Biotech, in brief

The US drug regulator, the Food and Drug Administration, paused new patients joining trials of Biohaven's experimental epilepsy drug opakalim on 4 September. The company disclosed it on Thursday. [1][2]

The regulator said it had too little information to judge the human risk from a metabolite, a substance the body makes as it breaks a drug down, spotted during testing in rodents. [1]

Biohaven said the finding may be specific to rodents and that what it means for people is uncertain. It extended the enrolment pause to sites outside the United States on its own. [1]

More than 1,200 people have already taken the drug, and the more than 600 patients already assigned to treatment can carry on. [1]

One fully enrolled late-stage trial is still due to report in the second half of 2026. Enrolment in a second one, RISE-2, is paused. [1][2]

Minter, an analyst at the investment bank William Blair, called the earlier licensing deal prudent but necessary for a company whose cash had been dwindling. [2]

Nine days before the hold, on 26 August, Biohaven sold worldwide rights to the drug to South Korea's SK Biopharmaceuticals for up to $795 million, with $350 million paid straight away. [1][2]

Biohaven said in its filing that it gave SK all its clinical and non-clinical data before signing. Minter said that suggests SK was comfortable with the metabolite issue. [2]

The deal has not closed and still needs a US antitrust filing. Biohaven's shares fell more than 13% before the market opened. [1]

Opakalim, also called BHV-7000, is aimed at focal epilepsy, where seizures start in one area of the brain and keep coming despite at least two suitable anti-seizure medicines. [1]

Biohaven is the second company of that name, spun out by the same managers after the first was sold to Pfizer in 2022. Its drugs have since failed in spinal muscular atrophy and in depression, and one was rejected by regulators. [2]

More animal data is due in the coming weeks, to test whether the rodent finding matters in people. [1]

Why it matters. Watch the animal data Biohaven expects in the coming weeks. [1] RBC Capital Markets analyst Leonid Timashev says the regulator's demand for more data itself adds risk, because a concerning signal might turn up in it. [2]

The Royal College of Physicians, the British body that sets standards for hospital doctors, is reviewing 20 cases at University Hospitals Coventry and Warwickshire in England. Brain cancer patients there were prescribed the chemotherapy drug temozolomide for long stretches with little or no evidence of benefit. [3] Guidelines say the drug is usually taken for six cycles after radiotherapy, about six months, but patients told the BBC they received it for years, one of them for 16. [3] Fifteen of the 20 reviewed cases were judged unsatisfactory overall, and none was judged good practice. [3]

Why it matters. More than 40 patients are represented by lawyers, and one father says he believes eight years of the drug left his son with irreversible neurological damage, liver problems and muscle pain. [3] The hospital trust says it has put new checks on who prescribes the drug and for how long. [3]

Boehringer Ingelheim's experimental pill satoprodil failed to beat a dummy pill in two mid-stage trials in major depression, published in Neuropsychopharmacology. [4] The drug damps down one part of the NMDA receptor, a switch on brain cells that researchers have linked to depression for years. [4] One trial gave it to 243 patients alongside their existing antidepressant; the other gave it alone to 225. [4] At six weeks, none of the three doses moved depression scores meaningfully further than the dummy pill did, although the drug was well tolerated. [4]

Why it matters. Together the two trials dosed 468 people, so this is a clear answer rather than a small one that could go either way. [4] The authors say it is the most thorough human evidence yet on this target and should steer future work on the brain's glutamate system. [4]

Anthropic, the American company behind the Claude artificial intelligence models, said it had disrupted several efforts this year by scientists using its models for research that could help build biological weapons. [5] In a report on misuse it said it could not tell whether the work was legitimate or harmful, because the same biology that produces vaccines can also engineer dangerous germs. [5] It shut the work down anyway. [5] Jacob Klein, who runs threat intelligence at the company, said nobody announces they want to build a weapon, and the situations are nuanced. [5]

Why it matters. Andrew Weber of the Council on Strategic Risks, who read the report before release, called the findings chilling examples of state-backed weapons developers using fast-improving AI. [5] Biological misuse has drawn less attention than AI-enabled cyberattacks, partly because earlier cases turned up only in research settings. [5]

Robert F. Kennedy Jr, the US health secretary, said on Friday that measles killed an infant in Lancaster County, Pennsylvania, citing the county coroner. [6] Last month he had asked for two Pennsylvania measles deaths to be taken off the national count kept by the CDC, the US disease agency. [6] He now disputes only the second, saying the coroner found that infant had a fatal inherited condition and was about two months old. [6] Pennsylvania's health department says it has confirmed two measles deaths this year, and the national data still shows none for 2026. [6]

Why it matters. The national tally is the number the rest of the country reads during a fast-growing outbreak, and the state and the US federal government are now publishing different answers. [6] Public health experts called the earlier removal request extraordinary political interference in a state health department's authority. [6]

Researchers followed 510 young people aged 4 to 17 for a year after they started second-generation antipsychotics, medicines used for severe mental illness, and most of them had never taken one before. [7] Blood tests confirmed who was actually taking them. [7] Total cholesterol, fasting blood sugar and blood fats rose in a way that weight and fat gain did not explain, most clearly with olanzapine and partly with risperidone, and less with quetiapine and aripiprazole. [7] Insulin resistance, by contrast, tracked closely with body composition. [7]

Why it matters. The authors say watching a young patient's weight alone therefore misses part of the damage, and that the risk differs by which of these drugs is chosen. [7]

University of Maryland researchers combined toxin-blocking proteins that western diamondback rattlesnakes carry in their own blood to avoid poisoning themselves. [8] Alone, each protein countered only part of a venom's effect and none prevented death. [8] Mixed, the best combinations were about ten times more potent in lab tests than the sheep-derived rattlesnake antivenom now used, and they neutralised venom from several viper species. [8] Today's antivenoms are made by injecting large animals with venom and harvesting their antibodies, which is costly, variable and can trigger severe immune reactions. [8]

Why it matters. Snakebite kills an estimated 80,000 to 140,000 people a year and leaves hundreds of thousands disabled, mostly in rural places where antivenom is hard to reach. [8] The work is in the laboratory, and the team expects veterinary use first, with human treatments later. [8]

Researchers reported TRI-611, a drug that does not block its target but sticks it to the cell's own disposal machinery so the cell destroys it. [9] The target is ALK, a stuck-together protein that drives one form of non-small-cell lung cancer. [9] TRI-611 degraded every version they tested, including ones that had already stopped responding to the approved ALK blockers, and it reaches the brain. [9] Tumours shrank in cell lines and in mice carrying human tumours, both under the skin and inside the skull. [9]

Why it matters. Patients whose ALK-driven lung cancer stops responding to the current pills have few options left, and this cancer often spreads to the brain. [9] The authors say it is the first drug of this kind to reach the clinical stage against a cancer-causing gene fusion. [9]

University College London researchers built a small artificial bladder lined with human cells and flushed with flowing urine, then infected it with the strain of E. coli behind most urinary tract infections. [10] In that setting the bacteria stuck harder to the lining, invaded it, and built protected pockets inside bladder cells that antibiotics could not reach. [10] A cocktail of phages, viruses that infect and kill bacteria, wiped out those hidden bacteria. [10] Hospitals normally test antibiotics in still, nutrient-rich liquid, which is nothing like a bladder. [10]

Why it matters. Urinary tract infections cause around 400 million cases a year, and repeated courses of antibiotics are one of the routes to drug-resistant bacteria. [10] Jennifer Rohn, who led the work, said the model shows why antibiotics that look powerful in standard tests often fall short in patients. [10]

Prostate tumours that stop responding to hormone-blocking treatment often survive by changing what kind of cell they are, dropping their gland-like genes and switching on stem-cell programmes. [11] University of Michigan researchers, writing in JCI Insight, paired two drug classes at that switch. [11] BET bromodomain inhibitors interfere with the new identity; DNMT inhibitors switch the lost gland genes back on. [11] Either alone slowed growth, but together they suppressed it further in cell lines and in tumours grown in mice, at doses well below the recommended ones. [11]

Why it matters. Prostate cancer is the second-leading cause of cancer death among men in the United States, and nearly everyone on hormone-blocking drugs eventually becomes resistant. [11] DNMT inhibitors are already approved for blood cancers, which shortens the route to a human trial. [11]

A team writing in Science Advances described HITMAN, tiny antennas that turn a weak magnetic field applied from outside the head into a sharp electric field right at a cell. [12] That electric field unfolds proteins and tears cell membranes. [12] In dishes it cut the survival of drug-resistant glioblastoma cells taken from patients by 52.2%, against 10% for temozolomide, the standard chemotherapy, while sparing neurons and support cells. [12] In mice with tumours grown in the brain it slowed growth and extended median survival by more than half, with no whole-body toxicity. [12]

Why it matters. Glioblastoma resists both radiation and chemotherapy, and surgery cannot remove a tumour that grows into healthy tissue. [12] Temozolomide is the same drug at the centre of the Coventry review above, which is a measure of how little has changed in this cancer. [3][12]

CADD522, an experimental cancer drug that blocks a protein called RUNX2, protected the bones of mice whose ovaries had been removed to imitate the menopause. [13] The same mice also lost body fat and reversed some of the changes in fat handling that follow the menopause. [13] The University of East Anglia team published the work in npj Drug Discovery. [13] Osteoporosis, a thinning of bone that makes fractures easy, affects about one in three women and one in five men over 50, and existing drugs are held back by side effects and awkward dosing. [13]

Why it matters. A molecule already in development for cancer has a shorter path to a bone trial than a new one does. [13] This was mice, and the researchers say the drug needs further development before anything reaches women. [13]

About two dozen biotech reverse mergers have been announced in 2026, more than double the 10 across all of 2025, on a count by the advisory firm JB Strategy Partners. [14] In a reverse merger a private drug developer takes over a listed company that has little left except its stock market listing, and inherits the listing. [14] Bankers call those companies shells. [14] Tim Opler of the investment bank Stifel said there is no question the move has taken off, and dealmakers are now hunting for shells worth buying. [14]

Why it matters. Testing a single molecule can cost hundreds of millions of dollars, so how a young drugmaker reaches public investors decides which medicines get funded at all. [14] Branden Berns, a partner at the law firm Gibson Dunn, said the danger is running out of high-quality shells. [14]

CSL Seqirus published phase 3 results in The Lancet for aQIVc, a flu vaccine for older adults that combines three approaches already used separately. [15] Those are an additive called MF59 that strengthens the immune response, manufacture in cell cultures rather than hens' eggs, and a higher dose. [15] The trial enrolled 7,699 adults aged 50 and over across eight countries. [15] It beat an adjuvanted egg-based vaccine on all four flu strains and every age group tested, and matched a recombinant vaccine on three of four strains in the over-50s. [15]

Why it matters. Older people respond less well to flu shots because the immune system weakens with age, which is why enhanced versions exist at all. [15] Britain's medicines regulator has already approved a three-strain version, and these figures come from the company's own announcement of the paper. [15]

ARPA-H, the US government's health research funding agency, awarded up to $4.4 million for a one-year pilot called PROPEL. [16] It is led by Silvi Rouskin's laboratory at Harvard Medical School with Jonathan Weissman's laboratory at MIT and the Whitehead Institute, alongside a company called RNAV8 Bio. [16] The idea uses the shapes an RNA message folds into: those folds set how much protein the message makes, and a small molecule that binds a fold can change the amount. [16] Bacteria already work this way, through elements called riboswitches. [16]

Why it matters. Most medicines act wherever their chemistry carries them, and most gene therapies change DNA permanently, so a dial that turns protein output up and down would be a different kind of control. [16] Devan Shah, who founded RNAV8 Bio, said the hard part has always been predicting what an RNA sequence actually does. [16]

Teijin, its subsidiary Teijin Regenet and Shinshu University Hospital signed a joint research agreement on manufacturing methods for cell and gene therapies in Japan. [17] They will work through the hospital's cell processing centre on automation, efficiency, quality testing and quality control. [17] The partners said Japan is short of both trained manufacturing staff and the facilities to make these treatments at scale. [17] CAR-T therapies, which re-engineer a patient's own immune cells to hunt cancer, are built one patient at a time, which is what makes the factory the bottleneck. [17]

Why it matters. A cell therapy that works in a trial is not a treatment anyone can get until somebody can make it repeatedly and to standard. [17]

Researchers followed 204 people aged 13 to 38 judged at clinical high risk of psychosis, testing them at two months, one year and two years. [18] At the start their symptom scores and their best functioning over the past year looked alike, but the groups then diverged. [18] Visuospatial learning and working memory measured at the start went on shaping symptoms and functioning for two years. [18] A brain-wave pattern called microstate D tracked outcomes too, and a model combining it with subtype picked out who developed psychosis. [18]

Why it matters. Everyone in this group carried the same label at the start and ended up in very different places, so a marker that separates them early decides who is watched and who is treated. [18] This is one group of 204 people and the participant data are not public. [18]

A team writing in Molecular Therapy Nucleic Acids reworked twin prime editing, a way of rewriting DNA that nicks the strands rather than cutting both, to make it more efficient and more accurate. [19] They then pointed it at spinocerebellar ataxia type 3, an inherited disease that wrecks balance and coordination and has no approved treatment. [19] The fault is a stretch of repeated DNA inside a single gene called ATXN3. [19] The edit makes the cell skip past the repeat and stop the protein early, producing a shortened version already known to be less toxic. [19]

Why it matters. This was done in human cell lines engineered to carry the fault, not in animals or people. [19] Type 3 is the most common of the spinocerebellar ataxias, and a single faulty copy inherited from one parent is enough to cause it. [19]

A neural network called LiSep LSTM, trained to predict septic shock on a US intensive care database, was tested on an independent European one from Amsterdam UMC. [25] It scored 0.8851 on a standard accuracy measure, slightly better than at home, and flagged patients a median of 47 hours before onset. [25] But its precision ranged from 0.01 to 0.17, which means the large majority of its alarms were false. [25] Training on both datasets together improved the scores. [25]

Why it matters. Sepsis kills when it is caught late, so a 47-hour warning is worth a great deal, and a ward that stops believing the alarm has gained nothing. [25] The authors say local recalibration and a way to handle false alarms are needed before anyone uses it. [25]

Using electronic records from a large German university hospital covering 2016 to 2022, researchers trained a model to predict which inpatients would fall, then grouped the flagged patients by which factors had driven each alert. [24] That produced nine clusters, each matching a recognisable reason people fall and each pointing at a different prevention step. [24] The model scored 0.95 on one standard accuracy measure and 0.36 on a second one that is harsher when the event is rare. [24] The authors say it needs testing at other hospitals first. [24]

Why it matters. Falls cause around 684,000 deaths a year worldwide and account for up to 1.5% of health spending, and one-size-fits-all prevention does not work because people fall for different reasons. [24]

The US drug regulator granted priority review to Genentech's Enspryng, known generically as satralizumab, for MOGAD, an autoimmune disease in which the body attacks the optic nerves, spinal cord or brain. [23] There is no approved treatment for it. [23] The filing rests on a phase 3 trial called METEOROID, which the company says cut the risk of relapse by 68% against a dummy injection in patients aged 12 and over. [23] The drug is already approved for a related condition, neuromyelitis optica spectrum disorder. [23]

Why it matters. Each MOGAD attack can leave permanent damage, including vision loss, weakness and confusion, so preventing relapses is most of what a treatment can offer. [23] The figures come from Genentech's own announcement rather than from a published paper. [23]

Researchers writing in Nature Biotechnology ran six proteomic ageing clocks, models that estimate biological age from the proteins in blood, over serum from a published 12-week trial of rentosertib, an experimental drug for scarring of the lungs. [22] All six read the treated patients as biologically younger. [22] The authors say the clocks on their own cannot separate an anti-ageing effect from the drug simply treating the disease. [22] They used pathway analysis instead, and found shifts in cell-ageing and metabolic processes alongside the drug's anti-scarring action. [22]

Why it matters. Standard trial designs cannot detect whether a drug for an age-related disease also slows ageing itself, and the authors argue for measuring both inside one study. [22]

Surrey and Borders Partnership NHS Foundation Trust in England joined a trial of a drug meant to prevent Alzheimer's disease rather than treat it. [21] The drug attacks beta amyloid, a sticky substance that builds up in the spaces between brain cells in the disease. [21] Blood tests will be used to find the people most likely to benefit, before symptoms appear. [21] Ramin Nilforooshan, the consultant psychiatrist leading the trial, said research has focused for decades on treating Alzheimer's after symptoms start. [21]

Why it matters. About 982,000 people in the UK are living with dementia, on an Alzheimer's Society estimate, and this trial asks whether the disease can be headed off rather than slowed once it has begun. [21]

Manus and Hal Alper's laboratory at the University of Texas at Austin finished a programme funded by BioMADE, a US bio-manufacturing body, in which industrial yeast were engineered to break their own cell walls at the end of fermentation. [26] Products made inside yeast normally have to be freed by grinding the cells or dissolving them in hazardous solvents. [26] At 300-litre pilot scale, engineered Yarrowia lipolytica cut the energy needed for mechanical separation by more than half. [26] The team also achieved self-digestion in a production strain of brewer's yeast. [26]

Why it matters. A growing share of proteins, oils, vitamins and pigments is made by fermentation, and getting the product back out of the cells is a large part of the bill. [26]

Sixteen hospital patients aged 60 and over with pneumonia completed a randomised, blinded pilot in which some took a probiotic capsule called resB Lung Support for 14 days alongside normal care and the rest took a dummy. [20] Stool samples were taken at the start, at day 15, and at day 30 or discharge. [20] The probiotic group showed more of several gut bacteria thought to be helpful, and less Corynebacterium and Streptococcus. [20] The authors call the trial exploratory, say the findings should be read cautiously, and ask for larger studies. [20]

Why it matters. Sixteen people is far too few to show that anyone got better, and the study measured bacteria rather than recovery. [20] It is a marker of how far the gut-and-lung idea has travelled from theory towards hospital wards. [20]