Larimar Therapeutics Discloses Lower Dose Data Of Investigational Drug For Rare Neurodegenerative Disorder, Shares Plummet

Larimar Therapeutics Inc LRMR announced preliminary topline data from the 25 mg cohort of its Phase 2 trial of CTI-1601 in participants with Friedreich's ataxia (FA). 

Data from the cohort indicate CTI-1601 was generally well tolerated and showed increases in frataxin (FXN) levels from baseline compared to the placebo in all evaluated tissues (skin and buccal cells) at day 14.

Daily subcutaneous injections of 25 mg CTI-1601 for 14 days increased frataxin levels from baseline compared to placebo in all evaluated tissues (skin and buccal cells).

Median placebo-adjusted increases from baseline of 3.5 pg/µg and 0.9 pg/µg in frataxin levels were observed in skin and buccal cells, respectively, with 14 days of daily dosing of CTI-1601.

Larimar has submitted the data from the trial's 25 mg cohort to FDA and has a meeting scheduled with the Agency for later this quarter to discuss the information needed to gain clearance to initiate a 50 mg cohort in the Phase 2 trial.

Larimar's Phase 2 data and non-interventional study results follow Phase 1 data that showed dose-dependent increases in frataxin levels in peripheral tissue with daily dosing of 50 and 100 mg of CTI-1601 for at least seven days and no detectable increase in FXN levels with daily dosing of 25 mg of CTI-1601 for only four days.

Price Action: LRMR shares are down 29.30% at $3.58 on the last check Monday.