The final results from a study of the experimental drug tegsediTM (inotersen) for the treatment of patients with hereditary ATTR amyloidosis with polyneuropathy have been published. The original article can be found here, at PR Newswire.
ATTR Amyloidosis
Amyloidosis is a condition caused by the protein amyloid building up in certain areas of the body, resulting in damage. The most common form of amyloidosis is AL amyloidosis, but ATTR amyloidosis is becoming more frequently diagnosed.
ATTR amyloidosis occurs when the amyloid deposits are made from the protein transthyretin (TTR). ATTR amyloidosis can either be hereditary (when it is caused by a heritable gene alteration) or acquired (not heritable).
In heritable ATTR (hATTR) amyloidosis, the amyloid deposits mainly affect the nerves and/or heart, although other areas such as the kidneys and eyes can also develop problems. Damage the peripheral nerves is known as polyneuropathy. Therefore, hereditary ATTR amyloidosis with polyneuropathy, the condition the drug was used on in the study, refers to a genetic, heritable form of ATTR amyloidosis in which the peripheral nerves are damaged.
About TegsediTM (inotersen)
Tegsedi is an antisense drug. Antisense drugs work by binding to RNA or DNA to stop cells from producing certain molecules; tegsedi is designed to reduce the amount of transthyretin (TTR) protein produced.
Tegsedi has been granted Orphan Drug and Fast Track statuses by the US FDA, and Orphan Drug status by the European Medicines Agency. It is currently being reviewed for marketing approval in the US, Canada, and EU.
The Published Study
The pivotal study NEURO-TTR, which looked at the effects of tegsedi on patients with hATTR amyloidosis with polyneuropathy, has been published in The New England Journal of Medicine.
NURO-TTR was a Phase 3 study that compared tegsedi to a placebo in 172 patients over 15 months. Using the modified Neuropathy Impairment Score and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy as measures of the drug’s effects, the study found that patients who took tegsedi showed more improvement than those who took the placebo.
Half of those given tegsedi reported an improved quality of life, and almost 40% showed improvement in a measure of neurological disease progression. The original article quotes Dr Gertz from the Mayo Clinic as saying,
“I believe tegsedi has promising potential to treat patients with this devastating disease.”
