A molecule in late-stage clinical trial
ManRos Therapeutics owns a molecule in late-stage clinical trial.
The probability for a molecule to become a drug increases at each stage of development.
Cystic Fibrosis (CF)
One molecule in phase 2 clinical trial
ManRos Therapeutics develops Roscovitine for its stimulatory effects of bactericidal activity in alveolar macrophages (which is reduced in macrophages of CF patients, explaining chronic infections, particularly by Pseudomonas aeruginosa). Thanks to a public funding (PHRC, ‘Programme Hospitalier de Recherche Clinique’) and the support from the Association ‘Vaincre la Mucoviscidose’, and following the green light from the French FDA homolog (ANSM), and in collaboration with the ‘Centre d’Investigations Cliniques’ from the CHU Brest, ManRos has started a clinical study to evaluate the safety and efficacy of Roscovitine in P. aeruginosa infected CF patients (10 hospitals, 36 patients, placebo controlled, double-blind).
A second generation molecule
ManRos Therapeutics develops other molecules along side with Roscovitine: M3 molecule analogs. These second generation molecules are designed to increase the efficiency of the Roscovitine while reducing its potential side effects.
Polycystic Kidney Disease (PKD)
The interest of ManRos for PKD started following the discovery, by a Genzyme team, of the arresting effects of Roscovitine on the development of kidney and liver cysts in mouse models of PKD. A collaboration was initiated which led to two publications and an option/ license agreement on CR8, a Roscovitine derived product. Unfortunately, for various reasons, this collaboration was stopped at the acquisition of Genzyme by Sanofi. ManRos Therapeutics nevertheless continued this project in three directions: proof of concept in animal models of PKD (and identification of molecular targets), combination of two different treatments and further optimization of Roscovitine derivatives.
Alzheimer’s Disease (AD) & Down Syndrome (DS)
AD and DS share a common therapeutic target, the DYRK1A kinase, which is involved in the cognitive deficits associated with these two diseases. ManRos is developing two classes of DYRK1A inhibitors, Leucettines and RCZ. ManRos has obtained proof of concept results in mice (in terms of prevention of cognitive deficits) with a Leucettine on two models of DS and two AD models. Optimization (medicinal chemistry, pharmacological properties) towards a drug candidate and further validation in animal models (including the identification of appropriate biomarkers) are the objectives of this AD/DS project. In addition, a chemically-induced model of AD is under development.