The Readout Damian Garde

An awkward IPO roadshow

It’s not the easiest time to be a biotech company trying to go public. The whole sector has underperformed the market this year. Drug pricing is a daily fixture in national news. And there’s a persistent sense that private firms might not be worth as much as their venture capitalists like to think.

Beam Therapeutics, a genome editing startup pitching a $100 million IPO, already had to deal with all of the above. Then came Monday and the news that its scientific founder just went and launched a rival CRISPR startup.

That means Beam and its bankers are likely to face questions from investors about whether the founder, the Broad Institute’s David Liu, will face scientific and financial conflicts going forward. Then there’s the fact that Liu’s new company is employing an approach to CRISPR that appears more powerful than the one on which Beam was founded, a topic sure to come up in the company’s many investor meetings.

Read more.

Vertex gets good news ahead of schedule

Everyone expected Vertex Pharmaceuticals to win approval for its latest cystic fibrosis treatment. No one expected it to happen in 2019.

Yesterday, the FDA approved Vertex’s Trikafta just three months after the company submitted it for review. The decision is five months ahead of schedule and marks one of the quickest FDA turnarounds in recent memory.

With the addition of Trikafta, Vertex now has a genetically defined therapy for roughly 90% of all patients with cystic fibrosis. And it has an enviable financial position. The company already banks nearly $4 billion each year from its portfolio of cystic fibrosis drugs, and analysts expect that figure to approach $7 billion by 2025.

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Bayer’s CRISPR experiment comes to an end

Back in 2016, when seemingly every pharma company needed a play in genome editing, German giant Bayer went with CRISPR Therapeutics, launching a joint venture and promising to spend $300 million on its work.

That endeavor will soon be no more. The company, Casebia Therapeutics, is going to cease operations as an independent entity and get absorbed by CRISPR Therapeutics, according to The Boston Globe. Yesterday’s announcement came four days after Bayer officials declined to tell the Globe whether Casebia was still in business.

There’s a long history in biotech of major drug makers buying into a hot new technology — whether RNAi, mRNA, or smart contact lenses — only to bail after a few years when the early returns don’t go quite as imagined. But it’s unclear whether that’s what happened at Bayer. As the Globe points out, the company is in the midst of a large-scale restructuring in an effort to save money, and its future in genome editing could be a casualty of that.

Akebia versus United States

Akebia Therapeutics believes it’s being wronged. Medicare has declined to cover its only product, a treatment for anemia related to kidney disease, which has put a drag on revenue. Now, after 12 months of meetings and pleadings, Akebia is taking the government to court.

As STAT’s Nicholas Florko reports, the company is suing CMS over its decision to consider Akebia’s drug, called Auryxia, as a quotidian mineral rather than a pharmaceutical product. The company argues that CMS violated the Administrative Procedure Act in excluding Auryxia from coverage, and the matter will eventually go before a judge.

“Patients deserve better,” Akebia CEO John Butler said. “The president said patients deserve better. His administration is not living up to that commitment and that’s why we had to bring suit.”

Read more.

More reads

  • How Joe Grogan, a former pharma lobbyist, upended Trump’s drug pricing agenda. (STAT)
  • Seattle Genetics breast cancer pill delayed tumor growth in Phase 3 trial. (STAT Plus)
  • Baker Brothers makes $730 million in one day on its biotech bet. (Bloomberg)
  • New CRISPR tool has the potential to correct almost all disease-causing DNA glitches, scientists report. (STAT)

Thanks for reading! Until tomorrow,

Tuesday, October 22, 2019


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