AI Earnings Predictions for Ultragenyx Pharmaceutical Inc. (RARE)

EX-99.1

2 rare-ex99_1.htm

EX-99.1

EX-99.1

Exhibit 99.1

Contacts Ultragenyx Pharmaceutical Inc.

Investors

Joshua Higa

ir@ultragenyx.com

Media Jess Rowlands media@ultragenyx.com

Ultragenyx Reports Third Quarter 2026 Financial Results, Announces Substantial Expense Reductions, and Reaffirms Profitability in 2027

Third quarter total revenue of $173 million,

CRYSVITA® revenue of $109 million and DOJOLVI® revenue of $30 million

GENGLYCOS™ and FAYUVI™ BLAs approved by FDA in the third quarter

Two Priority Review Vouchers (PRVs) under agreement for combined sales price of $413 million

Substantial reduction of expenses and headcount underway, expected to drive at least a 50% reduction in 2027 R&D expenses vs 2025

NOVATO, Calif. – November 03, 2026 – Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended September 30, 2026, announced substantial expense reductions, and reaffirmed its guidance for 2026 revenue and 2027 profitability.

“We received two BLA approvals from the FDA within one month for our first gene therapy treatments and the launches are progressing well with growing demand for these products,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “With substantial reductions in headcount and expenses, combined with two launches and continued growth in our base business, we expect meaningful profitability in 2027 and going forward.”

Third Quarter 2026 Revenue Highlights and 2026 Revenue Guidance

• Total revenue in the third quarter 2026 was $173 million. The company reaffirms its full year 2026 total revenue guidance of $730 million to $760 million, which excludes revenue from new product launches.

• CRYSVITA revenue in the third quarter 2026 was $109 million, consistent with expected ordering patterns. The company reaffirms its full year 2026 Crysvita revenue guidance of $500 million to $520 million.

• DOJOLVI revenue in the third quarter 2026 was $30 million. The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million.

• EVKEEZA® revenue in the third quarter 2026 was $24 million, driven by increased demand from new country launches and early access.

• MEPSEVII® revenue in the third quarter 2026 was $10 million.

Summary of Third Quarter 2026 Financial Results

Selected Financial Data (dollars in millions, except per share amounts), (unaudited)

Three Months Ended September 30,

Nine Months Ended September 30,

2026

2025

2026

2025

Total revenues

$

173

$

160

$

523

$

466

Operating expenses:

Cost of sales

32

28

96

80

Research and development

154

216

508

547

Selling, general and administrative

97

87

273

261

Total operating expenses

283

331

877

888

Net loss

$

(118

)

$

(180

)

$

(395

)

$

(446

)

Net loss per share, basic and diluted

$

(1.16

)

$

(1.81

)

$

(3.89

)

$

(4.55

)

Operating Expenses

Total operating expenses for the third quarter 2026 were $283 million, including $34 million of non-cash stock-based compensation.

Net Loss

Net loss for the third quarter 2026 was $118 million, or $1.16 per share basic and diluted, compared with a net loss for the third quarter 2025 of $180 million, or $1.81 per share basic and diluted.

Cash Balance and Net Cash Used in Operations

Cash, cash equivalents, and marketable securities were $401 million as of September 30, 2026. For the three months ended September 30, 2026, net cash used in operations was $43 million.

In October 2026, the Company entered into two definitive agreements to sell the PRVs received upon the approval of GENGLYCOS and FAYUVI for a combined price of $413 million. The sale of the GENGLYCOS PRV closed last week and the FAYUVI PRV sale is expected to close later this month. Net proceeds from both sales are expected to be approximately $350 million.

Initiation of substantial expense reductions and reaffirming profitability in 2027

With the recent completion of late-stage development programs, including first-ever FDA approvals in glycogen storage disease type Ia (GSDIa) and Sanfilippo syndrome type A, Ultragenyx is entering a new stage focused on commercial execution and a smaller, more targeted R&D footprint. To support this transition, the company has initiated substantial headcount and expense reductions and expects to reduce 2027 R&D expenses to less than $350 million and 2027 SG&A expenses to less than $375 million. Savings actions include:

• Reducing headcount by approximately 20%, resulting in a revised workforce of approximately 1,000 employees

• Completing wind-down of all ongoing studies for GTX-102 for Angelman syndrome

• Terminating UX701 for Wilson disease based on not achieving study goals

• Reducing expenses across the company, including reduction of real estate footprint

In addition to e

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EX-99.1

2 rare-ex99_1.htm

EX-99.1

EX-99.1

Exhibit 99.1

Contacts Ultragenyx Pharmaceutical Inc.

Investors

Joshua Higa

ir@ultragenyx.com

Media Jess Rowlands media@ultragenyx.com

Ultragenyx Reports First Quarter 2026 Financial Results and Corporate Update

First quarter total revenue of $136 million,

Crysvita® revenue of $93 million and Dojolvi® revenue of $18 million

Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path to profitability in 2027

GTX-102 for Angelman syndrome: New longer-term data from Phase 1/2 clinical study support durable and improving effects across multiple domains; Phase 3 data expected in the second half of 2026

NOVATO, Calif. – May 05, 2026 – Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended March 31, 2026 and reaffirmed its financial guidance for 2026.

“This is an important year for Ultragenyx with two BLAs under review and our Angelman Phase 3 readout coming soon. We have the opportunity to meaningfully accelerate our consistent commercial revenue growth over the last few years as we prepare for two potential gene therapy approvals and launches in two urgent diseases without any approved therapies,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “The latest long-term GTX-102 Phase 1/2 data further support the potential of the program, as it heads toward Phase 3 results later this year.”

First Quarter 2026 Revenue Highlights and 2026 Revenue Guidance

• Total revenue in the first quarter of 2026 was $136 million. The company reaffirms its full year 2026 total revenue guidance of $730 million to $760 million, which excludes revenue from potential new product launches.

• Crysvita revenue in the first quarter of 2026 was $93 million, consistent with expected seasonality in the U.S. and Canada and ordering patterns in Brazil. The company reaffirms its full year 2026 Crysvita revenue guidance of $500 million to $520 million.

• Dojolvi revenue in the first quarter 2026 was $18 million. The company reaffirms its full year 2026 Dojolvi revenue guidance of $100 million to $110 million.

• Evkeeza® revenue in the first quarter 2026 was $18 million, driven by increased demand from new country launches and early access.

• Mepsevii® revenue in the first quarter 2026 was $7 million.

Recent Clinical Milestones and 2026 Catalysts

• GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): As of a March 2026 Phase 1/2 data cut-off date, a total of 74 patients had been treated with GTX-102, with 66 patients continuing in the long-term extension (LTE) study. Phase 1/2 patients have been on continuous treatment for an average of more than three years, with some patients now in their fifth year, generally receiving the 14 mg quarterly maintenance dose. Patients have continued to show positive improvements across multiple domains and continued to gain ground developmentally. GTX-102 has maintained a consistent safety profile, sustained over multiple years of chronic treatment, while demonstrating no new cases of transient lower extremity weakness nor any other recurring drug-related serious adverse events. These updated efficacy and safety data are planned to be presented at a future scientific meeting.

The Phase 3 Aspire study, in patients with a full maternal UBE3A gene deletion, enrolled 129 patients, randomized 1:1 to GTX-102 or sham. Data from this study are expected in the second half of 2026.

Enrollment in the open-label Phase 2/3 Aurora study, evaluating GTX-102 in other genotypes and ages, began enrollment in October 2025 and is expected to complete enrollment in the second half of 2026.

• DTX401 (pariglasgene brecaparvovec) AAV8 gene therapy for the treatment of glycogen storage disease type Ia (GSDIa): In February 2026, the U.S. Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) seeking approval of DTX401 as a treatment for GSDIa, granted the BLA Priority Review, and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026. The FDA also recently informed the company that an Advisory Committee meeting is not anticipated at this time.

2

• UX111 (rebisufligene etisparvovec) AAV9 gene therapy for the treatment of Sanfilippo syndrome type A (MPS IIIA): In April 2026, the FDA accepted for review the resubmitted BLA seeking accelerated approval for UX111 as a treatment for MPS IIIA. The resubmitted BLA included substantial longer-term data that were presented in February at the WORLDSymposium™ 2026 and included up to eight years of follow-up. These data demonstrated further clinical improvement relative to th