International Top Pharmaceutical Awards

At the European Society of Cardiology (ESC) Congress 2024, results were presented from the SELECT trial, which evaluated the use of semaglutide for the treatment of obesity-related diastolic heart failure (HFpEF). Semaglutide is one of the first highly anticipated glucagon-like peptide-1 receptor agonist (GLP-1RA) therapies. In addition to its application within obesity, type 2 diabetes (T2D), and cardiovascular risk factors, it is also in pre-registration for HFpEF, pre-registration for chronic kidney disease (CKD), Phase III for peripheral artery disease (PAD) and peripheral vascular disease (PVD), and in Phase II for acute ischemic stroke. Semaglutide is a GLP-1RA therapy that offers efficacy and treatment duration advantages within the obesity and T2D market, among others. It is a long-acting injectable therapy that is self-administered subcutaneously once per week. The SELECT trial comprised two arms. The experimental arm involved subjects receiving semaglutide, and startin...

Oesophageal cancer is the 11th most diagnosed cancer and the 7th leading cause of cancer mortality. The disease starts in the oesophagus, the tube that moves food from the throat to the stomach, but symptoms often remain unnoticed until the cancer spreads. The two main types, oesophageal adenocarcinoma, and oesophageal squamous cell carcinoma, develop in different oesophageal regions and are driven by distinct genetic changes. The prevalence of oesophageal cancer Oesophageal cancer is more common in men than women, with global Age Standardised Incidence Rates (ASIR) of 7.6 and 2.6 respectively. The overall five-year survival rate is about 20% but varies from 5% to 50% and improves with early detection. Oesophageal cancer cases are projected to increase significantly, potentially surpassing 923,000 new cases annually by 2050. In 2022, nearly 511,000 new cases and 445,000 deaths were reported globally. Asia recorded the highest incidence in 2022, followed by Europe, North America, and...

In the clinical research industry , our mission is simple: to make the world a healthier place . For treatments to be effective and safe for all patients regardless of race, ethnicity or other factors, the industry is increasingly recognizing that clinical trials must be diverse and inclusive. Despite this growing consensus, most research still lacks adequate representation from minority groups. To foster inclusion, building trust is essential and requires a multifaceted approach across the industry. Why Diversity And Inclusion Matter In Clinical Research According to the National Institutes of Health , people can experience diseases differently based on factors like race, ethnicity, gender, living conditions, life experience, geography and more. Insufficient representation in participant populations and/or data from clinically relevan t populations can lead to inadequate medical product safety and efficacy. Inclusion also means ensuring quality care. Clinical trials can offer high-...

Lung cancer screening (LCS) is a crucial tool in the early detection and treatment of lung cancer. However, according to a new study in the Annals of Internal Medicine , current eligibility criteria may be excluding many individuals who could benefit most from screening. The study proposes a new set of criteria that could identify high-risk individuals more effectively, potentially reducing lung cancer mortality and addressing racial disparities in healthcare. The U.S. Preventive Services Task Force (USPSTF) currently recommends lung cancer screening for adults aged 50 to 80 years with a history of smoking 20 pack-years or more and who currently smoke or have quit within the last 15 years. While these criteria have been instrumental in guiding screening efforts, they are not without limitations. Many individuals who smoked for a long time at lower intensities, those who quit smoking over 15 years ago, and racial and ethnic minorities are disproportionately excluded from LCS, even th...

  I’m very proud – and always have been – of the advances that have been made in medicinal chemistry and drug discovery. Tremendous amounts of work (and tremendous amounts of public and private investment) have led us to all sorts of discoveries that have made a real difference in the lives of sick patients, and I am very glad indeed to have been a part of that effort for the last 35 years (and counting!) But pride shouldn’t make us unwilling to admit those areas where our knowledge thins out and disappears. There are several very large, very important blind spots in drug discovery that I hope can be illuminated. In no particular order, I would list: Where’d it go? Our lack of knowledge about where our compounds go insid e living cells. We know a fair amount about drug distribution and clearance in whole animals and in whole human patients (although still not enough). But intracellular distribution is usually a black box. Cells are tremendously complex spaces, full of distinct...

There’s a new weight loss drug candidate in town, and it’s targeted specifically at obese heart patients. Rivus Pharmaceuticals ’ HU6 led to statistically significan t weight loss in patients with obesity-related heart failure with preserved ejection fraction (HFpEF) in a mid-stage trial (the HuMAIN study). HFpEF patients have symptoms of HF, such as severely reduced exercise capacity, despite normal or near normal LV ejection fraction. This novel drug, Rivus says in its press release, “is designed to promote sustained body fat loss while preserving muscle mass.” HU6 is an oral, once-daily potentially first-in-class investigational treatment , a Controlled Metabolic Accelerator (CMA). Rivus’s CMAs are oral small molecules designed to increase resting metabolic rate, which results in increased consumption of energy, primarily from fat. Rivus says data from the HuMAIN study will be presented in a Late Breaking Clinical Trial Plenary Session at the Heart Failure Society of America (HFSA...

A day after the regulator denied approval of Lykos’ MDMA-assisted PTSD therapy, a medical journal pulled three studies related to the use of the psychedelic in the indication. The medical journal Psychopharmacology on Saturday retracted three papers related to the use of the psychiatric substance MDMA for the treatment of post-traumatic stress disorder. The decision to pull the studies was driven by “protocol violations amounting to unethical conduct” at one of the study sites, according to the journal’s retraction note. The authors of the studies also confirmed to Psychopharmacology that they knew of these breaches when they submitted their manuscript “but did not disclose this information to the journal or remove data generated by this site from their analysis.” Many of the authors failed to fully disclose a “potential competing interest, ” according to the retraction notice, while some of the researchers were affiliated with either the Multidisciplinary Association for Psychedelic...

Sanofi’s Sarclisa (isatuximab) plus standard of care has been shown to extend the time that some multiple myeloma patients remain stable without disease progression. This was demonstrated in a Phase III trial (NCT03617731) with 662 newly diagnosed patients who were eligible for a stem cell transplant. The trial found that adding Sarclisa to the standard drugs – lenalidomide, bortezomib, and dexamethasone (Rvd) – resulted in a statistically significant and clinically meaningful improvement in progression-free survival compared to RVd induction therapy. Sanofi said that these results reinforce the potential of Sarclisa as a backbone therapy when added to the current standard of care in various multiple myeloma patient populations. These recent findings build on data from 2021, where the trial met a different primary endpoint of minimal residual disease (MRD) negativity after induction therapy and before transplant. Around 50.1% of the newly diagnosed multiple myeloma patients achiev...