Axovant Announces Positive 12-month Data on AXO-Lenti-PD and Provides Updates Across Gene Therapy Pipeline Programs
January 13 2020 - 6:30AM
Axovant Gene Therapies Ltd. (NASDAQ: AXGT), a clinical-stage
company developing innovative gene therapies for neurological
diseases, today announced recent progress in its gene therapy
programs, including a 12-month update on its Parkinson’s program.
Major 2019 Accomplishments and Upcoming
2020 Milestones
AXO-Lenti-PD gene therapy in Parkinson’s disease:
- At 12 months post-dosing, patients
in the first dose cohort (4.2 x 106 TU) demonstrated an average
22-point change from baseline in motor function as assessed by the
UPDRS Part III “OFF” score, which represents a 37% improvement.
Individual patient improvements were 24-points and 20-points,
respectively. Previously, at 6 months post-dosing, these patients
demonstrated an average 17-point change from baseline, or 29%
improvement, on the same scale. The 12-month timepoint is
considered an important timeframe for assessment of therapeutic
response, differentiation from sham/placebo effect, and durability
of gene therapy in Parkinson’s disease.• AXO-Lenti-PD was observed
to be well-tolerated with no serious adverse events attributable to
the gene therapy.• Improvement in the UPDRS Part III “OFF”
score in the first cohort exhibited evidence of dose response when
compared to the low (n=3), medium (n=6), and high (n=6) dose
cohorts of ProSavin that were previously evaluated in a separate
Phase 1/2 study at 12 months (see Exhibit 1).• The UPDRS Part
II “OFF” score, which assesses activities of daily living, showed a
13-point average change from baseline, or a 44% improvement from
baseline to 12 months.• Only one of two patients in the first
cohort was able to record a Hauser diary. Improvements were
observed across various diary measures from baseline to 12 months
for the single patient.• The PDQ-39 score index, a
well-validated quality of life measure in Parkinson’s disease,
demonstrated an average 15-point change from baseline, or 30%
improvement from baseline to 12 months.
- Enrollment continues in the second
dose cohort of the SUNRISE-PD Phase 2 study of AXO-Lenti-PD. In Q1
2020, Axovant expects to present initial 6-month data from the
first two patients in the second cohort (1.4 x 107 TU) who were
administered a 3-fold higher dose of vector than the first dose
cohort.
- Based on the outcome of the
dose-escalation part of the study and successful development of a
suspension-based manufacturing process, Axovant expects to initiate
the randomized, sham-controlled part of the SUNRISE-PD Phase 2
study by the end of calendar year 2020.
- A recent placebo-controlled study
in a non-human primate model of Parkinson’s disease published in
Molecular Therapy: Methods and Clinical Development (Badin et.al.,
2019) compared two doses of AXO-Lenti-PD against control-group
animals receiving a null vector. The study demonstrated
statistically significant differences in PD clinical response
scores at 6-months in this diseased-animal model (p<0.0002 for
AXO-Lenti-PD vs. control), dose-dependent increases in PET
signaling using a 6-FMT radiotracer (p<0.001 for AXO-Lenti-PD
vs. control), and dose-dependent increases in gene expression for
AADC, TH, and CH1 in transduced striatal tissue.
AXO-AAV-GM1 gene therapy in GM1 gangliosidosis:
- Reported data from the first child
dosed with AXO-AAV-GM1 under an expanded access protocol, which
suggested tolerability and clinical improvement between baseline
and 6-months after vector administration with no reports of serious
adverse events attributable to the gene therapy.
- Continued enrollment in Part A of
the registrational study of late infantile and juvenile onset (Type
II) GM1 patients evaluating safety, tolerability, and exploratory
measures of efficacy. The study remains on track to report data
from Part A in mid-2020.
- Granted orphan drug designation
(ODD) by the U.S. Food and Drug Administration (FDA) in November
2019.
- Axovant co-authored the first
comprehensive retrospective study characterizing the natural
history of Type 1 GM1 gangliosidosis in Molecular Genetics and
Metabolism (Lang et.al., 2019) together with collaborators at the
National Institutes of Health (NIH). This paper describes a rapidly
progressive clinical course of Type 1 GM1 gangliosidosis, in which
almost all patients experience significant multi-organ system
dysfunction and neurodevelopmental regression between 6 and 18
months of age.
AXO-AAV-GM2 gene therapy in Tay-Sachs and Sandhoff diseases:
- In October 2019, at the European
Society of Gene and Cell Therapy (ESGCT) 27th Annual Congress, Dr.
Terry Flotte presented evidence of clinical disease stabilization
in two patients dosed with AXO-AAV-GM2 under an
investigator-initiated IND. Initial observations suggested that
AXO-AAV-GM2 was generally well-tolerated and associated with
improvement in bioactivity outcomes. In addition, data suggested
attainment of normal neurodevelopmental milestones and improvement
in myelination on brain MRI.
- The Company submitted an IND in
late 2019 to support the initiation of Axovant’s registrational
clinical trial in patients with GM2 gangliosidosis. Following the
review of the IND, the FDA did not raise concerns related to animal
toxicology or safety from the investigator-sponsored study. Rather,
due to CMC and device-related questions posed by the FDA beyond the
30-day review period, the Agency placed the IND on clinical hold.
Pending clearance by FDA, Axovant expects to initiate the
AXO-AAV-GM2 trial in 2020.
“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.
About Roivant
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
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.
Media and Investor Contact:
Parag MeswaniAxovant Gene Therapies(212)
547-2523investors@axovant.commedia@axovant.com
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/5e9dccfe-81e4-43e4-9d18-2f5e49111e72
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