Biotech, in brief

The FDA, the US drug regulator, gave accelerated approval this week to a head and neck cancer drug, Rybrevant Faspro. [1]

It is for adults whose cancer came back or spread after platinum chemotherapy and immunotherapy. [1]

The FDA says this is the first drug approved in 10 years for patients who already had platinum chemotherapy. [1]

Patients whose cancer has outlasted both chemotherapy and immunotherapy now have one more approved option. [1]

The next test is the studies the FDA ordered, which must show a real gain over time. [1]

Drug regulators call this accelerated approval: an early yes based on a stand-in measure, called a surrogate endpoint. [1]

Here the stand-in is how many tumours shrank and how long that lasted. [1] The FDA says a long-term benefit is not yet confirmed. [1]

A school can move a pupil up on one good test, then check whether next year's results hold.

The FDA's statement names no comparison group inside the trial, which was open-label, so people knew what was given. [1] Its 20% figure describes approved treatments in general. [1]

The FDA has required confirmatory studies, and its statement gives no date for them. [1]

The FDA approved Rybrevant Faspro on 102 patients and tumour shrinkage. [1]

An early approval lets it sell the drug in this cancer before the confirming studies finish. [1]

Whether Rybrevant Faspro helps patients over the long term.

The FDA says that is not yet confirmed, and its statement gives no date for the studies it ordered. [1]

Patients whose head and neck cancer had come back after platinum chemotherapy got their first newly approved drug in 10 years this week.

An infant in southwestern Wisconsin has died after catching measles, state officials said on Thursday. [3]

It is the US state's first measles death in 36 years. [3]

Measles is a viral disease that vaccines can prevent. [3] The coroner listed it as the cause of death. [3]

The state gave no other details, to protect the family's privacy. [3]

Wisconsin has 431 measles cases, and all but 3 were in unvaccinated people. [3] Nine people went to hospital. [3]

"My heart is broken for the family," said Kirsten Johnson, the state's health secretary. [3]

The US stopped local measles spread in 2000 by keeping vaccination rates high. [3]

Epidemiologists, who study how disease spreads, call this herd immunity: measles needs 95% coverage to prevent outbreaks. [3]

US vaccination rates have fallen below that 95% in recent years. [3] Below it, outbreaks are no longer prevented. [3]

A rumour dies when nearly everyone refuses to repeat it, and it spreads once enough people will.

How many Americans have died of measles this year.

The CDC, the US disease agency, has confirmed 4 deaths. Pennsylvania alone reports 5 and is checking a sixth. [3]

Medicare, the US health plan for older people, finalised a rule on Wednesday cutting hospital payments for some drugs. [4]

The cut hits drugs bought through 340B, a US programme of discounts for hospitals serving many vulnerable patients. [4]

Medicare usually pays a drug's average sales price plus 6%. [4] For 340B drugs it will now pay that price minus 28%. [4]

Regulators say that cuts drug spending by about $5.5 billion next year alone. [4]

The rule also sets one price for scans without contrast dye, such as CT, MRI and X-ray. [4]

A hospital unit and a doctor's office will be paid the same for them. [4] The agency says that saves over $9 billion in a decade. [4]

Medicare must keep its budget level, so payments for non-drug services rise by the same amount. [4] The American Hospital Association says the rule still adds stress on hospitals. [4]

Health economists call the gap between what a drug costs and what is paid for it a spread. [4]

A 340B hospital could buy a drug cheaply, then receive more from Medicare than it spent. [4] The new price shrinks that spread. [4]

A shop that buys at a members' discount and resells at full price keeps the difference.

Mehmet Oz, who runs Medicare's agency, calls this "rooting out waste". [4] The American Hospital Association calls it a "continued assault" on the programme. [4]

They sit outside 340B, and the budget rule moves the savings into payments for non-drug services, so they collect the rise without the cut. [4]

They lose part of the margin between what a drug costs them and what Medicare pays. [4]

Whether 340B hospitals come out behind, and what they would cut.

BioPharma Dive reports the drug cut and a matching rise in other payments, but no hospital-by-hospital figures or list of services at risk. [4]

Revolution Medicines sold $198 million of Rasonque, its pancreatic cancer drug, in the third quarter. [5]

The FDA only approved it on 26 August, so those figures cover just five weeks. [5]

Investors had expected $50 million to $100 million. [5]

More than 5,000 US patients received it, from about 2,700 prescribers. [5]

The company's research showed it could almost double survival for patients with spread pancreatic cancer. [5]

Rasonque's list price is $39,800 for 30 days. [5] The company says health plans covering over 90% of patients are paying. [5]

One analyst calls a first rush like this an "initial bolus": many patients arriving at once. [5]

Patients already on an early access programme moved over at launch, and that move is now finished. [5] The analyst says many were sicker and may stop treatment sooner. [5]

A new shop's first day is crowded by everyone who waited, so it says little about next month.

"The best oncology drug launch in history" is one analyst's phrase. [5] Revolution's own sales chief says five weeks is too early to judge. [5]

Plans are paying a list price BioPharma Dive says is far above that of Keytruda, the world's top-selling cancer medicine. [5]

Whether Rasonque's sales keep this pace.

The first five weeks include a one-off move from an early access programme, and analysts' next-quarter forecasts differ, $426 million against $460 million. [5]

Patient deaths and new government rules have slowed China's flood of rare-disease trials to a trickle, STAT reports. [6]

Since May, 164 trials have been approved under those rules, out of just over 1,000 submitted. [6]

Only 1 of the 164 is listed as a gene therapy or gene-editing programme for a rare genetic disease. [6]

Over the last decade, Chinese hospitals became skilled centres for diagnosing and treating rare diseases. [6]

Earlier rules let Chinese companies test drugs in people faster and more cheaply. [6] That drew US companies to China and pushed others to ask the FDA for a similar route. [6]

Families with rare genetic diseases have few gene trials to join, because just 1 such programme is listed so far. [6]

Tighter rules mean far fewer trials get through, and only one approved so far is a gene programme for a rare genetic disease. [6]

How many patients died in the trials that led China to tighten its rules.

The report we could read says only 'patient deaths', and the fuller story is behind a paywall. [6]

Abstracts out on Wednesday show a big blood-disease meeting will feature antibodies binding three targets instead of two. [7]

These "trispecifics" are being tested in multiple myeloma, a blood cancer. [7] Johnson & Johnson and AbbVie, two big drugmakers, are among the leaders. [7]

J&J gave its drug, ramantamig, with 2 other medicines to newly diagnosed patients. [7] All 20 checked after three cycles had no detectable diseased cells. [7]

AbbVie's drug, ABBV-2001, got responses in 75% to 85% of patients whose disease had returned or resisted treatment. [7]

Two Chinese firms report similar early numbers: Innovent 77%, and Shanghai Xianxiang as high as 86%. [7]

Amgen's Blincyto, cleared in 2014, was the first two-target antibody for a cancer. [7]

Drugmakers hope three grips give myeloma patients longer-lasting results, because tumours have tricks for resisting treatment. [7]

Pharmacologists, who study how drugs act, call these multispecific antibodies: one molecule with several grips. [7] Blincyto has 2 targets and ramantamig 3. [7]

Ramantamig grips CD3, a marker on immune cells, plus BCMA and GPRC5D on myeloma cells. [7] So one molecule grips both kinds of cell. [7]

A climber gripping with both hands is harder to shake off than one gripping with a single hand.

These results come from first-stage and mid-stage trials. [7] The phrase "strong early data" for J&J comes from RBC analysts, in a note to their clients. [7]

Johnson & Johnson's three-target antibody drew praise on 20 patients. [7]

Two trials of Arrowhead Pharmaceuticals' drug plozasiran were published on Wednesday in the New England Journal of Medicine. [8]

They tested 757 adults with severe hypertriglyceridemia, which means very high levels of a blood fat called triglycerides. [8]

Patients got the drug or a dummy at the start and at months 3, 6 and 9. [8]

At 12 months, median triglycerides were 79% and 81% below the start in the two drug groups. [8]

Against the dummy groups, the gaps were 45 and 47 percentage points. [8]

Pancreatitis, a sudden swelling of the pancreas, struck 1 per 100 patient-years on the drug and 5 on the dummy. [8]

Very high triglycerides are linked to more pancreatitis, so people with them have the most at stake. [8]

Geneticists call this RNA interference: a short strand of RNA stops a cell using one gene's message. [8]

Plozasiran blocks the message for APOC3. [8] With that message blocked, triglycerides in the blood fall. [8]

Cancelling an order before it reaches the kitchen stops the dish being made.

Arrowhead, which makes the drug, paid for both trials. [8] Medical problems during treatment hit 73% of patients in both groups alike. [8]

In two published trials of 757 adults with very high blood fats, attacks of pancreatitis came at 1 per 100 patient-years on plozasiran, against 5 on a dummy.

A small trial gave 56 people with colon cancer a short drug course before surgery, Nature Medicine reports. [9]

The drugs were botensilimab alone, or botensilimab with balstilimab. [9]

Half the patients had tumours with working DNA repair, a type long thought to resist this kind of drug. [9]

The goal was a major response: at most 10% of the tumour still alive at surgery. [9]

In the resistant type, 0% responded to one drug and 36% to both. [9]

In tumours with faulty DNA repair, 36% responded to one drug and 100% to both. [9]

Side effects linked to the immune system hit 29% on one drug and 54% on both. [9]

Up to 35% of people with colon cancer that has not spread see it return after treatment. [9]

Immunologists call these drugs immune checkpoint inhibitors: they lift brakes that stop immune cells attacking a tumour. [9]

Botensilimab targets CTLA-4 and balstilimab targets PD-1. [9] Adding the second drug took the resistant type from 0% to 36%. [9]

A door with two bolts opens only when both are drawn.

Each group held just 14 people, who were not assigned at random. [9] So that 36% could really be anywhere from about 13% to 65%. [9]

Why one drug is built to grab two or three targets at once

An ordinary antibody grips one target

A tumour can resist a drug aimed at one target

Drug designers give one molecule two or three grips

One grip holds an immune cell, and another holds the cancer cell

Extra grips leave the tumour fewer tricks, if early results last

One molecule can do a job two separate drugs cannot, because it holds an immune cell and a cancer cell at the same time.

Each step adds a grip to the same molecule. Blincyto was the first two-target cancer antibody, and ramantamig is still in mid-stage trials.

Its lead drug is a two-target antibody meant to pull an immune cell to a cancer cell, the same grip-and-hold step.

One molecule switches on two receptors, GLP-1 and glucagon, instead of a patient taking two drugs.

Here two separate drugs each lift one brake, and responses in the resistant tumours went from 0% to 36% when the second was added.

The drug itself stays on one target, and it worked longer in mice when a second drug acted at another site on that enzyme.

Most three-target results so far come from small early trials, and nobody yet knows if they last longer than two-target drugs.

A myeloma patient, a chemist in Shanghai and an investor in London all depend on these early numbers, and none of them can yet see how long the benefit lasts.

In a small colon cancer trial, all 14 patients with faulty-repair tumours who got two immune drugs before surgery had at most 10% of their tumour left alive.

University of Cambridge scientists and their spin-out, DIOSynVax, reported a broad bird flu vaccine in the journal Cell Reports Medicine. [10]

Named DVX-panH5Nx, it fully protected mice from lethal bird flu and gave better survival in ferrets. [10]

Only half the animals survived on the World Health Organization's existing vaccine, the type governments stockpile. [10]

It is an mRNA vaccine, and it has not yet been tested in people. [10]

Governments stockpile vaccines built against single strains that have already appeared. [10] Bird flu keeps changing as it infects birds and mammals. [10]

Countries planning for a pandemic could one day hold one vaccine that covers many strains, if human trials succeed. [10]

Immunologists call the part of a virus the body learns to recognise an antigen. [10]

The team fed an AI tool the gene sequences of logged bird flu viruses. [10] It designed antigens sharing features across the whole group, so one shot trains against many strains. [10]

A football team that practises against every style of play is ready for a rival it has never met.

The comparison and the phrase "game changer" both come from the team behind the vaccine, led by Jonathan Heeney. [10]

The next step is a human trial, and no date is given. [10]

California voters appeared on track on Thursday to pass Proposition 38, a ballot measure for immune research. [11]

It authorises $8.4 billion in bonds, money the state borrows, for immunology and immunotherapy research. [11]

Half, $4.2 billion, would go to one institute that the measure does not name. [11]

Experts call its long list of criteria "oddly specific". [11] Only the California Institute for Immunology & Immunotherapy appears to meet them. [11]

Gary Michelson, a billionaire and retired surgeon, co-founded that institute and put over $40 million into the Yes campaign. [11]

Backers said it would protect California from sudden turns in US science funding. [11] The Trump administration has cut and delayed research grants. [11]

Budget experts call this an earmark: money in a larger fund set aside for one recipient. [11]

The measure sets criteria, not a name. [11] Those criteria appear to point $4.2 billion to Michelson's institute. [11]

A job advert asking for one exact mix of skills can be written with one person in mind.

The institute went from no employees in 2024 to 3 last month, per US tax filings and CalMatters. [11] The No campaign had raised under $9,000 by mid-October. [11]

The measure's criteria appear to fit only that institute, so half the bonds would likely reach it. [11]

Whether Proposition 38 has formally passed.

STAT reported it only as on track, and the institute set to get half the money is still under construction. [11]

Democrats' win puts Bernie Sanders in line to lead the US Senate's health committee, STAT reports. [6]

He is a long-time critic of how the FDA pays for its drug reviews. [6]

The FDA funds those reviews with user fees collected from drug companies. [6]

Experts expect a much tougher line on the drug industry, as voters push for lower prices. [6]

The committee could change how the FDA deals with the drug companies it regulates. [6]

Political scientists call one fear about such fees regulatory capture: a regulator leaning towards those who pay it. [6]

Companies pay fees, the fees pay for reviews, and those reviews decide the same companies' drugs. [6] Sanders' committee could change that chain. [6]

A referee paid by one team's owner has to work harder to look fair.

Doctors and scientists won US races on Tuesday, including Abdul El-Sayed, an epidemiologist, for the Senate in Michigan. [12]

Pediatrician Annie Andrews flipped a Senate seat in South Carolina. [12] The current US Congress holds 20 physicians, which the next could pass. [12]

Amy Acton, Ohio's public health chief in the pandemic, was elected its governor over biotech billionaire Vivek Ramaswamy. [12]

Jasmine Clark, a microbiologist, will be the first Black woman with a science PhD in the US Congress. [12]

Candidates allied to MAHA, the health movement linked to US health secretary Robert Kennedy Jr, did badly. [13]

STAT counts at least 11 who lost, including Senator Roger Marshall of Kansas. [13]

Only 21% of voters said the Trump administration is making the nation healthier, in polling after the vote. [13]

Andrews says removing Kennedy is a top goal of hers. [13] Democrats also have questions about US vaccine policy and measles outbreaks. [13]

A voter poll analysed by KFF, a health research group, found 30% still support MAHA. [13] These counts come from an opinion article by two podcast hosts. [13]

A study in JAMA Network Open looked at US fast-route approvals for children's drugs from 2000 to 2020. [14]

The FDA gave accelerated approval to 26 drugs for 35 uses in children. [14]

The early studies behind them enrolled a median of 13 children. [14] The studies done after approval enrolled a median of 11. [14]

Full approval followed for 22 of the 35 uses, after a median of 58.1 months. [14]

Another 3 uses were withdrawn without ever reaching full approval. [14]

Children with serious diseases can get a drug sooner this way. [14] Its proof in children can stay thin for years. [14]

Pharmacologists call this extrapolation: judging a child's treatment largely from results in adults. [14]

The small check is often pharmacokinetic bridging, which asks whether the drug reaches matching levels in the body. [14] Studies like that need few children. [14]