- A Californian drug company has decided to stop developing its experimental medicine for a common yet challenging liver disease, now that an important clinical trial has delivered negative results.
- The mid-stage trial enrolled patients with nonalcoholic steatohepatitis, or NASH, a disease in which a buildup of liver fat progressively damages the organ. The main goal was to see whether that medicine from NGM Biopharmaceuticals, known as aldafermin, could improve liver scarring without making a patient's NASH worse. NGM said Monday this goal was not met.
- Based on the results, NGM does not plan to push aldafermin into more testing. Rather, the company said it will redirect resources that would have been used on a late-stage NASH program toward different projects. NGM has four other drugs at the Phase 2 stage of development, including ones for pancreatic cancer and an advanced form of eye disease.
NASH represents an enormous opportunity for drugmakers. It's estimated to affect millions of people in the U.S. alone — which, according to analysts, means a multibillion-dollar market awaits companies that can develop effective treatments.
So far, though, NASH and its complicated biological roots have proven difficult to conquer. No drugs are currently approved for the disease. And for companies trying to change that, setbacks have been more common than successes.
Within the last couple years, all of the most advanced experimental NASH medicines have run into major obstacles. Candidates from Gilead and the French firm GenFit each flunked key studies, while a closely watched tablet developed by Intercept Pharmaceuticals was rejected by the Food and Drug Administration.
NGM is the latest to notch a NASH failure, with the data released Monday showing there was not a large enough difference between treatment with its drug compared to a placebo, as measured by whether patients' fibrosis improved at least one stage without their NASH getting worse.
Specifically, the trial, which tested three different doses of aldafermin, found 19% of participants in the placebo arm hit its main goal, compared to 31%, 15% and 30% in the low-, medium- and high-dose aldafermin arms.
Though there were some positive findings, including one that showed participants in the high-dose arm had significantly better NASH resolution than those on placebo, it looks as though NGM is moving beyond aldafermin, which has been the biotech's lead asset for some time.
"Perhaps if this was our only asset, we might be really looking sideways at this data," David Woodhouse, the company's CEO, said on a Monday morning call with investors. "But from just a prudent resource allocation point of view, it seems like actually a relatively easy decision. Not one we were hoping to have to make, but the data is what the data is, and we're gonna follow it."
Investors, however, didn't take the update well. NGM's share value plummeted more than 40% Monday morning, erasing almost $1 billion in market valuation.
Looking ahead, NGM says it's focused on its four programs in mid-stage testing. They include: NGM621, for the treatment of geographic atrophy, a severe form of age-related macular degeneration; NGM120, for the treatment of metastatic pancreatic cancer and cancer-related cachexia; and MK-3655, which is being co-developed with Merck & Co. for the treatment of NASH.
NGM is also continuing to enroll a 48-week study that's evaluating aldafermin in NASH patients with severe fibrosis and compensated cirrhosis — an area that represents a "particularly acute unmet need, given this patient population's advanced disease," according to Woodhouse.