GTX-102

Ultragenyx Pharmaceutical Inc. (RARE) focuses on novel treatments for rare genetic diseases, with products like Crysvita and Mepsevii. It collaborates globally and reported a market cap of $4.031B, with a YTD return of 3.26% as of January 8, 2025.

H.C. Wainwright maintains a Buy rating for Ultragenyx Pharma with a $95 target, citing its Phase 3 Aspire study for GTX-102 targeting Angelman syndrome. Ultragenyx shows strong revenue growth and liquidity, with a 42% increase in Q3 2024 revenue but a $134M net loss. The company aims for GAAP profitability by 2026, with potential new therapies on the horizon.

Ultragenyx submitted a BLA to the FDA for UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway. The BLA is supported by data showing UX111's ability to reduce heparan sulfate levels in the CSF and improve cognitive development. UX111 is well-tolerated and has various designations in the U.S. and EU. Ultragenyx also dosed the first patient in the Aspire study for Angelman syndrome.

Ultragenyx Pharmaceutical initiates Phase 3 Aspire study to evaluate GTX-102, an investigational antisense oligonucleotide for Angelman syndrome. The study aims to confirm safety and efficacy in a randomized trial, with plans for further assessment in the Aurora study in 2025.

Ultragenyx Pharmaceutical Inc. announced the first patient dosed in its Phase 3 Aspire study evaluating GTX-102, an investigational antisense oligonucleotide for Angelman syndrome. The study aims to confirm GTX-102's safety and efficacy in a randomized trial, with primary endpoints focusing on cognition and multi-domain responder index. GTX-102 targets the UBE3A antisense transcript to reactivate the paternally inherited UBE3A gene.

Ultragenyx Inc. announced the first patient dosed in the Phase 3 Aspire study for GTX-102, an investigational treatment for Angelman syndrome. The study aims to confirm GTX-102's safety and efficacy in children aged 4-17. GTX-102 targets UBE3A-AS to reactivate UBE3A gene expression, addressing the syndrome's root cause. The Aurora study will further evaluate GTX-102 in different genotypes and age groups starting 2025.

Ultragenyx Pharmaceutical's Phase 1/2 data for GTX-102 in Angelman syndrome showed improvements across all domains, confirming the Phase 3 Aspire study's power to establish GTX-102's efficacy. At Week 48, patients demonstrated a mean change in Bayley-4 Cognition GSV score of +6.7, and 80% achieved clinically meaningful improvement in at least one domain. GTX-102 also showed a consistent safety profile.

Ultragenyx announces Phase 1/2 data for GTX-102, an investigational antisense oligonucleotide for Angelman syndrome, showing continued improvement across multiple domains at Week 48. The Phase 3 Aspire study will enroll 120 patients with full maternal UBE3A gene deletion, with primary and secondary endpoints focusing on cognition and multi-domain responder index. GTX-102 demonstrated a consistent safety profile and aims to reactivate paternal UBE3A expression in the CNS.

Ultragenyx announces Phase 1/2 data for GTX-102, an investigational antisense oligonucleotide for Angelman syndrome, showing improvements across all domains, confirming the Phase 3 Aspire study's power to establish efficacy. The Phase 3 program is on track to begin enrollment by end-of-year.

Phase 1/2 data show GTX-102 efficacy in Angelman syndrome, supporting Phase 3 Aspire study, with enrollment expected by end-of-year.