AlzeCure Pharma, a pharmaceutical company that develops a broad portfolio of small molecule candidate drugs for diseases affecting the central nervous system, with projects in both Alzheimer’s disease and pain, today announced that the company has received new data from the clinical phase I study (multiple ascending dose, MAD) with repeated dosing of the drug candidate ACD856, which is being developed against Alzheimer’s disease and other indications with cognitive dysfunction.
New data from a planned exploratory analysis of the MAD study show that ACD856 increases EEG activity in the brain. A clear difference can be seen after versus prior to administration of the substance. This result combined with previously reported data show that the substance not only crosses the blood-brain barrier, but also reaches and activates neural pathways in the brain, with the potential of having positive effects on cognition.
The MAD phase I study is AlzeCure’s third clinical study with ACD856, the company’s leading drug candidate within the NeuroRestore platform. The substance is under development as a symptom-relieving treatment for medical conditions where the cognitive ability is impaired, for example in Alzheimer’s disease. The primary study objective was to evaluate the drug candidate’s tolerability and safety after repeated dosing. As previously reported, ACD856 shows good safety and tolerability in both the SAD and MAD studies.
ACD856 and the other substances in the NeuroRestore platform stimulate several important signaling systems and signaling substances in the brain such as BDNF (Brain Derived Neurotrophic Factor) and NGF (Nerve Growth Factor), which can lead to improved cognition, something that has been demonstrated in previous preclinical studies. New preclinical results also show potential neuroprotective and disease-modifying effects with these substances. The biological mechanism behind NeuroRestore enables several indications, such as Alzheimer’s and Parkinson’s disease, but also traumatic brain injury and depression.
“These new data are very promising and show that the substance reaches and activates neural pathways in the brain, whose normal function is disrupted in diseases such as Alzheimer’s,” said Johan Sandin, CSO at AlzeCure Pharma.
“These are very good news that add to the previous positive data for ACD856 and further strengthens our commercial opportunities for this promising substance,” said Martin Jönsson, CEO.
Second orphan medicinal product designation for Neuro-Cells
The European Union has granted stem cell biotech Neuroplast an orphan medicinal product designation for the applicability of its stem cell technology platform to frontotemporal dementia (FTD), following a positive opinion from The European Medicines Agency (EMA). With the existing orphan disease designation (ODD) for traumatic spinal cord injury (TSCI), Neuro-Cells® is now approved for a fast-track development pathway with market exclusivity for both a trauma-induced and a chronic degenerative central nervous system disorder. This marks an important milestone in the development roadmap of Neuroplast’s Neuro-Cells® platform, as a stepping stone to other chronic neurodegenerative diseases such as Alzheimer’s, ALS and Parkinson’s Disease. The potential width in therapeutic applicability of the Neuroplast technology gives perspective to millions of people suffering from neurodegenerative diseases that currently have no outlook on effective treatment.
One technology addresses underlying mechanisms of multiple acute and chronic neurological disorders
Several conditions of the central nervous system, even when they seem unrelated at first and may have distinctive causes, have similar underlying disease mechanisms in common. These include unprogrammed cell death boosted by inflammation. Neuro-Cells, an autologous, bone-marrow derived Advanced Therapy Medicinal Product, addresses that disease mechanism by moderating inflammation of damaged cells in the central nervous system, to limit further impairment. The treatment objective in acute disorders is to limit impact of sudden injury, where the treatment objective in chronic disorders is to limit progression of the disease.
Neuroplast is already running a fast-track development pathway for traumatic spinal cord injury (TSCI), with a Phase II clinical trial in progress. This designation for frontotemporal dementia illustrates the broader applicability of the same technology for acute as well as chronic neurodegenerative disorders, paving the way to explore further applicability to conditions such as ALS, Alzheimer’s disease, traumatic brain injury, subarachnoid stroke and Parkinson’s Disease.
Orphan disease designation for FTD awarded based on pre-clinical evidence
Orphan disease designations are restricted to products for rare conditions for which there are no satisfactory methods of treatment authorized. It allows for a faster market authorization pathway and ten-year market exclusivity.
Frontotemporal dementia (FTD) is a degenerative condition in the brain that affect approximately 3.8 people in 10,000 persons in the EU. Typical survival rate lies between three and fourteen years from symptom onset, dependent on the FTD variant at play.
For this approval, the European Union followed the positive opinion from the EMA after the EMA followed positive recommendations from the Committee for Orphan Medicinal Products (COMP). COMP partly based their conclusions on the availability of pre-clinical evidence in mice, that showed decrease in neuroinflammation markers and rescue of cognitive and social behavioral deficits. Examples include reduction of anxiety, depressive-like behavior and abnormal social behavior.
Neuroplast CEO Johannes de Munter states:“This designation for frontotemporal dementia is an important milestone in expanding the Neuro-Cells development to a wider range of therapeutic areas. Using the same technology platform for traumatic spinal cord injury and frontotemporal dementia, illustrates an unusual range of acute and chronic neurological disorders that could potentially benefit from this.”
Neuroplast is open to discuss investor opportunities to effectuate the clinical pathways to a wider scope of neurological conditions.
Inhaled COVID-19 vaccines
Contrary to some media claims, neither CanSino Biologics’ Convidecia Air nor Bharat Biotech’s BBV154 are the first inhaled COVID-19 vaccines to market, according to GlobalData, which highlights that two such vaccines were approved in Russia in March and July this year. However, the leading data and analytics company notes that the trend towards inhaled vaccines is a positive move, as they have the potential to build ‘mucosal immunity’, where the COVID-19 virus is ‘blocked’ from entering the body via the nose, throat or lungs.
Janet Beal, Senior Research Analyst in Health Economics and Market Access at GlobalData, comments: “As the current set of intramuscular COVID-19 vaccines reduce the likelihood of severe infection but do not block transmission, and vaccinated individuals can pass on infection while asymptomatic, there remains a high risk of being stuck in a cycle of ‘waves’ of infection, despite high primary and booster vaccination rates, as we try to emerge from the pandemic. However, if these new inhaled vaccines generate mucosal immunity, as has been demonstrated for other inhaled or oral vaccines against several pathogens and allergens, they could effectively block COVID-19 viral entry across the mucosa and subsequent transmission, representing a step-change in our global emergence from the pandemic—a way to break the cycle.
“The current evidence base for such activity from human clinical trials of inhaled COVID-19 vaccines is still relatively limited, but these initial four inhaled vaccines are likely to be the first of several coming through trials—with developers in countries ranging from the US and Japan to Cuba, Iran and Mexico.”
GlobalData’s World Markets Healthcare has documented that the first inhaled vaccine for COVID-19 to reach the market was a nasal spray version of the widely-approved injectable vaccine Sputnik V, approved in March 2022. This was followed by Salnavac, a nasal drop form of Sputnik V, in July 2022; Convidecia Air on September 4, 2022; and, most recently, BBV154 on September 6, 2022.
“While both of these vaccines received emergency approvals from a relatively limited evidence base (Phase I/II safety and immunogenicity data only), both are closely based on the well-established injectable vaccine Sputnik V, approved in many countries around the world. The Gamaleya Institute is currently planning late-stage trials, following a similar scheme to the pathway followed for Sputnik V itself, for which protective efficacy was only fully confirmed after the vaccine had received its initial emergency approval for public use. Both of these inhaled vaccines would normally be considered to have promise as boosters, with convenient administration and the potential to block infection. However, in the current climate under international sanctions, their export and use beyond Russia looks unlikely in the near term.”
AlzeCure receives US patent for NeuroRestore ACD856
AlzeCure Pharma AB, a pharmaceutical company that develops a broad portfolio of small molecule candidate drugs for diseases affecting the central nervous system, with projects in both Alzheimer’s disease and pain, today announced that the United States Patent Office (USPTO) has issued a patent covering ACD856, which is being developed against Alzheimer’s disease and other disorders with cognitive impairment.
USPTO has announced that they have now approved the company’s patent application in the US, which refers to ACD856, the leading drug candidate in the NeuroRestore platform, which is being developed against Alzheimer’s disease. The patent number is US 11,352,332 and the patent is valid until 2039.
ACD856 and other substances in the NeuroRestore platform stimulate several important signaling systems and signaling molecules in the brain such as BDNF (Brain Derived Neurotrophic Factor) and NGF (Nerve Growth Factor), which can lead to improved cognition. Previous preclinical studies have shown that AlzeCure’s drug candidates strengthen communication between nerve cells and improve cognitive ability, including learning and memory functions. New preclinical results also show potential neuroprotective and disease-modifying effects with these substances. The biological mechanism behind NeuroRestore enables several indications, such as Alzheimer’s and Parkinson’s disease, but also depression.
“We are continuing to build a patent portfolio for our NeuroRestore program. This is an important step for ACD856, which is in clinical development against Alzheimer’s,” said Gunnar Nordvall, Head of Chemistry and IP.
“These are very good news and constitute an important milestone for both the project and the company and further strengthens our commercial opportunities for this promising substance, which has just generated new positive clinical data,” said Martin Jönsson, CEO.