Readout @ JPMDamian Garde

Hello again from #JPM20! Welcome back to a special edition of The Readout. Follow STAT's reporters on the ground: @adamfeuerstein, @damiangarde, @rebeccadrobbins@erbrod@pharmalot, and @matthewherper.

BioMarin’s plot to dominate gene therapy

JJ Bienaimé, the less-than-bashful CEO of BioMarin Pharmaceutical, has a less-than-bashful vision for his company: His company will win the arms race in gene therapy.

Thanks to years of infrastructure investment, BioMarin can now churn out 10,000 commercial-grade doses of gene therapy a year, something “nobody else is able to do in the field,” Bienaimé told STAT. In practical terms, that means valrox, BioMarin’s gene therapy for hemophilia A, could treat every eligible patient in the U.S. within about two years of winning approval, according to Bienaimé. If that approval comes later this year, as expected, BioMarin has a chance to seize the market before competitors Roche and Pfizer can sell a single infusion, he said.

And that’s just the first step. Bienaimé looks at BioMarin’s gene therapy division as a perpetual motion machine. Everything the company learned about making valrox went into its next gene therapy, targeting the rare disease phenylketonuria, and will be applicable to one-time treatments yet to come.

The backward DNA molecule above Union Square


(Adam Feuerstein/STAT)

You can’t miss it if you’re in the heart of San Francisco’s Union Square this week: a towering billboard urging conference goers to “discover health innovation in the UK.” It’s the kind of prime placement — no doubt costing a pretty penny — designed to make a statement to biotech insiders. 

The only problem: The molecules of the DNA pictured in the billboard are left-handed, or turned in the wrong direction. D-oh!

We asked the U.K.’s international trade department how the goof happened but haven’t heard back. 

AstraZeneca's fish oil drug fails

Amarin’s Vascepa is the last fish oil-derived drug standing after rivals AstraZeneca and Acasti Pharma suffered separate, significant setbacks on Monday. 

As Matt Herper reports, AstraZeneca was forced to stop a large, cardiovascular outcomes clinical trial of Epanova after an independent committee concluded it was “unlikely to demonstrate a benefit to patients.” Separately, a study of Acasti’s fish-oil drug failed to lower triglyceride levels compared to a placebo. 

Amarin benefits from its competitors’ misfortunes, but the stock only rose marginally because investors remain concerned about the possibility of generic versions of Vascepa reaching the market. Amarin is suing to stop that from happening, but a decision won’t be known until later this year.

Read more.

M&A withered

Monday at JPM came and went without significant buyouts or deals, and so, biotech stocks fell. The sector’s most closely followed stock index was on track to close down 2%. Gene therapy developer Uniqure — high on many investors’ takeout list — was among the sector’s biggest Monday losers. 

Logically, the absence of a large M&A deal during a single, 24-hour period means very little over the course of an entire year. If buyouts come to fruition next week or next month, no one will care about a snoozy JPM Monday. But biotech investing isn’t always logical. Sentiment often drives the trading narrative. And on Monday, healt hcare investors — tipsy on high expectations — were bummed out and selling stocks. 

Gilead’s M&A roadmap

During a 30-minute breakout session with investors, Gilead Sciences CEO Dan O’Day and CFO Andy Dickinson offered more details about the biotech’s plans to buy or partner with others. 

Does Gilead have plans to make acquisitions? Yes, said O’Day, although deals will be small to medium-sized — what he described as “bolt-on acquisitions.” Those hoping for Gilead to buy Vertex Pharmaceuticals will be disappointed. What types of smaller biotech companies might Gilead buy? O’Day said Gilead plans to stick with its strengths, meaning antivirals, immuno-modulation, and oncology. Gilead is not buying Amarin — sorry, Amaroids. 

In oncology, specifically, Gilead intends to focus partnerships and acquisitions on companies that compliment its existing Kite Pharma cell therapy business, O’Day said. That means Gilead isn’t interested in buying companies developing small molecules or targeted cancer drugs.

What about partnerships? Here, CFO Dickinson said Gilead has “transformational” intentions.  “We would like to do at least one more Galapagos-type partnership in the next couple of years,” said Dickinson, referencing the $5 billion tie-up announced last summer. “You can’t do a lot of those, but they deliver value over time.” 

More reads

  • Eyeing more data, Livongo will integrate continuous glucose monitor into diabetes coaching program (STAT Plus)
  • Biogen to buy early-stage potential Alzheimer's, Parkinson's treatment from Pfizer (Reuters)
  • FDA and NIH let clinical trial sponsors keep results secret and break the law (Science

Thanks for reading! More tomorrow.

Monday, January 13, 2020


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