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We have some news out of a meeting of the European Society of Cardiology, Roivant won a key autoimmune drug approval, and former Stanford president Marc Tessier-Lavigne is pushing back against revived allegations of research fraud.
RIP to Yayoi Kusama. Wearing manic polka dots today in tribute.
Roivant wins approval for autoimmune drug
The FDA yesterday approved Roivant and its subsidiary Priovant’s therapy for dermatomyositis, a rare autoimmune disease that causes muscle weakness and painful skin rashes. The once-daily pill, to be marketed as Lisraya, blocks TYK2 and JAK1 proteins. In a Phase 3 trial, the treatment improved symptoms and reduced patients’ reliance on steroids, STAT’s Adam Feuerstein writes. That said, the drug’s label carries a black box warning for serious infections, blood clots, and cardiovascular events.
CEO Matt Gline called Lisraya a “big boy” achievement, because the drug’s expected to win approval eventually for a wider swath of autoimmune diseases. If it succeeds, the company will be emulating biotechs such as Insmed and Argenx by turning drugs for “big orphan” diseases into multibillion-dollar franchises.
Read more.
Tessier-Levigne fights back against fraud claims
Former Stanford president Marc Tessier-Lavigne is mounting a fresh defense against allegations that he oversaw fraudulent research at Genentech and helped cover it up. He released previously private emails that he says undermine claims revived in “How to Rule the World,” a new book by former Stanford Daily reporter Theo Baker.
Tessier-Lavigne acknowledges that his influential 2009 Nature paper on neurodegeneration contained incorrect results, STAT’s Jonathan Wosen writes. But he argues they reflected the messy process of science rather than fabrication. Stanford’s investigation found no evidence that Tessier-Lavigne committed misconduct or covered up fraud, though it concluded that several studies he led contained manipulated data and criticized his handling of corrections.
Tessier-Lavigne resigned as Stanford president in 2023 and later retracted the Nature paper; he now runs the $1 billion-backed AI drug developer Xaira.
Read more.
AstraZeneca, Cytokinetics open ESC
The European Society of Cardiology’s annual conference kicked off this morning in Munich, and already we have some news:
AstraZeneca and partner Ionis Pharmaceuticals detailed the surprise failure of their silencer Wainua in an ATTR-CM trial. Investigators think the high proportion of patients who were already on a stabilizer drug at the start of the trial caused the miss, suggesting that adding a silencer on top of a stabilizer does not compound the benefits of treatment.The study has been weighing on Alnylam Pharmaceuticals, which sells a silencer called Amvuttra and is developing a next-gen silencer, and has boosted BridgeBio, the maker of a stabilizer called Attruby. Some analysts have said the data bolster the idea that stabilizers could be superior to silencers, though the different treatments haven’t been tested head-to-head. More here on the implications of the study, including the curious benefits seen in the monotherapy subgroup.
Cytokinetics reported the full data from its successful ACACIA-HCM study of the drug aficamten in non-obstructive hypertrophic cardiomyopathy. The medicine is already approved in the obstructive form of the disease as Myqorzo, and could be the first drug specifically cleared to treat nHCM, as Cytokinetics plans to submit it for approval before the end of the year. Still, the discussant described the benefits of the drug as “modest.” More here.
More reads
China approves J&J psoriasis pill Icotyde, says drugmaker, Reuters
With mRNA back in the spotlight, tRNA shows potential to treat genetic disease, FierceBiotech
Lilly’s move to hide Phase 1 pipeline draws mixed reviews, Endpoints
