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The Readout Damian Garde & Meghana Keshavan

A pandemic means society needs information fast. Can researchers provide it? Join STAT for a conversation about the role of real-world evidence in the Covid-19 crisis, featuring Deputy FDA Commissioner Amy Abernethy and Carolyn Magill, CEO of Aetion. The event is Thursday, Sept. 17, and you can sign up here.

FDA rejects Gilead’s blockbuster-in-waiting

Back before remdesivir became a household name, Gilead Sciences’ most-discussed drug was filgotinib, a treatment for inflammatory disease expected to bring in more than $1 billion a year by 2025. Last night, that all got put in jeopardy.

The FDA rejected Gilead’s application to sell filgotinib as a treatment for rheumatoid arthritis and said it wouldn’t reconsider until the company can provide more data on the drug’s effect on patients’ sperm counts. Gilead is running two studies to determine exactly that, but it won’t have results until some time next year.

The surprise rejection imperils Gilead’s long-term goal of building filgotinib into a major medicine. The plan was to start with rheumatoid arthritis and follow it with approvals in inflammatory bowel disease, psoriatic arthritis, ankylosing spondylitis, and eye inflammation. Until the FDA signs off on the drug’s safety, all of that is up in the air.

There’s probably going to be a new most expensive drug ever

By the end of the week, the FDA is expected to approve BioMarin Pharmaceutical’s gene therapy for hemophilia A, a one-time medicine that promises to revolutionize treatment of the rare bleeding disorder. It also promises to cost millions of dollars, reinvigorating the years-long conversation about how to value life-changing medicines.

The treatment, called Roctavian, demonstrated a 95% reduction in bleeding episodes in a four-year trial, and it also led to a 96% improvement in patients’ need for drugs that improve blood clotting. That latter point is important. Treating hemophilia A generally costs about $300,000 a year for a lifetime, a figure that doesn’t include the hospitalizations and surgeries many patients come to need. 

BioMarin hasn’t committed to a price but has said its gene therapy would be cost-effective at $3 million. That would make it the most expensive drug in the world, topping the $2.1 million cost of Novartis’s gene therapy Zolgensma. BioMarin believes it has enough data to make the case to payers that Roctavian is a long-term bargain. We’ll find out once the treatment is approved.

The avoidable dilemma facing Covid-19 vaccines

The drug industry is racing forward with potential vaccines for the novel coronavirus as governments around the world game plan for how to deploy them. But there’s a glaring hole in the process that could prove problematic.

As STAT’s Helen Branswell reports, none of the Covid-19 vaccine trials have included pregnant women or women who are breastfeeding, and only one of the vaccine makers that may end up supplying the American market, AstraZeneca, has started to test its vaccine in children.

That means if and when a vaccine succeeds in clinical trials, authorities might not yet know whether it works in these vulnerable populations. And experts warn time is getting tight to start finding those answers.

Read more.

Regeneron is tripling its footprint to prepare for success in Covid-19

If Regeneron Pharmaceuticals’ antibody treatment for Covid-19 works, millions of people are going to want it. Rather than waiting to find out, the company has signed a deal with Roche that will dramatically expand its manufacturing capacity to meet the potential demand.

Under the agreement, announced this morning, Regeneron expects to be able to make at least three and a half times as many doses of REGN-COV2, an antibody cocktail now in clinical trials with preliminary data expected next month. If the treatment works, Regeneron will handle distribution in the U.S., and Roche will preside over the rest of the world. In the meantime, the companies will split the costs of clinical development.

"It’s kind of an unusual deal," said Regeneron CEO Leonard Schleifer, "because your typical biotech deal, if this was a [contract manufactuer], we would be paying them money to do tech transfer, or if this were a typical licensing deal they would be paying us a big upfront. But this is a deal where both parties sort of realized that together we can serve more people in the U.S. and more people around the world with what we hope will be an important weapon in this pandemic fight."

Experts have endorsed the potential of therapeutic antibodies for Covid-19, but only if the drug industry can produce enough doses to make a difference in the pandemic. Regeneron’s agreement with Roche is designed to address that very concern.

Have we reached peak SPAC?

Biotech’s latest financial trend is the blank-check company, a publicly traded shell created to buy private firms and grant them Nasdaq listings without the trouble of an IPO. The latest one, from Casdin Capital and Corvex Management, is the fifth to launch this summer alone. It's called CM Life Sciences, and it's planning to raise $350 million to fund a future merger.

But considering the remarkable recent success of old-fashioned IPOs, are we sure there’s enough demand for all these blank checks?

To investors, the appeal of special purpose acquisition companies, or SPACs, is that a blue-chip fund like Casdin is likely better at evaluating private companies than the average stock picker looking at IPOs. And to the companies themselves, merging with a SPAC provides a path to the public markets that doesn’t involve hiring bankers and lawyers and spending months making the same pitch.

But each of those cases might be undermined by the current market for biotech companies. This year has already broken the all-time record for IPO proceeds, and the average company has traded up about 30% after its debut. That trend might convince would-be SPAC targets to go public on their own. And if the most promising companies see an IPO as the better path, SPACs might find themselves in a market for lemons, choosing between firms with no other options. That might scare off investors and deflate the SPAC bubble before it can really take off.

More reads

  • FDA finalizes penalties for clinical trial sponsors that don’t share data, but critics say they fall short. (STAT Plus)
  • ‘You can’t fix stupid’ — Trump trade advisor rips Kodak executives as dumbest ever. (CNBC)
  • Virtual event: Cracking Covid-19’s code with AI. (STAT)

Thanks for reading! Until tomorrow,

Wednesday, August 19, 2020

STAT

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