Remove Disease Remove Events Remove Small Molecule Remove Treatment
article thumbnail

Early trial data supports neurodegenerative disease-modifying drug

Drug Discovery World

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. All doses were well-tolerated and there were no serious adverse events.

Disease 130
article thumbnail

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.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

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. The efficiency of exon 7 inclusion in the mature SMN2 mRNA varies significantly among patients and poor inclusion efficiency correlates with higher disease severity.

article thumbnail

Reflections from Advanced Therapies Week 2023 

Drug Discovery World

Over the three days of Advanced Therapies Week (ATW) 2023 in Miami, Florida, US, from 18 to 20 January, DDW’s Megan Thomas heard from attendees about what they were most looking forward to learning from the event, as well as why they think the potential of cell and gene therapies outweighs the obstacles that must be overcome to achieve it.

Therapies 246
article thumbnail

Molecular photoswitch improves visual acuity in retinitis pigmentosa

Drug Discovery World

Topline results from a Phase I/II clinical trial have shown that Kiora’s light-restoring small molecule KIO-301 has the potential to meaningfully improve vision in patients with retinitis pigmentosa (RP). KIO-301 was safe and well tolerated with no ocular and non-ocular serious adverse events, nor any signs of retinal inflammation.

article thumbnail

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.

article thumbnail

Antibody Drug Conjugates: windows of opportunity

Drug Target Review

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.