I recently attended the Wall Street Journal’s 2024 Health Forum in Boston. It showcased a diversity of speakers who each spoke about the technology, business, and policy regulations that are impacting the health care industry. It was a fantastic conference and the speakers were excellent.

For investors, there were several publicly-traded companies who took the stage — including CRISPR Therapeutics (Nasdaq: CRSP), Vertex Pharmaceuticals (Nasdaq: VRTX), Alphabet (Nasdaq: GOOGL) and Moderna (Nasdaq: MRNA). There were also academic research leaders, innovative entrepreneurs, and policy decision-makers who gave a 360 degree holistic look at the industry.

For those interested in the nuance, here’s a link to my complete conference notes. But if you’re most interested in just hitting the high notes, here are my top three takeaways from this year’s WSJ Health Forum.

1) Ozempic: Light on Weight, But Heavily in Demand

A new class of drugs — including Novo Nordisk’s Ozempic and Eli Lilly’s Mounjaro — have just been approved to treat weight-loss. Even though they’ve only been on the market for less than three year, they’re already among the best-selling drugs in the history of the pharmaceutical industry.

These new drugs are GLP-1 inhibitors, which help the brain suppress appetite and keep patients from eating more than they need. The initial results thus far have been incredible. On average, patients have lost 16% of their weight when using Ozempic and around 22% when using Mounjaro. Those are comparable numbers to the weight lost from bariatric surgery.

Furthermore, it’s intriguing how the GLP-1 drugs (which are taken weekly, as an injection) can interact with different organs in the body. In the pancreas, they help to produce and regulate insulin — which can treat diabetes. In the brain, they suppress appetite — which can lead to weight loss. In the liver, they may help to prevent fatty buildups that could potentially be fatal.

And the demand for this new class of wonder drugs has been through the roof. Clinics and treatment facilities who can provide the prescriptions are reporting wait lists of over a month, just for new patients to be seen.

Yet despite the positive reception, questions remain. Scientifically, it’s been shown that the drugs work while they are being taken. However, as soon as patients stop taking the drugs, they often gain the weight right back again.

And even though they’ve received FDA-approval, insurance still rarely covers their cost for weight-loss — which is considered cosmetic/elective, rather than due to a medical need. When patients are paying out-of-pocket, Ozempic typically costs $700 or more on a monthly basis. Which is rather expensive and financially burdensome.

There will likely be upcoming improvements to the drugs, such as combination therapies to improve efficacy or improvements to improve longevity. Yet as long as these drugs are helping patients shed weight, they’ll continue packing on profits for two of the industry’s largest companies.

2) COVID: Accelerated (Some) Innovation

The COVID pandemic in 2020 had a huge impact on the medical world. There were more than 700 million reported COVID cases, and more than 5.5 billion people — 70% of the world’s total population — have now received a COVID-19 vaccine.

Moderna was one of the companies who developed and manufactured that vaccine. And as such, COVID quite significantly accelerated its innovation.

Before COVID, Moderna had 22 products in development. After COVID, it had 50.

After billions of people used its drug, there was a step change in the amount of data available related to its safety and efficacy. Moderna now better understands the coronavirus, which is already helping it prepare for future mutations.

Moderna’s parent organization, Flagship Pioneering, can also leverage its learnings from COVID to influence and steer its other companies. That’s helping it win partnerships with other large players, who are similarly interested in the data and the future potential.

This is a great reminder of how the big get bigger in the pharmaceutical industry. Large companies with plentiful resources can “try to figure something out 17 times…when most companies would give up after attempt number three.”

3) Gene Editing: Expensive, But Insurance Will Pay It

Two months ago, CRISPR Therapeutics and Vertex Pharmaceuticals made history by winning approval for the first gene-editing treatment. Its new drug Casgevy is a “one-and-done” treatment for sickle-cell disease, permanently fixing the genetic disorder that causes a patient’s red blood cells to be improperly shaped like sickles and can lead to an early death.

The drug isn’t cheap. Casgevy is priced at $2 million per treatment. That makes it one of the most expensive drugs in the history of the pharmaceutical industry.

Yet even with the expensive price tag, insurers are willing to pay it.

Gene editing offers a permanent solution. The treatment permanently alters the genome of the bone marrow’s stem cells — replacing the faulty cells with healthy cells that will replicate over time. That’s an important distinction from gene therapies, which often lose their durability after three or four years.

Ongoing chronic treatment for sickle cell disease also costs between $4 million to $6 million per patient over their lifetime. So even with the expensive price tag, Casgevy is still saving the health care system a ton of money.

So all eyes now are turned to the drug’s long-term durability. As long as Casgevy does, in fact, cure the disease, insurers will have no problem making the upfront, lump-sum payments. And those could fund a lot of exciting new opportunities in CRISPR and Vertex’s pipelines.

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