“The initiation of our CARDIO-TTRansform Phase 3 trial represents another important milestone further demonstrating Akcea and Ionis’ commitment to bring new treatment options to patients around the world living with TTR amyloidosis. Both the wild type and hereditary forms of ATTR cardiomyopathy are underdiagnosed and potentially fatal with limited treatment options available,” said
AKCEA-TTR-LRx is an antisense drug developed using Ionis’ proprietary LIgand Conjugated Antisense (LICA) technology platform and is designed to inhibit production of TTR. It was discovered by Ionis and is being co-developed by Ionis and Akcea. In a Phase 1 clinical trial, patients treated with AKCEA-TTR-LRx experienced reductions in TTR of up to 94 percent at the highest dose.
“TTR amyloidosis is an under-recognized cause of heart failure in many adults. While there have been some advances in standard of care for this disease over the last few years, it remains a significant area of unmet need for patients around the world. One important element of the CARDIO-TTRansform trial is that patients with ATTR cardiomyopathy will be able to continue with approved treatments as we assess AKCEA-TTR-LRx. We have the potential to bring a new range of treatment options to patients living with both the hereditary and wild type forms of this fatal disease,” said
CARDIO-TTRansform Phase 3 Study Design:
CARDIO-TTRansform is a global, double-blind, randomized, placebo-controlled Phase 3 cardiovascular outcome study. It is designed to compare AKCEA-TTR-LRx to placebo in patients with both wild type and hereditary ATTR cardiomyopathy who are on the current available standard of care. The primary composite endpoint is cardiovascular (CV) mortality and frequency of CV clinical events comparing the two study arms at Week 120. Secondary endpoints include the change from baseline in the 6-minute walk test and in the Kansas City Cardiomyopathy Questionnaire (KCCQ) scores, as well as the rates of CV mortality, CV clinical events, and all-cause mortality at Week 120.
AKCEA-TTR-LRx is also being studied in patients with polyneuropathy caused by hereditary TTR amyloidosis, or hATTR amyloidosis. For more information on the Phase 3 NEURO-TTRansform study, please visit www.NEURO-TTRansform.com or www.clinicaltrials.gov (NCT04136184).
AKCEA-TTR-LRx is an antisense drug that uses Ionis’ advanced LIgand Conjugated Antisense, or LICA, technology. It was discovered by Ionis and is being co-developed by Ionis and Akcea. AKCEA-TTR-LRx inhibits the production of the transthyretin (TTR) protein at its source. AKCEA-TTR-LRx is in development to treat a broad population of patients with both hereditary and wild-type forms of transthyretin amyloidosis, or ATTR amyloidosis.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to treat a broad range of diseases including cardiovascular diseases, neurological diseases, infectious diseases, pulmonary diseases and cancer.
To learn more about Ionis visit www.ionispharma.com and follow us on Twitter @ionispharma.
AKCEA AND IONIS FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and Ionis Pharmaceuticals, Inc. and the therapeutic and commercial potential of AKCEA-TTR-LRx. Any statement describing Akcea’s or Ionis’ goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of AKCEA-TTR-LRx or other of Akcea’s or Ionis’ drugs in development is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Akcea’s and 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 Akcea’s and Ionis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea and Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea’s and Ionis’ programs are described in additional detail in Akcea’s and Ionis’ quarterly reports on Form 10-Q and annual reports on Form 10-K, which are on file with the
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