Pharmaceutical Market Europe • April 2025 • 8-9
NEWS
Takeda/Protagonist’s rusfertide shows promise in rare blood cancer
Takeda and Protagonist Therapeutics have shared positive top-line results from a phase 3 trial of rusfertide in the rare blood cancer polycythaemia vera (PV).
The ongoing VERIFY study has been evaluating rusfertide against placebo as an add-on to standard-of-care treatment in phlebotomy-dependent PV patients.
As many as 160,000 people in the US are affected by PV, which causes the body to produce too many red blood cells and increases the risk of life-threatening cardiovascular and thrombotic events.
Rusfertide, which Takeda gained rights to last year, is thought to regulate iron homeostasis and control the absorption, storage and distribution of iron in the body.
VERIFY met its primary endpoint, with 77% of rusfertide-treated patients achieving a clinical response, defined as the absence of phlebotomy eligibility, compared to 33% of those receiving placebo during weeks 20 to 32.
The trial also met its first key secondary endpoint, which is the pre-specified primary endpoint for EU regulators, with a mean of 0.5 phlebotomies per patient in the rusfertide arm compared to 1.8 phlebotomies per patient in the placebo group during weeks zero to 32.
AbbVie shares late-stage results for Elahere in ovarian cancer
AbbVie’s Elahere (mirvetuximab soravtansine-gynx) has demonstrated consistent survival benefits in a subset of ovarian cancer patients, according to new results from a confirmatory late-stage trial of the drug.
The phase 3 MIRASOL study compared the efficacy and safety of Elahere against chemotherapy in women with folate receptor alpha-positive platinum-resistant ovarian cancer.
Ovarian cancer is the leading cause of death from gynaecological cancers in the US, with approximately 20,000 cases diagnosed every year and the majority of patients presenting with late-stage disease.
The final MIRASOL data analysis comes after the European Commission and US Food and Drug Administration approved the drug in November and March last year, respectively.
At a median follow-up of 30.5 months, median progression-free survival was 5.59 months for Elahere and 3.98 months for investigator’s choice chemotherapy.
Elahere was also associated with a higher objective response rate compared to chemotherapy, at 41.9% versus 15.9%, and demonstrated a 32% reduction in the risk of death compared to chemotherapy.
Other endpoints included safety and duration of response, which AbbVie said were consistent with the trial’s primary data analysis.
Pfizer/Arvinas’ phase 3 results for breast cancer treatment
Pfizer and Arvinas have shared promising top-line results from a late-stage study of their investigational protein degrader vepdegestrant in a subset of breast cancer patients.
The phase 3 VERITAC-2 trial has been evaluating the candidate against AstraZeneca’s endocrine therapy Faslodex (fulvestrant) in adults with oestrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer.
Patients enrolled to the trial had also experienced disease progression following prior treatment with cyclin-dependent kinase 4/6 inhibitors and endocrine therapy.
It is estimated that nearly 320,000 people will be diagnosed with breast cancer in the US this year, with ER-positive/HER2-negative disease accounting for approximately 70% of all cases.
VERITAC-2 met its primary endpoint in the cohort of patients with oestrogen receptor mutations, a common cause of acquired resistance, with vepdegestrant demonstrating a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to Faslodex. Statistical significance in PFS improvement was not reached in the intent-to-treat population.
Overall survival was immature at the time of the analysis and will continue to be assessed as a key secondary endpoint, the companies said.
UK government unveils plans to abolish NHS England
On 13 March, UK prime minister Sir Keir Starmer unveiled plans to bring management of the NHS “back into democratic control” by abolishing NHS England.
Sir Keir said the move will return the NHS “to the heart of government” and lead to a number of benefits, including cutting waiting times and freeing up money for front-line services.
NHS England was established in 2012 by the then-Conservative health secretary Andrew Lansley and formally came into being in April 2013.
It was set up as a quango, an organisation that operates independently from central government but is funded by public money, to help agree funding and priorities for the NHS, oversee the delivery of services, manage the NHS workforce and negotiate deals.
It is hoped that returning its functions to the Department of Health and Social Care will strip back layers of red tape and help deliver significant savings.
Sir James Mackey, newly appointed NHS England chief executive, will lead the transformation team, saying: “We [will] look to implement the three big shifts – analogue to digital, sickness to prevention and hospital to community – [to] build an NHS fit for the future.”
Boehringer launches CKD initiative to tackle health inequalities
This World Kidney Day (13 March 2025), Boehringer Ingelheim (BI) announced a new initiative aimed at detecting, diagnosing and treating chronic kidney disease (CKD) faster.
The SPOT CKD (screening, prevention, outreach and treatment for health equity) initiative will seek to reduce health inequalities by improving early diagnosis and providing proactive care to slow the progression of CKD in underserved communities.
It will run concurrently through collaborative working projects between BI with NHS Hampshire and Isle of Wight, and Health Innovation North East and North Cumbria working with North East and North Cumbria Integrated Care Board.
Approximately 10% of the UK population is affected by CKD, a progressive condition caused by decreased kidney function.
Research by Kidney Research UK has shown that people from lower socioeconomic groups are at a higher risk of developing CKD, and individuals from Black, Asian and minority ethnic populations are more likely to progress faster towards kidney failure.
SPOT CKD will support those at a higher risk of the disease and communities where accessing the right care can be more challenging due to socioeconomic disparities and limited resources.
Researchers discover unidentified genes linked to rare diseases
A study led by Queen Mary University of London has found 69 previously unidentified genetic determinants of rare diseases.
The research published in the journal Nature could improve rare disease diagnosis rates and pave the way for new treatments.
As many as 80% of rare disease patients remain undiagnosed after genomic sequencing due to the lack of evidence on disease-causing genetic variations.
The international team, which involved UCL researchers, developed an analytical framework to identify the causes of Mendelian diseases (genetic disorders caused by a single gene mutation) through rare variant gene burden analysis.
This was then applied to the genetic data of 34,851 patients and their family members recruited to Genomics England’s 100,000 Genomes Project.
The team discovered a total of 141 new associations, and prioritised 69 genes that were not previously linked to rare diseases. In 30 of these cases, the new genetic findings were supported by existing experimental evidence.
The strongest overall genetic and experimental evidence supported the newly discovered genetic variants for rare forms of diabetes, epilepsy, schizophrenia and Charcot-Marie-Tooth disease, as well as anterior segment ocular abnormalities.
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