The field of fatty liver, a metabolic disease characterized by scarring of the liver and is part of the metabolic syndrome that includes diabetes, obesity and heart disease, was once considered one of the hottest areas of investment in the medical sector, as it has no cure today. However, after several resounding failures in the field, including products of the companies Gilead, Intersept and Enanta, the enthusiasm of investors and the companies themselves from the field has declined, and some development plans in this field have been canceled.
However, whoever continues to bet on the grand prize, is a company Lonely The Israeli, which is traded on the Nasdaq at a value of $ 61 million.
Following promising results in Phase II of the clinical trials, the company has embarked on its Phase III trial, which will be conducted in several phases. In the first phase, 150 NASH patients, with a background of obesity and diabetes, will receive the treatment and undergo a biopsy in which the company will examine whether there is a reduction in the level of inflammation in the liver, as well as a slowdown in the development or stop of liver scarring. If good results are obtained from this experiment, which was conducted without a control group, the company will continue the experiment with a control group, which will be conducted in 1200 patients (800 in the experimental group and 400 placebo, and of course they do not know who).
The results of the controlled trial, if successful, will be submitted to the US Food and Drug Administration. Even if the product is approved, another 800 patients will enter for another trial, for follow-up.
The treatment is intended to be chronic, and in the trial participants will take the drug or placebo for an entire year. All patients will undergo a biopsy, which makes this experiment invasive and therefore its relatively slow and gradual onset. The need for a biopsy in the trial and the attempts to find new diagnostic solutions are among the things that have slowed the development in this market to date, but Gilmad decided to go for the most direct measure, even if it would make it difficult to recruit the subjects.
The experiment will be considered a success if it meets one of two main goals: the disappearance of the inflammation and stopping the scarring, or an improvement in the scarring without aggravation of the inflammation.
Improvement of at least one degree in the scar
The report today is from the first 16 patients in the first part of the trial, which is as stated open without a control group. In 15 of the 16 patients, an improvement in scarring was observed, after two days of treatment during the period. In honor of the current trial, the company increased the drug dose, but no abnormal side effects were observed. The decision to swallow the dose was made after in the previous experiment, only the high dose achieved a clinically significant result.
The biopsies were performed in 8 patients after 24 weeks, in 6 patients after 48 weeks and in 2 patients after 72 weeks. 50% of patients (8 in number) showed improvement of at least one degree in the scar (4 of 8 patients after 24 weeks of treatment, 3 of 6 patients after 48 weeks of treatment, and 1 of 2 patients after 72 weeks of treatment). Among three patients (19%) the scar improved in two degrees.
The data will be presented at the Liver Conference Digital Experience 2021, held from November 12-15, as part of the Liver Conference of the American Liver Research Association (AASLD).
Alan Bahrav, President and CEO of Gilmad, noted that “The data we present today provide initial clinical support for our hypothesis that higher exposure to the speaker leads to an improvement in the clinical benefit of the drug, and that the anti-fibrotic effect of the speaker may manifest as early as 24 weeks.
“The findings are a direct result of the hard work we have done to improve and optimize the drug since completing Phase 2b ARREST research, with the goal of reducing the risk in the Phase 3 clinical trial and bringing to market a leading drug for fatty liver and fibrosis,” added the rabbi.