Larimar Therapeutics Reports Positive Topline Phase 1 Clinical Trial Data Showing Dose-Dependent Increases in Frataxin Levels in Patients with Friedreich’s Ataxia

BALA CYNWYD, Pa., May 11, 2021 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for Friedreich’s ataxia (FA) and other complex rare diseases, today announced topline data from its Phase 1 multiple ascending dose (MAD) clinical trial (n=27) evaluating CTI-1601 as a treatment for FA.

FXN Change from Baseline in Buccal Cells Units: pg FXN / μg total protein Data presented as: n median (25th percentile, 75th percentile)
Dose Group	Day 4/7*						Day 13
Placebo (n=7)	-0.03		(-0.18, 0.97			)	0.35		(-0.02, 0.46	)
Cohort 1 Active (25 mg, n=6)	0.39		(0.01, 0.72			)	0.92		(0.48, 0.94	)
Cohort 2 Active (50 mg, n=6)	1.28		(1.09, 1.69			)	0.39		(0.35, 1.08	)
Cohort 3 Active (100 mg, n=7)	3.06		(1.70, 4.16			)	2.64		(1.93, 3.99	)
FXN Change from Baseline in Skin Biopsies# Units: pg FXN / μg total protein Data presented as: median (25th percentile, 75th percentile)
Dose Group	Day 4/7*						Day 13
Placebo (n=7)	N/A						0.92		(0.10, 1.17	)
Cohort 1 Active (25 mg, n=6)	N/A						0.87		(-0.07, 1.69	)
Cohort 2 Active (50 mg, n=6)	N/A						2.82		(1.93, 4.01	)
Cohort 3 Active (100 mg, n=7)	N/A						10.6		(7.20, 18.1	)
FXN Change from Baseline in Platelets Units: pg FXN / μg total protein Data presented as: median (25th percentile, 75th percentile)
Dose Group	Day 4/7*				Day 13
Placebo (n=7)	-0.38	(-1.23, 0.60		)	-0.65			(-1.71, 1.03		)
Cohort 1 Active (25 mg, n=6)	-0.15	(-2.18, 0.60		)	-0.53			(-1.01, 0.80		)
Cohort 2 Active (50 mg, n=6)	0.30	(-0.18, 0.75		)	-0.87			(-1.14, -0.70		)
Cohort 3 Active (100 mg, n=7)	2.54	(2.27, 2.70		)	3.55			(2.71, 4.93		)

Larimar Therapeutics Reports Positive Topline Phase 1 Clinical Trial Data Showing Dose-Dependent Increases in Frataxin Levels in Patients with Friedreich’s Ataxia