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The Readout Damian Garde & Meghana Keshavan

ALS drug shows a modest (but welcome) benefit in a clinical trial

A treatment for amyotrophic lateral sclerosis, dreamt up in a college dorm, slowed neurological decline in a mid-stage study, demonstrating a small but promising benefit in a disease with few other options.

As STAT’s Meghana Keshavan reports, on a 48-point scale of disease severity, ALS patients who took the experimental drug saw their condition decline on average about 2.9 points less than those taking placebo over that six-month period. The results, published yesterday in the New England Journal of Medicine, point to a modest benefit but will be welcome news for the ALS community.

The data are also a major milestone for Cambridge, Mass., biotech Amylyx, which is developing the drug. The company, formed in 2013 by two Brown University undergraduates, has raised about $75 million to get to this point.

Read more.

Sanofi and GSK are the latest to start a Covid-19 vaccine trial

Partners Sanofi and GlaxoSmithKline started a first clinical trial for their Covid-19 vaccine candidate, trailing a handful of rivals but bringing decades of experience to the process.

As STAT’s Helen Branswell reports, the two companies plan to enroll more than 400 volunteers in a Phase 1/2 trial conducted at 11 sites around the U.S. The study, which should wrap up in early December, would enable Phase 3 development.

Sanofi and GSK are months behind Moderna, AstraZeneca, and partners Pfizer and BioNTech, all of which are already in Phase 3. But the partners have what could amount to an advantage: Their Covid-19 vaccine uses the same technology behind Sanofi’s FDA-approved influenza vaccine, which might improve the odds of success.

Read more.

Biotech leaders: No more Covid-19 data by press release, please

A group of prominent biotech executives is asking its peers to be diligent and transparent in the process of developing treatments and vaccines for Covid-19. And when there’s data to share, describe them in detail — don’t just issue a vague press release.

As STAT’s Nicholas Florko reports, the recommendations come in an open letter organized by BIO, the industry’s largest trade group. In it, nine industry leaders lay out guiding principles for companies and regulators working as quickly as possible to advance projects that might end the pandemic.

The goal, according to BIO Chairman Jeremy Levin, is that all parties maintain the public trust, whether through diversity in clinical trials, clarity in press statements, or rigor in scientific review. Any deviations risk delegitimizing novel drugs and vaccines in the eyes of the world, which would defeat the purpose of developing them in the first place.

Read more.

Amarin might finally have hit the bottom

Amarin’s fortunes took a dive in March when a federal judge ruled that key patents covering its heart drug Vascepa were invalid. And the company’s appeal, argued at a hearing yesterday, doesn’t seem likely to succeed. But there’s an arguable silver lining: Amarin’s share price, which fell another 30% after that hearing, probably can’t get much worse.

As Cowen analyst Ken Cacciatore pointed out in a note to investors, Vascepa still has patent protection in Europe, where its peak sales could reach about $1.5 billion a year. Assuming Amarin has no chance of winning its U.S. appeal, the company would retain a sizable business opportunity overseas, one that might not be reflected in its roughly $2 billion market cap.

That might not be terribly comforting to investors who bought into Amarin when it traded north of $20 a share, but according to Cacciatore, it suggests things can’t get much worse for the oft-debated company.

More reads

  • How Trump could use a rare regulatory maneuver to lower drug prices before Election Day, and how it could backfire. (STAT Plus)
  • Casdin goes all-in on the SPAC game with $385 million offering. (Endpoints)
  • Placebo arms aren’t needed for challenge trials of Covid-19 vaccines. (STAT)
  • Tesla's Musk discusses vaccine project, car plant with German politicians. (Reuters)

Thanks for reading! Until tomorrow,

Thursday, September 3, 2020

STAT

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