A novel ulcerative colitis treatment - A phase III ready feasibility study | UCRxF | Proyecto | Ficha informativa | H2020 | CORDIS

Descripción del proyecto

Un fármaco biológico para la colitis ulcerosa

La colitis ulcerosa (CU o UC, por sus siglas en inglés) se asocia a una inflamación prolongada del colon que provoca dolor abdominal y diarrea. Para tratar la CU, el equipo del proyecto UCRxF, financiado con fondos europeos, propone el cobitolimod, un nuevo medicamento de ADN, que tiene un mecanismo de acción novedoso y activa el receptor de tipo toll 9 (TLR9). Los efectos secundarios de la activación del TLR9 incluyen la curación de las úlceras y la reducción de la inflamación de la mucosa, mientras que los resultados iniciales de los ensayos clínicos son alentadores con respecto a la seguridad y la eficacia del cobitolimod. Las actividades de UCRxF incluyen la evaluación toxicológica del fármaco y la mejora de la fabricación antes de pasar a los ensayos clínicos de fase III.

Objetivo

InDex Pharmaceuticals AB is a pharmaceutical development company based at the Karolinska Institutet Science Park in Stockholm, Sweden. The company´s primary asset is cobitolimod, which is in late phase clinical development as a new medication to treat moderate to severe active ulcerative colitis (UC); a chronic, debilitating disease with a high unmet medical need. UC is caused by chronic inflammation of the colon, and there is no cure.

Cobitolimod offers a novel and unique mechanism of action for the treatment of UC, and has demonstrated a higher efficacy and better safety profile in clinical studies than what has been reported for currently approved biological drugs. Cobitolimod is part of the broad InDex portfolio of DNA-based ImmunoModulatory Sequences (referred to as DIMS), which have the potential for treatment of various immunological diseases. Cobitolimod is currently being tested in a phase IIb clinical study with top line results expected in Q4 2018.

InDex’s business strategy is to out-license cobitolimod to a larger international pharmaceutical company prior to the initiation of clinical phase III development. This will generate revenues for InDex from upfront and milestone payments as well as royalties from third party sales, which can then be reinvested in the company to finance development of new compounds from the DIMS technology platform.

For clinical phase III, additional manufacturing and toxicology work as well as commercial preparations are essential. The planning and preparation of this work is the purpose of this feasibility study, which is a crucial part of the cobitolimod phase III ready programme, increasing the attractiveness of the asset in partnering discussions with pharmaceutical companies looking for innovative new products. A licensing deal will ensure that the development of this promising, innovative drug will continue in a fast and efficient way to reach patients with this debilitating disease.