June 26, 2023

The need-to-know this morning

Fibrogen said an experimental drug called pamrevlumab failed to improve lung function compared to a placebo in a Phase 3 study involving patients with idiopathic pulmonary fibrosis. Earlier this month, pamrevlumab also failed to achieve the efficacy goal in study for Duchenne multiple dystrophy.
Pfizer is advancing its oral, GLP-1 drug candidate danuglipron into late-stage clinical development for obesity and type 2 diabetes. A second GLP-1 drug, lotiglipron, is being discontinued.
Agios Pharmaceuticals said its sickle-cell disease drug mitapivat showed improved hemoglobin response compared to a placebo in a mid-stage clinical trial.
Moonlake Immunotherapeutics said its experimental antibody sonelokimab achieved the goals of a mid-stage study involving patients with hidradenitis suppurativa, a debilitating skin disease.
Biogen is convening a shareholder meeting today, highlighted by a vote on the nomination of Susan Langer to its board of directors.

losing weight

Lilly's GLP-1 pills are quite effective

The highest dose of Eli Lilly's experimental GLP-1 drug, orforglipron, led to nearly 15% in weight loss over 36 weeks, a new study in NEJM shows. By the end of the study, weight loss for participants hadn't plateaued — suggesting that even more weight loss is possible on the once-daily pill. The results were presented to a packed room this weekend at the American Diabetes Association conference in San Diego.

Orforglipron is part of a class of wildly popular drugs that mimic the effects of the glucagon-like peptide 1 hormone that creates feelings of satiety. The GLP-1 drugs already on the market exist in injection form; if the pills ultimately work as well, they could offer a more convenient option for many patients.

"There are distinctive advantages to these oral small molecules — they are less expensive to make and likely to be less expensive to the patient, which means they don't have to be 100% as good as [the injectables] to have a place in the market," one obesity expert who was not involved in the study told STAT.

BIOTECH

What's next for Moderna?

As the demand for Covid shots wanes, Moderna is trying to find its footing — and new uses for its mRNA-focused platform. The company still has star power accumulated during its pandemic heyday, and it has a plan — a pipeline of nearly 60 experimental drugs, ranging from personalized cancer vaccines to rare disease medicines. It's also been building out a global sales force and biomanufacturing network. But, as the Boston Globe's Robert Weisman reports, Moderna has an uphill climb all the same.

It must transition from a research-focused business to a multi-drug commercial company without losing its science edge, one MIT health care finance professor says. "In the parlance of Wall Street, it's always, 'What have you done for me now?'" he said. "They've got a really large pipeline of interesting therapeutics. So the world is waiting to see what's next."

public opinion

Nearly half of U.S. adults would spend $100 a month on new weight-loss drugs

The new wave of weight-loss medicines has nearly half the country champing at the bit. A survey from STAT and The Harris Poll found 47% of Americans would be willing to spend up to $100 a month for weight-loss drugs like Wegovy. Nearly one-quarter said they'd pay up to $250 per month, and another 17% said they'd pay as much as $500 a month for these medicines. That said, the majority — 84% — said they think insurance companies ought to pay for these medicines, which can cost up to $1,300 per month.

"When it comes to paying for these drugs it seems important to distinguish between people who have a medical need for treatment and those who have a cosmetic goal," a Vanderbilt professor who researchers pharmaceutical costs told STAT. "Understanding who gets these drugs and how much they pay will be important to watch."

hair loss

New drug for teens with alopecia areata

The FDA has approved Litfulo, a JAK inhibitor from Pfizer that can regrow hair in teenagers and adults with severe alopecia areata. This is the second such drug to win an FDA approval; Eli Lilly's JAK inhibitor Olumiant, or baricitinib, was green-lighted by regulators a year ago. Advocates are applauding Litfulo's approval, calling it significant advancement for the disease, particularly for teens — since this is the first time these drugs will be available for patients as young as 12.

"People living with alopecia areata are often misunderstood, and their experience is frequently trivialized as 'just hair,'" the CEO of the National Alopecia Areata Foundation said in a statement. "However, it is a serious autoimmune disease that can have considerable negative impact beyond the physical symptoms."

That said, JAK inhibitors interfere with inflammatory process in the body, and can elevate the risk of certain cancers, cardiovascular events, and severe infections.

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