Parkinson's gene therapy! 3 years after NBIb-1817 one-time drug: Continuously shorten OFF time/extend ON time, improve sports function!

September 14, 2020 // -- Neurocrine Biosciences and partner Voyager Therapeutics recently presented data from a three-year open label IB-1101 trial to assess the efficacy and safety of the one-time gene therapy NBIb-1817 (VY-AADC) at the 2020 International Conference on Parkinson's Disease and Movement Disorders (MDS).
results showed that in patients with Parkinson's disease, NBIb-1817 single-use treatment showed continuous improvement in motor function, including longer "ON" times without disturbing exercise symptoms, a decrease in the third part of the Unified Parkinson's Disease Assessment Scale (UPDRS) score, and a decrease in parkinson's medication.
specifically, in the PD-1101 trial, NBIb-1817 reduced the average "OFF" time for all three terminal Parkinson's patients by -1.91 hours, the average "ON" time by up to 2.23 hours, and no disturbing movement disorders three years after one-time dosing.
addition, 14 of the 15 patients treated with NBIb-1817 continued to improve in phases after three years, according to the improved Hoehn and Yahr scale.
the new data, as well as two-year data from another open-label IB trial PD-1102, were presented at the MDS Virtual Conference on September 12-16.
(PD) is a chronic, aggressive, debilitating neurodegenerative disease that affects about 6 million people worldwide.
The disease is characterized by the loss of dopamine and the degradation of neurons, accompanied by the loss of the aromatic L-amino acid detoxase (AADC) needed to synthesize dopamine in the brain, leading to damage associated with motor, neuropsychiatric and autonotic nerve function.
is a chemical "messenger" that is produced in the brain and is involved in controlling movement.
AADC enzyme converts lysobar into dopamine.
AADC enzyme decreases as Parkinson's progresses and the L-Doba is converted into dopamine in the brain.
NBIb-1817 is a recombinant adeno-related virus (AAV) serotonin 2 vector in study that encodes the human AADC gene and is designed to help brain cells produce AADC enzymes that convert lysopodoba into dopamine in brain cells.
NBIb-1817 promotes targeted drug use into the brain through in-surgery monitoring and magnetic resonance imaging (MRI).
in 3-year PD-1101 trial data, NBIb-1817 one-time treatment showed an average reduction of -0.15 to -1.91 hours in diary "OFF" time in 15 patients with advanced Parkinson's disease (Baseline: 4.28 to 4.93 hours), with an average increase of 0.26 to 2.23 hours (baseline: 10.32 to 10.46 hours) in the absence of disturbing movement disorders.
NBIb-1817 also showed continuous improvement in motor function after 3 years, with UPDRS Part III stop-and-ed scores, and the entire queue improved by -10.2 to -19.0 points (baseline: 35.8 to 38.2 points), according to the clinician's assessment.
demand for Parkinson's disease drugs was also reduced in patients with Queue 2 and Queue 3 (reduced daily equivalent doses of L-Doba, with averages of -322.0 and -441.2 mg/day, respectively; baselines of 1507.0 and 1477.0 mg/day, respectively).
two-year data from the PD-1102 trial in seven patients showed that NBIb-1817 reduced the diary "OFF" time by an average of -3.2 hours and increased the "ON" time by an average of 2.1 hours (baseline 9.3 hours and 6.6 hours, respectively).
In this study, NBIb-1817 showed continuous improvement in motor function after 2 years, with UPDRS Part III drug suspension score improved to -12.0 points (baseline 34.4) and reduced demand for Parkinson's disease drugs (reduced daily L-Doba equivalent dose, averaging -439.5 mg/day; baseline level 1500.9 mg/day).
safety data from two studies showed that NBIb-1817 was well-to-do and no drug-related serious adverse events (SAE) were studied.
most common adverse reactions are headache, loss of sensation and musculoskeletal pain (PD-1101), as well as upper respiratory tract infections, headaches, nausea and depression (PD-1102). Lead investigator on the
study, Dr. Chad Christine, M.D., a professor of neurology at the Will Institute of Neuroscience at the University of California, San Francisco, said: "Three years later, a single drug given by the one-time gene therapy NBIb-1817 showed that the 'OFF' time in Parkinson's patients continued to shorten and that the 'ON' time improved without disturbing movement disorders and other measurements of motor function.
that the motor function of people with Parkinson's disease is expected to deteriorate within three years makes these results very encouraging.
, the standard of treatment for advanced Parkinson's disease has not changed significantly, and we hope that NBIb-1817 has the potential to become the first gene therapy for Parkinson's disease.
. Eily Roberts, Chief Medical Officer, Neurocrine Biosciences, said, "We are pleased with the results of these studies that suggest that a single NBIb-1817 treatment may help restore the brain's ability to convert lysobar to dopamine.
hope that NBIb-1817 will help patients reduce OFF time, increase "ON" time, and improve exercise symptom control.
we plan to relaunch the enrollment of patients enrolled in the RESTORE-1 clinical trial of NBIb-1817 this year and look forward to further evaluating the application of NBIb-1817 in patients with Parkinson's disease.
Origin: Neurocrine Biosciences and Voyagers Present New Long-Term Three-Year Data Demonstrationing One-Time Treatment with An Investigation The Generapy Showed Sustained In Motor Function in Patients with Parkinson's Disease.