Advocates press government to exercise march-in rights for Xtandi

Advocates are pressing the Biden administration to license patents for Xtandi, a prostate cancer treatment, to other manufacturers so that lower-cost versions become available. In a letter to CMS, they said that two different federal laws — including march-in rights — would allow the U.S. government to authorize "qualified companies" to make and sell generic versions of the blockbuster drug.

Xtandi, which is sold in the U.S. by Astellas and Pfizer, is currently priced three to six times higher than it is in other high-income countries. In 2022, the average Medicare price was $130.66 per capsule; last year, that rose to $136.50. According to the CMS letter, the government payers spent more than $5 billion on this U.S. government-funded cancer drug.

The Biden administration rejected this petition a year ago, but since has issued a framework to assert march-in rights to lower costs of drugs created in part thanks to taxpayer dollars.

cancer

NCI director optimistic about research despite logistical hurdles

The director of the National Cancer Institute channeled Taylor Swift when speaking this week at the American Association for Cancer Research — speaking with optimism about the past, present, and future "eras" of cancer research. Kimryn Rathmell noted that scientists are generally shifting from more siloed research units to broader interdisciplinary teams, and that this is a trend that ought to continue.

"I am very optimistic," she said. "The kinds of questions that we can ask today are just unparalleled. I think that the way people are willing to work together and across boundaries has never been this strong or committed."

That said, she acknowledged that government funding for research scientists is stagnant or shrinking, saying that the agency is considering cutting contractor expenses and overall personnel. This is exacerbating the ongoing exodus of life scientists from academia.

"It's very worrisome," Rathmell said. "We absolutely need basic discovery science… we need people who are passionate about that to move into those fields and feel like that field is a career path for them."

drug pricing

The rationale for a 486% drug price hike

An injectable eye medicine called Triesence has been largely out of stock for years, despite being used to treat several diseases. Early last year, a small company called Harrow bought the treatment, and has plans to revamp and upgrade production of the drug — and increase the price by 486%. The drug was underpriced, and is "extremely challenging" and "tricky" to manufacture, CEO Mark Baum told STAT. It took "tens of millions of dollars" in production and regulatory costs in order to create a steady new supply of the medicine.

"It's a very important product in ophthalmology and right now, if you're a retina surgeon or general ophthalmologist, you don't have access to it. The drug has not been available for five years," Baum said. "So whether it's $160 or $944, it doesn't matter. There's no inventory at any distributor.… Some significant investments have to be made. It was a Model T vehicle in a Tesla Model S world."

venture capital

Biotech financing at its highest in two years

After a three-year slump, biotech startups just had an excellent fundraising quarter — raising $6.8 million in venture financing. That's the most since early 2022, according to data from HSBC. This isn't exactly enough to say the biotech sector has fully bounced back, an HSBC managing director told STAT.

"It's really hard to talk about this without saying it's like the tale of two cities, where you see a lot of really interesting momentum that's very positive, but on the other hand, you still have the frothiness of 2020 and 2021, in the form of higher valuations and maybe companies that, in a downturn, wouldn't have gotten the funding that they did, having to work their way through that next [investment] round," he said. "That is still happening, and that is going to happen throughout this year and into early next year."

gene therapy

AskBio CSO steps down, has new project in stealth

Jude Samulski, the early gene therapy pioneer, stepped down yesterday from his position as chief scientist of AskBio, the company he founded in 2001 to commercialize his work at a time when pharma and investors were fleeing the field. He will be replaced by Mansuo Shannon, who was chief scientist of the Eli Lilly subsidiary Prevail Therapeutics.

It's a notable departure, coming four years after Bayer bought AskBio and its pipeline of experimental therapies for up to $4 billion. At the time, Samulski said the company chose Bayer from a suite of suitors because the German pharma promised to keep its new subsidiary at arm's length, leaving Samulski and then-CEO and co-founder Sheila Mikhail in charge. Now, Mikhail and Sumalski are gone (Mikhail left after a cancer diagnosis). Prominent gene therapy researcher Kathy High also left last year, after joining in 2021.

Samulski, who will stay on AskBio's board, said he isn't done. "Retirement, I don't think so. Starting something new in stealth mode," he said in a text message, adding "You can publish that."

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