Wells Fargo analyst Tiago Fauth sets a price target of $18 for NASDAQ:WVE, suggesting a potential 75% increase.The price target was reduced from $21 to $18 following updates on AATD data for the 200 and 400 cohort.Wave Life Sciences' stock fluctuated between $7.49 and $8.20 today, with a trading volume of 3,322,679 shares.Wave Life Sciences (NASDAQ:WVE) is a biotechnology company focused on developing innovative therapies for genetic diseases. The company aims to address unmet medical needs through its prop ...

PresentationI will now turn the call over to Kate Rausch, Vice President of Corporate Affairs and Investor Relations.Good morning, and welcome to the Wave Life Sciences RestorAATion-2 Clinical Update Conference Call. [Operator Instructions] Also, as a reminder, this conference call is being recorded today.Kate RauschHead of Investor Relations Thank you, operator. This morning, we issued a press release announcing a positive update from our ongoing RestorAATion-2 trial of WVE-006 in alpha-1 antitrypsin defic ...

Wave Life Sciences Ltd. WVE stock is trading lower on Wednesday after the company shared data from the ongoing Phase 1b/2a RestorAATion-2 Trial of WVE-006 in alpha-1 antitrypsin deficiency.WVE is encountering selling pressure. Review the technical setup here.Alpha-1 antitrypsin (AAT) deficiency is an inherited genetic disorder that reduces the production of the AAT protein, which is crucial for protecting the lungs and liver.Also Read: Wave Life Sciences Stock Jumps On Positive Data From Muscle Disorder Dru ...

Wave Life Sciences (WVE) Update / Briefing September 03, 2025 08:30 AM ET Company ParticipantsPaul Bolno - President, CEO & DirectorChris Wright - Chief Medical OfficerJoseph Schwartz - Senior MD - Rare DiseasesErik Ingelsson - Chief Scientific OfficerBenjamin Paluch - Equity Research Senior Associate - BiotechYun Zhong - SVP - Equity ResearchCatherine Novack - Director - HealthcareSteve Seedhouse - Biotechnology Equity ResearchAngela Qian - Vice PresidentConference Call ParticipantsJoon Lee - MD & Senior B ...

WVE-006: RNA editing for AATD Positive RestorAATion-2 clinical data update Investor presentation September 3, 2025 Forward-looking statements This document contains forward-looking statements. All statements other than statements of historical facts contained in this document, including statements regarding possible or assumed future results of operations, preclinical and clinical studies, business strategies, research and development plans, collaborations and partnerships, regulatory activities and timing ...

Achieved durable production of serum AAT protein at levels associated with a lower risk of AATD liver and lung diseases following repeat 200 mg doses of WVE-006 (11.9 µM total AAT, 7.2 µM M-AAT) First-ever demonstration of therapeutically restored physiological serum AAT production in a Pi*ZZ individual during a non-drug related acute phase response (20.6 µM total AAT, 10.3 µM M-AAT) Single dose of 400 mg achieved 12.8 µM total AAT and 5.3 µM M-AAT; ongoing 400 mg multidose cohort has potential to deliver ...

Wave Life Sciences is dedicated to developing RNA-based medicines using its proprietary PRISM platform. This technology enables the company to design stereopure oligonucleotides -- specialized, chemically controlled strands of nucleic acids -- allowing greater control over drug properties like efficacy and safety. By leveraging this approach, it aims to create therapies for a wide range of genetic disorders, from rare conditions to broader public health challenges. Recently, Wave Life Sciences has built a d ...

Company Performance - Wave Life Sciences reported a quarterly loss of $0.31 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.29, and compared to a loss of $0.25 per share a year ago, indicating a negative earnings surprise of -6.90% [1] - The company posted revenues of $8.7 million for the quarter ended June 2025, missing the Zacks Consensus Estimate by 31.16%, and down from $19.69 million in the same quarter last year [2] - Over the last four quarters, Wave Life Sciences has surpassed consensus EPS estimates only once and has also topped consensus revenue estimates just once [2] Stock Performance - Wave Life Sciences shares have declined approximately 32.7% since the beginning of the year, contrasting with the S&P 500's gain of 8.3% [3] - The current Zacks Rank for Wave Life Sciences is 4 (Sell), indicating expectations of underperformance in the near future [6] Future Outlook - The consensus EPS estimate for the upcoming quarter is -$0.29 on revenues of $14.5 million, and for the current fiscal year, it is -$1.12 on revenues of $49.27 million [7] - The outlook for the Medical - Biomedical and Genetics industry, where Wave Life Sciences operates, is currently in the top 37% of over 250 Zacks industries, suggesting a favorable industry environment [8]

Financial Data and Key Metrics Changes - Revenue for Q2 2025 was $8.7 million, a decrease from $19.7 million in the prior year quarter, attributed to the timing of revenue recognized under the collaboration agreement with GSK [28] - Research and development expenses increased to $43.5 million from $40.4 million in the same period in 2024, driven by spending in the inhibin E program and RNA editing programs [28] - General and administrative expenses rose to $18 million from $14.3 million in the prior year quarter, primarily due to share-based compensation and other external expenses [28] - The net loss for Q2 2025 was $50.5 million, compared to a net loss of $32.9 million in the prior year quarter [28] - Cash and cash equivalents at the end of Q2 2025 were $208.5 million, down from $302.1 million as of December 31, 2024, with expectations to fund operations into 2027 [29] Business Line Data and Key Metrics Changes - The AATD clinical program is advancing with promising data from the RESTORATION trials, showing significant increases in circulating AAT levels [7][9] - The Enlight clinical program for obesity has expanded its second cohort from 8 to 32 participants due to favorable safety and tolerability results [10][11] - FORWARD 53 clinical results for DMD demonstrated a statistically significant improvement in time to rise, marking it as a best-in-class therapeutic option [13][14] Market Data and Key Metrics Changes - The company is actively engaging with the DMD community, highlighting the importance of their FORWARD 53 clinical results [12][13] - The obesity market is being targeted with the Enlight program, which is positioned to offer a differentiated approach compared to current standard care [22][24] Company Strategy and Development Direction - The company aims to unlock the potential of RNA medicines through proprietary oligonucleotide chemistry, focusing on both rare and common diseases [4][5] - Plans to initiate clinical development of new programs in 2026, with a focus on expanding their wholly owned discovery pipeline [12][25] - The strategy includes preparing for regulatory filings and engaging with the FDA for accelerated approval pathways [15][16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing clinical programs and the potential for upcoming data readouts to inform the therapeutic pipeline [31] - The company is encouraged by the engagement from the community and clinicians regarding their AATD program, indicating a strong need for effective therapies [6][9] - Future data from the RESTORATION and Enlight trials are expected to serve as key inflection points for the company [12][25] Other Important Information - The company welcomed Dr. Chris Wright as Chief Medical Officer, bringing extensive experience in drug development [5] - The company is focused on addressing both hepatic and extrahepatic targets with their RNA editing and siRNA programs [25][26] Q&A Session Summary Question: Can you elaborate on your reasons for expanding cohort two over advancing to cohort three sooner? - The decision to expand cohort two was based on safety data indicating the ability to dose escalate, and cohort two was modeled to align with weight loss similar to semaglutide [34][36] Question: Should we expect a linear dose response, or does the preservation of lean mass offset the weight loss aspect? - The expectation is that weight loss will be driven by fat without impacting muscle, but human data will clarify the translation of these effects [38] Question: What is your guidance on the different expectations from the two data readouts for the AATD program? - The focus will be on the multidose data, which is expected to show larger liver exposure and higher protein levels compared to single doses [44][46] Question: What is the desirable knockdown level for inhibin E? - The goal is to replicate preclinical data, aiming for a knockdown that aligns with therapeutic weight loss, and the company is open to continuing dose escalation beyond cohort three [51][52] Question: Can you provide specifics on the dosing completion for the two hundred milligram multidose? - All patients in the cohort have received their seven doses, and the study remains on track for data readout in the third quarter [58][59] Question: Is there anything qualitatively about the consistency of effect for the AATD program? - The company is encouraged by the consistency observed in preclinical models and early clinical data, indicating substantial protein production [65][66]

PART I - FINANCIAL INFORMATION [Item 1. Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) This section presents Wave Life Sciences Ltd.'s unaudited consolidated financial statements as of June 30, 2025, detailing balance sheets, operations, equity changes, and cash flows, showing decreased cash and revenue, and increased net loss [Unaudited Consolidated Balance Sheets](index=5&type=section&id=Unaudited%20Consolidated%20Balance%20Sheets) This section provides a snapshot of the company's financial position, detailing assets, liabilities, and equity as of June 30, 2025, compared to December 31, 2024 Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $208,481 | $302,078 | | Total current assets | $223,556 | $320,394 | | Total assets | $252,355 | $352,207 | | Total current liabilities | $87,408 | $110,953 | | Total liabilities | $105,215 | $134,818 | | Total shareholders' equity | $139,266 | $209,515 | [Unaudited Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Unaudited%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) This section details the company's financial performance, including revenue, expenses, and net loss for the three and six months ended June 30, 2025, and 2024 Consolidated Statements of Operations Highlights (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Revenue | $8,699 | $19,692 | $17,874 | $32,230 | | Research and development | $43,469 | $40,393 | $84,091 | $73,840 | | General and administrative | $17,989 | $14,296 | $36,346 | $27,845 | | Loss from operations | $(52,759) | $(34,997) | $(102,563) | $(69,455) | | Net loss | $(50,469) | $(32,923) | $(97,347) | $(64,481) | | Net loss per share | $(0.31) | $(0.25) | $(0.60) | $(0.50) | [Unaudited Consolidated Statements of Cash Flows](index=9&type=section&id=Unaudited%20Consolidated%20Statements%20of%20Cash%20Flows) This section outlines the cash inflows and outflows from operating, investing, and financing activities for the six months ended June 30, 2025, and 2024 Consolidated Statements of Cash Flows Highlights (in thousands) | Cash Flow Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(109,051) | $(60,900) | | Net cash used in investing activities | $(471) | $(469) | | Net cash provided by financing activities | $15,848 | $15,163 | | Net decrease in cash, cash equivalents, and restricted cash | $(93,573) | $(46,361) | [Notes to Unaudited Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Unaudited%20Consolidated%20Financial%20Statements) This section provides additional details and explanations for the financial statements, including business overview, liquidity, and revenue recognition - The company is a clinical-stage biotechnology firm focused on RNA medicines, with a diversified pipeline including programs in obesity, AATD, DMD, and HD[28](index=28&type=chunk) - As of June 30, 2025, the company had **$208.5 million in cash and cash equivalents** and expects this to be sufficient to fund operations for at least the next twelve months. An additional **$24.9 million** was raised post-quarter end via its at-the-market program[32](index=32&type=chunk) - Revenue from the GSK Collaboration Agreement was **$8.7 million for Q2 2025** and **$17.9 million for the first six months of 2025**. As of June 30, 2025, unsatisfied performance obligations under the agreement totaled approximately **$66.2 million**[53](index=53&type=chunk)[57](index=57&type=chunk) - The Takeda collaboration expired in October 2024, resulting in **no revenue from this agreement in 2025**, compared to **$0.8 million** in the first six months of 2024[60](index=60&type=chunk)[61](index=61&type=chunk) Research and Development Expenses by Program (in thousands) | Program | Three Months Ended June 30, 2025 | Six Months Ended June 30, 2025 | | :--- | :--- | :--- | | AATD program | $1,846 | $3,292 | | INHBE program (Obesity) | $3,515 | $6,054 | | DMD program | $2,681 | $5,719 | | HD program | $789 | $1,956 | | Other R&D expenses | $34,638 | $67,070 | [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=21&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's business, clinical pipeline progress, financial results, and liquidity, noting decreased revenue, increased expenses, and sufficient cash for the next twelve months [Overview and Current Programs](index=21&type=section&id=Overview%20and%20Current%20Programs) This section provides an overview of the company's clinical-stage biotech platform and updates on key development programs in obesity, AATD, DMD, and HD - The company is a clinical-stage biotech utilizing its PRISM® platform to develop RNA medicines across four modalities: RNA editing (AIMers), RNA interference (siRNA), splicing, and antisense silencing[72](index=72&type=chunk)[75](index=75&type=chunk) - **Obesity (WVE-007):** Initiated INLIGHT, a Phase 1 trial in obesity, in February 2025. Dosing is complete in the expanded 240 mg cohort, with data expected in Q4 2025[83](index=83&type=chunk)[85](index=85&type=chunk) - **AATD (WVE-006):** First-ever clinical demonstration of RNA editing in humans. Data from the 200 mg single and multidose cohorts of the RestorAATion-2 trial are expected in Q3 2025. The program is exclusively licensed to GSK[88](index=88&type=chunk)[89](index=89&type=chunk)[90](index=90&type=chunk) - **DMD (WVE-N531):** Positive 48-week data from the FORWARD-53 trial showed statistically significant functional improvement. The company plans to file a New Drug Application (NDA) in 2026 for accelerated approval with monthly dosing[97](index=97&type=chunk)[99](index=99&type=chunk) - **HD (WVE-003):** Positive Phase 1b/2a data showed significant reduction in mHTT. The company is preparing for a global Phase 2/3 study and expects to submit an IND in H2 2025[105](index=105&type=chunk)[107](index=107&type=chunk) [Results of Operations](index=30&type=section&id=Results%20of%20Operations) This section analyzes the company's financial performance for the three and six months ended June 30, 2025, compared to 2024, focusing on revenue, expenses, and net loss Comparison of Results for the Three Months Ended June 30 (in thousands) | Metric | 2025 | 2024 | Change | | :--- | :--- | :--- | :--- | | Revenue | $8,699 | $19,692 | $(10,993) | | Research and development | $43,469 | $40,393 | $3,076 | | General and administrative | $17,989 | $14,296 | $3,693 | | Net loss | $(50,469) | $(32,923) | $(17,546) | - The decrease in Q2 2025 revenue was primarily driven by lower revenue recognized under the GSK Collaboration Agreement[126](index=126&type=chunk) - The increase in Q2 2025 R&D expenses was mainly due to a **$6.8 million increase in unallocated R&D costs** and a **$1.9 million increase for the INHBE (obesity) program**, partially offset by decreases in spending on the HD, DMD, and AATD programs[128](index=128&type=chunk)[131](index=131&type=chunk) Comparison of Results for the Six Months Ended June 30 (in thousands) | Metric | 2025 | 2024 | Change | | :--- | :--- | :--- | :--- | | Revenue | $17,874 | $32,230 | $(14,356) | | Research and development | $84,091 | $73,840 | $10,251 | | General and administrative | $36,346 | $27,845 | $8,501 | | Net loss | $(97,347) | $(64,481) | $(32,866) | [Liquidity and Capital Resources](index=33&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the company's cash position, funding sources, and cash flow activities, confirming sufficient liquidity for the next twelve months - As of June 30, 2025, the company had cash and cash equivalents of **$208.5 million**. Subsequent to the quarter-end, it received an additional **$24.9 million** in net proceeds from its "at-the-market" equity program[140](index=140&type=chunk) - Management expects that existing cash and cash equivalents are sufficient to fund operations for at least the next twelve months[141](index=141&type=chunk) - Net cash used in operating activities increased to **$109.1 million** for the six months ended June 30, 2025, from **$60.9 million** in the same period of 2024, primarily due to a higher net loss and changes in operating assets and liabilities[145](index=145&type=chunk)[146](index=146&type=chunk) - Net cash provided by financing activities was **$15.8 million** for the first six months of 2025, mainly from **$12.4 million** in proceeds from the "at-the-market" equity program and **$3.0 million** from share option exercises[149](index=149&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=37&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company identifies primary market risks including interest rates, foreign exchange, inflation, and capital market volatility, none of which materially impacted financial results - The company's primary market risk exposures are from interest rates, foreign currency exchange rates, inflation, and capital market volatility[158](index=158&type=chunk) - Changes in foreign currency exchange rates and inflation did not have a material impact on financial results for the reported periods[160](index=160&type=chunk)[161](index=161&type=chunk) [Controls and Procedures](index=37&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded the company's disclosure controls and procedures were effective as of June 30, 2025, with no material changes to internal control over financial reporting - Based on an evaluation as of June 30, 2025, the principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures were effective[163](index=163&type=chunk) - There were no changes in internal control over financial reporting during the quarter that have materially affected, or are reasonably likely to materially affect, such controls[164](index=164&type=chunk) PART II - OTHER INFORMATION [Legal Proceedings](index=38&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any material legal proceedings - The company is not currently a party to any material legal proceedings[165](index=165&type=chunk) [Risk Factors](index=38&type=section&id=Item%201A.%20Risk%20Factors) This section refers readers to the comprehensive risk factors detailed in the company's 2024 Annual Report on Form 10-K - The company directs investors to review the risk factors detailed in its 2024 Annual Report on Form 10-K[166](index=166&type=chunk) [Other Information](index=38&type=section&id=Item%205.%20Other%20Information) This section discloses the adoption of Rule 10b5-1 trading plans by two executive officers, the CEO and CTO, for potential sales of company shares - During Q2 2025, two executive officers adopted Rule 10b5-1 trading plans for the sale of company securities[171](index=171&type=chunk) - On May 19, 2025, CEO Paul B. Bolno adopted a plan for the sale of up to **2,563,388 ordinary shares**, active until November 15, 2025[172](index=172&type=chunk) - On May 16, 2025, CTO Chandra Vargeese adopted a plan for the sale of up to **94,600 ordinary shares**, active until November 20, 2025[173](index=173&type=chunk)