Larimar's Ataxia Demonstrated Shows Better-Than-Expected Frataxin Increase, Analyst Says

Tuesday, 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.

Guggenheim has increased the price target from $12 to $14, with a Buy rating.

The analyst notes that the increase in frataxin was better than expected from the Phase 1 data, with a median placebo-adj increase from baseline of 3.5 pg/µg in the skin and 0.9 pg/µg in buccal cells.

The analyst writes that investors have shown some concern about the difference in the degree of frataxin increase between buccal cells and skin biopsy. The company noted that baseline frataxin levels in the skin are known to be higher than in buccal cells, and turnover is lower than in buccal cells, likely accounting for the lower increase reported in buccal cells. 

Guggenheim notes that management has always emphasized that buccal cells have the inconvenience of high variability and turnover and indicated skin biopsy as more reliable due to the lower cell turnover but practically less amenable method (since it's an invasive procedure).

Price Action: LRMR shares are up 0.32% at $3.66 on the last check Wednesday.