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BioWorld - Wednesday, December 31, 2025
Home » Topics » Clinical

Clinical
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Adamas’ MS therapy hits primary endpoint, and yet it …

Dec. 17, 2019
By Lee Landenberger
While the market gave Adamas Pharmaceuticals Inc. shares a thrashing Tuesday, newly installed CEO Neil McFarlane wouldn’t call it quits on the data generated from the phase III of ADS-5102, the Inroads study for treating multiple sclerosis patients with walking impairment.
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Lungs

What’s afta Trikafta? Proteostasis on the skids as phase II in CF ‘underwhelms’

Dec. 17, 2019
By Randy Osborne
Proteostasis Therapeutics Inc. CEO Meenu Chhabra pointed to a “new reality in cystic fibrosis [CF] research” faced by her firm and others: the challenge of enrolling the best patients.
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"Major depressive disorder" book, medical chart

Gemini Ascendant: Axsome questions, get good phase III answers in MDD

Dec. 16, 2019
By Randy Osborne
Fueled by new positive data, Axsome Therapeutics Inc. plans to seek approval during the second half of 2020 for its dextromethorphan/bupropion modulated delivery tablet, AXS-05, an oral NMDA receptor antagonist to treat major depressive disorder.
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insightec-exablate-neuro-12-13.png

Insightec low-intensity focused ultrasound opens blood-brain barrier in Alzheimer’s patients

Dec. 13, 2019
By Stacy Lawrence
There is no FDA-approved medication for Alzheimer’s disease. But there is some hope that if the blood-brain barrier could be more easily penetrated by drug candidates they would prove more effective. That is the line of research being pursued by Israeli company Insightec Ltd. via its Exablate Neuro that provides low-intensity focused ultrasound treatment.
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Transgene shows TG-4010 the guillotine after NSCLC trial miss

Dec. 13, 2019
By Michael Fitzhugh
Transgene SA is quitting development of one of its lead therapeutic vaccines after a combination of the candidate, TG-4010, with chemotherapy and Opdivo missed its primary endpoint in a phase II trial evaluating the combination as a first-line treatment for certain advanced non-squamous non-small-cell lung cancers.
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Cautious optimism for Biolinerx’s CXCR4 inhibitor in pancreatic cancer

Dec. 13, 2019
By David Ho
HONG KONG – Israel-based Biolinerx Ltd. has unveiled the latest data from its ongoing phase IIa COMBAT/KEYNOTE-202 study at the European Society of Medical Oncology Immuno-Oncology Congress 2019 which evaluates Biolinerx’s BL-8040, an inhibitor of the chemokine receptor CXCR4.
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Abdominal snowman melts for Iterum but proposition solid with sulopenem in UTI

Dec. 11, 2019
By Randy Osborne
Shares of Iterum Therapeutics plc (NASDAQ:ITRM) slid 36%, or $1.73, to close at $3.12 after the Dublin-based firm disclosed the much-anticipated but less-than-stellar results from the phase III trial called Sulopenem for Resistant Enterobacteriaceae, or SURE 3, testing oral and I.V. versions of the drug in complicated intra-abdominal infections (cIAI).
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ASH 2019

In all Candor, Darzalex has positive data

Dec. 10, 2019
By Lee Landenberger
ORLANDO, Fla. – As the enormous American Society of Hematology annual meeting wound down, the Janssen Pharmaceutical Cos. of Johnson & Johnson released phase III data showing that adding Darzalex to carfilzomib and dexamethasone, compared to carfilzomib and dexamethasone alone, significantly improved progression-free survival in patients with relapsed/refractory multiple myeloma.
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Sickle cell disease
ASH 2019

‘Multiple ways’ of fighting once-incurable sickle cell disease

Dec. 10, 2019
By Anette Breindl
ORLANDO, Fla. – At the 61st ASH annual meeting late-breaking abstracts session, researchers from Boston Children’s Hospital reported that three adult patients who had received an autologous transplant of gene-edited hematopoietic stem cells lacking BCL11A produced high levels of functional hemoglobin and had reduced disease symptoms for at least eight months after transplantation. 
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ASH 2019

Sanofi's sutimlimab delivers benefit for CAD patients in phase III study

Dec. 10, 2019
By Michael Fitzhugh
ORLANDO, Fla. – New research on sutimlimab, an investigational complement pathway inhibitor under development by Sanofi SA's Bioverativ unit, showed substantial benefits for people with the rare autoimmune disorder cold agglutinin disease (CAD).
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