Wave Life Sciences Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update
06 Mars 2024 - 1:30PM
Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage
biotechnology company focused on unlocking the broad potential of
RNA medicines to transform human health, today announced financial
results for the fourth quarter and full year ended December 31,
2023, and provided a business update.
“Following a year of execution and tremendous progress, we
entered 2024 well-capitalized and uniquely positioned to deliver
multiple high-impact clinical data readouts across three different
modalities and unlock the broad potential of our pipeline in both
rare and prevalent diseases,” said Paul Bolno, MD, MBA, President
and Chief Executive Officer of Wave Life Sciences. “We are rapidly
accelerating the development of our next generation GalNAc-siRNA
INHBE program in obesity and are encouraged by the emerging
preclinical data, which supports a potential best-in-class profile
for this program. These data demonstrate greater knockdown,
potency, and durability following a low, single dose, compared to
our first generation data, and importantly, demonstrate substantial
weight loss with decreases in fat mass and no loss of muscle
mass.”
Dr. Bolno continued, “In parallel with our efforts to advance
our INHBE program, we are continuing to progress our clinical
trials. We remain at the forefront of innovation in RNA editing and
expect to deliver proof-of-mechanism data for WVE-006 in AATD in
2024. These data would unlock our rapidly advancing pipeline of
wholly owned editing candidates. Beyond editing, we remain on track
to deliver multidose data for our novel, allele-selective HD
program in the second quarter and potentially registrational data
from our FORWARD-53 trial in DMD in the third quarter. The coming
months have the potential to be truly transformative for Wave.”
Recent Business Highlights
AATD and AIMer pipeline (RNA editing)
- Dose escalation in RestorAATion-1 clinical trial
ongoing; clinical program investigating WVE-006 as a first- and
best-in-class treatment for alpha-1 antitrypsin deficiency
(AATD).
- WVE-006 is a GalNAc-conjugated, subcutaneously delivered, RNA
editing oligonucleotide that is uniquely designed to address
AATD-related lung disease, liver disease, or both.
- The RestorAATion clinical program is evaluating WVE-006 for
AATD and includes healthy volunteers (RestorAATion-1), as well as
patients who have the homozygous Pi*ZZ mutation (RestorAATion-2).
It 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.
- In the fourth quarter of 2023, Wave initiated dosing in
RestorAATion-1, which resulted in a $20 million milestone payment
from GSK. Dose escalation is ongoing and WVE-006’s pharmacology in
healthy volunteers is translating as expected.
- Beyond WVE-006, Wave is advancing a pipeline of wholly owned
RNA editing therapeutics across a range of high-impact
GalNAc-hepatic and extra-hepatic targets. Utilizing its proprietary
“edit-verse,” which is powered by genetic datasets and deep
learning models, Wave has identified several new RNA editing
targets that leverage easily accessible biomarkers, offer efficient
paths to proof-of-concept in humans, and represent meaningful
commercial opportunities. Wave plans to share new preclinical data
for its advancing RNA editing programs in 2024.
- Expected upcoming milestone: Wave expects to
deliver proof-of-mechanism data from RestorAATion-2 in patients
with AATD in 2024.
Obesity (siRNA)
- Selected lead clinical candidate for INHBE program with
a potentially best-in-class profile for obesity and accelerated
clinical development timeline.
- Today, Wave announced the selection of its INHBE lead clinical
candidate, ahead of prior expectations, and now plans to submit a
clinical trial application for its INHBE program as early as
year-end 2024.
- Wave’s INHBE clinical candidate is a GalNAc-small interfering
RNA (siRNA) that utilizes Wave’s next generation siRNA format and
is designed to silence the INHBE (Inhibin βE) gene through RNA
knockdown with a goal of inducing lipolysis (fat-burning) while
preserving muscle mass to restore and maintain a healthy metabolic
profile.
- INHBE loss-of-function (LoF) heterozygous human carriers have a
favorable cardiometabolic profile, including reduced abdominal
obesity and reduced odds of type 2 diabetes and coronary artery
disease. Silencing INHBE is expected to recapitulate the
cardiometabolic profile of these LoF carriers and may also address
the limitations of GLP-1s as a monotherapy or be used as an adjunct
therapy with GLP-1s.
- Key data highlights from preclinical mouse models include:
- Highly potent silencing (ED50 < 1mg/kg)
- Durable silencing following one, low-single-digit dose
supporting every-six-month or annual subcutaneous dosing
- Weight loss with no loss of muscle mass
- Reduction in fat mass with preferential effects on visceral
fat, consistent with the profile of INHBE LoF carriers in human
genetics
- Expected upcoming milestone: Wave expects to
initiate a clinical trial for its INHBE candidate in the first
quarter of 2025.
DMD (exon skipping)
- Advancing FORWARD-53 clinical trial; multiple
presentations at MDA Clinical & Scientific Conference highlight
differentiated profile including industry-leading exon-skipping,
muscle tissue concentrations and myogenic stem cell
distribution.
- Wave’s WVE-N531 program for boys with Duchenne muscular
dystrophy (DMD) amenable to exon 53 skipping is designed to induce
production of endogenous, functional dystrophin protein.
- This week, at the 2024 MDA Clinical & Scientific
Conference, Wave presented multiple posters highlighting WVE-N531.
These posters featured data from Part A of Wave’s WVE-N531 trial,
demonstrating industry-leading exon skipping levels of 53% and
muscle tissue concentrations of 42 µg/g (42,000 ng/g), as well as
stem cell distribution in all study participants.
- WVE-N531 is currently being evaluated in the ongoing FORWARD-53
clinical trial of 11 boys with DMD, which is powered to evaluate
functional, endogenous dystrophin expression following 24 and 48
weeks of 10 mg/kg dosing administered every-other-week. The primary
endpoint is dystrophin protein levels, and the trial will also
evaluate pharmacokinetics, digital and functional endpoints, and
safety and tolerability.
- Pending positive results from the FORWARD-53 trial, the company
is planning to advance a broader DMD pipeline with PN-modified
oligonucleotides for skipping other exons, with the goal of
providing new treatment options for a larger population of boys
with DMD.
- Expected upcoming milestone: Wave expects to
deliver data, including dystrophin protein expression from muscle
biopsies at 24 weeks, in the third quarter of 2024.
HD (antisense silencing)
- WVE-003 and wild-type HTT sparing highlighted at annual
CHDI Conference.
- WVE-003 is a first-in-class investigational allele-selective
Huntington’s disease (HD) therapeutic designed to reduce mutant
huntingtin (mHTT) protein while also sparing healthy wild-type
huntingtin (wtHTT) protein. Due to the significance of wtHTT
function for the health of the central nervous system and the
potential for mHTT to disrupt wtHTT function, selectively lowering
mHTT while preserving wtHTT protein expression and function may
offer advantages over nonselective HTT-lowering approaches for the
treatment of HDrecent publication.
- WVE-003 has demonstrated single-dose reductions in mean mHTT in
cerebrospinal fluid of 35% compared to placebo, with preservation
of wtHTT, as previously shared in September 2022.
- The ongoing multi-dose portion of the SELECT-HD clinical trial
is evaluating a cohort of 24 patients with HD receiving 30 mg doses
of WVE-003 administered every eight weeks.
- Data from the ongoing SELECT-HD clinical trial will form the
basis for decision making for Wave’s advancement of this program,
including supporting an opt-in package for Takeda.
- Expected milestone: 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.
Financial Highlights
- Cash and cash equivalents were $200.4 million as of December
31, 2023, compared to $88.5 million as of December 31, 2022. The
increase in cash year-over-year is primarily due to the $170.0
million of cash received from the GSK Collaboration and the $93.6
million in net proceeds from the December 2023 Offering, partially
offset by the net loss for the year. Subsequent to December 31,
2023, Wave received $20 million in a milestone payment from GSK and
$14.0 million in net proceeds from the full exercise of the
greenshoe option related to the December 2023 financing.
- Wave expects that its current cash and cash equivalents will be
sufficient to fund operations into the fourth quarter of 2025.
Potential future milestone and other payments under the company’s
GSK and Takeda collaborations are not included in its cash
runway.
- Revenue was $29.1 million for the fourth quarter of 2023 as
compared to $1.2 million in the prior year quarter. Revenue was
$113.3 million in 2023 as compared to $3.6 million in 2022. The
increase in revenue was due to revenue earned from the company’s
collaborations with both GSK and Takeda.
- Research and development expenses were $34.1 million in the
fourth quarter of 2023 as compared to $31.1 million in the same
period in 2022. Research and development expenses for the full year
were $130.0 million in 2023, as compared to $115.9 million in
2022.
- General and administrative expenses were $13.7 million in the
fourth quarter of both 2023 and 2022. General and administrative
expenses for the full year were $51.3 million in 2023, as compared
to $50.5 million in 2022.
- Net loss was $16.3 million for the fourth quarter of 2023 as
compared to $43.7 million in the prior year quarter. Net loss for
the full year was $57.5 million for 2023 as compared to $161.8
million in 2022. Net loss significantly improved over the prior
year due to the substantial revenue earned from collaboration
partners in 2023.
Investor Conference Call and WebcastWave will
host an investor conference call today at 8:30 a.m. ET to
review the fourth quarter and full year 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-publications/events.
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 biotechnology company focused on unlocking the
broad potential of RNA medicines to transform human health. Wave’s
RNA medicines platform, PRISM™, combines multiple modalities,
chemistry innovation and deep insights in human genetics to deliver
scientific breakthroughs that treat both rare and prevalent
disorders. Its toolkit of RNA-targeting modalities includes
editing, splicing, RNA interference and antisense silencing,
providing Wave with unmatched capabilities for designing and
sustainably delivering candidates that optimally address disease
biology. Wave’s diversified pipeline includes clinical programs in
Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and
Huntington’s disease, as well as a preclinical program in obesity.
Driven by the calling to “Reimagine Possible”, Wave is leading the
charge toward a world in which human potential is no longer
hindered by the burden of disease. Wave is headquartered in
Cambridge, MA. For more information on Wave’s science, pipeline and
people, please visit www.wavelifesciences.com and follow Wave on X
(formerly Twitter) and LinkedIn.
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 and reporting of data and
completion of our clinical trials, including interactions with
regulators and 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
and pipeline compared to our competitors; our ability to design
compounds using various modalities and the anticipated benefits of
that approach; the breadth and versatility of our PRISM drug
discovery and development platform; 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
for certain of our programs to be best-in-class or first-in-class;
the potential benefits of our GalNAc-conjugated siRNA program
targeting INHBE; the potential benefits that our “edit-verse” may
provide us 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 benefits of RNA
medicines 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 the impact of any potential global macro events 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) |
|
|
|
|
|
|
|
|
|
|
December 31, 2023 |
|
|
December 31, 2022 |
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
200,351 |
|
|
$ |
88,497 |
|
Accounts receivable |
|
|
21,086 |
|
|
|
— |
|
Prepaid expenses |
|
|
9,912 |
|
|
|
7,932 |
|
Other current assets |
|
|
4,024 |
|
|
|
2,108 |
|
Total current assets |
|
|
235,373 |
|
|
|
98,537 |
|
Long-term assets: |
|
|
|
|
|
|
Property and equipment, net of accumulated depreciation of $42,709
and $37,846 as of December 31, 2023 and 2022, respectively |
|
|
13,084 |
|
|
|
17,284 |
|
Operating lease right-of-use assets |
|
|
22,637 |
|
|
|
26,843 |
|
Restricted cash |
|
|
3,699 |
|
|
|
3,660 |
|
Other assets |
|
|
156 |
|
|
|
62 |
|
Total long-term assets |
|
|
39,576 |
|
|
|
47,849 |
|
Total assets |
|
$ |
274,949 |
|
|
$ |
146,386 |
|
Liabilities, Series A
preferred shares and shareholders’ equity (deficit) |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
12,839 |
|
|
$ |
16,915 |
|
Accrued expenses and other current liabilities |
|
|
16,828 |
|
|
|
17,552 |
|
Current portion of deferred revenue |
|
|
150,059 |
|
|
|
31,558 |
|
Current portion of operating lease liability |
|
|
6,714 |
|
|
|
5,496 |
|
Total current liabilities |
|
|
186,440 |
|
|
|
71,521 |
|
Long-term liabilities: |
|
|
|
|
|
|
Deferred revenue, net of current portion |
|
|
15,601 |
|
|
|
79,774 |
|
Operating lease liability, net of current portion |
|
|
25,404 |
|
|
|
32,118 |
|
Other liabilities |
|
|
— |
|
|
|
190 |
|
Total long-term liabilities |
|
|
41,005 |
|
|
|
112,082 |
|
Total liabilities |
|
$ |
227,445 |
|
|
$ |
183,603 |
|
Series A preferred shares, no par
value; 3,901,348 shares issued and outstanding at December 31, 2023
and 2022 |
|
$ |
7,874 |
|
|
$ |
7,874 |
|
Shareholders’ equity
(deficit): |
|
|
|
|
|
|
Ordinary shares, no par value; 119,162,234 and 86,924,643 shares
issued and outstanding at December 31, 2023 and 2022,
respectively |
|
$ |
935,367 |
|
|
$ |
802,833 |
|
Additional paid-in capital |
|
|
129,237 |
|
|
|
119,442 |
|
Accumulated other comprehensive loss |
|
|
(124 |
) |
|
|
(29 |
) |
Accumulated deficit |
|
|
(1,024,850 |
) |
|
|
(967,337 |
) |
Total shareholders’ equity
(deficit) |
|
|
39,630 |
|
|
|
(45,091 |
) |
Total liabilities, Series A
preferred shares and shareholders’ equity (deficit) |
|
$ |
274,949 |
|
|
$ |
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 December 31, |
|
|
Twelve Months Ended December 31, |
|
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
Revenue |
|
$ |
29,056 |
|
|
$ |
1,239 |
|
|
$ |
113,305 |
|
|
$ |
3,649 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
34,074 |
|
|
|
31,078 |
|
|
|
130,009 |
|
|
|
115,856 |
|
General and administrative |
|
|
13,664 |
|
|
|
13,724 |
|
|
|
51,292 |
|
|
|
50,513 |
|
Total operating expenses |
|
|
47,738 |
|
|
|
44,802 |
|
|
|
181,301 |
|
|
|
166,369 |
|
Loss from operations |
|
|
(18,682 |
) |
|
|
(43,563 |
) |
|
|
(67,996 |
) |
|
|
(162,720 |
) |
Other income, net: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Dividend income and interest income, net |
|
|
1,844 |
|
|
|
825 |
|
|
|
7,928 |
|
|
|
1,571 |
|
Other income (expense), net |
|
|
582 |
|
|
|
(290 |
) |
|
|
1,878 |
|
|
|
7 |
|
Total other income, net |
|
|
2,426 |
|
|
|
535 |
|
|
|
9,806 |
|
|
|
1,578 |
|
Loss before income taxes |
|
|
(16,256 |
) |
|
|
(43,028 |
) |
|
|
(58,190 |
) |
|
|
(161,142 |
) |
Income tax benefit
(provision) |
|
|
— |
|
|
|
(681 |
) |
|
|
677 |
|
|
|
(681 |
) |
Net loss |
|
$ |
(16,256 |
) |
|
$ |
(43,709 |
) |
|
$ |
(57,513 |
) |
|
$ |
(161,823 |
) |
Net loss per share attributable
to ordinary shareholders—basic and diluted |
|
$ |
(0.15 |
) |
|
$ |
(0.47 |
) |
|
$ |
(0.54 |
) |
|
$ |
(2.05 |
) |
Weighted-average ordinary shares
used in computing net loss per share attributable to ordinary
shareholders—basic and diluted |
|
|
109,627,549 |
|
|
|
93,993,638 |
|
|
|
106,097,268 |
|
|
|
78,855,810 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other comprehensive loss: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
|
$ |
(16,256 |
) |
|
$ |
(43,709 |
) |
|
$ |
(57,513 |
) |
|
$ |
(161,823 |
) |
Foreign currency translation |
|
|
58 |
|
|
|
94 |
|
|
|
(95 |
) |
|
|
(210 |
) |
Comprehensive loss |
|
$ |
(16,198 |
) |
|
$ |
(43,615 |
) |
|
$ |
(57,608 |
) |
|
$ |
(162,033 |
) |
Investor Contact:Kate Rausch+1
617-949-4827krausch@wavelifesci.com
Media Contact:Alicia Suter+1
617-949-4817asuter@wavelifesci.com
Wave Life Sciences (NASDAQ:WVE)
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