- Shares in Akero Therapeutics fell in value by nearly two-thirds Tuesday after the biotechnology company released mixed study results for an experimental drug it’s developing for a prevalent form of fatty liver disease known as NASH.
- Akero’s therapy, known as efruxifermin, missed the main goal of the Phase 2b study, which compared rates of improvement in liver fibrosis with worsening of NASH between participants on one of two drug doses and those on placebo.
- While the results missed the trial’s bar for declaring success, Akero noted the data favored efruxifermin. Additionally, many more people treated with the drug saw their NASH resolve than did participants receiving placebo. The trial will run for two years in total.
Akero is one of many biotechnology companies that have sought to develop a drug for NASH, a potentially serious condition thought to affect millions of Americans.
To date, it has proved to be a difficult endeavor, with both small and large drugmakers hitting setbacks in clinical trials. Intercept Pharmaceuticals, which sparked a drug development race with promising trial data in 2014, recently had its NASH drug hopeful rejected by the Food and Drug Administration for the second time, spurring it to discontinue research.
Akero’s sliding share price suggests investors see the company as joining that group. The slide in share price Tuesday morning erased nearly $2 billion from the South San Francisco-based company.
But the biotech claims the topline miss is disguising more positive results overall. “We set a high bar with the primary endpoint after only 36 weeks of treatment,” said company CEO Andrew Cheng in Tuesday’s statement. “Viewing these data in their totality ... we believe [efruxifermin] has the potential to show additional improvements for patients after the long-term follow-up period is complete at Week 96.”
On the main goal, results showed that between one-fifth and one-quarter of study participants given either a 28 milligram or 50 milligram dose of efruxifermin experienced at least a one-stage improvement in their liver fibrosis with no worsening of NASH. That compared to 14% of those on placebo, a difference that wasn’t statistically significant.
Four people who received Akero’s drug in the study had an even greater three- or two-stage fibrosis improvement, which no one on placebo experienced.
Sixty-three and 60% of patients on the 28 milligram and 50 milligram doses of efruxifermin, respectively, had their NASH resolve, compared with only 26% on placebo. According to Akero, this is the highest response rate that’s been reported to date for NASH resolution among this group of patients.
The most common side effects in the study were “transient, mild-to-moderate” gastrointestinal events. Twelve participants dropped out of the study due to side effects, mostly due to diarrhea. None of the serious adverse events reported in the trial were judged by investigators to be related to efruxifermin.
Akero is planning to begin three more studies of efruxifermin, with the first patients enrolling by the end of this year.
Shares in 89bio, which is developing a similar drug for NASH, also fell sharply Tuesday morning on news of Akero’s data.