Wave Life Sciences Studies Fourth Quarter and Full Yr 2021 Monetary Outcomes and Supplies Enterprise Replace

Wave Life Sciences USA, Inc.

Medical information from a number of novel, PN-modified stereopure oligonucleotides for ALS/FTD, DMD, and HD anticipated in 2022

GalNAc-AIMers restore therapeutically related ranges of AAT for lung safety and scale back liver-damaging aggregates in preclinical examine; IND enabling toxicology research anticipated to provoke in 3Q 2022

FY2021 year-end money whole of $150.6 million offering runway into 2Q 2023

Wave to host investor convention name and webcast at 8:30 a.m. ET at present

CAMBRIDGE, Mass., March 03, 2022 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines firm dedicated to delivering life-changing therapies for individuals battling devastating illnesses, at present introduced monetary outcomes for the fourth quarter and full yr ended December 31, 2021 and supplied a enterprise replace.

“Wave achieved a number of important milestones in 2021, together with efficiently bringing PN chemistry into the clinic with the initiation of three new scientific trials with our next-generation RNA silencing and exon-skipping therapeutics, in addition to demonstrating the primary profitable protein restoration with AIMers in preclinical in vivo fashions for the remedy of alpha-1 antitrypsin deficiency, also referred to as AATD. These accomplishments have positioned us to ship a number of key datasets in 2022 to tell the potential of our novel oligonucleotides throughout tissues and modalities,” mentioned Paul Bolno, MD, MBA, President and Chief Government Officer of Wave Life Sciences.

“We proceed to quickly advance our AIMer RNA modifying capabilities and are poised to ship a first-in-class novel modality to deal with each lung and liver manifestations of AATD. We stay on observe to pick our first GalNAc-AIMer improvement candidate within the third quarter of this yr, and we’re main the way in which with RNA base modifying to deal with a wide selection of genetic illnesses, with doubtlessly much more expansive purposes by way of protein modulation. Lastly, our decade of funding in our PRISM platform has resulted in a sturdy and various pipeline, in addition to inside GMP manufacturing capabilities that may be scaled to assist our wants in addition to potential new companions,” continued Dr. Bolno.

Latest Enterprise Highlights and Upcoming Milestones

Medical silencing and exon skipping therapeutic packages:

Scientific publications:

  • In February 2022, Wave introduced two publications within the journal Nucleic Acids Analysis (NAR) supporting the incorporation of PN spine chemistry modifications (PN chemistry) in stereopure oligonucleotides as a major development for the therapeutic oligonucleotide discipline. Within the multitude of in vitro and in vivo (animal) research highlighted in Wave’s papers, PN chemistry was proven to dramatically enhance efficiency, distribution, and sturdiness of impact. The papers discover using PN chemistry in stereopure silencing oligonucleotides (publication hyperlink) for central nervous system (CNS) illnesses – designated as a Breakthrough Article by NAR — and stereopure splicing oligonucleotides (publication hyperlink) for neuromuscular illnesses.

WVE-N531 for Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping:

  • WVE-N531 (PN-modified splicing oligonucleotide) is being evaluated in an open-label, intra-patient dose escalation scientific trial. Dose escalation is ongoing and being guided by tolerability and plasma PK, with potential cohort enlargement knowledgeable by an evaluation of drug distribution in muscle and biomarkers, together with dystrophin, following a number of doses of WVE-N531.

  • When evaluating PN-modified compounds, together with WVE-N531, to first-generation PS/PO (non-PN-modified) compounds, PN chemistry constantly results in elevated exon-skipping exercise, will increase in muscle publicity, longer half-life, and extra sturdy results in preclinical mouse and non-human primate research. Primarily based on an evaluation of preliminary plasma PK from the beginning single dose of WVE-N531 in Wave’s ongoing scientific trial, there was a considerable improve in plasma concentrations and a transparent improve in plasma half-life as in comparison with suvodirsen, Wave’s first-generation PS/PO exon-skipping compound.

WVE-004 for C9orf72-associated amyotrophic lateral sclerosis (C9-ALS) and frontotemporal dementia (C9-FTD):

  • FOCUS-C9 is an ongoing, double-blind, adaptive, Part 1b/2a scientific trial of WVE-004. WVE-004 is an investigational stereopure PN-modified silencing oligonucleotide designed to selectively goal transcript variants containing a hexanucleotide repeat enlargement (G4C2) related to the C9orf72 gene for the remedy of C9-ALS and C9-FTD.

  • In January 2022, the Alzheimer’s Drug Discovery Basis (ADDF) and The Affiliation for Frontotemporal Degeneration (AFTD) introduced they’d partnered to assist Wave’s FOCUS-C9 scientific trial, particularly the analysis of fluid biomarkers, purposeful assessments, and digital biomarkers used within the examine, doubtlessly resulting in clinically significant endpoints to tell drug improvement for FTD. The choice to assist the FOCUS-C9 trial was made following a evaluation by members of the Deal with FTD Fund Joint Steering Committee of Wave’s Part 1b/2a examine plan, preclinical information supporting this system and experience of the examine workforce.

WVE-003 concentrating on SNP3 for Huntington’s illness (HD):

  • SELECT-HD is an ongoing, double-blind, adaptive, Part 1b/2a scientific trial of WVE-003. WVE-003 is an investigational stereopure PN-modified silencing oligonucleotide designed to selectively goal the mutant allele of the huntingtin (mHTT) gene, whereas leaving the wild-type (wholesome) HTT (wtHTT) protein comparatively intact.

  • In March 2022, Wave introduced on the CHDI Basis’s 17th Annual HD Therapeutics Convention, together with a poster titled “A novel quantitative wild-type huntingtin (wtHTT) protein biomarker technique for human cerebrospinal fluid” that highlights Wave’s wtHTT assay, which is meant to evaluate preservation of wtHTT protein in CSF within the setting of mHTT concentrating on, together with within the ongoing SELECT-HD scientific trial.

Upcoming scientific milestones:

  • Wave expects to share scientific information in 2022 for WVE-004, WVE-003, and WVE-N531 to offer perception into the scientific results of PN chemistry and allow decision-making for every program.

ADAR modifying therapeutic packages (RNA modifying utilizing endogenous ADAR enzymes)

Scientific shows:

  • In January 2022, Wave gave an oral presentation titled “In direction of the event of a therapeutic RNA modifying platform” on the 3rd Worldwide Convention on Base Modifying – Enzymes and Purposes Deaminet 2022, which highlighted Wave’s RNA modifying platform, the flexibility of AIMers to revive expression of purposeful protein in preclinical fashions in vivo and modulate protein-protein interactions in vitro.

  • Wave management will current on the upcoming 3rd RNA Modifying Summit on April 5 – 7, 2022 in Boston.

AATD program updates and upcoming milestones:

  • Wave at present introduced new preclinical information demonstrating restoration of purposeful AAT protein in a transgenic mouse mannequin with GalNAc-conjugated SERPINA1 AIMers. At 19 weeks, AIMer remedy resulted in roughly 60% RNA modifying of SERPINA1 transcript and circulating serum AAT ranges (18.5 uM) in AIMer handled mice that had been roughly 5-fold larger than PBS-treated controls.

  • At present, Wave additionally shared a histological evaluation that signifies discount of liver aggregates in a transgenic mouse mannequin at 19 weeks with AIMer remedy.

  • In November 2021, Wave introduced a poster at AASLD: The Liver Assembly, that included information demonstrating SERPINA1 AIMers obtain extremely particular RNA modifying in vivo, leading to wild-type, M-AAT protein circulating in serum that was purposeful in a neutrophil elastase inhibition assay.

  • Wave expects to pick an AATD AIMer improvement candidate and provoke IND-enabling toxicology research within the third quarter of 2022.

Fourth Quarter and Full Yr 2021 Monetary Outcomes and Monetary Steering

Wave reported a web lack of $34.8 million within the fourth quarter of 2021, as in comparison with $28.8 million in the identical interval in 2020. Wave reported a web lack of $122.2 million for the yr ended December 31, 2021, as in comparison with $149.9 million for the yr ended December 31, 2020.

Income earned beneath the Takeda Collaboration within the fourth quarter of 2021 was $1.8 million, as in comparison with $9.4 million for a similar interval in 2020. The lower in income year-over-year is especially as a result of modification of Wave’s collaboration with Takeda, which discontinued the Class 2 discovery analysis element of the Takeda Collaboration in change for an extra $22.5 million, which Wave obtained in October 2021 and accounted for within the third quarter of 2021. The Class 1 late-stage element of the Takeda Collaboration stays in impact and was unchanged by the modification. Throughout the yr ended December 31, 2021, Wave earned $41.0 million beneath the Takeda Collaboration, as in comparison with $20.1 million earned beneath the Takeda Collaboration and the Pfizer Collaboration through the yr ended December 31, 2020. The year-over-year improve is primarily pushed by recognition of income associated to the $22.5 million associated to the Takeda Modification.

Analysis and improvement bills had been $25.8 million within the fourth quarter of 2021 as in comparison with $30.0 million in the identical interval in 2020. Analysis and improvement bills had been $121.9 million in 2021, as in comparison with $130.9 million in 2020. The lower in analysis and improvement bills within the fourth quarter and full yr was primarily resulting from decreased exterior bills associated to our beforehand disclosed discontinued PRECISION-HD packages, partially offset by elevated inside and exterior bills associated to WVE-004, PRISM, together with ADAR modifying, and different ongoing packages.

Normal and administrative bills had been $12.1 million within the fourth quarter of 2021 as in comparison with $9.7 million in the identical interval in 2020. Normal and administrative bills had been $46.1 million in 2021, as in comparison with $42.5 million in 2020. The rise normally and administrative bills within the fourth quarter of 2021 and full yr was pushed by will increase in compensation-related and different exterior normal and administrative bills.

As of December 31, 2021, Wave had $150.6 million in money and money equivalents as in comparison with $184.5 million as of December 31, 2020. The lower in money and money equivalents was primarily resulting from Wave’s year-to-date web lack of $122.2 million, partially offset by the receipt of $54.9 million in web proceeds beneath Wave’s ATM fairness program and funds of $52.5 million obtained from our collaboration with Takeda.

Wave expects that its present money and money equivalents will allow the corporate to fund its working and capital expenditure necessities into the second quarter of 2023.

Investor Convention Name and Webcast
Wave administration will host an investor convention name at present at 8:30 a.m. ET to debate the corporate’s fourth quarter and full yr 2021 monetary outcomes and supply a enterprise replace. The convention name could also be accessed by dialing (866) 220-8068 (home) or (470) 495-9153 (worldwide) and coming into convention ID: 7694386. The reside webcast could also be accessed from the Investor Relations part of the Wave Life Sciences company web site at ir.wavelifesciences.com. Following the webcast, a replay will probably be out there on the web site.

About PRISM™
PRISM is Wave Life Sciences’ proprietary discovery and drug improvement platform that permits genetically outlined illnesses to be focused with stereopure oligonucleotides throughout a number of therapeutic modalities, together with silencing, splicing and modifying. PRISM combines the corporate’s distinctive means to assemble stereopure oligonucleotides with a deep understanding of how the interaction amongst oligonucleotide sequence, chemistry and spine stereochemistry impacts key pharmacological properties. By exploring these interactions by way of iterative evaluation of in vitro and in vivo outcomes and machine learning-driven predictive modeling, the corporate continues to outline design ideas which are deployed throughout packages to quickly develop and manufacture scientific candidates that meet pre-defined product profiles.

About Wave Life Sciences
Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines firm dedicated to delivering life-changing therapies for individuals battling devastating illnesses. Wave aspires to develop best-in-class medicines throughout a number of therapeutic modalities utilizing PRISM, the corporate’s proprietary discovery and drug improvement platform that permits the exact design, optimization, and manufacturing of stereopure oligonucleotides. Pushed by a resolute sense of urgency, the Wave workforce is concentrating on a broad vary of genetically outlined illnesses in order that sufferers and households could understand a brighter future. To search out out extra, please go to www.wavelifesciences.com and comply with Wave on Twitter @WaveLifeSci.

Ahead-Trying Statements
This press launch incorporates forward-looking statements regarding our objectives, beliefs, expectations, methods, aims and plans, and different statements that aren’t essentially based mostly on historic information, together with statements relating to the next, amongst others: the anticipated initiation, web site activation, affected person recruitment, affected person enrollment, dosing, era of knowledge for decision-making and completion of our adaptive scientific trials, and the announcement of such occasions; the protocol, design and endpoints of our ongoing and deliberate scientific trials; the long run efficiency and outcomes of our packages in scientific trials; future preclinical actions and packages; regulatory submissions; the progress and potential advantages of our collaborations with companions; the potential of our in vitro and in vivo preclinical information to foretell the habits of our compounds in people; our identification and anticipated timing of future product candidates and their therapeutic potential; the anticipated therapeutic advantages of our potential therapies in comparison with others; our means to design compounds utilizing a number of modalities and the anticipated advantages of that mannequin; the potential advantages of PRISM, together with our novel PN spine chemistry modifications, and our stereopure oligonucleotides in contrast with stereorandom oligonucleotides; the potential advantages of our novel ADAR-mediated RNA modifying platform capabilities, together with our AIMers, in comparison with others; anticipated advantages of our proprietary manufacturing processes and our inside manufacturing capabilities; the advantage of nucleic acid therapeutics usually; the power of our mental property; our assumptions based mostly on our stability sheet and the anticipated length of our money runway; our meant makes use of of capital; and our expectations relating to the influence of the COVID-19 pandemic on our enterprise. Precise outcomes could differ materially from these indicated by these forward-looking statements on account of varied vital elements, together with the next: our means to finance our drug discovery and improvement efforts and to lift extra capital when wanted; the flexibility of our preclinical packages to supply information adequate to assist our scientific trial purposes and the timing thereof; the scientific outcomes of our packages and the timing thereof, which can not assist additional improvement of product candidates; actions of regulatory companies, which can have an effect on the initiation, timing and progress of scientific trials, together with their receptiveness to our adaptive trial designs; our effectiveness in managing future scientific trials and regulatory interactions; the effectiveness of PRISM, together with our novel PN spine chemistry modifications; the effectiveness of our novel ADAR-mediated RNA modifying platform functionality and our AIMers; the continued improvement and acceptance of oligonucleotides as a category of medicines; our means to reveal the therapeutic advantages of our candidates in scientific trials, together with our means to develop candidates throughout a number of therapeutic modalities; our dependence on third events, together with contract analysis organizations, contract manufacturing organizations, collaborators and companions; our means to fabricate or contract with third events to fabricate drug materials to assist our packages and progress; our means to acquire, keep and defend our mental property; our means to implement our patents towards infringers and defend our patent portfolio towards challenges from third events; competitors from others creating therapies for comparable indications; our means to take care of the corporate infrastructure and personnel wanted to realize our objectives; the severity and length of the COVID-19 pandemic and variants thereof, and its destructive influence on the conduct of, and the timing of enrollment, completion and reporting with respect to our scientific trials; and another impacts on our enterprise on account of or associated to the COVID-19 pandemic, in addition to the knowledge beneath the caption “Danger Elements” contained in our most up-to-date Annual Report on Kind 10-Ok filed with the Securities and Trade Fee (SEC) and in different filings we make with the SEC every now and then. We undertake no obligation to replace the knowledge contained on this press launch to replicate subsequently occurring occasions or circumstances.

WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED BALANCE SHEETS

(In 1000’s, besides share quantities)

December 31, 2021

December 31, 2020

Property

Present belongings:

Money and money equivalents

$

150,564

$

184,497

Present portion of accounts receivable

30,000

Pay as you go bills

6,584

10,434

Different present belongings

5,416

5,111

Whole present belongings

162,564

230,042

Lengthy-term belongings:

Property and gear, web

22,266

29,198

Working lease right-of-use belongings

18,378

16,232

Restricted money

3,651

3,651

Different belongings

148

115

Whole long-term belongings

44,443

49,196

Whole belongings

$

207,007

$

279,238

Liabilities, Sequence A most well-liked shares and shareholders’ fairness

Present liabilities:

Accounts payable

$

7,281

$

13,795

Accrued bills and different present liabilities

14,861

11,971

Present portion of deferred income

37,098

91,560

Present portion of working lease legal responsibility

4,961

3,714

Whole present liabilities

64,201

121,040

Lengthy-term liabilities:

Deferred income, web of present portion

77,479

41,481

Working lease legal responsibility, web of present portion

24,955

25,591

Different liabilities

474

Whole long-term liabilities

102,434

67,546

Whole liabilities

$

166,635

$

188,586

Sequence A most well-liked shares, no par worth; 3,901,348 shares issued
and excellent at December 31, 2021 and 2020

$

7,874

$

7,874

Shareholders’ fairness:

Abnormal shares, no par worth; 59,841,116 and 48,778,678 shares issued
and excellent at December 31, 2021 and 2020, respectively

749,851

694,085

Extra paid-in capital

87,980

71,573

Amassed different complete earnings

181

389

Amassed deficit

(805,514

)

(683,269

)

Whole shareholders’ fairness

32,498

82,778

Whole liabilities, Sequence A most well-liked shares and shareholders’ fairness

$

207,007

$

279,238

WAVE LIFE SCIENCES LTD.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(In 1000’s, besides share and per share quantities)

Three Months Ended December 31,

Twelve Months Ended December 31,

2021

2020

2021

2020

Income

$

1,765

$

9,439

$

40,964

$

20,077

Working bills:

Analysis and improvement

25,761

30,033

121,875

130,944

Normal and administrative

12,114

9,719

46,105

42,510

Whole working bills

37,875

39,752

167,980

173,454

Loss from operations

(36,110

)

(30,313

)

(127,016

)

(153,377

)

Different earnings, web:

Dividend earnings and curiosity earnings, web

5

24

30

568

Different earnings, web

1,116

659

4,537

2,058

Whole different earnings, web

1,121

683

4,567

2,626

Loss earlier than earnings taxes

(34,989

)

(29,630

)

(122,449

)

(150,751

)

Earnings tax profit

204

841

204

841

Web loss

$

(34,785

)

$

(28,789

)

$

(122,245

)

$

(149,910

)

Web loss per share attributable to extraordinary
shareholders—fundamental and diluted

$

(0.61

)

$

(0.59

)

$

(2.36

)

$

(3.82

)

Weighted-average extraordinary shares utilized in computing
web loss per share attributable to extraordinary
shareholders—fundamental and diluted

57,190,742

48,777,001

51,825,566

39,227,618

Different complete earnings (loss):

Web loss

$

(34,785

)

$

(28,789

)

$

(122,245

)

$

(149,910

)

International forex translation

(77

)

88

(208

)

122

Complete loss

$

(34,862

)

$

(28,701

)

$

(122,453

)

$

(149,788

)

Investor Contact:
Kate Rausch
617-949-4827
[email protected]

Media Contact:
Alicia Suter
617-949-4817
[email protected]

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