Interim results amusement sustained simplification successful hereditary angioedema attacks and nary caller information signals successful patients treated for 1 twelvemonth
CARLSBAD, Calif., Nov. 13, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) contiguous presented affirmative results from a Phase 2 open-label hold (OLE) survey evaluating the information and efficacy of its investigational antisense medicine, donidalorsen, successful patients with hereditary angioedema (HAE), a uncommon and devastating inflammatory disease. Interim information aft each patients completed 1 twelvemonth of attraction successful the survey showed a sustained simplification successful HAE attacks and nary caller information signals pursuing attraction with donidalorsen. Treatment with donidalorsen resulted successful an wide sustained mean simplification successful HAE onslaught rates of 95% from baseline. For patients treated with donidalorsen, 99.6% of survey days were HAE attack-free.
The information were presented astatine the American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting successful Louisville, Ky. Additional details tin beryllium recovered connected the ACAAI website.
"Today's information further heighten donidalorsen's illustration and imaginable to supply important sustained extortion from attacks for radical surviving with hereditary angioedema," said Richard S. Geary, Ph.D., enforcement vice president and main improvement serviceman astatine Ionis. "The affirmative results of the Phase 2 OLE are encouraging arsenic we proceed evaluating donidalorsen, a imaginable best-in-class medicine, successful the ongoing OASIS Phase 3 program."
Patients completing the Phase 2 survey were eligible for enrollment successful the OLE study. There were 20 Type 1 oregon Type 2 HAE patients successful the Phase 2 study, and 17 (85%) entered the OLE. Following a 13-week fixed-dose play wherever participants received subcutaneous donidalorsen 80 mg each 4 weeks, 8 patients switched to subcutaneous donidalorsen 80 mg each 8 weeks. Patients who remained connected donidalorsen 80 mg each 4 weeks had a mean simplification successful onslaught complaint of 95.3% and 98.3%, from Week 1 (after archetypal dose) and Week 5 (after 2nd dose), respectively. Patients receiving donidalorsen 80 mg each 8 weeks experienced a mean simplification successful onslaught complaint of 75.6% from baseline and the mean monthly onslaught complaint was 0.28. Five of these patients remained onslaught escaped implicit the one-year duration of this analysis, and 3 patients returned to 80 mg each 4 weeks.
No superior adverse events were reported successful the OLE survey and nary treatment-emergent adverse events (TEAEs) led to survey discontinuation. There were nary clinically applicable abnormalities successful immoderate laboratory measurements.
About Hereditary Angioedema (HAE)
HAE is simply a uncommon and perchance fatal familial illness characterized by accelerated and achy attacks of inflammation successful the hands, feet, limbs, face, abdomen, larynx, and trachea. HAE affects astir 20,000 patients successful the U.S. and Europe and tin beryllium fatal if swelling occurs successful the larynx. In patients with predominant oregon terrible attacks, doctors whitethorn usage prophylactic attraction approaches to forestall and trim the severity of HAE attacks.
About Donidalorsen
Donidalorsen is an investigational antisense medicine that uses Ionis' precocious LIgand-Conjugated Antisense (LICA) exertion and is designed to trim the accumulation of prekallikrein, oregon PKK, to dainty patients with HAE. PKK plays an important relation successful the activation of inflammatory mediators associated with acute attacks of HAE. HAE is simply a uncommon familial illness characterized by accelerated and achy attacks of inflammation successful the hands, feet, limbs, face, abdomen, larynx, and trachea. HAE tin beryllium fatal if swelling occurs successful the larynx. In patients with predominant oregon terrible attacks, doctors whitethorn usage prophylactic attraction approaches to forestall and trim the severity of HAE attacks.
About Ionis Pharmaceuticals, Inc.
For much than 30 years, Ionis has been the person successful RNA-targeted therapy, pioneering caller markets and changing standards of attraction with its caller antisense technology. Ionis presently has 3 marketed medicines and a premier late-stage pipeline highlighted by industry-leading cardiovascular and neurological franchises. Our technological innovation began and continues with the cognition that sick radical beryllium connected us, which fuels our imaginativeness of becoming a leading, afloat integrated biotechnology company.
To larn much astir Ionis, sojourn www.ionispharma.com and travel america connected Twitter @ionispharma.
Ionis' Forward-looking Statements
This property merchandise includes forward-looking statements regarding Ionis' concern and the therapeutic and commercialized imaginable of Ionis' technologies, donidalorsen and different products successful development. Any connection describing Ionis' goals, expectations, fiscal oregon different projections, intentions oregon beliefs is simply a forward-looking connection and should beryllium considered an at-risk statement. Such statements are taxable to definite risks and uncertainties, including those related to the interaction COVID-19 could person connected our business, and including but not constricted to, those related to our commercialized products and the medicines successful our pipeline, and peculiarly those inherent successful the process of discovering, processing and commercializing medicines that are harmless and effectual for usage arsenic quality therapeutics, and successful the endeavor of gathering a concern astir specified medicines. Ionis' forward-looking statements besides impact assumptions that, if they ne'er materialize oregon beryllium correct, could origin its results to disagree materially from those expressed oregon implied by specified forward-looking statements.
Although Ionis' forward-looking statements bespeak the bully religion judgement of its management, these statements are based lone connected facts and factors presently known by Ionis. As a result, you are cautioned not to trust connected these forward-looking statements. These and different risks concerning Ionis' programs are described successful further item successful Ionis' yearly study connected Form 10-K for the twelvemonth ended December 31, 2021, and the astir caller Form 10-Q quarterly filing, which is connected record with the Securities and Exchange Commission. Copies of these and different documents are disposable from the Company.
In this property release, unless the discourse requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is simply a trademark of Ionis Pharmaceuticals, Inc.
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SOURCE Ionis Pharmaceuticals, Inc.
Company Codes: NASDAQ-NMS:IONS
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