Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices by Larimar Therapeutics, Inc. following a significant stock price drop after the release of clinical trial data [1] Group 1: Company Overview - Larimar Therapeutics, Inc. is publicly traded on NASDAQ under the ticker LRMR [1] - The company is currently conducting a long-term open-label study on Nomlabofusp for treating Friedreich's Ataxia [1] Group 2: Recent Developments - On September 29, 2025, Larimar announced "positive" data from its ongoing study, but also reported seven cases of anaphylaxis among participants [1] - Following the announcement, Larimar's stock price decreased by $1.72, representing a 33.66% decline, closing at $3.38 per share [1]

Group 1 - U.S. stock futures are higher, with Dow futures gaining approximately 100 points [1] - Quantum Computing Inc. raised $750 million from institutional investors in a market-priced private placement, leading to a sharp decline in its shares by 11.7% to $21.70 in pre-market trading [1] Group 2 - Rich Sparkle Holdings Ltd experienced a significant drop of 15% to $22.10 in pre-market trading after a 10% decline on Friday [3] - Nordic American Tankers Ltd fell 7.8% to $3.07 in pre-market trading [3] - Smurfit WestRock PLC shares decreased by 5.8% to $39.54 in pre-market trading [3] - Mesoblast Ltd shares declined 5.2% to $17.45 in pre-market trading, despite an 8% gain on Friday following the announcement of Ryoncil receiving a J-Code from Medicare & Medicaid Services [3] - Invesco Mortgage Capital Inc saw a decline of 4.4% to $7.42 in pre-market trading [3] - Inventiva ADR shares fell 4.2% to $6.26 in pre-market trading after the appointment of Andrew Obenshain as CEO [3] - International Paper Co dropped 4.2% to $45.21 in pre-market trading ahead of its third-quarter earnings release on October 30 [3] - Lufax Holding Ltd – ADR fell 3.7% to $3.85 in pre-market trading after an 8% decline on Friday [3] - Larimar Therapeutics Inc shares decreased by 3.3% to $4.71 in pre-market trading after a 14% jump on Friday [3]

Market Overview - U.S. stock futures are higher, with Dow futures gaining approximately 100 points [1] Quantum Computing Inc. - Quantum Computing Inc. (NASDAQ:QUBT) raised $750 million from institutional investors through a market-priced private placement led by top shareholders [1] - Shares of Quantum Computing fell sharply by 11.7% to $21.70 in pre-market trading [1] Other Stocks in Pre-Market Trading - Rich Sparkle Holdings Ltd (NASDAQ:ANPA) dropped 15% to $22.10 after a 10% decline on Friday [3] - Nordic American Tankers Ltd (NYSE:NAT) decreased by 7.8% to $3.07 [3] - Smurfit WestRock PLC (NYSE:SW) fell 5.8% to $39.54 [3] - Mesoblast Ltd (NASDAQ:MESO) declined 5.2% to $17.45, despite an 8% gain on Friday following the announcement of Ryoncil receiving a J-Code from Medicare & Medicaid Services [3] - Invesco Mortgage Capital Inc (NYSE:IVR) decreased by 4.4% to $7.42 [3] - Inventiva ADR (NASDAQ:IVA) fell 4.2% to $6.26 after appointing Andrew Obenshain as CEO [3] - International Paper Co (NYSE:IP) dropped 4.2% to $45.21 ahead of its third-quarter earnings release on October 30 [3] - Lufax Holding Ltd – ADR (NYSE:LU) fell 3.7% to $3.85 after an 8% decline on Friday [3] - Larimar Therapeutics Inc (NASDAQ:LRMR) decreased by 3.3% to $4.71 after a 14% jump on Friday [3]

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]