Author: Rachael Hansford

 A blood test for early cognitive decline?

Posted on December 21, 2024 by - Alzheimer's Disease, Cognition, Dementia

Identifying patients at risk for vascular brain injury before the onset of cognitive decline

21 December 2024: To identify and track blood vessel-related changes in the brain that contribute to cognitive impairment and dementia, researchers and clinicians typically rely on MRI to assess “downstream” biological markers – those at the end of a cascade of events. However, a multicentre study led by UCLA researchers could lead to a cost-effective blood test to detect changes occurring earlier in the process, potentially identifying at-risk patients at an earlier stage.

“We studied a protein in the blood that is crucial for blood vessel formation but also appears to play a role in vascular permeability linked to cognitive decline. By evaluating data from a large group of patients with varying vascular risk profiles and cognition ranging from unimpaired to mild dementia, we found that plasma levels of this protein, placental growth factor (PlGF), could potentially be used as a biomarker to screen for and monitor cognitive impairment and dementia,” said Jason Hinman, MD, PhD, a vascular neurologist at UCLA Health, Interim Co-Director of the Mary S. Easton Center for Alzheimer’s Research and Care at the David Geffen School of Medicine at UCLA, and senior author of an article in Alzheimer’s & Dementia: The Journal of the Alzheimer’s Association.

Dysfunctional cells lining blood vessels in the brain are increasingly recognised as a key driver of processes leading to cerebral small vessel disease (CSVD), a major contributor to cognitive decline and dementia. The leaky vessels are believed to allow fluid and inflammatory molecules to seep into brain tissue. CSVD is typically diagnosed through costly brain MRI, where areas of vascular-mediated brain injury appear as bright spots on clinical MRI sequences – called white matter hyperintensities, or WMH. WMH and other structural changes are late markers of vascular brain injury.

The researchers studied possible associations involving several factors: plasma levels of PlGF, a highly sensitive research MRI measure of fluid accumulation in the brain called white matter free water (FW), white matter hyperintensities, and patients’ scores on cognitive assessments. Results were consistent with models suggesting that elevated PlGF increases vascular permeability, leading to fluid accumulation in the brain’s white matter, development of white matter hyperintensities, and subsequent cognitive impairment.

A blood test for early cognitive decline?

“As a biomarker for cerebral small vessel disease and the vascular contributions to cognitive impairment and dementia (VCID), PlGF could be used as a cost-effective screening tool for identifying patients at risk of vascular brain injury before the subtle onset of cognitive decline,” said first author Kyle Kern, MD, a vascular neurologist at UCLA Health and researcher at the David Geffen School of Medicine at UCLA. “As a simple blood test, such a tool would be valuable not only for patients and clinicians but also for researchers identifying patients for clinical trials,” he added.

The study was conducted by researchers involved in MarkVCID, a multisite consortium established to validate candidate biomarkers for CSVD through the recruitment of participants from diverse racial and ethnic backgrounds, with a range of vascular risk factors, and across the spectrum of cognitive impairment. Participants were aged 55 or older and had undergone brain MRI and blood tests for PlGF levels.

The authors stated that while the study’s multicentre design and large, diverse sample support the use of PlGF as a biomarker, additional longitudinal studies are needed to reach conclusions about causation and timing in the relationships among PlGF, FW, WMH, and cognition. Ideally, PlGF could be used to screen younger populations for whom currently available treatments and lifestyle modifications may prevent or reverse the harmful effects of vascular injury before the onset of cognitive dysfunction. The research group is recruiting patients for future studies.

Tavapadon: A game-changing partial dopamine agonist?

Posted on December 20, 2024 by - Movement Disorders, Parkinson’s Disease

20 December 2024: AbbVie recently announced positive topline results from its pivotal Phase III TEMPO-2 trial evaluating investigational tavapadon as a flexible-dose monotherapy in early Parkinson’s disease (PD). Tavapadon, the first and only D1/D5 partial agonist in the dopamine agonist class, is under investigation as a once-daily treatment for PD. The trial met both its primary and secondary endpoints, suggesting that tavapadon has the potential to become a prominent treatment within the dopamine agonist market, says data and analytics company GlobalData.

Jos Opdenakker, Neurology Analyst at GlobalData, comments: “Tavapadon has displayed efficacy and tolerability both as a monotherapy and as an adjunctive therapy. In a phase III trial, patients treated with tavapadon alongside levodopa experienced a significant increase in total “ON” time without troublesome dyskinesia compared to those treated with Levodopa and placebo, highlighting tavapadon’s versatility and use potential in different clinical scenarios.”

The positive results for tavapadon in this pivotal study align with the sentiments of key opinion leaders (KOLs) recently interviewed by GlobalData, who held largely positive opinions on the drug. KOLs frequently cited the promising efficacy data of tavapadon and its unique position as a D1/D5 partial agonist. Furthermore, KOLs stated that tavapadon could be used for dyskinesia, as well as both as a monotherapy in early PD and as an adjunctive therapy in advanced PD.

Opdenakker continues: “There is concern over tavapadon’s market positioning, despite its unique stimulation profile. As a dopamine agonist, it would be entering a crowded market space within the PD treatment landscape and would face fierce competition from several cheaper genericised marketed in-class agents.”

There are six dopamine agonists currently available across the seven major pharmaceutical markets (the US, France, Germany, Italy, Spain, the UK, and Japan), several of which are available as generics, with two additional assets (AbbVie’s tavapadon and IRLAB Therapeutic’s mesdopetam) in the late-stage pipeline (Phases IIb–III).

Tavapadon has displayed efficacy and tolerability both as a monotherapy and as an adjunctive therapy. In a phase III trial, patients treated with tavapadon alongside levodopa experienced a significant increase in total “ON” time without troublesome dyskinesia compared to those treated with Levodopa and placebo.

GlobalData expects tavapadon to enter the market in 2028, and despite its impressive efficacy data, the drug’s success will likely be influenced by its safety profile. KOLs have noted that dopamine agonists are no longer the preferred adjunctive therapies for PD due to the risk of side effects such as hallucinations, delusions, and compulsive behavior. Tavapadon may become a success in the dopamine agonist market as a result of its improved safety profile coupled with strong efficacy data.

New mechanism for blood flow regulation in the brain

Posted on December 19, 2024 by - Alzheimer's Disease, Cognition, Research, Stroke

19 December, 2024: A study led by Mark Nelson, PhD, from the Larner College of Medicine at the University of Vermont, has identified a new mechanism, Electro-Calcium (E-Ca) Coupling, that integrates electrical and calcium signalling to regulate blood flow in the brain. Published in PNAS, the research redefines how capillaries ensure precise oxygen and nutrient delivery to active neurons.

Previously, electrical and calcium signalling were considered independent mechanisms. Nelson’s team discovered that these processes are interconnected. Electrical signals, facilitated by capillary endothelial Kir2.1 channels, spread rapidly across the capillary network, while calcium signals fine-tune local blood flow by triggering vascular responses. E-Ca coupling bridges these systems, with electrical waves enhancing calcium activity, enabling a synchronised response that adjusts blood flow both locally and across larger areas.

Advanced imaging and models showed that electrical signals amplify calcium activity by 76%, improving blood flow regulation. Simulated brain activity further increased calcium signalling by 35%, demonstrating a balanced distribution of blood across the capillary network. This mechanism ensures efficient delivery to areas of greatest demand.

Importantly, the team linked their findings to diseases like Alzheimer’s and small vessel disease, suggesting that restoring E-Ca coupling may correct cerebral blood flow deficits and slow cognitive decline. This research highlights capillaries’ crucial role in brain health and opens avenues for new treatments targeting disrupted blood flow in neurological conditions, including stroke and dementia.

This use-dependent increase in local blood flow (functional hyperemia), mediated by mechanisms collectively termed neurovascular coupling (NVC), is essential for normal brain function and represents the physiological basis for functional magnetic resonance imaging. Furthermore, deficits in cerebral blood flow (CBF) including functional hyperemia are an early feature of small vessel diseases (SVDs) of the brain and Alzheimer’s long before overt clinical symptoms.

Mark Nelson, Ph.D., from the Larner College of Medicine, University of Vermont

Sleep apnoea linked to changes in the brain

Posted on December 19, 2024 by - Sleep

18 December 2024: People with breathing problems during sleep may have a larger hippocampus, according to a study published in the December 18, 2024, online issue of Neurology®, the medical journal of the American Academy of Neurology. The study, which included mostly Latino people, also found that those with lower oxygen levels during sleep had changes in the deep parts of the brain, the white matter, a common finding of decreased brain health that develops with age.

Sleep disordered breathing is a range of conditions that cause abnormal breathing during sleep including snoring and obstructive sleep apnoea. Obstructive sleep apnoea is when a person stops breathing five or more times per hour. When breathing stops, it can lower oxygen levels, affecting the brain.

“Some studies have found sleep problems and lower oxygen levels during sleep have been linked to brain shrinkage while others have found a link to brain growth,” said study author Alberto R. Ramos, MD, of the University of Miami and Fellow of the American Academy of Neurology. “Both brain shrinkage and brain growth can harm memory and thinking by disrupting normal brain functions, increasing the risk of cognitive decline and dementia. Our study looked at Latino people, who have a higher risk of dementia when compared to non-Latino white people.”

The study involved 2,667 Latino people with an average age of 68.

At the start of the study, each participant was given a take-home sleep test that measured how often they stopped breathing, called apnoeas, and how often they had slow or shallow breathing, called hypopnoeas.

They were divided into three groups: those who had fewer than five sleep disruptions per hour, or no sleep problems; those with five to 15 disruptions, mild sleep problems; and those with more than 15, moderate to severe sleep problems

Researchers also measured oxygen levels in the bloodstream during sleep.

Both brain shrinkage and brain growth can harm memory and thinking by disrupting normal brain functions, increasing the risk of cognitive decline and dementia

Ten years later, participants had brain scans to measure brain volume and white matter hyperintensities, areas in the white matter where the brain tissue has been damaged.

After adjusting for factors such as age, sex, high blood pressure and whether a participant had insurance, they found that people in the group with the most sleep problems had 0.24 cubic centimeters (cm3) greater brain volume in the hippocampus than those with no sleep problems.

They also found that for each additional sleep disruption, there was a 0.006 cm3 increase of brain volume in the hippocampus. Researchers found lower oxygen during sleep was also associated with increased hippocampal volume as well as increased white matter hyperintensities.

“Our findings highlight the complex relationships between sleep health and brain ageing and show there is a need for longer studies that follow people beginning in middle age or earlier,” Ramos said. “A clear understanding of how brain volume is affected by sleep apnoea and other sleep disorders is essential so people can receive early and effective treatment, especially in people who may be at higher risk for dementia.”

A limitation of the study was that it only included Latino adults, so results may not be the same for other populations.

The study was supported by the National Institute on Aging.

Read articles in our sleep series.

Genetic testing for neurodevelopmental conditions

Posted on December 18, 2024 by - Genetics, Neurodevelopmental disorders

More personalised care for children

18 December 2024:Adding genetic testing to the evaluation of paediatric patients with neurodevelopmental disorders (NDD) resulted in more personalised care, including changes in medication, referrals to clinical trials or specialists, and surveillance for potential medical issues, according to a new UCLA Health study.

Children who present with neurodevelopmental differences, such as autism or global developmental delay, have high rates of co-occurring neuropsychiatric conditions, and almost half have an underlying genetic diagnosis. The current practice of relying on primary care doctors to refer patients to specialists can create delays in diagnoses and interventions for children with complex needs. “There was a gap in care for patients with rare genetic, neurodevelopmental, and psychiatric disorders,” said Dr. Julian Martinez, senior co-author of the study and medical geneticist at UCLA Health. “We started the UCLA Care and Research in NeuroGenetics (CARING) Clinic, where a psychiatrist, geneticist, neurologist, and psychologist can evaluate and treat a patient’s wide-ranging symptoms.”

Researchers working at the CARING clinic aimed to study whether genetic testing and subsequent treatment at a multidisciplinary clinic would impact the trajectory of care for children with NDD. They reviewed medical charts from 316 patients treated at the clinic from 2014 to 2019.

The study, published in Genetics in Medicine, found that of the 246 patients who underwent genetic testing, 42% had or were likely to have a diagnosis of a rare genetic condition. This genetic diagnosis led to medication changes in 14% of patients, a clinical trial referral in 3% of patients, and changes in medical surveillance for 30% of patients. The study also found that genetic testing led to specialty referrals in 70% of patients, with cardiac and gastrointestinal issues being the most common.

As we enter an era of more personalised medicine, combining expertise in genetics with specialised psychiatric and neurological care will become increasingly important for providing comprehensive care. We hope our model can help inform how healthcare systems adapt to meet these emerging needs – Dr. Aaron Besterman

“This study shows that taking a genetics-informed approach to neuropsychiatric care can make a meaningful difference for patients with neurodevelopmental disorders,” said Dr. Aaron Besterman, senior author of the study and former UCLA Health Postdoctoral Researcher, who now works at Rady Children’s Institute for Genomic Medicine. “As we enter an era of more personalised medicine, combining expertise in genetics with specialised psychiatric and neurological care will become increasingly important for providing comprehensive care. We hope our model can help inform how healthcare systems adapt to meet these emerging needs.”

With advancements in technology capturing the growing number of rare genetic diseases, Martinez said that the findings justify the need for genetic testing. “Early testing really transforms the care that a patient receives,” he added, noting that genomic medicine should be at the forefront of clinician training.

‘Digital twin’ brain tech for people with Parkinson’s given funding

Posted on December 13, 2024 by - Movement Disorders

£1m research project to advance ‘deep brain stimulation’ set to revolutionise Parkinson’s treatment and management

People with Parkinson’s disease are to benefit from cutting edge AI technology that allows neuroscientists to safely test medication and treatment on a ‘digital twin’ brain, thanks to a £1m funding boost for researchers at Manchester Met.

The project will develop bespoke digital representations of the brain in people with Parkinson’s – a ‘digital twin’ – enabling researchers to trial treatment options risk free and observe how the brain responds before treatments are administered.

Building on previous work funded by the Medical Research Council, the new three-year project will also involve the development of technology to enable people with Parkinson’s to monitor their own brain signals via an app.

This technology is similar to using ‘neurofeedback’ and will empower people with Parkinson’s to understand how factors like sleep, exercise or medication affect their symptoms.

Both strands of the project will advance ongoing research by Manchester Met neuroscientists into ‘deep brain stimulation’ (DBS) for Parkinson’s patients – when a device is placed under the skin of the chest and connected to wires inserted into the brain.

This allows electrodes to deliver high frequency stimulation to the target area, changing some of the signals in the brain that cause symptoms of Parkinson’s.

Following early success using DBS, the researchers hope that longer term investment and industry partnerships will enable both the ‘digital twin’ and neurofeedback tools to be used by the NHS and other healthcare providers to enhance clinical practice and empower patients to manage and monitor their own health. 

Professor of Neuroscience Nicola Ray said: “This work has the potential to revolutionise the treatment landscape for people with Parkinson’s and could transform the lives of those living with the condition.

“My colleague Dr Nelson Trujillo-Baretto, a computational neuroscientist also leading on the project, and I are excited about both complementary strands of this project, which harness our recent advancements into treatment using DBS. By combining cutting edge neuroscience, engineering and industry collaboration we hope to revolutionise the care and treatment of Parkinson’s disease.”

This work has the potential to revolutionise the treatment landscape for people with Parkinson’s and could transform the lives of those living with the condition

Nicola Ray, Professor of Neuroscience

Working alongside the senior research team at Manchester Met, including Professor of Image and Vision Computing Moi Hoon Yap and Professor of Computational Science Liangxiu Han, will be senior clinicians comprising Consultant Neurologist Dr Monty Silverdale at Salford Royal Hospital and Dr Antonella Macerollo at the Walton Centre.

The team will be boosted by the appointment of five new Manchester Met researchers whose roles will include leading the neuroimaging and computational studies, implementing new software platforms and liaising with the patients and public.

This research project is supported by Manchester Met’s Third Century Research Accelerator competition, which was launched this year to mark the University’s 200th year anniversary. The scheme is dedicated to funding impactful, interdisciplinary projects that advance knowledge on global and societal challenges.

Listen to Professor Nicola Ray and Dr Monty Silverdale talking about their research into deep brain stimulation on the University’s MetCast podcast here.

Read more ACNR Movement Disorder articles here.

Accelerated Approval in Huntington’s disease?

Posted on December 12, 2024 by - Huntington's disease, Movement Disorders

uniQure announces alignment with FDA on key elements of Accelerated Approval pathway for AMT-130 in Huntington’s disease

  • U.S Food and Drug Administration (FDA) agrees that data from ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a Biologics License Application (BLA) for Accelerated Approval
  • FDA agrees that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) may serve as an intermediate clinical endpoint for Accelerated Approval

10 December 2024: – uniQure N.V., a gene therapy company advancing therapies for patients with severe medical needs, has announced that the company reached agreement with the U.S Food and Drug Administration (FDA) on key elements of an Accelerated Approval pathway for AMT-130.

“We are very pleased to reach agreement with the FDA on core components of an Accelerated Approval pathway for AMT-130,” said M.D., Chief Medical Officer of uniQure. “Our alignment reflects the strength of our data and collaborative discussions with the staff and senior management at FDA’s Center for Biologics Evaluation and Research (CBER). This is an important milestone for the Huntington’s disease community as it puts us on the most rapid and efficient pathway to deliver a potentially life-changing therapy to people living with this devastating neurodegenerative disorder. We have initiated BLA readiness activities and look forward to further engaging with the FDA in the first half of 2025 to discuss our statistical analysis plan and the technical CMC requirements.”

As part of uniQure’s Regenerative Medicine Advanced Therapy (RMAT) Type B meeting held in late November, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission under the Accelerated Approval pathway, avoiding the need for an additional pre-submission study. The FDA also agreed that cUHDRS may be used as an intermediate clinical endpoint and that reductions in neurofilament light chain (NfL) measured in cerebrospinal fluid (CSF) may serve as supportive evidence of therapeutic benefit in the application for accelerated approval.

The FDA granted uniQure RMAT designation for AMT-130 in May 2024, stating that preliminary clinical data from the ongoing Phase I/II studies indicate AMT-130 has the potential to address unmet medical needs for the treatment of Huntington’s disease. In July 2024, uniQure presented interim data at 24 months showing durable, dose-dependent slowing of disease progression based on the cUHDRS of treated patients compared to a propensity-weighted natural history. These data also showed reductions in CSF NfL, a measure of neurodegeneration, in treated patients at 24 months compared to baseline.

About the Phase I/II Clinical Programme of AMT-130
uniQure is conducting two multi-centre, dose-escalating, Phase I/II clinical studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for the treatment of Huntington’s disease. In the U.S. study, a total of 26 patients with early manifest Huntington’s disease were randomised to treatment (n=6 low dose; n=10 high dose) or an imitation (sham) surgical procedure (n=10). Treated patients received a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of treated patients for five years. An additional four control patients crossed over to treatment.

The European open-label Phase Ib/II study of AMT-130 enrolled 13 patients with early manifest Huntington’s disease (n=6 low dose; n=7 high dose). A third cohort is enrolling an additional 12 patients across sites in the U.S. and EU. This cohort is randomised to explore both doses of AMT-130 in combination with immunosuppression, using the current, established stereotactic administration procedure. Additional details are available on http://www.clinicaltrials.gov (NCT0543017, NCT04120493).

AMT-130 was granted the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation, the first for Huntington’s disease.

Vamorolone for Duchenne Muscular Dystrophy

Posted on December 10, 2024 by - Neuromuscular

10 December 2024: The National Institute for Health and Care Excellence (NICE) has approved vamorolone (Agamree, Santhera) as a treatment option for Duchenne Muscular Dystrophy (DMD) in individuals aged 4 years and over, following a revised pricing agreement with the manufacturer. Despite initial rejection in March due to uncertainties in evidence and cost-effectiveness, further analyses and a revised discount secured its approval. This follows MHRA approval in January.

Approximately 1700 individuals in England with DMD could benefit from this new treatment option. Read the full guidance.

Key Benefits of Vamorolone:

  • Alternative to Corticosteroids: Vamorolone serves as an alternative to traditional corticosteroids like prednisone, which are associated with side effects such as osteoporosis and increased fracture risk.
  • Potentially Safer Profile: Vamorolone’s unique structure reduces adverse effects on growth and bone health, making it a potentially safer option, especially for patients transitioning from other corticosteroids.

The approval is supported by data from the VISION-DMD clinical trial, a phase 2b, double-blind study involving 121 children with DMD aged 4-6 years.

Key findings included:

  • Improved time-to-stand velocity over 24 weeks.
  • Minimal impact on height percentile and bone turnover markers compared to prednisone.

Vamorolone could offer important benefits to patients and their families because of its potential to reduce adverse events associated with current steroid treatments

Helen Knight, Director of Medicines Evaluation at NICE

Episodic migraine in children and adolescents

Posted on December 5, 2024 by - Headache

Teva presents positive efficacy and safety data of AJOVY® (fremanezumab) for the prevention of episodic migraine in children and adolescents from Phase 3 SPACE trial

  • AJOVY® (fremanezumab) significantly reduced monthly migraine days (MMD) and monthly headache days (MHD) versus placebo over a 12-week period in paediatric patients aged 6-17 years [1]
  • Efficacy consistent with fremanezumab pivotal Phase 3 and Real-World Evidence studies in adults with no new emergent safety signals observed
  • Full data presented as a late breaker at European Headache Congress (EHC) 4-7 December in Rotterdam, Netherlands

4 December 2024: Teva Pharmaceutical Industries Ltd presented positive data from its Phase 3 SPACE study evaluating the efficacy and safety of AJOVY® (fremanezumab) for the prevention of episodic migraine in children and adolescent patients aged 6-17 years [1]. The trial showed statistically significant superior efficacy compared to placebo over 12 weeks with a favourable safety profile [1] consistent with that observed in the adult population. 

Migraine is common among children, with an overall estimated prevalence of 7.7 [1]. The prevalence increases from 5% among children aged 5 to 10 years-old to approximately 15% among adolescents [2].  Migraine can cause significant disability in children and adolescents, leading to absence from school, impaired educational performance and missed social activities [2].

This is an important milestone for clinicians and young patients living with episodic migraine who currently have little treatment options available to them. This is the first Phase 3 trial of a CGRP-pathway treatment that has shown statistically superior efficacy with favourable safety and tolerability for the prevention of episodic migraine in children and adolescents.”

Professor Patricia Pozo-Rosich, Headache Unit and Research Group, Head of Section Neurology Department at Vall d’Hebron Hospital and Research Institute, Barcelona, one of the lead investigators

SPACE is a multicentre, double-blind study evaluating the efficacy and safety of fremanezumab in 237 children and adolescents with episodic migraine aged 6-17 years. The paediatric study participants had been diagnosed with migraine for 6 months or more, with a history of less than 14 headache days a month. The trial included subgroup analyses by age (6 -11 years and 12 -17 years) and by sex [1].

Highlights from the SPACE data showed that over 3 months fremanezumab achieved: [1]

  • Significant reduction in monthly migraine days (MMD) vs placebo (-2.5 vs -1.4; p=0.0210)
  • Significant reduction in monthly headache days (MHD) vs placebo (-2.6 vs -1.5; p=0.0172)
  • Significantly higher number of children achieving a 50% response rate vs placebo (47.2% vs 27.0%; p=0.0016)
  • Benefits were similar in both the age subgroups and between boys and girls

Fremanezumab also demonstrated a favourable safety profile, and was well tolerated with no safety signals: [1] 

  • Proportion of children reporting ≥1 adverse events (AEs) was similar between the treatment group vs placebo (55% vs 49%)
  • Proportion of patients with serious adverse events (SAEs) and AEs leading to treatment discontinuation was low at ≤3% and ≤1% respectively

“Over the last 30 years, the incidence of childhood migraine has increased but there has been little innovation in licenced treatments to manage this debilitating condition in children.” said Eric A. Hughes, MD, PhD, Executive Vice President, Global R&D and Chief Medical Officer, Teva Pharmaceuticals. “We’ve already seen the benefits of AJOVYin adultsand the SPACE trial has confirmed that children with episodic migraine can also benefit from AJOVY. This is a significant step forward for the care of migraine in children and adolescents who are having to live with this high burden.”

Teva continues to study the impact of fremanezumab in pediatric patients with chronic migraine and its long-term safety.

References

[1] Hershey, A., et al. Efficacy and Safety of Fremanezumab for the Preventive Treatment of Episodic Migraine in Children and Adolescents: a Phase 3, Randomised, Double-Blind, Placebo-Controlled Study. Presented at European Headache Congress (EHC); 4-7 December 2024, Rotterdam. AL026.

[2] Pediatric Migraine, An Update. Greene, Kaitlin. et al; Neurology clinics, Volume 37, Issue 4, 815-833. August 31, 2019.  https://doi.org/10.1016/j.ncl.2019.07.009

About SPACE

SPACE is a multicentre, randomised, double-blind, placebo-controlled, parallel-group study comparing the efficacy, safety, and tolerability of subcutaneous administration of fremanezumab versus placebo over a 12 week period for the preventive treatment of episodic migraine in 237 paediatric patients aged 6 to 17 years. 

About AJOVY®

AJOVY® is indicated for prophylaxis of migraine in adults who have at least 4 migraine days per month. AJOVY is available as a 225 mg/1.5 mL single dose injection in a pre-filled syringe or, in some countries, in a pre-filled pen. Two dosing options are available: 225 mg once monthly administered as one subcutaneous injection (monthly dosing), or 675 mg every three months (quarterly dosing), which is administered as three subcutaneous injections. AJOVY can be administered either by a healthcare professional or at home by a patient or caregiver. No starting dose is required to begin treatment.