University of Dundee

FDA Grants Accelerated Approval to Ipsen's Iqirvo, First Dual PPAR Agonist for Primary Biliary Cholangitis

3 months ago

• Ipsen's Iqirvo (elafibranor) receives FDA accelerated approval as the first dual PPAR alpha/delta agonist and first new therapy in over a decade for primary biliary cholangitis treatment. • In the ELATIVE trial, Iqirvo demonstrated significant efficacy with 51% of patients achieving cholestasis response compared to 4% on placebo, marking a substantial therapeutic advancement. • The drug will be available at $11,500 per month list price, entering a market estimated at $1.5 billion annually for UDCA-refractory PBC treatments.

Revolutionizing Drug Development: The Rise of Targeted Protein Degradation

4 months ago

Targeted Protein Degradation (TPD) represents a groundbreaking shift in pharmaceutical research, offering a novel approach to drug development by leveraging the body's natural protein degradation system. This method, which involves tagging disease-causing proteins for destruction by the proteasome, has shown promising results in clinical trials, particularly in cancer treatment. With over 36 drugs currently in clinical testing, TPD is paving the way for treatments that were previously unattainable, marking a significant advancement in the fight against complex diseases.

Scottish Patient Pioneers New Cancer Treatment in Europe

5 months ago

A Scottish woman has become the first in Europe to receive a groundbreaking personalised cancer vaccine, marking a significant step forward in cancer treatment. The vaccine, developed by Moderna and MSD, uses mRNA technology to target cancer cells specifically.

Clinical Trial Reporting Rates on the Rise Globally, but Gaps Persist

7 months ago

• Reporting rates for clinical trials are improving in the EU and the US, driven by stricter regulations and increased awareness. • Compliance varies significantly between countries, with Italy, the Netherlands, Spain, and France lagging in reporting results, especially for publicly funded trials. • Initiatives like the EU Trials Tracker and advocacy efforts are increasing transparency, but challenges remain in ensuring complete and accurate reporting. • Experts emphasize the ethical duty of scientists and funders to publish all trial information, regardless of outcome, for the benefit of patients and advancement of medical knowledge.

Novel PROTAC Degrader ACBI3 Shows Efficacy Against Multiple KRAS Mutant Cancers

8 months ago

• A newly developed small molecule, ACBI3, has demonstrated efficacy against 13 of the 17 most common KRAS mutant-driven cancers, offering a potential treatment option for a broader range of patients. • ACBI3, a PROTAC-based degrader, works by tagging the mutant KRAS proteins for degradation, offering a novel approach compared to traditional KRAS inhibitors that only target the G12C mutation. • Preclinical studies in mouse models have shown that ACBI3 induces effective tumor regression, validating KRAS degradation as a promising therapeutic strategy. • Boehringer Ingelheim has made ACBI3 available to the global research community through its opnMe portal to accelerate innovation in cancer research.

Inhaled Colistimethate Sodium Shows Efficacy in Non-Cystic Fibrosis Bronchiectasis

8 months ago

• Phase 3 PROMIS trials demonstrate that inhaled colistimethate sodium (CMS I-neb) significantly reduces pulmonary exacerbations in NCFB patients colonized with _P. aeruginosa_. • The PROMIS-I trial achieved a 39% reduction in annual pulmonary exacerbation rate compared to placebo (p=0.0010), with a 59% reduction in severe exacerbations. • CMS I-neb treatment led to clinically important improvements in quality of life and a reduction in _P. aeruginosa_ density among NCFB patients. • The treatment was generally well-tolerated, with bronchospasm occurring in fewer than 5% of patients, offering a potential new option for this underserved population.