Biotech, in brief

Novo Nordisk, the Danish maker of the weight-loss drug Wegovy, will pay $300 million up front to Jiangsu Hengrui Pharmaceuticals for an experimental weight-loss drug called HRS-1596. [1][2]

Hengrui can earn up to $2.3 billion more if the drug passes later tests and sells. That would take the deal to as much as $2.6 billion. [2][1]

Hengrui is China's largest drugmaker by stock-market value. It announced the deal on Tuesday. [2]

No person has taken the drug yet. China's regulator has cleared it for a phase 1 trial, the first small test of safety in people. [1][2]

It is designed as a pill taken once a week. The weight-loss pills sold now, Novo's Wegovy pill and Eli Lilly's Foundayo, are taken every day. [2][1]

Like Lilly's injection Zepbound, it copies two gut hormones, GLP-1 and GIP. They curb appetite and help the body handle sugar. [1][3]

Novo gets the rights everywhere except mainland China, Hong Kong, Macao and Taiwan. It says it will run trials around the world, but gave no dates. [2]

Novo has lost ground to Lilly in weight-loss drugs. It has installed a new chief executive, laid off thousands of workers and bought other companies' experimental drugs. [3]

Earlier this month it bought three experimental obesity drugs from the biotech company Kallyope. [3] Last week it agreed a 1.17 billion euro deal for Nanexa's way of making injections last longer. [1]

Hengrui has signed six licensing deals with US or European drugmakers since the start of 2025, more than any other Chinese drug company, by BioPharma Dive's count. [3]

Hengrui's obesity drugs are also the base of the startup Kailera Therapeutics, whose first sale of shares in April set a record. [1][3]

A Leerink Partners analyst said Kailera may have had the right to buy this drug first. He also said there is little data to judge it before tests in people. [3]

Why it matters. Watch the first human trial in China. It is the first test of whether a once-a-week pill of this kind is safe in people, and Novo has given no dates for its own trials. [1][2]

The Dutch company uniQure said on Tuesday that its one-time gene therapy AMT-130 slowed Huntington's disease by 44% after four years in 12 high-dose patients. [4][5] Huntington's is an inherited disease that destroys nerve cells. [5] The 44% was measured against a database of untreated patients, and it was not statistically significant, so chance could explain it. [5] At three years uniQure had reported 75%. [4] A second score, for daily tasks such as managing money, held at 61%, against 60% a year earlier. [4]

Why it matters. The US drug regulator is reviewing uniQure's request for early approval, filed this month on the three-year data. [4] Shares fell about 40% on Tuesday, and uniQure says the database lost its fastest-declining patients, which makes its drug look weaker. [4][5]

Novo Nordisk said on Tuesday that health records from 636,525 adults with type 2 diabetes showed a 6% lower risk of serious heart and blood-vessel events in people who raised their Ozempic dose. [6] They were compared with people who switched to Mounjaro, the rival drug from Eli Lilly. [6] Within 720 days, 36.9% had raised their Ozempic dose to 2 mg and only 5.7% had switched. [6] The results were shown at the European diabetes meeting in Milan. [6]

Why it matters. This was a study of records, not a trial, so nobody decided by chance who switched and who stayed. Novo and Lilly are fighting over a US obesity market that analysts put at $100 billion to $150 billion by 2030. [6]

Eli Lilly said on Tuesday that its daily weight-loss pill Foundayo lowered weight and blood sugar more than a higher, 25 mg dose of Novo's Ozempic pill, in adults with type 2 diabetes. [7] The two pills were never tested in one trial. Lilly compared results from two earlier late-stage studies. [7] It put Foundayo ahead by 1.5% to 2.4% more weight lost, and by 0.3% to 0.6% more off HbA1c, a measure of average blood sugar. [7]

Why it matters. Novo said people with type 2 diabetes lose less weight in studies than people with obesity alone. [7] Its 25 mg pill is not yet approved in the US for diabetes, and it expects a decision by the end of 2026. [7]

A phase 3 trial, the large final stage of testing, at 46 sites in China tried safiglipron, a daily pill that copies the gut hormone GLP-1. [9] It can be taken with food, at any time of day. [9] The 284 adults had early type 2 diabetes, managed with diet and exercise alone. [9] After 32 weeks, HbA1c, a measure of average blood sugar, fell 1.20 to 1.45 points more than on a dummy pill. [9] Between 71.4% and 77.8% reached the usual target, against 25% on the dummy. [9]

Why it matters. The trial's main measure was blood sugar, and weight fell only 0.65% to 3.56% more than on the dummy. [9] Side effects made 6.9% stop on the highest dose, against none on the dummy. [9]

BioNTech, the German company that first developed the COVID vaccine it sells with Pfizer, said on Monday it will close its sites in Idar-Oberstein, Marburg and Tuebingen. [10] It had tried since May to sell them, while moving its COVID vaccine production to Pfizer. [10] No buyer was found. [10] The wider plan, which also covered a site in Singapore, affects up to 1,860 jobs. [10]

Why it matters. BioNTech says it has agreed redundancy terms with its works council, the body that speaks for staff. [10] Its two co-founders said in March that they will leave by the end of this year to start a new company. [10]

Roche's experimental pill RO7589831 was tested in 88 people with advanced cancers that carry MSI, a fault in how cells fix errors when they copy DNA. [11] The pill blocks WRN, a protein that these cancer cells need to survive. [11] Of 66 patients whose tumours could be judged, seven saw them shrink clearly, and in 74.2% growth stopped for a while. [11] Those responses lasted a median of 10.2 months so far, and some were still going when the results were published in Nature Medicine on 29 September. [11]

Why it matters. This first test in people was about safety and dose, and nausea affected 54.5% of patients, mostly mildly. [11] MSI is found in 4% to 7% of advanced bowel cancers and 15% to 25% of advanced womb cancers, and immune drugs do not help all of those patients. [11]

A study paid for by Eli Lilly, published on 28 September in the International Journal of Obesity, compared tirzepatide, which Lilly sells as Zepbound, with Novo's semaglutide, sold as Wegovy. [8] The two drugs were never given in one trial. The authors lined up two separate trials, each against its own dummy group. [8] On that basis, the two higher tirzepatide doses took off 5.1 kg and 6.5 kg more than semaglutide, in adults with obesity but no diabetes. [8]

Why it matters. Six of the ten authors work for Lilly. [8] Lining up two trials cannot rule out that their patients differed, which is what a trial giving both drugs to one group, split by chance, is for.

Doctors at the MD Anderson Cancer Center in Texas gave 13 people with myeloma, a blood cancer that had come back, the antibody daratumumab after a stem-cell transplant late in their illness. [17] For six of them it was a second transplant, and 11 also took the pill pomalidomide. [17] The aim was to keep the cancer from coming back, a job called maintenance. [17] After a median of about four years of follow-up, half of them had gone 45.1 months before the cancer grew again. [17] Four years after the transplant, none had died. [17]

Why it matters. The study had one group and no comparison, and it was funded by Janssen, part of Johnson & Johnson. [17] Newer immune-cell treatments have challenged the place of late transplants in myeloma care. [17]

Gene therapies often use AAV, a small virus, to carry a new gene into cells, and it can land in the wrong tissue. [12] Researchers reported on 29 September in Nature Materials a coat that stops the virus entering any cell. [12] Different coats come off only in the liver, under near-infrared light, or where chemicals from inflamed tissue build up. [12] In mice with a damaged heart, the virus delivered a repair gene to the heart muscle. [12]

Why it matters. It has been tested only in mice, and most results in mice are never repeated in people. [12] The aim is to put a gene only where it is needed.

Lipid nanoparticles, tiny fat bubbles, carry mRNA into cells, but they work less well as the message gets longer, a team reported on 28 September in Nature Biotechnology. [13] Gene-editing tools are long messages. [13] The team screened 384 fat-like molecules with a long test message and picked the best, LC-1. [13] In mice it reached editing rates of up to 79% in the liver, 48% in the brain and 27% in the lung, up to four times the standard bubbles. [13]

Why it matters. It also carried a base editor, which changes one DNA letter, to genes including PCSK9, which affects cholesterol, and a cystic fibrosis fault. [13] All of this was in mice. [13]

COVID mRNA vaccines use a modified form of one RNA letter, called N1-methylpseudouridine, which stops the body attacking the mRNA itself and makes it last longer. [14] Earlier studies suggested it can make the cell's protein-building machine slip one letter along and make stray protein pieces. [14] A team reported on 28 September in Nature Communications that the slip happens at one short run of letters, UUUC. [14] Rewriting that run as UUCC or UUUU stopped it. [14]

Why it matters. The slip was measured in cells and test tubes, not in vaccinated people. [14] The authors say careful choice of letters can remove the risk from future mRNA medicines. [14]

Researchers at Baylor College of Medicine used AI and a large protein screen to find molecular glues that destroy VAV1, a signalling protein in immune cells. [15] A molecular glue sticks a target protein to the cell's own disposal system, so the cell breaks it down. [15] Their best compound, NGT-201-18, lowered VAV1 in human T cells and calmed their activity. [15] It also destroyed a second protein, LIMD1, that it was not aimed at. [15]

Why it matters. VAV1 is linked to blood cancers and to autoimmune diseases, where the immune system attacks the body. [15] The compounds have not yet been tested for safety or in animals with disease. [15]

Doctors in Varese, Italy, moved 30 patients with wet macular degeneration from faricimab to an 8 mg dose of aflibercept. [16] Wet macular degeneration is leaking blood vessels at the back of the eye, and a leading cause of lost sight in older people. [16] After three monthly injections the retina was less swollen, and eyesight stayed the same. [16] Patients then went longer between injections. [16]

Why it matters. The study had no comparison group and was partly paid for by Bayer. [16] Longer gaps matter because each dose is a needle into the eye, and frequent injections can make patients give up treatment. [16]