Mean muscle content-adjusted dystrophin expression of 9.0% and unadjusted dystrophin of 5.5%, with high consistency across participants, in a prespecified analysis; dystrophin was comprised of two isoforms consistent with Becker muscular dystrophy patients who display milder disease Data demonstrated meaningful improvement in serum biomarkers for muscle health, with localization of WVEN531 in myogenic stem cells and regeneration of myofibers Skeletal muscle concentrations of ~41,000 ng/g combined with 61-da ...

Wave Life Sciences (WVE) came out with a quarterly loss of $0.25 per share versus the Zacks Consensus Estimate of a loss of $0.16. This compares to loss of $0.20 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -56.25%. A quarter ago, it was expected that this biopharmaceutical company would post a loss of $0.18 per share when it actually produced a loss of $0.24, delivering a surprise of -33.33%. Over the last four quarters, ...

Clinical Development - The company initiated the RestorAATion clinical program for WVE-006 in Q4 2023, targeting Alpha-1 antitrypsin deficiency (AATD) with a focus on restoring wild-type AAT protein levels in serum [51]. - WVE-006 achieved serum AAT protein levels of up to 30 micromolar in preclinical models, restoring approximately 50% wild-type M-AAT protein and demonstrating a 3-fold increase in neutrophil elastase inhibition activity [53]. - The company is advancing WVE-007, a GalNAc-siRNA targeting INHBE for obesity, with preclinical data showing significant weight loss and fat reduction in diet-induced obesity models [54]. - WVE-N531 is designed to skip exon 53 in the dystrophin gene, addressing approximately 8-10% of Duchenne muscular dystrophy (DMD) cases, with positive proof-of-concept data from early trials [56]. - The Phase 2 trial for WVE-N531 (FORWARD-53) has been initiated, with results expected in Q3 2024, focusing on dystrophin protein levels and safety [58]. - WVE-003 is being developed for Huntington's disease, targeting a specific SNP associated with the disease, with the potential to address up to 80% of the HD population in future [59]. - The SELECT-HD trial showed a statistically significant reduction of up to 46% in cerebrospinal fluid mHTT levels with WVE-003, indicating potential benefits for early-stage HD patients [63]. Financial Performance - For the three months ended June 30, 2024, the company reported a net loss of $32.9 million, compared to a net loss of $21.1 million for the same period in 2023, reflecting an increase of $11.8 million [67]. - Total operating expenses for Q2 2024 were $54.7 million, up from $45.6 million in Q2 2023, marking an increase of $9.1 million [77]. - Research and development expenses rose to $40.4 million in Q2 2024, compared to $33.3 million in Q2 2023, an increase of approximately $7.1 million [82]. - The company has an accumulated deficit of $1,089.3 million as of June 30, 2024, up from $1,024.9 million at the end of 2023 [67]. - Revenue for the six months ended June 30, 2024, was $32.2 million, a decrease of approximately 8.1% from $35.0 million in the same period of 2023 [84]. - Total operating expenses increased to $101.7 million in the first half of 2024, up from $88.8 million in 2023, representing a rise of approximately 14.5% [84]. - Research and development expenses rose to $73.8 million in the first half of 2024, an increase of about 14.8% compared to $64.3 million in 2023 [84]. - General and administrative expenses were $27.8 million for the six months ended June 30, 2024, up from $24.5 million in 2023, reflecting an increase of approximately 13.5% [85]. - Net loss for the six months ended June 30, 2024, was $64.5 million, compared to a net loss of $48.5 million in 2023, indicating an increase in loss of approximately 33.0% [84]. - Cash and cash equivalents totaled $154.0 million as of June 30, 2024, with an accumulated deficit of $1,089.3 million [88]. - Net cash used in operating activities was $60.9 million for the six months ended June 30, 2024, compared to a net cash provided of $48.3 million in 2023 [89]. - Net cash provided by financing activities was $15.2 million in the first half of 2024, down from $36.9 million in the same period of 2023 [92]. Collaborations and Partnerships - The collaboration with GlaxoSmithKline (GSK) includes potential milestone payments of up to $525 million and double-digit tiered royalties for WVE-006, with a $20 million milestone payment already achieved [52]. - GSK selected two programs for development candidates, triggering a $12 million initiation payment [66]. - The GSK collaboration agreement generated revenue of $19.7 million in Q2 2024, a decrease from $22.1 million in Q2 2023 [78]. Future Outlook and Risks - The company expects to continue incurring significant operating losses and expenses in the foreseeable future [67]. - The company has not generated any product revenue since inception and does not expect to do so in the near future [68]. - The company anticipates continued significant expenses related to ongoing research and development activities and internal manufacturing [93]. - The company may face delays and issues in drug candidate development due to various risks, including regulatory challenges and safety issues [95]. - Future capital requirements for therapeutic programs will depend on research progress, costs, and the number of product candidates pursued [95]. - The company currently has no committed external funding sources, relying on potential future payments from collaborations with Takeda and GSK [95]. - The company is exposed to interest rate risk due to fluctuations in interest rates affecting cash and cash equivalents [97]. - Foreign currency exchange rate changes could materially impact the company's financial condition, although no significant effects were noted in the past two years [98]. - Inflation trends may lead to increased clinical trial, labor, and operating costs in the future [99]. - The company has no product revenues and relies on funds raised through equity offerings, which are influenced by capital market conditions [100]. Research and Development Strategy - The company maintains a robust pipeline of RNA-targeting modalities, leveraging novel chemistry to enhance the pharmacological properties of oligonucleotides [46]. - The company’s RNA medicines platform, PRISM®, combines multiple modalities and deep insights into human genetics to address both rare and prevalent disorders [46]. - The strategic focus on oligonucleotides allows the company to avoid risks associated with DNA editing and gene therapy, while leveraging established manufacturing processes [47].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______ to ______ Commission File Number: 001-37627 WAVE LIFE SCIENCES LTD. (Exact name of registrant as specified in its charter) Singapore (State or other juris ...

Exhibit 99.1 Wave Life Sciences Reports First Quarter 2024 Financial Results and Provides Business Update RestorAATion-2 clinical trial of WVE-006 in AATD patients underway; expected proof-of-mechanism data in patients with AATD remains on track for 2024 INHBE program for obesity expected clinical trial initiation in 1Q 2025; preclinical data demonstrate weight loss similar to semaglutide, fat loss with no loss of muscle mass, and curtailed rebound weight gain upon cessation of semaglutide, with potential f ...

Wave Life Sciences Ltd. (NASDAQ:WVE) Q4 2023 Earnings Call Transcript March 6, 2024 8:30 AM ET Company Participants Kate Rausch - Vice President, Investor Relations & Corporate Affairs Paul Bolno - President & Chief Executive Officer Anne-Marie Li-Kwai-Cheung - Chief Development Officer Kyle Moran - Chief Financial Officer Conference Call Participants Timur Ivannikov - Raymond James Salim Syed - Mizuho Joon Lee - Truist Joseph Schwartz - Leerink Partners Luca Issi - RBC Capital Andrew Fein - H.C. Wainwright ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File No. 001-37627 WAVE LIFE SCIENCES LTD. (Exact name of registrant as specified in its charter) Singapore 98-1356880 (State or other jurisdiction of i ...

Financial Performance - Revenue for the fourth quarter of 2023 was $29.1 million, a significant increase from $1.2 million in the prior year quarter, with total revenue for 2023 reaching $113.3 million compared to $3.6 million in 2022[13] - Total revenue for Q4 2023 reached $29,056 thousand, a significant increase from $1,239 thousand in Q4 2022, representing a growth of 2,241%[18] - For the full year 2023, revenue was $113,305 thousand, up from $3,649 thousand in 2022, marking a growth of 3,008%[18] - The net loss for Q4 2023 was $16.3 million, significantly improved from a net loss of $43.7 million in the prior year quarter, with a total net loss of $57.5 million for 2023 compared to $161.8 million in 2022[13] - The net loss for Q4 2023 was $16,256 thousand, an improvement from a net loss of $43,709 thousand in Q4 2022, indicating a reduction of 63%[18] - The comprehensive loss for the full year 2023 was $57,608 thousand, a decrease from $162,033 thousand in 2022, showing a reduction of 64%[18] Research and Development - Research and development expenses were $34.1 million in Q4 2023, up from $31.1 million in Q4 2022, with total R&D expenses for 2023 at $130.0 million compared to $115.9 million in 2022[13] - Research and development expenses for the full year 2023 totaled $130,009 thousand, up from $115,856 thousand in 2022, reflecting a 12% increase[18] - Wave anticipates delivering proof-of-mechanism data for WVE-006 in AATD in 2024, which would unlock its pipeline of RNA editing candidates[6] - The FORWARD-53 trial for WVE-N531 in DMD is on track to deliver data, including dystrophin protein expression, in the third quarter of 2024, with industry-leading exon skipping levels of 53% reported[9] - WVE-003 has demonstrated a 35% reduction in mean mutant HTT in cerebrospinal fluid compared to placebo, with ongoing multi-dose evaluations expected to report data in Q2 2024[9] - The company expects to initiate a clinical trial for its INHBE candidate in the first quarter of 2025, with a potentially best-in-class profile for obesity[9] Cash and Assets - Cash and cash equivalents were $200.4 million as of December 31, 2023, compared to $88.5 million as of December 31, 2022, primarily due to $170.0 million received from the GSK collaboration and $93.6 million in net proceeds from the December 2023 offering[13] - Wave's current cash and cash equivalents are expected to fund operations into the fourth quarter of 2025, excluding potential future milestone payments[13] - The total assets as of December 31, 2023, were $274,949 thousand, compared to $146,386 thousand as of December 31, 2022, an increase of 87%[16] Liabilities and Equity - Current liabilities increased to $186,440 thousand in 2023 from $71,521 thousand in 2022, a rise of 161%[16] - Shareholders' equity improved to $39,630 thousand in 2023 from a deficit of $45,091 thousand in 2022, indicating a positive shift[16] Milestones and Payments - The company received a $20 million milestone payment from GSK in Q4 2023 following the initiation of dosing in the RestorAATion-1 clinical trial[6] - The company reported a weighted-average of 109,627,549 ordinary shares used in computing net loss per share for Q4 2023, compared to 93,993,638 in Q4 2022[18]

Wave Life Sciences Ltd. (NASDAQ:WVE) Q3 2023 Earnings Conference Call November 9, 2023 8:30 AM ET Company Participants Kate Rausch - Vice President, Investor Relations & Corporate Affairs Paul Bolno - President & Chief Executive Officer Anne-Marie Li-Kwai-Cheung - Chief Development Officer Kyle Moran - Chief Financial Officer Conference Call Participants Steve Seedhouse - Raymond James Salim Syed - Mizuho Joseph Schwartz - Leerink Partners Eun Yang - Jefferies Operator Good morning and welcome to Wave Life ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______ to ______ Singapore (State or other jurisdiction of incorporation or organization) 7 Straits View #12-00, Marina One East Tower Singapore Commission F ...