- Part 1 of HALOS trial evaluating ION582 has completed
enrollment with results expected in mid-2024
- ION582 has been generally well tolerated at all dose levels,
and participants are continuing to Part 2, the long-term extension
portion of the study
- Encouraging EEG and clinical observations support continued
development
MIAMI, Nov. 11,
2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) today announced the completion of enrollment and
positive preliminary findings from Part 1 of the ongoing HALOS
Phase 1/2a study of ION582 (BIIB121) in Angelman syndrome. ION582
was generally well tolerated in the study and showed encouraging
electroencephalogram (EEG) activity trends and early signals of
positive clinical improvement, which will need to be confirmed upon
analysis of the full data set in mid-2024. The findings were
presented today at the FAST (Foundation for Angelman Syndrome
Therapeutics) Summit; the presentation is available here. Part 1 of
the HALOS trial is a three-month, open-label, multiple-ascending
dose study. Part 2 is a long-term extension study, which will
evaluate ION582 for an additional 12 months.
Angelman syndrome is caused by a loss of function in the UBE3A
gene. ION582 is designed to unsilence the paternal UBE3A allele in
order to increase production of the UBE3A protein in the brain.
Angelman syndrome affects an estimated one in 12,000 to 20,000
people globally1. It presents profound and severe
developmental delays in motor, language and cognitive functioning,
seizures and ataxia. It is a serious neuro-developmental
disorder that presents in early childhood, resulting in complete
dependence on a caregiver. There are no treatments specifically
approved for Angelman syndrome.
"We are encouraged by the completion of enrollment in the HALOS
trial in Angelman syndrome as well as the positive preliminary
clinical findings," said Frank
Bennett, Ph.D., executive vice president and chief
scientific officer of Ionis. "We look forward to reviewing results
from the study mid-year next year, which will help us define the
next stage of development for ION582. This community has an urgent
need for new treatment advances given the significant
neuro-developmental delays and challenges faced by people with
Angelman syndrome and their caregivers. We look forward to working
with the community, investigators, regulators and our partners at
Biogen on continued progress."
HALOS preliminary clinical findings shared at the meeting
included:
- HALOS is now fully enrolled with 51 patients at 11 global
sites.
- To date, ION582 has been generally well tolerated at all dose
levels. Adverse events in the trial were consistent with patient
medical histories, Angelman diagnosis or related to intrathecal
administration.
- In patients evaluable one month after the last Part 1 dose,
exploratory clinical findings included:
- A majority of patients showed an improvement in EEG activity.
Angelman syndrome is characterized by an increase in slow Delta
brain wave activity. Approximately 70% of patients showed a
reduction in slow-wave EEG delta activity and over 80% showed an
increase in faster frequency rhythms. While no direct comparisons
should be made, this improvement in EEG activity exceeds that
observed in natural history studies over the same time period.
- A majority of patients showed improvement in overall
functioning on the Angelman Syndrome Clinical Global Improvement
Change (SAS-CGI-C) scale, which evaluates clinicians' impressions
across 9 key functional areas.
- A majority of patients showed improvement on the total Bayley
score, which is a direct assessment of clinical functioning. While
no direct comparisons should be made, these changes went beyond
those seen in natural history studies over the same time
period.
ION582 has received Orphan Drug Designation in the U.S. and is
being developed by Ionis under a strategic collaboration with
Biogen. The HALOS Phase 1/2a trial is evaluating safety,
tolerability, pharmacokinetics and pharmacodynamics in addition to
certain clinical outcomes measures. ION582 is administered
intrathecally into the cerebral spinal fluid with a lumbar
puncture. For more information on the HALOS Study (NCT05127226),
visit clinicaltrials.gov.
About Ionis Pharmaceuticals, Inc.
For more than 30
years, Ionis has been a leader in RNA-targeted therapy, pioneering
new markets and changing standards of care. Ionis currently has
four marketed medicines and a promising late-stage pipeline
highlighted by cardiovascular and neurological franchises. Our
scientific innovation began and continues with the knowledge that
sick people depend on us, which fuels our vision to become the
leader in genetic medicine, utilizing a multi-platform approach to
discover, develop and deliver life-transforming therapies.
To learn more about Ionis visit www.ionispharma.com and
follow us on Twitter @ionispharma.
Ionis' Forward-looking Statements
This press release
includes forward-looking statements regarding Ionis' business and
the therapeutic and commercial potential of ION582, Ionis'
technologies, and other products in development. Any statement
describing Ionis' goals, expectations, financial or other
projections, intentions, or beliefs is a forward-looking statement
and should be considered an at-risk statement. Such statements are
subject to certain risks and uncertainties, including but not
limited to those related to our commercial products and the
medicines in our pipeline, and particularly those inherent in the
process of discovering, developing and commercializing medicines
that are safe and effective for use as human therapeutics, and in
the endeavor of building a business around such medicines. Ionis'
forward-looking statements also involve assumptions that, if they
never materialize or prove correct, could cause its results to
differ materially from those expressed or implied by such
forward-looking statements. Although Ionis' forward-looking
statements reflect the good faith judgment of its management, these
statements are based only on facts and factors currently known by
Ionis. As a result, you are cautioned not to rely on these
forward-looking statements. These and other risks concerning Ionis'
programs are described in additional detail in Ionis' annual
report on Form 10-K for the year ended December 31, 2022, and most recent Form 10-Q,
which are on file with the Securities and Exchange Commission.
Copies of these and other documents are available at
www.ionispharma.com.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc.
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1 Mertz LG,
Christensen R, Vogel I, Hertz JM, Nielsen KB, Gronskov K,
Ostergaard JR. Angelman syndrome in Denmark. birth incidence,
genetic findings, and age at diagnosis. Am J Med Genet A.
2013;161A:2197–203.
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SOURCE Ionis Pharmaceuticals, Inc.