Promising Experimental IPF Drug Receives Orphan Status

MediciNova, Inc.’s idiopathic pulmonary fibrosis (IPF) drug candidate MN-001 (tipelukast) was recently granted orphan drug status by the US Food and Drug Administration. As a result, MediciNova will have exclusive rights to market MN-001 to treat patients with IPF, should it be approved following clinical trials.

“We are very pleased to receive this orphan-drug designation, which is an important part of our development strategy for MN-001 in IPF,” stated Yuichi Iwaki, MD, PhD, President and Chief Executive Officer of MediciNova. “As we already have an open investigational new drug (IND) [application], we plan to finalize a protocol and submit it to the FDA in order to conduct a phase 2 clinical trial of MN-001 in IPF.”

MediciNova’s continued success is based on the mechanism of action of MN-001, otherwise known as tipelukast. As an orally active small molecule drug, MN-001 acts through several mechanisms that result in reduced fibrosis and inflammation when used in animal models. The key targets are leukotriene receptor, phosphodiesterases, and 5-lipoxygenase. These molecular pathways may be pathogenic factors in IPF.

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According to the company, the 5-LO/LT pathway in MN-001 is believed to be a pathogenic factor in the development of fibrosis. As a result, MN-001’s inhibitory effect on 5-LO and the 5-LO/LT pathway appears to offer a novel approach to treating fibrosis. MN-001 has been shown to down-regulate expression of genes that promote fibrosis including LOXL2, Collagen Type 1 and TIMP-1. MN-001 has also been shown to down-regulate expression of genes that promote inflammation including CCR2 and MCP-1. In addition, histopathological data shows that MN-001 reduces fibrosis in multiple animal models.

This is not the first time MediciNova has capitalized on MN-001. The company previously demonstrated high efficacy and tolerability of MN-001 in asthmatic patients during phase 2 trials. In total, MN-001 has been used in over 600 participants, with promising results for the intended treatments.