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Phase Ib/IIa trial in diabetic macular oedema successfully completed  

Drug Discovery World

Exonate, an mRNA therapy company focused on treatments for diabetic complications, has announced that its lead ophthalmology asset, EXN407, has achieved its prespecified endpoints in a Phase Ib/IIa study. During the trial, EXN407 met all safety and pharmacokinetic parameters and displayed encouraging signals of biological activity.

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Monoclonal antibody CB1 inhibitor for obesity enters Phase II trials

Drug Discovery World

Assessing psychiatric effects of CB1 inhibition The clinical trial will enrol 120 patients across four treatment groups. The first-generation CB1 inhibitor, rimonabant, was a small molecule that acted on the CNS. Eighty patients will receive either nimacimab 200mg or nimacimab-matching placebo subcutaneously once-weekly.

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Grant supports discovery of RNA-targeting Henipavirus therapeutics

Drug Discovery World

Under the grant, Evotec aims to discover and develop RNA-targeting small molecules as potential first-in-class therapeutics against Henipaviruses. PRROTECT is designed to accelerate R&D timelines for neutralising antibodies and create a flexible manufacturing network to deliver such therapeutics in the event of a future pandemic.

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A new drug approval for the vanguard of RNA-targeted small molecules

Dark Matter Blog

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. On the strength of those findings, nusinersen was approved by the FDA at the end of 2016 for the treatment of SMA , marketed under the commercial brand Spinraza.

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Early trial data supports neurodegenerative disease-modifying drug

Drug Discovery World

All doses were well-tolerated and there were no serious adverse events. We believe that the results from the Phase Ib MAD study directly support larger scale Phase II clinical development to more fully understand the potential for ALX-001 to become the first-ever disease-modifying small molecule for neurodegenerative diseases.”

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Looking beyond traditional oncogenic pathways to break cancer resistance

Drug Target Review

4 Another related and well described non-oncogene resistance mechanism is the histological transformation of EGFR-mutated non-small cell lung cancer (NSCLC) to small-cell lung cancer upon treatment with an EGFR inhibitor. This orally available small molecule binds to the bromodomain of CBP/p300 in a highly specific manner.

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Phase I clinical trial validates qEEG biomarker in CNS disorders

Drug Discovery World

The positive human qEEG biomarker results with apimostinel are consistent with prior biomarker observations with zelquistinel, Gate’s lead programme, which is currently being developed as a rapid-acting, once weekly, oral treatment for major depressive disorder (MDD). “We