Hey, people. At present, we discuss in regards to the decades-long pursuit to get MDMA accepted as a drugs for PTSD, prematurely of Lykos Therapeutics’ Sunday PDUFA date. Additionally, we see a bit of lady’s ultra-rare illness enhance after she was given a genetic medication designed only for her, and extra.
The necessity-to-know this morning
Merck acquired an experimental bispecific antibody from China-based Curon Biopharmaceutical for $700 million upfront, plus a further $600 million in potential milestone funds. The drug, referred to as CN201, is being studied in blood cancers, however Merck mentioned it sees the potential to focus on B-cell-associated autoimmune illnesses.
Avidity Biosciences mentioned its RNA-based drug produced 25% of regular dystrophin in sufferers with Duchenne muscular dystrophy brought on by a mutation in exon 44.
How a bespoke gene remedy labored
Susannah Rosen, a bit of lady with a uncommon and degenerative neurologic illness, acquired a bespoke remedy about two years in the past. Medical doctors injected her with stretches of genetic code focusing on a gene that induced her signs into the 8-year-old’s backbone, the place it might then make its option to her mind. A pair months later, she was capable of stand on her personal for the primary time in 4 years.
The remedy, based mostly on antisense oligonucleotides, was developed by the San Diego nonprofit n-Lorem. A brand new Nature examine reveals that the ASO remedy additionally diminished her seizures and improved her mobility. That mentioned, she’s nonetheless going through imaginative and prescient loss and cognitive difficulties.
Susannah’s case underscores how potent customized medication might be for individuals with ultra-rare illness — but additionally reveals that complexities with this sort of strategy can come up, significantly in scaling up these therapies for different sufferers.
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What it’s best to know in regards to the FDA’s looming MDMA resolution
The FDA has till Sunday to determine whether or not or not it can approve MDMA-assisted psychotherapy as a remedy for PTSD. It might additionally determine to postpone its resolution — however both manner, the decades-long effort to legalize the psychedelic has been contentious and polarizing.
Proponents, together with many fight veterans, say that MDMA can save lives. However critics query the ethics and the standard of information coming from Lykos Therapeutics, the corporate that submitted the MDMA remedy for FDA assessment.
If it’s accepted, MDMA would develop into the primary criminalized psychedelic to be acknowledged for its medical advantages because the battle on medication started. The end result might pave the way in which for different psychedelics, like psilocybin, that are additionally being researched as psychological well being remedies.
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Recursion merges with Exscientia
Two main AI drug improvement corporations are merging: Recursion Prescription drugs is shopping for Exscientia for $688 million. The bigger firm will nonetheless name itself Recursion, and CEO Chris Gibson will retain his function. David Hallett, Exscientia’s interim CEO, will probably be Recursion’s chief scientific officer.
This is among the first main mergers in AI drug improvement. The brand new firm is about to have 10 clinical-stage drug readouts throughout the subsequent 18 months, which embody partnerships with massive pharmaceutical corporations and NVIDIA, which invested $50 million in Recursion final 12 months.
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Why did Novo pull coronary heart failure paperwork for Wegovy?
How will we all know if the federal government will get an excellent deal within the first spherical of drug pricing negotiations? And has Allison considered something aside from enterprise capital for the final couple of months? We talk about all that and extra on this week’s episode of “The Readout LOUD.”
STAT’s Washington Correspondent John Wilkerson joins us to debate the primary spherical of drug pricing negotiations underneath the Inflation Discount Act, which is able to wrap up by Sept. 1. We additionally cowl the most recent information within the life sciences, together with the merger of AI drug improvement corporations Recursion Prescription drugs and Exscientia; Novo Nordisk’s resolution to pause its regulatory submission for Wegovy’s use in sufferers with coronary heart failure with preserved ejection fraction; and the state of biotech VC.
Pay attention right here.
Extra reads
23andMe exits drug improvement to step into weight reduction wave, Bloomberg
As Novavax comes full circle, it’s ‘actively exploring’ sale of Czech manufacturing plant, FierceBiotech
FDA approves Citius’ remedy for uncommon blood most cancers, Reuters
