Biotech, in brief

Pressure to prevent rather than repair

there is no effective treatment once the nerve damage has set in [2]

Distance from mice to people

every result here is in animals; the first human trial starts this month [2]

Caution about psychedelic drugs

New Zealand approved MDMA for severe post-traumatic stress disorder the next day [9]

1. 1A mushroom compound given before chemotherapy prevented its worst lasting side effect. In mice.
2. 2Breast cancer pill cleared for an earlier moment
3. 3Two gentler drugs beat full chemotherapy in leukaemia
4. 4AbbVie reports myeloma results
5. 5GSK pays for a Chinese cancer drug

Researchers at the MD Anderson Cancer Center in Texas gave mice two doses of psilocybin before each of six rounds of chemotherapy. [1][2]

Watch whether the human trial can be run the same way: it means giving a psychedelic to cancer patients before chemotherapy, then waiting to count nerve damage that never appears. [2]

The US drug regulator gave accelerated approval on 4 September to Etcamah, also called camizestrant, for advanced breast cancer that is hormone-receptor positive and HER2 negative. [4]

It is taken alongside one of three existing drugs.

What is new is the timing: it starts the moment a blood test finds an ESR1 mutation, which tumours pick up to escape the standard first treatment.

Fewer than 5% of patients have that mutation at diagnosis. Nearly 40% have it once the first treatment stops working. [4]

The regulator said plainly that nobody has yet confirmed whether starting at the blood test, rather than when the cancer is visibly growing, does patients any good.

It has ordered further studies to find out. [4]

A cure shows itself in a sick person getting better, but prevention can only be proved by counting an illness that never arrives.

the same step, one stage back: the US regulator approved camizestrant for the moment a blood test spots a resistance mutation, before the cancer is seen to grow, and said nobody has yet proved that treating then helps

to prove the human version works, a trial has to give the shot to healthy people living where the ticks are and then count the infections that never arrive

prevention that already worked is invisible, so the case for the missing quarter has to be made about a disease most of their parents have never seen

the same chain stops one step earlier: the markers can spot the disease before the symptoms, and there is nothing yet to give the person they find

Chemotherapy nerve damage that has already set in has no treatment at all, so the people in this month's trial are the ones nobody can help later.

Nobody swallowing one can tell whether it worked for them, and neither can the doctor who prescribed it.

Fewer than 5% of patients have that mutation at diagnosis and nearly 40% have it after the first treatment fails, so the approval moves treatment earlier and lengthens it. [4]

Adults newly diagnosed with acute myeloid leukaemia, a fast-moving blood cancer, are normally given intensive chemotherapy if they are fit enough for it.

In a trial of 172 such adults, half got that and half got azacitidine plus venetoclax, two milder drugs previously kept for people too frail for chemotherapy. [5]

The median time before the disease came back or the patient died was 14.5 months on the two drugs, against 6.2 months on chemotherapy. Severe infections hit 28% against 41%. [5]

It was a phase 2 trial of 172 people, funded by AbbVie, which sells venetoclax.

That is a real result and one paid for by a company with a stake in it. [5]

AbbVie reported results from a late-stage trial called Cervino of etentamig.

The drug grips a cancer cell with one arm and one of the patient's own immune cells with the other, holding the immune cell against the target. [6]

The 393 patients had multiple myeloma, a cancer of the bone marrow, and had already been through at least two rounds of treatment covering all three standard drug types.

More than 130,000 people worldwide are diagnosed with it each year. [6]

Myeloma has shifted from a quick death to a long illness, but it remains almost always incurable and nearly everyone relapses.

This is a drug for the point where the standard three types have all stopped working. [6]

GSK, a British drugmaker, agreed on 3 September to pay Hutchmed of China $110 million up front, and up to $1.19 billion more later, for most of the rights to a cancer drug called HMPL-A830. [7]

Drugs of this family normally tie a homing antibody to a poison.

This one ties the antibody to a small molecule that blocks KRAS, one of the commonest faults in human tumours.

Hutchmed says laboratory tests showed the two halves working better together than either alone. [7]

Almost all the money is conditional.

GSK is paying $110 million for a drug that has never been given to a person, and the rest only if it survives the trials. [7]

Doctors gave 773 women with triple-negative breast cancer, an aggressive form with no hormone target to aim at, an immune drug called atezolizumab on top of standard chemotherapy before surgery.

Another 777 got a dummy. [8] After four years, 3.3 percentage points more of the atezolizumab group were alive with no return of the cancer.

The trial's own statistics say that gap could be chance. Survival differed by 0.7 points, and serious side effects were 75.3% against 73.4%. [8]

A planned look at subgroups suggested the drug did help women whose cancer had reached the lymph nodes.

A subgroup finding inside a trial that missed its main goal is a lead for the next trial, not a result. [8]

New Zealand's medicines regulator, Medsafe, approved pharmaceutical-grade MDMA on 4 September for people with severe post-traumatic stress disorder, a lasting condition that can follow a terrifying event and that resists the usual treatments. [9]

Two psychiatrists won the right to prescribe it alongside talking therapy. MDMA is best known as a party drug.

New Zealand is only the second country to allow this, after Australia in 2023, and the same government backed magic mushrooms for severe depression last year. [9]

The approval went to named prescribers rather than onto the general list, so the number of people who can actually be given it stays very small. [9]

Four directors at the US Food and Drug Administration published a joint statement on 3 September restating that Good Clinical Practice applies wherever a drug trial is run. [10]

That is the rulebook covering consent, record-keeping and the safety of the people in a trial.

Drug companies increasingly run trials in countries where patients are easier to recruit and costs are lower, and the agency inspects those sites.

The heads of its drug, biologics, device and cancer centres all signed it. [10]

A regulator does not usually announce that its existing rules still apply unless its inspectors have been finding places where they were not followed. [10]

The US drug regulator has accepted four digital health products into a pilot called TEMPO that lets them be sold before they have marketing authorisation.

Products from two companies, Cadence and Limbic, are among them. [11]

These are devices that use generative artificial intelligence to make decisions about a patient's care, and the agency has not settled how to judge them.

The pilot feeds a Medicare experiment that pays for technology helping older Americans manage long-term conditions. [11]

The agency is using the real world as the trial. The products reach patients first, and the evidence about whether they help is gathered afterwards. [11]

Paul Knoepfler, a stem cell scientist who tracks the industry, wrote on 3 September that the US drug regulator has kept sending warning letters to clinics selling unproven stem cell treatments, which he had expected to stop. [12]

His worry was that the US health secretary, Robert F. Kennedy Jr., said on a podcast in 2025 that he had taken such treatments himself in the Caribbean.

The best available count puts more than 2,000 such firms in the United States. [12]

It is a rare case of an agency continuing to act against a business its own political leadership has personally used and praised. [12]

The US Department of Defense and the National Institutes of Health, the country's main funder of medical research, have signed an agreement to work together on biodefence and preparing for pandemics. [13]

The New York Times obtained a copy; it covers chemical and biological defence research.

Democratic members of Congress objected, warning that the arrangement could let the military reach billions of dollars that Congress had set aside for infectious disease work. [13]

Moving money from the purpose it was voted for to another one is normally a decision taken in public by a vote.

This one was taken by two agencies signing a document. [13]

Two former senior officials at the US Centers for Disease Control and Prevention wrote on 4 September about a dispute over the death count.

Debra Houry and Demetre Daskalakis say the health secretary and the agency's current leadership are arguing with Pennsylvania about whether deaths there count as measles deaths. [14]

States report notifiable diseases using case definitions agreed slowly by epidemiologists through a joint council with the agency. The authors say that process is now under threat. [14]

If states and the national agency stop agreeing on what a case is, the national count stops counting anything, and each state's figure becomes its own. [14]

More than a quarter of five-year-olds starting school in Birmingham this autumn have not had both doses of the MMR vaccine, which protects against measles, mumps and rubella, according to figures from the UK Health Security Agency. [15]

The city is bottom in the West Midlands at 74.1%, against 90.4% in Shropshire. Eight in every hundred five-year-olds there have had no vaccinations at all.

A single MMR dose already gives up to 93% protection. [15]

It is the lowest rate in the region, published in the same week that the United States is arguing over how to count the measles deaths it already has. [14][15]

Ada Yonath, the Israeli structural biologist who first worked out how to map the ribosome, has died aged 87. [16][17]

The ribosome is the machine inside every living cell that reads genetic instructions and builds proteins from them.

Yonath found a way to grow crystals of it good enough for X-ray analysis, which most of her field had thought impossible.

She shared the 2009 Nobel Prize in Chemistry for it, and was the fourth woman ever to win that prize. [16]

Her later work showed how antibiotics kill bacteria by jamming their ribosomes, and that is the basis on which new antibiotics are now designed. [16]

Researchers led by Ben Lehner at the Wellcome Sanger Institute in England changed almost every single letter of the genome of phi X, a virus that infects bacteria, and recorded what each change did. [18]

Phi X has 5,386 letters and 11 genes, and in the 1970s it was the first genome ever fully read.

Most of the changes harmed the virus, but the team cannot explain why a quarter of the lethal ones kill it. [18]

Artificial intelligence tools built to spot harmful mutations did badly here, on the most studied genome there is.

The finding is that these tools need more real experiments, not more computing. [18]

Scientists using RNA-targeting CRISPR, a version of gene editing that cuts a cell's messenger copies instead of its DNA, normally check how much they silenced with a laboratory method called RT-qPCR. [19]

A paper in Nature Biotechnology on 1 September found that the guide molecules steering the cut survive the extraction step and jam that measurement.

The silencing then looks stronger than it was, across every CRISPR system the authors tested. [19]

The authors recommend a different enzyme and a second independent method. That leaves figures measured the old way open to question, and there are a lot of them. [19]

All known life writes its genes with four letters.

Researchers at the University of California San Diego showed that RNA polymerase, the enzyme that reads DNA and copies it into RNA, can accurately read an eight-letter alphabet containing four synthetic additions. [20]

Detailed imaging showed the enzyme handling the artificial letters much as it handles natural ones. That suggests cells could run an expanded genetic system using machinery they already have. [20]

The point of extra letters is to let engineered cells build things natural chemistry cannot spell. The barrier has always been whether the cell's own machinery would read them. [20]

A ribosome normally bumps into a messenger molecule at random and builds whatever protein that message describes.

Researchers reported in Nature on 2 September that they had written a protein's instructions into the ribosome's own RNA, so the ribosome makes that one protein and nothing else. [21]

They showed bacterial ribosomes doing it. Beyond making a single product at scale, it bears on how protein-making could have worked before cells were organised. [21]

Biotechnology mostly makes proteins in vats of bacteria, and the yield depends on ribosomes finding the right message among all the others. This removes the search. [21]

Nature published a long piece on 2 September on how near researchers are to explaining why some infections leave people ill for years.

A 2024 study put the number of people worldwide with long COVID at 400 million. [22]

The article follows Amy Proal, who caught an infection in 2004, was told her blood work was fine, was sent to a psychiatrist, and was later diagnosed with ME/CFS, a condition of severe exhaustion after infection.

She now runs a research foundation. [22]

Illness after a virus was dismissed for decades precisely because the standard tests came back normal. The current search is for the trigger those tests miss. [22]

A very large study published in Nature on 2 September linked genetic differences to the Big Five personality traits, the standard psychological set that includes how outgoing and how conscientious a person is. [23]

The links found are small. They were, though, less disturbed by family background than genetic links to education or income are.

The team then connected the traits to taking up smoking, to COVID-19 infection risk, and to whether a person joins a research study at all. [23]

That last one matters for every other genetic study. If agreeable and open people are likelier to volunteer, every volunteer database is tilted before anyone measures anything in it. [23]

In February 2025 the US drug regulator dropped the elaborate blood-monitoring programme that had governed clozapine for more than 30 years.

Clozapine is the only approved drug for schizophrenia that has not responded to anything else, roughly one person in three with the illness, and the only one shown to cut suicide. [24]

A child psychiatrist wrote on 4 September that removing the paperwork has not removed the caution that kept doctors from prescribing it. [24]

The monitoring existed because clozapine can destroy a type of white blood cell. The writer's point is that the rule was blamed for a reluctance that has outlived it. [24]

Nine cancer drugs approved in 2024 came to market at more than $400,000 a year, according to a tally by the Institute for Clinical and Economic Review, a group that weighs what a medicine does against what it costs. [25]

One CAR-T therapy now lists at $600,000 for a single infusion and a rival at $550,000. A daily pill for gut cancer is $520,000 a year.

Two decades ago the prospect of a $100,000 cancer drug drew fierce criticism. [25]

The bills land on Medicare, on employers, on patients and on everyone paying US taxes and insurance premiums, and nothing caps them beyond what a company thinks it can charge. [25]

An opinion piece in the New York Times on 4 September, by a biotechnology editor at the magazine Works in Progress, argued that the machinery for testing new drugs is now what holds cures back. [26]

It opens with Annette Harlow, whose lung cancer treatment stopped working the day after Christmas in 2019 and who was told there was nothing left.

A message the next day offered her a phase 1 trial in Houston of sotorasib, aimed at a gene fault called KRAS that scientists had long thought untouchable.

She was among the first people in the world to take it, and was alive more than six years later. [26]

The case rests on one woman who reached a trial through a message that happened to arrive. The argument is that this should be the system rather than the exception. [26]

Ultragenyx's drug for Angelman syndrome, a genetic condition causing severe developmental delay, failed its late-stage trial this week. [27]

Researchers told STAT on 4 September that the miss does not condemn the whole idea.

Mark Zylka, an Angelman researcher at the University of North Carolina, said it says nothing about the other trials still running.

One failed trial, he added, does not mean 'this is a bad mechanism'. Other companies are still testing similar genetic medicines. [27]

After a failure, whether the idea died along with the drug is a separate question, and it is usually left unasked.

Here the researchers closest to it say it did not. [27]

Abivax, a French drugmaker, has strong late-stage results for obefazimod, a pill for ulcerative colitis, a disease that inflames and ulcerates the large intestine.

Investors spent most of 2026 expecting somebody to buy the company for around $20 billion. [28] Nine months in, nobody has.

Adam Feuerstein, STAT's biotech columnist, wrote on 3 September that takeovers across the industry are running unusually hot, which makes the absence of this one harder to explain. [28]

The people who bought the shares expecting a sale are the ones carrying the wait, and the article says they are unhappy about it. [28]

Roche, a Swiss drugmaker, told Fierce Biotech on 3 September it is excited about a drug licensed from Hanmi of South Korea that acts on a receptor called CRF2, rather than copying the gut hormone that Ozempic-style drugs imitate. [29]

Researchers have known for decades that switching that receptor on protects animals from wasting muscle. Roche's pitch is weight loss that keeps lean muscle.

All the evidence so far is in animals, and Hanmi is running the first human trial. [29]

Losing muscle along with the fat is the main complaint about the current weight-loss drugs, and every large drugmaker is now hunting a version that does not. [29]

Superluminal Medicines raised money for drugs that switch on G-protein-coupled receptors, a family of proteins many existing medicines already target. [30]

Its first candidate aims at MC4R, a receptor that sets appetite and how much energy the body burns, and should reach human testing by the end of the year.

Rhythm Pharmaceuticals already sells an MC4R drug, Imcivree, for rare inherited forms of obesity. Superluminal is starting with Bardet-Biedl syndrome and obesity caused by damage to the hypothalamus. [30]

It is aiming first at rare inherited obesity, where the fault is known and the patient group is small, before going anywhere near the general market. [30]

Biomedical engineers at Duke University in the United States made a material that can be injected into the cavity left after an ischaemic stroke, where blood flow was cut off and tissue died. [31]

In mice, the material drew in the animals' own immune cells, encouraged new blood vessels, supported nerve tissue and improved movement.

It appears to work partly by turning neutrophils, immune cells usually associated with damage, into helpful ones. The work is published in Cell Biomaterials. [31]

Stroke care is almost entirely about the first hours. This aims at the months afterwards, when the damage is settled and very little is offered. [31]

Researchers at the University at Buffalo in the United States made a gel that is injected as a liquid into an arthritic joint and turns into a slippery semi-solid at body temperature. [32]

It stays in place for several weeks, slowly releasing drug-carrying particles, and lubricates the joint while it is there.

Osteoarthritis is the wearing away of the cartilage that cushions a joint. Injected drugs normally wash out fast, which is why they have to be repeated. [32]

Keeping the drug inside the joint is also about keeping it out of the rest of the body, which is what limits how strong a dose can be given. [32]

Scientists in Brazil ran a year-long trial in people with severe periodontitis, a gum infection that destroys the bone holding teeth in place. [33] One group got the standard antibiotics.

The other got omega-3 with low-dose aspirin. About 58% of each group reached the treatment goal.

The work came from teams at Albert Einstein Israelite Hospital, Guarulhos University, the University of Taubate and the Ribeirao Preto dental school at the University of Sao Paulo. [33]

Resistance grows with every course of antibiotics prescribed anywhere, so a treatment that matches them without being one is worth more than the numbers alone suggest. [33]

Researchers fed mice sucralose and stevia, two zero-calorie sweeteners used in diet drinks. [34]

Both changed the animals' gut bacteria, cut the useful compounds those bacteria produce, and altered the activity of genes involved in handling energy and inflammation.

Some of those changes then appeared in later generations of mice that had never eaten either sweetener themselves. [34]

It is a mouse study and the same question has not been put to people.

What makes it unusual is the claim that a change carried into animals that never touched the stuff. [34]

Scientists in Japan tested pterostilbene, a compound found in blueberries and grapes, on cultured mouse muscle cells. [35]

It cut the abnormal fat that had built up inside those cells, both by speeding up fat breakdown and by stabilising a protein involved in handling fatty acids.