Pharmaceutical Market Europe • November 2024 • 8-9

NEWS

Eli Lilly shares positive
results for Alzheimer’s drug

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Eli Lilly has shared positive results from a late-stage study of its amyloid plaque-targeting Alzheimer’s disease (AD) drug donanemab in adults with early symptomatic cases of the neurodegenerative disorder.

The phase 3b TRAILBLAZER-ALZ 6 trial has been evaluating the impact of different dosing regimens of donanemab on the rates of amyloid-related imaging abnormalities with oedema/effusion (ARIA-E) and amyloid clearance in adults with early symptomatic AD, including those with mild cognitive impairment and the mild dementia stage of disease.

The data presented at this year’s Clinical Trials on AD Conference showed a reduction of ARIA-E in patients receiving a slightly modified titration of donanemab, with an incidence of 14% compared to 24% for those being treated with the once-monthly standard dosing regimen.

The largest ARIA-E reduction was seen in carriers of apolipoprotein E, a known genetic risk factor for developing AD. In these patients, 19% had ARIA-E on the modified titration compared to 57% on the standard regimen.

Lilly said it is discussing the results of the study with global regulators, with the intent to submit for a potential label update for the drug.


Roche presents two-year data
for SMA treatment Evrysdi

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Roche has presented two-year results from an ongoing study of its spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) in children who were treated pre-symptomatically as infants before six weeks of age.

Affecting approximately one in every 10,000 babies, SMA is a severe and progressive neuromuscular disease characterised by insufficient levels of the SMN protein.

Results from the RAINBOWFISH study presented at this year’s World Muscle Society Congress showed that 100% of Evrysdi-treated patients who had three or more SMN2 copies achieved standing and walking milestones, with most achieving these within World Health Organization windows of typical child development. Among those with two SMN2 copies, 100% could sit and 60% could stand and walk independently after two years of treatment.

Additionally, all patients were able to swallow and feed orally after two years of treatment, with none requiring permanent ventilation, and children in the study showed cognitive skills typical of those without SMA.

Roche leads the clinical development of Evrysdi, which is already approved in more than 100 countries, as part of a collaboration with the SMA Foundation and PTC Therapeutics.


Amgen’s Uplizna shows promise in generalised myasthenia gravis

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Amgen has announced promising top-line results from a phase 3 trial of its B cell-depleting therapy Uplizna (inebilizumab-cdon) in adults with generalised myasthenia gravis (gMG).

The MINT trial has been investigating the efficacy and safety of the drug in both acetylcholine receptor autoantibody-positive and muscle-specific kinase autoantibody-positive gMG patients.

Up to 100,000 people in the US are affected by myasthenia gravis, a rare autoimmune disorder that impairs neuromuscular communication and causes muscle weakness, with gMG accounting for approximately 85% of all cases.

Results from MINT, presented at this year’s American Association of Neuromuscular and Electrodiagnostic Medicine Annual Meeting, demonstrated a statistically significant change from baseline in Myasthenia Gravis Activities of Daily Living score for Uplizna compared with placebo at week 26 in the combined study population, meeting the primary endpoint.

The drug also demonstrated statistically significant and clinically meaningful changes from baseline compared to placebo for key secondary endpoints, including change in Quantitative Myasthenia Gravis score for the combined population at week 26.

Amgen said it is planning to file for US approval of Uplizna in this indication, followed by other key markets.


NHS England begins newborn screening programme

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NHS England has announced that hundreds of babies have begun to be tested for over 200 genetic conditions as part of a study led by Genomics England.

The Generation Study is evaluating the potential of identifying treatable, rare diseases shortly after a baby is born rather than when symptoms might appear later in childhood.

This could enable patients to benefit from earlier diagnosis and treatment that could help slow disease progression.

Up to 100,00 newborns in England will be offered whole genome sequencing using blood samples, usually taken from the umbilical cord shortly after birth.

More than 500 samples have already been collected at 13 NHS hospitals across the country, with plans to scale up to approximately 40 hospitals.

Thousands of children are born with a treatable rare condition in the UK every year, with genetic testing typically taking place when symptoms develop.

The NHS heel prick test is currently used to detect nine rare but serious health conditions in newborns. However, it is hoped that screening a baby’s entire genome could detect hundreds more treatable diseases in the baby’s first years of life.


Researchers target affected cells in motor neurone disease

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Researchers from the Francis Crick Institute and the UCL Queen Square Institute of Neurology have found a way to selectively target cells affected by motor neurone disease (MND).

Published in the journal Science, the study details how the team developed DNA molecules that contain ‘invisibility cloak’ sequences, which can only be removed by diseased cells.

It is hoped that preventing healthy cells from reading the molecules’ messages could lead to safer and more effective gene therapies for MND, which affects up to 5,000 adults in the UK at any one time, as well as other neurodegenerative diseases.

The team’s new approach centres around the activity of the TDP-43 protein, which resides near the DNA in healthy cells and helps them to correctly interpret the DNA’s genetic instructions. However, this protein gets stuck in distant parts of diseased cells, corrupting their interpretation of genetic messages.

Using artificial intelligence prediction tools and careful design, the researchers created DNA sequences that behave in the opposite way – the messages from the DNA are corrupted in healthy cells, but can be interpreted correctly in diseased ones.


BMA GP committee votes to phase out physician associates

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The British Medical Association (BMA) has announced that its general practitioners (GPs) committee for the UK has voted in favour of phasing out physician associates (PAs) in general practice.

An “overwhelming majority” of members voted in favour of the motion, declaring that the role of PAs in general practice is “fundamentally unsafe”.

There should be no new appointments of PAs and the role should be phased out, the motion states, adding that PAs are “inadequately trained” to manage undifferentiated patients and that these sessions should be immediately suspended.

PAs are healthcare professionals with a generalist healthcare education who support doctors in the diagnosis and management of patients. There are currently around 2,000 PAs working in general practice, according to the Royal College of GPs.

The BMA said those in existing PA roles should be given opportunities to retrain and take up other positions in the NHS.

The decision came under a month after the Royal College of GPs’ governing UK council voted to oppose the role of PAs working in general practice. The council also approved guidance to support practices already employing PAs.