What Ultragenyx Pharmaceutical (RARE) Said on Its Q3 Earnings Call

Key Points

• Ultragenyx received FDA approval for two gene therapies—GENGLYCOS for glycogen storage disease type Ia and FAYUVI for Sanfilippo syndrome type A—its first commercial gene therapy launches. It had activated 35 treatment centers and reported patient enrollment activity for both products.
• Pipeline setbacks included discontinuing GTX-102 after a failed phase III Angelman syndrome study and ending the UX701 Wilson disease program. The company plans to cut its workforce by about 20% and reduce other costs.
• Ultragenyx reaffirmed its 2026 revenue guidance of $730 million to $760 million, excluding the new launches, and its target of GAAP profitability and positive operating cash flow in 2027. It said proceeds from selling two priority review vouchers leave it with sufficient cash to reach profitability without further fundraising.

Ultragenyx Pharmaceutical NASDAQ: RARE said its third quarter included two U.S. gene therapy approvals, a failed late-stage Angelman syndrome study, the termination of its Wilson disease program and restructuring actions intended to support GAAP profitability in 2027.

Chief Executive Officer and President Emil Kakkis said the Food and Drug Administration approved GENGLYCOS for glycogen storage disease type Ia in August and FAYUVI for Sanfilippo syndrome type A in September. The approvals bring the company’s total number of approved medicines to six and mark its first two commercial gene therapies.

At the same time, Ultragenyx discontinued development of GTX-102 for Angelman syndrome following disappointing phase III results and ended the UX701 gene therapy program for Wilson disease. The company is reducing headcount by about 20%, bringing its workforce to approximately 1,000 employees, and is taking additional measures including reducing its real estate footprint and resizing general and administrative spending.

Gene Therapy Launches Begin

Kakkis described GENGLYCOS as the first FDA-approved treatment for glycogen storage disease type Ia, a condition in which patients may need to take cornstarch every few hours to avoid potentially life-threatening hypoglycemia. He said the treatment has the potential to reduce patients’ dependence on cornstarch.

FAYUVI is approved for Sanfilippo syndrome type A, a fatal neurological disease affecting children. Kakkis said the single outpatient intravenous infusion provides the first approved treatment option for affected families.

Chief Commercial Officer Erik Harris said Ultragenyx had activated 35 qualified treatment centers as of the end of October and remained on track to exceed 40 centers by year-end. Most of the centers are expected to administer both gene therapies.

Harris said the company has received a significant number of enrollment forms for both products and that some patients have been scheduled for infusions with approved reimbursement. Early payer decisions are being made on an individual basis, though the company anticipates broad coverage over time, he said.

During the question-and-answer session, Kakkis said demand for FAYUVI has been particularly urgent because patients progressively lose neurological function. He said patients and families are actively contacting physicians, while the company is also receiving requests from outside the United States. GENGLYCOS uptake is expected to develop more methodically because patients currently have an existing, though burdensome, treatment option, he said.

Pipeline Setbacks Prompt Restructuring

Ultragenyx said GTX-102 failed to demonstrate efficacy in its phase III study in Angelman syndrome despite what Kakkis called compelling phase II findings. The company will discontinue the program.

Kakkis said the phase III outcome reflected variability in the control group across multiple endpoints, making it difficult to distinguish treatment-related changes during the one-year randomized study. He said some phase II patients experienced what he described as transformative benefits over three to five years, but the company did not see a distinctive separation across multiple endpoints in the larger randomized trial.

The company also terminated UX701 for Wilson disease after determining the program did not meet its criteria for phase III advancement. Kakkis said some patients showed clinical activity and some were able to discontinue chelators and zinc therapy, but a majority did not achieve sufficient biochemical responses and withdrawal from standard-of-care treatment. He said the treatment showed a favorable safety profile across cohorts.

Ultragenyx plans to present data from the completed Wilson disease study at a future medical or scientific congress.

• Wind down all ongoing GTX-102 studies and related spending.
• Terminate UX701 for Wilson disease.
• Reduce workforce by approximately 20%.
• Lower real estate and general and administrative costs.

2026 Guidance Reaffirmed; Profitability Target Maintained

Chief Financial Officer Howard Horn reaffirmed Ultragenyx’s 2026 revenue guidance of $730 million to $760 million, excluding revenue from the new FAYUVI and GENGLYCOS launches. The company continues to expect 2026 CRYSVITA revenue of $500 million to $520 million and DOJOLVI revenue of $100 million to $110 million.

Ultragenyx also reaffirmed that combined research and development and selling, general and administrative expenses in 2026 should be flat to down low single digits compared with 2025. That outlook includes $30 million in severance and other restructuring charges recorded in the first half and about $25 million expected in the fourth quarter.

For 2027, the company expects R&D expense to be below $350 million, a reduction of more than 50% from 2025 levels. SG&A expense is expected to be below $375 million, representing an increase of up to 7% from 2025. Horn said most R&D spending will support commercial products, including post-marketing and regulatory activities.

The company reaffirmed its expectation for full-year GAAP net income profitability and positive operating cash flow in 2027. Horn said Ultragenyx received approximately $350 million in net proceeds from the sale of two priority review vouchers, after payments to licensors and fees. One transaction closed in October, and the second was expected to close before month-end. The proceeds will be reported as other income in 2026, but Horn said the company’s projected 2027 profitability does not include PRV income.

Horn added that Ultragenyx now has sufficient cash to reach profitability without additional fundraising.

Established Products and Remaining Development Programs

Harris said CRYSVITA continued to post steady growth through the first nine months of 2026. In Latin America, approximately 60 new patients began commercial treatment during the quarter, bringing the region’s total to more than 1,050 patients on therapy. He said demand remained strong in Brazil, Mexico, Argentina and Colombia.

DOJOLVI had nearly 700 reimbursed patients in North America and approximately 315 patients receiving treatment through named-patient or early-access programs across 14 countries in Europe, the Middle East and Africa. Harris said the product recently received conditional marketing authorization in the United Kingdom for the full LC-FAOD indication and that its Japanese launch has exceeded company expectations.

EVKEEZA revenue grew approximately 50% year to date compared with the same period in 2025, Harris said. More than 500 patients across over 20 countries outside the U.S. were receiving the treatment in territories commercialized by Ultragenyx.

Looking ahead, Ultragenyx plans to complete the phase III study of DTX301 for ornithine transcarbamylase deficiency, determine a regulatory path for setrusumab in osteogenesis imperfecta and advance selected earlier-stage programs. Kakkis said the company is also evaluating internal IND-ready programs for creatine transporter deficiency and CDKL5, while seeking to operate within the financial constraints needed to maintain profitability.

About Ultragenyx Pharmaceutical (NASDAQ:RARE)

Ultragenyx Pharmaceutical Inc is a biopharmaceutical company focused on developing and commercializing treatments for rare and ultra-rare diseases. The company concentrates on conditions with significant unmet medical needs, including genetic, metabolic and endocrine disorders, and uses biologics, small molecules and gene therapy approaches in its research and development programs.

Ultragenyx's commercial portfolio includes Crysvita (burosumab), a treatment for X-linked hypophosphatemia and related disorders; Mepsevii (vestronidase alfa), an enzyme replacement therapy for mucopolysaccharidosis VII; and Dojolvi (triheptanoin), a therapy for long-chain fatty acid oxidation disorders.

This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.