Biotech, in brief

Merck said its experimental eye drug remigromig did no worse than Lucentis, an established treatment, in Brunello, a trial that ran for a year. [1]

The patients had diabetic macular edema: swelling at the back of the eye in people with diabetes, which blurs the centre of their vision. [1]

Lucentis, sold by Roche's Genentech in the US, blocks VEGF, a signal that makes blood vessels in the eye grow and leak. [1]

Remigromig works another way. It copies norrin, a natural molecule that helps keep the wall between the blood and the retina sealed. [1]

The trial's main question was how well patients read an eye chart after 52 weeks. Both doses of remigromig were judged no worse than Lucentis. [1]

More patients on remigromig had new fragile blood vessels, bleeding into the gel inside the eye, or stopped because of side effects. [1]

The report gives no numbers for those side effects, and Merck says it is running more analyses. [1]

In an earlier, smaller trial run by EyeBio, the company that first made the drug, no side effects were linked to it. [1]

Merck's research chief, Dean Li, said 30% to 40% of patients do not respond to today's VEGF drugs, or stop responding. [1]

He said Merck expects eye doctors to mix VEGF drugs and non-VEGF drugs like remigromig. [1]

A Wells Fargo analyst asked Li whether Merck could beat Lucentis, and raised the argument that Merck had picked the weakest rival. Lucentis competes with Regeneron's Eylea and Roche's Vabysmo. [1]

Merck will present the data at the American Academy of Ophthalmology meeting next month and discuss them with regulators. [1]

Why it matters. Watch the full numbers at next month's eye-doctors' meeting, above all how many patients had bleeding inside the eye, and whether a second trial against Lucentis matches Brunello in March. [1]

Kyverna Therapeutics reported one-year results on 24 September for miv-cel, a CAR-T treatment. [2] CAR-T means taking a patient's own immune cells, changing them to hunt a target, and giving them back. [2] In 26 people with stiff person syndrome, a rare disease of rigid muscles and painful spasms, walking speed improved by a median 49%. [2] Eight of the 12 who needed a walking aid before still did not. [2] In a second disease, myasthenia gravis, all five patients followed for a year kept their improvement. [2]

Why it matters. If the US regulator approves it, it would be the first CAR-T for a disease where the immune system attacks the body. [2] Rivals Novartis and Bristol Myers Squibb paused similar trials in August after dangerous inflammation, including three patient deaths in Novartis's trials. [2]

Acadia Pharmaceuticals said remlifanserin missed its main goal in the first part of a trial in people with Alzheimer's disease who have hallucinations and delusions. [3] On the higher dose, a symptom score fell 12.6 points by week 6, against 10.4 on a dummy drug. [3] The result fell just short of the line trials use to rule out chance. [3] The drug was built to avoid a heart-rhythm risk of Acadia's older drug Nuplazid, and none was seen. [3]

Why it matters. Acadia will still run the final-stage part, with only the higher dose, and its shares opened 11% lower. [3] Analysts noted that drugs borrowed from other conditions for these patients can cause lasting movement problems in some of them. [3]

Nature Medicine published a trial of evorpacept in 127 people whose advanced stomach cancer is driven by a protein called HER2 and had already been treated. [4] Evorpacept blocks CD47, a signal cancer cells use to stop immune cells from eating them. [4] Added to three standard drugs, it shrank tumours in 40.3% of patients, against 26.6% without it. [4] Where a fresh biopsy still showed HER2, the figures were 54.8% and 23.1%. [4]

Why it matters. It beat the comparison group by the margin set in advance, but missed a second test against a 30% benchmark from past trials. [4] Blood-count side effects were more common, though overall safety was similar. [4]

Novartis, the Swiss drugmaker that leads the market in radioactive cancer drugs, agreed this week to pay BoomRay Therapeutics of Suzhou, China, up to $900 million. [5] The money covers an upfront payment plus milestones and royalties for a drug not yet tested in people. [5] These drugs carry a radioactive atom to a tumour. Novartis sells two: Pluvicto for prostate cancer and Lutathera for rarer hormone-cell tumours. [5]

Why it matters. The deal came a day after Telix, an Australian company, agreed to buy ITM, whose drug would compete with Lutathera. [5] Novartis had dropped a mid-stage radioactive drug in July after disappointing results. [5]

Shanghai Henlius, a Chinese drugmaker that built its business on cheaper copies of biologic drugs, agreed a deal worth up to $440 million with Amberstone, a US biotech. [6] Amberstone will design T-cell engagers for up to two targets Henlius chooses. [6] A T-cell engager is an antibody that ties the body's killer T cells to a cancer cell. [6] Most approved ones treat blood cancers. [6]

Why it matters. Their known danger is an immune overreaction called cytokine release syndrome. [6] Amberstone says its designs switch on in the acid around solid tumours and far less elsewhere, so far only in lab tests; its own first drug should reach people next year. [6]

The Trump administration has signed drug-price deals with 26 companies, tying some prices for Medicaid, the US health plan for people on low incomes, to what other rich countries pay. [7] Two separate plans for Medicare, the US government's health plan for older people, would also peg US prices to other countries. [7] A Lancet study estimated those plans would save about $11.6 billion a year at first. [7] Some drugmakers say their deals exempt them, and the study's lead author estimates exemptions could cut the savings by nearly 80%. [7]

Why it matters. The Medicare plans, called GLOBE and GUARD, are due to start on 1 October and 1 January 2027, but are not final. [7] The US government has not said which companies are exempt, and the deals' details are confidential. [7]

City Therapeutics, founded in 2023 in Cambridge, Massachusetts, filed on 24 September to sell shares on the Nasdaq stock market. [8] It makes drugs based on RNA, the molecule that carries a gene's instructions, for rare diseases. [8] Its lead drug, CITY-FXI, is in an early trial against thrombosis, harmful clots inside blood vessels. [8] It has raised about $238.8 million from investors so far. [8]

Why it matters. ADARx, another RNA drug company, was expected to list on Friday. [8] Reuters reported that autumn listings have struggled as rising interest rates on government bonds and worries about AI shares weigh on investors. [8]

Adagio Medical, which makes a device that freezes heart tissue to stop dangerous fast heart rhythms, let go 25 of its 43 full-time staff. [9] Its board is exploring a sale or merger. [9] Its shares closed at 23 cents on Wednesday, down 53%. [9] It had about $7.7 million in cash in June, and Nasdaq warned it in August about its finances. [9]

Why it matters. The device uses liquid nitrogen at minus 196 degrees Celsius, and Adagio is still asking the US regulator to approve it. [9] It sent in its main trial results in May. [9]

Researchers at Oregon Health & Science University asked 20 people with early Huntington's disease to eat only within a six-to-eight-hour window each day for 12 weeks. [10] Huntington's is an inherited brain disease with no approved treatment that slows it. [10] A disease-severity score improved by 0.5 points on average; it usually worsens about one point a year. [10] A blood marker of nerve damage fell 13%. [10]

Why it matters. The study was small and had no comparison group, so it cannot show the eating pattern slows the disease. [10] The team is seeking money for a proper trial against normal eating. [10]

Scientists at Canada's Centre for Addiction and Mental Health scanned the brains of people with long COVID and of healthy people. [11] Dopamine is a brain chemical involved in motivation and movement. [11] People with long COVID had much less of a marker of dopamine nerve endings in the striatum, a group of brain regions. [11] Losses in different areas matched different symptoms. [11]

Why it matters. Long COVID is estimated to affect about 5% of people worldwide and has no proven treatment. [11] The team plans a trial of drugs that boost dopamine in the coming months. [11]

A study in Nature Metabolism followed 887 children at high genetic risk of type 1 diabetes for up to six years. [12] Researchers read the DNA of gut microbes in 12,151 samples. [12] Children whose gut microbes stopped maturing early had about three times the risk of type 1 diabetes. [12] Type 1 diabetes happens when the immune system destroys the cells that make insulin. [12]

Why it matters. It shows a link, not a cause. [12] It suggests early microbes and a child's own genes act together in who gets the disease. [12]

A trial published on 22 September tested magnetic pulses to the brain, alongside occupational therapy, in 29 people with spinal cord injuries in the neck. [13] Half got real pulses and half got a fake version, three times a week for five weeks. [13] The real group gained more arm and hand strength, feeling and independence in daily life. [13]

Why it matters. The trial is small, with 14 and 15 patients in its two groups. [13] Scores for pain, muscle stiffness and low mood rose in both groups. [13]

Researchers described a test that checks up to 1,200 DNA targets from a single punch of a newborn's dried blood spot. [14] It was tried on samples from 2,069 babies in the Philippines. [14] It found gene changes behind conditions such as spinal muscular atrophy and fragile X, and signs of a severe immune disorder, in one run. [14] Results take 36 to 40 hours. [14]

Why it matters. The authors built it for countries where DNA sequencing is too costly, or too contested, for public newborn screening. [14]

Scientists at Berlin's Max Delbrueck Center imaged a muscle calcium channel, RyR1, at six stages of opening inside its natural membrane, using rabbit muscle. [15] The channel releases the calcium that makes a muscle contract. [15] Neighbouring channels touch and help each other open, which one scientist compared to cogs in a clock. [15]

Why it matters. Many gene changes behind malignant hyperthermia, a dangerous reaction to some anaesthetics, sit exactly where the channels touch. [15] That contact point could become a target for new drugs. [15]

Altruist Biologics, a Chinese company that makes drugs for other firms, said its Suzhou factory passed an inspection by the European Medicines Agency with no critical findings. [16] It is the first time the site has passed. [16] The inspection supports a client's application to sell a biologic drug in the European Union. [16] Altruist says it holds about 20% of China's capacity of this kind. [16]

Why it matters. European approval of the client's drug depended on this inspection. [16] The US regulator recently rejected ITM's cancer drug on manufacturing grounds, before Telix agreed to buy ITM. [5]