edasalonexent (CAT-1004) / Astria Therap - LARVOL DELTA

Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review and evidence synthesis. (PubMed, PLoS One) - "This study provides a current summary of prognostic indicators of disease progression in DMD, which will help inform the design of comparative analyses and future data collection initiatives in this patient population."

Biomarker • Journal • Review • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Assessing the ability of boys with Duchenne muscular dystrophy age 4-7 years to swallow softgel capsules: Clinical trial experience with edasalonexent. (PubMed, J Clin Pharm Ther) - "Swallowing of capsules was not a barrier for drug administration in young patients with DMD. Capsule formulations may be an acceptable alternative to liquid formulations for children as young as 4 years of age."

Clinical • Journal • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Infectious Disease • Muscular Dystrophy • Pediatrics

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial. (PubMed, J Neuromuscul Dis) - P3 | "Edasalonexent was generally well tolerated with a manageable safety profile at the dose of 100 mg/kg/day. Although edasalonexent did not achieve statistical significance for improvement in primary and secondary functional endpoints for assessment of DMD, subgroup analysis suggested that edasalonexent may slow disease progression if initiated before 6 years of age. (NCT03703882)."

Clinical • Journal • P3 data • Duchenne Muscular Dystrophy • Fibrosis • Gastrointestinal Disorder • Genetic Disorders • Immunology • Inflammation • Muscular Dystrophy • Pediatrics

Disease-modifying effects of edasalonexent, an NF-κB inhibitor, in young boys with Duchenne muscular dystrophy: Results of the MoveDMD phase 2 and open label extension trial: Running title: Edasalonexent for Duchenne muscular dystrophy. (PubMed, Neuromuscul Disord) - "Edasalonexent 100 mg/kg was associated with slowing of disease progression and preservation of muscle function compared to an off-treatment control period, with decrease in levels of NF-κB-regulated genes and improvements in biomarkers of muscle health and inflammation. These results support investigating edasalonexent in future trials and have informed the design of the edasalonexent phase 3 clinical trial in boys with Duchenne."

Clinical • Journal • P2 data • Duchenne Muscular Dystrophy • Gastrointestinal Disorder • Genetic Disorders • Immunology • Inflammation • Muscular Dystrophy

[VIRTUAL] Results from PolarisDMD, A Phase 3 Trial of Edasalonexent for Duchenne Muscular Dystrophy (MDA 2021) - "The trial did not meet its primary or secondary endpoints; however, there were trends toward improvement, particularly in the younger subgroup. While edasalonexent was generally safe and well-tolerated, the totality of evidence does not support further development of edasalonexent for DMD."

P3 data • Duchenne Muscular Dystrophy • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Muscular Dystrophy • Respiratory Diseases

Study of Edasalonexent With Midazolam and Deflazacort in Healthy Subjects (clinicaltrials.gov) - P1; N=26; Completed; Sponsor: Catabasis Pharmaceuticals; Active, not recruiting ➔ Completed

Clinical • Trial completion

GalaxyDMD: An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P3; N=130; Terminated; Sponsor: Catabasis Pharmaceuticals; Trial completion date: Sep 2022 ➔ Oct 2020; Enrolling by invitation ➔ Terminated; Trial primary completion date: Sep 2022 ➔ Oct 2020; The Phase 3 PolarisDMD trial did not meet the primary endpoint. As a result, activities related to the development of edasalonexent have stopped including the CAT-1004-302 Open-Label Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

Clinical • Trial completion date • Trial primary completion date • Trial termination • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

PolarisDMD: Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P3; N=131; Completed; Sponsor: Catabasis Pharmaceuticals; Active, not recruiting ➔ Completed

Clinical • Trial completion • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

Catabasis Pharmaceuticals Announces Top-Line Results for the Phase 3 PolarisDMD Trial of Edasalonexent in Duchenne Muscular Dystrophy (Businesswire) - P3, N=131; PolarisDMD (NCT03703882); Sponsor: Catabasis Pharmaceuticals; "Catabasis...today announced that the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) did not meet the primary endpoint, which was a change from baseline in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent compared to placebo. The secondary endpoint timed function tests (time to stand, 10-meter walk/run and 4-stair climb) also did not show statistically significant improvements...Data from the PolarisDMD trial will be further analyzed and are expected to be presented at an upcoming scientific conference and published."

P3 data: top line • Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals Presents Information On Edasalonexent, A Potential Foundational Therapy For Duchenne Muscular Dystrophy, At The Virtual 25th International Congress Of The World Muscle Society (Catabasis Press Release) - "The posters include new preclinical research findings supporting potential positive effects on cardiac function and preservation of bone health with edasalonexent in mouse models of DMD...also presented with baseline assessments from the Phase 3 PolarisDMD trial and long-term safety results from the Phase 2 MoveDMD trial and open-label extension...The primary endpoint of the Phase 3 PolarisDMD trial for edasalonexent is the North Star Ambulatory Assessment (NSAA), a validated measure of physical function designed for use in ambulatory boys affected by DMD."

P2 data • P3 data • Preclinical • Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals to Present During the Virtual 25th International Congress of the World Muscle Society (Businesswire) - "Catabasis...today announced that it will present information on the edasalonexent program, in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD), during the 25th International Congress of the World Muscle Society which begins September 28, 2020. During the Virtual Poster Session on October 1, 2020 from 12:30 – 2:30pm ET, Catabasis and collaborators will present five posters..."

Clinical data • P3 data • Preclinical • Duchenne Muscular Dystrophy

[VIRTUAL] In the global phase 3 polarisDMD trial for edasalonexent, standardized outcome measure training produces excellent test-retest variability in NSAA (WMS 2020) - No abstract available

Clinical • P3 data

[VIRTUAL] Edasalonexent treatment in young boys with Duchenne muscular dystrophy is associated with age-normative growth and normal adrenal function (WMS 2020) - No abstract available

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

[VIRTUAL] Edasalonexent maintains bone density and bone strength in the mdx mouse model of Duchenne muscular dystrophy (WMS 2020) - No abstract available

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

[VIRTUAL] Inhibition of NF-kB signaling by edasalonexent prevents the development of DMD-associated cardiomyopathy in mdx:Utrn+/- mice (WMS 2020) - No abstract available

Preclinical • Cardiomyopathy • Cardiovascular

Study of Edasalonexent With Midazolam and Deflazacort in Healthy Subjects (clinicaltrials.gov) - P1; N=26; Active, not recruiting; Sponsor: Catabasis Pharmaceuticals

Clinical • New P1 trial

Catabasis Pharmaceuticals to Host Virtual KOL Event on Edasalonexent and Duchenne Muscular Dystrophy (Businesswire) - "Catabasis...will be holding a virtual key opinion leader (KOL) event to discuss edasalonexent, currently in Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD), on Friday, September 11,.2020 at 9:00am ET."

Live event • Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals Reports Second Quarter 2020 Financial Results and Reviews Business Progress (Businesswire) - "Plans are progressing for the edasalonexent non-ambulatory trial in partnership with Duchenne UK and the trial is expected to initiate in 2021....Research and development expenses were $6.8 million for the three months ended June 30, 2020, compared to $5.2 million for the three months ended June 30, 2019."

Clinical • New trial • Duchenne Muscular Dystrophy

Catabasis Pharmaceuticals to Present at Parent Project Muscular Dystrophy Virtual Annual Conference (Businesswire) - "Catabasis...announced today that it will present information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) at the Parent Project Muscular Dystrophy (PPMD) Virtual Annual Conference...Dr. Donovan will also provide an overview of edasalonexent during the...session on Wednesday, July 22nd at 7:00pm ET during the live stream....Additionally, Catabasis will share three posters during the Virtual Poster Session, which will also be available in the On-Demand Library..."

Clinical • Clinical data • Live event • Duchenne Muscular Dystrophy

PolarisDMD: Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P3; N=131; Active, not recruiting; Sponsor: Catabasis Pharmaceuticals; Trial completion date: Jun 2020 ➔ Sep 2020; Trial primary completion date: Jun 2020 ➔ Sep 2020

Clinical • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Pediatrics

GalaxyDMD: An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P3; N=140; Enrolling by invitation; Sponsor: Catabasis Pharmaceuticals; Trial completion date: Jun 2022 ➔ Sep 2022; Trial primary completion date: Jun 2022 ➔ Sep 2022

Clinical • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Pediatrics

CAT-1004, an Oral Agent Targeting NF-kB: MoveDMD Trial Results in Duchenne Muscular Dystrophy (DMD) (ANA 2016) - "These results support a 12-week, double-blind, placebo-controlled trial in 30 boys aged 4–7 with DMD, with T2 MRI of leg muscles as the primary endpoint and functional measures as secondary endpoints. By reducing inflammation and muscle degeneration with potentially positive longer-term effects on muscle regeneration and function, CAT-1004 may have potential to be disease-modifying in DMD patients, regardless of mutation type."

Retrospective data • Biosimilar • Gene Therapies • Immunology

Catabasis Pharmaceuticals reports second quarter 2018 financial results and reviews business progress (Businesswire) - "Announced plans for edasalonexent Phase 3 PolarisDMD trial in Duchenne muscular dystrophy (DMD), expected to initiate in the second half of 2018 with top-line results expected in the second quarter of 2020. The trial design was informed by discussions with the U.S. Food and Drug Administration (FDA) as well as input from treating physicians and families of boys affected by Duchenne."

P3 data: top line • Trial initiation date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Catabasis Pharmaceuticals reports first quarter 2020 financial results and reviews business progress (Businesswire) - "The fully enrolled Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) is progressing with top-line results expected in Q4 2020....The fully enrolled Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD) is progressing with top-line results expected in Q4 2020."

NDA • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Catabasis Pharmaceuticals presents edasalonexent, a potential foundational therapy for Duchenne muscular dystrophy (Catabasis Press Release) - "Catabasis Pharmaceuticals, Inc....today shared information on the edasalonexent program in Phase 3 development for the treatment of Duchenne muscular dystrophy (DMD) in poster presentations at the 2020 Muscular Dystrophy Association (MDA) Virtual Poster Session...The three posters include an analysis of the baseline characteristics of patients enrolled in the Phase 3 PolarisDMD trial...Top-line results from the Phase 3 trial are expected in Q4 2020."

Clinical • P3 data: top line • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy