BMN 351 / BioMarin - LARVOL DELTA

Phase 1/2 Data Show Positive Dystrophin Expression and Favorable Tolerability With Nivudirsen (BMN 351) (PRNewswire) - "Additional presentations at WMS included data evaluating nivudirsen (BMN 351), an investigational antisense oligonucleotide for ambulatory boys ages 4 to 10 with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Included in these presentations was an update from the ongoing Phase 1/2 351-201 study, which was previously shared at the 19th International Congress on Neuromuscular Diseases in July. Results from 351-201 showed treatment with nivudirsen was generally well tolerated and led to consistently positive outcomes across multiple measures of therapeutic benefit, including expression of near full-length dystrophin."

P1/2 data • Duchenne Muscular Dystrophy

Phase 1/2 Results of an Antisense Oligonucleotide Therapy for Exon‑Skipping‑Amenable DMD (ICNMD 2026) - "Background: Nivudirsen (BMN 351) is an investigational phosphorothioate (PS) antisense oligonucleotide (ASO) that has been chemically modified to improve specificity and stability... Interim analyses up to 25 weeks demonstrated acceptable safety and positive dystrophin changes in boys with DMD receiving nivudirsen ≤9mg/kg. Enrollment of cohort 3 at the 12mg/kg nivudirsen dose is ongoing. Pharmacokinetic and pharmacodynamic modeling of PS-based ASOs predicts that dystrophin will continue to accumulate over time and reach steady-state levels that are approximately 2-fold higher than W25."

First-in-human • P1/2 data • Duchenne Muscular Dystrophy • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Infectious Disease • Influenza • Muscular Dystrophy • Respiratory Diseases • CST3

A Transcriptomics-Based Biomarker for Muscle Quality Measures BMN 351 Treatment Response in DMD Patient Biopsies (ICNMD 2026) - P1/2 | "The observed shift in transcriptome expression of muscle maturation and disease markers toward levels observed in healthy controls suggests that treatment with BMN 351 may be improving muscle quality. We anticipate that transcriptomics-based measures of treatment response will compliment dystrophin expression and functional assessments in future BMN 351 clinical trials."

Biomarker • Biopsy • Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • HSP90AA1

Orthogonal Methods Demonstrate Pharmacodynamic Responses in Duchenne Muscular Dystrophy Patients Treated with BMN 351 (ICNMD 2026) - P1/2 | "Background: BMN 351 is an antisense oligonucleotide therapeutic designed to exclude DMD exon 51 during mRNA splicing in muscle and induce synthesis of functional, near full-length dystrophin protein in amenable patients with Duchenne muscular dystrophy (DMD). Together, the data from these orthogonal methods provide a robust data set demonstrating early patient responses and support the potential for clinical benefit in patients treated with BMN 351."

Clinical • PK/PD data • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1/2 | N=18 | Active, not recruiting | Sponsor: BioMarin Pharmaceutical | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2026 ➔ Apr 2027 | Trial primary completion date: Sep 2026 ➔ Apr 2027

Enrollment closed • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=18 | Enrolling by invitation | Sponsor: BioMarin Pharmaceutical

New P2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

351-202: An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants with Duchenne Muscular Dystrophy (clinicaltrialsregister.eu) - P1/2 | N=3 | Not yet recruiting | Sponsor: Biomarin Pharmaceutical Inc.

New P1/2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: BioMarin Pharmaceutical | Trial completion date: Dec 2025 ➔ Sep 2026 | Trial primary completion date: Dec 2025 ➔ Sep 2026

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy. (PubMed, Nucleic Acid Ther) - "BMN 351 also improved gait scores and clinical and anatomical muscle pathology parameters compared with vehicle-treated hDMDdel52/mdx mice. The pharmacologic activity and safety of BMN 351 warrant further nonclinical and clinical development."

Journal • Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • HSP90AA1

BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal and Cardiac Muscle Lead to Preservation of Motor Function in a Mouse Model of Exon 51 Skip-Amenable Duchenne Muscular Dystrophy. (PubMed, Nucleic Acid Ther) - "Liver samples demonstrated findings consistent with ASO accumulation, to which mice are considered especially sensitive compared to humans and other non-clinical species. These results support further non-clinical and clinical development of BMN 351."

Journal • Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • HSP90AA1

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: BioMarin Pharmaceutical

New P1/2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

The antisense oligonucleotide BMN 351 durably ameliorates dystrophic phenotypes in a mouse model of exon 51–skip-amenable Duchenne muscular dystrophy (WMS 2023) - No abstract available

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Form 8-K BIOMARIN PHARMACEUTICAL For: Feb 25 (Streetinsider.com) - "BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies are underway with BMN 351, an oligonucleotide therapy that has demonstrated a high-level of protein expression in pre-clinical DMD models. The Company intends to determine timing of a potential IND filing at the end of the year based on results of ongoing IND-enabling studies."

Preclinical • Duchenne Muscular Dystrophy