liquid biopsy
Delfi unveils new data on lung cancer test
Delfi Diagnostics announced key data yesterday about its liquid biopsy test for lung cancer: The sensitivity of its test (the frequency at which it will flag positive when cancer is actually present) is 80%, and the specificity (how often the test flags negative when no cancer is detectable) is 58%.
Delfi's strategy is different than that of other liquid biopsy companies like Grail since it's focusing specifically on lung cancer, which has an established mode of screening through low-dose CT scans. Delfi's idea is to use a blood test to further stratify people who would already qualify for screening into higher-risk and lower-risk groups, motivating some to actually get the CT scans they already qualify for and allowing others to feel comfortable skipping them.
Read more from STAT's Matt Herper.
markets
Early-stage biotech unlikely to return to 'sugar high' days
From STAT's Jonathan Wosen: A report yesterday by Ernst & Young highlights the stark contrast in market performance between early-stage and late-stage biotechs.
Publicly traded U.S. and European biotechs with experimental drugs in Phase 1 trials lost 19% of their share value between December 2021 and March 2024, while companies with assets in late-stage trials saw their shares rise more than 20%.
A parallel trend was seen in venture capital. While a select number of biopharma companies raised huge rounds from VC firms last year, including mega-rounds of $270 million for RNA startup Orbital and $273 for Generate Biomedicines, the number of early-stage rounds in 2023 fell by 12%.
The report notes that lower interest rates could trigger a broader resurgence in the industry, but cautions that the industry is not likely to return to its "sugar high" days of 2020 and 2021, when companies with early-stage data could easily raise heaps of money at high valuations.
The industry overall is in a more precarious financial position — the fraction of companies with less than a year's worth of cash on hand has risen from 18% in 2021 to 31% in 2023.
However, some companies are benefiting from an unprecedented rise in private investments in public entities, or PIPEs, with deals during the first quarter of this year totaling $5.7 billion, an all-time high.
Regulation
Operation Warp Speed but for rare disease
From STAT's Jason Mast: The FDA said last week that it had selected the first participants in a program that top official Peter Marks once billed as an Operation Warp Speed for rare disease. The agency didn't disclose which groups were picked, but a couple companies disclosed their involvement Monday morning, including Denali Therapeutics and Neurogene.
The project is technically known as "Support for clinical Trials Advancing Rare Disease Therapeutics Pilot Program," or START — a less sexy name to distance the effort from the politics around the Covid vaccine effort and perhaps to reflect the program's dramatically smaller scope.
Rather than massive infusions of cash, companies will receive the ability to communicate with regulators more easily, so they can get better advice as they design studies and push toward approval. To be selected, sponsors had to be in clinical trials with either a gene or cell therapy or a more conventional drug focused on neurodegeneration. Denali is developing a new enzyme replacement for Hunter syndrome, a fatal neurodegenerative condition, while Neurogene is developing a gene therapy for Rett syndrome. Grace Sciences, a company developing a gene therapy for NGLY1 deficiency, an ultra-rare condition that affects the founder's daughter, also said it was selected.
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