On Monday, Larimar Therapeutics Inc (NASDAQ:LRMR) announced that the FDA removed the previous partial clinical hold on -nomlabofusp (CTI-1601) clinical program for Friedreich's Ataxia (FA).
Nomlabofusp is a novel protein replacement therapy designed to address the root cause of FA by delivering frataxin to mitochondria.
The FDA removed the partial clinical hold after reviewing data from the company's recently completed Phase 2 dose exploration study.
The review included data from the 25 mg and 50 mg cohorts in patients who received daily dosing of nomlabofusp for 14 days, followed by every other day dosing until day 28.
In the Phase 2 dose exploration study, nomlabofusp was generally well-tolerated throughout the four-week treatment period.
Nomlabofusp had a predictable pharmacokinetic profile and demonstrated dose-dependent increases in frataxin levels in skin and buccal cells.
All patients with quantifiable levels at baseline and Day 14 in the 50 mg cohort achieved frataxin levels in skin cells over 33% of the average level observed in healthy volunteers at Day 14, and 3 patients achieved levels greater than 50% of the average healthy volunteer level.
The long-term safety and tolerability, pharmacokinetics, and frataxin levels in peripheral tissues following nomlabofusp are currently being evaluated in the ongoing OLE study.
The OLE study will initially evaluate daily subcutaneous injections of 25 mg of nomlabofusp.
Larimar plans to dose escalate to 50 mg in the OLE study following additional characterization of frataxin PD at the 25 mg dose.
William Blair writes that the 50 mg dose can potentially increase FXN levels beyond the 5%-10% increase over baseline KOLs have suggested as a therapeutic threshold.
If necessary, dose escalation above 50 mg would require the submission of additional data for FDA review to support the increased dose.
Interim data from the OLE study is expected in the fourth quarter of 2024.
The analyst sees nomlabofusp as the leading therapy to boost FXN expression, which is differentiated from Biogen Inc's (NASDAQ:BIIB) Skyclarys and could potentially be used adjunctively pending safety data. William Blair reiterates the Outperform rating on Larimar.
Price Action: LRMR shares are up 10.66% at $8.05 at last check Tuesday.
Photo via Shutterstock