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].

In the Phase 2 dose exploration study, nomlabofusp was generally well-tolerated throughout the four-week treatment period. Nomlabofusp had a predictable pharmacokinetic profile and demonstrated dose-dependent increases in frataxin levels in skin and buccal cells. All patients with quantifiable levels at baseline and Day 14 in the 50 mg cohort achieved frataxin levels in skin cells over 33% of the average level observed in healthy volunteers at Day 14, and 3 patients achieved levels greater than 50% of the a ...

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 ...

• First patient dosed in open label extension (OLE) study with 25 mg daily dosing of nomlabofusp; interim data on track for Q4 2024 • Positive top-line Phase 2 dose exploration study data demonstrated nomlabofusp was generally well-tolerated with dose-dependent increases in tissue frataxin levels, reinforcing therapeutic potential • Biologics License Application ("BLA") submission targeted for 2H 2025; discussions initiated with Food and Drug Administration ("FDA") on potential to pursue accelerated approva ...

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].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 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 other jurisdiction of incorporation or organization) Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT O ...

1 August 2023 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 CTI-1601 and other planned product candidates, Larimar's planned research and development efforts, includi ...

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 June 30, 2023 or Commission File Number: 001-36510 (Exact name of registrant as specified in its charter) Delaware 20-3857670 (State or other jurisdiction of incorporation or organization) Three Bala Plaza East, Suite 506 19004 Bala Cynwyd, PA (Zip Code) (Address of principal executive offices) LA ...