BOSTON - Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., today announced that AIFA, or The Italian Medicines Agency, has granted approval for the reimbursement of TEGSEDI (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin (hATTR) amyloidosis.
'The approval of reimbursement for TEGSEDI in Italy is an exciting milestone as people living with hATTR amyloidosis with polyneuropathy will now have a much-needed treatment option available, and one that can be administered at home,' said Michael Pollock, SVP, head of Europe at Akcea.
'We are pleased that following the Italian Medicines Agency's detailed evaluation of TEGSEDI they are recognizing the serious unmet need of hATTR amyloidosis patients in the Country and the significance of the safety and efficacy data demonstrated thus far from this therapy, including positive long-term results from the NEURO-TTR open-label extension study that were recently published in the European Journal of Neurology,' said Massimo Boriero, VP, general manager of Italy at Akcea. 'We look forward to continue to work with the Italian Medicines Agency to rapidly bring TEGSEDI to appropriate patients.'
hATTR amyloidosis is an under-recognized, debilitating and progressive disease that is caused by the buildup of TTR proteins that misfold due to inherited genetic mutations. It is characterized by the deposition of amyloid fibrils throughout the body including in nervous tissue and can have a devastating impact on patients' quality of life.
TEGSEDI is a once-weekly, at-home subcutaneous injection that targets the polyneuropathy of hATTR amyloidosis at its source by silencing the defective gene in these patients and reducing abnormal production of the TTR protein. It is the first antisense oligonucleotide medicine available for patients in Italy with hATTR amyloidosis with polyneuropathy, and also the first treatment available to Italian patients that can be self-administered at home.
'hATTR is a complex disease and we understand how important it is for patients to have access to treatment as soon as possible as the disease can quickly progress. TEGSEDI is an important treatment option for the hATTR community, and long-term data shows that patients treated with TEGSEDI continue to experience improvements in measures of neuropathy and quality of life, with greater effects seen when treatment is started earlier in the disease lifecycle,' said Laura Obici, M.D., Fondazione IRCCS Policlinico San Matteo.
The Italian Medicines Agency's approval of reimbursement for TEGSEDI was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. Results from that study demonstrated that patients treated with TEGSEDI experienced significant benefit compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression.
In 2018, TEGSEDI was the first RNA-targeted therapeutic approved by the European Commission for adult patients with hATTR amyloidosis. Since then, TEGSEDI has continued to receive regulatory and reimbursement approvals across Europe and is now commercially available in 13 countries.
ABOUT TEGSEDI (INOTERSEN)
TEGSEDI was approved by the U.S. Food and Drug Administration (FDA) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis 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 also received marketing authorization in the European Union and Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis.
The approval is based on data from the NEURO-TTR study that was a Phase 3 randomized (2:1), double-blind, placebo-controlled, 15-month, international study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy. In NEURO-TTR, TEGSEDI demonstrated significant improvement compared to 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 experienced similar benefit regardless of subgroups such as age, sex, race, region, Neuropathy Impairment Score (NIS), Val30Met mutation status, and disease stage.
The approval is also based on data from the NEURO-TTR Open Label Extension (OLE) that is an ongoing study for patients who completed the NEURO-TTR study, designed to evaluate the long-term efficacy and safety of TEGSEDI.
ABOUT HEREDITARY TRANSTHYRETIN (hATTR) AMYLOIDOSIS
Hereditary ATTR amyloidosis 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 ATTR 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 AKCEA THERAPEUTICS, INC.
Akcea Therapeutics, Inc., a majority-owned affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), is a biopharmaceutical company focused on developing and commercializing medicines to treat patients with serious and rare diseases. Akcea is commercializing TEGSEDI (inotersen) and WAYLIVRA (volanesorsen), as well as advancing a mature pipeline of novel medicines, including AKCEA-APO(a)-LRx, vupanorsen (AKCEA-ANGPTL3-LRx), AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, with the potential to treat multiple diseases. All six medicines were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology. TEGSEDI is approved in the U.S., E.U., Canada and Brazil. WAYLIVRA is approved in the E.U. and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. Akcea is headquartered in Boston, Massachusetts, and is building the infrastructure to commercialize its medicines globally.
FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. Any statement describing Akcea's goals, expectations, financial or other projections, intentions or beliefs, including the commercial potential of Akcea's medicines 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 medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Akcea's 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 forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. In particular, we caution you that our forward-looking statements are subject to the ongoing and developing circumstances related to the COVID-19 pandemic, which may have a material adverse effect on our business, operations and future financial results. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea's programs are described in additional detail in Akcea's quarterly reports on Form 10-Q and annual reports on Form 10-K, 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', 'Akcea,' 'Company,' 'Companies' 'we,' 'our,' and 'us' refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.
Ionis Pharmaceuticals is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics, TEGSEDI and WAYLIVRA are trademarks of Akcea Therapeutics, Inc.
Contact:
Matt Roache
Tel: (617)-841-9535
Email: mroache@akceatx.com
AKCEA THERAPEUTICS, INC. 
