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Positive Phase 1 Results Of AKCEA-TTR-LRx Presented At The Heart Failure Society Of America Annual Meeting
Clinical Trials and Research

Positive Phase 1 Results of AKCEA-TTR-LRx Presented at the Heart Failure Society of America Annual Meeting

Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), presented data today from the Phase 1 clinical trial of AKCEA-TTR-LRx in a poster presentation at the Heart Failure Society of America 23rd Annual Scientific Meeting in Philadelphia, Pennsylvania.

AKCEA-TTR-LRx is an antisense drug developed using Ionis’ proprietary LIgand Conjugated Antisense (LICA) technology platform. It was discovered by Ionis and is being co-developed by Ionis and Akcea. AKCEA-TTR-LRx is designed to inhibit the production of transthyretin, or TTR, and is being studied in patients with both the hereditary and the wild type forms of TTR amyloidosis or ATTR.

AKCEA-TTR-LRx Phase 1 Study Results:
The goal of the Phase 1 study was to assess the effects of AKCEA-TTR-LRx on TTR levels in healthy volunteers. In the randomized, double-blind, placebo-controlled, dose-escalation study healthy volunteers were administered AKCEA-TTR-LRx or placebo via a single subcutaneous injection or once every four weeks for 13 weeks followed by an additional 13-week period where patients did not receive treatment.

Results from the Phase 1 study show:
• Patients who received injections of 45mg monthly achieved a mean reduction in TTR levels of 86% at Week 13.
• Patients who received injections of 90mg monthly achieved a mean reduction in TTR levels of 94% at Week 13.
• Patients who received a single 120mg injection achieved a mean TTR reduction of 86% at Week 4.
• All adverse events (AEs) were mild with the exception of one moderate AE (headache).
• No AEs led to an interruption in dosing.
• There were no severe adverse events in patients treated with AKCEA-TTR-LRx.

Sep 16, 2019
Amyloidosis Support Groups (ASG) Launches New Amyloidosis Patient Registry

Amyloidosis Support Groups (ASG) Launches new Amyloidosis Patient Registry

The Amyloidosis Patient Registry is a new resource for the entire amyloidosis community from Amyloidosis Support Groups. By compiling data on patient experiences with amyloidosis, the Registry will become the go-to resource for medical researchers and caregivers in their pursuit of earlier diagnosis, better treatment, and improved quality of life for those affected by this debilitating disease.
Your participation is the key to the Registry's success. By adding your data to those of other amyloidosis patients we expand the power of the Registry to make a difference for all of us. It is easy to participate and costs nothing but your time. After you register, you will fill out a series of surveys about amyloidosis and how it affects you and your family. The surveys don't have to be done in one visit. You can return to the surveys as often as you like to answer the questions.

Any information you share is private, secure, and completely controlled by you. Your data remains anonymous to researchers accessing the Registry unless you specifically allow them access to identifiable information like your name or contact information.

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