IONIS PHARMACEUTICALS, INC.

AstraZeneca and Ionis' Wainzua, a treatment for ATTR polyneuropathy, is set for EU approval after EMA's CHMP positive opinion. Wainzua, already approved in the US and Canada, reduces TTR levels by 82% and slows neurological impairment progression. It is also being developed for ATTR cardiomyopathy and is seen as a key drug for AZ's CVRM unit. Wainzua competes with Alnylam's Onpattro and Amvuttra, and Pfizer's Vyndamax/Vyndaqel.

AstraZeneca's Wainzua (eplontersen) recommended for EU approval by CHMP for treating hereditary transthyretin-mediated amyloidosis, based on NEURO-TTRansform Phase III trial results showing consistent and sustained benefits.

WAINZUA (eplontersen) recommended for EU approval for treating hereditary transthyretin-mediated amyloidosis in adults with stage 1 or 2 polyneuropathy, based on NEURO-TTRansform Phase 3 results showing consistent and sustained benefits.

Wainzua (eplotersen), produced by AstraZeneca and Ionis, recommended for approval in EU by EMA's CHMP for treating hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or 2 polyneuropathy. The CHMP opinion is based on the NEURO-TTransform phase 3 trial results showing sustained benefit on neuropathy impairment and serum transthyretin concentration over 66 weeks. The drug also improved quality of life, measured by the Norfolk QoL-DN questionnaire.

WAINZUA (eplontersen) recommended for EU approval for hATTR-PN treatment, based on NEURO-TTRansform Phase 3 results showing consistent benefits in neuropathy and QoL.

Wainzua (eplontersen) recommended for approval by EU's CHMP for treating hereditary transthyretin-mediated amyloidosis in adults with polyneuropathy, based on NEURO-TTRansform Phase III results showing consistent and sustained benefits in neuropathy impairment and quality of life versus placebo.

AstraZeneca and Ionis' Wainzua (eplontersen) recommended for approval by EU's CHMP for hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. Wainzua showed consistent and sustained benefits on serum transthyretin concentration, neuropathy impairment, and quality of life in the NEURO-TTRansform Phase III trial. If approved, it would be the only self-administered monthly treatment for ATTRv-PN in the EU.

AstraZeneca and Ionis’ Wainzua (eplontersen) recommended for approval by CHMP in EU for treating hereditary transthyretin-mediated amyloidosis in adults with stage 1 or 2 polyneuropathy. Wainzua demonstrated consistent and sustained benefits in NEURO-TTRansform Phase III trial, improving neuropathy impairment and quality of life. If approved, Wainzua would be the only self-administered monthly treatment in the EU for ATTRv-PN.

AstraZeneca and Ionis' Wainzua (eplontersen) recommended for approval by EU's CHMP for hereditary transthyretin-mediated amyloidosis in adults with stage 1 or 2 polyneuropathy. Wainzua, if approved, will be the only self-administered monthly treatment for ATTRv-PN in the EU. Based on Phase III results, Wainzua showed consistent benefits in improving neuropathy impairment and quality of life. Eplontersen is also under evaluation for transthyretin-mediated amyloid cardiomyopathy.

Wainzua (eplontersen) recommended for approval in the EU by CHMP for treating hereditary transthyretin-mediated amyloidosis in adults with polyneuropathy, based on NEURO-TTRansform Phase III results showing consistent and sustained benefits in neuropathy impairment and quality of life.