Wave Life Sciences Reports Third Quarter 2018 Financial Results and Provides Business Update
“During the third quarter we maintained strong momentum in advancing our three clinical programs and expect to announce our company’s first clinical readout later this year when we share topline safety results from the Phase 1 trial for WVE-210201, our investigational exon 51 skipping candidate for the treatment of Duchenne muscular dystrophy,” said
Third Quarter Highlights and Business Updates
- WVE-210201 DMD exon 51 skipping program
Wave expects to announce topline safety data from its Phase 1 clinical trial evaluating WVE-210201 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping by the end of the fourth quarter of 2018. As patients complete the Phase 1 trial, they have the option to enroll in an ongoing open-label extension study in which they continue to receive WVE-210201.
In addition, the company remains on track to deliver an interim analysis of dystrophin expression in muscle biopsies from its ongoing open-label extension study in the second half of 2019.
Wave has designed a global, pivotal, placebo-controlled Phase 2/3 efficacy and safety clinical trial of WVE-210201 in DMD patients amenable to exon 51 skipping, informed by ongoing discussions with regulatory authorities and the DMD patient community. The study will be powered to assess clinical efficacy and will include dystrophin expression readouts as part of interim and final analyses.
- WVE-N531 DMD exon 53 skipping program and additional DMD exon skipping programs
Wave is leveraging learnings from its ongoing DMD development and discovery efforts to advance WVE-N531, its preclinical program to target DMD in boys amenable to exon 53 skipping. Recent data presented at the 23rd International Annual Congress of the
World Muscle Societydemonstrated that WVE-N531 induced up to 71% dystrophin protein restoration in vitro in DMD patient-derived myoblasts compared with healthy human myoblasts as measured by Western Blot. Subject to submission of clinical trial applications and approval to proceed, Wave expects to deliver topline clinical data for WVE‑N531 in the second half of 2020.
The company is also expanding its DMD discovery programs to explore additional exon targets beyond its current exon 51 and exon 53 skipping programs, specifically including exons 44, 45, 52, 54 and 55.
- Pipeline expansion to rare, genetic eye diseases
Last month, Wave announced plans to design and advance stereopure oligonucleotide therapeutics for the potential treatment of inherited retinal diseases. Wave’s research in ophthalmology is assessing four inherited retinal diseases, which typically begin in childhood or adolescence and commonly lead to progressive vision loss: retinitis pigmentosa due to a P23H mutation in the RHO gene, Stargardt disease, Usher syndrome type 2A and Leber congenital amaurosis 10.
Wave’s decision to expand its therapeutic pipeline into ophthalmology is supported by its data presented at the 14th Annual Meeting of the
Oligonucleotide Therapeutics Societyin October 2018. The data demonstrate that a single intravitreal injection of stereopure oligonucleotide in the eye of non-human primates resulted in greater than 95% knockdown of a target RNA in the retina for at least four months. Based on these data, the company is working to design development candidates that could achieve a therapeutic effect with only two doses per year.
Wave expects to announce its first ophthalmology development candidate in the second half of 2019.
- PRECISION-HD Phase 1b/2a clinical trials
The PRECISION-HD program, which consists of two global Phase 1b/2a clinical trials evaluating investigational therapies WVE-120101 and WVE-120102 for patients with Huntington’s disease, remains on track to deliver topline data in the first half of 2019. An open-label extension study to assess safety, tolerability and efficacy using validated clinical outcome measures is planned for patients as they complete the ongoing Phase 1b/2a trials.
- Continued validation of Wave’s stereochemistry platform
Wave shared advancements related to its novel stereochemistry platform at the 14th Annual Meeting of the
Oligonucleotide Therapeutics Societyin October 2018. The company’s latest findings provide further validation of Wave’s platform to precisely design, optimize and manufacture stereopure oligonucleotides. Presentations included preclinical data demonstrating that Wave’s stereochemical control of antisense oligonucleotides enhances target efficacy and enables broad tissue distribution and exposure.
Third 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 third quarter of 2018 with
Wave expects that its existing cash and cash equivalents, together with expected and committed cash from existing collaborations, will enable it to fund its operating and capital expenditure requirements to the end of 2020.
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 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; 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 model; 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; 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 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 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
UNAUDITED CONSOLIDATED BALANCE SHEETS
(In thousands, except share amounts)
|September 30, 2018||December 31, 2017|
|Cash and cash equivalents||$||210,489||$||142,503|
|Current portion of accounts receivable||10,000||1,000|
|Prepaid expenses and other current assets||12,672||6,985|
|Total current assets||233,161||150,488|
|Accounts receivable, net of current portion||50,000||—|
|Property and equipment, net||37,722||27,334|
|Total long-term assets||91,416||31,355|
|Liabilities, Series A preferred shares and shareholders’ equity|
|Accrued expenses and other current liabilities||9,518||8,898|
|Current portion of capital lease obligation||—||16|
|Current portion of deferred rent||90||60|
|Current portion of deferred revenue||103,229||1,275|
|Current portion of lease incentive obligation||997||344|
|Total current liabilities||125,795||18,191|
|Deferred rent, net of current portion||5,084||4,214|
|Deferred revenue, net of current portion||69,494||7,241|
|Lease incentive obligation, net of current portion||8,229||3,094|
|Total long-term liabilities||84,302||16,168|
|Series A preferred shares, no par value; 3,901,348 shares issued and
outstanding at September 30, 2018 and December 31, 2017
|Ordinary shares, no par value; 29,426,176 and 27,829,079 shares issued
and outstanding at September 30, 2018 and December 31, 2017, respectively
|Additional paid-in capital||33,757||22,172|
|Accumulated other comprehensive income||181||116|
|Total shareholders’ equity||$||106,606||$||139,610|
|Total liabilities, Series A preferred shares and shareholders’ equity||$||324,577||$||181,843|
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||32,876||20,097||94,619||53,940|
|General and administrative||9,849||7,571||26,755||20,088|
|Total operating expenses||42,725||27,668||121,374||74,028|
|Loss from operations||(38,232||)||(26,353||)||(110,580||)||(71,233||)|
|Other income (expense), net:|
|Interest income (expense), net||5||1||16||5|
|Other income (expense), net||(468||)||(75||)||(384||)||(211||)|
|Total other income (expense), net||601||441||1,986||1,081|
|Loss before income taxes||(37,631||)||(25,912||)||(108,594||)||(70,152||)|
|Income tax benefit (provision)||—||418||(172||)||(1,035||)|
|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||(20||)||1||65||19|
Source: Wave Life Sciences