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Morning! Today, we discuss why disappointing data from Pfizer’s Duchenne muscular dystrophy trial might spell trouble for others developing gene therapies for the condition. Also, we see a huge cash influx for a startup conceptualized by the folks behind Karuna Therapeutics, and more.
The need-to-know this morning
- Catalent issued an open letter to customers, trying to tamp down concerns related to its pending acquisition by Novo Holdings.
- Novo Nordisk said oral semaglutide, the active ingredient in its diabetes medicine Rybelsus, demonstrated a 14% reduction in the risk of major cardiovascular events in a Phase 3 study of adults with Type 2 diabetes.
Pfizer’s Duchenne data rattle gene therapy makers
Pfizer suffered a blow earlier this month when new data showed that its gene therapy for Duchenne muscular dystrophy failed to help participants demonstrate significant improvement in motor function — even though they produced significant amounts of microdystrophin, a lab-built miniature version of the gene mutated in the disease.
But the results could also pose a much broader problem, STAT’s Jason Mast and Adam Feuerstein write. The Food and Drug Administration approved Sarepta Therapeutics’ Duchenne therapy on the basis of a belief that increasing microdystrophin levels were a sign the treatment was effective. Companies including RegenxBio and Solid Biosciences are developing their own treatments on this basis.
“The point is not only the level of expression of microdystrophin, but how functional it is,” the head of a gene therapy nonprofit told STAT. “A probable conclusion from these data is that this particular microdystrophin is not as efficient in humans as [Pfizer] had expected.
Seaport gets another $225M for mental health drugs
Seaport Therapeutics, a Boston-based mental health startup founded by the executives behind Karuna Therapeutics, just raised another $225 million in a Series B round — just six months after its $100 million Series A. Seaport is led by biotech veteran Daphne Zohar as well as former Karuna CEO Steven Paul, both of whom helped the company win approval for the novel schizophrenia treatment Cobenfy.
Seaport is developing therapies for depression, anxiety, and other mental health conditions by targeting nervous system receptors via the lymphatic system. This new fundraise will advance three drug candidates into trials, STAT’s Allison DeAngelis writes.
“We’re in the golden age of neuroscience, and neuropsychiatry, in particular,” Zohar said.
AI image analysis is changing cellular biology
Artificial intelligence is transforming our understanding of cell biology — merging molecular and cellular data to better understand how various cell processes work. Images are a gold mine that can yield information as rich as the genome, STAT’s Angus Chen writes, once you learn how to extract it.
Researchers like Anne Carpenter, an AI and cell biology researcher at the Broad Institute, and Cambridge Stem Cell Institute professor Sarah Teichmann spoke of the potential at the STAT Summit last week in Boston.
“We’re firmly in the spatial era for in vivo human cell and tissue characterization,” said Teichmann, who co-founded the international Human Cell Atlas Consortium. “One of the exciting things is to be able to connect molecular data to more cellular morphological data and tissue morphology.”
Controversy around 340B drug discount program
The 340B drug discount program was launched in 1992 to cut drug costs for hospitals and clinics serving low-income patients. A new analysis shows that last year the program grew 23.4%, with $63 billion in purchases, STAT’s Ed Silverman writes. The growth is intensifying the conflict between the pharmaceutical and hospital industries, with drugmakers restricting discounts saying they’re being abused.
“The data spotlight the increasing gulf between the growth of the 340B and the lack of any evidence that patients are benefitting from those discounts,” one pricing consult told STAT.