Core Points - The conference call is hosted by Larimar Therapeutics, with a focus on their business and clinical developments [1][2] - A slide deck and a news release are available on the company's website for further information [2] - The call includes forward-looking statements regarding the company's future business expectations, plans for nomlabofusp (formerly CTI-1601), and clinical trials [3] Company Developments - Larimar Therapeutics is discussing the development and commercialization of nomlabofusp, highlighting its significance in their business strategy [3] - The company is expected to provide insights into their planned clinical trials and overall business strategies during the call [3] Financial Position - The conference call will address the company's financial position and results of operations, indicating a focus on capital use and future financial expectations [3]

Group 1 - The conference call is hosted by Larimar Therapeutics, with a slide deck available for participants [2] - The call includes forward-looking statements regarding the company's business expectations, plans for nomlabofusp development, and clinical trials [3] - The company emphasizes that the forward-looking statements are based on current beliefs and assumptions, which involve risks and uncertainties [4]

Core Insights - Larimar Therapeutics Inc. is experiencing a decline in stock price following the release of data from its ongoing long-term open-label study of nomlabofusp for Friedreich's ataxia [1][7] Study Overview - The open-label study is assessing the safety, tolerability, pharmacokinetics, FXN levels in skin and buccal cells, exploratory pharmacodynamic markers, and clinical outcomes after long-term subcutaneous administration of nomlabofusp [2] - Initial dosing started at 25 mg daily, with an increase to 50 mg planned for the fourth quarter of 2024, and all new patients have been receiving the 50 mg dose since November 2024 [2] Patient Safety and Adverse Events - A total of 65 patients have received at least one dose of nomlabofusp across four completed studies and the ongoing open-label study [3] - Seven participants experienced anaphylaxis and were withdrawn from the study, with most events occurring on the first day of administration and within the first six weeks [3][4] - The company has modified its dosing regimen to include a 5 mg test dose followed by a 25 mg dose under observation [5] Clinical Outcomes - Trends towards improvement were noted in the modified Friedreich Ataxia Rating Scale (mFARS), FARS-Activities of Daily Living (ADL), 9 Hole Peg Test (9-HPT), and Modified Fatigue Impact Scale (MFIS) after one year of treatment [6] - Participants treated for one year showed a median improvement in mFARS score of 2.25, contrasting with a worsening of 1.00 in the FACOMS reference population [6] Future Plans - The company is on track to submit a marketing application for potential accelerated approval of nomlabofusp in Friedreich's ataxia in the second quarter of 2026 [7]

Summary of Larimar Therapeutics Conference Call (September 29, 2025) Company Overview - **Company**: Larimar Therapeutics (NasdaqGM:LRMR) - **Focus**: Development of nonlobofus, a potential disease-modifying therapy for Friedreich's ataxia (FA), a rare neurodegenerative disease caused by frataxin deficiency [doc id='12'][doc id='14']. Key Industry Insights - **Friedreich's Ataxia**: A progressive disease with severe symptoms including loss of mobility, blindness, and a life expectancy of 30-50 years. Current approved therapies do not address frataxin levels [doc id='13']. - **Unmet Need**: There is a significant demand for therapies that can modify the disease rather than just manage symptoms, as highlighted by patient advocates and key opinion leaders [doc id='14']. Core Findings from Clinical Trials - **Positive Data**: - Nonlobofus showed consistent improvements in clinical outcomes after one year of treatment, including a median improvement of 2.25 points in the modified Friedreich's Ataxia Rating Scale (mFARS) compared to a worsening in a reference population [doc id='9'][doc id='27']. - 100% of participants achieved skin frataxin levels above 50% of those found in healthy volunteers after six months [doc id='9'][doc id='37']. - **Safety Profile**: - Anaphylaxis was reported in seven participants, primarily occurring within the first six weeks of treatment. All participants recovered after standard treatment [doc id='10'][doc id='31']. - The most common adverse events were mild to moderate local injection site reactions [doc id='31']. Regulatory and Development Plans - **BLA Submission**: Larimar is targeting a Biologics License Application (BLA) submission in 2026, seeking accelerated approval based on skin frataxin levels as a surrogate endpoint [doc id='36'][doc id='41']. - **Dosing Regimen Changes**: The company has modified the dosing regimen to include a test dose to mitigate anaphylaxis risk, which has been agreed upon by the FDA [doc id='11'][doc id='63']. Financial Position - **Capital Raise**: As of June 30, 2025, Larimar reported pro forma cash of $203.6 million, providing a runway into Q4 2026 [doc id='41']. Additional Considerations - **Patient Enrollment**: The company is expanding its clinical program to include younger patients and those who have not previously participated in trials [doc id='34']. - **Comparative Safety**: Compared to other enzyme replacement therapies (ERTs), Larimar's safety profile is considered benign, with manageable allergic reactions [doc id='90']. Conclusion - Larimar Therapeutics is positioned to potentially change the treatment landscape for Friedreich's ataxia with nonlobofus, demonstrating promising clinical efficacy and a manageable safety profile. The company is actively working towards regulatory approval and expanding its clinical trials to address the unmet needs of patients with FA [doc id='40'][doc id='41'].

Core Insights - Larimar Therapeutics, Inc. (LRMR) announced positive data from a long-term open-label study evaluating nomlabofusp for Friedreich's ataxia, a rare neurological disease [1] - The study showed that nomlabofusp significantly increases skin frataxin levels, achieving tissue FXN levels comparable to over 50% of those found in healthy volunteers [2] - Consistent improvements were observed across four key clinical outcomes after one year of treatment, indicating potential clinical benefits [3] Study Details - Participants initially received a daily dose of 25 mg of nomlabofusp, which was later increased to 50 mg in Q4 2024 [1] - Anaphylaxis was reported in seven participants, prompting the company to consult experts and modify the starting dose regimen [3] Future Plans - The new dosing regimen is expected to be implemented in the open-label study in Q4 2025 [4] - Process performance qualification for the commercial scale drug substance is planned for Q4 2025, with a BLA submission anticipated in Q2 2026 [4] - Following the announcement, LRMR's stock price decreased by 30.72% to $3.53 on Nasdaq [4]

Larimar Therapeutics Program Update September 2025 1 pro Forward-Looking Statements This presentation contains forward-looking statements that are based on the beliefs and assumptions of Larimar Therapeutics, Inc. ( "Company") and on information currently available to management. All statements contained in this presentation other than statements of historical fact are forward-looking statements, including but not limited to Larimar's ability to develop and commercialize nomlabofusp (CTI-1601) and any other ...

Core Insights - Larimar Therapeutics announced positive results from an ongoing long-term open label study of nomlabofusp for treating Friedreich's ataxia, showing improvements in clinical outcomes and skin frataxin levels [1][2][3] Clinical Study Results - The open label study involved 65 participants, with 39 receiving nomlabofusp, and demonstrated consistent improvements across four key clinical outcomes compared to a reference population [3][12] - After one year of treatment, participants showed a median improvement in the modified Friedreich Ataxia Rating Scale (mFARS) score of -2.25, while the reference population experienced a worsening of 1.00 [12][14] - All participants who received nomlabofusp for at least six months achieved skin frataxin levels similar to asymptomatic carriers, with 10 out of 10 participants exceeding 50% of median levels found in healthy volunteers [3][8] Safety and Tolerability - Nomlabofusp was generally well-tolerated, with most adverse events being mild to moderate local injection site reactions [9][10] - Anaphylaxis was reported in 7 participants, all occurring within the first six weeks of treatment, leading to a modification of the starting dose regimen [3][9] Development Program Updates - Larimar plans to submit a Biologics License Application (BLA) seeking accelerated approval in Q2 2026 [3][20] - The dosing regimen has been modified to include a 5 mg test dose followed by a 25 mg dose, with plans to enroll younger patients in the future [15][20] Company Overview - Larimar Therapeutics is focused on developing treatments for complex rare diseases, with nomlabofusp as its lead candidate for Friedreich's ataxia [17]

Core Insights - Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with its lead compound nomlabofusp targeting Friedreich's ataxia [3]. Group 1: Company Overview - Larimar Therapeutics is listed on Nasdaq under the ticker LRMR and is dedicated to addressing complex rare diseases [3]. - The company is developing nomlabofusp as a potential treatment for Friedreich's ataxia and plans to utilize its intracellular delivery platform for designing other fusion proteins targeting additional rare diseases [3]. Group 2: Upcoming Events - Larimar will host a conference call and webcast on September 29, 2025, at 8:00 am EDT to discuss updates on the nomlabofusp clinical development program, including data from an ongoing long-term open label study [1][2].

Financial Performance - The company has not generated any revenue from product sales to date and does not expect to do so in the foreseeable future[151]. - Net loss for the three months ended June 30, 2025 was $26.2 million, an increase of $4.6 million from a net loss of $21.6 million in the same period of 2024[158]. - Net cash used in operating activities for the six months ended June 30, 2025 was $45.9 million, compared to $24.4 million in the same period of 2024[169]. - The accumulated deficit as of June 30, 2025 was $324.6 million, reflecting ongoing research and development costs[175]. - Other income (expense), net for the six months ended June 30, 2025 was $3.5 million, a decrease from $5.1 million in the same period of 2024[165]. - The company has not yet commercialized any products and does not expect to generate revenue from product sales for several years[174]. Capital Raising and Financial Resources - In February 2024, the company completed an underwritten public offering, issuing 19,736,842 shares at a price of $8.74 per share, resulting in net proceeds of approximately $161.8 million[139]. - The company has received net proceeds of approximately $65.1 million from a public offering completed after June 30, 2025, selling 21,562,500 shares at a price of $3.20 per share[141]. - The company anticipates that current cash resources, combined with net proceeds of $65.1 million from a July 2025 public offering, will fund operations into the fourth quarter of 2026[176]. - As of June 30, 2025, the company had cash, cash equivalents, and marketable securities totaling $138.5 million, excluding restricted cash of $0.6 million[175]. Research and Development - The company initiated human clinical studies of nomlabofusp in 2019, completing two Phase 1 studies and a Phase 2 dose exploration study[133]. - The ongoing open label study is currently enrolling participants, including adolescents and children aged 2-11 years[144]. - The company expects to increase its investment in research and development to advance nomlabofusp through additional clinical trials[143]. - Research and development expenses for the three months ended June 30, 2025 increased by $3.7 million to $23.4 million compared to $19.7 million in the same period of 2024[158]. - Research and development expenses for the six months ended June 30, 2025 increased by $17.3 million to $49.9 million compared to $32.6 million in the same period of 2024[163]. - The company continues to advance the development of nomlabofusp through additional clinical trials, including related manufacturing costs[179]. Regulatory and Clinical Development - The company received Orphan Drug Designation and Fast Track Designation from the FDA, as well as access to the EU's Priority Medicines Program[137]. - The company plans to submit a Biologics License Application (BLA) seeking accelerated approval in the second quarter of 2026[144]. - The company has engaged in multiple discussions with the FDA and EMA regarding the clinical development of nomlabofusp[135][136]. - The company seeks to obtain regulatory approvals for nomlabofusp and other potential product candidates[179]. Strategic Goals and Operations - The company aims to identify and advance the development of additional product candidates into clinical development and explore additional indications for existing candidates[179]. - The company plans to identify, acquire, or in-license other product candidates and technologies[179]. - The company intends to maintain, leverage, and expand its intellectual property portfolio[179]. - The company is focused on expanding its operational, financial, commercial, and management systems, including personnel to support clinical development and future commercialization efforts[179]. Miscellaneous - There are currently no off-balance sheet arrangements as defined under applicable SEC rules[181]. - The company is classified as a "smaller reporting company" and is not required to provide certain market risk disclosures[184].

Exhibit 99.1 study. • Strengthened Balance Sheet With $65.1 Million Public Offering: In July 2025, Larimar announced a public offering of common stock with net proceeds of $65.1 million supported by existing and new leading healthcare investors that extends its projected cash runway into the fourth quarter of 2026. Larimar Therapeutics Reports Second Quarter 2025 Financial Results *Pro forma cash, cash equivalents, and marketable securities of $203.6 million reflects $138.5 million of cash, cash equivalents ...