Full Press Release Details
Wave Life Sciences Reports Third Quarter 2024 Financial Results and Provides Business Update
Achieved first-ever RNA editing in humans in RestorAATion-2 trial of WVE-006
(GalNAc-AIMer) in alpha-1 antitrypsin deficiency and announced three wholly owned GalNAc-AIMer preclinical programs that offer
first-in-class approaches to address unmet needs in cardiometabolic diseases
WVE-007 (INHBE GalNAc-siRNA) clinical trial on track for 1Q 2025; preclinical data demonstrates opportunities for
monotherapy, for synergistic use with GLP-1s, and for maintenance to avoid rebound weight gain following cessation of GLP-1s
Received supportive initial feedback from FDA on WVE-003; FDA is engaged in discussing pathways to accelerated
approval and open to Wave s plan to evaluate biomarkers, including caudate atrophy, as an endpoint to evaluate HD progression; IND submission expected in 2H 2025
Delivered positive interim data from FORWARD-53 study of WVE-N531; expect
feedback on a pathway to accelerated approval from regulators, as well as the complete 48-week FORWARD-53 data, in 1Q 2025
Cash and cash equivalents of $310.9 million as of September 30, 2024, plus additional ~$28 million net proceeds received on October 1
from full exercise of greenshoe option, with runway expected into 2027
Investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., November 12, 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 third quarter ended September 30, 2024, and provided a business update.
Since the start of the third quarter, we have delivered positive clinical updates across DMD and AATD, unlocked RNA editing for Wave, and advanced our
novel, wholly owned pipeline focused on GalNAc-conjugated programs, said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. Our achievement of
proof-of-mechanism for WVE-006 marked a historic breakthrough for the oligonucleotide field with the first-ever in-human evidence of RNA editing, providing a strong foundation to expand our wholly owned editing pipeline. We are also advancing WVE-007 for obesity and WVE-N531 for DMD towards key milestones in the first quarter. In HD, we are encouraged by a potential path forward to bring the first-ever therapeutic approach that preserves healthy wild-type protein to people
living with HD, a devastating disease with limited treatment options. We continue to receive substantial interest on HD, including from potential strategic partners. With the continued demonstration of our novel chemistry in the clinic and cash
runway expected into 2027, we are well capitalized and well positioned to deliver multiple value inflection points across our portfolio as we continue to build a leading RNA medicines company.
Recent Business Highlights
Obesity (GalNAc-siRNA)
HD (allele-selective 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 third 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
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 timing and announcement of such events; the protocol, design and endpoints of our 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, future clinical data readouts and further validate of our platform; ongoing and future 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 progress and potential benefits of our collaborations; the potential achievement of
milestones under our collaborations and receipt of cash payments therefor; the potential commercial opportunities that our therapeutic candidates may address; 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; patient population estimates related to our therapeutic candidates; 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 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 and accelerated approval pathways, 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)
| September 30, 2024 | December 31, 2023 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 310,948 | $ | 200,351 | ||||
| Accounts receivable | 21,086 | |||||||
| Prepaid expenses | 10,572 | 9,912 | ||||||
| Other current assets | 2,995 | 4,024 | ||||||
| Total current assets | 324,515 | 235,373 | ||||||
| Long-term assets: | ||||||||
| Property and equipment, net of accumulated depreciation of $45,490 and $42,709 as of September 30, 2024 and December 31, 2023, respectively | 10,928 | 13,084 | ||||||
| Operating lease right-of-use assets | 19,119 | 22,637 | ||||||
| Restricted cash | 3,746 | 3,699 | ||||||
| Other assets | 196 | 156 | ||||||
| Total long-term assets | 33,989 | 39,576 | ||||||
| Total assets | $ | 358,504 | $ | 274,949 | ||||
| Liabilities, Series A preferred shares, and shareholders equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 12,781 | $ | 12,839 | ||||
| Accrued expenses and other current liabilities | 14,642 | 16,828 | ||||||
| Current portion of deferred revenue | 135,907 | 150,059 | ||||||
| Current portion of operating lease liability | 7,398 | 6,714 | ||||||
| Total current liabilities | 170,728 | 186,440 | ||||||
| Long-term liabilities: | ||||||||
| Deferred revenue, net of current portion | 18,490 | 15,601 | ||||||
| Operating lease liability, net of current portion | 19,772 | 25,404 | ||||||
| Total long-term liabilities | 38,262 | 41,005 | ||||||
| Total liabilities | $ | 208,990 | $ | 227,445 | ||||
| Series A preferred shares, no par value; 3,901,348 shares issued and outstanding at September 30, 2024 and December 31, 2023 | $ | 7,874 | $ | 7,874 | ||||
| Shareholders equity: | ||||||||
| Ordinary shares, no par value; 148,392,939 and 119,162,234 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively | $ | 1,139,714 | $ | 935,367 | ||||
| Additional paid-in capital | 153,196 | 129,237 | ||||||
| Accumulated other comprehensive loss | (159 | ) | (124 | ) | ||||
| Accumulated deficit | (1,151,111 | ) | (1,024,850 | ) | ||||
| Total shareholders equity | $ | 141,640 | $ | 39,630 | ||||
| Total liabilities, Series A preferred shares, and shareholders equity | $ | 358,504 | $ | 274,949 |
WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS)
(In thousands, except share and per share amounts)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Revenue | $ | (7,676 | ) | $ | 49,214 | $ | 24,554 | $ | 84,249 | |||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 41,197 | 31,642 | 115,037 | 95,935 | ||||||||||||
| General and administrative | 15,042 | 13,128 | 42,887 | 37,628 | ||||||||||||
| Total operating expenses | 56,239 | 44,770 | 157,924 | 133,563 | ||||||||||||
| Income (loss) from operations | (63,915 | ) | 4,444 | (133,370 | ) | (49,314 | ) | |||||||||
| Other income, net: | ||||||||||||||||
| Dividend income and interest income | 1,798 | 1,960 | 6,425 | 6,084 | ||||||||||||
| Other income, net | 337 | 171 | 684 | 1,296 | ||||||||||||
| Total other income, net | 2,135 | 2,131 | 7,109 | 7,380 | ||||||||||||
| Income (loss) before income taxes | (61,780 | ) | 6,575 | (126,261 | ) | (41,934 | ) | |||||||||
| Income tax benefit (provision) | 677 | 677 | ||||||||||||||
| Net income (loss) | $ | (61,780 | ) | $ | 7,252 | $ | (126,261 | ) | $ | (41,257 | ) | |||||
| Less: net income attributable to participating securities | $ | $ | (257 | ) | $ | $ | ||||||||||
| Net income (loss) attributable to ordinary shareholders, basic and diluted | $ | (61,780 | ) | $ | 6,995 | $ | (126,261 | ) | $ | (41,257 | ) | |||||
| Net income (loss) per share attributable to ordinary shareholders basic | $ | (0.47 | ) | $ | 0.07 | $ | (0.97 | ) | $ | (0.39 | ) | |||||
| Weighted-average ordinary shares used in computing net income (loss) per share attributable to ordinary shareholders basic | 132,563,467 | 106,025,063 | 130,470,603 | 104,529,266 | ||||||||||||
| Net income (loss) per share attributable to ordinary shareholders diluted | $ | (0.47 | ) | $ | 0.07 | $ | (0.97 | ) | $ | (0.39 | ) | |||||
| Weighted-average ordinary shares used in computing net income (loss) per share attributable to ordinary shareholders diluted | 132,563,467 | 106,975,231 | 130,470,603 | 104,529,266 | ||||||||||||
| Other comprehensive income (loss): | ||||||||||||||||
| Net income (loss) | $ | (61,780 | ) | $ | 7,252 | $ | (126,261 | ) | $ | (41,257 | ) | |||||
| Foreign currency translation | 120 | (32 | ) | (35 | ) | (153 | ) | |||||||||
| Comprehensive income (loss) | $ | (61,660 | ) | $ | 7,220 | $ | (126,296 | ) | $ | (41,410 | ) |