Interim outcomes present sustained discount in hereditary angioedema assaults and no new security alerts in sufferers handled for one 12 months
CARLSBAD, Calif., Nov. 13, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) in the present day offered constructive outcomes from a Part 2 open-label extension (OLE) examine evaluating the security and efficacy of its investigational antisense medication, donidalorsen, in sufferers with hereditary angioedema (HAE), a uncommon and devastating inflammatory illness. Interim information in any case sufferers accomplished 1 12 months of therapy within the examine confirmed a sustained discount in HAE assaults and no new security alerts following therapy with donidalorsen. Remedy with donidalorsen resulted in an general sustained imply discount in HAE assault charges of 95% from baseline. For sufferers handled with donidalorsen, 99.6% of examine days have been HAE attack-free.
The info have been offered on the American School of Allergy, Bronchial asthma & Immunology (ACAAI) Annual Scientific Assembly in Louisville, Ky. Extra particulars will be discovered on the ACAAI website.
“At present’s information additional improve donidalorsen’s profile and potential to offer vital sustained safety from assaults for individuals dwelling with hereditary angioedema,” mentioned Richard S. Geary, Ph.D., govt vp and chief growth officer at Ionis. “The constructive outcomes of the Part 2 OLE are encouraging as we proceed evaluating donidalorsen, a possible best-in-class medication, within the ongoing OASIS Part 3 program.”
Sufferers finishing the Part 2 examine have been eligible for enrollment within the OLE examine. There have been 20 Kind 1 or Kind 2 HAE sufferers within the Part 2 examine, and 17 (85%) entered the OLE. Following a 13-week fixed-dose interval the place contributors acquired subcutaneous donidalorsen 80 mg each 4 weeks, eight sufferers switched to subcutaneous donidalorsen 80 mg each eight weeks. Sufferers who remained on donidalorsen 80 mg each 4 weeks had a imply discount in assault charge of 95.3% and 98.3%, from Week 1 (after first dose) and Week 5 (after second dose), respectively. Sufferers receiving donidalorsen 80 mg each eight weeks skilled a imply discount in assault charge of 75.6% from baseline and the imply month-to-month assault charge was 0.28. 5 of those sufferers remained assault free over the one-year length of this evaluation, and three sufferers returned to 80 mg each 4 weeks.
No critical antagonistic occasions have been reported within the OLE examine and no treatment-emergent antagonistic occasions (TEAEs) led to review discontinuation. There have been no clinically related abnormalities in any laboratory measurements.
About Hereditary Angioedema (HAE)
HAE is a uncommon and doubtlessly deadly genetic illness characterised by speedy and painful assaults of irritation within the palms, toes, limbs, face, stomach, larynx, and trachea. HAE impacts roughly 20,000 sufferers within the U.S. and Europe and will be deadly if swelling happens within the larynx. In sufferers with frequent or extreme assaults, medical doctors might use prophylactic therapy approaches to forestall and cut back the severity of HAE assaults.
Donidalorsen is an investigational antisense medication that makes use of Ionis’ superior LIgand-Conjugated Antisense (LICA) know-how and is designed to scale back the manufacturing of prekallikrein, or PKK, to deal with sufferers with HAE. PKK performs an vital function within the activation of inflammatory mediators related to acute assaults of HAE. HAE is a uncommon genetic illness characterised by speedy and painful assaults of irritation within the palms, toes, limbs, face, stomach, larynx, and trachea. HAE will be deadly if swelling happens within the larynx. In sufferers with frequent or extreme assaults, medical doctors might use prophylactic therapy approaches to forestall and cut back the severity of HAE assaults.
About Ionis Prescribed drugs, Inc.
For greater than 30 years, Ionis has been the chief in RNA-targeted remedy, pioneering new markets and altering requirements of care with its novel antisense know-how. Ionis at the moment has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading cardiovascular and neurological franchises. Our scientific innovation started and continues with the information that sick individuals depend upon us, which fuels our imaginative and prescient of changing into a number one, absolutely built-in biotechnology firm.
To study extra about Ionis, go to www.ionispharma.com and observe us on Twitter @ionispharma.
Ionis’ Ahead-looking Statements
This press launch consists of forward-looking statements relating to Ionis’ enterprise and the therapeutic and industrial potential of Ionis’ applied sciences, donidalorsen and different merchandise in growth. Any assertion describing Ionis’ objectives, expectations, monetary or different projections, intentions or beliefs is a forward-looking assertion and ought to be thought-about an at-risk assertion. Such statements are topic to sure dangers and uncertainties, together with these associated to the impression COVID-19 might have on our enterprise, and together with however not restricted to, these associated to our industrial merchandise and the medicines in our pipeline, and significantly these inherent within the strategy of discovering, creating and commercializing medicines which are secure and efficient to be used as human therapeutics, and within the endeavor of constructing a enterprise round such medicines. Ionis’ forward-looking statements additionally contain assumptions that, in the event that they by no means materialize or show right, might trigger its outcomes to vary materially from these expressed or implied by such forward-looking statements.
Though Ionis’ forward-looking statements mirror the great religion judgment of its administration, these statements are based mostly solely on information and elements at the moment identified by Ionis. Because of this, you might be cautioned to not depend on these forward-looking statements. These and different dangers regarding Ionis’ applications are described in further element in Ionis’ annual report on Type 10-Okay for the 12 months ended December 31, 2021, and the latest Type 10-Q quarterly submitting, which is on file with the Securities and Change Fee. Copies of those and different paperwork can be found from the Firm.
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SOURCE Ionis Prescribed drugs, Inc.
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