Biopharmaceutical company MediciNova recently announced that MN-001 (tipelukast) has been approved for clinical trials by the U.S. Food and Drug Administration (FDA) for the treatment of moderate to severe idiopathic pulmonary fibrosis (IPF).
MN-001 is an orally-available small molecule compound, demonstrated to have anti-inflammatory and anti-fibrotic activities. It acts through different mechanisms, namely by down-regulating the expression of genes known to promote fibrosis (e.g., LOXL2, TIMP-1 and Collagen Type 1) and genes known to promote inflammation (e.g., MCP-1 and CCR2). In terms of histology, MN-001 was also found to be able to reduce fibrosis in several animal models.
It is important to highlight that due to the fact that MN-001 has been proven to be generally safe and well-tolerated by study participants without any serious adverse reactions, the FDA has cleared the drug to proceed to its first clinical trial, a Phase 2 study. The IPF protocol was filed under the open Investigational New Drug Application from MediciNova in FDA’s Division of Pulmonary, Allergy, and Rheumatology Products (DPARP). In addition, the FDA has granted MN-001 orphan drug designation, which gives MediciNova exclusive marketing of the drug in the event of its approval for IPF treatment.
“We are very pleased that this important regulatory step is now completed, as we can now pursue clinical development of MN-001 in IPF,” stated the President and CEO of MediciNova, Dr. Yuichi Iwaki, in a press release.
IPF is a serious progressive disease in which the alveoli and the lung tissue close to the alveoli is damaged, becomes thick and scarred, compromising oxygen transfer between the lungs and the bloodstream. The generation of this scar tissue is known as fibrosis. IPF is characterized by a shortness of breath that gradually worsens, with respiratory failure being the main cause of death associated with IPF. There is no cure for this disease and it is estimated that 128,000 individuals in the U.S. suffer from IPF, with approximately 48,000 new cases diagnosed every year. IPF has a poor prognosis and around two-thirds of the patients succumb to death within five years after being diagnosed.