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The Readout Damian Garde & Meghana Keshavan

Remdesivir gets a price

After a long wait, Gilead Sciences has set a price for remdesivir, the first medicine shown to work against Covid-19. Now the debate over whether that price is fair can begin.

For all governments in the developed world, including the U.S. government’s Medicaid program and the Department of Veterans Affairs, Gilead will charge $2,340 for a five-day course. U.S. insurers will pay 33% more, or $3,120. Countries in the developing world will get the drug at greatly reduced prices through generic manufacturers to which Gilead has licensed production.

There has been speculation about the price for months, with the Institute for Clinical and Economic Review offering up arguments for a price anywhere between $10 and $5,080, and some Wall Street analysts making their own estimates.

“We spent a lot of time and considerable care and discussion about how to approach the pricing of this medicine,” Gilead CEO Daniel O’Day told STAT. “At this price it’s significantly below the value it brings to patients and to society. There is no doubt of that in my mind.”

Major drug makers commit $1 billion to novel antibiotics

After years of neglect, research into antibiotic resistance is getting a major boost. Several large drug makers are creating a $1 billion for-profit venture to acquire or invest in small antibiotic companies and their nascent products, STAT’s Ed Silverman exclusively reports

The effort will be formally announced on July 9 by the leaders of Pfizer, Merck, Eli Lilly, Novo Nordisk, Boehringer Ingelheim, Bayer Pharmaceuticals, and MerckKGaA, among others. Government officials from Germany, Sweden, the U.K., and France — along with representatives from the Wellcome Trust and Pew Charitable Trusts — will also be present, according to two people familiar with the plans. It’ll also have support from the World Health Organization and the European Investment Bank. 

“At present, it’s a fund organized mostly by large pharmaceutical companies that will be owners,” one source told STAT. “This keeps the [antibiotic] pipeline alive. In the oncology world, $1 billion is chump change, but $1 billion from for-profit companies to take a small number of assets and commercialize them is a lifesaver."

Read more.

Patient deaths in Audentes gene therapy study increase worry over high-dose treatments

Two patients in an Audentes Therapeutics gene therapy trial have died, STAT’s Matthew Herper reports — heightening safety concerns over high-dose forms of these medicines. 

The company told patient groups in a June 23 letter, obtained by STAT, that the patients died after having serious liver problems that appeared related to the company’s experimental treatment for X-linked myotubular myopathy, a rare condition that causes muscle weakness. Most patients with the disease don’t survive childhood. 

Although previous data from the study showed that the gene therapy improved patients’ ability to breathe, CEO Natalie Holles told STAT that the company has stopped enrolling patients in the study because of the deaths. The FDA then placed a clinical hold on the trial a few weeks ago. 

“This unfortunate event may be a wake-up call to do the basic studies to support clinical development as higher and higher doses of [gene therapy] are administered,” one gene therapy consultant told STAT. 

Read more.

Base editing successful in monkeys

CRISPR base editing has passed its first substantive test, STAT’s Sharon Begley writes. The tool, which can edit genomes on a letter-by-letter basis, was used to knock out two cholesterol-associated genes in monkeys — and found that LDL and triglyceride levels plummeted 60% and 65%, respectively.

The study comes from Verve Therapeutics — but it also bodes well for Beam Therapeutics, which is also developing CRISPR base editors for a number of different diseases. Verve actually licensed some of Beam’s technology for this experiment, which was carried out in 14 macaques. 

“Our goal is to develop a one-and-done genome editing medicine for heart disease,” Verve CEO Sekar Kathiresan told STAT.

A single PDF keeps data from trial participants

Participation in clinical trials is daunting for many people — in large part because they’re unable to see their own data. Harvard geneticist George Church, along with Resilience Project director Jason Bobe, propose an overhaul for clinical trial data distribution — and say that it’ll begin with eradicating one surprising roadblock: a single PDF

A document that materialized on the Centers of Medcare and Medicaid Services site in 2014 revises how Clinical Laboratory Improvement Amendments (CLIA) are interpreted — and “clumsily” deems that sharing trial data with participants is not copacetic. 

“Without a more supportive policy environment, even small-scale experiments in more transparent and equitable research practices that involve sharing data from research laboratories are unlikely to happen,” Church and Bobe write.

Read more.

More reads

  • Sinopharm says second Covid-19 vaccine found to be safe. (Bloomberg)
  • U.K.'s RenalytixAI aims for dual listing with $86M Nasdaq IPO. (FierceBiotech)
  • With peanut patch in FDA purgatory, DBV plans layoffs to stretch cash. (Xconomy)

Thanks for reading! More tomorrow,

Damian

Monday, June 29, 2020

STAT

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