Ionis Presents Positive Results from Phase 3 NEURO-TTRansform Trial at International Symposium on Amyloidosis

  • Eplontersen from Ionis and AstraZeneca demonstrated a statistically significant and clinically meaningful change from baseline for the co-primary and secondary endpoints at week 35 compared to the external placebo group
  • Eplontersen achieved an 81.2% reduction from baseline in the co-primary endpoint of serum transthyretin (TTR) concentration, demonstrating reduced TTR protein production
  • Eplontersen showed a favorable safety and tolerability profile

CARLSBAD, CALIFORNIA., September 7, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today presented positive results from a planned 35-week interim analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca’s eplontersen in patients with hereditary transthyretin-mediated amyloid disease Polyneuropathy (ATTRv-PN). In the study, eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for its co-primary and secondary efficacy endpoints compared to the external placebo group. Eplontersen showed a favorable safety and tolerability profile. The study results were presented today at the International Symposium on Amyloidosis (ISA) in Heidelberg, Germany.

In the study, eplontersen achieved a mean reduction in the co-primary endpoint of serum transthyretin (TTR) concentration by 81.2% (pQuality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), demonstrating that treatment with eplontersen significantly improved patient-reported quality of life compared to the external placebo group (S

Eplontersen showed a favorable safety and tolerability profile. In the study, the rate of treatment-emergent adverse events (TEAEs) in the eplontersen group was either lower or similar compared to placebo in all main categories. There were no TEAEs of particular interest that led to drug discontinuation.

The study data is consistent with the clinical profile of Ionis’ other LICA programs, further confirming the Company’s profile LIgandCconjugated Antisense technology enables Ionis to deliver potentially transformative treatments across a broad spectrum of unmet medical needs.

“Eplontersen demonstrated a clinically meaningful improvement in neuropathy impairment and quality of life compared to baseline. The significant efficacy, combined with a favorable safety and tolerability profile, indicates that eplontersen has the potential to be an important therapeutic option for patients living with this debilitating and fatal disease,” said Teresa CoelhoMD, Neurologist and Neurophysiologist at Hospital Santo António, Centro Hospitalar Universitário do Porto, Portugal and investigator for the NEURO-TTRansform study. dr Coelho presented data from the interim analysis at the ISA.

“The promising results of NEURO-TTRansform demonstrate that eplontersen had a positive impact on disease progression and improved quality of life in a significant number of patients with the disease,” said Eugene SchneiderMD, Ionis Executive Vice President and Chief Clinical Development Officer.

Based on the study results, Ionis and AstraZeneca will seek regulatory approval of eplontersen for ATTRv-PN and plan to submit a new drug application to the US Food and Drug Administration this year.

As part of a global development and commercialization agreement with AstraZeneca, eplontersen will be jointly developed and commercialized by both companies in the US and will be developed and commercialized in the rest of the world by AstraZeneca (excluding Latin America).

Eplontersen has been granted orphan drug status in the United States. It is also currently being evaluated in the Phase 3 CARDIO-TTRansform trial for amyloid transthyretin cardiomyopathy (ATTR-CM), a systemic, progressive, and fatal disease that progresses to progressive heart failure and death within four years off Diagnosis.

About NEURO-TTTransform (NCT04136184)

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 the efficacy of eplontersen to the historical placebo arm of TEGSEDI® (inotersen) NEURO-TTR pivotal study that Ionis completed in 2017. The final primary endpoint analysis will be completed at week 66 and all patients will be followed through week 85 when they have the opportunity to progress to the open-label extension study.

About Eplontersen

Eplontersen is an investigational antisense drug that uses Ionis’ advanced technology LIgandCconjugated Antisense or LICA, a technology designed to inhibit the production of the protein transthyretin (TTR) at its source. Eplontersen, to be given to patients via a self-administered auto-injector, 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 characterized by the abnormal formation of TTR protein and the aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including the peripheral nerves, heart, and gut , caused 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 a progressive decline in quality of life, severely interfering with activities of daily living. The disease often progresses rapidly and can lead to premature death. The median survival time is 4.7 years after diagnosis.

About Ionis Pharmaceuticals, Inc.

For more than 30 years, Ionis has been at the forefront of RNA-targeted therapy, pioneering new markets and changing the standard of care with its novel antisense technology. Ionis currently has three marketed drugs and a world-class late-stage pipeline highlighted by industry-leading cardiovascular and neurology franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, fueling our vision to become a leading, fully integrated biotechnology company.

To learn more about Ionis, visit and follow us on Twitter @ionispharma.

Ionis Forward-Looking Statements

This press release contains forward-looking statements regarding Ionis’ business and the therapeutic and commercial potential of Ionis’ technologies, eplontersen and other products in development. Any statement that describes Ionis’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered a statement that involves risk. Such statements are subject to certain risks and uncertainties, including those related to the impact that COVID-19 may have on our business and including but not limited to those relating to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing, and commercializing drugs that are safe and effective for use as human therapeutics, and seeking to build a business around such drugs. Ionis’ forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause results to differ materially from those expressed or implied by such forward-looking statements.

Although Ionis’ forward-looking statements reflect management’s good faith judgment, these statements are based only on facts and factors currently known to Ionis. Therefore, you are cautioned not to rely on these forward-looking statements. These and other risks related to Ionis’ programs are described in more detail in Ionis’ Annual Report on Form 10-K for the current year December 31, 2021, and most recent quarterly filing of Form 10-Q on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.

In this press release, “Ionis”, “company”, “we”, “our” and “us” refer to Ionis Pharmaceuticals and its subsidiaries unless the context otherwise requires.

Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc.

SOURCE Ionis Pharmaceuticals, Inc.


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