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BioWorld - Sunday, December 28, 2025
Home » Topics » Clinical, BioWorld

Clinical, BioWorld
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Doctor examining patient's hand

Addex shares fall 48% as recruitment delays scupper pivotal trial in Parkinson’s

June 17, 2022
By Cormac Sheridan
Shares in Addex Therapeutics Ltd. (Zurich:ADXN) dropped 48% June 17 on news that the company had terminated a pivotal phase IIb/III study of dipraglurant as a potential treatment for dyskinesia in Parkinson’s disease.
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Heart and kidneys
Newco news

Vectus shows lead candidate reverses heart, kidney fibrosis

June 16, 2022
By Tamra Sami
From 20 years of research on metabolic change as a result of salt intake, Karen Duggan discovered in 2003 that a naturally occurring molecule in the human body, native vasoactive intestinal peptide (VIP), was capable of reversing fibrosis caused by hypertension and other chronic diseases such as diabetes. From that discovery, Vectus Biosystems Ltd. was founded, and the company has developed a new class of mimetic drug candidates and a drug library based on VIP.
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Elderly hands holding broken brain structure

Alzheimer’s research moving to combination therapies after another amyloid failure, this time in prevention trial

June 16, 2022
By Richard Staines
Alzheimer’s experts have suggested that future research may involve combination therapies after yet another trial failure involving a drug targeting amyloid – in this case Roche Holding AG’s crenezumab. Originally discovered by Swiss biotech AC Immune SA, of Lausanne, crenezumab failed to slow or prevent cognitive decline in people with a certain mutation that causes early onset in a closely watched trial.
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Despite some non-significant data, Pfizer plunges ahead with Paxlovid NDA

June 15, 2022
By Lee Landenberger
A day after announcing it would pump $120 million into a Michigan manufacturing facility for Paxlovid (nirmatrelvir/ritonavir), Pfizer Inc. produced new data it plans to use in an U.S. NDA submission for treating COVID-19. While some data for the oral antiviral fell short of statistical significance, other companies are not far behind Pfizer in their drive for FDA acceptance using similar approaches.
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GSK research lab

After falling behind in first COVID wave, Sanofi and GSK on verge of breakthrough with booster

June 13, 2022
By Richard Staines
Sanofi SA and GSK plc were beat to market during the first wave of COVID-19 by vaccines from upstarts such as Biontech SE and Moderna Inc. – but the French and U.K. vaccine specialists are gaining traction in the race to develop booster shots against newer variants.
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Acute myeloid leukemia illustration

Daiichi Sankyo’s quizartinib doubles overall survival in patients with newly diagnosed FLT3-ITD-positive AML

June 13, 2022
By Tamra Sami
Daiichi Sankyo Co. Ltd.’s quizartinib met the primary endpoint of overall survival in the pivotal phase III Quantum-First study, which tested the addition of quizartinib to chemotherapy vs. chemotherapy alone for adults with newly diagnosed FLT3-ITD-positive acute myeloid leukemia (AML).
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Day One’s phase II findings with ‘drug to beat’ may inaugurate new era in pediatric brain cancer

June 13, 2022
By Randy Osborne
Day One Biopharmaceuticals Inc.’s phase II data with tovorafenib from the Firefly-1 study in pediatric brain cancer dazzled Wall Street, and shares (NASDAQ:DAWN) closed $14.96, up $8.34, or 126%.
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Sickle cell disease

At EHA 2022, sickle cell disease headway and headwinds

June 13, 2022
By Anette Breindl
At the European Hematology Association’s annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
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Mast cell releasing histamine during allergic response

EHA 2022: Best in class? Cogent soars on early data in mastocytosis

June 10, 2022
By Jennifer Boggs
Analysts have already started tagging Cogent Biosciences Inc.’s bezuclastinib as potentially best in class, after the company presented impressive, though early stage, data at the European Hematology Association Congress in Vienna demonstrating promising efficacy and a possibly differentiating safety profile for the selective KIT D816V inhibitor in advanced systemic mastocytosis.
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Trevi heavy as Algernon bears down in IPF cough space

June 10, 2022
By Randy Osborne
A paper published March 22 in the journal Chest highlighted research that found patients with interstitial lung disease (ILD) who had poor quality of life specifically related to cough were more likely to be hospitalized for respiratory issues, need a lung transplant and die. The findings in 1,447 patients matter because they back previous investigations and provide a way of predicting the advance of ILD, of which idiopathic pulmonary fibrosis (IPF) is a prominent type. A handful of companies are working on drugs to tackle cough in IPF, often an early symptom of the disease.
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