Axovant Gene Therapies to Present AXO-Lenti-PD Program Update During Virtual Parkinson’s Disease R&D Day on October 30, 2020
- Patient-level data in cohort 2 of SUNRISE-PD study demonstrates consistent, clinically meaningful outcomes and evidence of dose response
- Delays in manufacturing process expected to postpone the start of enrollment in randomized, controlled study
- Virtual R&D Day webcast on
October 30th at 11:30 AM Eastern time
Axovant’s Parkinson’s disease R&D Day will be moderated by Chief R&D Officer,
AXO-Lenti-PD Program Updates
- Summary of available individual patient-data from the second dose cohort at the 6-month timepoint following one-time dosing with AXO-Lenti-PD gene therapy demonstrate consistent treatment outcomes including:
- Favorable safety and tolerability profile, with no serious adverse events attributable to gene therapy
- Improvement in Hauser diary “Good ON time” and “OFF time” changes from baseline for all 4 patients
- Improvement in UPDRS Part II and Part III “OFF” score in 2 evaluable patients
- Reduction in Levodopa-equivalent daily dose (LEDD) from baseline
- 23-point improvement from baseline to 12 months in UPDRS Part III “OFF” score observed for the first patient in cohort 2 who has reached the 12-month evaluation, which was performed as a remote assessment.
- Based on new information received from our 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, the Company believes that it is unlikely that its planned randomized, sham-controlled trial of AXO-Lenti-PD will enroll 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 on program timelines in the first quarter of 2021 or as program timelines are clarified.
R&D Day Information
- To register for the R&D webcast, please click here.
- An 8-K with a copy of the R&D Day presentation has been filed with the
Securities and Exchange Commission(SEC).
- A live audio webcast of the R&D Day can be accessed through the Events & Presentations section of the company's website at investors.axovant.com. An archived replay of the webcast will be available on the company's website following the event.
About Axovant Gene Therapies
Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinson’s disease.
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Axovant Gene Therapies Ltd.