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Biogen Advances Investigational SMA Therapy to Registrational Trials After Positive Phase 1 Data

The Pharma Data

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

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The rising impact of biomarkers in early clinical development

Drug Target Review

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

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Novo Nordisk: Phase 3 Data Shows Mim8 Well-Tolerated After Switch from Emicizumab in Hemophilia A

The Pharma Data

The study marks a significant step forward in the treatment landscape for haemophilia A, demonstrating not only clinical safety and pharmacokinetic stability, but also strong patient preference for the Mim8 pen-injector delivery system. Throughout the 26-week study period, the investigational therapy was well-tolerated.

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How GPCR agonists, including antibodies, are shaping the future of metabolic care

Drug Target Review

Confo Therapeutics , led by CEO Dr Cedric Ververken, is at the forefront of developing innovative GPCR-targeted therapies using its proprietary ConfoBody ® platform. VHH-based therapies have been clinically-validated many times with multiple products in numerous indications, including for long-term dosing.

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UCB’s GEMZ Phase 3 Trial Shows Promising Results for Fenfluramine in Treating CDKL5 Deficiency Disorder

The Pharma Data

The GEMZ study is a randomized, double-blind, placebo-controlled, fixed-dose, multi-center clinical trial designed to assess the safety, efficacy, and pharmacokinetics of fenfluramine in children and adults aged 1 to 35 years diagnosed with CDD who experience uncontrolled seizures.

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Amgen to Present Phase 2 Maritide Data at ADA 85th Scientific Sessions

The Pharma Data

The presentations will also include new insights from Amgen’s Phase 3 FOURIER study of Repatha® (evolocumab) and the VESALIUS-REAL study exploring real-world lipid management practices. The findings are expected to inform healthcare systems and providers about adherence gaps and optimization strategies in statin and non-statin therapy use.

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Amgen Unveils Phase 2 Obesity Trial Results for Monthly Maritide at ADA’s 85th Scientific Sessions

The Pharma Data

These pivotal findings were unveiled at the 85th Scientific Sessions of the American Diabetes Association (ADA) and were concurrently published in The New England Journal of Medicine , underscoring the significant promise of MariTide in the growing field of anti-obesity therapies.

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