Akcea and Ionis Present Positive Early-Stage Data for Experimental ATTR Therapy

Rare Daily Staff

Akcea Therapeutics and Ionis Pharmaceuticals presented positive data from a 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.

AKCEA-TTR-LRx is an antisense drug developed using Ionis’ proprietary technology platform. It was discovered by Ionis and is being co-developed by Ionis and its affiliate Akcea. It 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.

Hereditary transthyretin amyloidosis (hATTR) is a progressive, systemic, and fatal inherited diseases that is 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, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow.

The progressive accumulation of TTR amyloid deposits in tissues and organs leads to sensory, motor, and autonomic dysfunction. Patients with hATTR amyloidosis often experience nerve degeneration and damage to the heart. Ultimately, hATTR amyloidosis results in death within three to 15 years of symptom onset.

The goal of the phase 1 study of AKCEA-TTR-LRx was to assess its effects 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 that patients who received monthly injections achieved a mean reduction of TTR levels of 86 percent to 94 percent depending on dosage. All adverse events were mild with the exception of one moderate headache event.

“These results are encouraging as we expand our commitment to the TTR amyloidosis community through development of AKCEA-TTR-LRx for patients with both hereditary and wild type forms of ATTR amyloidosis,” said Louis O’Dea, chief medical officer at Akcea. “Our goal is to improve patients’ lives with a safe and efficacious therapy that targets and reduces TTR protein at its source. The convenience of monthly administration would also represent a significant advantage for patients living with this debilitating and fatal disease.”

Akcea and Ionis also plan to initiate the phase 3 program for AKCEA-TTR-LRx later this year.

Photo: Louis O’Dea, chief medical officer at Akcea

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