Relief for MediciNova as NASH drug clears phase 2 trial

Shares rebound after last week's disappointment for ibudilast in methamphetamine dependence. (Nasdaq)

Drug repositioning specialist MediciNova has stopped a mid-stage trial of its nonalcoholic steatohepatitis (NASH) drug tipelukast early on the strength of positive data, shrugging off a pipeline disappointment last week.

The California-based biotech said it will now push the accelerator on the development of tipelukast (MN-001) on the strength of the study, which enrolled patients with NASH and other stages of nonalcoholic fatty liver disease (NAFLD) who had elevated blood triglycerides, a biomarker thought to be linked to fat accumulation in the liver.

In the phase 2 trial, tipelukast was able to cut triglycerides levels significantly in these patients, from a mean of 260.1 mg/dL before treatment to 185.2 mg/dL eight weeks later, meeting one the primary endpoints in the trial. Further details will be presented at the European Association for the Study of the Liver later this month.

Last week, MediciNova’s stock took a tumble after its ibudilast failed a phase 2 trial drug in methamphetamine dependence, but that disappointment has been quickly offset by the new tipelukast data in a much bigger target population. Shares in the company rocketed more than 40% after-hours on news of the new trial, more than offsetting its earlier declines.

NASH is a form of chronic liver disease caused by a buildup of fat in the liver and is thought to affect around 15 million Americans. If left untreated, it can cause scarring, or fibrosis, in the liver, which can eventually lead to liver failure or cancer.

It has been tipped to replace alcoholism and viral hepatitis as the leading cause of liver transplants in the coming years That has led to a scramble among biopharma companies to develop therapies for the disease, which some forecasters predict could develop into a $35 billion market.

The positive phase 2 trial for tipelukast—originally discovered by Japanese drugmaker Kyorin and licensed by MediciNova in 2002—could ignite interest among big pharma companies, which have been snapping up promising NASH candidates at pace in the last few years.

The compound is an oral small-molecule inhibitor of the PDE3 and 4 receptors, 5-lipoxygenase, and phospholipase C, as well as an antagonist of leukotriene D4 receptor. Preclinical studies in animal models have suggested the drug can reduce the formation of fibrosis in advanced NASH whilst also tackling the inflammation that characterizes the disease, a double whammy which could make it an attractive therapeutic prospect.

The drug is also in a phase 2 trial for idiopathic pulmonary fibrosis, another potentially big opportunity, but has run into some roadblocks in the past. It was previously abandoned as a treatment for asthma—for commercial reasons when montelukast went off-patient—as well as intestinal cystitis because of a lack of efficacy in trials.

MedicNova’s CEO Yuichi Iwaki, M.D., Ph.D., said, “We are thrilled with the results of this study, which showed a large reduction in triglycerides. Based on the results of this study, along with the triglyceride data we have from prior clinical studies of MN-001 in other indications, we believe that MN-001 has potential to benefit a wide range of patients with hypertriglyceridemia, not limited to those with NASH and NAFLD.”