MELBOURNE, Australia — Neuren Pharmaceuticals Limited today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Phelan-McDermid syndrome (PMS). Significant improvement was observed by both clinicians and caregivers from treatment, across multiple efficacy measures. Improvements were consistently seen across many of the core PMS characteristics....
treatment News
MONTREAL, Québec — Neurenati Therapeutics is delighted to announce the successful closing of its seed funding round, securing 1.2 million $ from Genson Capital, on the 2024 Rare Disease Day. Neurenati, a Québec-based biotech company, focuses on the development of various rare diseases therapies, including pediatric conditions. Combined with the...
SCHLIEREN, Switzerland – AL-S Pharma AG, a biotech company jointly founded by Neurimmune and TVM Capital Life Science, announced today enrollment of the first patient in a multicenter, placebo-controlled Phase 2 clinical trial to evaluate AP-101 in patients with amyotrophic lateral sclerosis (ALS). AP-101 is a human antibody directed against misfolded...
Highlights: Last Open-Label Extension (OLE) study patient successfully completes 12-month treatment period Treatment with NUZ-001 continues to be well-tolerated and shows promising results in extending the life expectancy of patients with MND/ALS Key findings compared to untreated matched controls from the PRO-ACT Historical Database were: NUZ-001 significantly increased survival (χ2=14.1, p=0.00017)...
MELBOURNE, Australia — Neurizon Therapeutics Limited (ASX: NUZ & NUZOA) (“Neurizon” or “the Company”), a clinical-stage biotech company dedicated to advancing treatments for neurodegenerative diseases, is pleased to provide the following update on preparations for entry into the HEALEY ALS Platform Trial, along with encouraging new data showing that NUZ-001 slows the...
NUZ-001 and its active metabolite NUZ-001 Sulfone demonstrated significant neuroprotective effects in a zebrafish model of Huntington’s disease Treatment prevented hallmark developmental and morphological abnormalities, protected against neuronal cell death, restored delayed haemoglobin production, and rescued BDNF expression following Htt protein knockdown Neurizon plans to initiate additional validation studies in mammalian...
Neurizon has successfully developed a new oral liquid formulation of NUZ-001 for the treatment of ALS The liquid formulation is designed to support patients with all stages of ALS, particularly those with swallowing difficulties such as bulbar onset, ensuring broader access to therapy Developed as part of Neurizon’s patient-centric innovation strategy,...
High levels of NUZ-001 and its major active metabolite NUZ-001 Sulfone cross the blood-brain barrier in a rodent PK study In a repeat of the TDP-43 aggregation assay designed to extend the dose-response curve, both compounds significantly prevented the aggregation of TDP-43 in a patient-derived iPSC neuronal model of ALS...
MELBOURNE, Australia — Neurizon® Therapeutics Limited (ASX: NUZ & NUZOA) (“Neurizon” or “the Company”), a clinical-stage biotech company dedicated to advancing innovative treatments for neurodegenerative diseases, is pleased to report positive topline results from the Open-Label Extension (OLE) study of its lead candidate NUZ-001, for the treatment of amyotrophic lateral sclerosis (ALS), the...
MELBOURNE, Australia — Neurizon® Therapeutics Limited (ASX: NUZ & NUZOA; OTCQB: NUZTF) (“Neurizon” or “the Company”), a clinical-stage biotech company dedicated to advancing innovative treatments for neurodegenerative diseases, is pleased to announce that the Sean M. Healey & AMG Center for ALS at Mass General Brigham has received clearance from the...