Larimar Therapeutics, Inc.
For the period ended Aug 4, 2026. Filed Aug 4, 2026.
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Aug 4, 2026 · Filed Aug 4, 2026
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EX-99.1 · Consistent Long-term Safety Profile
Consistent Long-term Safety Profile.
Longer-term dosing was generally well tolerated with thirteen adults on treatment for one year, seven for 18 months and three for two years.
The most common adverse events remained mild-to-moderate local injection site reactions, which decreased in frequency over time and did not lead to any withdrawals from the study.
Twenty-one participants discontinued since study initiation in January 2024.
Ten participants experienced anaphylaxis and discontinued the study, including nine participants with prior nomlabofusp exposure; all participants who experienced anaphylaxis responded to standard therapy and all returned to their usual state of health with no further sequelae.
Three participants experienced generalized urticaria and discontinued the study, with no new occurrences observed following initiation of antihistamine therapy.
Other discontinuations included three associated with other adverse events and five discontinuations unrelated to treatment, primarily due to logistical factors.
Of the eleven participants who had previously not been exposed to nomlabofusp, one had anaphylaxis.
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Sustained Increases in Skin FXN Levels Comparable to Asymptomatic Carriers. Skin frataxin (FXN) levels increased following nomlabofusp administration, with 82% (9/11) of participants achieving levels above those in asymptomatic carriers by six months, 100% (9/9) reaching this threshold at one year, and 100% (3/3) maintaining it through 18 months.
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