These two biotechnology stocks could be gearing up for a run. Growth stocks have been outpacing value and dividend stocks since 2008, and this performance gap has only widened as the pandemic's impact has waned. Technological breakthroughs are opening new markets in high- demand areas, fueling investor interest in a wide array of next-generation platforms. Biotechnology, among the best-performing industries until interest rates flipped in 2022, is rife with top growth stocks trading at rock-bottom valuation ...

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].

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 ...

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 ...

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 ...

Larimar Therapeutics Reports Fourth Quarter and Full Year 2023 Operating and Financial Results and Provides Update on Nomlabofusp Development Bala Cynwyd, PA, March 14, 2024 – Larimar Therapeutics, Inc. ("Larimar") (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its fourth quarter and full year 2023 operating and financial results. "This year we made tremendous progress across key clinical and regulatory milestones for our nom ...

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 ...