March 20, 2024

The need-to-know this morning

On Monday, the Food and Drug Administration approved a gene therapy for children with a fatal neurodegenerative disease called metachromatic leukodystrophy, or MLD. Today, Orchard Therapeutics said it would charge $4.25 million for the treatment, called Lenmeldy, making it the most expensive drug in history. But for the 40-100 children born with MLD each year, Lenmeldy will be the only lifeline away from a progressive, fatal disease.
Acelyrin reported preliminary results from an early study investigating an antibody treatment for thyroid eye disease that can be administered with a simple skin injection.

Venture

A bright idea in cancer raises $150 million

Biotech startup Clasp Therapeutics has a plan to turn the body's natural defenses against cancer while minimizing the risk to healthy bystander cells, an idea that helped the nascent company raise $150 million in venture dollars.

As STAT's Allison DeAngelis reports, Clasp is working in the field of T-cell engagers, medicines designed to grab onto tumors and immune cells at the same time, exposing cancerous tissues to the full weight of the immune system. Prior generations of T-cell engagers have had problems with accuracy: Many proteins on the surface of cancer cells are also found elsewhere in the body, meaning a poorly calibrated therapy will wreak havoc on tumors and healthy tissues alike.

Clasp, by contrast, is targeting cancer-specific proteins inside of tumor cells. The company's T-cell engagers, still in preclinical development, are meant to latch onto the telltale signs of cancer and drag immune cells into the fight.

Read more.

In the pipeline

Biotech's year of neuroscience is off to a rough start

Back at January's J.P. Morgan Morgan Health Care Conference, when mood and weather alike were sunny, there was a common refrain among executives, investors, and analysts: Neuroscience is in for a big year in 2024.

Two months later, that prediction is looking a little dicey. First, the FDA decided to again delay approval for Eli Lilly's Alzheimer's disease therapy, a decision that could have wide implications in the industry. Then, Acadia Pharmaceuticals lost about a quarter of its value when the company's approved psychiatric treatment failed in a schizophrenia study, derailing its plans to expand the market. And then Amylyx Pharmaceuticals' closely watched ALS study came up negative, a crushing disappointment that puts the entire company's future in jeopardy.

To be fair, it's only March, and things could still turn around for the long-promised neuroscience renaissance. In the second half of this year, Neumora will have Phase 3 data on a novel approach to treating depression that could bring about a desperately needed new medicine, and Karuna Therapeutics, now owned by Bristol Myers Squibb, is expected to win approval for the first new approach to treating schizophrenia in decades.

AI

Novo is building a really big computer

The Novo Nordisk Foundation, whose controlling stake in the maker of Wegovy has made it the wealthiest charitable foundation in the world, is getting into AI.

Specifically, it's putting up about $90 million to construct Gefion, a Danish supercomputer running on hardware and software from the trillion-dollar tech firm Nvidia. The idea is to put "extreme AI computing power" to the task of "drug discovery, disease diagnosis, and treatment," Novo Nordisk Foundation CEO Mads Krogsgaard Thomsen said in a statement.

Gefion, once it comes online later this year, will be made available to Danish researchers in the public and private sectors, with any resulting revenue invested back into the project.

Biotech

How many radiopharma companies are even left?

Yesterday's news that AstraZeneca will pay $2 billion for a targeted radiation company called Fusion Pharmaceuticals marks the latest high-dollar deal in the booming world of radiopharmaceuticals. It also takes yet another biotech company off the board of potential acquisition targets.

Pharma's recent interest in radiopharmaceuticals, which use targeted isotopes to radiate tumors while sparing healthy tissues, dates back to Novartis and a pair of acquisitions, in 2017 and 2018, in which it traded roughly $6 billion for a place in the field. In the years since, Bayer has bought in, and so have Eli Lilly and, most recently, Bristol Myers Squibb.

That gold rush has helped put radiopharma companies on the map — and correspondingly thinned their ranks. Shares of Lantheus, among Fusion's few remaining competitors, rose about 4% on the news yesterday, while Perspective Therapeutics, a radiopharma firm with a market capitalization below $1 billion, went up more than 15%.

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