Ultragenyx reports Q2 2026 results, cites two PDUFA dates and Angelman readout ahead
The company reaffirmed full-year 2026 revenue guidance and flagged upcoming FDA decisions for DTX401 and UX111 alongside pivotal Phase 3 data for GTX-102 in Angelman syndrome.
Ultragenyx Pharmaceutical reported financial results for the quarter ended June 30, 2026 on August 4, 2026. For DTX401 (pariglasgene brecaparvovec), an AAV8 gene therapy for glycogen storage disease type Ia, the FDA accepted the company's Biologics License Application for review in February 2026 and set a PDUFA action date of August 23, 2026.1 For UX111 (rebisufligene etisparvovec), an AAV9 gene therapy for Sanfilippo syndrome type A, the FDA accepted the resubmitted BLA for accelerated approval review in April 2026, with a PDUFA date of September 19, 2026.1
In the Angelman syndrome program, the Phase 3 Aspire study of GTX-102 (apazunersen), an antisense oligonucleotide, enrolled 129 patients with a full maternal UBE3A gene deletion, randomized 1:1 to drug or sham.1 Data are expected in the September or October timeframe.1 Separately, the open-label Phase 2/3 Aurora study, which is testing GTX-102 in additional genotypes and age groups, started enrolling in October 2025 and is expected to finish enrollment in the second half of 2026.1
Other pipeline updates: enrollment finished for the fourth dosing cohort in the pivotal Cyprus2+ study of UX701 for Wilson disease, with data from this stage expected in the fourth quarter of 2026.1 The FDA cleared the IND for UX016, a sialic acid prodrug for GNE myopathy, and an externally funded Phase 1/2 study is expected to begin in the second half of 2026.1 The Phase 3 Enh3ance study of DTX301 for OTC deficiency continues following patients through 64 weeks, with second primary endpoint data on treatment burden reduction expected in the first half of 2027.1
On finances, total revenue for the quarter was $214 million, and the company reaffirmed full-year 2026 total revenue guidance of $730 million to $760 million, a figure that does not include revenue from any new product launches.1 Cash, cash equivalents and marketable securities stood at $436 million as of June 30, 2026, with $97 million in net cash used in operations during the quarter.1 The company also reaffirmed guidance for combined R&D and SG&A expenses to be flat to down low single digits in 2026 versus 2025, and to decrease by at least 15% in 2027.1
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