Business
Wave Life Sciences Reports First Quarter 2022 Financial Results and Provides Business Update
Delivered first clinical data demonstrating target engagement and translation of PN chemistry’s impact in clinic; Adapting ongoing Phase 1b/2a FOCUS-C9

About this update from Wave Life Sciences Ltd.
[{"type":"text","content":"Delivered first clinical data demonstrating target engagement and translation of PN chemistry’s impact in clinic; Adapting ongoing Phase 1b/2a FOCUS-C9 clinical trial to optimize dose level and frequency, with additional single and multidose data expected throughout 2022 Clinical data also expected in 2022 from Huntington’s disease (WVE-003) and Duchenne muscular dystrophy (WVE-N531) trials Robust preclinical datasets for first-in-class AATD program demonstrate restoration of levels of AAT relevant for potential lung protection and reduction of liver-damaging aggregates with GalNAc AIMers; IND enabling toxicology studies for lead AATD candidate on-track to initiate in 3Q 2022 Wave to host investor conference call and webcast at 8:30 a.m. ET today CAMBRIDGE, Mass., May 12, 2022 (GLOBE NEWSWIRE) -- 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 first quarter ended March 31, 2022 and provided a business update. “Thus far in 2022, Wave has achieved several important milestones, with the highlight being our first clinical data demonstrating successful target engagement with WVE-004 in the ongoing FOCUS-C9 clinical trial for C9-ALS and C9-FTD. We observed potent and durable reductions of the poly(GP) biomarker with low single doses of WVE-004, demonstrating that our preclinical data for PN-containing oligonucleotides are beginning to translate in the clinic,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “These initial results are compelling and reinforce the potential of our unique oligonucleotide platform and our expectation to see the advantages of PN chemistry manifest in our other pipeline programs. We also continue to rapidly advance our WVE-003 program for HD and WVE-N531 program for DMD towards initial data updates later this year. We are also pleased with the recognition we are receiving with our endogenous RNA editing modality, which is being highlighted through scientific presentations and our recent Nature Biotechnology publication. Alpha-1 antitrypsin deficiency (AATD) is uniquely suited for an RNA editing therapeutic, and our AATD program is rapidly advancing towards clinical development with IND enabling studies on...