October 18, 2023

The need-to-know this morning

BrainStorm Cell Therapeutics withdrew its FDA application for NurOwn, the company's investigational treatment for ALS, and acknowledged it will need to conduct another pivotal trial.
Activist investor Carl Icahn is suing Illumina, claiming the company breached its "fiduciary duties" in acquiring Grail for more than $7 billion.
Ardelyx won FDA approval for its chronic kidney disease treatment, a drug that had previously been rejected.

Markets

Are we sure GLP-1s are going to reshape global capitalism?

It made sense, at least arguably, when the rise of medicines like Ozempic sharply shifted biotech stocks. And you could make a case, over some protest, that the recent crash of dialysis companies was warranted.

But now analysts from every corner of Wall Street are cranking out research notes on the disparate, dramatic, and often debatable implications of GLP-1 drugs' growing popularity. Buy Bumble, sell McDonald's. Short Pepsi, go long Louis Vuitton. Put your money in sectors that cater to a svelte and sated brand of consumer, and get out of the ones that rely on excess and compulsion.

"The baseline thesis is a future in which everyone is skinny and has no appetite — basically every other person looks like Christy Turlington," Mizuho analyst Jared Holz said. "And if that's the case, then you don't want to be owning some of these stocks."

The only problem, Holz and other analysts said, is that the baseline thesis doesn't make a lot of sense.

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Regulatory

The case against Cassava

The years-long debate over Cassava Sciences and its investigational treatment for Alzheimer's disease took a turn last week, when an independent investigation found evidence "highly suggestive of deliberate scientific misconduct" in the drug's development.

Now, STAT's Adam Feuerstein writes, the FDA needs to intervene. Cassava is in the process of running two Phase 3 studies on its medicine, called simufilam, which means patients are currently receiving an unapproved product that resulted from what appears to be an alarmingly questionable development process.

"Patient safety is paramount to the FDA, so stopping Cassava's two Phase 3 studies is the ethically correct thing to do," Adam writes. "The FDA has the power to place clinical holds on experimental drugs when there's evidence of an unacceptable safety risk to study participants."

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In the lab

Wegovy doesn't work for everyone, and no one's quite sure why

The well-publicized effects of GLP-1 treatments — 15% to 20% weight loss in clinical trials — are just averages, meaning some people lose quite a bit more, and some don't lose much at all.

As STAT's Elaine Chen reports, it remains unclear just why there's so much variation, and researchers are probing human genetics in hopes of finding some clues.

Because genetics play a significant role in whether people develop obesity, and because early data also show that genetics may contribute to how people respond to bariatric surgery, "that would argue that there's probably going to be a genetic contribution" to the amount of weight loss people experience on obesity drugs, said Lee Kaplan, director of the Obesity, Metabolism, and Nutrition Institute at Massachusetts General Hospital.

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R&D

Nkarta's pivot is going over well

Shares of the biotech company Nkarta more than doubled in value yesterday after the company said it would reduce its spending and press forward with a cell therapy designed to treat lupus.

The news is that NKX019, an off-the-shelf cell therapy, won the FDA's blessing to enter a Phase 1 trial enrolling patients with systemic lupus erythematosus, an autoimmune disorder. At the same time, Nkarta said it would lay off an undisclosed number of staff members and implement "cost containment measures" that will extend its cash runway into 2026.

The lupus program opens a new front in Nkarta's efforts to turn immune cells called natural killer cells into powerful medicines. The company is developing similar therapies for types of leukemia and lymphoma.

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