Jupiter Neurosciences, Inc. strategic partnership with Zina Biopharmaceutica

October 22, 2025. The company develops JNS101, which is in Phase II trial for the treatment of Friedreich's Ataxia, a rare inherited disease that causes damage to the nervous system, as well as mobility dysfunctions; and JNS108 that is in Phase II trial for treating mild cognitive impairment/early Alzheimer’s disease. It is also involved in the development of JNS102, which is in Phase II trial for the treatment of mucopolysaccharidosis type 1; and JNS107 that is in Phase II trial for treating mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes syndrome. In addition, the company develops JNS115, which is in Phase IIa trial for the treatment of Parkinson’s disease. It has a strategic partnership with Zina Biopharmaceuticals, LLC to advance Phase 2a clinical trial to evaluate the safety, tolerability, and pharmacokinetics of Resveratrol (JOTROLTM) in individuals with Parkinson’s disease; and with Aquanova AG to develop a series of consumer-focused nutritional products targeting longevity, aging, and healthspan. The company was formerly known as Jupiter Orphan Therapeutics, Inc. and changed its name to Jupiter Neurosciences, Inc. in August 2021. Jupiter Neurosciences, Inc. was incorporated in 2016 and is headquartered in Jupiter, Florida.

Exploring the pleiotropic effects of lncRNA in different repeat expansion disorders

Soumalya Das, Shubhi Khandelwal, Sakshi Shukla, Amit Kumar, Exploring the pleiotropic effects of lncRNA in different repeat expansion disorders, Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, 2025, 168089, ISSN 0925-4439, doi:10.1016/j.bbadis.2025.168089.

A Probabilistic Deep Ensemble Framework for the Objective Assessment of Friedreich Ataxia

Maneesha Randeniya, Kanishka Ranaweera, Thang Ngo, et al. A Probabilistic Deep Ensemble Framework for the Objective Assessment of Friedreich Ataxia. TechRxiv. October 16, 2025. DOI: 10.36227/techrxiv.176062677.75204670/v1

There was strong agreement between the proposed ensemble and clinical mFARS scores, reflected in a Pearson correlation of 0.81, Spearman's correlation of 0.76, R² of 0.46, RMSE of 10.30, and MAE of 8.79 on the unseen test set. Uncertainty was systematically evaluated using Uncertainty Characteristics Curves (UCC), where the ensemble achieved an Area Under UCC (AUUCC) of 8.83.

Chapter 23 - Omaveloxolone: a nuclear factor erythroid 2-related factor 2 activator for Friedreich’s ataxia

Ziquan Zhao, Junjie Wang, Mengchen Lu, Qidong You, Zhengyu Jiang, Chapter 23 - Omaveloxolone: a nuclear factor erythroid 2-related factor 2 activator for Friedreich’s ataxia, Editor(s): Bin Yu, Peng Zhan, Drug Discovery Stories, Volume 2, Elsevier, 2026, Pages 331-344, ISBN 9780443338854, doi:10.1016/B978-0-443-33885-4.00038-X. 

 Nuclear factor erythroid 2-related factor 2 (NRF2) is an important cytoprotective transcription factor that affects the fate of the cell. Impairment of the NRF2 signaling pathway has been regarded as a major contributor to the pathophysiology of FRDA, and targeting NRF2 activation has become an attractive strategy for the treatment of FRDA.

Targeting rare splicing defects: Antisense oligonucleotides offer a therapeutic strategy in FRDA

Targeting rare splicing defects: Antisense oligonucleotides offer a therapeutic strategy in FRDA, Kerkhof, Laurie M.C. et al., Molecular Therapy Nucleic Acids, Volume 36, Issue 4, 102723 doi:10.1016/j.omtn.2025.102723

Using patient-derived cells, the authors demonstrated that antisense oligonucleotides (ASOs) targeting splicing regulatory elements effectively restore splicing deficits and increase frataxin expression. While encouraging in cell-based studies, this strategy is limited to a small subset of individuals with FRDA carrying these rare mutations and requires functional validation in disease-relevant tissues.

Analysis of a Modified Version of the Inventory of Non-Ataxia Signs Over 12 Years in Patients with Friedreich's Ataxia in the EFACTS Study

Lischewski, S.A., Dogan, I., Giunti, P., Parkinson, M.H., Mariotti, C., Durr, A., Ewenczyk, C., Boesch, S., Nachbauer, W., Klopstock, T., Stendel, C., de Rivera Garrido, F.J.R., Schöls, L., Fleszar, Z., Klockgether, T., Grobe-Einsler, M., Giordano, I., Rai, M., Pandolfo, M., Jacobi, H., Hilgers, R.-D., Schulz, J.B., Reetz, K. and the EFACTS Study Group (2025), Analysis of a Modified Version of the Inventory of Non-Ataxia Signs Over 12 Years in Patients with Friedreich's Ataxia in the EFACTS Study. Mov Disord. doi:10.1002/mds.70084

Participants were drawn from the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS). The modified INAS count (presence/absence, 0–16 scale) and modified INAS sum (severity-weighted, 0–84 scale) were evaluated using linear mixed-models and standardized response means (SRMs). Items rare (<5%) and uncharacteristic in Friedreich's ataxia were excluded (chorea, myoclonus, fasciculations, resting tremor, rigidity)

LEXEO Message to the Friedreich Ataxia Community

Message to the Friedreich Ataxia Community. October 7, 2025​.

This morning, we were pleased to share positive interim clinical data for LX2006, our investigational gene therapy, that includes both cardiac and neurological outcome measures. LX2006 (AAVrh10hFXN) is currently being studied in the Lexeo-sponsored SUNRISE-FA Phase 1/2 clinical trial and the Weill Cornell Medicine investigatorinitiated Phase 1A trial for the treatment of Friedreich ataxia cardiomyopathy.

Frataxin depletion leads to decreased soma size and activation of AMPK metabolic pathway in dorsal root ganglia sensory neurons

Frataxin depletion leads to decreased soma size and activation of AMPK metabolic pathway in dorsal root ganglia sensory neurons Olivier Griso, Deepika Mokkachamy Chellapandi, Amelie Weiss, Ioannis Manolaras, Helene Puccio. bioRxiv 2025.10.07.680891; doi:10.1101/2025.10.07.680891 

Our findings uncover AMPK-mTOR dysregulation as a key driver of neuronal growth impairment in FA. This robust neuronal model provides new insights into proprioceptive neuron vulnerability and offers a platform for therapeutic discovery.

Synthesis and Biological Profile of Omaveloxolone: The Cornerstone for Friedreich Ataxia Treatment

Cordaro, M.; Neri, G.; Ansari, S.A.M.K.; Buccheri, R.; Scala, A.; Piperno, A. Synthesis and Biological Profile of Omaveloxolone: The Cornerstone for Friedreich Ataxia Treatment. Int. J. Mol. Sci. 2025, 26, 9747. doi:10.3390/ijms26199747 

This review provides a comprehensive overview of the therapeutic potential of omaveloxone (OMA) for the treatment of Friedreich’s ataxia (FA), along with an analysis of the historical development and current status of the synthetic strategies for OMA production. OMA activates the nuclear factor-2-(erythroid-2)-related (Nrf2) pathway in vitro and in vivo, in both animal models and humans. The Nrf2 pathway plays a crucial role in the cellular response to oxidative stress. Furthermore, OMA has been shown to mitigate mitochondrial dysfunction, restore redox homeostasis and downregulate nuclear factor-κB (NF-κB), a key mediator of inflammatory responses. Through these mechanisms, OMA contributes to tissue protection and inflammation reduction in patients with FA. The review also highlights future perspective, focusing on the challenges associated with OMA reprofiling through innovative drug delivery approaches and its potential repurposing for diseases beyond FA.

Lexeo Therapeutics Stock Rallies On Discussions With FDA To Expedite Friedreich’s Ataxia Drug Approval Process

Published Oct 07, 2025. https://stocktwits.com 

The company stated that data from a planned pivotal study will be pooled with data from the ongoing Phase I/II studies of LX2006 to support an approval application to the U.S. Food and Drug Administration for the therapy. 

 Lexeo Therapeutics (LXEO) announced on Tuesday that the company is considering a smaller pivotal study for LX2006 in the treatment of Friedreich's ataxia (FA) cardiomyopathy, scheduled to begin in the first half of 2026, pending finalization of the study protocol. 

 The data from the planned pivotal study will be pooled with data from the ongoing Phase I/II studies of LX2006 to support an approval application to the U.S. Food and Drug Administration for the therapy, the company stated after discussions with the agency.