BALA CYNWYD, Pa. - Larimar Therapeutics, Inc. (NASDAQ:LRMR), a biotechnology firm engaged in developing treatments for rare diseases, announced today that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold on its nomlabofusp (CTI-1601) program, a potential treatment for Friedreich’s Ataxia (FA). The FDA's decision followed a review of Phase 2 data, which included a four-week, placebo-controlled dose exploration study.
Nomlabofusp is a protein replacement therapy aimed at the underlying cause of FA by delivering frataxin to mitochondria. The recent study assessed the safety and pharmacokinetics of nomlabofusp, with patients receiving doses of either 25 mg or 50 mg. The medication was reported to be generally well-tolerated, and it showed dose-dependent increases in frataxin levels in skin and buccal cells.
The company's President and CEO, Carole Ben-Maimon, MD, expressed optimism about the FDA's decision, stating that the clearance to dose escalate to 50 mg in the ongoing open label extension (OLE) study is a significant step forward. The OLE study is currently evaluating the long-term safety and frataxin levels following daily administration of nomlabofusp.
Interim data from the OLE study is expected in the fourth quarter of 2024, with a Biologics License Application (BLA) submission targeted for the second half of 2025. Larimar plans to proceed with dose escalation to 50 mg after additional characterization of frataxin pharmacodynamics at the 25 mg dose. Any further dose increase above 50 mg would require the submission of additional data for FDA review.
Nomlabofusp has received Rare Pediatric Disease designation, Fast Track designation, and Orphan Drug designation by the FDA, along with similar designations from European regulatory agencies.
InvestingPro Insights
Larimar Therapeutics (NASDAQ:LRMR), despite the positive news regarding the FDA's decision, presents a mixed financial picture as per the latest metrics from InvestingPro. The company's market capitalization currently stands at $463.84 million, indicating a relatively small-cap status in the biotechnology sector. A critical insight for investors is the company's negative P/E ratio, which is currently -7.55, reflecting that the company is not generating profit relative to its share price. This aligns with the InvestingPro Tips that suggest analysts do not expect the company to be profitable this year, and net income is anticipated to drop.
On the positive side, one of the InvestingPro Tips highlights that Larimar Therapeutics holds more cash than debt on its balance sheet, which could provide some financial stability as the company continues to invest in its clinical programs. Additionally, the company has experienced a significant return over the last year with a 101.64% price total return, although it has seen a price decline of 38.81% over the last three months.
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