Financial Performance - The net loss for the second quarter of 2024 was $21.6 million, or $0.34 per share, compared to a net loss of $8.4 million, or $0.19 per share, for the same period in 2023[6]. - The company reported a net loss of $36.3 million, or $0.62 per share, for the first six months of 2024, compared to a net loss of $14.9 million, or $0.34 per share, for the same period in 2023[7]. - Net loss for Q2 2024 was $21,627,000, compared to a net loss of $8,366,000 in Q2 2023, representing an increase of 158%[14]. - Net loss per share for Q2 2024 was $0.34, compared to $0.19 in Q2 2023[14]. - Comprehensive loss for Q2 2024 was $21,752,000, compared to $8,354,000 in Q2 2023[14]. - Net loss for the six months ended June 30, 2024, was $36,281,000, compared to $14,892,000 for the same period in 2023[14]. Research and Development Expenses - Research and development expenses for the second quarter of 2024 were $19.7 million, an increase of $13.8 million from $5.9 million in the second quarter of 2023, primarily due to increased manufacturing costs for nomlabofusp[6]. - Research and development expenses for the first six months of 2024 were $32.6 million, up from $10.4 million in the same period of 2023, reflecting increased costs associated with nomlabofusp[7]. - Research and development expenses for Q2 2024 were $19,682,000, a significant increase of 234% compared to $5,875,000 in Q2 2023[14]. - Research and development expenses for the six months ended June 30, 2024, totaled $32,621,000, up from $10,437,000 in the same period of 2023[14]. Operating Expenses - General and administrative expenses for the second quarter of 2024 were $4.9 million, compared to $3.7 million in the second quarter of 2023, an increase of $1.2 million[6]. - Total operating expenses for Q2 2024 reached $24,599,000, up 156% from $9,620,000 in Q2 2023[14]. - Total operating expenses for the six months ended June 30, 2024, were $41,333,000, compared to $17,257,000 in the prior year[14]. Cash Position - As of June 30, 2024, Larimar Therapeutics reported cash, cash equivalents, and marketable securities totaling $226.1 million, providing a projected cash runway into 2026[5]. Clinical Development - The company plans to initiate a pharmacokinetic (PK) run-in study in adolescents by year-end 2024, with an initial enrollment of 12-15 patients[3]. - All 7 sites for the open label extension (OLE) study have been activated, with interim data expected in Q4 2024[4]. - Larimar has been selected by the FDA to participate in the START pilot program for nomlabofusp, which may aid in achieving the timeline for the Biologics License Application (BLA) submission targeted for the second half of 2025[2]. - Larimar plans to initiate a global confirmatory study by mid-2025, with potential sites in the U.S., Europe, U.K., Canada, and Australia[1]. Shareholder Information - Weighted average common shares outstanding increased to 63,801,792 in Q2 2024 from 43,897,603 in Q2 2023[14]. - Other income (expense), net for Q2 2024 was $2,972,000, compared to $1,254,000 in Q2 2023[14].

START is a new milestone-driven program designed to accelerate development of novel therapies intended to address an unmet medical need for rare diseases Nomlabofusp was selected based on potential for clinical benefit in a rare neurodegenerative disease and demonstrated development program readiness START pilot program is intended to improve development efficiency through enhanced communication with the FDA Nomlabofusp is expected to be one of three CDER programs and one of six total programs selected by t ...

Group 1: Industry Overview - Growth stocks have been outperforming value and dividend stocks since 2008, with the gap widening post-pandemic [1] - Biotechnology has been one of the best-performing industries until interest rates changed in 2022, with many top growth stocks currently trading at low valuations [2] Group 2: Avidity Biosciences - Avidity Biosciences is pioneering a new approach to RNA therapeutics through antibody-oligonucleotide conjugates (AOCs), combining RNA therapeutic precision with monoclonal antibody specificity [3] - The lead candidate, Del-desiran (AOC 1001), targets myotonic dystrophy type 1, affecting around 40,000 individuals in the U.S., with a pivotal trial expected to launch soon [4] - Major clinical updates from the trial are anticipated around mid-2025, with successful outcomes potentially unlocking significant shareholder value [5] - The company has a cash runway extending into 2026, minimizing short-term dilution risk, though clinical setbacks could negatively impact share prices [6] Group 3: Larimar Therapeutics - Larimar Therapeutics is developing a protein replacement therapy, nomlabofusp, for Friedreich's ataxia (FA), a rare genetic disease, highlighting the high stakes in this treatment area [7] - The stock has increased over 55% this year due to the FDA lifting a partial clinical hold, positive trial updates, and a successful capital raise extending its cash runway into 2026 [8] - Larimar's market capitalization of $459 million may not reflect its value proposition, given the $2 billion-plus market opportunity for FA treatments, with the unique profile of nomlabofusp potentially allowing for significant market share capture [9] - The company's future is highly dependent on the success of its FA therapy, making it a high-risk but potentially high-reward investment [10]

Core Viewpoint - Larimar Therapeutics, Inc. has received FDA approval to lift the partial clinical hold on its nomlabofusp (CTI-1601) clinical program, leading to an 8.5% increase in share price on May 21 [1]. Company Overview - Larimar's lead candidate, nomlabofusp, is being developed for treating Friedreich's ataxia (FA), a rare and fatal nervous system disease caused by frataxin deficiency [2]. - The company has no marketed products currently, making the successful development of nomlabofusp its primary focus [7]. Clinical Study Details - In February 2024, Larimar completed a four-week, placebo-controlled phase II dose exploration study for nomlabofusp, which was well-tolerated [3]. - The FDA's review included data from 25 mg and 50 mg cohorts, with patients receiving daily dosing for 14 days, followed by alternate day dosing until day 28 [3]. Efficacy Results - At day 14, patients treated with 50 mg of nomlabofusp achieved frataxin levels exceeding 33% of the average levels seen in healthy volunteers, with three patients exceeding 50% [4]. Ongoing Studies - Nomlabofusp (25 mg) is currently being evaluated in an open label extension (OLE) study, which will initially investigate daily subcutaneous injections [5]. - The company plans to escalate dosing to 50 mg in the OLE study after further pharmacodynamics characterization at the 25 mg dose, with interim data expected in Q4 2024 [6]. Market Context - Biogen currently markets Skyclarys, the first approved treatment for FA, which was approved by the FDA in February 2023 and in Europe in February 2024 [8]. - The acquisition of Reata Pharmaceuticals by Biogen in September 2023 added this treatment to its portfolio, with ongoing launches in the U.S. and EU [8]. Stock Performance - Year-to-date, Larimar's shares have increased by 73.4%, contrasting with a 6.5% decline in the industry [4].

Core Viewpoint - The U.S. FDA has lifted the partial clinical hold on Larimar Therapeutics' nomlabofusp (CTI-1601) clinical program, which is being developed for the treatment of Friedreich's Ataxia (FA) [1][2] Group 1: Clinical Development - Nomlabofusp is a novel protein replacement therapy aimed at delivering frataxin to mitochondria, addressing the root cause of FA [1][4] - The FDA's decision followed a review of data from a completed four-week, placebo-controlled Phase 2 dose exploration study, which included 25 mg and 50 mg cohorts [1][2] - The Phase 2 study showed that nomlabofusp was generally well-tolerated, with a predictable pharmacokinetic profile and dose-dependent increases in frataxin levels in skin and buccal cells [2][3] Group 2: Ongoing Studies - An ongoing open-label extension (OLE) study is evaluating the long-term safety and pharmacokinetics of nomlabofusp, initially using a 25 mg dose [3][7] - The company plans to escalate the dose to 50 mg in the OLE study after further characterization of frataxin pharmacodynamics at the 25 mg dose [2][3] - Interim data from the OLE study is expected in the fourth quarter of 2024, with a Biologics License Application (BLA) submission targeted for the second half of 2025 [7] Group 3: Regulatory Designations - Nomlabofusp has received several designations from the FDA, including Rare Pediatric Disease designation, Fast Track designation, and Orphan Drug designation [4][5] - The European Commission has also granted Orphan Drug Designation, and the European Medicines Agency has awarded a PRIME designation [4]

About Nomlabofusp (CTI-1601) Nomlabofusp is a recombinant fusion protein intended to deliver human frataxin to the mitochondria of patients with Friedreich's ataxia who are unable to produce enough of this essential protein. Nomlabofusp has been granted Rare Pediatric Disease designation, Fast Track designation and Orphan Drug designation by the U.S. Food and Drug Administration (FDA), Orphan Drug Designation by the European Commission, and a PRIME designation by the European Medicines Agency. About Larimar ...

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 For the quarterly period ended March 31, 2024 or ☐ 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 ...

[Executive Summary & Business Update](index=1&type=section&id=Executive%20Summary%20%26%20Business%20Update) Larimar Therapeutics achieved significant Q1 2024 progress, advancing nomlabofusp's clinical program with positive Phase 2 data, initiating an OLE study, and planning a BLA submission by H2 2025, supported by successful financing [Q1 2024 Overview and CEO Commentary](index=1&type=section&id=Q1%202024%20Overview%20and%20CEO%20Commentary) Larimar Therapeutics made significant Q1 2024 progress, advancing nomlabofusp's clinical program with positive Phase 2 data, initiating an OLE study, and planning a BLA submission by H2 2025, supported by successful financing - CEO Dr. Carole Ben-Maimon stated a strong start to 2024, achieving key milestones supporting the late-stage advancement of the nomlabofusp clinical program[3](index=3&type=chunk) - Phase 2 dose exploration study data for nomlabofusp showed the drug was generally well-tolerated and demonstrated dose-dependent increases in tissue frataxin levels, potentially addressing the root cause of Friedreich's ataxia (FA)[3](index=3&type=chunk) - The company has initiated an OLE study, with plans to report interim data in **Q4 2024** to provide long-term safety and tissue frataxin level information for nomlabofusp[3](index=3&type=chunk) - The company aims to submit a Biologics License Application (BLA) in **H2 2025** and is discussing an accelerated approval pathway with the FDA[3](index=3&type=chunk) - Recent successful capital injection extended the company's cash runway into **2026**, laying the groundwork for advancing potential therapies to increase frataxin levels in FA patients[3](index=3&type=chunk) [Recent Operational and Clinical Milestones](index=1&type=section&id=Recent%20Operational%20and%20Clinical%20Milestones) Larimar Therapeutics achieved key Q1 2024 milestones, including launching the nomlabofusp OLE study, reporting positive Phase 2 data, discussing accelerated approval with the FDA, securing **$161.8 million** in financing, and appointing a VP of Commercial - The first patient has received a **25 mg daily dose** of nomlabofusp in the Open-Label Extension (OLE) study, with interim data expected in **Q4 2024**[5](index=5&type=chunk)[12](index=12&type=chunk) - Positive topline data from the Phase 2 dose exploration study showed nomlabofusp was generally well-tolerated and dose-dependently increased tissue frataxin levels, with significant increases in skin and buccal cell frataxin levels, particularly in the **25 mg and 50 mg dose groups**[5](index=5&type=chunk)[6](index=6&type=chunk) - By Day 14, all patients receiving **50 mg** nomlabofusp (with quantifiable baseline and Day 14 levels) achieved skin cell frataxin levels **above 33%** of the healthy volunteer average, with **three patients exceeding 50%**[6](index=6&type=chunk) - The company has initiated discussions with the FDA regarding tissue frataxin levels as a potential novel surrogate endpoint to support an accelerated approval pathway, with a BLA submission planned for **H2 2025**[5](index=5&type=chunk)[6](index=6&type=chunk)[7](index=7&type=chunk) - Successfully raised **$161.8 million** through a public offering, increasing cash, cash equivalents, and marketable securities to **$239.0 million** as of March 31, 2024, extending the cash runway into **2026**[5](index=5&type=chunk)[8](index=8&type=chunk)[12](index=12&type=chunk) - Larimar appointed Frank Nazzario as Vice President of Commercial in **March 2024**, beginning to build its commercial team, with Mr. Nazzario bringing nearly **30 years** of leadership experience in rare disease drug launches[12](index=12&type=chunk) [Financial Performance](index=2&type=section&id=Financial%20Performance) Larimar Therapeutics' Q1 2024 financial performance was marked by a **$14.7 million** net loss, driven by increased R&D and G&A expenses, despite a strong cash position of **$239.0 million** following a public offering [First Quarter 2024 Financial Results Overview](index=2&type=section&id=First%20Quarter%202024%20Financial%20Results%20Overview) As of March 31, 2024, Larimar Therapeutics held **$239.0 million** in cash, cash equivalents, and marketable securities, reporting a **net loss of $14.7 million** for Q1 2024, primarily due to increased R&D and G&A expenses Q1 2024 Key Financial Data | Metric | Q1 2024 (Millions USD) | Q1 2023 (Millions USD) | YoY Change (Millions USD) | YoY Change (%) | | :------------------------------------------ | :-------------------------- | :-------------------------- | :-------------------- | :------------- | | Cash, Cash Equivalents, and Marketable Securities (End of Period) | 239.0 | N/A | N/A | N/A | | Net Loss | (14.7) | (6.5) | (8.2) | 126.2% | | Net Loss Per Share | (0.27) | (0.15) | (0.12) | 80.0% | | Research and Development Expenses | 12.9 | 4.6 | 8.3 | 180.4% | | General and Administrative Expenses | 3.8 | 3.1 | 0.7 | 22.6% | - Research and development expenses increased by **$8.3 million**, primarily driven by a **$5.7 million** increase in nomlabofusp manufacturing costs, **$1.0 million** in clinical costs related to the OLE study initiation, **$1.0 million** in personnel expenses, **$0.3 million** in consulting fees, and **$0.2 million** in stock-based compensation expense[9](index=9&type=chunk) - General and administrative expenses increased by **$0.7 million**, primarily driven by a **$0.2 million** increase in personnel expenses, **$0.2 million** in legal fees, and **$0.1 million** in stock-based compensation expense[10](index=10&type=chunk) [Condensed Consolidated Balance Sheet](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheet) As of March 31, 2024, Larimar Therapeutics' total assets significantly increased to **$248.2 million**, primarily due to a substantial rise in cash and marketable securities, reflecting capital injection from a public offering Condensed Consolidated Balance Sheet (Unaudited, in Thousands USD) | Assets | March 31, 2024 | December 31, 2023 | | :-------------------------------- | :------------- | :-------------- | | **Current Assets:** | | | | Cash and cash equivalents | 110,125 | 26,749 | | Marketable securities, short-term | 117,171 | 60,041 | | Prepaid expenses and other current assets | 3,657 | 3,385 | | **Total Current Assets** | **230,953** | **90,175** | | Marketable securities, long-term | 11,711 | — | | Property and equipment, net | 604 | 684 | | Operating lease right-of-use assets | 2,920 | 3,078 | | Restricted cash | 1,339 | 1,339 | | Other assets | 678 | 659 | | **Total Assets** | **248,205** | **95,935** | | **Liabilities and Stockholders' Equity** | | | | **Current Liabilities:** | | | | Accounts payable | 1,918 | 1,283 | | Accrued expenses | 10,098 | 7,386 | | Operating lease liabilities, current | 825 | 837 | | **Total Current Liabilities** | **12,841** | **9,506** | | Operating lease liabilities | 4,520 | 4,709 | | **Total Liabilities** | **17,361** | **14,215** | | **Stockholders' Equity:** | | | | Common stock | 64 | 43 | | Additional paid-in capital | 434,013 | 270,150 | | Accumulated deficit | (203,208) | (188,554) | | Accumulated other comprehensive income (loss) | (25) | 81 | | **Total Stockholders' Equity** | **230,844** | **81,720** | | **Total Liabilities and Stockholders' Equity** | **248,205** | **95,935** | [Condensed Consolidated Statements of Operations](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Larimar Therapeutics' operating and net losses widened in Q1 2024 compared to the prior year, primarily due to significant increases in research and development and general and administrative expenses Condensed Consolidated Statements of Operations (Unaudited, in Thousands USD, Except Per Share Data) | | For the Three Months Ended March 31, | | :-------------------------------------- | :------------- | :------------- | | | **2024** | **2023** | | **Operating expenses:** | | | | Research and development | 12,939 | 4,562 | | General and administrative | 3,795 | 3,075 | | **Total operating expenses** | **16,734** | **7,637** | | **Operating loss** | **(16,734)** | **(7,637)** | | Other income, net | 2,080 | 1,111 | | **Net loss** | **(14,654)** | **(6,526)** | | Net loss per share, basic and diluted | (0.27) | (0.15) | | Weighted-average common shares outstanding, basic and diluted | 53,553,707 | 43,897,603 | | **Comprehensive loss:** | | | | Net loss | (14,654) | (6,526) | | Other comprehensive income (loss): | | | | Unrealized gain (loss) on marketable securities | (106) | 31 | | **Total other comprehensive income (loss)** | **(106)** | **31** | | **Total comprehensive loss** | **(14,760)** | **(6,495)** | [Company Information & Disclosures](index=2&type=section&id=Company%20Information%20%26%20Disclosures) This section provides essential information about Larimar Therapeutics, including its strategic focus, forward-looking statements, and key contact details for investors and the company [About Larimar Therapeutics](index=2&type=section&id=About%20Larimar%20Therapeutics) Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with nomlabofusp for Friedreich's ataxia and plans to leverage its intracellular delivery platform for other rare conditions - Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases[11](index=11&type=chunk) - The company's lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia[11](index=11&type=chunk) - Larimar plans to leverage its intracellular delivery platform to design other fusion proteins targeting additional rare diseases caused by deficiencies in intracellular bioactive compounds[11](index=11&type=chunk) [Forward-Looking Statements](index=3&type=section&id=Forward-Looking%20Statements) This press release contains forward-looking statements based on management's beliefs and assumptions, involving risks and uncertainties that could cause actual results to differ materially from projections - This press release contains forward-looking statements based on management's beliefs, assumptions, and current information, regarding the company's ability to develop and commercialize nomlabofusp and other product candidates[13](index=13&type=chunk) - Forward-looking statements cover R&D efforts, nomlabofusp clinical trial timing, interactions with the FDA, overall development plans, business strategy, financing capabilities, capital use, operating results, financial condition, and future operational plans and objectives[13](index=13&type=chunk) - These statements involve risks, uncertainties, and other factors that could cause actual results, performance, or achievements to differ materially from those expressed or implied by forward-looking statements, including product development activities, the success, cost, and timing of non-clinical studies and clinical trials, and ongoing interactions with the FDA[14](index=14&type=chunk) - Additional risks include preliminary clinical trial results differing from final outcomes, early data not being predictive, potential FDA disagreement with development strategies, impacts of public health crises, ability to optimize and scale manufacturing processes, obtaining regulatory approvals, developing sales and marketing capabilities, and raising necessary capital[14](index=14&type=chunk) [Investor and Company Contacts](index=3&type=section&id=Investor%20and%20Company%20Contacts) This section provides contact information for Larimar Therapeutics' investor relations and chief financial officer, facilitating stakeholder access to further information - Investor Contact: Joyce Allaire (LifeSci Advisors), Email: jallaire@lifesciadvisors.com, Phone: (212) 915-2569[15](index=15&type=chunk) - Company Contact: Michael Celano (Chief Financial Officer), Email: mcelano@larimartx.com, Phone: (484) 414-2715[15](index=15&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or ☐ 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 (State or other jurisdiction of inco ...

Financial Performance - Larimar reported a net loss of $13.0 million, or $0.30 per share, for Q4 2023, compared to a net loss of $9.4 million, or $0.21 per share, in Q4 2022[6]. - For the full year 2023, the company reported a net loss of $36.9 million, or $0.84 per share, compared to a net loss of $35.4 million, or $1.37 per share, in 2022[9]. - The net loss for Q4 2023 was $12,993,000, compared to a net loss of $9,425,000 in Q4 2022, reflecting a 38% increase in losses year-over-year[22]. - The company reported a net loss of $36,949,000 for the year ended December 31, 2023, compared to a net loss of $35,355,000 in 2022, which is a 4% increase[22]. - The net loss per share for Q4 2023 was $0.30, an improvement from $0.21 in Q4 2022[22]. Expenses - Research and development expenses for Q4 2023 were $10.6 million, up from $7.2 million in Q4 2022, driven by increases in drug manufacturing, consulting, and personnel expenses[7]. - General and administrative expenses for the full year 2023 were $14.1 million, compared to $12.3 million in 2022, primarily due to increases in stock-based compensation and personnel expenses[13]. - Total operating expenses for Q4 2023 were $14,162,000, an increase from $10,439,000 in Q4 2022, representing a 35% increase year-over-year[22]. - Research and development expenses for the year ended December 31, 2023, were $27,670,000, up from $24,250,000 in 2022, indicating a 10% increase[22]. - Total operating expenses for the year ended December 31, 2023, were $41,758,000, compared to $36,526,000 in 2022, marking a 14% increase[22]. - General and administrative expenses for Q4 2023 were $3,514,000, up from $3,221,000 in Q4 2022, indicating a 9% increase[22]. Cash and Assets - As of December 31, 2023, the company had cash, cash equivalents, and marketable securities totaling $86.8 million[6]. - The total assets of the company as of December 31, 2023, were $95.9 million, down from $126.4 million in 2022[19]. Funding and Future Outlook - The company raised approximately $161.6 million in net proceeds through a public offering of common stock in February 2024, extending its operating runway into 2026[5]. - The company continues to focus on research and development to drive future growth despite the increased operating losses[22]. Clinical Developments - Positive top-line data from the Phase 2 dose exploration study of nomlabofusp showed dose-dependent increases in frataxin levels, with all patients treated with 50 mg achieving skin frataxin levels greater than 33% of healthy volunteers[4]. - Discussions with the FDA are ongoing regarding the use of tissue frataxin levels as a novel surrogate endpoint to support a Biologics License Application (BLA) submission targeted for the second half of 2025[11]. - The first patient in the open-label extension (OLE) study was dosed in March 2024, with interim data expected in Q4 2024[5]. Other Income - Other income for Q4 2023 was $1,169,000, compared to $1,014,000 in Q4 2022, showing a 15% increase[22]. - The weighted average common shares outstanding for Q4 2023 were 43,905,603, slightly up from 43,897,241 in Q4 2022[22].