Ionis Completes Patient Enrollment in Cohort 1 of P-III (REVEAL) Study of Obudanersen in Angelman syndrome (AS)

Shots: Ionis has completed enrollment in Cohort 1 (n=136; 2 to <18yrs.) of the global P-III (REVEAL) study assessing obudanersen (ION582) in pts with AS with genetic confirmation of either a UBE3A deletion or UBE3A mutation The Cohort 2 comprises adult pts (18 to ≤50yrs.) living with AS, with enrollment expected to be completed in…

ByRidhi RastogiJuly 7, 2026

NEWS

Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Shots: The US FDA has granted BTD to ION582 (UBE3A protein expression stimulant) for the treatment of Angelman syndrome Designation was based on P-I/II (HALOS) trial assessing ION582 in pts with Angelman syndrome, which showed improvements across all functional domains incl. communication, cognition & motor function; safety was favorable Additionally, ION582 is being investigated in…

ByRidhi RastogiSeptember 12, 2025

NEWS

Ultragenyx Completes Patient Enrolment in P-III (Aspire) Trial of GTX-102 for Angelman Syndrome

Shots: Ultragenyx has completed enrolment in its P-III (Aspire) trial to assess GTX-102 (apazunersen) in Angelman syndrome pts with confirmation of full maternal UBE3A gene deletion; Aurora study in more ages & genotypes to begin in H2'25 Trial will evaluate GTX-102 (intrathecal, 8mg QM for 3mos., then up to 14mg Q4M) vs a sham treatment…

ByRidhi RastogiAugust 1, 2025

NEWS

MavriX Bio Reports the US FDA’s IND Clearance of MVX-220 for Angelman Syndrome

Shots: The US FDA has approved IND application of MVX-220 for the treatment of Angelman Syndrome (AS) to initiate P-I/II (ASCEND-AS) trial, with first pts dosing expected in H2’25 The P-I/II trial will evaluate the safety, tolerability, & effectiveness of MVX-220 in both adult & pediatric pts with multiple AS genotypes, incl. rare types like uniparental…

ByRidhi RastogiMay 13, 2025

Ionis Completes Patient Enrollment in Cohort 1 of P-III (REVEAL) Study of Obudanersen in Angelman syndrome (AS)

Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Ultragenyx Completes Patient Enrolment in P-III (Aspire) Trial of GTX-102 for Angelman Syndrome

MavriX Bio Reports the US FDA’s IND Clearance of MVX-220 for Angelman Syndrome

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