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FDA's Project Optimus Drives Dose Optimisation in Oncology Trials

FDA's Project Optimus Drives Dose Optimisation in Oncology Trials

The FDA's Project Optimus, launched in 2021, continues to influence oncology trial designs, as discussed at the AACR 2026. The initiative aims to refine dose selection in cancer treatments, moving away from the traditional maximum tolerated dose approach. Instead, it emphasizes selecting doses based on efficacy, safety, and tolerability. This shift is crucial for newer cancer therapies like targeted treatments and immunotherapies, where the effective dose may be lower than the toxicity threshold. The project encourages more structured, comparative evidence generation in early-phase trials, integrating patient-reported outcomes to better reflect patient experiences and improve treatment tolerability.
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Rznomics Secures FDA RMAT Designation for RZ-001 in Hepatocellular Carcinoma Treatment

Rznomics Secures FDA RMAT Designation for RZ-001 in Hepatocellular Carcinoma Treatment

Rznomics Secures FDA RMAT Designation for RZ-001 in Hepatocellular Carcinoma Treatment
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Harbour BioMed Receives FDA Clearance for New Cancer Treatment Trial

Harbour BioMed Receives FDA Clearance for New Cancer Treatment Trial

Harbour BioMed Receives FDA Clearance for New Cancer Treatment Trial
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Epicrispr Biotechnologies Partners with Forge Biologics for Gene Therapy Trials in FSHD

Epicrispr Biotechnologies Partners with Forge Biologics for Gene Therapy Trials in FSHD

Epicrispr Biotechnologies Partners with Forge Biologics for Gene Therapy Trials in FSHD
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Life Sciences Companies Invest in Longevity Research to Extend Healthspan

Life Sciences Companies Invest in Longevity Research to Extend Healthspan

Life Sciences Companies Invest in Longevity Research to Extend Healthspan
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FDA's Project Optimus Drives Dose Optimization in Oncology Trials

FDA's Project Optimus Drives Dose Optimization in Oncology Trials

The FDA's Project Optimus, launched five years ago, remains a focal point in oncology research, as discussed at the AACR 2026 conference. The initiative aims to improve dose selection in early-phase oncology trials by balancing efficacy, safety, and tolerability. Traditional dose-finding methods, which escalate doses until toxicity is intolerable, are being reevaluated in light of new treatment modalities like targeted therapies and immunotherapies. The FDA encourages more structured, comparative evidence generation to support dose selection, emphasizing patient-reported outcomes to better capture tolerability.
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FDA to Reconsider Rejection of Ebvallo Cell Therapy for Rare Disease

FDA to Reconsider Rejection of Ebvallo Cell Therapy for Rare Disease

FDA to Reconsider Rejection of Ebvallo Cell Therapy for Rare Disease
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Altasciences and Certara Partner to Enhance Early Drug Development with Model-Informed Approaches

Altasciences and Certara Partner to Enhance Early Drug Development with Model-Informed Approaches

Altasciences and Certara Partner to Enhance Early Drug Development with Model-Informed Approaches
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Intellia Therapeutics Advances CRISPR Gene Editing with FDA Application for Hereditary Angioedema Treatment

Intellia Therapeutics Advances CRISPR Gene Editing with FDA Application for Hereditary Angioedema Treatment

Intellia Therapeutics Advances CRISPR Gene Editing with FDA Application for Hereditary Angioedema Treatment
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Stoke Therapeutics Reports Positive Data on Zorevunersen for Dravet Syndrome

Stoke Therapeutics Reports Positive Data on Zorevunersen for Dravet Syndrome

Stoke Therapeutics Reports Positive Data on Zorevunersen for Dravet Syndrome
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Harbour BioMed Receives FDA Clearance for HBM7004 to Treat Advanced Solid Tumors

Harbour BioMed Receives FDA Clearance for HBM7004 to Treat Advanced Solid Tumors

Harbour BioMed Receives FDA Clearance for HBM7004 to Treat Advanced Solid Tumors
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Actio Biosciences Initiates Trial for KCNT1-Related Epilepsy Treatment

Actio Biosciences Initiates Trial for KCNT1-Related Epilepsy Treatment

Actio Biosciences has announced the initiation of the KYRON Phase 1b/2 clinical trial for ABS-1230, a treatment for KCNT1-related epilepsy. This rare and severe pediatric epileptic encephalopathy is characterized by treatment-resistant seizures and developmental delays. ABS-1230 is a small molecule inhibitor targeting the genetic cause of the disease. The trial, accepted into the FDA's Rare Disease Evidence Principles process, aims to evaluate the safety, tolerability, and efficacy of ABS-1230 in reducing seizure burden. The study will involve children and young adults aged one month to 21 years.