Core Viewpoint - Larimar Therapeutics, Inc. is progressing towards the submission of a Biologics License Application (BLA) for nomlabofusp, a potential treatment for Friedreich's Ataxia, with a planned submission in the second quarter of 2026 following FDA recommendations for safety data inclusion [1][4][9] FDA Recommendations - The FDA has provided clear recommendations for the safety database, requiring at least 30 participants with continuous exposure for 6 months, including a subset of at least 10 participants with 1-year exposure, primarily from the 50 mg dose group [4][5] - The FDA is open to the use of skin frataxin (FXN) concentrations as a reasonably likely surrogate endpoint for the BLA submission, acknowledging the relationship between increased skin FXN and relevant tissues [5][9] Clinical Development Progress - Enrollment in the open label extension (OLE) study is ongoing, with plans to include adolescents and patients who have not previously participated in clinical studies [2][4] - Data from the OLE study, expected in September 2025, will include results from 30-40 participants who received at least one dose of nomlabofusp, focusing on the 50 mg dose [4][9] - Adolescent pharmacokinetic (PK) run-in data is also anticipated in September 2025 from 14 participants, some of whom received a placebo [4][9] Global Phase 3 Study - Activities for the global Phase 3 study are ongoing, with site identification and qualification in the U.S., Europe, U.K., Canada, and Australia [4][9] - The Phase 3 study is intended to serve as a confirmatory study to verify clinical benefit as required by the FDA's accelerated approval pathway [9] Company Overview - Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with nomlabofusp as its lead compound targeting Friedreich's Ataxia [7]

Core Viewpoint - Larimar Therapeutics, Inc. is set to host a conference call and webcast on June 23, 2025, to discuss regulatory updates regarding its nomlabofusp clinical development program for Friedreich's Ataxia [1] Company Overview - Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases [3] - The company's lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's Ataxia [3] - Larimar plans to utilize its intracellular delivery platform to design additional fusion proteins targeting other rare diseases characterized by deficiencies in intracellular bioactive compounds [3] Conference Call and Webcast Details - The conference call and webcast will take place on June 23, 2025, at 8:00 am EDT [2] - Participants can access the event via a provided link or by phone using specific domestic and international numbers [2] - An archived version of the webcast will be available on the Larimar website following the live event [2]

[PART I - FINANCIAL INFORMATION](index=6&type=section&id=PART%20I%20-%20FINANCIAL%20INFORMATION) [Financial Statements](index=6&type=section&id=Item%201.%20Financial%20Statements) This section presents the unaudited condensed consolidated financial statements for Larimar Therapeutics as of March 31, 2025 [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The balance sheet as of March 31, 2025, shows a decrease in total assets to $170.2 million and in total stockholders' equity to $144.3 million Condensed Consolidated Balance Sheets (in thousands) | | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Total current assets** | $165,485 | $195,304 | | **Total assets** | $170,175 | $200,225 | | **Total current liabilities** | $22,134 | $24,356 | | **Total liabilities** | $25,904 | $28,413 | | **Total stockholders' equity** | $144,271 | $171,812 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For Q1 2025, the company reported a net loss of $29.3 million, a significant increase from the $14.7 million loss in Q1 2024 Q1 2025 vs Q1 2024 Statement of Operations (in thousands, except per share data) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Research and development | $26,552 | $12,939 | | General and administrative | $4,636 | $3,795 | | **Total operating expenses** | **$31,188** | **$16,734** | | **Loss from operations** | **($31,188)** | **($16,734)** | | Other income, net | $1,907 | $2,080 | | **Net loss** | **($29,281)** | **($14,654)** | | **Net loss per share, basic and diluted** | **($0.46)** | **($0.27)** | [Condensed Consolidated Statements of Changes in Stockholders' Equity](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Changes%20in%20Stockholders%27%20Equity) Stockholders' equity decreased from $171.8 million to $144.3 million in Q1 2025, primarily due to the quarterly net loss - Total stockholders' equity decreased by **$27.5 million** during Q1 2025, from $171.8 million to $144.3 million, mainly due to the net loss[20](index=20&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities increased to $26.5 million in Q1 2025, reflecting higher R&D spending Q1 2025 vs Q1 2024 Cash Flows (in thousands) | | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($26,540) | ($10,411) | | Net cash provided by (used in) investing activities | $14,448 | ($68,364) | | Net cash provided by financing activities | $— | $162,151 | [Notes to Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes detail the development of nomlabofusp for Friedreich's ataxia, positive regulatory interactions, and the company's liquidity position - The company's lead product candidate, nomlabofusp, is a recombinant fusion protein intended to deliver human frataxin (FXN) to the mitochondria of patients with Friedreich's ataxia (FA)[26](index=26&type=chunk) - The FDA selected nomlabofusp for its START Pilot Program and is open to considering FXN concentration as a reasonably likely surrogate endpoint (RLSE), with a **BLA submission targeted for the end of 2025**[33](index=33&type=chunk)[39](index=39&type=chunk) - As of March 31, 2025, the company had **$157.5 million** in cash, cash equivalents, and marketable securities, sufficient to fund operations into the **second quarter of 2026**[48](index=48&type=chunk)[50](index=50&type=chunk) - In February 2024, the company completed an underwritten public offering, raising net proceeds of approximately **$161.8 million**[91](index=91&type=chunk)[92](index=92&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A)](index=28&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the clinical progress of nomlabofusp, the significant increase in R&D expenses, and the company's liquidity and capital resources [Overview and Program Update](index=28&type=section&id=Overview) Larimar is a clinical-stage biotech company advancing nomlabofusp for Friedreich's ataxia with positive trial data and favorable regulatory feedback - The FDA is open to considering **FXN concentration as a reasonably likely surrogate endpoint (RLSE)** to support a future marketing application for nomlabofusp[149](index=149&type=chunk) - A global **Phase 3 study is on track to be initiated by mid-2025**[150](index=150&type=chunk) - An update on OLE trial data, including participants dosed for over a year, is planned for **September 2025**[145](index=145&type=chunk) - In March 2025, the OLE protocol was amended to include premedication for the first month of dosing to reduce the risk of allergic reactions, including anaphylaxis[145](index=145&type=chunk) [Results of Operations](index=33&type=section&id=Results%20of%20Operations) The net loss for Q1 2025 increased to $29.3 million, driven by a $13.6 million rise in R&D expenses for the nomlabofusp program Comparison of Operations for Q1 2025 and Q1 2024 (in thousands) | | 2025 | 2024 | Increase (Decrease) | | :--- | :--- | :--- | :--- | | Research and development | $26,552 | $12,939 | $13,613 | | General and administrative | $4,636 | $3,795 | $841 | | **Total operating expenses** | **$31,188** | **$16,734** | **$14,454** | | **Net loss** | **($29,281)** | **($14,654)** | **($14,627)** | - The **$13.6 million increase in R&D expenses** was primarily driven by a $7.1 million increase in nomlabofusp manufacturing costs and a $2.8 million increase in clinical costs[171](index=171&type=chunk) [Liquidity and Capital Resources](index=33&type=section&id=Liquidity%20and%20Capital%20Resources) The company held $157.5 million in cash and equivalents as of March 31, 2025, which is expected to fund operations into Q2 2026 - As of March 31, 2025, the company had an accumulated deficit of **$298.4 million** and cash, cash equivalents, and marketable securities of **$157.5 million**[182](index=182&type=chunk) - Current cash, cash equivalents, and marketable securities are anticipated to fund operations into the **second quarter of 2026**[184](index=184&type=chunk) - Net cash used in operating activities increased to **$26.5 million** in Q1 2025 from $10.4 million in Q1 2024, reflecting increased R&D activity[175](index=175&type=chunk)[176](index=176&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=36&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a "smaller reporting company," the company is not required to provide quantitative and qualitative disclosures about market risk - As a **"smaller reporting company,"** Larimar is not required to provide information regarding market risk[190](index=190&type=chunk) [Controls and Procedures](index=36&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of March 31, 2025 - The Chief Executive Officer and Chief Financial Officer concluded that the company's disclosure controls and procedures are **effective** as of March 31, 2025[193](index=193&type=chunk) - **No material changes** in internal control over financial reporting occurred during the quarter[195](index=195&type=chunk) [PART II - OTHER INFORMATION](index=37&type=section&id=PART%20II%20-%20OTHER%20INFORMATION) [Legal Proceedings](index=37&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any legal proceedings that would have a material adverse effect on its business - As of the report date, there are **no threatened or pending legal actions** that could reasonably be expected to have a material adverse effect on the company[196](index=196&type=chunk) [Risk Factors](index=37&type=section&id=Item%201A.%20Risk%20Factors) This section directs investors to the risk factors discussion in the company's Annual Report on Form 10-K for the year ended December 31, 2024 - Investors are advised to carefully consider the risk factors described in the company's **2024 Annual Report on Form 10-K**[197](index=197&type=chunk) [Other Required Disclosures (Items 2-6)](index=37&type=section&id=Other%20Required%20Disclosures) The company reports no unregistered sales of equity, no defaults on senior securities, and no new officer trading plans during the quarter - There were **no unregistered sales of equity securities** or use of proceeds during the quarter[198](index=198&type=chunk) - **No defaults upon senior securities** were reported[199](index=199&type=chunk) - No directors or officers adopted, modified, or terminated a **Rule 10b5-1 trading arrangement** during the quarter ended March 31, 2025[201](index=201&type=chunk)

Financial Performance - The company reported a net loss of $29.3 million, or $0.46 per share, for Q1 2025, compared to a net loss of $14.7 million, or $0.27 per share, for Q1 2024, indicating a 99% increase in net loss year-over-year[8] - Total operating expenses rose to $31,188,000 in Q1 2025, up from $16,734,000 in Q1 2024, marking an increase of 86%[20] - Net loss for Q1 2025 was $29,281,000, compared to a net loss of $14,654,000 in Q1 2024, reflecting a 99% increase in losses[20] - Net loss per share, basic and diluted, was $0.46 in Q1 2025, compared to $0.27 in Q1 2024, indicating a 70% increase in loss per share[20] - Total comprehensive loss for Q1 2025 was $29,375,000, compared to $14,760,000 in Q1 2024, representing a 99% increase[20] - Other income, net, decreased to $1,907,000 in Q1 2025 from $2,080,000 in Q1 2024, a decline of 8%[20] - Unrealized loss on marketable securities was $94,000 in Q1 2025, slightly improved from a loss of $106,000 in Q1 2024[20] Expenses - Research and development expenses for Q1 2025 were $26.6 million, up from $12.9 million in Q1 2024, primarily due to a $7.1 million increase in manufacturing costs for nomlabofusp[9] - General and administrative expenses increased to $4.6 million in Q1 2025 from $3.8 million in Q1 2024, driven by a $0.7 million rise in personnel expenses[10] - Research and development expenses increased to $26,552,000 in Q1 2025 from $12,939,000 in Q1 2024, representing a 105% increase[20] Cash Position - As of March 31, 2025, Larimar Therapeutics reported cash, cash equivalents, and marketable securities totaling $157.5 million, with a projected cash runway into the second quarter of 2026[5][8] - Total liabilities as of March 31, 2025, were $25.9 million, a decrease from $28.4 million as of December 31, 2024[17] Development Plans - Larimar plans to submit a Biologics License Application (BLA) by the end of 2025, seeking accelerated approval for nomlabofusp, with a global Phase 3 study expected to initiate in mid-2025[3][5] - The FDA has indicated openness to considering skin FXN concentration as a surrogate endpoint for accelerated approval, which could support the BLA submission[5][6] - Data from the ongoing open-label extension study, including results from the 50 mg dose cohort, is expected to be reported in September 2025[12] - The company completed dosing in the adolescent pharmacokinetic run-in study, with data from this cohort anticipated in the upcoming program update[6][12] - Larimar plans to transition to a lyophilized formulation of nomlabofusp in mid-2025, which is intended for commercialization[12] Shareholder Information - Weighted average common shares outstanding increased to 63,964,008 in Q1 2025 from 53,553,707 in Q1 2024, a rise of 19%[20]

Core Viewpoint - Larimar Therapeutics is advancing its clinical development program for nomlabofusp, aiming for a Biologics License Application (BLA) submission by the end of 2025, with a focus on potential accelerated approval for treating Friedreich's ataxia (FA) [2][4][12] Company Updates - The company reported a net loss of $29.3 million for Q1 2025, compared to a net loss of $14.7 million in Q1 2024, reflecting increased research and development expenses [6][18] - As of March 31, 2025, Larimar had a strong balance sheet with $157.5 million in cash, cash equivalents, and marketable securities, providing a cash runway into Q2 2026 [4][6] - The FDA has indicated openness to considering skin FXN concentration as a surrogate endpoint for accelerated approval, which could support the BLA submission [4][5] Clinical Development - Larimar plans to initiate a global Phase 3 study in mid-2025, following regulatory feedback on the study protocol [2][12] - The company has completed dosing in an adolescent pharmacokinetic (PK) run-in study and expects to present data from this cohort in September 2025 [5][12] - The ongoing open-label extension (OLE) study is expected to provide topline data on the 50 mg dose in September 2025 [5][12] Financial Performance - Research and development expenses for Q1 2025 were $26.6 million, significantly higher than $12.9 million in Q1 2024, driven by increased manufacturing and clinical costs [7][18] - General and administrative expenses rose to $4.6 million in Q1 2025 from $3.8 million in Q1 2024, primarily due to higher personnel and consulting fees [8][18]

Financial Performance - For the years ended December 31, 2024 and 2023, the company reported net losses of $80.6 million and $36.9 million, respectively, with an accumulated deficit of $269.2 million as of December 31, 2024[194]. - The company has not generated any commercial revenue to date and may never become profitable, relying on regulatory approval and successful commercialization of its lead drug candidate, nomlabofusp[198]. - The company has incurred significant losses since its inception in 2016 and expects to continue incurring net operating losses for the foreseeable future[194]. - The company has net operating loss carryforwards of $198.2 million as of December 31, 2024, which may be limited in their use due to ownership changes[209]. - The company may need to raise additional funding to complete the development and commercialization of nomlabofusp, which could lead to dilution of existing stockholders[207]. Development and Clinical Trials - The company is currently developing nomlabofusp, having completed two Phase 1 studies and a Phase 2 dose exploration study, with ongoing trials in patients with Friedreich's Ataxia (FA)[213]. - The company anticipates significant increases in expenses related to clinical trials and research and development for nomlabofusp and any future product candidates[203]. - The ongoing Phase 2 open-label extension trial and run-in study in adolescent patients with FA may face delays or termination due to safety issues or negative interim results[222]. - The clinical development process for nomlabofusp is lengthy and expensive, with a high historical failure rate for product candidates in the industry[217]. - The company has completed two Phase 1 clinical trials and a four-week, placebo-controlled Phase 2 dose exploration study for its lead product candidate, nomlabofusp[222]. Regulatory Challenges - Regulatory approval for nomlabofusp may be delayed or limited, and there is no assurance of successful market acceptance or profitability[216]. - The FDA and other regulatory authorities may require additional data or impose post-marketing commitments, affecting the commercialization timeline[233]. - The FDA has acknowledged the unmet need for treatments for FA and is open to considering FXN levels as a surrogate endpoint for accelerated approval, with a target for BLA submission by the end of 2025[239]. - The FDA's accelerated approval program allows for the approval of drugs for serious conditions based on surrogate endpoints, but there is no guarantee that the data generated will support clinical benefit predictions[256]. - The company has not yet obtained regulatory approval for nomlabofusp, which may require substantial further clinical development before submission to the FDA[281]. Market and Competition - The company currently has no marketed proprietary products and has not advanced any product candidate beyond Phase 2 clinical trials, impacting its ability to commercialize products independently[226]. - The company may face competition from omaveloxolone, which was approved for the treatment of FA in February 2023 and February 2024, necessitating compelling advantages in efficacy and safety for nomlabofusp[306]. - The company anticipates increasing competition from biosimilars in both the United States and Europe, which could exert downward pressure on product prices and sales[309]. - The commercial success of nomlabofusp will depend on market acceptance among the medical community, which is influenced by factors such as adverse side effects and availability of alternative treatments[301]. - The prevalence of Friedreich's Ataxia (FA) is estimated to be approximately three times greater in the European Union than in the United States, representing a significant potential market for nomlabofusp[297]. Manufacturing and Supply Chain - The manufacturing of nomlabofusp is complex and may face challenges that could delay clinical trials and commercialization[249]. - The company has not yet manufactured its product candidates on a commercial scale, which could hinder its ability to launch products successfully[368]. - The manufacturing process for nomlabofusp is currently dependent on third-party manufacturers, which poses risks related to quality and supply continuity[369]. - Any interruption in supply from approved manufacturers could significantly disrupt commercial supply and delay product development[376]. - Changes in manufacturing methods during product development may lead to additional costs and delays, affecting clinical trial outcomes[378]. Cybersecurity and Data Privacy - The company has implemented comprehensive security measures to protect its internal computer systems and those of third-party service providers, but vulnerabilities remain due to the complexity and size of these systems[340]. - Cyberattacks and data breaches pose significant risks, potentially leading to material disruptions in product development and operations, with substantial recovery costs[341]. - Compliance with data privacy regulations such as GDPR and HIPAA is essential, with non-compliance potentially resulting in substantial fines and operational challenges[356]. - The rise of shadow IT practices among employees increases security risks, as unauthorized software may not adhere to established cybersecurity standards[358]. - The complexity of maintaining compliance across multiple jurisdictions increases operational burdens, requiring continuous updates to security policies and IT infrastructure[356]. Strategic Partnerships and Collaborations - The company may pursue acquisitions or strategic alliances, but successful integration is crucial for realizing benefits[317]. - The company is dependent on collaborative arrangements and strategic alliances, which may expose it to risks such as lack of control over resource allocation and potential financial difficulties of collaborators[381]. - The company may need to rely on third-party collaborations for commercialization in foreign markets, which introduces additional risks and uncertainties[298]. - Competition for collaborators is significant, and the success of collaborations depends on various factors including clinical trial results[320]. - The company relies on consultants and advisors for development and commercialization strategy, which may limit their availability due to other commitments[314]. Intellectual Property - The company’s commercial success relies on obtaining and maintaining issued patents and protecting proprietary technology; failure to do so could harm its competitive advantage and profitability[384]. - The company faces uncertainty regarding the issuance and enforceability of its patent applications, which could impact its ability to protect its products from competitors[388]. - The company’s ability to enforce patent rights is challenged by the difficulty in detecting infringement and the potential high costs of litigation[390]. - The company relies on unpatented trade secrets and technological innovation, which may be compromised if confidentiality agreements are breached[392]. - The company may not be able to successfully commercialize nomlabofusp before relevant patents expire, affecting its market position[392].

Financial Performance - The company reported a net loss of $28.8 million, or $0.45 per share, for Q4 2024, compared to a net loss of $13.0 million, or $0.30 per share, for Q4 2023[7]. - For the full year 2024, the company reported a net loss of $80.6 million, or $1.32 per share, compared to a net loss of $36.9 million, or $0.84 per share, for 2023[10]. - Net loss for the year was $80,604,000 in 2024 compared to $36,949,000 in 2023, reflecting a 118.5% increase in losses[21]. - The company reported a net loss per share of $1.32 in 2024, compared to $0.84 in 2023, indicating a worsening loss per share by 57.1%[21]. Research and Development - Research and development expenses for Q4 2024 were $26.7 million, a significant increase from $10.6 million in Q4 2023, primarily due to a $15.0 million rise in nomlabofusp manufacturing costs[8]. - Research and development expenses for the full year 2024 were $73.3 million, up from $27.7 million in 2023, driven by a $36.1 million increase in nomlabofusp manufacturing costs[11]. - Research and development expenses rose significantly to $73,278,000 in 2024 from $27,670,000 in 2023, an increase of 164.5%[21]. Cash and Assets - As of December 31, 2024, the company had cash, cash equivalents, and marketable securities totaling $183.5 million, with a projected cash runway into the second quarter of 2026[5]. - Total assets increased to $200,225,000 in 2024 from $95,935,000 in 2023, representing a growth of 108.5%[19]. - Total current assets surged to $195,304,000 in 2024, up from $90,175,000 in 2023, marking a 116.5% increase[19]. Equity and Liabilities - Total stockholders' equity increased to $171,812,000 in 2024 from $81,720,000 in 2023, a growth of 109.8%[19]. - Total liabilities rose to $28,413,000 in 2024 from $14,215,000 in 2023, an increase of 100.0%[19]. Income and Other Financial Metrics - Other income for the full year 2024 was $10.3 million, compared to $4.8 million in 2023, primarily due to increased interest income[13]. - Other income, net, improved to $10,286,000 in 2024 from $4,809,000 in 2023, an increase of 114.5%[21]. - General and administrative expenses increased to $17,612,000 in 2024 from $14,088,000 in 2023, a rise of 25.5%[21]. Future Plans and Studies - The company plans to submit a Biologics License Application (BLA) seeking accelerated approval by the end of 2025[5]. - The ongoing open label extension (OLE) study continues to enroll participants, with data from the 50 mg dose expected to be reported in September 2025[6]. - The company is on track to initiate a global Phase 3 study in mid-2025, with feedback obtained from both FDA and EMA on the study protocol[6]. - The FDA has indicated openness to considering skin FXN concentration as a surrogate endpoint for accelerated approval, which could support the BLA submission[6]. Shareholder Information - The weighted average common shares outstanding increased to 61,256,084 in 2024 from 43,901,241 in 2023, a growth of 39.5%[21].

Core Viewpoint - Larimar Therapeutics is making significant progress in its nomlabofusp program, with plans for a Biologics License Application (BLA) submission by the end of 2025, aiming for accelerated approval based on promising clinical data and FDA feedback regarding skin frataxin (FXN) concentrations as a potential surrogate endpoint [2][4][11]. Clinical and Regulatory Progress - The company is enthusiastic about recent interactions with the FDA, which are aligned with its approach to using skin FXN concentrations as a novel surrogate endpoint for Friedreich's ataxia (FA) [2][4]. - Larimar is actively enrolling participants in its open label extension (OLE) study, with some participants receiving treatment for up to one year, and plans to report data from this study in September 2025 [2][11]. - The company is on track to initiate a global Phase 3 trial in mid-2025, having received feedback from both the FDA and European Medicines Agency (EMA) on the trial protocol [4][11]. Financial Results - As of December 31, 2024, Larimar reported cash, cash equivalents, and marketable securities totaling $183.5 million, providing a cash runway into the second quarter of 2026 [4][7]. - The company reported a net loss of $28.8 million for Q4 2024, compared to a net loss of $13.0 million in Q4 2023, and a full-year net loss of $80.6 million for 2024, up from $36.9 million in 2023 [7][10]. - Research and development expenses for Q4 2024 were $26.7 million, significantly higher than $10.6 million in Q4 2023, primarily due to increased manufacturing costs for nomlabofusp [8][12]. Upcoming Developments - Larimar plans to provide an update on the OLE study data in September 2025, focusing on participants receiving the 50 mg dose of nomlabofusp [11]. - The company has amended the OLE protocol to include premedication to mitigate the risk of allergic reactions, including anaphylaxis [11]. - The FDA has accepted the data supporting the transition to a lyophilized form of nomlabofusp, which is expected to be introduced into the clinical development program in mid-2025 [11].

Core Insights - Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases [3] - The company will present and participate in 1x1 investor meetings at the Leerink Partners Global Healthcare Conference from March 10 to 12, 2025 [1] - Larimar's lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia [3] Presentation Details - The presentation is scheduled for March 10, 2025, from 3:40 to 4:10 PM EST [2] - A webcast link for the presentation is provided, and a replay will be available for 30 days on the company's website [2] Company Overview - Larimar Therapeutics is developing an intracellular delivery platform to design fusion proteins targeting rare diseases characterized by deficiencies in intracellular bioactive compounds [3] - For more information, the company’s website is available at https://larimartx.com [3]

Core Viewpoint - Larimar Therapeutics has initiated dosing of adolescents aged 12-17 in a pediatric pharmacokinetic (PK) run-in study for Friedreich's ataxia, marking a significant step in evaluating the safety and pharmacokinetics of their lead compound, nomlabofusp [1][2]. Group 1: Study Details - The PK run-in study involves a randomized 2:1 allocation of participants to receive either nomlabofusp or a placebo for seven days [2]. - Following the assessment of safety and PK data, participants will be eligible to transition into the ongoing open-label extension (OLE) study [2][7]. - A second cohort of children aged 2-11 years is planned to begin in the first half of 2025 [2][7]. Group 2: Future Expectations - Long-term data from the OLE study, including 50 mg data from adults and available data from adolescents, is expected to be reported in mid-2025 [2][7]. - The OLE study will evaluate safety, tolerability, pharmacokinetics, FXN levels, and exploratory pharmacodynamic markers following long-term administration of nomlabofusp [3]. Group 3: Company Overview - Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with nomlabofusp as its lead compound for Friedreich's ataxia [4]. - The company aims to utilize its intracellular delivery platform to design additional fusion proteins targeting other rare diseases characterized by deficiencies in intracellular bioactive compounds [4].