Larimar tumbles as safety concerns resurface in rare disease drug study update
BUZZ-Larimar tumbles as safety concerns resurface in rare disease drug study update Updates
** Larimar Therapeutics' LRMR.O shares fall 15.2% to $3.10
** LRMR has begun rolling submission for accelerated U.S. FDA approval of its drug nomlabofusp, a daily injection for Friedreich's ataxia, a rare and progressive nerve disease
** Company says FDA has signaled that LRMR's existing data is sufficient to support a full review, with a potential mid-2027 launch, if approved
** Company says about 21 of the 43 patients in the ongoing study have dropped out
** William Blair analysts say a key investor concern remains safety, with the discontinuation rate rising to 51.2% from 35.9% in the open-label study
** Larimar reported cases of anaphylaxis in its latest study update, after similar safety concerns flagged last year
** Ten of 43 patients experienced anaphylaxis, a sudden and severe allergic reaction that can cause breathing problems, swelling and a drop in blood pressure
** Leerink Partners analysts said "safety remains the main caveat," with anaphylaxis "remains the key AE to manage" despite progress on the regulatory front
** Long-term study data shows patients on the drug for one year improved protein levels and signs of clinical benefit
** As of last close, stock down ~4% YTD
(Reporting by Kamal Choudhury in Bengaluru)
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Larimar tumbles as safety concerns resurface in rare disease drug study update