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Wave Life Sciences Reports Second Quarter 2023 Financial Results and Provides Business Update
Preparing for imminent submission of clinical trial applications (CTAs) for GalNAc-conjugated AATD candidate (WVE-006), the industry’s first RNA editing clinical candidate
Novel DMD therapeutic candidate (WVE-N531) with best-in-class exon skipping clinical data is on track to enter a potentially registrational Phase 2 clinical study in 2H 2023, with dystrophin data expected in 2024
“R&D Day” virtual event planned for
Cash and cash equivalents of
Investor conference call and webcast at
“In the second quarter, we continued to rapidly advance our pipeline of RNA medicines towards meaningful inflection points. We are on the cusp of moving the industry’s first RNA editing therapeutic candidate, WVE-006 for alpha-1 antitrypsin deficiency or AATD, into the clinic, which will mark an important milestone not only for Wave, but also for individuals living with AATD and the entire RNA medicines field. WVE-006 has the potential to transform the standard of care for people living with AATD, and the goal of our initial clinical trial will be delivering early clinical proof-of-concept via measurement of validated serum surrogate markers,” said
Recent Business Highlights
- Presented new preclinical data for WVE-006 supporting its potential to be a first-and best-in-class treatment for alpha-1 antitrypsin deficiency (AATD). In
May 2023 , at theAmerican Society of Gene & Cell Therapy (ASGCT) Annual Meeting, Wave presented data demonstrating that treatment with WVE-006 improved several markers of liver disease in mice, including a decrease in liver inflammation and hepatocyte turnover and reduction in the size of PAS-D positive globules. WVE-006 is uniquely designed to address both liver and lung disease related to AATD and is poised to be the industry’s first RNA editing candidate to reach the clinic. The company expects to submit clinical trial applications (CTAs) for WVE-006 imminently.
- Highlighted clinical data for WVE-N531 to DMD community at Parent Project Muscular Dystrophy (PPMD) Annual Conference. In
June 2023 , in an oral presentation at thePPMD Conference , Wave highlighted its clinical data from the proof-of-concept study of WVE-N531, including the highest level of exon skipping seen in any DMD trial, high muscle concentrations, and a safe and tolerable profile. Wave is on track to initiate dosing in Part B, a potentially registrational Phase 2 trial of WVE-N531, in 2023, which will be powered to evaluate functional dystrophin expression following 24 and 48 weeks of biweekly dosing. The primary endpoint will be dystrophin protein levels, and the study will also evaluate safety and tolerability, pharmacokinetics, and functional endpoints. Data are expected in 2024. If successful, WVE-N531 has potential to become a near-term wholly owned commercial opportunity for Wave and would enable accelerated development of additional exon skipping candidates for other mutations.
- Multi-dose portion of SELECT-HD study of WVE-003 for HD underway. In the second quarter, Wave initiated the multi-dose portion of its ongoing Phase 1b/2a SELECT-HD clinical trial of WVE-003 (allele-selective silencing oligonucleotide) for Huntington’s disease, and dosing is currently underway.
- Emerging best-in-class siRNA designs highlighted at ASGCT. Also at the
May 2023 ASGCT Annual Meeting, Wave highlighted its novel siRNA formats in a poster presentation titled “PN modification of stereopure GalNAc-siRNAs enhances durability of human HSD17B13 silencing in transgenic mouse model.” Wave’s GalNAc-siRNAs were more potent and had better durability of effect in mice compared with reference GalNAc-siRNA designs, which were driven by improved Ago2 loading. These data were also included in Wave’sNucleic Acids Research publication fromApril 2023 , which can be accessed here.
Wave Life Sciences added to Russell Indexes. InJune 2023 , the company joined the Russell 2000® and Russell 3000® Indexes as part of the annual reconstitution.
Anticipated Upcoming Milestones and Events
WVE-N531 for DMD:
- Initiate dosing in Part B of potentially registrational Phase 2 clinical trial in 2023
- Deliver data from Part B in 2024
WVE-006 for AATD:
- Submit CTAs for first-in-human study in 2H 2023
WVE-003 for HD:
- Deliver additional single-dose and available multi-dose biomarker and safety clinical data in 2H 2023
Platform and Pipeline:
- “R&D Day” virtual event to be held on
September 28, 2023 , during which Wave will demonstrate how it is continuing to extend its leadership in RNA editing and share preclinical data on new wholly owned programs - Advance collaboration activities with GSK, with potential for additional cash inflows in 2023 and beyond
Second Quarter 2023 Financial Results
Wave reported a net loss of
Research and development expenses were
General and administrative expenses were
As of
Investor Conference Call and Webcast
Wave will host an investor conference call today at 8:30 a.m. ET to review second quarter 2023 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-and-presentations. 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
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 of data and completion of our clinical trials, 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; 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 compared to others; our ability to design compounds using multiple modalities and the anticipated benefits of that approach; the breadth and versatility of PRISM; 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 status and progress of our programs relative to potential competitors; anticipated benefits of our proprietary manufacturing processes and our internal manufacturing capabilities; the benefit of nucleic acid therapeutics generally; the strength of our intellectual property and the data that support our IP; the anticipated duration of our cash runway; our intended uses of capital; and our expectations regarding any potential global macro events beyond our control 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
UNAUDITED CONSOLIDATED BALANCE SHEETS (In thousands, except share amounts) |
||||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 172,974 | $ | 88,497 | ||||
| Prepaid expenses | 9,012 | 7,932 | ||||||
| Other current assets | 2,722 | 2,108 | ||||||
| Total current assets | 184,708 | 98,537 | ||||||
| Long-term assets: | ||||||||
| Property and equipment, net of accumulated depreciation of as of |
14,983 | 17,284 | ||||||
| Operating lease right-of-use assets | 24,805 | 26,843 | ||||||
| Restricted cash | 3,668 | 3,660 | ||||||
| Other assets | 1,821 | 62 | ||||||
| Total long-term assets | 45,277 | 47,849 | ||||||
| Total assets | $ | 229,985 | $ | 146,386 | ||||
| Liabilities, Series A preferred shares and shareholders’ equity (deficit) | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 12,379 | $ | 16,915 | ||||
| Accrued expenses and other current liabilities | 10,429 | 17,552 | ||||||
| Current portion of deferred revenue | 111,133 | 31,558 | ||||||
| Current portion of operating lease liability | 6,285 | 5,496 | ||||||
| Total current liabilities | 140,226 | 71,521 | ||||||
| Long-term liabilities: | ||||||||
| Deferred revenue, net of current portion | 104,540 | 79,774 | ||||||
| Operating lease liability, net of current portion | 28,875 | 32,118 | ||||||
| Other liabilities | 190 | 190 | ||||||
| Total long-term liabilities | 133,605 | 112,082 | ||||||
| Total liabilities | $ | 273,831 | $ | 183,603 | ||||
| Series A preferred shares, no par value; 3,901,348 shares issued and outstanding at |
$ | 7,874 | $ | 7,874 | ||||
| Shareholders’ equity (deficit): | ||||||||
| Ordinary shares, no par value; 98,566,816 and 86,924,643 shares issued and outstanding at |
$ | 839,675 | $ | 802,833 | ||||
| Additional paid-in capital | 124,601 | 119,442 | ||||||
| Accumulated other comprehensive income (loss) | (150 | ) | (29 | ) | ||||
| Accumulated deficit | (1,015,846 | ) | (967,337 | ) | ||||
| Total shareholders’ equity (deficit) | $ | (51,720 | ) | $ | (45,091 | ) | ||
| Total liabilities, Series A preferred shares and shareholders’ equity (deficit) | $ | 229,985 | $ | 146,386 | ||||
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (In thousands, except share and per share amounts) |
||||||||||||||||
| Three Months Ended |
Six Months Ended |
|||||||||||||||
| 2023 | 2022 | 2023 | 2022 | |||||||||||||
| Revenue | $ | 22,106 | $ | 375 | $ | 35,035 | $ | 2,125 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 33,314 | 29,733 | 64,293 | 57,203 | ||||||||||||
| General and administrative | 12,265 | 12,806 | 24,500 | 25,180 | ||||||||||||
| Total operating expenses | 45,579 | 42,539 | 88,793 | 82,383 | ||||||||||||
| Loss from operations | (23,473 | ) | (42,164 | ) | (53,758 | ) | (80,258 | ) | ||||||||
| Other income, net: | ||||||||||||||||
| Dividend income and interest income, net | 2,251 | 124 | 4,124 | 150 | ||||||||||||
| Other income, net | 118 | 744 | 1,125 | 998 | ||||||||||||
| Total other income, net | 2,369 | 868 | 5,249 | 1,148 | ||||||||||||
| Loss before income taxes | (21,104 | ) | (41,296 | ) | (48,509 | ) | (79,110 | ) | ||||||||
| Income tax provision | — | — | — | — | ||||||||||||
| Net loss | $ | (21,104 | ) | $ | (41,296 | ) | $ | (48,509 | ) | $ | (79,110 | ) | ||||
| Net loss per share attributable to ordinary shareholders—basic and diluted |
$ | (0.20 | ) | $ | (0.62 | ) | $ | (0.47 | ) | $ | (1.25 | ) | ||||
| Weighted-average ordinary shares used in computing net loss per share attributable to ordinary shareholders—basic and diluted |
105,462,414 | 66,479,293 | 103,768,971 | 63,514,426 | ||||||||||||
| Other comprehensive loss: | ||||||||||||||||
| Net loss | $ | (21,104 | ) | $ | (41,296 | ) | $ | (48,509 | ) | $ | (79,110 | ) | ||||
| Foreign currency translation | (100 | ) | (142 | ) | (121 | ) | (228 | ) | ||||||||
| Comprehensive loss | $ | (21,204 | ) | $ | (41,438 | ) | $ | (48,630 | ) | $ | (79,338 | ) | ||||
Investor Contact:
+1 617-949-4827
krausch@wavelifesci.com
Media Contact:
+1 617-949-4817
asuter@wavelifesci.com
Source: Wave Life Sciences USA, Inc.