Beware declarations of Covid-19 victory

The early days of the coronavirus crisis brought an onslaught of press releases from microcap biotech companies, each promising that its previously obscure pipeline held the key to stopping Covid-19. Now, many of those would-be world-savers are due to present actual data, and it would be wise to approach with skepticism.

As STAT’s Adam Feuerstein reports, CytoDyn, which spent all of 2019 as a penny stock, is nearing the end of a clinical trial testing its repurposed HIV drug in Covid-19. And the company looks poised to declare a spurious victory.

The trial, which enrolled 75 patients, defines success as improving fever, muscle ache, shortness of breath, and cough. Those are imprecise metrics that don’t necessarily lend themselves to definitive results. By contrast, Gilead Sciences’ remdesivir study looked at death, intubation, use of oxygen, and hospitalization.

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Remember aducanumab?

Not long ago, the fate of Biogen’s long-debated treatment for Alzheimer’s disease looked like the odds-on biggest biotech story of 2020. Various things transpired since then, but yesterday the company gave the world a timetable on the future of the oft-delayed aducanumab.

Biogen finally filed its controversial treatment for FDA approval, checking a box that has little importance in itself but sets in motion a series of events. Next, the FDA has 60 days to either accept or reject Biogen’s filing. Then, it will decide whether to give aducanumab a six- or 10-month review. Some time in between, the agency will almost certainly convene a panel of outside experts to debate the merits of the drug.

And those merits, as you might recall, are complicated. In one Phase 3 trial, Biogen’s treatment met its goal of slowing cognitive decline. In a second, it failed. Both studies were terminated early because they appeared hopeless. Months later, Biogen emerged with a take that, if you look at all of the data, aducanumab worked for those who got the highest dose for the longest period of time. At last, we’re going to find out whether the FDA agrees.

Genome editing finally breaks into the power source

Mitochondria, the tiny organelles that provide energy to cells, have long evaded the tools of DNA editing, much to the frustration of scientists. But a new technique, derived from a bacterial toxin, suggests cellular power plants can be tinkered with after all, lighting the way to potential treatments for rare and devastating diseases.

As STAT’s Sharon Begley reports, scientists at the Broad Institute and the University of Washington paired their expertises to edit mitochondrial DNA in lab dishes. The efficiency is only so-so, hitting roughly 20% to 40% of its genetic targets. But if the rapid advance of CRISPR-based genome editing is a guide, further study could hone the new method into a potent tool.

“With this technology, mitochondrial research will enter a golden age,” said biologist Fyodor Urnov of the University of California’s Innovative Genomics Institute.

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How hydroxychloroquine looms over the FDA’s credibility

Scientists have by and large moved on from the idea that the generic malaria drug hydroxychloroquine can help patients with Covid-19. But President Trump, a vocal advocate for the drug, wants it back in the equation, citing a problematic study and demanding the FDA “act now.” How the agency responds could speak volumes about its role in the ongoing crisis.

As STAT’s Matthew Herper reports, the latest support for hydroxychloroquine comes from an observational study with more than a few confounding details, which experts widely dismissed. But the White House disagrees, forcing the FDA into an uncomfortable position: If it bows to Trump and endorses hydroxychloroquine, it could undermine its authority on the much more important question of whether any of the in-development coronavirus vaccines should be approved.

“The FDA cannot afford another misstep if it wants to maintain credibility with American people, which is going to be so essential when doing a broad vaccine program, should we identify a safe and effective vaccine for Covid,” said Luciana Borio, who served as the FDA’s acting chief scientist from 2017 to 2019.

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More reads

• AbbVie may face fewer wrinkles after judge suggests a 10-year ban on a Botox rival. (STAT Plus)
• A swift big pharma endorsement for Kiadis. (Evaluate Vantage)
• Drug prices steadily rise amid pandemic, data shows. (Politico)
• RA Capital's SPAC prices upsized $118 million IPO. (Renaissance Capital)