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 2a clinical trial

ManRos Therapeutics develops Seliciclib (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’, ManRos Therapeutics, in collaboration with the Center for Clinical Investigation from the CHRU of Brest, received approval from the French Health Authority (ANSM) to conduct a clinical study to evaluate the safety and efficacy of Seliciclib (Roscovitine) in P. aeruginosa infected CF patients (13 hospitals, 36 patients, placebo-controlled, double-blind). The trial was launched in April 2016. Results are expected to be available in autumn 2018.

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 several classes of DYRK1A inhibitors, including Leucettines, a family of derivatives of a marine sponge natural product. ManRos has obtained proof of concept results in mice (in terms of prevention of cognitive deficits) with a Leucettine on three models of DS and three 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, based on the “Alzheimerogen” activity of some pyrazole insecticides.