Biotech, in brief

On 23 September, ten outside experts who advise the FDA, the US drug regulator, voted on Galleri, a blood test made by the US company Grail. [1][2]

Galleri looks for signs of many kinds of cancer in one blood draw, in adults aged 50 and over. [1] No test that looks for many cancers at once is approved in the US. [1][4]

The panel voted three times. [2] All ten said the test is safe. [1] Six of ten said it works, and seven said its benefits outweigh its risks, with one abstaining. [1][2]

The FDA does not have to follow the panel, but it weighs its votes heavily. [1] A final decision is expected in the coming months. [1]

Galleri has been sold in the US since 2021 through a regulatory exception, without FDA approval. [3] About 117,000 people took it in the first half of this year, outside research studies. [3]

Most buyers pay the $949 list price themselves. [3] Grail lobbied for a law that lets Medicare, the US government's health insurance for older people, pay for the test from 2028, if the FDA approves it. [3]

In Grail's US study, the test flagged 35% of the cancers found within a year. [1] It raised a false alarm in about 15 of every 10,000 people without cancer. [5]

The test caught late cancers far more often than early ones. [5] In the US study it flagged 21% of stage 1 cancers, the earliest, and 63% of stage 4, the most spread. [5]

A paper in Nature Medicine on 22 September covered more than 35,000 people. [4] The test found a cancer signal in 287 of them, and about 60% of those were later diagnosed with cancer. [4]

When the test did find cancer, it named the right part of the body about 91% of the time. [4]

In the US study, 159 of 218 people who were checked after a positive result had at least one invasive procedure. [5] Among those who turned out not to have cancer, 47.8% had one. [5]

In February, a big trial in the UK found the test did not cut the number of cancers diagnosed at stage 3 or 4. [3][6] No trial has shown that tests like it reduce cancer deaths. [3]

Why it matters. The FDA decides in the coming months; watch whether it follows the panel, and whether it limits the test to people at higher risk of cancer, as one researcher suggested. [1][4]

Heidi Overton, President Trump's pick to run the FDA, goes before the US Senate health committee on 24 September. [9] In an opening statement STAT saw in advance, she says Trump told her to go fast. [8] She says slow FDA rules are pushing drug investment to China. [8] She backs Operation Trailblazer, an FDA effort started this year to speed up clinical trials. [8] The agency has lost more than 3,000 staff in the past year. [9]

Why it matters. If the US Senate confirms her, her view of speed will shape decisions like the one on Grail's test. The last FDA head, Marty Makary, resigned in May after clashes with officials in the Trump administration. [9]

Longeveron's stem-cell treatment, laromestrocel, did not help babies born with hypoplastic left heart syndrome, where the left side of the heart is too small to work. [10] In a mid-stage trial, 40 infants got the cells during their second heart operation. [10] After 12 months, the right side of their hearts pumped no better than in babies who had surgery alone. [10] No side effects were judged to come from the cells. [10]

Why it matters. Longeveron's shares fell 60%, and it had $10.1 million in cash at the end of June. [10] It is cutting costs and has hired an investment bank to review all its options. [10]

Roche's monthly injection sefaxersen met its main goal in a late-stage trial in IgA nephropathy, a disease in which the immune system damages the kidneys' filters. [11] After 37 weeks, patients on it leaked less protein into their urine than patients on a dummy injection. [11] Protein in the urine is a sign of kidney damage. [11] The trial stays blinded until week 105 to measure kidney function itself. [11]

Why it matters. It would compete with Novartis's Fabhalta, a twice-daily pill that blocks the same protein, factor B, and is already fully approved. [11] Roche hopes the early result earns a faster approval. [11]

Immunovant stopped testing its antibody IMVT-1402 in cutaneous lupus, a form of lupus that inflames the skin. [12] In 57 adults, skin scores after 12 weeks were no better than on a dummy drug. [12] The drug blocks FcRn, a protein that keeps the body's own antibodies in the blood for longer. [12] The company said it stopped because of the result and because rival drugs are further ahead. [12]

Why it matters. Immunovant is still testing the same drug in five other diseases, including Graves' disease and myasthenia gravis. [12] It caused no safety worries in this trial. [12]

China's government wants a quarter of the world's first-in-class drugs, meaning drugs that work in a way no earlier drug does, to come from China by 2030. [13] The goal is in a five-year plan published on 18 September by China's drug regulator and eight other agencies. [13] It also wants 50 Chinese drugmakers each selling at least $1.5 billion a year. [13] A GSK executive said at a panel that its deals with Chinese companies such as Hansoh are paying off. [14]

Why it matters. Chinese companies are now a major source of new drugs for US and European drugmakers. [13][14] The US Treasury is drafting rules that would probably keep most of those deals legal. [13]

A planned Trump administration order that would let political appointees veto grants from the NIH, the US government's medical research agency, is looking dead. [15] That is according to Senator Susan Collins and a person familiar with the decision. [15] Collins, a Republican who leads the US Senate's spending committee, wrote on Tuesday urging officials not to go ahead. [15] Democrats, including Senator Patty Murray, also objected. [15]

Why it matters. The panel would have included Russell Vought, President Trump's budget director, who had accused the NIH of funding work that does not match the Trump administration's goals. [15] Collins wrote that grants should be decided on scientific merit, not political ideology. [15]

Enveda, a Colorado company that uses AI to find drug-like molecules in plants and microbes, raised $311 million from investors. [16] It has three drugs in human testing. [16] One, ENV-294, is an eczema pill that shrank the area and severity of the rash by 68% on average after 28 days, in a small early trial. [17] Another, ENV-308, copies a hormone the body releases after exercise. [16] It is being tested for keeping weight off after people stop weight-loss drugs. [16][17]

Why it matters. The round is as big as some of this year's largest biotech share sales on the stock market. [16] Its drug results so far come from small early trials. [17]

Basecamp Research raised $140 million to train its EDEN AI models, which design pieces of DNA. [18] It wants to use them to make cell therapies that are simpler and cheaper to produce than today's. [18] In a study not yet checked by other scientists, half of the DNA-inserting enzymes EDEN designed worked in human cells. [18] Investors include Nvidia's venture arm and Anthropic, the company that makes the Claude AI models. [18]

Why it matters. It is one of two AI drug-discovery rounds announced on 23 September, alongside Enveda's. [16][18] Basecamp's results so far are all in the lab, not in patients. [18]

The US Department of War, the country's defence department, gave Flagship Pioneering a $47 million contract over five years. [19] Flagship starts new biotech companies. [19] The money is for medical countermeasures: vaccines, drugs and tests for use after a biological or chemical attack, or a new natural outbreak. [19] The first programmes are at Apriori Bio, which uses AI to predict how viruses change, and Prologue Medicines, which designs proteins. [19]

Why it matters. The contract adds military money to AI biology. [19] It comes in the same week that US civilian research money at the NIH was the subject of a fight over who decides grants. [15]

Boehringer Ingelheim, a German drugmaker, expanded its deal with Variant Bio, a Seattle company, adding more than $250 million in possible payments. [20] Variant uses AI on genetic data from people in many parts of the world to find new drug targets. [20] The deal began in January, worth $120 million, for kidney disease, and now covers heart disease too. [20] It came a day after Boehringer signed a deal worth up to $1 billion with another AI company, Envisagenics. [20]

Why it matters. Boehringer already sells Jardiance, a drug for heart failure and kidney disease. [20] The companies did not say how much is paid now and how much depends on future results. [20]

Researchers at Boston Children's Hospital made tiny fat bubbles, called liposomes, that release a drug very slowly. [21] They filled them with tetrodotoxin, a nerve poison from pufferfish, and injected them near a nerve in rats' legs. [21] The leg stayed numb for two to three weeks. [21] A numbing drug sold today lasted four to eight hours. [21] No harm was seen at the injection site or elsewhere in the body. [21]

Why it matters. The team says it could one day replace opioids for pain after surgery. [21] Tetrodotoxin is not approved for patients, and the tests so far are only in rats. [21]

Many antibody drugs are made by living cells grown in a nutrient liquid, and the liquid is costly. [22] Researchers at MIT used an electric field to pull waste out of used liquid so it can be reused. [22] In tests they recovered 87.5% of it. [22] Factories that recycle the liquid today usually reuse less than half, and reusing it without cleaning can slow the cells down. [22]

Why it matters. The method is still at pilot stage. [22] The researchers say it still has to be checked for any effect on the quality of the drug. [22]

Researchers at Sartorius built a control system for tanks of hamster-ovary cells, the standard cells used to make antibody drugs. [23] It adjusts three flows at once: fresh food in, old cells out and product out. [23] It raised production by 68% while keeping at least 95% of the cells alive. [23] Much of the testing was done on digital copies of the process. [23]

Why it matters. It lets a factory switch between running steadily and squeezing out more product without stopping the run. [23] It stayed stable even when the computer model did not match the real process well. [23]

Drug companies and investors are returning to osteoporosis, the thinning of bones that makes them break easily in older adults. [24] Lilly, Merck, Novartis and GSK each once had a drug for it. [24] Awkward rules for taking the drugs and safety worries held their use back. [24] Many people who could be treated take none of today's drugs. [24] A long-awaited change in US regulation has renewed interest, and Lilly is hiring scientists for the work. [24]

Why it matters. Venture investors are now making bone health one of their priorities, a turn for a field most drug companies had left. [24]

Hundreds of studies that companies promised to run after their drugs or devices were approved are listed by the FDA as delayed, an analysis by KFF Health News found. [25] Some are more than a decade late. [25] For some, the maker was still writing a plan for the study. [25] The FDA uses these studies to settle questions left open at approval. [25]

Why it matters. Until they report, patients and whoever pays, including Medicare, may be paying for products that do more harm than good. [25] Galleri's biggest open question, whether it saves lives, also needs longer studies. [4]