Eli Lilly to take big swings in Alzheimer’s, ALS and gene therapy

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Heart disease. Hearing loss. Addiction. Chronic pain. Alzheimer’s. ALS. These are some of the areas where Eli Lilly, flush with cash from its GLP-1 drugs, wants to make big bets. 

These are the ideas that are “hiding in plain sight,” said Lilly Chief Scientific Officer Dan Skovronsky. They’re places where other pharmaceutical companies might not want to go because they’re hard problems to solve. 

“As right now really the biggest health-care company in the world, probably the biggest health-care company in the world ever, we have an obligation,” Skovronsky said. “Investors have given us that vote of confidence. We see that as an obligation to invest in some of these big problems that are hiding in plain sight, to try and make a difference for the health of your community.” 

Lilly’s tirzepatide, sold as Mounjaro for diabetes and Zepbound for obesity, has transformed the company. The company’s sales have grown almost 60% since Mounjaro was approved in 2022. Lilly’s stock price has rocketed 268% higher in the last three years, giving the company a market cap of $823 billion – the highest of any health-care company. 

Now the company wants that success to translate to other disease areas.

Lilly’s already testing whether its drug Kisunla can prevent Alzheimer’s disease. Kisunla is a monoclonal antibody that removes amyloid plaques from the brain, which are associated with the memory-robbing disease. It’s currently approved to treat people in the early stages of Alzheimer’s. 

The company’s recruiting seniors at churches, Walmart parking lots and other venues to give them a blood test and see if they’re at risk of the disease. Some of the people in the trial will receive Kisunla and others will receive a placebo. Once enough of the participants are diagnosed with Alzheimer’s, Lilly will look and see if there’s a difference between people who received its drug and people who received a placebo. 

“If we can prevent it, even in half of the patients, that will be a revolution in how we think about diagnosing and treating Alzheimer’s,” Skovronsky said. “It’d probably mark a major inflection point in how these kinds of medicines are used.” 

A vial of Eli Lilly’s Alzheimer’s drug sold under the brand name Kisunla.

Source: Eli Lilly

Lilly’s also investing heavily in gene therapy at a time when the field is facing significant uncertainty. Last summer, the company opened the $700 million Lilly Institute for Genetic Medicine in Boston, away from Lilly’s headquarters in Indianapolis and in one of the U.S. epicenters for this work. 

Biopharma companies large and small are struggling to turn the potential of scientific breakthroughs like gene-editing technology Crispr-Cas9 into blockbuster drugs. One of the holdups has been figuring out how to get the treatments to their destinations inside the body, or delivery, as it’s known in the industry. 

“The reality is that local delivery is going to be sort of a small application to human health, but once we crack that, and I think Lily is the company that can crack it, because when we think about delivering to other tissues, it’s not just the genetic medicine, it’s how do you package it? How do you target it with antibodies or small molecules? Those are things we’re really good at,” Skovronsky said. 

Lilly is uniquely positioned to take the big swings. Whether it can hit them remains to be seen. 


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