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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 goal is to boost SMN protein production more effectively and offer a more convenient, once-a-year dosing schedule , thereby improving long-term disease management and patient compliance. These reductions were sustained through one year , highlighting salanersen’s potential to meaningfully slow disease progression. Source link

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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. Safety Profile and Tolerability: No Surprises, GI Events Mostly Mild In terms of safety, the Phase 2 study did not identify any new safety signals.

Trials 40
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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.

Trials 40
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Therapeutic Oligos 2025 Keynote Speakers Announced

Elrig

Prof Rory Johnson, Associate Professor, University College Dublin, and Dr Shalini Andersson, Vice President Nucleic Acid Therapeutics, AstraZeneca will lead this years event focussed on drugging the undruggable.

RNA 59
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The future of mental health treatment: Zelquistinel’s role

Drug Target Review

By adjusting the activity of this receptor, these drugs can influence brain functions associated with mood, cognition and pain, positioning them as potential treatments for conditions like depression, cognitive disorders and neurological diseases. The drug’s pharmacokinetics (PK) and pharmacodynamics (PD) are closely linked.

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Navigating Regulatory Hurdles in Drug Development

DrugBank

Regulatory agencies require pharmaceutical companies to submit preclinical and clinical trial data covering toxicology, pharmacokinetics, pharmacodynamics, and long-term safety monitoring. These submissions contain comprehensive data on clinical efficacy, pharmacokinetics, pharmacodynamics, and risk-benefit assessments.