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Just caught LRMR jumping over 40% today - Larimar Therapeutics got FDA Breakthrough Therapy Designation for their Nomlabofusp candidate. It's designed to treat Friedreich ataxia, this rare genetic disorder that affects muscle coordination and mobility. Pretty niche area, but apparently only around 5,000 people in the US have it, so the upside could be significant if this works out.
What caught my attention is they're planning to file for accelerated approval next month, with data expected in Q2. If it gets approved, Nomlabofusp could be the first real disease-modifying therapy for Friedreich ataxia patients. The FDA apparently confirmed their approach using skin FXN levels as a way to measure if the treatment actually works. They're targeting a US launch in the first half of 2027 if everything goes smoothly.
Stock was trading between $1.61 and $5.37 over the past year, so this is a decent move. The clinical data they showed seems to have convinced regulators they're onto something. Worth keeping an eye on if you're into biotech plays - could be one of those rare disease wins that actually pans out.