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

Benzinga · 10/16 18:59

Oppenheimer has initiated coverage on Larimar Therapeutics (NASDAQ: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 (NASDAQ: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 (NASDAQ: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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