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The rising impact of biomarkers in early clinical development

Drug Target Review

Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms. A biomarker is a measurable indicator of a biological process, disease state, or response to a treatment.

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

The Pharma Data

Designed using the same fundamental mechanism as Biogen’s approved SMA treatment SPINRAZA (nusinersen), salanersen represents a next-generation approach aimed at greater potency and less frequent dosing—potentially requiring only once-yearly administration. Treatment-naïve individuals, to evaluate efficacy as a first-line therapy.

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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. It is characterized by early-onset, treatment-resistant seizures, beginning as early as six weeks of age, and profound global neurodevelopmental impairment.

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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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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. MariTide represents a new generation of obesity therapeutics.

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BeOne Medicines Gains CHMP Backing for BRUKINSA Tablet Formulation

The Pharma Data

A Patient-Centered Innovation in B-Cell Cancer Treatment Zanubrutinib, marketed as BRUKINSA, is a next-generation Bruton’s tyrosine kinase (BTK) inhibitor approved for the treatment of various B-cell malignancies.