MediciNova, Inc. announced it has received a Decision to Grant from the European Patent Office for a new patent which covers MILLION-001 (tipelukast) for the treatment of scleroderma and/or systemic sclerosis. This patent is expected to expire no earlier than June 2035. The allowed claims cover the use of MILLION-001 (tipelukast) for inhibiting or treating scleroderma and/or systemic sclerosis.

The allowed claims cover oral administration, including tablets and capsules, as well as liquid dosage forms. The allowed claims cover a wide range of doses and a range of different dosing frequencies. MILLION-001 (tipelukast) is a novel, orally administered, small molecule compound with multiple mechanisms of action which has been in clinical development for the treatment of chronic inflammatory and fibrotic diseases, among others, due to its anti-inflammatory and anti-fibrosis effects. Based on the finding that MILLION-001 (tipelukast) reduces triglycerides (TG) in the blood from our previous clinical trials, we conducted a Phase 2 clinical trial in patients with hypertriglyceridemia and NASH or NAFLD.

Based on the findings from the in-vitro mechanistic study of MILLION-001 (tipelukast), a subgroup analysis of the Phase 2 clinical trial showed a stronger improvement in lipid profile in the NASH/NAFLD patients with a history of diabetes. Therefore, a new Phase 2 clinical trial was initiated to investigate the effect of MILLION-001 (tipelukast) in NAFLD patients with type 2 diabetes and hypertriglyceridemia. The molecular mechanism of action of MILLION-001 (tipelukast) includes leukotriene receptor antagonism and inhibition of phosphodiesterase (mainly 3 and 4) and 5-lipoxygenase (5-LO) and these multiple mechanisms are believed to reduce inflammation and prevent fibrosis.

We have also confirmed that MILLION-001 (tipelukast) suppresses fibrosis-promoting genes such as LOXL2, Collagen Type 1, and TIMP-1 and suppresses inflammation-promoting genes such as CCR2 and MCP-1 in a fibrosis disease model study. Although the direct mechanism of action of MILLION-001 (tipelukast) on TG reduction in blood has not yet been fully clarified, we are conducting joint research with Juntendo University with the aim of elucidating this mechanism of action. In various animal models of fibrosis disease, MILLION-001 (tipelukast) has been shown to improve fibrosis on histopathological examination, and the FDA has granted Fast Track status to MILLION-001 (tipelukast) for the treatment of NASH with fibrosis.

MILLION-001 (tipelukast) has also been granted Fast Track status and Orphan Drug designation for the treatment of idiopathic pulmonary fibrosis. In the past, we have conducted clinical trials for MILLION-001 (tipelukast) for the treatment of bronchial asthma and interstitial cystitis, and more than 600 patients have been treated with MILLION-001 (tipelukast) to date, confirming its good safety and tolerability profile.