Larimar Therapeutics Reports First Quarter 2025 Financial Results

BALA CYNWYD, Pa., April 30, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today reported its first quarter 2025 operating and financial results.

Completed Dosing of Adolescents in Pharmacokinetic (PK) Run-In Study.

Potential for Accelerated Approval Pathway Based on Skin FXN Concentrations. 

Planned Upcoming Regulatory Discussions. 

50 mg OLE Data Expected in September 2025. 

Planned Transition to Lyophilized Form of Nomlabofusp. 

BLA Submission and Initiation of Global Phase 3 Study on Track

Gene circuits enable more precise control of gene therapy

Anne Trafton | MIT News Publication Date:April 28, 2025 

Very few gene therapy treatments have been approved by the FDA. One of the challenges to developing these treatments has been achieving control over how much the new gene is expressed in cells — too little and it won’t succeed, too much and it could cause serious side effects. 

To demonstrate this system, the researchers designed ComMAND circuits that could deliver the gene FXN, which is mutated in Friedreich’s ataxia — a disorder that affects the heart and nervous system. They also delivered the gene Fmr1, whose dysfunction causes fragile X syndrome. In tests in human cells, they showed that they could tune gene expression levels to about eight times the levels normally seen in healthy cells. 

Without ComMAND, gene expression was more than 50 times the normal level, which could pose safety risks. Further tests in animal models would be needed to determine the optimal levels, the researchers say.

Model-guided design of microRNA-based gene circuits supports precise dosage of transgenic cargoes into diverse primary cells

Model-guided design of microRNA-based gene circuits supports precise dosage of transgenic cargoes into diverse primary cells. Love, Kasey S. et al., Cell Systems, Volume 0, Issue 0, 101269 DOI: 10.1016/j.cels.2025.101269

In a therapeutic context, supraphysiological expression of transgenes can compromise engineered phenotypes and lead to toxicity. To ensure a narrow range of transgene expression, we developed a single-transcript, microRNA-based incoherent feedforward loop called compact microRNA-mediated attenuator of noise and dosage (ComMAND).

ComMAND effectively sets levels of the clinically relevant transgenes frataxin (FXN) and fragile X messenger ribonucleoprotein 1 (Fmr1) within a narrow window.

Progression of Friedreich Ataxia to Scoliosis and Cardiomyopathy: Utility of Real-World Data From Medical Claims (P12-5.032)

Progression of Friedreich Ataxia to Scoliosis and Cardiomyopathy: Utility of Real-World Data From Medical Claims (P12-5.032) April 8, 2025 issue 104 (7_Supplement_1) doi:10.1212/WNL.0000000000210504 

 Real-world data from medical claims can augment evidence from natural history studies in FA to allow for analyses of a broader range of commercially insured patients and explore patient characteristics, practice patterns, and outcomes not currently available.

Timing and Frequency of Onset Symptoms in Friedreich Ataxia Based on Real-world Medical Claims Data (P12-5.031)

Pravin Khemani, Shakti V. Nayar, Boyang Bian, Daniel Gomes, Sarah England, James McKay, Tony Wang, and Robin L. Avila​; Neurology® April 8, 2025 issue 104 (7_Supplement_1). doi:10.1212/WNL.0000000000210523

Real-world data from medical claims can augment evidence from prospectively designed natural history studies in helping to identify FA-related events at the time of disease onset and prior to FA diagnosis, allowing for additional insights on the early burden of disease.

Employment and work ability in individuals living with rare diseases: a systematic literature review

Bougas, N., Kangas, T., Vanthomme, K. et al. Employment and work ability in individuals living with rare diseases: a systematic literature review. Orphanet J Rare Dis 20, 193 (2025). doi:10.1186/s13023-025-03691-7 

These results show that individuals with rare diseases tend to have poor work outcomes, but methodological limitations hamper the understanding of health selection effects. Implications for future research and policy-making are discussed.

Biogen are withdrawing from the NICE appraisal for Skyclarys

Biogen. 24th April 2025​, Following your request for information, I want to inform you that Biogen has withdrawn omaveloxolone from the National Institute for Health and Care Excellence (NICE) appraisal process. This follows the news of the medicine’s approval by the Medicines and Healthcare products Regulatory Agency (MHRA) on 23rd April.

Artificial Intelligence‐Based Virtual Assistant for the Diagnostic Approach of Chronic Ataxias

Alessandro, L., Bianciotti, N., Salama, L., Volmaro, S., Navarrine, V., Ameghino, L., … & Rossi, M. Artificial Intelligence‐Based Virtual Assistant for the Diagnostic Approach of Chronic Ataxias. Movement Disorders. 22 March 2025. doi:10.1002/mds.30168

A non-commercial virtual assistant was developed using advanced algorithms, decision trees, and large language models. In the validation process, 453 clinical cases from the literature were selected from 151 causes of chronic ataxia. The diagnostic accuracy was compared with that of 21 neurologists specializing in movement disorders and GPT-4. Usability regarding time and number of questions needed were also evaluated.

Dietary and lifestyle interventions for the management of hereditary ataxias

Yang Wenyao , Thompson Bruce , Kwa Faith A. A. ;Dietary and lifestyle interventions for the management of hereditary ataxias. Frontiers in Nutrition Volume 12 - 2025. DOI=10.3389/fnut.2025.1548821 

 This review explains recent findings on dietary and nutraceutical interventions, as well as lifestyle modifications such as exercise and rehabilitation programs for HA. It outlines common types of HA, including Friedreich ataxia, spinocerebellar ataxias, ataxia with vitamin E deficiency, ataxia-telangiectasia, and studies on a mixed cohort of patients with HA.

MHRA approves first UK treatment for Friedreich’s ataxia, omaveloxolone

The Medicines and Healthcare products Regulatory Agency (MHRA) has today, 23 April, approved omaveloxolone (Skyclarys), the first treatment for patients aged 16 and over, in the UK for a rare neurodegenerative movement disorder called Friedreich’s ataxia.