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GTX-102

Generic Name
GTX-102
Drug Type
Biotech

Advanced Drug Monograph

Access comprehensive pharmacological analysis, clinical applications, and therapeutic considerations.

Background

GTX-102 is an antisense oligonucleotide designed to target and inhibit the expression of UBE3A-AS. It is being investigated for the treatment of Angelman Syndrome.

Clinical Trials
Related News

Ionis Advances Antisense Therapy for Angelman Syndrome to Phase 3 Following Promising Clinical Results

• Ionis Pharmaceuticals is moving forward with Phase 3 trials for ION582, an antisense therapy designed to unsilence the paternal UBE3A gene in Angelman syndrome patients, after Biogen declined to license the drug. • In the Phase 1/2 HALOS study, 97% of participants receiving medium or high doses of ION582 showed improvements in communication, cognition, and motor function, with favorable safety profiles. • The race to develop treatments for Angelman syndrome is intensifying, with Ultragenyx's GTX-102 already in Phase 3 trials and Neuren Pharmaceuticals advancing an oral synthetic peptide, highlighting significant progress in addressing this rare genetic disorder.

Phase 3 Trial Success: GTx-104 Shows Promise in Treating Brain Hemorrhage with Reduced Hypotension Risk

• Grace Therapeutics' Phase 3 STRIVE-ON trial of GTx-104, an injectable nimodipine formulation, met its primary endpoint with 19% reduction in clinically significant hypotension compared to oral nimodipine in aSAH patients. • The novel IV formulation demonstrated superior drug delivery, with 54% of GTx-104 patients achieving ≥95% relative dose intensity compared to only 8% with oral nimodipine, alongside reduced ICU stays and ventilator days. • Grace Therapeutics plans to submit New Drug Application (NDA) in first half of 2025, potentially offering the first major innovation in aSAH treatment in nearly 40 years.

Ultragenyx Announces Positive 2024 Revenue and Advances Rare Disease Pipeline

• Ultragenyx reported preliminary 2024 revenue of $555 million to $560 million, exceeding previous guidance, driven by strong sales of Crysvita and Dojolvi. • The company anticipates total revenue between $640 million and $670 million in 2025, projecting continued growth and pipeline advancement. • Key clinical programs are progressing, including setrusumab for osteogenesis imperfecta (Phase 3) and GTX-102 for Angelman syndrome (Phase 3). • Ultragenyx expects a PDUFA decision for UX111 in Sanfilippo syndrome type A and plans to file a BLA for DTX401 in Glycogen Storage Disease Type Ia in 2025.

Ultragenyx Initiates Phase 3 Aspire Study of GTX-102 for Angelman Syndrome

• Ultragenyx has commenced the Phase 3 Aspire study to evaluate GTX-102, an experimental treatment for Angelman syndrome, in children aged 4 to 17. • The Aspire study is a randomized, sham-controlled trial with a 48-week primary efficacy analysis period, measuring cognitive improvement via the Bayley-4 cognitive raw score. • Ultragenyx is also anticipating an interim analysis from the Phase 3 Orbit study for setrusumab, expected around the end of 2024 or early 2025. • Analysts maintain a strong buy consensus on Ultragenyx stock, with price targets ranging from $48 to $140 per share, reflecting optimism about the company's pipeline.

Ultragenyx Doses First Patient in Phase 3 Aspire Study of GTX-102 for Angelman Syndrome

• Ultragenyx has initiated the Phase 3 Aspire study to evaluate GTX-102 for Angelman syndrome, a rare neurogenetic disorder. • The Aspire study will enroll 120 children aged 4-17 with Angelman syndrome, assessing cognitive and motor function improvements. • GTX-102 is an investigational antisense oligonucleotide designed to restore UBE3A protein expression by targeting UBE3A-AS. • The Aurora study is planned for 2025 to evaluate GTX-102 in other Angelman syndrome genotypes and age groups.

Ultragenyx's UX111 Gene Therapy for Sanfilippo Syndrome Type A Receives FDA Priority Review

• The FDA has granted priority review to Ultragenyx's Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A (MPS IIIA). • The FDA's decision is expected by August 18, 2025, and the agency is not planning an advisory committee meeting for this application. • UX111 has demonstrated statistically significant improvements in cognitive and communication skills, correlated with reduced heparan sulfate levels in cerebrospinal fluid. • UX111, if approved, would be the first-ever treatment for Sanfilippo syndrome type A, addressing a critical unmet need for this rare, neurodegenerative disease.

Ultragenyx's GTX-102 Shows Sustained Improvement in Angelman Syndrome Patients in Phase 1/2 Data

• Ultragenyx's GTX-102, an investigational antisense oligonucleotide, demonstrates continued improvement across multiple domains in Angelman syndrome patients at Week 48 in Phase 1/2 data. • The Phase 1/2 data supports the Phase 3 Aspire study, indicating it is adequately powered to establish the efficacy of GTX-102. • Patients in the Dose Expansion Cohorts showed a mean change in Bayley-4 Cognition Growth Scale Value (GSV) score from baseline of +6.7 at Week 48. • Approximately 80% of patients achieved clinically meaningful net improvement in at least one domain, with GTX-102 demonstrating a consistent and acceptable safety profile.

Ultragenyx's GTX-102 Shows Promise in Angelman Syndrome Phase 1/2 Trial

• Ultragenyx presented positive Phase 1/2 data for GTX-102, an antisense oligonucleotide targeting Angelman syndrome, at the FAST Global Science Summit. • Patients in the Dose Expansion Cohorts demonstrated continued improvement across multiple domains at Week 48, with a mean change in Bayley-4 Cognition GSV score of +6.7. • Approximately 80% of patients achieved clinically meaningful net improvement in at least one domain, with GTX-102 demonstrating a consistent and acceptable safety profile. • The global Phase 3 Aspire study, enrolling approximately 120 patients, will assess GTX-102's efficacy using the Bayley-4 cognitive raw score as the primary endpoint.

Ultragenyx's GTX-102 Shows Promise in Angelman Syndrome Phase 1/2 Data

• Ultragenyx presented Phase 1/2 data supporting the Phase 3 Aspire study of GTX-102 for Angelman syndrome at the FAST Global Science Summit. • Data showed continued improvement across multiple domains at Week 48, with a mean change in Bayley-4 Cognition Growth Scale Value (GSV) score of +6.7. • Week 48 data demonstrated a total net response of +2.0 (p < 0.0001) on the Multi-domain Responder Index (MDRI), indicating clinically meaningful improvement. • The Phase 3 Aspire study, designed to enroll approximately 120 patients, is on track to begin enrollment by the end of the year.

Ultragenyx's GTX-102 Shows Positive Phase 1/2 Data for Angelman Syndrome, Phase 3 Enrollment to Begin by Year-End

• Ultragenyx announced positive Phase 1/2 data for GTX-102, an investigational antisense oligonucleotide for Angelman syndrome, at the FAST Global Science Summit. • Data showed improvements across multiple domains, with a mean change in Bayley-4 Cognition Growth Scale Value (GSV) score of +6.7 from baseline at Week 48. • Week 48 data from expansion cohorts showed a total net response of +2.0 (p < 0.0001) on the Multi-domain Responder Index (MDRI), with 80% of patients achieving clinically meaningful improvement in at least one domain. • The Phase 3 Aspire study, designed to enroll approximately 120 patients, is on track to begin enrollment by the end of the year.

Ultragenyx Reports Positive Q3 2024 Results and Clinical Updates

• Ultragenyx's Q3 2024 total revenue increased by 42% year-over-year to $139 million, driven by Crysvita and Dojolvi sales growth, reaffirming its 2024 revenue guidance of $530-$550 million. • The FDA granted Breakthrough Therapy Designation for setrusumab (UX143) in osteogenesis imperfecta, based on promising Phase 2 clinical trial results showing a significant reduction in fracture rate. • Phase 3 follow-up data for DTX401 in glycogen storage disease type Ia (GSDIa) demonstrated a substantial 62% mean reduction in cornstarch intake among crossover patients. • Ultragenyx anticipates filing a J-NDA for Dojolvi in Japan by mid-2025 and a BLA submission for DTX401 in mid-2025, marking significant progress in expanding treatment options for rare diseases.

Ultragenyx's Setrusumab Receives FDA Breakthrough Therapy Designation for Osteogenesis Imperfecta

• Ultragenyx's setrusumab (UX143) has been granted Breakthrough Therapy Designation by the FDA to reduce fracture risk in osteogenesis imperfecta (OI) patients. • The FDA's decision was based on positive data from the Phase 2 Orbit study and Phase 2b ASTEROID study, demonstrating a significant reduction in fracture rates. • Setrusumab, a fully human monoclonal antibody, inhibits sclerostin to enhance bone formation, density, and strength in OI patients. • The Breakthrough Therapy Designation aims to expedite the development and review of setrusumab, offering hope for an approved treatment option for OI.

Acasti Completes Enrollment in Phase 3 STRIVE-ON Trial of GTX-104 for aSAH

• Acasti Pharma has completed patient enrollment in its Phase 3 STRIVE-ON trial evaluating GTX-104 for aneurysmal subarachnoid hemorrhage (aSAH). • The STRIVE-ON trial is comparing intravenous GTX-104 to oral nimodipine in 100 patients hospitalized with aSAH across 25 U.S. hospitals. • GTX-104, an injectable formulation of nimodipine, aims to address challenges associated with oral administration and improve patient outcomes. • Acasti anticipates STRIVE-ON data readout in early 2025 and plans to submit an NDA to the FDA in the first half of 2025.

Ultragenyx Announces Preliminary 2023 Revenue and 2024 Financial Guidance

Ultragenyx Pharmaceutical Inc. has reported its preliminary unaudited 2023 revenue results, with total revenue ranging between $430 million to $435 million. The company also provided its 2024 revenue guidance, expecting total revenue to be between $500 million to $530 million. Key highlights include significant revenue from Crysvita and Dojolvi, and a year-end 2023 cash balance of approximately $776 million. Ultragenyx anticipates several important clinical milestones in 2024, including advancements in its gene therapy programs and Phase 3 studies for various genetic diseases.
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