Gary Nachman

Jay Olson

David Lebowitz

Jessica Fye

Michael Ulz

Yanan Zhu

Akash Tewari

Andy Chen

Chi Meng Fong

Luca Issi

Yaron Werber

Avi Nova

Cassie

Debjit Chattopadhyay

Gena Huidong Wang

Jacob Roberge

Jason Gerberry

Lily Young

Mani Foroohar

Steven Eynav

Zaki Molvi

- Ionis Pharmaceuticals is advancing its transition to a fully integrated commercial-stage biotechnology company, with recent independent launches of **Tringolsys** for FCS and **Donzera** for HAE.  **[874015_2170642_1]** **[874015_2170642_3]** 
- The company anticipates **three new commercial launches in 2025**, including wholly-owned **olazarsen** for severe hypertriglyceridemia and **zilganeursen** for Alexander disease, alongside partnered bepiraversin for chronic HBV.  **[874015_2170642_3]** **[874015_2170642_61]**  **Olazarsen** is positioned as a potential "blockbuster opportunity" with Phase 3 data showing **72% mean placebo-adjusted reductions in triglycerides** and **85% reduction in acute pancreatitis**, targeting an anticipated launch in **H2 2025**.  **[874015_2170642_4]** **[874015_2170642_41]** 
- Ionis projects a strong financial outlook with **$2 billion in cash at year-end** and a clear path to **sustained positive cash flow**, with cash flow breakeven anticipated in **2028**.  **[874015_2170642_51]** **[874015_2170642_53]**  The company forecasts potential **peak annual revenues exceeding $5 billion** from product revenues and royalties, including **over $3 billion** from late-stage wholly-owned medicines and **over $2 billion** from partnered medicine royalties.  **[874015_2170642_51]** **[874015_2170642_52]**

Ionis Pharmaceuticals Outlines Commercial Strategy and Financial Outlook at Innovation Day 2025

- Ionis Pharmaceuticals is hosting its 2025 Innovation Day, highlighting **two independent launches** in less than nine months for **TRYNGOLZA® (olezarsen)** and **DAWNZERA™ (donidalorsen)**.  **[874015_3343f969fdb04bc5b1f83baf9ec6ee7b_0]** **[874015_3343f969fdb04bc5b1f83baf9ec6ee7b_1]** 
- The company's pipeline includes plans for **two more independent launches in 2026** and **four partner launches by the end of 2027**, with significant advancements such as the planned NDA submission for **olezarsen in severe hypertriglyceridemia (sHTG) by year-end** and **zilganersen for Alexander disease in Q1 2026**.  **[874015_3343f969fdb04bc5b1f83baf9ec6ee7b_0]** **[874015_3343f969fdb04bc5b1f83baf9ec6ee7b_1]** **[874015_3343f969fdb04bc5b1f83baf9ec6ee7b_2]** 
- Ionis has a strong financial foundation with **~$2 billion in cash and short-term investments** (2025 financial guidance) and projects **>$5 billion in potential annual peak revenue**.  **[874015_3343f969fdb04bc5b1f83baf9ec6ee7b_2]** 
- The company has a **clear path to sustained positive cash flow** and expects to reach **cash flow breakeven in 2028**.  **[874015_3343f969fdb04bc5b1f83baf9ec6ee7b_0]** **[874015_3343f969fdb04bc5b1f83baf9ec6ee7b_2]**

Ionis Pharmaceuticals Showcases Pipeline and Financial Outlook at Innovation Day

- Ionis Pharmaceuticals, Inc. announced **positive topline results** from the pivotal study of zilganersen in children and adults with Alexander disease (AxD).  **[874015_0001140361-25-035634_ef20055880_ex99-1.htm:0]** 
- Zilganersen 50 mg demonstrated **statistically significant and clinically meaningful stabilization** on the primary endpoint of gait speed, showing a mean difference of **33.3%** compared to control at week 61 (p=**0.0412**).  **[874015_0001140361-25-035634_ef20055880_ex99-1.htm:0]** **[874015_0001140361-25-035634_ef20055880_8k.htm:2]** 
- This marks the **first time** an investigational medicine has shown a positive disease-modifying impact in Alexander disease.  **[874015_0001140361-25-035634_ef20055880_ex99-1.htm:0]** **[874015_0001140361-25-035634_ef20055880_8k.htm:2]** 
- Ionis plans to submit a new drug application (NDA) to the U.S. Food and Drug Administration in **Q1 2026**.  **[874015_0001140361-25-035634_ef20055880_ex99-1.htm:0]** **[874015_0001140361-25-035634_ef20055880_8k.htm:2]**

Ionis Announces Positive Topline Results for Zilganersen in Alexander Disease

- **Arrowhead Pharmaceuticals** has filed a Complaint for Declaratory Judgment against **Ionis Pharmaceuticals** in the United States District Court for the District of Delaware.  **[874015_06e1c1686ed24b07bb8903c3be9d68bb_0]** 
- The lawsuit seeks to declare Ionis’s United States Patent No. **9,593,333** invalid and not infringed by Arrowhead’s investigational drug, **plozasiran**, which is currently under FDA review.  **[874015_06e1c1686ed24b07bb8903c3be9d68bb_0]** 
- Arrowhead believes Ionis's allegations of infringement are baseless and is not seeking monetary relief, but rather a declaratory decree.  **[874015_06e1c1686ed24b07bb8903c3be9d68bb_0]** 
- **Plozasiran** is an investigational RNA interference therapeutic for familial chylomicronemia (FCS) and has received **Breakthrough Therapy Designation**, **Orphan Drug Designation**, and **Fast Track Designation** from the U.S. FDA.  **[874015_06e1c1686ed24b07bb8903c3be9d68bb_1]**

Ionis Pharmaceuticals Faces Patent Infringement Lawsuit from Arrowhead

IONIS PHARMACEUTICALS (IONS)