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 ...

Core Insights - Larimar Therapeutics, Inc. reported its second quarter 2025 operating and financial results, highlighting progress in its nomlabofusp program aimed at treating Friedreich's ataxia [2][3] Financial Performance - As of June 30, 2025, the company had cash, cash equivalents, and marketable securities totaling $203.6 million, which includes $138.5 million in cash and $65.1 million from a public offering [1][7] - The net loss for the second quarter of 2025 was $26.2 million, or $0.41 per share, compared to a net loss of $21.6 million, or $0.34 per share, for the same period in 2024 [8] - Research and development expenses for the second quarter of 2025 were $23.4 million, up from $19.7 million in the second quarter of 2024, primarily due to increased clinical trial activity [9] - General and administrative expenses decreased to $4.4 million in the second quarter of 2025 from $4.9 million in the same quarter of 2024 [10] Clinical Development - The company is advancing its nomlabofusp program towards potential registration, with ongoing enrollment in an open-label study and plans to submit a Biologics License Application (BLA) in the second quarter of 2026 [3][5] - Initial data from the 50 mg dose in the open-label study and adolescent pharmacokinetic (PK) run-in study are expected in September 2025 [6][5] - The FDA has recommended that the safety database for the BLA include at least 30 participants with continuous study drug exposure for 6 months, with a subset of at least 10 participants for 1 year [12] Strategic Initiatives - Larimar has identified global sites for its Phase 3 trial and expects to initiate patient recruitment later this year [5][12] - The company has published two peer-reviewed articles supporting the mechanism of action of nomlabofusp and the use of skin frataxin concentrations as a surrogate endpoint [12][5] - The balance sheet has been strengthened through a recent capital raise, extending the projected cash runway into the fourth quarter of 2026 [7][12]

Core Viewpoint - Larimar Therapeutics, Inc. has successfully closed a public offering of 21,562,500 shares at a price of $3.20 per share, raising gross proceeds of $69.0 million before expenses [1][2]. Group 1: Offering Details - The offering included the full exercise of the underwriters' option to purchase an additional 2,812,500 shares [1]. - The shares were offered under a shelf registration statement declared effective by the SEC on May 24, 2024 [3]. Group 2: Use of Proceeds - The net proceeds from the offering will be utilized to support the development of nomlabofusp and other pipeline candidates, as well as for working capital and general corporate purposes, including research and development and pre-commercialization expenses [2]. Group 3: Company Overview - Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with its lead compound nomlabofusp aimed at treating Friedreich's ataxia [5]. - The company plans to leverage its intracellular delivery platform to design additional fusion proteins targeting other rare diseases characterized by deficiencies in intracellular bioactive compounds [5].

Core Viewpoint - Larimar Therapeutics, Inc. has announced a public offering of 18,750,000 shares of common stock at a price of $3.20 per share, aiming to raise approximately $60.0 million in gross proceeds before expenses [1][2]. Group 1: Offering Details - The offering consists of 18,750,000 shares priced at $3.20 each, with expected gross proceeds of $60.0 million [1]. - Underwriters have a 30-day option to purchase an additional 2,812,500 shares at the public offering price [1]. - The offering is expected to close around July 31, 2025, pending customary closing conditions [1]. Group 2: Use of Proceeds - Net proceeds from the offering will be used to support the development of nomlabofusp and other pipeline candidates, as well as for working capital and general corporate purposes, including research and development and pre-commercialization expenses [2]. Group 3: Company Overview - Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases [5]. - The company's lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia [5]. - Larimar plans to utilize its intracellular delivery platform to design additional fusion proteins targeting rare diseases characterized by deficiencies in intracellular bioactive compounds [5].

Core Viewpoint - Larimar Therapeutics, Inc. has initiated an underwritten public offering of its common stock and pre-funded warrants, aiming to raise capital for the development of its lead compound, nomlabofusp, and other pipeline candidates [1][2]. Group 1: Offering Details - The public offering includes an option for underwriters to purchase an additional 15% of the securities at the public offering price [1]. - The offering is subject to market conditions, and there is no guarantee regarding its completion or the final terms [1][3]. - The offering is made under a shelf registration statement that was declared effective by the SEC on May 24, 2024 [3]. Group 2: Use of Proceeds - The net proceeds from the offering will be utilized to support the development of nomlabofusp, other pipeline candidates, and for general corporate purposes, including research and development expenses [2]. Group 3: Company Overview - Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with nomlabofusp being developed for Friedreich's ataxia [5]. - The company plans to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases [5].

Industry Overview - The uncertainty surrounding tariffs and trade measures has impacted economic growth, with President Trump threatening to impose tariffs as high as 200% on pharmaceutical imports to encourage domestic production [1] - Despite these threats, the biotech sector has remained stable, with many global pharmaceutical companies already investing in domestic manufacturing [1] Current Trends - The Zacks Medical-Drugs industry is showing promising trends due to positive pipeline and regulatory developments [2] - Key areas of innovation include rare diseases, next-generation oncology treatments, obesity, immunology, and neuroscience, attracting significant investor interest [3] M&A Activity - M&A activity in the sector remains healthy, indicating growth potential for companies like Catalyst Pharmaceuticals, Zevra Therapeutics, Theravance Biopharma, Aldeyra Therapeutics, and Larimar Therapeutics [3] Industry Characteristics - The Zacks Medical-Drugs industry consists of small to medium-sized drug companies that produce medicines for human and veterinary use, often relying on collaboration payments for revenue [4] Factors Influencing Future Growth - The success of key pipeline candidates in clinical studies can significantly impact stock prices, with successful innovations acting as catalysts [5] - Strong collaboration with larger drugmakers is a positive indicator for small pharma companies, especially when equity investments are involved [6] - Investment in technology and personalized medicine is crucial for smaller companies to thrive in a changing healthcare landscape [7] Performance Metrics - The Zacks Medical-Drugs industry currently holds a Zacks Industry Rank of 96, placing it in the top 39% of 245 Zacks industries, indicating strong prospects [9] - Year-to-date, the industry has risen 5.6%, outperforming the Zacks Medical sector, which has decreased by 2.9%, while slightly underperforming the S&P 500, which has risen by 6.9% [11] Valuation - The industry is currently trading at a trailing 12-month price-to-sales ratio of 2.32, compared to the S&P 500's 5.66 and the Zacks Medical sector's 2.37 [12] Company Highlights - **Catalyst Pharmaceuticals**: Lead drug Firdapse shows strong demand; stock has risen 0.4% this year; earnings estimate for 2025 increased from $2.23 to $2.25 [16][17] - **Theravance Biopharma**: Strong sales from Yupelri; stock has risen 22.6% this year; earnings estimate for 2025 declined from 3 cents to 1 cent [20][21] - **Larimar Therapeutics**: Developing nomlabofusp for Friedreich's ataxia; stock has declined 18.3% this year; loss estimate for 2025 narrowed from $1.90 to $1.89 [24][25] - **Zevra Therapeutics**: Early adoption of Miplyffa exceeds expectations; stock has risen 53.4% this year; earnings estimate for 2025 increased from 31 cents to 76 cents [28][29] - **Aldeyra Therapeutics**: Focused on RASP modulators; stock has declined 0.4% this year; loss estimate for 2025 narrowed from $1.03 to 90 cents [31][32]

Core Insights - Larimar Therapeutics is advancing its lead compound, nomlabofusp, as a potential treatment for Friedreich's ataxia, with a Biologics License Application (BLA) submission planned for Q2 2026 seeking accelerated approval [1][2] Company Overview - Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with nomlabofusp being its lead compound targeting Friedreich's ataxia [3] Research Findings - Two peer-reviewed articles have been published that provide evidence of nomlabofusp's mechanism of action, demonstrating its ability to increase frataxin (FXN) levels in relevant tissues after administration [2][6] - The data from these studies support the potential use of skin FXN concentrations as a reasonably likely surrogate endpoint for the FDA's consideration in the accelerated approval process [1][2] Future Plans - The company is focused on executing near-term catalysts to advance nomlabofusp as the first potential disease-modifying therapy for patients with Friedreich's ataxia [2]

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']