Events Pharmacokinetics Small Molecule Treatment 
Drug Discovery World
DECEMBER 12, 2023
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.
Drug Discovery World
MARCH 15, 2024
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.
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Drug Discovery World
JANUARY 4, 2024
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).
Drug Target Review
JUNE 12, 2023
With 13 preclinical candidates and three AI-designed drugs currently undergoing clinical trials, Insilico is spearheading a revolution in cancer treatment and beyond. Can you provide a summary of the key findings and implications of the preclinical studies on ISM6331 for the treatment of advanced solid tumours?
The Pharma Data
AUGUST 29, 2020
Phase II results are promising for treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder; 1,2 second Phase II study with LNP023 monotherapy in anti-C5 naïve PNH patients ongoing . All patients required RBC transfusions prior to starting LNP023 treatment. “In Peffault de Latour.
The Pharma Data
DECEMBER 20, 2020
Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. ALS-4 is a novel small molecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus.
The Pharma Data
NOVEMBER 4, 2020
CTP-543: An Investigational Treatment for Moderate to Severe Alopecia Areata. The presentation highlighted that in the open label extension study, treatment with CTP-543 shows continued maintenance of hair regrowth relative to the hair growth shown in the Company’s previously conducted Phase 2 trials in patients with alopecia areata.
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