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].

Biotech Analysis Central Service - The Biotech Analysis Central service offers a deep-dive analysis of pharmaceutical companies, with a monthly subscription fee of $49 and a yearly plan available at a 33.50% discount, priced at $399 per year [1] - The service includes a library of 600+ Biotech investing articles, a model portfolio of 10+ small and mid-cap stocks with detailed analysis, live chat, and a range of analysis and news reports to assist healthcare investors [2] Larimar Therapeutics, Inc - Larimar Therapeutics, Inc (NASDAQ: LRMR) was previously discussed in a Seeking Alpha article titled "Larimar: Stronger Buy Now More Than Ever After Partial Clinical Hold Completely" [2] Analyst and Seeking Alpha Disclosures - The analyst has no stock, option, or derivative positions in any mentioned companies and no plans to initiate such positions within the next 72 hours [3] - Seeking Alpha clarifies that past performance does not guarantee future results and that their analysts are third-party authors, including both professional and individual investors, who may not be licensed or certified by any regulatory body [4]

Core Insights - Larimar Therapeutics, Inc. released initial data from a long-term study on nomlabofusp for Friedreich's Ataxia, showing significant increases in tissue FXN levels [1][2] - The data indicates a promising potential for nomlabofusp to transform treatment for this rare neurological disease, with analysts projecting over 200% upside for the stock [2] Group 1: Study Results - The study evaluated daily subcutaneous injections of 25 mg of nomlabofusp, resulting in a mean change of 1.32 pg/μg in buccal cells and 9.28 pg/μg in skin cells at Day 90 [1] - Tissue FXN levels increased from 15% of healthy volunteers at baseline to 30% in buccal cells and from 16% to 72% in skin cells by Day 90 [2] - FXN levels reached steady-state by Day 30 in buccal cells [2] Group 2: Analyst Perspectives - William Blair considers the biomarker data for the 25 mg dose to be stronger than expected, nearing the 50% FXN level seen in unaffected healthy individuals [3] - The analyst anticipates that escalating the dose to 50 mg will further enhance FXN levels to at least 30% of normal levels in approximately 90% of patients [3] Group 3: Safety and Stock Performance - Concerns regarding the safety of nomlabofusp have contributed to stock weakness, with four patients withdrawing from the study due to logistical challenges and serious adverse events [4] - Despite these concerns, William Blair confirmed no CNS-related adverse events in toxicology studies and maintained an Outperform rating for the stock [5] - LRMR stock experienced an 11.8% decline, trading at $4.262 [6]

Core Insights - Larimar Therapeutics, Inc. released initial data from a long-term OLE study evaluating nomlabofusp for Friedreich's Ataxia, showing promising results in increasing tissue FXN levels [1][2] Group 1: Study Overview - The ongoing long-term OLE study involves daily subcutaneous injections of 25 mg of nomlabofusp in participants with Friedreich's Ataxia, with 14 patients treated for up to 260 days (mean 99 days) [1] - More than 50% of the patients in the study were non-ambulatory [2] Group 2: Efficacy Results - Tissue FXN levels showed a mean change from baseline of 1.32 pg/μg in buccal cells and 9.28 pg/μg in skin cells at Day 90 [2] - Nomlabofusp increased tissue FXN levels from 15% of healthy volunteers at baseline to 30% in buccal cells and from 16% to 72% in skin cells at Day 90 [2] - Steady-state levels of tissue FXN were reached by Day 30 in buccal cells [2] Group 3: Safety and Tolerability - Nomlabofusp was generally well tolerated, with two participants experiencing serious adverse events that resolved within 24 hours [3] - The most common adverse events were mild injection site reactions, which were brief and self-limited [4] Group 4: Future Developments - Dose escalation to 50 mg daily has been initiated in 6 participants [5] - Screening for adolescents with Friedreich's Ataxia is ongoing for a pediatric PK run-in study, with dosing expected in early 2025 [5] Group 5: Market Reaction - Following the release of the study data, LRMR stock declined by 21.5%, trading at $4.85 [6]

Core Insights - Larimar Therapeutics announced positive initial data from the ongoing open label extension (OLE) study of nomlabofusp, indicating it is generally well tolerated and shows potential clinical benefits for patients with Friedreich's ataxia (FA) [1][2][4] Group 1: Study Results - Daily subcutaneous injections of 25 mg nomlabofusp were administered to 14 participants for up to 260 days, with a mean treatment duration of 99 days [4] - Tissue frataxin (FXN) levels increased significantly, with buccal cells showing a rise from 15% of healthy volunteers at baseline to 30% at Day 90, and skin cells from 16% to 72% [1][5] - Early trends towards improvement in clinical outcomes were observed, including decreased values in modified Friedreich Ataxia Rating Scale (mFARS) and other clinical measures at Day 90 [5][6] Group 2: Pharmacokinetics and Safety - Pharmacokinetic data indicated that nomlabofusp levels in plasma reached a steady state by Day 30, with no further accumulation noted [1][5] - The treatment was generally well tolerated, with serious adverse events occurring in two participants that resolved within 24 hours [5][6] Group 3: Future Plans and Developments - The company has initiated dose escalation to 50 mg daily in the OLE study for six participants, with plans to increase the dose for all participants [1][6] - A pediatric pharmacokinetic run-in study is ongoing, with dosing expected to start early 2025, and adolescents completing the study will transition into the OLE [1][6] - A global confirmatory/registration study is planned to initiate in mid-2025, with a Biologics License Application (BLA) submission targeted for the second half of 2025 [1][7] Group 4: Financial Position - As of September 30, 2024, the company reported a strong balance sheet with $203.7 million in cash and investments, providing a projected runway into the second quarter of 2026 [1][6]

Core Insights - Larimar Therapeutics announced positive initial data from the ongoing open label extension (OLE) study of nomlabofusp, indicating it is generally well tolerated and shows potential clinical benefits for patients with Friedreich's ataxia (FA) [1][2] Group 1: Study Results - Daily subcutaneous injections of 25 mg nomlabofusp were well tolerated for up to 260 days in 14 participants [1] - Tissue frataxin (FXN) levels increased significantly, with buccal cells rising from 15% to 30% and skin cells from 16% to 72% at Day 90 [1][2] - Early trends towards improvement in clinical outcomes were observed at Day 90, suggesting potential clinical benefits across a broad spectrum of FA patients [1][4] Group 2: Pharmacokinetics and Safety - Pharmacokinetic data indicated that nomlabofusp levels in plasma reached a steady state by Day 30, with no further accumulation noted [1][4] - The most common adverse events were mild injection site reactions, with serious adverse events occurring in two participants that resolved within 24 hours [3][2] Group 3: Development Plans - The dose of nomlabofusp has been escalated to 50 mg daily for six participants, with plans to increase the dose for all current participants [5][2] - Screening for adolescents in a pediatric pharmacokinetic study is ongoing, with dosing expected to start in early 2025 [1][5] - A global confirmatory/registration study is planned to initiate in mid-2025, with a Biologics License Application (BLA) submission targeted for the second half of 2025 [1][6] Group 4: Financial Position - As of September 30, 2024, the company reported a strong balance sheet with $203.7 million in cash and investments, providing a runway into the second quarter of 2026 [1]

Core Viewpoint - Larimar (LRMR) shares have increased by 16.4% in the past four weeks, closing at $7.74, with a potential upside indicated by Wall Street analysts' price targets suggesting a mean estimate of $22.67, representing a 192.9% upside [1] Price Targets and Estimates - The mean estimate consists of nine short-term price targets with a standard deviation of $6.93, where the lowest estimate of $14 indicates an 80.9% increase, and the highest estimate suggests a 365.1% surge to $36 [2] - A low standard deviation among price targets indicates a high degree of agreement among analysts regarding the stock's price movement direction [7] Earnings Estimates and Analyst Sentiment - Analysts are optimistic about LRMR's earnings prospects, as indicated by a positive trend in earnings estimate revisions, which has shown a strong correlation with near-term stock price movements [9] - The Zacks Consensus Estimate for the current year has risen by 14.4% over the past month, with three estimates increasing and no negative revisions [10] - LRMR holds a Zacks Rank 1 (Strong Buy), placing it in the top 5% of over 4,000 ranked stocks based on earnings estimates, indicating strong potential for upside [11]

Product Development - The company is focused on developing nomlabofusp, a treatment for Friedreich's ataxia, which aims to increase frataxin levels in patients [68]. - The Phase 2 trial of nomlabofusp showed dose-dependent increases in FXN levels in all evaluated tissues after 14 days of daily dosing [70]. - The FDA lifted the partial clinical hold on nomlabofusp in May 2024, allowing further clinical development [70]. - The ongoing open label extension study for nomlabofusp is evaluating safety, pharmacokinetics, and frataxin levels in patients [70]. - The company plans to initiate a global confirmatory study by mid-2025, targeting a Biologics License Application filing in the second half of 2025 [70]. - The company has received multiple designations for nomlabofusp, including orphan drug designations in the U.S. and EU, and participation in the FDA's START pilot program [70]. Financial Performance - The company has not generated any revenue from product sales to date and does not expect to in the foreseeable future [78]. - Net loss for the three months ended September 30, 2024, was $15.5 million, an increase of $6.4 million from a net loss of $9.1 million in the same period of 2023 [82]. - Research and development expenses for the three months ended September 30, 2024, increased by $7.3 million to $13.9 million compared to $6.6 million in the same period of 2023 [82]. - General and administrative expenses for the three months ended September 30, 2024, rose by $0.6 million to $4.3 million compared to $3.8 million in the same period of 2023 [85]. - Research and development expenses for the nine months ended September 30, 2024, increased by $29.5 million to $46.5 million compared to $17.0 million in the same period of 2023 [87]. - General and administrative expenses for the nine months ended September 30, 2024, increased by $2.5 million to $13.1 million compared to $10.6 million in the same period of 2023 [88]. - Net cash used in operating activities for the nine months ended September 30, 2024, was $48.9 million, compared to $24.1 million in the same period of 2023 [92]. - Net cash provided by financing activities for the nine months ended September 30, 2024, was $161.8 million, primarily from a common stock offering [94]. - As of September 30, 2024, the company had an accumulated deficit of $240.3 million and cash, cash equivalents, and marketable securities of $203.7 million [96]. - The company expects to incur significant expenses and operating losses for the foreseeable future as it continues to advance the development of its product candidates [96]. Capital Raising - In February 2024, the company completed a public offering, issuing 19,736,842 shares at $8.74 per share, raising approximately $161.8 million [72]. - The company completed a public offering in February 2024, issuing 19,736,842 shares and receiving net proceeds of approximately $161.8 million [96]. Risks and Compliance - The company is subject to risks common to pre-commercial biotechnology firms, including regulatory approval and funding challenges [71]. - The company qualifies as a "smaller reporting company" and is not required to provide certain market risk disclosures [102]. - No recent accounting pronouncements applicable to the company were mentioned in the financial statements [100].

Exhibit 99.1 Larimar Therapeutics Reports Third Quarter 2024 Operating and Financial Results • Nomlabofusp program update expected mid-December to include available safety, pharmacokinetic (PK) and frataxin data, as well as available clinical outcomes observations from patients with Friedreich's ataxia (FA) receiving 25 mg of nomlabofusp daily for 30-180 days in ongoing open label extension (OLE) study • Initiation of PK run-in study in adolescents on track by year-end 2024 • Initiation of global confirmato ...

Nomlabofusp program update expected mid-December to include available safety, pharmacokinetic (PK) and frataxin data, as well as available clinical outcomes observations from patients with Friedreich's ataxia (FA) receiving 25 mg of nomlabofusp daily for 30-180 days in ongoing open label extension (OLE) study Initiation of PK run-in study in adolescents on track by year-end 2024 Initiation of global confirmatory/registration study planned mid-2025 Biologics License Application (BLA) submission for nomlabofu ...