Fresh findings from a large clinical trial are helping build the case that an experimental drug for cardiovascular illness could also be useful in combating Alzheimer’s disease.
The drug, known as obicetrapib, is designed to block a protein that regulates cholesterol levels. Three late-stage studies have shown patients treated with it experienced significant reductions in LDL — or “bad” — cholesterol. The drug’s developer, NewAmsterdam Pharma, disclosed near the end of last year data from the latest of those experiments, titled “Broadway,” which suggested obicetrapib may also offer some protection from heart disease-related complications like strokes.
Now, NewAmsterdam is highlighting results from a pre-planned “sub-study” of Broadway focused on biological markers of Alzheimer’s.
There, the thinking is that obicetrapib’s lipid-lowering effects may safeguard neurons. Researchers believe high levels of LDL cholesterol in the brain contribute to the accumulation of amyloid and tau, the two proteins most closely linked to Alzheimer’s. And notably, the gene APOE4, which creates a protein that transports cholesterol throughout the brain, is the strongest genetic risk factor for the disease.
NewAmsterdam said the sub-study evaluated 1,727 patients, including 367 who carried the APOE4 gene. The main measure researchers looked at was a type of tau protein called “p-tau217.” Over a yearlong treatment period, levels of this protein diminished significantly across both the broader group and the APOE4 segment, according to NewAmsterdam.
Previously, the company ran a small, mid-stage study wherein 13 patients at the early stages of Alzheimer’s were given obicetrapib and followed for about six months. The experiment showed reductions in two cholesterol derivatives with ties to the disease.
The new findings differentiate obicetrapib from other drugs and “strongly support a potential preventive strategy for Alzheimer's disease,” NewAmsterdam’s CEO Michael Davidson said in a statement.
The outcome stoked excitement on Wall Street, too. Matt Phipps, an analyst at the investment firm William Blair, wrote in a note to clients that there are “clearly caveats to this analysis” since the sub-study wasn’t conducted in Alzheimer’s disease patients. Even so, taken together with the data from that smaller trial, his team thinks these latest results “intriguing” and may ultimately provide “significant long-term upside” to NewAmsterdam’s stock price.
Jefferies analyst Dennis Ding echoed that idea in his own note. While investors have been “lukewarm” on the opportunity in Alzheimer’s because of the slow uptake of Biogen and Eisai’s products, “this could be an interesting call option over time” and might attract interest from strategic dealmakers.
Others were even more bullish. Roanna Ruiz, of Leerink Partners, argued that the Broadway results not only help obicetrapib stand apart from other oral drugs and “PCSK9s” — a separate, closely watched class of cholesterol-lowering medicines — but they also allow NewAmsterdam to “pursue the blockbuster Alzheimer's market.”
TD Cowen’s Tyler Van Buren went as far as to write the data suggest obicetrapib “can potentially be the first preventative therapy for Alzheimer's disease patients.”
NewAmsterdam shares were up about 6% at market’s open Monday, though they flattened out by mid-afternoon to trade around $20.20 apiece.
NewAmsterdam plans to present more detailed data from its sub-study during the Alzheimer’s Association International Conference in Toronto at the end of July. The company said in January that data from its trio of late-stage studies should support approval filings to global drug regulators. A submission to the European Medicines Agency is planned for the second half of this year.
