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Unassigned

New Medicines

ProSavin Treatment of late-stage Parkinson's disease

Information

ProSavin
New molecular entity
Sio Gene Therapies
Sio Gene Therapies

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
None
Nov 20Sio announces that, based on new information received from its manufacturing partner, Oxford Biomedica in mid-October regarding delays in CMC data and third-party fill/finish issues, the development of a suspension-based manufacturing process for AXO-Lenti-PD will take longer than expected. As a result, Sio believes that it is unlikely that its planned randomised, sham-controlled trial of AXO-Lenti-PD will enrol patients by the end of calendar year 2021. Manufacturing of several GMP batches is now underway and planned at Oxford Biomedica with a goal of generating material for use in future clinical trials as soon as possible. The Company expects to provide an update in Q1 21 or as program timelines are clarified [7].
Jul 20Oxford Biomedica enters into clinical supply agreement with a subsidiary of Axovant Gene Therapies (now Sio Gene Therapies), for AXO-Lenti-PD. Under the terms of the agreement, Oxford Biomedica will manufacture GMP batches of AXO-Lenti-PD for Axovant to support the ongoing and future clinical development of the gene therapy [6].
Dec 18Positive feedback from a meeting with the US FDA which reaffirmed the SUNRISE-PD clinical study to be a continuation of the previous ProSavin program and a part of a single development program.[2]
Jun 18Oxford BioMedica enters into an agreement with Axovant Sciences, wherein the latter in-licensed exclusive worldwide rights for development and commercialisation of OXO 102 (also known as AXO Lenti PD) [6].

Category

LentiVector® delivery system which delivers the three genes required for dopamine synthesis, AADC, TH and CH1 to neurons and glial cells that do not normally produce this neurotransmitter, thus creating new dopamine-producing cells.[2,3]
Parkinson's disease is the second most common neurodegenerative disorder after Alzheimers disease. Typically develops between the ages of 55 and 65 years and occurs in 1-2% of people over the age of 60 years, rising to 3.5% at age 85-89 years. About 0.3% of the general population is affected, and the prevalence is higher among men than women, with a ratio of 1.5 to 1 [1].
Treatment of late-stage Parkinson's disease
Intrastriatal
Parenteral

Trial or other data

Dec 20PI/II SUNRISE-PD trial is due to complete collection of primary outcome data in Jun 22 [7].
Oct 20Sio Gene Therapies intends to initiate the sham-controlled part of the SUNRISE-PD trial in 2021. Updated efficacy and safety results released show AXO-Lenti-PD was well-tolerated with no treatment-related serious adverse events at 6 months. 21-point mean improvement in UPDRS Part III “OFF” score, a 40% improvement from baseline based on the two evaluable patients in the study, exceeding predefined criteria for success. Greater than 2-hour improvement from baseline in both diary “good ON time” and diary OFF time assessments [6].
Apr 20Axovant (now called Sio Gene Therapies) completes dosing of all patients in second-cohort of SUNRISE-PD trial [6].
Jan 20UK trial sites Cambridge, London [5]
Mar 19Positive interim results from the first cohort of 2 pts with advanced PD in PII SUNRISE-PD trial. They both received a single administration of the lowest dose of the therapy. It was well-tolerated and no serious adverse events were observed. At 3 months after receiving the therapy, improvements of an average of 55% from baseline were observed, based on the UPDRS Total OFF score. The company now plan to advance to higher dose cohorts [4]
Feb 19Axovant has dosed two patients in the SUNRISE-PD PI/II trial.[2]
Nov 18Axovant dosed the second pt in the SUNRISE-PD PI/II trial of OXB 102, in the UK (OXB-102-01; NCT03720418). The two part trial is designed to evaluate safety and tolerability, as well as efficacy data including standard measures of motor function in PD pts. Part A is an open-label dose-escalation phase in which pts are enrolled in cohorts and will receive one of three escalating doses of AXO-Lenti-PD. Part B is a randomised, double-blind phase in which pts will be randomiaed to an active group receiving the selected dose from Part A, or to a control group receiving an imitation surgical procedure. The trial intends to enrol approximately 30 pts in France and the UK. The trial may expand to the US during the part-B portion. Primary outcome measures are based on safety; as measured by incidence of treatment emergent adverse events and serious adverse events and by clinical laboratory analysis, vital signs, physical examination and changes in brain MRI findings. Secondary outcome measures are changes after 6 months iin; Unified Parkinson´s Disease Rating Scale (UPDRS) scores, motor fluctuations and dyskinesia rating scale all vs. baseline. The trial currently has an estimated primary completion date of June 2022 [2,3].