EMA approved clinical trial of AT-001 (NPI-001) in Iceland

REYKJAVIK, ICELAND and FORT WORTH, TX – March 12, 2024 – Arctic Therapeutics ehf,  an Icelandic drug development company and partner Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced approval by the European Medicines Agency (EMA) for initiation of the first clinical trial evaluating AT-001 (Nacuity’s NPI-001) for the treatment of Hereditary Cystatin C Amyloid Angiopathy (HCCAA) in Iceland.

HCCAA is an ultra rare, serious condition characterized by the accumulation of amyloid in the brain's blood vessels, which can lead to cerebral hemorrhage, stroke, and progressive neurological deterioration, including dementia. Current treatment options are severely limited, focusing primarily on symptom management rather than addressing the underlying cause of the disease. AT-001 is a differentiated, purified, prescription-quality, GMP-grade N-acetylcysteine amide (NACA).

“EMA's approval paves the way for a comprehensive investigation into the safety and efficacy of a novel therapeutic approach designed to halt or reverse the progression of HCCAA. Our study aims to explore the potential of AT-001 in modifying 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 entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of Nacuity’s NPI-001 under the name AT-001 for HCCAA in Iceland and related forms of amyloid angiopathies (CAAs) worldwide. Nacuity is also currently evaluating NPI-001 in Phase 1/2 clinical trials in Australia for retinitis pigmentosa associated with Usher syndrome and cystinosis.

“Our proprietary NPI-001 has potential to address the myriad diseases and conditions where oxidative stress plays a role. EMA approval for the evaluation of NPI-001 tablets for treatment of HCCAA represents most importantly, a step forward in addressing HCCAA, a life threatening and underserved rare disease, and secondly, a key pharmaceutical quality milestone for the companies in the global development of NPI-001,” said G. Michael Wall, Ph.D., SVP & CSO, Nacuity.

About Nacuity Pharmaceuticals, Inc.

Nacuity Pharmaceuticals, Inc. (https://www.nacuity.com/), is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to attentuate oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has three highly differentiated clinical programs ongoing in retinitis pigmentosa, cataracts and cystinosis with the potential to be first-of-a-kind therapies and gateways to wider applications. Nacuity has operations in Fort Worth, TX, USA, and Australia, and extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org).

About Arctic Therapeutics ehf

Arctic Therapeutics ehf (AT) (https://www.arctictherapeutics.com/) is an Icelandic drug development company, established in 2015 as a spin-off from the US-based Center for Applied Genomics (CAG), a research center at the Children's Hospital of Philadelphia. AT leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world’s most challenging diseases. The company has operations in Iceland, USA, and multiple collaborations across Europe.


Nacuity Media Contact
Julia Clements

Arctic Media Contact
Gulli Arnason