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The Readout

Why wait for FDA approval to sell your drug?

Thanks to the recently enacted “right-to-try” law, patients have easier access to therapies not yet approved by the FDA — and companies have the right to charge them in exchange.

Enter BrainStorm Cell Therapeutics, which told Bloomberg that it plans to offer its investigational drug for amyotrophic lateral sclerosis for hundreds of thousands of dollars. That drug, NurOwn, failed to beat placebo in a phase 2 study. BrainStorm is pressing ahead into phase 3 with a narrower patient population and, thanks to right to try, is now set to become a “semi-commercial enterprise with modest profits,” Bloomberg reports.

To STAT’s Adam Feuerstein, Brainstorm’s precedent “is extremely dangerous and potentially exploitive,” creating a world in which “unscrupulous drug companies will be incentivized to skip the traditional clinical trial and FDA review process altogether.”

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What do you think?


Is BrainStorm CEO Chaim Lebovits right in saying that it’s OK to charge patients for unapproved therapies because companies “have to have an incentive” to develop drugs? Will “right-to-try” create an avenue for corporate fundraising that will eventually speed up the process of developing new drugs?

Or will it seed a generation of fly-by-night biotech companies able to squeeze dollars out of desperate patients while pressing forward with questionably efficacious drug candidates almost destined to fail in clinical trials?

This is fine so long as the companies are running confirmatory trials.

This basically nullifies the FDA and puts patients at risk.

The market doesn’t care about DimonBezosBuffetCare anymore


Back in January, when the capitalist Super Friends who run Amazon, JP Morgan, and Berkshire Hathway made their foray into health care, hospitals, PBMs, and insurance companies lost billions in market cap in a matter of hours. Yesterday, when the trio’s health care endeavor announced that famed surgeon Atul Gawande would serve as CEO, nothing happened.

The world’s health care incumbents — the ones Jeff Bezos and company were expected to disrupt — traded flat on the announcement. That’s perhaps because the appointment of Gawande suggests the group “is looking not at the drug value chain in isolation, but more broadly at the overall health care system across payers and providers of care delivery,” wrote Leerink analyst Ana Gupte. Which is to say the immediate risks to the likes of Aetna and Express Scripts are limited.

Anyway, what might Gawande actually do while in charge of this medicinal cerberus? STAT’s Megan Thielking and Andrew Joseph dug into his prodigious written output on a quest for clues, which you can find here.

Investors think Wave has crashed


Yesterday, we talked about how Sarepta Therapeutics' early results with a gene therapy for Duchenne muscular dystrophy could be bad news for Wave Life Sciences and its hopes of developing a drug for a segment of DMD patients. But if you believe a group of investors surveyed by Mizuho, Wave is already doomed.

Analyst Salim Sayed polled 40 investors, and 90 percent of them said Sarepta's gene therapy would be a first option for DMD patients, followed by Wave's chronic therapy in extreme cases. The idea that Wave's drug would be a first-line therapy is "the dumbest thing I have ever heard," one investor wrote. "Game over," read another comment, followed by "I think [Wave] is dead."

That doesn't exactly bode well for Wave, which has lost about a quarter of its market value since Sarepta's gene therapy data became public.

Attn: haters


Caption (Credit)

That's Cindy Eckert, former CEO of Sprout Pharmaceuticals, the company that introduced the world to a controversial and questionably effective female libido drug called Addyi. Addyi is back, as you may have read, and well now you've been warned.

More reads

  • Takeda’s CEO on China, cancer drugs, and Alzheimer’s disease. (Bloomberg)
  • Number of drug makers lobbying Congress on pricing issues skyrockets. (STAT Plus)
  • There go those drug companies inventing new diseases again. (Forbes)
  • Latecomer Sanofi looks to catch next wave of cancer therapies. (Reuters)

Thanks for reading! More tomorrow,


Thursday, June 21, 2018


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