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A CRISPR milestone, Mylan's 'broken model,' & victory for the Broad

  

 

The Readout

Good morning, all. Damian here with news of a CRISPR milestone, an update on the former Mylan, and a consideration of "aggression" as a financial concept.

Intellia’s CRISPR drug impresses in preliminary results

Intellia Therapeutics’ CRISPR-based therapy for a genetic liver disorder reduced the levels of a disease-causing protein by 93% in a small study, the company said, surpassing earlier results reported last year.

As STAT’s Adam Feuerstein reports, the updated study results remain preliminary but provide further evidence that Intellia’s treatment, NTLA-2001, can be delivered through a one-time infusion and safely edit the damaged DNA of liver cells inside the body. 

They also represent a milestone for genome editing: Intellia is the first biotech company to report clinical data on a so-called “in vivo” CRISPR treatment, in which the editing is done inside the body rather than in a lab.

Read more.

It looks like the Broad Institute is about to make some CRISPR money

After years of legal drama, the fight over just who invented CRISPR genome editing appears to have ended. Yesterday, the U.S. Patent and Trademark Office ruled in favor of the Broad Institute of Harvard and MIT and against the University of California, Berkeley.

As STAT’s Megan Molteni reports, the PTO dismissed UC’s appeal of an earlier decision that went in favor of the Broad. UC can still appeal, but the likely outcome is that companies without a license agreement with the Broad — including Intellia Therapeutics and CRISPR Therapeutics — will have to negotiate one.

“This is pretty bad news for anyone who has a license for Berkeley’s technology,” said Jacob Sherkow, a patent attorney and professor at the University of Illinois College of Law who has closely followed the dispute. “They have over 100 patent claims that just got wiped off the face of the earth.”

Read more.

Depends on what you mean by ‘aggressive’

Aurinia Pharmaceuticals, maker of the approved lupus treatment Lupkynis, is providing biotech investors a lesson in semantics.

Last month, when asked about the company’s yet-to-be-announced sales projections for 2022, CEO Peter Greenleaf said Aurinia had no plans to be conservative and that Wall Street should expect “aggressive numbers.” Those projections came yesterday, and they were about 30% below analysts’ consensus, news that sent Aurinia’s stock price down roughly 25%.

What happened to the aggression? On a conference call with analysts yesterday, Greenleaf defended his usage, noting that Aurinia is predicting Lupkynis sales to grow about 200% in 2022, which strikes him as plenty aggressive. 

All is not well at the former Mylan

The company formerly known as Mylan is struggling to generate cash, so it sold off the part of its business that consistently generates cash. The reaction was not positive.

Viatris, formed by the 2020 merger of Mylan and Pfizer’s generics business, said yesterday that it would sell its biosimilars division to the Indian firm Biocon for about $3.3 billion, valuing the business at about four times its annual revenue. Viatris’ share price fell about 25% on the news, reflecting disappointment in the decision and the company’s projection that revenue would fall by about 4% in 2022.

Selling off biosimilars leaves Viatris with a shrinking baseline business of low-margin generics and over-the-counter products. To Cowen analyst Ken Cacciatore, the fact that Viatris is shaking things up suggests the company knows it’s presiding over a “broken model,” but “the assets, capabilities and competency are what they are, and we have no confidence that the transition will either be successful, or that this management team will be able to create any meaningful value from these levels.”

More reads

  • Pfizer made Trump’s vaccine push harder than it needed to be, former Warp Speed official alleges in new book. STAT
  • Pharma dealmaking hit by greater scrutiny of prices and competition. Financial Times
  • Strong evidence is becoming a business imperative for mental health tech startups. STAT+
  • Wall Street regulator to strengthen disclosures, boost transparency around short selling. Reuters

Thanks for reading! Until tomorrow,

@damiangarde
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Tuesday, March 1, 2022

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