The third quarter of 2025 was an active and eventful quarter for AlzeCure Pharma. In July, we received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for our pain drug Painless ACD440 for the rare disease erythromelalgia. Furthermore, we carried out a rights issue of SEK 48.5 million, with an overallotment option of SEK 10 million. The rights issue was backed by all major shareholders, as well as by the company’s Board of Directors and management group through subscription commitments. The rights issue was oversubscribed to 212% and generated total proceeds of SEK 58.5 million. We are very pleased and proud of this strong outcome and wish to thank all participants in the rights issue for their confidence in the plans on which the issue was based.
We also presented and published new data for our pain projects ACD440 and TrkA-NAM. During the quarter, we further strengthened our organization by appointing Dr. Cecilia Wadell as Head of Development. She brings extensive experience in AlzeCure’s therapeutic areas and will be responsible for leading our development efforts.
Our pain projects in the Painless platform, ACD440 and TrkA-NAM, continue to make good progress. With our TRPV1 antagonist ACD440, we have previously obtained positive clinical Phase IIa results in patients with chronic peripheral neuropathic pain (nerve injury pain). During the quarter, we also presented an expanded analysis of clinical data from the study at the pain congress for neuropathic pain. In July, we released a new publication presenting the results from our Phase IIa clinical trial.
During the third quarter, we received Orphan Drug Designation (ODD) from the FDA for ACD440 for the rare and chronic pain condition erythromelalgia. This represents a further endorsement and validation of the project. In the US alone, between 35,000 and 70,000 individuals3) suffer from erythromelalgia, experiencing burning pain that causes significant suffering for patients. There are currently no approved or curative treatments for the disease. It is also very encouraging that already in June we received positive feedback from the FDA regarding a potential Phase IIb/III registrational study for erythromelalgia. We are now continuing to work with this feedback and are simultaneously progressing with the out-licensing of the project.
Orphan Drug Designation offers several important advantages, including the possibility of 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 this promising project. In addition, the price of orphan drugs in the US is very high, with a median price of approximately SEK 2 million (about USD 218,000) for one year of treatment.4) 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 is also approximately 17 times higher than for other pharmaceuticals. On August 27, we held a seminar in which we discussed erythromelalgia, our initiatives, the significance of obtaining orphan drug designation and the overall orphan drug market. A recording of the seminar is available on our website and other channels.5)
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 September, we presented new preclinical data on the lead drug candidate in the project, ACD137, in an osteoarthritis model at the international pain congress NeuPSIG. The results showed significant pain relief in both movement-induced and evoked pain, as well as a significant anti-inflammatory effect.
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.
After receiving the grant of EUR 2.5 million from the European Innovation Council (EIC) Accelerator in the spring for a Phase II study in Alzheimer’s patients with NeuroRestore ACD856, we are now continuing the development of the project. The grant has been of great importance to AlzeCure, both financially and as a validation of the project itself. The EIC has also offered us the opportunity to potentially receive additional funding through a direct investment in the company, which we are now exploring further and working to secure. An investment from the EIC could accelerate the continued development of both the project and the company.
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.7) 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 Alzstatin are gamma-secretase modulators (GSM), which reduce the production of the harmful protein amyloid-beta-42 that generates plaques in the brain. GSM for the treatment of Alzheimer’s have received growing attention during the year 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 that it intends to present clinical interim results from its Phase II study in 2026. A second clinical study with Roche’s GSM compound has also been initiated by the Banner Institute, where the compound is being combined with an antibody treatment (donanemab from Eli Lilly).8)
We view these studies and initiatives as positive and validating for our Alzstatin project. They further strengthen interest both in GSM as a drug class and in our Alzstatin GSM project. In the field of Alzheimer’s, the medical need for effective treatments remains very significant. Studies show that only 5–8% of Alzheimer’s patients seen at memory clinics are regarded as appropriate candidates for prescription of the newly developed antibody therapies.9) 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.
With the new capital from the rights issue, we will be able to develop the business and advance our projects. The focus is to prepare for a Phase II clinical trial with our Alzheimer’s project 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 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.
To lead our development work, we hired Dr Cecilia Wadell as Head of Development during the third quarter. Cecilia brings extensive experience from both Big Pharma and biotech, as well as from CROs. She has worked at companies such as AstraZeneca, Medivir and Wilson Therapeutics. With her experience in the development of both traditional and orphan drugs, she contributes highly valuable expertise to our projects. We are delighted to welcome Cecilia to the AlzeCure team.
With strong progress and many positive events during the year, I look forward to working with my colleagues and our partners to ensure continued success in 2025.
Stockholm, November 2025
Martin Jönsson
CEO of AlzeCure Pharma AB