People who almost certainly had Alzheimer’s were also included in the study, but in this group, a placebo outperformed Cassava’s drug.

The conclusion, of course, is obvious: Simufilam is inactive. It’s an inert compound no more effective than a placebo. Cassava’s assertion that simufilam is showing “disease-modifying activity” falls apart given its study was opened to people who should have been ineligible because they were misdiagnosed with Alzheimer’s. Whether that was done intentionally or unwittingly isn’t known, but it’s certainly troubling and makes the case for immediate, regulatory intervention even stronger.

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Hi! Today we see that prime editing works nicely in monkeys, learn more about the potential new bill to speed treatments for life-threatening diseases, and find that a Regeneron (formerly Decibel) therapy may restore hearing in children.

The need-to-know this morning
• Sanofi said it will spin out its consumer health unit and cut costs in other areas in order to increase spending on research and development of new medicines. Separately, the French pharma giant reported third-quarter earnings and revenue that fell short of analyst consensus. Sanofi reiterated its financial forecast for the remainder of the year, but new, long-range guidance for 2024 and 2025 implies financial results lower than current analyst estimates.
• Abbvie reported adjusted third-quarter earnings of $2.95 per share, beating the consensus estimate. Revenue was $13.93 billion, down 6% year over year but better than consensus. Sales of the arthritis medicine Humira fell 36% from the previous year to $3.5 billion, largely due to generic competition, but were still in line with consensus. The company raised financial guidance for the remainder of the year.
• The FDA approved a new treatment for ulcerative colitis made by Eli Lilly. As Jonathan Wosen reports, the drug, called Omvoh, is the first to target an immune signaling pathway that plays a key role in sustaining the chronic, gastrointestinal disease.

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The drugs include Bristol Myers Squibb’s blood thinner Eliquis, Boehringer Ingelheim and Eli Lilly’s diabetes drug Jardiance, Johnson & Johnson’s blood thinner Xarelto, Merck’s diabetes drug Januvia, AstraZeneca’s diabetes drug Farxiga, Novartis’ heart failure treatment Entresto, Amgen’s rheumatoid arthritis drug Enbrel, Johnson & Johnson and AbbVie’s blood cancer treatment Imbruvica, J&J’s anti-inflammatory medicine Stelara, and Novo Nordisk insulins that go by names including Fiasp and NovoLog.

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MIAMI — Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye.

Antonio, who’s been legally blind for much of his 14 years, can see again.

The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio’s eyes?

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The reasons can vary, from quality control failures at manufacturing plants to surging demand, including significant interest that has at times squelched availability of drugs taken for weight loss.

But the problem is not easily fixed. Most active pharmaceutical ingredients are made in China, and boosting production in the U.S. is not like flipping a switch. Many of the drugs in short supply are generics made in India, where regulators often find serious production lapses. So what to do? Along with a colleague, Marta Wosińska, a senior fellow in economic studies at The Brookings Institution, has three ideas to alleviate shortages of generic sterile injectable medicines, in particular. They suggest the U.S. government should provide incentives to upgrade facilities and create a buffer inventory. But they also argue hospital purchasing is in need of an overhaul. We discussed the possibilities; our conversation has been lightly edited. 

I’ve been writing about drug shortages on and off for many years, but the problem never goes away. And now, it seems even worse. Obviously, something has to change. But what exactly has been lacking with our policies to date?

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At issue is a maneuver called alternative funding, which a growing number of drugmakers contend exploits their charitable programs. Basically, a plan sponsor excludes certain expensive drugs from coverage and taps an outside vendor to help patients obtain the medicines for free from patient assistance programs run by drug makers or foundations.

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Almost a year ago, three top investors at Lux Capital and Obvious Ventures announced they were leaving the firms, teasing on Twitter that they were starting “something new.”

That something is a new venture capital firm, which launched Wednesday with $350 million for its first fund.

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But research presented at the American Society of Hematology meeting in New Orleans suggested that a second-generation drug called zanubrutinib from BeiGene is about to unseat ibrutinib as “the king on the block” for the treatment of chronic lymphocytic leukemia, said Catherine Diefenbach, medical director of the lymphoma program at the NYU Langone’s Perlmutter Cancer Center.

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