Atlas Molecular Pharma announces the start of human clinical trial

The Basque company begins the first phase I clinical trial with ATL-001 for the treatment of congenital erythropoietic porphyria (CEP)

Atlas Molecular Pharma (Atlas), a spin-off from the CIC bioGUNE research center and led by Óscar Millet and Joaquín Castilla, was founded in 2015 with the aim of developing new pharmacological chaperones for the treatment of rare metabolic diseases.

The biotech company, which closed a €3M round led by Inveready a year ago to advance the clinical development of its first candidate, announced today the start of its first human clinical trial with ATL-001. ATL-001 acts as a pharmacological chaperone by stabilizing and restoring the function of a protein that causes Congenital Erythropoietic Porphyria.

CEP is an ultra-rare, severe and debilitating pathology with no therapeutic alternative on the market and no drugs in development, representing a clear unmet medical need. With this Phase I, double-blind, randomized, and placebo-controlled trial, the safety of the drug will be measured in healthy volunteers as a primary objective and the pharmacokinetic profile as a secondary objective.

Trial participants will receive a certain dose of ATL-001 for 5 consecutive days with the aim of finding the recommended dose. To this end, four cohorts are planned to include 8 healthy subjects each. The drug dose will be escalated as successive cohorts are involved in the trial. To carry out this clinical phase, a single North American center has been selected: Hassman Research Institute located in New Jersey, United States.

In the words of Óscar Millet, CEO of Atlas: “Our goal at Atlas Molecular Pharma is to develop innovative therapies for rare and neglected metabolic diseases, and we are very excited to start the first human clinical trial with ATL-001 for congenital erythropoietic porphyria. We hope that this study will bring us closer to an effective treatment for a devastating disease that currently lacks adequate therapeutic options.

In January 2018 and May 2018, the EMA and FDA granted ATL-001 orphan drug designation for the treatment of Congenital Erythropoietic Porphyria. Once this clinical phase is completed, Atlas will administer the recommended dose to patients suffering from Congenital Erythropoietic Porphyria in a Phase II clinical trial.