-- Intepirdine Program to be Discontinued Based
on MINDSET, HEADWAY, and Gait and Balance Study Results ----
Company to Advance to Larger Confirmatory Nelotanserin DLB
Study Focused on Motor Function and Psychosis ---- Conference
Call Today at 8:00 a.m. EST --
Axovant Sciences (NASDAQ:AXON) today announced that its
investigational drug intepirdine did not meet its primary efficacy
endpoints in the Phase 2b HEADWAY and pilot Phase 2 Gait and
Balance studies. Separately, the Company’s investigational drug
nelotanserin met its prespecified primary endpoint of safety in the
pilot Phase 2 Visual Hallucination study. In addition, in this
exploratory study, nelotanserin treatment resulted in a positive
trend in efficacy in a prespecified intent to treat (ITT) analysis
of the motor function scale, the Unified Parkinson's Disease Rating
Scale (UPDRS) Part III. Nelotanserin treatment also resulted in an
efficacy signal in a post-hoc subset analysis of patients with
higher baseline scores on the Scale for the Assessment of Positive
Symptoms - Parkinson's Disease (SAPS-PD).
In the HEADWAY study of intepirdine in patients with dementia
with Lewy bodies (DLB), neither 35 mg nor 70 mg of intepirdine
resulted in statistically significant improvements after 24 weeks
of treatment compared with placebo-treated patients. In motor
function, as measured by the UPDRS Part III, 35 mg of intepirdine
caused a 2.01 point worsening versus placebo (p=0.158) and 70 mg of
intepirdine caused a 0.74 point improvement versus placebo
(p=0.607). In cognition, as measured by the Alzheimer’s Disease
Assessment Scale-Cognitive Subscale (ADAS-Cog), 35 mg of
intepirdine caused a 0.47 point worsening versus placebo (p=0.653)
and 70 mg of intepirdine caused a 0.67 point improvement versus
placebo (p=0.527). In global function, as measured by the Clinician
Interview-Based Impression of Change plus caregiver interview
(CIBIC+), 35 mg of intepirdine caused a 0.15 point improvement
versus placebo (p=0.395) and 70 mg of intepirdine caused a 0.07
point improvement versus placebo (p=0.701).
Previously defined co-primary endpoints for the HEADWAY study
were the CIBIC+ and a computerized cognitive battery (CCB).
Intepirdine did not achieve statistical significance on the CCB,
with 35 mg of intepirdine resulting in a composite z-score of a
0.38 worsening versus placebo (p=0.452) and 70 mg of intepirdine
resulting in a composite z-score of a 0.36 improvement versus
placebo (p=0.471).
In the study of intepirdine in patients with dementia and gait
impairment, 35 mg of intepirdine did not result in any improvement
in gait speed (1.90 cm per second worsening versus placebo,
p=0.357). Intepirdine was generally well tolerated in these
studies.
In the pilot study of nelotanserin in patients with DLB and
Parkinson’s disease dementia (PDD) who were experiencing visual
hallucinations, the primary endpoint was safety, including an
assessment of symptoms as measured by the UPDRS. On this primary
endpoint, nelotanserin was generally well tolerated. A number of
exploratory efficacy assessments were conducted, including the
UPDRS Part III; the Scale for the Assessment of Positive Symptoms
(SAPS); SAPS-PD; the Patient Global Impression of Change of Visual
Hallucinations (PGIC-VH); and an internally developed patient
diary. In a prespecified ITT analysis, nelotanserin treatment
versus placebo (n=27) resulted in a 3.12 point improvement in the
UPDRS Part III with a positive trend (p=0.075, unadjusted).
Notably, in a prespecified analysis of the DLB patient subset
(n=19), nelotanserin improved the UPDRS Part III by 4.00 points
(p=0.041, unadjusted).
Although nelotanserin did not significantly improve the SAPS-PD
(n=27) in the entire efficacy evaluable population (0.88 point
improvement, p=0.519, unadjusted), in a post-hoc subset analysis of
patients with a baseline SAPS-PD score greater than 8.0 (n=19),
indicating greater severity, nelotanserin treatment at 40 mg for
two weeks followed by 80 mg for two weeks resulted in a 1.21 point
improvement (p=0.011, unadjusted). Further analyses of these data
will be conducted which could yield new insights into the effects
of nelotanserin.
No other trends of improvement were seen on the full SAPS,
PGIC-VH, or in the patient diary.
“Based on the totality of intepirdine data to date, there is no
evidence to support its further development. We are incredibly
disappointed and saddened for the millions of people living with
these difficult conditions, and are deeply grateful to the
patients, caregivers and investigators who participated in our
trials,” said David Hung, M.D., chief executive officer of Axovant.
“For nelotanserin, we are encouraged by the positive trend in motor
improvement observed on the UPDRS, especially in patients with DLB,
and by the efficacy signal in patients with more severe SAPS-PD
scores at baseline, particularly given the short treatment period
at what we believe is the correct therapeutic dose. Therefore, we
intend to discuss a larger confirmatory nelotanserin study with the
FDA that is focused on DLB patients with motor deficits and more
severe baseline psychotic symptoms. Separately, we will be working
with Roivant to build our pipeline to develop other new therapies
for patients with neurological conditions who so desperately need
them.”
“We at Roivant remain fully supportive of Axovant and its
mission,” said Vivek Ramaswamy, founder and chief executive officer
of Roivant Sciences and director of Axovant. “We appreciate the
continued commitment of David and his team to finding and
developing therapies to help patients in need, and Roivant is
equally committed to finding ways to assist Axovant in executing on
attractive strategic options.”
Axovant will work with investigators to appropriately conclude
the HEADWAY and MINDSET extension studies.
About the Intepirdine HEADWAY StudyThis global,
multi-center, randomized, double-blind, placebo-controlled,
parallel-group Phase 2b HEADWAY study evaluated the efficacy,
safety and tolerability of two doses of intepirdine (35 mg and 70
mg) over a 24-week treatment period in 269 patients with DLB.
Individuals receiving stable background therapy for DLB were
allowed to participate in the study. The primary efficacy endpoint
was the change from baseline to week 24 in motor function as
measured by the UPDRS Part III. The co-secondary endpoints were the
change from baseline to week 24 in cognition as measured by the
ADAS-Cog and the change in baseline in global function as measured
by the CIBIC+. Individuals who completed the HEADWAY study were
eligible to receive intepirdine in an extension study.
About the Intepirdine Gait and Balance Study
This multi-center, randomized, double-blind, placebo-controlled,
crossover pilot Phase 2 study evaluated the safety of intepirdine
35 mg daily and its effect on gait and balance in 38 patients with
Alzheimer’s disease, DLB or PDD who were experiencing gait
impairment. All study participants were on stable background
cholinesterase inhibitor therapy for at least six weeks prior to
screening. The study evaluated quantitative measures of gait and
balance that are clinically-relevant predictors of fall risk. The
primary efficacy endpoint was gait speed on an electronic walkway
system after two weeks of treatment. Exploratory endpoints assessed
the effects of intepirdine 35 mg compared with placebo on gait
variability, balance and freezing of gait, using a variety of
standardized tests. With the crossover study design, every patient
received double-blind placebo for two weeks and double-blind
intepirdine for two weeks in a randomized order.
About the Nelotanserin Visual Hallucinations
Study This multi-center, randomized, double-blind,
placebo-controlled, pilot Phase 2 crossover study evaluated the
safety, tolerability, and efficacy of nelotanserin over a four-week
treatment period and enrolled 30 patients with DLB and PDD who were
experiencing frequent and recurrent visual hallucinations. With the
crossover design, every patient received placebo for four weeks and
nelotanserin for four weeks (two weeks of a 40 mg dose followed by
two weeks of an 80 mg dose). Study participants were allowed to be
on stable antipsychotic treatments, stable anti-Parkinson’s disease
treatments, and stable background cholinesterase inhibitor therapy
for at least four weeks prior to screening.
The prespecified primary endpoint of the pilot study was safety
including assessment of extrapyramidal symptoms as measured by the
change in UPDRS scores. In addition, there were multiple
exploratory efficacy assessments in the study that included UPDRS
Part III, SAPS, SAPS-PD, PGIC-VH and an internally developed
patient diary, that evaluated the effects of nelotanserin over a
four-week treatment period. Individuals who completed this study
were eligible to receive nelotanserin in an extension study.
About DLBDementia with Lewy bodies (DLB) is a
progressive neurodegenerative disorder characterized by the
aggregation of Lewy bodies, abnormal deposits of a protein called
alpha-synuclein. Lewy bodies build up in areas of the brain that
regulate behavior, cognition and movement. DLB is the second most
prevalent cause of neurodegenerative dementia in elderly patients.
Approximately 1.1 million patients in the United States have DLB.
Patients with DLB can present with a range of symptoms including
fluctuations in cognition, attention and alertness; Parkinson’s
symptoms; visual hallucinations; and REM sleep behavior disorder
(RBD), in which people physically act out their dreams, impacting
their quality of life and endangering their bed partners. No
therapies are approved for the treatment of DLB in the United
States or Europe.i
About Nelotanserin Nelotanserin is a selective
inverse agonist of the 5-HT2A receptorii that was discovered by
Arena Pharmaceuticals, Inc.
Teleconference/Webcast DetailsTo participate in
the live conference call today, January 8, at 8:00 a.m. EST, please
dial 1-833-652-5918 from the U.S. and Canada or +1
409-767-9227 internationally, and use the passcode 3467829. The
live call is being webcast and can be accessed on the “Events and
Presentations” page of the “Investors” section of the Company’s
website at http://investors.axovant.com. A replay of the webcast
will be available for 30 days following the live event.
About Axovant SciencesAxovant is a
clinical-stage biopharmaceutical company focused on developing and
commercializing innovative medicines to broadly address multiple
forms of dementia and related neurological disorders. Axovant is
developing a pipeline of product candidates that focuses on the
cognitive, functional and behavioral aspects of debilitating
conditions such as Alzheimer's disease, Lewy body dementia and
other neurological disorders. For more information, visit
www.axovant.com.
Forward-Looking Statements This press release
contains forward-looking statements, including statements regarding
Axovant’s plans for the development of nelotanserin, the
discontinuation of intepirdine development, the conclusion of the
HEADWAY and MINDSET extension studies, and the development of its
pipeline. Forward-looking statements can be identified by the words
“believe,” “anticipate,” “continue,” “estimate,” “project,”
“expect,” “plan,” “potential,” “intends,” “will,” “would,” “could,”
“should” or the negative or plural of these words or other similar
expressions that are predictions or indicate future events, trends
or prospects. Forward-looking statements are subject to risks and
uncertainties that could cause actual results to differ materially
and reported results should not be considered as an indication of
future performance. These risks and uncertainties include, but are
not limited to: risks associated with the success, cost and timing
of our product development activities and clinical trials,
increased regulatory requirements, and interim results or other
preliminary analyses do not ensure that later or final results in a
clinical trial or in related or similar clinical trials will
replicate those interim results. There can be no assurance that any
of our product candidates will ever receive regulatory approval or
be successfully commercialized. For a further description of the
risks and uncertainties that could cause actual results to differ
from those expressed in these forward-looking statements, as well
as risks relating to Axovant’s business in general, see the “Risk
Factors” section of our quarterly report on Form 10-Q filed with
the Securities and Exchange Commission (SEC) on November 2, 2017,
and other filings that Axovant makes with the SEC from time to
time. These forward-looking statements are based on information
available to Axovant as of the date of this press release and speak
only as of the date of this release. Axovant disclaims any
obligation to update these forward-looking statements, except as
may be required by law.
Contacts:Investors &
MediaSamina Bari Vice President, Corporate
Affairsinvestors@axovant.com media@axovant.com
917.509.9655
MediaBrian ReidW2O
Groupbreid@w2ogroup.com 402.875.0525
i Alzheimer's Association. Dementia with Lewy bodies.
http://www.alz.org/dementia/dementia-with-lewy-bodies-symptoms.asp.
Accessed January 7, 2018.ii The Journal of Pharmacology and
Experimental Therapeutics. Nelotanserin, a novel selective human
5-hydroxytryptamine2A inverse agonist for the treatment of
insomnia. http://www.ncbi.nlm.nih.gov/pubmed/19841476. 2010
Jan;332(1):281-90. doi: 10.1124/jpet.109.160994. Epub 2009 Oct
19.
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