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Ionis Pharmaceuticals Inc - Stock Quote IONS

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Morningstar's Ionis Pharmaceuticals Inc Stock Analysis

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Maintaining Our $62 Fair Value Estimate for Ionis Following Positive Phase 3 Eplontersen Data

Karen Andersen, CFA Sector Strategist

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.

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Ionis Pharmaceuticals Inc's Company Profile

Business Description

Ionis Pharmaceuticals is the leading developer of antisense technology to discover and develop novel drugs. Its broad clinical and preclinical pipeline targets a wide variety of diseases, with an emphasis on cardiovascular, metabolic, neurological, and rare diseases. Ionis and partner Biogen brought Spinraza to market in 2016 as a treatment for a rare neuromuscular disorder, spinal muscular atrophy. Ionis subsequently brought two additional drugs to market via its cardiovascular-focused subsidiary Akcea, including ATTR amyloidosis drug Tegsedi (2018) and cardiology drug Waylivra (Europe, 2019).

2855 Gazelle Court
Carlsbad, CA, 92010
T +1 760 931-9200
Sector Healthcare
Industry Biotechnology
Most Recent Earnings Mar 31, 2022
Fiscal Year End Dec 31, 2022
Stock Type Distressed
Employees 660

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