Axovant Gene Therapies to Present AXO-Lenti-PD Program Update During Virtual Parkinson’s Disease R&D Day on October 30, 202...
29 Outubro 2020 - 5:01PM
Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage
company developing innovative gene therapies, today announced
program updates to be presented as part of its virtual R&D Day
on Friday, October 30, 2020 at 11:30 AM Eastern time, for the
Company’s AXO-Lenti-PD gene therapy for Parkinson’s disease.
Axovant’s Parkinson’s disease R&D Day will be moderated by
Chief R&D Officer, Gavin Corcoran,
M.D., and will feature presentations on the
current treatment landscape, unmet medical need for people living
with Parkinson’s disease, and a summary of data from the second
cohort of the Phase 2 SUNRISE-PD trial for AXO-Lenti-PD.
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.
Dr. Stéphane Palfi, M.D., Ph.D., Professor of Neurosurgery and
Head of the Neurosurgery Department at Henri Mondor Medical Center,
Paris University, commented, "As an investigator in this program
for several years, I am grateful to all the patients and families
who have participated in the study. I am increasingly encouraged by
the emerging body of evidence generated so far in the SUNRISE-PD
study as suggestive of clinically meaningful improvements and
dose-dependent responses following one-time administration of
AXO-Lenti-PD gene therapy. New therapeutic approaches are urgently
needed to address the suffering and disability of patients with
Parkinson’s disease, and I believe AXO-Lenti-PD has the potential
to meaningfully improve their lives."
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. 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.
Forward-Looking Statements
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 "will," "expect," "believe," "estimate,"
and other similar expressions are intended to identify
forward-looking statements. For example, all statements Axovant
makes regarding costs associated with its operating activities are
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 impact of the Covid-19 pandemic on our operations, the
initiation and conduct of preclinical studies and clinical trials;
the availability of data from clinical trials; the scaling up of
manufacturing, the expectations for regulatory submissions and
approvals; the continued development of our gene therapy product
candidates and platforms; 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 August 11, 2020, 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.
Contacts:
Investors
Parag MeswaniAxovant Gene Therapies Ltd.(212)
547-2523investors@axovant.com
Media
Josephine Belluardo, Ph.D. LifeSci Communications(646)
751-4361jo@lifescicomms.commedia@axovant.com
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