Full Press Release Details
Wave Life Sciences Reports Third Quarter 2021 Financial Results and Provides Business Update
Strengthened balance sheet with approximately $52 million; focusing additional investment in RNA editing programs led by hepatic
Optimized AIMers for AATD program demonstrate potent, highly specific RNA editing and restoration of functional AAT protein
substantially above therapeutic threshold; potential for best-in-class, potent and durable RNA editing in vivo in multiple preclinical models and tissues
Dosing ongoing in three clinical programs (WVE-004, WVE-003,
WVE-N531); data being generated through 2022 to enable decision-making
investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., November 10, 2021 (GLOBE NEWSWIRE) Wave Life Sciences Ltd.
(Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced financial results for the third quarter ended September 30, 2021 and provided a
In the third quarter, we achieved several important milestones including providing a comprehensive update on our potentially best-in-class ADAR editing capability and the initiation of dosing in three clinical trials evaluating our next-generation stereopure
PN-modified oligonucleotides, said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. RNA editing is a novel therapeutic modality that greatly expands our landscape
of addressable genetically defined diseases. We are leading the way in this new field and quickly working toward announcing our first ADAR editing development candidate for our alpha-1 antitrypsin deficiency
program next year. With this program, we are on a path to generate proof of principle that we can harness human biological machinery to edit RNA for the treatment of genetic diseases of the liver, CNS, and beyond.
Our robust and diversified pipeline is driven by our PRISM platform, which enables a unique ability to design and optimize oligonucleotides with novel,
stereopure backbone modifications, including PN chemistry. We expect data being generated from our three ongoing clinical trials will enable us to make decisions on next steps for the programs next year. Finally, we recently strengthened our balance
sheet via our at-the-market facility and funds received from Takeda under the terms of the amendment, leaving us well-capitalized to deliver on our portfolio, including
advancing our first ADAR editing program toward the clinic and expanding our AIMer pipeline to include additional indications.
capability recent events and upcoming milestones
Leading RNA editing capability using AIMers to harness endogenous ADAR enzymes
Alpha-1 antitrypsin deficiency (AATD) program with ADAR editing:
Clinical silencing and exon skipping programs and upcoming milestones
WVE-004 for C9orf72-associated amyotrophic lateral sclerosis (C9-ALS) and
frontotemporal dementia (C9-FTD):
WVE-003 targeting SNP3 for Huntington s disease (HD):
WVE-N531 for Duchenne muscular dystrophy (DMD) amenable to exon
clinical milestones:
Corporate developments
Third quarter 2021 financial results and financial guidance
Wave reported a net loss of $6.2 million in the third quarter of 2021 as compared to $33.1 million in the same period in 2020.
Revenue earned during the three months ended September 30, 2021 was $36.4 million, as compared to $3.4 million for the three months ended
September 30, 2020. The increase in revenue year-over-year is primarily driven by the $22.5 million paid as part of the amendment to Wave s collaboration agreement with Takeda, which was recognized as revenue in the three months ended
September 30, 2021, as well as the recognition of the remaining revenue related to Category 2 research support payments previously paid by Takeda.
Research and development expenses were $31.1 million in the third quarter of 2021 as compared to $28.3 million in the same period in 2020. The
increase in research and development expenses in the third quarter was primarily due to increased external expenses related to preclinical programs and compensation-related expenses, partially offset by decreased external expenses related to our
discontinued programs.
General and administrative expenses were $12.9 million in the third quarter of 2021 as compared to $9.6 million in the
same period in 2020. The increase in general and administrative expenses in the third quarter of 2021 was driven by increases in compensation-related and other external general and administrative expenses.
As of September 30, 2021, Wave had $123.9 million in cash and cash equivalents as compared to $184.5 million as of December 31, 2020. The
decrease in cash and cash equivalents was mainly due to Wave s year-to-date net loss of $87.5 million, partially offset by the receipt of
$21.2 million in proceeds under Wave s ATM equity program through September 30, 2021.
Subsequently, in October 2021 Wave received an additional $52.1 million in cash, including
$22.5 million from Takeda under the terms of the amendment to Wave s collaboration agreement with Takeda, and $29.6 million in proceeds under its ATM equity program from a block sale of ordinary shares based on interest received from
new and existing shareholders following its Analyst and Investor Research Webcast in September 2021.
Wave expects that its existing cash and cash
equivalents will enable the company to fund its operating and capital expenditure requirements into the second quarter of 2023.
Wave management will host an investor conference call today at 8:30 a.m. ET to discuss the company s third quarter and 2021
financial results and provide a business update. The conference call may be accessed by dialing (866) 220-8068 (domestic) or (470) 495-9153 (international) and entering
conference ID: 6995569. The live webcast may be accessed from the Investor Relations section of the Wave Life Sciences corporate website at ir.wavelifesciences.com. Following the webcast, a replay will be available on the website.
PRISM is Wave Life Sciences proprietary discovery and drug development platform that enables genetically defined diseases to be targeted with stereopure
oligonucleotides across multiple therapeutic modalities, including silencing, splicing and editing. PRISM combines the company s unique ability to construct stereopure oligonucleotides with a deep understanding of how the interplay among
oligonucleotide sequence, chemistry and backbone stereochemistry impacts key pharmacological properties. By exploring these interactions through iterative analysis of in vitro and in vivo outcomes and machine learning-driven predictive
modeling, the company continues to define design principles that are deployed across programs to rapidly develop and manufacture clinical candidates that meet pre-defined product profiles.
About Wave Life Sciences
Wave Life Sciences (Nasdaq:
WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop
best-in-class medicines across multiple therapeutic modalities using PRISM, the company s proprietary discovery and drug development platform that enables the
precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future.
To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.
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 for decision-making and
completion of our adaptive clinical trials, and the announcement of such events; the protocol, design and endpoints of our ongoing and planned 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 with partners; the potential of our in vitro and in vivo 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 therapeutic benefits of our potential therapies compared to others; our ability to design compounds using multiple modalities and the
anticipated benefits of that model; the potential benefits of PRISM, including our novel PN backbone chemistry modifications, and our stereopure oligonucleotides compared with stereorandom oligonucleotides; the potential benefits of our novel
ADAR-mediated RNA editing platform capabilities, including our AIMers, compared to others; the benefit of nucleic acid therapeutics generally; the strength of our intellectual property; our assumptions based on our balance sheet and the anticipated
duration of our cash runway; our intended uses of capital; and our expectations regarding the impact of the COVID-19 pandemic 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; our ability to maintain the company
infrastructure and personnel needed to achieve our goals; the clinical results of our programs, which may not support further development of product candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of
clinical trials, including their receptiveness to our adaptive trial designs; our effectiveness in managing future clinical trials and regulatory interactions; the effectiveness of PRISM, including our novel PN backbone chemistry modifications; the
effectiveness of our novel ADAR-mediated RNA editing platform capability and our AIMers; the continued development and acceptance of oligonucleotides as a class of medicines; 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 similar indications; the severity and duration of the COVID-19 pandemic and its negative
impact on the conduct of, and the timing of enrollment, completion and reporting with respect to our clinical trials; and any other impacts on our business as a result of or related to the COVID-19 pandemic,
as well as 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, 2021 | December 31, 2020 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 123,896 | $ | 184,497 | ||||
| Accounts receivable | 22,500 | 30,000 | ||||||
| Prepaid expenses | 7,627 | 10,434 | ||||||
| Other current assets | 3,964 | 5,111 | ||||||
| Total current assets | 157,987 | 230,042 | ||||||
| Long-term assets: | ||||||||
| Property and equipment, net | 24,020 | 29,198 | ||||||
| Operating lease right-of-use assets | 14,639 | 16,232 | ||||||
| Restricted cash | 3,651 | 3,651 | ||||||
| Other assets | 215 | 115 | ||||||
| Total long-term assets | 42,525 | 49,196 | ||||||
| Total assets | $ | 200,512 | $ | 279,238 | ||||
| Liabilities, Series A preferred shares and shareholders equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 7,443 | $ | 13,795 | ||||
| Accrued expenses and other current liabilities | 11,364 | 11,971 | ||||||
| Current portion of deferred revenue | 8,736 | 91,560 | ||||||
| Current portion of operating lease liability | 4,097 | 3,714 | ||||||
| Total current liabilities | 31,640 | 121,040 | ||||||
| Long-term liabilities: | ||||||||
| Deferred revenue, net of current portion | 107,606 | 41,481 | ||||||
| Operating lease liability, net of current portion | 22,477 | 25,591 | ||||||
| Other liabilities | 1,014 | 474 | ||||||
| Total long-term liabilities | $ | 131,097 | $ | 67,546 | ||||
| Total liabilities | $ | 162,737 | $ | 188,586 | ||||
| Series A preferred shares, no par value; 3,901,348 shares issued and outstanding at September 30, 2021 and December 31, 2020 | $ | 7,874 | $ | 7,874 | ||||
| Shareholders equity: | ||||||||
| Ordinary shares, no par value; 51,998,032 and 48,778,678 shares issued and outstanding at September 30, 2021 and December 31, 2020, respectively | $ | 716,118 | $ | 694,085 | ||||
| Additional paid-in capital | 84,254 | 71,573 | ||||||
| Accumulated other comprehensive income | 258 | 389 | ||||||
| Accumulated deficit | (770,729 | ) | (683,269 | ) | ||||
| Total shareholders equity | $ | 29,901 | $ | 82,778 | ||||
| Total liabilities, Series A preferred shares and shareholders equity | $ | 200,512 | $ | 279,238 |
WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share amounts)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Revenue | $ | 36,423 | $ | 3,450 | $ | 39,199 | $ | 10,638 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 31,086 | 28,275 | 96,114 | 100,911 | ||||||||||||
| General and administrative | 12,944 | 9,590 | 33,991 | 32,791 | ||||||||||||
| Total operating expenses | 44,030 | 37,865 | 130,105 | 133,702 | ||||||||||||
| Loss from operations | (7,607 | ) | (34,415 | ) | (90,906 | ) | (123,064 | ) | ||||||||
| Other income, net: | ||||||||||||||||
| Dividend income and interest income, net | 6 | 23 | 25 | 544 | ||||||||||||
| Other income, net | 1,371 | 1,292 | 3,421 | 1,399 | ||||||||||||
| Total other income, net | 1,377 | 1,315 | 3,446 | 1,943 | ||||||||||||
| Loss before income taxes | (6,230 | ) | (33,100 | ) | (87,460 | ) | (121,121 | ) | ||||||||
| Income tax provision | ||||||||||||||||
| Net loss | $ | (6,230 | ) | $ | (33,100 | ) | $ | (87,460 | ) | $ | (121,121 | ) | ||||
| Net loss per share attributable to ordinary shareholders basic and diluted | $ | (0.12 | ) | $ | (0.86 | ) | $ | (1.75 | ) | $ | (3.36 | ) | ||||
| Weighted-average ordinary shares used in computing net loss per share attributable to ordinary shareholders basic and diluted | 50,709,877 | 38,364,224 | 50,017,521 | 36,021,256 | ||||||||||||
| Other comprehensive income (loss): | ||||||||||||||||
| Net loss | $ | (6,230 | ) | $ | (33,100 | ) | $ | (87,460 | ) | $ | (121,121 | ) | ||||
| Foreign currency translation | (11 | ) | 23 | (131 | ) | 34 | ||||||||||
| Comprehensive loss | $ | (6,241 | ) | $ | (33,077 | ) | $ | (87,591 | ) | $ | (121,087 | ) |