During the first quarter of 2026, 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). In addition, we successfully advanced the Phase Ib clinical trial with ACD856, further expanding the therapeutic dose window to increase our opportunities to address multiple indications. During the quarter, we also continued preparing for a potential registrational study for the pain project Painless ACD440.
In February, ACD440 was granted orphan drug designation by the European Medicines Agency. We also remained active in business development at international conferences and published new data from our NeuroRestore project at the world-leading Alzheimer’s conference AD/PD 2026, which was held in Copenhagen in March.
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. This grant is of great significance for AlzeCure, both financially and as a validation of the project itself. The EIC made its first payment to the project in December. During the first quarter, we used part of the funding to prepare for the launch of the Phase IIa trial, which is expected to begin later this year.
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 linked to cognitive deterioration in several diseases, including not only Alzheimer’s disease but also depression, traumatic brain injury, sleep disorders, and Parkinson’s disease. Previous preclinical and clinical results with ACD856 have demonstrated a very favorable safety and tolerability profile, supporting a large potential therapeutic window. To leverage this potential, we have initiated clinical trials to further increase the dose in humans. The trials are proceeding according to plan and are expected to be completed during the first half of 2026.
These studies also address the feedback we have received in our discussions with other pharmaceutical companies. One area where we are seeing growing interest is depression—an indication for which we have also published positive preclinical results.1) Depression is one of the most common disorders globally, affecting approximately 280 to 350 million people, and there remains a significant need for improved treatments. About one-third of patients are currently treatment-resistant and do not respond satisfactorily to existing therapies. In addition, side effects associated with current treatments further drive the demand for new alternatives. We therefore hope that NeuroRestore may become a possible treatment option for patients suffering from depression.
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 capacity, as well as to be disease-modifying. This is of great importance for patients with Alzheimer’s and other neurodegenerative diseases. At AD/PD 2026 in Copenhagen, held March 17–21, we also presented and published new data for NeuroRestore ACD856 relating to BDNF and NGF signaling in models linked to Alzheimer’s disease and depression.2)
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 “Best-in-Class” molecule, and over the past year we generated additional data supporting this achievement.3) ACD680 is estimated to have patent protection until 2045 with an additional five years of exclusivity in the US, which is very valuable and increases the project’s attractiveness.
In the field of Alzheimer’s, the medical need for effective treatments remains very significant. Studies show that only 5–8 percent 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 therapy, thereby addressing a high unmet medical need for patients, their families, and the healthcare system.
Our pain projects ACD440 and TrkA-NAM also 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 of 2025, we presented an expanded analysis of clinical data from the study at the neuropathic pain congress5) and last year we also presented the results from the Phase IIa clinical trial in a new scientific publication.6)
ACD440 has previously been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for the rare and chronic pain disorder erythromelalgia. 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 and 70,000 individuals7) are affected by erythromelalgia, which manifests as burning pain. The disease causes great suffering for patients, including both adults and children from as early 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 quotes for this type of trial, which have been requested in connection with discussions on out-licensing.
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 enhanced and extended market exclusivity, which enhances our competitive advantages and the conditions for out-licensing. In addition, orphan drugs in the US are very expensive, with a median price of approximately SEK 2 million for one year of treatment, which is about 17 times higher than other medications.8) The orphan drug market has expanded rapidly in recent years, growing at roughly twice the pace of the overall pharmaceutical market.
Our second pain project, TrkA-NAM, focuses on arthritis of the knee. Over 300 million people are currently estimated to 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 associated pain. In the fall of 2025, 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.
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, and the Alzheimer’s project Alzstatin ACD680, for Phase I clinical trials. We are also intensifying our efforts on business development with the aim of securing an out-licensing agreement for one or more of our projects.
During the first quarter of 2026, we obtained orphan drug designation from the European Medicines Agency (EMA) for our pain compound Painless ACD440, which means we now hold designation in both Europe and the US—a clear strength for the project. Our main focus during the spring has otherwise been on preparing the Phase IIa trial in Alzheimer’s patients with NeuroRestore ACD856 to enable study initiation during 2026.
With a strong and active start to the year, and many positive events in 2025, I look forward to working with my colleagues and our partners to ensure a successful 2026.
Stockholm, May 2026
Martin Jönsson
CEO of AlzeCure Pharma AB