August 8, 2024

The need-to-know this morning

Eli Lilly boosted 2024 revenue guidance by $3 billion on strong Mounjaro, Zepbound sales.
Apellis Pharmaceuticals announced positive results from a Phase 3 study of its approved drug Empaveli in patients with two types of rare kidney disease. The company plans to seek FDA approval in early 2025. Its marketing partner Sobi will also apply for European approval next year.
Merck halted a Phase 3 clinical trial of its anti-TIGIT drug vibostolimab in small cell lung cancer after independent monitors determined the drug combined with Keytruda would not prolong patient survival compared to standard treatment.

health policy

Trump's real health care agenda isn't in Project 2025 — it's in his own words

Former President Trump has sought to distance himself from Project 2025 — despite efforts by Democrats to link him to the Heritage Foundation plan. So STAT dove deep into Trump's own remarks on health care issues, taken from his "Agenda47" series and campaign statements, to better understand the policies he might advance if he reclaims the White House.

Turns out he's had plenty to say on subjects like prescription drug shortages and drug pricing.

"It strikes me as infinitely more likely that the Trump administration would maintain Medicare negotiation and try to use that as a mechanism to implement their most favored nation idea," said Ben Ippolito, a senior fellow in economic policy studies at the right-leaning American Enterprise Institute.

gene therapy

Sarepta CEO: We're 'pretty brilliant'

On its earnings call last night, Sarepta Therapeutics was not asked directly why it demanded a Duchenne patient advocacy group censor a video to delete comments critical of the company that were made by a mother of a young adult with Duchenne, as STAT recently reported. One analyst on the call brought up the issue indirectly, asking Sarepta CEO Doug Ingram why there was a disconnect between some in the Duchenne community and the company.

"I think anybody who has been watching us carefully over the last seven or eight years will know that we've been wholly committed to bringing a better life to the community. And we've, at the risk of being a bit immodest, we've been pretty brilliant in our ability to do that," Ingram said.

"The great bulk of the patient community fully understands that and is cheering us along … right-minded people are getting it," he added.

For the second quarter, Sarepta reported Duchenne drug sales of $360.5 million, including $122 million for its gene therapy called Elevidys — a drop-off from the prior quarter and missing Wall Street expectations. The company insisted there were "no bottlenecks" being encountered with the Elevidys launch, arguing that it takes three to five months for patients to receive treatment after an initial request.

The recent FDA approval and label expansion that cleared Elevidys use by nearly all Duchenne patients will accelerate sales towards the end of the year, the company said. It provided 2025 sales guidance in the range of $2.9 billion to $3.1 billion, lower than the current analyst consensus of $3.2 billion.

crispr

Delivery is the priority in CRISPR therapies

Now seasoned with a decade's experience, CRISPR researchers are focused keenly on delivery: They're trying to develop in-vivo editing methods that can reliably deliver gene-editing therapies to hard-to-reach tissues like neurons, muscles, and blood stem cells.

And they're moving past viral vectors alone to deliver CRISPR-based therapies. There's been a surge of interest in using new lipid nanoparticles, exosome-based delivery systems, and virus-based particles to ferry these treatments to the desired tissues.

Recent research using exosomes, or extracellular vesicles, to deliver CRISPR therapies has been particularly interesting in Alzheimer's disease — "however, challenges like ensuring specificity, overcoming supply chain issues, and reducing costs are still critical areas that need ongoing research," one drug delivery expert told STAT's Megan Molteni.

Rare disease

Vertex, U.K. reach deal on CRISPR-based medicine

Beta thalassemia patients in England can now receive the CRISPR-based medicine Casgevy, now that its maker, Vertex Pharmaceuticals, has struck a reimbursement deal with U.K. health officials. The one-time treatment edits genes in the stem cells in a patient's blood, offering a potential cure for the disease.

Casgevy's U.S. list price is $2.2 million, though the deal between England and Vertex means it will be offered through the NHS at an undisclosed discount.

Read more.

patents

How the drug patenting system works

Biopharma companies guard their patents closely in order to secure the most bang for the inordinate amount of research bucks it takes to develop a new drug. The lure of market exclusivity is strong: Without patents, companies might never recoup the money they pour into R&D.

In a new video, STAT's Anna Yeo explains the process by which the U.S. Patent and Trademark Office grants intellectual property rights to drugmakers — and how that ultimately impacts biopharma economics.

Watch here.

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