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The Readout Damian Garde & Meghana Keshavan

Maybe no one’s buying Uniqure after all

Uniqure, among the most talked-about potential buyout targets in biotech, just did something companies don’t do when they’re for sale.

The Dutch biotech sold the rights to its lead asset, a gene therapy for hemophilia, in exchange for $450 million in cash and the promise of $1.6 billion more provided it meets a series of goals. The news sent Uniqure’s shares down about 8%. 

Not only does the deal deflate any near-term hopes for a Uniqure acquisition; it also casts some doubt on the market for gene therapy companies. If the reports of Uniqure shopping itself around were correct, and all it came up with was a $450 million partnership, that could be a sign that there's a disconnect between investor expectations and pharmaceutical reality.

Prosecutors say Regeneron ran an Eylea racket

There’s nothing to stop drug companies from pouring money into charities that end up paying patients’ co-pays. What they can’t do is specify that those charities only cover certain drugs. And that, according to federal prosecutors, is exactly what Regeneron Pharmaceuticals did.

As STAT’s Ed Silverman reports, Regeneron is accused of paying tens of millions of dollars to the Chronic Disease Fund under the guarantee that its money would only go toward co-pays for Eylea, the company’s injectable treatment for macular degeneration. That helped Regeneron stave off competition, according to authorities. Regeneron denies the allegations.

This is hardly the first time a drug company has been accused of using a patient charity as a commercial crutch. Over the past two years alone, drug makers including Novartis, Alexion Pharmaceuticals, and Pfizer have settled similar allegations with the government.

Read more.

A novel way around cancer’s defenses

Tumors have devised a way to disarm one of the body’s most reliable weapons against infection. So scientists made an evolutionary tweak, creating a new treatment that looks promising enough to build a company around.

As STAT’s Elizabeth Cooney reports, researchers at the Yale School of Medicine homed in on interleukin-18, a powerful cytokine that normally galvanizes an immune response to unwanted presences, like tumors. Cancer, in its infinite trickery, has evolved to nullify IL-18 with a decoy receptor. So the researchers screened hundreds of millions of mutated IL-18s until they came up with one that could escape the decoy and trigger the immune system in the process.

The result, dubbed DR-18, performed well in mouse studies, fighting tumors and stimulating immune cells. Now they’ve formed a venture-backed startup, Simcha Therapeutics, with plans to begin human studies next year.

Read more.

Three years after failing in Alzheimer’s, Merck is investing more in neuroscience

Back 2017, the decisive failure of a Merck drug called verubecestat helped bring about the end of an entire wing of Alzheimer’s disease research. Now, Merck is gradually building back a neuroscience pipeline, partnering with a startup on some early-stage projects.

As STAT’s Adam Feuerstein reports, Merck signed a deal with Yumanity Therapeutics in hopes of finding novel treatments for amyotrophic lateral sclerosis, or ALS, and frontotemporal lobar dementia, a group of related conditions resulting from the degeneration of brain cells.

Yumanity, founded in 2014 by biotech entrepreneur Tony Coles, is building on the discoveries of Susan Lindquist, a well-known researcher and National Medal of Science recipient who used yeast as a model organism to study neurodegenerative diseases caused by misfolded proteins.

Read more.

More reads

  • As approval decision nears, fate of Intercept’s NASH drug clouded by murky FDA review delays. (STAT Plus)
  • Biotech suddenly bests FAANG stocks in rally to all-time high. (Bloomberg)
  • A pill to fight alcoholism causes an uproar in France over a regulator’s view of a clinical trial. (STAT Plus)

Thanks for reading! Until tomorrow,

Thursday, June 25, 2020

STAT

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