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Good morning, everyone. Damian here with the latest twist for a futuristic cancer therapy, big money for longevity research, and a dispatch from London.
The need-to-know this morning
• Karuna Therapeutics said its application seeking the approval of a new treatment for schizophrenia was accepted by the Food and Drug Administration. The agency will review the drug, called KarXT, and render a decision on or before Sept. 26, 2024. Karuna has conducted two successful Phase 3 clinical trials showing KarXT reduced psychosis in people with schizophrenia.
Decoding the CAR-T safety scare
The FDA is investigating whether CAR-T cancer treatment, which involves genetically modifying immune cells, can lead to rare cases of blood cancer, the agency said yesterday, news that puzzled experts in the field.
The FDA isn’t restricting the use of any approved CAR-T medicines and noted that their benefits continue to outweigh any potential risks. The agency is reviewing reports of patients developing lymphoma in their T cells, which means looking into data on CAR-T products from Bristol Myers Squibb, Gilead Sciences, and Novartis.
CAR-T scientists said they were surprised by the development and unclear as to what might explain it. One possibility relates to the viruses used to engineer patients’ T cells, which could be straying from their targets. It’s also possible that earlier lines of therapy predisposed certain patients to developing lymphoma.
Saudi longevity fund is looking for biotech ideas
Hevolution, a Saudi Arabian investment firm, is launching an incubator to fund longevity research, planning to launch new startups around the most promising ideas.
As STAT’s Allison DeAngelis reports, the plan is to find 10 research initiatives and give each one $1 million over three years to refine their science. Representatives from Hevolution, Novo Nordisk, 5AM Ventures, RA Capital, and EvoTec will then evaluate each project, and the best among them will grow into venture-backed startups.
The effort is part of Hevolution’s broader goal of putting about $1 billion a year into anti-aging and longevity research, including grants for academic institutions and investments in biotech companies. The fund plans to invest in about 25 startups over the next three years.
Flagship opens a European front
In the more than two decades since it was founded, Flagship Pioneering has grown into a symbol of the biotech boom in Cambridge, and, specifically, in Kendall Square. But like many in their early 20s, the venture firm is venturing out.
Yesterday, Flagship marked the opening of its new office in London, which it says will be a hub for its operations in the U.K. and Europe. The expansion was announced earlier this year — with a subsequent announcement of another Flagship office in Singapore — but the firm and the U.K. cemented their pact at an event last night at the Royal Society, in a room lined with old books and where the WiFi password referenced Newton and a certain gravitationally motivated fruit.
“We used to happily say our entire ecosystem is a bicycle ride away from our offices,” Flagship CEO Noubar Afeyan said at the event. But now, Afeyan said, Flagship was embarking on an “experiment” with its Europe and Asia-Pacific hubs.
The firm — which this month unveiled a new company split between two Cambridges (that’s Massachusetts and the U.K.) called Quotient Therapeutics — was not coming in with a set plan for how it will operate, Afeyan said, but instead aims to learn from the existing scientific and investment infrastructure in the U.K. But Flagship does have particular ambitions, including building partnerships that could mean running clinical trials with the NHS, collaborating with U.K. research groups, and attracting U.K. investors into its fundraising.
Good help is hard to find
Back in May, Acelyrin was a roughly $2 billion company with a promising anti-inflammatory drug that looked like a potential pipeline in a product. Six months and two disappointments later, Acelyrin is worth just a quarter of its former value.
It started in September, when izokibep, Acelyrin’s drug, failed to outperform placebo at clearing lesions for patients with the common skin disease hidradenitis suppurativa, news that cut more than 60% off of the company’s stock price. That setback led Acelyrin to double down on its quality control, the company said, which is where the latest problem emerged.
It turns out that a third-party contractor made a mistake in izokibep’s next big test, a study in psoriatic arthritis, that resulted in some patients getting the drug in the wrong dosing sequence, the company said yesterday. Acelyrin said it is still working out the implications of that error, but the resulting 30% drop in the company’s stock price suggests investors are losing hope in one of 2023’s few IPO successes.
• Vertex pushes a donation program to widen access to cystic fibrosis treatments, but advocates say it’s not enough, STAT
• Bayer sees no quick fixes after bankers game out breakup, Bloomberg
• Neuroscience has to grapple with a long legacy of racism if it wants to move into the future, STAT