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The Readout Damian Garde & Meghana Keshavan

We'll be live-blogging today's FDA advisory committee for the Johnson & Johnson Covid-19 vaccine. Check it out here.

Sarepta gets third Duchenne approval

Sarepta’s new drug for Duchenne muscular dystrophy, Amondys 45, won FDA approval yesterday. It’s the company’s third marketed treatment for the degenerative muscle disease — and is aimed at treating about 8% of patients with a specific mutation of the Duchenne gene.

When combined with Sarepta’s other Duchenne drugs, STAT’s Adam Feuerstein writes, the company’s therapeutics could treat about 30% of all patients with the rare disease.

Amondys 45, much like Exondys 51 and Vyondys 53, helps produce a small amount of the muscle protein dystrophin that’s missing in these patients. However, there’s no real evidence that more dystrophin actually improves muscle function in Duchenne, or slows the progression of the disease. Sarepta has been tasked with pursuing follow-on studies that show the patients actually benefit from the treatment, which will likely cost as much as $1 million per year.

Read more. 

Why can’t kids get Covid-19 vaccines?

Is there hope for biotech penny stocks? And when are feces medicine? 

We discuss all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. First, pediatric cancer advocate Nancy Goodman joins us to discuss why she believes Covid-19 vaccine trials need to be sped up for teens and children. Then, Wall Street Journal reporter Greg Zuckerman calls in to explain the unlikely story behind Novavax, a former biotech penny stock now on the verge of making history. Finally, we embark on a lightning round, featuring quick takes on the confirmation process for a new health secretary and the future of therapeutic stool.

Listen here.

Biologics patents suddenly tenuous

A new ruling may force biologics makers to re-examine their patent protections: A U.S. appeals court recently invalidated two Amgen patents for its cholesterol-lowering antibody drug, Repatha — favoring Sanofi’s claim that its own PCSK9 didn’t step on Amgen’s IP toes.

The ruling could have far-reaching implications for the broader pharmaceutical industry, STAT’s Ed Silverman writes, as it calls into question what kind of patent claims companies can make about any monoclonal antibody drugs.

“I think this is a huge deal,” one expert in life science patent law told STAT.

Read more.

Moderna CMO Tal Zaks stepping down in September

After spearheading the development of Moderna's Covid-19 vaccine, Tal Zaks, the company's storied chief medical officer, is set to leave later this year.

Zaks has been with Moderna for six years, helping the company grow from a mysterious unicorn into a biopharmaceutical powerhouse. It's unclear why he’s leaving now, or where he’s headed: Moderna disclosed his impending departure at the end of its quarterly financial report this week.   

What is clear is that Zaks is leaving a good deal richer than he was when he arrived.

More reads

  • FDA approves storage, transport of Pfizer-BioNTech vaccine at higher temperatures. (Reuters)
  • Merck to buy Cambridge autoimmune drug developer Pandion for $1.85 billion. (Beta Boston)

Thanks for reading! More next week,


Friday, February 26, 2021


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