Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, is
presenting two posters this week on the health burden and
diagnostic challenges of Fragile X syndrome (FXS). These data are
being presented at the American Society for Experimental
Neurotherapeutics (ASENT) 2020 Meeting, which is being held in
Bethesda, MD on March 2nd through March 5th, 2020.
The first poster presents health state utility
indices that estimate the severity of pediatric disorders including
FXS, and the potential benefit of Zygel (CBD transdermal gel;
ZYN002) in children and adolescents with FXS. Joseph M. Palumbo,
MD, FAPA, MACPsych, Chief Medical Officer of Zynerba, will also
present these data during the ASENT Pipeline Data Blitz on March 4.
The second poster speaks to the initial family experience in FXS,
expanding upon the existing knowledge of patient presentation,
diagnosis and understanding of FXS; the protracted journey to
diagnosis; and the high prevalence of comorbid conditions. A copy
of the posters are available on the Zynerba corporate website at
http://zynerba.com/publications/.
Post Hoc Analysis - An Open-Label Study of
Transdermal Cannabidiol (ZYN002) for the Treatment of Fragile X
Syndrome in Children and Adolescents: Estimating Health State
Utility Scores
- Poster number: 29
- Poster presentation time: Tuesday, March 3 from 5:00 to 6:00 PM
and Wednesday, March 4 from 5:00 to 7:00 PM.
- Oral presentation time: Wednesday, March 4 from 1:00 to 3:00 PM
during the ASENT Pipeline Data Blitz session.
FXS is a rare genetic condition characterized by a range of
developmental, neuropsychiatric, and behavioral symptoms. The
spectrum and severity of FXS symptoms result in a high clinical,
humanistic, and economic burden on patients and caregivers,
including important healthcare resource utilization and associated
costs. Health state utility indices (HUI) are used in clinical and
economic analyses of therapies with potential impact on
health-related quality of life (HRQoL) and enable comparison of
HRQoL across conditions. Health state utility is measured on a 0 to
1 scale in which 0 represents death and 1 represents complete
health; the lower the score, the more significant the impact of the
disease to HRQoL. The Aberrant Behavior Checklist - Community
Utility Index (ABC-UI) - a utility index specific to FXS - was
derived from the Aberrant Behavior Checklist - Community for FXS
(ABC-CFXS) to measure the HRQoL benefit of treatments for FXS. The
ABC-UI, created and subsequently published in the peer-reviewed
journal Quality of Life Research, in 2015, established an algorithm
that calculates utility index score based on ABC-CFXS items
pertaining to the core symptom domains of FXS.
“The mean health state utility index score for FXS in this
seminal analysis was calculated to be 0.57, estimating a
significant disease-related impact on HRQoL in FXS which may be as
robust as, or perhaps even more impactful, than that described in
the published literature for other debilitating pediatric
conditions in measures of HUI”, said Dr. Palumbo. “We will work to
confirm our initial observations in future analyses.”
The objective of the analysis undertaken was to evaluate the
potential benefit of Zygel on the ABC-UI in pediatric and
adolescent patients with FXS through post hoc analysis of data from
the Phase 2 open label FAB-C (Treatment of Fragile
X Syndrome Anxiety and Behavioral
Challenges with CBD) trial. Individual
patient-level data from the FAB-C study were mapped to the ABC-UI
algorithm to generate a utility index score for each patient.
“Fragile X syndrome is a debilitating diagnosis, and for the
first time we have estimated the health state utility index scores
of patients with FXS, in the context of the published HUI
literature, helping to clarify the significance of this
disorder,” continued Dr. Palumbo. “Further, we observed
statistically significant improvements in the health state utility
index scores of patients treated with Zygel in the 12-week
exploratory Phase 2 FAB-C trial compared to baseline, suggesting a
potential broad spectrum of benefit of the drug in the important
domains of the ABC-CFXS that were incorporated into the utility
index.”
Improvement in ABC-UI Score with Zygel
Treatment
As shown in Figure 1 below, compared to their baseline scores,
patients on Zygel experienced significant (P < 0.01) improvement
in their mean ABC-UI beginning at week 4 and this improvement was
maintained through weeks 8 and 12.
Figure 1. Mean ABC-UI Score at Each Timepoint
during Treatment with Zygel (*P<0.01) is available
at https://www.globenewswire.com/NewsRoom/AttachmentNg/3deced17-5fcf-46db-ab60-b87b5cad47c6
The authors of the poster concluded that:
- Treatment with Zygel significantly improved health state
utility index scores in pediatric and adolescent patients with FXS,
suggesting a potential broad spectrum of benefit of Zygel in the
important domains of the ABC-CFXS that were incorporated into the
utility index;
- The correlation of the ABC-UI scores with Clinical Global
Impression of Severity (CGI-S) scores in these patients suggests
that the ABC-UI appropriately reflects symptom severity in FXS;
and
- The estimated health state utility index score of 0.57 in
patients with FXS enrolled in FAB-C appear to describe a poor
baseline level of HRQoL, despite standard of care, highlighting the
considerable impact of FXS symptoms on patient HRQoL.
Fragile X Syndrome Diagnosis and Patient Journey: The
Caregiver’s Perspective
- Poster number: 28
- Poster presentation time: Tuesday, March 3 from 5:00 to 6:00 PM
and Wednesday, March 4 from 5:00 to 7:00 PM.
“Children with Fragile X syndrome generally remain undiagnosed
until approximately three years of age, and the path to diagnosis
is complex,” said Dr. Palumbo. “These children also have a
high prevalence of comorbid conditions which complicates the
diagnosis. Today’s standard of care includes counseling and therapy
and the use of traditional prescription medications that aren’t
specifically indicated for FXS. This information supports the
importance of testing for the disorder early in the diagnostic
journey and bringing novel treatments that specifically treat the
behavioral symptoms of FXS through the FDA approval process for the
benefit of patients and their families.”
Core FXS clinical symptoms include social avoidance/withdrawal,
anxiety, irritability, deficits in learning and cognition, and
sleep difficulties. These symptoms are frequently compounded by
comorbid conditions, including autism and
attention-deficit/hyperactivity disorder. Parents often first
recognize the initial symptoms and developmental delays, leading to
subsequent clinical diagnosis of FXS utilizing genetic testing for
mutations in the FMR1 gene. Early diagnosis of FXS is important to
facilitate treatment and coordinate the multidisciplinary
supportive care and educational interventions required to manage
the symptoms of FXS. Unfortunately, diagnosis of FXS is often
delayed. A 2008 study reported a delay of 24 to 26 months between
initial symptoms and diagnosis, and mean age at diagnosis has
remained delayed over time (32 months in 2018 vs 38 months in
2008).
Zynerba utilized a 30-minute, anonymized, quantitative online
survey, conducted in the United States from May 3 to June 12, 2019,
to characterize the patient journey in FXS surrounding diagnosis
and clinical experiences. Thirty-five (35) predominantly female
(80%) primary caregivers of children with FXS completed the survey.
The children of these caregivers were 3 to 17 years of age, had a
full FMR1 mutation, and exhibited socially avoidant behaviors.
Path to Diagnosis
The mean age of children with FXS at the time of diagnosis was
36 months. When asked to rate the top 3 factors prompting
caregivers to schedule an initial visit with a physician, the most
common were:
- Cognitive/intellectual developmental delays;
- Issues with speech and/or motor skills; and
- Social avoidance/social unresponsiveness.
Most caregivers scheduled an initial visit with a physician
within 6 months of noticing symptoms (82.9%). The first physician
seen was usually a primary care physician (family doctor or general
pediatrician, 71.4%), but formal diagnosis of FXS was most often
made by a specialist (80.0%), most frequently geneticists and
neurologists/pediatric neurologists.
Current Experience with FXS
As shown in Figure 2 below, the most frequently experienced
current symptoms of FXS were social avoidance and attention
challenges. Most caregivers (85.7%) rated the severity of FXS at
the time of the survey as severe (45.7%) or moderate (40.0%).
Figure 2. Current FXS Symptoms is available
at: https://www.globenewswire.com/NewsRoom/AttachmentNg/da488eea-61ea-45b1-b5f0-6d98870d0423
Children of the surveyed caregivers were receiving a mean 1.94
treatments. Seventy-seven (77%) of children were currently
receiving counseling/therapy; 46% were receiving traditional
prescription treatment; and 31.4% were receiving nonprescription
treatment/supplements. The most commonly received prescription
medications were antidepressants/selective serotonin reuptake
inhibitors (SSRIs) and stimulants. In addition, 77% of the children
were reported to have comorbid conditions, the most common being
autism spectrum disorder (66%), attention-deficit/hyperactivity
disorder (26%), and sleep disorders (20%).The authors concluded
that:
- The results of this survey expanded upon existing knowledge of
the initial presentation/diagnosis and experience of FXS, finding
an average age of 3 years at initial diagnosis, a high prevalence
of comorbid conditions, and standard of care consisting primarily
of counseling/therapy and traditional prescription medications;
and
- While caregivers of children with FXS often notice a variety of
initial symptoms early and seek help from a health care
professional, it is not until subsequent physician visits, often
involving a specialist, that a formal diagnosis is made.
About Zynerba Pharmaceuticals, Inc. Zynerba
Pharmaceuticals is the leader in pharmaceutically-produced
transdermal cannabinoid therapies for rare and near-rare
neuropsychiatric disorders. We are committed to improving the lives
of patients and their families living with severe, chronic health
conditions including Fragile X syndrome, autism spectrum disorder,
22q11.2 deletion syndrome, and a heterogeneous group of rare and
ultra-rare epilepsies known as developmental and epileptic
encephalopathies. Learn more at www.zynerba.com and follow us on
Twitter at @ZynerbaPharma.
Cautionary Note on Forward-Looking
Statements
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. We may, in some cases, use terms such as “predicts,”
“believes,” “potential,” “proposed,” “continue,” “estimates,”
“anticipates,” “expects,” “plans,” “intends,” “may,” “could,”
“might,” “will,” “should” or other words that convey uncertainty of
future events or outcomes to identify these forward-looking
statements. Such statements are subject to numerous important
factors, risks and uncertainties that may cause actual events or
results to differ materially from the Company’s current
expectations. Management’s expectations and, therefore, any
forward-looking statements in this press release could also be
affected by risks and uncertainties relating to a number of other
factors, including the following: the Company’s cash and cash
equivalents may not be sufficient to support its operating plan for
as long as anticipated; the Company’s ability to obtain additional
funding to support its clinical development programs; the results,
cost and timing of the Company’s clinical development programs,
including any delays to such clinical trials relating to enrollment
or site initiation; clinical results for the Company’s product
candidates may not be replicated or continue to occur in additional
trials and may not otherwise support further development in a
specified indication or at all; actions or advice of the U.S. Food
and Drug Administration and foreign regulatory agencies may affect
the design, initiation, timing, continuation and/or progress of
clinical trials or result in the need for additional clinical
trials; the Company’s ability to obtain and maintain regulatory
approval for its product candidates, and the labeling under any
such approval; the Company’s reliance on third parties to assist in
conducting pre-clinical and clinical trials for its product
candidates; delays, interruptions or failures in the manufacture
and supply of the Company’s product candidates the Company’s
ability to commercialize its product candidates; the size and
growth potential of the markets for the Company’s product
candidates, and the Company’s ability to service those markets; the
Company’s ability to develop sales and marketing capabilities,
whether alone or with potential future collaborators; the rate and
degree of market acceptance of the Company’s product candidates;
the Company’s expectations regarding its ability to obtain and
adequately maintain sufficient intellectual property protection for
its product candidates; and the timing and outcome of current and
future legal proceedings. This list is not exhaustive and these and
other risks are described in the Company’s periodic reports,
including the annual report on Form 10-K, quarterly reports on Form
10-Q and current reports on Form 8-K, filed with or furnished to
the Securities and Exchange Commission and available
at www.sec.gov. Any forward-looking statements that the
Company makes in this press release speak only as of the date of
this press release. The Company assumes no obligation to update
forward-looking statements whether as a result of new information,
future events or otherwise, after the date of this press
release.
Zynerba ContactWill Roberts, VP Investor
Relations and Corporate
Communications484.581.7489robertsw@zynerba.com
Media contactMolly DevlinEvoke
KYNE215.928.2199Molly.Devlin@evokegroup.com
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