Full Press Release Details
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 for dosing 1 2 times per year
Continued momentum in GSK collaboration; advancing first two GSK collaboration programs following successful target validation; both programs utilize
Wave s GalNAc-siRNA format and discovery collaboration continues to span all Wave modalities, including RNA editing
Clinical data on track
for first-in-class, allele-selective HD program (expected 2Q 2024) and potentially registrational FORWARD-53 trial in DMD
Cash and cash equivalents of $181 million as of March 31, 2024; with an additional $12 million aggregate
initiation payment earned under GSK collaboration subsequent to quarter-end for advancement of programs; runway expected into 4Q 2025
Investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., May 9, 2024 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, 2024, and provided a business update.
Since our last update, we have made excellent progress across our multimodal pipeline of novel RNA medicines and continued to advance our collaboration
with GSK. We are on-track to deliver key data sets this year, which enable opportunities to unlock the broad potential of our RNA editing, silencing and splicing capabilities, said Paul Bolno, MD, MBA,
President and Chief Executive Officer of Wave Life Sciences. In RNA editing, we are advancing WVE-006, having used data from healthy volunteers in RestorAATion-1
to identify a starting dose in our RestorAATion-2 study that is expected to engage target in patients. With RestorAATion-2 now underway, we remain on track to deliver
expected proof of mechanism data in patients with AATD this year, which would represent the first-ever clinical demonstration of RNA editing and be an important milestone for the alpha-1 community, as well as
serve to validate our wholly owned RNA editing pipeline.
Dr. Bolno continued, In addition to pioneering the field of RNA editing, we are
working expeditiously to advance our GalNAc-siRNA INHBE program in obesity and expect to initiate a clinical trial in the first quarter of next year. In DMD and HD, we are approaching clinical data readouts as we plan to deliver multidose data for
our allele-selective HD program in the second quarter and potentially registrational data from our FORWARD-53 trial in DMD in the third quarter. Our efforts to accelerate development of our INHBE program and
advance our pipeline and collaborations have laid a strong foundation for Wave s future and we look forward to demonstrating our leadership in RNA medicines as we reimagine what s possible for science, for medicine, and for human
Recent Business Highlights
AATD and AIMer pipeline (RNA editing)
HD (antisense silencing)
Financial Highlights
Investor Conference Call and Webcast
Wave will host an investor conference call today at 8:30 a.m. ET to review the first quarter 2024 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 following audio-conferencing link: available here. Once registered, participants will receive the dial-in
information. Following the live event, an archived version of the webcast will be available on the Wave Life Sciences website.
About 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 prevalent 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
Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington s disease, as well as a preclinical program in obesity. 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 announcement of such events; the protocol, design and endpoints of our clinical trials; the future performance and results of
our programs in clinical trials; ongoing and future preclinical activities and programs; regulatory submissions; the progress and potential benefits of our collaborations; the potential achievement of milestones under our collaborations and receipt
of cash payments therefor; the potential of our preclinical data to predict the behavior of our compounds in humans; our identification and expected timing of future product candidates and their therapeutic potential; the anticipated benefits of our
therapeutic candidates and pipeline compared to our competitors; our ability to design compounds using various modalities and the anticipated benefits of that approach; the breadth and versatility of our PRISM 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; the potential benefits of our GalNAc-conjugated
siRNA program targeting INHBE; the potential benefits that our edit-verse may provide us, including identifying new RNA editing targets; 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 adaptive trial designs, which may affect the initiation, timing and progress of clinical trials; our effectiveness in managing regulatory interactions and future clinical trials; the effectiveness of PRISM; 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.
WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED BALANCE SHEETS
(In thousands, except share amounts)
| March 31, 2024 | December 31, 2023 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 180,922 | $ | 200,351 | ||||
| Accounts receivable | 21,086 | |||||||
| Prepaid expenses | 11,139 | 9,912 | ||||||
| Other current assets | 4,706 | 4,024 | ||||||
| Total current assets | 196,767 | 235,373 | ||||||
| Long-term assets: | ||||||||
| Property and equipment, net of accumulated depreciation of $43,687 and $42,709 as of March 31, 2024 and December 31, 2023, respectively | 12,418 | 13,084 | ||||||
| Operating lease right-of-use assets | 21,502 | 22,637 | ||||||
| Restricted cash | 3,715 | 3,699 | ||||||
| Other assets | 868 | 156 | ||||||
| Total long-term assets | 38,503 | 39,576 | ||||||
| Total assets | $ | 235,270 | $ | 274,949 | ||||
| Liabilities, Series A preferred shares, and shareholders equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 11,730 | $ | 12,839 | ||||
| Accrued expenses and other current liabilities | 6,621 | 16,828 | ||||||
| Current portion of deferred revenue | 140,586 | 150,059 | ||||||
| Current portion of operating lease liability | 6,936 | 6,714 | ||||||
| Total current liabilities | 165,873 | 186,440 | ||||||
| Long-term liabilities: | ||||||||
| Deferred revenue, net of current portion | 12,536 | 15,601 | ||||||
| Operating lease liability, net of current portion | 23,598 | 25,404 | ||||||
| Total long-term liabilities | 36,134 | 41,005 | ||||||
| Total liabilities | $ | 202,007 | $ | 227,445 | ||||
| Series A preferred shares, no par value; 3,901,348 shares issued and outstanding at March 31, 2024 and December 31, 2023 | $ | 7,874 | $ | 7,874 | ||||
| Shareholders equity: | ||||||||
| Ordinary shares, no par value; 122,321,384 and 119,162,234 shares issued and outstanding at March 31, 2024 and December 31, 2023, respectively | $ | 949,877 | $ | 935,367 | ||||
| Additional paid-in capital | 132,118 | 129,237 | ||||||
| Accumulated other comprehensive loss | (198 | ) | (124 | ) | ||||
| Accumulated deficit | (1,056,408 | ) | (1,024,850 | ) | ||||
| Total shareholders equity | $ | 25,389 | $ | 39,630 | ||||
| Total liabilities, Series A preferred shares, and shareholders equity | $ | 235,270 | $ | 274,949 |
WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share amounts)
| Three Months Ended March 31, | ||||||||
| 2024 | 2023 | |||||||
| Revenue | $ | 12,538 | $ | 12,929 | ||||
| Operating expenses: | ||||||||
| Research and development | 33,447 | 30,979 | ||||||
| General and administrative | 13,549 | 12,235 | ||||||
| Total operating expenses | 46,996 | 43,214 | ||||||
| Loss from operations | (34,458 | ) | (30,285 | ) | ||||
| Other income, net: | ||||||||
| Dividend income and interest income | 2,535 | 1,873 | ||||||
| Other income, net | 365 | 1,007 | ||||||
| Total other income, net | 2,900 | 2,880 | ||||||
| Loss before income taxes | (31,558 | ) | (27,405 | ) | ||||
| Income tax benefit (provision) | ||||||||
| Net loss | $ | (31,558 | ) | $ | (27,405 | ) | ||
| Net loss per share attributable to ordinary shareholders basic and diluted | $ | (0.24 | ) | $ | (0.27 | ) | ||
| Weighted-average ordinary shares used in computing net loss per share attributable to ordinary shareholders basic and diluted | 129,271,678 | 102,056,712 | ||||||
| Other comprehensive loss: | ||||||||
| Net loss | $ | (31,558 | ) | $ | (27,405 | ) | ||
| Foreign currency translation | (74 | ) | (21 | ) | ||||
| Comprehensive loss | $ | (31,632 | ) | $ | (27,426 | ) |