Design Therapeutics (NASDAQ: DSGN) on Wednesday reported preliminary data from a phase 1 trial of DT-216 in patients with Friedreich ataxia (FA).
FA is a genetic, progressive, neurodegenerative movement disorder, where a person experiences symptoms such as difficulty walking, fatigue, mood changes, and slow speech, among others.
The company said that the data showed that DT-216 was generally well tolerated and was able to overcome the problem of frataxin (FXN) that causes FA, with a greater than two-fold increase in FXN mRNA in the group with the highest response.
The primary and secondary objectives of the phase 1 single-escalating dose (SAD) study were to evaluate the safety and tolerability, and pharmacokinetics (PK) of DT-216. In the trial, 39 patients were placed in 6 dose groups from 25 mg to 600 mg and were randomized to receive DT-216 (N = 26) or placebo (N = 13).
Design said 16 patients on DT-216 and eight on placebo reported at least one treatment-emergent adverse event (TEAE).
Most adverse events (AEs) were mild and transient and there were no treatment-related adverse events (SAEs). Three patients had visible thrombosis at the imaging site, two mild and one mild, according to the company.
Design stated that a single dose of DT-216 in all groups 100-mg and above resulted in an increase in FXN mRNA levels at 24 hours after the dose.
Individual patient responses to a single dose of DT-216 ranging from 100-mg to 600-mg resulted in an increase in FXN mRNA over 24 hours ranging from 1.24 to 2.62-fold, according to the company.
The results showed that there was no increase in FXN protein from the primary blood mononuclear cells (PBMCs) from patients who were given a single dose of DT-216 or placebo.
The company added that the data from this study will support the advancement of DT-216 in the ongoing advanced dose (MAD) phase 1 and phase 2 clinical trials in patients with FA, which will start in 2023.
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