Ionis and Akcea win the prestigious Prix Galien Award for TEGSEDI® (inotersen) as the best biotechnology product
“We are delighted to accept this award for TEGSEDI – a treatment that has already brought substantial benefits to those devastated by polyneuropathy associated with inherited transthyretin amyloidosis,” said Brett P. Monia, Ph.D., Managing Director of Ionis and Founding Scientist of Ionis who led the discovery and development of TEGSEDI. “Our vision in the discovery and development of TEGSEDI was to improve the lives of patients living with the devastating effects of this rare genetic disease which affects the lives of generations of families. I would like to thank our teams at Akcea and Ionis for their tireless work and dedication to developing TEGSEDI and giving hope to patients around the world. “
The Prix international Galien Award is awarded each year by the Galien Foundation. The Prix Galien USA committee comprises renowned leaders from the biomedical industry and academia, who are jointly responsible for evaluating the nominees. To qualify, each candidate must be approved by the United States Food and Drug Administration for marketing within the past five years and demonstrate significant potential to advance human health globally. Since its creation in 1970, the Prix Galien has been considered the equivalent of the Nobel Prize for the biopharmaceutical and medical industries.
ABOUT HEREDITARY TRANSTHYRETIN AMYLIDDOSE (hATTR)
Hereditary ATTR amyloidosis is a serious, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and the aggregation of TTR amyloid deposits in various tissues and organs of the body, including peripheral nerves, the heart, and the intestinal tract. The gradual build-up of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy and / or cardiomyopathy, as well as other manifestations of the disease. Hereditary ATTR amyloidosis causes significant morbidity and a gradual decline in quality of life, severely affecting activities of daily living. The disease often progresses quickly and can lead to premature death. Median survival is 4.7 years after diagnosis. Additional information on hereditary ATTR amyloidosis, including a full list of organizations supporting the hATTR amyloidosis community around the world, can be found at www.hattrchangethecourse.com or by visiting www.hATTRGuide.com.
IN REGARDS TOTEGSEDI® (INOTERSEN)
TEGSEDI has been approved by the United States Food and Drug Administration (FDA) for the treatment of hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR) in adults. TEGSEDI, discovered and developed by Ionis Pharmaceuticals, is the world’s first and only subcutaneous RNA targeting drug designed to reduce the production of human transthyretin (TTR) protein. TEGSEDI has also received marketing authorization in the European Union and Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with inherited transthyretin amyloidosis.
The approval is based on data from the NEURO-TTR study which was an international, phase 3, randomized (2: 1), double-blind, placebo-controlled, 15-month study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy. In NEURO-TTR, TEGSEDI demonstrated significant improvement over placebo in measures of neuropathy and quality of life as measured by the modified neuropathy impairment score +7 (mNIS + 7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. Patients treated with TEGSEDI showed similar benefit regardless of subgroups such as age, sex, race, region, neurological impairment score (NIS), Val30Met mutation status, and stage of the disease. sickness.
The approval is also based on data from the open-label NEURO-TTR (OLE) extension which is an ongoing study for patients who have completed the NEURO-TTR study, designed to assess efficacy and safety in the long term. term of TEGSEDI.
For full TEGSEDI prescribing information, please visit www.TEGSEDI.com.
About Akcea Therapeutics, Inc.
Akcea Therapeutics, Inc., is a wholly owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-based therapies. Akcea markets TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen), and with Ionis is advancing a mature pipeline of new drugs discovered by Ionis and based on Ionis proprietary antisense technology. TEGSEDI is approved in the US, EU, Canada, and Brazil, and WAYLIVRA is approved in the EU. For more information on Akcea, please visit www.akceatx.com.
ABOUT IONIS PHARMACEUTICALS, INC.
As a leader in the discovery and development of RNA-targeted drugs, Ionis has created an efficient and widely applicable drug discovery platform called antisense technology that can treat diseases for which no other therapeutic approach can. has proven to be effective. Our drug discovery platform has served as a springboard for concrete promises and fulfilled hopes 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 therapy approved for the treatment of polyneuropathy in adults with hereditary amyloidosis. transthyretin. We are targeting all patients we have not yet reached with a pipeline of over 40 new drugs designed to potentially treat a wide range of diseases, including neurological, cardio-renal, metabolic, infectious and pulmonary diseases. To learn more about Ionis, visit www.ionispharma.com and follow us on Twitter @ionispharma.
This press release contains forward-looking statements regarding the therapeutic and commercial potential of TEGSEDI (inotersen) and developing Ionis technologies and products, including the business of Akcea Therapeutics, Inc., a wholly-owned subsidiary of Ionis. Any statement describing Ionis’s objectives, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered a risky statement. These statements are subject to certain risks and uncertainties, including those relating to the impact COVID-19 may have on our business, and including, but not limited to, those relating to our commercial products and our medicines. pipeline, and in particular those inherent in the process of discovering, developing and commercializing safe and effective drugs for human therapeutic purposes, and in building a business around these drugs. Ionis’ forward-looking statements also involve assumptions which, if they do not materialize or prove to be correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis’s forward-looking statements reflect the good faith judgment of its management, such 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 relating to Ionis’ programs are described in more detail in the Ionis Annual Report on Form 10-K for the year ended. December 31, 2019, and the most recent quarterly filing of Form 10-Q, which are filed with the SEC. Copies of these and other documents are available from the Company.
Ionis Pharmaceutical® is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics® TEGSEDI??® are registered trademarks of Akcea Therapeutics, Inc.
SOURCE Akcea Therapeutics, Inc .; Ionis Pharmaceuticals, Inc.