Background

Arctic Therapeutics is developing AT-001, an oral treatment targeting neurodegenerative diseases like Alzheimer’s by disrupting amyloid plaques and preventing toxic protein formations.

The project is rooted in applied genomics and builds on the safe, well-known molecule N-acetylcysteine (NAC), with improved properties in its derivative, NAC Amide (NACA). AT-001 has shown promising results in early studies for Hereditary Cystatin C Amyloid Angiopathy (HCCAA), a rare form of familial dementia.

The drug crosses the blood-brain barrier, a major hurdle in neurodegenerative treatments. Arctic Therapeutics collaborates closely with leading genomics institutions and top-tier neurology experts.

Commercial Potential

Dementia affects over 55 million people globally, with costs expected to reach €2.8 trillion by 2030. AT-001 is positioned to capture part of the €1.4B serviceable market for familial dementia and Alzheimer’s treatments. Its oral administration, strong early data, and safer profile compared to current amyloid-targeting therapies make it an attractive candidate for broader neurodegenerative disease markets. If successful, AT-001 could lead Arctic to expand into other major indications like atopic dermatitis and psoriasis through its pipeline.

Achievements

AT-001 has received approval from the European Medicines Agency (EMA) for a registration study. Early Phase IIa trial results in HCCAA patients showed reduced amyloid deposits, halted disease progression, and no new serious events. The company secured exclusive rights for NACA development in neurological disorders through a partnership with Nacuity Pharmaceuticals. Arctic Therapeutics also built a strong leadership team and a network of high-profile collaborators from Harvard, UCL, and major stroke research centers.

Highlights

- AT-001 disrupts amyloid plaques and prevents toxic protein formation.

- Oral administration with good blood-brain barrier penetration.

- Strong early Phase IIa results in rare dementia (HCCAA).

- Exclusive licensing deal with Nacuity Pharmaceuticals for NACA in neurology.

- Backed by senior researchers from Harvard, UCL, and Mass General.

Senior Review

All three senior reviewers support funding Arctic Therapeutics, highlighting its strong team, safe drug profile, and clear alignment with Cerebrum DAO’s mission. They agree AT-001 has strong potential if Phase IIb/III trials are successful, despite risks typical of neurodegenerative disease studies. Reviewers noted that AT-001 builds on a clinically established molecule (NAC) and emphasized the de-risked path using HCCAA as a proof-of-concept model. Overall, the project is seen as highly promising but will require careful execution in future clinical trials to reach full commercial potential.