Wave Life Sciences Ltd.

Good morning and welcome to the Wave Life Sciences first quarter 2021 financial results conference call. At this time, all participants are in a listen-only mode. As a reminder, this call is being recorded and webcast. I'll now turn the call over to Kate Rausch, Head of Investor Relations at Wave Life Sciences. Please go ahead.

Thank you, Operator. Good morning, and thank you for joining us today to discuss our recent business progress and review Wave's first quarter 2021 operating results. On the call with me today is Dr. Paul Bolno, WAVE's President and Chief Executive 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 first quarter financial results and provided a business update. This news release and a slide presentation to accompany this webcast are available in the investor section of our website, www.wavelifesciences.com. 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 issued today and in our SEC filings, including our annual report on Form 10-K for the year ended December 31, 2020. We undertake no obligation to update or revise any forward-looking statements for any reason. I'd now like to turn the call over to Paul. Paul?

Thanks, Kate. Good morning to everyone on the call, and thank you for joining us. During the call today, I will provide some opening remarks, after which Mike will give an update on our clinical trials, and Kyle will briefly review our financials. It has been an incredibly productive start to the year for WAVE as we advance three-and-a-half-generation stereopure oligonucleotides into clinical development. We have formally initiated clinical trials for WBE004, our C9OR72 candidate in ALS and FTD, and WBE003, our SNP3 candidate in Huntington's disease. We've also received important regulatory approvals towards initiating our third PN chemistry program targeting exon 53 in DMV, WBEN531. These clinical trials are designed to enable rapid proof of concept using biomarker-driven adaptive designs and are the first investigative candidates designed with our novel PN backbone chemistry modification. Next year, we expect that data from these clinical trials will enable decision-making about next steps for these programs, as well as provide insight into PN chemistry across different modalities, tissue types, and targets. We've also made substantial progress with our endogenous ADAR editing capabilities. which we believe is the most advanced in its class. We've generated a breadth of RNA editing data demonstrating activity across in vivo and in vitro systems, including in vivo editing in the central nervous system. Much of this data is being presented in an oral presentation tomorrow, May 14th, at the ASGCT annual meeting. Our first data editing program for alpha-1 antitrust and disease has generated promising initial results, and we are on track to share in vivo data this quarter. Our PRISM platform is unique and differentiated from others developing RNA therapeutics. At our foundation, we set out to embrace rather than ignore the reality and importance of stereochemistry that exists in each and every oligonucleotide. In choosing to control for the three-dimensional orientations of backbone linkages and advance single isomer therapeutics, we can apply the principles of rational drug design to our pipeline candidates, which is impossible with mixture-based oligonucleotides. This resolution enables us to define distinct profiles for our stereocure molecules, and we now have several years of clinical data to further inform our platform. Earlier this year, we announced the discontinuation of our remaining first-generation programs following the results of the PRECISION-HD trials. While we only saw modest and inconsistent reductions of mutant Huntington, it is important to note there were no clinically meaningful trends in disease progression or laboratory values, such as elevations in CSF white blood cells, proteins, and neurofilaments, light chain, or NFL. There were, however, suggestions of allele selectivity, underscoring the precision enabled by our platform. In our next generation programs, We've prioritized the use of in vivo models during preclinical development to ensure we advance clinical candidates that will reach the desired site of action and engage targets. In addition to the wealth of data collected over the past several years, we're also leveraging an influx of new talent in oligonucleotide therapeutics to further advance our understanding of design principles, pharmacology, and toxicology. The application of PN backbone chemistry modifications in the context of controlling stereochemistry was a major advancement that emerged from our platform. And based on what we have seen preclinically, this innovation has the potential to significantly improve the profiles of therapeutic oligonucleotides, independent of sequence, tissue type, or modality. Separately, our ADAR editing capability further expands our toolkit beyond silencing and splicing, enabling us to select the best modality to address the root cause of genetic diseases. We anticipate sharing more on PN chemistry and ADAR editing at a research day later this year. Our current pipeline is comprised of programs designed with next generation of PRISM, including PN chemistry. I am extremely proud of how quickly we have advanced this innovation to the clinic, and we are rapidly approaching the first of many opportunities for clinical proof of concept of PN chemistry. I'd now like to turn the call over to Mike Panzara for an update on our neurology programs. Mike. Thanks, Paul.