Ionis Pharmaceuticals Inc 8-K, filed Jun 21, 2022
Ionis Pharmaceuticals Inc (IONS)
8-K
2022-06-21
For: 2022-06-21
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Added on
April 07, 2026
About this 8-K
Ionis Pharmaceuticals Inc filed this 8-K on June 21, 2022.
Items reported
- Item 8.01: Other events
- Item 9.01: Financial statements and exhibits
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
PURSUANT TO SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
PURSUANT TO SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of report (Date of earliest event reported): June 21, 2022
(Exact Name of Registrant as Specified in Charter)
(State or Other Jurisdiction of Incorporation)
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(Commission File No.)
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(IRS Employer Identification No.)
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(Address of Principal Executive Offices and Zip Code)
Registrant’s telephone number, including area code: (760 ) 931-9200
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following
provisions:
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
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Securities registered pursuant to Section 12(b) of the Act:
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Title of each class
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Trading symbol
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Name of each exchange on which registered
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“
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (Section 230.405 of this
chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (Section 240.12b-2 of this chapter).
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or
revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
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Item 8.01
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Other Events.
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On June 21, 2022, Ionis Pharmaceuticals, Inc. issued a press release announcing that eplontersen met its co-primary and secondary endpoints in an interim
analysis of the Phase 3 NEURO-TTRansform study for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
A copy of this press release is attached as Exhibit 99.1 to this Current Report and incorporated herein by reference.
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Item 9.01.
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Financial Statements and Exhibits.
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(d) Exhibits.
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Exhibit No.
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Description
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Press Release dated June 21, 2022.
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104
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Cover Page Interactive Data File (embedded within the Inline XBRL document).
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SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on
its behalf by the undersigned, thereunto duly authorized.
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Ionis Pharmaceuticals, Inc.
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Dated: June 21, 2022
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By:
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/s/ Patrick R. O’Neil
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Patrick R. O’Neil
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Executive Vice President, Chief Legal Officer and General Counsel
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Exhibit 99.1
Ionis announces eplontersen met co-primary and secondary endpoints in interim analysis of the Phase 3 NEURO-TTRansform study for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)
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Eplontersen showed statistically significant and clinically meaningful improvements in mNIS+7 and Norfolk QoL-DN
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Eplontersen demonstrated a favorable safety profile
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Ionis and AstraZeneca expect to file a New Drug Application this year
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CARLSBAD, Calif., June 21, 2022 – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from a 35-week interim analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca’s eplontersen in patients with
hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for the co-primary endpoints of percent change in serum transthyretin (TTR)
concentration and the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, versus the historical placebo group. Eplontersen also met its key secondary endpoint of change from baseline in the Norfolk
Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), showing treatment with eplontersen significantly improved patient-reported quality of life versus the historical placebo group.
In the study, eplontersen demonstrated a favorable safety and tolerability profile with no specific concerns. The study data are consistent
with the clinical profile seen across Ionis’ other LICA programs, further validating how advancements in the company’s LIgand-Conjugated Antisense technology position Ionis
to deliver potentially transformative treatments for a range of unmet medical needs.
Based on these study results, the companies will seek regulatory
approval for eplontersen for ATTRv-PN and plan to file a new drug application with the U.S. Food and Drug Administration this year.
“These encouraging data reinforce the safety profile of
eplontersen and demonstrate clear evidence of its potential to provide much needed therapeutic benefit to patients living with hereditary transthyretin-mediated amyloid polyneuropathy,” said Teresa Coelho, M.D., a neurologist and
neurophysiologist at Hospital Santo António, Centro Hospitalar Universitário do Porto, Portugal and an investigator for the NEURO-TTRansform study.
“We are pleased that the data in the NEURO-TTRansform study demonstrate eplontersen had a positive impact on disease progression, including improvement in
neuropathy impairment and quality of life in a substantial number of patients. These highly statistically significant and clinically meaningful results put us on the cusp of providing a new therapeutic option for polyneuropathy patients living with this debilitating and fatal disease,” said Eugene Schneider, M.D., Ionis’ executive vice president and chief clinical development officer. “We are
grateful to the patients, families and clinicians who are participating in NEURO-TTRansform. Without their commitment the eplontersen program would not have progressed as successfully as it has.”
The data from the 35-week interim analysis will be presented at an upcoming medical meeting later this year.
As part of a global development and commercialization agreement between
Ionis and AstraZeneca, eplontersen is being jointly developed and commercialized by both companies in the U.S. and will be developed and commercialized in the rest of the world by AstraZeneca (with the exception of Latin America).
Eplontersen was granted Orphan Drug Designation in the U.S. It is also
currently being evaluated in the Phase 3 CARDIO-TTRansform study for amyloid transthyretin cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.
For more information on the NEURO-TTRansform study, please visit: https://clinicaltrials.gov/ct2/show/NCT04136184.
About the NEURO-TTRansform Study
NEURO-TTRansform is a global, open-label, randomized study evaluating the efficacy and safety of eplontersen in patients with hereditary
transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The study has enrolled adult patients with stage 1 or stage 2 polyneuropathy and will compare efficacy of eplontersen to the historical placebo arm from the TEGSEDI® (inotersen)
NEURO-TTR registrational study that Ionis completed in 2017. The final primary endpoint analysis will be completed at week 66 and all patients will be followed until week 85 when they will have the option to transition into the open label extension
study.
The co-primary efficacy endpoints at week 66 are:
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Percent change from baseline in serum TTR concentration
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Change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression
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Change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN)
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About Eplontersen
Eplontersen is an investigational antisense medicine that uses Ionis' advanced LIgand-Conjugated Antisense,
or LICA, technology designed to inhibit the production of the transthyretin (TTR) protein at its source. Eplontersen, which is planned to be delivered to patients via an autoinjector, is in development to treat all types of ATTR, a systemic,
progressive and fatal disease.
About Hereditary Transthyretin Amyloidosis (ATTRv)
Hereditary transthyretin amyloidosis (ATTRv) is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and
aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract. The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable
peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. Hereditary transthyretin amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting
activities of daily living. The disease often progresses rapidly and can lead to premature death. The median survival is 4.7 years following diagnosis.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense
technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people
depend on us, which fuels our vision of becoming a leading, fully integrated biotechnology company.
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 Ionis' technologies,
eplontersen 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 the impact COVID-19 could have on our business, and 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 Dec. 31, 2021,
and the most recent Form 10-Q quarterly filing, which are on file with the SEC. 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" refers to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.
Ionis Investor Contact: 760-603-2331
Ionis Media Contact: 760-603-4679
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