portfolio. Crysvita contributed $156 million, with notable patient growth in Latin America and Türkiye. Dojolvi added $27 million, with ~675 patients on reimbursed therapy in North America. Evkeeza showed significant growth, up 50% year-over-year to $21 million, now treating over 500 patients across 25 countries outside the US. The company emphasized its global commercial infrastructure's scale and expertise in rare disease commercialization, which supports continued product growth and prepares for future launches.

    02

    Gene Therapy Launch Preparations and Market Access

    The company is actively preparing for the potential launches of two gene therapies, DTX401 for GSDIa and UX111 for Sanfilippo syndrome type A, with PDUFA dates in August and September 2026, respectively. Preparations include working with Qualified Treatment Centers (QTCs) and engaging with payers. Management noted strong engagement from QTCs and positive recognition from payers regarding the significant unmet need and urgency to treat for both diseases, aiming to minimize barriers for eligible patients post-approval.

    03

    Angelman Syndrome (GTX-102) Phase III Update

    The Phase III Aspire study for GTX-102 in Angelman syndrome has completed its 48-week patient visits, and the database is currently undergoing cleaning and locking. Top-line results are anticipated in September or October 2026. The study's primary statistical alpha is split between the Bayley-4 cognitive raw score (80%) and the Multi-Domain Responder Index (MDRI) (20%), with either endpoint achieving statistical significance leading to a successful study. Management expressed confidence in the study design and the clinical meaningfulness of the endpoints.

    04

    Osteogenesis Imperfecta (UX143) Regulatory Discussions

    Results from the Phase III Orbit and Cosmic studies for UX143 (setrusumab) in osteogenesis imperfecta did not meet the primary endpoints of annualized fracture reductions. However, the studies showed clear signals of biologic activity, statistically meaningful improvements in bone mineral density, and reductions in fractures in certain bones and patient populations. Discussions with regulatory agencies are ongoing; the MHRA (UK) may require a new randomized study, while the FDA indicated openness to alternative approaches to fracture analysis but requires further definition of additional clinical data needed for a BLA.

    05

    Path to Profitability and Capital Allocation

    Ultragenyx reiterated its clear path to profitability in 2027, driven by three primary factors: continued double-digit revenue growth from current products and contributions from potential new launches, disciplined expense management (with combined R&D and SG&A expenses expected to decrease by at least 15% in FY27), and incremental non-dilutive capital from the monetization of Priority Review Vouchers (PRVs) associated with DTX401 and UX111, if approved. The company's cash balance stood at $436 million as of June 30, 2026.

    AI-generated summary of the company’s earnings call. Not investment advice.