MN-001 is a novel, orally bioavailable small molecule compound thought to exert its effects through several mechanisms to produce its anti-inflammatory activity in preclinical models, including leukotriene (LT) receptor antagonism, inhibition of phosphodiesterase (PDE) 3 and 4, and inhibition of 5-lipoxygenase (5-LO). It is postulated that inhibition of the 5-LO pathway exerts anti-inflammatory actions, which has implications in various inflammatory diseases such as arthritis, osteoarthritis, and allergy. Recently, 5-LO has been postulated as a pathogenic factor in fibrotic changes. MN-001's inhibitory effect on 5-LO and the 5-LO/LT pathway is considered to be a novel approach to treat fibrosis.

Previously, MediciNova evaluated MN-001 for its potential clinical efficacy in asthma and had positive Phase 2 results. MN-001 has been exposed to more than 600 subjects and considered generally safe and well-tolerated.

Every Breath Counts: Idiopathic Pulmonary Fibrosis, a documentary to mark Pulmonary Fibrosis Awareness Month, will air on the Discovery Channel on September 13th and 27th at 8:00 am ET/PT.

Supported by Boehringer Ingelheim Pharmaceuticals, Inc. and created together with the Pulmonary Fibrosis Foundation and the Coalition for Pulmonary Fibrosis, the documentary aims at increasing public awareness and understanding of idiopathic pulmonary fibrosis (IPF). The documentary can also be viewed at www.everybreathcountsfilm.com.

The treatment uses a microRNA mimic, miR-29, which is delivered to lung tissue intravenously. In mouse models, miR-29 not only blocked pulmonary fibrosis, it reversed fibrosis after several days.

The findings were published Sept. 19 in the journal EMBO Molecular Medicine.

“The mimic, when injected into the blood, goes to the lung and it has a sustained effect. We are very impressed that it can reverse fibrosis, not only prevent it,” said Naftali Kaminski, M.D., a professor at Yale School of Medicine and section chief of pulmonary, critical care, and sleep medicine. He is a corresponding author of the study.

"Our preclinical data has demonstrated robust improvements in pathology and breadth of response in key bio-markers implicated in the progression of this deadly disease. After thoroughly reviewing both the pre-clinical and phase I safety data, our scientific advisers strongly recommended to advance the investigation of PBI-4050 for IPF", stated Mr. Pierre Laurin, President and Chief Executive Officer of ProMetic. "We are very enthusiastic about the potential of PBI-4050 to offer breakthrough therapy clinical benefits to patients suffering from this deadly medical condition", added Mr. Laurin.

ProMetic is first initiating its IPF clinical program this quarter in Canada and intends to include other already identified sites in the United Kingdom and US shortly thereafter. ProMetic is currently preparing a request for an IPF Orphan Drug Designation ("ODD") to be filed with the US Food and Drug Administration ("FDA").

"The ability of PBI-4050 to reduce or improve some of the most recognized inflammatory and fibrotic indicators, typically representative of lesions and scars in the lungs, provides a compelling case to aggressively investigate the use of this oral therapy to treat IPF patients", said Dr. John Moran, ProMetic's Chief Medical officer.

In a gold standard animal model proven to emulate pulmonary fibrosis in humans, PBI-4050 performed favorably compared to Pirfenidone, the only commercially approved product for such medical use. PBI-4050 significantly reduced tissue scarring in the lungs otherwise observed in non-treated animals. Moreover, the combination of PBI-4050 and Pirfenidone generated unprecedented reduction of fibrotic markers, indicating the potential for clinically significant improvement and stabilization in lung function. ProMetic has also successfully completed its PBI-4050 Phase I clinical trial in 40 healthy volunteers where it was found to be safe and very well tolerated without any serious adverse events reported.

Yuichi Iwaki, MD, PhD, President and Chief Executive Officer of MediciNova, Inc., commented, "We are very pleased to receive this orphan-drug designation, which is an important part of our development strategy for MN-001 in IPF. As we already have an open IND, we plan to finalize a protocol and submit it to FDA in order to conduct a Phase 2 clinical trial of MN-001 in IPF."