Pharmaceutical Market Europe • October 2022 • 8-9

NEWS

AstraZeneca’s Farxiga data shows ‘significant’ mortality benefits in heart failure patients

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AstraZeneca (AZ) has announced new data from two phase 3 trials showing Farxiga (dapagliflozin) ‘significantly lowers’ the risk of cardiovascular (CV) death in patients with heart failure, potentially leading to a significant increase in patients who could benefit from the SGLT2 inhibitor.

The reduction in risk of CV death was consistent across pre-specified subgroups and is the first analysis to demonstrate a mortality benefit with a heart failure medication in patients with heart failure across the left ventricular ejection fraction (LVEF) range.

The pre-specified, patient-level, pooled analysis from the phase 3 DAPA-HF and DELIVER trials showed the SGLT2 inhibitor reduced the risk of CV death by 14% over the median follow-up of 22 months, death from any cause by 10% and hospitalisation for heart failure by 29%.

Moreover, the data showed Farxiga reduced the composite of death from CV causes, myocardial infarction, or stroke by 10% in patients with heart failure, irrespective of LVEF.

Of the estimated 64 million heart failure patients, roughly half have reduced ejection fraction – the measurement of the heart’s ability to pump oxygen-rich blood into the body – which is equal to or less than 40%. The remainder have mildly reduced or preserved ejection fraction.


AbbVie’s Imbruvica gets FDA approval for paediatric patients with chronic graft-versus-host disease

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AbbVie’s Imbruvica (ibrutinib) has been approved by the US Food and Drug Administration (FDA) for the treatment of paediatric patients one year and older with chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy, the company announced.

cGVHD occurs when donated peripheral blood or bone marrow stem cells view the recipient’s body as foreign and the donated cells launch an immune attack on the body, having major impacts on major organs, including the skin, eyes, mouth and liver.

Around 35% of the estimated 8,000 patients who undergo life-saving allogeneic haematopoietic stem cell transplant (HSCT) per year develop cGVHD that requires systemic treatment, AbbVie reported.

Imbruvia is a once-daily oral medication jointly developed and commercialised by Janssen Biotech and Pharmacyclics.

The application was supported by the iMAGINE phase 1/2 study, an open-label, multicentre, single-arm trial of Imbruvica for the treatment of paediatric patients aged from one years to less than 22 years with moderate or severe cGVHD.

The study demonstrated an Overall Response Rate (ORR) through week 25 of 60% in patients with a median age of 13 years with relapsed/refractory (R/R) moderate to severe cGVHD. Moreover, no new safety signals were observed.

The approval is the company’s first paediatric indication for Imbruvica, and marks the first approved treatment option for children under 12 years with cGVHD.


Pfizer announces positive phase 3 results of RSV vaccine candidate in older adults

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Pfizer has announced positive topline results from a phase 3 clinical trial investigating its bivalent respiratory syncytial virus (RSV) candidate, RSVpreF, to treat adults aged 60 years and older.

There is currently no US approved vaccine for RSV, a contagious virus characterised by several mild, cold-like symptoms.

The phase 3 RENOIR trial is a global, randomised, double-blind, placebo-controlled study designed to assess the efficacy, immunogenicity and safety of a single dose of RSVpreF in adults 60 years old and over.

The study showed the vaccine was 85.7% effective among participants with three or more symptoms, and 66.7% for two or more symptoms, according to an interim analysis carried out by an external data monitoring panel. Moreover, the vaccine was well tolerated and no safety concerns were observed.

Based on these results, Pfizer plans to submit a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for RSVpreF and to prepare submissions for other regulatory authorities in the coming months.

In March 2022, Pfizer announced RSVpreF received Breakthrough Therapy Designation from the FDA for the prevention of RSV-associated lower respiratory tract disease caused by RSV in infants from birth up to six months old by active immunisation of pregnant women.


Manus Bio awarded $2m by Bill & Melinda Gates Foundation to combat malaria

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Manus Bio has received a fourth award of $2m from the Bill & Melinda Gates Foundation to accelerate the development of a scalable and cost-effective production route for artemisinin, a key therapeutic for treating malaria.

The $2m funding will enable the natural products biomanufacturer to begin scaling up the unique biological process it has developed towards the artemisinin precursor, dihydroartemisinic acid, to enable process simplification for large-scale, commercial manufacturing of artemisinin.

Malaria remains one of the world’s biggest public health issues, with the World Health Organization (WHO) reporting an estimated 241 million cases of malaria in 2020, and estimated 627,000 malaria deaths. It is, however, curable and preventable.

WHO recommends artemisinin or one of its derivatives formulated in combination therapies as frontline treatments for all cases of malaria, particularly for Plasmodium falciparum malaria – the most deadly malaria parasite globally and the most prevalent in Africa.

However, availability of the plant from which it is traditionally extracted – the Artemisia annua plant or ‘sweet wormwood’ – is subject to agricultural instabilities and vulnerabilities.

Manus states that dihydroartemisinic acid can be produced using fermentation, which provides a more stable and sustainable source for making artemisinin than through agricultural extraction.

The company has already applied its BioAssemblyLine cell engineering platform to develop a fermentation-based approach for artemisinin production through the support of the foundation.

In a landmark move in the fight against malaria, GSK was awarded a contract by UNICEF for the first-ever supply of a malaria vaccine this year, with a value of up to £170m.

The award will lead to 18 million doses of RTS,S/AS01 being available over the next three years, potentially saving thousands of lives every year, UNICEF reported.


Alzheimer’s Research UK calls for urgent action to prioritise investment in last-stage dementia research

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Alzheimer’s Research UK has called for urgent action to prioritise investment in last-stage dementia research after official figures revealed ‘shockingly low’ participation in trials compared to other major health conditions.

The charity outlined that only 61 participants were recruited to late-stage dementia drug trials supported by the National Institute for Health and Care Research (NIHR) Clinical Research Network in England last year – around 100 times fewer than for cancer, and around ten times fewer than for stroke or coronary heart disease.

This is in spite of the increasing number of potential dementia treatments in trials across the world, with twice as many disease-modifying treatments for dementia being studied in phase 3 trials than in 2014, and more than two-thirds of people in the UK reporting a willingness to participate in dementia research, the charity reported.

Releasing the figures to coincide with World Alzheimer’s Month, Alzheimer’s Research UK claims that there is a ‘unique opportunity’ for the new Prime Minister to ‘join up’ clinical research and dementia diagnoses in the NHS.

The charity is now calling on the new Prime Minister to take five key steps to improve the speed of finding treatments that will benefit people living with dementia in the UK, including to keep dementia research a priority, enable earlier and more accurate diagnosis in the NHS and improve participation and diversity in dementia research.


BHF announces drug combination could delay need for high-risk surgery in patients with Marfan syndrome

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The combination of beta-blockers and angiotensin receptor blockers can delay the need for surgery to correct potentially deadly aortic enlargement in people with Marfan syndrome, according to research funded by the British Heart Foundation (BHF).

Marfan syndrome affects the connective tissues responsible for holding the body’s cells in place, causing patients to have weak blood vessels. One of the most serious complications is weakness of the aorta, which can expand over time, increasing the risk that it will tear or burst. If this happens, immediate, life-saving surgery is required.

The Marfan Treatment Trialists’ (MTT) Collaboration analysed the results of seven previous clinical trials involving 1,442 people with no prior aortic surgery, aiming to understand more about the effects of two medications.

The MTT found that individually, both medicines helped to slow the growth of the aorta, but a combination of the two was more effective than when they were used alone.

Specifically, the combination therapy may reduce the rate of change by at least half and potentially by much more than this, which, if maintained over a sustained period, would be expected to delay the need for surgery substantially, the researchers said.

The findings were presented at the European Society of Cardiology Congress in Barcelona and published in The Lancet.