Wave Life Sciences Corporate Presentation May 8, 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 thereof, competitive position, potential growth opportunities ...

PART I - FINANCIAL INFORMATION [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) This section presents the unaudited consolidated financial statements for Q1 2025, detailing financial position, operations, equity, and cash flows with accompanying notes [Unaudited Consolidated Balance Sheets](index=5&type=section&id=Unaudited%20Consolidated%20Balance%20Sheets) As of March 31, 2025, total assets decreased to **$288.3 million** from **$352.2 million** at year-end 2024, primarily due to reduced cash and cash equivalents Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $243,075 | $302,078 | | Total current assets | $257,976 | $320,394 | | Total assets | $288,343 | $352,207 | | **Liabilities & Equity** | | | | Total current liabilities | $87,367 | $110,953 | | Total liabilities | $108,666 | $134,818 | | Total shareholders' equity | $171,803 | $209,515 | [Unaudited Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Unaudited%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For Q1 2025, the company reported revenue of **$9.2 million** and a net loss of **$46.9 million**, an increase from the **$31.6 million** net loss in Q1 2024 Statement of Operations Summary (in thousands, except per share data) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Revenue | $9,175 | $12,538 | | Research and development | $40,622 | $33,447 | | General and administrative | $18,357 | $13,549 | | Loss from operations | $(49,804) | $(34,458) | | Net loss | $(46,878) | $(31,558) | | Net loss per share | $(0.29) | $(0.24) | [Unaudited Consolidated Statements of Series A Preferred Shares and Shareholders' Equity](index=7&type=section&id=Unaudited%20Consolidated%20Statements%20of%20Series%20A%20Preferred%20Shares%20and%20Shareholders%27%20Equity) Total shareholders' equity decreased to **$171.8 million** by March 31, 2025, primarily due to the quarterly net loss, partially offset by capital inflows - Total shareholders' equity decreased by **$37.7 million** during Q1 2025, from **$209.5 million** to **$171.8 million**, mainly due to the quarterly net loss of **$46.9 million**[21](index=21&type=chunk) [Unaudited Consolidated Statements of Cash Flows](index=8&type=section&id=Unaudited%20Consolidated%20Statements%20of%20Cash%20Flows) The company used **$63.0 million** in cash for operating activities during Q1 2025, resulting in a net decrease in cash, cash equivalents, and restricted cash of **$59.0 million** Cash Flow Summary (in thousands) | Activity | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(63,039) | $(33,432) | | Net cash used in investing activities | $(165) | $(417) | | Net cash provided by financing activities | $4,155 | $14,510 | | **Net decrease in cash** | **$(58,991)** | **$(19,413)** | [Notes to Unaudited Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Consolidated%20Financial%20Statements) The notes detail the company's business, liquidity, collaboration agreements, and a breakdown of research and development expenses by program - The company expects its existing cash and cash equivalents of **$243.1 million** as of March 31, 2025, to be sufficient to fund operations for at least the next twelve months[30](index=30&type=chunk) - Under the GSK Collaboration Agreement, the company recognized **$9.2 million** in revenue for Q1 2025. As of March 31, 2025, the total unsatisfied transaction price allocated to performance obligations is **$74.45 million**[53](index=53&type=chunk)[57](index=57&type=chunk) - The Takeda collaboration expired in October 2024. Consequently, no revenue was recognized under this agreement in Q1 2025, compared to **$0.3 million** in Q1 2024[59](index=59&type=chunk)[60](index=60&type=chunk) Research and Development Expenses by Program (Q1 2025, in thousands) | Program | Expense | | :--- | :--- | | AATD program | $1,446 | | INHBE program (Obesity) | $2,539 | | DMD program | $3,038 | | HD program | $1,167 | | Other R&D expenses | $32,432 | | **Total R&D Expenses** | **$40,622** | [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=21&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's RNA medicines platform, clinical pipeline progress, and financial performance, including revenue, net loss, and liquidity - The company is advancing a diversified pipeline of RNA medicines, with clinical programs in obesity, alpha-1 antitrypsin deficiency (AATD), Duchenne muscular dystrophy (DMD), and Huntington's disease (HD)[71](index=71&type=chunk) - Key clinical updates include: - **Obesity (WVE-007):** Phase 1 trial initiated, with clinical data expected in the second half of 2025 - **AATD (WVE-006):** Positive proof-of-mechanism data demonstrated; further data from multidose and single dose cohorts expected in Q3 2025 and fall 2025 - **DMD (WVE-N531):** Positive 48-week data showed significant functional improvement; plan to file for accelerated approval in 2026 - **HD (WVE-003):** Positive Phase 1b/2a data showed potent mHTT reduction; preparing for a global Phase 2/3 study with an IND submission planned for the second half of 2025[82](index=82&type=chunk)[86](index=86&type=chunk)[94](index=94&type=chunk)[102](index=102&type=chunk)[104](index=104&type=chunk) Comparison of Operations (Q1 2025 vs Q1 2024, in thousands) | Item | Q1 2025 | Q1 2024 | Change | | :--- | :--- | :--- | :--- | | Revenue | $9,175 | $12,538 | $(3,363) | | Research and development | $40,622 | $33,447 | $7,175 | | General and administrative | $18,357 | $13,549 | $4,808 | | **Net loss** | **$(46,878)** | **$(31,558)** | **$(15,320)** | - As of March 31, 2025, the company had cash and cash equivalents of **$243.1 million**, which is expected to be sufficient to fund operations for at least the next twelve months[130](index=130&type=chunk)[131](index=131&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=35&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company identifies primary market risks including interest rate, foreign currency, inflation, and capital market risks, impacting operations and future funding - The company's main market risks are identified as interest rate risk on cash holdings, foreign currency risk from international operations, inflation risk on operating costs, and capital market risk related to future funding needs[149](index=149&type=chunk) - The company does not currently hedge its foreign currency exposure and acknowledges that continued global inflation could lead to appreciable increases in clinical trial and labor costs[151](index=151&type=chunk)[152](index=152&type=chunk) [Controls and Procedures](index=36&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal control over financial reporting - Based on an evaluation as of March 31, 2025, the principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures were effective at the reasonable assurance level[155](index=155&type=chunk) - There were no changes in internal control over financial reporting during the first quarter of 2025 that have materially affected, or are reasonably likely to materially affect, these controls[156](index=156&type=chunk) PART II - OTHER INFORMATION [Legal Proceedings](index=37&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not currently a party to any material legal proceedings - As of the filing date, the company is not involved in any material legal proceedings[158](index=158&type=chunk) [Risk Factors](index=37&type=section&id=Item%201A.%20Risk%20Factors) This section refers readers to the 'Risk Factors' section of the company's Annual Report on Form 10-K for a comprehensive discussion of potential risks and uncertainties - For a detailed discussion of risk factors, the report refers to Item 1A of the company's 2024 Annual Report on Form 10-K[159](index=159&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=37&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reports that there were no unregistered sales of its equity securities and no repurchases during the first quarter of 2025 - The company made no unregistered sales of equity securities and did not repurchase any of its equity securities during the three months ended March 31, 2025[160](index=160&type=chunk)[161](index=161&type=chunk) [Defaults Upon Senior Securities](index=37&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reports that there have been no defaults upon its senior securities - No defaults upon senior securities were reported[162](index=162&type=chunk) [Mine Safety Disclosures](index=37&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company's business - This section is not applicable[163](index=163&type=chunk) [Other Information](index=37&type=section&id=Item%205.%20Other%20Information) This section discloses that several officers and directors adopted Rule 10b5-1 trading plans in March 2025 for the pre-planned sale of company shares - During Q1 2025, certain officers and directors adopted Rule 10b5-1 trading plans for the sale of company securities[164](index=164&type=chunk) - On March 13, 2025, CFO Kyle Moran adopted a plan for the sale of up to **296,000 shares**, and three board members adopted plans for the sale of an aggregate of **313,845 shares**[165](index=165&type=chunk)[166](index=166&type=chunk)[167](index=167&type=chunk)[168](index=168&type=chunk) [Exhibits](index=40&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including certifications by the Principal Executive Officer and Principal Financial Officer, and Inline XBRL documents - The report includes certifications from the Principal Executive Officer and Principal Financial Officer (Exhibits 31.1, 31.2, and 32) and Inline XBRL data files[170](index=170&type=chunk)

Exhibit 99.1 Wave Life Sciences Reports First Quarter 2025 Financial Results and Provides Business Update Dosing complete in the first two cohorts of INLIGHT trial in obesity of WVE-007 (INHBE siRNA), designed to induce healthy weight loss by reducing fat without impacting muscle; clinical data on track for 2H 2025 Dosing underway in second single dose cohort (400 mg) and multidosing (200 mg) ongoing in RestorAATion-2 clinical trial of WVE-006 in individuals with PiZZ AATD; data from the complete 200 mg mul ...

Core Insights - Wave Life Sciences Ltd. has made significant progress in its clinical trials, including the completion of dosing in the INLIGHT trial for obesity and positive data from the FORWARD-53 trial for Duchenne muscular dystrophy (DMD) [1][2][3] - The company is on track to deliver multiple clinical datasets in 2025, which will showcase its capabilities in RNA medicines [2] - Financial results for Q1 2025 indicate a net loss of $46.9 million, with cash and cash equivalents of $243.1 million, expected to fund operations into 2027 [6][7][14] Obesity - WVE-007, a GalNAc-conjugated siRNA, aims to induce healthy weight loss by silencing INHBE mRNA, with dosing completed in the first two cohorts of the INLIGHT trial [3][4] - The trial assesses safety, tolerability, pharmacokinetics, and metabolic health, with clinical data expected in the second half of 2025 [3][4] - WVE-007 has shown potential for significant weight loss without muscle loss, and when combined with semaglutide, it doubled weight loss [4] Alpha-1 Antitrypsin Deficiency (AATD) - WVE-006 is being evaluated in the RestorAATion-2 trial, with ongoing dosing in both single and multiple cohorts [1][3] - Data from the 200 mg multidose and single dose cohorts are expected in Q3 2025, while data from the 400 mg cohort is anticipated in fall 2025 [1][4] - The trial aims to demonstrate the safety and efficacy of WVE-006 in individuals with the PiZZ mutation [4] Duchenne Muscular Dystrophy (DMD) - WVE-N531 has shown statistically significant improvements in muscle health and function in the FORWARD-53 trial, with a 3.8-second improvement in Time-to-Rise [7] - The drug has received Orphan Drug Designation and is expected to file for accelerated approval in 2026 [7] - All participants in the trial have opted to continue into the extension phase, receiving monthly doses [7] Huntington's Disease (HD) - WVE-003 is positioned as a first-in-class treatment for HD, demonstrating allele-selective reduction of mutant HTT protein while preserving wild-type HTT [7] - The company is preparing for a potentially registrational Phase 2/3 study, with an IND submission expected in the second half of 2025 [7][5] Financial Overview - Revenue for Q1 2025 was $9.2 million, down from $12.5 million in the same quarter of the previous year [6][14] - Research and development expenses increased to $40.6 million from $33.4 million year-over-year, while general and administrative expenses rose to $18.4 million from $13.5 million [6][14] - The net loss for the quarter was $46.9 million, compared to a loss of $31.6 million in the prior year [6][14]

Core Viewpoint - Wave Life Sciences (WVE) is anticipated to report a year-over-year decline in earnings despite an increase in revenues for the quarter ended March 2025, which could significantly influence its stock price based on actual results compared to estimates [1][2]. Company Summary - The consensus estimate indicates a quarterly loss of $0.27 per share for Wave Life Sciences, reflecting a year-over-year change of -12.5%. Revenues are projected to be $13.97 million, representing an 11.4% increase from the previous year [3]. - Over the last 30 days, the consensus EPS estimate has been revised 0.43% lower, indicating a reassessment by analysts regarding the company's earnings prospects [4]. - The Most Accurate Estimate for Wave Life Sciences is lower than the Zacks Consensus Estimate, resulting in an Earnings ESP of -1.89%. The company currently holds a Zacks Rank of 3, making it challenging to predict an earnings beat [10][11]. Earnings Surprise History - In the last reported quarter, Wave Life Sciences was expected to post a loss of $0.17 per share but instead reported earnings of $0.17, resulting in a surprise of +200%. However, the company has only beaten consensus EPS estimates once in the last four quarters [12][13]. Industry Context - Rhythm Pharmaceuticals, another company in the Zacks Medical - Biomedical and Genetics industry, is expected to report a loss of $0.69 per share for the same quarter, indicating a year-over-year change of +70.6%. Its revenues are expected to be $40.18 million, up 54.7% from the previous year [17]. - The consensus EPS estimate for Rhythm Pharmaceuticals has been revised 0.7% lower in the last 30 days, leading to an Earnings ESP of -12.84% and a Zacks Rank of 3, complicating predictions for an earnings beat [18].

CAMBRIDGE, Mass., May 01, 2025 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, will host a live webcast and conference call at 8:30 a.m. ET on Thursday, May 8, 2025, to review the company’s first quarter 2025 financial results and provide business updates. The webcast and conference call may be accessed by visiting “Investor Events” on the investors section of the Wave Life ...

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Company Overview - Wave Life Sciences (WVE) shares increased by 6.1% to $10.02, following a significant trading volume, contrasting with an 8.9% decline over the past four weeks [1] - The company is focused on developing treatments for Duchenne muscular dystrophy (DMD) [2] Clinical Study Results - The stock's rally was driven by positive results from the phase II FORWARD-53 study, which assessed WVE-N531 for DMD treatment [2] - The study met all objectives, showing sustained exon skipping, muscle concentrations, and dystrophin restoration over 48 weeks, with a 61-day tissue half-life supporting monthly dosing [2] - Treatment with WVE-N531 was reported to be safe and well-tolerated [2] Financial Expectations - The upcoming quarterly report is expected to show a loss of $0.26 per share, reflecting an 8.3% year-over-year decline, while revenues are projected at $13.26 million, a 5.8% increase from the previous year [3] - The consensus EPS estimate for the quarter has been revised 9.8% higher in the last 30 days, indicating potential price appreciation [4] Industry Context - Wave Life Sciences operates within the Zacks Medical - Biomedical and Genetics industry, where another company, Agios Pharmaceuticals (AGIO), has seen a 12.6% decline over the past month [4] - Agios Pharmaceuticals' EPS estimate has decreased by 5.4% over the past month, indicating a challenging outlook compared to the previous year [5]

Core Insights - Wave Life Sciences announced positive results from the Phase 2 FORWARD-53 trial of WVE-N531, demonstrating significant improvements in muscle health and function in boys with Duchenne muscular dystrophy (DMD) [1][4][10] Group 1: Trial Results - The FORWARD-53 trial achieved all goals, showing a statistically significant and clinically meaningful improvement of 3.8 seconds in Time-to-Rise (TTR) compared to natural history, the largest effect observed relative to any approved dystrophin restoration therapy at 48 weeks [1][3][12] - A 28.6% reduction in muscle fibrosis was observed between weeks 24 and 48 (p<0.01), alongside a 50% decrease in serum creatine kinase (CK) levels (p<0.001) [3][12] - Dystrophin expression stabilized between 24 and 48 weeks, averaging 7.8%, with 88% of boys achieving over 5% average dystrophin [1][8] Group 2: Safety and Tolerability - WVE-N531 was reported to be safe and well-tolerated, with no serious adverse events and all treatment-related adverse events being mild to moderate [1][8] - The trial included 11 boys, all of whom advanced to the extension portion of the study receiving monthly doses of WVE-N531 [7][8] Group 3: Regulatory and Future Plans - Following feedback from the FDA, Wave plans to file a New Drug Application (NDA) in 2026 for accelerated approval of WVE-N531, supported by data for monthly dosing [1][5][6] - Wave expects to submit multiple clinical trial applications (CTAs) for other DMD candidates targeting different exons in 2026, aiming to establish a best-in-class exon skipping franchise [1][6][10] Group 4: Market Opportunity - WVE-N531 and other exon skipping programs could address approximately 40% of the DMD population, representing a market opportunity exceeding $2.4 billion in the United States alone [6][10]

Wave Life Sciences Ltd. (NASDAQ:WVE) Q4 2024 Earnings Conference Call March 4, 2025 8:30 AM ET Company Participants Kate Rausch - VP, IR & Corporate Affairs Paul Bolno - President & CEO Erik Ingelsson - Chief Scientific Officer Kyle Moran - Chief Financial Officer Conference Call Participants Ron Feiner - JPMorgan Salim Syed - Mizuho Joon Lee - Truist Securities Joe Schwartz - Leerink Partners Roger Song - Jefferies Catherine Novack - Jones Trading Ryan Deschner - Raymond James Madison El-Saadi - B. Riley O ...