Larimar Therapeutics(LRMR) - 2024 Q1 - Quarterly Results

Executive Summary & Business Update 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 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] - 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] - 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] - The company aims to submit a Biologics License Application (BLA) in H2 2025 and is discussing an accelerated approval pathway with the FDA[3] - 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] Recent Operational and Clinical Milestones 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][12] - 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][6] - 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] - 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][6][7] - 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][8][12] - 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] Financial Performance 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 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] - 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] Condensed Consolidated Balance Sheet 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 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 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 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] - The company's lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia[11] - 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] Forward-Looking Statements 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] - 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] - 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] - 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] Investor and Company Contacts 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] - Company Contact: Michael Celano (Chief Financial Officer), Email: mcelano@larimartx.com, Phone: (484) 414-2715[15]