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Friday, Oct 3, 2025
Mugglehead Investment Magazine
Alternative investment news based in Vancouver, B.C.
Larimar shares jump on positive drug data in Friedreich’s Ataxia
Larimar shares jump on positive drug data in Friedreich’s Ataxia
Image via Dall-E.

Medical and Pharmaceutical

Larimar Therapeutics shares jump on positive FDA guidance

The company released new long-term study results for its experimental drug nomlabofusp

Larimar Therapeutics’ (NASDAQ: LRMR) have gained nearly 28 per cent over the past five days, climbing from about USD$3.35 to USD$4.28. The rally follows positive FDA guidance on its Friedreich’s ataxia drug, nomlabofusp, and reflects continued investor enthusiasm despite minor daily pullbacks.

Despite some intraday retracements, the stock’s overall momentum points to sustained bullish sentiment. Announced on Wednesday, the price fluctuations come despite earlier concerns over anaphylaxis cases in the trial, as investors responded positively to the drug’s efficacy signals and the company’s proactive safety measures.

The drug is aimed at treating Friedreich’s ataxia (FA), a rare genetic disease that leads to progressive nerve damage and serious physical decline.

Larimar’s announcement has drawn attention because the results suggest that nomlabofusp may both boost the key protein missing in FA patients and slow the disease’s progression.

In total, 65 patients across four completed studies and one ongoing open-label trial received at least one dose of nomlabofusp. Of these, 39 patients are enrolled in the current long-term study.

Fourteen participants have now taken the drug for at least six months, and eight have continued therapy for more than a year. According to Larimar, this group shows encouraging signs of both biological improvement and functional stability.

FA results from low levels of frataxin (FXN), a protein vital to energy production in cells. Patients develop progressive problems with balance, movement, and coordination. Many lose the ability to walk and face shortened life expectancy. Because FA stems directly from insufficient FXN, scientists believe restoring protein levels could slow or even change the course of disease.

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Larimar has shown consistent improvements

The company reported that all 10 patients who had skin protein data at six months reached FXN levels above 50 per cent of those found in healthy individuals. Importantly, this level matches the average of asymptomatic carriers, who do not suffer from FA. In simple terms, nomlabofusp appears to push FXN production into a range where the disease might not progress.

Larimar has shown consistent improvements across four clinical outcome measures after one year of therapy. These included mFARS, which tracks motor skills, FARS-ADL, which measures daily function, the 9-Hole Peg Test for hand coordination, and the Modified Fatigue Impact Scale.

When compared with patients from a separate natural history study who were not on treatment, the nomlabofusp group showed a meaningful difference. Instead of worsening over a year, patients showed directional improvement.

The company stressed that these results, while early, suggest the potential for clinical benefit. For patients and families facing a relentless disease with no approved treatments targeting its root cause. Further, this represents a hopeful signal.

Safety remains a central focus. Larimar reported that daily injections of nomlabofusp were generally well tolerated over long periods. Most side effects were mild to moderate injection site reactions.

However, the open-label study did report seven cases of anaphylaxis, a serious allergic reaction. Most of these episodes happened during the first day of treatment, and all occurred within the first six weeks. Every patient recovered with standard care, but all seven were withdrawn from the study.

In addition to these cases, three patients stopped due to widespread hives, one due to a seizure, one due to a fainting event, and two for reasons unrelated to treatment.

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FDA reviewed the update and agreed with Larimar’s plan

Larimar’s management emphasized that the risk of anaphylaxis is not unique to nomlabofusp. Many protein-based therapies carry similar risks. Still, the company consulted experts and adjusted the starting dose regimen to improve safety.

The U.S. Food and Drug Administration reviewed the update and agreed with Larimar’s revised plan.

Carole Ben-Maimon, the company’s chief executive officer, described the findings as both encouraging and validating. She noted that raising FXN levels above the carrier threshold could represent a turning point for patients.

“Achieving tissue FXN levels equivalent to more than 50 per cent of those found in healthy volunteers means participants are at levels found in asymptomatic carriers who do not develop the disease,” Ben-Maimon said.

The Friedreich’s Ataxia Research Alliance (FARA), a key patient advocacy group, also voiced optimism.

“Treatment approaches, like nomlabofusp, that target the root cause of FA by FXN supplementation are of great interest to the FA community,” said Jennifer Farmer, CEO of FARA.

The ongoing open-label study is designed to track safety, tolerability, and drug levels in the body. Patients began on 25 milligrams daily and later escalated to 50 milligrams. Since late 2024, all new participants have started directly on the 50 milligram dose. Importantly, about half of enrolled patients were non-ambulatory when they began the study, meaning they already had significant disease progression.

As of August 27, 2025, 25 participants remained on active treatment, with some receiving daily injections for up to 527 days. The average treatment duration was 154 days at the time of data cut-off.

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Larimar plans to file Biologics License Application

Larimar has stayed in close contact with the FDA through the Support for Clinical Trials Advancing Rare Disease Therapeutics, also known as the START pilot program. This initiative aims to help speed development for treatments targeting rare conditions. According to the company, the agency has reviewed the safety findings, FXN data, and functional outcomes and supports the ongoing strategy.

The company plans to file a Biologics License Application seeking accelerated approval in the second quarter of 2026. Accelerated approval allows earlier access for patients when a therapy addresses an unmet medical need and shows strong biological evidence of potential benefit. This comes even before long-term clinical results are fully available.

Friedreich’s ataxia affects roughly one in 50,000 people in the United States and has no approved disease-modifying therapies. Current care focuses on managing symptoms rather than addressing the root cause. Consequently, Larimar’s progress with nomlabofusp represents one of the most closely watched efforts in the FA research community.

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