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Akcea and Ionis Announce Initiation of NEURO-TTRansform Phase 3 Clinical Trial for AKCEA-TTR-LRx in Patients with Polyneuropathy Driven by Hereditary TTR Amyloidosis
BOSTON and CARLSBAD, Calif., Nov. 25, 2019 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals,
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"BOSTON and CARLSBAD, Calif., Nov. 25, 2019 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today announced initiation of the NEURO-TTRansform Phase 3 clinical trial for AKCEA-TTR-LRx in patients with polyneuropathy caused by h856ereditary TTR amyloidosis, or hATTR amyloidosis.\n\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n\n “The initiation of the TTRransform Phase 3 program represents an expansion of our dedication and commitment to the ATTR community. The NEURO-TTRansform study is the first of two Phase 3 studies that we expect to initiate with AKCEA-TTR-LRx. Our Phase 1 data are encouraging and we are excited about the possibility of delivering a significant advancement for people living with hATTR amyloidosis,” said Damien McDevitt, Ph.D., interim chief executive officer at Akcea. 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. “The Phase 1 data of AKCEA-TTR-LRx are consistent with the clinical profile seen across our other LICA programs, highlighting the potential of LICA-engineered therapies to address both rare and more common diseases,” said Brett P. Monia, Ph.D., chief operating officer of Ionis. “We remain dedicated to advancing this important clinical development program as rapidly as possible and are hopeful about the prospect of bringing a new safe and effective treatment to people living with the devastating symptoms of hATTR amyloidosis in the years ahead.” NEURO-TTRansform Phase 3 Study Design NEURO-TTRansform is a global, open-label, randomized study evaluating the efficacy and safety of AKCEA-TTR-LRx in patients with polyneuropathy due to hATTR amyloidosis. The study will enroll 140 adult patients with stage 1 or stage 2 polyneuropathy and will be compared to the historical placebo arm from the TEGSEDI® (inotersen) NEURO-TTR Phase 3 study that was completed in 2017. Of the total 140 participants, 20 patients will begin on TEGSEDI and move to AKCEA...