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The Readout Damian Garde

So good we're sending this twice: This time as "The Readout" instead of "Morning Rounds," an equally great newsletter. Apologies for the earlier misfire.

The price of Zolgensma vs. the price of a child's life



Tina Anderson’s son, Malachi, was born with the most severe form of spinal muscular atrophy. 

“You can take him home, love him, take lots of pictures,” she remembers doctors saying, “but he won’t make it to his first birthday.” 

But thanks to Zolgensma, the $2.1 million gene therapy that was recently approved to treat this devastating disease, Malachi will soon celebrate his fourth birthday. Despite the controversy around the therapy’s stunningly high cost, Anderson is grateful that it’s an option available for families like her own. 

“To me, you cannot put a price on your child’s life,” Anderson, who got her son into a clinical trial for the treatment, told STAT’s Damian Garde. “If tomorrow we were told to pay back everything, we would. We would figure it out. Because our son is now alive and well.” 

Read more.

Moderna (finally) shows data on its cancer vaccine

After years of deep stealth and billions in capitalization, Moderna Therapeutics finally has released some data. It’s early stage, to be sure, but the company’s personalized cancer vaccine — which was administered alone and in conjunction with Keytruda — appears to have helped the patient’s body recognize and attack the unique proteins populating the outside of tumor cells. 

Moderna now will move on to kick off a Phase 2 trial with its partner, Merck, enrolling 150 patients with melanoma that’s been surgically removed. The idea here is to delay relapse.

Moderna’s cancer vaccine is tailor-made to an individual patient’s tumor. The entire manufacturing process, which involves tumor biopsy, genetic sequencing, vaccine building, and administration, takes about two months. 

Read more.

Impressive breast cancer results may or may not change protocol

Novartis breast cancer drug Kisqali impressively extends breast cancer survival among young women who develop the most common form of the disease, a new JAMA study shows. Adding Kisqali to hormone-blocking therapy resulted in a 29% reduction in death. After three and a half years of treatment, about 70% of women receiving the combination therapy survived, compared to just 42% who received standard therapy alone. 

Kisqali is a CDK 4/6 inhibitor — a relatively new class of breast cancer drugs. Pfizer’s Ibrance is a similar drug, and brought in some $4.1 billion last year; Kisqali, on the other hand, had sales of just $235 million. These results may or may not change standard of care, and increase Novartis sales of the drug. Trials of CDK 4/6 inhibitors in breast cancer “have all showed very similar results,” one breast cancer oncologist told STAT’s Matt Herper. “To a first- and second-order analysis, the drugs are interchangeable in terms of clinical efficacy as far as we can tell.”      

Read more.


On the heels of ASCO, another staple industry confab kicks off: BIO begins this week in Philadelphia. One notable change? Digital health companies are increasingly taking center stage, STAT’s Kate Sheridan writes, underscoring just how serious biopharma takes tech. 

Meanwhile, an annual controversial biotech bash is scheduled yet again. The infamous “Party at BIO Not Associated with BIO,” or PABNAB, which last year drew ire for featuring topless dancers with company logos painted on their bodies, has a “Gladiators and Goddesses” theme this year. Nothing debasing could happen at a glorified toga party… right?

Read more.

More reads

  • Merck's Keytruda lifts survival rates for lung cancer. (Financial Times)
  • Grail shows impressive, if early, results for cancer blood test. (STAT)
  • The Affordable Care Act erased a racial disparity in health care, a new study says. (STAT)
  • AstraZeneca's dynamic duo wants to dominate the market for cancer drugs. (STAT)

Thanks for reading! More tomorrow,


Monday, June 3, 2019


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