Clinical experience with the AKT1 inhibitor miransertib in two children with PIK3CA-related overgrowth syndrome
The clinical and radiographic symptoms of Proteus syndrome are highly variable, as are its orthopaedic manifestations.
IAG, Image Analysis Group worked alongside Arqule (acq. by Merck) and outstanding academics led by Dr. Irvine to support the development of novel imaging-based system for assessment of the drug efficacy.
Its implementation in our platform DYNAMIKA and subsequent application in the trial led to clearer definitions of the radiographic changes in these patients and ability to derive quantitative measurable outcomes in this small trial.
This publication reports the first paediatric case series of the use of Miransertib in two children with PROS (PIK3CA-related overgrowth spectrum).
Objective clinical response was observed in patient one, and improvement in key qualitative outcomes was reported in patient two. This case series highlights the potential therapeutic utility of Miransertib in selected paediatric patients with severe PROS, and further demonstrates the potential for re-purposing targeted therapies for the treatment of rare diseases.
An open label, Phase 1/2 study of Miransertib in children with PROS and PS is underway to more accurately assess the efficacy of Miransertib in the treatment of PROS disorder (NCT03094832).
IAG contributed to this investigation led by Arqule (now acquired by Merck) by designing and deploying a imaging based scoring system and conducting the review of all patients in DYNAMIKA. The scoring systems allowed to assess volumetric and morphological changes in series of MRI analysis, and revealed up to 15% reduction in calculated volumes of fatty overgrowth between the treatment commencement and the last follow-up.
Title: ‘Clinical experience with the AKT1 inhibitor Miransertib in two children with PIK3CA-related overgrowth syndrome’
Journal: Orphanet Journal of Rare Diseases, February 2021
Authors: Karina Forde, Nicoletta Resta, Carlotta Ranieri, David Rea, Olga Kubassova, Mark Hinton, Katrina A. Andrews, Robert Semple, Alan D. Irvine & Veronika Dvorakova
Our goal is to accelerate novel drug development by using the right analytical tools and modern trial infrastructure. We take a broader view on the assets’ development and bring expertise in study design, execution, and commercialization. As needed, we deploy AI, Machine Learning, and smart image analysis methods to ensure the speed and cost-effectiveness of clinical programs. IAG’s team supports investors and biotech executive teams with deploying the right strategies for early efficacy assessments, objective response prediction and critical analysis of advanced treatment manifestations. Thus, lowering the investment risks into advanced therapies while helping to accelerate study outcomes.
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