Wednesday, April 18, 2018

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of the latest in biotech. For more in-depth coverage of biopharma, subscribe to STAT Plus. On Twitter: @damiangarde@megkesh, and @statnews.

How much does Ultragenyx's new drug actually cost?

When drug companies win FDA approval, they tell the world the list price of their new drug and then trip over themselves to explain that almost no one will pay that full price thanks to the bramble of middlepeople who stand between manufacturers and patients.

Ultragenyx did it differently. When the company won approval for its second rare disease treatment yesterday, it disclosed that the list price of 10 mg of the drug would cost $3,400, and then it outlined a bunch of math and started estimating net prices. For the average child, the net price will be around $160,000 per year, the company said, and for adults, it will reach about $200,000 per year.

That’s interesting because net prices reflect rebates and discounts that are usually confidential and inconsistent from payer to payer. By coming right out and saying something other drug companies only hint at, Ultragenyx is conceivably giving up negotiating power with the institutions that will decide whether its drug is worth the money.

And where’d they get those net price numbers?

“We're not providing background details on our calculations,” CFO Shalini Sharp told analysts on a conference call, “but I can assure you we're making very appropriate assumptions based on what we've seen in clinical trials.”

What happened to that Alzheon IPO?

Remember Alzheon? It's a small company that believes a once-failed treatment for Alzheimer's disease can be refashioned into a winner with a few tweaks and a lot of money.

The last version of that story didn't end particularly well, and now Alzheon's reboot appears to be struggling to find an audience.

In March, the company filed to raise $100 million in an IPO. In April, it went out on the requisite roadshow in which biotech companies make their case to investors from I-95 to Sandhill Road in hopes of actually going public.

And then came word the IPO would price last week. And then nothing. And then the company didn't respond to a request for comment yesterday.


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What it's like to ‘hit a tree’ on the road to biotech riches

April has been a cruel month for Incyte. The company found out that epacadostat, its star cancer drug, was essentially useless when added to blockbuster immunotherapy, a late-stage failure that cut about $4 billion out of its market cap.

But life goes on in the retrofitted Delaware shopping mall Incyte calls its headquarters.

“When you are the first with a new mechanism, you are the one doing the trailblazing and you can hit a tree in the process,” CEO Hervé Hoppenot told Bloomberg, pivoting from his earlier penchant for nautical metaphors.

And yet there could be more trees ahead. Incyte’s last sure thing, an Eli Lilly-partnered treatment for rheumatoid arthritis, got rejected by the FDA last year, and now the two companies have to hope their renewed pitch can win over regulators this summer. 

What's going to happen with GW Pharma?

The next big catalyst in biotech belongs to GW Pharmaceuticals, a company poised to win the first-ever FDA approval for a cannabinoid drug.

That's if it wins over the FDA.

Yesterday, the agency released briefing documents suggesting that regulators see no major issues with GW's drug, meant to treat a rare seizure disorder. And, considering the company's stock price rose 12 percent the same day, Wall Street seems confident that GW will skate through the pre-approval panel discussion slated for Thursday.

What do you think? How will that panel of FDA advisers vote on GW's drug?

Unanimously positive
Split, but positive
Split, but negative
Unanimously negative

More reads

  • Trade secrets battle between Alnylam, Dicerna is just one of many. (Boston Globe)
  • FDA official insists U.S. is catching up to Europe on biosimilar regulations, approvals. (STAT Plus)
  • Someone hacked into a Sangamo exec's email 11 weeks ago. (SEC)
  • Coke versus Pepsi of drug makers? Wall Street doesn't buy it. (Bloomberg)

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Thanks for reading! Until tomorrow,


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