An Open-Label, Single Dose Study to Assess the Breast Milk Pharmacokinetics of Omaveloxolone in Healthy Lactating Women

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026​. Poster Number: 316 T. An Open-Label, Single Dose Study to Assess the Breast Milk Pharmacokinetics of Omaveloxolone in Healthy Lactating Women. Hamim Zahir, PhD, Biogen, Inc., Lucy Wu, PhD, Biogen, Inc., Susie Sinks, Biogen, Inc., Andre Arizpe, PharmD, Biogen, Inc., Sarah Chambers Gurson, Biogen, Inc., Konstantine Skordos, Biogen, Inc.

Objective: To determine the relative estimated infant exposure of omaveloxolone through breast milk following a single dose of 150 mg in healthy lactating women.

Conclusions: The results indicate that after oral administration of a single dose of 150 mg omaveloxolone, breast milk–fed infants may be exposed to a small fraction.(<1%) of the adult dose. The safety findings observed in this study are consistent with the known safety profile for omaveloxolone.

Diagnoses and Procedures Observed Prior to Friedreich Ataxia Diagnosis: A Real-World US Medical Claims Analysis

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Poster Number: 291 T. Diagnoses and Procedures Observed Prior to Friedreich Ataxia Diagnosis: A Real-World US Medical Claims Analysis. Alicia Henriquez, MD, Seattle Children's Hospital, Meredith Hatcher, Texas Movement Disorder Specialists, Georgetown, TX, USA, Sarah Doss, University of Nebraska Medical Center, Omaha, NE, USA, Partha S. Ghosh, MD, Boston Children's Hospital, Pravin Khemani, MD, Swedish Neuroscience Institute, Seattle, WA, USA, Sarah M. England, PhD, Biogen, Inc., Queeny Ip, Komodo Health, Xinshuo Ma, Komodo Health, Saila Chen, Komodo Health, Robin Avila, PhD, Biogen, Alexandra DiDonato, PharmD, Biogen. 

Objective: To characterize diagnoses and procedures received by patients prior to diagnosis of FA. 

Conclusions: Medical claims data enhance our understanding of the clinical journey of patients leading to FA diagnosis. Prior to FA diagnosis, patients encounter various healthcare professionals, receive a variety of diagnoses, and experience a multitude of procedures, which may lead to delayed time to definitive FA diagnosis. Expediting the diagnostic pathway for FA through timely referrals enables earlier intervention, which is imperative for reducing disease burden and enhancing patient quality of life.

High prevalence of pathogenic expanded composite repeats in Friedreich ataxia: Need for a more accurate diagnostic testing strategy

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026​. Poster Number: 295 T. High prevalence of pathogenic expanded composite repeats in Friedreich ataxia: Need for a more accurate diagnostic testing strategy. Sanjay Bidichandani, MBBS, PhD, University of Oklahoma, Morgan Devore, MS, University of Oklahoma, Christina Lam, BS, University of Oklahoma, David Lynch, MD, PhD, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA. High prevalence of pathogenic expanded composite repeats in Friedreich ataxia: Need for a more accurate diagnostic testing strategy. 

Objectives: To determine the true prevalence and variety of pathogenic composite alleles in FRDA by characterizing the precise sequence of the expanded alleles in a large prospective series of patients. To overcome the expense and unwieldy nature of whole genome longread sequencing by developing a robust, logistically-feasible, and cost-effective testing strategy to accurately detect and characterize these missing pathogenic alleles in FRDA.

US Medical Claims Database of Patients With Friedreich Ataxia Prescribed Omaveloxolone: Clinical Events From Diagnosis to Initiation of Treatment

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Poster Number: 296 T. US Medical Claims Database of Patients With Friedreich Ataxia Prescribed Omaveloxolone: Clinical Events From Diagnosis to Initiation of Treatment. Sheng-Han Kuo, PhD, Columbia University Medical Center, New York, NY, USA, Liana S. Rosenthal, MD, PhD, Johns Hopkins University School of Medicine, Boyang Bian, PhD, Biogen, Inc., Alexandra DiDonato, PharmD, Biogen, Sarah M. England, PhD, Biogen, Inc., Daniel Gomes, Voxanalytica, Nicholas D’Alberto, Biogen, Inc., James McKay, PhD, Biogen, Inc., Tony Wang, PhD, Voxanalytica, Robin Avila, PhD, Biogen. 

Objective: To describe clinical events and patient profiles in individuals with FA prior to omaveloxolone initiation using US medical claims.

Conclusions: These findings highlight that patients with FA experienced progressive worsening of symptoms, including bulbar, respiratory, and mobility impairments, between initial diagnosis and initiation of omaveloxolone. This underscores the importance of identifying decision points that should prompt earlier treatment intervention.

Long-Term Safety and Tolerability of Omaveloxolone in Patients With Friedreich Ataxia: Up to 6-Year Data From the MOXIe Open-Label Extension

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026​.Poster Number: 298 T. Long-Term Safety and Tolerability of Omaveloxolone in Patients With Friedreich Ataxia: Up to 6-Year Data From the MOXIe Open-Label Extension. Theresa Zesiewicz, MD, MD, University of South Florida Ataxia Research Center, Tampa, Florida, USA, Susan Perlman, MD, University of California, Los Angeles, Martin B. Delatycki, Victorian Clinical Genetics Services, Murdoch Children’s Research Institute, J. Chad Hoyle, Ohio State University College of Medicine, Sylvia Boesch, MD, Medical University of Innsbruck, Wolfgang Nachbauer, MD, PhD, Medical University of Innsbruck, Paola Giunti, MD, PhD, University College Hospital, George Wilmot, MD, PhD, Emory University School of Medicine, SH Subramony, MD, University of Florida Health, Katherine Mathews, MD, University of Iowa, Iowa City, Iowa, USA, Syed Farooq, Biogen International GmbH, Shobhana Natarajan, PhD, Biogen, Inc., Rose M. Domingo-Horne, MD, Biogen, Inc., Andre Arizpe, PharmD, Biogen, Inc., Jonathan Smith, MSc, Biogen Idec Ltd, Nicolas Folschweiller, Biogen, David Lynch, MD, PhD, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA. 

Objective: To evaluate the long-term safety and tolerability of omaveloxolone treatment in the MOXIe OLE.Conclusions: This updated analysis of the MOXIe OLE provides up to 6 years of continuous long-term safety and tolerability data regarding omaveloxolone use in patients with FA. 

Safety findings were consistent with the previously known safety profile.

Assessing Real-World Experiences on SKYCLARYS® (Omaveloxolone) (ARIES) in Patients With Friedreich Ataxia: An Observational Study

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026​. Poster Number: 292 T. Assessing Real-World Experiences on SKYCLARYS® (Omaveloxolone) (ARIES) in Patients With Friedreich Ataxia: An Observational Study. Elissa Hult, Biogen, Inc., Sarah M. England, PhD, Biogen, Inc., Boyang Bian, PhD, Biogen, Inc., James McKay, PhD, Biogen, Inc., Tami Sova, Biogen, Inc., Molly Scannell Bryan, PicnicHealth, Robin Avila, PhD, Biogen. 

Objective: To understand omaveloxolone treatment experience through PROs, the associated longitudinal clinical outcomes, and HCRU in the US real-world setting. 

Conclusions: This currently enrolling study will provide a means to longitudinally follow patients with FA treated with omaveloxolone in real-world clinical practice without the need for clinical sites. The findings will increase understanding of the real-world experience with omaveloxolone use in patients underrepresented in clinical trials and the impact on long-term patient experience and outcomes.

Interim Analysis of mFARS Trajectories Across 5 Years in the MOXIe Extension: Impact of Omaveloxolone Initiation Timing in Friedreich Ataxia

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026, Poster Number: 297 T. Interim Analysis of mFARS Trajectories Across 5 Years in the MOXIe Extension: Impact of Omaveloxolone Initiation Timing in Friedreich Ataxia. Theresa Zesiewicz, MD, MD, University of South Florida Ataxia Research Center, Tampa, Florida, USA, David Lynch, MD, PhD, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA, Claudia Yang Santos, PhD, Biogen, Inc., Susie Sinks, Biogen, Inc., Syed Farooq, Biogen International GmbH, Jonathan Smith, MSc, Biogen Idec Ltd, Shobhana Natarajan, PhD, Biogen, Inc., Nicolas Folschweiller, Biogen. 

Objective: Compare mFARS score trajectories between patients who initially received omaveloxolone in MOXIe Part 2 and patients who started omaveloxolone 1 year later in the OLE. 

Conclusions: The omaveloxolone-omaveloxolone group that started treatment 1 year before the placebo-omaveloxolone group showed numerically slower mFARS progression, relatively. Nevertheless, all patients receiving omaveloxolone experienced sustained benefit in slowing of mFARS decline throughout the analysis period.

Efficacy and Safety of a Novel Investigational AAV FXN Gene Therapy (SGT-212) for the Treatment of Friedreich’s Ataxia

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026​. Poster Number: 299 T. Efficacy and Safety of a Novel Investigational AAV FXN Gene Therapy (SGT-212) for the Treatment of Friedreich’s Ataxia. Matthew Harmelink, MD, Solid Biosciences, Brandon Chan, PhD, Solid Biosciences, Jun Lee, PhD, Solid Biosciences, Jessica Boehler, PhD, Solid Biosciences, Jamie Marshall, PhD, Solid Biosciences, Gourav Choudhury, PhD, DABT, Franklin Labs, Heather Born, PhD, GEMMA Biotherapeutics, Juliette Hordeaux, DVM, PhD, DECVP, GemmaBio, James Wilson, MD, PhD, GemmaBio, Jessie Hanrahan, PhD, Solid Biosciences, Gabriel Brooks, MD, Solid Biosciences, Nicholas Christoforou, PhD, Solid Biosciences. 

Approach: SGT-212, Solid Biosciences’ investigational AAV gene therapy for FA, expresses full-length, human FXN under a ubiquitous promoter. Preclinical evaluation of SGT-212 assessed neurologic and cardiac outcomes in conditional Fxn knockout mouse models targeting the nervous system (nKO) and heart (cKO). Long-term safety and biodistribution were assessed in non-human primates (NHPs) to support a first-in-human clinical trial using a dual route of administration via intraparenchymal dentate nucleus (IDN) and intravenous (IV) infusions. This multisystem, dual route approach was designed to target the neurologic, cardiac and systemic manifestations of FA.

Conclusions: These nonclinical studies demonstrate that a one-time administration of SGT-212 increases FXN expression in disease-relevant tissues, improves neurologic and cardiac phenotypes in mouse models, and is well tolerated in NHPs. Altogether, this positive, nonclinical data package supports the Phase 1b FALCON trial (NCT07180355), a first-in-human evaluation of SGT-212, which is actively screening participants.

Interim observations from a long-term open label study evaluating daily nomlabofusp administration in patients with Friedreich’s ataxia

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Poster Number: 419 O. Russell Clayton, DO, Larimar Therapeutics Inc., Erin Coyle, Larimar Therapeutics Inc., Flavia De Toni, PhD, Larimar Therapeutics Inc., Mohamed Hamdani, Larimar Therapeutics Inc. 

Objective: Evaluate interim observations related to FXN levels, clinical measures, and safety in patients with FA receiving long term administration of nomlabofusp. 

Conclusion: In patients with FA, daily administration of nomlabofusp for 6 months resulted in increased skin FXN to levels that were within the range expected in asymptomatic carriers with no phenotypic expression of disease. After 1 year of nomlabofusp treatment, values from clinical measures trended lower, suggesting a potential for clinical improvement in the context of increased FXN levels. There is a risk of anaphylaxis during early treatment, particularly in patients who had past exposure to nomlabofusp. Long term treatment with nomlabofusp appeared to be well tolerated.

Unmet Needs of Individuals With Friedreich Ataxia Cardiomyopathy: Insights From the Lexeo Friedreich Ataxia Cardiac Advisory Council

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Poster Number: 293 T. Cardiomyopathy: Insights From the Lexeo Friedreich Ataxia Cardiac Advisory Council. Joslyn Crowe, MSW, MA, Lexeo Therapeutics, Aly Bourbeau, Lexeo FA Cardiac Advisory Council, Marc Likins, Lexeo FA Cardiac Advisory Council, Amalia Maranhao, Lexeo FA Cardiac Advisory Council, Madelyn Raynsford, Lexeo FA Cardiac Advisory Council, Nilomi Shah, PharmD, Lexeo Therapeutics. 

 The FA Cardiac Advisory Council meeting aimed to understand crucial gaps and shared goals related to heart health, entry points in cardiac care and diagnostic challenges. A pre-meeting survey was completed by 10 council members. 

 Conclusions: The FA Cardiac Advisory Council identified key collaborative opportunities to increase knowledge around FA-CM and improve cardiac care by: sharing insights and stories on their lived experiences with FA-CM, developing materials to better inform newly diagnosed individuals with FA, building a community for those impacted by FA-CM via digital and social media channels, and providing on clinical trial experience.