Oppenheimer has initiated coverage on Larimar Therapeutics LRMR, a clinical-stage biotechnology company focused on developing treatments for rare diseases. The analysts view the company's lead program, Nomlabofusp, as a differentiated, disease-modifying therapy that targets the key pathogenic mechanism of frataxin (FXN) deficiency in Friedreich's ataxia (FA). Lower FXN levels predict early onset, disease severity, and faster progression. Friedreich's ataxia is a rare neurodegenerative disease affecting ~20, ...

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, 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 o ...

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].

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 ...