Dyscorban (ifetroban) / Cumberland - LARVOL DELTA

Cumberland Pharmaceuticals Closes Strategic Transaction with Apotex (PRNewswire) - "Under the terms of the agreement, Apotex has acquired Cumberland's line of branded pharmaceuticals for cash consideration of $100 million funded at closing, which followed approval by Cumberland's shareholders...Following the closing, Cumberland will focus its resources on developing ifetroban, a potent thromboxane antagonist currently being studied across clinical programs targeting serious rare and progressive diseases."

Licensing / partnership • Cardiomyopathy • Duchenne Muscular Dystrophy • Idiopathic Pulmonary Fibrosis • Systemic Sclerosis

Cumberland Pharmaceuticals…shared updated results from its Phase 2 FIGHT DMD trial evaluating ifetroban, a novel oral therapy for Duchenne muscular dystrophy (DMD) heart disease, at the annual Parent Project Muscular Dystrophy (PPMD) conference (PRNewswire) - "Through 36 months of treatment across both the Phase 2 trial and the open-label extension, ifetroban maintained a favorable safety profile in DMD patients with no treatment-related serious adverse events or new safety concerns. Evaluation of novel blood biomarkers identified a 30% reduction in MYL3 and a 50% reduction in MYOD1 (circulating markers of heart muscle and cell damage), alongside a 2.4-fold increase in FGF16 and a 2.1-fold increase in TSPAN7 (cardioprotective and tissue repair proteins) with ifetroban treatment compared with placebo."

P2 data • Duchenne Muscular Dystrophy

CPI-IFE-007: Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=46 | Completed | Sponsor: Cumberland Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Cumberland Pharmaceuticals Receives FDA Fast Track Designation for its Ifetroban Duchenne Muscular Dystrophy Program (Yahoo Finance) - "Cumberland previously announced positive results from its Phase 2 FIGHT DMD trial evaluating oral thromboxane receptor antagonist ifetroban in DMD heart disease, demonstrating a 5.4% improvement in left ventricular ejection fraction (LVEF) over 12 months of treatment."

Fast track • Cardiovascular • Duchenne Muscular Dystrophy

Early Cardiac Dysfunction in Duchenne Muscular Dystrophy: A Case Report and Literature Update. (PubMed, Int J Mol Sci) - "This case report presents a rare instance of early-onset cardiac involvement in a 3-year-old male with a confirmed deletion in exon 55 of the dystrophin gene...Genetic mechanisms and genotype-phenotype correlations related to cardiac involvement were reviewed, highlighting emerging therapies such as exon skipping, vamorolone, ifetroban, and rimeporide...While current DMD care standards improve survival, optimizing management through early intervention and novel therapies remains essential. Further research is needed to better understand genotype-phenotype correlations and improve cardiac outcomes for patients with DMD."

Journal • Review • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Heart Failure • Muscular Dystrophy

CPI-IFE-007: Oral Ifetroban in Subjects with Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=48 | Active, not recruiting | Sponsor: Cumberland Pharmaceuticals | Trial completion date: Jan 2025 ➔ Dec 2025 | Trial primary completion date: Jan 2025 ➔ Sep 2025

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

CPI-IFE-007: Oral Ifetroban in Subjects with Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=48 | Active, not recruiting | Sponsor: Cumberland Pharmaceuticals | Recruiting ➔ Active, not recruiting

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

CPI-IFE-007: Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=48 | Recruiting | Sponsor: Cumberland Pharmaceuticals | Trial completion date: Aug 2024 ➔ Dec 2024 | Trial primary completion date: Aug 2024 ➔ Dec 2024

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

Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2 | N=48 | Recruiting | Sponsor: Cumberland Pharmaceuticals | Trial completion date: Jul 2023 ➔ Aug 2024 | Trial primary completion date: Jul 2023 ➔ Aug 2024

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

Ifetroban Reduces Coronary Artery Dysfunction in a Mouse Model of Duchenne Muscular Dystrophy. (PubMed, Am J Physiol Heart Circ Physiol) - P2 | "Overall, our data demonstrate enhanced coronary arterial vasoconstriction to TPr activation in young mdx mice, a phenotype that could be reversed with ifetroban. These data could have important therapeutic implications for improving cardiovascular function in DMD."

Journal • Preclinical • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy