Larimar Therapeutics' New Drug Could Transform Treatment For Rare Neurological Disease, Analyst Sees Over 200% Upside For Stock

Oppenheimer has initiated coverage on Larimar Therapeutics LRMR, a clinical-stage biotechnology company focused on developing treatments for rare diseases.

The analysts view the company’s lead program, Nomlabofusp, as a differentiated, disease-modifying therapy that targets the key pathogenic mechanism of frataxin (FXN) deficiency in Friedreich’s ataxia (FA).

Lower FXN levels predict early onset, disease severity, and faster progression.

Friedreich’s ataxia is a rare neurodegenerative disease affecting ~20,000 patients globally.

Also Read: Larimar Therapeutics’ Friedreich’s Ataxia Investigational Drug Differentiated From Biogen’s Marketed Drug.

Oppenheimer expects open-label extension (OLE) interim data in the fourth quarter of 2024.

Larimar intends to expand into the pediatric population and pursue accelerated approval with a potential FDA marketing application submission in the second half of 2025 under the FDA’s START Pilot Program.

Oppenheimer has initiated with an Outperform rating and a price target of $26.

The analysts assume a 60% chance of accelerated approval, modeling the U.S. launch in 2027 and the European launch in 2028 for FA, estimating $1.2 billion in global revenues in 2030.

Following early 2023 approval, Skyclarys, developed by Reata and acquired by Biogen Inc BIIB for $7.3 billion in 2023, has generated ~$278 million in global sales.

Oppenheimer says that Nomlabofusp stands out from its competitors due to its potential to modify the disease.

Although Reata/Biogen’s Skyclarys has received approval for patients aged over 16 with FA and PTC Therapeutics Inc’s PTC vatiquinone is approved for patients over 7, Larimar’s strategy to include pediatric and adolescent FA patients in its Phase 2 OLE trial enables it to address a larger patient demographic and tackle the progressive aspects of the disease.

Price Action: LRMR stock is up 2.56% at $7.61 at last check Wednesday.

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