suvodirsen (WVE-210201) / Wave Life Sciences - LARVOL DELTA

Advances in Dystrophinopathy Diagnosis and Therapy. (PubMed, Biomolecules) - "Pharmacological therapy for dystrophinopathies comprises glucocorticoids (prednisone, prednisolone, and deflazacort), vamorolone, and ataluren...Eteplirsen, an antisense-oligonucleotide drug for skipping exon 51 from the Dystrophin gene, is available on the market, which may help up to 14% of Duchenne muscular dystrophy patients. There are various FDA-approved exon skipping drugs including ExonDys-51 for exon 51, VyonDys-53 and Viltolarsen for exon 53 and AmonDys-45 for exon 45 skipping. Other antisense oligonucleotide drugs in the pipeline include casimersen for exon 45, suvodirsen for exon 51, and golodirsen for exon 53 skipping. Advances in the diagnosis and therapy of dystrophinopathies offer new perspectives for their early discovery and care."

Journal • Review • Becker Muscular Dystrophy • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Osteoporosis • Rheumatology

Wave Life Sciences announces positive phase 1 results for WVE-210201 in Duchenne muscular dystrophy (DMD) (GlobeNewswire) - “Wave Life Sciences…announced that the safety and tolerability data from the WVE-210201 Phase 1 clinical trial in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping support initiation of a Phase 2/3 clinical trial....Wave has selected a dose for its planned Phase 2/3 clinical trial of WVE-210201, which it intends to initiate in 2019....Wave plans to present the results from the WVE-210201 Phase 1 clinical trial, as well as details of the Phase 2/3 study design, at upcoming scientific meetings.”

Clinical protocol • New P2/3 trial • P1 data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Wave Life Sciences reports fourth quarter and full year 2019 financial results and provides business update (GlobeNewswire) - "...Wave announced the discontinuation of suvodirsen development for patients with Duchenne muscular dystrophy (DMD). Wave plans to present additional findings from the Phase 1 open-label extension study of suvodirsen at the 2020 MDA Clinical and Scientific Conference in Orlando, Florida...Wave reported a net loss of $56.8 million in the fourth quarter of 2019...reported a net loss of $193.6 million for the year ended December 31, 2019...including manufacturing, in preparation for the potential commercialization of suvodirsen."

Commercial • Discontinued • P1 data

DYSTANCE 51: Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2/3; N=6; Terminated; Sponsor: Wave Life Sciences Ltd.; Suspended ➔ Terminated; Lack of efficacy

Clinical • Trial termination

DYSTANCE 51: Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2/3; N=6; Suspended; Sponsor: Wave Life Sciences Ltd.; N=150 ➔ 6; Trial completion date: Dec 2022 ➔ Mar 2020; Recruiting ➔ Suspended; Trial primary completion date: Dec 2021 ➔ Dec 2019

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Trial suspension

Wave Life Sciences Reports Third Quarter 2019 Financial Results and Provides Business Update (GlobeNewswire, Wave Life Sciences) - "Fast Track designation for suvodirsen received from the U.S. FDA. Interim analysis of dystrophin expression from suvodirsen open-label extension study expected in 4Q 2019."

Clinical data • Commercial • Preclinical • Regulatory

DYSTANCE 51: Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2/3; N=150; Recruiting; Sponsor: Wave Life Sciences Ltd.; Not yet recruiting ➔ Recruiting; Trial completion date: Aug 2021 ➔ Dec 2022; Trial primary completion date: Aug 2021 ➔ Dec 2021

Clinical • Enrollment open • Trial completion date • Trial primary completion date

Wave Life Sciences announces Fast Track designation from U.S. FDA for suvodirsen (GlobeNewswire) - "Wave Life Sciences Ltd....today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to suvodirsen for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping....Wave remains on track to deliver an interim analysis of dystrophin expression from muscle biopsies in boys receiving suvodirsen in the OLE study in the fourth quarter of 2019. Pending positive clinical dystrophin expression data, the company expects to file for an accelerated approval of suvodirsen in the U.S. in the second half of 2020."

BLA • Clinical data • Fast track designation

Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy (clinicaltrialsregister.eu) - P2/3; N=150; Sponsor: Wave Life Sciences UK Limited

Clinical • New P2/3 trial

Wave Life Sciences Reports First Quarter 2019 Financial Results and Provides Business Update (GlobeNewswire, Wave Life Sciences) - “Wave’s PRECISION-HD program, which consists of two global Phase 1b/2a clinical trials evaluating investigational therapies WVE-120101 and WVE-120102 for patients with Huntington's disease (HD), is continuing to enroll patients globally….These results are expected to include a summary of clinical safety results, the degree of mutant huntingtin protein lowering in cerebrospinal fluid (CSF) and the ratio of total huntingtin versus mutant huntingtin protein in CSF to assess wild-type huntingtin protein.”

Clinical protocol • Enrollment status

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1; N=36; Completed; Sponsor: Wave Life Sciences Ltd.; Active, not recruiting ➔ Completed

Clinical • Trial completion

DYSTANCE 51: A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 (Suvodirsen) in Ambulatory Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2/3; N=150; Not yet recruiting; Sponsor: Wave Life Sciences Ltd.

Clinical • New P2/3 trial

"$WVE interim analysis of dystrophin expression from WVE-210201 study in DMD in 2H'19. $SRPT" (@BioStocks)

Biomarker

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1; N=40; Active, not recruiting; Sponsor: Wave Life Sciences Ltd.; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed