August 9, 2024

The need-to-know this morning

Merck acquired an experimental bispecific antibody from China-based Curon Biopharmaceutical for $700 million upfront, plus an additional $600 million in potential milestone payments. The drug, called CN201, is being studied in blood cancers, but Merck said it sees the potential to target B-cell-associated autoimmune diseases.
Avidity Biosciences said its RNA-based drug produced 25% of normal dystrophin in patients with Duchenne muscular dystrophy caused by a mutation in exon 44.

n-of-1

How a bespoke gene therapy worked

Susannah Rosen, a little girl with a rare and degenerative neurologic disease, received a bespoke treatment about two years ago. Doctors injected her with stretches of genetic code targeting a gene that caused her symptoms into the 8-year-old's spine, where it could then make its way to her brain. A couple months later, she was able to stand on her own for the first time in four years.

The treatment, based on antisense oligonucleotides, was developed by the San Diego nonprofit n-Lorem. A new Nature study shows that the ASO treatment also reduced her seizures and improved her mobility. That said, she's still facing vision loss and cognitive difficulties.

Susannah's case underscores how potent personalized medicine can be for people with ultra-rare disease — but also shows that complexities with this kind of approach can arise, particularly in scaling up these therapies for other patients.

psychedelics

What you should know about the FDA's looming MDMA decision

The FDA has until Sunday to decide whether or not it will approve MDMA-assisted psychotherapy as a treatment for PTSD. It could also decide to postpone its decision — but either way, the decades-long effort to legalize the psychedelic has been contentious and polarizing.

Proponents, including many combat veterans, say that MDMA can save lives. But critics question the ethics and the quality of data coming from Lykos Therapeutics, the company that submitted the MDMA treatment for FDA review.

If it's approved, MDMA would become the first criminalized psychedelic to be recognized for its medical benefits since the war on drugs began. The outcome could pave the way for other psychedelics, like psilocybin, which are also being researched as mental health treatments.

artificial intelligence

Recursion merges with Exscientia

Two leading AI drug development companies are merging: Recursion Pharmaceuticals is buying Exscientia for $688 million. The larger company will still call itself Recursion, and CEO Chris Gibson will retain his role. David Hallett, Exscientia's interim CEO, will be Recursion's chief scientific officer.

This is one of the first major mergers in AI drug development. The new company is set to have 10 clinical-stage drug readouts within the next 18 months, which include partnerships with large pharmaceutical companies and NVIDIA, which invested $50 million in Recursion last year.

podcast

Why did Novo pull heart failure paperwork for Wegovy?

How will we know if the government gets a good deal in the first round of drug pricing negotiations? And has Allison thought about anything other than venture capital for the last couple of months? We discuss all that and more on this week's episode of "The Readout LOUD."

STAT's Washington Correspondent John Wilkerson joins us to discuss the first round of drug pricing negotiations under the Inflation Reduction Act, which will wrap up by Sept. 1. We also cover the latest news in the life sciences, including the merger of AI drug development companies Recursion Pharmaceuticals and Exscientia; Novo Nordisk's decision to pause its regulatory submission for Wegovy's use in patients with heart failure with preserved ejection fraction; and the state of biotech VC.

Listen here.

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