Pharmaceutical Market Europe • December 2024 • 8-9

NEWS

UCB shares ‘encouraging’ data for Alzheimer’s candidate

Image

UCB has announced “encouraging data” from a phase 2a study of its investigational anti-tau antibody bepranemab in Alzheimer’s disease (AD) patients.

The TOGETHER study has been evaluating two dose levels of the drug in patients with prodromal-to-mild cases of the neurodegenerative disorder.

Results presented at this year’s Clinical Trials on AD meeting showed that the primary endpoint of Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) total score at week 80 was not met.

However, the company highlighted benefits across key secondary endpoints, with bepranemab slowing the rate of tau accumulation versus placebo in key brain regions by 33-58% at week 80, and slowing cognitive decline by 21-25% versus placebo in the same period.

Improvements also were observed in patients with low tau burden at baseline and APOε4 non-carriers.

A post hoc analysis of these two subgroups showed that high-dose bepranemab slowed the rate of tau accumulation versus placebo in key brain regions by 63-67% at week 80, and slowed clinical disease progression by 29%, as measured by the change in CDR-SB, at week 80.


J&J’s Balversa granted MHRA approval in bladder cancer

Image

Johnson & Johnson (J&J) has announced that its pan-FGFR tyrosine kinase inhibitor Balversa (erdafitinib) has been granted marketing authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat a subset of bladder cancer patients.

The drug has been approved as an oral monotherapy for adults with unresectable or metastatic cases of urothelial carcinoma, the most common form of the disease, who are harbouring susceptible FGFR3 genetic alterations.

Eligible patients will also have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor in the unresectable or metastatic treatment setting.

The MHRA’s decision, which came less than three months after the European Commission approved Balversa for the same indication, was based on positive results from cohort 1 of the late-stage THOR trial.

J&J’s drug demonstrated a median overall survival of 12.1 months compared to 7.8 months for chemotherapy. It also showed an improvement in progression-free survival compared to chemotherapy, at 5.6 months versus 2.7 months, and had a confirmed objective response rate of 35.3% compared to 8.5% for chemotherapy.


AstraZeneca and Merck share results for neurofibromatosis drug

Image

AstraZeneca (AZ) and Merck – known as MSD outside the US and Canada – have shared positive results from a late-stage study of Koselugo (selumetinib) in adults with neurofibromatosis type 1 (NF1), a rare genetic condition affecting an estimated 1.7 million people globally.

The phase 3 KOMET trial has been evaluating the oral MEK inhibitor in NF1 patients who have symptomatic, inoperable plexiform neurofibromas (PNs).

In up to 50% of NF1 patients, tumours develop on the nerve sheaths (PNs) and cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.

KOMET met its primary endpoint, with Koselugo demonstrating a statistically significant and clinically meaningful improvement in objective response rate versus placebo. This was defined as the percentage of patients whose PNs disappeared or achieved at least a 20% reduction in tumour volume by cycle 16.

The safety profile of Koselugo was also shown to be consistent with that observed in clinical trials among children and adolescents, with no new safety signals identified. The new data for the drug will now be shared with regulatory authorities.


ICR researchers identify potential blood cancer treatment approach

Image

Researchers led by the Institute of Cancer Research (ICR) have identified a potential new treatment approach to slow down the progression of acute myeloid leukaemia (AML).

The discovery published in the EMBO Journal centres around disrupting the function of a certain protein complex that cancer cells rely on to maintain their abnormal growth.

More than 3,100 people are diagnosed with AML, an aggressive form of blood cancer, in the UK every year. The disease is challenging to treat due to its diverse genetic mutations, rapid progression and resistance to existing therapies.

Experiments in cell cultures conducted by the research team showed that inhibiting BPTF, a specific cancer-driving protein within the nucleosome-remodelling factor (NURF) complex, can impair AML’s ability to flourish.

The NURF complex has previously been studied in other types of cancer but not in AML until recently, while BPTF has already been linked to MYC, another gene well known to promote cancer growth in many other types of cancer.

Ultimately, the results identify the formation of an alternative, previously unknown, NURF complex and suggest it could be a potential drug target in AML.


WHO announces endemic pathogens for urgent vaccine development

Image

A World Health Organization (WHO) study has listed the top endemic pathogens for which new vaccines are “urgently needed”.

The study details the 17 pathogens that regularly cause diseases in communities and represents the first global effort to “systematically prioritise” endemic pathogens based on criteria such as antimicrobial resistance risk, regional disease burden and socioeconomic impact, WHO said.

Alongside reconfirming long-standing priorities for vaccine research and development, including for malaria, the study identifies pathogens such as Group A streptococcus and Klebsiella pneumoniae as top disease control priorities in all regions.

Vaccines for the pathogens are at different stages of development, WHO outlined, with Group A streptococcus, hepatitis C virus, HIV-1 and Klebsiella pneumoniae on the list of those where vaccine research is needed.

The list of pathogens where vaccines need to be further developed includes cytomegalovirus, influenza virus (broadly protective vaccine), Leishmania species, non-typhoidal Salmonella, norovirus, Plasmodium falciparum (malaria), Shigella species and Staphylococcus aureus.

Meanwhile, the pathogens where vaccines are approaching regulatory approval, policy recommendation or introduction are dengue virus, Group B streptococcus, extra-intestinal pathogenic E. coli, Mycobacterium tuberculosis and respiratory syncytial virus.


UK study shows RSV vaccine could reduce antibiotic use

Image

A UK study has demonstrated that interventions to reduce respiratory syncytial virus (RSV) infections could help to reduce antibiotic prescribing and therefore antimicrobial resistance (AMR).

The new analysis from the UK Health Security Agency (UKHSA), Imperial College London and Oxford Population Health found that RSV infections lead to 640,000 antibiotic prescriptions a year.

RSV is characterised by several mild, cold-like symptoms. Although most people can recover within a week or two, it can cause severe illness in certain groups, including young infants and older adults. Despite antibiotics not working for viruses, they are sometimes prescribed when it is not possible to determine if the infection is bacterial or viral.

After examining data from general practice antibiotic prescriptions alongside laboratory-confirmed respiratory infections from 2015 to 2018, the research team found that approximately 2.1% of antibiotic prescriptions in English GPs were attributable to RSV infections, with the largest number prescribed to those aged over 75 years.

The research, funded by the Medical Research Foundation and National Institute for Health and Care Research, follows the launch of a national RSV vaccination programme in September.