Full Press Release Details
Wave Life Sciences Reports Second Quarter 2020 Financial Results and Provides Business Update
Data from 32 mg cohorts of both PRECISION-HD trials and
PRECISION-HD OLE trials expected in 1Q 2021
C9orf72 and SNP3 clinical trial applications on
track to be submitted in 4Q 2020
ADAR editing advancing with additional in vivo data expected at upcoming Analyst &
Investor Research Webcast
Wave to host investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., August 10, 2020 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 second quarter ended June 30, 2020 and provided a business update.
In the second quarter, despite persistent global challenges due to the COVID-19 pandemic, we continued to
execute on our clinical and preclinical neurology programs. Our clinical, laboratory and manufacturing teams have proven to be adaptable and resilient as they work amidst new operating environments, said Paul Bolno, MD, MBA, President and
Chief Executive Officer of Wave Life Sciences. We are on track to submit clinical trial applications for our C9orf72 program for amyotrophic lateral sclerosis and frontotemporal dementia and our SNP3 program for HD in the fourth quarter of
this year, both of which are designed with a novel chemistry application from our PRISM platform. During our upcoming Analyst & Investor Research Webcast on August 25, we plan to share exciting updates on our platform, including
advances in oligonucleotide chemistry and our promising ADAR editing modality, and new preclinical data for our C9orf72 program.
PRECISION-HD programs for Huntington s disease (HD): Wave is developing a unique
portfolio of investigational stereopure oligonucleotides designed to selectively target the mutant allele of the huntingtin (mHTT) gene, while leaving the wild-type (wtHTT) protein relatively intact.
PRECISION-HD trials:
SNP3 program for HD: Wave is advancing a third allele-selective HD program, which is designed to selectively target an undisclosed SNP on the mHTT mRNA
transcript (SNP3), while leaving the wild-type (wtHTT) protein relatively intact.
C9orf72 program for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia
(FTD): Wave s C9orf72 program is designed to selectively target the transcripts containing the hexanucleotide repeat expansion (G4C2) in the C9orf72 gene.
Central nervous system (CNS) programs in
collaboration with Takeda: Wave is leveraging its learnings from PRISMTM, its proprietary discovery and drug development platform, to design additional stereopure oligonucleotides with
optimized profiles for CNS indications, including Alzheimer s disease, Parkinson s disease and others, as part of its ongoing collaboration with Takeda.
ADAR editing: Wave is advancing a novel RNA editing platform capability
using endogenous ADAR (adenosine deaminases acting on RNA) enzymes via free uptake (non-viral, no nanoparticles) of A-to-I base
editing oligonucleotides, which has the potential to be a best-in-class RNA editing modality.
Strengthening leadership team: In May 2020, Wave appointed Kenneth Rhodes, PhD, as Senior Vice President, Therapeutics Discovery. Dr. Rhodes is
responsible for defining the strategy and guiding discovery research to design new therapeutic candidates and advance them to the clinic, with an initial focus on neurological diseases.
Analyst and Investor Research Webcast: Wave is scheduled to hold an Analyst and Investor Research Webcast to discuss its latest PRISM platform
advancements and neurology-focused oligonucleotide pipeline on Tuesday, August 25th, from 10:00 a.m. 11:30 a.m.
Second Quarter 2020 Financial Results and Financial Guidance
Wave reported a net loss of $40.5 million in the second quarter of 2020 as compared to $41.9 million in the same period in 2019.
Research and development expenses were $31.5 million in the second quarter of 2020 as compared to $41.6 million in the same period in 2019. The
decrease in research and development expenses in the second quarter was primarily due to decreased external expenses related to suvodirsen due to our December 2019 decision to discontinue the program, partially offset by increased external expenses
related to our clinical and preclinical activities, including our HD and C9orf72 programs for ALS and FTD.
General and administrative expenses were
$10.2 million in the second quarter of 2020 as compared to $11.6 million in the same period in 2019. The decrease in general and administrative expenses in the second quarter of 2020 was mainly driven by decreased headcount resulting from
the workforce reduction implemented in February 2020.
As of June 30, 2020, Wave had $94.1 million in cash and cash equivalents as compared to
$147.2 million as of December 31, 2019. The decrease in cash and cash equivalents was mainly due to Wave s year-to-date net loss of $88 million,
partially offset by the receipt of $20 million in research support funding from Takeda under our collaboration and $12 million in net proceeds under our
at-the-market equity program.
Wave expects that its existing cash and
cash equivalents, together with expected and committed cash from its existing collaboration will enable the company to fund its operating and capital expenditure requirements into the fourth quarter of 2021.
Investor Conference Call and Webcast
will host an investor conference call today at 8:30 a.m. ET to discuss the company s second quarter 2020 operating results and provide a business update. The conference call may be accessed by dialing (866)
220-8068 (domestic) or +1 (470) 495-9153 (international) and entering conference ID 4791316. 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. 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 artificial
intelligence-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
About Wave Life Sciences
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 PRISMTM, 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 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 commencement, patient enrollment, data readouts and completion of our 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 of future 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 anticipated benefits of our proprietary manufacturing processes and our
internal manufacturing capabilities; the potential benefits of PRISM and our stereopure oligonucleotides compared with stereorandom oligonucleotides; the benefit of nucleic acid therapeutics generally; the strength of our intellectual property; the
anticipated duration of our cash runway; 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 severity and duration of the COVID-19 pandemic and its potentially negative impact on the conduct of, and the timing of enrollment, completion and reporting with respect to, our clinical trials; any other impacts on our business as a result of
or related to the COVID-19 pandemic; 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; our effectiveness in managing future clinical trials and regulatory interactions; the effectiveness of PRISM; 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; and competition from others developing therapies for similar indications, 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)
| June 30, 2020 | December 31, 2019 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 94,054 | $ | 147,161 | ||||
| Current portion of accounts receivable | 30,000 | 20,000 | ||||||
| Prepaid expenses | 6,452 | 9,626 | ||||||
| Other current assets | 16,328 | 8,689 | ||||||
| Total current assets | 146,834 | 185,476 | ||||||
| Long-term assets: | ||||||||
| Accounts receivable, net of current portion | 30,000 | |||||||
| Property and equipment, net | 33,096 | 36,368 | ||||||
| Operating lease right-of-use assets | 17,201 | 18,101 | ||||||
| Restricted cash | 3,650 | 3,647 | ||||||
| Other assets | 3,170 | 10,658 | ||||||
| Total long-term assets | 57,117 | 98,774 | ||||||
| Total assets | $ | 203,951 | $ | 284,250 | ||||
| Liabilities, Series A preferred shares and shareholders equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 13,910 | $ | 9,073 | ||||
| Accrued expenses and other current liabilities | 8,220 | 16,185 | ||||||
| Current portion of deferred revenue | 84,849 | 89,652 | ||||||
| Current portion of operating lease liability | 3,473 | 3,243 | ||||||
| Total current liabilities | 110,452 | 118,153 | ||||||
| Long-term liabilities: | ||||||||
| Deferred revenue, net of current portion | 61,081 | 63,466 | ||||||
| Operating lease liability, net of current portion | 27,513 | 29,304 | ||||||
| Other liabilities | 1,520 | 1,721 | ||||||
| Total long-term liabilities | $ | 90,114 | $ | 94,491 | ||||
| Total liabilities | $ | 200,566 | $ | 212,644 | ||||
| Series A preferred shares, no par value; 3,901,348 shares issued and outstanding at June 30, 2020 and December 31, 2019 | $ | 7,874 | $ | 7,874 | ||||
| Shareholders equity: | ||||||||
| Ordinary shares, no par value; 35,732,154 and 34,340,690 shares issued and outstanding at June 30, 2020 and December 31, 2019, respectively | $ | 551,543 | $ | 539,547 | ||||
| Additional paid-in capital | 65,070 | 57,277 | ||||||
| Accumulated other comprehensive income | 278 | 267 | ||||||
| Accumulated deficit | (621,380 | ) | (533,359 | ) | ||||
| Total shareholders equity | $ | (4,489 | ) | $ | 63,732 | |||
| Total liabilities, Series A preferred shares and shareholders equity | $ | 203,951 | $ | 284,250 |
The accompanying notes are an integral part of the unaudited consolidated financial statements.
WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share amounts)
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Revenue | $ | 3,027 | $ | 7,628 | $ | 7,188 | $ | 10,654 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 31,478 | 41,605 | 72,636 | 81,718 | ||||||||||||
| General and administrative | 10,205 | 11,640 | 23,201 | 22,541 | ||||||||||||
| Total operating expenses | 41,683 | 53,245 | 95,837 | 104,259 | ||||||||||||
| Loss from operations | (38,656 | ) | (45,617 | ) | (88,649 | ) | (93,605 | ) | ||||||||
| Other income (expense), net: | ||||||||||||||||
| Dividend income | 135 | 1,544 | 520 | 2,968 | ||||||||||||
| Interest income (expense), net | (2 | ) | 8 | 1 | 19 | |||||||||||
| Other income (expense), net | (2,005 | ) | 2,123 | 107 | 4,476 | |||||||||||
| Total other income (expense), net | (1,872 | ) | 3,675 | 628 | 7,463 | |||||||||||
| Loss before income taxes | (40,528 | ) | (41,942 | ) | (88,021 | ) | (86,142 | ) | ||||||||
| Income tax provision | ||||||||||||||||
| Net loss | $ | (40,528 | ) | $ | (41,942 | ) | $ | (88,021 | ) | $ | (86,142 | ) | ||||
| Net loss per share attributable to ordinary shareholders basic and diluted | $ | (1.15 | ) | $ | (1.22 | ) | $ | (2.53 | ) | $ | (2.58 | ) | ||||
| Weighted-average ordinary shares used in computing net loss per share attributable to ordinary shareholders basic and diluted | 35,212,291 | 34,260,298 | 34,836,898 | 33,433,322 | ||||||||||||
| Other comprehensive income (loss): | ||||||||||||||||
| Net loss | $ | (40,528 | ) | $ | (41,942 | ) | $ | (88,021 | ) | $ | (86,142 | ) | ||||
| Foreign currency translation | 5 | 30 | 11 | 127 | ||||||||||||
| Comprehensive loss | $ | (40,523 | ) | $ | (41,912 | ) | $ | (88,010 | ) | $ | (86,015 | ) |
The accompanying notes are an integral part of the unaudited consolidated financial statements.