Connect with us

News

Interview: The US company appealing Europe’s rejection of daily Alzheimer’s pill

Published

on

Despite having its application for a new, daily Alzheimer’s pill rejected by Europe’s regulators the CEO of US drug company Anavex Life Sciences is appealing this decision.

A Phase IIb/III trial  found over one-third of patients with mild Alzheimer’s – those with a Mini-Mental State Examination (MMSE) score of between 20 and 28 – experienced a slowing of their ‘cognitive decline’.

And, for the majority of the population – those with the common sigma-1 gene – the results were even more dramatic, slowing decline by 49.8%.

Despite this success, in December last year, the European medical authorities rejected an application for the drug – known as Blarcamesine – saying the study ‘failed to demonstrate effectiveness and safety’.

Within days Anavex initiated a challenge to the decision. It has called for a re-examination of the evidence and this is now being undertaken by the European Medicines Agency (EMA).

Speaking to Agetech World Dr Christopher Missling, president and CEO of Anavex, said: “The trial data showed that patients actually improved their quality of life.

“The Alzheimer’s patients had a higher quality of life at the end of the trial than at baseline.”

What Is Blarcamesine?

Unlike other treatments targeting Alzheimer antagonists, such as amyloid-beta or tau pathology, Blarcamesine acts upstream by activating sigma-1 receptors.

This permits the restoration of autophagy – the intracellular recycling and cleaning system which is impaired in pathologies such as Alzheimer’s.

Alzheimer treatments such as Leqembi – which has been approved in over 50 counties and targets amyloid-beta plaques in the brain to slow Alzheimer progression – require regular hospital infusions and carry the risk of brain swelling and bleeding.

Blarcamesine is a pill, taken orally, with no evidence of damaging side effects, such as brain swelling or micro-bleeding, eliminating the need for frequent MRI monitoring required for other drugs.

Dr Missling said: “None of this would be required with Blarcamesine, which is a once-daily, simple oral pill you can ship anywhere.

“The efficacy is also extremely favourable; we see a double or more benefit of cognition and function compared to those injectable antibodies. So, it potentially offers a strong advantage not only in safety and convenience but also in efficacy.”

Why was it rejected by the EU?

A statement from the EMA outlined its position: “In December 2025, EMA’s human medicines committee, the Committee for Medicinal Products for Human Use (CHMP), concluded that the main study failed to demonstrate effectiveness and safety of Blarcamesine Anavex in patients with early Alzheimer’s disease who do not have a mutation in the sigma-1 gene.”

It went on to say that Anavex has requested a ‘re-examination of EMA’s opinion issued on December 11, 2025…(and) the agency will re-examine its opinion and issue a final recommendation’.

Concerns raised by the CHMP focused on trial methodology, possible side effects in the nervous system, and impurities that could potentially cause cancer.

Dr Missling highlighted how a lengthening of the titration process had addressed the mild-dizziness issue and the impurity concerns, which centred on an acceptable threshold for nitrosamines, has also been negated.

And, he highlighted how the amyloid-beta plaque-targeting drugs Leqembi and  Kisunla – which have been fully approved in the US – were eventually approved in Europe after a similar re-examination process .

USA application

Anavex has started a dialogue with the US Food and Drug Administration which has requested access to all of its trial data and if approved in the USA it will open-up the potential for global market authorisation.

The potential size of the market for Blarcamesine is huge, with the number of adults suffering from Alzheimer’s disease expected to grow from around 60 million to 150 million by 2050, as the global population ages.

Dr Missling on how Blarcamesine works

“Blarcamesine activates the Sigma-1 receptor in vivo, which has been confirmed in several peer-reviewed publications and established with a PET study demonstrating dose-dependent activation in the brain.

“The sigma-1 is an integral membrane protein involved in restoring cellular homeostasis. It activates an upstream compensatory process – autophagy – through sigma-1 activation.

“Autophagy gets impaired over time during aging and especially during pathologies like Alzheimer’s and Parkinson’s. This is a very important process, which is nothing else but the recycling of neurons who cannot get rid of their ‘trash’, if you like; they have to recycle it. If this mechanism is impaired, those cells eventually die.

“It stands at the top of many cascades of this complex pathology, for example, on top of A-beta aggregation or Tau aggregation.

“That’s why it’s intriguing to try to approach this from a more comprehensive upstream viewpoint.

Blarcamesine is a small molecule you can take once a day. It restores homeostasis, reactivates impaired autophagy, and lets the body function as it does in a healthy fashion.”

Continue Reading
Click to comment

Leave a Reply

Your email address will not be published. Required fields are marked *

News

Medtronic targets US$784m MiniMed IPO

Published

on

Less than a year after unveiling plans to spin off its diabetes division, Medtronic has confirmed it is seeking to raise US$784 million through an initial public offering of its MiniMed business.

The company intends to offer 28 million ordinary shares at an expected price range of US$25 to US$28 per share.

Underwriters will be granted a 30-day option to purchase up to a further 4.2 million shares at the IPO price.

Once listed, the newly independent company will trade on the Nasdaq under the ticker symbol “MMED”, implying a potential valuation of as much as US$7.86 billion.

Medtronic announced last year that it would separate its US$2.8 billion diabetes unit to create what it described as the only company in the sector to offer a fully integrated insulin management ecosystem.

The business centres on the MiniMed 780G insulin pump, alongside its continuous glucose monitoring systems and smart insulin pen technology.

The diabetes division employs more than 8,000 people and is headquartered in Northridge, California, near Los Angeles.

As the group’s smallest segment by revenue, the spin-off forms part of a wider strategy to streamline Medtronic’s portfolio.

That restructuring has included the formation of kidney care-focused joint venture Mozarc Medical with DaVita in 2023, as well as the company’s exit from the ventilator market the following year.

Continue Reading

News

Stem cell therapy improves frailty mobility

Published

on

A stem cell therapy improved mobility in older adults with age-related frailty after nine months in a phase 2b trial, compared with placebo.

Frailty is a condition in which older people become less able to cope with everyday or sudden stress, leaving them more vulnerable to illness, injury and poor outcomes after surgery.

The study tested laromestrocel, an intravenous therapy derived from donor bone-marrow mesenchymal stem cells.

A total of 148 ambulatory adults with frailty took part, with researchers assessing physical performance and patient-reported outcomes.

Participants receiving the therapy walked further in the six-minute walk test, a standard measure of physical capacity.

After nine months, the treatment group walked an average of 63.4 metres more than those given placebo, a result described as clinically meaningful. At six months, the improvement was 41.3 metres but did not reach statistical significance.

The trial was conducted by Longeveron, a Miami-based clinical stage biotechnology company developing regenerative cell therapies for rare paediatric and chronic age-related conditions.

Joshua M. Hare, chief science officer at Longeveron, said: “We are highly encouraged by these Phase 2b results that demonstrate the potential of stem cell therapy to improve the condition of patients with ageing-related frailty.

“Those with Ageing Frailty are disproportionately compromised in their ability to cope with every day and acute stressors, are at high vulnerability to disease and injury, and are at increased risk for poor outcomes and death after surgery.

“This development area is at the core of Longeveron’s mission advancing stem cell therapies addressing life threatening conditions in the most vulnerable populations children and the elderly.”

Researchers also identified a potential biomarker, meaning a measurable biological indicator, linked to treatment response.

Higher doses of laromestrocel were associated with reductions in soluble TIE-2, a protein involved in blood vessel signalling.

Laromestrocel is being evaluated across several conditions.

The company said the findings point to a possible stem cell therapy approach for managing reduced mobility and other features of age-related frailty.

Continue Reading

News

Agetech investment and innovation round-up

Published

on

Global market to hit US$740bn in 2026, longevity needs lifts? ‘little brain’s’ big role, ageing ethics questioned…and more

IRISH insight and analysis company Research and Markets estimates the global ageing economy will surpass US$740bn this year.

These projections by the company, which is based next to the Guinness Brewery in Dublin, come in its latest paper, entitled: Longevity Market Report 2026-2036.

It takes a holistic view of the ageing economy encompassing consumer wellness, institutional healthcare, technology and regenerative medicine.

In a press release, accompanying the launch Research And Markets, say: “The longevity market is undergoing a structural shift, moving beyond predominantly consumer-driven wellness offerings toward institutionally funded healthcare solutions.

“Insurers, employers, health systems and pharmaceutical companies are increasingly integrating longevity-focused strategies to address the challenges of ageing populations, rising chronic disease burden and long-term cost sustainability.

“This evolution is accelerating demand for integrated platforms that enable early risk identification, targeted prevention and ongoing clinical engagement across the life course.”

Canadian researchers at McGill University say they have found a direct link between age‑related declines in the ‘little brain’ and worsening motor skills.

‘Little brain’ levers

Lead research author Eviatar Fields, a McGill doctoral student in the Integrated Program in Neuroscience, highlights how diminishing neuron activity in the cerebellum – at the base of the skull and known as the little brain – can impact gait, balance and agility.

The research pinpointed how changes in Purkinje cells – a key type of cerebellar neuron – drive this decline and translate into measurable changes in behaviour and physical function.

“By demonstrating how the changes that happen to Purkinje cells in age are causally linked to changes in gait, motor co-ordination and balance, our work provides new avenues for therapies that may prevent or delay motor aging.”

“This provides new hope for extending health span and ultimately improving quality of life and independence in elderly people,” said Mr Fields.

German lift company TK Elevator, is projecting a surge in demand as the global population ages and people find it increasingly difficult to use the stairs.

“As populations age – and that’s happening in Europe, it’s going to happen in China, everywhere else – there’s a need to put in elevators,” said Uday Yadav, its chief executive, speaking to the FT.

Longevity lifts

There are 22 million lifts worldwide, of which 30 per cent are more than 20 years old and potentially ripe to be refitted, he added.

TK Elevator, which was sold by German industrial conglomerate Thyssenkrupp to private equity firms Advent and Cinven for €17.2bn in 2020, is said to be looking at a potential €25bn market listing. Its revenues topped €9bn last year.

Researchers funded by the American Heart Association say the amino acid Taurine increased the life expectancy of mice, and monkeys by up to 25%.

Taurine is one of the most abundant amino acids within our bodies. It is secreted naturally and can be found in foods such as turkey, chicken, shellfish, and dairy.

It has the ability to lower blood pressure, act as an anti-inflammatory agent, and support cardiovascular health, but the concentration within human blood decreases as we age.

As well as longevity, the mice that were fed taurine exhibited improved bone density, muscle mass, pancreas function, and gut health.

Ethical questions

British GP and Medical Director Rammya Mathew has questioned the ethics of longevity highlighting how patients are being charged hefty sums of money ‘for investigations that are often unnecessary, of uncertain benefit, or unsupported by robust evidence’.

She added: “This is framed as empowering patients with knowledge, but it risks crossing the line into over-medicalisation of healthy people.”

The article published in the British Medical Journal continues: “I have watched this field with growing interest, particularly as an increasing number of high profile clinicians, some of whom have held senior roles in the NHS, move into private longevity medicine.

“Practising privately is not unethical in itself. But it does place doctors in an environment where the evidence base is often less clear, commercial pressures are more explicit, and the temptation to conflate innovation with benefit is real.”

Levels of the Nicotinamide Adenine Dinucleotide (NAD+) – a vital coenzyme found in every human cell – are the target of new research by the Nestlé Institute of Health Sciences.

The research published in the Nature Metabolism Journal indicates that certain NAD+ precursors can boost cellular energy levels and influence gut microbiome activity.

The study discovered that NAD+ precursors – nicotinamide riboside (NR), nicotinamide mononucleotide (NMN) increased circulating NAD+ concentrations.

NAD+ levels decline with age in multiple tissues – muscle, liver, brain and skin – by as much 65% from young adulthood to old age.

This contributes to hallmarks of aging like mitochondrial dysfunction, reduced energy production, impaired DNA repair, increased inflammation, and cellular senescence.

Ongoing research has shown that restoring NAD+ (via precursors) improves mitochondrial function, metabolic health, and resilience.

Continue Reading

Trending

Agetech World