Full Press Release Details
Wave Life Sciences Reports Second Quarter 2025 Financial Results and Provides Business Update
WVE-006, the leading GalNAc-RNA editing oligonucleotide designed to correct AAT protein and address both lung and
liver manifestations of AATD, rapidly advancing in RestorAATion-2 study of AATD patients; multi-dosing is complete in first cohort (200 mg) and single dosing is complete in second cohort (400 mg)
RestorAATion-2 clinical data from complete 200 mg single and multidose dose cohorts remain on track for 3Q 2025;
data from complete 400 mg single dose cohort remain on track for fall 2025
WVE-007, an INHBE GalNAc-siRNA
designed to induce fat loss with muscle preservation, dosing is complete in expanded Cohort 2 (240 mg; from 8 to 32 individuals) of INLIGHT trial at dose predicted to be therapeutically active based on preclinical weight loss data
Expansion of INLIGHT Cohort 2 was triggered by favorable safety and tolerability as well as robust Activin E reduction in Cohort 1 (75 mg; n=8); Cohort 1
and 2 data expected in 4Q 2025 and dosing underway in Cohort 3 (400 mg) with data expected in 1Q 2026
Cash and cash equivalents of
$208.5 million as of June 30, 2025, with runway expected into 2027
Investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., July 30, 2025 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 second quarter ended June 30, 2025, and provided a business update.
We have rapidly advanced our RestorAATion-2 study following positive
proof-of mechanism data last year where we observed mean total AAT protein that met the level that has been the basis for regulatory approval for AAT augmentation therapies following a single, lowest planned
dose of WVE-006, our GalNAc-RNA editing candidate for AATD, said Paul Bolno, MD, MBA, President and Chief Executive Officer at Wave Life Sciences. We remain on track to share two comprehensive data
sets from our RestorAATion-2 trial this year, beginning with multidose data in the third quarter, which will inform the therapeutic potential of WVE-006, and our
pipeline of wholly-owned GalNAc-RNA editing programs.
Dr. Bolno continued, We are also advancing our INLIGHT trial of WVE-007, our INHBE GalNAc-siRNA that uses our propriety chemistry and best-in-class design, in individuals living with overweight and
obesity. Since our last update, we have expanded Cohort 2 based on the favorable safety and tolerability data and robust target engagement we observed in Cohort 1, our lowest single dose cohort, and dosed 24 additional patients in Cohort 2. This
achievement confirms the successful clinical translation of our siRNA platform and preclinical modeling. It also strengthens our conviction in upcoming data from Cohort 2, which tests a dose that is projected to be therapeutically active based on
preclinical weight loss data. Additionally, WVE-N531, for DMD, and WVE-003, for HD, remain on track, both of which have demonstrated potential best-in-class therapeutic profiles. Our consistent execution in the clinic has positioned us to deliver multiple robust data sets in the second half of 2025 that carry the
potential to further extend our leadership in RNA medicines.
Recent Business Highlights and Expected Milestones
AATD (Alpha-1 antitrypsin deficiency)
Emerging wholly owned siRNA and RNA editing pipeline
DMD (Duchenne muscular dystrophy)
HD (Huntington s disease)
Financial Highlights
Corporate Highlights
Investor Conference Call and Webcast
Wave will host an investor conference call today at 8:30 a.m. ET to review the second 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: https://ir.wavelifesciences.com/events-publications/events. Analysts planning to participate during
the Q&A portion of the live call can join the conference call at the audio-conferencing link here. Following the live event, an archived version of the webcast will be available on 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, obesity, Duchenne
muscular dystrophy, and Huntington s disease, 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 www.wavelifesciences.com and follow Wave on
X (formerly Twitter) and LinkedIn.
Forward-Looking Statements
This 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, including the anticipated therapeutic benefits of such programs; 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, and the timing and
announcement of data related to such preclinical activities; 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 status and progress of our programs and 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 benefits of RNA medicines generally; 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; anticipated benefits of our proprietary manufacturing processes and our internal manufacturing capabilities; 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; any potential macro economic events, including changes in economic policies; 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 Rausch
VP, Corporate Affairs and Investor Relations
Director, Investor Relations
Senior Director, Corporate Communications
WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED BALANCE SHEETS
(In thousands, except share amounts)
| June 30, 2025 | December 31, 2024 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 208,481 | $ | 302,078 | ||||
| Accounts receivable | 1,617 | 1,422 | ||||||
| Prepaid expenses | 6,700 | 9,544 | ||||||
| Other current assets | 6,758 | 7,350 | ||||||
| Total current assets | 223,556 | 320,394 | ||||||
| Long-term assets: | ||||||||
| Property and equipment, net of accumulated depreciation of $47,918 and $46,329 as of June 30, 2025 and December 31, 2024, respectively | 9,037 | 10,128 | ||||||
| Operating lease right-of-use assets | 15,250 | 17,870 | ||||||
| Restricted cash | 3,784 | 3,760 | ||||||
| Other assets | 728 | 55 | ||||||
| Total long-term assets | 28,799 | 31,813 | ||||||
| Total assets | $ | 252,355 | $ | 352,207 | ||||
| Liabilities, Series A preferred shares, and shareholders equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 14,664 | $ | 16,262 | ||||
| Accrued expenses and other current liabilities | 13,026 | 21,081 | ||||||
| Current portion of deferred revenue | 51,582 | 65,972 | ||||||
| Current portion of operating lease liability | 8,136 | 7,638 | ||||||
| Total current liabilities | 87,408 | 110,953 | ||||||
| Long-term liabilities: | ||||||||
| Deferred revenue, net of current portion | 4,232 | 6,099 | ||||||
| Operating lease liability, net of current portion | 13,575 | 17,766 | ||||||
| Total long-term liabilities | 17,807 | 23,865 | ||||||
| Total liabilities | $ | 105,215 | $ | 134,818 | ||||
| Series A preferred shares, no par value; 3,901,348 shares issued and outstanding at June 30, 2025 and December 31, 2024 | $ | 7,874 | $ | 7,874 | ||||
| Shareholders equity: | ||||||||
| Ordinary shares, no par value; 155,673,292 and 153,037,286 shares issued and outstanding at June 30, 2025 and December 31, 2024, respectively | $ | 1,191,029 | $ | 1,175,181 | ||||
| Additional paid-in capital | 167,603 | 156,454 | ||||||
| Accumulated other comprehensive loss | (161 | ) | (262 | ) | ||||
| Accumulated deficit | (1,219,205 | ) | (1,121,858 | ) | ||||
| Total shareholders equity | $ | 139,266 | $ | 209,515 | ||||
| Total liabilities, Series A preferred shares, and shareholders equity | $ | 252,355 | $ | 352,207 |
WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share amounts)
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Revenue | $ | 8,699 | $ | 19,692 | $ | 17,874 | $ | 32,230 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 43,469 | 40,393 | 84,091 | 73,840 | ||||||||||||
| General and administrative | 17,989 | 14,296 | 36,346 | 27,845 | ||||||||||||
| Total operating expenses | 61,458 | 54,689 | 120,437 | 101,685 | ||||||||||||
| Loss from operations | (52,759 | ) | (34,997 | ) | (102,563 | ) | (69,455 | ) | ||||||||
| Other income, net: | ||||||||||||||||
| Interest income | 2,372 | 2,092 | 5,247 | 4,627 | ||||||||||||
| Other income (expense), net | (82 | ) | (18 | ) | (31 | ) | 347 | |||||||||
| Total other income, net | 2,290 | 2,074 | 5,216 | 4,974 | ||||||||||||
| Loss before income taxes | (50,469 | ) | (32,923 | ) | (97,347 | ) | (64,481 | ) | ||||||||
| Income tax benefit (provision) | ||||||||||||||||
| Net loss | $ | (50,469 | ) | $ | (32,923 | ) | $ | (97,347 | ) | $ | (64,481 | ) | ||||
| Net loss per share attributable to ordinary shareholders basic and diluted | $ | (0.31 | ) | $ | (0.25 | ) | $ | (0.60 | ) | $ | (0.50 | ) | ||||
| Weighted-average ordinary shares used in computing net loss per share attributable to ordinary shareholders basic and diluted | 163,987,640 | 129,527,003 | 163,266,106 | 129,399,340 | ||||||||||||
| Other comprehensive loss: | ||||||||||||||||
| Net loss | $ | (50,469 | ) | $ | (32,923 | ) | $ | (97,347 | ) | $ | (64,481 | ) | ||||
| Foreign currency translation | 43 | (81 | ) | 101 | (155 | ) | ||||||||||
| Comprehensive loss | $ | (50,426 | ) | $ | (33,004 | ) | $ | (97,246 | ) | $ | (64,636 | ) |