March 2024 • PharmaTimes Magazine • 9
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Neurona Therapeutics has announced that it has secured $120m in funding to help advance its lead candidate, NRTX-1001, a regenerative neural cell therapy currently being assessed for drug-resistant mesial temporal lobe epilepsy (MTLE).
Affecting around 50 million people globally, MTLE is the most common form of focal epilepsy, which can trigger ongoing seizures despite medical treatment.
Furthermore, for people with seizures resistant to anti-seizure drugs, epilepsy surgery can be an option. However, the surgical options are not available or effective for everyone and can be tissue-destructive with significant adverse effects.
The financing was provided through seed funding, co-led by Viking Global Investors and Cormorant Asset Management, as well as other institutional supporters including US Investments, UCB Ventures, Euclidean Capital, the Column Group and Alexandria Venture Investments.
Neurona’s lead candidate is a cell therapy comprised of inhibitory GABAergic interneurons, which could potentially address the underlying hyperactive neural networks in epileptic seizures.
Previous results from a phase 1/2 study of NRTX-1001, which Neurona presented at the annual meeting of the American Epilepsy Society in December last year, showed a more than 95% drop in overall seizure frequency for over a year after treatment.
In addition, the candidate was safe overall, with adverse events considered mild to moderate in severity.
Closed Loop Medicine has shared positive results of its novel drug and proprietary technology software, developed to personalise treatment for hypertension, or raised blood pressure, patients.
Findings from the study were published in the Journal of the American Heart Association. Currently the leading preventable cause of morbidity and premature death worldwide, hypertension occurs when the pressure in the blood vessels is abnormally high.
The first-line recommended anti-hypertensive drug, amlodipine, is usually discontinued by around 20% of patients due to unwanted effects, including peripheral oedema – the retention of fluid in the leg.
The results from the PERSONAL-COVIDBP clinical trial demonstrated the capabilities of the techbio company’s integrated precision care solution, CLM-HT01, to control blood pressure while minimising adverse reactions and supporting medical adherence in patients with primary hypertension.
CTM-HT01 is a single label combination product in development that is linked with Closed Loop Medicine’s software as a medical device in combination with amlodipine, which works to support more effective, precise and affordable disease management solutions to improve patient outcomes.
Researchers adjusted and personalised drug dosing using information recorded by patients into a smartphone app, which was transmitted securely to a clinician.
Results from the study demonstrated a significant reduction in hypertension, with the majority of patients achieving blood pressure control and minimising side effects, including those who were previously intolerant of conventional amlodipine doses.
MAIA Biotechnology has announced positive results from its preclinical studies for THIO, a treatment for small cell lung cancer.
The treatment was reported to have shown a profound and persistent tumour regression in advanced, in vivo cancer models. Meanwhile, the telomere-targeting agent effectively decreased cancer-initiating cells and diminished tumour-initiation potential in vitro and in vivo.
In addition, THIO was highly effective in combination with ionising radiation treatment regiments.
Researchers from Imperial College London have revealed that a blood test can pinpoint the underlying cause of brain injury in newborn babies.
The study could lead to a simple test to quickly diagnose brain injury in newborns and help with treatment decisions.
Researchers took blood samples from 35 babies born with hypoxic-ischaemic encephalopathy, before or shortly after birth, in Italy, and 99 babies in South Asia, shortly after birth.
The Joint Committee on Vaccination and Immunisation has announced its advice to the UK government in relation to the 2024 spring COVID-19 vaccine programme.
The committee’s advice encourages the government to offer the vaccine to those at high risk of serious disease and most likely to benefit from vaccination.
Multiple variants of the SARS-CoV-2 tend to co-circulate at different times of the year. In August and September 2023, the most dominant circulating variants were from the EG and XBB lineages.
A new programme led by Queen Mary University of London and funded by the Alzheimer’s Society has received positive trial results that could benefit carers and patients living with dementia.
The New Interventions for Independence in Dementia (NIDUS-Family) programme was successful in supporting patients and carers to achieve their goals compared to those who received usual care.
Dementia affects more than 944,000 people in the UK.
Researchers from King’s College London have predicted that the number of cases and deaths from intracerebral haemorrhage (ICH) is set to increase across Europe and the UK by 2050. ICH is a condition that occurs when haematoma is formed within the brain parenchyma with or without blood extension into the ventricles.
The study estimates a 59.4% increase in deaths related to ICH in people aged 70 to 95 and over in the UK by 2050.
The Academy of Medical Sciences has published a report that highlights evidence of declining health among children under five years in the UK and has called on policymakers to take urgent action.
The new report highlights that children’s health has been overlooked in policy, the health service and research from preconception through pregnancy to the first five years of life.
According to the report, which involved perspectives from parents and carers, infant survival rates are worse than in 60% of similar countries and, between 2019 and 2022, the total number of children living in extreme poverty tripled.