Atlas Molecular Pharma (Derio), has received FDA approval to initiate clinical development of its first candidate, ATL-001

Atlas Molecular Pharma (Derio), a company dedicated to the identification and development of novel pharmacological chaperones through a proprietary platform, has received FDA approval to initiate clinical development of its first candidate, ATL-001.

ATL-001 is an orally administered chemical molecule that stabilizes the enzyme UROIIIS, which is key in the pathology known as congenital erythropoietic porphyria (CEP). In CEP patients, UROIIIS has mutations that destabilize its structure and it is unable to perform its function properly, leading to the accumulation of a series of toxic proteins that are responsible of the disease. The company saw a significant improvement of the pathology phenotype in vivo when ATL-001 was administered through stabilization of UROIIIS.

The company applied for the IND Application to the FDA during the summer months, ultimately obtaining a favorable agency response in September. This will allow Atlas to initiate clinical development of the molecule in the United States. The company will conduct a Phase I study in healthy volunteers and then, if no toxicity is observed, a Phase II study in patients

This is an important milestone for the company, whose ultimate goal is to provide an effective treatment for patients suffering from this ultrarare and devastating disease.