MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the JASDAQ Market of the Tokyo Stock Exchange (Code Number: 4875), today announced that following completion of an agreement with Penn State (Dr. Rebecca Bascom, Principal Investigator) and IRB (Institutional Review Board) approval by Penn State Milton S. Hershey Medical Center, MediciNova will initiate a Phase 2 clinical trial of MN-001 (tipelukast) to treat moderate to severe idiopathic pulmonary fibrosis (IPF).
Yuichi Iwaki, MD, PhD, President and Chief Executive Officer of MediciNova, Inc., commented, “We are very pleased to have completed the IRB review period and the agreement with Penn State and look forward to initiating patient enrollment this Fall.” Dr. Iwaki further commented “MN-001 development is addressing an unmet medical need in targeting the most severely afflicted IPF patients.”
About the IPF Trial
The Phase 2 trial is a single-center, randomized, placebo-controlled, double blind 6-month study followed by an open-label extension phase to evaluate the efficacy, safety, and tolerability of MN-001 in subjects with moderate to severe IPF. Approximately fifteen qualifying subjects will be randomly assigned in a 2:1 ratio to MN-001 750 mg or matching placebo orally administered twice a day for 26 weeks (double-blind treatment phase). After completion of the double-blind treatment phase, subjects will participate in the open-label extension (OLE) phase for an additional 6 months. Subjects who were in the placebo group will be administered MN-001 750 mg twice a day for remainder of the OLE Phase. Subjects randomized to the MN-001 group will continue on MN-001 for additional 6 months. A follow-up visit will occur within 4 weeks after last dose.
The primary efficacy endpoints of the study are to evaluate the effect of MN-001 on 1) change from baseline of forced vital capacity (FVC) and FVC% predicted up to 26 weeks, and 2) the semiannual rate of decline of disease activity based on forced vital capacity (FVC). Secondary endpoints include safety and tolerability, semiannual rate of decline on disease activity based on the 6-minute walk test (6MWT), change from baseline on disease activity based on Modified Medical Research Council Dyspnea Score (MMRC), change from baseline on quality of life (QOL) measured by A Tool to Assess Quality of Life in Idiopathic Pulmonary Fibrosis (ATAQ-IPF), frequency of worsening IPF, and time to first worsening of IPF.
FDA (U.S. Food and Drug Administration) has already approved the protocol for this clinical trial of MN-001 (tipelukast) for the treatment of moderate to severe IPF. Importantly, due to safety data from previous clinical studies of MN-001, FDA agreed that MediciNova may proceed with a Phase 2 study as the first clinical trial of MN-001 in IPF. The IPF protocol was filed under MediciNova’s open IND (Investigational New Drug Application) in FDA’s Division of Pulmonary, Allergy, and Rheumatology Products (DPARP). FDA granted orphan-drug designation to MN-001 for the treatment of IPF, which will provide MediciNova with seven years of marketing exclusivity if MN-001 is approved for IPF, as previously announced. Additionally, the FDA granted Fast-Track Designation to MN-001 for the treatment of IPF, which expedites the review process and emphasizes frequent communication between the FDA and the sponsor throughout the drug development process.
Pulmonary fibrosis (PF) is a progressive disease characterized by scarring of the lungs that thickens the lining, causing an irreversible loss of the tissue’s ability to transport oxygen. The causes of PF vary and can be due to anti-cancer drug therapy or exposure to chemicals. Idiopathic pulmonary fibrosis (IPF) is one type of PF without a clear cause. According to the Coalition for Pulmonary Fibrosis, IPF affects approximately 128,000 individuals in the U.S., with about 48,000 new cases diagnosed annually. The prognosis for IPF is poor and about two-thirds of IPF patients die within five years of diagnosis.
About MN-001 (tipelukast)
MN-001 (tipelukast) is a novel, orally bioavailable small molecule compound thought to exert its effects through several mechanisms to produce its anti-inflammatory and anti-fibrotic activity in preclinical models, including leukotriene (LT) receptor antagonism, inhibition of phosphodiesterases (PDE) (mainly 3 and 4), and inhibition of 5-lipoxygenase (5-LO). The 5-LO/LT pathway has been postulated as a pathogenic factor in fibrosis development and MN-001’s inhibitory effect on 5-LO and the 5-LO/LT pathway is considered to be a novel approach to treat 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.
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 is considered generally safe and well-tolerated.