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The Readout Damian Garde

At Novartis, who knew what — and when?

Yesterday's revelation that Novartis's much-vaunted gene therapy approval was based, in part, on falsified data was a shocking turn for a company in the midst of a self-described culture change. And for Vas Narasimhan, the CEO presiding over the Swiss company's evolution, the news raises uncomfortable questions.

The gene therapy is Zolgensma, acquired in an $8.7 billion deal meant to set the tone for Narasimhan’s tenure. According to the FDA, people inside Novartis became aware of data manipulation as early as March, but no one informed regulators until late June, more than a month after Zolgensma won approval.

Now Narasimhan, who has enjoyed a rising profile in an industry otherwise beset by criticism, needs to explain who knew what, and when.

Read more.

CAR-T is expensive. CMS is still figuring things out

The FDA approved the first CAR-T treatments, Novartis’s Kymriah and Gilead’s Yescarta, in 2017, but CMS is still struggling to find a way to pay for these cutting-edge, costly therapies.

The missteps have been many: The agency still doesn’t have a uniform policy for covering these therapies; it's balked at shelling out extra cash to make hospitals whole; and it was forced in July 2018 to walk away from a closely watched deal to pay for Kymriah only when it works as intended.

CMS Administrator Seems Verma told STAT's Nicholas Florko that she, too, is frustrated by the government’s inability to figure out how to pay for these treatments, and she warns it could take years before things are finally smoothed out. “It’s a great microcosm into the complexities of government price setting,” she told STAT.

Read more.

Editas has a permanent CEO again

It's official: After serving as Editas Medicine's interim CEO and president since January, biotech veteran Cynthia Collins has been appointed to the jobs "permanently," board chair James Mullen said in a statement on Tuesday.

The move comes less than two weeks after Editas began enrolling patients in a clinical trial of its lead drug candidate, which uses CRISPR-Cas9 to treat a form of congenital blindness. As the first trial using CRISPR to edit the DNA of patients inside their body (rather than cells isolated from blood, CRISPR'ed in the lab, and then returned), it's being watched with equal parts hope and fear as a harbinger of whether CRISPR can live up to sky-high expectations.

Collins, who in a statement praised Editas's "limitless potential," was previously the CEO of Human Longevity and Clarient Diagnostics, with turns at Beckman Coulter and Sequoia Pharmaceuticals.

Why is Bluebird sitting on results of a CAR-T drug critical to its future?

For the past year, Bluebird Bio has been tracking long-term outcomes from multiple myeloma patients treated with its experimental CAR-T therapy called bb2121. So, why haven’t those data been disclosed publicly?

To do so would be a “distraction to the team,” said Bluebird CEO Nick Leschly, referring to the company’s employees. Not everyone agrees. As Adam Feuerstein notes, the success or failure of bb2121 will depend on its ability to keep patients in remission and prolong survival.

By sitting on these important data, Bluebird is raising questions about bb2121’s future.

Read more.

Patient charities favor the insured and cover costlier drugs

Patient assistance programs are designed to do just that: help people obtain the medicines they need. But a new JAMA study finds that last year, a whopping 97% of such programs run by the six largest organizations didn’t help people who had no insurance. And the drugs they did cover were more likely to be expensive brand-name medicines than generic versions.

Not every program disclosed its data and two charities told STAT’s Ed Silverman they objected to the JAMA study’s conclusions. Among those that did reveal their spending, the cost for each Medicare Part D beneficiary for these drugs amounted to $1,157, compared with $367 for medications not covered. “The programs are relying on health insurance to pay most of the bill,” said study co-author Gerard Anderson of Johns Hopkins Bloomberg School of Public Health. “And the drug company benefits because the drug gets sold — and also makes more money if it’s a more expensive drug.”

Read more.

More reads

  • Mallinckrodt puts off planned generics spinoff amid opioid cases (Bloomberg
  • Regeneron beats second-quarter profit estimates on Eylea boost (Reuters)
  • GSK offloads rights to develop vaccines for Ebola and Marburg viruses (STAT)

Thanks for reading! Until tomorrow,


Wednesday, August 7, 2019


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