Investors & Media Press Releases
Axovant Announces Clinical Updates from AXO-AAV-GM2 and AXO-Lenti-PD Studies
-R&D Day featuring leading physicians and scientists in Parkinson’s disease, GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs disease) will be webcast today at
-Approximately 25% decrease in GM2 ganglioside in CSF observed at end of three-month period in the first child dosed with AXO-AAV-GM2-
-Interim data from additional patients in the AXO-AAV-GM2 program expected in H2 2019-
-First patient expected to be dosed in second cohort of SUNRISE-PD study of AXO-Lenti-PD in Q2 2019-
AXO-AAV-GM2 for Tay-Sachs disease
Axovant recently reported three-month data from an investigator-initiated study administering investigational AXO-AAV-GM2 gene therapy in a patient with advanced infantile Tay-Sachs disease. Additional independent assays and samplings were conducted to further evaluate the biological activity of AXO-AAV-GM2. There was a reduction of approximately 25% in GM2 ganglioside from baseline in the cerebral spinal fluid (CSF). This suggests that the previously announced approximately 3-fold increase from baseline in β-Hexosaminidase A enzyme activity in the CSF is associated with a decrease in GM2 ganglioside, which accumulates in Tay-Sachs disease patients and is believed to cause disease progression.
Interim data from additional patients is expected in the second half of 2019.
AXO-Lenti-PD for Parkinson’s disease
Axovant’s previously reported interim results from the first cohort of the SUNRISE-PD Phase 2 study of AXO-Lenti-PD in Parkinson’s disease patients demonstrated that the product was generally well-tolerated with no serious adverse events reported at the low dose (4.2 x 106 TU). In addition, consistent improvements over the study period were observed across multiple motor function and dyskinesia scales, including improvements on the UPDRS Part III OFF score of 14-points and 36-points, respectively, in the two patients studied in the low-dose cohort. Axovant plans to proceed to the second cohort of the SUNRISE-PD study, at a higher dose of 1.4 x 107 TU, with the first subject expected to be dosed in the second quarter of 2019.
R&D Day Webcast Information
The R&D Day event will be webcast live on
About Axovant Gene Therapies
Axovant, part of the Roivant family of companies, is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases. The company’s current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), Parkinson’s disease, oculopharyngeal muscular dystrophy (OPMD), amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit www.axovant.com.
Roivant Sciences aims to improve health by rapidly delivering innovative medicines and technologies to patients. It does this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. For more information, please visit www.roivant.com.
Forward Looking Statements and Information
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “expect,” “plan,” “anticipate,” “believe,” “intend,” “future,” or “continue” and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the following are forward looking: the success and timing of its ongoing development of AXO-Lenti-PD, AXO-AAV-GM2 and its other product candidates; the anticipated start dates, durations and completion dates of its ongoing and future clinical trials; the anticipated designs of its future clinical studies; and the success of its interactions with the
Source: Axovant Sciences Ltd.