April 25, 2024

The need-to-know this morning

AstraZeneca reported first-quarter sales and profits that topped analyst estimates, buoyed by its blockbuster cancer drugs.
It's a busy day for pharma earnings: Merck, Sanofi, Daiichi Sankyo, Astellas, and Bristol Myers Squibb.
Regeneron Pharmaceuticals and Mammoth Biosciences are partnering to develop new CRISPR-based gene therapies.

drug development

A hospital takes a gene therapy's approval into its own hands

What happens if, as an academic, you develop a promising gene therapy, but no company is willing to take it to market? Well, one option is — you do it yourself.

That's what British researcher Claire Booth and her hospital are attempting. The unusual step reflects the crisis in commercial gene therapy development. Many companies have abandoned promising therapies for ultra-rare diseases because they often don't see a possibility of making a return on these medicines, which are immensely expensive to design and are running into reimbursement obstacles, STAT's Andrew Joseph reports.

Booth's drug is for an extremely rare immunodeficiency syndrome called ADA-SCID. It has already been through clinical trials, appearing to effectively cure 48 out of 50 patients. Orchard Therapeutics had licensed the therapy from the researchers in 2016, only to hand it back a few years later.

Read more on how Booth is trying to carry out this effort and how it could open the door for more academically developed therapies.

pharma

BMS lays off workers as earnings take a hit

Bristol Myers Squibb will cut 2,200 jobs — or 6% of its workforce — by the end of the year, as it embarks on an aggressive cost-cutting program aiming to save $1.5 billion by the end of 2025, CEO Christopher Boerner said in an exclusive interview.

The company this morning reported an adjusted loss of $4.40 per share in the first quarter, a result that was impacted by its recent acquisitions. Analysts had estimated an adjusted loss of $3.43 per share. BMS also lowered its 2024 forecast to a range of $0.40 to $0.70 per share from $7.10 to $7.40 per share.

Talking to STAT's Matt Herper, Boerner said, "We've had success progressing the science over the last number of years, and that's given us a lot of options as a company. And when you have a lot of options, it forces you to prioritize." BMS is looking across the company, including in cell therapy and other areas, as it makes cuts, he said.

Read more on the CEO's thoughts about the company's pipeline and the potential to use CAR-T in autoimmune diseases.

drug pricing

Bernie Sanders targets 'outrageously high price' of Ozempic

Senate health committee chair Bernie Sanders said he launched an investigation yesterday into the "outrageously high price" of Novo Nordisk's blockbuster diabetes and obesity drugs Ozempic and Wegovy.

In a letter he sent to CEO Lars Fruergaard Jørgensen, he pointed out that the list prices of the drugs in the U.S. ($969 and $1,349 per month) are much more than what's charged in other countries. He also cited a recent study that shows the drugs can be manufactured at less than $5 a month.

Even though pharmacy benefit managers can negotiate lower net prices, coverage of the drugs will still lead to spikes in insurance premiums, Sanders said.

Sanders asked if Novo would "substantially reduce" both the list and net prices of the drugs and asked for documents related to their pricing.

lobbying

Another company leaves BIO trade group

Takeda Pharmaceuticals has left the biotech industry's main lobbying group, the fourth departure of a major member since December, STAT's John Wilkerson reports.

Takeda follows Pfizer, UCB, and WuXi AppTech in leaving BIO (though the circumstances of WuXi's departure were unique).

The loss of these members coincides with a drop in lobbying spending by BIO, which spent $1.76 million in the first quarter of this year, down 27% from the same period last year.

Drugmakers have been re-evaluating their memberships in trade groups amid a tough economic environment. PhRMA has also seen some departures recently.

Alzheimer's

Leqembi launch gains momentum after slow start

After a tepid rollout, usage is now ramping up for Biogen's Alzheimer's drug Leqembi, as medical centers finish the logistical setup needed to administer weekly infusions of the drug, executives said when reporting earnings yesterday.

Leqembi, which is co-marketed with Eisai, brought in $19 million in sales in the first quarter, nearly triple the amount in the fourth quarter of last year. This was also higher than the "whisper number" based on prescription data that suggested sales of $16 million, STAT's Adam Feuerstein writes.

Many doctors remain skeptical of Leqembi, though, as the drug carries risks like brain swelling, and it's not clear how meaningful the benefits will be for patients.

Skyclarys, the treatment for Friedreich's ataxia that Biogen got in its acquisition of Reata Pharmaceuticals last year, also beat expectations. It brought in $78 million in the first quarter, following a disappointing performance in the fourth quarter last year.

Read more on another of Biogen's drugs that beat expectations and how the company is thinking about M&A this year.

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