US approves Voceryq, the first plasmid DNA immune treatment, for a rare airway disease caused by HPV

The stories

13 stories on this beat today.

Every story told the same way: what happened, who is involved, why it matters, how it works, the truth and the good news.

1. 01

   US approves Voceryq, the first plasmid DNA immune treatment, for a rare airway disease caused by HPV

   7 of 26

   patients with three or more earlier operations who then needed none for a year

   27% of that group, in an open trial of 32 adults [1]

   32

   adults in the trial behind the approval

   each had needed at least two operations in the year before [1]

   2

   plasmids in each dose, one for HPV proteins and one for IL-12

   brief electric pulses push them into the cells [1]

   What happened

   • The FDA, the US drug regulator, approved Voceryq on Friday for a rare airway disease. [1]
   • The disease, recurrent respiratory papillomatosis, grows wart-like lumps in the airways. [1]
   • A virus causes the growths: HPV, types 6 and 11. [1]
   • It is the first US approval for an immune treatment built from plasmid DNA. [1]
   • Voceryq carries 2 plasmids, small pieces of DNA first taken from bacteria. [1]
   • One plasmid holds the instructions for proteins from the two HPV types. [1]
   • The other holds IL-12, a molecule that strengthens the immune response. [1]
   • The aim is for the immune system to attack cells the virus has infected. [1]
   • Doctors inject it into muscle with a device called CELLECTRA. [1]
   • Brief electric pulses open tiny pores in the cells, letting the DNA in. [1]
   • In an open trial, where everyone knew what they got, 32 adults took Voceryq. [1]
   • Each had needed at least 2 operations in the year before. [1]
   • Of 26 patients with three or more earlier operations, 7 needed none in the next year. [1]
   • That is 27% of that group. [1]
   • Common side effects were sore injection sites, headache, tiredness and higher blood pressure. [1]
   • The FDA also gave it Breakthrough status and labelled it a drug for a rare disease. [1]

   7 of 26

   needed no operation for a year

   Who is involved

   The drug company Inovio Pharmaceuticals makes Voceryq and won the approval. [1]

   • Inovio Pharmaceuticals the drug company that makes Voceryq; it won the approval and must now run trials to confirm the benefit
   • FDA US drug regulator
   • The FDA the US drug regulator; it gave the accelerated approval on a count of operations
   • Adults with recurrent respiratory papillomatosis people with HPV growths in the airway who often need operation after operation

   How it unfolded

   The growths form most often on the vocal cords, inside the voice box. [1]

   They cause hoarseness and a lasting cough, and in bad cases trouble breathing. [1]

   Surgery can cut the growths out, but the virus stays in the tissue. [1]

   So many patients need operation after operation for years. [1]

   In some cases the disease can turn into cancer over time. [1]

   1. First year operations counted
   2. After that not yet measured

   Why it matters

   Patients may face fewer throat operations, the FDA says. [1]

   How it works

   Regulators call this accelerated approval: a yes given on an early sign of benefit. [1]

   Here the early sign is fewer operations, which the FDA judges likely to mean real benefit. [1]

   The truth

   • What is really going on The FDA approved Inovio's Voceryq because fewer patients needed throat operations in the first year of an open trial of 32 adults, and Inovio must still prove the benefit in more trials. [1]
   • Why it works on people The word first in a regulator's headline makes an approval sound bigger than its evidence: here it describes the kind of medicine, not how well it works.
   • The trial counted operations only in the first year, so longer effects are unknown. [1]
   • Who gains Inovio Pharmaceuticals — It can sell Voceryq now on an accelerated approval, before the confirming trials finish. [1]
   • Who gains Adults with recurrent respiratory papillomatosis — They get a new option beside repeated surgery, built to make the immune system attack infected cells. [1]
   • Nobody knows yet

     How long Voceryq's effect lasts.

     The trial counted operations only in the first year after treatment, and the trials that must confirm the benefit have not reported. [1]

   • Nobody knows yet

     How Voceryq compares with surgery alone.

     The FDA describes the trial as open-label and gives results for treated patients only, with no comparison group named. [1]

   Good news

   • Seven of 26 adults with a rare airway disease, who had each needed three or more operations, needed none for a year after Voceryq, which the US approved on Friday.

   What comes next

   Inovio must now run more trials to confirm the benefit. [1]

1. 02

   Gilead pauses two lymphoma cell-treatment trials

   What happened

   • Gilead Sciences, a big drugmaker, paused two trials of its cell treatment KITE-753. [2]
   • KITE-753 is a CAR-T: a patient's own immune cells, rebuilt to attack cancer. [2]
   • It is meant for diffuse large B-cell lymphoma, a cancer of white blood cells. [2]
   • Gilead told investors on 29 October that it agreed a partial hold with the FDA. [2]
   • The paused trials are PALISADES-1, a mid-stage test, and PALISADES-2, a final-stage one. [2]
   • A small number of patients had grade 3 or 4 ICANS, a serious brain side effect. [2]
   • ICANS can bring fever, confusion and tremors. [2]
   • Gilead's cell-treatment chief, Cindy Perettie, said no patient died. [2]

   Who is involved

   • Gilead Sciences drugmaker
   • Cindy Perettie head of Gilead's cell-treatment arm, Kite

   How it unfolded

   In an early trial last year, KITE-753 was tested with a sister treatment, KITE-363. [2]

   Cancer could no longer be found in 71% of those patients. [2]

   Why it matters

   The pause hits Gilead's cell-treatment arm, whose sales fell 18% to $355 million last quarter. [2]

   How it works

   Drug-safety reviewers pause a trial to check whether a harm comes from the drug or chance. [2]

   Why a vaccine can treat an infection the body already has

   Most vaccines raise antibodies that stop a virus outside cells; once it hides inside them, killer T cells have to find the infected cells.

   The twist

   A vaccine given after infection does not block the virus; it teaches the body to find and kill its own infected cells.

   The picture

   Antibodiesoutside cells

   vsvs

   Killer T cellsinfected cells

   Step by step

   1. 1 A virus like HPV gets inside cells and lives there
   2. 2 Antibodies work in blood and body fluids, so they cannot reach it
   3. 3 Infected cells show small pieces of the virus on their surface
   4. 4 Killer T cells that have learned those pieces destroy the infected cells
   5. 5 A treatment like Voceryq teaches T cells the pieces, with IL-12 to boost them

   The same force, elsewhere today

   Where this chain is also running, in today's other stories.

   • Gilead pauses two lymphoma cell-treatment trials

     KITE-753 skips the teaching step: T cells are taken out and rebuilt to find cancer, and that attack caused serious brain side effects in a few patients.

   • Blocking a protein slows pancreatic tumours in mice

     The tumour survives by wearing out the killer T cells that reach it, and blocking ANO6 left more of them able to kill.

   Where you've seen this

   Shingles shots for older adults

   the chickenpox virus already hides in nerve cells, so the shot boosts the T cells that keep it down

   Cancer vaccines

   they show T cells pieces of a patient's own tumour so the cells can hunt what surgery left

   Rabies shots after a bite

   they work only because the virus travels slowly to the brain, so antibodies can still catch it first

   The catch

   Trained T cells do not always win: in Voceryq's trial, 7 of 26 patients needed no operation for a year, so most still needed one.

   And the whole of it

   Most adults carry viruses that hide in their cells, like the one behind cold sores, and T cells keep them in check without anyone noticing. The patients in today's stories are people for whom that checking stopped working, and doctors are now trying to restart it.

   The truth

   • What is really going on Gilead paused two trials of its own immune-cell treatment after serious brain side effects in a few patients. [2]
   • Gilead has not said how long the review will take. [2]
   • Who pays Patients waiting for Gilead's PALISADES trials — The two trials are on partial hold while Gilead reviews the brain side effects, with no end date given. [2]
   • Nobody knows yet

     What lies behind the brain side effects in KITE-753's later trials.

     Gilead says it saw differences in grade 3 and 4 ICANS between phase 1 and phase 2 and is still investigating. [2]

1. 03

   Kenya to free its last Ebola contacts

   What happened

   • Kenya plans to release 46 people from Ebola quarantine on 3 November. [11]
   • By then they will have finished the 21 days of checks Kenya requires. [11]
   • Kenya says it has found no new case in the last 10 days. [11]
   • Health teams are still following 9 contacts who are now in other countries. [11]
   • Posts online claimed Kenya's health minister, Aden Duale, had announced a lockdown. [12]
   • Fact-checkers found the claim false: Kenya's health ministry has repeatedly ruled out a lockdown. [12]

   9Cases

   ++

   2Deaths

   ++

   46Contacts leaving

   How it unfolded

   Kenya confirmed its first ever Ebola case on 6 October. [12]

   It has had 9 cases and 2 deaths, all linked to that first patient. [11]

   1. 6 Oct first case confirmed
   2. 3 Nov last contacts released

   Why it matters

   The virus reached Kenya from Congo, where confirmed cases have passed 10,000. [12][13]

   How it works

   Epidemiologists call this contact tracing: find everyone exposed, then watch them for symptoms. [11]

   The truth

   • People leaving quarantine are to get an agreed package of support, the official says. [11]
   • Nobody knows yet

     Whether Kenya's Ebola outbreak is over.

     No new case has been found in 10 days, but the last 46 contacts finish monitoring only on 3 November and 9 contacts abroad are still followed. [11]

   Good news

   • Every Kenyan case after the first was already in quarantine when symptoms began. [12]
   • Kenya has found no new Ebola case in 10 days, and every case after its first was already in quarantine when symptoms began.
2. 04

   Central African Republic screens its river border

   What happened

   • The Central African Republic has stepped up Ebola checks on its river border with Congo. [13]
   • At a pier in Bangui, the capital, canoe passengers step off one at a time. [13]
   • They wash their hands, hear about Ebola and have their temperature checked. [13]

   Who is involved

   The two countries share a border over 1,500 km long, largely unwatched. [13]

   Dr Valentin Nebanga, a Central African health official, says the same communities live on both sides. [13]

   • Dr Valentin Nebanga health official, Central African Republic
   • Fishers
   • Canoe ferrymen

   How it unfolded

   Congo's Ebola cases have passed 10,000, and cases have reached Kenya and Nigeria. [13]

   Why it matters

   Fishers and canoe ferrymen on the Oubangui river earn their living on the water. [13]

   Neige, a fishmonger, says river workers "shall be the first to fall victim". [13]

   How it works

   Public health workers call this entry screening: checking for fever where people cross. [13]

   The truth

   • Trade on the river carries on despite the risk, the report says. [13]
3. 05

   AstraZeneca plans $1.5 billion a year in savings

   What happened

   • AstraZeneca, Britain's biggest drugmaker, launched a plan to save $1.5 billion a year. [3]
   • It will put most of the savings into drugs still in development. [3]
   • It plans to back more than 180 projects in development. [3]
   • The plan will cost up to $2 billion in one-off charges. [3]
   • It is due to be largely complete by the end of 2029. [3]

   2025 sales$59bn

   2030 aim$80bn

   How it unfolded

   Sales of Farxiga, its diabetes and heart drug, fell 25% to $1.6 billion. [3]

   The fall came after cheaper copies went on sale in the US. [3]

   More of its big brands lose patent protection by the mid-2030s, STAT reports. [4]

   Why it matters

   AstraZeneca aims for $80 billion in yearly sales by 2030, from almost $59 billion in 2025. [3]

   How it works

   Drugmakers call this a patent cliff: sales drop fast once copies are allowed. [3]

   The truth

   • Staff may pay for the savings: job cuts are possible, though none are announced. [3]
   • Who gains AstraZeneca's research teams — Most of up to $1.5 billion a year in savings moves into more than 180 projects in development. [3]
   • Who pays AstraZeneca staff — The restructuring could bring job cuts, though none have been announced. [3]
   • Nobody knows yet

     Whether AstraZeneca's savings plan means job cuts, and where.

     Euronews notes that cuts could follow, but none have been announced, and the plan runs to the end of 2029. [3]

4. 06

   Diet and exercise put young adults' diabetes into remission

   What happened

   • A trial in England and Canada tested a strict diet plus exercise against type 2 diabetes. [5]
   • In type 2 diabetes blood sugar runs too high; starting young makes it harsher. [5]
   • The 96 volunteers were aged 18 to 45, had obesity, and did not use insulin. [5]
   • Fifty followed the diet and exercise plan for 24 weeks; 46 got usual care. [5]
   • After 24 weeks, 27 of the 50, or 54%, were in remission. [5]
   • On usual care, 2 of 46, about 4%, were. [5]
   • Remission means blood sugar stays below the diabetes line without diabetes drugs. [5]
   • Side effects such as constipation and headaches hit 43 of the 50, mostly early on. [5]

   Diet and exercise54%

   Usual care4%

   How it unfolded

   1. 24 weeks remission measured
   2. Next longer studies

   Why it matters

   Type 2 diabetes is becoming more common among younger adults worldwide, the researchers say. [5]

   How it works

   Doctors call this an open-label trial: the volunteers knew which group they were in. [5]

   The truth

   • Remission may not be a permanent cure, and the trial lasted only 24 weeks. [5]
   • Who pays Volunteers on the strict diabetes diet — 43 of the 50 had at least one side effect, such as constipation, headaches or dizziness. [5]
   • Nobody knows yet

     Whether the diabetes remission lasts outside a trial.

     The trial ran for 24 weeks under close supervision, and its authors say longer studies are needed. [5]

   Good news

   • In a trial in England and Canada, 27 of 50 young adults with type 2 diabetes were in remission after 24 weeks of diet and exercise, against 2 of 46 on usual care.

   What comes next

   The researchers say longer studies must show whether remission lasts. [5]

5. 07

   India shuts a Sun Pharma eye-drop plant

   What happened

   • Indian regulators ordered Sun Pharmaceutical to stop making eye products at its Assam plant. [4]
   • The order also stops the company selling and distributing those eye products. [4]
   • A joint national and state inspection found failures in the plant's quality system. [4]
   • The order came on 20 August; STAT passed on a Reuters report of it on Friday. [4]

   Who is involved

   • India's national drug regulator
   • Assam's drug controller

   How it unfolded

   In July, Sun Pharma had itself recalled about 11 eye-care brands. [4]

   1. 20 Aug shutdown ordered
   2. Friday STAT relays Reuters' report

   Why it matters

   Sun Pharma had expected the site to become one of its largest sterile-medicine hubs. [4]

   How it works

   Drug plants must run a quality system: rules meant to keep each product safe. [4]

   The truth

   • The reports do not say which faults inspectors found or which products were hit. [4]
   • Who pays Sun Pharmaceutical — It cannot make or sell eye products from a plant it expected to be one of its largest sterile-medicine hubs. [4]
   • Nobody knows yet

     What exactly failed at Sun Pharma's Assam plant.

     The reports say only that its quality system failed, not which faults or which products. [4]

6. 08

   Novartis drops a US filing for its heart drug

   What happened

   • Novartis, the Swiss drugmaker, will not seek US approval for its heart drug pelacarsen. [14]
   • In September, pelacarsen failed to protect heart health in a big final-stage trial. [14]
   • It was meant to lower a particle in the blood called Lp(a). [14]
   • It was once seen as a possible multibillion-dollar product. [14]
   • Novartis is not yet sure whether to go on with a second drug aimed at Lp(a). [14]

   Who is involved

   • Novartis Swiss drugmaker

   How it unfolded

   A week after pelacarsen failed, del-desiran, a muscle-disease drug, failed too. [14]

   1. September pelacarsen fails
   2. A week later del-desiran fails

   Why it matters

   Novartis faces patent expiry on big drugs and needs new ones, BioPharma Dive reports. [14]

   The truth

   • Investors paid first: the two failures pushed Novartis shares down nearly 15%. [14]
   • Who pays Novartis shareholders — Two failed final-stage trials in September pushed its shares down nearly 15%. [14]
7. 09

   One gene edit for many diseases, tried in mice

   What happened

   • David Liu, a gene-editing scientist, spoke this week at ESGCT, Europe's gene and cell therapy meeting. [10]
   • His idea is fewer treatments that each work for many genetic diseases. [10]
   • About 30% of genetic diseases have at least one early stop signal in a gene. [10]
   • That stop signal cuts the protein short, so the body lacks a full working copy. [10]
   • Liu's team edited a spare tRNA, a helper molecule, to read past that signal. [10]
   • Humans carry 418 tRNA genes, more than they need. [10]
   • In mice with Hurler syndrome, an inherited illness, it restored about 7% of enzyme activity. [10]
   • Liu suggested that much "can rescue the disease". [10]

   30 of 100

   of genetic diseases carry an early stop signal

   Who is involved

   • David Liu gene-editing scientist

   Why it matters

   One treatment could then serve many rare diseases that share the same stop signal. [10]

   How it works

   Geneticists call this fault a nonsense mutation: a stop signal in the wrong place. [10]

   The truth

   • Liu warned that most genetic diseases are still too rare for companies to pursue. [10]

   What comes next

   The Broad Institute and two partners set up a nonprofit to connect rare-disease patients with treatments. [10]

8. 10

   Blocking a protein slows pancreatic tumours in mice

   What happened

   • Scientists at Fox Chase Cancer Center found that a protein, ANO6, helps pancreatic cancer grow. [9]
   • Pancreatic tumours sit in dense tissue with few working blood vessels. [9]
   • Support cells around the tumour pass cholesterol to cancer cells, which nibble their surface. [9]
   • ANO6 is what makes that transfer possible. [9]
   • The same contact wears out killer T cells, immune cells that destroy cancer. [9]
   • In mice, two cheap drugs that block ANO6's work, niclosamide and clofazimine, cut tumour weight. [9]
   • The team published the work in Nature Cancer, a research journal. [9]
   • This was in mice, and it has not been tested in patients. [9]

   Why it matters

   The finding could explain part of why pancreatic cancer resists immune treatments. [9]

   How it works

   Cancer biologists call the cells around a tumour its microenvironment, and here it feeds the tumour. [9]

   The tumour survives by wearing out the killer T cells that reach it, and blocking ANO6 left more of them able to kill.

   The idea, in story 02: Why a vaccine can treat an infection the body already has →

   The truth

   • Both drugs also hit other targets, so ANO6 may not explain all of their effect. [9]
   • Nobody knows yet

     Whether blocking ANO6 would help people with pancreatic cancer.

     The results are in mice, and both drugs used also act on other targets. [9]

9. 11

   Setara starts with $90 million for nerve-disease pills

   What happened

   • Setara Biosciences launched on Friday with $90 million, starting with a drug for a nerve disease. [6]
   • Its first drug, STAR-0871, targets Charcot-Marie-Tooth disease, which damages nerves in the limbs. [6]
   • The pill blocks TRPV4, a tiny protein tunnel on cells that runs overactive in patients. [6]
   • Setara plans a small early trial in patients by the end of the year. [6]
   • It will also test the pill in overactive bladder. [6]

   Who is involved

   David Goldstein, who led Actio, now runs Setara. [6]

   • David Goldstein chief executive, Setara

   How it unfolded

   Jazz Pharmaceuticals bought Actio in August for $1.3 billion, for its epilepsy drug. [6]

   Why it matters

   Drugmakers have long struggled to change the course of this nerve disease. [6]

   How it works

   Measuring slow nerve damage in a trial is harder than counting seizures, says Goldstein. [6]

   The truth

   • Jazz bought Actio's lead drug and also holds a stake in Setara. [6]
   • Who gains Jazz Pharmaceuticals and Deerfield Management — Having backed Actio, they now also fund Setara, which carries on the drugs Jazz did not buy. [6]
10. 12

    A record of how 100 million immune cells respond

    What happened

    • Parse Biosciences and Nomic Bio will build Cascade, a dataset of 100 million immune cells. [8]
    • Parse, owned by QIAGEN, reads which genes are active in single cells. [8]
    • Nomic measures the proteins that cells release. [8]
    • Cascade will record how cells react to over 150,000 drug and immune-signal conditions. [8]
    • GSK, Pfizer and Sanofi, three big drugmakers, have signed up as partners. [8]
    • Charlie Roco of Parse says biological AI "has hit a data bottleneck". [8]

    Who is involved

    • GSK
    • Pfizer
    • Sanofi

    Why it matters

    Drug companies want such data to build AI models that predict how cells respond. [8]

    How it works

    Biologists call Parse's method single-cell sequencing: reading genes cell by cell, not as a mixture. [8]

    The truth

    • The data is offered to the drug company partners, the release says. [8]
    • Who gains GSK, Pfizer and Sanofi — As partners in Cascade they are offered data on how 100 million immune cells respond to drugs. [8]
11. 13

    Opentrons raises $40 million for lab robots

    What happened

    • Opentrons Labworks, which makes lab robots, raised $40 million from investors led by Avego. [7]
    • It has put more than 11,000 robots in labs in over 80 countries. [7]
    • The money will go into AI systems that plan, run and learn from experiments. [7]
    • Its customers include university groups, testing labs and big drug companies. [7]

    Who is involved

    • Universities
    • Testing labs
    • Drug companies

    How it unfolded

    Earlier this year it began using Nvidia's AI tools to train its robots. [7]

    Why it matters

    Drugmakers and AI companies are moving lab work onto machines, its lead investor says. [7]

    The truth

    • The claim that labs are settling on Opentrons comes from its own chief executive. [7]