Latest NEURO-TTR Trial Data Confirm Inotersen’s Potential to Treat FAP Patients

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by Alice Melao |

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anesthesia and FAP

The latest results of the Phase 3 NEURO-TTR study continue to demonstrate the therapeutic potential of inotersen for patients with hereditary TTR amyloidosis (hATTR) with polyneuropathy, also known as familial amyloid polyneuropathy (FAP).

The investigational drug developed by Ionis Pharmaceuticals showed clinically meaningful benefits and improved quality of life in patients with FAP.

The findings were part of an oral presentation titled, “Safety and Efficacy of Inotersen in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (NEURO-TTR),” at the 142nd annual meeting of the American Neurological Association (ANA) in San Diego.

“Based on benefit observed in neurological and quality of life endpoints, inotersen treatment has shown the potential to effectively change the relentless progression of this disease and offers a convenient, at-home administration method, providing patients further liberation from the burden of hATTR,” Annabel Wang, MD, associate professor of neurology at the University of California, Irvine, and NEURO-TTR trial investigator, said in a press release.

The Phase 3 NEURO-TTR trial (NCT01737398) is an international study at 24 clinical centers to evaluate inotersen’s safety and effectivness in treating FAP patients. The study included 172 patients who randomly received weekly subcutaneous injections of 300 mg of the investigative drug or a placebo for up to 15 months. To date, more than 80% of patients have completed the study.

The most recent results showed that patients who received inotersen experienced a benefit of 11.68 points on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) after 66 weeks of treatment compared to the placebo group.

Treated patients also showed significant improvements in symptoms, as demonstrated by a 19.73 point benefit on the modified Neuropathy Impairment Score +7 (mNIS+7) compared to the placebo. Significant symptom improvements promoted by inotersen already had been reported after only eight months of treatment.

These clinically meaningful results were found to be independent of TTR gene status (mutated or normal), disease severity (stage 1 or 2), and previous administration of alternative therapies.

“The pivotal inotersen data represent new hope for patients suffering with hATTR,” Wang said. “A 20-point benefit in mNIS+7 is unprecedented and could mean the difference between the ability to walk and being confined to a wheelchair for patients suffering from this debilitating and fatal disease.”

Overall, the treatment was found to be well-tolerated, with an acceptable safety profile. Low blood platelet counts (thrombocytopenia) and renal dysfunction were reported but were manageable.

Patients who have completed the NEURO-TTR trial can continue treatment in an open-label extension study (NCT02175004) that will evaluate the safety and tolerability of long-term inotersen treatment. To date, more than 95% of patients who have completed NEURO-TTR trial are continuing treatment in the extension study.