Catherine Novack

Joon Lee

Salim Syed

Jiale Song

Joseph Schwartz

Lisa Walter

Madison El-Saadi

Madison Wynne El-Saadi

Ron Feiner

Ananda Ghosh

Andrew Fein

Anthony

Cheng Li

Eric Joseph

Jenny Leigh Gonzalez-Armenta

Joe Schwartz

Luca Issi

Martin Auster

Nicholas Econom

Roger Song

Ryan Deschner

Samantha Semenkow

Steven Seedhouse

Steve Seedhouse

Tiago Fauth

Yun Jung

Yun Zhong

- Wave Life Sciences is progressing a diverse pipeline of RNA therapeutics, including an **INHIBIN-E siRNA obesity program** in Phase 1, an **RNA editing program for Alpha-1 Antitrypsin Deficiency (AATD)**, an **exon-skipping program for Duchenne Muscular Dystrophy (DMD)**, and an **allele-selective ASO for Huntington's Disease**.  **[1631574_2156165_0]** **[1631574_2156165_1]** **[1631574_2156165_5]** **[1631574_2156165_10]** **[1631574_2156165_12]** 
- The **INHIBIN-E siRNA obesity program** has preclinical data showing monotherapy efficacy comparable to semaglutide, an additive effect with semaglutide, and prevention of weight regain, with upcoming **Q4 readouts for cohort one and two**.  **[1631574_2156165_1]** **[1631574_2156165_12]** 
- For **AATD**, the RNA editing program achieved **13 micromolar of total AATD** and **65% conversion to M protein** from C, with a 60% decrease in C protein, and a **Q1 2026 readout for the MAD cohort** is anticipated.  **[1631574_2156165_5]** **[1631574_2156165_12]** 
- The **DMD exon 53 program** demonstrated high and consistent dystrophin levels (around **7.8%**) and a statistically significant improvement in time to rise.  **[1631574_2156165_10]** 
- The company plans to file CTAs in **2026** for a new **PLMP3 program targeting MASH**, leveraging its RNA editing approach for a large liver disease population.  **[1631574_2156165_9]**

Wave Life Sciences Provides Updates on Clinical Programs and Upcoming Catalysts

- Wave Life Sciences' mission is to **unlock the broad potential of RNA medicines** to transform human health.  **[1631574_0001193125-25-214176_d76224dex991.htm:1]** 
- The company is developing a multi-modal RNA medicines platform (PRISM®) with a robust pipeline including **WVE-007** (obesity), **WVE-006** (Alpha-1 antitrypsin deficiency), **WVE-N531** (Duchenne muscular dystrophy), and **WVE-003** (Huntington's disease).  **[1631574_0001193125-25-214176_d76224dex991.htm:1]** **[1631574_0001193125-25-214176_d76224dex991.htm:2]** 
- Wave Life Sciences is **well-capitalized with a cash runway into 2027**.  **[1631574_0001193125-25-214176_d76224dex991.htm:1]** 
- Key upcoming milestones include **WVE-007 data in 4Q 2025**, **WVE-006 data in 1Q 2026**, an **NDA submission for WVE-N531 in 2026**, and an **IND application for WVE-003 in 2H 2025**.  **[1631574_0001193125-25-214176_d76224dex991.htm:12]**

Wave Life Sciences Ltd. provides corporate presentation on RNA medicines and pipeline updates

- WVE-006, a treatment for Alpha-1 Antitrypsin Deficiency (AATD), demonstrated a **favorable safety profile** across all tested doses and regimens in the Restoration 1 and 2 clinical trials, with all drug-related adverse events being mild to moderate in intensity.  **[1631574_tx_382747_2]** 
- In the Restoration 2 trial, WVE-006 showed efficient and durable RNA editing, with the 200 mg multi-dose cohort achieving a mean max MAAT protein proportion of around **64%** of total AAT.  **[1631574_tx_382747_3]** **[1631574_tx_382747_5]**  A single 200 mg dose also enabled a ZZ individual to produce a **dynamic AAT response** during an acute phase event, with serum total AAT increasing to over **20 micromolar**.  **[1631574_tx_382747_3]** **[1631574_tx_382747_4]** 
- The 400 mg single dose cohort achieved serum total AAT of **12.8 micromolar** and MAAT of **5.3 micromolar**, with circulating MAAT reaching **47.2%** of total AAT and ZAAT decreasing by **49%**.  **[1631574_tx_382747_4]** 
- The data supports **monthly or less frequent dosing** for WVE-006, with complete data from the 400 mg multiple-dose cohort expected in **Q1 2026**.  **[1631574_tx_382747_4]** **[1631574_tx_382747_9]** 
- Wave Life Sciences is eligible for substantial milestone payments of up to **$3.3 billion** from its collaboration with GSK, with anticipated payments in 2025 and beyond.  **[1631574_tx_382747_5]**

Wave Life Sciences provides WVE-006 clinical trial update

Wave Life Sciences (WVE)