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Fast Track designation for suvodirsen received from the U.S. FDA
Interim analysis of dystrophin expression from suvodirsen open-label extension study expected in 4Q 2019
Topline data from PRECISION-HD2 clinical trial expected by year-end
Key hires and board expansion mark continued progress towards commercial preparedness in the U.S.
CAMBRIDGE, Mass., Nov. 05, 2019 (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, 2019 and provided a business update.
“We continued our strong execution in the third quarter and, as a result, we are on track to deliver key clinical data readouts in the fourth quarter from our open-label extension study of suvodirsen in Duchenne muscular dystrophy and from PRECISION-HD2, the first of our two Phase 1b/2a trials in Huntington’s disease,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “In anticipation of our potential launch of suvodirsen in the United States and future late-stage programs, we hired a Chief Commercial Officer with proven experience in neurology and rare disease and named three new Board members with deep expertise in clinical development, product commercialization and government and payor relations.”
“As we prepare for our near-term milestones, we continue to advance our platform, PRISM, with new preclinical programs such as our SNP3 program in Huntington’s disease and our lead ophthalmology program for Usher Syndrome Type 2A,” continued Dr. Bolno. “In addition, we’re very excited about the latest modality to emerge from PRISM, ADAR-mediated RNA editing, which we presented during our Analyst and Investor Research Day in early October.”
Wave is committed to building a fully integrated genetic medicines company led by its clinical and preclinical programs for the treatment of neuromuscular, central nervous system and ophthalmologic diseases.
Suvodirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping
Additional exon skipping programs for patients with Duchenne muscular dystrophy
Central nervous system (CNS) diseases
PRECISION-HD clinical program evaluating WVE-120101 and WVE-120102 in Huntington’s disease
Allele-selective approach to treating Huntington’s disease
CNS disease pipeline
PRISM: next generation modalities
Third Quarter 2019 Financial Results and Financial Guidance
Wave reported a net loss of $50.7 million in the third quarter of 2019 as compared to $37.6 million in the same period in 2018. The increase in net loss in the third quarter of 2019 was largely driven by increased research and development efforts and continued organizational growth to support Wave’s corporate goals.
Research and development expenses were $44.6 million in the third quarter of 2019 as compared to $32.9 million in the same period in 2018. The increase in research and development expenses in the third quarter of 2019 was primarily due to increased external expenses related to our suvodirsen clinical activities as well as increased investments in PRISM and other research and development expenses.
General and administrative expenses were $12.5 million in the third quarter of 2019 as compared to $9.8 million in the same period in 2018. The increase in general and administrative expenses in the third quarter of 2019 was mainly driven by continued organizational growth to support Wave’s corporate goals.
As of September 30, 2019, Wave had $209.0 million in cash and cash equivalents as compared to $174.8 million as of December 31, 2018. The increase in cash and cash equivalents was mainly due to the $161.8 million in net proceeds from the January 2019 follow-on offering, partially offset by Wave’s year-to-date net loss of $136.9 million.
Wave expects that its existing cash and cash equivalents, together with expected and committed cash from existing collaborations, will enable Wave to fund its operating and capital expenditure requirements to the end of 2020.
About PRISM ™
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 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.
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 potential commercial launch of our product candidates; 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 facility; our future growth and anticipated transition to a fully integrated commercial-stage company; 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; and the anticipated duration of our cash runway. 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 continue to build and 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 processes; 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 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 uses, 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, 2019||December 31, 2018|
|Cash and cash equivalents||$||209,009||$||174,819|
|Current portion of accounts receivable||20,000||10,000|
|Prepaid expenses and other current assets||21,249||17,454|
|Total current assets||250,258||202,273|
|Accounts receivable, net of current portion||30,000||50,000|
|Property and equipment, net||37,204||39,931|
|Operating lease right-of-use assets||18,527||—|
|Total long-term assets||96,954||93,667|
|Liabilities, Series A preferred shares and shareholders’ equity|
|Accrued expenses and other current liabilities||13,738||14,736|
|Current portion of deferred rent||—||115|
|Current portion of deferred revenue||96,322||100,945|
|Current portion of lease incentive obligation||—||1,156|
|Current portion of operating lease liability||3,132||—|
|Total current liabilities||133,411||130,041|
|Deferred rent, net of current portion||—||5,132|
|Deferred revenue, net of current portion||59,196||68,156|
|Lease incentive obligation, net of current portion||—||9,247|
|Operating lease liability, net of current portion||30,165||—|
|Total long-term liabilities||$||91,154||$||84,677|
|Series A preferred shares, no par value; 3,901,348 shares issued and
outstanding at September 30, 2019 and December 31, 2018
|Ordinary shares, no par value; 34,284,217 and 29,472,197 shares issued
and outstanding at September 30, 2019 and December 31, 2018, respectively
|Additional paid-in capital||52,290||37,768|
|Accumulated other comprehensive income||282||153|
|Total shareholders’ equity||$||114,773||$||73,348|
|Total liabilities, Series A preferred shares and shareholders’ equity||$||347,212||$||295,940|
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,|
|Research and development||44,585||32,876||126,303||94,619|
|General and administrative||12,523||9,849||35,064||26,755|
|Total operating expenses||57,108||42,725||161,367||121,374|
|Loss from operations||(54,179||)||(38,232||)||(147,784||)||(110,580||)|
|Other income, net:|
|Interest income, net||6||5||25||16|
|Other income (expense), net||2,239||(468||)||6,715||(384||)|
|Total other income, net||3,453||601||10,916||1,986|
|Loss before income taxes||(50,726||)||(37,631||)||(136,868||)||(108,594||)|
|Income tax provision||—||—||—||(172||)|
|Net loss per share attributable to ordinary
shareholders—basic and diluted
|Weighted-average ordinary shares used in
computing net loss per share attributable to
ordinary shareholders—basic and diluted
|Other comprehensive income (loss):|
|Foreign currency translation||2||(20||)||129||65|
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