During the fourth quarter of 2025, our focus was on preparing the Phase II study in Alzheimer’s patients with NeuroRestore ACD856, for which we were awarded a grant of EUR 2.5 million from the European Innovation Council (EIC). The first disbursement of the grant was made in early December. During the quarter, we also worked on preparations for a potential registrational study for the pain project Painless ACD440, for which we received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) in February 2026. We have continued to actively participate in business development congresses and to publish new data for several of our projects at world-leading scientific conferences.
In the spring of 2025, we received a grant of EUR 2.5 million from the European Innovation Council (EIC) Accelerator for a Phase IIa clinical trial in Alzheimer’s patients with NeuroRestore ACD856, enabling us to continue development of the project. The grant is of great importance to AlzeCure, both financially and as a validation of the project itself. In December, the EIC made its first disbursement to the project. The EIC has also offered us the opportunity for potential additional funding through a direct investment in the company, which we are continuing to pursue. Such an investment could accelerate the continued development of the project and provide further validation for the company.
Previous preclinical and clinical results with ACD856 have also demonstrated a very good safety and tolerability profile, enabling a broad potential therapeutic window for ACD856. To leverage this opportunity, we have initiated clinical studies to further increase the dose in humans. These studies, expected to be completed in the first half of 2026, also address feedback received from pharmaceutical companies interested in in-licensing. The activities may broaden the scope of the compound, including within depression, an area of growing interest in which we have also published positive preclinical results.¹
NeuroRestore ACD856 is a “Trk-PAM,” a novel type of drug that enhances the brain’s BDNF and NGF signaling. Impaired function in these signaling pathways is associated with reduced cognition in several different diseases, such as Alzheimer’s disease, sleep disorders, traumatic brain injury, and Parkinson’s disease. Previous clinical studies have shown that the compound is safe, efficiently absorbed in the brain, and activates neuronal pathways important for both cognition and depression. Preclinical data also demonstrate positive effects on neuronal communication, learning, and memory, as well as protective and anti-inflammatory properties, including improved mitochondrial function. ACD856 has a unique mechanism of action and the potential to improve both learning and memory, as well as to have disease-modifying properties. This is of great importance for patients with Alzheimer’s and other neurodegenerative diseases.
Alzstatin, our disease-modifying and preventive treatment in tablet form for Alzheimer’s disease, continues to be developed according to plan. The drug candidate ACD680 is in preclinical development and is being prepared to enter clinical trials. The results indicate that with ACD680 we potentially have a so-called “Best-in-Class” molecule, and during the year we generated additional data supporting this achievement.² ACD680 is expected to have a long patent term, until 2045, as well as an additional five years of exclusivity in the US, which is very valuable and increases the project’s attractiveness.
The compounds in the Alzstatin program are “gamma-secretase modulators” (GSM) that reduce the production of the harmful protein amyloid-beta-42, which, among other effects, forms amyloid plaques in the brain. GSMs for the treatment of Alzheimer’s have received growing attention in recent years as the target mechanism has been validated by the Swiss pharmaceutical company Roche, which is also developing a GSM compound, RG6289 (nivegacetor). Roche has announced its intention to present clinical interim results from its ongoing Phase II study in 2026. A second clinical study with Roche’s GSM compound was initiated in 2025 by Martin Jönsson, CEO, at the Banner Institute, where they are also combining the compound with an antibody therapy (donanemab from Eli Lilly).³ We see these Roche studies and initiatives as positive and validating for our Alzstatin GSM project.
During the fourth quarter of 2025, our focus was on preparing the Phase II study in Alzheimer’s patients with NeuroRestore ACD856, for which we were awarded a grant of EUR 2.5 million from the European Innovation Council (EIC). The fourth quarter rounded off an active and highly successful 2025. In addition to receiving a grant from the European Innovation Council (EIC) for our study in Alzheimer’s patients, we were also granted Orphan Drug Designation from the US Food and Drug Administration (FDA) for our pain candidate Painless ACD440. Furthermore, during the year we carried out a successful rights issue that was oversubscribed to 212%, which was a strong endorsement for the company.
In the field of Alzheimer’s, the medical need for effective treatments remains very high. Studies show that only 5–8% of Alzheimer’s patients seen at memory clinics are suitable for prescription of the newly developed and approved antibody therapies.⁴ As a result, both NeuroRestore and Alzstatin could become highly attractive treatments in their own right, while also serving as a complement to antibody therapies, thereby addressing a high unmet medical need for patients, their families, and the healthcare system.
Our pain projects ACD440 and TrkA-NAM continue to make good progress. With the TRPV1 antagonist ACD440, we have previously obtained positive clinical Phase IIa results in patients with chronic peripheral neuropathic pain (nerve injury pain). In the fall, we presented an expanded analysis of clinical data from the study at the neuropathic pain congress,⁵ and during the year we also presented the results from the Phase IIa clinical trial in a new scientific publication.⁶ ACD440 has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the rare and chronic pain disorder erythromelalgia. Recently, in February 2026, we also received the corresponding designation from the European Medicines Agency (EMA). These classifications are further clear validations of the project.
In the US alone, between 40,000–70,000 individuals⁷ suffer from erythromelalgia, causing burning pain and significant distress for patients, including both adults and children as young as 3–4 years of age. There are currently no approved or curative treatments for the disease. We have previously received positive feedback from the FDA regarding a potential Phase IIb/III registrational study for erythromelalgia. During the quarter, we continued to advance the project and obtained initial study quotes requested as part of ongoing out-licensing discussions, a key focus for the company.
Orphan Drug Designation provides several highly important advantages, including the opportunity to obtain accelerated or conditional approval, as well as priority review. In addition, it provides stronger and extended market exclusivity, which enhances our competitive advantages and the conditions for out-licensing. In addition, the price of orphan drugs in the US is very high, with a median annual treatment price of approximately SEK 2 million (about USD 218,000).⁸ The orphan drug market has expanded rapidly in recent years, growing at roughly twice the pace of the overall pharmaceutical market. Pricing within the orphan drug segment in the US is also approximately 17 times higher than for other pharmaceuticals.
Our second pain project, TrkA-NAM, focuses on arthritis of the knee. Over 300 million people currently suffer from the disease, and the patient population is growing due to factors such as an aging population and obesity-related problems. TrkA-NAM is being developed to reduce peripheral NGF signaling and thus pain. In the fall, we presented new preclinical data for ACD137, the lead drug candidate in the project, in an osteoarthritis model at the international NeuPSIG pain conference. The results showed significant pain relief in both movement-induced and evoked pain, as well as a significant anti-inflammatory effect.⁹
The analgesic effect of ACD137 was as potent as that of the anti-NGF antibody tanezumab, which has demonstrated significant and robust pain relief in patients in several clinical trials. ACD137 was also shown to protect against articular cartilage damage, with significant improvements in several structural parameters for cartilage and the knee joint, suggesting a protective effect on knee joint function. The compound has previously demonstrated powerful analgesic effects in several different preclinical studies, in models for both neuropathic and nociceptive pain, indicating a wide range of applications for the compound.
We are now preparing ACD137 for further preclinical safety studies. With the available funds from the successful capital raise completed in August 2025, we will be able to drive the business development activities and projects forward. Our main focus going forward will be to plan and prepare for the Phase II clinical study with NeuroRestore ACD856. Furthermore, we continue to prepare both our pain project in knee osteoarthritis, TrkA-NAM ACD137, as well as the Alzheimer’s project Alzstatin ACD680, for Phase I clinical trial. At the same time, we remain strongly focused on business development with the aim of executing an out-licensing agreement for one or more of our projects.
With strong progress and many positive events in 2025, I look forward to working with my colleagues and our partners to ensure a successful 2026.
Stockholm, February 2026
Martin Jönsson
CEO of AlzeCure Pharma AB