Wave Life Sciences Reports Third Quarter 2023 Financial Results and Provides Business Update
November 09 2023 - 7:30AM
Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA
medicines company committed to delivering life-changing treatments
for people battling devastating diseases, today announced financial
results for the third quarter ended September 30, 2023, and
provided a business update.
“The team has made tremendous progress during the past quarter
as we solidified our leadership in RNA editing through the
initiation of our clinical trial evaluating the first-ever RNA
editing therapeutic, WVE-006. Additionally this quarter, we
advanced our WVE-N531 (DMD) and WVE-003 (HD) clinical development
programs and expanded our RNA therapeutic capabilities beyond rare
diseases to common diseases with the announcement of our first
GalNAc-conjugated siRNA program targeting INHBE to treat metabolic
disorders, including obesity,” said Paul Bolno, MD, MBA, President
and Chief Executive Officer of Wave Life Sciences. “As we shared
during our recent R&D Day, we will sustain our leadership in
RNA editing by building a pipeline of wholly owned editing
candidates, each of which will have efficient paths to clinical
proof-of-concept and represent significant commercial
opportunities. With multiple, high impact clinical data readouts
expected over the course of 2024 and plans to expeditiously deliver
on a growing pipeline of RNA-targeting modalities, Wave is truly at
an exciting inflection point.”
Recent Business Highlights
- Initiated RestorAATion clinical program investigating
WVE-006 as a treatment for alpha-1 antitrypsin deficiency
(AATD). Wave recently initiated RestorAATion following
approval of multiple clinical trial applications (CTAs). WVE-006 is
uniquely designed to address AATD-related lung disease, liver
disease, or both. The RestorAATion clinical program includes
healthy volunteers (RestorAATion-1), as well as individuals with
AATD who have the homozygous PiZZ mutation (RestorAATion-2) and is
designed to provide an efficient path to proof-of-mechanism as
measured by restoration of wild-type alpha-1 antitrypsin (M-AAT)
protein in serum. Wave expects to initiate dosing with WVE-006 in
healthy volunteers in the fourth quarter of 2023 and deliver
proof-of-mechanism data in individuals with AATD in 2024.
- Announced plans to advance a wholly owned pipeline of
RNA editing candidates. Wave is utilizing its proprietary
“edit-verse,” which is powered by genetic datasets and deep
learning models and designed to identify new RNA editing targets
and edit sites. These targets leverage easily accessible
biomarkers, offer efficient paths to proof-of-concept in humans,
and represent meaningful commercial opportunities. Wave
demonstrated in vivo and in vitro proof-of-concept data on several
of these new targets, achieving at least 2-fold mRNA upregulation
in liver and kidney targets and more than 60% mRNA correction in
liver and lung targets.
- Announced new, wholly owned GalNAc-conjugated siRNA
program targeting INHBE for metabolic disorders, including obesity,
along with preclinical in vivo
proof-of-concept data. INHBE loss-of-function
heterozygous human carriers exhibit a healthy metabolic profile,
including reduced waist-to-hip ratio and reduced odds of type 2
diabetes and coronary artery disease, and INHBE reduction of 50% or
greater is expected to restore a healthy metabolic profile. Wave
presented in vivo data at its recent R&D Day demonstrating
INHBE silencing well beyond the 50% therapeutic threshold, which
led to substantially lower body weight and reduction of visceral
fat in diet-induced obesity mice as compared to control. These are
the first in vivo data to demonstrate INHBE silencing is consistent
with the phenotypes of heterozygous loss-of-function carriers. Wave
has identified potent and highly specific leads and expects to
select an INHBE clinical candidate in the fourth quarter of 2024.
The INHBE program is Wave’s first wholly owned program to emerge
from its collaboration with GSK.
- Advancing potentially registrational Phase 2 FORWARD-53
clinical trial for WVE-N531. Wave remains on track to
initiate dosing in FORWARD-53 in 2023 and enroll up to 10 boys in
the trial. The clinical trial is powered to evaluate functional,
endogenous dystrophin expression following 24 and 48 weeks of
bi-weekly dosing. The primary endpoint will be dystrophin protein
levels, and the trial will also evaluate pharmacokinetics, digital
and functional endpoints, and safety and tolerability. Pending
positive results from this trial, the company is planning to
advance a broader DMD pipeline with PN-modified splicing
oligonucleotides for skipping other exons, with the goal of
providing new treatment options for a larger population of boys
with DMD. Data from FORWARD-53 are expected in 2024.
- Delivered first clinical data in DMD demonstrating
uptake in satellite cells after three biweekly doses. At
Wave’s annual R&D Day held in September 2023, the company
highlighted clinical data from Part A of the Phase 1b/2a trial
indicating that WVE-N531 was present in myogenic satellite cells,
which is important for potential muscle regeneration. These are the
first clinical data in DMD to demonstrate uptake in satellite cells
at this early time point and further support the potential
differentiation of WVE-N531 from other therapeutics, including gene
therapies.
- Achievement of a non-clinical milestone under Takeda
collaboration; NHP data supports broad distribution of WVE-003 in
CNS. WVE-003 is the most advanced investigational HD
therapeutic designed to reduce mutant huntingtin (mHTT) protein
while also sparing healthy, wild-type huntingtin (wtHTT) protein,
and previously demonstrated single-dose reductions in mean CSF mHTT
of 35% as compared to placebo, with preservation of wtHTT, in
September 2022. In the third quarter of 2023, Wave achieved a
milestone in its collaboration with Takeda, which pertained to the
positive results from a non-clinical study of WVE-003 in non-human
primates (NHPs) and resulted in a payment of $7 million to Wave in
the fourth quarter of 2023. This study showed significant tissue
exposure levels of WVE-003 in the deep brain regions, including
striatum, and bolstered Wave’s existing datasets that confirm the
ability of its oligonucleotides to distribute to the areas of the
CNS important for HD.
- Completed enrollment in 30 mg multi-dose cohort
of Phase 1b/2a SELECT-HD clinical trial for
WVE-003 – the most advanced allele-selective investigational
therapeutic for individuals with HD. Wave recently
completed enrollment in the 30 mg multi-dose cohort of the
SELECT-HD clinical trial, which is evaluating doses administered
every eight weeks. Having rolled over patients from the single-dose
cohort and fully enrolled the 30 mg multi-dose cohort, Wave will
now evaluate the completed single-dose and multi-dose cohorts
simultaneously. Wave expects to report data from the 30 mg
multi-dose cohort with extended follow-up, along with all
single-dose data, in the second quarter of 2024. These data are
expected to enable decision making on the program and support the
company’s opt-in package for Takeda.
Anticipated Upcoming Milestones
WVE-006 for AATD:
- Initiate dosing in healthy volunteers in RestorAATion clinical
program in 4Q 2023
- Deliver proof-of-mechanism data from RestorAATion clinical
program in 2024
WVE-N531 for DMD:
- Initiate dosing in potentially registrational FORWARD-53 Phase
2 clinical trial in 2023
- Deliver data from FORWARD-53 clinical trial in 2024
WVE-003 for HD:
- Deliver data from 30 mg multi-dose cohort with extended
follow-up, along with all single-dose data, in 2Q 2024
INHBE program for metabolic disorders, including obesity
- Select a clinical candidate in 4Q 2024
Platform and Pipeline:
- Advance collaboration activities with GSK, with potential for
additional cash inflows in 2023 and beyond
- Select five new clinical candidates by year-end 2025, including
INHBE
Third Quarter 2023 Financial Results
Wave reported a net income of $7.3 million in the third quarter
of 2023, as compared to a net loss of $39.0 million in the same
period in 2022. The year-over-year change was primarily driven by
the $48.9 million increase in revenue recognized under its
collaborations. Under the Takeda collaboration, Wave earned $7.0
million dollars for the achievement of a WVE-003 non-clinical
milestone. Additionally, under the Takeda collaboration, Wave
recognized $28.0 million relating to the discontinuation of WVE-004
and under the GSK Collaboration, recognized $14.3 million.
Research and development expenses were $31.6 million in the
third quarter of 2023, as compared to $27.6 million in the same
period in 2022. The increase in research and development expenses
was primarily driven by increased external expenses related to all
three of Wave’s clinical programs.
General and administrative expenses were $13.1 million in the
third quarter of 2023, as compared to $11.6 million in the same
period in 2022. The increase was primarily due to increased
professional and consulting expenses as well as other general
expenses.
As of September 30, 2023, Wave had $139.9 million in cash and
cash equivalents, as compared to $88.5 million as of December 31,
2022. Subsequent to the quarter end, Wave received the $7.0 million
for the achievement of the previously discussed milestone. The
company expects that its current cash and cash equivalents will be
sufficient to fund operations into 2025.
Investor Conference Call and WebcastWave will
host an investor conference call today at 8:30 a.m. ET to
review third quarter 2023 financial results and pipeline updates. A
webcast of the conference call can be accessed by visiting
“Investor Events” on the investor relations section of
the Wave Life Sciences website:
https://ir.wavelifesciences.com/events-and-presentations. Analysts
planning to participate during the Q&A portion of the live call
can join the conference call at the following audio-conferencing
link: available here. Once registered, participants will receive
the dial-in information. Following the live event, an archived
version of the webcast will be available on the Wave Life
Sciences website.
About Wave Life SciencesWave Life Sciences
(Nasdaq: WVE) is a clinical-stage RNA 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 X
(formerly Twitter) @WaveLifeSci.
Forward-Looking Statements 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 initiation, site activation, patient recruitment,
patient enrollment, dosing, generation of data and completion of
our clinical trials, including any potential registration based on
these data, and the announcement of such events; the protocol,
design and endpoints of our 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; the potential achievement
of milestones under our collaborations and receipt of cash payments
therefor; the potential of our preclinical data to predict the
behavior of our compounds in humans; our identification and
expected timing of future product candidates and their therapeutic
potential; the anticipated benefits of our therapeutic candidates
compared to others; our ability to design compounds using multiple
modalities and the anticipated benefits of that approach; the
breadth and versatility of PRISM; the expected benefits of our
stereopure oligonucleotides compared with stereorandom
oligonucleotides; the potential benefits of our RNA editing
capability, including our AIMers, compared to others; the potential
benefits of our GalNAc-conjugated siRNA program targeting INHBE,
the potential benefits that our “edit-verse” map may offer to
identify new RNA editing targets; the status and progress of our
programs relative to potential competitors; anticipated benefits of
our proprietary manufacturing processes and our internal
manufacturing capabilities; the benefit of nucleic acid
therapeutics generally; the strength of our intellectual property
and the data that support our IP; the anticipated duration of our
cash runway; our intended uses of capital; and our expectations
regarding any potential global macro events beyond our control 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; the clinical results of our
programs and the timing thereof, which may not support further
development of our product candidates; actions of regulatory
authorities and their receptiveness to our adaptive trial designs,
which may affect the initiation, timing and progress of clinical
trials; our effectiveness in managing regulatory interactions and
future clinical trials; the effectiveness of PRISM; the
effectiveness of our RNA editing capability and our AIMers; 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 the indications we
are pursuing; our ability to maintain the company infrastructure
and personnel needed to achieve our goals; and 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, 2023 |
|
|
December 31, 2022 |
|
Assets |
|
|
|
|
|
Current
assets: |
|
|
|
|
|
Cash and cash equivalents |
$ |
139,942 |
|
|
$ |
88,497 |
|
Accounts receivable |
|
7,000 |
|
|
|
— |
|
Prepaid expenses |
|
7,514 |
|
|
|
7,932 |
|
Other current assets |
|
4,116 |
|
|
|
2,108 |
|
Total
current assets |
|
158,572 |
|
|
|
98,537 |
|
Long-term assets: |
|
|
|
|
|
Property and equipment, net of accumulated depreciation of $41,596
and $37,846 as of September 30, 2023 and
December 31, 2022, respectively |
|
13,770 |
|
|
|
17,284 |
|
Operating lease right-of-use assets |
|
23,738 |
|
|
|
26,843 |
|
Restricted cash |
|
3,683 |
|
|
|
3,660 |
|
Other assets |
|
155 |
|
|
|
62 |
|
Total
long-term assets |
|
41,346 |
|
|
|
47,849 |
|
Total
assets |
$ |
199,918 |
|
|
$ |
146,386 |
|
Liabilities, Series A preferred shares and shareholders’
equity (deficit) |
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
Accounts payable |
$ |
12,608 |
|
|
$ |
16,915 |
|
Accrued expenses and other current liabilities |
|
12,624 |
|
|
|
17,552 |
|
Current portion of deferred revenue |
|
68,251 |
|
|
|
31,558 |
|
Current portion of operating lease liability |
|
6,497 |
|
|
|
5,496 |
|
Total
current liabilities |
|
99,980 |
|
|
|
71,521 |
|
Long-term liabilities: |
|
|
|
|
|
Deferred revenue, net of current portion |
|
105,380 |
|
|
|
79,774 |
|
Operating lease liability, net of current portion |
|
27,170 |
|
|
|
32,118 |
|
Other liabilities |
|
— |
|
|
|
190 |
|
Total
long-term liabilities |
|
132,550 |
|
|
|
112,082 |
|
Total
liabilities |
$ |
232,530 |
|
|
$ |
183,603 |
|
Series A
preferred shares, no par value; 3,901,348 shares
issued and outstanding at September 30, 2023 and
December 31, 2022 |
$ |
7,874 |
|
|
$ |
7,874 |
|
Shareholders’ equity (deficit): |
|
|
|
|
|
Ordinary shares, no par value; 99,011,901 and 86,924,643 shares
issued and outstanding at September 30, 2023 and
December 31, 2022, respectively |
$ |
841,405 |
|
|
$ |
802,833 |
|
Additional paid-in capital |
|
126,885 |
|
|
|
119,442 |
|
Accumulated other comprehensive loss |
|
(182 |
) |
|
|
(29 |
) |
Accumulated deficit |
|
(1,008,594 |
) |
|
|
(967,337 |
) |
Total
shareholders’ deficit |
$ |
(40,486 |
) |
|
$ |
(45,091 |
) |
Total
liabilities, Series A preferred shares and shareholders’
deficit |
$ |
199,918 |
|
|
$ |
146,386 |
|
WAVE LIFE SCIENCES
LTD.UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS AND COMPREHENSIVE INCOME (LOSS)
(In thousands, except share and per share
amounts)
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
Revenue |
$ |
49,214 |
|
|
$ |
285 |
|
|
$ |
84,249 |
|
|
$ |
2,410 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
31,642 |
|
|
|
27,575 |
|
|
|
95,935 |
|
|
|
84,778 |
|
General and administrative |
|
13,128 |
|
|
|
11,609 |
|
|
|
37,628 |
|
|
|
36,789 |
|
Total operating expenses |
|
44,770 |
|
|
|
39,184 |
|
|
|
133,563 |
|
|
|
121,567 |
|
Income (loss) from
operations |
|
4,444 |
|
|
|
(38,899 |
) |
|
|
(49,314 |
) |
|
|
(119,157 |
) |
Other income (expense),
net: |
|
|
|
|
|
|
|
|
|
|
|
Dividend income and interest income, net |
|
1,960 |
|
|
|
596 |
|
|
|
6,084 |
|
|
|
746 |
|
Other income (expense), net |
|
171 |
|
|
|
(701 |
) |
|
|
1,296 |
|
|
|
297 |
|
Total other income (expense),
net |
|
2,131 |
|
|
|
(105 |
) |
|
|
7,380 |
|
|
|
1,043 |
|
Income (loss) before income
taxes |
|
6,575 |
|
|
|
(39,004 |
) |
|
|
(41,934 |
) |
|
|
(118,114 |
) |
Income tax benefit
(provision) |
|
677 |
|
|
|
— |
|
|
|
677 |
|
|
|
— |
|
Net income (loss) |
$ |
7,252 |
|
|
$ |
(39,004 |
) |
|
$ |
(41,257 |
) |
|
$ |
(118,114 |
) |
Less: net income attributable to participating
securities |
$ |
(257 |
) |
|
|
— |
|
|
|
— |
|
|
|
— |
|
Net income (loss) attributable
to ordinary shareholders, basic and diluted |
$ |
6,995 |
|
|
$ |
(39,004 |
) |
|
$ |
(41,257 |
) |
|
$ |
(118,114 |
) |
Net income (loss) per share
attributable to ordinary shareholders—basic |
$ |
0.07 |
|
|
$ |
(0.42 |
) |
|
$ |
(0.39 |
) |
|
$ |
(1.60 |
) |
Weighted-average ordinary
shares used in computing net income (loss) per
share attributable to ordinary
shareholders—basic |
|
106,025,063 |
|
|
|
93,900,484 |
|
|
|
104,529,266 |
|
|
|
73,754,417 |
|
Net income (loss) per share
attributable to ordinary
shareholders—diluted |
$ |
0.07 |
|
|
$ |
(0.42 |
) |
|
$ |
(0.39 |
) |
|
$ |
(1.60 |
) |
Weighted-average ordinary
shares used in computing net income (loss) per
share attributable to ordinary
shareholders—diluted |
|
106,975,231 |
|
|
|
93,900,484 |
|
|
|
104,529,266 |
|
|
|
73,754,417 |
|
Other comprehensive income
(loss): |
|
|
|
|
|
|
|
|
|
|
|
Net income (loss) |
$ |
7,252 |
|
|
$ |
(39,004 |
) |
|
$ |
(41,257 |
) |
|
$ |
(118,114 |
) |
Foreign currency translation |
|
(32 |
) |
|
|
(76 |
) |
|
|
(153 |
) |
|
|
(304 |
) |
Comprehensive income
(loss) |
$ |
7,220 |
|
|
$ |
(39,080 |
) |
|
$ |
(41,410 |
) |
|
$ |
(118,418 |
) |
Investor Contact:Kate Rausch+1
617-949-4827krausch@wavelifesci.com
Media Contact:Alicia Suter+1
617-949-4817asuter@wavelifesci.com
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