During the recent meeting of the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) that took hold from October 13th to 15th (https://www.ectrims-congress.eu/2021.html) , Accure Therapeutics has presented four posters summarizing the progress of ACT-01 as a new disease-modifying therapy for Multiple Sclerosis (MS). ACT-01 is a small chemical with neuroprotective activity that has shown positive results in animal models of neuroinflammation and neurodegeneration. Accure Therapeutics is developing ACT-01 to treat MS, including the common relapse of Acute Optic Neuritis. The ECTRIMS is the most attended international meeting about MS. Therefore, most of the basic and clinical research is presented and discussed, an ideal forum for disclosing the latest results of the ACT-01 program for this disease. A summary of the 4 posters is presented below:
Considering that ACT-01 mechanism of action is activating the trophic factor pathways such as IGF-1 or BDNF, it was essential to assess if after a brief therapy with ACT-01, the beneficial effects of ACT-01 in terms of axonal protection were sustained along time. Accure Therapeutics researchers demonstrated that after five days of treatment in the lysolecithin-induced demyelination model of the optic nerve, the benefits in terms of axonal protection were extended and even increased up to one month later. Such results provide a therapeutic rationale for treating MS patients with intermittent therapy (e.g., once per week or month) to protect the brain and delay disability accumulation.
Combination therapy is becoming the preferred approach for chronic diseases. In the case of MS, patients and neurologists have expressed their preference for stopping the inflammatory response with immunomodulatory drugs while protecting the brain or even regenerating it with new neuroprotective therapies. Using the Experimental Autoimmune Encephalitis model in rodents, the scientists demonstrated that the combination with most approved immunomodulatory drugs for MS was safe and well-tolerated. Even more importantly, the combination of ACT-01 with fingolimod or dimethyl-fumarate showed an enhanced efficacy (synergistic effect). Such results provide an ideal scenario for treating people with MS with combination therapy that significantly delays disability accumulation.
Accure Therapeutics presented the ACT-01 phase 1 trial results in healthy volunteers and showed that ACT-01 was safe and well-tolerated when administered for 2h intravenously up to 5 consecutive days. In addition, the pharmacokinetic studies confirmed the linear behavior of the drug across species, which allows predicting dose behavior in clinical studies.
Based on the positive preclinical and phase 1 study results, Accure Therapeutics has recently started a phase 2 trial for evaluating the safety and efficacy of ACT-01 in patients with Acute Optic Neuritis. The poster describes the study design and protocol to inform the medical community about the ongoing trial.
Accure Therapeutics is a private translational R&D engine at clinical stage in the Central Nervous System (CNS) field. Based in Barcelona (Spain), it was launched in 2020 with a Series A funding led by Alta Life Sciences Spain I and supported by the Centre for Technological and Industrial Development (CDTI). This European company with an international mindset boasts a unique portfolio of three new chemical entity programs pursuing innovative targets – with potential to accommodate others. Accure aims to develop new disease modifying drugs to treat serious conditions such as optic neuritis, multiple sclerosis, Parkinson’s disease and epilepsy. With an experienced business and scientific team, Accure Therapeutics is one of the few companies that operate in an agnostic fashion on initial science to deliver cutting-edge drugs in CNS.