INS1201 / Insmed - LARVOL DELTA

ASCEND: A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P1 | N=12 | Active, not recruiting | Sponsor: Insmed Gene Therapy LLC | Recruiting ➔ Active, not recruiting

Enrollment closed • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy. (PubMed, Mol Ther Adv) - "Toxicology evaluations concluded that INS1201 was well tolerated at all tested doses, with the no observed adverse effect level set at the maximum feasible dose administered for both species (up to 8.0E+11 vg/mouse and 3.05E+14 vg/NHP). Collectively, these findings provided support for an INS1201 phase 1 human clinical study."

Journal • Preclinical • Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Inflammation • Muscular Dystrophy

Progress and Anticipated Milestones:…INS1201 (PRNewswire) - "Insmed continues to enroll the Phase 1 ASCEND clinical study of INS1201, an intrathecally delivered gene therapy for patients with Duchenne muscular dystrophy (DMD)."

Enrollment status • Duchenne Muscular Dystrophy

Design of the Phase 1 ASCEND Study to Investigate the Safety and Biodistribution of INS1201 Gene Therapy in Males With Duchenne Muscular Dystrophy (ASGCT 2025) - "The ASCEND study will assess the safety and biodistribution of INS1201 to inform RP2D selection. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Clinical • Gene therapy • P1 data • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

CSF Delivery of INS1201 AAV9-Micro-Dystrophin Demonstrates Long-Term Durability of Efficacy in a Mouse Model of DMD (ASGCT 2025) - "These findings support ongoing studies to evaluate the potential of INS1201 as a therapeutic option for DMD. Disease Focus of Abstract:Muscular Dystrophy (all forms)"

Preclinical • Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Immunology • EP400

Recent Progress and Anticipated Milestones by Program (PRNewswire) - "Insmed completed the Phase 2b study of treprostinil palmitil inhalation powder (TPIP) in pulmonary arterial hypertension (PAH), with topline data anticipated in June of 2025; The Company plans to initiate a Phase 3 study of TPIP in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) in the second half of 2025...Insmed initiated the Phase 1 ASCEND clinical study of INS1201, an intrathecally-delivered gene therapy for patients with Duchenne muscular dystrophy (DMD), in the first quarter of 2025, and expects to dose the first patient in the second quarter of 2025; The Company's next two gene therapy candidates, which target amyotrophic lateral sclerosis (ALS) and Stargardt disease, are currently advancing toward the clinic."

New P3 trial • P2b data • Pipeline update • Trial status • Amyotrophic Lateral Sclerosis • Duchenne Muscular Dystrophy • Genetic Disorders • Interstitial Lung Disease • Ophthalmology • Pulmonary Arterial Hypertension

ASCEND: A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Insmed Gene Therapy LLC | Not yet recruiting ➔ Recruiting | Initiation date: Feb 2025 ➔ May 2025

Enrollment open • Trial initiation date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Insmed Reports Fourth-Quarter and Full-Year 2024 Financial Results and Provides Business Update (PRNewswire) - "Insmed plans to initiate a clinical study of INS1201 in the first half of 2025."

P1 data • Duchenne Muscular Dystrophy