Ultragenyx Pharmaceutical (RARE) Leerink Global Healthcare Conference 2026 summary

Commercial performance and pipeline priorities

• Achieved double-digit year-over-year growth for Crysvita, with strong performance from Dojolvi and Evkeeza, and steady revenue from MEPSEVII supporting pipeline development.

• Focused on advancing the gene therapy pipeline, with key programs including GSDIa, Sanfilippo, OTC, and Wilson disease, and a PDUFA date set for GSDIa.

• Emphasizing depth and maturity in the clinical pipeline, aiming to bring new therapies to market and expand the pipeline further.

Angelman syndrome program (GTX-102)

• Phase 3 study uses Bayley Cognition Score without caregiver input to minimize placebo effect, differing from Phase 1/2 design.

• Achieved a 10.9-point improvement in raw Bayley scores in treated patients, with a clinically meaningful difference defined as a 5-point change.

• Study over-enrolled to 129 patients to ensure robust statistical power, accounting for variability in neurology trials.

• FDA supports MDRI as a secondary primary endpoint, applying alpha and recognizing its value in capturing multi-domain development.

• Dose selection for pivotal trial informed by animal and Phase 1/2 clinical data, with potency claims based on observed clinical effects.

GTX-102 mechanism and Aurora study

• Greater than 80% knockdown of the antisense molecule is needed for at least 50% protein restoration, and GTX-102 achieves this threshold.

• Approximately 35% protein expression from the paternal allele is considered sufficient for clinical benefit.

• Aurora Phase 3 study is open-label, includes broader patient genotypes, and aims to support a full label through extrapolation to Aspire data.