Atlas Molecular Pharma successfully completes phase one in humans of its treatment for Congenital Erythropoietic Porphyria Erythropoietica Congenita
The final results of ATL-001, the biotech company's lead candidate, have shown that it is able to stabilize UROIIIS and recover its enzymatic activity.
Atlas Molecular Pharma, a spin-off from CIC-Biogune backed by Inveready, under Óscar Millet (CEO) leadership has successfully completed its first human Phase I trial with its lead candidate, ATL-001. This biotech company focuses on discovering and developing new pharmacological chaperones for the treatment of rare and ultra-rare diseases.
ATL-001 targets a deficiency in the uroporphyrinogen III synthase (UROIIIS) enzyme, a critical part of the heme biosynthetic pathway. This deficiency leads to Congenital Erythropoietic Porphyria, a severe and debilitating condition. ATL-001 can stabilize UROIIIS and recover its enzymatic activity.
The completion of Phase I, which involved 40 healthy volunteers, marks a significant milestone for Atlas Molecular Pharma. Preclinical in vivo studies had already shown promising results, significantly improving the disease phenotype. The Phase I trial has now confirmed the drug's safety and tolerability in humans.
With these outcomes, the company is poised to begin Phase II trial in the United States. This next phase will assess the efficacy of ATL-001 in patients suffering from Congenital Erythropoietic Porphyria. The successful transition to Phase II underscores the potential of ATL-001 to become the first pharmacological treatment for this rare disease.
Atlas Molecular Pharma continues to advance its mission of developing innovative therapies that can transform the lives of patients with rare diseases and ultra-rare diseases.