On Monday, Larimar Therapeutics, Inc. (NASDAQ:LRMR) released initial data from the ongoing long-term OLE study evaluating daily subcutaneous injections of 25 mg of nomlabofusp self-administered or administered by a caregiver in participants with Friedreich’s Ataxia.
Tissue FXN levels showed a mean change from baseline of 1.32 pg/μg in buccal cells and 9.28 pg/μg in skin cells at Day 90.
25 mg of nomlabofusp increased and maintained tissue FXN levels over time, increasing from a mean level of 15% of healthy volunteers at baseline to 30% in buccal cells and from 16% to 72% in skin cells at Day 90.
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Tissue FXN levels appear to reach steady-state levels by Day 30 in buccal cells (inside of the mouth and lips).
William Blair views this biomarker data as stronger than expected for the 25 mg dose, approaching the 50% FXN bar for unaffected healthy persons. The analyst says it continues to bode well for dose escalation to 50 mg once daily, cleared for all new and existing patients in the OLE.
Based on in silico simulations of nomlabofusp treatment, the company has suggested that the 50 mg dose will boost FXN levels to at least 30% of normal levels in ~90% of patients.
The analyst attributes the stock weakness to potential nomlabofusp safety concerns.
Four patients withdrew from the study in total. Two patient discontinuations were related to logistical challenges with protocol compliance, and two patients had serious adverse events, which led to withdrawal, though both SAEs resolved within 24 hours.
William Blair confirmed with management that the NHP toxicology studies did not report any CNS-related adverse events or seizures. The analyst reiterated the Outperform rating.
Price Action: LRMR stock is down 11.8% at $4.262 at last check Tuesday.
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