Biotech, in brief

Bristol Myers Squibb (BMS), a big US drugmaker, said on Friday that a patient died in an early trial of zola-cel, its experimental cell treatment for multiple sclerosis. [1]

Multiple sclerosis (MS) is a disease in which the immune system attacks the coating around nerve fibres. That breaks the signals between the brain and the body. [1]

Zola-cel is a CAR-T treatment. A patient's own immune cells are collected, changed in a lab to hunt cells carrying a protein called CD19, and put back in one drip. [1][2]

The patient had a fast-relapsing form of MS. Later, they developed movement problems that steroids and an antibody drip did not fix. [1][2]

Doctors then gave a high dose of cyclophosphamide, a strong chemotherapy drug. The patient developed septic pneumonia, a lung infection that set off a reaction through the whole body, and died the next day, 44 days after the treatment. [1][2]

BMS says no other patient has died among the 190 people given zola-cel in its early trials for immune diseases. [1][2]

The MS trial, called Breakfree-2, treated 66 people: 30 with relapsing MS and 36 with forms of MS that worsen steadily. [1][2]

Its main goal was to measure safety. 42.4% of patients had cytokine release syndrome, an immune over-reaction, and five had brain-inflammation symptoms that cleared in about two days. [1]

BMS said all 60 patients given the second dose level were still off their other MS medicines at the last check, nine of them for more than a year. [2]

In September BMS paused its zola-cel studies to review safety. It did so after Novartis, a Swiss drugmaker, paused eight trials of rap-cel, its own cell treatment. [1]

BMS shares were about 2% lower in early trading on Friday. [1]

Why it matters. The next thing that decides zola-cel's future is what BMS's safety review finds and when it ends the pause on its studies that began in September. [1]

India's government will cap the mark-up that hospitals, distributors and chemists add to 137 more cancer drugs at 30%, from next month. [3] It acted after India's supreme court called the mark-ups extortion. [3] The case began with Sarojam Radhakrishnan, a retired bank clerk in Kerala who could not afford her breast-cancer drugs; she died in 2022. [3] India's drug-price body found mark-ups on some cancer drugs averaging 170%, and more than 700% in the worst cases. [3] India's government says prices will fall by up to 70%. [3]

Why it matters. Patients are expected to save 2,500 crore rupees (about 199 million pounds) a year in a country with about 1.56 million new cancer cases a year. [3] The court has given India's government until 27 October to say why the cap covers only cancer drugs. [3]

Takeda, a Japanese drugmaker, showed final-trial results for arcotatug tavatecan at ESMO, Europe's biggest cancer meeting, in Madrid. [4] It is an antibody-drug conjugate: an antibody that carries a cell-killing drug straight to cancer cells. [4] The trial had 464 people in China and Japan with advanced stomach cancer and at least two earlier treatments. [4] Patients on the drug lived a median 10.2 months, against 6.9 months on the usual drugs. [4] One patient died suddenly of an unknown cause, and Takeda is still looking into it. [4]

Why it matters. Takeda plans to ask Japan's regulator for approval, and Innovent, the Chinese company that made the drug, has already applied in China. [4] Innovent could earn more than $10bn from Takeda if this drug and a second one succeed. [4]

BMS and SystImmune said iza-bren cut the risk of the cancer growing or death by 59% against chemotherapy, in a final trial of 383 patients. [5] They had a kind of breast cancer fed by hormones, and had already had chemotherapy. [5] The cancer was held back for a median 8.2 months, against 4.0 months on chemotherapy. [5] The trial was run in China by Sichuan Biokin, SystImmune's parent company. [5]

Why it matters. Nobody knows yet whether iza-bren helps people live longer, because the survival numbers are not mature. [5] BMS and SystImmune develop the drug together under a licence deal. It has now shown a benefit in five final trials for five different uses. [5]

Pathos, a New York company that uses AI to choose and steer drug trials, raised $237m in a round led by BMS. [6][8] The round values Pathos at about $3bn. [6] Pathos will pay Tempus, a health-data company, $55m over five years for records from hundreds of thousands of people with brain and nerve diseases. [6][7] For three years, only BMS may use Pathos's AI for brain-disease drugs, starting with Alzheimer's. [6][8]

Why it matters. Pathos's chief executive says the aim is to raise the odds that a drug succeeds in its trials. [7] Its own drugs include a late-stage cancer drug licensed from Alphamab Oncology, another drug company. [7]

Roche, a Swiss drugmaker, will pay Zymeworks $55m up front for ZW209, a drug in its first trial in people. [9] ZW209 grabs a cancer cell with one arm and an immune cell with two others, to switch the immune cell on harder. [9] It aims to beat Imdelltra, an Amgen drug approved in the US in 2024 for small-cell lung cancer. [9] Roche will pay $40m more when the second stage of testing starts, and up to $718m in later payments. [9]

Why it matters. The only evidence so far comes from lab tests and from mice given human tumours. [9] Small-cell lung cancer is fast and hard to treat, and Roche already sells Tecentriq for it. [9]

Boehringer Ingelheim, a German drugmaker, showed early results for obrixtamig, a drug that links immune cells to small-cell lung cancer cells. [12] The trial tests it together with topotecan, a chemotherapy that gave 84% of patients serious side effects in an earlier look. [12] Among 60 patients, those who got less than 80% of the planned chemo did at least as well: 72% had their tumours shrink, against 61% of those who got more. [12]

Why it matters. The result eases a worry that patients who cannot take the full chemo dose do worse. [12] Boehringer has two final trials running, and its drug and Zymeworks's both aim to beat Amgen's Imdelltra. [12][9]

GSK, a British drugmaker, showed new early results for mo-rez, an antibody-drug conjugate, at ESMO. [11] In 45 women with ovarian cancer that no longer responds to platinum chemotherapy, 62% had their tumours shrink. [11] The cancer was held back for a median 11.1 months, against the six to eight months GSK says these patients usually get. [11] But 71% had serious side effects, mostly low blood counts. [11]

Why it matters. GSK already runs five final trials of mo-rez, and has changed their rules on preventing low blood counts. [11] It licensed the drug from Hansoh, a Chinese company. [11]

Zentalis, a US biotech, showed results for azenosertib, a pill that stops cancer cells from pausing when their DNA is damaged, so the damage builds up until they die. [10] In 48 women whose ovarian cancer carried a protein called Cyclin E1, 37.2% had their tumours shrink. [10] They lived a median 15.9 months. [10] Single-drug chemotherapy shrinks these tumours in only 4% to 12% of women, its medical chief said. [10]

Why it matters. Side effects were common, and 43.1% of patients needed a lower dose. [10] Two patients died of infections linked to low white blood cells, which led the FDA, the US drug regulator, to pause the drug briefly in 2024. [10] A bigger part of the trial reports in the first half of next year. [10]

Roche gave the numbers behind a final-trial win it announced a month ago for sefaxersen, a monthly injection for IgA nephropathy. [13] That is a kidney disease in which the immune system slowly damages the kidneys' filters. [13] After 37 weeks, protein leaking into urine, a sign of kidney damage, fell by 43.1% compared with a dummy injection. [13] Roche expects yearly sales of one to two billion Swiss francs at its peak. [13]

Why it matters. The trial now continues for two years to measure kidney function itself. [13] Novartis already sells Fabhalta, a twice-daily pill that blocks the same protein, factor B. [13]

China's drug regulator approved batoclimab, a Harbour BioMed injection, for generalised myasthenia gravis. [14] That is a rare disease in which the immune system's antibodies block the signals from nerves to muscles, causing weakness. [14] In the final trial, 25.4% of patients reached minimal or no symptoms at some visit, against 4.7% on a dummy injection. [14] The most common side effect was swelling in the arms or legs, in 38.8% of patients. [14]

Why it matters. Harbour BioMed says it is the first drug of its kind approved by China's regulator after finishing its whole clinical testing. [14] The company says the disease badly affects whether patients can work. [14]

Star Therapeutics, a US biotech, raised $200m from investors on Friday. [15] It earlier built two companies around single drugs. [15] Vega was sold to Incyte in a deal worth up to $2bn, and Electra raised $350m by selling shares to the public in September. [15] Star will now go quiet and work on early drugs for blood and immune diseases. [15]

Why it matters. Investors are paying for the team's record before it has named a drug to show. [15] One investor, Janus Henderson, said it does not often back companies at such an early stage. [15]

A US judge let PhRMA, the drugmakers' trade group, and AbbVie, BMS and Boehringer Ingelheim join a lawsuit over a US government trial scheme. [19] The scheme, due to start on 1 January, lets drugmakers pay rebates later instead of giving discounts up front. [19] It covers medicines that hospitals buy through 340B, a US discount programme. [19] Hospitals sued this month to block the scheme. [19]

Why it matters. The judge had earlier refused to let the drugmakers in, so they can now argue for the scheme before it starts. [19] Which side wins decides when hospitals get the money off. [19]

Newron, a drug company near Milan in Italy, finished signing up patients for its final trial of evenamide in schizophrenia that has not responded to other drugs. [16] Of 996 people screened, 618 qualified and were given one of two doses or a dummy pill, on top of their usual drugs. [16] Evenamide is meant to calm the brain chemical glutamate when too much of it is released. [16]

Why it matters. First results after 12 weeks are due in early 2027, and the trial runs for a year. [16]

Nxera Pharma, a Japanese drug company, has asked South Korea's drug regulator to approve vamorolone for Duchenne muscular dystrophy. [17] That is an inherited disease in which the muscles waste away. [17] Vamorolone is a new kind of steroid drug from Santhera, a Swiss company, which licensed it to Nxera in January. [17] Nxera aims for approval in 2027 and sales in 2028. [17]

Why it matters. The filing uses a fast review route South Korea granted in June for serious and rare diseases. [17]

Summit Therapeutics agreed to use Lunit's AI to study tumour tissue slides from trials of ivonescimab, its cancer drug. [18] Ivonescimab blocks two things at once: a brake on immune cells and a signal that feeds blood vessels to tumours. [18] The AI reads ordinary stained slides to map the immune cells around a tumour. [18]

Why it matters. The companies hope the slides can show which patients are most likely to respond, so trials can pick them. [18]

Researchers in Ireland, London and Barcelona studied data from about 2,000 bowel tumours. [20] They focused on the most common kind, which responds poorly to newer drugs. [20] They built an AI tool to combine genes, proteins and other data on each tumour. [20] It found weak points, and in lab models trametinib, a drug already sold for melanoma, slowed tumours when combined with other drugs. [20] The paper is in the journal Cancer Cell. [20]

Why it matters. The findings are only in lab models so far, and the team says the next step is finding who would respond. [20] Re-using approved drugs could reach patients faster than a new one. [20]

Congo began the first vaccine trial of its current Ebola outbreak on Friday, in Bunia, the main city of the province where the outbreak is centred. [21][22] The first volunteers got Ervebo, a vaccine approved against the Zaire kind of Ebola. [21] This outbreak is caused by the Bundibugyo kind, which has no approved vaccine or treatment. [21][22] The trial, called Tumaini, vaccinates the people most at risk around each patient. [21] It will later add two vaccines made for Bundibugyo, one from the University of Oxford and one from Moderna. [21][22]

Why it matters. Congo has counted more than 9,700 cases and more than 4,600 deaths since May, and the outbreak has reached Uganda, Kenya and Nigeria. [21] The WHO's emergencies director said success would be the first clear proof that any vaccine protects against Bundibugyo. [22]

Kenya's health ministry said on Friday that a 65-year-old woman had died of Ebola, the country's second death. [23][24] She was a close relative of the first patient, a man who arrived from Congo and died on 5 October. [23][24] She fell ill while she was already in quarantine. [23] Seven other patients are being treated, among them three health workers and a hospital cleaner. [23] Kenya's ministry says all the cases are in one group, with no sign of spread in the wider community. [23]

Why it matters. Nigeria's health minister said the virus in Nigeria's first case, found this week, looks closely related to the one in Kenya's first patient. [21][24] The US has warned its citizens against travelling to Kenya. [23]

An insulin used by people with type 1 diabetes is in short supply in Pakistan, a health official told the National Assembly, its parliament, on Friday. [25] Nelson Azeem, a parliamentary secretary for health, said its German maker halted supplies while it upgrades its products. [25] Deliveries fell from 3,500 kits to 1,000 and could fall further during a year-long upgrade. [25] He said other insulins that work the same way are available, and that the shortage reaches Asia, Australia and Europe. [25]

Why it matters. Syeda Amna Batool, a member of the assembly, said the other insulins cost at least 20% more and many patients cannot easily see a doctor to switch. [25] Officials had said on Tuesday that only one brand was missing. [25]