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BioWorld - Tuesday, December 30, 2025
Home » Topics » Clinical

Clinical
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Biogen trumpets another trial-failure-that-isn’t-a-failure, this time in ALS

Oct. 18, 2021
By Richard Staines
Biogen Inc.’s phase III trial of tofersen in a form amyotrophic lateral sclerosis (ALS) has missed its main goal, but the company said it is still talking with regulators after seeing positive trends in the data.
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Valneva vaccine vials

Regulatory submissions ahead on solid phase III data for Valneva’s COVID-19 vaccine

Oct. 18, 2021
By Nuala Moran
LONDON – Valneva SA’s COVID-19 vaccine, VLA-2001, prompted a stronger immune response with fewer side effects than Astrazeneca plc’s product, according to headline data from the phase III trial. Shares in Valneva (NASDAQ:VALN) shot up by nearly 40% to close Oct. 18 at $39.21 when the data were announced, partly repairing the damage on Sept. 13 when the U.K. government cancelled a $1.65 billion contract with the French vaccines specialist for 100 million doses.
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In the clinic for Oct. 18, 2021

Oct. 18, 2021
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Akeso, Astrazeneca, Eli Lilly, Medigen, Merck, Protalix, RDIF, TG.
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Astrazeneca finds anti-CTLA4, anti-PD-L1 drug combo extends OS in liver cancer trial

Oct. 15, 2021
By Michael Fitzhugh
In trials name-checking some of the world’s highest peaks, longest rivers and most famous seas, Astrazeneca plc has long sought the best use of its CTLA4 inhibitor tremelimumab. Now, after a disappointing cruise along the first-line bladder cancer trial Danube and travails in non-small-cell lung cancer with the trials Mystic, Neptune and Arctic, a trek to the endpoint of its phase III trial Himalaya has yielded top-line success. A single, high priming dose of tremelimumab added to the company's immune checkpoint inhibitor, Imfinzi (durvalumab), led to a statistically significant overall survival (OS) benefit vs. Nexavar (sorafenib, Bayer AG and Amgen Inc.) in the first-line treatment for patients with unresectable hepatocellular carcinoma.
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DNA data illustration

Fabric Genomics AI quickly identifies 90% of rare disease genomic variants

Oct. 14, 2021
By Annette Boyle
Fabric Genomics Inc.’s Gem artificial intelligence algorithm plus whole genome and whole exome data detected more than 90% of disease-causing variants in infants with rare diseases, a study in Genome Medicine demonstrated. The full process from blood sample to shortlist of causative variants and likely diseases takes just a matter of hours and the time to interpret whole genomes is condensed to about 15 minutes.
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Too early to say ‘WEE1,’ but strides made in validating approach to cancer

Oct. 14, 2021
By Randy Osborne
Recent news that The University of Texas MD Anderson Cancer Center is teaming up with Schrödinger Inc. to work on the latter’s WEE1 inhibitor – along with data disclosed at the 2021 meeting of the European Society for Medical Oncology (ESMO) – shone a light on the gatekeeper checkpoint kinase, which a number of players are busy exploring, though nothing in the class has been approved so far.
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In the clinic for Oct. 14, 2021

Oct. 14, 2021
Clinical updates, including trial initiations, enrollment status and data readouts and publications: 60 Degrees, Alimera, Anavex, Apnimed, Atara, Biogen, BMS, Brainstorm, Crispr, EMD, Eyepoint, Faraday, First Wave, Genentech, Horizon, I-Mab, Immune-Onc, Immunitybio, Neoimmunetech, Neurobo, Oncolytics, Oryzon, Outlook, Resverlogix, Rhizen, Sanofi, Smart Immune, TC, Tizona, Ultimovacs, Union, VBI, Veru, Vistagen.
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Newco news

Khondrion heading for pivotal trial as mitochondrial disease competition mounts

Oct. 13, 2021
By Richard Staines
Mitochondrial disease, where the tiny energy factories in cells aren’t functioning properly, can have devastating and sometimes fatal consequences. As there are no approved treatments, patients rely on strategies such as strict diets and conserving energy to try and control symptoms that can affect organs including the brain, kidneys, muscles, pancreas and heart. Netherlands-based biotech Khondrion BV is one of a group of companies that is making progress against this group of rare inherited conditions that affects around one in every 4,000 people.
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ECTRIMS opens: Biogen therapies did not reduce COVID-19 antibody responses in MS patients

Oct. 13, 2021
By Lee Landenberger
Biogen Inc. opened the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting with data showing those treated with its multiple sclerosis (MS) therapies had an effective antibody response to COVID-19 vaccination.
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In the clinic for Oct. 13, 2021

Oct. 13, 2021
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Advaccine, Aerie, Agenus, Apeiron, Arcturus, Ascletis, Astellas, BMS, Inflarx, Inhibrx, Inovio, Istari, Otonomy, Vtv.
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