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.

Dexterity Outcomes in Friedreich Ataxia as Measured by mFARS and FA-ADL

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Poster Number: 294 T. Dexterity Outcomes in Friedreich Ataxia as Measured by mFARS and FA-ADL. Sheng-Han Kuo, PhD, Columbia University Medical Center, New York, NY, USA, Syed Farooq, Biogen International GmbH, Claudia Yang Santos, PhD, Biogen, Inc., Shobhana Natarajan, PhD, Biogen, Inc., Jonathan Smith, MSc, Biogen Idec Ltd, Richard Lawson, MSc, Biogen. 

Objective: To assess the impact of Friedreich ataxia (FA) on the activities of daily living associated with dexterity at different levels of disease severity.

Conclusions: Dexterity outcomes measured by FA-ADL have a strong association with mFARS score, suggesting that mFARS can also capture dexterity progression in FA.

Patient-reported experiences during long-term omaveloxolone treatment in Friedreich ataxia: survey design and planned qualitative analysis

MDA Clinic & Scientific Conference 2026. March 8 – 11, 2026. Patient-reported experiences during long-term omaveloxolone treatment in Friedreich ataxia: survey design and planned qualitative analysis. Susan Perlman, MD, University of California, Los Angeles, Theresa Zesiewicz, MD, MD, University of South Florida, Matthew Lafleur, MA, Bionews, Inc., Pensacola, FL, USA, Libby Schwers, BA, With Love, Libby, Des Moines, Iowa, USA, Jennifer Farmer, MS, Friedreich's Ataxia Research Alliance, Pearl WU, MS, Biogen, Claudia Yang Santos, PhD, Biogen, Inc., Kyle Fowler, MBiolSci, Syneos Health Consulting, Zurich, Switzerland, Fiona Thomas, MBChB, Syneos Health Consulting, London, UK, David Lynch, MD, PhD, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA. 

This qualitative survey will explore patient-reported lived experiences and perceptions of functional outcomes among adults (≥18 years) with Friedreich Ataxia (FA) receiving long-term omaveloxolone treatment in the United States.