Pharmaceutical Market Europe • January 2025 • 8-9
NEWS
AbbVie’s Parkinson’s drug shows promise in phase 3 trial
AbbVie has shared promising top-line results from a phase 3 study of its investigational Parkinson’s disease (PD) candidate tavapadon in adults and said it is “on track” to submit a new drug application to the US Food and Drug Administration in 2025.
The TEMPO-2 trial evaluated the D1/D5 dopamine receptor partial agonist as a flexible-dose monotherapy in patients with early stages of the neurodegenerative disorder.
More than ten million people worldwide are living with PD, which is characterised by symptoms such as tremor, muscle rigidity, slowness of movement and difficulty with balance.
TEMPO-2 met its primary endpoint, with tavapadon-treated patients experiencing a statistically significant improvement from baseline in the Movement Disorder Society – Unified Parkinson’s Disease Rating Scale parts two and three combined score compared to those receiving placebo at week 26.
The study’s key secondary endpoint was also met, demonstrating a statistically significant and clinically meaningful improvement in motor aspects of experiences of daily living in the tavapadon group compared to placebo at week 26, and the drug’s safety profile was consistent with prior clinical trials.
Regeneron shares phase 3 data for rare blood disorder
Regeneron Pharmaceuticals has shared positive data from a phase 3 trial of pozelimab plus cemdisiran (poze-cemdi) in patients with the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH).
Results from an exploratory cohort of the ACCESS-1 trial were presented at the American Society of Hematology annual meeting and support the continued development of the combination treatment in PNH and other complement-mediated diseases, Regeneron said.
Affecting up to 1.5 people per million in the US, PNH is caused by a genetic mutation that results in the body’s complement system attacking its red blood cells.
Data showed that 96% of poze-cemdi-treated patients achieved adequate lactate dehydrogenase (LDH), a biomarker used to measure the degree of haemolysis, control across study visits (weeks eight to 26) on average, compared to 80% of those receiving AstraZeneca’s C5 inhibitor ravulizumab, marketed under the brand name Ultomiris.
Of the patients in the poze-cemdi group, 93% achieved LDH normalisation across study visits on average, compared to 65% of those in the ravulizumab arm, and an 84% decrease in LDH from baseline was observed for Regeneron’s combination compared to 74% with ravulizumab.
Sanofi’s MS candidate given FDA breakthrough designation
Sanofi’s investigational Bruton’s tyrosine kinase (BTK) inhibitor tolebrutinib has been given breakthrough therapy designation by the US Food and Drug Administration (FDA) as a treatment for adults with non-relapsing secondary progressive multiple sclerosis (MS).
The decision was supported by positive results from the late-stage HERCULES study, in which the drug delayed the time to onset of six-month confirmed disability progression by 31% compared to placebo.
The number of patients in the trial who experienced confirmed disability improvement also increased in the tolebrutinib arm, at 10% versus 5% in the placebo cohort.
Affecting approximately 2.9 million people worldwide, MS is a chronic condition that affects nerves in the central nervous system.
Sanofi’s tolebrutinib is designed to block the action of the BTK enzyme, which is essential for the survival and activation of a type of white blood cell thought to be involved in the inflammation caused by MS.
The drug is being assessed in various forms of the disease and is now the first and only brain-penetrant BTK inhibitor in MS to be designated breakthrough therapy by the FDA.
ICR study reveals how drug resistance develops in prostate cancer
Researchers from the Institute of Cancer Research (ICR) have found a way to predict how long prostate cancer patients will respond to the PARP inhibitor olaparib.
The research published in the journal Cancer Cell could help doctors personalise treatments and pave the way for the development of drugs that prevent resistance.
AstraZeneca and Merck & Co’s – known as MSD outside the US and Canada – olaparib already holds approvals to treat certain cases of prostate cancer under the brand
name Lynparza.
Funded by AstraZeneca, Prostate Cancer UK, Movember, Cancer Research UK and Prostate Cancer Foundation, the researchers analysed blood samples from 25 patients with advanced prostate cancer who took part in the phase 2 TOPARP-B trial and had all initially responded well to olaparib.
They were able to see that patients with a high number of certain DNA changes called reversion mutations had an average survival time of 13.9 months, compared to 21.4 months for those with a lower number of these changes.
According to the ICR, this is the first research to study the link between reversion mutations and clinical outcomes for prostate cancer patients.
Novo Nordisk to establish $1.2bn rare disease production site
Novo Nordisk has announced an investment of 8.5bn Danish kroner, or approximately $1.2bn, to establish a new rare disease production facility in Odense, Denmark.
Construction on the new site, which will include a production facility and warehouse, has begun and is scheduled to be completed in 2027, with the investment expected to eventually create 400 permanent jobs.
The modular and flexible facility will be specifically designed to accommodate multiple product types within rare diseases, including drugs for the inherited blood disorder haemophilia.
Henrik Wulff, executive vice president, product supply, quality and IT of Novo Nordisk, said: “The facility will utilise advanced technology and innovative equipment to ensure the highest quality to patients and meet the growing global demand for our… medicines.”
The announcement came just two weeks after Novo unveiled plans to invest 2.9bn Danish kroner, approximately $407m, to establish a quality control laboratory in Denmark.
The laboratory in Hillerød, which is also expected to be completed in 2027, is set to serve as a central hub for quality control operations in the country.
Eli Lilly announces $3bn expansion to US injectables facility
Eli Lilly has announced a $3bn expansion to the US manufacturing facility that it acquired from Nexus Pharmaceuticals last year.
The investment in Kenosha County, Wisconsin is aimed at extending the company’s global injectable product manufacturing network to help it meet the growing demand for its diabetes and obesity medicines, as well as future pipeline drugs across multiple therapeutic areas.
The expanded facility, which brings Lilly’s total planned investment in Wisconsin to $4bn, will focus on manufacturing injectable medicines, device assembly and packaging.
Lilly said it will use advanced automation, including guided vehicles, robotics and production equipment, throughout the site to accelerate processes and increase accuracy, which the company said will allow employees to “focus on making safe, high-quality medicines”.
Construction on the expansion is expected to begin this year and, once complete, the facility is set to add 750 highly skilled jobs to the current 100-plus workforce at the Wisconsin location.
The commitment represents Lilly’s single largest US manufacturing investment outside its home state of Indiana, explained Edgardo Hernandez, executive vice president and president of Lilly manufacturing operations.
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