Arctic Therapeutics ehf, an Icelandic drug development company, in collaboration with Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company specializing in treatments for retinitis pigmentosa, cataracts, and other oxidative stress-related diseases, has received approval from the European Medicines Agency (EMA) to initiate the first clinical trial of AT-001 (also known as Nacuity’s NPI-001) for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA) in Iceland.

HCCAA is an extremely rare and severe condition characterized by the accumulation of amyloid in the brain's blood vessels, leading to cerebral hemorrhage, stroke, and progressive neurological deterioration, including dementia. The current treatment options are limited, primarily focusing on managing symptoms rather than addressing the root cause of the disease. AT-001, a purified, prescription-grade N-acetylcysteine amide (NACA), offers a novel therapeutic approach.

"We are pleased to announce EMA's approval, marking a significant milestone in investigating the safety and efficacy of AT-001 in halting or reversing the progression of HCCAA. Our study aims to explore the potential of AT-001 in altering the disease's course, improving patient outcomes, and ultimately, saving lives," said Ivar Hakonarson, CEO and co-founder of Arctic.

In September 2022, Nacuity and Arctic signed an exclusive licensing agreement and strategic partnership for the development and commercialization of Nacuity’s NPI-001, rebranded as AT-001, for HCCAA in Iceland and related forms of amyloid angiopathies (CAAs) worldwide. Nacuity is also currently conducting Phase 1/2 clinical trials in Australia for retinitis pigmentosa associated with Usher syndrome and cystinosis using NPI-001.

"Our proprietary NPI-001 holds promise in addressing various diseases and conditions associated with oxidative stress. EMA's approval for evaluating NPI-001 tablets for HCCAA treatment represents a significant step forward in combating HCCAA and underscores a crucial pharmaceutical quality milestone for our global development efforts," stated G. Michael Wall, Ph.D., SVP & CSO, Nacuity.

 

Source: globenewswire.com

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