NGM Bio raises funds to launch clinical trial of aldafermin for PSC
Findings, if positive, expected to support application seeking therapy's approval
NGM Biopharmaceuticals has raised $122 million in financing, which the California-based company will use to help launch a planned registrational clinical trial to test aldafermin, its treatment candidate for primary sclerosing cholangitis (PSC).
A registrational trial is one in which the findings, if positive, are expected to be used to support a regulatory application seeking the therapy’s approval.
The new funding also will be used to advance the clinical development of NGM120, the company’s experimental treatment for hyperemesis gravidarum, a rare complication of pregnancy characterized by uncontrolled nausea and vomiting.
“At the beginning of this year, we unveiled our strategy to advance clinical development efforts for two serious, rare conditions characterized by significant unmet patient need,” David J. Woodhouse, PhD, CEO of NGM Bio, said in a company press release. “This Series A capital strengthens our financial position, enabling us to progress our planned registrational trial of aldafermin in primary sclerosing cholangitis and to evaluate NGM120 in a proof-of-concept study for hyperemesis gravidarum.”
Both trials are planned to start in the last months of this year, but no further details on their design or enrollment sites were provided.
Aldafermin aims to improve live function, ease itching in PSC
NGM Bio said the PSC trial will use proposed surrogate measures of treatment efficacy, and further, that should such results be positive, they would be used to ask the U.S. Food and Drug Administration (FDA) to grant aldafermin accelerated approval.
Accelerated approval is a regulatory mechanism in which the FDA allows a medication to be sold based on early clinical data showing the treatment is likely to be effective. Full approval is dependent on additional clinical trial data confirming the therapy’s efficacy.
PSC is a chronic form of cholangitis, or inflammation of the bile ducts, a series of tubes that carry the digestive fluid bile out of the liver, where it’s made, to the intestines.
In individuals with PSC, chronic inflammation leads to scarring in the bile ducts that blocks the normal flow of bile. As a result, bile builds up in the liver, which damages the liver and can set the stage for serious complications like liver failure or cancer. Excess bile also can leak into the bloodstream, which leads to itching, a common PSC symptom.
To date, no treatments have been approved for PSC.
Aldafermin, formerly NGM282, is a molecule that mimics the activity of fibroblast growth factor 19 (FGF19), a gastrointestinal hormone that helps regulate the production of bile acids, a component of bile.
As such, the therapy is expected to reduce bile acid production and limit the toxic buildup of bile that drives PSC. That, in turn, would improve liver function and health and ease PSC symptoms like itching.
With this Series A investment, [The Column Group] is delighted to extend our investment in NGM and support the compelling opportunities presented by aldafermin and NGM120 as potential treatments for PSC and [hyperemesis gravidarum], respectively.
In a mouse model of PSC, the therapy was shown to reduce levels of several liver damage markers, including alkaline phosphatase (ALP), and to lessen liver inflammation and bile duct scarring.
In a previous international Phase 2 trial (NCT02704364), 62 adults with PSC were randomly assigned to receive a subcutaneous, or under-the-skin, injection of either one of two doses of aldafermin or a placebo, once daily for about three months.
That study’s main goal was to assess whether aldafermin was superior to the placebo at reducing ALP levels. While the trial failed to meet its goal, the results showed that the experimental treatment, particularly its higher dose, led to significantly greater drops in blood levels of other liver damage markers, bile acids, and markers of liver scarring.
This funding round for NGM Bio, part of a Series A financing, was led by The Column Group (TCG), with participation from a select group of investors.
“With this Series A investment, TCG is delighted to extend our investment in NGM and support the compelling opportunities presented by aldafermin and NGM120 as potential treatments for PSC and [hyperemesis gravidarum], respectively,” said Peter Svennilson, founder and managing partner of TCG, who has now joined NGM Bio’s board of directors.
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