Wave Life Sciences Reports First Quarter 2018 Financial Results and Provides Business Update
“The strong momentum we achieved in 2017 with our clinical development programs and as an organization continued through the first quarter,” said
First Quarter Highlights and Business Update
- Ongoing and planned clinical trials on track
The 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’s manufacturing facility completes inaugural good manufacturing practice (GMP) campaigns
Wave is now producing clinical material in the GMP manufacturing suite at its
Lexington, Massachusettsfacility. The company recently completed manufacturing runs and material from these campaigns will support the development programs, including those for ALS and FTD.
July 2017, 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.
- Takeda collaboration takes effect: at least
$230 millionin committed cash
April 2018, Wave’s global strategic collaboration with Takeda Pharmaceutical Company Limitedto 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 millionin cash under the collaboration. In addition, Takeda is required to fund at least $60 millionof Wave research over a four-year period to advance multiple preclinical targets.
- Data presented in April at scientific and medical conferences
Wave presented preclinical in vivo data at the
European Association for the Studyof the Liver’s annual meeting, the International Liver Congress2018, 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.
- Neuromuscular research collaboration with Deep Genomics established to identify novel splicing targets
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.
- Pfizer nominates maximum number of hepatic targets; collaboration moving toward candidate selection
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
Research and development expenses were
General and administrative expenses were
Wave ended the first quarter of 2018 with
The company expects that its cash and cash equivalents, together with the
About Wave Life Sciences
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
UNAUDITED CONSOLIDATED BALANCE SHEETS
(In thousands, except share amounts)
|March 31, 2018||December 31, 2017|
|Cash and cash equivalents||$||110,491||$||142,503|
|Prepaid expenses and other current assets||7,470||7,985|
|Total current assets||117,961||150,488|
|Property and equipment, net||28,778||27,334|
|Total long-term assets||32,460||31,355|
|Liabilities, Series A preferred shares and shareholders’ equity|
|Accrued expenses and other current liabilities||6,461||8,898|
|Current portion of capital lease obligation||—||16|
|Current portion of deferred rent||70||60|
|Current portion of deferred revenue||1,275||1,275|
|Current portion of lease incentive obligation||478||344|
|Total current liabilities||16,298||18,191|
|Deferred rent, net of current portion||4,591||4,214|
|Deferred revenue, net of current portion||5,819||7,241|
|Lease incentive obligation, net of current portion||4,185||3,094|
|Total long-term liabilities||16,200||16,168|
|Series A preferred shares, no par value; 3,901,348 shares issued and outstanding
at March 31, 2018 and December 31, 2017
|Ordinary shares, no par value; 27,993,337 and 27,829,079 shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively||$||311,591||$||310,038|
|Additional paid-in capital||26,602||22,172|
|Accumulated other comprehensive income||165||116|
|Total shareholders’ equity||$||110,049||$||139,610|
|Total liabilities, Series A preferred shares and shareholders’ equity||$||150,421||$||181,843|
WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share amounts)
|Three Months Ended March 31,|
|Research and development||29,196||14,740|
|General and administrative||8,001||5,850|
|Total operating expenses||37,197||20,590|
|Loss from operations||(35,775)||(20,207)|
|Other income (expense), net:|
|Interest income (expense), net||7||3|
|Other income (expense), net||343||(72)|
|Total other income (expense), net||706||221|
|Loss before income taxes||(35,069)||(19,986)|
|Income tax provision||(172)||(1,110)|
|Net loss per share attributable to ordinary shareholders—basic and diluted||$||(1.26)||$||(0.90)|
|Weighted-average ordinary shares used in computing net loss per share
attributable to ordinary shareholders—basic and diluted
|Other comprehensive income (loss):|
|Foreign currency translation||$||49||$||15|
Source: Wave Life Sciences