Jonathan Wolleben

Myles Minter

Patrick Dolezal

Samantha Semenkow

Yatin Suneja

- Larimar Therapeutics reported positive long-term open-label study data for nonlobofus in Friedreich's ataxia (FA), demonstrating consistent directional improvements in key clinical outcome measures, including a median **2.25 improvement in mFARS** after one year of dosing compared to a median **1.0 point worsening** in a reference population.  **[1374690_2160836_1]** **[1374690_2160836_5]** **[1374690_2160836_7]** 
- The study also showed that **100% of participants** achieved skin frataxin levels greater than 50% of healthy volunteers by six months.  **[1374690_2160836_1]** **[1374690_2160836_7]** 
- While **anaphylaxis was reported in 7 participants**, mostly on the initial day of administration, all recovered after standard treatment, and Larimar's modified dosing regimen was **agreed upon by the FDA**.  **[1374690_2160836_1]** **[1374690_2160836_2]** **[1374690_2160836_2]** **[1374690_2160836_6]** 
- Larimar Therapeutics is targeting a **BLA submission in 2026** for nonlobofus, seeking accelerated approval based on skin frataxin levels.  **[1374690_2160836_2]** **[1374690_2160836_2]** **[1374690_2160836_7]** **[1374690_2160836_8]** 
- The company has **$203.6 million in pro forma cash** as of June 30, 2025, with a projected cash runway into **Q4 2026**.  **[1374690_2160836_7]**

Larimar Therapeutics Announces Encouraging Nonlobofus Open-Label Study Results and 2026 BLA Target

- Larimar Therapeutics' nomlabofusp open-label study data indicates therapeutic potential for Friedreich's Ataxia (FA), with **100% of participants** achieving skin FXN levels over **50%** of healthy volunteer median levels by 6 months.  **[1374690_2160667:5]** **[1374690_2160667:9]** 
- The study also demonstrated a median mFARS score improvement of **2.25** after one year, compared to a median worsening of **1.00** in the FACOMS natural history reference population.  **[1374690_2160667:5]** **[1374690_2160667:11]** 
- Despite **7 cases of anaphylaxis** among 39 participants in the open-label study, Larimar modified its starting dose regimen with FDA agreement, and nomlabofusp was generally well tolerated with long-term daily dosing.  **[1374690_2160667:5]** **[1374690_2160667:12]** 
- Larimar continues to target a Biologics License Application (BLA) submission for accelerated approval in **Q2 2026**, with a U.S. launch anticipated in **early 2027**.  **[1374690_2160667:5]** **[1374690_2160667:13]**

Larimar Therapeutics Provides Nomlabofusp Program Update and Targets Q2 2026 BLA Submission

- Larimar Therapeutics announced **positive data** from its ongoing long-term open-label study of nomlabofusp for Friedreich's ataxia, showing **consistent directional improvement** across four key clinical outcomes and **increased skin FXN levels** in all participants at 6 months.  **[1374690_0001193125-25-221538_d24800dex991.htm:0]** **[1374690_0001193125-25-221538_d24800dex992.htm:2]** 
- The company reported **7 cases of anaphylaxis** in the open-label study, primarily occurring within the first six weeks of dosing; Larimar has since **modified its starting dose regimen**, which was agreed upon by the FDA.  **[1374690_0001193125-25-221538_d24800dex991.htm:0]** **[1374690_0001193125-25-221538_d24800dex992.htm:2]** **[1374690_0001193125-25-221538_d24800dex992.htm:5]** 
- Larimar is targeting a **Biologics License Application (BLA) submission for accelerated approval in Q2 2026**, with a **U.S. launch targeted for early 2027**.  **[1374690_0001193125-25-221538_d24800dex991.htm:0]** **[1374690_0001193125-25-221538_d24800dex991.htm:4]** **[1374690_0001193125-25-221538_d24800dex992.htm:5]** 
- As of **June 30, 2025**, the company held **$203.6 million in pro forma cash and investments**, projecting a cash runway into **Q4 2026**.  **[1374690_0001193125-25-221538_d24800dex992.htm:2]**

Larimar Therapeutics (LRMR)