- Two-year treatment with donidalorsen in the Phase 2
open label study resulted in consistent, sustained protection from
HAE attacks
- Completed enrollment in the Phase 3 OASIS-HAE
study
- Topline Phase 3 results expected in H1
2024
CARLSBAD, Calif., June 1, 2023
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today
announced positive clinical progress with donidalorsen, its
late-stage investigational prophylactic therapy for hereditary
angioedema (HAE). Topline two-year open-label extension (OLE)
results continue to demonstrate consistent efficacy and safety,
with an overall sustained mean reduction in HAE attack rates of 96%
from baseline through two years across dosing groups. The company
also announced that it has completed enrollment in the Phase 3
OASIS-HAE study, which is evaluating the safety and efficacy of
donidalorsen in preventing angioedema attacks. Topline data from
the study are expected in the first half of 2024. HAE is a
rare and potentially fatal genetic disease characterized by severe
and potentially fatal swelling of the arms, legs, face and
throat1,2.
"By completing enrollment in the Phase 3 study, we are one step
closer to bringing a potentially transformative and differentiated
prophylactic treatment to HAE patients," said Richard S. Geary, Ph.D., executive vice
president and chief development officer at Ionis. "We are also
encouraged by the long-term safety and durable efficacy results
seen in patients treated for two years in our ongoing open-label
extension study. We look forward to the Phase 3 data readout in the
first half of 2024 and are advancing our go-to-market preparations
to commercialize donidalorsen."
The two-year Phase 2 OLE results will be presented at an
upcoming medical congress.
In the Phase 2 study, through week 17, donidalorsen 80 mg
monthly demonstrated a 90% reduction in angioedema attacks compared
with placebo after the first dose, and a 97% reduction in
angioedema attacks starting with the second dose. The Phase 2
results also showed a significant improvement in quality of life as
assessed by the Angioedema Quality of Life Questionnaire (AE-QoL),
in the patients treated with donidalorsen. Donidalorsen continues
to demonstrate a favorable safety and tolerability profile with
added two-year OLE data.
About OASIS-HAE
OASIS-HAE is a multi-center, double-blind, randomized,
placebo-controlled study of monthly and bimonthly subcutaneous
injections of donidalorsen or placebo in 84 participants, age 12
and above, with Type 1 and Type 2 hereditary angioedema
(HAE). The study is designed to evaluate the safety and
efficacy of donidalorsen in participants with HAE and the effect of
donidalorsen on the severity and pattern of HAE attacks and its
impact on quality of life (QoL). Participants were randomized
in a 2:1 ratio to Cohort A (donidalorsen or placebo every four
weeks) or Cohort B (donidalorsen or placebo every eight weeks),
respectively. Within each cohort, participants were randomized in a
3:1 ratio to receive donidalorsen or matching-placebo. The primary
endpoint is the time-normalized number of investigator-confirmed
HAE attacks from week one to 25. A key secondary endpoint is change
in the Angioedema Quality of Life Questionnaire (AE-QoL) total
score at week 25. Additional information about OASIS-HAE
(NCT05139810) and the Phase 2 open-label extension study
(NCT04307381) may be found at ClinicalTrials.gov.
About Hereditary Angioedema (HAE)
HAE is a rare and potentially fatal genetic disease
characterized by severe and potentially fatal swelling of the arms,
legs, face and throat1,2. HAE is estimated to affect
more than 20,000 patients in the U.S. and Europe. In the U.S., doctors frequently use
prophylactic treatment approaches to prevent and reduce the
severity of HAE attacks in patients.
About Donidalorsen
Donidalorsen is an investigational
LIgand-Conjugated Antisense (LICA) medicine
designed to target the prekallikrein, or PKK, pathway. PKK plays an
important role in the activation of inflammatory mediators
associated with acute attacks of hereditary angioedema (HAE). By
inhibiting the production of PKK, donidalorsen could be an
effective prophylactic approach to preventing HAE attacks.
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 with
its novel antisense technology. 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
donidalorsen, 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 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 Dec. 31, 2022, and the most recent Form 10-Q
quarterly filing, which are on file with the Securities and
Exchange Commission. Copies of these and other documents are
available from the Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark
of Ionis Pharmaceuticals, Inc.
1 Cicardi M, et al. Allergy. 2012;67(2):
147-157.
2 Zuraw BL. N Engl J Med. 2008;359(10): 1027-1036.
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SOURCE Ionis Pharmaceuticals, Inc.