About four months after varoglutamstat failed in Alzheimer’s disease, Vivoryon Therapeutics NV disclosed data from the Viviad phase IIb study with the same compound in kidney disease that provided cause for hope – and Anne Doering, chief financial officer, said the new data “reinforce our strategic shift.”

As the hunt goes on for a better treatment in wet age-related macular degeneration (AMD), landmark analyses of two batches of phase II gene therapy data billed as positive were disclosed during the American Society of Retina Specialists annual meeting in Stockholm, where 4D Molecular Therapeutics Inc. and Adverum Biotechnologies Inc. offered findings.

Two weeks after reporting positive top-line results in first-line head and neck cancer with its lead candidate, eftilagimod alfa, in combination with Merck & Co. Inc.’s Keytruda (pembrolizumab), Immutep Ltd. presented updated data indicating the treatment could offer a non-chemotherapy option for patients with negative PD-L1 expression. Data from cohort B of the Tacti-003 (Keynote-PNC-34) phase IIb trial, which included 31 evaluable patients with PD-L1 expression of less than 1, as measured by Combined Proportion Score, showed a 35.5% objective response rate per RECIST 1.1 assessment.

AI-driven clinical trial design company Opyl Ltd. formed a partnership with L39 Capital Pty Ltd. to establish a $100 million biotech fund that will showcase the predictive power of Opyl’s Trialkey software in selecting successful biotech and pharmaceutical stocks. Melbourne, Australia-based Opyl leverages AI to elevate clinical trial design and forecast outcomes, empowering clinical researchers, biopharma companies and investors to enhance trial design, drug development and market delivery.

Kazia Therapeutics Ltd.'s stock shot up 248% on July 10 after the Sydney-based company reported that paxalisib showed a 3.8-month improvement in overall survival, a 33% improvement, for newly diagnosed unmethylated patients with glioblastoma (GBM) compared to the concurrent standard-of-care arm in the pivotal GBM-Agile phase II/III study.

Nonalcoholic steatohepatitis was renamed, for the first time in 34 years, to metabolic dysfunction associated steatohepatitis (MASH), but a name change is far from being the biggest development in the field, according to experts at Bioplus Interphex (BIX) Korea 2024.

AI-driven clinical trial design company Opyl Ltd. formed a partnership with L39 Capital Pty Ltd. to establish a $100 million biotech fund that will showcase the predictive power of Opyl’s Trialkey software in selecting successful biotech and pharmaceutical stocks. Melbourne, Australia-based Opyl leverages AI to elevate clinical trial design and forecast outcomes, empowering clinical researchers, biopharma companies and investors to enhance trial design, drug development and market delivery.

Interim data from two early stage Friedreich’s ataxia (FA) cardiomyopathy studies from Lexeo Therapeutics Inc. hit the mark by reducing heart muscle thickness, a key cause of death among patients with the rare disease. The results came from the Sunrise-FA phase I/II study and an investigator-initiated phase Ia study of LX-2006, an adeno-associated virus-mediated gene therapy encoding the human frataxin gene. The drug is designed to improve frataxin protein expression to improve mitochondrial cell function.

Two weeks after reporting positive top-line results in first-line head and neck cancer with its lead candidate, eftilagimod alfa, in combination with Merck & Co. Inc.’s Keytruda (pembrolizumab), Immutep Ltd. presented updated data indicating the treatment could offer a non-chemotherapy option for patients with negative PD-L1 expression. Data from cohort B of the Tacti-003 (Keynote-PNC-34) phase IIb trial, which included 31 evaluable patients with PD-L1 expression of less than 1, as measured by Combined Proportion Score, showed a 35.5% objective response rate per RECIST 1.1 assessment.