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The Readout Damian Garde & Meghana Keshavan

How do you describe a one-in-a-million risk?

What’s heparin-induced thrombocytopenia? And why’s it so hard to find drugs for Covid-19?

We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. First, Science Magazine reporter Kai Kupferschmidt joins us to discuss the U.S. decision to press pause on the Johnson & Johnson Covid-19 vaccine and what researchers have learned about rare cases of dangerous clotting. Then biotech analyst Josh Schimmer talks with us about why the drug industry has had such a hard time coming up with treatments for Covid-19 and offers some tips that might help in the next pandemic.

Listen here.

FDA wants more data on a promising ALS drug, frustrating patients

The Food and Drug Administration wants Amylyx Pharmaceuticals to run another clinical trial of its treatment for ALS, declining to consider approving the treatment based on the results of a single, positive clinical trial completed last September.

As STAT’s Adam Feuerstein reports, the news is troubling to patient advocates, who have long pressured the agency to speed up the regulatory process for ALS drugs. They argued that Amylyx’s study, which demonstrated a modest but statistically significant benefit over placebo, provides enough evidence for the FDA to consider granting an approval.

Adding to their frustration, regulators in Europe and Canada have agreed to review the drug, called AMX0035, based on the single study. That means ALS patients in other countries around the world could have access to AMX0035 as early as next year while their counterparts in the U.S. await data from a Phase 3 trial.

Read more.

Activists have come for GSK

The hedge fund Elliott Management, famed for its shareholder activism, has amassed a multibillion-dollar stake in GlaxoSmithKline, the Financial Times reported yesterday, news that could portend a public battle for the storied British drug maker.

Elliott’s move comes at an awkward time for GSK, which is in the process of spinning out its consumer products division to focus on drugs and vaccines. That’s exactly the sort of corporate mitosis activist investors tend to agitate for, making it unclear what changes Elliott might have in mind.

In any case, it was warmly received by investors, who sent GSK’s stock up about 8% yesterday. The company began 2021 with an ambitious plan to launch 10 blockbuster medicines over the next five years, a goal that came under immediate strain when two investigational cancer drugs ran into clinical setbacks. As SVB Leerink analyst Geoffrey Porges wrote in a note to investors yesterday, that puts GSK in the unenviable situation of needing to buy something but lacking the balance sheet to do it.

Remember Samumed?

As recently as 2018, it was a $12 billion company with plans to cure baldness and reverse arthritis, and it counted IKEA and Gary Cohn among its investors. Three years later it has a new name, a new CEO, and $120 million in new financing.

It’s now called Biosplice, and former Chief Financial Officer Cevdet Samikoglu has taken over for CEO Osman Kibar, who remains executive director. The latest funding, announced yesterday, comes from a more biotech-involved syndicate than the investors that got Samumed to its sector-leading valuation. Biosplice didn’t disclose whether it was able to sell shares at the same price it commanded in 2018.

Either way, it would appear things have not gone entirely to plan. Three years ago, when the then-Samumed raised $438 million, Kibar told Endpoints that the money would pay its way to a first FDA approval, “plus launch cost, plus some extra cushion.” Instead, Biosplice’s arthritis medicine is in a pair of Phase 3 trials that are no longer recruiting patients. The company hasn’t said when it expects data.

More reads

  • Next-generation Covid-19 vaccines are supposed to be better. Some experts worry they could be worse. (STAT)
  • U.S. preparing for 1-year Covid-19 booster shots. (Reuters)
  • Thermo Fisher CEO sees PPD buy as ‘all about speed’ in new drugs. (Bloomberg)
  • International team creates first chimeric human-monkey embryos. (STAT)

Thanks for reading! Until next week,

Friday, April 16, 2021

STAT

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