Full Press Release Details
Wave Life Sciences Reports First Quarter 2018 Financial Results and Provides Business Update
CAMBRIDGE, Mass., May 9, 2018 Wave Life Sciences Ltd. (NASDAQ: WVE), a biotechnology company focused on delivering
transformational therapies for patients with serious, genetically-defined diseases, today announced financial results for the first quarter ended March 31, 2018, and provided a business update.
The strong momentum we achieved in 2017 with our clinical development programs and as an organization continued through the first quarter, said
Paul Bolno, MD, MBA, Chief Executive Officer and President of Wave Life Sciences. We are seeing significant interest from patients and the medical community in our first three clinical studies in Huntington s disease and Duchenne muscular
dystrophy and look forward to initiating three additional development programs by the end of the year.
First Quarter Highlights and Business
PRECISION-HD program, which consists of two global Phase 1b/2a clinical trials evaluating WVE-120101 and WVE-120102 for patients
with Huntington s disease, continues to enroll patients and the company is on track to report topline data in the first half of 2019.
The global Phase 1 clinical trial, testing WVE-210201 for the treatment of Duchenne muscular dystrophy
(DMD) patients amenable to exon 51 skipping, continues to enroll patients. Safety data from the trial are anticipated in the third quarter of 2018.
The company intends to initiate clinical trials of WVE-3972-01
in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) in the fourth quarter of 2018. Wave s next DMD development program will target exon 53, with clinical trials expected to initiate in the first quarter of 2019.
Wave is now producing clinical material in the GMP manufacturing suite at its Lexington, Massachusetts facility. The company recently completed
manufacturing runs and material from these campaigns will support the development programs, including those for ALS and FTD.
Wave took occupancy of the new facility which was designed to provide greater independence and flexibility in conducting clinical trials, secure material for current and future development activities and provide the capability for commercial-scale
manufacturing. The company is continuing to build out the 90,000-square foot facility with process development, quality control and analytical development laboratories.
April 2018, Wave s global strategic collaboration with Takeda Pharmaceutical Company Limited to discover, develop and commercialize nucleic acid therapies for disorders of the central nervous system took effect when Wave closed on the issuance
and sale of 1,096,892 ordinary shares to Takeda and received aggregate cash proceeds of $60 million. Wave also received an upfront payment of $110 million in cash under the collaboration. In addition, Takeda is required to fund at least
$60 million of Wave research over a four-year period to advance multiple preclinical targets.
preclinical in vivo data at the European Association for the Study of the Liver s annual meeting, the International Liver Congress 2018, demonstrating that controlling stereochemistry increases both the duration and the potency of
GalNAc-conjugated apolipoprotein C-III (APOC3) antisense oligonucleotides, thereby potentially enhancing the pharmacological properties of stereopure oligonucleotides for APOC3 targeting in the clinic.
At the 70th Annual Meeting of the American Academy of Neurology, Dr. Robert H. Brown, Jr., Chair and Professor of Neurology at
the University of Massachusetts Medical School, presented data from preclinical studies of WVE-3972-01, Wave s investigational stereopure antisense oligonucleotide
designed to target the pathogenic allele of the C9ORF72 gene for the treatment of ALS and FTD. In preclinical in vivo studies, WVE-3972-01 demonstrated a
potent, sustained and preferential knockdown of toxic biomarkers associated with ALS and FTD.
In April, Wave formed a collaboration with Deep Genomics, Inc. to identify novel therapies to be developed by Wave for the treatment of genetic
neuromuscular disorders. Under the collaboration, the companies will analyze and test oligonucleotides against potential therapeutic targets within multiple genes implicated in neuromuscular disorders. The collaboration aims to increase the size of
patient populations that may be eligible for treatment by expanding the universe of druggable splicing targets beyond DMD and spinal muscular atrophy.
On May 7, 2018, Wave announced that Pfizer recently nominated the fourth and fifth final hepatic targets under the collaboration agreement
between the two companies to develop genetically targeted therapies for the treatment of metabolic hepatic diseases, such as nonalcoholic steatohepatitis. Once candidates have been developed, Pfizer may elect to exclusively license the programs and
undertake further development and potential commercialization.
First Quarter 2018 Financial Results and Financial Guidance
Wave reported a net loss of $35.2 million in the first quarter of 2018 as compared to $21.1 million in the same period in 2017. The increase in net
loss in the first quarter of 2018 was primarily driven by increases in research and development efforts, infrastructure investments and employee headcount to support Wave s corporate goals.
Research and development expenses were $29.2 million in the first quarter of 2018 as compared to $14.7 million in the same period in 2017. The
increase in research and development expenses in the first quarter of 2018 was primarily driven by increases in research, preclinical and clinical investments, further expansion of our manufacturing capabilities and facility-related expenses to
continue to advance Wave s expanding pipeline.
General and administrative expenses were $8.0 million in the first quarter of 2018 as compared
to $5.9 million in the same period in 2017. The increase in general and administrative expenses in the first quarter of 2018 was primarily driven by the continued growth in Wave s employee headcount, as well as increases in
facility-related expenses and other general operating expenses.
Wave ended the first quarter of 2018 with $110.5 million in cash and cash equivalents as compared to
$142.5 million as of December 31, 2017. The decrease in cash and cash equivalents in the first quarter of 2018 was primarily the result of Wave s quarterly net loss of $35.2 million.
The company expects that its cash and cash equivalents, together with the $170.0 million of cash received from Takeda in April 2018, will enable it to
fund its operating and capital expenditure requirements to the end of 2020.
About Wave Life Sciences
Wave Life Sciences is a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases. Its
chemistry platform enables the creation of highly specific, well characterized oligonucleotides designed to deliver superior efficacy and safety across multiple therapeutic modalities. The company s pipeline is initially focused on neurological
disorders and extends across several other therapeutic areas. For more information, please visit www.wavelifesciences.com.
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 duration of our clinical trials; the protocol,
design and endpoints of our clinical trials; the future performance and results of our programs in clinical trials; 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 in clinical trials; our identification of future candidates and their therapeutic potential; the anticipated therapeutic benefits of our potential therapies compared to others; our advancing of
therapies across multiple modalities and the anticipated benefits of that strategy; the anticipated benefits of our manufacturing process and our internal manufacturing facility; our future growth; the potential benefits of our stereopure compounds
compared with stereorandom compounds, our drug discovery platform and nucleic acid therapeutics generally; 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: 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 success of our platform in identifying viable candidates; the continued development and acceptance of nucleic acid therapeutics as a
class of drugs; 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 our
collaborators and partners; our ability to manufacture drug material; 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; our ability to finance our drug discovery and development efforts and to raise additional capital when needed; 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)
| March 31, 2018 | December 31, 2017 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 110,491 | $ | 142,503 | ||||
| Prepaid expenses and other current assets | 7,470 | 7,985 | ||||||
| Total current assets | 117,961 | 150,488 | ||||||
| Long-term assets: | ||||||||
| Property and equipment, net | 28,778 | 27,334 | ||||||
| Restricted cash | 3,612 | 3,610 | ||||||
| Other assets | 70 | 411 | ||||||
| Total long-term assets | 32,460 | 31,355 | ||||||
| Total assets | $ | 150,421 | $ | 181,843 | ||||
| Liabilities, Series A preferred shares and shareholders equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 8,014 | $ | 7,598 | ||||
| Accrued expenses and other current liabilities | 6,461 | 8,898 | ||||||
| Current portion of capital lease obligation | 16 | |||||||
| Current portion of deferred rent | 70 | 60 | ||||||
| Current portion of deferred revenue | 1,275 | 1,275 | ||||||
| Current portion of lease incentive obligation | 478 | 344 | ||||||
| Total current liabilities | 16,298 | 18,191 | ||||||
| Long-term liabilities: | ||||||||
| Deferred rent, net of current portion | 4,591 | 4,214 | ||||||
| Deferred revenue, net of current portion | 5,819 | 7,241 | ||||||
| Lease incentive obligation, net of current portion | 4,185 | 3,094 | ||||||
| Other liabilities | 1,605 | 1,619 | ||||||
| Total long-term liabilities | 16,200 | 16,168 | ||||||
| Total liabilities | $ | 32,498 | $ | 34,359 | ||||
| Series A preferred shares, no par value; 3,901,348 shares issued and outstanding at March 31, 2018 and December 31, 2017 | $ | 7,874 | $ | 7,874 | ||||
| Shareholders equity: | ||||||||
| Ordinary shares, no par value; 27,993,337 and 27,829,079 shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively | $ | 311,591 | $ | 310,038 | ||||
| Additional paid-in capital | 26,602 | 22,172 | ||||||
| Accumulated other comprehensive income | 165 | 116 | ||||||
| Accumulated deficit | (228,309 | ) | (192,716 | ) | ||||
| Total shareholders equity | $ | 110,049 | $ | 139,610 | ||||
| Total liabilities, Series A preferred shares and shareholders equity | $ | 150,421 | $ | 181,843 |
WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share amounts)
| Three Months Ended March 31, | ||||||||
| 2018 | 2017 | |||||||
| Revenue | $ | 1,422 | $ | 383 | ||||
| Operating expenses: | ||||||||
| Research and development | 29,196 | 14,740 | ||||||
| General and administrative | 8,001 | 5,850 | ||||||
| Total operating expenses | 37,197 | 20,590 | ||||||
| Loss from operations | (35,775 | ) | (20,207 | ) | ||||
| Other income (expense), net: | ||||||||
| Dividend income | 356 | 290 | ||||||
| Interest income (expense), net | 7 | 3 | ||||||
| Other income (expense), net | 343 | (72 | ) | |||||
| Total other income (expense), net | 706 | 221 | ||||||
| Loss before income taxes | (35,069 | ) | (19,986 | ) | ||||
| Income tax provision | (172 | ) | (1,110 | ) | ||||
| Net loss | $ | (35,241 | ) | $ | (21,096 | ) | ||
| Net loss per share attributable to ordinary shareholders basic and diluted | $ | (1.26 | ) | $ | (0.90 | ) | ||
| Weighted-average ordinary shares used in computing net loss per share attributable to ordinary shareholders basic and diluted | 27,919,063 | 23,531,788 | ||||||
| Other comprehensive income (loss): | ||||||||
| Foreign currency translation | $ | 49 | $ | 15 | ||||
| Comprehensive loss | $ | (35,192 | ) | $ | (21,081 | ) |