Here is the other competing studio, only us are missing, and the...

Here is the other competing studio, only us are missing, and the Conference begins tomorrow:

Ionis announces positive detailed results from the HALOS Study of ION582 in people with Angelman syndrome
July 22, 2024 at 7:00 AM EDT

• ION582 showed robust and consistent benefit in communication, cognition and motor function in a broad patient population evaluated with a comprehensive set of assessment tools that collect input from parents and clinicians
• 97% of patients in the medium and high dose groups saw improvement in overall Angelman syndrome symptoms as measured by the SAS-CGI-C
• Improvements on the Bayley-4 in cognition, communication and motor function exceeded those observed in natural history studies
• Ionis plans to initiate Phase 3 development in H1 2025
• Ionis to host webcast on Monday, July 22 at 8:00am ET

CARLSBAD, Calif., July 22, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582 in people with Angelman syndrome (AS) demonstrating consistent and encouraging clinical improvement on measures assessing all functional domains including communication, cognition and motor function. Overall, 97% of people in the medium and high dose groups saw an improvement in overall AS symptoms as measured by the Symptoms of Angelman Syndrome–Clinician Global Impression-Change (SAS-CGI-C). ION582 showed favorable safety and tolerability at all dose levels in the study. Detailed results will be presented in a company webcast today and at the 2024 Angelman Syndrome Foundation (ASF) Family Conference in Sandusky, Ohio on July 24, 2024.
"Ionis looks forward to collaborating with investigators, regulators and members of the Angelman syndrome community to initiate Phase 3 development for ION582 in the first half of 2025," said Brett Monia, Ph.D., chief executive officer of Ionis. "Ionis has pioneered the discovery and development of groundbreaking medicines for serious neurological conditions including spinal muscular atrophy and amyotrophic lateral sclerosis. These encouraging results from the HALOS study position ION582 to be the cornerstone of Ionis' next wave of transformational, wholly owned medicines for neurological conditions, which currently includes five clinical-stage programs."

AS is a serious, rare neurodevelopmental disorder that is caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide and presents in early childhood as profound and severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. ION582 is an investigational antisense medicine designed to unsilence the normal paternal UBE3A gene to increase production of the UBE3A protein in the brain.
"Angelman syndrome is a serious neurodevelopmental disorder with life-long impairments and dependence on caregivers, for which we currently have only supportive care," said Lynne Bird, M.D., professor of clinical pediatrics at UC San Diego and HALOS study investigator. "We are very encouraged by these promising data with ION582, showing consistent improvements over what we observe in the natural course of the disease."

HALOS Study Results
HALOS included 51 people with AS, and allowed participants aged two-50 to enroll. Results presented today are from the final timepoint of the completed MAD portion of the study at six months. These results include:

• ION582 showed favorable safety and tolerability at all dose levels.
• Evidence of consistent benefit observed across all ages and genotypes as well as clinical improvement observed across key functional areas:
  • Improvements in communication, cognition and motor function exceeding the Angelman Syndrome Natural History Study (NHS) were observed on the Bayley-4, an objective and direct clinician-administered assessment of clinical functioning. See details in Table 1 below.
  • Clinical improvements were observed across key functional areas in the Vineland-3 and Observer-Reported Communication Ability (ORCA), which are both parent-reported assessment tools.
  • 97% of participants showed clinically meaningful overall improvement on the SAS-CGI-C, which evaluates clinicians' impressions of AS symptoms in study participants.

https://ir.ionis.com/news-releases/...-positive-detailed-results-halos-study-ion582


https://www.angelman.org/2024-conference/


Will our data be out tomorrow morning?????