Wave Life Sciences Reports First Quarter 2024 Financial Results and Provides Business Update
09 Mai 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 first quarter ended March 31, 2024, and provided a
business update.
“Since our last update, we have made excellent progress across
our multimodal pipeline of novel RNA medicines and continued to
advance our collaboration with GSK. We are on-track to deliver key
data sets this year, which enable opportunities to unlock the broad
potential of our RNA editing, silencing and splicing capabilities,”
said Paul Bolno, MD, MBA, President and Chief Executive Officer of
Wave Life Sciences. “In RNA editing, we are advancing WVE-006,
having used data from healthy volunteers in RestorAATion-1 to
identify a starting dose in our RestorAATion-2 study that is
expected to engage target in patients. With RestorAATion-2 now
underway, we remain on track to deliver expected proof of mechanism
data in patients with AATD this year, which would represent the
first-ever clinical demonstration of RNA editing and be an
important milestone for the alpha-1 community, as well as serve to
validate our wholly owned RNA editing pipeline.”
Dr. Bolno continued, “In addition to pioneering the field of RNA
editing, we are working expeditiously to advance our GalNAc-siRNA
INHBE program in obesity and expect to initiate a clinical trial in
the first quarter of next year. In DMD and HD, we are approaching
clinical data readouts as we plan to deliver multidose data for our
allele-selective HD program in the second quarter and potentially
registrational data from our FORWARD-53 trial in DMD in the third
quarter. Our efforts to accelerate development of our INHBE program
and advance our pipeline and collaborations have laid a strong
foundation for Wave’s future and we look forward to demonstrating
our leadership in RNA medicines as we reimagine what’s possible for
science, for medicine, and for human health.”
Recent Business Highlights
AATD and AIMer pipeline (RNA editing)
- RestorAATion-2 underway; clinical program investigating
WVE-006 as a first- and best-in-class treatment for alpha-1
antitrypsin deficiency (AATD)
- WVE-006 is Wave’s GalNAc-conjugated, subcutaneously delivered,
RNA editing oligonucleotide that is uniquely designed to address
AATD-related lung disease, liver disease, or both. WVE-006 does not
use a lipid-nanoparticle (LNP) delivery system.
- The RestorAATion-2 clinical trial is now underway.
RestorAATion-2 is a Phase 1b/2a open label study designed to
evaluate the safety, tolerability, pharmacodynamics (PD) and
pharmacokinetics (PK) of WVE-006 in patients with AATD who have the
homozygous Pi*ZZ mutation. The trial includes both single ascending
dose (SAD) and multiple ascending dose (MAD) portions. 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.
- Wave’s progress in dose-escalating healthy volunteers in
RestorAAtion-1 enabled the quick identification of a starting dose
level in RestorAATion-2 that, based on preclinical data, is
expected to engage target in patients.
- In addition to WVE-006, Wave continues to advance its pipeline
of wholly owned RNA editing therapeutics across a range of
high-impact GalNAc-hepatic and extra-hepatic targets. Powered by
genetic datasets and deep learning models, Wave is utilizing its
proprietary “edit-verse” to identify new RNA editing targets that
leverage easily accessible biomarkers, offer efficient paths to
proof-of-concept in humans, address diseases of high unmet need,
and represent meaningful commercial opportunities. Wave plans to
share new preclinical data from its wholly owned RNA editing
pipeline in 2024.
- Expected upcoming milestone: Wave expects to
deliver proof-of-mechanism data from RestorAATion-2 in patients
with AATD in 2024
Obesity (siRNA)
- Advancing lead clinical candidate for INHBE program
with a potentially best-in-class profile for obesity toward
anticipated clinical trial initiation in 1Q 2025
- Wave’s wholly owned 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, 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
limitations of GLP-1s.
- Wave’s INHBE GalNAc-siRNA has demonstrated highly potent (ED50
< 1mg/kg) and durable silencing following one, low-single-digit
dose supporting every-six-month or annual subcutaneous dosing in
preclinical mouse models. Data also demonstrated weight loss with
no loss of muscle mass and a reduction in fat mass with
preferential effects on visceral fat, consistent with the profile
of INHBE LoF carriers in human genetics.
- In an ongoing, head-to-head study in diet-induced obesity mice,
Wave has observed a weight loss effect from a single dose of its
INHBE GalNAc-siRNA similar to semaglutide. In addition, treatment
with Wave’s INHBE GalNAc-siRNA upon cessation of semaglutide
treatment curtailed expected rebound weight gain. The company plans
to share additional preclinical data later this year.
- Expected upcoming milestone: Wave expects to
initiate a clinical trial for its INHBE candidate in the first
quarter of 2025.
GSK Collaboration
- Advancing first two collaboration programs recently
selected by GSK following target validation; programs utilize
Wave’s next generation GalNAc siRNA format
- In April 2024, GSK selected its first two Collaboration
Programs, which are in hepatology, to advance to development
candidates following achievement of target validation. GSK will
provide an aggregate initiation payment of $12 million to
Wave.
- The first two GSK Collaboration Programs utilize Wave’s
GalNAc-siRNA formats. Under the agreement, GSK can advance up to
eight programs leveraging Wave’s PRISM platform and multiple
RNA-targeting modalities, including RNA editing, with target
validation ongoing in multiple therapy areas.
- In total, Wave is eligible for up to $3.3 billion in potential
milestone payments, as well as tiered royalties on net sales, for
GSK’s eight Collaboration Programs and WVE-006, for which GSK has
an exclusive global license.
DMD (exon skipping)
- Advancing FORWARD-53 clinical trial toward potentially
registrational 24-week dystrophin data in the third quarter of
2024
- 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.
- In Part A of Wave’s WVE-N531 trial, WVE-N531 demonstrated
industry-leading exon skipping levels of 53%, muscle tissue
concentrations of 42 µg/g (~42,000 ng/g), and myogenic 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
endogenous, functional 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 of 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 SELECT-HD multi-dose data with extended
follow-up remains on track for 2Q 2024; first-in-class program
designed to lower mutant HTT while sparing wild-type HTT
- 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 HD.
- 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 upcoming 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.
Corporate
- Dr. Erik Ingelsson appointed CSO; builds on Wave's
recent progress advancing innovative genetic targets and adds
experience to accelerate rapid identification and translation of
unique genetic insights
- Today, Wave announced the appointment of Erik Ingelsson, MD,
PhD, as Chief Scientific Officer (CSO). Dr. Ingelsson joins Wave to
drive the emerging therapeutic portfolio strategy, including
growing its genetics and genomics capabilities for identifying new,
high impact targets and leveraging Wave’s multimodal platform to
advance transformative RNA medicines. Dr. Ingelsson brings deep
expertise in genetics and drug discovery, as well as substantial
experience in metabolic diseases, such as obesity, MASH and
cardiovascular disease.
- Most recently, Dr. Ingelsson served as Senior Vice President,
Head of Target Discovery, at GSK, and prior to that, was SVP of
Genomic Sciences at GSK. Previously, he was a Professor of Medicine
at Stanford University. (See May 9, 2024 press release here)
Financial Highlights
- Cash and cash equivalents were $180.9 million as of March 31,
2024, compared to $200.4 million as of December 31, 2023.
Subsequent to March 31, 2024, GSK advanced two programs to
candidate development, triggering a $12.0 million aggregate
initiation payment to Wave. 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 to Wave under its GSK and Takeda collaborations are not
included in its cash runway.
- Revenue was $12.5 million for the first quarter of 2024, as
compared to $12.9 million in the first quarter of 2023. The slight
decrease in revenue was due to decreased revenue from the Takeda
collaboration. Revenue from the GSK collaboration was consistent
for the first quarter of 2024 and 2023.
- Research and development expenses were $33.4 million in the
first quarter of 2024, as compared to $31.0 million in the first
quarter of 2023. General and administrative expenses were $13.5
million in the first quarter of 2024, as compared to $12.2 million
in the first quarter of 2023.
- Net loss was $31.6 million for the first quarter of 2024, as
compared to $27.4 million for the first quarter of 2023.
Investor Conference Call and WebcastWave will
host an investor conference call today at 8:30 a.m. ET to
review the first quarter 2024 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; ongoing and 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, including identifying 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 and our ability to fund future operations; 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) |
|
|
|
March 31, 2024 |
|
|
December 31, 2023 |
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
180,922 |
|
|
$ |
200,351 |
|
Accounts receivable |
|
|
— |
|
|
|
21,086 |
|
Prepaid expenses |
|
|
11,139 |
|
|
|
9,912 |
|
Other current assets |
|
|
4,706 |
|
|
|
4,024 |
|
Total current assets |
|
|
196,767 |
|
|
|
235,373 |
|
Long-term assets: |
|
|
|
|
|
|
Property and equipment, net of accumulated depreciation of $43,687
and $42,709 as of March 31, 2024 and December 31, 2023,
respectively |
|
|
12,418 |
|
|
|
13,084 |
|
Operating lease right-of-use assets |
|
|
21,502 |
|
|
|
22,637 |
|
Restricted cash |
|
|
3,715 |
|
|
|
3,699 |
|
Other assets |
|
|
868 |
|
|
|
156 |
|
Total long-term assets |
|
|
38,503 |
|
|
|
39,576 |
|
Total assets |
|
$ |
235,270 |
|
|
$ |
274,949 |
|
Liabilities, Series A
preferred shares, and shareholders’ equity |
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
Accounts payable |
|
$ |
11,730 |
|
|
$ |
12,839 |
|
Accrued expenses and other current liabilities |
|
|
6,621 |
|
|
|
16,828 |
|
Current portion of deferred revenue |
|
|
140,586 |
|
|
|
150,059 |
|
Current portion of operating lease liability |
|
|
6,936 |
|
|
|
6,714 |
|
Total current liabilities |
|
|
165,873 |
|
|
|
186,440 |
|
Long-term liabilities: |
|
|
|
|
|
|
Deferred revenue, net of current portion |
|
|
12,536 |
|
|
|
15,601 |
|
Operating lease liability, net of current portion |
|
|
23,598 |
|
|
|
25,404 |
|
Total long-term
liabilities |
|
|
36,134 |
|
|
|
41,005 |
|
Total liabilities |
|
$ |
202,007 |
|
|
$ |
227,445 |
|
Series A preferred shares, no
par value; 3,901,348 shares issued and outstanding at
March 31, 2024 and December 31, 2023 |
|
$ |
7,874 |
|
|
$ |
7,874 |
|
Shareholders’ equity: |
|
|
|
|
|
|
Ordinary shares, no par value; 122,321,384 and 119,162,234 shares
issued and outstanding at March 31, 2024 and December 31,
2023, respectively |
|
$ |
949,877 |
|
|
$ |
935,367 |
|
Additional paid-in capital |
|
|
132,118 |
|
|
|
129,237 |
|
Accumulated other comprehensive loss |
|
|
(198 |
) |
|
|
(124 |
) |
Accumulated deficit |
|
|
(1,056,408 |
) |
|
|
(1,024,850 |
) |
Total shareholders’
equity |
|
$ |
25,389 |
|
|
$ |
39,630 |
|
Total liabilities, Series A
preferred shares, and shareholders’ equity |
|
$ |
235,270 |
|
|
$ |
274,949 |
|
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, |
|
|
|
2024 |
|
|
2023 |
|
Revenue |
|
$ |
12,538 |
|
|
$ |
12,929 |
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
|
33,447 |
|
|
|
30,979 |
|
General and administrative |
|
|
13,549 |
|
|
|
12,235 |
|
Total operating expenses |
|
|
46,996 |
|
|
|
43,214 |
|
Loss from operations |
|
|
(34,458 |
) |
|
|
(30,285 |
) |
Other income, net: |
|
|
|
|
|
|
Dividend income and interest income |
|
|
2,535 |
|
|
|
1,873 |
|
Other income, net |
|
|
365 |
|
|
|
1,007 |
|
Total other income, net |
|
|
2,900 |
|
|
|
2,880 |
|
Loss before income taxes |
|
|
(31,558 |
) |
|
|
(27,405 |
) |
Income tax benefit
(provision) |
|
|
— |
|
|
|
— |
|
Net loss |
|
$ |
(31,558 |
) |
|
$ |
(27,405 |
) |
Net loss per share
attributable to ordinary shareholders—basic and diluted |
|
$ |
(0.24 |
) |
|
$ |
(0.27 |
) |
Weighted-average ordinary
shares used in computing net loss per share attributable to
ordinary shareholders—basic and diluted |
|
|
129,271,678 |
|
|
|
102,056,712 |
|
Other comprehensive loss: |
|
|
|
|
|
|
Net loss |
|
$ |
(31,558 |
) |
|
$ |
(27,405 |
) |
Foreign currency translation |
|
|
(74 |
) |
|
|
(21 |
) |
Comprehensive loss |
|
$ |
(31,632 |
) |
|
$ |
(27,426 |
) |
|
Investor Contact:Kate Rausch+1
617-949-4827krausch@wavelifesci.com
Media Contact:Alicia Suter+1
617-949-4817asuter@wavelifesci.com
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