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The Readout Damian Garde & Meghana Keshavan

The newsletter is taking a break Monday in observance of President's Day. We'll be back Tuesday.

Amicus’s rare disease drug misses the mark, but company sees hope

Amicus Therapeutics’ late-stage treatment for the rare condition Pompe disease failed to beat the standard of care in a key clinical trial, but the company believes it can still make its case to regulators.

As STAT’s Adam Feuerstein reports, Amicus’s drug, called AT-GAA, was numerically superior to Sanofi’s Lumizyme when it came to how far patients with Pompe could walk after one year of treatment, but the difference didn’t cross the threshold of statistical significance, meaning it could be due to random chance.

But it was close, Amicus CEO John Crowley said. Had patients in the AT-GAA group walked two meters further, at the median, the study would have met its primary endpoint, he said. The company plans to submit for FDA approval in the second quarter. 

Read more.

How many effective Covid-19 drugs are just waiting to be found?

When will we have enough vaccine doses? And what do Redditors think about biotech?

We discuss all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. First, we check in with our colleague Helen Branswell for her view on the current state of the Covid-19 pandemic and the ongoing vaccine rollout. Then, we’re joined by David Fajgenbaum, a physician and scientist at the University of Pennsylvania, to talk about his work to identify and develop treatments for people with Covid-19. Lastly, we embark on a lightning round, covering Gilead Sciences’ latest setback, a schism in Alzheimer’s disease research, and a preview of the newest podcast from STAT.

Listen here.

Maybe machine learning can build a better gene therapy

Gene therapies rely on harmless human viruses to deliver their corrective payloads to the right places in the body. The problem with those viruses is that their ubiquity means quite a few people will have developed antibodies against them, making those people unlikely to benefit from a gene therapy they might need.

As STAT’s Katie Palmer reports, machine learning might be able to help. Dyno Therapeutics, a startup co-founded by George Church, used machine learning models to comb through loads of those harmless viruses to isolate the ones that are both unlikely to alarm the immune system and structurally sound enough to carry a whole gene.

The results, published in Nature, suggest Dyno’s method might make it easier to craft viable viral delivery vehicles for future gene therapies. The company has already formed partnerships with Novartis, Sarepta Therapeutics, and Roche to do exactly that. 

Read more.

Regeneron is back into the rare disease business

Regeneron Pharmaceuticals won FDA approval for a drug that treats a rare genetic disease causing dangerously high cholesterol levels, marking a commercial departure for a company that has made its name — and billions of dollars — treating more widespread conditions.

The drug is evinacumab, approved yesterday under the brand name Evkeeza, and it treats homozygous familial hypercholesterolemia, or HoFH, a genetic disorder that results in potentially deadly levels of bad cholesterol despite maximum doses of existing medicines. In a pivotal trial, Evkeeza led to a 49% average reduction in bad cholesterol compared to placebo, an unprecedented result for patients with the disease.

The commercial concern comes from the fact that HoFH affects only about 1,300 people in the U.S., and, because there are other cholesterol-lowering options out there, evinacumab may be reserved for only the most severe patients. Evkeeza is dosed based upon a patient’s weight, meaning in its list price will vary, but Regeneron expects it to cost $450,000 a year on average. The challenge for Regeneron is to learn from the experience with its first rare-disease treatment, Arcalyst, which is a commercial nonentity after 13 years on the market.

More reads

  • Arthritis drug cuts deaths in hospitalized Covid-19 patients, major study shows. (STAT)
  • 'A game changer’: Drug brings weight loss in patients with obesity. (New York Times)
  • Dear NHS: You need a control arm for Grail’s cancer test. (STAT)
  • 'Our gratitude always': From China's CanSino, Mexico welcomes biggest vaccine shipment yet. (Reuters)

Thanks for reading! Until next week,

Friday, February 12, 2021


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