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The Readout Damian Garde & Meghana Keshavan

Are we sure Moderna’s worth $23 billion?

Back in January, the erstwhile unicorn Moderna, despite having no marketed products, carried the fairly controversial market value of $6 billion. Five months and one global crisis later, the company’s worth has nearly quadrupled. But do the numbers add up?

As STAT’s Adam Feuerstein explains, Moderna’s staggering rise relies on a slew of assumptions that have some investors nervous. For one, the company’s early-stage coronavirus vaccine has to actually work, which is no small feat. But then Moderna has to make a successful product out of it, which will involve global production, negotiating with governments, and dealing with the very real possibility of competition from larger companies.

And even if all of that works out, there are even more questions.

Read more.

MyoKardia’s heart drug looks like a ‘game changer’

In a pivotal trial, a drug from MyoKardia significantly improved the symptoms of a dangerous, inherited heart disease, lighting the way for FDA approval.

As STAT’s Matthew Herper reports, MyoKardia’s treatment improved blood oxygen capacity for patients with hypertrophic cardiomyopathy, a progressive disease that reduces the heart’s ability to pump blood throughout the body. The data, which sent MyoKardia’s share price up more than 55% yesterday, are yet to be published or reviewed by outside experts.

If the results hold up, it would spell “a potential game changer” for patients with hypertrophic cardiomyopathy, said Harlan Krumholz, a professor of cardiology at the Yale School of Medicine who was not involved with the study. The current standard of care is a surgery that comes with significant risk of complications and death.

Read more.

Genentech’s next top scientist isn’t averse to math

Aviv Regev, a pioneering researcher in the field of computational biology, will be Genentech’s next head of R&D, becoming one of the very few women directing science at a major drug company.

As STAT’s Elizabeth Cooney reports, Regev will depart her post at the Broad Institute of MIT and Harvard to join Genentech Aug. 1. In her roughly 14 years at the Broad, Regev was instrumental in bridging the worlds of data science and biology. She is part of a group that created the Human Cell Atlas, an international collaboration of scientists working to understand all cell types in the human body, a process that weds machine learning with fundamental biology.

Leaving academia for industry, Regev said she wants to translate the kinds of discoveries that marked her career at the Broad into products that can help patients. “I have realized that the blend of deep biology, sophisticated computation, and new ways to marry the two — should now complete its arc with patient biology and therapies,” Regev wrote in an email to colleagues.

Read more.

Long-delayed NASH data turns out negative

After months of delays with confusing explanations, French drug maker Genfit finally revealed data on its treatment for the prevalent liver disease NASH. And the data are bad.

In a study enrolling about 1,000 patients, Genfit’s drug failed its primary goal of resolving the inflammatory symptoms of NASH without worsening fibrosis, the liver scarring that results from the disease. The response rate wasn’t meaningfully different from placebo, and neither was its improvement in fibrosis nor its effect on metabolic symptoms.

In other words, Genfit’s drug was a total miss, which should winnow the competitive field in NASH, widely considered a blockbuster opportunity. The next big NASH news will come June 9, when a panel of FDA advisers will vote one whether a drug from Intercept Pharmaceuticals deserves approval. 

More reads

  • A secret experiment revealed: In a medical first, doctors treat Parkinson’s with a novel brain cell transplant. (STAT)
  • Dyno spins out of Harvard's laboratories with an aim to make gene therapy better. (BioPharma Dive)
  • Biotechnology experts who understand both science and investing are skeptical of the stock market’s strength. (MarketWatch)
  • New research sheds light on sex imbalances in diseases like schizophrenia and lupus. (STAT)

Thanks for reading! Until tomorrow,

Tuesday, May 12, 2020

STAT

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