Wave Life Sciences Ltd
For the period ended Jun 30, 2024. Filed Aug 8, 2024.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2024 · Filed Aug 8, 2024
WVE$3.37+4.5%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-03-31; filed 2024-05-10.
0000950170-24-057457
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2024-06-30; filed 2024-08-08.
0000950170-24-093341
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 150 previous paragraphs and 162 current paragraphs.
105 unchanged; 34 reworded; 15 added; 11 removed; 0 previous and 8 current paragraphs rearranged.
Tables are not compared: 3 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (15)
Current report
HD: WVE-003 is an allele-selective oligonucleotide designed to lower mutant huntingtin (“mHTT”) protein and preserve healthy, wild-type huntingtin (“wtHTT”) protein.
Current report
There are currently no disease modifying therapies for HD, which affects over 200,000 individuals across all disease stages in the United States and Europe.
Current report
The SELECT-HD trial was a global, multicenter, randomized, double-blind, placebo-controlled Phase 1b/2a clinical trial to assess the safety and tolerability of WVE-003 in people with a confirmed diagnosis of HD who are in the early stages of the disease and carry SNP3 in association with their cytosine-adenine-guanine (“CAG”) expansion. Additional objectives included assessing pharmacokinetics and exploratory pharmacodynamic and clinical endpoints.
Current report
In June 2024, we announced positive clinical data from the Phase 1b/2a SELECT-HD study of WVE-003. Results from the multi-dose portion of the trial, which evaluated three doses of 30mg WVE-003 administered every eight weeks, showed clear translation of target engagement to clinic with statistically significant, potent, durable and allele-selective reductions in cerebrospinal fluid (“CSF”) mHTT of up to 46% and preservation of healthy protein. This cohort also revealed a statistically significant correlation between mHTT reductions and slowing of caudate atrophy, indicating a potential benefit of allele-selective mHTT reductions. Structural brain magnetic resonance imaging (“MRI”) changes such as caudate atrophy are well-characterized measures of disease progression and neurodegeneration in HD. WVE-003 was generally safe and well-tolerated, with mild-to-moderate adverse events (“AEs”) and no Serious AEs.
Current report
We have submitted an opt-in package to our partner, Takeda, and initiated engagement with regulators on a clinical development path to accelerated approval. We expect to receive a decision from Takeda on their option right, as well as feedback from regulators by year-end.
Current report
Other Income (Expense), Net
Current report
Other income, net for the three months ended June 30, 2024 and 2023 was $2.1 million and $2.4 million, respectively, and consisted primarily of dividend income.
Current report
Income Tax Benefit (Provision)
Current report
Comparison of the six months ended June 30, 2024 and 2023
Current report
Revenue
Current report
Research and development expenses were $73.8 million for the six months ended June 30, 2024, compared to $64.3 million for the six months ended June 30, 2023. The increase of approximately $9.5 million was due to the following:
Current report
General and administrative expenses were $27.8 million for the six months ended June 30, 2024, as compared to approximately $24.5 million for the six months ended June 30, 2023. The increase of $3.3 million was primarily driven by increases in compensation related expenses and professional fees.
Current report
Other Income (Expense), Net
Current report
Other income, net for the six months ended June 30, 2024 and 2023 was $5.0 million and $5.2 million, respectively, and consisted primarily of dividend income, as well as estimated refundable tax credits.
Current report
Income Tax Benefit (Provision)
Removed paragraphs (11)
Previous report
HD: WVE-003 is a selective mutant huntingtin (“mHTT”)-lowering SNP3 antisense silencing oligonucleotide.
Previous report
The SELECT-HD trial, which incorporates learnings from our prior HD programs, is a multicenter, randomized, double-blind, placebo-controlled Phase 1b/2a clinical trial to assess the safety and tolerability of intrathecally administered WVE-003 for patients with early manifest HD. Additional objectives include measurement of mHTT and wtHTT protein in cerebrospinal fluid (“CSF”) and exploratory pharmacokinetic, pharmacodynamic, clinical and magnetic resonance imaging (“MRI”) endpoints. The SELECT-HD trial is designed to be adaptive, with dose level and dosing frequency being guided by an independent committee.
Previous report
In September 2022 (data cut-off: August 29, 2022), we announced a positive update from SELECT-HD driven by the observation of reductions in mean CSF mHTT protein in cerebrospinal fluid (“CSF”) after trial participants received either a single 30 or 60 mg dose of WVE-003. Additionally, wtHTT protein levels appeared consistent with allele-selectivity. Single doses (30 mg, 60 mg, and 90 mg) of WVE-003 appeared generally safe and well-tolerated. Based on the SELECT-HD data, we adapted the trial to expand the single-dose cohorts, and the multi-dose portion is underway.
Previous report
We have completed enrollment in the 30 mg multi-dose cohort, which is evaluating doses of WVE-003 administered every eight weeks, and we plan to evaluate the completed single-dose and multi-dose cohorts simultaneously. We expect 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 form the basis for decision making for our advancement of this program, including supporting an opt-in package for Takeda Pharmaceutical Company Limited (“Takeda”).
Previous report
Research and Development Expenses
Previous report
General and Administrative Expenses
Previous report
Research and Development Expenses
Previous report
General and Administrative Expenses
Previous report
Other Income, Net
Previous report
For the three months ended March 31, 2024 and 2023, other income, net was $2.9 million for both periods and consisted primarily of dividend income.
Previous report
Income Tax Provision
Reworded paragraphs (34)
Previous wording
Obesity: Lead clinical candidate is a GalNAc-conjugated RNAi oligonucleotide targeting inhibin βE (“INHBE”).
Current wording
Obesity: WVE-007 is a GalNAc-conjugated RNAi oligonucleotide targeting inhibin βE (“INHBE”).
Previous wording
(1) A discovery collaboration which enables us to advance up to three programs leveraging targets informed by GSK’s novel insights, the first of which is our INHBE program for obesity and other metabolic disorders;
Current wording
(1) A discovery collaboration which enables us to advance up to three programs leveraging targets informed by GSK’s novel insights, the first of which is our INHBE program (WVE-007) for obesity and other metabolic disorders;
Previous wording
WVE-006 is first-in-class in AATD and is the most advanced program currently in development using an oligonucleotide to harness an endogenous enzyme for RNA editing. In the fourth quarter of 2023, we initiated our RestorAATion clinical program investigating WVE-006 as a treatment for AATD. The RestorAATion clinical program includes both healthy volunteers (“RestorAATion-1”), as well as patients 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. 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 individuals with AATD who have the homozygous PiZZ mutation. The trial includes both single ascending dose (“SAD”) and multiple ascending dose (“MAD”) portions. In April 2024, we announced that we had received approval for our first CTA for RestorAATion-2. RestorAAtion-2 is underway and we expect to deliver proof-of-mechanism data in patients with AATD in 2024.
Current wording
WVE-006 is first-in-class in AATD and is the most advanced program currently in development using an oligonucleotide to harness an endogenous enzyme for RNA editing. In the fourth quarter of 2023, we initiated our RestorAATion clinical program investigating WVE-006 as a treatment for AATD. The RestorAATion clinical program includes both healthy volunteers (“RestorAATion-1”), as well as patients 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. 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 individuals with AATD who have the homozygous PiZZ mutation. The trial includes both single ascending dose (“SAD”) and multiple ascending dose (“MAD”) portions. In the third quarter of 2024, we initiated dosing in the single dose portion of the first dose cohort of RestorAATion-2. We expect to deliver proof-of-mechanism data in patients with AATD in the fourth quarter of 2024.
Previous wording
Under our GSK collaboration, GSK received an exclusive global license for WVE-006, with clinical development and commercial responsibilities transitioning to GSK after we complete the RestorAATion-2 trial. In December 2023, we announced that we achieved the first WVE-006 milestone in our collaboration with GSK, resulting in a $20 million payment. Under the terms of the collaboration, we are eligible to receive up to $505 million in additional development, launch, and commercial milestone payments, as well as double-digit tiered royalties up to the high teens, as a percentage of net sales for WVE-006.
Current wording
Under our GSK collaboration, GSK received an exclusive global license for WVE-006, with clinical development and commercial responsibilities transitioning to GSK after we complete the RestorAATion-2 trial. Under the terms of the collaboration, we are eligible to receive up to $525 million in development, launch, and commercial milestone payments, as well as double-digit tiered royalties up to the high teens, as a percentage of net sales for WVE-006. In December 2023, we announced that we achieved the first WVE-006 milestone in our collaboration with GSK, resulting in a $20 million payment.
Previous wording
Preclinical data show that treatment with WVE-006 resulted in serum AAT protein levels of up to 30 micromolar in an established AATD mouse model (NSG-PiZ). WVE-006 also led to restoration of approximately 50% wild-type M-AAT protein in serum and a 3-fold increase in neutrophil elastase inhibition activity, indicating that the restored M-AAT protein was functional. Wave’s AATD AIMers are highly specific to SERPINA1 RNAin vitro andin vivo based on transcriptome-wide analyses.
Current wording
Preclinical data show that treatment with WVE-006 resulted in serum AAT protein levels of up to 30 micromolar in an established AATD mouse model (NSG-PiZ). WVE-006 also led to restoration of approximately 50% wild-type M-AAT protein in serum and a 3-fold increase in neutrophil elastase inhibition activity, indicating that the restored M-AAT protein was functional. Our AATD AIMers are highly specific to SERPINA1 RNAin vitro andin vivo based on transcriptome-wide analyses.
Previous wording
If we are successful in the clinic with WVE-006, we will validate our clinical approach to AATD and demonstrate the feasibility of RNA editing as a therapeutic modality in humans.
Current wording
If we are successful in the clinic with WVE-006, this would validate our clinical approach to AATD and demonstrate the feasibility of RNA editing as a therapeutic modality in humans.
Previous wording
Our first wholly owned program to emerge from our collaboration with GSK is a GalNAc-conjugated siRNA silencing program targeting INHBE for obesity. There are approximately 174 million people in the United States and Europe with obesity, and therapeutic options beyond GLP-1 receptor agonists are needed. GLP-1 receptor agonists lead to weight loss at the expense of muscle, suppress the general reward system, and are associated with a poor tolerability profile, with 68% drop-off after one year. Heterozygous INHBE loss-of-function human carriers exhibit a healthy metabolic profile, including reduced waist-to-hip ratio and reduced odds of developing type 2 diabetes or coronary artery disease, and reduction of INHBE by 50% or more is expected to restore a healthy metabolic profile. In connection with our 2023 Research and Development Day, we sharedin vivo proof of concept data in diet-induced obesity mice demonstrating INHBE silencing well beyond the anticipated 50% therapeutic threshold, which led to substantially lower body weight and reduction of visceral fat as compared to controls. These are the first data to demonstrate INHBE silencingin vivo in an animal model is consistent with the phenotypes of heterozygous loss-of-function carriers.
Current wording
Our first wholly owned program to emerge from our collaboration with GSK is WVE-007, a GalNAc-small interfering RNA (“siRNA”) that is designed to silence the Inhibin βE gene (“INHBE”) to induce lipolysis (fat-burning) while preserving muscle mass to restore and maintain a healthy metabolic profile. There are approximately 174 million people in the United States and Europe with obesity, and therapeutic options beyond GLP-1 receptor agonists are needed. GLP-1 receptor agonists lead to weight loss at the expense of muscle, suppress the general reward system, and are associated with a poor tolerability profile and 68% drop-off after one year. Heterozygous INHBE loss-of-function human carriers exhibit a healthy metabolic profile, including reduced waist-to-hip ratio and reduced odds of developing type 2 diabetes or coronary artery disease, and reduction of INHBE by 50% or more is expected to restore a healthy metabolic profile. In connection with our 2023 Research and Development Day, we sharedin vivo proof of concept data in diet-induced obesity mice demonstrating INHBE silencing well beyond the anticipated 50% therapeutic threshold, which led to substantially lower body weight and reduction of visceral fat as compared to controls. These are the first data to demonstrate INHBE silencingin vivo in an animal model is consistent with the phenotypes of heterozygous loss-of-function carriers.
Previous wording
In March 2024, we announced the selection of our INHBE lead clinical candidate, which utilizes our next generation GalNAc-siRNA format. This next generation format results in more potent and durable siRNA silencing and when applied to our INHBE program resulted in: 1) highly potent INHBE silencing (ED50 < 1 mg/kg), durable silencing following one, low-single digit dose in a diet-induced obesity (“DIO”) mouse model supporting every-six-month or annual subcutaneous dosing; 2) weight loss with no loss of muscle mass; and 3) reduction in fat mass, with preferential effect to the visceral fat, consistent with the profile of INHBE LoF in human genetics. In an ongoing, head-to-head study in DIO mice, we have observed a weight loss effect from a single dose of our INHBE GalNAc-siRNA similar to semaglutide. In addition, treatment with our INHBE GalNAc-siRNA upon cessation of semaglutide treatment curtailed expected rebound weight gain. We expect to initiate a clinical trial for our INHBE candidate in the first quarter of 2025.
Current wording
WVE-007 utilizes our next generation GalNAc-siRNA format. In preclinical diet-induced obesity (“DIO”) mouse models, our INHBE GalNAc-siRNA has demonstrated highly potent INHBE silencing (ED50 < 1 mg/kg), durable silencing following one, low-single digit dose supporting every-six-month or annual subcutaneous dosing in humans, weight loss with no loss of muscle mass and reduction in fat mass, with preferential effect to the visceral fat, consistent with the profile of INHBE LoF in human genetics. In a head-to-head study in DIO mice, we have observed a weight loss effect from a single dose of our INHBE GalNAc-siRNA similar to semaglutide. In addition, treatment with our INHBE GalNAc-siRNA upon cessation of semaglutide treatment curtailed expected rebound weight gain. Additionally, in a separate ongoing study in DIO mice, when administered in combination with semaglutide, a single dose of our INHBE GalNAc-siRNA doubled the weight loss observed with semaglutide alone and this effect was sustained throughout the duration of the study. We plan to share additional preclinical data later this year and expect to initiate a clinical trial for WVE-007 in the first quarter of 2025.
Previous wording
In HD, we are currently advancing WVE-003, a stereopure antisense oligonucleotide designed to selectively target an undisclosed single nucleotide polymorphism (“SNP”), “mHTT SNP3”, associated with the disease-causing mHTT mRNA transcript within the Huntingtin (“HTT”) gene. Approximately 40% of the HD population carries SNP3 according to published literature (Carroll et al., Molecular Therapy, 2011).
Current wording
In HD, we are currently advancing WVE-003, a stereopure allele-selective oligonucleotide designed to selectively target an undisclosed single nucleotide polymorphism (“SNP”), “mHTT SNP3”, associated with the disease-causing mHTT mRNA transcript within the Huntingtin (“HTT”) gene. Approximately 40% of the HD population carries SNP3 according to published literature (Carroll et al., Molecular Therapy, 2011), and up to 80% of HD may be addressed in the future with other SNP-targeted candidates.
Previous wording
WVE-003 incorporates our novel PN chemistry. Targeting mRNA with SNP3 allows us to lower expression of transcript from the mutant allele, while leaving the healthy transcript relatively intact, thereby preserving wild-type (healthy) huntingtin (“wtHTT”) protein, which is important for neuronal function. Only an allele-selective approach to mHTT lowering has the potential to both protect the reservoir of wtHTT protein and decrease the mHTT to wtHTT ratio in neurons, potentially releasing wtHTT from the inhibitory actions of mHTT. Our allele-selective approach may also enable us to address the premanifest HD patient population in the future. In preclinical studies, WVE-003 showed dose-dependent and selective reduction of mHTT mRNAin vitro, as well as potent and durable knockdown of mHTT mRNA and proteinin vivo in mouse models.
Current wording
WVE-003 incorporates our proprietary PN chemistry. Targeting mRNA with SNP3 allows us to lower expression of transcript from the mutant allele, while leaving the healthy transcript relatively intact, thereby preserving wild-type (healthy) huntingtin (“wtHTT”) protein, which is important for neuronal function. Only an allele-selective approach to mHTT lowering has the potential to both protect the reservoir of wtHTT protein and decrease the mHTT to wtHTT ratio in neurons, potentially releasing wtHTT from the inhibitory actions of mHTT. Our allele-selective approach may also enable us to address HD patient populations in early stages of disease prior to onset of clinical symptoms. In preclinical studies, WVE-003 showed dose-dependent and selective reduction of mHTT mRNAin vitro, as well as potent and durable knockdown of mHTT mRNA and proteinin vivo in mouse models.
Previous wording
In the third quarter of 2023, we achieved a milestone in our collaboration with Takeda, which pertained to the positive results from a non-clinical study of WVE-003 in non-human primates and resulted in a payment of $7.0 million to us 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 our existing datasets that confirm the ability of our oligonucleotides to distribute to the areas of the CNS important for HD.
Current wording
In the third quarter of 2023, we achieved a milestone in our collaboration with Takeda Pharmaceutical Company Limited (“Takeda”), which pertained to the positive results from a non-clinical study of WVE-003 in non-human primates and resulted in a payment of $7.0 million to us. This study showed significant tissue exposure levels of WVE-003 in the deep brain regions, including striatum and bolstered our existing datasets that confirm the ability of our oligonucleotides to distribute to the areas of the CNS important for HD.
Previous wording
We expect to select five new clinical candidates by the end of 2025, including our INHBE candidate for obesity.
Current wording
We expect to select five new clinical candidates by the end of 2025, including WVE-007, our INHBE candidate for obesity.
Previous wording
We have never been profitable, and since our inception, we have incurred significant operating losses. Our net loss was $31.6 million and $27.4 million for the three months ended March 31, 2024 and 2023, respectively. As of March 31, 2024 and December 31, 2023, we had an accumulated deficit of $1,056.4 million and $1,024.9 million, respectively. We expect to continue to incur significant expenses and operating losses for the foreseeable future.
Current wording
We have never been profitable, and since our inception, we have incurred significant operating losses. Our net loss for the three months ended June 30, 2024 and 2023 was $32.9 million and $21.1 million, respectively. Our net loss for the six months ended June 30, 2024 and 2023 was $64.5 million and $48.5 million, respectively. As of June 30, 2024 and December 31, 2023, we had an accumulated deficit of $1,089.3 million and $1,024.9 million, respectively. We expect to continue to incur significant expenses and operating losses for the foreseeable future.
Previous wording
Revenue for the three months ended March 31, 2024 and 2023 was $12.5 million and $12.9 million, respectively, and is comprised of revenue earned under the GSK Collaboration Agreement and the Takeda Collaboration Agreement. The revenue earned under the GSK Collaboration Agreement was consistent year-over-year. There was a slight decrease in the revenue earned under the Takeda Collaboration Agreement year-over-year.
Current wording
Revenue for the three months ended June 30, 2024 and 2023 was $19.7 million and $22.1 million, respectively, and is comprised of revenue earned under the GSK Collaboration Agreement and the Takeda Collaboration Agreement. There was a slight decrease in the revenue earned under both the GSK Collaboration Agreement and the Takeda Collaboration Agreement year-over-year.
Previous wording
Includes expenses related to other research and development programs, identification of potential drug discovery candidates, compensation-related expenses, internal manufacturing expenses, equipment repairs and maintenance expense, facility-related expenses and other operating expenses, which are not allocated to specific programs.
Current wording
Includes expenses related to discovery and development programs, identification of potential drug discovery candidates, compensation-related expenses, internal manufacturing expenses, equipment repairs and maintenance expense, facility-related expenses and other operating expenses, which are not allocated to specific programs.
Previous wording
Research and development expenses were $33.4 million for the three months ended March 31, 2024, compared to $31.0 million for the three months ended March 31, 2023. The increase of approximately $2.5 million was due to the following:
Current wording
Research and development expenses were $40.4 million for the three months ended June 30, 2024, compared to $33.3 million for the three months ended June 30, 2023. The increase of approximately $7.1 million was due to the following:
Previous wording
an increase of $1.6 million in external expenses related to our AATD program, WVE-006 (RNA editing);
Current wording
an increase of $1.0 million in external expenses related to our AATD program, WVE-006 (RNA editing);
Previous wording
an increase of $2.7 million in external expenses related to our DMD programs, including WVE-N531 (splicing);
Current wording
an increase of $1.8 million in external expenses related to our DMD programs, including WVE-N531 (splicing);
Previous wording
a decrease of $0.9 million in external expenses related to our HD programs, including WVE-003 (silencing);
Current wording
a decrease of $0.5 million in external expenses related to our HD programs, including WVE-003 (silencing);
Previous wording
an increase of $1.7 million in other research and development expenses(1), including INHBE, RNA editing, PRISM, and other internal and external research and development expenses that are not allocated on a program-by-program basis or are related to other discovery and development programs, and the identification of potential drug discovery candidates, mainly due to increases in compensation-related expenses and other external research and development expenses, partially offset by decreases in facilities-related expenses; and
Current wording
an increase of $8.0 million in other research and development expenses(1), including INHBE, RNA editing, PRISM, and other internal and external research and development expenses that are not allocated on a program-by-program basis or are related to other discovery and development programs, and the identification of potential drug discovery candidates, mainly due to increases in compensation-related expenses and facilities-related expenses, partially offset by decreases in other external research and development expenses; and
Previous wording
a decrease of $2.7 million in external expenses related to our discontinued ALS and FTD program, WVE-004.
Current wording
a decrease of $3.3 million in external expenses related to our discontinued ALS and FTD program, WVE-004.
Previous wording
General and administrative expenses were $13.5 million for the three months ended March 31, 2024, as compared to $12.2 million for the three months ended March 31, 2023. The increase is primarily driven by increases in professional fees and other external general and administrative expenses.
Current wording
General and administrative expenses were $14.3 million for the three months ended June 30, 2024, as compared to $12.3 million for the three months ended June 30, 2023. The increase is primarily driven by increases in compensation related expenses and administrative expenses.
Previous wording
Since our inception, we have not generated any product revenue and have incurred recurring net operating losses. To date, we have primarily funded our operations through public and other registered offerings of our ordinary shares and other securities, collaborations with third parties and private placements of debt and equity securities. Through March 31, 2024, we have received an aggregate of approximately $1,330.2 million in net proceeds from these transactions, consisting of $741.6 million in net proceeds from public and other registered offerings of our ordinary shares and other securities, $499.3 million from our collaborations and $89.3 million in net proceeds from private placements of our debt and equity securities.
Current wording
Since our inception, we have not generated any product revenue and have incurred recurring net operating losses. To date, we have primarily funded our operations through public and other registered offerings of our ordinary shares and other securities, collaborations with third parties and private placements of debt and equity securities. Through June 30, 2024, we have received an aggregate of approximately $1,342.7 million in net proceeds from these transactions, consisting of $742.1 million in net proceeds from public and other registered offerings of our ordinary shares and other securities, $511.3 million from our collaborations and $89.3 million in net proceeds from private placements of our debt and equity securities.
Previous wording
As of March 31, 2024, we had cash and cash equivalents totaling $180.9 million, restricted cash of $3.7 million and an accumulated deficit of $1,056.4 million.
Current wording
As of June 30, 2024, we had cash and cash equivalents totaling $154.0 million, restricted cash of $3.7 million and an accumulated deficit of $1,089.3 million. Subsequent to June 30, 2024, we received $12.7 million in net proceeds under our at-the-market equity program.
Previous wording
Our operating lease commitments as of March 31, 2024 total approximately $36.1 million, of which $7.0 million is related to payments in 2024 and approximately $29.1 million is related to payments beyond 2024.
Current wording
Our operating lease commitments as of June 30, 2024 total approximately $33.7 million, of which approximately $4.6 million is related to payments in 2024 and approximately $29.1 million is related to payments beyond 2024.
Previous wording
On March 3, 2022, we filed a new universal shelf registration on Form S-3 with the SEC, which was declared effective by the SEC on May 4, 2022, pursuant to which we registered for sale up to $500.0 million of any combination of our ordinary shares, debt securities, warrants, rights and/or units from time to time and at prices and on terms that we may determine, which we refer to as the “2022 Form S-3.” The 2022 Form S-3 includes a prospectus covering up to approximately $132.0 million in ordinary shares that had not yet been issued or sold under our Sales Agreement with Jefferies at the time the 2022 Form S-3 was declared effective. As of March 31, 2024, we have $311.7 million in securities available for issuance under the 2022 Form S-3, including approximately $128.7 million in ordinary shares available for issuance under our at-the-market equity program.
Current wording
On March 3, 2022, we filed a new universal shelf registration on Form S-3 with the SEC, which was declared effective by the SEC on May 4, 2022, pursuant to which we registered for sale up to $500.0 million of any combination of our ordinary shares, debt securities, warrants, rights and/or units from time to time and at prices and on terms that we may determine, which we refer to as the “2022 Form S-3.” The 2022 Form S-3 includes a prospectus covering up to approximately $132.0 million in ordinary shares that had not yet been issued or sold under our Sales Agreement with Jefferies at the time the 2022 Form S-3 was declared effective. As of August 7, 2024, we have $298.2 million in securities available for issuance under the 2022 Form S-3, including approximately $115.2 million in ordinary shares available for issuance under our at-the-market equity program.
Previous wording
During the three months ended March 31, 2024, operating activities used $33.4 million of cash, due to our net loss of $31.6 million and changes in operating assets and liabilities of $6.9 million, offset by non-cash charges of $5.1 million. The largest changes in operating assets and liabilities were the $21.1 million decrease in accounts receivable related to collection of accounts receivable under the GSK Collaboration Agreement, the $12.5 million decrease in deferred revenue, and the $10.2 million decrease in accrued expenses and other current liabilities.
Current wording
During the six months ended June 30, 2024, operating activities used $60.9 million of cash, due to our net loss of $64.5 million and changes in operating assets and liabilities of $7.1 million, offset by non-cash charges of $10.7 million.
Previous wording
During the three months ended March 31, 2023, operating activities provided $85.5 million of cash, primarily due to the $126.4 million increase in deferred revenue, driven by our GSK Collaboration Agreement, which became effective in January 2023, partially offset by our net loss of $27.4 million, the $9.9 million decrease in accrued expenses and other current liabilities and the $4.7 million decrease in accounts payable.
Current wording
During the six months ended June 30, 2023, operating activities provided $48.3 million of cash, due to our net loss of $48.5 million, offset by non-cash charges of $9.9 million and changes in operating assets and liabilities of $86.9 million. The largest change in operating assets and liabilities was a $104.3 million increase in deferred revenue, mainly driven by our GSK Collaboration Agreement, which became effective in January 2023.
Previous wording
During the three months ended March 31, 2023, investing activities used $0.5 million of cash, related to purchases of property and equipment.
Current wording
During the six months ended June 30, 2024, investing activities used $0.5 million of cash, related to purchases of property and equipment.
Previous wording
During the three months ended March 31, 2024, investing activities used $0.4 million of cash, related to purchases of property and equipment.
Current wording
During the six months ended June 30, 2023, investing activities used $0.6 million of cash, related to purchases of property and equipment.
Previous wording
During the three months ended March 31, 2024, net cash provided by financing activities was $14.5 million, primarily due to the $14.0 million in net proceeds from the January 2024 exercise of the underwriters’ option to purchase an additional 3,000,000 shares under the December 2023 Offering.
Current wording
During the six months ended June 30, 2024, net cash provided by financing activities was $15.2 million, which was primarily due to the $14.0 million in net proceeds from the January 2024 exercise of the underwriters’ option to purchase an additional 3,000,000 shares under the December 2023 Offering. Additionally, we received $0.5 million in net proceeds from sales under our at-the-market equity program.
Previous wording
During the three months ended March 31, 2023, net cash provided by financing activities was $35.1 million, which was primarily due to the GSK Equity Investment (as defined in Note 5).
Current wording
During the six months ended June 30, 2023, net cash provided by financing activities was $36.9 million, which was primarily due to the GSK Equity Investment (as defined in Note 5).
Previous wording
respond to the impacts of local and global health epidemics, the conflict involving Russia and Ukraine, the conflict in the Middle East, global economic uncertainty, rising inflation, rising interest rates or market disruptions on our business; and
Current wording
respond to the impacts of the local and global health epidemics, the conflict involving Russia and Ukraine, the conflict in the Middle East, global economic uncertainty, rising inflation, rising interest rates or market disruptions on our business; and
Previous wording
the impacts of local and global health epidemics, the conflict involving Russia and Ukraine, the conflict in the Middle East, global economic uncertainty, rising inflation, rising interest rates or market disruptions on our business;
Current wording
the impacts of the local and global health epidemics, the conflict involving Russia and Ukraine, the conflict in the Middle East, global economic uncertainty, rising inflation, rising interest rates or market disruptions on our business;
Rearranged current paragraphs (8)
Current report — wording carried from elsewhere
Revenue for the six months ended June 30, 2024 and 2023 was $32.2 million and $35.0 million, respectively, and is comprised of revenue earned under the GSK Collaboration Agreement and the Takeda Collaboration Agreement. There was a slight decrease in the revenue earned under both the GSK Collaboration Agreement and the Takeda Collaboration Agreement year-over-year.
Current report — wording carried from elsewhere
Includes expenses related to discovery and development programs, identification of potential drug discovery candidates, compensation-related expenses, internal manufacturing expenses, equipment repairs and maintenance expense, facility-related expenses and other operating expenses, which are not allocated to specific programs.
Current report — wording carried from elsewhere
an increase of $2.6 million in external expenses related to our AATD program, WVE-006 (RNA editing);
Current report — wording carried from elsewhere
an increase of $4.5 million in external expenses related to our DMD programs, including WVE-N531 (splicing);
Current report — wording carried from elsewhere
a decrease of $1.4 million in external expenses related to our HD programs, including WVE-003 (silencing);
Current report — wording carried from elsewhere
an increase of $9.7 million in other research and development expenses(1), including INHBE, RNA editing, PRISM, and other internal and external research and development expenses that are no allocated on a program-by-program basis or are related to other discovery and development programs, and the identification of potential drug discovery candidates, mainly due to increases in compensation-related expenses and facilities-related expenses, partially offset by decreases in other external research and development expenses; and
Current report — wording carried from elsewhere
a decrease of $5.9 million in external expenses related to our discontinued ALS and FTD program, WVE-004.
Current report — wording carried from elsewhere
During the six months ended June 30, 2024 and 2023, we recorded no income tax provision. We maintained a full valuation allowance for the six months ended June 30, 2024 and 2023 in all jurisdictions due to uncertainty regarding future taxable income.