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LUMINA Study Update
Lead Author: Lauren Kett, MD, PhD and Sabrina Paganoni, MD, PhD (Co-Presenters)
Published: 2025 Annual NEALS Meeting
Date: October 8, 2025
Poster: Characterizing the CSF Biomarker Signature of Calpain-2 Activity in ALS and the Biomarker Impact of Calpain-2 Inhibition in a Preclinical Model of ALS
Lead Author: Robert Bowser, PhD (Presenter)
Published: 2025 Annual NEALS Meeting
Date: October 8, 2025
Presentation: Characterizing the CSF Biomarker Signature of Calpain-2 Activity in ALS and the Biomarker Impact of Calpain-2 Inhibition in a Preclinical Model of ALS
Lead Author: Robert Bowser, PhD (Presenter)
Published: 2025 Annual NEALS Meeting
Date: October 8, 2025
Update on the Phase 2b Portion of the ORION Trial in Progressive Supranuclear Palsy (PSP)
Lead Author: Anne-Marie Wills, MD (Presenter)
Published: International Congress of Parkinson’s Disease and Movement Disorders 2025
Date: October 7, 2025
Poster: Reduction in Rate of Hypoglycemic Events with Avexitide in Post-Bariatric Hypoglycemia: Results from the Phase 2 and 2B Studies
Lead Author: Marilyn Tan, MD, FACE (Presenter)
Published: Endocrine Society’s Annual Meeting (ENDO 2025)
Date: July 13, 2025
Presentation: Reduction in Rate of Hypoglycemic Events with Avexitide in Post-Bariatric Hypoglycemia: Results from the Phase 2 and 2B Studies
Lead Author: Marilyn Tan, MD, FACE (Presenter)
Published: Endocrine Society’s Annual Meeting (ENDO 2025)
Date: July 13, 2025
Population PK (PopPK) and Pharmacokinetic/Pharmacodynamic (PK/PD) Analysis of Avexitide in Individuals with Post-Bariatric Hypoglycemia
Lead Author: Kelly Fox, MD (Presenter)
Published: Endocrine Society’s Annual Meeting (ENDO 2025)
Date: July 12, 2025
A Phase 1, Multicenter, Randomized, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of AMX0114 in ALS (LUMINA)
Lead Author: Sabrina Paganoni, MD, PhD
Published: The Canadian Neurological Sciences Federation (CNSF) Congress
Date: June 11, 2025
Survival Results from the Global Phase 3 Trial (PHOENIX) Evaluating Sodium Phenylbutyrate and Ursodoxicoltaurine in ALS
Lead Author: Leonard H. van den Berg, MD, PhD (Presenter)
Published: European Network to Cure ALS (ENCALS) 2025 Meeting
Date: June 5, 2025
48-Week Results from the HELIOS Trial: A Phase 2, Open-Label Study Evaluating an Oral, Fixed-Dose Combination of Sodium Phenylbutyrate and Taurursodiol in Wolfram Syndrome
Lead Author: Fumihiko Urano, MD, PhD
Published: Joint Congress of the European Society for Pediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE)
Date: May 12, 2025