Wave Life Sciences Ltd.

Good morning and welcome to the Wave Life Sciences third quarter 2021 financial results conference call. At this time, our participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you need to press star 1 on your telephone. As a reminder, this call is being recorded in webcast. I'll now turn the call over to Kate Rausch, head of investor relations at Wave Life Sciences. Please go ahead.

Thank you, Misty. Good morning, and thank you for joining us today to discuss our recent business progress and review Wade's third quarter 2021 operating results. Joining me in the room today is prepared remarks for Dr. Paul Bono, Wade's President and Chief Executive Officer, Dr. Chandra Varghese, Chief Technology Officer, Dr. Mike Panzera, Chief Medical Officer, Head of Therapeutics Discovery and Development, and Kyle Moran, Chief Financial Officer. This morning, we issued a news release detailing our third quarter financial results and provided a business update. This news release and a slide presentation to accompany this webcast will be available in the Investor section of our website, www.wavelifesciences.com, following the call. Before we begin, I would like to remind you that discussions during this conference call will include forward-looking statements. These statements are subject to a number of risks and uncertainties that could cause our actual results to differ materially from those described in these forward-looking statements. The factors that could cause actual results to differ are discussed in the press release issue today and in our SEC filing, including our annual report on Form 10-K for the year ended December 31, 2020, and our quarterly report on Form 10-Q for the quarter ended September 30, 2021. We undertake no obligation to update or revise any forward-looking statement for any reason. I now like to turn the call over to Paul. Paul?

Thanks, Kate. Good morning, and thank you for joining us. Today, I will start with opening remarks after which Chandra will walk through how we are building a pipeline of RNA editing therapeutics with Amherst. Mike will then provide an update on our therapeutic program and turn it to Kyle to discuss our financials. During the third quarter, we achieved several important milestones and made progress advancing our therapeutic pipeline, bringing us closer to our goal of delivering life-changing treatments for people battling devastating diseases. Most recently, we held our annual analyst and investor research webcast on September 28th, during which we formally introduced our aimers for RNA editing oligonucleotides and shared the most mature in vivo RNA editing data set generated to date. This includes an update on our alpha one antitrypsin deficiency or AATP program and use of aimers to restore functional AAT protein well above the therapeutic threshold. In parallel, We share these data in multiple posters and presentations at the 2021 OTS and TIDES annual meetings. Following these exciting and promising updates, we raised approximately $30 million in proceeds from an aggregate block sale of ordinary shares through our ATM equity program, with participation based on interest received from both new and existing investors. Coupled with the cash received from Cicada under the terms of our CNS collaboration amendment announced last month, We strengthened our balance sheet with approximately $52 million in October, putting us in a position to accelerate the momentum of our emerging AMER pipeline, leading with hepatic indications. We continue to execute on advancing our clinical therapeutic pipeline and initiated dosing in three clinical trials in the third quarter. Focus C9 evaluating WVE-004 in ALS and STD, Select HD evaluating WVE-003 in HD, and a clinical trial evaluating WVE-N531 and exon 53 amenable CMD. Each of these innovative, adaptive clinical trials is designed to accelerate time to proof of concept. We expect clinical data being generated through 2022 in these trials to enable decision-making on next steps for each of these programs, as well as to help define our future portfolio and platform investments. Our ongoing clinical and emerging clinical programs include silencing modalities in CNS, splicing in muscle, and RNA editing in liver. As we continue to advance these programs, clinical data will enable us to further unlock value through additional targets within these tissue types using these three modalities. As you can see on the right-hand side of the slide, we believe ADAR editing has the potential to represent a substantial portion of our portfolio over time. RNA editing is a novel therapeutic modality, setting up an opportunity to deliver first-in-class innovative AMER therapeutics. Our initial focus is on using AMERS to correct driver mutations and restore protein expression or correct protein function, such as with AATD or Rett syndrome. AMERS can also be used to modulate protein function, including disrupting protein-protein interactions and modifying post-translational modifications for treatment of haploid disease. loss of function disorders, to name some examples. During our research webcast, we shared an in vitro data exemplifying how AMERS can modulate protein-protein interactions using the KEEP1 NRF2 system. We believe clinical proof of principle with our AETD program also serves to de-risk these applications, which represent large patient populations. We've deliberately designed a portfolio that is diversified to reflect the breadth of our platform with differentiated candidates that address diseases of high unmet need. This robust portfolio is led by our clinical programs, OO4 in ALS and FDD, OO3 in HD, and 531 in DMD. These ongoing trials all include biomarker assessments and clinical data, which will enable potential paths to registration and unlock value for additional wholly-owned pipeline programs. As a reminder, Takeda has a 50-50 option to WVE-004, WVE-003, and ATAXA-3. I'd now like to turn the call over to Chandra Varghese to discuss our AMERS. Chandra.