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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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Early trial data supports neurodegenerative disease-modifying drug

Drug Discovery World

The Phase Ib study examined the safety, tolerability and pharmacokinetics of twice-daily oral doses of ALX-001 in 32 heathy adult participants aged 50-80. All doses were well-tolerated and there were no serious adverse events. The findings were presented at the AD/PD 2024 Conference in Lisbon.

Disease 130
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Looking beyond traditional oncogenic pathways to break cancer resistance

Drug Target Review

At TOLREMO, we have developed a proprietary modular phenotypic screening platform that leverages transcriptional reprogramming events associated with non-genetic resistance to deliver new chemical scaffolds and identify novel resistance regulators. Inhibiting KDM5A, a regulator of cell proliferation, poses another interesting approach.

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Positive Phase I results for first-in-kind small molecule complement inhibitor

Drug Discovery World

ANX1502 is a first-in-kind oral, selective small molecule inhibitor that targets the active form of C1s responsible for propagating classical pathway activation in association with C1q. Across all doses evaluated, ANX1502 was generally well tolerated with mild to moderate treatment-emergent adverse events (TEAEs).

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Game-changing pan-TEAD inhibitor for solid tumours

Drug Target Review

The current version of Chemistry42 uses over 40 generative models, including generative autoencoders and generative adversarial networks as well as both structure-based and ligand-based drug design to generate and optimise de novo small molecules.

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Study offers possible treatment for frontotemporal dementia

Drug Discovery World

VES001 is a first-in-class oral, brain-penetrant, small molecule sortilin inhibitor rationally designed as a potentially disease-modifying treatment for FTD(GRN). High levels of safety and tolerability of VES001 were observed across the doses tested, with no serious or treatment-emergent adverse events reported.

Treatment 147
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Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)

The Pharma Data

LNP023 also demonstrated a favorable safety and tolerability profile with no serious treatment-related infections or thromboembolic events. Following the cut-off date for the presented data, one participant, who had severe lymphopenia at study entry, discontinued treatment due to a serious adverse event (AE) of lymphoproliferative disorder.