Summary of Laramar Therapeutics Conference Call Company Overview - **Company**: Laramar Therapeutics - **Focus**: Development of nonlobofusp (formerly CTI-16-01) for the treatment of Friedreich's Ataxia (FA), a rare neurodegenerative disease [doc id='13'][doc id='16'] Industry Context - **Disease**: Friedreich's Ataxia (FA) is characterized by low levels of frataxin, leading to severe neurological symptoms and a life expectancy of 30 to 50 years [doc id='15'][doc id='14'] - **Current Treatments**: The FDA approved omevaloxolone in 2023, but it does not affect frataxin levels, highlighting the unmet need for therapies that address the underlying deficiency [doc id='16'] Key Regulatory Updates - **FDA Recommendations**: Laramar received FDA guidance on the safety database for the Biologics License Application (BLA) submission, requiring data from at least 30 participants exposed to the drug for six months and 10 participants for one year [doc id='7'][doc id='8'] - **BLA Submission Timeline**: The company plans to submit the BLA in Q2 2026, with a U.S. launch targeted for early 2027 [doc id='25'][doc id='31'] Clinical Development - **Clinical Trials**: Ongoing studies include a global Phase 3 trial and an open-label extension study to evaluate long-term safety and efficacy [doc id='11'][doc id='12'] - **Patient Population**: The Phase 3 study will include patients aged 2 to 40, with a focus on younger patients [doc id='28'][doc id='29'] - **Efficacy Data**: Initial data from the 25 mg dose showed increases in frataxin levels and early trends towards clinical improvement [doc id='19'][doc id='20'] Safety Profile - **Adverse Events**: Nonlobofusp has been generally well tolerated, with mild injection site reactions being the most common adverse events [doc id='20][doc id='21'] - **Allergic Reactions**: Anaphylaxis has been reported, particularly in patients with prior exposure, leading to the introduction of antihistamine premedication [doc id='21'][doc id='22'] Financial Position - **Cash Balance**: As of March 31, the company reported $158 million in cash, sufficient to support operations through the BLA filing [doc id='61'] - **Funding Strategy**: Laramar is exploring non-dilutive financing options, including royalty financing [doc id='61] Future Plans - **Expansion of Studies**: The company plans to enroll children aged 2 to 11 directly into the open-label study, pending FDA discussions [doc id='26'][doc id='105'] - **Data Reporting**: Upcoming data cuts in September will include safety and pharmacokinetic data from 30 to 40 participants [doc id='26'][doc id='39'] Conclusion - Laramar Therapeutics is making significant progress in the development of nonlobofusp for FA, with clear regulatory guidance from the FDA and a robust clinical program aimed at addressing the unmet needs of patients with this debilitating disease [doc id='30'][doc id='31']

Larimar Therapeutics Nomlabofusp Regulatory Update June 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 ...

BALA CYNWYD, Pa., June 20, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that the Company will host a conference call and webcast to discuss regulatory updates for the Company’s nomlabofusp clinical development program for the treatment of Friedreich’s Ataxia on Monday, June 23, 2025 at 8:00 am EDT. Conference Call and Webcast DetailsTo access the webcast on Mond ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___ to ____ Commission File Number: 001-36510 LARIMAR THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 20-3857670 (State or other jurisdiction of incorporation or organizati ...

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]

FDA stated as part of a START pilot program meeting that it is open to considering skin FXN concentration as a reasonably likely surrogate endpoint in support of an accelerated approval BLA seeking accelerated approval planned to be submitted by year-end 2025; global Phase 3 study planned to initiate in mid-2025 Completed dosing in adolescent PK run-in study; topline 50 mg dose data from the OLE study and data from adolescent cohort planned for program update in September 2025 Strong balance sheet of $157. ...

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]