The state’s celebrated cluster of biotechs has hatched breakthrough medicines for everything from COVID-19 and cystic fibrosis to multiple rare diseases. It has pioneered cutting-edge research in exotic fields like messenger RNA, gene editing, and gene therapies.
But when it comes to what is arguably the hottest space in drug development today — controlling weight and diabetes for millions of people worldwide — the brainy innovators from Kendall Square have missed out on the booming market, at least so far.
Instead, the field, which has scientific roots in Massachusetts, is dominated by a pair of global pharma giants: Denmark’s Novo Nordisk, maker of the blockbuster drugs Wegovy and Ozempic, and Indianapolis-based Eli Lilly, which produces the fast-selling Mounjaro.
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The drugs, which are injected once a week, yield an average weight loss of 15 to 20 percent by targeting brain receptors that make people feel hungry. (Wegovy is approved for weight loss in the United States; Ozempic and Mounjaro are approved for diabetes, but also result in weight loss.) The drugs can cost more than $1,000 a month, and most health insurers aren’t yet covering them for obesity.
Despite the resistance from insurers, and worries it may be too soon to gauge long-term side effects, combined sales for the new class of appetite-suppressing drugs, called GLP-1, are projected to top $18 billion this year. That dwarfs the $8 billion in revenue forecasted for cholesterol drugs, a market that includes lower-cost generics.
So why has Massachusetts’ vaunted biotech sector been late to the game? Part of it is the nature of developing diabetes and obesity drugs.
Those treatments require long-term commitment, steady investment, and “perseverance in the field for decades,” said Daniel Skovronsky Lilly’s chief scientific and medical officer, playing to the strengths of deep-pocketed Big Pharma companies. In contrast, the smaller biotechs that abound in Massachusetts are backed by venture capital firms with shorter time horizons to advance new treatments.
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“The big companies can do it, and they can do it in fields that nobody else wanted to work in,” Skovronsky told life sciences leaders at the STAT Summit in Boston last week.
Because earlier weight loss drugs showed far less benefits, many business analysts had questioned the wisdom of investing heavily in their development. “For many years, the commercial wizards would have said there’s no opportunity,” Skovronsky said.
But many in the industry say the field is still young and the current leaders could be overtaken by future treatments that are more effective or easier to administer, creating an opening for Massachusetts biotechs. Biopharma history is replete with follow-on therapies that displaced market leaders. Gilead Science’s hepatitis C treatment Solvaldi, for example, supplanted Incivek, an antiviral medicine sold by Boston-based Vertex Pharmaceuticals.
Indeed, a number of drug makers, including Lilly and Pfizer, already are testing weight loss pills that could be taken orally once a day.
Several biotechs in Massachusetts, meanwhile, are seeking to elbow their way into the lucrative market.
“Once the door’s opened, everybody’s running through it,” said David Meeker, chief executive of Rhythm Pharmaceuticals. “Massachusetts companies are taking new approaches the big guys aren’t taking yet.”
Rhythm, based in Boston’s Back Bay, is carving out its own niche. It won Food and Drug Administration approval for a self-administered daily injection called Imcivree that manages weight in children and adults with obesity linked to ultrarare genetic defects.
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So far, the drug, which costs about $300,000 per patient annually, treats only several hundred people in the United States. But the company has developed a genetic testing program and is working with doctors to identify more patients with genetic disorders who could be treated.
“These are rare diseases lost within a common disease,” said Meeker, a veteran of the rare disease business at pioneering biotech Genzyme and, later, at Sanofi, the French drug maker that acquired it.
Across the river in Cambridge, biotech Scholar Rock, cofounded by Harvard medical professor and entrepreneur Timothy Springer, announced this month that it’s developing an experimental drug that could be used in combination with existing treatments to allow patients to continue building muscle as they lose weight. Patients now taking the GLP-1s typically lose muscle mass along with weight.
“Our goal is to optimize weight loss by preserving and increasing lean muscle,” said Jay Backstrom, chief executive of Scholar Rock, which plans to begin clinical trials in 2025. “If you look at the current therapies . . . as the weight is being reduced, muscle mass is being reduced as well.”
Another drug discovery program is underway at Fractyl Health, a Lexington biotech that has raised $300 million from big-time backers like General Catalyst and Bessemer Venture Partners. Fractyl is testing in pigs and mice a GLP-1-based therapy that modifies patients’ genes to control appetite; the company plans to seek FDA approval by the end of next year to begin clinical trials in humans.
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If successful, the treatment would only have to be administered once, addressing a major problem with today’s drugs. Many people stop taking the weekly injections after about a year and then quickly regain the weight they lost.
“It has the potential to be a real step forward in treatment,” said Harith Rajagopalan, the chief executive of Fractyl. “If you can take this treatment once and have a long-term effect, you can make sure these people are going to benefit for a long time.”
Ironically, while biotechs here are playing catchup in the race to market new weight loss treatments, some of the key scientific findings that underpin the GLP-1 therapies emerged from Boston.
GLP-1 hormones, which control the insulin that maintains healthy sugar levels, were discovered in the lab of endocrinologist Joel Habener at the Massachusetts General Research Institute, which was granted patents on its discoveries in the 1990s. The lab’s work was credited at last week’s STAT Summit by Lotte Bjerre Knudsen, chief scientific adviser for Novo Nordisk, which is reaping the benefits.
Even as Novo and Lilly capitalize on the obesity drug boom, Lilly’s Skovronsky acknowledged the potential for better weight loss drugs that can also reduce the prevalence of other diseases, such as hypertension, heart attacks, and strokes. “It will take time to reach the full potential,” he said. “There will be multiple generations of products.”
That’s a welcome forecast for Massachusetts biotechs scrambling to cash in on the weight loss bonanza. As the field expands, more drug makers are expected to jump in. Investment bank Goldman Sachs has projected the market will more than double to $44 billion by the end of the decade.
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“Given the scale of the market and the opportunity, a lot of companies are coming into the space,” said Backstrom at Scholar Rock. “The market size will accommodate more than two large companies.”
Robert Weisman can be reached at robert.weisman@globe.com.