Analyst Note| Karen Andersen, CFA |
We're not making any changes to our $62 Ionis fair value estimate following positive top-line interim results from a phase 3 study of Astra-partnered eplontersen in polyneuropathy, as the drug remains on track to be filed later this year and approved in 2023. After 35 weeks of treatment, the drug led to significant differences from historical controls on co-primary endpoints (transthyretin concentration and neuropathic disease progression) as well as a key secondary endpoint, quality of life. However, we had already assumed trial success in this indication. In addition, collaboration terms (roughly 20% royalty from Astra) and competition (Alnylam received approval of Amvuttra last week), limit cash flow impact. In this indication, we think Alnylam has the edge, with Amvuttra's every-three-month dosing (versus monthly dosing for eplontersen), although more details on the extent of eplontersen's benefit to disease progression will determine uptake. We continue to assign Ionis a narrow moat, based on the breadth and potential of its late-stage pipeline in neurology, cardiology, and rare diseases.