LM-014 for an Inborn Error of Metabolism
Disease and Prevalence
The targeted inborn error of metabolism is ultra-rare (~1-2 per 100,000 newborns). The majority of patients present with clinical signs and symptoms within the first few days or weeks of birth and overall prognosis is poor. Those patients advancing into childhood or later may develop liver and/or kidney failure. Complications may present in other organ systems as well.
Pathogenesis and LM-014 Mechanism of Action
The defective protein is well-characterized and the gene mutation leading to the disorder is inherited in an autosomal recessive manner. LM-014 is a recombinant protein with targeting sequences facilitating uptake in critical compartments.
Current Treatment & Unmet Need
Like many inborn error of metabolism disorders, standard-of-care treatments include dietary restrictions. Nonetheless, the unmet medical need is substantial given potential mortality at young ages and, at later stages, the need for organ transplant(s) in some patients and CNS complications in many. There are no approved therapeutics nor known drug candidates in late-stage clinical development.
LM-014 is a new molecular entity and First-in-Class drug candidate with a unique target product profile including administration to all ages with minimal sides effects and with a positive impact on multiple organ systems.
LM-014 is a novel, patented protein replacement therapy for a life-threatening inherited metabolic disorder for which there is no approved therapy.