- Shares of Akero Therapeutics were up by as much as 27% Tuesday morning after the company revealed its main drug succeeded in a mid-stage study of patients with a liver disease known as NASH.
- With NASH, or nonalcoholic steatohepatitis, built-up of liver fat progressively damages the organ. Akero's data show study participants who were given AKR-001 experienced greater liver fat reduction than those given a placebo. The company tested three doses of the drug, each of which significantly outperformed placebo.
- Akero expects to report liver biopsy results and topline safety data sometime in the next three months. The company, though, doesn't know how the coronavirus pandemic will affect this data collection. Already, the virus has caused Akero to delay the start of an additional part of its study that would evaluate AKR-001 in NASH patients with more severe liver damage.
Wall Street analysts see a multibillion-dollar opportunity in NASH, which is thought to affect millions of people in the U.S.
That large patient pool, coupled with there being no drugs approved for NASH, has attracted attention and investment from both big pharmas and venture capitalists. Apple Tree Partners, for example, founded Akero and co-led the $65 million Series A financing that got the company off the ground. Last summer, Akero raised $98 million in net proceeds from an initial public offering.
Though Akero's stock price has fluctuated since it went public, Tuesday's data release provided a bump that offset some of the coronavirus-related decline seen so far in March.
The data pertained to four study measurements. The first two were absolute reduction in liver fat and relative reduction in liver fat. If a patient started the trial with 20% liver fat and ended with 15%, the absolute reduction would be 5% and the relative reduction would be 25%.
Results showed that, on average, study participants experienced absolute reductions of 12% to 14%, depending on the dose of AKR-001 they were given. For relative reduction, the average was between 63% and 72%. The trial also looked at the number of patients who achieved a 30% or greater relative reduction in fat, as well as levels of an enzyme known as ALT, which is found at higher levels in patients with unhealthy livers.
While difficult to compare across trials, Akero's efficacy data, at least on the surface, seem to hold up against rival NASH drugmakers. A Phase 2 study of a drug from Madrigal Pharmaceuticals found mean relative fat reduction of 37% for patients in the treatment group versus 9% for those in the placebo group. Viking Therapeutics, meanwhile, disclosed in mid-2018 data from a 'super'-responder analysis showing that its drug VK2809 reduced liver fat content by 50% or more in two-thirds of patients.
Data from Akero's Phase 2a BALANCED study
|Measure (mean)||Placebo (n=21)||AKR-001 28 mg (n=19)||50 mg (n=20)||70 mg (n=20)|
|Abs. reduction in liver fat (%)||-0.3||-12.3||-13.4||-14.1|
|Rel. reduction in liver fat (%)||0||-63||-71||-72|
|≥30% rel. reduction in fat (%)||10||84||85||75|
|Reduction in ALT (U/L)||-6||-24||-30||-32|
*AKR-001 doses given once weekly
Akero's CEO Andrew Cheng told BioPharma Dive the next step for AKR-001 would be a Phase 2b trial, the details of which would need to ironed out with the Food and Drug Administration.
However, COVID-19 presents an obstacle to doing more research.
"In terms of the current situation, it's tricky," Cheng said. "If we were to enroll or begin screening, it would be sometime later in the year, hopefully by the time the pandemic is resolved or greatly diminished."