Biotech, in brief

From Thursday, doctors in England can prescribe Enhertu for HER2-low advanced breast cancer, which affects about 1,000 women a year there. [1]

Doctors in Northern Ireland can prescribe it now too, and Wales is expected to follow. [1]

The drug treats HER2-low breast cancer, where the cancer cells carry only a little of a marker called HER2. [1] Enhertu is an antibody that locks onto that marker and carries a chemotherapy drug to the cell.

The makers, Daiichi Sankyo and AstraZeneca, have not substantially lowered their price since the drug was first refused, the BBC understands. [1]

What changed is the ceiling. NICE, which decides which medicines the NHS in England pays for, raised its upper limit from 30,000 pounds to 35,000 for one extra year of good health. [1]

That ceiling rose largely because of a trade deal with the United States, under which the UK government agreed to spend 25% more on medicines. [1]

At the end of August NICE also changed how it measures a patient's quality of life. [1]

Breast Cancer Now, the charity that campaigned for the drug, says thousands of people missed out and many died waiting. [1]

In the EU, advisers recommended the same drug on 17 September for a different, earlier group: patients with HER2-positive early breast cancer who still have disease after pre-surgery treatment. [2]

In that trial, DESTINY-Breast05, 92.4% of patients on Enhertu were free of returning cancer at three years, against 83.7% on trastuzumab emtansine, an older drug of the same kind. [2]

The lungs of 9.6% of patients on Enhertu became inflamed, and two of those cases were fatal. [2]

Why it matters. Wales is expected to follow England, and the European Commission still has to turn its advisers' opinion into an approval; watch whether other medicines NICE refused at the old ceiling are looked at again.

The US drug regulator approved Aqneursa for the loss of balance and coordination caused by ataxia-telangiectasia, an inherited disease that damages the nervous system from early childhood and also raises the risk of cancer. [3] It is the first treatment approved for that symptom. [3] The same drug was approved in 2024 for a different rare disease, Niemann-Pick type C. [3] In a 73-patient trial each person took the drug for 12 weeks and a dummy for 12 weeks, and scored better on a balance and speech scale while on the drug. [3]

Why it matters. Children with this disease have had nothing approved for the part of it that takes away their walking and their speech. The regulator gave the drug orphan status, the label used for medicines aimed at very small numbers of patients, and approval went to IntraBio. [3]

Novo Nordisk, the Danish maker of the weight-loss drug semaglutide, has bought three early-stage obesity programmes from Kallyope, a New York biotech. [4] None of them works the way semaglutide does. Semaglutide copies a gut hormone that tells the brain you are full; these three act on other targets. [4] The lead one, a weekly injection called K-554, is ready to enter its first human trial. [4] Novo would not give financial terms. [4]

Why it matters. Novo has been losing ground to Eli Lilly in obesity drugs, and buying in outside programmes is faster than growing its own. The day before, it signed a separate deal worth up to $1.4 billion with Orbis Medicines. [4]

Electra Therapeutics, a biotech near San Francisco, sold 23.3 million shares on Thursday evening and raised $350 million, above the $296.6 million it had told investors to expect days earlier. [5] It had planned to sell 21.7 million shares at about $15 each. [6] Most of the money goes to one drug, ipsoprubart: $220 million is set aside to carry it through a global trial in a rare condition where the immune system overreacts and attacks the body. [5]

Why it matters. In an earlier 12-patient study of that syndrome when it follows cancer, all 12 patients were alive after eight weeks. The trial is not due to finish recruiting until 2027, so the money is committed years before any answer. [5]

The US National Institutes of Health is preparing a clinical research centre on its campus in Bethesda, Maryland, to study people who believe a vaccine made them ill, including Covid vaccines. [8] Officials discussed the plan at an internal meeting and it could open in mid-October. [8] The amount of money has not been set. [8] The centre was first proposed in a 2025 report by a White House commission chaired by Robert F. Kennedy Jr., who runs the US health department and has long questioned vaccine safety. [8]

Why it matters. A patient advocate who developed lasting side effects after a Covid vaccine told Science it should have happened long ago. A paediatrician at Vanderbilt University called the effort laudable and said that if it is not done scientifically it will become anti-vaccine propaganda. [8]

The US health department has not decided whether Covid vaccines will be supplied through Vaccines for Children, the federal programme that gives free shots to children who are uninsured, on Medicaid, or Native American. [12] The programme reaches about half of the country's children. [12] States cannot order doses until the department signs off. [12] The hold-up is tied to the committee that normally votes on which vaccines the programme covers, which has been in legal limbo since a judge ruled in March that its members were not qualified. [12]

Why it matters. Children whose parents have insurance, or who can pay, can still be vaccinated. So the delay falls on the poorer half of America's children and on nobody else. [12]

The US Food and Drug Administration is trying to add more than 2,000 staff, a year after the cost-cutting team run by Elon Musk cut 3,500 jobs there in April 2025 and set off a wider exodus. [13] The head of the agency's biologics centre told drug manufacturers at a conference on Monday that it is hiring very actively. [13] STAT reports the push is stuck in bureaucratic delays caused by the same cuts it is meant to repair. [13]

Why it matters. A year ago the agency lost 3,500 people and more left after them. [13] Now it is advertising more than 2,000 posts and cannot fill them quickly, because of those same cuts. [13]

Around 80% of clinical trials miss the date by which they meant to have enrolled their patients, and fewer than 5% of adult cancer patients who are eligible for a trial take part. [15] Each delayed day of a late-stage cancer trial costs between $600,000 and $8 million in lost sales, and slow enrolment is estimated to cost the industry $50 billion a year. [15] A research-policy director at Merck told a Boston conference this week that US trial sites are already full. [15] The US regulator has opened a pilot to speed up first-in-human trials. [16]

Why it matters. A patient who cannot get into a trial loses the chance of a treatment that is not yet on sale anywhere. Drug companies at the conference said the answer is to run trials in ordinary community clinics rather than big teaching hospitals. [15]

Researchers analysed genetic and health data from more than 3.3 million people who had used a consumer DNA test, and found an inherited change in a gene called EGFR, known as T790M, in about one person in 15,850. [9] People carrying it had 25 times the odds of lung cancer, and no raised risk of 17 other cancers that were checked. [9] The variant spread in the United States from a single family line in the Southern Appalachian region about 200 years ago. [9]

Why it matters. Most lung cancer is caused by smoking, and this one is not. Lung cancer in people who never smoked is a growing share of cases, and the researchers say carriers of this variant may be the people that genetic testing and scanning would help. [9]

Researchers at WEHI in Melbourne, working with the Royal Melbourne Hospital, studied about 900 human gut samples and mini-guts grown from patients. [10] They found that in people with inflammatory bowel disease the cells lining the gut stay primed to die, even when the patient feels well and the disease looks controlled. [10] Inflammatory bowel disease covers Crohn's disease and ulcerative colitis, and affects around 180,000 Australians. [10] The work was published in Science. [10]

Why it matters. Doctors currently judge whether the disease is under control by how the patient feels. This says a test could look for something still going on underneath, though finding the fault is not the same as showing it starts a flare-up. [10]

Two economists followed the same adults with diabetes through more than ten years of a national US survey, from 2012 to 2023, and looked at what happened after they started a GLP-1 drug such as Ozempic. [11] They found little measurable change in depression symptoms, distress, self-rated health, employment or marriage. [11] Users did look different from non-users at first, but those gaps mostly disappeared once the same people were tracked over time. [11] It is a working paper, not yet reviewed by other researchers. [11]

Why it matters. Insurers and government programmes are deciding whether to widen who can get these drugs, and part of the case has been that the gains spread beyond blood sugar and weight. This study looked for those gains and did not find them. [11]

People taking GLP-1 weight-loss drugs are buying less food, eating smaller portions and buying smaller clothes, and food companies are trying to adjust. [17] Dan Sherry, a 58-year-old nurse in Holland, Michigan, was prescribed Ozempic in spring 2024 at 210 pounds and now weighs 160. [17] He eats out less, drinks less alcohol, usually leaves half a restaurant meal, and buys more meat and protein shakes. [17] He says his grocery bill is 10 to 15% lower. [17]

Why it matters. Food companies have built their sales on how much people eat. A medicine prescribed for something else is now changing that from outside their industry. [17]

BPC-157, a peptide sold online for pain, healing and muscle growth, has not been approved in the United States and has almost no evidence from human trials. [14] A data firm searched more than 15 million medical records and found 1,039 patients whose doctors had typed it into their notes by hand, because the compound has no drug code to track it by. [14] New confirmed users rose 33-fold between 2020 and 2026. [14] In July a panel of advisers to the US regulator, encouraged by health secretary Robert F. Kennedy Jr., voted to let pharmacies make it. [14]

Why it matters. The regulator had previously restricted making BPC-157 because of what it called significant safety risks and insufficient human data. Analysts estimate peptides like it could become a $2 billion to $3 billion market if they are legitimised. [14]

Doctors treated five patients whose pancreatic neuroendocrine tumours had kept growing after several other treatments. [18] They combined a therapy that carries a radioactive atom to the tumour with sunitinib, a pill that blocks the blood vessels feeding it. [18] Three patients had near-complete responses on scans and two had partial ones. [18] The rest of the paper is laboratory work in tumour cells, showing each treatment makes the other lethal at a lower dose. [18]

Why it matters. Five patients is a pilot, not a trial, and there was no comparison group. These tumours do respond to the radioactive treatment but rarely for long, and this is a first look at whether an existing pill extends that. [18]

Cellares, which builds automated factories for cell therapies, agreed to move GenomeFrontier's manufacturing onto its Cell Shuttle machine. [19] GenomeFrontier, based in Taiwan, is developing GF-CART01 for blood cancers including diffuse large B-cell lymphoma, and makes it without using a virus to carry genes into the cells. [19] The company says it has clinical results in Taiwan and is now recruiting for a first trial in the United States. [19] It is Cellares' first development partnership in Asia. [19]

Why it matters. CAR-T treatments are built one patient at a time, largely by hand, which is slow, variable and expensive. That cost is a large part of why so few patients ever receive one. [19]

Definium Therapeutics said on Monday that a single 100-microgram dose of DT120, a fast-dissolving LSD tablet, cut anxiety scores by 9.8 points after 12 weeks, against 4.7 points for a dummy pill. [7] The trial, called Panorama, enrolled 245 patients and also compared that dose against a 50-microgram dose meant to be felt but not to help. [20] It is the third late-stage win in a row, after an anxiety trial in August and a depression trial in June. [7] Definium plans to file for US approval in the first half of 2027. [7]

Why it matters. A patient who takes a psychedelic knows they have taken it, which is why the low-dose arm matters: it tests whether the benefit comes from the drug or from knowing. [20] Definium shares rose 5%. [7]