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The Readout Damian Garde & Meghana Keshavan

Your guide to the coming quarter in biotech

Over the next three months, we’re going to find out whether the next wave of Covid-19 drugs have a shot, how the FDA feels about the first gene therapy for hemophilia, and whether, at last, Biogen is going to keep its promise in Alzheimer’s disease.

STAT rounded up all the major biotech events in the cards for the third quarter. That includes a bevy of expected data on treatments and vaccines for the novel coronavirus and a pair of weighty regulatory events that, were there no pandemic, would be the talk of the industry.

First, BioMarin is awaiting an FDA decision on what would be the first one-time treatment for hemophilia A. The company has mapped out a strategy for world domination in the competitive space, but the first step is convincing regulators that the benefits of its gene therapy outweigh the risks. Then, Biogen has pledged to submit its long-delayed Alzheimer’s treatment for approval in the third quarter. Whether it can meet that timeline has sweeping reputational importance for the company.

Read more.

When are we getting that coronavirus vaccine data?

Speaking of long-awaited data, we still don’t know just how well Moderna’s investigational coronavirus vaccine works.

As you might recall, Moderna issued a press release worth $1.3 billion that described, in qualitative terms, the early returns from its vaccine. The full data would be published in a matter of “weeks, not months,” Moderna CEO Stéphane Bancel said one month and two weeks ago

The timing depends not on Moderna but on its partners at the NIH, which ran the Phase 1 study. An NIH spokeswoman said there's no set timeline for publication. But there’s more than a little immediacy. Some time in July, Moderna plans to start enrolling about 30,000 people in a Phase 3 trial. As it stands, those potential volunteers have only a press release of information to go on.

The FDA might have shifted the goalposts in NASH

Yesterday, Intercept Pharmaceuticals revealed that the FDA rejected its treatment for NASH, a prevalent fatty-liver disease. But the implications could go far beyond a single company, changing the definition of success in one of the drug industry’s biggest targets.

As STAT’s Adam Feuerstein reports, Intercept’s drug significantly reduced liver scarring, a hallmark of NASH, in a pivotal trial. But that wasn’t enough to convince the FDA, which concluded that the “predicted benefit” of the company’s drug “does not sufficiently outweigh the potential risks,” Intercept said in a statement.

That could mean that the FDA is unwilling to approve NASH treatments based on scarring alone, which would affect a multitude of drugs in development. Conversely, the rejection could stem from an issue specific to Intercept’s drug. Until there’s more information, no one can be sure.

Read more.

Gilead could live to regret its pricing decision

Yesterday, Gilead Sciences set a price for the Covid-19 treatment remdesivir that is “well below” its actual value, according to the company’s CEO, in order to ensure “broad and equitable access.” To activists, that’s a tacit admission that Gilead has been overcharging for years. To investors, it’s a concession that could undermine the company’s business. And to the industry at large, it’s a precedent that might change how drugs are priced.

SVB Leerink analyst Geoffery Porges warned that Gilead’s decision “will change all future expectations for the treatment of this disease and may also influence pricing in other diseases.” If drug companies change their definition of fair value for Covid-19, why not diabetes, cancer, or Alzheimer’s disease?

What’s worrisome to Porges is welcome news to James Krellenstein, co-founder of the HIV-prevention advocacy group PrEP4All. Gilead’s decision proves that drug companies can make ample profits without charging the maximum the market will bear, making remdesivir an opportunity to put pressure on the industry.

Read more.

More reads

  • A new FDA tool aims to inform cancer care, but experts say it has a glaring gap. (STAT)
  • CanSino's Covid-19 vaccine candidate approved for military use in China. (Reuters)
  • A little-known biotech working on a Covid-19 vaccine has surged 304% in two days. (Business Insider)
  • Trouble in paradise: The Senate’s bipartisan accord on drug prices is broken, says Sen. Chuck Grassley. (STAT)

Thanks for reading! Until tomorrow,

Tuesday, June 30, 2020

STAT

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