Atlas Molecular Pharma to Begin Dosing First Patients in Phase 2 Trial of ATL-001 for Congenital Erythropoietic Porphyria
Atlas Molecular Pharma, a Basque biotech developing pharmacological chaperone therapies for rare diseases, announces that the first three patients enrolled in its Phase 2 clinical trial of ATL-001 are scheduled to begin treatment in September. It will be the first time the drug is administered to patients with Congenital Erythropoietic Porphyria (CEP), following a successful Phase 1 safety trial.
Congenital Erythropoietic Porphyria (CEP), also known as Günther disease, is an extremely severe and debilitating ultra-rare genetic disorder caused by a deficiency in the enzyme uroporphyrinogen III synthase (UROIIIS), a key component of the heme biosynthetic pathway. Patients with CEP face lifelong, often severe symptoms, including extreme skin photosensitivity, chronic anemia, and progressive organ damage. To date, CEP has no approved treatment on the market.
ATL-001, developed using Atlas Molecular Pharma's proprietary CHASSYS™ platform, is a pharmacological chaperone designed to stabilize the defective UROIIIS enzyme and restore its activity, and is the first therapeutic option designed to target the underlying cause of the disease rather than only its symptoms. The candidate holds Orphan Drug Designation from both the US Food and Drug Administration (FDA) and the European Commission.
In its Phase 1 trial, ATL-001 was evaluated in 40 healthy volunteers and demonstrated a favorable safety and tolerability profile, confirming earlier preclinical findings and paving the way for testing in the target patient population.
The ongoing Phase 2 trial is a defining step for the company: for the first time, ATL-001 will be tested directly in patients living with CEP, a disease for which no approved treatment currently exists. The three enrolled patients have completed the trial's screening assessments and the run-in period of 6 months and are scheduled to begin treatment in September. Following the study protocol, patients will receive a lower induction dose during the first 12 weeks of treatment, followed by the full dose for the subsequent 36 weeks. The study will evaluate the safety, tolerability, and preliminary efficacy of ATL-001 in this patient population.
Atlas Molecular Pharma is a biotechnology company and spin-off of CIC bioGUNE, headquartered at the Bizkaia Science and Technology Park (Spain). The company is dedicated to the development of innovative therapies for rare and ultra-rare diseases, such as congenital erythropoietic porphyria, prion diseases and tyrosinemia type I. Its technological platform includes second-generation pharmacological chaperones. The company develops its programs through an open innovation model, in collaboration with an international network of academic and clinical centers and industry companies.