Major 2019 Accomplishments and Upcoming 2020 Milestones
AXO-Lenti-PD gene therapy in Parkinson's disease:
AXO-AAV-GM1 gene therapy in GM1 gangliosidosis:
AXO-AAV-GM2 gene therapy in Tay-Sachs and Sandhoff diseases:
"In 2019, Axovant advanced its vision of creating a new genetic medicines company around an experienced leadership team, innovative gene therapy pipeline, and patient-focused mission. We are unique in having three promising clinical-stage gene therapy programs focused on life-threatening neurodegenerative diseases," said Pavan Cheruvu, M.D., Chief Executive Officer of Axovant. "I am particularly excited to share a new 12-month data update on safety and efficacy from the first dose cohort of our Parkinson's disease program, which highlights the potential for AXO-Lenti-PD gene therapy to produce both durable and clinically meaningful improvements over the currently available standard of care in Parkinson's disease. We believe that a 12-month evaluation is a key regulatory timepoint in the assessment of treatment durability and separation from placebo effect. In the first quarter of 2020, we expect to present 6-month data from two patients in the second cohort treated with a higher dose of AXO-Lenti-PD. Axovant is committed to advancing each of our gene therapy programs with a sense of urgency on behalf of our patient communities. We believe we are well-positioned to deliver on the promise of these programs for patients and families affected by Parkinson's disease, GM1 gangliosidosis, and Tay-Sachs and Sandhoff diseases."
About Axovant Gene Therapies
Axovant Gene Therapies, 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 neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), and Parkinson's disease. 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.
In 2018, Axovant licensed exclusive worldwide rights from the University of Massachusetts Medical School (UMMS) for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.
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.
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," "would," "should," "expect," "believe," "estimate," and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the initiation, timing, progress, and reporting of results of its preclinical programs, clinical trials, and research and development programs; costs associated with its operating activities; its ability to advance its gene therapy product candidates into and successfully initiate, enroll, and complete clinical trials; the potential clinical utility of its product candidates; its ability to continue to develop its gene therapy platforms; its ability to develop and manufacture its products and successfully transition manufacturing processes; its ability to perform under existing collaborations with, among others, Oxford BioMedica, the University of Massachusetts Medical School, and the National Institutes of Health (NIH); and the timing and likelihood of its regulatory filings and agency interactions to advance its gene therapy programs and clinical studies, are all forward-looking. All forward-looking statements are based on estimates and assumptions by Axovant's management that, although Axovant believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Axovant expected. Such risks and uncertainties include, among others, the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; interactions and submissions with regulatory agencies; Axovant's scientific approach and general development progress; and the availability or commercial potential of Axovant's product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Axovant's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 8, 2019, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.
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