In an indication that has proved difficult for biopharma to conquer, Regulus Therapeutics Inc. disclosed further positive data from its ongoing phase Ib study testing RGLS-8429 in autosomal dominant polycystic kidney disease and laid out its plans to move straight into a phase III trial later this year, with the potential for an accelerated U.S. approval.

With positive results from a phase III study, ITM Isotope Technologies Munich SE said it has reached a landmark. The clinical trial of ITM-11, a synthetic, targeted radiotherapeutic agent for treating inoperable, progressive grade 1 or grade 2 gastroenteropancreatic neuroendocrine tumors, met its primary endpoint of prolonging progression-free survival when compared to the mTOR inhibitor everolimus, a targeted molecular therapy and a standard of care.

A new bioprinting platform to create tissues that can change shape as a result of forces generated by the cells, similar to what happens naturally during organ development, was developed by researchers from the University of Galway, Ireland.

What a difference 60 weeks can make. That’s the lesson Akero Therapeutics Inc. shared with the rollout of what executives called “unprecedented” data from the phase IIb Symmetry trial testing efruxifermin (EFX), its FGF21 receptor agonist, in patients with compensated cirrhosis due to metabolic dysfunction-associated steatohepatitis (MASH). While earlier findings had shown only a trend in improvement at 36 weeks, full 96-week results showed more than doubling of earlier effect size, hitting the primary endpoint and sending shares of Akero (NASDAQ:AKRO) up 97% to close Jan. 27 at $51.71.

In December 2024 BioWorld recorded 212 clinical trial updates, an increase from 180 in November and closely aligning with 219 in October. The month saw 16 successful phase III outcomes, along with two mixed results and two trial failures. Eli Lilly and Co. reported that its FDA-approved weight-loss drug Zepbound (tirzepatide) outperformed Novo Nordisk A/S’s Wegovy (semaglutide) in the phase IIIb Surmount-5 trial.

As investors look ahead to data shortly from Veru Inc. with enobosarm, the weight-loss space remains hot, with Novo Nordisk A/S reporting favorable top-line results from a phase Ib/IIa trial evaluating amycretin, a unimolecular GLP-1 and amylin receptor agonist for once weekly subcutaneous administration in people overweight or with obesity.

Accunea Ltd. is looking to reduce the number of kidneys that are discarded with its bioanalysis technology Renosure, which can detect critical changes in organ function in real-time, both before and after transplantation. The company is looking to raise £1.6 million (US$2 million) to develop the device which could transform the lives of people requiring a kidney transplant.

George Medicines, a new spinout from Australia’s George Institute for Global Health, could offer patients better control of their blood pressure as well as fewer side effects thanks to an ultra-low-dose triple combination. A polypill that combines multiple medicines into a single tablet, GMRx2 was developed out of a 20-year research program at The George Institute for Global Health. The single pill is a combination of three best-in-class medicines: telmisartan, amlodipine and indapamide.