Last year, AT secured approval from EMA for the initiation of an Investigational Medicinal Product (IMP) registration study targeting Hereditary Cystatin C Amyloid Angiopathy (HCCAA). This approval is not just a pivotal step for us but also lays the groundwork for extending our research into more common forms of dementia, including AD. The Phase IIb/III clinical trial for AT-001 in HCCAA has successfully reached its one-year mark. Enrollment was completed in December 2024, with 15 patients enrolled from the original target cohort of up to 25. The trial follows an open-label, single-arm protocol with dosage escalation based on tolerability and biomarker response. Throughout the study, patient safety has remained a top priority, with scheduled Data Safety Monitoring Board (DSMB) reviews confirming adherence to safety protocols. No major safety concerns have been reported, and all patients remain actively engaged in the study. Preliminary biomarker analysis suggests modulation trends consistent with the expected therapeutic mechanism of AT-001, though full statistical evaluation will be conducted at the trial’s completion. All safety and observable efficacy measures appear strong, and the study remains on track with no major deviations. The final patient is expected to complete 12 months of treatment by the end of the year, at which point the full safety and efficacy dataset will be analyzed to support further clinical and regulatory decisions. AT firmly believes this will result in a market approval in Iceland to treat HCCAA, as well as opening the possibility of off-label prescription for other forms of familial dementia in Europe, anchored by the strong safety profile of the treatment.
AT’s commercial model is geared towards partnering with an established pharma major to bring the AT-001 to market, tapping into the experience and infrastructure of a leading player driving consolidation in the wider therapeutic area (TA). We strongly believe that it is extremely important to demonstrate a clear differentiation from approved and emerging rival treatments ahead of any out-licensing agreement. This will put AT in a much stronger position to secure and negotiate a more favorable out-licensing agreement with partners to take our assets the last mile to market. It is important to note that we are defining our competition from an asset perspective – not from a company perspective, since both Biogen and Lilly are possible co-development partners, despite having competing assets approved in certain geographies. This work is progressing well, and we have identified all major approved and emerging competing assets from information available in the public domain, as well as shaping the differentiation based on our business intelligence work.
1. We have commenced soft outreach via our proprietary network, as working towards aligning our value proposition across online and offline channels and material. This includes a revamped website, with messaging geared towards BD&L teams at possible pharma partners consolidating or investing in our core therapeutic areas (TA). When completed, this sharpened messaging will be amplified via digital channels to generate inbound conversations, providing air cover ahead of direct outreach.
2. The news of the closing of our oversubscribed €26.5 million Series A financing was widely reported across top-tier trade, tech, and financial publications, including Endpoints and Bloomberg. This heightened exposure has led to an influx of inbound interest and discussions – not only around our frontrunners but also across our broader portfolio. Regarding AT-001, we have already started to engage with pharma majors on a possible collaboration – including Roche/Genentech and Merck & Co (MSD Group in North America).
