Full Press Release Details
Wave Life Sciences Reports Second Quarter 2022 Financial Results and Provides Business Update
WVE-006 announced as investigational development candidate for AATD with CTA submissions expected in
first-in-class RNA editing candidate and most
advanced program currently in development using an oligonucleotide to harness an endogenous enzyme for editing
clinical programs, including WVE-004, WVE-003, and WVE-N531, expected in 2H 2022
Positive target engagement data in C9-ALS/FTD with single low doses of WVE-004 highlighted at ENCALS conference
$70 million financing in June 2022 strengthened
balance sheet and extended cash runway to the end of 2023
Wave to host investor conference call and webcast at 8:30 a.m. ET today
CAMBRIDGE, Mass., August 11, 2022 Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company
committed to delivering life-changing treatments for people battling devastating diseases, today announced financial results for the second quarter ended June 30, 2022 and provided a business update.
At Wave, we are urgently working to advance our lead programs for the treatment of devastating genetic diseases. Since the start of the second quarter
of 2022, we have achieved multiple important milestones, including positive target engagement data in our FOCUS-C9 clinical trial and the selection of our first RNA editing, or AIMer , development
candidate, WVE-006 for the treatment of AATD, said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. RNA editing is a novel therapeutic modality and Wave is leading
this field with our AIMers. These RNA base editing therapeutics are being advanced with and without GalNAc conjugation and avoid the need for large, complex delivery vehicles, such as lipid nanoparticles or viral vectors. Our recent financing
ensures we are well-capitalized to execute on several key milestones, including data announcements from three ongoing clinical trials and the advancement of WVE-006 into clinical development.
Recent Pipeline and Business Highlights
Alpha-1 antitrypsin deficiency clinical candidate (WVE-006) selected; program designed to correct mutant AATD transcript to address both liver and lung manifestations of
FOCUS-C9 clinical trial of WVE-004 for C9-ALS and C9-FTD continues to advance; previously announced positive target engagement data highlighted at ENCALS meeting
SELECT-HD clinical trial for WVE-003 in Huntington s disease (HD) continues to advance; only allele-selective clinical program designed to reduce mutant HTT and spare
Phase 1b/2a clinical trial of WVE-N531 in Duchenne muscular dystrophy (DMD) amenable to exon
53 skipping continues to advance; dosing underway at fourth dose level
Extended cash runway with successful financing
Key Anticipated Upcoming Milestones
WVE-006 (GalNAc-conjugated AIMer) for AATD:
WVE-004 for C9-ALS and C9-FTD:
Recent Scientific Publications
Second Quarter 2022 Financial Results and Financial Guidance
Wave reported a net loss of $41.3 million in the second quarter of 2022, as compared to $38.8 million in the same period in 2021.
Wave recorded revenue of $0.4 million for the second quarter of 2022, as compared to $2.8 million in the same period in 2021.
Research and development expenses were $29.7 million in the second quarter of 2022 as compared to $31.6 million in the same period in
2021. The decrease in research and development expenses in the second quarter was primarily due to decreased external expenses related to our previously disclosed discontinued PRECISION-HD programs, partially
offset by increased share-based compensation expense and increased external expenses related to AATD.
General and administrative expenses
were $12.8 million in the second quarter of 2022 as compared to $11.0 million in the same period in 2021. The increase in general and administrative expenses in the second quarter of 2022 was primarily due to increases in
compensation-related expenses, primarily from share-based compensation, offset by decreases in other external general and administrative expenses.
of June 30, 2022, Wave had $148.2 million in cash, cash equivalents and short-term investments. As of December 31, 2021, Wave had $150.6 million in cash and cash equivalents. The net decrease is primarily due to Wave s
year to date operating cash burn, partially offset by $65.5 million net proceeds from its financing in June.
Wave expects that its existing cash,
cash equivalents and short-term investments will enable the company to fund its operating and capital expenditure requirements to the end of 2023.
Investor Conference Call and Webcast
Wave management will host an investor conference call today at 8:30 a.m. ET to discuss the company s second quarter 2022 financial results and provide a
business update. The webcast of the conference call may be accessed by visiting Events on the investor relations section of the Wave Life Sciences corporate website:
ir.wavelifesciences.com/events-and-presentations.
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. It is recommended that participants register at least 15 minutes in advance of the
call. 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 the FOCUS-C9 Clinical Trial
The FOCUS-C9 trial is an ongoing, global, multicenter, randomized, double-blind, placebo-controlled Phase
1b/2a clinical trial to assess the safety and tolerability of single- and multiple-ascending intrathecal doses of WVE-004 for people with C9-ALS and/or C9-FTD. Additional objectives include measurement of poly(GP) DPR proteins in the cerebrospinal fluid (CSF), plasma and CSF pharmacokinetics (PK), and exploratory biomarkers and clinical outcomes. The FOCUS-C9 trial is designed to be adaptive, with dose escalation and dosing frequency being guided by an independent committee.
In an initial data analysis, reductions in poly(GP) were observed across all active treatment groups (10 mg, n=2 patients; 30 mg, n=4 patients; 60 mg, n=3
patients), reaching statistical significance versus placebo (n=3 patients) after single 30 mg doses, with a 34% reduction in poly(GP) at day 85 (p=0.011). At the time of analysis, none of the patients dosed with 60 mg had reached day 85. As the
poly(GP) reduction in the 30 mg single dose cohort does not appear to have plateaued, Wave will extend the observation period from approximately three months (85 days) to approximately six months to identify the maximum reduction of poly(GP) and
duration of effect of low single doses. Based on the durability and potency observed in the 30 mg cohort, FOCUS-C9 has been adapted to include additional patients receiving 20 mg and 30 mg single doses of WVE-004. Adverse events (AEs) were balanced across treatment groups, including placebo, and were mostly mild to moderate in intensity. Four patients (including one on placebo) experienced severe and/or serious
adverse events; three were reported by the investigators to be related to ALS or administration, and one was reported by the investigator to be related to study drug. There were no treatment-associated elevations in CSF white blood cell counts or
protein and no other notable laboratory abnormalities were observed.
Support for FOCUS-C9 is provided by the
Alzheimer s Drug Discovery Foundation.
About Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease in which the progressive degeneration of motor neurons in the brain and spinal cord
leads to the inability to initiate or control muscle movement. People with ALS may lose the ability to speak, eat, move and breathe. ALS affects as many as 20,000 people in the United States.
Frontotemporal dementia (FTD) is a fatal neurodegenerative disease in which progressive nerve cell loss in the brain s frontal lobes and temporal lobes
leads to personality and behavioral changes, as well as the gradual impairment of language skills. It is the second most common form of early-onset dementia after Alzheimer s disease in people under the age of 65. FTD affects as many as 70,000
people in the United States.
A hexanucleotide repeat expansion (G4C2) is the most common known genetic cause of the sporadic and inherited forms of ALS and FTD. The expansion leads to production of modified sense and antisense transcripts that can form nuclear RNA
foci and encode dipeptide protein repeats (DPRs), which are believed to drive disease pathology. Additionally, the G4C2 expansion can
decrease expression of C9orf72 protein, affecting regulation of neuronal function and the immune system.
In the United States, mutations of the C9orf72 gene are present in approximately 40% of familial ALS cases
and ~8-10% of sporadic ALS cases. In FTD, the mutations appear in 38% of familial cases and 6% of sporadic cases.
About Huntington s Disease
disease (HD) is a debilitating and ultimately fatal autosomal dominant neurological disorder, characterized by cognitive decline, psychiatric illness, and chorea. HD causes nerve cells in the brain to deteriorate over time, affecting thinking
ability, emotions, and movement. HD is caused by an expanded cytosine-adenine-guanine (CAG) triplet repeat in the huntingtin (HTT) gene that results in production of mutant HTT (mHTT) protein. Accumulation of mHTT causes progressive loss of neurons
in the brain. Wild-type, or healthy, HTT (wtHTT) protein is critical for neuronal function and suppression may have detrimental long-term consequences. Approximately 30,000 people in the United States have symptomatic HD and more than 200,000 others
are at risk for developing the disease. There are currently no approved disease-modifying therapies available.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic neuromuscular disorder caused predominantly by out-of-frame deletions in the dystrophin gene, resulting in absent or defective dystrophin protein. Dystrophin protein is needed for normal muscle maintenance and operation.
Because of the genetic mutations in DMD, the body cannot produce functional dystrophin, which results in progressive and irreversible loss of muscle function, including the heart and lungs. Worldwide, DMD affects approximately one in 5,000 newborn
boys. Approximately 8%-10% of DMD patients have mutations amenable to treatment with an exon 53 skipping therapy. Exon skipping aims to address the underlying cause of DMD by promoting the production of
dystrophin protein to stabilize or slow disease progression
PRISM is Wave Life Sciences proprietary discovery and drug development platform that enables genetically defined diseases to be targeted with stereopure
oligonucleotides across multiple therapeutic modalities, including silencing, splicing and editing. PRISM combines the company s unique ability to construct stereopure oligonucleotides with a deep understanding of how the interplay among
oligonucleotide sequence, chemistry and backbone stereochemistry impacts key pharmacological properties. By exploring these interactions through iterative analysis of in vitro and in vivo outcomes and machine
learning-driven predictive modeling, the company continues to define design principles that are deployed across programs to rapidly develop and manufacture clinical candidates that meet pre-defined product
About Wave Life Sciences
Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases. Wave aspires to develop
best-in-class medicines across multiple therapeutic modalities using PRISM, the company s proprietary discovery and drug development platform that enables the
precise design, optimization, and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, the Wave team is targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future.
To find out more, please visit www.wavelifesciences.com and follow Wave on Twitter @WaveLifeSci.
Forward-Looking 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
adaptive clinical trials, and the announcement of such events; the protocol, design and endpoints of our ongoing and future clinical trials; the future performance and results of our programs in clinical trials; future preclinical activities and
programs; regulatory submissions; the progress and potential benefits of our collaborations with partners; the potential of our 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 therapeutic benefits of our potential therapies compared to others; our ability to design compounds using multiple modalities and the anticipated benefits of that approach; the
potential benefits of PRISM, including our novel PN backbone chemistry modifications, and our stereopure oligonucleotides compared with stereorandom oligonucleotides; the potential benefits of our novel ADAR-mediated RNA editing platform
capabilities, including our AIMers, compared to others; the status and progress of our novel 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; our assumptions based on our balance sheet and the anticipated duration of our cash runway; our intended uses of capital; and our expectations regarding the
impact of the COVID-19 pandemic on our business. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including the following: