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The rising impact of biomarkers in early clinical development

Drug Target Review

A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. Diagnostic biomarkers typically confirm or establish a diagnosis and are often used in selecting patient populations for clinical trials.

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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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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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The evolution of AI in drug discovery: learning from history’s mistakes (Part 2)

Drug Target Review

Promising areas for AI implementation When discussing the most transformative AI applications in drug discovery, Sujeegar identifies a particularly ambitious goal: developing AI models that can simulate human pharmacokinetics and pharmacodynamics (PK/PD) using only preliminary laboratory data.

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

The Pharma Data

The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. The full dataset is expected to be presented at an upcoming international scientific conference, which will provide greater clarity on the magnitude of fenfluramine’s clinical benefits.

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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. The MARITIME program includes 72-week chronic weight management trials that will assess safety, efficacy, and tolerability in individuals with obesity or overweight, both with and without T2D.

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Gamcemetinib

New Drug Approvals

Gamcemetinib CAS 1887069-10-4 CC-99677 , OS2IR8TV1O Molecular Weight 469.94 nM) and cell based assays ( EC 50 =89 nM).