Wave Life Sciences Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update
Wave Life Sciences (WVE) announces lead clinical candidate selection for potential best-in-class obesity treatment with promising clinical trial updates. Financially, the company reports strong cash position and revenue growth, with positive outlook.
Positive
Wave Life Sciences selects lead clinical candidate for potential best-in-class obesity treatment with INHBE program
Clinical trial updates for WVE-006, WVE-N531, and WVE-003 show promising results in various therapeutic areas
Cash and cash equivalents of $200 million as of December 31, 2023, provide strong financial position
Revenue growth to $29.1 million in the fourth quarter of 2023, compared to $1.2 million in the prior year quarter
Net loss significantly improves in 2023 to $57.5 million from $161.8 million in 2022
The financial results reported by Wave Life Sciences, with a significant improvement in net loss from $161.8 million in 2022 to $57.5 million in 2023, highlight a strategic pivot towards a more sustainable fiscal trajectory. This improvement is largely attributed to the increased revenue from collaborations, which grew from $3.6 million in 2022 to $113.3 million in 2023. The company's cash position, bolstered by milestone payments and exercise of the greenshoe option, offers a runway into 4Q 2025. This financial stability is critical for sustaining ongoing and future clinical trials without immediate pressure for additional capital raising.
However, investors should scrutinize the sustainability of these revenue streams, as they are heavily reliant on milestone payments from strategic partnerships rather than product sales. The long-term viability of Wave Life Sciences will depend on the successful commercialization of its clinical candidates and the ability to maintain a balanced expenditure on research and development, which currently stands at $130.0 million for the full year 2023.
The announcement of Wave Life Sciences' clinical trial advancements, particularly in RNA editing and exon-skipping technologies, underscores the company's commitment to innovation in genetic medicine. The initiation of the RestorAATion-1 clinical trial for alpha-1 antitrypsin deficiency (AATD) and the expected proof-of-mechanism data in 2024 are pivotal for validating the efficacy of WVE-006, a potentially first-in-class treatment. The implications for patients with AATD are profound, as this therapy could address both lung and liver disease associated with the condition.
Similarly, the potential registrational FORWARD-53 trial for Duchenne muscular dystrophy (DMD) and the SELECT-HD clinical trial for Huntington's disease (HD) represent significant advancements in treating these debilitating diseases. The focus on preserving muscle mass in obesity treatment and sparing wild-type huntingtin protein in HD therapy demonstrates a nuanced approach to treatment that could offer improved outcomes over existing therapies.
The biotechnology sector is highly competitive, with innovation serving as a key differentiator among companies. Wave Life Sciences' positioning with multiple high-impact clinical data readouts in the pipeline places it in a potentially advantageous position within the industry. The selection of the INHBE lead clinical candidate for obesity signals a strategic move to address a prevalent health issue with a large market potential. Given the obesity epidemic and the limitations of current treatments, a successful therapy that induces weight loss without muscle mass loss could capture significant market share.
Additionally, the company's advancements in DMD and HD treatments cater to niche markets with unmet medical needs, offering the potential for strong market penetration. However, the success of these programs will hinge on clinical outcomes and regulatory approvals. It is also important for investors to monitor the competitive landscape, as other companies may be working on similar treatments that could impact Wave Life Sciences' market position.
03/06/2024 - 07:30 AM
INHBE lead clinical candidate selected; potential best-in-class treatment for obesity with potent and durable silencing, weight loss with no loss of muscle mass, reduction of visceral fat, and every-six-month or annual subcutaneous dosing; clinical trial initiation expected 1Q 2025
Dose escalation ongoing in RestorAATion-1 clinical trial of WVE-006, industry’s first-ever RNA editing candidate in the clinic; GalNAc pharmacology translating as expected and proof-of-mechanism data from patients with AATD in RestorAATion-2 on track for 2024
Dystrophin data from potentially registrational FORWARD-53 trial of WVE-N531 in DMD on track for 3Q 2024; industry-leading exon skipping levels, muscle tissue concentrations, and myogenic stem cell distribution achieved in Part A clinical trial
WVE-003 multi-dose data in HD with extended follow-up on track for 2Q 2024; first-in-class program designed to lower mutant HTT while sparing wild-type HTT
Cash and cash equivalents of $200 million as of December 31, 2023; additional $34 million received in 1Q 2024 in a milestone payment from GSK and net proceeds from full exercise of greenshoe option, with runway expected into 4Q 2025; additional anticipated payments under existing collaborations in 2024 and beyond
Investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., March 06, 2024 (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 fourth quarter and full year ended December 31, 2023, and provided a business update.
“Following a year of execution and tremendous progress, we entered 2024 well-capitalized and uniquely positioned to deliver multiple high-impact clinical data readouts across three different modalities and unlock the broad potential of our pipeline in both rare and prevalent diseases,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “We are rapidly accelerating the development of our next generation GalNAc-siRNA INHBE program in obesity and are encouraged by the emerging preclinical data, which supports a potential best-in-class profile for this program. These data demonstrate greater knockdown, potency, and durability following a low, single dose, compared to our first generation data, and importantly, demonstrate substantial weight loss with decreases in fat mass and no loss of muscle mass.”
Dr. Bolno continued, “In parallel with our efforts to advance our INHBE program, we are continuing to progress our clinical trials. We remain at the forefront of innovation in RNA editing and expect to deliver proof-of-mechanism data for WVE-006 in AATD in 2024. These data would unlock our rapidly advancing pipeline of wholly owned editing candidates. Beyond editing, we remain on track to deliver multidose data for our novel, allele-selective HD program in the second quarter and potentially registrational data from our FORWARD-53 trial in DMD in the third quarter. The coming months have the potential to be truly transformative for Wave.”
Recent Business Highlights
AATD and AIMer pipeline (RNA editing)
Dose escalation in RestorAATion-1 clinical trial ongoing; clinical program investigating WVE-006 as a first- and best-in-class treatment for alpha-1 antitrypsin deficiency (AATD). WVE-006 is a GalNAc-conjugated, subcutaneously delivered, RNA editing oligonucleotide that is uniquely designed to address AATD-related lung disease, liver disease, or both. The RestorAATion clinical program is evaluating WVE-006 for AATD and includes healthy volunteers (RestorAATion-1), as well as patients who have the homozygous Pi*ZZ mutation (RestorAATion-2). It is designed to provide an efficient path to proof-of-mechanism as measured by restoration of wild-type alpha-1 antitrypsin (M-AAT) protein in serum. In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK. Dose escalation is ongoing and WVE-006’s pharmacology in healthy volunteers is translating as expected. Beyond WVE-006, Wave is advancing a pipeline of wholly owned RNA editing therapeutics across a range of high-impact GalNAc-hepatic and extra-hepatic targets. Utilizing its proprietary “edit-verse,” which is powered by genetic datasets and deep learning models, Wave has identified several new RNA editing targets that leverage easily accessible biomarkers, offer efficient paths to proof-of-concept in humans, and represent meaningful commercial opportunities. Wave plans to share new preclinical data for its advancing RNA editing programs in 2024. Expected upcoming milestone: Wave expects to deliver proof-of-mechanism data from RestorAATion-2 in patients with AATD in 2024.Obesity (siRNA)
Selected lead clinical candidate for INHBE program with a potentially best-in-class profile for obesity and accelerated clinical development timeline. Today, Wave announced the selection of its INHBE lead clinical candidate, ahead of prior expectations, and now plans to submit a clinical trial application for its INHBE program as early as year-end 2024. Wave’s INHBE clinical candidate is a GalNAc-small interfering RNA (siRNA) that utilizes Wave’s next generation siRNA format and is designed to silence the INHBE (Inhibin βE) gene through RNA knockdown with a goal of inducing lipolysis (fat-burning) while preserving muscle mass to restore and maintain a healthy metabolic profile. INHBE loss-of-function (LoF) heterozygous human carriers have a favorable cardiometabolic profile, including reduced abdominal obesity and reduced odds of type 2 diabetes and coronary artery disease. Silencing INHBE is expected to recapitulate the cardiometabolic profile of these LoF carriers and may also address the limitations of GLP-1s as a monotherapy or be used as an adjunct therapy with GLP-1s. Key data highlights from preclinical mouse models include: Highly potent silencing (ED50 < 1mg/kg) Durable silencing following one, low-single-digit dose supporting every-six-month or annual subcutaneous dosing Weight loss with no loss of muscle mass Reduction in fat mass with preferential effects on visceral fat, consistent with the profile of INHBE LoF carriers in human genetics Expected upcoming milestone: Wave expects to initiate a clinical trial for its INHBE candidate in the first quarter of 2025.DMD (exon skipping)
Advancing FORWARD-53 clinical trial; multiple presentations at MDA Clinical & Scientific Conference highlight differentiated profile including industry-leading exon-skipping, muscle tissue concentrations and myogenic stem cell distribution. Wave’s WVE-N531 program for boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping is designed to induce production of endogenous, functional dystrophin protein. This week, at the 2024 MDA Clinical & Scientific Conference, Wave presented multiple posters highlighting WVE-N531. These posters featured data from Part A of Wave’s WVE-N531 trial, demonstrating industry-leading exon skipping levels of 53% and muscle tissue concentrations of 42 µg/g (42,000 ng/g), as well as stem cell distribution in all study participants. WVE-N531 is currently being evaluated in the ongoing FORWARD-53 clinical trial of 11 boys with DMD, which is powered to evaluate functional, endogenous dystrophin expression following 24 and 48 weeks of 10 mg/kg dosing administered every-other-week. The primary endpoint is dystrophin protein levels, and the trial will also evaluate pharmacokinetics, digital and functional endpoints, and safety and tolerability. Pending positive results from the FORWARD-53 trial, the company is planning to advance a broader DMD pipeline with PN-modified oligonucleotides for skipping other exons, with the goal of providing new treatment options for a larger population of boys with DMD. Expected upcoming milestone: Wave expects to deliver data, including dystrophin protein expression from muscle biopsies at 24 weeks, in the third quarter of 2024.HD (antisense silencing)
WVE-003 and wild-type HTT sparing highlighted at annual CHDI Conference. WVE-003 is a first-in-class investigational allele-selective Huntington’s disease (HD) therapeutic designed to reduce mutant huntingtin (mHTT) protein while also sparing healthy wild-type huntingtin (wtHTT) protein. Due to the significance of wtHTT function for the health of the central nervous system and the potential for mHTT to disrupt wtHTT function, selectively lowering mHTT while preserving wtHTT protein expression and function may offer advantages over nonselective HTT-lowering approaches for the treatment of HDrecent publication. WVE-003 has demonstrated single-dose reductions in mean mHTT in cerebrospinal fluid of 35% compared to placebo, with preservation of wtHTT, as previously shared in September 2022. The ongoing multi-dose portion of the SELECT-HD clinical trial is evaluating a cohort of 24 patients with HD receiving 30 mg doses of WVE-003 administered every eight weeks. Data from the ongoing SELECT-HD clinical trial will form the basis for decision making for Wave’s advancement of this program, including supporting an opt-in package for Takeda. Expected milestone: Wave expects to report data from the 30 mg multi-dose cohort with extended follow-up, along with all single-dose data, in the second quarter of 2024.Financial Highlights
Cash and cash equivalents were $200.4 million as of December 31, 2023, compared to $88.5 million as of December 31, 2022. The increase in cash year-over-year is primarily due to the $170.0 million of cash received from the GSK Collaboration and the $93.6 million in net proceeds from the December 2023 Offering, partially offset by the net loss for the year. Subsequent to December 31, 2023, Wave received $20 million in a milestone payment from GSK and $14.0 million in net proceeds from the full exercise of the greenshoe option related to the December 2023 financing. Wave expects that its current cash and cash equivalents will be sufficient to fund operations into the fourth quarter of 2025. Potential future milestone and other payments under the company’s GSK and Takeda collaborations are not included in its cash runway. Revenue was $29.1 million for the fourth quarter of 2023 as compared to $1.2 million in the prior year quarter. Revenue was $113.3 million in 2023 as compared to $3.6 million in 2022. The increase in revenue was due to revenue earned from the company’s collaborations with both GSK and Takeda. Research and development expenses were $34.1 million in the fourth quarter of 2023 as compared to $31.1 million in the same period in 2022. Research and development expenses for the full year were $130.0 million in 2023, as compared to $115.9 million in 2022. General and administrative expenses were $13.7 million in the fourth quarter of both 2023 and 2022. General and administrative expenses for the full year were $51.3 million in 2023, as compared to $50.5 million in 2022. Net loss was $16.3 million for the fourth quarter of 2023 as compared to $43.7 million in the prior year quarter. Net loss for the full year was $57.5 million for 2023 as compared to $161.8 million in 2022. Net loss significantly improved over the prior year due to the substantial revenue earned from collaboration partners in 2023. Investor Conference Call and Webcast 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 www.wavelifesciences.com and follow Wave on X (formerly Twitter) and LinkedIn .
Forward-Looking Statements
WAVE LIFE SCIENCES LTD. UNAUDITED CONSOLIDATED BALANCE SHEETS (In thousands, except share amounts) December 31, 2023 December 31, 2022 Assets Current assets: Cash and cash equivalents $ 200,351 $ 88,497 Accounts receivable 21,086 — Prepaid expenses 9,912 7,932 Other current assets 4,024 2,108 Total current assets 235,373 98,537 Long-term assets: Property and equipment, net of accumulated depreciation of $42,709 and $37,846 13,084 17,284 Operating lease right-of-use assets 22,637 26,843 Restricted cash 3,699 3,660 Other assets 156 62 Total long-term assets 39,576 47,849 Total assets $ 274,949 $ 146,386 Liabilities, Series A preferred shares and shareholders’ equity (deficit) Current liabilities: Accounts payable $ 12,839 $ 16,915 Accrued expenses and other current liabilities 16,828 17,552 Current portion of deferred revenue 150,059 31,558 Current portion of operating lease liability 6,714 5,496 Total current liabilities 186,440 71,521 Long-term liabilities: Deferred revenue, net of current portion 15,601 79,774 Operating lease liability, net of current portion 25,404 32,118 Other liabilities — 190 Total long-term liabilities 41,005 112,082 Total liabilities $ 227,445 $ 183,603 Series A preferred shares, no par value; 3,901,348 shares issued $ 7,874 $ 7,874 Shareholders’ equity (deficit): Ordinary shares, no par value; 119,162,234 and 86,924,643 shares issued $ 935,367 $ 802,833 Additional paid-in capital 129,237 119,442 Accumulated other comprehensive loss (124 ) (29 ) Accumulated deficit (1,024,850 ) (967,337 ) Total shareholders’ equity (deficit) 39,630 (45,091 ) Total liabilities, Series A preferred shares and shareholders’ equity (deficit) $ 274,949 $ 146,386
WAVE LIFE SCIENCES LTD. UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) (In thousands, except share and per share amounts) Three Months Ended December 31, Twelve Months Ended December 31, 2023 2022 2023 2022 Revenue $ 29,056 $ 1,239 $ 113,305 $ 3,649 Operating expenses: Research and development 34,074 31,078 130,009 115,856 General and administrative 13,664 13,724 51,292 50,513 Total operating expenses 47,738 44,802 181,301 166,369 Loss from operations (18,682 ) (43,563 ) (67,996 ) (162,720 ) Other income, net: Dividend income and interest income, net 1,844 825 7,928 1,571 Other income (expense), net 582 (290 ) 1,878 7 Total other income, net 2,426 535 9,806 1,578 Loss before income taxes (16,256 ) (43,028 ) (58,190 ) (161,142 ) Income tax benefit (provision) — (681 ) 677 (681 ) Net loss $ (16,256 ) $ (43,709 ) $ (57,513 ) $ (161,823 ) Net loss per share attributable to ordinary $ (0.15 ) $ (0.47 ) $ (0.54 ) $ (2.05 ) Weighted-average ordinary shares used in computing 109,627,549 93,993,638 106,097,268 78,855,810 Other comprehensive loss: Net loss $ (16,256 ) $ (43,709 ) $ (57,513 ) $ (161,823 ) Foreign currency translation 58 94 (95 ) (210 ) Comprehensive loss $ (16,198 ) $ (43,615 ) $ (57,608 ) $ (162,033 )
Investor Contact: krausch@wavelifesci.com
Media Contact: asuter@wavelifesci.com
What is the lead clinical candidate selected by Wave Life Sciences for potential best-in-class obesity treatment?
Wave Life Sciences has selected its lead clinical candidate for its INHBE program, showcasing a potentially best-in-class profile for obesity.
What are the key data highlights from preclinical mouse models regarding the INHBE program?
Key data highlights include highly potent silencing, durable silencing supporting every-six-month or annual dosing, weight loss with no loss of muscle mass, and reduction in fat mass.
What is the expected milestone for Wave Life Sciences regarding the INHBE program?
Wave Life Sciences expects to initiate a clinical trial for its INHBE candidate in the first quarter of 2025.
What is the WVE program by Wave Life Sciences for boys with Duchenne muscular dystrophy (DMD)?
Wave Life Sciences' WVE-N531 program for DMD aims to induce production of functional dystrophin protein in boys amenable to exon 53 skipping.
When does Wave Life Sciences expect to deliver data from the FORWARD-53 trial in DMD?
Wave Life Sciences expects to deliver data, including dystrophin protein expression from muscle biopsies at 24 weeks, in the third quarter of 2024.
What financial highlights were reported by Wave Life Sciences for the fourth quarter of 2023?
In the fourth quarter of 2023, Wave Life Sciences reported cash and cash equivalents of $200.4 million, revenue of $29.1 million, and a net loss of $16.3 million.
