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Geron's long-awaited chance, all-important obesity data, & one way to diversify trials

 

The Readout

Good morning, all. Damian here with news from a long-struggling biotech company, reason for caution on the drug industry's latest sure thing, and a little international intrigue.

After decades of disappointment, Geron gets a shot

Geron, a biotech firm that has spent the last 30 years without developing an approved drug, might finally change that after its experimental drug for a chronic form of blood cancer reached its primary endpoint in a late-stage clinical trial.

As STAT’s Jason Mast reports, Geron’s imetelstat helped about 40% of patients with myelodysplastic syndrome live free of blood transfusions for at least eight weeks, the company said yesterday. That beat the 15% observed in the placebo group, a statistically significant difference.

Now Geron’s future will rest in the hands of regulators, who will decide whether imetelstat’s benefits outweigh its significant risks. In the study, nearly two-thirds of patients on the drug saw their platelet counts drop dramatically, and just over two-thirds saw significant declines in infection-fighting immune cells.

Read more.

What if losing weight isn’t all that beneficial?

The projected $90 billion market for next-generation weight-loss medicines depends on the idea that lowering a patient’s body mass index will lead to the kind of long-term improvements in health that insurers would be willing to pay for. But what if it doesn’t?

We’ll find out later this year when Novo Nordisk reports data from a five-year, 17,500-patient study testing whether the obesity treatment Wegovy can reduce the number of cardiovascular problems relative to placebo. Decades of medical conventional wisdom suggest it’ll work, but there are no prospective, randomized trials establishing a link between obesity and cardiovascular disease. And there’s no guarantee the trial, which is relatively short for an outcomes study, will favor Wegovy.

The potential implications are massive. Wegovy is part of a new class of medicines called incretin mimetics which have proved to spur dramatic weight loss for patients diagnosed with obesity, leading analysts to forecast billions in future revenue. But without long-term data linking those effects to broader health improvements, payers will continue to limit their use, making Wall Street’s sales forecasts all but impossible.

International intrigue in NASH

Viking Therapeutics, a U.S. company developing a drug for the prevalent liver disease NASH, is suing a would-be Chinese partner, claiming the firm orchestrated a “ruse” to steal its trade secrets and rapidly develop a rival drug.

The lawsuit, filed last week, accuses Ascletis Pharma of offering to partner with Viking, signing a disclosure agreement to view the company’s trade secrets, and then using that intellectual property to design a NASH treatment of its own. “Rather than exploring a legitimate business opportunity with Viking, which was the express purpose of the [confidentiality agreement], defendants purposely misled Viking into revealing Viking trade secrets … to advance defendants’ secret development of a competing drug,” the lawsuit states.

In a statement issued yesterday, Ascletis said it had not yet been served with the lawsuit but, based on what’s publicly available, the company “believes that such allegations have no merit and will vigorously defend against the complaints.”

One way to diversify trials: pay up

There has long been evidence that paying people to participate in research can help diversify clinical trials. But a new study finds that if the amount is too small, compensation can have the opposite effect.

As STAT’s Usha Lee McFarling reports, researchers at the University of Chicago sent invitations for a Covid-19 study to people in zip codes that varied by both income and racial and ethnic makeup, randomizing them to offer no incentive, a $100 enticement, or the promise of $500.

With no incentive, the rate of participation was just 6%, and most respondents came from higher-income and white households. Curiously, when $100 was offered, overall participation increased to 17%, but the incentive widened the gap between groups: More than 20% of higher-income and white households participated, while participation of groups from lower-income and non-white households was about 10%. Offering a $500 incentive narrowed the gap almost completely; participation in all households increased to nearly 30%, which is considered a high rate for mail-in surveys.

Read more.

More reads

  • 3 challenges to watch in global health in 2023, STAT
  • Antibody drugs could target infectious diseases — if costs come down, Washington Post
  • Drugmakers raise prices on at least 350 drugs in U.S. in January, Reuters

Thanks for reading! Until tomorrow,

@damiangarde
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Wednesday, January 4, 2023

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