Design Therapeutics has reported positive outcomes from the double-masked, randomised Phase I trial of its GeneTAC small molecule, DT-168, in healthy volunteers.
The single and multiple-ascending dose (SAD/MAD) trial assessed the tolerability, safety, and systemic pharmacokinetics (PK) of the eye drop formulation, which is aimed at decreasing the expression of the mutant transcription factor 4 (TCF4) gene, resulting in Fuchs endothelial corneal dystrophy (FECD).
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The randomised, placebo-controlled trial involved 24 healthy participants who received either a placebo or DT-168 in single and multiple ascending doses.
The treatment was administered two times a day for seven days, with a maximum dose of two 0.5% drops.
According to the company, the eye drops were well tolerated by all participants, without any serious or ocular adverse events, and no treatment discontinuations due to these events were reported.
In addition, PK analysis indicated systemic exposure below the quantitation limit across all timepoints and dose groups.
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By GlobalDataConcurrently with this trial, the company carried out reference range trials that showed distinct splicing in the corneal endothelium between unaffected eye donors and samples collected from FECD patients with the mutant TCF4 gene.
Design Therapeutics CEO and chairperson Pratik Shah said: “DT-168 represents a differentiated opportunity for FECD, a disease with millions of patients facing progressive vision loss and no approved disease modifying therapies.
“Leveraging a convenient eye drop formulation, DT-168 is designed to address the underlying genetic cause of FECD by targeting the repeat expansion that drives disease progression.”
Building on these outcomes, Design is planning a Phase II biomarker trial to further investigate the tolerability, corneal endothelium biomarkers and safety in FECD patients.
This upcoming trial will involve FECD patients carrying the TCF4 mutation who are awaiting corneal transplant surgery.
Subjects will be given 0.5% eye drops twice a day for approximately four weeks before surgery. Post-surgery, the corneal tissues will be tested for RNA biomarkers including abnormal gene splicing known as spliceopathy.
This trial is scheduled to commence in the second half of this year, with data expected in 2026.
Design’s GeneTAC molecules are designed to modulate the expression of specific genes to address the root cause of various diseases.
