Latest news with #WVE-007
Yahoo
15-07-2025
- Business
- Yahoo
Wave Life Sciences Unveils Promising Preclinical Data for Obesity Treatment WVE-007 at ADA Scientific Sessions
Wave Life Sciences Ltd. (NASDAQ:WVE) is one of the best low priced pharma stocks to buy now. From June 20 to 23, Wave Life Sciences presented preclinical data for WVE-007, which is its investigational GalNAc-siRNA targeting INHBE mRNA, at the American Diabetes Association's 85th Annual Scientific Sessions in Chicago. The oral presentation was delivered on June 21 and highlighted WVE-007 as a potential novel and distinct obesity treatment designed to induce healthy weight loss by reducing fat while preserving muscle mass. WVE-007 functions by silencing INHBE mRNA, which in turn reduces the production of Activin E protein. Activin E is a lipolysis suppressor that is upregulated in obesity. By inhibiting Activin E, WVE-007 promotes lipolysis, or fat breakdown. A biotechnology laboratory filled with computers and equipment to support research. Human genetics provides strong support for INHBE as a therapeutic target. Individuals with a protective loss-of-function variant in one copy of the INHBE gene exhibit a healthier cardiometabolic profile, which includes less abdominal fat, lower triglycerides, and reduced risk of type 2 diabetes/T2D and cardiovascular disease/CAD. These individuals also show favorable associations with liver traits, such as reductions in cT1 (reflecting liver inflammation and fibrosis) and ALT (reflecting liver damage), without impacting liver fat. Wave Life Sciences Ltd. (NASDAQ:WVE) is a clinical-stage biotechnology company that designs, develops, and commercializes ribonucleic acid/RNA medicines through PRISM, which is a discovery and drug development platform. While we acknowledge the potential of WVE as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey. Error while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Yahoo
03-07-2025
- Business
- Yahoo
H.C. Wainwright Reaffirms Buy Rating on Wave Life Sciences After ADA Data Release
Wave Life Sciences Ltd. (NASDAQ:WVE) ranks among the best CRISPR stocks to buy. On June 23, H.C. Wainwright maintained its $22 price target and Buy rating on Wave Life Sciences Ltd. (NASDAQ:WVE). The rating, in line with the strong analyst consensus on the company, follows new preclinical evidence that was presented at the 85th ADA meeting in Chicago. The firm cited data from WAVE's WVE-007 program that showed that in diet-induced obese mice, lowering INHBE mRNA with a single subcutaneous injection of INHBE-03 led to considerable weight loss. More specifically, the preclinical findings showed that the only way treated mice lost weight was by losing fat mass in their epididymal white adipose tissue; in contrast, the quadricep muscle mass, which is a crucial therapeutic differentiator, was constant. Wave Life Sciences Ltd. (NASDAQ:WVE) is a clinical-stage biotechnology company that employs PRISM, a platform for drug research and discovery, to create and market RNA (ribonucleic acid) therapies. While we acknowledge the potential of WVE as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. Read More: and Disclosure: None.
Business Upturn
20-06-2025
- Health
- Business Upturn
Wave Life Sciences Announces Oral Presentation of Preclinical Data Supporting WVE-007's Mechanism (INHBE) to Reduce Fat, Preserve Muscle, and Induce Healthy Weight Loss at ADA's Annual Scientific Sessions
Presentation to highlight WVE-007 (INHBE GalNAc-siRNA) as a potential novel and unique obesity treatment leading to healthy weight loss and supporting preclinical data demonstrating potent and durable reduction in Activin E resulting in fat loss with muscle preservation New preclinical data demonstrate that a single dose of INHBE siRNA leads to lower inflammation of adipose tissue with strong suppression of pro-inflammatory M1 macrophages in visceral fat in DIO mice, highlighting potential mechanistic insights into the risk reduction for type 2 diabetes (T2D) and coronary artery disease (CAD) suggested by human genetics CAMBRIDGE, Mass., June 20, 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, today announced the presentation of preclinical data supporting WVE-007, its GalNAc-siRNA designed to silence INHBE mRNA, an obesity target with strong evidence from human genetics. The data demonstrate the ability of Wave's long-acting GalNAc-siRNA to reduce INHBE mRNA and Activin E protein, induce weight loss mainly through reduction of fat mass, and reduce pro-inflammatory macrophage recruitment in a diet-induced obese (DIO) mouse model. The data were highlighted today in an oral presentation at the American Diabetes Association's 85th Annual Scientific Sessions, taking place June 20 to 23, in Chicago. 'These exciting preclinical data highlighted in today's presentation both recapitulate human genetics findings and continue to support the potential of WVE-007 to drive weight reduction by reducing Activin E to induce lipolysis – or fat breakdown, preferentially reducing visceral fat, and decreasing inflammation of adipose tissue – all without impacting lean muscle mass. These data suggest a highly differentiated therapeutic profile with lower visceral fat, less insulin resistance and less inflammation, supporting potential for risk reduction of T2D, CAD and other obesity-related co-morbidities,' said Erik Ingelsson, MD, PhD, Chief Scientific Officer. 'Silencing of INHBE is an entirely orthogonal mechanism from GLP-1s, which are centrally acting and impact the digestive system and central nervous system to decrease appetite. If these preclinical data translate in the clinic, WVE-007 has the potential to transform the obesity treatment paradigm by delivering healthy weight loss, preservation of muscle mass, and infrequent dosing of once or twice a year. We are evaluating WVE-007 in our ongoing INLIGHT clinical trial in adults living with overweight or obesity, and we are on track to deliver the first clinical data in the second half of this year.' Human genetics provides strong evidence for INHBE as a therapeutic target. Individuals who have a protective loss-of-function variant in one copy of the INHBE gene have a healthier cardiometabolic profile, including less abdominal fat, lower triglycerides, and lower risk of type 2 diabetes and cardiovascular disease. These heterozygous carriers also exhibit favorable associations with liver traits, including reductions in cT1 (reflecting liver inflammation and fibrosis) and ALT (reflecting liver damage), with no impact on liver fat. The oral presentation titled, 'siRNA-INHBE Silencing in Mice Recapitulates Human Genetic Data and Demonstrates Improved Healthy Weight Loss Profile,' highlighted results from studies in DIO mice that evaluated monotherapy (INHBE-siRNA alone) as well as combination (INHBE siRNA and semaglutide), and maintenance (INHBE siRNA when semaglutide treatment is discontinued) treatment settings. Key results are as follows: A single dose of INHBE-siRNA led to robust target engagement, including reduction of INHBE mRNA and Activin E protein, a lipolysis suppressor that is upregulated in obesity. Liver INHBE mRNA was strongly correlated with serum Activin E levels following INHBE-siRNA treatment. A single dose of INHBE-siRNA led to weight loss on par with semaglutide. There was a decrease in diet-induced visceral adipose mass and shrinkage of adipocytes compared with PBS treatment, supporting the restoration of healthy adipose tissue with this mechanism of action. Muscle mass was preserved. Infiltration of activated macrophages in visceral adipose was significantly decreased by a single dose of INHBE-siRNA compared with PBS controls. INHBE-siRNA also significantly reduced proinflammatory M1 macrophage (CD11c positive) recruitment while sustaining levels of anti-inflammatory M2 macrophages in visceral fat, indicating an overall shift away from a pro-inflammatory state. When administered as an add-on to semaglutide, a single dose of INHBE-siRNA doubled the amount of weight loss, highlighting potential for combination treatment. Wave's INHBE siRNA curtailed rebound weight gain when semaglutide treatment was discontinued, highlighting its potential as an off-ramp and maintenance treatment following GLP-1 treatment. The full presentation can be accessed by visiting 'Scientific Presentations' on the Investors section of the Wave Life Sciences website: About Wave Life Sciences Wave Life Sciences (Nasdaq: WVE) is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave's RNA medicines platform, PRISM®, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and common disorders. Its toolkit of RNA-targeting modalities includes editing, splicing, RNA interference and antisense silencing, providing Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave's diversified pipeline includes clinical programs in Alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, Huntington's disease, and Obesity, as well as several preclinical programs utilizing the company's broad RNA therapeutics toolkit. Driven by the calling to 'Reimagine Possible', Wave is leading the charge toward a world in which human potential is no longer hindered by the burden of disease. Wave is headquartered in Cambridge, MA. For more information on Wave's science, pipeline and people, please visit and follow Wave on X (formerly Twitter) and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding our expectations for WVE-007 and the anticipated therapeutic benefits thereof; the anticipated timing of clinical data from our INLIGHT clinical trial of WVE-007; the novelty of our approach to silence INHBE mRNA in order to achieve healthy, sustainable weight loss and the potential for once- or twice-yearly dosing; the potential benefits of WVE-007 over existing obesity therapies; the potential of WVE-007's mechanism (INHBE) as a novel and unique obesity treatment to induce fat loss, preserve muscle, and drive weight loss; our understanding of our preclinical data for WVE-007 and our expectations of how such data will translate in humans; beliefs that Wave's portfolio of RNA medicines is differentiated, potentially best-in-class and potentially transformative; the broad potential of Wave's RNA medicines pipeline and oligonucleotide chemistry and any benefits that may arise as a result thereof. The words 'may,' 'will,' 'could,' 'would,' 'should,' 'expect,' 'plan,' 'anticipate,' 'intend,' 'believe,' 'estimate,' 'predict,' 'project,' 'potential,' 'continue,' 'target' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release and actual results may differ materially from those indicated by these forward-looking statements as a result of these risks, uncertainties and important factors, including, without limitation, the risks and uncertainties described in the section entitled 'Risk Factors' in Wave's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as amended, and in other filings Wave makes with the SEC from time to time. Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances. Contact:Kate RauschVP, Corporate Affairs and Investor Relations +1 617-949-4827
Business Insider
11-06-2025
- Business
- Business Insider
Wave Life Sciences assumed with an Outperform at Raymond James
Raymond James assumed coverage of Wave Life Sciences (WVE) with an Outperform rating and $14 price target as part of a broader research note on selection Biotech names. Risk/reward skews favorably across the sector, though while companies with high PoS – probability of success – lead assets allow for higher conviction, highly attractive skews on less de-risked assets may deliver the most outsized returns, the analyst tells investors in a research note. For the company, the firm is positive on the risk-reward opportunity ahead of second-half catalysts for its two most important clinical-stage programs – WVE-007 in obesity, which features a novel mechanism with differentiating qualities, and WVE-006 in AATD, with second half updates that will include larger cohorts at repeat and higher doses, Raymond James added. Confident Investing Starts Here:
Yahoo
08-05-2025
- Business
- Yahoo
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 multidose and single dose cohorts expected in 3Q 2025; data from complete 400 mg single dose cohort expected in the fall of 2025 Delivered positive data from FORWARD-53 clinical trial of WVE-N531 in exon 53 amenable DMD including statistically significant and clinically meaningful improvement in TTR, substantial improvements in muscle health; NDA submission for accelerated approval with monthly dosing planned for 2026 IND submission expected 2H 2025 for potentially registrational WVE-003 Phase 2/3 study in HD with caudate atrophy as a primary endpoint Cash and cash equivalents of $243.1 million as of March 31, 2025, with runway expected into 2027 Investor conference call and webcast at 8:30 a.m. ET today CAMBRIDGE, Mass., May 08, 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, today announced financial results for the first quarter ended March 31, 2025, and provided a business update. 'We've continued our consistent execution across modalities as we completed dosing in the first two cohorts of our INLIGHT trial in obesity, advanced our RestorAATion-2 trial in AATD, and delivered positive data from our FORWARD-53 clinical trial in DMD. We are on track to deliver multiple clinical datasets this year that will further demonstrate our broad capabilities across modalities and our leadership in RNA medicines,' said Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences. 'Our RestorAATion-2 trial of WVE-006, a subcutaneously-dosed GalNAc-conjugated RNA editing oligonucleotide, continues to advance and we are on track to deliver data from multiple dose cohorts this year, which will inform the therapeutic potential of WVE-006 and our pipeline of wholly-owned GalNAc-RNA editing programs. In obesity, we are evaluating WVE-007, our INHBE GalNAc-siRNA in our ongoing INLIGHT clinical trial, and are on track to deliver the first clinical data in the second half of this year. This program has potential to transform the obesity treatment paradigm with healthy weight loss, preservation of muscle mass, and infrequent dosing of once or twice a year.' Dr. Bolno continued, 'In DMD, we delivered the first-ever substantial improvements in muscle health with an exon skipping therapy and showed statistically significant and clinically meaningful functional data from our FORWARD-53 trial of WVE-N531 in March. We have been engaged with the community in discussing our recent clinical results and are excited by the potential to bring a meaningful new potential treatment option to boys with DMD. In HD, our WVE-003 program has industry-leading CSF mutant lowering, and remains the only program to have successfully demonstrated allele-selective knockdown with wild-type HTT preservation in the clinic. We are actively engaged with both the HD community and prospective strategic partners, as we continue to prepare for our potentially registrational Phase 2/3 study.' Recent Business Highlights and Expected Milestones Obesity WVE-007 is a GalNAc-conjugated small interfering RNA (GalNAc-siRNA) designed to silence INHBE mRNA, an obesity target with strong evidence from human genetics. WVE-007 is Wave's first siRNA candidate to enter clinical development and uses Wave's best-in-class proprietary oligonucleotide chemistry. INLIGHT is an ongoing, first-in-human, placebo-controlled, clinical trial evaluating WVE-007 in adults living with overweight or obesity and assesses safety, tolerability, pharmacokinetics, biomarkers for target engagement, body weight and composition, and metabolic health. Today, Wave announced that it has completed dosing in the first and second single dose cohorts of INLIGHT. Next week, in oral presentations at the 32nd European Congress on Obesity (ECO) and the American Society of Gene and Cell Therapies (ASGCT) 28th Annual Meeting, Wave will highlight its preclinical data supporting WVE-007's potential in multiple treatment settings with potential for dosing once or twice a year, including: A single dose of Wave's INHBE siRNA led to weight loss on par with semaglutide, but with no muscle loss. When administered as an add-on to semaglutide, a single dose of Wave's INHBE siRNA doubled the amount of weight loss. Wave's INHBE siRNA curtailed rebound weight gain when semaglutide treatment was discontinued, highlighting its potential as an off-ramp and maintenance treatment following GLP-1 treatment. Expected milestones: Wave expects to deliver clinical data from INLIGHT in the second half of 2025, including safety, tolerability and biomarkers reflective of healthy weight loss. AATD (Alpha-1 antitrypsin deficiency) WVE-006 is a GalNAc-conjugated, subcutaneously delivered, A-to-I RNA editing oligonucleotide (AIMer) that is uniquely designed to address alpha-1 antitrypsin deficiency (AATD)-related lung disease, liver disease, or both. RestorAATion clinical program: Multi-dosing is complete in RestorAATion-1 (healthy volunteers) at a dose level greater than those planned for any cohort in its ongoing RestorAATion-2 study. RestorAATion-2 is a Phase 1b/2a open-label study with both single and multiple ascending dose portions, which is evaluating the safety, tolerability, pharmacodynamics and pharmacokinetics of WVE-006 in individuals with AATD who have the homozygous Pi*ZZ mutation. Multi-dosing is ongoing in the first cohort of RestorAATion-2, where patients are receiving 200 mg subcutaneous doses every two weeks. Dosing is also underway in the second single dose cohort at 400 mg. In October 2024, Wave delivered proof-of-mechanism data from a single, lowest dose of WVE-006 from the first two patients in the ongoing RestorAATion-2 clinical study, representing the first-ever clinical demonstration of RNA editing in humans. Circulating wild-type M-AAT protein in plasma reached a mean of 6.9 micromolar, representing more than 60% of total AAT. Mean total AAT protein increased to 10.8 micromolar, meeting the level that has been the basis for regulatory approval for AAT augmentation therapies. Expected milestones: Wave expects to share data from the complete 200 mg multidose and single dose cohorts of RestorAATion-2 in the third quarter of 2025, and data from the complete 400 mg single dose cohort in the fall of 2025. Emerging wholly owned siRNA and RNA editing pipeline Wave is advancing new targets across multiple disease areas to expand its pipeline of wholly owned programs in both rare and common diseases. Wave's pipeline of preclinical candidates utilize Wave's proprietary chemistry to achieve best-in-class silencing using siRNA and RNA editing in a variety of hepatic and extrahepatic tissues, including in the CNS with multiple AIMers such as MECP2. Within RNA editing, Wave has demonstrated the ability to correct single variants to restore wild-type protein function and to increase the stability of the mRNA transcript to upregulate protein levels. Wave's wholly owned RNA editing pipeline includes programs that use GalNAc conjugation and have efficient clinical paths to proof-of-concept. These include PNPLA3 mRNA correction to potentially address the nine million homozygous I148M carriers in the US and Europe at risk for a variety of liver diseases, and mRNA upregulation (LDLR) and mRNA correction (APOB), which together would address approximately one million people living with heterozygous familial hypercholesterolemia (HeFH) in the US and Europe. Next week, in an oral presentation at the ASGCT 28th Annual Meeting, Wave plans to share preclinical data demonstrating proof-of-principle for the use of AIMers in lung indications, including cystic fibrosis (CF). Available therapies for CF cannot address stop codon mutations in the CFTR gene. In human bronchial epithelial cells with CFTR mutation W1282X, CFTR AIMers increased expression of CFTR mRNA 3-fold and restored up to 50% of functional wild-type CFTR protein levels. Expected milestones: Wave plans to share new preclinical data from hepatic and extra-hepatic RNA editing programs in 2025 and to initiate clinical development of additional RNA editing programs, including PNPLA3, LDLR, and APOB, in 2026. DMD (Duchenne muscular dystrophy) WVE-N531 is an exon skipping oligonucleotide being developed as a disease modifying treatment for boys with Duchenne muscular dystrophy amenable to exon 53 skipping. WVE-N531 was designed using Wave's best-in-class oligonucleotide chemistry modifications, including PN backbone chemistry. WVE-N531 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food & Drug Administration. In March 2025, Wave announced positive 48-week data from its Phase 2, open-label FORWARD-53 clinical trial of WVE-N531, which included: Statistically significant and clinically meaningful improvement of 3.8 seconds in Time-to-Rise vs. natural history with largest effect observed relative to any approved dystrophin restoration therapy at 48 weeks; additional functional benefits observed in other outcome measures including NSAA. First-ever demonstration of substantial improvements in muscle health with exon skipping – statistically significant reduction in fibrosis driven by decreases in inflammation and necrosis, coupled with transition from regenerative to mature muscle; decreases in creatine kinase and circulating inflammatory biomarkers. Dystrophin expression stabilized between 24 and 48 weeks and averaged 7.8%, with 88% of boys above 5% average dystrophin. WVE-N531 remains safe and well-tolerated with no Serious Adverse Events. All participants in FORWARD-53 elected to advance to the extension portion of the clinical trial, which is currently ongoing with boys receiving monthly doses of WVE-N531. To augment monthly data and ensure a monthly regimen at a potential launch, Wave is also expanding FORWARD-53 to include additional boys who will be dosed monthly. Also in March 2025, Wave announced that the company met with the U.S. Food and Drug Administration (FDA) on WVE-N531 to discuss its interim 24-week data and initial plans for the confirmatory trial, where the Agency confirmed that the accelerated approval pathway using dystrophin expression as a surrogate endpoint remains open. Expected milestones: Wave plans to file a New Drug Application (NDA) in 2026 to support accelerated approval of WVE-N531 with monthly dosing. Wave expects to submit clinical trial applications (CTAs) for additional exon skipping programs in 2026. HD (Huntington's disease) WVE-003 is a first-in-class, allele-selective oligonucleotide for the treatment of Huntington's disease (HD). In the SELECT-HD clinical trial, data demonstrated the first-ever allele-selective reduction in CSF mHTT protein and preservation of healthy, wtHTT with multiple doses of WVE-003, as well as a statistically significant correlation between mHTT reduction and slowing of caudate atrophy. By reducing mHTT at the mRNA and protein level, WVE-003 addresses underlying drivers of neurodegeneration. In addition, by sparing wtHTT protein, which is critical to the health of the central nervous system, WVE-003 is uniquely positioned to address presymptomatic HD patients, as well as symptomatic patients. Wave has received supportive initial feedback from FDA, who recognize the severity of HD and are receptive to and engaged with Wave regarding a potential pathway to accelerated approval. Preparation is ongoing for a potentially registrational, global Phase 2/3 study of WVE-003 in adults with SNP3 and HD using caudate atrophy as a primary endpoint. Expected milestones: Wave expects to submit an Investigational New Drug (IND) application for a potentially registrational Phase 2/3 study of WVE-003 in HD in the second half of 2025. Financial Highlights Cash and cash equivalents were $243.1 million as of March 31, 2025, compared to $302.1 million as of December 31, 2024. Wave expects that its current cash and cash equivalents will be sufficient to fund operations into 2027. Potential future milestones and other payments to Wave under its GSK collaboration are not included in its cash runway. Revenue recognized was $9.2 million for the first quarter of 2025 as compared to $12.5 million in the prior year quarter. Research and development expenses were $40.6 million in the first quarter of 2025 as compared to $33.4 million in the same period in 2024. General and administrative expenses were $18.4 million in the first quarter 2025 as compared to $13.5 million in the same period in 2024. Net loss was $46.9 million for the first quarter of 2025 as compared to $31.6 million in the prior year quarter. Investor Conference Call and WebcastWave will host an investor conference call today at 8:30 a.m. ET to review the first quarter 2025 financial results and pipeline updates. A webcast of the conference call can be accessed by visiting 'Investor Events' on the investor relations section of the Wave Life Sciences website: Analysts planning to participate during the Q&A portion of the live call can join the conference call by dialing (833) 630-1956 (domestic) or (412) 317-1837 (international). Following the live event, an archived version of the webcast will be available on the Wave Life Sciences website. About Wave Life SciencesWave Life Sciences (Nasdaq: WVE) is a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health. Wave's RNA medicines platform, PRISM®, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and common disorders. Its toolkit of RNA-targeting modalities includes editing, splicing, RNA interference and antisense silencing, providing Wave with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology. Wave's diversified pipeline includes clinical programs in Alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, Huntington's disease, and Obesity, as well as several preclinical programs utilizing the company's broad RNA therapeutics toolkit. Driven by the calling to 'Reimagine Possible', Wave is leading the charge toward a world in which human potential is no longer hindered by the burden of disease. Wave is headquartered in Cambridge, MA. For more information on Wave's science, pipeline and people, please visit and follow Wave on X (formerly Twitter) and LinkedIn. Forward-Looking StatementsThis press release contains forward-looking statements concerning our goals, beliefs, expectations, strategies, objectives and plans, and other statements that are not necessarily based on historical facts, including statements regarding the following, among others: the anticipated initiation, site activation, patient recruitment, patient enrollment, dosing, generation and reporting of data and completion of our clinical trials, including interactions with regulators and any potential registration based on these data, and the timing and announcement of such events; the protocol, design, endpoints, dose levels and dosing frequency for our investigational therapeutics in clinical trials; the future performance and results of our programs in clinical trials; our expectations with respect to how our clinical data successes to date may predict success for our future therapeutic candidates and data readouts and may further validate our platform; preclinical activities and programs and their potential to transition into clinical-stage programs; the potential of our preclinical data to predict the behavior of our compounds in humans; regulatory submissions and timing for regulatory feedback; the submission of marketing approval applications to regulators, approval thereof, and the potential commercialization of our late-stage programs; the progress and potential benefits of collaborations and strategic partnerships; the potential achievement of milestones under any collaborations; our identification of future product candidates and their therapeutic potential; the anticipated benefits of our therapeutic candidates and pipeline compared to our competitors; the potential unmet medical needs and addressable patient population estimates related to our therapeutic candidates; our ability to design compounds using the most appropriate of our multiple modalities and the anticipated benefits of that approach; the breadth and versatility of our drug discovery and development platform; the expected benefits of our stereopure oligonucleotides compared with stereorandom oligonucleotides; the potential benefits of our RNA editing capability, including our AIMers, compared to others; the potential for certain of our programs to be best-in-class or first-in-class or to change the existing treatment paradigm or show substantial benefits over existing standards of care; the status and progress of our programs relative to potential competitors; anticipated benefits of our proprietary manufacturing processes and our internal manufacturing capabilities; the benefits of RNA medicines generally; the strength of our intellectual property and the data that support our IP; the anticipated duration of our cash runway and our ability to fund future operations; our intended uses of capital; and our expectations regarding the impact of any potential global macro events on our business. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including the following: our ability to finance our drug discovery and development efforts and to raise additional capital when needed; the ability of our preclinical programs to produce data sufficient to support our clinical trial applications and the timing thereof; the clinical results of our programs and the timing thereof, which may not support further development of our product candidates; actions of regulatory authorities and their receptiveness to our trial designs and accelerated approval pathways, which may affect the initiation, timing and progress of clinical trials; our effectiveness in managing interactions with regulatory authorities; the effectiveness of our drug discovery and development platform; the effectiveness of our RNA editing capability and our AIMers; our ability to demonstrate the therapeutic benefits of our candidates in clinical trials, including our ability to develop candidates across multiple therapeutic modalities; our dependence on third parties, including contract research organizations, contract manufacturing organizations, collaborators and partners; our ability to manufacture or contract with third parties to manufacture drug material to support our programs and growth; our ability to obtain, maintain and protect our intellectual property; our ability to enforce our patents against infringers and defend our patent portfolio against challenges from third parties; competition from others developing therapies for the indications we are pursuing; our ability to maintain the company infrastructure and personnel needed to achieve our goals; and the information under the caption 'Risk Factors' contained in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) and in other filings we make with the SEC from time to time. We undertake no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances. Contact:Kate RauschVP, Corporate Affairs and Investor Relations+1 617-949-4827 Investors:InvestorRelations@ Media:MediaRelations@ WAVE LIFE SCIENCES CONSOLIDATED BALANCE SHEETS(In thousands, except share amounts) March 31, 2025 December 31, 2024 Assets Current assets: Cash and cash equivalents $ 243,075 $ 302,078 Accounts receivable — 1,422 Prepaid expenses 8,062 9,544 Other current assets 6,839 7,350 Total current assets 257,976 320,394 Long-term assets: Property and equipment, net of accumulated depreciation of $47,027 and $46,329 as of March 31, 2025 and December 31, 2024, respectively 9,566 10,128 Operating lease right-of-use assets 16,581 17,870 Restricted cash 3,772 3,760 Other assets 448 55 Total long-term assets 30,367 31,813 Total assets $ 288,343 $ 352,207 Liabilities, Series A preferred shares, and shareholders' equity Current liabilities: Accounts payable $ 14,358 $ 16,262 Accrued expenses and other current liabilities 7,813 21,081 Current portion of deferred revenue 57,312 65,972 Current portion of operating lease liability 7,884 7,638 Total current liabilities 87,367 110,953 Long-term liabilities: Deferred revenue, net of current portion 5,584 6,099 Operating lease liability, net of current portion 15,715 17,766 Total long-term liabilities 21,299 23,865 Total liabilities $ 108,666 $ 134,818 Series A preferred shares, no par value; 3,901,348 shares issued and outstanding at March 31, 2025 and December 31, 2024 $ 7,874 $ 7,874 Shareholders' equity: Ordinary shares, no par value; 154,093,313 and 153,037,286 shares issued and outstanding at March 31, 2025 and December 31, 2024, respectively $ 1,179,336 $ 1,175,181 Additional paid-in capital 161,407 156,454 Accumulated other comprehensive loss (204 ) (262 ) Accumulated deficit (1,168,736 ) (1,121,858 ) Total shareholders' equity $ 171,803 $ 209,515 Total liabilities, Series A preferred shares, and shareholders' equity $ 288,343 $ 352,207 WAVE LIFE SCIENCES CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS(In thousands, except share and per share amounts) Three Months Ended March 31, 2025 2024 Revenue $ 9,175 $ 12,538 Operating expenses: Research and development 40,622 33,447 General and administrative 18,357 13,549 Total operating expenses 58,979 46,996 Loss from operations (49,804 ) (34,458 ) Other income, net: Interest income 2,875 2,535 Other income, net 51 365 Total other income, net 2,926 2,900 Loss before income taxes (46,878 ) (31,558 ) Income tax benefit (provision) — — Net loss $ (46,878 ) $ (31,558 ) Net loss per share attributable to ordinary shareholders—basic and diluted $ (0.29 ) $ (0.24 ) Weighted-average ordinary shares used in computing net loss per share attributable to ordinary shareholders—basic and diluted 162,527,026 129,271,678 Other comprehensive loss: Net loss $ (46,878 ) $ (31,558 ) Foreign currency translation 58 (74 ) Comprehensive loss $ (46,820 ) $ (31,632 ) Sign in to access your portfolio
