DYN 101 / Ionis, Dynacure - LARVOL DELTA

Clinical, histopathological, and biomarker characterization of XLMTM and ADCNM: Operational lessons, screening and baseline data of the Unite-CNM study. (PubMed, J Neuromuscul Dis) - P1/2 | "We present baseline clinical, functional, and biomarker findings from the terminated Unite-CNM trial (NCT04033159), a basket study of the antisense oligonucleotide DYN101, designed to modulate DNM2 expression in adolescents and adults with XLMTM or ADCNM.MethodsScreening and baseline evaluations included medical history, patient-reported outcomes, quantitative muscle strength and motor function, respiratory testing, muscle ultrasound and histology, and biomarker analyses (DNM2 protein, creatinine, creatine kinase, cystatin C, myostatin, and microRNAs) in plasma and muscle.ResultsTwenty-six patients were screened (15 DNM2, 11 MTM1); 14 entered the study...Muscle pathology was comparable across groups. Biomarker analyses showed altered DNM2 protein, reduced myostatin, and specific abnormalities in creatinine, creatine kinase, cystatin C, and microRNAs.ConclusionThis dataset provides valuable phenotypic, functional, and biomarker characterization of adult XLMTM and..."

Biomarker • Journal • CNS Disorders • Infectious Disease • Myositis • Novel Coronavirus Disease • CST3

DyNaMic: Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies (clinicaltrials.gov) - P1/2 | N=0 | Withdrawn | Sponsor: Dynacure | Trial completion date: Jun 2023 ➔ Nov 2025 | Not yet recruiting ➔ Withdrawn | Trial primary completion date: Mar 2023 ➔ Sep 2025

Trial completion date • Trial primary completion date • Trial withdrawal • Myositis

Unite-CNM: Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (clinicaltrials.gov) - P1/2 | N=16 | Terminated | Sponsor: Dynacure | Trial completion date: Dec 2022 ➔ Jun 2022 | Recruiting ➔ Terminated | Trial primary completion date: Dec 2022 ➔ Jun 2022; Based on tolerability findings at the low dose level thus far, continuation of dosing or even dose escalation is not possible.

Trial completion date • Trial primary completion date • Trial termination • Myositis

Unite-CNM: Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (clinicaltrials.gov) - P1/2; N=18; Recruiting; Sponsor: Dynacure; Trial completion date: Apr 2022 ➔ Dec 2022; Trial primary completion date: Apr 2022 ➔ Dec 2022

Clinical • Trial completion date • Trial primary completion date • Myositis

DyNaMic: Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies (clinicaltrials.gov) - P1/2; N=9; Not yet recruiting; Sponsor: Dynacure

Clinical • New P1/2 trial • Myositis

Unite-CNM: Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (clinicaltrials.gov) - P1/2; N=18; Recruiting; Sponsor: Dynacure SAS; Trial completion date: Apr 2021 ➔ Apr 2022; Trial primary completion date: Apr 2021 ➔ Apr 2022

Clinical • Trial completion date • Trial primary completion date

Unite-CNM: Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (clinicaltrials.gov) - P1/2; N=18; Recruiting; Sponsor: Dynacure SAS; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

Unite-CNM: Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (clinicaltrials.gov) - P1/2; N=18; Not yet recruiting; Sponsor: Dynacure SAS

Clinical • New P1/2 trial

An early phase human drug trial (called phase 1/2) to investigate DYN101 in patients ≥ 16 years of age with a rare muscle disease group called the centronuclear myopathies caused by genetic changes in DNM2 or MTM1 for the drug’s safety, tolerability, how the body processes the drug as well as interactions of the drug with the body and preliminary effects of the drug. (clinicaltrialsregister.eu) - P1/2; N=18; Sponsor: Dynacure SAS

Clinical • New P1/2 trial