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Eplontersen Met Co-Primary and Secondary Endpoints in Interim Analysis of the NEURO-TTRansform Phase III Trial for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN)
Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed AstraZeneca and Ionis’ eplontersen met its co-primary endpoints in a planned interim analysis at 35 weeks. In the trial, eplontersen reached a statistically significant and clinically meaningful change from baseline for its co-primary endpoint of percent change in serum transthyretin (TTR) concentration, reducing TTR protein production. Eplontersen also reached its co-primary endpoint of change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, versus external placebo group.
High-level results showed the trial also met its secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) showing treatment with eplontersen significantly improved patient-reported quality of life versus external placebo group. In the trial, eplontersen demonstrated a favorable safety and tolerability profile with no specific concerns.
people & placesVan Selby, MDDr. Van Selby is a cardiologist who spec...
educationLocalized AmyloidosisAmyloidosis is a rare disease caused by ...
educationUnderstanding Transthyretin AmyloidosisTransthyretin amyloidosis is a slowly pr...
people & placesJack Khouri, MDJack Khouri is a Hematologist/Oncologist...
people & placesMichelle O’Shaughnessy, MDMichelle O’Shaughnessy, MB BCh, MS, ...
educationImaging of Focal Amyloid Depositions in the Head, Neck, and Spine: AmyloidomaAmyloidomas are benign tumorlike lesions...
news & meetingsNew, More Accessible Staging System Developed to Predict Survival for Patients With AL AmyloidosisAL amyloidosis is a rare condition cause...
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