Mifcare, a biotechnology company focused on research and development of drug candidates for the treatment of pulmonary hypertension, autoimmune inflammatory diseases and cancer, recently reported encouraging preclinical data for its most advanced compound, MFC1040 for pulmonary arterial hypertension (PAH).

Mifcare designs small molecules that selectively inhibit the macrophage migration inhibitory factor (MIF), a cytokine strongly involved in PAH.

MFC1040 is a MIF inhibitor that blocks vascular cell proliferation and perivascular inflammation, two key processes that contribute to the development and progression of PAH.

In experiments, Christophe Guignabert from INSERM U999 discovered that MIF is increased in animal models and patients with PAH, which contributes to the intense vascular remodeling of pulmonary vessels. The researchers also reported that treatment with MFC1040 regressed established pulmonary vascular remodeling in the monocrotaline (MCT) PAH mouse model.

Specifically, mice with PAH that received a single daily dose of MFC1040 for two weeks had marked decreases in average pulmonary arterial pressure (mPAP), and improvements in cardiac structure and function. Treatment with MFC1040 decreased the degree of pulmonary arterial wall thickness in the animals.

“These encouraging preclinical data suggest that MFC1040 may provide therapeutic benefit for people suffering from PAH and reinforce our commitment to target inflammation which is considered as a primary component of PAH pathogenesis” said Gael Jalce, chief executive officer at Mifcare, in a recent press release.

Mifcare is planning to begin a Phase 1 trial next year.

PAH is a life-threatening disease characterized by increased blood pressure in the pulmonary artery, pulmonary vein, or pulmonary capillaries, which leads to shortness of breath, dizziness, fainting, leg swelling and other symptoms. Although the exact mechanisms leading to the onset and progression of the disease are still not yet fully understood, inflammation in and around the pulmonary arteries is strongly suspected to facilitate its development.