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Lilly's FDA rejection, billionaires in biotech, & a novel cancer target

January 20, 2023
National Biotech Reporter & Biotech Correspondent

Good morning, all. Damian here with the latest twist in Alzheimer's disease research, some promising developments in oncology, and a novel idea in antibiotics.

 

Biotech

FDA rejects Lilly's Alzheimer's drug

The FDA rejected Eli Lilly's attempt to win a speedy approval for its Alzheimer's disease treatment, the company said yesterday, holding off until an ongoing Phase 3 study concludes later this year.

As STAT's Adam Feuerstein reports, the agency declined to approve donanemab, an antibody treatment, because Lilly's submitted data did not include enough patients who had taken the medicine for at least a year, according to the company. Lilly had submitted results from a Phase 2 study in hopes of winning accelerated approval.

The future of donanemab now rests on the results from a larger, Phase 3 study expected to read out in the second quarter. While the FDA's rejection delays the potential launch of Lilly's drug, donanemab's commercial potential was always dependent on its Phase 3 data — and whether the results are strong enough to convince Medicare to pay for it.

Read more.


Oncology

The cancer target that could unlock powerful treatments

A host of novel therapies have led to dramatic benefits for patients with blood cancers, but replicating those results for solid tumors has proved vexingly difficult. The emergence of a new biological target, a protein found almost exclusively on cancer cells, promises a path forward.

As STAT's Angus Chen reports, that protein is called mesothelin, or MSLN, and it turns up in ovarian and pancreatic cancers but few healthy tissues. That makes it an ideal target for newfangled treatments like cell therapy and bispecific antibodies, which have proved potent in blood cancer but have found little success in other tumor types.

The idea is not to attack mesothelin directly but rather to use it as a bullseye. Cell therapies like CAR-T can be designed to find and kill any cell in the body carrying the protein, while bispecifics would bind to mesothelin and link it with immune cells that would eradicate it.

Read more.


podcast

Where do new drugs come from?

Does science need billionaires? And do normal people care about messenger RNA?

We cover all that and more this week on "The Readout LOUD," STAT's biotech podcast. Journalist Nathan Vardi joins us to talk about his new book delving into the race to develop the lifesaving cancer drug now called Imbruvica, involving a Scientologist CEO and secretive investor seeking redemption after the worst trade of his life. We also discuss the latest news in the life sciences, including new vaccines for a vexing infection and the future of mRNA.

Listen here.



Regulatory

An American idea ruffles feathers in Europe

For more than a decade, the U.S. has incentivized drug companies to invent much-needed therapies by offering a sellable coupon for every qualifying drug approved. But a similar enticement, proposed in Europe, has run into objections.

As STAT's Ed Silverman reports, the EU idea is meant to encourage the development of novel antibiotics by offering companies a voucher that adds a year of patent exclusivity to any medicine. It's a variation on the U.S. model, which awards a voucher redeemable for an expedited FDA review.

The concern, voiced by more than a dozen members of the EU, is that large drugmakers could buy such vouchers and apply the added exclusivity to expensive, best-selling medicines, forestalling generic competition across the continent and driving up healthcare costs. "It doesn't incentivize directly the development of new antimicrobials or the availability throughout the EU," said Kris Boers, Belgium's health and pharma counselor. "... "We need better measures, new business models, and direct financial incentives."

Read more.


M&A

A deal's a deal

Investors' long-predicted spike in drug-industry dealmaking hasn't come to pass, and the sector's latest acquisition is a reminder of just how far biotech has fallen in the eyes of its pharmaceutical peers.

Yesterday, Sun Pharmaceutical said it would pay $8 a share for Concert Pharmaceuticals and its late-stage treatment for alopecia. The offer is a roughly 33% premium to Concert's average stock price over the last month, and it could pay out another $3.50 per share if the company's drug meets certain milestones.

But zooming out, Sun's offer is a 20% discount to where Concert was trading in early 2021, and for shareholders who have held on since the company's 2014 IPO, the buyout would represent a nearly 45% haircut.


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More reads

  • 'A cascade of failures': FDA cites Indian drugmaker for numerous quality control problems, STAT
  • Biotech companies hunt for cash as VC funding dries up, Bloomberg 
  • Moderna CEO in talks with China to supply Covid vaccines, Reuters

Thanks for reading! Until next week,


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