Larimar Therapeutics Inc. (NASDAQ: LRMR) is trading lower on Monday after the company released data from the ongoing long-term open-label (OL) study evaluating daily subcutaneous 25 mg and 50 mg injections of nomlabofusp self-administered or administered by a caregiver in participants with Friedreich’s ataxia (FA).
The OL study is evaluating the safety and tolerability, pharmacokinetics (PK), and FXN levels in skin and buccal cells, along with exploratory pharmacodynamic (PD) markers (lipid profiles) and clinical outcomes following long-term subcutaneous administration of nomlabofusp.
Participants were initially administered 25 mg of nomlabofusp daily. The dose was increased to 50 mg in the fourth quarter of 2024, with all newly enrolled patients receiving the 50 mg dose since November 2024.
Also Read: PTC Therapeutics Faces FDA Rejection On Rare Disease Treatment Over Efficacy Concerns
The company stated on Monday that 65 patients have received at least one dose of nomlabofusp in 4 completed studies and the ongoing OL study. Seven OL study participants experienced anaphylaxis and were withdrawn from the study.
Most of the events occurred on the initial day of administration, and all occurred within the first 6 weeks of dosing; all participants returned to their usual state of health after receiving standard treatment.
Larimar consulted its experts and decided to modify its starting dose regimen.
The new dosing regimen will include a 5 mg test dose followed by a 25 mg dose one hour later under observation. Nomlabofusp 25 mg will then be administered once daily for 30 days, after which the dose will be increased to 50 mg once daily.
Larimar plans to enroll patients aged 2-11 years directly into the OL study.
Other discontinuations include three cases of generalized urticaria, one seizure (the same event as reported in December 2024), one vasovagal event, and two non-treatment-related discontinuations.
Trends towards improvement were observed in the modified Friedreich Ataxia Rating Scale (mFARS), FARS-Activities of Daily Living (ADL), 9 Hole Peg Test (9-HPT), and Modified Fatigue Impact Scale (MFIS) at 1 year relative to baseline.
OL study participants treated for 1 year with nomlabofusp daily demonstrated a median improvement in mFARS score of 2.25 relative to a worsening of 1.00 observed in patients in the Friedrich’s Ataxia Clinical Outcomes Measure Study (FACOMS) reference population.
Directional improvements in the other three clinical outcomes (FARS-ADL, 9-HPT, MFIS) were also observed in OL study participants, while worsening in these outcomes was observed in the FACOMS reference population.
The marketing application submission to request potential accelerated approval for nomlabofusp in FA is still on track for the second quarter of 2026.
Price Action: LRMR stock is down 23.75% to $3.89 as of the last check on Monday.
Read Next:
Beyond Meat (BYND) Stock Dives On Debt Restructuring: What Investors Need To Know
Photo: Shutterstock