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Lysogene's trial failure, mRNA cancer vaccines, & passing the PASTEUR Act

 

 

The Readout

Good morrow, it's Meghana. Today we have two companies with unfortunate trial results, and some support for the PASTEUR Act — legislation that would support the development of drugs for superbugs. 

An expert on why Congress should approve the PASTEUR Act

Superbugs are a looming threat for the global population — with estimates that drug-resistant infections could kill 10 million people each year by 2050. By contrast, Covid-19 killed an estimated 3 million people in 2020. Yet policymakers have yet to address the superbug crisis in a major way. Scientists need to focus on developing potent new antimicrobial drugs now, but they’re “dangerously behind schedule,” opines Emory University infectious disease expert Carlos del Rio. And it’s not the science that’s the problem: There are antibiotics that have made it through early trials. But the market — or lack thereof — remains a problem.

That’s why del Rio is urging Congress to pass the PASTEUR Act — that is, the Pioneering Antimicrobial Subscriptions to End Upsurging Resistance Act. The legislation would allow the makers of antimicrobial drugs to enter a subscription model with the federal government.

“It’s similar to Netflix — subscribers can use as much or as little of the product as they need, rather than buying the medicine per dose,” del Rio writes. “By paying antimicrobial makers a predetermined amount of money for the availability of their treatment, these companies don’t need to rely on the number of antimicrobial drugs sold to stay afloat financially.”

Read more.

A less-than-stellar readout for Lysogene

Lysogene’s Phase 2/3 trial in patients with Sanfilippo syndrome just failed — a brutal blow to the flailing gene therapy company. Although there was a statistically significant improvement in cognitive function among the six youngest participants in the trial, a treatment effect wasn’t seen in the remaining 12 in the study, FierceBiotech writes.

A year ago, Sarepta ended a deal with Lysogene, in which it would hold the rights outside of Europe for the treatment for the inherited neurodegenerative disorder. Today, Lysogene has just about $6.3 million cash in hand — just enough to last until the end of January. That said, Lysogene believes that the encouraging results in young children could be enough to fundraise with.

Using mRNA vaccines for cancer

Covid isn’t the only application for mRNA vaccines. There’s another with a lot of potential: cancer. In this situation, of course, the mRNA vaccine would be used as a therapeutic instead of as a preventative measure, but the idea would still be to educate and rally the immune response. “The tumor cells are already there. And you have to build an army of immune cells which is big enough,” BioNTech’s CMO told STAT. “It’s a numbers game to conquer a tumor, which means you have to be able to induce very strong immune responses.”

Biotech companies are either doing this by targeting antigens that are shared across patients, or by creating cancer vaccines tailored to an individual. Some of the more advanced players in this cancer vaccine game include BioNTech, CureVac, and Moderna.

Read more.

Osteoarthritis flops for Biosplice

Biosplice — née Samumed — was once considered one of the richest of biotech unicorns. In 2018, it had a $12 billion valuation, more than Moderna or Grail. Now, however, Biosplice’s experimental osteoarthritis drug has failed two late-stage trials — the company’s most advanced medicine in its pipeline. The drug lorevivint failed to reduce pain in patients, though the company does plan to reevaluate the drug in another Phase 3 trial in patients with less-advanced osteoarthritis.

The biotech, whose small molecules were targeted toward indications ranging from neurological to oncological to musculoskeletal, has taken some serious lumps. In February, the company quietly laid off a solid chunk of its workforce — 22% — and cut a hair loss program in men. 

Read more.

More reads

  • Watch: This small-town pharmacy may be a model for more affordable drugs, STAT
  • Sporting night vision googles, a scientist probes the internal clocks that help parasites infect people, STAT
  • Pfizer CEO says Covid vaccine will remain ‘free for all Americans,’ overlooking indirect costs, STAT
  • Holmes gets more than 11 years in prison for Theranos scam, STAT
  • U.S. midterms dampen Big Pharma hopes for drug price policy change, Reuters

Thanks for reading! Until tomorrow,

@megkesh
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Monday, November 21, 2022

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