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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. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.

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

The Pharma Data

The interim data, presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, California, offer a strong signal of therapeutic potential. Phase 1 Study Overview The Phase 1 clinical program was structured as a single ascending dose study to assess safety, tolerability, and pharmacokinetics of salanersen.

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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 ADA’s annual meeting is one of the most influential forums for showcasing scientific and clinical advancements in diabetes and metabolic disease research.

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

The Pharma Data

In tandem with the Phase 2 results, Amgen also shared detailed data from its Phase 1 Pharmacokinetics Low Dose Initiation (PK-LDI) study. This study explored the effects of beginning treatment with lower starting doses of MariTide to improve tolerability while maintaining clinical benefit.

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

Drug Target Review

G protein-coupled receptors (GPCRs) represent a cornerstone of modern drug discovery due to their crucial role in regulating human physiology and their involvement in numerous diseases. A suitable pharmacokinetics profile can be achieved with the addition of half-life extending moieties to the molecule.

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Therapeutic Oligos 2025 Keynote Speakers Announced

Elrig

It will also cover recent advancements in broadening their application, including innovative approaches to ensure their safety and efficacy, the use of targeted delivery to reach disease-relevant tissues as well as success stories in bringing therapeutic oligonucleotides to the clinic.

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