Orphazyme reported results from the phase 2 ORARIGAU-01 trial, evaluating arimoclomol in Gaucher disease (GD) type 1 and 3 patients naive to enzyme and/or substrate replacement therapy. In the trial Arimoclomol demonstrated a marked and clinically meaningful...
Metabolic Disease
Category
AHA-Backed Study Reveals Ongoing Lack of Minority & Female Participation in Cardiometabolic Clinical Trials
Unfortunately, yet another report reveals the dearth of women and minorities, especially African Americans’ representation in clinical trials in the critically important cardiovascular and metabolic therapeutic domain. The results of a recent study published in the...
FDA Places Clinical Hold on LogicBio’s Gene Therapy Treatment
LogicBio Therapeutics announced that the U.S. FDA has placed a clinical hold on the Investigational New Drug (IND) submission for LB-001 for the treatment of methylmalonic acidemia (MMA) pending the resolution of certain clinical and nonclinical questions. LogicBio...
Sanofi Reports Positive Results from Phase 3 Trials of Olipudase Alfa for Nieman-Pick Disease
Sanofi reported positive results from two phase 3 trials of olipudase alfa, an investigational recombinant human acid sphingomyelinase, for the treatment of acid sphingomyelinase deficiency (ASMD), also known as Nieman-Pick disease, in adult and pediatric patients....
Akcea and Ionis Report Positive Phase 2 Data for AKCEA-ANGPTL3-LRx; Asset Transferred to Pfizer
Akcea Therapeutics along with its parent company, Ionis Pharmaceuticals, announced positive topline results from the Phase 2 study of AKCEA-ANGPTL3-LRx in patients with hypertriglyceridemia, type 2 diabetes and non-alcoholic fatty liver disease (NAFLD). The study met...
Acasti Pharma Reports Topline Results for Phase 3 TRILOGY 1 Trial of CaPre for Severe Hypertriglyceridemia
Acasti Pharma announced topline results from the phase 3 trial, TRILOGY 1, which was designed to determine the efficacy of CaPre compared to placebo in lowering fasting triglyceride (TG) levels in patients with severe hypertriglyceridemia. Despite positive results in...
Allena Pharma Reports Data from Phase 3 URIROX-1 Trial of Reloxaliase for Enteric Hyperoxaluria
Allena Pharmaceuticals announced positive topline results from URIROX-1, a Phase 3 pivotal trial evaluating reloxaliase in patients with enteric hyperoxaluria (EH). The primary endpoint was achieved, showing a statistically significant reduction in urinary oxalate in...
Clinuvel Receives FDA Approval for Scenesse for Use in Patients with the Rare Disorder Erythropoietic Protoporphyria
Clinuvel announced the U.S FDA has approved Scenesse (afamelanotide) to increase pain-free light exposure in adult patients with a history of phototoxic reactions (damage to skin) from erythropoietic protoporphyria (EPP). With this approval, Scenesse is the first...
Moderna Therapeutics Receives IND Approval for mRNA-3927, its Investigational mRNA Therapeutic for Propionic Acidemia
Moderna Therapeutics announced the U.S. Food and Drug Administration (FDA) has completed its review of the Investigational New Drug (IND) application for mRNA-3927, its investigational mRNA therapeutic for propionic acidemia (PA), a rare metabolic disease. With the...
North Carolina Clinical Investigator Site Joins ProScientos NASH PASS Initiative
High Point Clinical Trials Center LLC, the largest independent clinical research unit in North Carolina, announced its participation in the NASH PASS network. NASH PASS is organized and managed by metabolic disease specialist CRO ProSciento. Who is High Point Clinical...
High Point Clinical Trials Center Participates in NASH Pass Initiative Focusing on Accelerating Clinical Trial Recruitment
High Point Clinical Trials Center, LLC (HPCTC), the largest independent clinical research site in North Carolina, reports it has partnered with ProSciento, a specialized CRO exclusively focused on metabolic diseases, to join as a NASH PASS site partner. ProSciento has...
Australian Researchers Identify Connection Between Hidradenitis Suppurativa & Metabolic Syndrome
A large group of prominent Australian researchers conducted a comprehensive meta-analysis study of Hidradenitis Suppurativa and its potential association with Metabolic Syndrome. TrialSite News has a significant reader community interested in Hidradenitis Suppurativa....
University of British Columbia Study to Identify Cause of Rare Genetic Metabolic Disorder
The University of British Columbia (UBC), BC Children’s Hospital, and an international team of investigators published in the New England Journal of Medicine a rarely-seen type of DNA mutation as the potential cause of an inherited metabolic disorder. Often the result...
Pfizer Invests $51 Million to Access Gene Therapy Platform for Liver Disorders
FINSMES reports Pfizer has invested $51 million for a 15% stake in Vivet Therapeutics, a France-based gene therapy venture dedicated to developing inherited liver disorder treatments. Pfizer secured an exclusive option to buy all outstanding shares. Pfizer’s Monika...
Virginia Commonwealth University Study Highlights Liver & Heart Interaction
Anne Dreyfuss writing for Virginia Commonwealth University (VCU) reports that its researchers have collaborated on a clinical trial that identifies indicators for nonalcoholic fatty liver disease—a typically asymptomatic disease caused by fat buildup in the liver and...
Children’s Hospital LA Investigator Secures $1.3 Million for Stem Cell Therapy for Liver Failure
As reported in EurekAlert!, Children’s Hospital Los Angeles Investigator Tracy Grikscheit MD has received $1.3 million to study the use of pluripotent stem cells on liver-based metabolic disease. The grant was awarded by the California Institute of Regenerative...