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 first quarter ended March 31, 2021 and provided a
business update.
“Despite our PRECISION-HD results at the end of the first
quarter, it has been a productive start of the year for Wave and
our team remains focused on advancing our clinical trials for
ALS/FTD, HD and DMD. These new trials mark the transition of our
next-generation programs into the clinic. We expect clinical data
that will provide insight into PN chemistry and enable decision
making on next steps for these programs next year,” said Paul
Bolno, MD, MBA, President and Chief Executive Officer of Wave Life
Sciences. “We have a deep and diverse pipeline of RNA therapeutics,
each designed with our PN chemistry, which has been shown to
increase potency, exposure and durability compared to our
first-generation compounds in preclinical studies. We continue to
produce compelling in vivo data, and we are advancing multiple
programs for CNS indications, including Alzheimer’s disease,
Parkinson’s disease and others, in collaboration with our partner
Takeda. Our ADAR editing capability demonstrates the diversity of
our genetic medicines toolkit and we are well-positioned to be
leaders in the RNA editing field. We look forward to providing more
updates on ADAR editing, including the first in vivo data from our
AATD program, in the first half of this year.”
Recent Business Highlights and Upcoming
Milestones
WVE-004 (C9orf72) for amyotrophic lateral sclerosis
(ALS) and frontotemporal dementia (FTD):
- WVE-004 is an investigational antisense oligonucleotide
designed to selectively target transcript variants containing a
hexanucleotide repeat expansion (G4C2) in the C9orf72 gene, which
is one of the most common genetic causes of the sporadic and
inherited forms of ALS and FTD. WVE-004 uses novel PN backbone
chemistry modifications.
- In February 2021, Wave published in Nature Communications the
results of initial work to identify and validate its targeting
strategy to achieve variant-selective knockdown of
expansion-containing C9orf72 transcripts.
- In April 2021, during a platform presentation at the American
Academy of Neurology (AAN) 2021 Virtual Annual Meeting, Wave
highlighted preclinical in vivo data for WVE-004, which
demonstrated potent and durable knockdown of more than 90% of
polyGP dipeptide repeat (DPR) proteins in the spinal cord and at
least 80% in the cortex, an effect that persisted for at least six
months. C9orf72 protein was relatively unchanged over the same time
period.
- This week, at the European Network to Cure ALS (ENCALS) meeting
being held May 12 – May 14, Wave is presenting a poster introducing
its FOCUS-C9 Phase 1b/2a trial design for WVE-004. The FOCUS-C9
trial is a global, multicenter, randomized, double-blind,
placebo-controlled Phase 1b/2a clinical trial to assess the safety
and tolerability of intrathecal doses of WVE-004 for patients with
C9-ALS and/or C9-FTD. Additional objectives include measurement of
polyGP proteins in the cerebrospinal fluid (CSF), plasma and CSF
pharmacokinetics, and exploratory biomarker and clinical endpoints.
The FOCUS-C9 trial is designed to be adaptive and includes single-
and multiple-ascending dose portions, with dose escalation and
dosing frequency being guided by an independent safety
committee.
- Wave has received regulatory and ethics approvals and site
activation is underway for the FOCUS-C9 clinical trial, and Wave
expects to initiate dosing in 2021.
WVE-003 (SNP3) for Huntington’s disease
(HD):
- WVE-003 is Wave’s next-generation HD candidate and Wave’s first
HD candidate that uses PN chemistry. WVE-003 is designed to
selectively target the mutant allele of the huntingtin (mHTT) gene,
while leaving the wild-type (wtHTT) protein relatively intact.
Wave’s approach to HD is guided by the recognition that, in
addition to a gain of function of the mHTT protein, people with
this disease have less wtHTT protein, leaving them with a smaller
protective reservoir of healthy protein than unaffected
individuals. A growing body of scientific evidence suggests that
preserving as much of this essential protein as possible, when in
the setting of stress from toxic mHTT protein, may be important for
favorable clinical outcomes.
- In April 2021, at the 16th Annual CHDI Foundation Huntington’s
Disease Therapeutic Conference, Wave highlighted preclinical data
for WVE-003, which showed selective reduction of mHTT mRNA in vitro
and potent and durable knockdown of mHTT mRNA in vivo. Wave also
introduced the design for the Phase 1b/2a clinical trial of
WVE-003, called SELECT-HD. The multicenter, randomized,
double-blind, placebo-controlled trial will assess the safety and
tolerability of intrathecally administered WVE-003 for patients
with early manifest Huntington’s disease. Additional objectives
include measurement of mHTT and wtHTT protein and exploratory
pharmacokinetic, pharmacodynamic, clinical and MRI endpoints. The
trial is designed to be adaptive, with dose escalation and dosing
frequency being guided by an independent safety committee.
- Wave has received regulatory and ethics approvals and site
activation is underway for the SELECT-HD clinical trial, and Wave
expects to initiate dosing in 2021.
WVE-N531 for Duchenne muscular dystrophy (DMD) amenable
to exon 53 skipping:
- WVE-N531 is Wave’s first splicing candidate to incorporate PN
chemistry, which Wave advanced following results of an in vivo
study in double knock-out mice (dKO) that showed that an
oligonucleotide designed with PN chemistry appeared to
significantly increase dystrophin production and substantially
improve survival, compared to oligonucleotides designed with Wave’s
first-generation chemistry.
- In March 2021, Wave initiated clinical development of WVE-N531
with the submission of a clinical trial
application.
- Wave has received regulatory approval for a clinical trial of
WVE-N531 to assess initial safety and dystrophin production in
patients with DMD amenable to exon 53 skipping. Wave expects to
initiate dosing in this trial in 2021.
ADAR editing:
- In March 2021, Wave presented a poster at the 2021 Keystone
eSymposia on Precision Engineering of the Genome, Epigenome and
Transcriptome highlighting the breadth of RNA editing data
generated using its ADAR editing capability to date. This
presentation illustrated editing activity across in vivo and in
vitro systems, including in vivo editing in the CNS, using
conjugated and non-conjugated oligonucleotides. Wave will also
present these data in an oral presentation at the 24th American
Society of Gene and Cell Therapy (ASGCT) Annual Meeting being held
this week, May 11 – 14, 2021.
- Wave expects to present additional ADAR editing data at
scientific congresses in 2021.
Alpha-1 antitrypsin deficiency (AATD) program with ADAR
editing:
- Wave’s AATD program, its first ADAR editing program, uses an
oligonucleotide to correct the single RNA base mutation in mRNA
coded by the SERPINA1 Z allele. ADAR editing may provide an ideal
approach to treating AATD by increasing circulating levels of
healthy alpha-1 antitrypsin (AAT) protein and reducing aggregation
in the liver, thus simultaneously addressing both the lung and
liver manifestations of the disease.
- To support the continued development of its AATD program, Wave
has developed a proprietary humanized SERPINA1/ADAR model. Wave
expects to share in vivo data from this model in the first half of
2021 and plans to submit these data for presentation at a
scientific congress in 2021.
First Quarter 2021 Financial Results and Financial
GuidanceWave reported a net loss of $42.5 million in the
first quarter of 2021 as compared to $47.5 million in the same
period in 2020.
Research and development expenses were $33.4 million in the
first quarter of 2021 as compared to $41.2 million in the same
period in 2020. The year-over-year decrease was primarily due to
the decrease in external expenses related to Wave’s suvodirsen
program, which was discontinued in December 2019, but had wind-down
costs throughout 2020, as well as decreases in compensation-related
expenses and other external expenses, partially offset by the
increases in external expenses related to Wave’s clinical and
preclinical activities related to its HD programs and its C9orf72
program for ALS and FTD.
General and administrative expenses were $10.1 million in the
first quarter of 2021, as compared to $13.0 million in the same
period in 2020. The year-over-year decrease was driven by decreases
in compensation-related expenses and other external expenses.
Wave ended the first quarter of 2021 with $148.5 million in cash
and cash equivalents, as compared to $184.5 million as of December
31, 2020. The decrease in cash and cash equivalents was mainly due
to Wave’s year-to-date net loss, partially offset by the receipt of
$8.0 million in net proceeds under Wave’s at-the-market equity
program. In April 2021, Wave received an additional $30.0 million
in committed research support under its collaboration with
Takeda.
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 second quarter of
2023.
Investor Conference Call and WebcastWave
management will host an investor conference call today at 8:30 a.m.
ET to discuss the company’s first quarter and 2021 financial
results and provide a business update. The conference call may be
accessed by dialing (866) 220-8068 (domestic) or (470) 495-9153
(international) and entering conference ID: 7430859. 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.
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, including
silencing, splicing and editing. 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 machine
learning-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 SciencesWave 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.
Forward-Looking StatementsThis 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 adaptive 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 product
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, including our novel
PN backbone chemistry modifications, and our stereopure
oligonucleotides compared with stereorandom oligonucleotides; the
potential benefits of our novel ADAR-mediated RNA editing platform
capabilities compared to others; 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 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, including their
receptiveness to our adaptive trial designs; our effectiveness in
managing future clinical trials and regulatory interactions; the
effectiveness of PRISM, including our novel PN backbone chemistry
modifications ; the effectiveness of our novel ADAR-mediated RNA
editing platform capability; 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; competition from
others developing therapies for similar indications; the severity
and duration of the COVID-19 pandemic and its negative impact on
the conduct of, and the timing of enrollment, completion and
reporting with respect to, our clinical trials; and any other
impacts on our business as a result of or related to the COVID-19
pandemic, 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, 2021 |
|
December 31, 2020 |
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
$ |
148,535 |
|
$ |
184,497 |
|
Current portion of accounts receivable |
|
30,000 |
|
|
30,000 |
|
Prepaid expenses |
|
10,430 |
|
|
10,434 |
|
Other current assets |
|
5,580 |
|
|
5,111 |
|
Total current assets |
|
194,545 |
|
|
230,042 |
|
Long-term assets: |
|
|
|
|
|
|
Property and equipment, net |
|
27,370 |
|
|
29,198 |
|
Operating lease right-of-use assets |
|
15,720 |
|
|
16,232 |
|
Restricted cash |
|
3,651 |
|
|
3,651 |
|
Other assets |
|
1,361 |
|
|
115 |
|
Total long-term assets |
|
48,102 |
|
|
49,196 |
|
Total assets |
$ |
242,647 |
|
$ |
279,238 |
|
Liabilities, Series A
preferred shares and shareholders’ equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
$ |
13,418 |
|
$ |
13,795 |
|
Accrued expenses and other current liabilities |
|
6,661 |
|
|
11,971 |
|
Current portion of deferred revenue |
|
24,763 |
|
|
91,560 |
|
Current portion of operating lease liability |
|
3,838 |
|
|
3,714 |
|
Total current liabilities |
|
48,680 |
|
|
121,040 |
|
Long-term liabilities: |
|
|
|
|
|
|
Deferred revenue, net of current portion |
|
108,278 |
|
|
41,481 |
|
Operating lease liability, net of current portion |
|
24,587 |
|
|
25,591 |
|
Other liabilities |
|
407 |
|
|
474 |
|
Total long-term liabilities |
|
133,272 |
|
|
67,546 |
|
Total liabilities |
$ |
181,952 |
|
$ |
188,586 |
|
Series A preferred shares, no par
value; 3,901,348 shares issued and outstanding at March 31, 2021
and December 31, 2020 |
$ |
7,874 |
|
$ |
7,874 |
|
Shareholders’ equity: |
|
|
|
|
|
|
Ordinary shares, no par value; 49,854,651 and 48,778,678 shares
issued and outstanding at March 31, 2021 and December 31, 2020,
respectively |
$ |
702,649 |
|
$ |
694,085 |
|
Additional paid-in capital |
|
75,636 |
|
|
71,573 |
|
Accumulated other comprehensive income |
|
269 |
|
|
389 |
|
Accumulated deficit |
|
(725,733 |
) |
|
(683,269 |
) |
Total shareholders’ equity |
$ |
52,821 |
|
$ |
82,778 |
|
Total liabilities, Series A
preferred shares and shareholders’ equity |
$ |
242,647 |
|
$ |
279,238 |
|
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, |
|
|
2021 |
|
2020 |
|
Revenue |
$ |
— |
|
$ |
4,161 |
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
33,393 |
|
|
41,158 |
|
General and administrative |
|
10,078 |
|
|
12,996 |
|
Total operating expenses |
|
43,471 |
|
|
54,154 |
|
Loss from operations |
|
(43,471 |
) |
|
(49,993 |
) |
Other income, net: |
|
|
|
|
|
|
Dividend income and interest income, net |
|
11 |
|
|
388 |
|
Other income, net |
|
996 |
|
|
2,112 |
|
Total other income, net |
|
1,007 |
|
|
2,500 |
|
Loss before income taxes |
|
(42,464 |
) |
|
(47,493 |
) |
Income tax provision |
|
— |
|
|
— |
|
Net loss |
$ |
(42,464 |
) |
$ |
(47,493 |
) |
Net loss per share attributable
to ordinary shareholders—basic and diluted |
$ |
(0.86 |
) |
$ |
(1.38 |
) |
Weighted-average ordinary shares
used in computing net loss per share attributable to ordinary
shareholders—basic and diluted |
|
49,101,606 |
|
|
34,461,505 |
|
|
|
|
|
|
|
|
Other comprehensive income
(loss): |
|
|
|
|
|
|
Net loss |
$ |
(42,464 |
) |
$ |
(47,493 |
) |
Foreign currency translation |
|
(120 |
) |
|
6 |
|
Comprehensive loss |
$ |
(42,584 |
) |
$ |
(47,487 |
) |
Investor Contact:Kate
Rausch617-949-4827krausch@wavelifesci.com
Media Contact:Alicia
Suter617-949-4817asuter@wavelifesci.com
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