Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) on Thursday reported mixed fourth-quarter earnings and initiated a strategic restructuring plan.
“The year ahead marks an important turning point for the company, as we approach two potential product launches and a pivotal data readout that, together, could significantly accelerate our commercial revenue trajectory,” said Emil Kakkis, CEO and president of Ultragenyx.
“We are implementing a strategic restructuring plan to reduce our operating expenses and ensure our resources are squarely aligned with our highest-impact opportunities, while leading the future of rare disease with multiple first-ever treatments,” Kakkis commented on Thursday.
FDA Update
Tucked in its earnings press release, the company said it received an Incomplete Response Letter (IRL) from the U.S Food and Drug Administration (FDA) regarding its resubmitted marketing application.
The application seeks approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy for Sanfilippo syndrome type A (MPS IIIA).
The IRL requests additional supportive documentation related to its CRL CMC responses, which the company will provide in a resubmission.
William Blair wrote, “While the IRL is frustrating, we think it will ultimately be promptly resolved.”
Earnings Snapshot
Ultragenyx reported a fourth-quarter loss of $1.29 per share, missing the consensus of $1.14.
Sales $207 million jumped from $165 million a year ago, beating the consensus of $196.97 million.
Crysvita’s revenue was $145 million. Dojolvi revenue reached $32 million, and Evkeeza sales were $17 million.
Ultragenyx has initiated a strategic restructuring plan designed to reduce its headcount and expenses.
The significant reduction and partial reinvestment of expenses, and the planned growth in revenue from current and new product launches, are designed to keep the company on its path to profitability in 2027.
The company announced a 10% workforce reduction, impacting approximately 130 employees.
Outlook
Ultragenyx forecasts fiscal 2026 sales of $730 million-$760 million versus the consensus of $786.93 million.
Crysvita revenue is expected to be between $500 million and $520 million, reflecting growing underlying global demand partially offset by the expected timing of ordering patterns in Brazil.
Dojolvi revenue to be $100 million-$110 million.
Restructuring Drives Cost Discipline Through 2027
- In 2026, combined R&D and SG&A expenses are expected to be flat to down low single digits versus 2025.
- This includes the impact of spend reductions and approximately $50 million for severance, manufacturing, and other non-recurring restructuring charges.
- In 2027, R&D expenses are expected to decrease from 2025 levels by 38%, or approximately $280 million, driven by the completion of multiple phase 3 studies and the reduction of early-stage research efforts.
- On a combined basis, 2027 R&D and SG&A expenses are expected to decrease at least 15% versus 2025.
RARE Price Action: Ultragenyx Pharmaceutical shares were down 10.21% at $21.10 at the time of publication on Friday, according to Benzinga Pro data.
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